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Positive Phase 1b/2 Results from Ongoing REC-4881 TUPELO Trial Demonstrate Rapid and Durable Reductions in Polyp Burden in Familial Adenomatous Polyposis (FAP) at 25 Weeks

  • REC–4881 (4 mg QD) achieved rapid clinical activity, with 75% of evaluable patients showing reductions in total polyp burden and a 43% median reduction after 12 weeks of treatment (n=12)
  • After 12 weeks off therapy (week 25 of the study), 82% of evaluable patients (9 of 11) maintained a durable reduction in total polyp burden, with a 53% median reduction observed from baseline
  • Natural history analysis showed that 87% of untreated FAP patients – who resembled the inclusion criteria of TUPELO – had annualized polyp–burden increase, 10% remained stable, and 3% showed modest decrease—underscoring the disease’s progressive trajectory (n=55)
  • 40% of patients (4 out of 10) achieved a ≥1–point improvement in Spigelman stage—a clinically meaningful measure of upper GI disease severity to assess surveillance and clinical management
  • REC–4881 (4 mg QD) has a safety profile consistent with MEK1/2 inhibition, with the majority of treatment–related adverse events being Grade 1 or 2, Grade 3 events occurring in 15.8% of the safety–evaluable patients, and no Grade ≥4 TRAEs reported to date
  • First clinical validation of the Recursion OS, demonstrating how unbiased phenotypic and mechanistic insights—such as MEK1/2 rescue of APC loss–of–function—can translate to novel, differentiated therapeutics for diseases like FAP with no approved therapy and high prevalence of >50,000 patients in US and EU5
  • Next steps: Engage the FDA in the 1H26 to define a potential registration pathway, and in parallel, expand the population from ≥55 to ≥18 years old, and further optimize dosing schedule

SALT LAKE CITY, Dec. 08, 2025 (GLOBE NEWSWIRE) — Recursion (Nasdaq: RXRX), a clinical–stage TechBio company decoding biology to radically improve lives, today announced positive Phase 1b/2 data from the ongoing TUPELO trial of REC–4881, an investigational allosteric MEK1/2 inhibitor for familial adenomatous polyposis (FAP).

Through an unbiased phenotypic screen of thousands of compounds, the earliest version of the Recursion OS identified selective MEK1/2 inhibition as a highly specific mechanism capable of reversing APC loss–of–function signatures. Using high–content cellular phenomics driven by AI, REC–4881 emerged as one of the strongest phenotypic rescue hits, reverting APC–deficient cells toward a healthy state and suppressing ERK/MAPK hyperactivation downstream of APC loss. Guided by this AI–driven insight, Recursion in–licensed REC–4881 from Takeda and redirected REC–4881—originally evaluated clinically in solid tumors—as a mechanistically aligned therapeutic candidate for FAP. REC–4881 is now the first MEK1/2 inhibitor ever studied clinically for this disease.

“The durable polyp burden reduction demonstrated by REC–4881—especially the sustained effect seen at Week 25, 12 weeks after completing therapy—is highly encouraging for the FAP community,” said Jessica Stout, D.O., Assistant Clinical Professor, University of Utah School of Medicine, and Principal Investigator of the TUPELO study. “Given the near–100% lifetime risk of colorectal cancer and the absence of any approved medical therapies, patients today often face a lifetime of intensive surveillance and life–altering surgeries. These Phase 2 results provide a meaningful basis for hope and support the potential for REC–4881 to offer a much–needed non–surgical option for this debilitating, life–long disease.”

“These Phase 2 results mark a meaningful validation of the Recursion OS,” said Chris Gibson, Ph.D., Co–Founder and CEO of Recursion. “An unbiased phenotypic insight from our platform and driven by AI—linking MEK1/2 inhibition to APC loss–of–function biology—has now translated into rapid, substantial, and durable reductions in polyp burden in patients. This is a powerful example of how even the earliest versions of the Recursion OS can uncover therapeutic opportunities in diseases with no approved pharmacotherapy options. And since this discovery, we’ve only added to the breadth, depth, and power of the Recursion OS; we believe this is the first of many potential medicines that will advance as our flywheel of discovery accelerates.”

In the Phase 2 portion of the study, REC–4881 demonstrated rapid and durable reductions in polyp burden, with 43% median reduction in evaluable patients after 12 weeks of treatment. 75% of evaluable patients had a reduction in polyp burden in this same period. Importantly, the effect persisted well beyond the dosing period: 82% of evaluable patients (9 of 11) maintained reductions at Week 25—12 weeks after stopping therapy—with a 53% median decrease from baseline. These results are especially meaningful when considered alongside real–world natural history analyses showing that untreated FAP patients are expected to experience increases when left untreated.

“This program reflects a full validation cycle of the Recursion OS: an unbiased phenotypic signal identifying MEK1/2 inhibition as a rescue mechanism for APC loss–of–function, followed by mechanistic confirmation, clinical translation, and now encouraging human data in a disease with no approved medical therapies,” said Najat Khan, Ph.D., Chief R&D and Commercial Officer and incoming President and CEO. “REC–4881, an allosteric MEK1/2 inhibitor, represents a first precision–medicine approach for the causal biology of FAP. In TUPELO, we are seeing rapid, substantial, and durable reductions in polyp burden — including sustained benefit after patients stop therapy. Equally important, our ClinTech and real–world data capabilities have been instrumental in guiding this program — from refining eligibility to contextualizing a single–arm dataset with a first–of–its–kind natural history study.”

About FAP

FAP is one of the most clinically significant hereditary colorectal cancer syndromes and is caused by inactivating mutations in the APC gene, leading to the growth of hundreds to thousands of gastrointestinal polyps and a near 100% risk of developing colorectal cancer before the age of 40 if left untreated. With no approved pharmacotherapies, excisions followed sequentially by debilitating, life altering surgeries remain the only option to remove polyps and polyp burdened organs, typically involving colectomy in the early 20s. While this procedure removes immediate colon cancer risk, it does not address the underlying disease biology and does not prevent future polyp formation. Patients will continue to develop polyps in the rectum or upper GI tract, with approximately 50% of patients requiring subsequent life–altering surgical procedures to manage the persistent disease. Current treatment approaches lead to substantial long–term loss of quality of life, affecting continence, fertility, and long–term gastrointestinal function in relatively young patients. Approximately 90% of FAP patients go on to develop duodenal adenomas, with 6% eventually undergoing a high–morbidity risk duodenectomy to control polyp growth. FAP affects an estimated >50,000 individuals across the US and EU5 (France, Germany, Italy, Spain, and the UK).

Background on REC–4881 and Recursion’s Platform Insights

REC–4881 was discovered using one of the earliest versions of the Recursion OS (v0.1), through an unbiased, high–content AI–driven phenotypic screening approach in APC–deficient human cell models. Because FAP is driven by loss–of–function mutations in the APC gene, the platform was designed to identify molecules capable of rescuing APC–dependent biology—guided entirely by cellular phenotype, without presupposing any specific mechanism. Using AI/ML to extract and compare over a thousand morphological features that distinguish “diseased” from “healthy” states, the Recursion OS screened numerous investigative compounds and identified REC–4881 as one of the most robust phenotype–rescuing hits. In follow–up assays, REC–4881 consistently reverted APC–deficient cells toward a healthy–state phenotype and demonstrated potent, selective, and concentration–dependent MEK1/2 inhibition that was not seen across hundreds of other oncogenes and tumor suppressor models tested.

Importantly, the Recursion OS highlighted MEK1/2 inhibition as a mechanistic strategy to exploit a therapeutic vulnerability arising from APC loss in FAP—a disease area where MEK1/2 inhibition had not previously been investigated as a therapeutic strategy in a clinical setting. Based on this novel insight, Recursion in–licensed REC–4881 from Takeda, where it had been evaluated in solid tumors, and redirected it as the first MEK1/2 inhibitor advanced clinically for FAP. This program represents the power of a phenotype–first AI–driven discovery model: the platform surfaced a mechanistically aligned therapeutic opportunity solely through scaled high–dimensional exploration.

Today, the Company is using the Recursion OS 2.0 platform—including proprietary ClinTech capabilities of large–scale real–world evidence (RWE) analytics—to further advance the REC–4881 program. This includes a comprehensive natural history collaboration with Amsterdam University Medical Center, home to one of the largest and longest–running FAP registries, as well as analysis of more than 1,000 US FAP patients and 250,000 physician notes processed through Recursion’s custom LLM–based pipeline. Together, these data reinforce the relentlessly progressive nature of FAP, highlight the absence of spontaneous polyp regression, and demonstrate the substantial burden of repeated polyp–removal procedures and major surgeries experienced by real–world patients.

ClinTech insights also helped refine the design of the ongoing TUPELO trial, including expanding age eligibility from ≥55 to ≥18. This expansion was based on a thorough risk–benefit analysis, enabling evaluation of REC–4881 in younger patients who represent a substantial portion of FAP patients. REC–4881 has received Fast Track and Orphan Drug designations from the US FDA, as well as Orphan Drug designation from the European Commission.

Results of the Phase 2 Data for Ongoing REC–4881 Trial

Efficacy and Durability Findings

As of the November 25, 2025 data cutoff in the open–label Phase 2 portion of TUPELO, treatment with REC–4881 (4 mg QD) demonstrated meaningful and durable reductions in polyp burden in patients with FAP.

Week 13 Assessment

  • REC–4881 produced a median 43% reduction in total polyp burden among 12 efficacy–evaluable patients.
  • The majority of evaluable patients responded, with 75% showing reductions in polyp burden after 12 weeks of therapy.
  • 40% of patients (4 out of 10) achieved a ≥1–point improvement in Spigelman stage—a clinically meaningful measure of upper GI disease severity to assess surveillance and clinical management.
  • Other investigational agents currently under evaluation in separate studies generated approximately 17–29% median reduction in polyp burden after 12 months of treatment; no off–treatment durability was reported (Biodexa press release, June 24 2024)

Figure 1: Waterfall plot showing percent change from baseline in total polyp burden at Week 13 for efficacy–evaluable patients receiving REC–4881 (4 mg QD)

Week 25 Assessment

  • After 12 weeks of treatment, patients went off treatment for an additional 12–weeks. Durability of effect was maintained during the off–treatment observation period with 82% of patients responding (>0% reduction; 9 out of 11) at Week 25.
  • 73% achieved durable ≥30% reductions in polyp burden with a 53% median reduction in total polyp burden observed.
  • 40% of patients (4 out of 10) maintained a ≥1–point improvement in Spigelman stage from baseline.

Figure 2: Waterfall plot showing percent change from baseline in total polyp burden at Week 25 for efficacy–evaluable patients receiving REC–4881 (4 mg QD)

Safety Summary

As of the data cutoff, treatment with 4 mg REC–4881 demonstrated a safety profile generally consistent with prior MEK1/2 inhibitors.

  • Across the combined Phase 1b and Phase 2 safety cohorts (n=19), 94.7% of patients reported at least one treatment–related adverse event (TRAE), the majority of which were Grade 1/2 in severity. The most frequent TRAEs (≥10%) included: dermatitis acneiform / rash and blood CPK increase.
  • Grade 3 TRAEs occurred in 15.8% of patients; no Grade ≥4 TRAEs have been reported to date.
  • Treatment modifications were infrequent, with 2 of 19 patients experiencing dose interruption.

Figure 3: Summary of adverse events across Phase 1b and Phase 2 of the TUPELO trial

About the TUPELO Trial Design and Expanded Population
The Phase 1b/2 TUPELO trial is evaluating the safety, tolerability, pharmacokinetics (PK), and efficacy of REC–4881 monotherapy in patients with familial adenomatous polyposis (FAP).

Study Design and Analysis

Efficacy is assessed via upper and lower endoscopy at baseline, Week 13 (on–treatment), and Week 25 (off–treatment).

  • The primary endpoint is percent change from baseline in polyp burden, which is the sum of all polyp diameters in the GI.
  • The Efficacy Evaluable Population includes patients with measurable disease at baseline who received ≥75% of study drug and had at least one post–baseline endoscopic assessment. Disease staging uses the Spigelman system for upper GI polyposis and the InSiGHT classification for the lower GI tract.

Natural History Analyses
To better understand the natural history of FAP and to contextualize the single–arm efficacy of REC–4881, we collaborated with Amsterdam University Medical Center to analyze a registry of ~200 patients with FAP. 55 of these patients met the key inclusion criteria of TUPELO. We also leveraged our clinical development technology (ClinTech) platform to analyze US electronic health records (EHR), including physician notes, of ~1,000 FAP patients to assess disease progression and treatment patterns in the US. Both studies revealed high patient burden and progressive natural history of the disease. Results of the studies suggest that the natural history of FAP is to progress with relentless precancerous polyp progression: 87% of untreated patients in the registry experienced annualized increase in polyp burden. 10% were stable and 3% experienced a modest decrease in polyp burden. Mean increase of 60% and median increase of 28% in annualized polyp burden was observed. At least 75% of patients in the US EHR database had a major invasive surgery along with frequent polypectomies during follow–up.

Next Steps
Recursion plans to expand the population from ≥55 to ≥18 years old and further optimize dosing schedule. In parallel, the Company intends to engage the FDA in 1H26 to define a potential registration pathway.

Forward Looking Statements
This document contains information that includes or is based upon “forward–looking statements” within the meaning of the Securities Litigation Reform Act of 1995, including, without limitation, those regarding Recursion’s anticipated engagement with the FDA; the clinical relevance of the TUPELO trial data and obtaining additional confirmatory data; advancing potential transformational therapies for FAP and beyond; subsequent REC–4881 studies, including expanded enrollment and alternate dosing schedule, and their results and advancing Recursion’s REC–4881 program further; the size of the potential FAP patient population; Recursion OS and other technologies potential and advancement of the future of medicine; business and financial plans and performance; and all other statements that are not historical facts. Forward–looking statements may or may not include identifying words such as “plan,” “will,” “expect,” “anticipate,” “intend,” “believe,” “potential,” “continue,” and similar terms. These statements are subject to known or unknown risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements, including but not limited to: challenges inherent in pharmaceutical research and development, including the timing and results of preclinical and clinical programs, where the risk of failure is high and failure can occur at any stage prior to or after regulatory approval due to lack of sufficient efficacy, safety considerations, or other factors; our ability to leverage and enhance our drug discovery platform; our ability to obtain financing for development activities and other corporate purposes; the success of our collaboration activities; our ability to obtain regulatory approval of, and ultimately commercialize, drug candidates; our ability to obtain, maintain, and enforce intellectual property protections; cyberattacks or other disruptions to our technology systems; our ability to attract, motivate, and retain key employees and manage our growth; inflation and other macroeconomic issues; and other risks and uncertainties such as those described under the heading “Risk Factors” in our filings with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10–K and Quarterly Reports on Form 10–Q. All forward–looking statements are based on management’s current estimates, projections, and assumptions, and Recursion undertakes no obligation to correct or update any such statements, whether as a result of new information, future developments, or otherwise, except to the extent required by applicable law.

About Recursion
Recursion (NASDAQ: RXRX) is a clinical stage TechBio company leading the space by decoding biology to radically improve lives. Enabling its mission is the Recursion OS, a platform built across diverse technologies that continuously generate one of the world’s largest proprietary biological and chemical datasets. Recursion leverages sophisticated machine–learning algorithms to distill from its dataset a collection of trillions of searchable relationships across biology and chemistry unconstrained by human bias. By commanding massive experimental scale — up to millions of wet lab experiments weekly — and massive computational scale — owning and operating one of the most powerful supercomputers in the world, Recursion is uniting technology, biology and chemistry to advance the future of medicine. Recursion is headquartered in Salt Lake City, where it is a founding member of BioHive, the Utah life sciences industry collective. Recursion also has offices in Montréal, New York, London, and the Oxford area. Learn more at www.recursion.com, or connect on X and LinkedIn.

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Miltenyi Biomedicine stellt die Primäranalyse der zulassungsrelevanten DALY-2-EU-Studie zur Zweitlinientherapie des rezidivierten/refraktären großzelligen B-Zell-Lymphoms auf der 67. Jahrestagung der American Society of Hematology (ASH) vor

  • Die Ergebnisse der DALY 2–EU–Studie zeigen, dass Zamtocabtagen Autoleucel (Zamto–cel) bei Patienten mit rezidiviertem/refraktärem großzelligem B–Zell–Lymphom (r/r LBCL) eine klinisch bedeutsame Überlegenheit gegenüber der Chemoimmuntherapie aufweist1
  • Zamto–cel wurde von der Mehrheit der Patienten gut vertragen. Die DALY 2–EU–Studie umfasste eine Hochrisikopopulation, die durch ein höheres Alter und klinisch ungünstige Krankheitsmerkmale gekennzeichnet war
  • Eine Herstellungsdauer von 12 Tagen führte zu einer Vene–zu–Vene–Zeit von 14–16 Tagen und verringerte die Wahrscheinlichkeit, dass eine Bridging–Therapie erforderlich war.

BERGISCH GLADBACH, Deutschland, Dec. 08, 2025 (GLOBE NEWSWIRE) — Miltenyi Biomedicine hat heute die Ergebnisse der zulassungsrelevanten DALY 2–EU–Studie bekanntgegeben. In dieser Studie wurde die Wirksamkeit und Sicherheit von Zamtocabtagen Autoleucel (Zamto–cel) im Vergleich zur Standard–Chemoimmuntherapie (R–GemOx oder Pola–BR) als Zweitlinientherapie bei Patienten mit rezidiviertem oder refraktärem großzelligem B–Zell–Lymphom (r/r LBCL) beurteilt, die aufgrund ihres Alters, von Komorbiditäten oder anderer medizinischer Gründe nicht für eine Transplantation infrage kamen.

Die Primäranalyse zeigte, dass Zamto–cel bei transplantationsungeeigneten Patienten mit hohem Risiko für eine rasche Krankheitsprogression eine signifikante und klinisch relevante Überlegenheit gegenüber der Chemoimmuntherapie (R–GemOx) aufwies.1 Diese Studienpopulation war durch ein höheres Alter und klinisch ungünstige Krankheitsmerkmale gekennzeichnet: Das mediane Alter betrug 74 Jahre, 57 % der Patienten hatten einen hohen International Prognostic Index (IPI ≥ 3) und 67 % befanden sich im Stadium III/IV der Erkrankung. Zamto–cel wurde in dieser überwiegend älteren Population mit hohem Risiko gut vertragen.1

Dr. Peter Borchmann, Hauptprüfer der DALY 2–EU–Studie und stellvertretender medizinischer Direktor der Abteilung für Hämatologie und Onkologie am Universitätsklinikum Köln, erklärte: „Zamto–cel zeigte bei transplantationsungeeigneten Patienten mit Hochrisikoerkrankung eine klinisch bedeutsame und statistisch signifikante Überlegenheit gegenüber R–GemOx, indem es das ereignisfreie Überleben verbesserte und gleichzeitig ein günstiges Verträglichkeitsprofil aufwies. Diese Ergebnisse unterstreichen das Potenzial von Zamto–cel als wichtige neue Behandlungsoption für eine klinisch vulnerable Patientengruppe mit begrenzten therapeutischen Möglichkeiten.“

Dr. Toon Overstijns, Chief Executive Officer von Miltenyi Biomedicine, fügte hinzu: „Die Ergebnisse der DALY 2–EU–Studie sind ein wichtiger Meilenstein in unserem Engagement für die Weiterentwicklung von Zell– und Gentherapien. Zamto–cel, die erste Tandem–CD20–CD19–gerichtete–, nicht kryokonservierte CAR–T–Zelltherapie, zeigte einen bedeutenden klinischen Nutzen mit vielversprechender Wirksamkeit und Sicherheit. Damit sind wir der Bereitstellung dringend benötigter Behandlungsoptionen für Patienten mit Hochrisiko–Lymphomen einen Schritt näher.“

  • Zamto–cel ist die erste Tandem–CD20–CD19–gerichtete, nicht kryokonservierte chimäre Antigenrezeptor–T–Zelltherapie (CAR–T). Die Hauptmechanismen für ein Rezidiv nach Behandlungen mit CD19–gerichteten CAR–T–Zelltherapien sind die begrenzte Persistenz der CAR–T–Zellen, die Hemmung ihrer Funktion sowie die CD19–spezifische Antigenflucht. Um das Risiko eines Rezidivs aufgrund einer CD19–Antigenflucht zu minimieren, nutzt Zamto–cel ein duales Antigen–Targeting von CD20 und CD19. Zamto–cel hat eine Herstellungsdauer von 12 Tagen, was zu einer Vene–zu–Vene–Zeit von 14 bis 16 Tagen führt. Dadurch wird die Wahrscheinlichkeit verringert, dass eine Bridging–Therapie notwendig wird

Primäre Ergebnisse der DALY 2–EU–Studie1

Zum Zeitpunkt des Datenstichtags wurden die Patienten randomisiert und der Behandlung mit Zamto–cel (n = 82) oder der Behandlung mit R–GemOx/ Pola–BR (n = 86) zugewiesen. Die Studie erlaubte zudem ein Crossover: 29 Patienten erhielten Zamto–cel, nachdem sie nicht auf R–GemOx (n = 28) bzw. nicht auf Pola–BR (n = 1) angesprochen hatten

Wirksamkeitsergebnisse (bewertet durch das verblindete unabhängige Überprüfungsgremium, BIRC)

  • Das mediane ereignisfreie Überleben (EFS) betrug für Zamto–cel 6,2 Monate (95 % CI 3,8–13,8) im Vergleich zu 2,5 Monaten (95 %–KI 2,0–3,3) für R–GemOx (HR 0,39; 95 %–KI 0,27–0,58; p < 0,0001).
  • Das mediane progressionsfreie Überleben (PFS) war bei Zamto–cel mit 8,5 Monaten (95 % CI 3,8–16,8) signifikant länger als bei R–GemOx mit 3,3 Monaten (95 %–KI 2,0–3,8) (HR 0,43 [95 %–KI 0,28–0,65]; p < 0,0001).
  • In der Intent–to–treat–Population (ITT) betrug die Gesamtansprechrate (ORR) für Zamto–cel 72 % mit einer vollständigen Ansprechrate (CRR) von 54 % im Vergleich zu einer ORR von 45 % und einer CRR von 14 % für R–GemOx.

Sicherheitsergebnisse1
Zamto–cel wurde in dieser älteren Patientenpopulation mit hohem Risiko gut vertragen

  • Ein Zytokin–Freisetzungssyndrom (CRS) von Grad ≥ 3 wurde bei vier Patienten (5,3 %) beobachtet.
  • Ein Immuneffektorzell–assoziiertes Neurotoxizitätssyndrom (ICANS) von Grad 3 trat bei einem Patienten (1,3 %) auf.

Über DALY 2–EU2
DALY 2–EU (NCT04844866) ist eine zulassungsrelevante, randomisierte, multizentrische, offene Phase–II–Studie, die in zwölf Ländern innerhalb der EU durchgeführt wird. In der Studie wird die Sicherheit und Wirksamkeit von gentechnisch veränderten autologen T–Zellen untersucht, die einen gegen CD20 und CD19 gerichteten chimären Antigenrezeptor (Zamtocabtagen Autoleucel, Zamto–cel) exprimieren, im Vergleich zur Chemoimmuntherapie (CIT) (Rituximab, Gemcitabin undOxaliplatin [R–GemOx]) oder Polatuzumab Vedotin plus Bendamustin/Rituximab (Pola–BR) als Zweitlinientherapie bei primär rezidiviertem/refraktärem großzelligem B–Zell–Lymphom (r/r LBCL). Nach unserem Kenntnisstand ist dies die einzige randomisierte CAR–T–Studie, die bisher bei dieser Patientengruppe durchgeführt wurde.

Teilnehmen konnten Erwachsene mit r/r LBCL, die innerhalb von 24 Monaten nach Beginn ihrer Erstlinientherapie refraktär waren oder ein Rezidiv entwickelt hatten, mindestens eine Anthrazyklin– und eine Rituximab–haltige Therapie erhalten hatten und für eine Stammzelltransplantation nicht infrage kamen.

Die Teilnehmer wurden im Verhältnis 1:1 randomisiert und erhielten entweder Zamto–cel oder CIT (R–GemOx/Pola–BR). Zamto–cel wurde als einzelne, nicht kryokonservierte Infusion in einer Dosis von 2,5 x 10^6 CAR–transduzierten T–Zellen pro Kilogramm Körpergewicht nach Lymphodepletion mit Fludarabin und Cyclophosphamid verabreicht. Die Patienten, die in den Vergleichsarm randomisiert wurden, erhielten entweder R–GemOx oder Pola–BR.

Der primäre Endpunkt der Studie ist das ereignisfreie Überleben (EFS), das durch einen verblindeten, unabhängigen Überprüfungsausschuss (BIRC) bewertet wird und definiert ist als die Zeit von der Randomisierung bis zum objektiven Fortschreiten der Erkrankung, dem Nichterreichen eines partiellen (PR) oder kompletten Ansprechens (CR) in oder nach Woche 8, das zur Einleitung einer neuen Anti–Lymphom–Therapie führt, oder Tod jeglicher Ursache. Sekundäre Endpunkte umfassen das progressionsfreie Überleben (PFS), die beste Komplettansprechrate (CRR), die Dauer des kompletten Ansprechens (DOR) und das Gesamtüberleben (OS).

Diese Daten werden im Rahmen einer vorab geplanten EFS–Zwischenanalyse nach einer medianen Nachbeobachtungszeit von 17 Monaten veröffentlicht. Weitere Analysen mit längeren Nachbeobachtungszeiträumen sind geplant und werden auf zukünftigen Tagungen vorgestellt.

Die Ergebnisse der DALY–2–EU–Studie ergänzen frühere Veröffentlichungen zu Zamto–cel bei anderen Indikationen und Populationen, darunter:

  • DALY II USA (NCT04792489), eine multizentrische, offene, einarmige Phase–II–Studie mit Zamto–cel bei Patienten mit r/r DLBCL, die bereits mindestens zwei vorherige Therapielinien erhalten haben, darunter einen monoklonalen Anti–CD20–Antikörper und ein Anthrazyklin–haltiges Behandlungsschema, und bei denen eine messbare Erkrankung gemäß der Lugano–Klassifikation 2014 vorliegt. Die ORR in der auswertbaren Patientenpopulation (n = 59), bewertet durch ein unabhängiges Radiologie–Gremium, betrug 72,9 % (95 %–KI, 59,7–83,6) mit einer CRR von 49,2 % (95 %–KI, 35,9–62,5).
  • In der klinischen Studie DALY II USA wurde außerdem eine spezielle Kohorte für r/r Lymphome des Zentralnervensystems eröffnet. In dieser Kohorte von 16 Patienten zeigte sich eine Gesamtansprechrate von 80 % bzw. 100 % und eine vollständige Ansprechrate von 50 % bzw. 100 % in der PCNSL– (primäres ZNS–Lymphom) bzw. SCNSL–Gruppe (sekundäres ZNS–Lymphom).
  • Zamto–cel wird derzeit beim r/r Mantelzelllymphom (MCL) und bei der r/r Richter–Transformation (RT) untersucht

Über Zamtocabtagen Autoleucel (Zamto–cel)
Zamto–cel ist eine sich in der Erprobung befindende autologe chimäre Antigenrezeptor (CAR)–T–Zell–Therapie, die sowohl auf CD20 als auch auf CD19 abzielt. Sie wird in klinischen Studien zur Behandlung von rezidivierten oder refraktären B–Zell–Malignomen untersucht, darunter großzelliges B–Zell–Lymphom (LBCL), diffuses großzelliges B–Zell–Lymphom (DLBCL), primäres und sekundäres Lymphom des Zentralnervensystems (ZNS), Mantelzell–Lymphom (MCL), Richter–Transformation (RT) und andere B–Zell–Neoplasien.

Zamto–cel wird unter Verwendung der proprietären Plattform von Miltenyi, einem geschlossenen, automatisierten System, hergestellt. Die Herstellungsdauer von 12 Tagen ermöglicht eine Vene–zu–Vene–Zeit von 14–16 Tagen, wodurch die Notwendigkeit einer Bridging–Therapie reduziert und der Zugang zu Zelltherapien für Hochrisikopatienten mit dringendem Therapiebedarf erleichtert wird. Da keine Kryokonservierung erforderlich ist, entfallen kryokonservierungsbedingte logistische Schritte und Kosten.

Über Miltenyi Biomedicine
Miltenyi Biomedicine hat es sich zum Ziel gesetzt, Patienten mit schweren Erkrankungen Zugang zu innovativen Krebsbehandlungen und regenerativen Therapien zu verschaffen. Mithilfe modernster Technologien entwickelt das Unternehmen neue Ansätze für schwer behandelbare Blutkrebserkrankungen und nutzt das Potenzial der CAR–Technologie, um die Patientenversorgung grundlegend zu verbessern. Derzeit untersucht Miltenyi Biomedicine sein erstes Zelltherapieprodukt.

Über Miltenyi Biotec
Miltenyi Biotec ist weltweit führend in Technologien und Dienstleistungen für patientenspezifische Zell– und Gentherapien und setzt wissenschaftliche Entdeckungen in praktische Behandlungen für die personalisierte Medizin um. Mit über 35 Jahren Erfahrung unterstützt das Unternehmen biomedizinische Entdeckungen und überträgt sie in klinische Anwendungen, wodurch Patienten Zugang zu neuen Therapien erhalten. Mit seinen integrierten Lösungen, darunter GMP–zertifizierte Zellfabriken, bietet Miltenyi Biotec Therapieentwicklern über seine globale CDMO–Sparte Miltenyi Bioindustry kompetente Beratung von der Prozessentwicklung bis zur Kommerzialisierung.

Kontakt
Miltenyi Biomedicine
Jasmine Oberwalleney
Friedrich–Ebert–Straße 68
51429 Bergisch Gladbach, Deutschland
[email protected]

Referenzen

  1. Borchmann P, et al. Zamtocabtagene–autoleucel, a tandem CD20–CD19 directed CAR–T cell therapy as second–line treatment for Relapsed/Refractory large B–cell lymphoma: primary analysis of the randomized pivotal DALY 2–EU study. Vorgestellt auf der Jahrestagung der American Society of Hematology (ASH). Abstract #abs25–738.
  2. ClinicalTrials.Gov. Efficacy and Safety of MB–CART2019.1 vs. SoC in Lymphoma Patients (DALY 2–EU). Verfügbar unter: https://clinicaltrials.gov/study/NCT04844866. Zugriff im September 2025.

MAT–GL–ZA–0003

Erstellungsdatum: Dezember 2025


GLOBENEWSWIRE (Distribution ID 1001143511)

À l’occasion de la 67e réunion annuelle de l’American Society of Hematology (ASH), Miltenyi Biomedicine présente l’analyse primaire de l’essai pivot DALY 2-EU pour le traitement de seconde ligne du lymphome à grandes cellules B en rechute ou réfractaire

  • Les résultats de l’essai DALY 2–EU révèlent que le zamtocabtagene autoleucel (zamto–cel) présente une supériorité cliniquement significative par rapport à la chimio–immunothérapie chez les patients atteints d’un lymphome à grandes cellules B en rechute ou réfractaire (LGCB r/r)1
  • Le zamto–cel a été bien toléré par la majorité des patients. L’étude DALY 2–EU portait sur une population à haut risque, caractérisée par un âge avancé et des caractéristiques cliniques de maladie à haut risque
  • Un temps de fabrication de 12 jours a permis d’obtenir un délai veine à veine de 14 à 16 jours, réduisant ainsi la nécessité d’instaurer un traitement de transition.

BERGISCH GLADBACH, Allemagne, 08 déc. 2025 (GLOBE NEWSWIRE) — Miltenyi Biomedicine annonce ce jour les résultats de l’essai pivot DALY 2–EU visant à évaluer l’efficacité et la sécurité d’emploi du zamtocabtagene autoleucel (zamto–cel) par rapport à la chimio–immunothérapie conventionnelle (R–GemOx ou Pola–BR) dans le cadre d’un traitement de seconde ligne de patients atteints d’un lymphome à grandes cellules B, en rechute ou réfractaire (LGCB r/r) et inéligibles à une greffe en raison de leur âge, de leurs comorbidités ou d’autres raisons médicales.

L’analyse primaire a révélé que le zamto–cel présentait une supériorité significative et cliniquement pertinente par rapport à la chimio–immunothérapie (R–GemOx) chez les patients inéligibles à une greffe et présentant un risque élevé de progression rapide de la maladie.1 Cette population d’étude s’est caractérisée par un âge avancé et des caractéristiques cliniques de maladie à haut risque : l’âge médian était de 74 ans, 57 % des patients présentaient un indice pronostique international élevé (IPI ≥ 3) et 67 % présentaient une maladie de stade III/IV. Le zamto–cel a été bien toléré dans cette population majoritairement âgée et à haut risque.1

Le Dr Peter Borchmann, investigateur principal de l’essai DALY 2–EU et directeur médical adjoint du service d’hématologie et d’oncologie de l’hôpital universitaire de Cologne, en Allemagne, a déclaré : « Le zamto–cel a démontré une supériorité cliniquement pertinente et statistiquement significative par rapport au R–GemOx chez les patients inéligibles à une greffe et présentant une maladie à haut risque, améliorant la survie sans événement tout en conservant un profil de tolérance favorable. Ces résultats démontrent le bénéfice potentiel du zamto–cel en tant que nouvelle option thérapeutique majeure pour une population de patients cliniquement vulnérables dont les choix thérapeutiques sont limités. »

Le Dr Toon Overstijns, directeur général de Miltenyi Biomedicine, a fait la déclaration suivante : « Les résultats de l’essai DALY 2–EU marquent une étape décisive dans notre engagement visant à faire progresser les thérapies cellulaires et géniques. Le zamto–cel, première thérapie cellulaire CAR–T tandem ciblant les antigènes CD20 et CD19 (dirigée) non cryoconservée, a démontré un bénéfice clinique significatif avec une efficacité et une sécurité prometteuses, nous rapprochant de l’étape de mise à disposition de nouvelles options thérapeutiques pour les patients atteints de lymphomes à haut risque. »

  • Le zamto–cel est la première thérapie cellulaire CAR–T (récepteurs antigéniques chimériques) tandem ciblant les antigènes CD20 et CD19 (dirigée) non cryoconservée. Les principaux mécanismes de rechute après des traitements par thérapie cellulaire CAR–T anti–CD19 sont les suivants : persistance limitée des cellules CAR–T, inhibition de la fonction des cellules CAR–T et échappement immunologique de l’antigène CD19. Afin de réduire le risque de rechute due à l’échappement de l’antigène CD19, le zamto–cel utilise un double ciblage antigénique des antigènes CD20 et CD19. Le temps de fabrication du zamto–cel est de 12 jours. Cela se traduit par un délai veine à veine de 14 à 16 jours et permet de réduire la nécessité d’instaurer un traitement de transition

Principaux résultats de l’essai DALY 2–EU1

À la date de gel des données, les patients ont été randomisés pour recevoir soit le zamto–cel (n = 82), soit le R–GemOx/PolaBR (n = 86). Au cours de cet essai, le transfert des patients était autorisé : 29 patients ont reçu le zamto–cel après l’absence de réponse avec le R–GemOx (n = 28) ou le Pola–BR (n = 1).

Résultats d’efficacité (évalués par le comité d’évaluation indépendant [CEI] en aveugle)

  • La survie sans événement (SSE) médiane pour le zamto–cel était de 6,2 mois (IC à 95 % : 3,8–13,8) contre 2,5 mois (IC à 95 % : 2,0–3,3) pour le R–GemOx (RR 0,39 ; IC à 95 % : 0,27–0,58 ; p < 0,0001).
  • La survie sans progression (SSP) médiane était significativement plus longue avec le zamto–cel, à 8,5 mois (IC à 95 % : 3,8–16,8), contre 3,3 mois (IC à 95 % : 2,0–3,8) pour le R–GemOx (RR 0,43 [IC à 95 % : 0,28–0,65] ; p < 0,0001).
  • Dans la population en intention de traiter (ITT), le taux de réponse globale (TRG) était de 72 % avec un taux de réponse complète (TRC) de 54 % pour le zamto–cel, contre un TRG de 45 % et un TRC de 14 % pour le R–GemOx.

Résultats de sécurité1
Le zamto–cel a été bien toléré dans cette population de patients âgés à haut risque

  • Un syndrome de relargage des cytokines (SRC) de grade ≥ 3 a été signalé chez 4 patients (5,3 %).
  • Un syndrome de neurotoxicité associée aux cellules effectrices immunitaires (ICANS) de grade 3 est survenu chez 1 patient (1,3 %).

À propos de l’essai DALY 2–EU2
L’essai DALY 2–EU (NCT04844866) est un essai pivot, randomisé, multicentrique, en ouvert, de phase II, mené dans 12 pays de l’UE, visant à évaluer la sécurité et l’efficacité des lymphocytes T autologues génétiquement modifiés exprimant un récepteur antigénique chimérique spécifique anti–CD20 et anti–CD19, le zamtocabtagene autoleucel (zamto–cel), par rapport à la chimio–immunothérapie (CIT) (rituximab, gemcitabine et oxaliplatine [R–GemOx]) ou au polatuzumab vedotin plus bendamustine/rituximab [Pola–BR]), en tant que traitement de deuxième intention du lymphome à grandes cellules B primitif en rechute/réfractaire (LGCB r/r). À notre connaissance, il s’agit de la seule étude randomisée sur la thérapie cellulaire CAR–T menée à ce jour auprès de cette population de patients.

Les patients éligibles étaient des adultes atteints d’un LGCB r/r réfractaire ou ayant rechuté dans les 24 mois suivant le début de leur traitement en première intention, ayant reçu au moins un schéma à base d’anthracycline et de rituximab et inéligibles à une greffe de cellules souches.

Les participants ont été randomisés selon un rapport de 1/1 pour recevoir soit du zamto–cel, soit une CIT (R–GemOx/Pola–BR). Le zamto–cel a été administré sous forme de perfusion unique non cryoconservée à une dose de 2,5 × 10^6 lymphocytes T transduits par CAR par kg de poids corporel après lymphodéplétion par fludarabine et cyclophosphamide. Les patients randomisés dans le bras comparateur ont reçu soit du R–GemOx, soit du Pola–BR.

Le critère d’évaluation principal de l’essai est la survie sans événement (SSE), évaluée par un comité d’évaluation indépendant [CEI] en aveugle, définie comme le temps écoulé entre la randomisation et la progression objective de la maladie, l’absence de réponse partielle (RP) ou de réponse complète (RC) à la semaine 8 ou au–delà, entraînant l’instauration d’un nouveau traitement anti–lymphome ou le décès (toutes causes confondues). Les critères d’évaluation secondaires comprennent la survie sans progression (SSP), le taux de réponse complète (TRC), la durée de la réponse complète (DR) et la survie globale (SG).

Ces données seront communiquées dans le cadre d’une analyse intermédiaire préplanifiée de la SSE après un suivi médian de 17 mois. Des analyses supplémentaires sont prévues avec des périodes de suivi plus longues et seront présentées lors de prochaines réunions.

Les résultats de l’essai DALY 2–EU viennent s’ajouter aux publications précédentes sur le zamto–cel dans d’autres indications et populations, notamment :

  • L’essai DALY II USA (NCT04792489), un essai de phase II multicentrique, en ouvert, à bras unique portant sur le zamto–cel chez des patients présentant un LDGCB r/r après au moins deux lignes de traitement antérieures, y compris un anticorps monoclonal anti–CD20 et un schéma à base d’anthracycline, et une maladie mesurable selon la classification de Lugano 2014. Le TRG dans la population de patients évaluables (n = 59), tel qu’évalué par un comité radiologique indépendant, était de 72,9 % (IC à 95 %, 59,7–83,6) avec un TRC de 49,2 % (IC à 95 %, 35,9–62,5).
  • Dans le cadre de l’essai clinique DALY II USA, une cohorte dédiée au lymphome du système nerveux central r/r a été ouverte. Dans cette cohorte de 16 patients, les données ont révélé un taux de réponse globale de 80 % et 100 % et un taux de réponse complète de 50 % et 100 % dans le LPSNC (lymphome primaire du SNC) et le LSSNC (lymphome secondaire du SNC) respectivement.
  • Le zamto–cel est actuellement à l’étude dans le traitement du lymphome à cellules du manteau (LCM) r/r et du syndrome de Richter (SR) r/r

À propos du zamtocabtagene autoleucel (zamto–cel)
Le zamto–cel est un traitement expérimental à base de lymphocytes T autologues à récepteurs antigéniques chimériques (CAR) conçu pour cibler à la fois les antigènes CD20 et CD19. Il fait actuellement l’objet d’essais cliniques pour le traitement des tumeurs malignes à cellules B en rechute ou réfractaires, notamment le lymphome à grandes cellules B (LGCB), le lymphome diffus à grandes cellules B (LDGCB), le lymphome primaire et secondaire du système nerveux central (SNC), le lymphome à cellules du manteau (LCM), le syndrome de Richter (SR) et d’autres tumeurs à cellules B.

Le zamto–cel est fabriqué à l’aide de la plateforme exclusive de Miltenyi, un système clos et automatisé. Le temps de fabrication de 12 jours se traduit par un délai veine à veine de 14 à 16 jours, ce qui réduit la nécessité d’instaurer un traitement de transition et augmente la capacité à recevoir une thérapie cellulaire pour les patients à haut risque ayant des besoins thérapeutiques urgents. Sa formulation non cryoconservée permet d’éliminer les étapes logistiques et les coûts liés à la cryoconservation.

À propos de Miltenyi Biomedicine
Miltenyi Biomedicine s’engage à mettre les traitements anticancéreux innovants et les thérapies régénératives à disposition des patients atteints de maladies graves. Grâce à une technologie de pointe, la société innove de manière indépendante pour traiter les cancers hématologiques difficiles à soigner et exploiter le potentiel de la technologie CAR afin de transformer la prise en charge des patients. Le premier actif de thérapie cellulaire de Miltenyi Biomedicine est en cours d’étude.

À propos de Miltenyi Biotec
Miltenyi Biotec est un leader mondial dans le domaine des technologies et des services innovants pour les thérapies cellulaires et géniques spécifiques aux patients. Il transforme les découvertes scientifiques en traitements pratiques pour une médecine personnalisée. Fort de plus de 35 ans d’expertise, il soutient les découvertes biomédicales et les transforme en applications cliniques, améliorant ainsi l’accès des patients à de nouvelles thérapies. Grâce à ses solutions intégrées, notamment ses usines cellulaires certifiées BPF, Miltenyi Biotec fournit des conseils d’experts aux développeurs de thérapies, de manière efficace, depuis le développement des processus jusqu’à la mise sur le marché, par l’intermédiaire de sa division CDMO mondiale Miltenyi Bioindustry.

Contact
Miltenyi Biomedicine
Jasmine Oberwalleney
Friedrich–Ebert–Strasse 68
51429 Bergisch Gladbach, Allemagne
[email protected]

Références

  1. Borchmann P, et al. Zamtocabtagene–autoleucel, a tandem CD20–CD19 directed CAR–T cell therapy as second–line treatment for Relapsed/Refractory large B–cell lymphoma: primary analysis of the randomized pivotal DALY 2–EU study. Présenté à l’occasion de la réunion annuelle de l’American Society of Hematology (ASH). Résumé n° abs25–738.
  2. ClinicalTrials.Gov. Efficacy and Safety of MB–CART2019.1 vs. SoC in Lymphoma Patients (DALY 2–EU). Disponible à l’adresse suivante : https://clinicaltrials.gov/study/NCT04844866. Consulté en septembre 2025.

MAT–GL–ZA–0003

Date de préparation : décembre 2025


GLOBENEWSWIRE (Distribution ID 1001143511)

Miltenyi Biomedicine divulga análise primária do estudo principal do DALY 2-EU para linfoma de células B grandes recidivante/refratário de segunda linha na 67ª Reunião Anual da Sociedade Americana de Hematologia (ASH)

  • Os resultados do DALY 2–UE mostram que o autoleucel de zamtocabtageno (zamto–cel) demonstrou superioridade clinicamente significativa em relação à quimioimunoterapia em pacientes com linfoma de células B grandes recidivante/refratário (r/r LBCL)1
  • O Zamto–cel foi bem tolerado na maioria dos pacientes. O estudo DALY 2–UE incluiu uma população de estudo de alto risco, caracterizada por idade avançada e doenças de alto risco
  • Um tempo de fabricação de 12 dias resultou em um tempo de veia a veia de 14–16 dias, reduzindo a probabilidade de terapia de ponte.

BERGISCH GLADBACH, Alemanha, Dec. 08, 2025 (GLOBE NEWSWIRE) — A Miltenyi Biomedicine divulgou hoje os resultados do estudo DALY 2–EU que avalia a eficácia e a segurança do zamtocabtagene autoleucel (zamto–cel) em comparação com a quimioimunoterapia padrão (R–GemOx ou Pola–BR) em pacientes com linfoma de células B grandes de segunda linha, recidivante ou refratário (r/r LBCL) inelegíveis para transplante devido à idade, comorbidades ou outras razões médicas.

A análise primária mostrou que o zamto–cel demonstrou superioridade clinicamente significativa à quimioimunoterapia (R–GemOx) em pacientes inelegíveis para transplante com alto risco de progressão rápida da doença.1 Esta população do estudo foi caracterizada por idade mais avançada e doença de alto risco: com idade média de 74 anos, 57% dos pacientes com alto Índice Prognóstico Internacional (IPI≥ 3) e 67% com doença em estágio III/IV. O Zamto–cel foi bem tolerado nesta população predominantemente mais velha e de alto risco.1

O Dr. Peter Borchmann, pesquisador principal do estudo DALY 2–EU e Diretor Médico Assistente do Departamento de Hematologia e Oncologia do University Hospital of Cologne, Alemanha, disse: “O Zamto–cel demonstrou superioridade clínica e estatisticamente significativa ao R–GemOx em pacientes inelegíveis para transplante com doença de alto risco, aumentando a sobrevida sem eventos, mantendo um perfil de tolerabilidade favorável. Esses resultados destacam o potencial do zamto–cel como uma nova opção de tratamento importante para uma população de pacientes clinicamente vulneráveis com opções terapêuticas limitadas.”

O Dr. Toon Overstijns, Diretor Executivo da Miltenyi Biomedicine, disse: “Os resultados do DALY 2–EU são um marco importante para o nosso compromisso com o avanço das terapias celulares e genéticas. Zamto–cel – o primeiro CD20–CD19 em tandem (dirigido), terapia com células CAR–T não criopreservadas – demonstrou benefício clínico significativo com eficácia e segurança promissoras, aproximando–nos de fornecer opções de tratamento muito necessárias para pacientes com linfomas de alto risco.”

  • O Zamto–cel é a primeira terapia celular com receptor de antígeno quimérico T (CAR–T) não criopreservado CD20–CD19 (direcionado) em tandem. Os principais mecanismos para recidiva após tratamentos com terapias de células CAR–T direcionadas ao CD19 são a persistência limitada das células CAR–T, inibição da função das células CAR–T e escape de antígeno imunológico CD19. Para minimizar o risco de recaída devido ao escape do antígeno CD19, o zamto–cel utiliza o direcionamento duplo do antígeno CD20 e CD19. O Zamto–cel tem um tempo de fabricação de 12 dias, resultando em um tempo de veia a veia de 14 a 16 dias e reduzindo a probabilidade da terapia de ponte

DALY 2–EU – Resultados primários 1

No ponto de corte dos dados, os pacientes foram distribuídos aleatoriamente para receber zamto–cel (n=82) ou R–GemOx/PolaBR (n=86). O estudo permitiu o cruzamento, 29 pacientes receberam zamto–cel após falha em alcançar uma resposta com R–GemOx (n=28) ou Pola–BR (n=1)

Resultados da eficácia (avaliados pelo comitê de revisão independente cego (BIRC))

  • A sobrevida sem eventos (EFS) mediana do zamto–cel foi de 6,2 meses (IC95% 3,8–13,8) em comparação com 2,5 meses (IC95% 2,0–3,3) do R–GemOx (HR 0,39; IC95% 0,27–0,58; p<0,0001).
  • A sobrevida sem progressão mediana (PFS) foi significativamente mais alta com o zamto–cel aos 8,5 meses (IC95% 3,8–16,8) em comparação com 3,3 meses (IC95% 2,0–3,8) do R–GemOx (HR 0,43 [IC95% 0,28–0,65]; p<0,0001).
  • Na população com intenção de tratamento (ITT), a taxa de resposta geral (ORR) foi de 72% com uma taxa de resposta completa (CRR) de 54% do zamto–cel em comparação com 45% de ORR e 14% de CRR do R–GemOx.

Resultados de Segurança 1
O Zamto–cel foi bem tolerado nesta população de pacientes idosos com alto risco

  • Foi relatada síndrome de liberação de citocinas (CRS) de grau ≥ 3 em 4 pacientes (5,3).
  • A Síndrome de neurotoxicidade associada a células efetoras imunes de grau 3 (ICANS) ocorreu em 1 paciente (1,3%).

Sobre o DALY 2–EU2
O DALY 2–EU (NCT04844866) é um estudo principal, randomizado, multicêntrico, aberto de Fase II conduzido em 12 países dentro da UE, que avalia a segurança e eficácia das células T autólogas geneticamente modificadas que expressam receptor de antígeno quimérico específico anti–CD20 e anti–CD19, zamtocabtageno autoleucel (zamto–cel), em comparação com quimioimunoterapia (CIT), (rituximabe, gencitabina e oxaliplatina (R.GemOx)) ou polatuzumabe vedotina mais bendamustina/rituximabe (Pola–BR)), como uma terapia de segunda linha para linfoma primário de células B grandes recidivante/refratário (r/r LBCL). Até onde sabemos, é o único estudo randomizado de CAR–T nesta população de pacientes até o momento.

Pacientes adultos elegíveis com LBCL r/r refratários ou recidivantes dentro de 24 meses a partir do início do tratamento de primeira linha, receberam pelo menos uma antraciclina e um regime contendo rituximabe, sendo inelegíveis para um transplante de células–tronco.

Os participantes foram randomizados 1:1 para receber zamto–cel ou CIT (R–GemOx/Pola–BR). O Zamto–cel foi administrado como uma infusão única não criopreservada com uma dose de 2,5 x 10^6 células T transduzidas por CAR por kg de peso corporal após linfodepleção com fludarabina e ciclofosfamida. Os pacientes randomizados para o braço comparador receberam R–GemOx ou Pola–BR.

O objetivo primário do estudo é a sobrevida sem eventos (EFS) avaliada por um comitê de revisão independente cego (BIRC), definido como o tempo desde a randomização até a progressão objetiva da doença, falha em atingir uma resposta parcial (PR) ou resposta completa (CR) na ou além da Semana 8, levando a uma nova terapia anti–linfoma ou morte por qualquer causa. Os desfechos secundários incluem sobrevida sem progressão (PFS), melhor taxa de resposta completa (CRR), duração da resposta completa (DOR) e sobrevida global (OS).

Esses dados serão relatados como parte de uma análise provisória pré–planejada da EFS após um acompanhamento médio de 17 meses. Análises adicionais são planejadas com períodos de acompanhamento mais longos e apresentação em reuniões futuras.

Os resultados do DALY 2–UE foram divulgados após publicações anteriores do zamto–cel em outras indicações e populações, incluindo:

  • DALY II USA (NCT04792489), um estudo multicêntrico, aberto, de Fase II de braço único de zamto–cel em pacientes com r/r DLBCL após pelo menos duas linhas anteriores de tratamento, incluindo anticorpo monoclonal anti–CD20 e regime contendo antraciclina e doença mensurável por classificação de Lugano 2014. A ORR na população de pacientes avaliáveis (n=59) avaliada por um Comitê Independente de Radiologia foi de 72,9% (IC 95%, 59,7–83,6) com uma CRR de 49,2% (IC 95%, 35,9–62,5).
  • No ensaio clínico DALY II USA, foi aberta uma coorte dedicada para linfoma do sistema nervoso central r/r. Nesta coorte de 16 pacientes, os dados mostraram uma taxa de resposta geral de 80% e 100% e uma taxa de resposta completa de 50% e 100% no PCNSL (linfoma primário do SNC) e SCNSL (linfoma secundário do SNC), respectivamente.
  • O Zamto–cel está sendo explorado no linfoma de células do manto r/r (MCL) e na transformação de Richter r/r (RT)

Sobre o zamtocabtagene autoleucel (zamto–cel)
Zamto–cel é uma terapia de células T de receptor de antígeno quimérico autólogo (CAR) de investigação projetada para direcionar CD20 e CD19. Ele está sendo estudado em ensaios clínicos para o tratamento de malignidades de células B recidivantes ou refratárias, incluindo linfoma de células B grandes (LBCL), linfoma de células B grandes difusas (DLBCL), linfoma primário e secundário do sistema nervoso central (SNC), linfoma de células do manto (MCL), transformação de Richter (RT) e outras neoplasias de células B.

O Zamto–cel é fabricado na plataforma proprietária da Miltenyi, um sistema fechado e automatizado. O tempo de fabricação de 12 dias resulta em um tempo de veia a veia de 14–16 dias, reduzindo a necessidade de terapia de ponte e aumentando a capacidade de recebimento de terapia celular em pacientes de alto risco com necessidades terapêuticas urgentes. Sua formulação não criopreservada elimina as etapas e os custos logísticos da criopreservação.

Sobre a Miltenyi Biomedicine
A Miltenyi Biomedicine está empenhada em tornar tratamentos inovadores contra o câncer e terapias regenerativas acessíveis a pacientes com doenças graves. Com sua tecnologia de ponta, a empresa inova de forma independente para lidar com cânceres de sangue difíceis de tratar e utilizar o potencial da tecnologia CAR para transformar o atendimento ao paciente. A Miltenyi Biomedicine está pesquisando seu primeiro ativo de terapia celular.

Sobre a Miltenyi Biotec
A Miltenyi Biotec é líder global em tecnologias e serviços inovadores para terapias celulares e genéticas específicas de pacientes, transformando descobertas científicas em tratamentos práticos para medicina personalizada. Com mais de 35 anos de experiência, a empresa apoia descobertas biomédicas e as traduz em aplicações clínicas, aumentando o acesso dos pacientes a novas terapias. A Miltenyi Biotec, com suas soluções integradas, e fábricas de células com certificação GMP, fornece orientação especializada para desenvolvedores de terapias de forma eficiente, desde o desenvolvimento do processo até a comercialização por meio da sua divisão CDMO global da Miltenyi Bioindustry.

Contact
Miltenyi Biomedicine
Jasmine Oberwalleney
Friedrich–Ebert–Strasse 68
51429 Bergisch Gladbach, Alemanha
[email protected]

Referências

  1. Borchmann P, et al. Zamtocabtagene–autoleucel, a tandem CD20–CD19 directed CAR–T cell therapy as second–line treatment for Relapsed/Refractory large B–cell lymphoma: primary analysis of the randomized pivotal DALY 2–EU study. Presented at American Society of Hematology (ASH) Annual Meeting. Abstract #abs25–738.
  2. ClinicalTrials.Gov. Efficacy and Safety of MB–CART2019.1 vs. SoC in Lymphoma Patients (DALY 2–EU). Disponível em: https://clinicaltrials.gov/study/NCT04844866. Acessado em setembro de 2025

MAT–GL–ZA–0003

Data da Redação: Dezembro de 2025


GLOBENEWSWIRE (Distribution ID 1001143511)

Miltenyi Biomedicine presents primary analysis of the pivotal DALY 2-EU trial for second-line relapsed/refractory large B-cell lymphoma at the 67th American Society of Hematology (ASH) Annual Meeting

  • DALY 2–EU results show zamtocabtagene autoleucel (zamto–cel) demonstrated clinically meaningful superiority over chemoimmunotherapy in patients with relapsed/refractory large B–cell lymphoma (r/r LBCL)1
  • Zamto–cel was well–tolerated in the majority of patients. DALY 2–EU included a high–risk study population, characterized by older age and clinically high–risk disease features
  • A 12–day manufacturing time resulted in a vein–to–vein time of 14–16 days, reducing the likelihood for bridging therapy.

BERGISCH GLADBACH, Germany, Dec. 07, 2025 (GLOBE NEWSWIRE) — Miltenyi Biomedicine today announced results from the pivotal DALY 2–EU trial evaluating the efficacy and safety of zamtocabtagene autoleucel (zamto–cel) compared with standard chemoimmunotherapy (R–GemOx or Pola–BR) in patients with second–line, relapsed or refractory large B–cell lymphoma (r/r LBCL) who were transplant–ineligible due to age, comorbidities, or other medical reasons.

The primary analysis showed that zamto–cel demonstrated significant and clinically meaningful superiority over chemoimmunotherapy (R–GemOx) in transplant–ineligible patients who were at a high risk of rapid disease progression.1 This study population was characterized by older age and clinically high–risk disease features: the median age was 74 years, 57% of patients had a high International Prognostic Index (IPI≥ 3) and 67% presented with stage III/IV disease. Zamto–cel was well tolerated in this predominantly older and high–risk population.1

Dr. Peter Borchmann, Lead investigator of DALY 2–EU trial and Assistant Medical Director in the Department of Hematology and Oncology at the University Hospital of Cologne, Germany, said: “Zamto–cel demonstrated clinically meaningful and statistically significant superiority over R–GemOx in transplant–ineligible patients with high–risk disease, improving event–free survival while maintaining a favorable tolerability profile. These findings highlight the potential of zamto–cel as an important new treatment option for a clinically vulnerable patient population with limited therapeutic choices.”

Dr. Toon Overstijns, Chief Executive Officer of Miltenyi Biomedicine, said: “The DALY 2–EU results mark an important milestone in our commitment to advancing cell and gene therapies. Zamto–cel – the first tandem CD20–CD19 (directed), non–cryopreserved CAR–T cell therapy – demonstrated meaningful clinical benefit with promising efficacy and safety, bringing us closer to providing much needed treatment options for patients with high–risk lymphomas.”

  • Zamto–cel is the first tandem CD20–CD19 (directed) non–cryopreserved chimeric antigen receptor T (CAR–T) cell therapy. The main mechanisms for relapse after treatments with CD19–directed CAR–T cell therapies are the limited persistence of CAR–T cells, inhibition of CAR–T cell function, and CD19 immunological antigen escape. To minimize the risk of relapse due to CD19 antigen escape, zamto–cel utilizes dual antigen targeting of CD20 and CD19. Zamto–cel has a 12–day manufacturing time, resulting in a vein–to–vein time of 14–16 days and reducing the likelihood for bridging therapy

DALY 2–EU Primary results1

At the data cutoff, patients were randomly assigned to receive zamto–cel (n=82) or R–GemOx/ PolaBR (n=86). The trial allowed for crossover, 29 patients received zamto–cel following failure to achieve a response with either R–GemOx (n=28) or Pola–BR (n=1)

Efficacy Results (assessed by the blinded independent review committee (BIRC))

  • The median event–free survival (EFS) for zamto–cel was 6.2 months (95% CI 3.8–13.8) compared to 2.5 months (95% CI 2.0–3.3) for R–GemOx (HR 0.39; 95% CI 0.27–0.58; p<0.0001).
  • The median progression–free survival (PFS) was significantly longer with zamto–cel at 8.5 months (95% CI 3.8–16.8) versus 3.3 months (95% CI 2.0–3.8) for R–GemOx (HR 0.43 [95% CI 0.28–0.65]; p<0.0001).
  • In the intent–to–treat (ITT) population, the overall response rate (ORR) was 72% with a 54% complete response rate (CRR) for zamto–cel compared to 45% ORR and 14% CRR for R–GemOx.

Safety Results1
Zamto–cel was well–tolerated in this elderly patient population with high risk

  • Grade ≥ 3 cytokine release syndrome (CRS) was reported in 4 patients (5.3).
  • Grade 3 Immune effector cell–associated neurotoxicity syndrome (ICANS) occurred in 1 patient (1.3%).

About DALY 2–EU2
DALY 2–EU (NCT04844866) is a pivotal, randomized, multi–center, open–label Phase II trial conducted in 12 countries within the EU, evaluating the safety and efficacy of genetically engineered autologous T–cells expressing anti–CD20 and anti–CD19 specific chimeric antigen receptor, zamtocabtagene autoleucel (zamto–cel), compared to chemoimmunotherapy (CIT), (rituximab, gemcitabine, and oxaliplatin (R.GemOx)) or polatuzumab vedotin plus bendamustine/rituximab (Pola–BR)), as a second–line therapy for primary relapsed/refractory large B–cell lymphoma (r/r LBCL). To our knowledge, is the only CAR–T randomized study in this patient population to date.

Eligible patients were adults with r/r LBCL who were refractory or relapsed within 24 months from the start of their first–line treatment, had received at least an anthracycline and a rituximab–containing regimen and were ineligible for a stem–cell transplant.

Participants were randomized 1:1 to receive either zamto–cel or CIT (R–GemOx/Pola–BR). Zamto–cel was administered as a single non–cryopreserved infusion at a dose of 2.5 x 10^6 CAR–transduced T cells per kg body weight after lymphodepletion with fludarabine and cyclophosphamide. Patients randomized to the comparator arm received either R–GemOx or Pola–BR.

The primary endpoint of the trial is event–free survival (EFS) assessed by a blinded independent review committee (BIRC), defined as the time from randomization to objective disease progression, failure to achieve a partial response (PR) or complete response (CR) at or beyond Week 8, leading to a new anti–lymphoma therapy or death from any cause. Secondary endpoints include progression–free survival (PFS), best complete response rate (CRR), duration of complete response (DOR), and overall survival (OS).

These data will be reported as part of a pre–planned EFS interim analysis after a median follow–up of 17 months. Additional analyses are planned with longer follow–up periods and will be presented at future meetings.

DALY 2–EU results join previous zamto–cel publications in other indications and populations, including:

  • DALY II USA (NCT04792489), a multicenter, open label, single–arm Phase II trial of zamto–cel in patients with r/r DLBCL after at least two prior lines of treatment, including anti–CD20 monoclonal antibody and anthracycline–containing regimen and measurable disease per Lugano 2014 classification. The ORR in the evaluable patient population (n=59) as assessed by an Independent Radiology Committee was 72.9% (95% CI, 59.7–83.6) with a CRR of 49.2% (95% CI, 35.9–62.5).
  • In the DALY II USA clinical trial, a dedicated cohort for r/r central nervous system lymphoma was opened. In this cohort of 16 patients, the data showed an overall response rate of 80% and 100% and a complete response rate of 50% and 100% in the PCNSL (primary CNS lymphoma) and SCNSL (secondary CNS lymphoma) respectively.
  • Zamto–cel is being explored in r/r Mantle cell lymphoma (MCL) and r/r Richter’s transformation (RT)

About zamtocabtagene autoleucel (zamto–cel)
Zamto–cel is an investigational autologous chimeric antigen receptor (CAR) T–cell therapy designed to target both CD20 and CD19. It is being studied in clinical trials for the treatment of relapsed or refractory B–cell malignancies, including large B–cell lymphoma (LBCL), diffuse large B–cell lymphoma (DLBCL), primary and secondary central nervous system (CNS) lymphoma, mantle cell lymphoma (MCL), Richter’s transformation (RT), and other B–cell neoplasms.

Zamto–cel is manufactured using Miltenyi’s proprietary platform, a closed, automated system. The manufacturing time of 12 days results in a vein–to–vein time of 14–16 days, reducing the need for bridging therapy and increasing the ability to receive cellular therapy for high risk patients with urgent therapeutic needs. Its non–cryopreserved formulation eliminates cryopreservation–related logistical steps and costs.

About Miltenyi Biomedicine
Miltenyi Biomedicine is committed to making innovative cancer treatments and regenerative therapies accessible to patients with serious diseases. Leveraging cutting–edge technology, the company innovates independently to address hard–to–treat blood cancers and harness the potential of CAR technology to transform patient care. Miltenyi Biomedicine is currently investigating its first cell therapy asset.

About Miltenyi Biotec
Miltenyi Biotec is a global leader in innovating technologies and services for patient–specific cell and gene therapies, transforming scientific discoveries into practical treatments for personalized medicine. With over 35 years of expertise, it supports biomedical discoveries and translates them into clinical applications, enhancing patient access to new therapies. Miltenyi Biotec, with its integrated solutions, including GMP–certified cell factories, provides expert guidance to therapy developers efficiently from process development to commercialization through its Miltenyi Bioindustry global CDMO division.

Contact
Miltenyi Biomedicine
Jasmine Oberwalleney
Friedrich–Ebert–Strasse 68
51429 Bergisch Gladbach, Germany
[email protected]

References

  1. Borchmann P, et al. Zamtocabtagene–autoleucel, a tandem CD20–CD19 directed CAR–T cell therapy as second–line treatment for Relapsed/Refractory large B–cell lymphoma: primary analysis of the randomized pivotal DALY 2–EU study. Presented at American Society of Hematology (ASH) Annual Meeting. Abstract #abs25–738.
  2. ClinicalTrials.Gov. Efficacy and Safety of MB–CART2019.1 vs. SoC in Lymphoma Patients (DALY 2–EU). Available at: https://clinicaltrials.gov/study/NCT04844866. Accessed September 2025.

MAT–GL–ZA–0003

Date of Preparation: December 2025


GLOBENEWSWIRE (Distribution ID 1001143400)

Sabin Vaccine Institute’s Investigational Marburg Vaccine Delivered to Ethiopia for Outbreak Response

WASHINGTON, Dec. 04, 2025 (GLOBE NEWSWIRE) — The Sabin Vaccine Institute (Sabin) has sent more than 640 doses of its investigational cAd3–Marburg Vaccine to Ethiopia to support the country’s response to its first–ever outbreak of Marburg virus disease. Marburg is a highly contagious hemorrhagic fever disease and can have a high case fatality rate of up to 88%. There are currently no licensed vaccines or treatments for Marburg.

Soon after Marburg was confirmed as the virus causing a hemorrhagic fever outbreak in Ethiopia’s southern region, the Ethiopia Ministry of Health engaged Sabin and the U.S. government to request access to Sabin’s investigational cAd3–Marburg Vaccine. The U.S. government approved this request. The Biomedical Advanced Research and Development Authority (BARDA), part of U.S. Department of Health and Human Services’ Administration for Strategic Preparedness and Response (ASPR), funds the development and manufacture of Sabin’s investigational vaccine candidate.

Sabin and the Ethiopia Ministry of Health have entered into a clinical trial agreement under which Sabin is providing BARDA–funded investigational cAd3–Marburg Vaccine doses for a two–cohort Phase 2, rapid response, open–label, randomized trial to assess safety, efficacy, and immunogenicity. Under this approved protocol, Cohort A is limited to high–risk health care and front–line workers or individuals who have had direct contact with infected persons within 21 days, the incubation period for Marburg virus disease. All other health care and front–line workers and contacts will be randomized in Cohort B, some receiving vaccine on Day 1 and others on Day 22 of the trial.

“The Ethiopian Ministry of Health reached out to Sabin early in the outbreak, and we have been working in partnership to support Minister of Health Dr. Mekdes Daba and her team,” says Sabin Chief Executive Officer Amy Finan. “We’ve built on our previous outbreak response experience to quickly assist our Ethiopian colleagues as requested.”

In addition to coordinating directly with the Minister of Health and the Armauer Hansen Research Institute (AHRI), Sabin is also working closely with manufacturing partner ReiThera, clinical research organization IQVIA and the Coalition for Epidemic Preparedness Innovations (CEPI) – the same organizations that came together to support Rwanda’s response to their 2024 outbreak. With authorization from ASPR, Sabin provided cAd3–Marburg Vaccine to the Rwanda Biomedical Center for use in a Phase 2 clinical trial.

Ethiopia’s Ministry of Health has confirmed 13 Marburg infections, including eight deaths, in their most recent update. Another three deaths are suspected but not confirmed. Officials continue to express concern about the outbreak’s proximity to fragile health settings in nearby Kenya and South Sudan.

In addition to Sabin’s single–dose cAd3–Marburg Vaccine being used in a Phase 2 outbreak clinical trial in Rwanda, the vaccine is also in Phase 2 clinical trials in the U.S., Uganda and Kenya. More than 2000 trial participants have received the vaccine and no significant safety concerns have been reported. Results from Phase 1 clinical trials and nonclinical studies indicate that the vaccine is safe and elicits rapid, robust immune responses.

The Sabin cAd3–Marburg Vaccine doses have been supported in whole or in part with federal funds from the U.S. Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under contract numbers 75A50119C00055 and 75A50123C00010. 

Once rare, Marburg virus disease outbreaks have surged in Africa in recent years, with incidents reported in 2023 in Tanzania and Equatorial Guinea, in 2024 in Rwanda and in 2025 in Tanzania. Marburg is transmitted from fruit bats to humans, spreading from person to person through contact with infected bodily fluids. 

About the Sabin Vaccine Institute

The Sabin Vaccine Institute is a leading advocate for expanding vaccine access and uptake globally, advancing vaccine research and development, and amplifying vaccine knowledge and innovation. Unlocking the potential of vaccines through partnership, Sabin has built a robust ecosystem of funders, innovators, implementers, practitioners, policy makers and public stakeholders to advance its vision of a future free from preventable diseases. As a non–profit with three decades of experience, Sabin is committed to finding solutions that last and extending the full benefits of vaccines to all people, regardless of who they are or where they live. At Sabin, we believe in the power of vaccines to change the world. For more information, visit www.sabin.org and follow us on X, @SabinVaccine.

About the cAd3 Platform 

In August 2019, Sabin announced exclusive agreements with GSK for Sabin to advance the development of the prophylactic candidate vaccines against the deadly Zaire ebolavirus, Sudan virus, and Marburg virus. The three candidate vaccines were initially developed collaboratively by the U.S. National Institutes of Health and Okairos, which was acquired by GSK in 2013. The candidate vaccines, based on GSK’s proprietary cAd3 (Chimpanzee Adenovirus Type 3) platform, were further developed by GSK, including the Phase 2 development for the Zaire ebolavirus vaccine. Under the agreements between GSK and Sabin, Sabin exclusively licensed the technology for all three candidate vaccines and acquired certain patent rights specific to these vaccines. Sabin is developing the cAd3–Marburg Vaccine and cAd3–Sudan Vaccine, both are in Phase 2 clinical trials in the US and Africa.  

Media Contact: 
Monika Guttman 
Media Relations Specialist 
Sabin Vaccine Institute 
+1 (202) 662–1841 
[email protected] 

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/c29e0887–de65–45d0–9d2f–4365a8bc78ea


GLOBENEWSWIRE (Distribution ID 9597202)

NeOnc Technologies Reports Updated Clinical Results

CALABASAS, Calif., Nov. 12, 2025 (GLOBE NEWSWIRE) — NeOnc Technologies Holdings, Inc. (NTHI) (“NeOnc” or the “Company”), a multi–Phase 2 clinical–stage biopharmaceutical company developing novel therapies for central nervous system (CNS) cancers today announced updated clinical results from its current Phase 1/2a and compassionate care studies involving a cohort of 24 patients with recurrent WHO Grade III/IV isocitrate dehydrogenase 1 (IDH1)–mutant astrocytoma treated with its investigational intranasal agent, NEO100.

Clinical results provided support that treatment with intranasally delivered NEO100 resulted in a significant radiographic response, confirmed by contrast–enhanced and perfusion MRI, in 5 of 24 patients (21%)—a rate that markedly exceeds the <8% response typically observed with salvage therapies for recurrent gliomas. Additionally, results indicated that 44% of patients in this Phase 2a study achieved six–month progression–free survival (PFS–6), surpassing the 21–31% benchmark reported in historical datasets for IDH1–mutant recurrent high–grade gliomas. Furthermore, 8 of 24 patients (33%) remained alive ≥18 months after initiation of NEO100, demonstrating long term survival.

In addition, no significant toxicity was reported with intranasal administration of NEO100, even with prolonged, chronic intranasal administration, underscoring its potential utility as a therapeutic option for this patient population.

Amir F. Heshmatpour, Executive Chairman, President & CEO of NeOnc Technologies Holdings, Inc., stated: “With these data, we believe NeOnc stands at the threshold of a true game–changer for one of medicine’s greatest unmet needs—recurrent IDH1–mutant hi grade gliomas. For the first time, we’re witnessing evidence of meaningful radiographic responses and support for durable survival in patients who previously had few, if any, options. As we approach full enrollment of the NEO100 trial—and our full data readout six months post–enrollment—we believe this may represent a potential paradigm shift in the treatment of brain cancer. The data will speak for itself—and for the patients whose lives we are determined to transform.”

Mr. Heshmatpour added: “We view this trial as a pivotal value inflection point for NeOnc and for the broader neuro–oncology field signaling a potential move beyond palliation toward measurable, durable disease control and a possible new standard of care in malignant gliomas.”

Founder, Dr. Thomas Chen, Vice–Chairman and Chief Medical Officer noted that, “The data suggest that intranasal NEO100 may be the first central nervous system (CNS)–penetrant metabolic therapy capable of inducing durable response and multi–year survival in recurrent IDH1 mutant gliomas.”

Dr. Henry Friedman of Duke University emphasized the implications of these findings, stating, “The results from NEO100 signify a potential paradigm shift in the treatment of recurrent IDH1–mutant gliomas.”

Key Data Highlights:

  • Radiographic Response: 21% (5 of 24 patients) achieved significant radiographic remission confirmed by contrast–enhanced, T2–FLAIR, and perfusion MRI. This response rate exceeds the <8% typically observed with salvage therapies for recurrent gliomas.
  • Progression–Free Survival (PFS–6): 44% of patients achieved 6–month progression–free survival (PFS–6), outperforming historical benchmarks of 21–31% for IDH1–mutant recurrent high–grade gliomas.
  • Long Term Survival: 33% (8 of 24) demonstrated durable survival ≥18 months post–initiation of NEO100.
  • Tolerability: No significant toxicity observed even with prolonged, chronic intranasal administration.

Study Context: All patients in the combined analysis (1 compassionate–use case, 5 from Phase 1, and 18 from Phase 2a) had confirmed WHO recurrent Grade III/IV IDH1–Mutant Astrocytoma and were included in the analysis if they had been enrolled in the study for at least six months prior to data cutoff. Patients received intranasal NEO100, and results were assessed using Response Assessment in Neuro–Oncology (RANO) criteria.

Collectively, these findings indicate that NEO100 could represent a first–in–class, CNS–penetrant metabolic therapy potentially inducing significant radiographic response and extending survival in patients with recurrent Grade III/IV IDH1–Mutant Astrocytoma, thereby potentially offering a major advancement over past approaches predominantly limited to palliative care.

ABOUT NEONC TECHNOLOGIES HOLDINGS, INC.
NeOnc Technologies Holdings, Inc. is a clinical–stage life sciences company focused on the development and commercialization of central nervous system therapeutics that are designed to address the persistent challenges in overcoming the blood–brain barrier. The company’s NEO™ drug development platform has produced a portfolio of novel drug candidates and delivery methods with patent protections extending to 2038. These proprietary chemotherapy agents have demonstrated positive effects in laboratory tests on various types of cancers and in clinical trials treating malignant gliomas. NeOnc’s NEO100™ and NEO212™ therapeutics are in Phase II human clinical trials and are advancing under FDA Fast–Track and Investigational New Drug (IND) status. The company has exclusively licensed an extensive worldwide patent portfolio from the University of Southern California consisting of issued patents and pending applications related to NEO100, NEO212, and other products from the NeOnc patent family for multiple uses, including oncological and neurological conditions.

For more about NeOnc and its pioneering technology, visit neonc.com.

Important Cautions Regarding Forward Looking Statements
This press release contains “forward–looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward–looking statements can be identified by terminology such as “may,” “will,” “should,” “intend,” “expect,” “plan,” “budget,” “forecast,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “evaluating,” or similar words. Statements that contain these words should be read carefully, as they discuss our future expectations, projections of future results of operations or financial condition, or other forward–looking information.

Examples of forward–looking statements include, among others, statements regarding whether a definitive agreement will be reached with Quazar. These statements reflect our current expectations based on information available at this time, but future events may differ materially from those anticipated.

The “Risk Factors” section of our most recent Annual Report on Form 10–K filed with the Securities and Exchange Commission, along with other cautionary language in that report or in our subsequent filings, outlines important risks and uncertainties. These may cause our actual results to differ materially from the forward–looking statements herein, including but not limited to the failure to finalize the agreement with Quazar, modifications to its terms, or alternative uses of proceeds.

We assume no obligation to revise or update any forward–looking statements, whether as a result of new information, future developments, or otherwise, except as required by applicable securities laws and regulations.

“NEO100” and NEO “212” are registered trademarks of NeOnc Technologies Holdings, Inc.

Company Contact:
[email protected]

Investor Contact:
James Carbonara
Hayden IR
(646)–755–7412
[email protected]


GLOBENEWSWIRE (Distribution ID 9573872)

Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome

HAIFA, Israel, Sept. 18, 2025 (GLOBE NEWSWIRE) — Minovia Therapeutics Ltd. (“Minovia” or the “Company”), a clinical–stage biotechnology company developing novel therapies to treat mitochondrial diseases and combat age–related decline, announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to the Company’s lead investigational compound, MNV–201 for Myelodysplastic Syndrome (MDS), a serious age–related hematopoietic disease. This designation is in addition to the existing FDA Fast Track and Rare Pediatric Disease Designations for MNV–201 in the treatment of Pearson Syndrome, an ultra–rare and life–threatening mitochondrial disorder affecting children and for which the Company is currently conducting a Phase 2 clinical trial.

“The Fast Track Designation in MDS is further validation of the urgency and potential for our science, which targets the mitochondria, a critical multi–functional organelle. FDA designations such as the FTD help us to decrease the potential time to market and provide additional benefits across the FDA process that, we expect, will prove both medically and financially valuable,” said Minovia Co–founder and CEO, Natalie Yivgi–Ohana, Ph.D.

FDA’s Fast Track Designation is designed to accelerate the development and review of therapies for serious or life–threatening conditions with unmet medical need. The designation provides Minovia with the opportunity for increased FDA interactions, potential eligibility for priority review, and the opportunity for a rolling submission of a future Biologics License Application (BLA) for MNV–201.  

The Company also recently announced entry into a definitive business combination agreement (the “Business Combination Agreement”) with Launch One Acquisition Corp. (Nasdaq: LPAA, “Launch One”), a publicly traded special purpose acquisition company. Following the expected closing of the transaction contemplated by this Business Combination Agreement (the “Business Combination”), projected for late 2025, the combined company will operate as Minovia Therapeutics and trade on Nasdaq under a new ticker symbol.

About MDS

MDS is defined by ineffective hematopoiesis resulting in blood cytopenia, and clonal instability with a risk of evolution to Acute Myeloid Leukemia (AML). Patients with MDS collectively have a high symptom burden and are also at risk of death from complications of cytopenia and AML. The goals of therapy for patients with MDS are to improve cytopenia, reduce disease–associated symptoms and the risk of disease progression and death, thereby improving both quality of life and lifespan. The median age at diagnosis of MDS is ~70 years, but surprisingly some of the Pearson Syndrome patients develop MDS in a much higher prevalence relative to the disease population. About 15% of the MDS patients will present with Sideroblastic Anemia, the most common symptom in Pearson Syndrome. Minovia developed novel blood biomarkers to measure mitochondrial health and has been able to demonstrate for the first time that MDS is presumably an age–related mitochondrial disease. As such, Minovia is currently conducting a Phase Ib study of MNV–201 in low–risk MDS patients. Six out of the nine expected patients in the study have been dosed so far.

About MNV–201

MNV–201 is a first–in–class cell therapy that uses Minovia’s proprietary Mitochondrial Augmentation Technology (MAT) to add healthy, energy–producing mitochondria into a patient’s own stem cells — aiming to restore organ function and improve health. In early–stage clinical studies, MAT has demonstrated a strong safety profile and signs of multi–system benefit in patients with Pearson Syndrome, including improvements in growth, muscle function, hematologic stability, and improved quality of life.

About Minovia Therapeutics

Minovia Therapeutics, chaired by John Cox, is a clinical–state biotechnology company working on treatments to replace dead or defective mitochondria with new healthy mitochondria, helping people with mitochondrial diseases and fighting aging. Minovia’s main treatment, MNV–201, is already being tested for Pearson Syndrome and Myelodysplastic Syndrome. Minovia is also developing ways to help people live longer, healthier lives. Based in Haifa, Israel, where it operates a GMP facility for mitochondrial drug substance and drug product manufacturing for clinical trials related to its therapy, Minovia is planning to expand operations to the U.S. For more information, visit www.minoviatx.com.

About Launch One Acquisition Corp.

Launch One Acquisition Corp. is a company set up to merge with and take public an exciting business in healthcare or technology. Listed on Nasdaq under the ticker LPAA, Launch One is led by experienced leaders who want to support game–changing solutions. For more information, contact Jurgen van de Vyver at [email protected].

Additional Information and Where to Find It

In connection with the Business Combination and the Business Combination Agreement, among Launch One, Minovia and Mito US One Ltd., a newly formed Israeli company limited by shares (“Pubco”), and certain other parties named therein. Launch One and Minovia intend to file relevant materials with the U.S. Securities and Exchange Commission (“SEC”), including a Registration Statement on Form F–4 of Pubco (the “Registration Statement”), which will include a proxy statement/prospectus of Launch One, and will file other documents regarding the proposed Business Combination with the SEC. This communication is not intended to be, and is not, a substitute for the proxy statement/prospectus or any other document that Launch One has filed or may file with the SEC in connection with the proposed Business Combination. The Registration Statement has not been filed or declared effective by the SEC. Following such filing and upon such declaration of effectiveness, the definitive proxy statement/prospectus contained within the Registration Statement and other relevant materials for the proposed Business Combination will be mailed or made available to stockholders of Launch One as of a record date to be established for voting on the proposed Business Combination.

Before making any voting or investment decision, investors and stockholders of Launch One are urged to carefully read, when they become available, the entire Registration Statement, the proxy statement/prospectus, and any other relevant documents filed with the SEC, as well as any amendments or supplements to these documents, and the documents incorporated by reference therein, because they will contain important information about Launch One, Minovia, Pubco and the proposed Business Combination . Launch One’s investors and stockholders and other interested persons will also be able to obtain copies of the Registration Statement, the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, other documents filed with the SEC that will be incorporated by reference therein, and all other relevant documents filed with the SEC by Launch One and/or Pubco in connection with the Business Combination, without charge, once available, at the SEC’s website at www.sec.gov, or by directing a request to Launch One or Minovia at the addresses set forth below.

Participants In the Solicitation

Launch One, Minovia, Pubco and their respective directors, executive officers, other members of management and employees may be deemed participants in the solicitation of proxies from Launch One’s stockholders with respect to the Business Combination. Investors and security holders may obtain more detailed information regarding the names, and interests in the Business Combination, of Launch One’s directors and officers in Pubco's and Launch One’s filings with the SEC, including, when filed with the SEC, the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, amendments and supplements thereto, and other documents filed with the SEC. Such information with respect to Minovia’s directors and executive officers will also be included in the proxy statement/prospectus. You may obtain free copies of these documents as described above under the heading “Additional Information and Where to Find It.”

Non–Solicitation

This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the potential transaction and shall not constitute an offer to sell or a solicitation of an offer to buy the securities of Launch One, Pubco, or Minovia, nor shall there be any sale of any such securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act of 1933, as amended.

Forward–Looking Statements

This press release includes certain statements that may be considered forward–looking statements within the meaning of the federal securities laws. Forward–looking statements include, without limitation, statements about future events or Minovia’s, Launch One's, or Pubco's future financial or operating performance. For example, statements regarding the development and regulatory approval of MNV–201, the implications of Fast Track Designation, RPD and PRVs and the timing of future clinical trials or potential applications are forward–looking statements. In some cases, you can identify forward–looking statements by terminology such as “may,” “should,” “could,” “might,” “plan,” “possible,” “project,” “strive,” “budget,” “forecast,” “expect,” “intend,” “will,” “estimate,” “anticipate,” “believe,” “predict,” “potential” or “continue,” or the negatives of these terms or variations of them or similar terminology.

These forward–looking statements regarding future events and the future results of Minovia or Launch One are based on current expectations, estimates, forecasts, and projections about the industry in which Minovia or Launch One operates, as well as the beliefs and assumptions of Minovia’s and Launch One's management. These forward–looking statements are only predictions and are subject to, without limitation, (i) known and unknown risks, including the risks and uncertainties indicated from time to time in the final prospectus of Launch One relating to its initial public offering filed with the SEC, including those under “Risk Factors” therein, and other documents filed or to be filed with the SEC by Launch One or Pubco; (ii) uncertainties; (iii) assumptions; and (v) other factors beyond Minovia’s or Launch One's control that are difficult to predict because they relate to events and depend on circumstances that will occur in the future. They are neither statements of historical fact nor promises or guarantees of future performance. Therefore, Minovia’s actual results may differ materially and adversely from those expressed or implied in any forward–looking statements and Minovia and Launch One therefore caution against relying on any of these forward–looking statements.

These forward–looking statements are based upon estimates and assumptions that, while considered reasonable by Minovia and its management, as the case may be, are inherently uncertain and are inherently subject to risks, variability and contingencies, many of which are beyond Minovia’s or Launch One's control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (i) the occurrence of any event, change or other circumstances that could give rise to the termination of the Business Combination Agreement and any subsequent definitive agreements with respect to the Business Combination; (ii) the outcome of any legal proceedings that may be instituted against Launch One, Minovia, Pubco, or others following the announcement of the Business Combination and any definitive agreements with respect thereto; (iii) the inability to complete the Business Combination due to the failure to obtain consents and approvals of the shareholders of Launch One and Minovia, to obtain financing to complete the Business Combination or to satisfy other conditions to closing, or delays in obtaining, adverse conditions contained in, or the inability to obtain necessary regulatory approvals required to complete the transactions contemplated by the Business Combination Agreement; (iv) changes to the proposed structure of the Business Combination that may be required or appropriate as a result of applicable laws or regulations or as a condition to obtaining regulatory approval of the Business Combination; (v) projections, estimates and forecasts of revenue and other financial and performance metrics, projections of market opportunity and expectations, and the estimated implied enterprise value of Minovia; (vi) Minovia’s ability to scale and grow its business, and the advantages and expected growth of Minovia; (vii) Minovia’s ability to source and retain talent, and the cash position of Minovia following closing of the Business Combination; (viii) the ability to meet stock exchange listing standards in connection with, and following, the consummation of the Business Combination; (ix) the risk that the Business Combination disrupts current plans and operations of Minovia as a result of the announcement and consummation of the Business Combination; (x) the ability to recognize the anticipated benefits of the Business Combination, which may be affected by, among other things, competition, the ability of Minovia to grow and manage growth profitably, maintain key relationships and retain its management and key employees; (xi) costs related to the Business Combination; (xii) changes in applicable laws, regulations, political and economic developments; (xiii) the possibility that Minovia may be adversely affected by other economic, business and/or competitive factors; (xiv) Minovia’s estimates of expenses and profitability; (xv) the failure to realize estimated shareholder redemptions, purchase price and other adjustments; and (xvi) other risks and uncertainties set forth in the filings by Launch One and Minovia with the SEC. There may be additional risks that neither Launch One nor Minovia presently know or that Launch One and Minovia currently believe are immaterial that could also cause actual results to differ from those contained in the forward–looking statements. Any forward–looking statements made by or on behalf of Launch One or Minovia speak only as of the date they are made. Neither Launch One nor Minovia undertakes any obligation to update any forward–looking statements to reflect any changes in their respective expectations with regard thereto or any changes in events, conditions or circumstances on which any such statements are based.  

Contact

Minovia Therapeutics Ltd.
Natalie Yivgi Ohana, Co–Founder and CEO
+972–74–7039954
[email protected]

Launch One Acquisition Corp.
Jurgen van de Vyver
[email protected]
+1–510–692–9600

Investor Relations
Dave Gentry, CEO
RedChip Companies
+1–407–644–4256
[email protected]

Investor Relations
Jules Abraham
Managing Director, Communications
CORE IR
1–212–655–0924
[email protected]


GLOBENEWSWIRE (Distribution ID 9531105)

Minovia Therapeutics Announces $350,000 Grant from Countdown for a Cure Foundation to Develop Mitochondria Blood-Based Biomarkers

HAIFA, Israel, Aug. 27, 2025 (GLOBE NEWSWIRE) — Minovia Therapeutics Ltd. (“Minovia” or the “Company”), a clinical–stage biotechnology company developing novel therapies to treat mitochondrial diseases and combat age–related decline, announces that it has been chosen to receive a $350,000 grant from Countdown for a Cure for a research proposal related to the development of novel mitochondrial blood–based biomarkers.

Minovia Chief Scientific Officer Dr. Noa Sher, commented, “We are grateful to the Countdown for a Cure Foundation, whose funds are expected to be instrumental in our advancing development of blood–based functional mitochondrial biomarkers. These biomarkers will enable identification of patients who may benefit from our mitochondrial augmentation technology, or MAT, proprietary platform, as well as patient follow–up after MAT treatment. Given how critical mitochondria are to human health, we envision a world in which assessment of mitochondrial biomarkers is available in routine checkups for individuals of all age groups. We look forward to optimizing this technology.”

In addition to the novel MAT–based mitochondrial therapies under development, Minovia has set dual goals of developing biomarkers to quantify mitochondrial content, quality and function and of using these biomarkers to determine mitochondrial scores in healthy individuals relative to mitochondrial disease patients. For this reason, Minovia opened a clinical trial in Sheba Medical Center to collect blood samples from both healthy volunteers and patients suffering from mitochondrial diseases. The Countdown for a Cure grant will fund clinical operations for blood sample collection from approximately 30 patients with primary mitochondrial diseases, and 140 samples from healthy controls. The samples collected will be analyzed in Minovia’s labs with its newly developed biomarkers, and a “MitoScore” will be determined for each sample. In addition, the Countdown for a Cure funding will support the development of new biomarkers using novel research tools.

Mitochondrial dysfunction is known to occur in rare genetic mitochondrial diseases, as well as in chronic and age–related diseases. There is a profound unmet need for treatment of these devastating diseases, as there are currently no approved therapies for mitochondrial dysfunction and no functional tests to diagnose and quantify mitochondrial dysfunction in individuals of all ages.

The Company also recently announced entry into a definitive business combination agreement (the “Business Combination Agreement”) with Launch One Acquisition Corp. (Nasdaq: LPAA, “Launch One”), a publicly traded special purpose acquisition company. Following the expected closing of the transaction contemplated by this Business Combination Agreement (the “Business Combination”), projected for late 2025, the combined company will operate as Minovia Therapeutics and trade on Nasdaq under a new ticker symbol.

About Minovia Therapeutics

Minovia Therapeutics, chaired by John Cox, is a clinical–state biotechnology company working on treatments to replace dead or defective mitochondria with new healthy mitochondria, helping people with mitochondrial diseases and fighting aging. Its main drug product, MNV–201, is already being tested for Pearson Syndrome and Myelodysplastic Syndrome. Minovia is also developing ways to help people live longer, healthier lives. Based in Haifa, Israel, where it operates a GMP facility for mitochondrial drug substance and drug product manufacturing for clinical trials related to its therapy, Minovia is planning to expand to the U.S. For more information, visit www.minoviatx.com.

About Countdown for a Cure

Countdown For A Cure (CFAC) is a non–profit organization dedicated to advancing mitochondrial research and medicine, improving the lives of those affected by diseases linked to mitochondrial dysfunction. Founded in 2024 by Mitzi and Jeff Solomon, who experienced mitochondrial dysfunction and its impact on their family firsthand, CFAC partners with leading research institutions and patient advocacy groups to fund cutting–edge science and provide critical support to families. Mitzi and Jeff are driven by an unwavering commitment to supporting families, accelerating research, and uniting a community determined to make a tangible difference in the lives of those affected by mitochondrial disease and all diseases connected to mitochondrial dysfunction.

About Launch One Acquisition Corp.

Launch One Acquisition Corp. is a company set up to merge with and take public an exciting business in healthcare or technology. Listed on Nasdaq under the ticker LPAA, Launch One is led by experienced leaders who want to support game–changing solutions. For more information, contact Jurgen van de Vyver at [email protected].

Additional Information and Where to Find It

In connection with the Business Combination and the Business Combination Agreement, among Launch One, Minovia and Mito US One Ltd., a newly formed Israeli company limited by shares (“Pubco”), and certain other parties named therein. Launch One and Minovia intend to file relevant materials with the U.S. Securities and Exchange Commission (“SEC”), including a Registration Statement on Form F–4 of Pubco (the “Registration Statement”), which will include a proxy statement/prospectus of Launch One, and will file other documents regarding the proposed Business Combination with the SEC. This communication is not intended to be, and is not, a substitute for the proxy statement/prospectus or any other document that Launch One has filed or may file with the SEC in connection with the proposed Business Combination. The Registration Statement has not been filed or declared effective by the SEC. Following such filing and upon such declaration of effectiveness, the definitive proxy statement/prospectus contained within the Registration Statement and other relevant materials for the proposed Business Combination will be mailed or made available to stockholders of Launch One as of a record date to be established for voting on the proposed Business Combination.

Before making any voting or investment decision, investors and stockholders of Launch One are urged to carefully read, when they become available, the entire Registration Statement, the proxy statement/prospectus, and any other relevant documents filed with the SEC, as well as any amendments or supplements to these documents, and the documents incorporated by reference therein, because they will contain important information about Launch One, Minovia, Pubco and the proposed Business Combination. Launch One’s investors and stockholders and other interested persons will also be able to obtain copies of the Registration Statement, the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, other documents filed with the SEC that will be incorporated by reference therein, and all other relevant documents filed with the SEC by Launch One and/or Pubco in connection with the Business Combination, without charge, once available, at the SEC’s website at www.sec.gov, or by directing a request to Launch One or Minovia at the addresses set forth below.

Participants In the Solicitation

Launch One, Minovia, Pubco and their respective directors, executive officers, other members of management and employees may be deemed participants in the solicitation of proxies from Launch One’s stockholders with respect to the Business Combination. Investors and security holders may obtain more detailed information regarding the names, and interests in the Business Combination, of Launch One’s directors and officers in Pubco's and Launch One’s filings with the SEC, including, when filed with the SEC, the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, amendments and supplements thereto, and other documents filed with the SEC. Such information with respect to Minovia’s directors and executive officers will also be included in the proxy statement/prospectus. You may obtain free copies of these documents as described above under the heading “Additional Information and Where to Find It.”

Non–Solicitation

This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the potential transaction and shall not constitute an offer to sell or a solicitation of an offer to buy the securities of Launch One, Pubco, or Minovia, nor shall there be any sale of any such securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act of 1933, as amended.

Forward–Looking Statements

This press release includes certain statements that may be considered forward–looking statements within the meaning of the federal securities laws. Forward–looking statements include, without limitation, statements about future events or Minovia’s, Launch One's, or Pubco's future financial or operating performance. For example, statements regarding the development and regulatory approval of MNV–201, the implications of Fast Track Designation, RPD and PRVs and the timing of future clinical trials or potential applications are forward–looking statements. In some cases, you can identify forward–looking statements by terminology such as “may,” “should,” “could,” “might,” “plan,” “possible,” “project,” “strive,” “budget,” “forecast,” “expect,” “intend,” “will,” “estimate,” “anticipate,” “believe,” “predict,” “potential” or “continue,” or the negatives of these terms or variations of them or similar terminology.

These forward–looking statements regarding future events and the future results of Minovia or Launch One are based on current expectations, estimates, forecasts, and projections about the industry in which Minovia or Launch One operates, as well as the beliefs and assumptions of Minovia’s and Launch One's management. These forward–looking statements are only predictions and are subject to, without limitation, (i) known and unknown risks, including the risks and uncertainties indicated from time to time in the final prospectus of Launch One relating to its initial public offering filed with the SEC, including those under “Risk Factors” therein, and other documents filed or to be filed with the SEC by Launch One or Pubco; (ii) uncertainties; (iii) assumptions; and (v) other factors beyond Minovia’s or Launch One's control that are difficult to predict because they relate to events and depend on circumstances that will occur in the future. They are neither statements of historical fact nor promises or guarantees of future performance. Therefore, Minovia’s actual results may differ materially and adversely from those expressed or implied in any forward–looking statements and Minovia and Launch One therefore caution against relying on any of these forward–looking statements.

These forward–looking statements are based upon estimates and assumptions that, while considered reasonable by Minovia and its management, as the case may be, are inherently uncertain and are inherently subject to risks, variability and contingencies, many of which are beyond Minovia’s or Launch One's control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (i) the occurrence of any event, change or other circumstances that could give rise to the termination of the Business Combination Agreement and any subsequent definitive agreements with respect to the Business Combination; (ii) the outcome of any legal proceedings that may be instituted against Launch One, Minovia, Pubco, or others following the announcement of the Business Combination and any definitive agreements with respect thereto; (iii) the inability to complete the Business Combination due to the failure to obtain consents and approvals of the shareholders of Launch One and Minovia, to obtain financing to complete the Business Combination or to satisfy other conditions to closing, or delays in obtaining, adverse conditions contained in, or the inability to obtain necessary regulatory approvals required to complete the transactions contemplated by the Business Combination Agreement; (iv) changes to the proposed structure of the Business Combination that may be required or appropriate as a result of applicable laws or regulations or as a condition to obtaining regulatory approval of the Business Combination; (v) projections, estimates and forecasts of revenue and other financial and performance metrics, projections of market opportunity and expectations, and the estimated implied enterprise value of Minovia; (vi) Minovia’s ability to scale and grow its business, and the advantages and expected growth of Minovia; (vii) Minovia’s ability to source and retain talent, and the cash position of Minovia following closing of the Business Combination; (viii) the ability to meet stock exchange listing standards in connection with, and following, the consummation of the Business Combination; (ix) the risk that the Business Combination disrupts current plans and operations of Minovia as a result of the announcement and consummation of the Business Combination; (x) the ability to recognize the anticipated benefits of the Business Combination, which may be affected by, among other things, competition, the ability of Minovia to grow and manage growth profitably, maintain key relationships and retain its management and key employees; (xi) costs related to the Business Combination; (xii) changes in applicable laws, regulations, political and economic developments; (xiii) the possibility that Minovia may be adversely affected by other economic, business and/or competitive factors; (xiv) Minovia’s estimates of expenses and profitability; (xv) the failure to realize estimated shareholder redemptions, purchase price and other adjustments; and (xvi) other risks and uncertainties set forth in the filings by Launch One and Minovia with the SEC. There may be additional risks that neither Launch One nor Minovia presently know or that Launch One and Minovia currently believe are immaterial that could also cause actual results to differ from those contained in the forward–looking statements. Any forward–looking statements made by or on behalf of Launch One or Minovia speak only as of the date they are made. Neither Launch One nor Minovia undertakes any obligation to update any forward–looking statements to reflect any changes in their respective expectations with regard thereto or any changes in events, conditions or circumstances on which any such statements are based.

Contacts:

Minovia Therapeutics Ltd.
Natalie Yivgi Ohana, Co–Founder and CEO
+972–74–7039954
[email protected]

Launch One Acquisition Corp.
Jurgen van de Vyver
[email protected]
+1–510–692–9600

Investor Relations
Dave Gentry, CEO
RedChip Companies
+1–407–644–4256
[email protected]

Investor Relations
Jules Abraham
Managing Director, Communications
CORE IR
1–212–655–0924
[email protected]


GLOBENEWSWIRE (Distribution ID 9518763)

Dr. Falk Pharma und Allianthera (Suzhou) Biopharmaceuticals gehen strategische Partnerschaft für die Entwicklung eines neuartigen AhR-Agonisten ein

Freiburg (Deutschland), Suzhou (China) und Boston (USA), 30. Juli 2025: 

Dr. Falk Pharma GmbH und Allianthera (Suzhou) Biopharmaceuticals Co., Ltd. geben strategische Partnerschaft für die Entwicklung eines neuartigen AhR–Agonisten zur Behandlung mittelschwerer bis schwerer Colitis ulcerosa bekannt.

Dr. Falk Pharma GmbH, ein forschungsbasiertes Pharmaunternehmen und Spezialist für Verdauungs– und Stoffwechselmedizin, und Allianthera (Suzhou) Biopharmaceuticals Co., Ltd., sowie sein verbundenes Unternehmen Allianthera Boston, Inc., ein Biotechnologieunternehmen mit Fokus auf klinischer Forschung und Entwicklung neuartiger Arzneimittel im Bereich Immunologie und entzündlicher Erkrankungen (Allianthera), geben die Unterzeichnung eines Vertrages über die gemeinsame Entwicklung, mögliche Lizenzierung, Herstellung und Vermarktung des neuartigen, niedermolekularen Aryl–Hydrocarbon–Rezeptor–(AhR–)Agonisten ATB102 bekannt, der gegenwärtig eine klinische Studie der Phase I in den USA durchläuft.

Im Rahmen des neu unterzeichneten Vertrags werden Dr. Falk Pharma und Allianthera gemeinsam an der Entwicklung von ATB102 zur Behandlung entzündlicher Darmerkrankungen mit anfänglichem Fokus auf refraktärer, mittelschwerer bis schwerer Colitis ulcerosa (CU) arbeiten. Dr. Falk Pharma erhält die Exklusivrechte für die Lizenzierung, Herstellung und Vermarktung von ATB102 auf der ganzen Welt, mit Ausnahme von Festlandchina, Hongkong, Macau und Taiwan. Allianthera erhält eine Einmalzahlung bei Vertragsunterzeichnung, Zahlungen bei der Erreichung wesentlicher Entwicklungsmeilensteine sowie eine Lizenzgebühr und anschließend, während der Vermarktung, bestimmte Meilensteinzahlungen und gestaffelte Lizenzgebühren.

Bei dem von Allianthera entwickelten Wirkstoff ATB102 handelt es sich um einen auf den Darm beschränkten AhR–Agonisten, der einen neuen therapeutischen Ansatz bei entzündlichen Darmerkrankungen darstellt. Er richtet sich speziell gegen Entzündungen und Schleimhautschäden im Gastrointestinaltrakt, mit besonderem Schwerpunkt auf der Behandlung refraktärer, mittelschwerer bis schwerer CU. Aus präklinischen Daten geht hervor, dass ATB102 die Immunhomöostase unterstützt, die Integrität der Schleimhautbarriere wiederherstellt und zudem antifibrotischen und antioxidativen Nutzen mit sich bringt, was diesen Wirkstoff zu einer potenziellen neuen Therapieoption für Patient*innen macht, die nicht auf bestehende Therapien ansprechen oder ein Rezidiv erlitten haben.

Dr. Falk Pharma wird, in Zusammenarbeit mit seiner 100%igen Tochtergesellschaft Losan Pharma GmbH, einem international führenden Auftragsentwicklungs– und –herstellungspartner (CDMO) und Galenikexperten, eine innovative Formulierung von ATB102 mit gezielter Freisetzung im Darm entwickeln, um die derzeitige Formulierung, die auf einer sofortigen Freisetzung beruht, zu ergänzen.

„Der Aryl–Hydrocarbon–Rezeptor stellt einen wahrhaft neuartigen Wirkmechanismus dar, um auf den erheblichen unerfüllten medizinischen Bedarf bei entzündlichen Darmerkrankungen, und insbesondere bei Colitis ulcerosa, einzugehen. Das ATB–Team hat einen unserer Ansicht nach erstklassigen AhR–Agonisten entdeckt, der hinsichtlich der Sicherheit, Anreicherung im Darm und therapeutischen Wirkung optimiert ist. Wir freuen uns sehr auf die Zusammenarbeit mit Dr. Falk Pharma, einem Unternehmen mit tiefgehender Fachkompetenz in Formulierung und klinischer Entwicklung. Wir sind davon überzeugt, dass wir dieses Programm durch die Kombination unserer komplementären Stärken beschleunigen können und den Patient*innen damit schneller als je zuvor eine dringend benötigte Therapie von hoher Qualität zur Verfügung stellen können“, erklärt Yuanhua Ding, CEO von Allianthera.

Dr. Kai Pinkernell, Geschäftsführer des Geschäftsbereichs Science & Innovation bei Dr. Falk Pharma, fügt hinzu: „Wir sind begeistert, unsere F&E–Pipeline um ATB102 ergänzen zu können, und freuen uns darauf, das Potenzial dieses neuartigen Moleküls in zukünftigen klinischen Studien gemeinsam mit Allianthera zu erforschen. Die Partnerschaft mit Allianthera steht für die Kraft wissenschaftlicher Exzellenz, gegenseitiges Vertrauen und eine gemeinsame Vision, Menschen mit entzündlichen Darmerkrankungen zu besserer Lebensqualität zu verhelfen. Diese Zusammenarbeit spiegelt nicht nur unsere wachsende weltweite Entwicklung und unser Engagement für Innovation in Bereichen mit hohem unerfüllten medizinischen Bedarf wider, sondern wird auch das Wirkstoffportfolio von Dr. Falk Pharma für die Behandlung von Erkrankungen des Verdauungstrakts erweitern.“

Über Colitis ulcerosa
Colitis ulcerosa gehört zur Gruppe der chronisch entzündlichen Darmerkrankungen (CED). Weitere Erkrankungen aus dieser Gruppe sind Morbus Crohn sowie die mikroskopischen Kolitiden lymphozytäre und kollagene Kolitis.

Colitis ulcerosa ist auf den Dickdarm beschränkt und beginnt typischerweise im Rektum, wobei sich die Entzündung kontinuierlich über den restlichen Teil des Dickdarms ausbreiten kann. Die genaue Ursache der Colitis ulcerosa ist noch unklar; jedoch werden derzeit eine Fehlfunktion des Immunsystems, genetische Faktoren, ein Ungleichgewicht des Darmmikrobioms sowie eine Dysregulation der Integrität der intestinalen Schleimhaut oder Umweltfaktoren als mögliche Auslöser untersucht.

Weitere Informationen finden Sie auf der Seite der Dr. Falk Pharma: https://de.drfalkpharma.com/de/indikationen/colitis–ulcerosa/

Über Allianthera
Allianthera wurde im November 2020 mit einem Forschungs– und Entwicklungszentrum in Suzhou, China, und einer Tochtergesellschaft in Boston, USA, gegründet. Allianthera hat es sich zum Ziel gesetzt, weltweit führend in therapeutischer Innovation zu werden, indem es sein umfassendes pathophysiologisches Wissen und seine führende Expertise in der Forschung und Entwicklung neuartiger Wirkstoffe mit Technologien von Kooperationspartnern kombiniert und gleichzeitig einzigartige Stärken in verschiedenen Regionen, in Ost oder West, nutzt. Das Unternehmen baut durch interne F&E–Programme und externe Partnerschaften ein starkes Wirkstoffportfolio auf, wobei der anfängliche Fokus auf der Behandlung immunologischer und entzündlicher Krankheiten mit hohem ungedecktem medizinischem Bedarf liegt.

Weitere Informationen zu Allianthera finden Sie auf LinkedIn: https://www.linkedin.com/company/allianthera–suzhou–biopharmaceutical–co–ltd

Partnerschaften mit Dr. Falk Pharma
Dr. Falk Pharma kooperiert mit zuverlässigen Partnern, um innovative Behandlungskonzepte mit Potenzial zu entwickeln und umzusetzen. Diese können aus jeglichen pharma–kologischen Konzepten hervorgehen: von „Small Molecules“ bis hin zu Biologika oder neuartigen Drug–Delivery–Technologien. In seinen Partnerschaftsprojekten betrachtet das Familienunternehmen verschiedenste Entwicklungsschritte in allen Phasen der prä–klinischen/klinischen Entwicklung und Vermarktung, die ihren Ursprung in der akademischen Forschung, im klinischen Einsatz, in Start–ups oder auch in etablierten Firmen haben können.

Dr. Falk Pharma ist ein Branchenführer für innovative Arzneimittelformulierungen, die Wirkstoffe zu bestimmten funktionalen Abschnitten des Magen–Darm–Traktes transportieren. Viele ihrer Produkte gelten als Behandlungsstandard. Das Unternehmen baut auf die langjährige enge Zusammenarbeit mit renommierten klinischen Zentren und wichtigen akademischen und klinischen Meinungsbildnern in den Bereichen Gastroenterologie, Hepatologie und Stoffwechselerkrankungen.

Mehr Informationen und Kontaktdaten gibt es unter https://de.drfalkpharma.com/de/partnering/

Über die Dr. Falk Pharma GmbH
Die Dr. Falk Pharma GmbH mit Sitz in Freiburg entwickelt und vertreibt seit über 60 Jahren innovative Arzneimittel für verschiedene Erkrankungen der Leber, der Gallenwege, des Darms und der Speiseröhre. Als internationaler Spezialist für Verdauungs– und Stoffwechselmedizin bringt das Unternehmen Ärztinnen und Ärzte, Wissenschaftler*innen und Patient*innen zusammen, um neue und wirkungsvolle Ansätze der Versorgung der Betroffenen zu entwickeln. Im Fokus der Forschungsinvestitionen und Studien steht das Ziel, die klinische Praxis und das Leben der Patient*innen nachhaltig zu verbessern. Das stetig wachsende Familienunternehmen mit globaler Vernetzung und 10 Tochtergesellschaften in Europa und Australien forscht und entwickelt in Freiburg. Hergestellt werden die pharmazeutischen Produkte in Europa, größtenteils in Deutschland, Frankreich, Italien und der Schweiz. Das mit der Region Breisgau tief verbundene Unternehmen beschäftigt rund 1400 Mitarbeiter*innen, davon 340 in Freiburg.

Weitere Informationen über die Dr. Falk Pharma finden Sie unter: www.drfalkpharma.de


GLOBENEWSWIRE (Distribution ID 1001120434)