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Apex Labs Granted Israel MoH Approval to Expand Phase 2b Macrodose Psilocybin PTSD Clinical Trial

  • Israel’s Ministry of Health (MoH) approval to add additional sites to APEX SUMMIT–90 160 patient phase 2b macrodose clinical trial:
    • Tel Aviv University (TAU)’s Institute for Psychedelic Research located at the Sagol Brain Institute (SGI) in Tel–Aviv Sourasky Medical Center.
    • Be'er Yaakov Mental Hospital (Merhavim) Center for Psychedelic Studies.
  • For more information or to register visit clinicaltrials.gov (Canada) and mytrials.gov (Israel).

VANCOUVER, British Columbia, June 12, 2025 (GLOBE NEWSWIRE) — Apex Labs Ltd. (APEX or the Company), a pharmaceutical company transforming the standard of mental health care with psilocybin is pleased to announce the approval by the Israeli MoH and IRBs to open two additional clinical trial sites for SUMMIT–90. The trial is a double–blind, placebo controlled phase 2b study evaluating multiple doses of APEX–90, a psilocybin macrodose utilizing APEX’s US patent pending capsule. APEX–90 is administered in–clinic with study–assisted psychotherapy for severe depression within diagnosed PTSD. Israel is facing a severe mental health crisis: 44% of adults report depression and 42% PTSD, far above the 8–13% depression and 6–10% PTSD rates seen in the US and Canada.

This MoH approval leverages the expertise of TAU’s renowned SGI and Merhavim Hospital, which both have a rich history of pioneering research in neurological sciences. Their cutting–edge facilities and teams profound understanding of PTSD dynamics are poised to add patient recruitment expertise.

“I am honoured to have been able to facilitate this new partnership; another example of building important bridges between Canada and Israel in innovative clinical research, which will result in advancing patient access to emerging treatments,” says Sharon J. Fraenkel, TAU Canada’s CEO for Ottawa, Quebec, and Atlantic Canada, on behalf of the organization.

“As someone deeply connected to Israel, witnessing the toll of PTSD among my loved ones, I'm driven to lead research that brings hope and healing,” says Alysa Langburt, APEX’s VP of Global Clinical Development. “This marks more than a clinical milestone, it represents a fundamental step towards transforming the mental health landscape in Canada and Israel, where the need has never been greater. Through our incredible partnerships, we aim to catalyze a shift in access, care and outcomes for those suffering with PTSD.”

“SUMMIT–90 offers a beacon of hope for the significant numbers suffering from PTSD in Canada and Israel,” says Tyler Powell, co–Founder and CEO of APEX. “It underscores our commitment to global mental health innovation and our belief in the opportunity for clinically proven psilocybin therapies to transform mental health care.”

About Apex Labs Ltd.
APEX is a patient–driven pharmaceutical company focused on revolutionizing the standard of mental health care with psilocybin. APEX's strategy is two–pronged, clinical evaluation of drug assets alongside a robust Early Access Program. APEX recognizes and prioritizes Veterans as a patient base with the most severe unmet medical need.

Visit apexlabs.com for more information and follow APEX on LinkedIn, Twitter and Instagram.

Forward–Looking Statements
This release contains certain “forward–looking statements” and certain “forward–looking information” as defined under applicable Canadian securities laws. Forward–looking statements and information can generally be identified by the use of forward–looking terminology such as “may”, “will”, “expect”, “intend”, “estimate”, “anticipate”, “believe”, “continue”, “plans” or similar terminology. Forward–looking statements and information are based on forecasts of future results, estimates of amounts not yet determinable and assumptions that, while believed by management to be reasonable, are inherently subject to significant business, economic and competitive uncertainties and contingencies. Forward–looking statements and information are subject to various known and unknown risks and uncertainties, many of which are beyond the ability to control or predict, that may cause the Company's actual results, performance or achievements to be materially different from those expressed or implied thereby, and are developed based on assumptions about such risks, uncertainties and other factors set out here in, including but not limited to: receiving authorization of Health Canada Dealers Licence; filing US provisional patent, the Company evaluating the safety and efficacy of APEX–52 (psilocybin) and APEX–90 (psilocybin) in treating depression in Veterans and patients with Post–Traumatic Stress Disorder; statements related to APEX–52 and APEX–90, including manufacturing, dosing, and trial details; statements made by the Company's executives with respect to Health Canada's Dealer's Licence and capsule patent filing; the Company's efforts around the Early Access Program; statements made relating to Canadian Veteran patients; approvals by the Israeli Ministry of Health and ethics; the inherent risks involved in the general securities markets; uncertainties relating to the availability and costs of financing needed in the future; the inherent uncertainty of cost estimates and the potential for unexpected costs and expenses, currency fluctuations; regulatory restrictions, liability, competition, loss of key employees and other related risks and uncertainties. The Company undertakes no obligation to update forward–looking information except as required by applicable law. Such forward–looking information represents managements' best judgment based on information currently available. No forward–looking statement can be guaranteed and actual future results may vary materially. Accordingly, readers are advised not to place undue reliance on forward–looking statements or information.

SOURCE Apex Labs Ltd.


GLOBENEWSWIRE (Distribution ID 9467934)

Dr. Falk Pharma announces positive results from its pivotal phase 3 trial on norucholic acid in primary sclerosing cholangitis

Freiburg, May 7th, 2025

Study results demonstrate superiority of norucholic acid (NCA) over placebo in the combined primary endpoint. There is currently no approved medicine to treat primary sclerosing cholangitis. The results of the 96–week analysis of the NUC–5 trial will be presented at 2025 EASL Congress in Amsterdam.

Dr. Falk Pharma, a research–based pharmaceutical company specializing in digestive and metabolic medicine, today announced positive results from its pivotal, phase 3 trial (NUC–5) on norucholic acid (NCA) in primary sclerosing cholangitis (PSC).

NUC–5 (NCT03872921) is a double–blind, placebo–controlled trial enrolling 301 patients with PSC, who receive either 1,500 mg NCA or placebo for a total of 192 weeks. At the primary data analysis after 96 weeks of treatment, the primary endpoint of combined partial normalization of blood levels of a liver enzyme linked to PSC (alkaline phosphatase) and no worsening of disease stage on histology was achieved by a statistically significantly greater proportion of patients receiving NCA than placebo. Significant superiority of NCA was also observed in multiple secondary endpoints. The safety results revealed similar rates of study patients with adverse events and serious adverse events between the NCA and placebo groups.

The topline results from NUC–5 will be presented during the Late Breaker session on Saturday, May 10, 2025 at the EASL Congress in Amsterdam.

PSC is a rare, progressive disease in which the immune system attacks the bile ducts in the liver, which leads to fibrosis, or the formation of scar tissue. A considerable proportion of patients develop bile duct, liver, or colorectal cancer, while many others eventually progress to cirrhosis of the liver. No approved pharmaceutical treatment is currently available, and the most effective treatment option is liver transplantation.

“Finding a medicine that effectively treats PSC has been a challenge in the field of hepatology for decades” said Prof. Michael Trauner, Head of the Division of Gastroenterology and Hepatology at the Medical University of Vienna, Austria and principal investigator of the trial. “After so many disappointments in this space, the first positive results from a phase 3 study on PSC is a watershed moment for people with PSC, their families, physicians and the entire PSC community. The results of this study will not only advance patient care but will also give researchers new insights into the disease itself”.

“We are very excited about the positive results of the NUC–5 trial, which was the largest clinical trial on PSC to date which compared biopsies from patients before and after treatment” said Dr. Kai Pinkernell, Managing Director Science and Innovation for Dr. Falk Pharma. “A trial of this duration and involvement is a major undertaking, and we thank all of the participating patients, investigators, and trial staff for their dedication to this trial”.

About norucholic acid

Norucholic acid is an engineered bile acid derivative. Unlike endogenous bile acids, it undergoes no meaningful amidation with glycine or taurine, allowing NCA to be absorbed from bile by cholangiocytes and subsequently re–secreted into bile by hepatocytes in a process called cholehepatic shunting. This together with putative direct anti–inflammatory and anti–fibrotic mechanisms are thought to confer protective effects in PSC. In a previous phase 2 trial, 12 weeks of treatment with NCA was shown to significantly reduce levels of alkaline phosphatase (ALP).

About NUC–5

NUC–5 is a randomized, double–blind, placebo–controlled trial enrolling 301 patients with biopsy–confirmed PSC and levels of ALP at least 1.5–fold greater than the upper limit of normal (ULN). The combined primary endpoint was partial normalization of ALP to <1.5–fold ULN and no worsening of disease stage by histology (Ludwig classification). The key secondary endpoint was partial normalization of ALP to <1.5–fold ULN and no worsening of disease stage by histology (modified Nakanuma staging).

At week 96, 15.1% of patients receiving NCA achieved the primary endpoint compared to 4.2% of placebo patients (p = 0.0048). Similarly, 15.1% of NCA patients versus 5.1% of placebo patients achieved the key secondary endpoint (p = 0.0086). NCA treatment led to improvement by at least 1 Ludwig stage for 25.2% of NCA patients compared to 10.5% of placebo patients (p = 0.0217). Furthermore, worsening by at least one Ludwig stage was observed in 40.4% of placebo patients compared to 20.3% of NCA patients (p = 0.0069). Blood levels of multiple liver enzymes improved under NCA but not placebo. NCA was well tolerated, with similar rates of serious adverse events between the two arms.

NUC–5 is still ongoing, with patients receiving continuous double–blind treatment with either NCA or placebo for an additional 96 weeks. Additional results will be reported after the conclusion of all 192 weeks of double–blind treatment. Patients completing all 192 weeks of double–blind treatment will have the option of receiving open–label treatment with NCA for up to 72 weeks.

Read the abstract here (LBO–001)

About Dr. Falk Pharma GmbH

Dr. Falk Pharma GmbH has been developing and marketing innovative medicines to treat a wide range of gastrointestinal disorders like inflammatory bowel disease or eosinophilic esophagitis as well as hepatobiliary disorders such as primary biliary cholangitis for over 60 years. As the international experts in digestive and metabolic medicine, the company brings together physicians, scientists, and patients to devise new and powerful approaches to patient care. Dr. Falk Pharma engages in pre–clinical and clinical stage research that aims to meaningfully improve therapeutic practice as well as patient health and well–being. A family–owned business with a global presence, Dr. Falk Pharma has ten affiliates in Europe and Australia and is continuously growing. The company has its headquarters and R&D facilities in Freiburg, Germany, its pharmaceutical products are manufactured in Europe, mainly at sites in Germany, France, Italy and Switzerland. Dr. Falk Pharma GmbH employs approximately 1400 individuals globally and 340 in Freiburg.

Further information on Dr. Falk Pharma can be found online: https://drfalkpharma.com


GLOBENEWSWIRE (Distribution ID 1001095027)

Dr. Falk Pharma gibt positive Ergebnisse seiner zulassungs-relevanten Phase-III-Studie zu Norucholsäure bei primär sklerosierender Cholangitis bekannt

Freiburg, den 7. Mai 2025

Die Studienergebnisse zeigen die Überlegenheit von Norucholsäure (NCA) gegenüber Placebo im kombinierten primären Endpunkt. Es gibt derzeit kein zugelassenes Arzneimittel zur Behandlung von primär sklerosierender Cholangitis. Die Ergebnisse der Studie NUC–5 nach 96 Wochen der Analyse werden auf dem EASL–Kongress 2025 in Amsterdam vorgestellt.

Dr. Falk Pharma, ein forschungsbasiertes Pharmaunternehmen und Spezialist für Verdauungs– und Stoffwechselmedizin, gab heute positive Ergebnisse seiner zulassungsrelevanten Phase–III–Studie (NUC–5) zu Norucholsäure (NCA) bei primär sklerosierender Cholangitis (PSC) bekannt.

NUC–5 (NCT03872921) ist eine doppelblinde, placebokontrollierte klinische Studie, in die 301 Patient*innen mit PSC aufgenommen wurden, welche für insgesamt 192 Wochen entweder 1500 mg NCA oder Placebo erhalten. Bei der primären Datenanalyse nach 96 Behandlungswochen wurde der kombinierte primäre Endpunkt aus teilweiser Normalisierung der Blutwerte eines mit PSC verknüpften Leberenzyms (alkalische Phosphatase) und keiner Verschlechterung des histologisch bestätigten Krankheitsstadiums von einem statistisch signifikant größeren Anteil der Patient*innen in der NCA–Gruppe als in der Placebogruppe erreicht. Die signifikante Überlegenheit von NCA wurde auch bei mehreren sekundären Endpunkten festgestellt. Die Sicherheitsdaten ergaben in der NCA–Gruppe und der Placebogruppe ähnliche Raten von unerwünschten Ereignissen und schwerwiegenden unerwünschten Ereignissen.

Die wichtigsten Ergebnisse aus NUC–5 werden auf dem EASL–Kongress in Amsterdam in der Late Breaker Session am Samstag, dem 10. Mai 2025 vorgestellt.

PSC ist eine seltene, progrediente Krankheit, bei der das Immunsystem die Gallengänge in der Leber angreift, was zu Fibrose, d. h. zur Bildung von Narbengewebe führt. Ein erheblicher Anteil der Patient*innen entwickelt eine Krebserkrankung (Gallengangs–, Leber– oder Kolorektalkarzinom), während bei vielen anderen Patient*innen die Krankheit schließlich zu Leberzirrhose fortschreitet. Derzeit steht keine zugelassene pharmakologische Therapie zur Verfügung. Die wirksamste Behandlung besteht in der Lebertransplantation.

„Ein Arzneimittel zu finden, mit dem sich PSC wirksam behandeln lässt, stellt seit Jahrzehnten eine Herausforderung auf dem Gebiet der Hepatologie dar“, erklärt Prof. Michael Trauner, Leiter der klinischen Abteilung für Gastroenterologie und Hepatologie an der Medizinischen Universität Wien und leitender Prüfarzt der Studie. „Nach so vielen Enttäuschungen in diesem Bereich sind die ersten positiven Ergebnisse einer Phase–III–Studie zu PSC ein Wendepunkt für Menschen mit PSC, deren Familien, Ärztinnen, Ärzte und die gesamte PSC–Gemeinschaft. Die Ergebnisse dieser Studie werden nicht nur zu Fortschritten in der Patientenversorgung führen, sondern den Wissenschaftlern auch neue Erkenntnisse über die Krankheit selbst liefern.“

„Wir sind begeistert von den positiven Ergebnissen der NUC–5–Studie, der bisher größten klinischen Studie zu PSC, in der vor und nach der Behandlung entnommene Biopsieproben der Patient*innen verglichen wurden“, so Dr. Kai Pinkernell, Managing Director Science & Innovation bei Dr. Falk Pharma. „Eine klinische Studie mit dieser Dauer und Beteiligung ist eine große Aufgabe und wir danken allen teilnehmenden Patient*innen, Prüfärzt*innen und Studienmitarbeitenden für ihr Engagement bei dieser Studie.“

Über Norucholsäure

Norucholsäure ist ein semi–synthetisches Gallensäurederivat. Im Gegensatz zu endogener Gallensäure erfährt NCA mit Glycin oder Taurin keine bedeutsame Amidierung, wodurch NCA von Cholangiozyten aus der Gallenflüssigkeit aufgenommen und anschließend von Hepatozyten in einem als „cholehepatisches Shunting“ bezeichneten Prozess wieder in die Gallenflüssigkeit abgegeben wird. Es wird angenommen, dass dies zusammen mit mutmaßlichen direkten entzündungshemmenden und antifibrotischen Mechanismen für eine schützende Wirkung bei PSC sorgt. In einer früheren Phase–II–Studie wurde durch eine 12–wöchige Behandlung mit NCA nachweislich eine signifikante Senkung der alkalischen Phosphatase (ALP)–Werte erzielt.

Über NUC–5

NUC–5 ist eine randomisierte, doppelblinde, placebokontrollierte Studie mit 301 Teilnehmenden mit anhand einer Biopsieprobe bestätigter PSC und ALP–Werten, die bei mindestens dem 1,5–Fachen des oberen Normalwerts (ULN) liegen. Der kombinierte primäre Endpunkt war eine teilweise Normalisierung des ALP–Werts auf unterhalb des 1,5–fachen ULN und keine Verschlechterung des histologisch bestätigten Krankheitsstadiums (Klassifikation nach Ludwig). Der sekundäre Hauptendpunkt war eine teilweise Normalisierung des ALP–Werts auf unterhalb des 1,5–fachen ULN und keine Verschlechterung des histologisch bestätigten Krankheitsstadiums (modifiziertes Staging nach Nakanuma).

Nach 96 Wochen erreichten 15,1 % der mit NCA behandelten Patient*innen den primären Endpunkt, im Vergleich zu 4,2 % der Patient*innen in der Placebogruppe (p = 0,0048). Ebenso erreichten 15,1 % der Patient*innen in der NCA–Gruppe und 5,1 % der Patient*innen in der Placebogruppe den wichtigsten sekundären Endpunkt (p = 0,0086). Die Behandlung mit NCA führte bei 25,2 % der Patient*innen zu einer Verbesserung der Fibrose (um mindestens ein Stadium nach Ludwig), im Vergleich zu 10,5 % der Patient*innen in der Placebogruppe (p = 0,0217). Darüber hinaus war bei 40,4 % der Placebopatient*innen eine Verschlechterung um mindestens ein Stadium nach Ludwig zu beobachten, im Vergleich zu 20,3 % der NAC–Patient*innen (p = 0,0069). Die Blutwerte mehrerer Leberenzyme verbesserten sich unter NCA, jedoch nicht unter Placebo. NCA wurde gut vertragen, in beiden Behandlungsarmen traten ähnliche Raten an schwerwiegenden unerwünschten Ereignissen auf.

NUC–5 läuft noch und die Teilnehmenden erhalten für weitere 96 Wochen eine kontinuierliche doppelblinde Behandlung mit entweder NCA oder Placebo. Weitere Ergebnisse werden nach Abschluss der gesamten 192 Wochen der doppelblinden Behandlung veröffentlicht. Patient*innen, die alle 192 Wochen der doppelblinden Behandlung abschließen, erhalten die Möglichkeit einer unverblindeten Behandlung mit NCA für bis zu eineinhalb weitere Jahre.

Abstrakt hier lesen (LBO–001)

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 1001095027)

Sabin Begins Marburg Vaccine Trial in U.S.

WASHINGTON, April 16, 2025 (GLOBE NEWSWIRE) — The Sabin Vaccine Institute has launched a multi–site Phase 2 clinical trial in the U.S. for its Marburg vaccine candidate, administering doses to the first participants in Melbourne, Florida. This trial builds on ongoing Phase 2 testing in Kenya and Uganda, with initial findings from that research expected in the coming months.

Sabin’s vaccine development efforts, including clinical trials, are becoming increasingly critical as Marburg outbreaks grow in frequency, underscoring the urgent need for vaccines to protect those at highest risk. Sabin supported an open–label Phase 2 clinical trial sponsored by the Rwanda Biomedical Centre (RBC) by supplying the investigational vaccine during Rwanda’s 2024 Marburg outbreak. More than 1,700 individuals — primarily frontline health care workers — were vaccinated, with first doses arriving within nine days of the outbreak being declared. Data from the RBC trial will be shared with Sabin to support the vaccine’s licensure.

Rwanda’s outbreak ended on December 20 with a case fatality rate of 23%, lower than the historical average of 50%. Fatality rates in outbreaks can vary due to multiple factors, including greater surveillance, prompt detection, supportive care, and the overall response effort. On January 20, Tanzania declared an outbreak of Marburg virus disease, which ended on March 13.

Currently, there are no approved vaccines for Marburg virus disease.

For the U.S. clinical trial that began this week, Sabin will recruit 200 volunteers aged 18 to 70 across four locations – in addition to Melbourne, the vaccine will be tested at sites in Dallas, Texas; Huntsville, Alabama; and Peoria, Illinois. The randomized, placebo–controlled, double–blind trial will continue to evaluate safety and immunogenicity, and will monitor vaccinated volunteers for a year.

“Recent outbreaks highlight the urgent need to strengthen our defenses against this deadly and unforgiving disease,” says Amy Finan, Sabin’s Chief Executive Officer. “Sabin’s Phase 2 clinical trials will generate essential data to move this vaccine closer to licensure — and offer a potentially life–saving tool where none exists.”

Marburg is a filovirus, in the same family as the virus that causes Ebola. Like Ebola, Marburg virus disease spreads via direct contact with the blood or other bodily fluids of infected individuals. It is highly virulent and causes hemorrhagic fever.

“Conducting clinical trials in Africa is key to evaluating the vaccine in regions where Marburg and other filoviruses are most common or endemic,” notes Kelly Warfield, Sabin’s President of Research & Development. “The U.S. trial will give us vital safety and immune response data for non–endemic populations, helping us better prepare for outbreaks and spread of this disease.”

Based on the cAd3 platform, Sabin’s single–dose investigational Marburg vaccine was found to be promising in Phase 1 clinical and non–clinical studies, with results showing it to be safe, while eliciting rapid and robust immune responses.

The Marburg vaccine trials are supported by the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, under multi–year contracts between the organizations.

BARDA and Sabin began working together in September 2019 to develop monovalent vaccine candidates for Marburg virus and Sudan virus diseases. To date, Sabin has received around $252 million in contract awards from BARDA for furthering vaccine research and development against these two disease threats.

A Phase 2 clinical trial for Sabin’s Sudan virus vaccine is underway in Uganda and Kenya. The cAd3 Sudan vaccine candidate will also be tested among adult volunteers in the U.S. later this year.

This project has been supported in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Center for the Biomedical Advanced Research and Development Authority (BARDA), under contract numbers 75A50119C00055 and 75A50123C00010.

For information about Sabin’s Phase 2 Marburg vaccine trials, visit:
Clinicaltrials.gov — NCT06620003 (U.S. trial)
Clinical.trials.gov — NCT05817422 (Uganda and Kenya trial)
Pan African Clinical Trials Registry — PACTR202306534727467 (Uganda and Kenya trial)

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 Sabin’s Vaccine R&D and 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.

Media Contact:
Monika Guttman
Senior Media Relations Specialist
Sabin Vaccine Institute
+1 (202) 621–1691
[email protected]

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/cdb8a6cd–2ff2–49bd–aeb4–43e859c1b44f


GLOBENEWSWIRE (Distribution ID 9423434)

Minovia Therapeutics Announces FDA Clearance of Second IND Application, for a Phase II Clinical Trial of Lead Product MNV-201 in Pearson Syndrome

MNV–201 is Minovia’s second generation mitochondrial cell therapy product composed of autologous hematopoietic stem cells enriched with allogeneic mitochondria

Rare Pediatric Designation granted

MNV–201 is also being studied in a Phase Ib for low–risk Myelodysplastic Syndrome; Preliminary clinical data demonstrate safety and efficacy

HAIFA, Israel, April 03, 2025 (GLOBE NEWSWIRE) — Minovia Therapeutics Ltd, a clinical stage biopharmaceutical company advancing mitochondrial therapies for primary and secondary mitochondrial diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its second Investigational New Drug (IND) application for MNV–201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. The IND supports the initiation of a Phase II clinical trial of MNV–201 in pediatric patients with Pearson Syndrome, a primary mitochondrial disease.

Based on previous clinical experience from the 1st generation product, MNV–101 (autologous hematopoietic stem cell product augmented with syngeneic maternal mitochondria), Minovia designed this phase II study with change in growth (height SDS) as primary endpoint. According to the natural history study recently published by Dr. Rebecca Ganetzky from CHOP, all patients with Pearson Syndrome suffer from failure to thrive and do not respond to growth hormone. Natural history shows an annual reduction of 0.5 units in height SDS, while MNV–101 treated patients showed stabilization or improvement, with no decline of height SDS at the 6 and 12 month follow up time points in a comparable subset of patients. This change in growth correlated with an improved International Pediatric Mitochondrial Disease Scale (IPMDS), which measures how the patient feels and functions (R2=0.9; p=0.0036). Linear growth was also suggested as an objective and clinically meaningful endpoint for a pivotal trial in Pearson by the FDA in early interactions.

“The FDA’s clearance of our IND marks an important achievement for Minovia, allowing us to clinically evaluate our allogeneic mitochondrial cell therapy approach and proceed with the Phase II clinical program for this first–in–class allogeneic mitochondrial therapy for Pearson Syndrome patients,” said Natalie Yivgi Ohana, PhD, CEO of Minovia. “We are pleased to have safely dosed three Pearson patients enrolled in an ongoing study under the Israeli Ministry of Health. We look forward to treating additional patients under this IND, as well as to learning about the potential of MNV–201 to improve growth in this patient population.”

“We are pleased that our cumulative interactions with the FDA enabled alignment on requirements for the entire MNV–201 program, including preclinical, CMC, and clinical aspects,” said Noa Sher, PhD, CSO of Minovia. “Early clinical and regulatory experience with MNV–101 shaped the current program and enabled a successful IND submission.”

The Phase II clinical trial is an open–label, single dose study to evaluate the safety and efficacy of MNV–201 in pediatric subjects diagnosed with Pearson Syndrome. The trial will also enable assessment of efficacy in improving growth and quality of life. The study is expected to enroll three additional patients up to a total of 6 patients. For more information visit clinicaltirals.gov

About MNV–201
MNV–201 is an autologous hematopoietic stem cell product enriched with allogeneic mitochondria. MNV–201 aims to restore mitochondrial function in patient hematopoietic stem cells, resulting in improved differentiation and function. Preclinical research suggests the potential for safe dosing with low immunogenicity risk and scalable manufacturing to address the significant number of patients who are potentially eligible for MNV–201 therapy.

About Pearson Syndrome
Pearson Syndrome is a multisystem progressive pediatric mitochondrial disease caused by single large–scale mitochondrial deletions (SLSMDS) of mitochondrial DNA (mtDNA), with consequent defects in the mitochondrial respiratory chain function. Pearson Syndrome classically presents in the first year of life with bone marrow failure and exocrine pancreatic dysfunction. Patients have macrocytic sideroblastic anemia that is frequently transfusion–dependent and may be accompanied by thrombocytopenia and neutropenia. Pancreatic dysfunction occurs secondary to fibrosis and leads to chronic diarrhea, malabsorption, and failure to thrive. Pearson Syndrome is marked by accumulating organ system involvement and worsening disease: variable other organ involvement can occur, including renal tubulopathy, liver cholestasis and/or fibrosis, adrenal insufficiency, diabetes mellitus, cardiomegaly, and/or cardiac conduction defects. Pearson Syndrome is universally fatal and since there is no effective therapy, the diagnosis of Pearson Syndrome is one of the worst diagnoses that a caregiver must deliver to parents of an affected infant. MNV–201 aims to reduce disease–associated symptoms and the risk of disease progression and death, thereby improving both lifespan and quality of life.

About Minovia Therapeutics
Minovia Therapeutics Ltd. is a clinical stage biotechnology company advancing mitochondrial therapies for primary–genetic and age–related mitochondrial diseases. Minovia's clinical stage product candidate, MNV–201, is composed of mobilized peripheral blood, autologous CD34+ cells enriched with allogeneic, cryopreserved placental derived mitochondria, produced by Minovia's proprietary Mitochondrial Augmentation Technology (MAT). The enrichment of hematopoietic stem cells with healthy and functional mitochondria aims to restore stem cells function of patients suffering mitochondrial dysfunction, caused both by mtDNA mutations or deletions in pediatric patients suffering from primary mitochondrial diseases, or in adults with age–related diseases. MNV–201 is currently in clinical studies for pediatric patients with single–large scale mtDNA deletion syndromes (Pearson Syndrome and Kearn Sayre Syndrome) with five patients successfully dosed; and in Low Risk Myelodysplastic Syndrome. For more information, please visit www.minoviatx.com or follow the Company LinkedIn.

Contact Information: Natalie Yivgi Ohana, Co–Founder and CEO

Phone: +972–74–7039954

Email: [email protected]


GLOBENEWSWIRE (Distribution ID 9416391)

Society for Clinical Research Sites (SCRS) e Fortrea Fazem Parceria para Promover a Colaboração na Pesquisa Clínica

DURHAM, N.C., Feb. 20, 2025 (GLOBE NEWSWIRE) — A Society for Clinical Research Sites (SCRS) e a Fortrea (Nasdaq: FTRE), uma organização líder global de pesquisa por contrato (CRO), tem o prazer de anunciar o patrocínio da Fortrea do grupo de trabalho SCRS Collaborate Forward.

Composto por 16 organizações líderes de Parceiros de Impacto Global, o grupo de trabalho Collaborate Forward explorará e desenvolverá as melhores práticas para reduzir os encargos administrativos em todo o ecossistema de pesquisa clínica. O grupo está empenhado em promover transparência e colaboração para enfrentar os desafios enfrentados pelos centros de pesquisa clínica. O aprimoramento dos processos internos visa tornar os sites mais sustentáveis e os ensaios mais eficientes – levando, em última análise, a uma experiência mais simplificada para os pacientes.

O patrocínio da Fortrea é um investimento significativo para a promoção da inovação em todo o setor e reflete a dedicação da empresa em colocar os sites na vanguarda do planejamento de ensaios clínicos.

“Estamos entusiasmados com a parceria com a SCRS para lançar e apoiar este grupo de trabalho”, disse Mike Clay, vice–presidente sênior de Entrega Global de Projetos da Fortrea. “Os ensaios clínicos estão se tornando cada vez mais complexos, e a indústria enfrenta uma pressão crescente para acelerar a inovação para os pacientes. Acreditamos que a colaboração com centros de pesquisa clínica é essencial para a disponibilidade de eficiências e ganhos de produtividade que irão agilizar o processo de testes clínicos. Esta iniciativa desenvolverá soluções tangíveis que patrocinadores de estudos clínicos, CROs, fornecedores, sites e grupos de defesa do paciente podem apoiar. Como CRO líder, temos orgulho de estar na vanguarda desse esforço, garantindo que os sites permaneçam centrais para impulsionar o progresso e promover maior colaboração em toda a indústria para a oferta de tratamentos que mudem mais rapidamente a vida dos pacientes.”

“A pesquisa clínica requer uma interdependência única para gerar os melhores resultados. O Collaborate Forward compartilhará sucessos de parcerias que impactam as pessoas, os processos e a tecnologia que melhoram a pesquisa clínica hoje ”, acrescentou Sean Soth, vice–presidente sênior de Estratégia e Parcerias de Negócios Globais da SCRS. “É um prazer ter a Fortrea como patrocinadora da Collaborate Forward. Essa parceria ressalta o valor da colaboração intersetorial e do esforço coletivo necessário para impulsionar um progresso significativo para a criação de um ecossistema de ensaios clínicos mais conectado e eficiente.”

O Collaborate Forward se concentrará inicialmente na inicialização do estudo, mostrando as vantagens da colaboração por meio de histórias convincentes, estudos de caso e insights baseados em dados. O grupo se reunirá regularmente para trocar ideias, avaliar as tendências do setor e desenvolver ferramentas pragmáticas que patrocinadores e CROs possam implementar em suas organizações. As atualizações sobre o progresso do grupo de trabalho serão compartilhadas ao longo de 2025 por meio de Summits e publicações da SCRS Site Solutions, destacando os principais resultados e realizações colaborativas.

A SCRS convida patrocinadores e CROs comprometidos com a sustentabilidade do site a se juntarem a esse esforço e contribuírem para moldar um cenário de pesquisa clínica mais eficaz e sinérgico. Para mais informação sobre como participar, contacte Brian Egan.

Sobre a Society for Clinical Research Sites

A Society for Clinical Research Sites (SCRS) é a principal organização de defesa dedicada a unificar a voz da comunidade global de centros de pesquisa clínica. Representando mais de 11.000 sites de pesquisa em todo o mundo, a SCRS facilita colaborações e conversas da indústria dedicadas à defesa, educação, orientação e conexão focadas no site. A SCRS é uma defensora ativa e influente de sites em iniciativas do setor para garantir que a perspectiva dos sites seja ouvida e valorizada. Saiba mais e participe em myscrs.org. Nossa voz. Nossa comunidade. Seu sucesso.

Sobre a Fortrea

A Fortrea (Nasdaq: FTRE) é fornecedora líder global de soluções para o desenvolvimento clínico para a indústria de ciências da vida. Fazemos parcerias com grandes e emergentes empresas biofarmacêuticas, de biotecnologia, de dispositivos médicos e de diagnóstico para impulsionar a inovação na saúde que acelera terapias que mudam a vida dos pacientes. A Fortrea fornece gerenciamento de testes clínicos de fase I–IV, farmacologia clínica e serviços de consultoria. As soluções da Fortrea utilizam suas três décadas de experiência abrangendo mais de 20 áreas terapêuticas, sua dedicação ao rigor científico, insights excepcionais e uma forte rede de pesquisadores. Nossa equipe talentosa e diversificada que trabalha em cerca de 100 países é dimensionada para fornecer soluções focadas e ágeis para clientes de todo o mundo. Saiba mais sobre como a Fortrea está se tornando uma força transformadora de pipeline para pacientes em Fortrea.com e siga–nos em LinkedIn e X (ex–Twitter).

Contatos da SCRS:

Marissa Hill (Mídia) – 267–865–3296, [email protected]
Brian Egan (Mídia) – 518–207–6965, [email protected]

Contatos da Fortrea:

Galen Wilson (Mídia) – 703–298–0802, [email protected]
Kate Dillon (Mídia) – 646–818–9115, [email protected]


GLOBENEWSWIRE (Distribution ID 9362056)

La Society for Clinical Research Sites (SCRS) et Fortrea s’associent pour promouvoir la collaboration dans le domaine de la recherche clinique

DURHAM, N.C., 20 févr. 2025 (GLOBE NEWSWIRE) — La Society for Clinical Research Sites (SCRS) et Fortrea (Nasdaq : FTRE), l’une des principales organisations de recherche contractuelle (ORC) à l’international, ont le plaisir d’annoncer que Fortrea parraine désormais le groupe de travail Collaborate Forward de la SCRS.

Le groupe de travail Collaborate Forward, composé de 16 grandes organisations Global Impact Partner, a pour mission d’explorer et de développer les meilleures pratiques pour réduire la charge administrative dans l’ensemble du secteur de la recherche clinique. Le groupe s’engage à favoriser la transparence et la collaboration pour relever les défis auxquels sont confrontés les sites de recherche clinique. Il cherche à rendre les sites plus durables et les essais plus efficaces en améliorant les processus internes, ce qui permettra aux patients de bénéficier d’une expérience plus sereine.

Le parrainage de Fortrea constitue un investissement important dans la promotion de l’innovation à travers le secteur et témoigne de son engagement à donner la priorité aux sites lors de la planification des essais cliniques.

« Nous sommes ravis de travailler en partenariat avec la SCRS pour créer et accompagner ce groupe de travail », déclare Mike Clay, vice–président senior de Global Project Delivery chez Fortrea. « Les essais cliniques deviennent de plus en plus complexes et le secteur fait face à une pression croissante pour accélérer l’innovation au service des patients. Nous sommes convaincus qu’une collaboration avec les sites de recherche clinique est essentielle pour réaliser des gains d’efficacité et de productivité qui permettront de simplifier le processus d’essai clinique. Cette initiative permettra de mettre au point des solutions concrètes auxquelles les promoteurs d’études cliniques, les ORC, les fournisseurs, les sites et les groupes de défense des intérêts des patients pourront adhérer. En tant qu’ORC de premier plan, nous sommes fiers de participer à cet effort, en veillant à ce que les sites restent au cœur du progrès et en encourageant une plus grande collaboration à travers le secteur, afin de mettre plus rapidement à la disposition des patients des traitements qui changeront leur vie. »

« La recherche clinique nécessite une interdisciplinarité particulière pour générer les meilleurs résultats. Collaborate Forward partagera les réussites de partenariats qui ont un impact sur les personnes, les processus et la technologie qui améliorent la recherche clinique aujourd’hui », ajoute Sean Soth, vice–président senior de la stratégie et des partenariats commerciaux mondiaux chez la SCRS. « Nous sommes ravis de pouvoir compter sur Fortrea en tant que promoteur fondateur de Collaborate Forward. Ce partenariat souligne l’importance de la collaboration interdisciplinaire et de l’effort collectif nécessaire pour réaliser des progrès significatifs dans la création d’un système d’essais cliniques plus connecté et plus efficace. »

Collaborate Forward se concentrera d’abord sur le lancement d’études, en présentant les avantages de la collaboration par le biais de témoignages convaincants, d’études de cas et d’informations fondées sur des données. Le groupe se réunira régulièrement pour échanger des points de vue, évaluer les tendances du secteur et développer des outils pragmatiques que les promoteurs et les ORC pourront mettre en œuvre au sein de leurs organisations. Des informations sur les progrès réalisés par le groupe de travail seront communiquées tout au long de l’année 2025 lors des sommets SCRS Site Solutions ainsi que dans des publications, mettant en évidence les principaux résultats et les réussites en matière de collaboration.

La SCRS invite les promoteurs et les ORC impliqués dans la durabilité des sites à se joindre à cet effort et à contribuer à la création d’un environnement de recherche clinique plus efficace et plus dynamique. Pour plus d’informations sur les modalités de participation, veuillez contacter Brian Egan.

À propos de la Society for Clinical Research Sites

La Society for Clinical Research Sites (SCRS) est la principale organisation de défense des intérêts des sites de recherche clinique à l’échelle internationale. Représentant plus de 11 000 sites de recherche dans le monde, elle facilite les collaborations au sein du secteur et les conversations sur la défense des intérêts des sites, l’éducation, le mentorat et les relations. La SCRS est un porte–parole actif et influent pour les sites dans les initiatives du secteur, qui s’assure que le point de vue des sites est entendu et pris en compte. Pour en savoir plus et participer, consultez le site myscrs.org. Notre voix. Notre communauté. Votre succès.

À propos de Fortrea

Fortrea (Nasdaq : FTRE) est un fournisseur mondial de premier plan de solutions de développement clinique pour le secteur des sciences de la vie. Fortrea s’associe à des sociétés établies et émergentes du domaine biopharmaceutique, de la biotechnologie, des dispositifs médicaux et des diagnostics pour stimuler l’innovation en matière de santé, et accélérer la mise au point de traitements révolutionnaires pour les patients. Fortrea propose des services de gestion d’essais cliniques de phase I à IV, de pharmacologie clinique et de consulting. Nos solutions s’appuient sur 30 ans d’expérience dans 20 disciplines thérapeutiques, une passion pour la rigueur scientifique, des connaissances exceptionnelles et un solide réseau de centres de recherche. Notre équipe talentueuse et diversifiée, qui travaille dans une centaine de pays, est dimensionnée pour fournir des solutions ciblées et flexibles à nos clients, partout dans le monde. Pour en savoir plus sur la manière dont Fortrea est un moteur d’influence du pipeline au patient, rendez–vous sur Fortrea.com et suivez–nous sur LinkedIn et X (anciennement Twitter).

Contacts SCRS :

Marissa Hill (Média) – 267–865–3296, [email protected]
Brian Egan (Média) – 518–207–6965, [email protected]

Contacts Fortrea :

Galen Wilson (Média) – 703–298–0802, [email protected]
Kate Dillon (Média) – 646–818–9115, [email protected]


GLOBENEWSWIRE (Distribution ID 9362056)

Society for Clinical Research Sites (SCRS) und Fortrea gehen eine Partnerschaft zur Förderung der Zusammenarbeit in der klinischen Forschung ein

DURHAM, North Carolina, Feb. 20, 2025 (GLOBE NEWSWIRE) — Die Society for Clinical Research Sites (SCRS) und Fortrea (Nasdaq: FTRE), ein führendes globales Auftragsforschungsinstitut (Contract Research Organization, CRO), freuen sich, die Förderung der Arbeitsgruppe „SCRS Collaborate Forward“ durch Fortrea bekanntzugeben.

Die Arbeitsgruppe „Collaborate Forward“, die aus 16 führenden Global Impact Partner–Organisationen besteht, wird bewährte Verfahren zur Reduzierung des Verwaltungsaufwands im gesamten Bereich der klinischen Forschung untersuchen und entwickeln. Die Gruppe setzt sich für Transparenz und Zusammenarbeit ein, um die Herausforderungen zu bewältigen, mit denen klinische Forschungszentren konfrontiert sind. Durch die Verbesserung interner Prozesse sollen die Standorte nachhaltiger und die Studien effizienter werden, was letztlich zu einem reibungsloseren Ablauf für die Patienten führt.

Die Förderung durch Fortrea stellt eine bedeutende Investition in die Förderung branchenweiter Innovationen dar und spiegelt das Engagement des Unternehmens wider, Prüfzentren an die Spitze der Planung klinischer Studien zu bringen.

„Wir freuen uns, mit der SCRS zusammenzuarbeiten, um diese Arbeitsgruppe ins Leben zu rufen und zu unterstützen“, so Mike Clay, Senior Vice President of Global Project Delivery bei Fortrea. „Klinische Studien werden immer komplexer und die Branche steht unter zunehmendem Druck, Innovationen für Patienten zu beschleunigen. Wir sind der Meinung, dass die Zusammenarbeit mit klinischen Forschungszentren der Schlüssel zu mehr Effizienz und Produktivitätssteigerungen ist, die den Prozess klinischer Studien optimieren werden. Diese Initiative wird konkrete Lösungen entwickeln, hinter denen sich Sponsoren klinischer Studien, CROs, Anbieter, Prüfzentren und Patientenvertretungsgruppen versammeln können. Als führendes Auftragsforschungsinstitut sind wir stolz darauf, bei diesen Bemühungen an vorderster Front zu stehen und sicherzustellen, dass die Prüfzentren weiterhin eine zentrale Rolle bei der Förderung des Fortschritts und der Förderung einer stärkeren branchenweiten Zusammenarbeit spielen, um Patienten schneller lebensverändernde Behandlungen zukommen zu lassen.“

„Klinische Forschung erfordert eine einzigartige Interdependenz, um die besten Ergebnisse zu erzielen. Collaborate Forward wird über Erfolge von Partnerschaften berichten, die sich auf die Menschen, Prozesse und Technologien auswirken und die klinische Forschung heute verbessern“, fügte Sean Soth, Senior Vice President, Strategy and Global Business Partnerships, SCRS, hinzu. „Wir freuen uns, Fortrea als Gründungssponsor von Collaborate Forward begrüßen zu dürfen. Diese Partnerschaft unterstreicht den Wert branchenübergreifender Zusammenarbeit und der gemeinsamen Anstrengungen, die erforderlich sind, um einen bedeutenden Fortschritt bei der Schaffung eines stärker vernetzten und effizienteren Ökosystems für klinische Studien zu erzielen.“

Collaborate Forward wird sich zunächst auf den Beginn von Studien konzentrieren und die Vorteile der Zusammenarbeit durch fesselnde Geschichten, Fallstudien und datengestützte Erkenntnisse aufzeigen. Die Gruppe wird regelmäßig zusammenkommen, um Erkenntnisse auszutauschen, Branchentrends zu bewerten und pragmatische Tools zu entwickeln, die Sponsoren und CROs in ihren Organisationen umsetzen können. Über den Fortschritt der Arbeitsgruppe wird im Laufe des Jahres 2025 auf den SCRS Site Solutions Summits und in Veröffentlichungen berichtet, in denen die wichtigsten Ergebnisse und Erfolge der Zusammenarbeit hervorgehoben werden.

Die SCRS lädt Sponsoren und CROs, die sich für die Nachhaltigkeit von Standorten einsetzen, ein, sich diesen Bemühungen anzuschließen und zur Gestaltung einer effektiveren und synergetischeren klinischen Forschungslandschaft beizutragen. Für weitere Informationen zur Teilnahme wenden Sie sich bitte an Brian Egan.

Über die Society for Clinical Research Sites

Die Society for Clinical Research Sites (SCRS) ist die führende Interessenvertretung, die sich dafür einsetzt, der weltweiten Gemeinschaft der klinischen Forschungszentren eine gemeinsame Stimme zu verleihen. Die SCRS vertritt mehr als 11.000 Forschungszentren weltweit und fördert die Zusammenarbeit und den Austausch in der Branche, die sich für standortbezogene Interessenvertretung, Bildung, Mentoring und Vernetzung einsetzen. Die SCRS ist ein aktiver und einflussreicher Fürsprecher für Standorte in Brancheninitiativen, um sicherzustellen, dass die Perspektive der Prüfzentren gehört und geschätzt wird. Erfahren Sie mehr und engagieren Sie sich unter myscrs.org. Unsere Stimme. Unsere Gemeinschaft. Ihr Erfolg.

Über Fortrea

Fortrea (Nasdaq: FTRE) ist ein weltweit führender Anbieter von Lösungen für die klinische Entwicklung in der Life–Science–Branche. Wir arbeiten mit aufstrebenden und großen biopharmazeutischen, biotechnologischen, medizintechnischen und diagnostischen Unternehmen zusammen, um Innovationen im Gesundheitswesen voranzutreiben, die das Angebot lebensverändernder Therapien für Patienten beschleunigen. Fortrea bietet Management von klinischen Studien der Phasen I–IV, klinische Pharmakologie und Beratungsdienste an. Die Lösungen von Fortrea basieren auf drei Jahrzehnten Erfahrung in mehr als 20 Therapiegebieten, einer Leidenschaft für wissenschaftliche Strenge, außergewöhnlichen Erkenntnissen und einem starken Netzwerk von Prüfzentren. Unser talentiertes und vielfältiges Team, das in etwa 100 Ländern tätig ist, ist darauf ausgelegt, Kunden weltweit fokussierte und flexible Lösungen zu bieten. Erfahren Sie mehr darüber, wie Fortrea von der Pipeline zum Patienten zu einer transformativen Kraft wird, unter Fortrea.com und folgen Sie uns auf LinkedIn und X (ehemals Twitter).

SCRS – Kontakte:

Marissa Hill (Medien) – 267–865–3296, [email protected]
Brian Egan (Medien) – 518–207–6965, [email protected]

Kontakt zu Fortrea:

Galen Wilson (Medien) – 703–298–0802, [email protected]
Kate Dillon (Medien) – 646–818–9115, [email protected]


GLOBENEWSWIRE (Distribution ID 9362056)

Society for Clinical Research Sites (SCRS) and Fortrea Partner to Advance Collaboration in Clinical Research

DURHAM, N.C., Feb. 20, 2025 (GLOBE NEWSWIRE) — The Society for Clinical Research Sites (SCRS) and Fortrea (Nasdaq: FTRE), a leading global contract research organization (CRO), are pleased to announce Fortrea’s sponsorship of the SCRS Collaborate Forward working group.

Comprising 16 leading Global Impact Partner organizations, the Collaborate Forward working group will explore and develop best practices to reduce administrative burdens across the clinical research ecosystem. The group is committed to fostering transparency and collaboration to tackle challenges faced by clinical research sites. By improving internal processes, it aims to make sites more sustainable and trials more efficient—ultimately leading to a smoother experience for patients.

Fortrea’s sponsorship marks a significant investment in fostering industry–wide innovation and reflects the company’s dedication to placing sites at the forefront of clinical trial planning.

“We are excited to partner with SCRS to launch and support this working group,” said Mike Clay, senior vice president of Global Project Delivery at Fortrea. “Clinical trials are becoming increasingly complex, and the industry faces mounting pressure to accelerate innovation for patients. We believe that collaboration with clinical research sites is key to unlocking efficiencies and productivity gains that will streamline the clinical trial process. This initiative will develop tangible solutions that clinical study sponsors, CROs, vendors, sites and patient advocacy groups can rally behind. As a leading CRO, we are proud to be at the forefront of this effort, ensuring that sites remain central to driving progress and fostering greater industry–wide collaboration to bring life–changing treatments to patients faster.”

“Clinical research requires a unique interdependency to generate the best outcomes. Collaborate Forward will share partnership successes that impact the people, process and technology improving clinical research today,” added Sean Soth, senior vice president, Strategy and Global Business Partnerships, SCRS. “We are pleased to welcome Fortrea as the charter sponsor of Collaborate Forward. This partnership underscores the value of cross–industry collaboration and the collective effort needed to drive meaningful progress in creating a more connected and efficient clinical trial ecosystem.”

Collaborate Forward will initially focus on study startup, showcasing the advantages of collaboration through compelling stories, case studies and data–driven insights. The group will convene regularly to exchange insights, assess industry trends and develop pragmatic tools that sponsors and CROs can implement within their organizations. Updates on the working group's progress will be shared throughout 2025 via SCRS Site Solutions Summits and publications, highlighting key findings and collaborative achievements.

SCRS invites sponsors and CROs committed to site sustainability to join this effort and contribute to shaping a more effective and synergistic clinical research landscape. For more information on how to participate, please contact Brian Egan.

About The Society for Clinical Research Sites

The Society for Clinical Research Sites (SCRS) is the leading advocacy organization dedicated to unifying the voice of the global clinical research site community. Representing more than 11,000 research sites globally, SCRS facilitates industry collaborations and conversations dedicated to site–focused advocacy, education, mentorship and connection. SCRS is an active and influential champion for sites in industry initiatives to ensure that the perspective of sites is heard and valued. Learn more and get involved at myscrs.org. Our voice. Our community. Your success. 

About Fortrea

Fortrea (Nasdaq: FTRE) is a leading global provider of clinical development solutions to the life sciences industry. We partner with emerging and large biopharmaceutical, biotechnology, medical device and diagnostic companies to drive healthcare innovation that accelerates life changing therapies to patients. Fortrea provides phase I–IV clinical trial management, clinical pharmacology and consulting services. Fortrea’s solutions leverage three decades of experience spanning more than 20 therapeutic areas, a passion for scientific rigor, exceptional insights and a strong investigator site network. Our talented and diverse team working in about 100 countries is scaled to deliver focused and agile solutions to customers globally. Learn more about how Fortrea is becoming a transformative force from pipeline to patient at Fortrea.com and follow us on LinkedIn and X (formerly Twitter).

SCRS Contacts:

Marissa Hill (Media) – 267–865–3296, [email protected]
Brian Egan (Media) – 518–207–6965, [email protected]

Fortrea Contacts:

Galen Wilson (Media) – 703–298–0802, [email protected]
Kate Dillon (Media) – 646–818–9115, [email protected]


GLOBENEWSWIRE (Distribution ID 9361361)

Zenas BioPharma Announces Key 2024 Accomplishments and 2025 Business Objectives to Support the Global Development and Commercialization of Therapies for Autoimmune Diseases

– Advancing Phase 2 and Phase 3 trials of obexelimab, a unique CD–19 x FcγRIIb inhibitor of B cell function–

–Topline results from Phase 2 Trial in Relapsing Multiple Sclerosis (MoonStone) expected in third quarter 2025–

–Topline results from pivotal Phase 3 Trial in Immunoglobulin G4–Related Disease (INDIGO) expected year–end 2025–

– Enrollment of Phase 2 Trial in Systemic Lupus Erythematosus (SunStone) expected to be completed in 2025–

– Out–licensed greater–China anti–IGF–1R Thyroid Eye Disease programs to Zai Lab –

WALTHAM, Mass., Feb. 05, 2025 (GLOBE NEWSWIRE) — Zenas BioPharma, Inc. (“Zenas” or the “Company”) (Nasdaq: ZBIO), a clinical–stage global biopharmaceutical company committed to being a leader in the development and commercialization of therapies for autoimmune diseases, today announced its 2024 accomplishments, outlined its key business objectives for 2025 and announced preliminary unaudited cash balance as of year–end 2024.

“Based upon the progress achieved across all of our corporate goals and objectives during 2024, we enter 2025 with an opportunity to achieve major value–driving milestones with the anticipated results from our ongoing obexelimab Phase 2 and Phase 3 clinical trials,” said Lonnie Moulder, Founder and Chief Executive Officer of Zenas. “We are extremely proud of the accomplishments of our dedicated team as we enter the year well–financed and able to focus on execution, and achievement of our key objectives for the year.”

The Company enters 2025 well–capitalized to deliver its key milestones with approximately $350 million in cash, cash equivalents, and short–term investments as of December 31, 2024, 1 which is expected to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2026.

2024 Accomplishments and Recent Achievements

During 2024, the Company achieved the following objectives and announced a recent business development transaction:

  • Completed enrollment of the Phase 3 INDIGO trial, a global Phase 3 registration–directed, randomized, double–blind placebo–controlled trial of obexelimab in patients with Immunoglobulin–G4 Related Disease (IgG4–RD), the largest clinical trial ever conducted in this patient population.
  • Initiated the Phase 2 MoonStone trial, a Phase 2, multicenter, randomized, double–blind, placebo–controlled trial, to evaluate the efficacy and safety of obexelimab in patients with Relapsing Multiple Sclerosis (RMS).
  • Initiated the Phase 2 SunStone trial, a Phase 2, multicenter, randomized, double–blind, placebo–controlled trial, to evaluate the efficacy and safety of obexelimab to reduce disease activity in patients with Systemic Lupus Erythematosus (SLE).
  • Provided initial data from the Phase 2 SApHiAre trial, a global, multicenter, open–label safety and dose confirmation run–in period (SRP) to evaluate the safety and activity of obexelimab in patients with warm Autoimmune Hemolytic Anemia (wAIHA). Obexelimab achieved clinical proof–of–mechanism by increasing hemoglobin levels and red blood cells, and decreasing LDH and total bilirubin levels. Obexelimab was well tolerated in the SRP.
  • Completed an upsized Series C and initial public offering, raising approximately $458.7 million in aggregate gross proceeds to fund its planned activities for obexelimab and the Company’s growth strategy.
  • Bolstered its leadership team with the appointments of Chief Commercial Officer, Orlando Oliveira, and Chief Legal Officer, Jeff Held.
  • Out–licensed ZB005, a human IgG4 monoclonal antibody designed to bind only to the active form of C1s, for which Zenas held the development and commercialization rights in China, Hong Kong, Macau and Taiwan (Greater China) through an exclusive license with Dianthus.
  • The Company recently out–licensed regional rights to its thyroid eye disease programs, including ZB001, an insulin–like growth factor–1 receptor (anti–IGF–1R) monoclonal antibody, to Zai Lab (Zai).  Zenas received an upfront fee and is eligible to receive milestone payments and royalties in the future, as consideration for an exclusive sublicense to Zai to develop and commercialize ZB001 and related programs in Greater China.

Anticipated 2025 Clinical Milestones for Obexelimab

Obexelimab is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. This unique mechanism of action and self–administered, subcutaneous once–weekly injection regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. Obexelimab has been evaluated in five completed clinical trials in a total of 198 patients who received obexelimab either as an intravenous infusion or as a subcutaneous injection. Obexelimab was well tolerated and demonstrated clinical activity across these five clinical trials, providing the Company an initial clinical proof of concept for obexelimab as a potent B cell inhibitor for the treatment of patients living with certain autoimmune diseases.

During 2025, the Company expects to achieve the following key clinical milestones:

  • Report the 12–week primary endpoint results in the third quarter of 2025 from the Phase 2 MoonStone trial in patients with RMS.

The role of B cells in the pathogenesis of multiple sclerosis including RMS has been demonstrated through the successful clinical development, approval and clinical use of anti–CD20 B cell targeting therapies of other companies, including OCREVUS® (ocrelizumab) and KESIMPTA® (ofatumumab), which selectively deplete CD20–expressing B cells. The Company believes obexelimab’s unique mechanism of action to potently inhibit but not deplete a broader B cell lineage than CD20, nonclinical data, and a subcutaneous injection regimen, supports its potential for the treatment of RMS.

  • Following an initial screening period, patients in the MoonStone trial are being randomized 2:1 to 250 mg of obexelimab or placebo administered as a subcutaneous injection every seven days for a 12–week treatment period.
  • The primary objective of this double–blinded portion of the trial will be to assess the change from baseline in the cumulative number of gadolinium (Gd) enhancing lesions identified on T1–weighted magnetic resonance imaging (MRI).
  • Upon completion of the 12–week period, patients will enter an open–label period where patients on placebo will receive obexelimab treatment for at least three months, and patients initially randomized to obexelimab will continue treatment.
  • Important secondary endpoints during this open–label period include using standardized assessments, novel 3D imaging and biomarkers, including serum neurofilament light chain (NfL), to evaluate the impact of obexelimab on disease progression.

More information on the Phase 2 MoonStone trial (NCT06564311) is available at clinicaltrials.gov

  • Report topline results year–end 2025 from the Phase 3 INDIGO trial in patients with IgG4–RD.

IgG4–RD is a chronic fibro–inflammatory disease that can affect virtually all organ systems, including the pancreas, biliary tract, salivary and lacrimal glands, lungs, and kidneys. Patients with IgG4–RD may present with a single organ involved but more frequently present with multiple organ involvement. As the disease progresses and patients experience new or worsening symptoms (i.e., flares), lesions develop in additional organs and the cellular inflammation characterizing early disease moves toward a more fibrotic stage, which can lead to major irreversible tissue damage and ultimately organ failure. We estimate that the currently diagnosed population of IgG4–RD patients in the U.S. is approximately 20,000, with comparable prevalence rates globally.

Despite the growing recognition of IgG4–RD and advances in the understanding of its pathophysiology, there are no approved therapies for the treatment of this disease and there remains high unmet medical need. The current standard of care is treatment with glucocorticoids (GCs). Although GCs are initially effective, treatment with GCs can often result in various complications and co–morbidities. Most patients can relapse within 12 months of discontinuing GC treatment, and maintenance therapy with GCs has not been shown to prevent recurrence of disease.

The pathogenesis of IgG4–RD suggests that B cell–targeted therapies may provide therapeutic benefit. Although not approved by any regulatory authorities to treat IgG4–RD, certain B cell depleting agents (e.g., rituximab) are occasionally administered to patients with IgG4–RD. However, B cell depleting agents are often associated with infections, including serious opportunistic infections, and can compromise a patient’s ability to mount a response to vaccinations.

The reported evidence for the role of B cells in the pathogenesis of IgG4–RD, the observed effects of B cell targeting agents in previous trials in IgG4–RD, the data from our Phase 2 IgG4–RD trials with obexelimab, and its unique, non–depleting mechanism and once–weekly, subcutaneous injection regimen support its development in patients with IgG4–RD.

  • INDIGO is the largest clinical trial conducted in patients living with IgG4–RD and is designed to evaluate the safety and efficacy of obexelimab in approximately 190 patients with active IgG4–RD and being conducted at approximately 100 sites in 20 countries.
  • Following an initial screening period, patients were randomized 1:1 to 250 mg of obexelimab or placebo administered as a subcutaneous injection every seven days for 52 weeks, followed by an opportunity for eligible patients to continue in an open–label extension period where all patients will receive treatment with obexelimab.
  • The primary efficacy endpoint of INDIGO is the time to first IgG4–RD flare, as determined per protocol by the investigator and the adjudication committee.
  • Secondary endpoints include annualized flare rate, the proportion of patients achieving complete remission, and use and quantity of rescue medication, including GCs.

More information on the Phase 3 INDIGO trial (NCT05662241) is available at clinicaltrials.gov

  • Complete enrollment in 2025 in the Phase 2 SunStone trial in patients with SLE and report topline results in the first half of 2026.

The crucial role of B cells in SLE pathogenesis is well recognized, from producing autoantibodies to abnormal regulation of immune responses. Moreover, SLE is an autoimmune disease characterized by B cell dysfunction, the production of autoantibodies toward cellular and nuclear components, and multiorgan damage caused by immune complex deposition and inflammation within affected tissues. Current treatments are limited in number and modestly effective. Obexelimab has demonstrated clinical activity in a prior Phase 2 double–blind, randomized trial demonstrating proof–of–concept in the overall trial population and increased response in patients who maintained higher systemic exposure to obexelimab, and also in biomarker–defined subpopulations. Coupled with the safety data obtained to date, we believe these data provide support for the development of obexelimab in patients with SLE.

  • Patients with active SLE determined at screening by the investigator and adjudication committee are randomized 1:1 to obexelimab 250 mg or placebo administered as a subcutaneous injection every seven days for 24 weeks.
  • The 250 mg once–weekly subcutaneous injection dose has been selected to maximize the potential for clinical activity as higher systemic exposure (Ctrough) correlated with greater clinical activity in the prior Phase 2 trial in SLE.
  • The primary endpoint is the percentage of responders, defined by BILAG–based Composite Lupus Assessment, with a reduction of SLE disease activity at week 24.
  • Biomarker analysis is planned to be conducted in all patients, including baseline RNA expression profiles to immunophenotype patients and evaluation of their differential responses to treatment.

More information on the Phase 2 SunStone trial (NCT06559163) is available at clinicaltrials.gov

About Zenas BioPharma, Inc.

Zenas is a clinical–stage global biopharmaceutical company committed to becoming a leader in the development and commercialization of transformative therapies for patients with autoimmune diseases. Our core business strategy combines our experienced leadership team with a disciplined product candidate acquisition approach to identify, acquire and develop product candidates globally that we believe can provide superior clinical benefits to patients living with autoimmune diseases. Zenas’ lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb, which are broadly present across B cell lineage, to inhibit the activity of cells that are implicated in many autoimmune diseases without depleting them. We believe that obexelimab’s unique mechanism of action and self–administered, subcutaneous injection regimen may broadly and effectively address the pathogenic role of B cell lineage in chronic autoimmune disease. For more information about Zenas BioPharma, please visit www.zenasbio.com and follow us on LinkedIn.

Forward looking statements

This press release contains “forward–looking statements” which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are forward–looking statements. In some cases, forward–looking statements can be identified by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward–looking statements contain these words. Forward–looking statements include, but are not limited to, statements concerning Zenas’s plans, objectives, expectations and intentions; the timing and results of ongoing and future clinical trials, including expectations on the timing of reporting INDIGO trial topline results, the 12–week primary endpoint data for the MoonStone trial and the anticipated timing of completing enrollment and reporting topline results for the SunStone trial; its growth strategy; the Company’s preliminary unaudited cash, cash equivalents and short–term investments as of December 31, 2024; and cash runway guidance. The forward–looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company’s actual results to differ materially from those anticipated in the forward–looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company’s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies, many of which already have approved therapies in the Company’s current indications; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third–party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers, many of which are located outside of the United States, including the Company’s current sole contract manufacturing organization for drug substance and drug product, WuXi Biologics (Hong Kong) Limited, which is located in China; and other risks and uncertainties described in the section “Risk Factors” in the Company’s Quarterly Report on Form 10–Q for the quarter ended September 30, 2024, as well as other information we file with the Securities and Exchange Commission. The forward–looking statements in this press release are inherently uncertain, speak only as of the date of this press release and may prove incorrect. These statements are based upon information available to the Company as of the date of this press release and while the Company believes such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that the Company has conducted an exhaustive inquiry into, or review of, all potentially available relevant information. Because forward–looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company’s control, these forward–looking statements should not be relied upon as guarantees of future events. The events and circumstances reflected in the forward–looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward–looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward–looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

The Zenas BioPharma word mark and logos are trademarks of Zenas BioPharma, Inc. or its affiliated companies.

Investor Contact:

Matthew Osborne

Investor Relations and Corporate Communications
[email protected]

Media Contact:

Argot Partners
[email protected]

1 This amount is preliminary and unaudited and is subject to completion of the Company’s financial closing procedures. As a result, this amount may differ materially from the amount that will be reflected in the Company’s consolidated financial statements for the year ended December 31, 2024.


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