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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)

Dr. Falk Pharma and Allianthera (Suzhou) Biopharmaceuticals Forge Strategic Partnership for Novel AhR Agonist Development

Freiburg (Germany), Suzhou (China) and Boston (USA), July 30, 2025: 

Dr. Falk Pharma GmbH and Allianthera (Suzhou) Biopharmaceuticals Co., Ltd. Announce Strategic Partnership to Develop a Novel AhR Agonist for the Treatment of Moderate to Severe Ulcerative Colitis 

Dr. Falk Pharma GmbH, a research–based pharmaceutical company specializing in digestive and metabolic medicine, Allianthera (Suzhou) Biopharmaceuticals Co., Ltd., and its affiliate Allianthera Boston, Inc., a clinical stage biotechnology company focusing on novel drug research and development in immunology and inflammatory diseases, (Allianthera), are pleased to announce the signing of an agreement on the co–development, license option, manufacturing, and commercialization of the novel small molecule ATB102, an aryl hydrocarbon receptor (AhR) agonist currently undergoing a phase 1 clinical trial in the United States.

Under the terms of the newly signed agreement, Dr. Falk Pharma and Allianthera will collaborate to develop ATB102 for inflammatory bowel disease (IBD), with an initial focus on refractory moderate–to–severe ulcerative colitis (UC). Dr. Falk Pharma will enjoy the exclusive rights to license, manufacture, and commercialize ATB102 worldwide, excluding Mainland China, Hong Kong, Macau and Taiwan. As part of the agreement, Allianthera will receive a signing fee, significant development milestone payments as well as a licensing fee, followed by sales milestone payments and tiered royalties.

ATB102, developed by Allianthera, is a gut–enriched AhR agonist which represents a new therapeutic approach for IBD. It is designed to specifically target inflammation and mucosal damage within the gastrointestinal tract, with a particular emphasis on treating refractory moderate–to–severe UC. In pre–clinical research, ATB102 supports immune homeostasis, restores mucosal barrier integrity, and confers anti–fibrotic and anti–oxidative benefits, making it a potential new option for patients unresponsive to or relapsed from existing therapies.

Dr. Falk Pharma, in cooperation with its fully–owned subsidiary Losan Pharma GmbH, a leading contract development and manufacturing organisation (CDMO) partner and formulation expert, will develop an innovative colonic–release formulation of ATB102 to complement the current immediate release formulation.

“The aryl hydrocarbon receptor represents a truly novel mechanism of action to address the significant unmet needs in inflammatory bowel disease – especially in ulcerative colitis. The ATB team has discovered what we believe to be the best–in–class AhR agonist optimized for safety, gut enrichment, and therapeutic impact. We are delighted to join forces with Dr. Falk Pharma, who has deep expertise in formulation and clinical development. By combining our complementary strengths, we are confident that we will accelerate this program and deliver a much–needed, high–quality therapy to patients faster than ever before,” said Yuanhua Ding, CEO of Allianthera.

Kai Pinkernell, M.D., Managing Director, Science & Innovation at Dr. Falk Pharma, added, “We are excited about the addition of ATB102 to our R&D pipeline and to further exploring the potential of this novel molecule in future clinical trials together with Allianthera. The partnership with Allianthera reflects the strength of scientific excellence, mutual trust and a shared vision to better the lives of patients suffering from IBD. This collaboration not only reflects our growing global development and commitment to innovation in areas of high unmet medical need, it will also enhance Dr. Falk Pharma´s portfolio to treat digestive diseases and conditions.”


About Ulcerative Colitis
Ulcerative colitis is one of the disorders known as inflammatory bowel disease (IBD). Other disorders within this group of conditions include Crohn’s disease and two forms of microscopic colitis, called lymphocytic and collagenous colitis.

Ulcerative colitis is confined to the colon and typically begins in the rectum, spreading continuously throughout the rest of the large intestine. The exact cause of ulcerative colitis is still unclear; however immune system dysfunction, genetic factors, gut microbiome imbalance and dysregulation of the intestinal mucosal integrity as well as environmental factors are currently being investigated as potential contributors.

For more information, visit Dr. Falk Pharma’s website: https://drfalkpharma.com/en/indications/ulcerative–colitis/


About Allianthera 
Allianthera was established in November 2020 with a research and development (R&D) center in Suzhou, China, and a subsidiary in Boston, USA. Allianthera strives to become a global leader in therapeutic innovation by combining their deep disease biology knowledge and industry–leading R&D expertise with partners’ technologies while leveraging unique strengths in different geographies, East or West. The company is building a strong portfolio through internal R&D programs and external partnerships with an initial focus on diseases with unmet medical needs in immunology and inflammatory diseases.

Further information on Allianthera can be found on LinkedIn: https://www.linkedin.com/company/allianthera–suzhou–biopharmaceutical–co–ltd


About partnering with Dr. Falk Pharma
Dr. Falk Pharma engages in a variety of collaboration and partnership models to develop and deliver innovative treatment concepts with great potential. These treatments can be based on any type of pharmacological approach, from small molecules to biologics to novel drug delivery technologies. The company is involved in partnership projects across the range of phases and partners, from all stages of pre–clinical/clinical development and marketing as well as with academic researchers, start–ups, and established companies.

Dr. Falk Pharma is an industry leader in innovative pharmaceutical formulations that deliver active substances to specific functional segments of the gastrointestinal tract. Many of their products have attained standard–of–care status. The company enjoys strong, long–standing collaborations with renowned clinical centres and with academic and clinical key opinion leaders in the fields of gastroenterology, hepatology and metabolic diseases.

For more information go to https://drfalkpharma.com/en/partnering/


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 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 1001120434)

Entera Bio Receives FDA Agreement on BMD as Primary Endpoint for EB613 Registrational, Phase 3 Study in Post-Menopausal Women with Osteoporosis

JERUSALEM, July 28, 2025 (GLOBE NEWSWIRE) — Entera Bio Ltd. (NASDAQ: ENTX), a leader in the development of oral peptides and protein replacement therapies, announced today that in a written response to a Type A meeting request, the U.S. Food and Drug Administration (FDA) agreed with the Company’s proposal that the NDA marketing application filing for EB613 would be supported by a single multinational, randomized, double–blind, placebo–controlled, 24 month phase 3 study in women with postmenopausal osteoporosis, where change in total hip BMD is evaluated as the primary endpoint, and incidence of new or worsening vertebral fractures is evaluated as the key secondary endpoint. This marks a shift from precedent placebo–controlled phase 3 studies of new osteoporosis drugs which required incidence of fracture as the primary endpoint.

“This regulatory update is a major milestone for Entera and the entire osteoporosis community,” said Miranda Toledano, CEO of Entera. “Our alignment with the FDA reflects the strength of our data and collaborative discussions. Importantly, it allows us to advance our clinical development program without having to wait for FDA’s qualification of the Study to Advance Bone Mineral Density as a Regulatory Endpoint (SABRE), which is still expected this year. We thank the FDA and the Review Team at the Division of Endocrinology for their constructive approach. We also thank the SABRE team for paving the path to innovation for osteoporosis treatment,” said Toledano.

“Osteoporosis afflicts more women than heart attack, stroke and breast cancer combined. Over 200 million women globally are estimated to have osteoporosis and remain vastly undertreated, despite efficacious injectable anabolic (bone forming) treatments. One in two women over the age of 50 will suffer a fracture due to osteoporosis. No new drug for osteoporosis has been approved by FDA since 2019; and innovation has stalled for close to a decade due to the size, duration, cost and ethical constraints associated with fracture endpoint studies. In a silent disease, patient and clinician access to novel and alternative forms of validated mechanisms of action is important. We are developing EB613 as the first oral, once–daily anabolic tablet treatment to potentially serve this unmet medical need. EB613 is intended to increase skeletal mass, improve bone microarchitecture and reduce the risk of fracture,” said Miranda Toledano, CEO of Entera.

About EB613

Substantial evidence supports the efficacy of anabolic therapies over bisphosphonates for lowering fracture risk in osteoporosis patients at high risk. However, all available anabolic therapies are administered by subcutaneous (SC) injection and used in a minority of eligible patients. EB613 (oral PTH (1–34), teriparatide), is being developed as the first oral, once–daily anabolic tablet treatment for osteoporosis. EB613 completed a phase 2, 6–month, 161–patient, placebo–controlled study that met all biomarker and BMD endpoints without significant safety concerns in women with postmenopausal osteoporosis or low BMD (JBMR 2024). EB613 produced rapid dose–proportional increases in biochemical markers of bone formation, reductions in markers of bone resorption, and increased lumbar spine, total hip, and femoral neck BMD. The effects of EB613 on trabecular and cortical bone using 3D–DXA showed increases with EB613 compared with placebo in a variety of indices, including integral volumetric BMD and trabecular volumetric BMD, cortical thickness, and cortical surface BMD. Mechanistically, the findings suggest that bone strengthening, and fracture resistance may occur rapidly with EB613. Furthermore, the data are consistent with those of published subcutaneous teriparatide at the 6–month time point. Further abstracts have been submitted to ASBMR and NAMS 2025 conferences.

About Entera Bio

Entera is a clinical stage company focused on developing oral peptide and protein replacement therapies for significant unmet medical needs where an oral tablet form holds the potential to transform the standard of care. The Company leverages on a disruptive and proprietary technology platform (N–Tab™) and its pipeline of first–in–class oral peptide programs targeting PTH(1–34), GLP–1 and GLP–2. The Company’s most advanced product candidate, EB613 (oral PTH(1–34), teriparatide), is being developed as the first oral, osteoanabolic (bone building) once–daily tablet treatment for post–menopausal women with low BMD and high–risk osteoporosis. A placebo–controlled, dose–ranging Phase 2 study of EB613 tablets (n= 161) met primary (PD/bone turnover biomarker) and secondary endpoints (BMD). The EB612 program is being developed as the first oral PTH(1–34) tablet peptide replacement therapy for hypoparathyroidism. Entera is also developing the first oral oxyntomodulin, a dual targeted GLP1/glucagon peptide, in tablet form for the treatment of obesity and metabolic syndromes; and first oral GLP–2 peptide as an injection–free alternative for patients suffering from rare malabsorption conditions such as short bowel syndrome in collaboration with OPKO Health. For more information on Entera Bio, visit www.enterabio.com or follow us on LinkedIn, Twitter, Facebook, Instagram.

About SABRE

The Study to Advance BMD as a Regulatory Endpoint (SABRE) initiative, which started as a public private partnership sponsored by the FNIH in 2013, has amassed the strongest evidence to date that treatment–related gains in Bone Mineral Density (BMD) reliably and quantitatively predict fracture–risk reduction. In November 2023, the SABRE team submitted a full qualification package to FDA’s Biomarker Division as part of the Drug Development Tool Biomarker Qualification Pathway to potentially qualify BMD as a surrogate endpoint to fracture; in March 2024, the FDA Biomarker Division indicated to the SABRE project team that a decision would be issued within 10 months. The single most important predictor of osteoporotic fractures in postmenopausal women without a previous fracture is BMD. Treatment guidelines in the U.S. strongly recommend pharmacologic therapy for patients with a BMD T–score of –2.5 or lower in the spine, femoral neck, total hip. SABRE final FQP meta–analysis included data from 22 randomized, placebo–controlled trials (63,000 participants across seven drug classes) and showed that treatment–related gains in total–hip BMD explain 72% of the observed fracture–risk reduction. The R2 for this correlation was 0.73—double the correlation between blood pressure and stroke (R² = 0.37), which is the well accepted basis for the value of antihypertensive therapy.

Cautionary Statement Regarding Forward Looking Statements

Various statements in this presentation are “forward–looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements (other than statements of historical facts) in this presentation regarding our prospects, plans, financial position, business strategy and expected financial and operational results may constitute forward–looking statements. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward–looking statements. Forward–looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Forward–looking statements should not be read as a guarantee of future performance or results and may not be accurate indications of when such performance or results will be achieved.

Important factors that could cause actual results to differ materially from those reflected in Entera’s forward–looking statements include, among others: changes in the interpretation of clinical data; results of our clinical trials; the FDA’s interpretation and review of our results from and analysis of our clinical trials; unexpected changes in our ongoing and planned preclinical development and clinical trials, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for our product candidates; the potential disruption and delay of manufacturing supply chains; loss of available workforce resources, either by Entera or its collaboration and laboratory partners; impacts to research and development or clinical activities that Entera may be contractually obligated to provide; overall regulatory timelines; the size and growth of the potential markets for our product candidates; the scope, progress and costs of developing Entera’s product candidates; Entera’s reliance on third parties to conduct its clinical trials; Entera’s ability to establish and maintain development and commercialization collaborations; Entera’s operation as a development stage company with limited operating history; Entera’s competitive position with respect to other products on the market or in development for the treatment of osteoporosis, hypoparathyroidism, short bowel syndrome, obesity, metabolic conditions and other disease categories it pursues; Entera’s ability to continue as a going concern absent access to sources of liquidity; Entera’s ability to obtain and maintain regulatory approval for any of its product candidates; Entera’s ability to comply with Nasdaq’s minimum listing standards and other matters related to compliance with the requirements of being a public company in the United States; Entera’s intellectual property position and its ability to protect its intellectual property; and other factors that are described in the “Cautionary Statement Regarding Forward–Looking Statements,” “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Entera’s most recent Annual Report on Form 10–K filed with the SEC, as well as Entera’s subsequently filed Quarterly Reports on Form 10–Q and Current Reports on Form 8–K. There can be no assurance that the actual results or developments anticipated by Entera will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Entera. Therefore, no assurance can be given that the outcomes stated or implied in such forward–looking statements and estimates will be achieved. Entera cautions investors not to rely on the forward–looking statements Entera makes in this presentation. The information in this presentation is provided only as of the date of this presentation, and Entera undertakes no obligation to update or revise publicly any forward–looking statements, whether as a result of new information, future events or otherwise, except to the extent required by law.


GLOBENEWSWIRE (Distribution ID 9501141)

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 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)

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)

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)