Passage Bio Out-licenses Three Pediatric Gene Therapy Programs to GEMMA Biotherapeutics and Enters New Research Collaboration
Passage Bio (NASDAQ: PASG) has out-licensed three pediatric gene therapy programs to GEMMA Biotherapeutics, a new company co-founded by Dr. James M. Wilson. The deal includes exclusive, worldwide rights for PBGM01 (GM1 gangliosidosis), PBKR03 (Krabbe disease), and PBML04 (metachromatic leukodystrophy). Passage Bio will receive initial payments of $10 million, with potential for up to $124 million in additional milestones and future royalties.
The company also entered a new strategic research collaboration with GEMMA Biotherapeutics, focusing on CNS indications including Huntington's disease. This transaction is expected to extend Passage Bio's operating cash runway to the end of Q2 2026, allowing the company to focus on advancing its lead asset, PBFT02, in multiple adult neurodegenerative diseases.
Passage Bio (NASDAQ: PASG) ha ceduto in licenza tre programmi di terapia genica pediatrica a GEMMA Biotherapeutics, una nuova azienda co-fondata dal Dr. James M. Wilson. L'accordo include diritti esclusivi e mondiali per PBGM01 (gangliosidosi GM1), PBKR03 (malattia di Krabbe) e PBML04 (leucodistrofia metacromatica). Passage Bio riceverà un pagamento iniziale di 10 milioni di dollari, con la possibilità di fino a 124 milioni di dollari in pagamenti aggiuntivi e diritti futuri.
L'azienda ha anche avviato una nuova collaborazione strategica di ricerca con GEMMA Biotherapeutics, concentrandosi su indicazioni nel sistema nervoso centrale, inclusa la malattia di Huntington. Si prevede che questa transazione estenda la liquidità operativa di Passage Bio fino alla fine del secondo trimestre del 2026, consentendo all'azienda di concentrarsi sullo sviluppo del suo principale asset, PBFT02, in diverse malattie neurodegenerative dell'adulto.
Passage Bio (NASDAQ: PASG) ha relegado tres programas de terapia génica pediátrica a GEMMA Biotherapeutics, una nueva empresa cofundada por el Dr. James M. Wilson. El acuerdo incluye derechos exclusivos a nivel mundial para PBGM01 (gangliosidosis GM1), PBKR03 (enfermedad de Krabbe) y PBML04 (leucodistrofia metacromática). Passage Bio recibirá pagos iniciales de 10 millones de dólares, con posibilidad de hasta 124 millones de dólares en hitos adicionales y regalías futuras.
La empresa también ha iniciado una nueva colaboración de investigación estratégica con GEMMA Biotherapeutics, enfocándose en indicaciones del SNC, incluida la enfermedad de Huntington. Se espera que esta transacción extienda la liquidez operativa de Passage Bio hasta finales del segundo trimestre de 2026, lo que permitirá a la empresa concentrarse en avanzar su activo principal, PBFT02, en varias enfermedades neurodegenerativas en adultos.
Passage Bio (NASDAQ: PASG)가 세 가지 소아 유전자 치료 프로그램을 GEMMA Biotherapeutics에 라이선스를 부여했습니다. 이 새로운 회사는 James M. Wilson 박사가 공동 설립했습니다. 이 거래는 PBGM01 (GM1 신경세포성 비대형), PBKR03 (크라베병) 및 PBML04 (탈수초 경화증)에 대한 독점적인 전 세계적 권리를 포함합니다. Passage Bio는 1000만 달러의 초기 지급금을 받을 예정이며, 최대 1억 2400만 달러의 추가 이정표 및 미래 로열티 가능성이 있습니다.
이 회사는 또한 GEMMA Biotherapeutics와 함께 새로운 전략적 연구 협력을 시작했으며, 헌팅턴병을 포함한 CNS 적응증에 초점을 맞추고 있습니다. 이 거래는 Passage Bio의 운영 자금 수명을 2026년 2분기 말까지 연장할 것으로 예상되며, 이 회사는 여러 성인 신경퇴행성 질환에서 주요 자산인 PBFT02의 개발에 집중할 수 있습니다.
Passage Bio (NASDAQ: PASG) a octroyé des licences pour trois programmes de thérapie génique pédiatrique à GEMMA Biotherapeutics, une nouvelle société co-fondée par le Dr. James M. Wilson. L'accord comprend des droits exclusifs et mondiaux pour PBGM01 (gangliosidose GM1), PBKR03 (maladie de Krabbe) et PBML04 (leucodystrophie métachromatique). Passage Bio recevra des paiements initiaux de 10 millions de dollars, avec un potentiel de jusqu'à 124 millions de dollars en jalons supplémentaires et redevances futures.
L'entreprise a également lancé une nouvelle collaboration de recherche stratégique avec GEMMA Biotherapeutics, axée sur des indications dans le système nerveux central, y compris la maladie de Huntington. Cette transaction devrait étendre la durée de trésorerie opérationnelle de Passage Bio jusqu'à la fin du deuxième trimestre 2026, permettant ainsi à l'entreprise de se concentrer sur l'avancement de son actif phare, le PBFT02, dans plusieurs maladies neurodégénératives chez l'adulte.
Passage Bio (NASDAQ: PASG) hat drei Programme zur gentherapeutischen Behandlung von Kindern lizenziert an GEMMA Biotherapeutics, ein neues Unternehmen, das von Dr. James M. Wilson mitgegründet wurde. Der Vertrag umfasst die exklusiven weltweiten Rechte für PBGM01 (GM1-Gangliosidose), PBKR03 (Krabbe-Krankheit) und PBML04 (metachromatische Leukodystrophie). Passage Bio wird eine Anfangszahlung von 10 Millionen Dollar erhalten, mit der Möglichkeit von bis zu 124 Millionen Dollar in zusätzlichen Meilensteinen und zukünftigen Lizenzgebühren.
Das Unternehmen hat außerdem eine neue strategische Forschungskollaboration mit GEMMA Biotherapeutics begonnen, die sich auf ZNS-Indikationen wie die Huntington-Krankheit konzentriert. Es wird erwartet, dass diese Transaktion die Betriebsmittel von Passage Bio bis Ende Q2 2026 verlängert, was dem Unternehmen ermöglicht, sich auf die Weiterentwicklung seines Hauptprodukts PBFT02 bei mehreren neurodegenerativen Erkrankungen des Erwachsenen zu konzentrieren.
- Out-licensing deal potentially worth up to $134 million plus royalties
- Extended cash runway to Q2 2026
- New strategic research collaboration for CNS indications
- Ability to focus resources on lead asset PBFT02
- Discontinuation of preclinical research programs
- Termination of remaining options for future CNS research programs with University of Pennsylvania
Insights
The out-licensing deal between Passage Bio and GEMMA Biotherapeutics marks a significant strategic shift for Passage Bio, with substantial financial implications. The transaction includes
Crucially, this agreement is expected to extend Passage Bio's cash runway to the end of Q2 2026, a significant extension that provides the company with ample time to advance its lead asset, PBFT02 and explore additional indications. This extended runway is particularly valuable in the current economic climate, where biotech funding can be challenging to secure.
The strategic pivot to focus on adult neurodegenerative diseases while out-licensing pediatric programs allows Passage Bio to streamline its pipeline and potentially reduce R&D expenses. However, investors should note that this also means the company is now more dependent on the success of PBFT02.
The new research collaboration with GEMMABio, including options for four novel CNS indications, provides Passage Bio with additional shots on goal without immediate financial burden. This could be seen as a risk-mitigating strategy, diversifying the company's potential future pipeline.
Overall, while this deal reduces Passage Bio's direct exposure to the pediatric rare disease market, it significantly improves the company's financial position and focuses its resources on potentially more lucrative adult indications. Investors should monitor the progress of PBFT02 closely, as it now becomes even more critical to Passage Bio's future prospects.
This transaction represents a significant shift in Passage Bio's strategic focus, moving away from pediatric lysosomal storage diseases to concentrate on adult neurodegenerative conditions. The out-licensing of PBGM01, PBKR03 and PBML04 to GEMMA Biotherapeutics allows these promising therapies to continue development under a dedicated rare disease-focused entity, while Passage Bio redirects its resources to PBFT02 and other adult indications.
The collaboration with GEMMABio on Huntington's disease research is particularly intriguing. Huntington's is a challenging target that has seen several high-profile clinical failures in recent years. If Passage Bio can make progress in this area, it could significantly enhance its position in the neurodegenerative disease space.
The option to initiate research programs in four novel CNS indications provides Passage Bio with valuable pipeline expansion opportunities. This flexibility could be important in the rapidly evolving field of gene therapy, allowing the company to pivot towards the most promising areas as new data emerges.
However, the termination of the collaboration with the University of Pennsylvania's Gene Therapy Program marks the end of a significant partnership. While this aligns with the company's new focus, it may limit access to cutting-edge academic research in the future.
From a technical perspective, the transfer of clinical trial materials and product supply to GEMMABio suggests that the out-licensed programs were at a relatively advanced stage. This could potentially accelerate GEMMABio's development timeline, benefiting Passage Bio through milestone payments and royalties if the programs succeed.
Overall, this strategic realignment positions Passage Bio as a more focused player in the adult neurodegenerative disease space, with a streamlined pipeline and extended cash runway. The success of this strategy will largely depend on the clinical progress of PBFT02 and the company's ability to leverage its new collaboration with GEMMABio effectively.
Passage Bio's strategic pivot reflects broader trends in the gene therapy and rare disease markets. The decision to out-license pediatric programs while focusing on adult neurodegenerative diseases aligns with market dynamics that favor larger patient populations and potentially more lucrative commercial opportunities.
The rare disease market, particularly for pediatric conditions, is characterized by small patient populations and complex reimbursement landscapes. By shifting focus to adult neurodegenerative diseases, Passage Bio is targeting conditions with larger patient pools and potentially easier market access. This could lead to more attractive commercial prospects if their therapies prove successful.
The formation of GEMMA Biotherapeutics, co-founded by Dr. James M. Wilson, a pioneer in gene therapy, is noteworthy. This new entity's focus on rare pediatric diseases could potentially fill a gap in the market, as larger companies often shy away from these ultra-rare indications. The involvement of Dr. Wilson lends significant credibility to GEMMABio and may attract investor interest.
The research collaboration on Huntington's disease is particularly interesting from a market perspective. Despite several clinical setbacks in recent years, Huntington's remains a high-value target due to the lack of effective treatments and the devastating nature of the disease. Success in this area could position Passage Bio as a leader in a potentially lucrative market segment.
The option to explore four novel CNS indications provides Passage Bio with flexibility to adapt to evolving market trends. This could be important in the fast-moving field of gene therapy, where new discoveries can quickly shift the competitive landscape.
Overall, this strategic realignment positions Passage Bio to potentially capture larger market opportunities while maintaining exposure to the rare disease space through its partnership with GEMMABio. The success of this strategy will depend on the clinical progress of PBFT02 and the company's ability to effectively leverage its new collaborations and research options.
Granted exclusive, worldwide rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy to GEMMA Biotherapeutics, a new company co-founded by Dr. James M. Wilson
Entered new strategic research and collaboration agreement with GEMMA Biotherapeutics to advance genetic medicines for CNS indications, including Huntington’s disease
Transaction expected to extend operating cash runway to the end of Q2 2026
PHILADELPHIA, Aug. 01, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced that it has entered into a series of agreements to out-license its three clinical-stage pediatric lysosomal storage disease programs to GEMMA Biotherapeutics, Inc. (GEMMABio), a newly formed company co-founded by James M. Wilson, M.D., Ph.D. Through the transaction, the company granted GEMMABio exclusive, worldwide rights for the development and commercialization of PBGM01 for the treatment of GM1 gangliosidosis, PBKR03 for the treatment of Krabbe disease, and PBML04 for the treatment of metachromatic leukodystrophy.
Under the terms of the out-license agreements, the company will receive initial payments of
"We are excited to have out-licensed our pediatric lysosomal storage disease programs to a company deeply committed to advancing these promising therapies for underserved patient communities,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “This transaction is a key initiative in fulfilling our strategy to focus on advancing our lead asset, PBFT02, in multiple adult neurodegenerative diseases while continuing our preclinical research in high potential CNS indications. We have further extended our cash runway, providing us critical time and resources to continue to generate meaningful clinical data for PBFT02 and explore its potential in additional patient populations.”
Additionally, the company entered a new strategic research, collaboration, and license agreement with GEMMA Biotherapeutics. Under this agreement, GEMMABio will undertake specific preclinical and IND-enabling activities for the company’s research program including for Huntington’s disease, which was previously managed by the University of Pennsylvania’s Gene Therapy Program. Furthermore, GEMMABio will provide Passage Bio with options to initiate new research programs in four novel central nervous system (CNS) indications.
"By establishing GEMMA Biotherapeutics, we are able to further development of promising genetic medicines for rare patient populations, and we are thrilled to advance the three therapies from Passage Bio, each of which targets a devastating pediatric disease,” said James M. Wilson, M.D., Ph.D. “We also look forward to continuing our research and collaboration efforts with Passage Bio, which will leverage our combined expertise to explore new frontiers in CNS research. Together, we are poised to make a profound impact on the lives of patients around the world.”
In connection with the agreements with GEMMABio, Passage Bio amended its strategic collaboration with the University of Pennsylvania’s Gene Therapy Program to discontinue its preclinical research programs, terminate its remaining options to future CNS research programs, and terminate its funding of the discovery research program, effective July 31, 2024.
Overall, the net impact of the transaction is expected to extend the company’s operating cash runway to the end of Q2 2026.
About Passage Bio
Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression.
To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: www.passagebio.com.
About GEMMA Biotherapeutics
GEMMA Biotherapeutics (“GEMMABio”), a new therapeutics company, will serve as a research and innovation company to speed the research of and global access to life-changing advanced therapies for those living with rare diseases.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including the receipt of future payments under the GEMMABio agreements; our expectations about GEMMABio’s ability to execute key initiatives; our expectations about cash runway; and the ability of our product candidates to treat their respective target CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
For further information, please contact:
Passage Investors:
Stuart Henderson
Passage Bio
267.866.0114
shenderson@passagebio.com
Passage Bio Media:
Mike Beyer
Sam Brown Inc. Healthcare Communications
312.961.2502
mikebeyer@sambrown.com
GEMMA Biotherapeutics:
Kristen Love
klove@briancom.com
FAQ
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