Gain Therapeutics Announces Formation of Clinical Advisory Board to Support Continued Advancement of Lead Drug Candidate GT-02287
Gain Therapeutics, a clinical-stage biotechnology company, announced the formation of a Clinical Advisory Board (CAB) to support the development of its lead drug candidate, GT-02287, for Parkinson's disease. The CAB includes experts in trial design, genetics, and biomarkers and will assist in progressing GT-02287 through potential Phase 2 and Phase 3 clinical trials.
CEO Gene Mack emphasized the strategic importance of this step, expressing confidence in the CAB's collective expertise to advance the treatment. Key members include:
- Karl Kieburtz, M.D., M.P.H., a neurology professor with extensive experience in clinical trials and advisory roles.
- Roy Alcalay, M.D., M.S., Chief of the Movement Disorders Division at Tel Aviv Sourasky Medical Center, focusing on biomarkers and genetics.
- Samuel Broder, M.D., former Director of the National Cancer Institute, known for his work in developing anti-cancer agents and HIV treatments.
- Wilma D.J. Van de Berg, Ph.D., a senior translational neuroscientist specializing in neuropathology and biomarker research.
Gain Therapeutics, una società di biotecnologie in fase clinica, ha annunciato la formazione di un Clinical Advisory Board (CAB) per supportare lo sviluppo del suo principale candidato farmaco, GT-02287, per il morbo di Parkinson. Il CAB include esperti in progettazione di studi clinici, genetica e biomarcatori, e assisterà nel portare avanti GT-02287 attraverso i potenziali trial clinici di Fase 2 e Fase 3.
Il CEO Gene Mack ha sottolineato l'importanza strategica di questo passo, esprimendo fiducia nell'expertise collettiva del CAB per far progredire il trattamento. I membri chiave includono:
- Karl Kieburtz, M.D., M.P.H., professore di neurologia con ampia esperienza in studi clinici e ruoli di consulenza.
- Roy Alcalay, M.D., M.S., Capo della Divisione dei Disturbi del Movimento presso il Tel Aviv Sourasky Medical Center, con focus su biomarcatori e genetica.
- Samuel Broder, M.D., ex Direttore del National Cancer Institute, noto per il suo lavoro nello sviluppo di agenti anti-cancro e trattamenti per l'HIV.
- Wilma D.J. Van de Berg, Ph.D., neuroscienziata traslazionale senior specializzata in neuropatologia e ricerca sui biomarcatori.
Gain Therapeutics, una empresa de biotecnología en etapa clínica, anunció la formación de un Clinical Advisory Board (CAB) para apoyar el desarrollo de su principal candidato a fármaco, GT-02287, para la enfermedad de Parkinson. El CAB incluye expertos en el diseño de ensayos, genética y biomarcadores y ayudará a avanzar en GT-02287 a través de los potenciales ensayos clínicos de Fase 2 y Fase 3.
El CEO Gene Mack enfatizó la importancia estratégica de este paso, expresando confianza en la experiencia colectiva del CAB para avanzar en el tratamiento. Los miembros clave incluyen:
- Karl Kieburtz, M.D., M.P.H., profesor de neurología con amplia experiencia en ensayos clínicos y roles de asesoría.
- Roy Alcalay, M.D., M.S., Jefe de la División de Trastornos del Movimiento en el Tel Aviv Sourasky Medical Center, enfocado en biomarcadores y genética.
- Samuel Broder, M.D., ex Director del National Cancer Institute, conocido por su trabajo en el desarrollo de agentes anti-cáncer y tratamientos para el VIH.
- Wilma D.J. Van de Berg, Ph.D., científica neurotraslacional senior especializada en neuropatología e investigación de biomarcadores.
Gain Therapeutics는 임상 단계의 생명공학 회사로, 파킨슨병을 위한 주요 약물 후보인 GT-02287의 개발을 지원하기 위해 임상 자문 위원회(CAB)를 구성했다고 발표했습니다. CAB는 임상 시험 설계, 유전학 및 바이오마커의 전문가들로 구성되어 있으며, GT-02287을 잠재적인 2상 및 3상 임상 시험으로 진전시키는 데 도움을 줄 것입니다.
CEO인 Gene Mack은 이 단계의 전략적인 중요성을 강조하면서 CAB의 집단적인 전문성이 치료를 발전시키는 데 기여할 것이라고 확신했습니다. 주요 멤버들은 다음과 같습니다:
- Karl Kieburtz, M.D., M.P.H.는 임상 시험 및 자문 역할에서 풍부한 경험을 가진 신경학 교수입니다.
- Roy Alcalay, M.D., M.S.는 Tel Aviv Sourasky Medical Center의 운동 장애 부서장으로 바이오마커 및 유전학에 중점을 두고 있습니다.
- Samuel Broder, M.D.는 전 국가 암 연구소의 이사로, 항암제 및 HIV 치료제 개발로 유명합니다.
- Wilma D.J. Van de Berg, Ph.D.는 신경병리학과 바이오마커 연구를 전문으로 하는 고위 번역 신경 과학자입니다.
Gain Therapeutics, une entreprise de biotechnologie en phase clinique, a annoncé la formation d'un Clinical Advisory Board (CAB) pour soutenir le développement de son principal candidat médicament, GT-02287, pour la maladie de Parkinson. Le CAB comprend des experts en conception d'essais cliniques, en génétique et en biomarqueurs et aidera à faire progresser GT-02287 à travers les essais cliniques potentiels de Phase 2 et Phase 3.
Le PDG Gene Mack a souligné l'importance stratégique de cette étape, exprimant sa confiance dans l'expertise collective du CAB pour faire avancer le traitement. Les membres clés incluent :
- Karl Kieburtz, M.D., M.P.H., professeur de neurologie avec une vaste expérience dans les essais cliniques et les rôles de conseil.
- Roy Alcalay, M.D., M.S., chef de la division des troubles du mouvement au Tel Aviv Sourasky Medical Center, se concentrant sur les biomarqueurs et la génétique.
- Samuel Broder, M.D., ancien directeur du National Cancer Institute, connu pour son travail sur le développement d'agents anticancéreux et de traitements du VIH.
- Wilma D.J. Van de Berg, Ph.D., scientifique senior en neurosciences translationnelles spécialisée dans la neuropathologie et la recherche sur les biomarqueurs.
Gain Therapeutics, ein biopharmazeutisches Unternehmen in der klinischen Phase, hat die Bildung eines Clinical Advisory Board (CAB) bekannt gegeben, um die Entwicklung seines führenden Arzneimittelkandidaten, GT-02287, zur Behandlung von Morbus Parkinson zu unterstützen. Das CAB besteht aus Experten für Studiendesign, Genetik und Biomarker und wird dabei helfen, GT-02287 durch die möglichen klinischen Studien der Phase 2 und Phase 3 voranzubringen.
CEO Gene Mack betonte die strategische Bedeutung dieses Schrittes und äußerte Vertrauen in die kollektive Expertise des CAB zur Förderung der Therapie. Zu den Schlüsselmitgliedern gehören:
- Karl Kieburtz, M.D., M.P.H., ein Neurologie-Professor mit umfassender Erfahrung in klinischen Studien und Beratungstätigkeiten.
- Roy Alcalay, M.D., M.S., Leiter der Abteilung für Bewegungsstörungen am Tel Aviv Sourasky Medical Center, der sich auf Biomarker und Genetik spezialisiert hat.
- Samuel Broder, M.D., ehemaliger Direktor des National Cancer Institute, bekannt für seine Arbeit an der Entwicklung von Antitumormitteln und HIV-Behandlungen.
- Wilma D.J. Van de Berg, Ph.D., eine leitende Übersetzungsneurowissenschaftlerin, die sich auf Neuropathologie und Biomarkerforschung spezialisiert hat.
- Formation of a Clinical Advisory Board with leading experts to support GT-02287 development.
- Strategic step forward for advancing GT-02287 into potential Phase 2 and Phase 3 clinical trials.
- No immediate financial metrics or clinical trial results provided.
BETHESDA, Md., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced the formation of its Clinical Advisory Board (CAB) to support the continued development of the Company’s lead drug candidate, GT-02287, in Parkinson’s disease. The CAB is composed of leading experts in Parkinson’s disease trial design, genetics, and biomarkers and will work closely with Gain leadership to progress GT-02287 through potential Phase 2 and Phase 3 clinical development.
“The formation of Gain’s new Clinical Advisory Board marks a significant strategic step forward for Gain, as GT-02287 advances into Parkinson’s disease patients,” stated Gene Mack, CEO of Gain Therapeutics. “We are confident that the collective expertise of this Clinical Advisory Board will be invaluable as we strive to deliver a meaningful new treatment option for Parkinson's disease patients.”
Gain Scientific Advisory Board Members
Karl Kieburtz, M.D., M.P.H. is a Professor of Neurology (part time) at the University of Rochester, was the founding Director of the Center of Health & Technology (CHET), and served as the Director of the Clinical and Translational Science Institute and Senior Associate Dean for Clinical Research at the University of Rochester. Dr. Kieburtz also served as the Chair of the Parkinson Study Group Executive Committee and chaired the FDA’s Peripheral and Central Nervous System Advisory Committee. He has been global Principal Investigator for more than 50 multi-center and multi-national NIH, industry, and Foundation sponsored clinical trials, including the large NIH-sponsored NET-PD study. He was elected as a Fellow in the American Association for the Advancement of Sciences in 2014. In 2008, he co-founded Clintrex Research Corporation, now a part of Tox Strategies, which provides scientific and regulatory advisory services to companies developing CNS therapies.
Roy Alcalay, M.D., M.S. is Chief of the Movement Disorders Division at Tel Aviv Sourasky Medical Center in Tel Aviv, Israel. He moved from Columbia University where he holds a part-time associate professorship. He obtained his medical degree from Tel Aviv University, his neurology training from the Harvard University residency program at Massachusetts General Hospital and Brigham and Women’s Hospital, and his training in movement disorders at Columbia University. He earned a Master’s degree in biostatistics (patient-oriented research track) from Columbia University. His research focuses on biomarkers and genetics in Parkinson’s disease. He is the principal investigator of the Parkinson’s Foundation-funded PD GENEration and a member of The Michael J. Fox Foundation-funded PPMI executive steering committee. His research is funded by the Michael J. Fox Foundation, the Parkinson’s Foundation, and the Silverstein Foundation for Parkinson’s with GBA.
Samuel Broder, M.D. is the former Director of the National Cancer Institute (NCI) where he oversaw the development of numerous anti-cancer therapeutic agents. Dr. Broder helped launch multiple large-scale clinical trials investigating the prevention, diagnosis, and treatment of cancer, and he inaugurated the highly successful Specialized Program of Research Excellence (SPORE). In the 1980s, his laboratory was responsible for developing the first 3 agents approved by FDA specifically to treat HIV (Retrovir® (AZT), Videx® (ddI), and HIVID® (ddC). He joined the Celera Corporation at its founding in 1998, as Chief Medical Officer and Executive VP for Medical Affairs and helped advance the human genome project. His most recent executive position was as SVP, Health Sector at Intrexon Corp. He is the author or co-author of over 340 scientific publications and an inventor on many patents. He has received numerous awards related to his research in cancer and AIDS. He was elected to the National Academy of Medicine in 1993. He graduated from the University of Michigan Medical School in Ann Arbor in 1970, completed an internship and residency in Internal Medicine at Stanford University, and completed subspecialty training in medical oncology at the National Cancer Institute in Bethesda, Maryland.
Wilma D.J. Van de Berg, Ph.D. is a senior associate professor, senior translational neuroscientist, and neuroanatomist at the Department of Anatomy and Neurosciences, Amsterdam UMC, Amsterdam. She is the chair of the research section of Clinical Neuroanatomy and Biobanking and a leader in the field of neuropathology, brain biobanking, and biomarker research in Parkinson’s disease. Her research focuses on cellular disease mechanisms underpinning clinical heterogeneity, alpha-synuclein aggregation, and disease progression in Parkinson’s and related neurodegenerative disorders. She leads various initiatives to identify novel biomarkers and therapeutic strategies aiming to slow down or halt disease progression in Parkinson’s disease, including the multicenter longitudinal cohort study ‘Profiling Parkinson’s’ (ProPARK). In 2021, she co-founded the Dutch Parkinson Scientists association (www.dutchparkinsonscientists.nl) to stimulate Parkinson’s research and provide a platform of Parkinson scientists to meet and exchange expertise in the Netherlands. She is the founder and director of the ‘Normal Aging Brain Collection Amsterdam’ (NABCA; www.nabca.eu) and is involved in several international collaborative networks, such as ‘PD-RISK’ (NRF)), Global Parkinson’s Genetics Consortium (GP2), NEUROCOV, COST ‘IMMUPARKNET’, EPND, and 4DPD-OMICS.
About GT-02287
Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.
Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggests that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease.
Gain’s lead program in Parkinson’s disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate, GT-02287 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma exposure in the projected therapeutic range, CNS exposure, and target engagement and modulation of GCase enzyme.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology.
Forward-Looking Statements
This release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287; and the potential therapeutic and clinical benefits of the Company’s product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company’s business in general, please refer to the Company’s Form 10-K for the year ended December 31, 2023 and Form 10-Q for the quarter ended September 30, 2024. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.
Investor Contacts:
Apaar Jammu and Chuck Padala
ajammu@gaintherapeutics.com
chuck@lifesciadvisors.com
Media Contacts:
Russo Partners
Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.com
elio.ambrosio@russopartnersllc.com
(760) 846-9256
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