Gain Therapeutics Announces Formation of Clinical Advisory Board to Support Continued Advancement of Lead Drug Candidate GT-02287

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:
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