Gain Therapeutics Presents Phase 1 GT-02287 Data at International Congress of Parkinson’s Disease and Movement Disorders Demonstrating Increase in GCase Activity in Healthy Volunteers
Company to Host Webinar to Discuss Results at 8:30 AM ET Today
BETHESDA, Md., Sept. 30, 2024 (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 presentation of a late-breaking abstract at the International Congress of Parkinson’s Disease and Movement Disorders. The poster and oral platform presentation summarize the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of single- and multiple-ascending doses of GT-02287, the Company’s clinical stage lead drug candidate, in a Phase 1 first-in-human study in healthy volunteers. The International Congress of Parkinson’s Disease and Movement Disorders? is being held September 27 - October 1 in Philadelphia, PA.
“The fact that we saw an increase in GCase activity in healthy volunteers, who we assume have homeostatic mechanisms to counter-regulate a pharmacologically-induced increase in GCase enzymatic activity, indicates that it will have more pronounced effect on GCase in people with Parkinson’s disease (PD) and further confirms our conviction that GT-02287 may be able to slow or stop the progression of PD. We hope that one day we can deliver this drug to those that need it and help them improve their everyday quality of life,” commented Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain.
Gene Mack, interim Chief Executive Officer and current Chief Financial Officer of Gain, continued, “We remain enthusiastic about the promising profile of GT-02287 and look forward to initiating a trial in people with PD by the end of Q4 2024, with the goal of demonstrating safety and tolerability in patients with PD and obtaining proof of mechanism based on relevant biomarkers.”
The late-breaker, titled, “The novel glucocerebrosidase chaperone GT-02287 in development for GBA-PD is safe and well tolerated in healthy volunteers at oral doses that produce plasma exposures in the projected therapeutic range,” was presented on-site by Jonas Hannestad and included results from the Phase 1 study of GT-02287. The single and multiple dose levels tested were safe and generally well tolerated, with no serious adverse events or Grade 3 (severe) adverse events observed, and no other safety signals detected. The PK profile of GT-02287 was linear across the tested dose ranges, and plasma exposures at daily doses of 7.7 mg/kg and above were within the projected therapeutic range. GT-02287 was measurable in cerebrospinal fluid (CSF) at levels in line with rodent levels at effective doses, demonstrating CNS exposure. Notably, GCase activity in dried blood spots increased approximately 30% in subjects who received GT-02287 but not in those who received placebo, demonstrating target engagement and modulation of GCase enzyme. GCase activity continued to increase 12 hours post-dose at 14 days, the furthest time point analyzed in the study. These results support the continued development of GT-02287 and its potential as a disease-modify treatment for Parkinson’s disease.
A PDF of the poster presented at the International Congress of Parkinson’s Disease and Movement Disorders is available on the Science and Technology section of the Company’s website at https://gaintherapeutics.com/science-and-technology/posters.
Webinar Details
Gain Therapeutics will host a webinar on Monday, September 30 at 8:30 AM ET to review results presented at the International Congress of Parkinson’s Disease and Movement Disorders. To register, please click here: https://lifescievents.com/event/gaintherapeutics/
About GT-02287 Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease 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 mouse models of GBA1-PD, including that presented at FENS Forum 2024 in June describing improvement in cognitive performance in addition to motor performance after administration of GT-02287, suggests that GT-02287 may have the potential to slow 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 for the treatment of Parkinson’s disease with or without a GBA1 mutation, is currently being evaluated in a Phase 1 clinical trial.
Leveraging AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models, the company’s Magellan? drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Its AI and machine-learning tools and virtual screening capabilities leverage the emerging on-demand compound libraries covering vast chemical spaces of over five trillion compounds to identify and select suitable small molecule hits for experimental validation.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced 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.
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