Gain Therapeutics presents Phase 1 GT-02287 data

Gain Therapeutics 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, 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 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. 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 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 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.