BETHESDA -
No discontinuations or serious adverse events were reported. After database lock, topline data from this Phase 1, randomized, double-blind, placebo-controlled study is expected to be released in August, and full safety analysis and plasma pharmacokinetics are expected to be presented at a future congress.
'The successful completion of the SAD/MAD in 72 healthy subjects with no serious adverse events or discontinuations related to adverse events and the ability to achieve therapeutic plasma levels with oral dosing further confirm our belief in GT-02287's potential to be a transformative therapy for Parkinson's disease patients,' commented Gain's Executive Chairman,
The MAD part of the Phase 1 study was initiated in February, and all four MAD cohorts have completed daily oral dosing for 14 days. GT-02287 was well tolerated up to and including the highest planned dose level, and no safety signals have been detected in the 32 healthy volunteers who participated in the MAD part. Of the adverse events that occurred, 90% were mild and 10% were moderate with no Grade 3 or higher events reported. The favorable safety and tolerability profile and the appropriate range of plasma exposures achieved after oral administration further bolster GT-02287's best-in-class potential.
The primary objective of this Phase 1 clinical trial with single- and multiple ascending doses was to evaluate the safety and tolerability of GT-02287 administered orally once daily in healthy adults. The secondary objective was to evaluate the pharmacokinetics of SAD and MAD dose levels to identify recommended doses for further clinical development in people with Parkinson's disease. The SAD part of the Phase 1 clinical trial concluded in April with positive results and no serious adverse events. The SAD part of the Phase 1 clinical trial enrolled 40 healthy participants across five separate cohorts - all of which were completed at the planned dose levels with no premature discontinuations or safety signals.
GT-02287 has been shown to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and dysfunctional due to GBA1 gene mutations, the most common genetic risk factor for the development of Parkinson's disease. Compelling preclinical data in mouse models of GBA1-PD, including that presented at
About GT-02287
Gain's lead program in Parkinson's disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson's
About
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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