Gain Therapeutics will present oral data on its lead candidate, GT‑02287, at the 3rd International GBA1 Meeting scheduled for May 22‑23 in Phoenix, Arizona. The presentation will detail the company’s latest Phase 1b results, including target engagement and biomarker reductions in patients with or without GBA1 mutations.
The Phase 1b study has shown that GT‑02287 engages its target, glucocerebrosidase, and reduces key biomarkers such as glucosylsphingosine and neurofilament light chain. Early clinical signals suggest a disease‑modifying effect, supporting the company’s hypothesis that the drug could shift Parkinson’s treatment from symptom relief to slowing disease progression.
Gain’s management will also attend the 7th World Parkinson’s Congress from May 24‑27, also in Phoenix, to share these findings and discuss the strategy for advancing GT‑02287 into a Phase 2 trial later in 2026. The company is preparing for a Phase 2 start in Q3 2026, pending FDA review.
"The data from our Phase 1b study furthers our hypothesis that GT‑02287 is among the first disease‑modifying therapies promising to shift the treatment paradigm in PD from symptom relief to halting or slowing symptom progression, targeting the causative biology of PD to enable a more durable and predictable treatment effect for those living with PD," said Gene Mack, President and CEO.
The announcement underscores Gain’s progress toward a Phase 2 launch and highlights the company’s proprietary Magellan™ platform and funding support from the Michael J. Fox Foundation and the Silverstein Foundation. Presenting at these conferences positions GT‑02287 as a potential disease‑modifying therapy for Parkinson’s disease and signals a key communication milestone for the company.
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