Gain Therapeutics extends Phase 1b Parkinson’s drug trial to 12 months

Published 04/09/2025, 13:10
Gain Therapeutics extends Phase 1b Parkinson’s drug trial to 12 months

BETHESDA, Md. - Gain Therapeutics, Inc. (NASDAQ:GANX), a clinical-stage biotech company with a market capitalization of $66 million, received approval from Australian ethics committees to extend dosing in its ongoing Phase 1b clinical study of GT-02287, the company announced Thursday. While the company maintains a strong balance sheet with more cash than debt, InvestingPro data shows it’s rapidly burning through available funds.

The extension will allow participants who completed the initial 90-day treatment period to continue receiving the experimental Parkinson’s disease therapy for up to 12 months total. According to the company, most participants who have completed the initial treatment period have expressed interest in joining the extension.

Separately, an independent data monitoring committee found no safety concerns in its review of interim safety data and recommended continuing the study without modifications.

"The approval by Australian authorities of the dosing extension and the DMC’s positive recommendation represent important milestones in GT-02287’s clinical development program," said Gene Mack, President and CEO of Gain Therapeutics. The company’s stock has shown strong momentum, delivering an impressive 85% return over the past year. According to InvestingPro analysis, two analysts have recently revised their earnings expectations upward for the upcoming period, though profitability remains a challenge.

GT-02287 is an oral, brain-penetrant small molecule designed to treat Parkinson’s disease with or without GBA1 mutations. The drug aims to restore function of the lysosomal enzyme glucocerebrosidase, which becomes impaired in Parkinson’s disease.

The ongoing Phase 1b trial is evaluating the safety and tolerability of GT-02287 after three months of dosing in people with Parkinson’s disease. The extension will further assess long-term safety, tolerability, functional changes, and biomarker activity.

The extended study will collect additional data intended to support Phase 2 planning, according to the press release statement. Analyst price targets for GANX range from $5 to $12, reflecting optimistic expectations for the company’s development pipeline. For deeper insights into GANX’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively on InvestingPro, which covers over 1,400 US stocks with detailed analysis and actionable intelligence.

In other recent news, Gain Therapeutics has announced several key developments. The company reached its enrollment target ahead of schedule for the Phase 1b clinical study of GT-02287, its lead asset in Parkinson’s disease treatment, with 16 participants by the end of June. This accelerated timeline allows for biomarker analysis to occur in the fourth quarter of 2025, earlier than initially planned. Gain Therapeutics also plans to release cerebrospinal fluid data on key biomarkers in the fourth quarter of this year, as part of its ongoing clinical trials.

In addition, Gain Therapeutics announced an underwritten public offering of common stock and warrants to fund further development of GT-02287 and general corporate purposes. The company has increased its authorized common shares from 50 million to 100 million following a stockholder vote. Analyst firm H.C. Wainwright has reiterated its Buy rating with an $8 price target on Gain Therapeutics, while BTIG has lowered its price target to $9 due to the timing of biomarker data release. These developments mark significant progress in Gain Therapeutics’ clinical and corporate strategies.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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