Gain Therapeutics reports progress in Parkinson's disease study

Published 10/04/2025, 12:06
Gain Therapeutics reports progress in Parkinson's disease study

BETHESDA, Md. - Gain Therapeutics, Inc. (NASDAQ:GANX), a biotechnology firm with a market capitalization of $45.85 million, has shared updates on its clinical-stage drug candidate GT-02287, showing potential as a disease-modifying treatment for Parkinson's disease (PD). The company, whose stock currently trades at $1.65, presented new preclinical evidence at the AD/PD™ 2025 International Conference held from April 1-5 in Vienna, Austria. According to InvestingPro data, analysts have set price targets ranging from $5 to $12, suggesting significant potential upside if the company's clinical trials succeed.

The oral presentation highlighted GT-02287's ability to alleviate motor deficits and prevent complex behavioral deficits in animal models of both GBA1 and idiopathic PD. These effects persisted after the withdrawal of the compound, suggesting long-term benefits. The poster detailed the design of the ongoing Phase 1b clinical study, which aims to assess the safety, tolerability, and pharmacokinetics of GT-02287 in people with PD. InvestingPro analysis reveals the company maintains a healthy current ratio of 2.97, indicating strong ability to fund its ongoing research, though it's worth noting the company is currently burning through cash rapidly.

Gain Therapeutics anticipates conducting an early biomarker analysis from the Phase 1b study in Q2 2025, with full analysis expected in Q4 2025. This will inform the planning of a Phase 2 study in the second half of the year.

Dr. Joanne Taylor, Senior Vice President of Research, expressed confidence in the compound's disease-modifying potential, supported by both biomarker and behavioral data. Dr. Jonas Hannestad, Chief Medical Officer, noted the strong enrollment for the Phase 1b study and the company's eagerness to see the translation of preclinical effects to PD patients.

GT-02287 has demonstrated favorable safety and tolerability in a Phase 1 study involving healthy volunteers. The ongoing Phase 1b trial is evaluating the drug's safety profile over three months of dosing in PD patients across seven Australian sites.

Gain Therapeutics' research in PD has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation for Parkinson's with GBA, and the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency.

The company's approach focuses on allosteric small molecule therapies, with GT-02287 targeting the restoration of glucocerebrosidase (GCase) function, a lysosomal enzyme impaired in PD. This press release statement provides the basis for the updates on GT-02287's development and the ongoing clinical trial. With the next earnings report scheduled for May 7, 2025, investors seeking deeper insights into Gain Therapeutics' financial health and growth prospects can access comprehensive analysis through InvestingPro, which offers exclusive access to over 30 additional financial metrics and expert insights not covered in this article.

In other recent news, Gain Therapeutics has regained compliance with Nasdaq's Market Value of Listed Securities requirement. The company had previously been notified of falling below the $50 million threshold necessary for continued listing on The Nasdaq Global Market. By January 14, 2025, Gain Therapeutics successfully raised its market value above this benchmark, securing its position on the exchange. Additionally, Scotiabank analysts have initiated coverage on Gain Therapeutics, assigning a Sector Outperform rating and setting a price target of $12.00. The analysts' optimism is centered around the potential of Gain Therapeutics' investigational drug, GT-02287, which is in Phase 1b trials for Parkinson's Disease. They consider the development of GT-02287 a high-risk/high-reward opportunity, with significant value implications expected from an interim analysis in 2025. These developments reflect ongoing interest and confidence in Gain Therapeutics' strategic direction and financial health.

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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