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BETHESDA, Md. - Gain Therapeutics, Inc. (NASDAQ:GANX), a $56.8 million market cap biotech company, announced Thursday it has begun a nine-month extension of its Phase 1b study for GT-02287, the company’s experimental Parkinson’s disease treatment. According to InvestingPro data, the company’s stock has experienced a challenging period, declining about 27% year-to-date, though it maintains a favorable cash position with more debt than cash on its balance sheet.
The extension allows participants to continue treatment beyond the initial 90-day dosing period, with more than half of the study’s 21 participants opting to continue. The original Phase 1b study is expected to complete in December 2025. Analysts following the company maintain an optimistic outlook, with price targets ranging from $5 to $12 per share, significantly above the current trading price of $1.58.
Early results from the study will be presented at the International Congress of Parkinson’s Disease and Movement Disorders in Honolulu on October 7. Additional data, including functional changes measured by the Movement Disorder Society Unified Parkinson’s Disease Rating Scale and biomarker information from cerebrospinal fluid and blood samples, will be available in the fourth quarter of 2025.
"Early observations related to safety, tolerability, pharmacokinetics, and functional impact are encouraging," said Gene Mack, President and Chief Executive Officer of Gain Therapeutics, in a press release statement.
GT-02287 is an orally administered small molecule designed to restore the function of glucocerebrosidase (GCase), an enzyme that becomes impaired due to GBA1 gene mutations or age-related factors. GBA1 mutations represent the most common genetic abnormality associated with Parkinson’s disease.
The drug candidate previously demonstrated favorable safety and tolerability in a Phase 1 study with healthy volunteers, showing increased GCase activity at clinically relevant doses.
The current Phase 1b trial is evaluating the safety and tolerability of GT-02287 in people with Parkinson’s disease with or without a GBA1 mutation across seven clinical sites in Australia.
The development program 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. While the company’s overall financial health score is categorized as ’WEAK’ by InvestingPro, which offers comprehensive analysis through its Pro Research Reports, the external funding support helps offset the significant cash burn rate typical of clinical-stage biotech companies. Investors can access detailed financial analysis and 8 additional ProTips for GANX through an InvestingPro subscription.
In other recent news, Gain Therapeutics has received approval to extend its Phase 1b clinical trial for the Parkinson’s disease therapy GT-02287 to 12 months, allowing participants who completed the initial 90-day period to continue treatment. The company has also announced that it reached its original enrollment target of 16 participants ahead of schedule, with enrollment remaining open for additional candidates. Analysts at H.C. Wainwright have reiterated a Buy rating on Gain Therapeutics with an $8 price target, while BTIG has adjusted its price target to $9 from $10, maintaining a Buy rating as well. The company plans to release cerebrospinal fluid data on key biomarkers in the fourth quarter, which may include GCase-deficient patients. In a move to raise funds, Gain Therapeutics announced a public offering of common stock and warrants, aiming to advance the development of GT-02287 and support other corporate purposes. The trial’s accelerated enrollment means that biomarker analysis will occur earlier than expected, with full results anticipated in the fourth quarter of 2025.
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