BETHESDA, Md. – Gain Therapeutics, Inc. (NASDAQ:GANX), a $69 million market cap biotech company currently trading at $1.92 per share, announced Monday that its lead drug candidate GT-02287 showed encouraging early results in a Phase 1b clinical study for Parkinson’s disease patients. According to InvestingPro data, the company maintains a strong balance sheet with more cash than debt, though it’s currently experiencing rapid cash burn typical of early-stage biotechs.
The company presented interim data at the International Congress of Parkinson’s Disease and Movement Disorders revealing improvements in MDS-UPDRS scores, a standard measure of Parkinson’s disease severity, among the first nine participants after 90 days of treatment. This development has caught analysts’ attention, with InvestingPro showing that two analysts have recently revised their earnings estimates upward, with price targets ranging from $5 to $12.
According to the company, GT-02287 was generally well-tolerated with no treatment-emergent serious adverse events reported. Some participants experienced transient increases in liver enzymes that normalized despite continued treatment.
“The stability and trending improvements we have observed in MDS-UPDRS scores was not something we were expecting to see with 90 days of dosing,” said Gene Mack, President and Chief Executive Officer of Gain Therapeutics, in the press release.
The study included 21 participants with varying treatment backgrounds. Mean MDS-UPDRS scores at baseline were 5.8, 7.4, and 24.7 for Parts I, II, and III, respectively. Several participants showed improvement in Parts II and III scores after 90 days of treatment, while Part I scores remained stable.
Plasma concentrations of the drug were within projected therapeutic ranges and comparable to those observed in the company’s earlier Phase 1 study with healthy volunteers.
The data monitoring committee has twice recommended continuation of the study, and Australian health authorities have approved an extension allowing patients to be treated for up to 12 months.
GT-02287 is an allosteric enzyme modulator designed to restore the function of glucocerebrosidase (GCase), which becomes impaired in Parkinson’s disease, particularly in patients with GBA1 mutations.
Gain Therapeutics plans to host a webinar with key opinion leaders on October 14 to further discuss the results.
In other recent news, Gain Therapeutics has extended its Phase 1b study for GT-02287, an experimental treatment for Parkinson’s disease. The extension, approved by Australian ethics committees, allows participants to continue receiving the therapy for up to 12 months, beyond the initial 90-day period. This extension comes as more than half of the study’s 21 participants have opted to continue treatment. Meanwhile, H.C. Wainwright has reiterated its Buy rating for Gain Therapeutics, maintaining an $8 price target, reflecting a positive outlook on the company’s progress. In contrast, BTIG adjusted its price target for Gain Therapeutics to $9 from $10, citing the timing of biomarker data release as a factor, while still maintaining a Buy rating. The company plans to release cerebrospinal fluid data on key biomarkers in the fourth quarter. Additionally, Gain Therapeutics announced a public offering of common stock and warrants, intending to use the proceeds to advance the development of GT-02287 and for general corporate purposes. These developments indicate ongoing advancements and strategic financial planning by the company.
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