Small-Cap Gain Therapeutics Stock Falls On Phase 1 Data From Its Lead Asset For Parkinson's Disease

Thursday, Gain Therapeutics, Inc. GANX released results from the Phase 1 study to evaluate the safety, tolerability, and pharmacokinetics of GT-02287.

The company previously announced that no discontinuations or serious adverse events were reported when the study concluded.

Also Read: EXCLUSIVE: Gain Therapeutics Highlights Lead Program Data Showcasing Its Impact On Cellular Death For Treating Parkinson’s Disease.

The Phase 1 study enrolled 72 healthy volunteers. A review of unblinded data after database lock confirmed that single and multiple doses of GT-02287 were safe and generally well tolerated up to and including the highest planned dose levels across all age groups

GT-02287 was present in the cerebrospinal fluid (CSF), and peripheral target engagement was demonstrated.

The favorable safety and tolerability profile at oral dose levels that resulted in therapeutic plasma levels, CNS exposure, and target engagement.

“On the heels of this data, we expect to initiate a trial in people with Parkinson’s disease by Q4 2024 with the goal of demonstrating safety and tolerability in patients with Parkinson’s disease and to obtain proof of mechanism based on relevant biomarkers. We anticipate having data from Parkinson’s disease patients by mid-point 2025,” commented Jonas Hannestad, Chief Medical Officer of Gain.

In February, Gain Therapeutics released preclinical data demonstrating that its clinical-stage GCase regulator GT-02287 provided neuroprotection and restored motor function in Parkinson’s disease models following delayed administration.

Price Action: GANX stock is down 13.40% at $1.16 at the last check on Thursday.

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