Company: GANX
Filing Date: 2025-11-07
Form Type: S-3/A
Source: 0001104659-25-108472
Chunk: 7

Company: Gain Therapeutics, Inc.
Filing Date: 2025-11-07
Form: S-3/A
Chunk 7
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 in the summer of 2025 with interim data from the study expected by mid-2025. The Phase 1b open-label trial will assess the safety and tolerability of 13.5 mg/kg/day of GT-02287 for three months in patients with GBA1-PD or idiopathic Parkinson’s disease. Secondary endpoints include pharmacokinetics, GCase modulation, levels of GCase substrates, and other biomarkers in plasma and cerebrospinal fluid. The primary goal of the

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Phase 1b trial is to assess the safety and tolerability of GT-02287. Upon successful completion we expect to begin planning a Phase 2 study during the second half of 2025.

In preparation for the treatment of Parkinson’s disease patients, we initiated a chronic (6 months in rodents and 9 months in non-rodents) preclinical toxicity study in July 2024, enabling the conduct of clinical studies in patients with a GT-02287 treatment duration beyond three months. The 6- and 9-month studies will be completed in the second quarter of 2025 and the third quarter of 2025, respectively.

In October 2025, the Company presented early results from its Phase 1b clinical study in patients with Parkinson’s Disease at the International Congress of Parkinson’s Disease and Movement Disorders ® . GT-02287 was generally well tolerated, with no treatment-emergent serious adverse events observed. Additionally, participants in the open label trial appear to be benefitting from stabilization and trending improvements in their MDS-UPDRS scores after 90 days of GT-02287 administration. In September 2025, the company elected to extend the open label study for an additional 9 months to allow participants interested in remaining on GT-02287 to continue on the study. The majority of participants have elected to remain on the open label extension.

Our Magellan™ Platform

We use our computational target and drug discovery platform, Magellan™, to discover novel allosteric binding sites on proteins implicated in a disease and to identify proprietary small molecules that bind these sites to modulate protein function and treat the underlying cause of the disease. We believe that Magellan™ is uniquely suited to identify allosteric binding sites on the protein surface, which are different from the active (or orthosteric) binding site where the natural ligand of the protein binds. Targeting an allosteric binding site instead of the active