Company: GANX
Filing Date: 2025-04-10
Form Type: 8-K
Source: 0001104659-25-033797
Chunk: 1

Company: Gain Therapeutics, Inc.
Filing Date: 2025-04-10
Form: 8-K
Item: Item 8.01
Chunk 1
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Item 8.01. Other Events.

On April 10, 2025, the Company announced
the presentation of additional preclinical data and design of the Company’s Phase 1b clinical study of GT-02287. An early biomarker
analysis from the currently enrolled participants in the Phase 1b study will be conducted mid-year and will include samples from participants
both with and without the GBA1 mutations for purposes of informing Phase 2 planning during the second half of 2025. Complete analysis
of the Phase 1b endpoints, available after all patients have completed 90 days of treatment, is anticipated sometime during
the fourth quarter of 2025.

Clinical Stage Glucocerebrosidase Modulator,
GT-02287, Shows Disease Modifying Potential in Preclinical Models Of Both GBA1 and Idiopathic Parkinson’s Disease

In animal models of both GBA1 and idiopathic Parkinson’s
disease, GT-02287 is able to rescue motor deficits and prevent the development of deficits in complex behaviors such as nesting. These
effects persist even following withdrawal of the compound, suggesting a disease-modifying potential of GT-02287. The new preclinical data
that were presented additionally support a disease-modifying potential of GT-02287. Several biomarkers of disease progression, including
aggregated α-synuclein, IRE-1 (a marker of ER stress), LAMP-1 (a marker of lysosomal integrity), Miro1 (a marker of damaged
mitochondrial destined for mitophagy), phospho-Tau (a marker of neurodegeneration), and Iba-1 (a marker of neuroinflammation) were statistically
reduced upon treatment with GT-02287 and remained reduced for several days following drug washout demonstrating a long-lasting effect.

Design of a Phase 1b Study to Evaluate the
GCase-targeting Molecule GT-02287 in GBA1-PD and Sporadic PD

The study will assess safety and tolerability
of GT-02287 during three months of dosing in people with PD, using adverse events, laboratory tests, electrocardiograms, and vital signs
as the key safety endpoints. Additionally, the study will characterize the single-dose and steady-state plasma pharmacokinetics (PK) profile
of GT-02287. Exploratory endpoints include various biomarkers in the cerebrospinal fluid (CSF), dried blood spots, plasma, and whole