Gain Therapeutics (GANX) Corporate presentation summary

Company overview and platform

• Founded in 2017, IPO in March 2021, with headquarters in Bethesda, MD and offices in Switzerland and Spain.

• Employs 25 people and is led by Dr. Khalid Islam as Founder and Executive Chairman.

• Proprietary Magellan platform uses AI-supported structural biology to identify novel allosteric binding sites on disease-related proteins.

• Focuses on generating first-in-class or best-in-class product candidates using allosteric mechanisms.

• Partners with leading medical and scientific organizations.

Pipeline and lead asset

• Pipeline includes programs for Parkinson's, Gaucher's, dementia with Lewy bodies, Alzheimer's, and lysosomal storage disorders.

• Lead asset GT-02287 targets GCase for Parkinson's, with additional indications in development.

• Completed Phase 1a in healthy volunteers and Phase 1b in Parkinson's patients, with 84% joining a 9-month extension.

• Phase 2 trial for GT-02287 in Parkinson's planned to start in Q3 2026.

• Holds global rights to GT-02287 with patent protection through 2038.

Clinical data and efficacy

• GT-02287 restores GCase function, improves neuronal survival, and shows disease-modifying effects in preclinical and clinical models.

• Phase 1b demonstrated statistically significant biomarker evidence of central target engagement and clinical improvement via MDS-UPDRS scores.

• Patients with high baseline CSF GluSph showed greater improvement in motor scores after 90 and 150 days of dosing.

• GT-02287 led to an average 81% reduction in CSF GluSph after 90 days in high baseline group.

• Well-tolerated in both healthy volunteers and PD patients, with no severe adverse events.