Gain Therapeutics (GANX) Corporate presentation summary

Company overview

• Founded in 2017, with IPO in March 2021 and headquarters in Bethesda, MD, plus 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-driven structural biology to identify novel allosteric binding sites for drug discovery.

• Partners with leading medical and scientific organizations.

• Listed on NASDAQ with ticker GANX.

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, Gaucher's, and related disorders.

• GT-02287 has completed Phase 1a and 1b trials, showing disease-modifying potential in Parkinson's disease.

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

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

Clinical data and efficacy

• Phase 1b trial in Parkinson's patients showed statistically significant biomarker evidence of central target engagement and clinical improvement (MDS-UPDRS scores).

• GT-02287 demonstrated well-tolerated safety profile in healthy volunteers and patients, with no severe adverse events.

• Patients with high baseline CSF GluSph had an average 81% reduction after 90 days of treatment and a 6.2-point improvement in MDS-UPDRS II+III scores.

• GT-02287 reduced DDC levels in high GluSph group, indicating impact on dopaminergic neuron function.

• Animal models confirmed disease-modifying effects, improving neuronal survival and motor function.