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 integrates AI-supported structural biology and proprietary algorithms to identify novel allosteric binding sites on disease-implicated 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

• Lead asset GT-02287 targets Parkinson's disease, with additional programs in Gaucher's, dementia with Lewy bodies, Alzheimer's, and lysosomal storage disorders.

• GT-02287 completed Phase 1a in healthy volunteers and Phase 1b in Parkinson's patients, with 84% of patients joining a 9-month extension study.

• Biomarker evidence supports disease-modifying potential for both GBA1 and idiopathic Parkinson's disease.

• Full worldwide rights to GT-02287, with patent protection through 2038 and additional applications under review.

• Supported by grants from Michael J. Fox Foundation, Silverstein Foundation, and Innosuisse.

Clinical data and efficacy

• GT-02287 binds allosterically to chaperone GCase enzyme, restoring function and improving neuronal survival in preclinical models.

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

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

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

• Phase 2 trial is planned to start in Q3 2026.