Gain Therapeutics (GANX) investor relations material

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-driven structural biology to identify novel allosteric binding sites on disease-related proteins.

• Focuses on developing 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, Gaucher's, and related disorders.

• GT-02287 has completed Phase 1a in healthy volunteers and Phase 1b in Parkinson's patients.

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

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

Clinical data and mechanism of action

• GT-02287 is an allosteric modulator that restores GCase function, improving neuronal survival and reducing toxic substrates.

• Phase 1b showed statistically significant biomarker evidence of disease modification and clinical improvement in MDS-UPDRS scores.

• Patients with high baseline CSF GluSph had an average 81% reduction and greater motor score improvements.

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

• Preclinical and animal models confirm disease-modifying effects in both GBA1 and idiopathic PD.