Gain Therapeutics (GANX) Corporate presentation summary

Company overview

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

• Employs 23 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.

Pipeline and technology

• Magellan platform supports a pipeline targeting neurodegenerative and lysosomal storage disorders, including Parkinson's, Gaucher's, and Alzheimer's diseases.

• Rexaceract is the lead asset, targeting GCase for Parkinson's, with additional candidates in preclinical stages.

• Allosteric modulation of GCase aims to restore enzyme function and neuronal survival.

Clinical development and results

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

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

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

• Rexaceract was well-tolerated, with no severe adverse events and therapeutic CNS exposure achieved.