Gain Therapeutics (GANX) Maxim Group’s 2024 Healthcare Virtual Summit summary

Leadership and company overview

• Interim CEO and CFO Gene Mack joined in April and became CEO in June, bringing extensive experience in biotech and finance.

• The company is transitioning from preclinical to clinical stage, now able to discuss human and patient data.

Scientific and clinical progress

• GT-02287 is a GCase modulator aiming to be the first disease-modifying therapy for Parkinson's disease.

• The drug targets the underlying disease mechanism by modulating GCase, impacting toxic substrate clearance, ER stress, and mitochondrial health.

• Recent multiple ascending dose studies in healthy volunteers showed significant GCase modulation at predicted therapeutic doses.

• Data presented at major conferences, including Movement Disorders and Society for Neuroscience, support the clinical potential.

Patient population and trial design

• GT-02287 was initially focused on GBA1 Parkinson's patients but now includes idiopathic patients due to observed GCase elevation in healthy volunteers.

• Phase 1b will enroll both GBA1 and idiopathic Parkinson's patients, with dosing expected to start early next year.

• The trial will dose 10-15 patients for three months, with potential for protocol extension and expansion to Phase 2.