Gain Therapeutics (GANX) The 38th Annual Roth Conference summary

Unmet needs and therapeutic approach

• Current Parkinson's treatments focus on symptomatic relief, mainly using L-DOPA and related drugs, but do not address underlying disease biology.

• The aim is to develop disease-modifying therapies by targeting glucocerebrosidase (GCase) to slow or halt neurodegeneration.

• A new therapeutic backbone is envisioned, combining disease modification with symptomatic treatments for longer-lasting benefits.

Magellan platform and drug discovery

• The Magellan platform automates complex binding kinetic calculations, enabling rapid identification of small molecules that stabilize target proteins.

• Unlike traditional approaches, the focus is on promoting protein function (gain of function) rather than inhibition.

• GT-02287 was selected for its ability to stabilize and chaperone GCase, supporting its function in lysosomes and mitochondria.

Clinical results and biomarker analysis

• Early clinical studies showed GT-02287 is safe and well-tolerated, with minor nausea in healthy volunteers but not in Parkinson's patients.

• Biomarker analysis revealed reductions in glucosylsphingosine in CSF, indicating effective GCase engagement and potential disease modification.

• MDS-UPDRS scores showed stability, with some patients responding more quickly, especially those with high glucosylsphingosine.

• Physicians are interested in biomarker data, though remain cautious about short-term clinical outcomes due to Parkinson's slow progression.