Early Trial Success Signals Promise for GT-02287 in Parkinson’s Disease Treatment

In an interview with Pharmaceutical Executive, Gene Mack, CEO, GAIN Therapeutics, shared promising updates on the company’s lead candidate, GT-02287, a novel therapy in development for Parkinson’s disease. Enrollment for the Phase Ib open-label trial wrapped three months ahead of schedule, driven by growing clinician confidence in the drug’s safety and therapeutic potential. Originally designed for patients with a GBA1 gene mutation—a known disruptor of the GCase enzyme—GT-02287 has shown early signs of benefit across a broader Parkinson’s population. With biomarker data expected in Q4 2025, GAIN is preparing for a pivotal Phase II trial in early 2026 while weighing financing options and potential strategic partnerships, all hinging on forthcoming efficacy and biomarker insights.

Pharmaceutical Executive: What drove the acceleration in enrollment for the GT-02287 trial, allowing you to complete it three months ahead of schedule?

Gene Mack: To be fair, we weren’t looking to enroll a large number of patients, but the pace of enrollment picked up as the study progressed—and that’s a very positive sign. When initiating clinical trials with an investigational drug, especially in centers with no prior experience using it, there’s often some initial hesitation. Even with all the necessary approvals and regulatory safeguards in place, clinicians can be cautious about enrolling patients, particularly in a disease like Parkinson’s, where few therapies have delivered meaningful disease-modifying efficacy.

While treatments like levodopa have certainly benefited patients, there’s still a significant unmet need. Clinicians on the front lines are responsible for their patients’ outcomes and naturally want to see how early participants respond before fully committing. As the trial continued, we observed growing comfort among clinicians, which translated into increased enrollment. This is a common dynamic in clinical research—momentum builds as confidence in the therapy grows. In our case, it reflected increasing enthusiasm and optimism about the potential of GT-02287, which was very encouraging.

Full Interview Summary: Enrollment for the Phase Ib trial of GT-02287, a novel therapeutic candidate for Parkinson’s disease, was completed three months ahead of schedule. While the trial targeted a small cohort, momentum increased as clinicians grew more comfortable with the drug’s safety profile and potential. Initially cautious due to the investigational nature of the therapy, sites became more confident in recommending the study, reflecting growing optimism about GT-02287’s promise.

The ongoing Phase Ib trial is an open-label pilot involving 15 to 20 patients with Parkinson’s disease, including those with idiopathic PD and those with a GBA1 gene mutation. This gene mutation disrupts the production of glucocerebrosidase (GCase), a key enzyme that GT-02287 is designed to restore. Although the drug was initially developed for GBA1-mutated patients, early findings suggest it may benefit a broader PD population. The Phase Ib readout, expected in Q4 2025, will include biomarker data from cerebrospinal fluid to assess differential efficacy between patient subtypes.

Looking ahead, a larger, double-blind Phase II trial is planned for early 2026, enrolling 100–200 patients to validate efficacy signals in a blinded setting and mitigate bias seen in open-label designs.

GT-02287 was developed using the company’s Magellan AI platform, which identifies novel binding pockets on target proteins and screens both known and hypothetical molecules. Magellan extends drug discovery into previously unexplored chemical space by predicting interactions that can be synthesized and tested.

Partnership discussions are ongoing with large pharma, biotech, and mid-cap firms. Final decisions around financing or partnering will depend on Phase Ib results. The company is prepared to either self-fund the $50–60M Phase II trial or advance through a strategic collaboration, depending on the biomarker and efficacy data in Parkinson’s patients.