Future Partnership Plans for GT-02287 and the Magellan Platform in Parkinson’s Research

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: Are there plans for partnerships or licensing agreements tied to GT-02287 or the Magellan platform?

Gene Mack: We’ll need to make several important decisions in the fourth quarter, once we receive the first relevant data set from Parkinson’s disease patients. Depending on the efficacy results and biomarker impact of GT-02287, we’ve been actively engaging with potential partners—including large pharma, mid-cap pharma, large biotech, and some mid-cap biotech companies. There’s a healthy level of interest among companies focused on neurodegenerative diseases.

That said, neurodegenerative disease remains a very challenging area. Unlike oncology or autoimmune diseases, conditions like Alzheimer’s and Parkinson’s have seen less progress, so there’s considerable risk aversion given the high failure rate in this space. We’ve been discussing this program with potential partners for several years, and now we’re approaching a critical stage.

At this point, the industry may choose to collaborate with us to advance the molecule, or we may decide to finance further development ourselves through capital markets. Both options are on the table, and we’ll see what the Phase 1b results indicate. After that, we’ll engage with investors and partners to determine the next steps.

Our Phase II study is expected to cost around $50 to $60 million. We could either fund this ourselves, partner the asset, or pursue a combination of both.

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.