Gain Therapeutics completes enrollment in phase 1b study to evaluate GT-02287 in PD

Gain Therapeutics completes enrollment in phase 1b study to evaluate GT-02287 in PD

By, Admin 3 July 2025

Overview

Gain Therapeutics, Inc, a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, provided an update on the progress of its ongoing phase 1b clinical study evaluating the safety and tolerability of GT-02287 in people with Parkinson’s disease with or without a GBA1 mutation. The study, which aimed to enroll 15-20 participants, reached 16 participants on June 30th, 2025.

Statement from Gene Mack: president & CEO, Gain Therapeutic

  • We are extremely pleased we’ve reached our target enrollment for our phase 1b trial of GT-02287, even with an initial two-month delay in protocol implementation,"" said Gene Mack, president and CEO of Gain Therapeutics. “The faster-than-anticipated enrollment means all those participants will reach their 90-day visit in time to facilitate biomarker analysis of all CSF samples taken by fourth quarter of this year instead of the first quarter of 2026. Based on the greater relevance of CSF biomarkers as proof of drug activity, and considering our new timeline, Gain is expected to provide full results on 90-day biomarker evidence from all CSF and blood samples together during 4Q 2025, which is also earlier than originally planned.”
  • Mack continued, ""We are grateful to the patients and clinicians whose strong interest in novel, potentially disease modifying treatments for Parkinson’s disease, together with previous evidence of GT-02287 target engagement from our phase 1 healthy volunteer study, led to completing enrollment of the study before the end of the summer as originally planned. Current treatments for Parkinson’s disease are aimed at alleviating symptoms while there are no treatments available to impact the eventual progression of Parkinson’s disease beyond transient symptom relief. There are over 1 million Americans with Parkinson’s disease making this population of individuals among the larger populations with a significant unmet need.”

Phase 1b Extension to Inform Gain’s Phase 2 Planning in 2026

  • Additionally, to facilitate the continued interest on the part of clinicians and patients currently aware of the phase 1b study in Australia, Gain will extend the screening window for participants through July 31st, 2025. 
  • Gain is also planning to approach the local health authorities in Australia to extend the dosing period of the phase 1b beyond the 90-day period currently allowed in the protocol. 
  • Long-term chronic toxicology studies required to support this extension of dosing are near completion, and Gain is expected to provide an update on the progress of the extension before the end of 3Q of 2025. 
  • Gain believes that the extension of the phase 1b will also better inform phase 2 protocol design and planning for early 2026.

About the lead candidate: GT-02287

  • Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. 
  • The orally administered, brain-penetrant small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. 
  • In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced ER stress, lysosomal and mitochondrial pathology, aggregated a-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. 
  • In rodent models of both GBA1-PD and idiopathic PD, GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as nesting.

Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggest that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease.

The outcomes

  • Results from a phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with a >50% increase in glucocerebrosidase (GCase) activity among those receiving GT-02287 at clinically relevant doses.
  • GT-02287 is currently being evaluated in a phase 1b clinical trial for the treatment of Parkinson’s disease with or without a GBA1 mutation. 
  • The primary endpoint of the trial, which is currently enrolling participants across 7 sites in Australia, is to evaluate the safety and tolerability of GT-02287 after 3 months of dosing in people with Parkinson’s disease.

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