BETHESDA, Md., June 29, 2026
Gain Therapeutics announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for GT-02287, enabling the initiation of a Phase 2 clinical trial for patients with Parkinson’s disease, including those with and without a GBA1 mutation. The Phase 2 study is expected to begin during the third quarter of 2026 across clinical sites in the United States, Australia, and Europe.
The regulatory milestone follows encouraging Phase 1a and Phase 1b clinical results, which demonstrated target engagement, favorable safety and tolerability, biomarker improvements, and early signs of clinical activity. GT-02287 also becomes the first allosteric small-molecule therapy developed using Gain Therapeutics’ proprietary Magellan™ AI drug discovery platform to receive FDA IND authorization, highlighting the growing role of artificial intelligence in accelerating innovative drug development for neurodegenerative diseases.
Phase 1 Results Support Advancement Into Phase 2
Gain Therapeutics reported that GT-02287 continued to demonstrate a strong safety profile during an ongoing Phase 1b extension study, with all 16 participants remaining on treatment through Day 150 and an independent Data Monitoring Committee recommending continuation without modifications. Biomarker analyses showed an average 81% reduction in cerebrospinal fluid glucosylsphingosine (GluSph) among participants with elevated baseline levels after 90 days of treatment, indicating sustained target engagement. Investigators also observed reductions in DOPA decarboxylase (DDC), another biomarker associated with Parkinson’s disease biology.
Clinical assessments further supported these findings, with participants exhibiting improvements or stabilization in Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part II and III scores, particularly among patients with elevated baseline GluSph levels. In addition, several participants reported encouraging improvements in balance, gait, smell, taste, sleep quality, and other motor and non-motor symptoms, providing further rationale for advancing the program into larger efficacy studies.
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GT-02287 Targets the Underlying Biology of Parkinson’s Disease
Unlike conventional Parkinson’s therapies that primarily address symptoms, GT-02287 is designed to target the underlying disease mechanisms responsible for neurodegeneration. The orally administered, brain-penetrant small molecule functions as an allosteric modulator of glucocerebrosidase (GCase), restoring activity of the lysosomal enzyme impaired by GBA1 mutations and age-related cellular stress. Dysfunction of GCase contributes to the accumulation of alpha-synuclein, neuroinflammation, mitochondrial dysfunction, and progressive neuronal loss, all hallmarks of Parkinson’s disease.
Preclinical studies demonstrated that GT-02287 restored enzyme function, reduced pathological protein aggregation, improved lysosomal and mitochondrial health, lowered neurodegeneration biomarkers, and significantly improved motor performance in animal models. These findings suggest the therapy may have the potential to slow or halt disease progression, addressing one of the largest unmet needs in Parkinson’s disease treatment, where no currently approved therapies have demonstrated disease-modifying effects.
AI Drug Discovery Platform Drives Future Pipeline Growth
The FDA’s IND clearance also represents an important validation of Gain Therapeutics’ Magellan™ AI drug discovery platform, which is designed to identify novel allosteric small-molecule therapies capable of restoring dysfunctional proteins involved in complex diseases. Beyond Parkinson’s disease, GT-02287 is also being explored for potential applications in Gaucher’s disease, dementia with Lewy bodies, and Alzheimer’s disease, while the company continues to advance additional preclinical programs targeting lysosomal storage disorders, metabolic diseases, and oncology.
Gain’s Parkinson’s program has received funding support from organizations including The Michael J. Fox Foundation for Parkinson’s Research, The Silverstein Foundation for Parkinson’s with GBA, and the Eurostars-2 program supported by the European Union. With FDA IND clearance now secured, Gain Therapeutics is preparing to launch its global Phase 2a clinical trial later this year, marking a significant step toward developing a potential disease-modifying therapy for Parkinson’s disease.
Source: Gain Therapeutics, press release
