Gain Therapeutics’ GT-02287 was found to be safe and tolerable in the Phase I trial of the therapy in healthy volunteers.

GT-02287 is an allosteric protein modulator which is being developed as a treatment for Parkinson’s disease associated with GBA1 gene mutation, one of the common mutations in patients with Parkinson’s. It is found in as many as 15% of patients with the disorder. The GBA1 gene mutation leads to the production of misfolded and impaired Glucocerebrosidase (GCase) lysosomal protein enzyme. GT-02287 restores the function of GCase enzyme, thereby, potentially slowing disease progression.

The single-centre, placebo-controlled Phase I trial evaluated the safety and tolerability of GT-02287 in healthy volunteers. The trial first evaluated the therapy in a single ascending dose (SAD) cohort before progressing to a multiple ascending dose (MAD) phase in February following the approval from the Bellberry Human Research Ethics Committee (HREC) in Australia.

The SAD cohort enrolled 40 patients and evaluated five doses of the therapy. Gain was sparse on providing trial detail but noted that the therapy had a “good safety and tolerability profile”, and that the therapy was well tolerated up to and including the highest planned dose level, with no serious adverse events. The MAD cohort is expected to be completed in the second quarter of this year.

“Current therapies only treat symptoms whereas GT-02287 has the potential to slow or stop disease progression in PD by treating the underlying cause of the disease,” said Gain chief medical officer Jonas Hannestad.

“We expect to initiate a first-in-patient cohort in the Phase I clinical trial in the second half of 2024 that can provide proof of mechanism in patients with PD based on relevant biomarkers and potentially other exploratory measures.”

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Following the news, Gain’s shares rose by 13% on market open on 24 April, compared to market open on the previous day. The company’s market cap stands at $51.7m.

In February, Gain presented preclinical data showing that GT-02287 restored motor function and substantially reduced plasma levels of neurofilament light chain (NfL), an emerging neurodegeneration biomarker, in mice models.

Gain received funding from the Michael J. Fox Foundation for Parkinson’s Research (MJFF), the Silverstein Foundation for Parkinson’s with GBA, the Eurostars-2 joint program, the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency, for developing GT-02287.

Other Parkinson’s therapies currently in development include Bayer’s gene therapy AB-1005 currently in Phase Ib, IRLAB Therapeutics’ alpha 2 and 5HT 7 receptor inhibitor, pirepemat, currently in Phase IIb along with Phase I Parkinson’s therapy HL192 developed by HanAll Biopharma, NurrOn Pharmaceuticals and Daewoong Pharmaceutical.