Gain Updates Parkinson’s Pipeline, Promotes Executives; Cash Runway Into Q2 2027

(RTTNews) – Gain Therapeutics, Inc. (GANX) President and CEO, Gene Mack on Thursday provided investors with an update on the company’s progress of its clinical pipeline, leadership initiatives, and cash position.

Clinical Pipeline Update

Gain’s lead Parkinson’s disease candidate, Rexaceract, continued to demonstrate a favorable safety and tolerability profile in the Phase 1b trial and its open-label extension, with positive results maintained through five months of treatment.

Rexaceract (GT-02287) is an orally administered, brain penetrant small molecule and a beta-glucocerebrosidase (GCase) positive allosteric modulator designed to restore lysosomal GCase function impaired by GBA1 gene mutations. It is being developed for the treatment of Parkinson’s disease with or without GBA1 mutation.

As of March 2024, 14 of 16 participants had completed five months of dosing (Day 150), while an independent Data Monitoring Committee recommended the trial continue without modification. Results from the Phase 1b study are expected in the fourth quarter of 2026.

The company said previously reported reductions in cerebrospinal fluid glucosylsphingosine (GluSph) levels and early clinical benefits were largely maintained through at least five months of treatment.

Gain added that Rexaceract reduced cerebrospinal fluid (CSF) GluSph levels by an average of 81% after 90 days in patients with elevated baseline GluSph levels, a biomarker associated with GCase dysfunction. The GCase substrate GluSph is considered a significant biomarker for Gaucher’s disease, and an emerging biomarker for Parkinson’s disease.

Furthermore, participants with elevated baseline CSF GluSph levels following 90 days of Rexaceract treatment showed decreased DOPA decarboxylase (DDC) levels.

Participants with elevated GluSph baseline after 150 days of treatment, demonstrated clinical benefits with a 4.8-point improvement over those with lower baseline levels in combined Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) part 2 and 3 scores.

The company said this biomarker improvements were accompanied by early signs of clinical benefit.

The U.S. FDA authorized the Investigational New Drug (IND) for Rexaceract in the second quarter of 2026, enabling the company to initiate patient enrollment in a Phase 2 trial in early Parkinson’s disease by the third quarter of 2026.

The IND authorization was supported by positive Phase 1 results showing rexaceract was well tolerated and demonstrated biomarker and early clinical activity.

Gain’s second pipeline candidate, GT-04686, demonstrated positive preclinical results in Parkinson’s disease animal models. The candidate showed activity in both in vitro and in vivo models, including increase in GCase activity, lipid substrate depletion in patient fibroblasts harboring both mutated and wildtype GBA1, and restoration of motor and non-motor function.

The company expects to select its initial therapeutic indication by year-end 2026 and is advancing IND-enabling activities ahead of clinical development.

Organizational Update

Gain’s Board of Directors has agreed to appoint Joanne Taylor as Chief Scientific Officer (CSO) and Terenzio Ignoni as Chief Operating Officer (COO).

Taylor joined Gain Therapeutics as Senior Vice President in September 2020 and brings over three decades of experience in the pharmaceutical industry. Prior to that, she served as Vice President and Head of Neuroscience at Prescient Healthcare Group. She also held multiple roles in Eisai’s London Research Laboratories and European Headquarters.

Ignoni joined Gain in 2021 as Senior Vice President, Quality and Chemistry Manufacturing and Controls (CMC), and has served as Senior Vice President, Technical Operations and Development since 2022. He previously held senior quality leadership roles at Jazz Pharmaceuticals, Gentium, and Zambon Group.

Cash Position

Cash, cash equivalents and marketable securities as of March 31, 2026, amounted to $16.5 million, which the company expects will fund operations into second quarter of 2027.

Gain shares have traded between $1.41 and $4.34 over the last year.

GANX closed Thursday’s trade down 1.19% at $1.66.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.