BETHESDA, Md., Aug. 11, 2026 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today reported financial results for the quarter ended June 30, 2026, and provided a corporate update.
"The second quarter marked an important period of execution for Gain, highlighted by FDA authorization of our IND for rexaceract, which positions us to advance into Phase 2 clinical development," said Gene Mack, President and CEO of Gain Therapeutics. "The encouraging biomarker and clinical data generated to date continue to support the potential disease-modifying properties of rexaceract, and we were pleased to further share these findings with the scientific community at the 3rd International GBA1 Meeting. We believe these data further strengthen the therapeutic rationale for rexaceract across both idiopathic and GBA1 Parkinson's disease."
Mr. Mack continued, "We remain focused on completing the ongoing nine-month extension of our Phase 1b study, with final data expected to be presented at the International Congress of Parkinson's Disease and Movement Disorders in Korea in October 2026, while advancing preparations for our Phase 2 clinical program. As of June 30, 2026, 14 of the 16 participants that elected to enroll in the Phase 1b nine-month extension had completed Day 270 with seven of those crossing the Day 360 mark. To date, MDS-UPDRS scores continue to demonstrate that patients with high baseline CSF GluSph experience an early and durable response to rexaceract that persists through Day 270 and Day 360, while the overall patient group continues to benefit from non-progression of their Parkinson's symptoms through Day 360. We continue to believe rexaceract has the potential to shift the treatment paradigm beyond symptom management by addressing the underlying biology of Parkinson's disease, and we look forward to achieving additional clinical and regulatory milestones throughout the remainder of the year."
Second Quarter 2026 and Recent Corporate and Pipeline Highlights
Pipeline Updates
Rexaceract
-- Presented additional data from the Phase 1b clinical study of rexaceract
(GT-02287) that supported its disease-modifying potential via
demonstrating the restoration of glucocerebrosidase (GCase) biology
at the 3rd International GBA1 Meeting 2026 in May 2026 in Phoenix, AZ.
-- As of May 2026, all 16 participants enrolled in the Phase 1b,
nine-month extension remained on study and had completed five
months of dosing (Day 150).
-- MDS-UPDRS scores remained stable and durable across the overall
study population after 150 days of treatment with rexaceract.
-- Participants with elevated baseline levels of glucosylsphingoshine
(GluSph) in cerebrospinal fluid $(CSF)$ continued to benefit more
than those with low baseline levels of GluSph in CSF after 150
days of dosing with rexaceract, with a difference of 4.8 points in
the sum of MDS-UPDRS Part II and Part III scores between the two
groups at Day 150.
-- Participants provided unsolicited descriptions of perceived
benefit after 90 days of dosing with rexaceract. Perceived
benefits most commonly described included four instances of
improved sense of smell and taste, four instances of improved
balance or gait, and three instances of improved sleep.
-- Rexaceract accepted as the International Nonproprietary Name $(INN)$ for
GT-02287, establishing the compound's official generic name as it
continues to advance through clinical development.
GT-04686
-- GT-04686 is moving towards IND-enabling studies and ultimately, clinical
development.
-- Current activities are focused on optimizing the program and
generating the data necessary to support IND-enabling studies.
-- The advancement of GT-04686 presents an opportunity to bolster the
Company's pipeline and create additional long-term value for
shareholders.
-- Initial therapeutic indication for GT-04686 expected to be
selected by year end.
-- GT-04686 is the second allosteric modulator developed from Gain's
Magellan$(TM)$ AI drug discovery platform that is planned for entry
into the clinic.
Corporate Updates
Regulatory
-- The U.S. Food and Drug Administration (FDA) completed its review of an
Investigational New Drug $(IND)$ application for rexaceract.
-- Acceptance of the IND submission paves the way for initiation of
rexaceract Phase 2 clinical development in the U.S., expected
during Q3 2026.
-- The acceptance follows positive Phase 1 results for rexaceract in
both healthy volunteers and people with Parkinson's disease
demonstrating both biomarker and clinical evidence of activity
with favorable safety and tolerability.
-- Rexaceract is the first allosteric modulator developed from Gain's
Magellan(TM) AI drug discovery platform to receive IND clearance.
Management Changes
-- Promotion of Dr. Joanne Taylor to Chief Scientific Officer, reinforcing
the Company's scientific leadership and advancing its research and
development strategy.
-- Promotion of Terenzio Ignoni to Chief Operating Officer, strengthening
the Company's executive leadership and operational execution.
Upcoming Anticipated Milestones
-- Phase 2 clinical trial of rexaceract in people with Parkinson's disease
expected to begin in Q3 2026.
-- Final results from Phase 1b clinical study of rexaceract expected in Q4
2026.
-- GT-04686 initial target indication expected in Q4 2026.
Q2 2026 Financial Results
Research and development (R&D) expenses decreased by $0.6 million to $2.1 million for the three months ended June 30, 2026, compared to $2.8 million for the three months ended June 30, 2025. The decrease in research and development expenses was primarily related to lower costs associated with our lead program compound GT-02287 (rexaceract) for the treatment of Parkinson's Disease and optimization of the pipeline, together with lower research and development personnel expenses, partially offset by the expiration of the grant awarded by Innosuisse under the Swiss Accelerator program in April 2026 and unfavorable foreign exchange currency translation as the Swiss franc and Australian dollar strengthened against the U.S. dollar.
General and administrative (G&A) expenses increased by $0.2 million to $2.5 million for the three months ended June 30, 2026, compared to $2.3 million for the three months ended June 30, 2025. The increase in general and administrative expenses for the period was primarily attributable to higher professional fees, higher personnel costs, and unfavorable foreign exchange currency translation as the Swiss franc strengthened against the U.S. dollar.
Net loss for the three months ended June 30, 2026, was $0.11 per share, basic and diluted, compared to $0.19 per share, basic and diluted, for the three months ended June 30, 2025.
Cash and cash equivalents were $13.1 million as of June 30, 2026, compared to $20.8 million as of December 31, 2025.
About Rexaceract
Gain Therapeutics' lead drug candidate rexaceract, formerly known as 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 a beta-glucocerebrosidase (GCase) positive allosteric modulator, antiparkinsonian, that restores the function of the lysosomal enzyme 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, by stabilizing and chaperoning it to the lysosomes and mitochondria. In preclinical models of PD, rexaceract restored GCase enzymatic function, reduced endoplasmic reticulum stress, lysosomal and mitochondrial pathology, aggregated <ALPHA>-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, rexaceract 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 rexaceract, suggest that rexaceract may have the potential to slow or stop the progression of Parkinson's disease.
Results from a Phase 1 study of rexaceract in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with an increase in GCase activity among those receiving rexaceract at clinically relevant doses.
Rexaceract 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 enrolled participants across seven sites in Australia, is to evaluate the safety and tolerability of rexaceract after three months of dosing in people with Parkinson's disease. The Phase 1b study extension allows participants to continue to be treated with rexaceract for up to a total of 12 months.
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