Presented initial data from Phase 1b study suggesting GT-02287 has a disease-slowing effect consistent with the preclinical models in vivo and the proposed mechanism of action
Completed enrollment of 21 participants in Phase 1b study evaluating GT-02287 in Parkinson's Disease with or without GBA1 mutations during 3Q 2025
Received approval from the Australian health authorities to extend the duration of the Phase 1b study, allowing participants to continue treatment with GT-02287 for a total of 12 months; with a majority of subjects electing to participate in the study extension
Analysis of functional changes and biomarker activity at 90 days expected to be available during 4Q 2025
BETHESDA, Md., Nov. 12, 2025 (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 September 30, 2025, and provided a corporate update.
"We are encouraged by the progress made during the third quarter of 2025 and remain on track to report the analysis of both functional changes and biomarker activity during the fourth quarter of 2025. We are excited to better understand the impact GT-02287 has on the biology of Parkinson's disease from this analysis and look forward to presenting what we learn. We presented early clinical findings last month at the 2025 MDS conference in Hawaii suggesting GT-02287 has a disease-slowing effect as evidenced by stabilization and improvement in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores that appears after approximately 30 days of administration according to our data," said Gene Mack, President and CEO of Gain. "We are further encouraged by the strong engagement we have seen in the Phase 1b study, with approximately 80% of participants having joined or confirmed their interest in the study extension phase. We look forward to generating additional feedback from this study extension that we can incorporate into our Phase 2 planning, which is already underway."
Important highlights during the quarter included completion of enrollment for the company's Phase 1b study evaluating lead candidate GT-02287 in Parkinson's disease with or without a GBA1 mutation. Of the 21 participants enrolled, which is a number that surpassed the original target of 15 participants, 16 have completed 90 days of dosing. The remaining 5 participants are expected to complete dosing in December 2025. Eligible participants that have completed 90 days of treatment with GT-02287 continue to transition into the Phase 1b study extension, which commenced September 2025.
Recent data presented at the International Congress of Parkinson's Disease and Movement Disorders$(R)$ demonstrated improvement in MDS-UPDRS scores after 90 days of dosing in the first nine participants enrolled and suggest a disease-slowing effect consistent with the preclinical models in vivo and the proposed mechanism of action of GT-02287. Accordingly, Gain expects a full 90-day analysis, including functional changes scored according to MDS-UPDRS and biomarker data from cerebrospinal fluid and blood, from participants that were enrolled as of June 30, 2025, to be available in Q4 2025 as previously expected.
Mr. Mack continued, "We are deeply grateful to the patients, investigators, and clinical sites for their continued commitment to advancing this important program. Participating in a Parkinson's disease trial is never an easy undertaking but the interest we continue to see underscores the significant unmet need for a disease-modifying therapy. We hope to shift the treatment paradigm and deliver a life-changing treatment to those that need it, and we believe we are well-positioned to do so."
Third Quarter 2025 and Recent Corporate and Clinical Program Highlights
Clinical Program Highlights
-- Presented initial clinical data in October 2025 that supported the
disease-modifying potential of GT-02287 in people with Parkinson's
disease at International Congress of Parkinson's Disease and Movement
Disorders(R) held in Honolulu, HI.
-- GT-02287 was generally well tolerated, with no treatment-emergent
serious adverse events observed.
-- Several participants experienced an improvement in their UPDRS
Part II and III scores after 90 days of dosing with GT-02287 while
mean Part I scores remained unchanged.
-- The mean improvement in Parts II and III by Day 90, which was not
observed by Day 30, suggests that GT-02287 has a disease-slowing
effect, consistent with preclinical models in vivo and the
proposed mechanism of action.
-- Plasma pharmacokinetics $(PK)$ profile was consistent across all 14
participants sampled, was within the projected therapeutic range,
and comparable to exposures observed in healthy volunteers in the
Phase 1 study.
Corporate Updates
-- Received Australian approval to extend dosing by an additional nine
months in the ongoing Phase 1b study, beyond the 90-day period originally
allowed in the protocol. A review of interim safety data by the
independent data monitoring committee resulted in no safety concerns
identified and a recommendation to continue the Phase 1b study with no
changes.
-- Completed enrollment in the Phase 1b study for GT-02287 in people with
Parkinson's disease regardless of GBA1 status. 21 participants have been
enrolled as of September 30, 2025, surpassing original target enrollment
of 15 participants.
-- Commenced Phase 1b study extension allowing participants to continue
treatment with GT-02287 for a total of 12 months. Eligible participants
continue to transition into the study extension to further assess
long-term safety and tolerability, functional changes scored according to
MDS-UPDRS, and biomarker levels.
-- Hosted a KOL event highlighting current thinking on Parkinson's disease
clinical outcome scales and biomarkers as well as the Company's initial
Phase 1b data. A replay of the event can be found here.
-- Completed an underwritten public offering that resulted in approximately
$7.1 million of net proceeds.
Upcoming Anticipated Milestones
-- Analysis of functional changes scored according to MDS-UPDRS and
biomarker levels in cerebrospinal fluid and blood samples from
participants who completed 90 days of the Phase 1b study evaluating
GT-02287 in people with Parkinson's disease expected in the fourth
quarter of 2025.
-- IND submission to FDA, expected by year end 2025, facilitating expansion
into Phase 2 clinical development of GT-02287 to include clinical sites
in the United States.
-- Results from Phase 1b study extension expected the second half of 2026.
Q3 2025 Financial Results
Research and Development (R&D) expenses increased by $0.2 million to $2.8 million for the three months ended September 30, 2025, as compared to $2.6 million for the three months ended September 30, 2024. The increase in R&D expenses was primarily related to costs associated with the ongoing Phase 1b clinical trial and unfavorable foreign exchange currency translation as the Swiss franc and Australian dollar strengthened against the U.S. dollar. The increase was partially offset by optimization of our pipeline costs and lower research and development personnel costs.
General and Administrative (G&A) expenses increased by $0.1 million to $1.9 million for the three months ended September 30, 2025, as compared to $1.8 million for the three months ended September 30, 2024. The increase in G&A expenses for the period was primarily attributable to higher stock-based compensation, higher personnel costs, and unfavorable foreign exchange currency translation as the Swiss franc and Australian dollar strengthened against the U.S. dollar. The increases were partially offset by lower legal and professional fees related to general corporate matters.
Net loss for the three months ended September 30, 2025, was $0.15 per share, basic and diluted, compared to $0.17 per share, basic and diluted, for the three months ended September 30, 2024.
Cash and cash equivalents were $8.8 million as of September 30, 2025, compared to $10.4 million as of December 31, 2024.
About 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 <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, 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.
(MORE TO FOLLOW) Dow Jones Newswires
November 12, 2025 07:00 ET (12:00 GMT)
Comments