Gain Therapeutics (NASDAQ: GANX) shares early Phase 1b Parkinson’s study data

Rhea-AI Filing Summary

Gain Therapeutics reported preliminary results from its ongoing Phase 1b study of GT-02287 in people with Parkinson’s disease, with or without a GBA1 mutation. After 90 days of dosing, GT-02287 has been safe and generally well tolerated, with no treatment-emergent serious adverse events. Some participants showed transient increases in alkaline phosphatase and other liver enzymes, which returned to normal even while treatment continued.

The 21 participants include treatment-naïve patients, individuals on deep brain stimulation, and most on levodopa and other Parkinson’s drugs. Several participants experienced improvements in their MDS-UPDRS Part II and III scores by Day 90, while mean Part I scores were unchanged. The company notes that the mean improvement in Parts II and III by Day 90, not seen by Day 30, suggests a potential disease-slowing effect consistent with preclinical models and the proposed mechanism of GT-02287. Plasma pharmacokinetics were consistent across sampled participants and within the projected therapeutic range.

A data monitoring committee has twice recommended continuing the study, and Australian health authorities have approved an extension allowing patients to be treated for up to 12 months.

Insights

Biotech clinical development analyst

Early Phase 1b Parkinson’s data for GT-02287 look encouraging on safety and exploratory efficacy.

The update shows GT-02287 was safe and generally well tolerated over 90 days in people with Parkinson’s disease, with no treatment-emergent serious adverse events. Transient liver enzyme elevations that normalized on treatment are important, as they outline a manageable safety signal that regulators and clinicians will watch closely.

Exploratory efficacy signals include improvements in MDS-UPDRS Part II and III scores by Day 90, changes that were not seen by Day 30. The company links this pattern to a possible disease-slowing effect consistent with its preclinical models and proposed mechanism, though these findings remain preliminary and from a small cohort.

The consistent plasma pharmacokinetic profile within the projected therapeutic range, together with the data monitoring committee’s recommendations to continue and Australian approval of an extension to treat patients for up to 12 months, supports ongoing development. Subsequent company filings may provide longer-duration safety and efficacy data as patients remain on GT-02287 beyond 90 days.

8-K Event Classification

Item 8.01 — Other Events

1 item

Item 8.01 Other Events Other

Voluntary disclosure of events the company deems important to shareholders but not covered by other items.

Understanding 8-K Material Events →

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

PURSUANT TO SECTION 13 OR 15(d) OF THE

SECURITIES EXCHANGE ACT OF 1934

Date of Report (Date of earliest event reported): October 6, 2025

GAIN THERAPEUTICS, INC.

(Exact name of registrant as specified in its charter)

Delaware		001-40237		85-1726310
(State or other jurisdiction of incorporation)		(Commission File Number)		(IRS Employer Identification No.)

4800 Montgomery Lane, Suite 220

Bethesda, Maryland 20814

(Address of principal executive offices) (Zip Code)

(301) 500-1556

(Registrant’s telephone number, including area code)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR§230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2).

Emerging growth company x

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.0001	GANX	The Nasdaq Stock Market LLC

Item 8.01 Other Information

On October 6, 2025, Gain Therapeutics, Inc. (the “Company”) presented preliminary data from the Company’s ongoing Phase 1b clinical study of GT-02287 in people with Parkinson’s disease (“PD”) with or without a GBA1 mutation at the International Congress of Parkinson’s Disease and Movement Disorders.

Preliminary Findings

After 90 days of dosing, GT-02287 has been safe and generally well tolerated, with no treatment-emergent serious adverse events observed. A transient increase in alkaline phosphatase and other liver enzymes has been observed in some participants and normalized despite ongoing dosing. Of the 21 study participants, two are treatment-naïve, two are on deep brain stimulation, and 18 are on levodopa and/or dopamine agonists or other PD drugs.

Mean MDS-UPDRS scores at baseline were 5.8, 7.4, and 24.7 for Parts I, II, and III, respectively. 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 the preclinical models in vivo and the proposed mechanism of action of GT-02287, supporting continued development. The plasma pharmacokinetics (PK) profile was consistent across all 14 participants sampled, was within the projected therapeutics range, and comparable to exposures observed in healthy volunteers in the Phase 1 study.

At two different meetings, the data monitoring committee has recommended continuation of the study and more recently, Australian health authorities have approved the Phase 1b study extension for patients who can now be treated for up to 12 months.

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	GAIN THERAPEUTICS, INC.
Dated: October 6, 2025	By:	/s/ Gene Mack
	Name: Gene Mack
	Title: Chief Executive Officer

FAQ

What did Gain Therapeutics (GANX) report about GT-02287 in Parkinson’s disease?

Gain Therapeutics reported preliminary Phase 1b data for GT-02287 in people with Parkinson’s disease, indicating the drug has been safe and generally well tolerated over 90 days with no treatment-emergent serious adverse events and showing exploratory improvements in movement and daily living scores.

How safe was GT-02287 in the Phase 1b Parkinson’s study for GANX?

GT-02287 was described as safe and generally well tolerated after 90 days of dosing, with no treatment-emergent serious adverse events. Some participants had transient increases in alkaline phosphatase and other liver enzymes, which normalized despite continued treatment.

Did Gain Therapeutics (GANX) see any signs of efficacy from GT-02287?

Several participants experienced improvements in MDS-UPDRS Part II and III scores after 90 days of GT-02287 treatment, while mean Part I scores remained unchanged. The company notes that mean improvement in Parts II and III at Day 90, not seen at Day 30, suggests a potential disease-slowing effect consistent with preclinical models.

How many patients were included in Gain Therapeutics’ Phase 1b GT-02287 study?

The preliminary data cover 21 participants with Parkinson’s disease, including treatment-naïve individuals, patients on deep brain stimulation, and others on levodopa and dopamine agonists or other Parkinson’s disease drugs.

What did regulators and monitors decide about continuing the GT-02287 trial for GANX?

The data monitoring committee has twice recommended continuation of the Phase 1b study, and Australian health authorities have approved an extension of the study to allow patients to be treated with GT-02287 for up to 12 months.

What did Gain Therapeutics say about GT-02287 pharmacokinetics in the Phase 1b study?

The plasma pharmacokinetic profile of GT-02287 was consistent across the 14 participants sampled, was within the projected therapeutic range, and was comparable to exposures observed in healthy volunteers in the earlier Phase 1 study.