subpage banner


Gain Therapeutics Announces Dosing of First Two Subjects in Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease

First small molecule allosteric binder discovered with Gain’s computational drug discovery platform enters clinical development

BETHESDA, Md., Oct. 04, 2023 (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 announced dosing of the first two subjects in a Phase 1 clinical trial of GT-02287, Gain’s lead drug candidate for the treatment of GBA1 Parkinson’s disease. The Company expects to complete this trial in the first half of 2024.

“Initiating first-in-human dosing with GT-02287 is an important milestone for Gain as we enter a new era as a clinical-stage company,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics. “I am very proud of the work accomplished by the entire Gain team to get us to this stage today, and we are eager to advance our understanding of the safety, tolerability and effect of GT-02287 in humans. This represents another major step forward toward providing a treatment for Parkinson’s patients and their families impacted by this devastating disease.”

Compelling preclinical data demonstrated that GT-02287 can restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and dysfunctional due to a GBA1 gene mutation, the most common genetic risk factor for the development of Parkinson’s disease. Restoring GCase function with GT-02287 was shown to have profound effect in animal models of Parkinson’s disease on the entire disease cascade, including a neuroprotective effect on dopaminergic neurons and improvement of motor deficiencies. Based on these data, GT-02287 has the potential to slow or even stop the progression of Parkinson’s disease.

“Today marks an important step for Gain in the journey to bring a novel, potentially disease-modifying therapy to patients for whom only symptom-focused therapeutics exist,” said Dr. Robin Ely MD, Director, Integrative and Regenerative Medicine; Founder, National Gaucher Foundation; and Clinical-Scientific Consultant to NGF Global Diagnostic and Treatment Initiative. “If GT-02287 proves successful in disrupting the disease process in GBA1 Parkinson’s, its fundamental mechanism of action could play a crucial role in addressing various neurodegenerative diseases, including Gaucher, idiopathic Parkinson’s, dementia with Lewy bodies, and Alzheimer’s disease.”

The Phase 1 clinical trial is a single center, randomized, double-blind, placebo-controlled, single- and multiple-ascending dose (SAD/MAD) study to evaluate the safety and tolerability of GT-02287 administered orally once daily in healthy adults. The secondary objective is to evaluate the pharmacokinetics of SAD and MAD dose levels to identify a maximum tolerated dose (MTD) and identify recommended doses for further clinical development in the setting of GBA1 Parkinson’s disease. In addition, as an exploratory endpoint, this study will look at GCase target engagement and activity in blood, which may provide an early clinical validation of the effect of GT-02287 on GCase.

Gain’s broad pipeline of novel allosteric therapies, including GT-02287, was discovered via the Company’s SEE-Tx® drug discovery platform. Designed to leverage AI-supported 3D structural biology and supercomputer-powered proprietary physics-based models, Gain is exploiting the untapped opportunities of allosteric binding sites and allosteric modulators to treat disease.

About GT-02287

Gain Therapeutics’ lead drug candidate, GT-02287, is in development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to a GBA1 gene mutation, the most common genetic abnormality associated with PD. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.

The program has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF), The Silverstein Foundation for Parkinson’s with GBA, and InnoSuisse.

About GBA1 Parkinson’s Disease

GBA1 Parkinson’s disease is caused by mutations in the GBA1 gene, found in up to 15% of patients with Parkinson’s disease and making it the primary genetic risk factor. The mutation causes dysfunctional misfolding of the lysosomal enzyme glucocerebrosidase (GCase), reducing its activity in the brain and leading to the subsequent accumulation of α-synuclein and neurodegeneration of dopamine cells. Patients with GBA1-PD tend to have earlier onset and faster symptom progression than sporadic PD, a progressive neurodegenerative disease characterized by a motor syndrome consisting of bradykinesia (slowness of movement), rigidity, resting tremors, and postural instability. With current therapies treating only the symptoms of Parkinson’s disease without affecting the underlying progression of the disease, there is an unmet need to develop novel disease-modifying therapies such as GT-02287 that have the potential to slow or stop disease progression and help improve outcomes in this patient population.

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287, in development for the treatment of GBA1 Parkinson’s disease, is currently being evaluated in a Phase 1 clinical trial.

Leveraging AI-supported structural biology, proprietary algorithms and supercomputer-powered physics-based models, the company’s SEE-Tx® discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. For more information, please visit and follow us on LinkedIn.

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical facts are “forward-looking statements”. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "goal, " "intend," "seek, " "potential" or "continue," the negative of these terms and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1 clinical study for GT-02287; and the potential therapeutic and clinical benefits of the Company’s product candidates. These forward-looking statements are based on the Company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s preclinical and future clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. Many factors may cause differences between current expectations and actual results, including the impacts of the post-COVID-19 environment and other global and macroeconomic conditions on the Company’s business; clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the section titled “Risk Factors,” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 23, 2023 and its other documents subsequently filed with or furnished to the Securities and Exchange Commission from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Investor & Media Contact:

Susan Sharpe
Linnden Communications
(919) 602-2330
This email address is being protected from spambots. You need JavaScript enabled to view it.