Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease

BETHESDA, Md., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (Nasdaq: GANX), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, announces preclinical data demonstrating that its clinical-stage GCase regulator GT-02287 provided neuroprotection and restored motor function in Parkinson’s disease models following delayed administration. The data was accepted as a late-breaker abstract and will be presented at the 20^th Annual WORLDSymposium™ being held in San Diego this week.

“We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer. “We are currently conducting a Phase 1 clinical trial of GT-02287 in healthy adults to evaluate its safety, tolerability, and pharmacokinetics, and plan to commence treatment of patients in an extension of that clinical trial in Q3 of this year.”

The preclinical study showed that GT-02287 restored motor function in a mouse model, even following a delayed administration of the drug candidate after the initial toxic insult mimicking the effects of GBA1 Parkinson’s disease. Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14 to day 27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.

Rescue of locomotor impairment was reflected in the complete reversal of plasma levels of Neurofilament Light Chain (NfL) to the level of the control arm in the study, which also suggests a neuroprotective effect of GT-02287. The emerging neurodegeneration biomarker NfL was previously accepted by the U.S. Food and Drug Administration (“FDA”) for the accelerated approval of tofersen for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS) and was recommended by the FDA as an exploratory endpoint for neuronopathic mucopolysaccharidosis II (MPS II) clinical trials.

Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed .

About GT-02287

Gain Therapeutics’ lead drug candidate, GT-02287, currently being evaluated in a Phase 1 clinical trial, has the potential to be a disease-modifying treatment of GBA1 Parkinson’s disease (GBA1-PD) and other neurodegenerative diseases. The orally administered small molecule that crosses the blood-brain barrier is an allosteric protein modulator that restores the function of the lysosomal protein glucocerebrosidase (GCase). In GBA1-PD patients, this enzyme 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 and reduced aggregated α-synuclein, neuroinflammation and neuronal death, and caused increased striatal dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker of 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 degeneration of dopamine-producing neuronal 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 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 Magellan™ discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Magellan is the next generation of Gain’s original SEE-Tx^® (Site-Directed Enzyme Enhancement Therapy) platform, which was enhanced and expanded with new AI and machine-learning tools and virtual screening capabilities to access the emerging on-demand compound libraries covering vast chemical spaces of over 50 billion compounds.

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.

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