Gain Therapeutics Announces the Initiation of the Multiple Ascending Dose (MAD) Part of the Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease
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Insights
GT-02287's progression into the Multiple Ascending Dose (MAD) stage of Phase 1 clinical trials signifies an important milestone for Gain Therapeutics, particularly in the context of developing treatments for GBA1 Parkinson’s disease. The transition from Single Ascending Dose (SAD) to MAD cohorts, sanctioned by the Bellberry Human Research Ethics Committee (HREC) in Australia, reflects a positive safety and tolerability profile which is a critical factor in the drug development process. This progression not only demonstrates the potential viability of GT-02287 but also reinforces the company's adherence to the projected clinical trial timeline.
From a medical research perspective, the therapeutic target of GT-02287, the lysosomal enzyme glucocerebrosidase (GCase), is significant. GCase dysfunction due to GBA1 gene mutations is a well-established risk factor for Parkinson’s disease. The restoration of GCase function could, therefore, be a transformative approach in the treatment of this neurodegenerative disorder. The preclinical data suggesting complete restoration of motor function and normalization of plasma NfL levels, a biomarker associated with neurodegeneration, point towards GT-02287's potential to modify disease progression rather than merely alleviating symptoms.
The initiation of the MAD part of Phase 1 clinical trials for GT-02287 could have significant financial implications for Gain Therapeutics. The positive preclinical data and subsequent approval to proceed by HREC enhance investor confidence, potentially impacting the company's stock valuation. For biotechnology firms, especially those in the clinical stage like Gain Therapeutics, successful trial milestones are critical value drivers. They not only attract potential partnerships or funding opportunities but also pave the way for future commercialization prospects.
However, it's important for investors to consider the inherent risks and volatility associated with clinical-stage biotech investments. The long-term success of GT-02287 will depend on continued positive results in subsequent trial phases and ultimately regulatory approval. It's also worth noting that the biotech sector is highly competitive and the success of new drug candidates is uncertain until all clinical trials are completed and regulatory hurdles are cleared.
In the broader context of the pharmaceutical industry, the development of treatments for Parkinson’s disease represents a substantial market opportunity. The disease affects millions worldwide and the market is characterized by a high unmet medical need. The advancement of GT-02287 into later stages of clinical trials may position Gain Therapeutics favorably within this space. The uniqueness of GT-02287's mechanism, targeting the GBA1 mutation, differentiates it from other Parkinson's treatments and could capture a niche market segment if proven effective.
Moreover, the focus on the neurodegeneration biomarker NfL in the preclinical studies indicates a shift towards precision medicine in neurodegenerative diseases, which is a growing trend in the industry. The ability to demonstrate disease-modifying effects through biomarkers could not only enhance the drug's profile but also aid in the stratification of patient populations for targeted therapies. This could lead to more efficient clinical trials and a more personalized approach to treatment, which is increasingly valued in the medical community and by payers.
BETHESDA, Md., Feb. 27, 2024 (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, announces the initiation of the Multiple Ascending Dose (MAD) part of the Phase 1 clinical trial of GT-02287, Gain’s lead drug candidate for the treatment of GBA1 Parkinson’s disease.
Based on the safety and tolerability profile observed in the Single Ascending Dose (SAD) cohorts of the study, the Bellberry Human Research Ethics Committee (HREC) in Australia approved the start of the MAD part of the study in parallel with the completion of the last SAD cohorts. Following the HREC approval, administration of GT-02287 in the MAD part of the study has been initiated.
“Our Phase 1 clinical trial of GT-02287 remains on track and according to plan,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics. “We are pleased with the safety and tolerability profile observed in the SAD cohorts and expect to complete the MAD part of the Phase 1 clinical trial in Q2.”
GT-02287 has been shown to 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. Compelling preclinical data presented at WORLDSymposium™ earlier in February 2024 demonstrated that treatment with GT-02287 completely restored motor function and reduced plasma levels of the emerging neurodegeneration biomarker NfL back to normal levels. Based on these data, GT-02287 may have the potential to slow or even stop the progression of Parkinson’s 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
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 GainTherapeutics.com 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.
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