Gain Therapeutics, Inc. (Nasdaq: GANX), a pioneering biotechnology company specializing in innovative allosteric small molecule therapies, has made significant strides in the fight against Parkinson’s disease (PD). The Company’s lead drug candidate, GT-02287, has demonstrated extraordinary potential in mitigating the effects of Parkinson’s disease across two distinct preclinical models of the ailment. These findings suggest that GT-02287 has the capacity to alleviate Parkinson’s disease pathology and enhance motor function.
A notable highlight is the substantial reduction of plasma Neurofilament Light Chain (NfL) levels, an emerging biomarker for neurodegeneration. This advancement was demonstrated in a mouse model of GBA1-PD, a discovery that could have far-reaching implications for understanding neurodegenerative conditions.
These groundbreaking findings were presented at the International Congress of Parkinson’s Disease and Movement Disorders®, held in Copenhagen, Denmark from August 27 to 31, 2023.
Dr. Joanne Taylor, Senior Vice President of Research at Gain, expressed her excitement, stating, “The data from these preclinical models highlight GT-02287’s potential as an oral allosteric regulator of GCase. The restoration of enzymatic function and reduction in Parkinson’s disease-related pathologies are promising indicators. Additionally, the significant reduction in plasma NfL levels, a key neurodegeneration biomarker, holds promise for future clinical trials.”
Of significant importance is the substantial decrease in plasma NfL levels observed in the GBA1-PD model, signifying a potential advancement in neurodegeneration assessment. The second poster, “GT-02287, a brain-penetrant structurally targeted allosteric regulator,” scheduled for August 30th, will delve into GT-02287’s impact on a PD animal model, showcasing its potential to restore protein function and counteract neurotoxic effects.
Matthias Alder, Chief Executive Officer of Gain Therapeutics, emphasized the significance of these results. He stated, “The data from both posters solidify the potential of GT-02287 to restore GCase protein function, shield against Parkinson’s disease’s neurological impact, and reduce neurodegeneration, as indicated by the emerging biomarker NfL. These compelling findings pave the way for Phase 1 clinical trials with GT-02287, marking a pivotal step towards advancing a potentially groundbreaking treatment for GBA1 Parkinson’s disease.” Gain Therapeutics’ achievements underscore its pivotal role in shaping the landscape of Parkinson’s disease research and treatment.
GAIN Therapeutics:
https://www.gaintherapeutics.com/
https://www.marketscreener.com/quote/stock/GAIN-THERAPEUTICS-INC-120780704/
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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 Company’s observations based upon the reported preclinical data; the Company’s expectations regarding the timing of commencement of a Phase 1 clinical program for GT-02287 for GBA1 Parkinson’s disease; and the potential therapeutic and clinical benefits of GT-02287 for the treatment of GBA1 Parkinson’s disease.
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 risk that observations from preclinical studies are not indicative or predictive of results in clinical trials; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; the impacts of the post-COVID-19 environment and other global and macroeconomic conditions on the Company’s business; the Company’s financial position; 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.
Source:
MDM Worldwide: https://www.mdmworldwide.com/
Contact: David Zazoff dzazoff@mdmworldwide.com
MDM Worldwide
264 W 40th St
New York NY 10018
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