Gain Therapeutics Receives Approval to Commence Phase 1 Clinical Study of GT-02287 from the Human Research Ethics Committee (HREC) in Australia
September 12 2023 - 8:25AM
Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”),
a biotechnology company leading the discovery and development of
the next generation of allosteric small molecule therapies, today
announced that the Company has received approval from the Bellberry
Human Research Ethics Committee (HREC) in Australia to initiate a
Phase 1 clinical study of its lead drug candidate GT-02287 in
development for GBA1-Parkinson’s disease (GBA1-PD). Dosing in
healthy volunteers to assess the safety and pharmacokinetics of
GT-02287 is expected to begin in the near term.
“Following the recent presentation of encouraging preclinical
data at the International Congress of Parkinson's Disease and
Movement Disorders® showing the robust effect of GT-02287 against
PD-associated pathology and its ability to significantly decrease
plasma NfL levels, an emerging biomarker for neurodegeneration, we
are pleased to receive HREC’s approval to enable the advancement of
GT-02287 into the clinic,” said Matthias Alder, Chief Executive
Officer of Gain Therapeutics. “This approval is a key step for Gain
and further validates our differentiated allosteric approach and
our computational drug discovery platform as we endeavor to bring a
novel, potentially disease-modifying therapeutic to patients with
GBA1-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 available, 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) and The Silverstein
Foundation for Parkinson’s with GBA. A Phase 1 trial of GT-02287 is
scheduled to begin in the near term.
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 subsequent
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 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, has been approved for
clinical testing in Australia.
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 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 commencement of dosing of subjects in 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 SharpeLinnden Communications(919)
602-2330susan@linndencom.com
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