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
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.
Investor & Media
Contact:
Susan SharpeLinnden Communications(919)
602-2330susan@linndencom.com
Gain Therapeutics (NASDAQ:GANX)
過去 株価チャート
から 4 2024 まで 5 2024
Gain Therapeutics (NASDAQ:GANX)
過去 株価チャート
から 5 2023 まで 5 2024