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 20th 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 here.
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
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 trial for
GT-02287 and the treatment of Parkinson’s patients in that clinical
trial; and the potential therapeutic and clinical benefits of the
Company’s product candidates including GT-02287. 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 Contact:
CORE IR(516)
222-2560 ir@gaintherapeutics.com
Media Contacts:
Russo Partners Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(212)
845-4242
Gain Therapeutics (NASDAQ:GANX)
Historical Stock Chart
From Apr 2024 to May 2024
Gain Therapeutics (NASDAQ:GANX)
Historical Stock Chart
From May 2023 to May 2024