Gain Therapeutics, Inc., (Nasdaq: GANX), a clinical-stage
biotechnology company leading the discovery and development of the
next generation of allosteric small molecule therapies, today
announced it is holding a 2024 Research & Development update on
recent in vivo data for GT-02287 along with valuable insights from
Key Opinion Leaders.
Event Details
“Beyond Symptomatic Treatment of
Parkinson’s Disease: Gain Therapeutics R&D Update with KOL
& Analyst Insights 2024”
Date: February 22nd, 2024
Time: 11:00 am - 12:00 pm EST
Register for the Event HERE.
Send any questions you’d like answered in our Q&A to
ir@gaintherapeutics.com.
Discussion Topics
GBA-associated Parkinson’s Disease & other
Synucleinopathies: current unmet medical needs and needed
therapies.
From biology to classification: understanding Parkinson’s
Disease.
Discussion of recent in vivo data for GT-02287 as a
best-in-class small molecule, for the treatment of GBA-Parkinson’s
Disease and other neurodegenerative diseases.
Speakers
Gain Therapeutics
Matthias Alder - Chief Executive
Officer
Joanne Taylor – Senior Vice
President, Research
Key Opinion Leaders
Professor Roy Alcalay
- Prof. Alcalay is Chief of the
Movement Disorders Division at Tel Aviv Sourasky Medical Center in
Tel Aviv, Israel. He also holds an associate professorship in
clinical neurology at Columbia University. His research focuses on
biomarkers and genetics in Parkinson’s Disease. He is a member of
The Michael J. Fox Foundation-funded Parkinson’s Progression
Markers Initiative (PPMI) executive steering committee, and the PI
of the Parkinson’s Foundation-funded PD GENEration study.
Professor Tiago Fleming Outeiro
- Prof. Outeiro is Professor and
Director of the Department of Experimental Neurodegeneration at the
University Medical Center Göttingen in Germany. He is also
Professor at Newcastle University. His research interests are
focused on the understanding of the molecular mechanisms of
neurodegenerative diseases such as Parkinson’s, Huntington’s, and
Alzheimer’s disease. He is Chair of the Task Force on the
Biological Definition of Parkinson’s Disease of Internation
Parkinson and Movement Disorder Society.
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 PartnersNic Johnson and Elio
Ambrosionic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(212)
845-4242
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