Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain,” or the “Company”),
a biotechnology company leading the discovery and development of
allosteric small molecule therapies, today releases the following
letter to stockholders from its President and Chief Executive
Officer Matthias Alder.
Dear Fellow Stockholders,
With the start of a new year, I want to take a
moment to reflect on Gain’s progress over the last 12 months and to
share our strategy and plans for 2024.
Research and development of pharmaceutical
products for the treatment of neurodegenerative diseases has seen
tremendous progress in recent years, with the approval of new drugs
for the treatment of Alzheimer’s disease, multiple sclerosis and
ALS. The pharmaceutical industry is taking note as evidenced by the
recent acquisition of Cerevel Therapeutics for $8.7 billion by
AbbVie, and the acquisition of preclinical-stage Caraway
Therapeutics by Merck with an announced deal value of $610
million.
In Parkinson’s disease, the second most common
neurodegenerative disease after Alzheimer’s disease, new insights
into the root cause of the disease have led to a number of drug
candidates advancing in clinical development, but there are no
approved therapies available to patients that can slow or stop the
progression of the disease. At Gain, we seek to address this high
unmet medical need with our clinical-stage lead drug candidate
GT-02287, which we believe has disease-modifying potential for the
treatment of Parkinson’s disease and other neurodegenerative
diseases.
Successful Transition to a
Clinical-Stage Biotech Company
In 2023, we advanced GT-02287 through
preclinical development and initiated the company’s first clinical
trial in September 2023 on time and on plan. The Phase 1 clinical
trial is a single center, randomized, double-blind,
placebo-controlled, single-ascending dose (SAD) and
multiple-ascending dose (MAD) study to evaluate the safety and
tolerability of GT-02287 administered orally in healthy adults. The
dose escalation of the SAD phase is underway, and the MAD phase of
the study is expected to begin in Q1 2024. The SAD/MAD part of the
Phase 1 clinical trial is expected to be completed by mid-2024 with
results expected to be reported in the second half of the year.
In Q3 2024, we plan to start treatment of a
cohort of 12-15 Parkinson’s patients as an extension of the ongoing
Phase 1 clinical trial to establish a biomarker-based clinical
proof of concept, replicating the effects of GT-02287 on biomarkers
we have observed in our preclinical in vivo studies. We expect data
from this study to be achieved in late 2024 or early 2025 ahead of
starting a Phase 2 clinical trial in the first half of 2025.
Cutting-Edge Science and Best-in-Class
Preclinical Data Package
In 2023, we made several data presentations of
results of our GBA1 program in preclinical models of Parkinson’s
disease and Alzheimer’s disease. For example, we presented
new data demonstrating a reduction of the plasma neurodegeneration
biomarker NfL after administration of GT-02287 in a GBA1
Parkinson’s disease model at the International Congress of
Parkinson's Disease and Movement Disorders® last August. NfL is an
emerging biomarker of neurodegeneration that was recently accepted
by the FDA as a surrogate endpoint in the agency’s accelerated
approval of a drug for the treatment of certain ALS patients.
In that same preclinical model, GT-02287 also restored
β-glucocerebrosidase (GCase) enzymatic function, reduced aggregated
α-synuclein, neuroinflammation and neuronal death, increased
dopamine levels and improved motor function.
We presented additional data on our allosteric
GCase modulators at the March 2023 International Conference on
Alzheimer’s and Parkinson’s Diseases. In that poster presentation,
we showed results that we believe support the disease-modifying
potential of allosteric GCase regulators for the treatment of
Alzheimer’s disease. Finally, we presented results of a preclinical
model that we believe support the disease-modifying potential of a
GCase-targeting small molecule for neuronopathic Gaucher disease at
last year’s 19th Annual WORLDSymposium.™ The data generated in an
animal model of neuronopathic Gaucher disease show that GT-02329
restores GCase activity, depletes accumulation of toxic lipid
substrates, reduces neuroinflammation, and improves neuromuscular
function.
Additionally, we published data in PLOS ONE on
our allosteric small molecule modulators of the GLB1 enzyme showing
significantly restored β-Gal function and reduced intracellular
toxic substrates as part of our program for GM1 gangliosidosis.
These results were generated in collaboration with the Institute
for Research in Biomedicine in Bellinzona, Switzerland.
In 2024, we expect to present further data on
GT-02287 from preclinical models at scientific conferences,
starting with the 20th Annual WORLDSymposium™ being held February
4-9, 2024, in San Diego, CA. At that conference, we will present
new in vitro and in vivo data that were accepted as a late-breaker
abstract titled, “GT-02287, a clinical stage GCase enhancer,
displays neuroprotection and restores motor function in preclinical
models of Parkinson’s disease following delayed administration.” In
addition, the abstract was selected for an oral presentation at the
conference on February 9, 2024.
Based on the preclinical data we have generated
with GT-02287 across numerous in vitro and in vivo models of
Parkinson’s disease, we believe that GT-02287 has a best-in-class
preclinical profile with evidence of improving the entire disease
cascade caused by GCase dysfunction and offering the potential to
slow or even stop the progression of this devastating
neurodegenerative disease.
Magellan™ Platform
During the course of 2023, we significantly
upgraded our computational drug discovery platform. The platform
now provides for an integrated, efficient workflow of existing and
newly added tools and programming technologies that enable the
identification of new binding sites on proteins and screening of
the 50+ billion chemical spaces that have become available through
providers like Enamine. Over the last 18 months, we also expanded
the application of the platform beyond its original focus of
enhancing and restoring enzyme function to new modalities that
disrupt the function of target proteins.
Based on the added capabilities and expanded
application, we feel that the original platform name SEE-Tx
(Site-specific Enzyme Enhancement Therapy) no longer adequately
represents our platform technology. Magellan, the name of our
next-generation platform, reflects its enhanced capabilities that
enable the exploration of new protein biology and a vast chemical
space to discover new allosteric small molecule therapies. Based on
the unique combination of physics-based models and integrated
AI/ML-enhanced virtual screening capabilities, we believe Magellan
is highly differentiated in the field of computational drug
discovery platforms and positions us to continue building our own
pipeline of potential first-in-class drug candidates. In addition,
we are excited about the prospect of deploying Magellan in drug
discovery collaborations with academic institutions, biotechnology
companies, CROs, and pharma companies, and will provide updates on
our progress in these efforts throughout 2024.
Cash Runway into 2025
In late November 2023, we completed a CMPO/PIPE
financing that raised gross proceeds of $10.1 million. Based on our
operational plans, we are now positioned to fund our operations
into 2025, which we expect will enable the achievement of a
biomarker-based clinical proof of concept of GT-02287 in the
previously mentioned Phase 1 patient cohort. In addition, we
received grants totaling approximately $3.4 million during the year
to fund R&D activities for our pipeline programs. In 2024, we
plan to continue tapping into grant opportunities to provide
non-dilutive funding for our pipeline programs.
Strategic Partnering and Business
Development
In 2023, we continued our active engagement with
potential industry partners for our pipeline programs and platform
technology. Following the J.P. Morgan Healthcare conference in
early January, I am optimistic about our strategic partnering
efforts, and we will continue to pursue opportunities for one or
more of our R&D assets, whether for our lead drug candidate
GT-02287, our earlier pipeline programs, or the Magellan platform
or a combination thereof. We believe that potential transactions
for any of our assets could provide significant non-dilutive
funding and/or increase the inherent value of Gain by advancing
partnered programs through value inflection points.
Organizational Development
With the transition from a discovery and
research-stage company to a clinical-stage biotech company, we are
continuously evaluating the organization and adding new skills
required to achieve our goals in the next 2-3 years while
maintaining our focus on prudent cash management. Our new status as
a clinical company has necessitated the search for a Chief Medical
Officer who will be charged with advancing the development of the
clinical strategy for GT-02287 and leading the clinical development
team and related functions at Gain. We look forward to updating you
once we have secured the appropriate candidate. Also, individual
career objectives can lead to changes in organizations.
Unfortunately, we are saying goodbye to Dr. Xavi Barril, our
current Chief Technology Officer, who has been instrumental in
guiding the evolution of our computational drug discovery platform.
Xavi is leaving the world of academia as a professor at the
University of Barcelona and his part-time engagement with Gain to
join a major pharmaceutical company. As we wish him luck, the
Magellan platform remains in good hands with an experienced team
led by Dr. Elena Cubero, who has been working with Xavi and the
platform over the last 10 years.
Outlook for 2024
In 2024, we expect to achieve a series of
important value inflection points with Gain, which are summarized
below:
Q1 2024:
- Poster and
Platform Presentation of new data in preclinical model of
Parkinson’s disease at WORLDSymposium
- Start of MAD
cohort in Phase 1 clinical trial with GT-02287
Q2 2024
- Completion of
MAD cohort of Phase 1 clinical trial; potential for showing effect
on GCase levels and activation
Q3 2024
- Start of
Parkinson’s patient cohort in Phase 1 clinical trial
Q4 2024 / Q1 2025
- Completion of
patient cohort; potential to show biomarker-based clinical proof of
concept
I look forward to updating you as we progress
Gain’s business and programs throughout this year and thank you for
your continued support along the way to providing new treatments
for debilitating neurodegenerative diseases.
Sincerely,
Matthias AlderPresident and CEOGain
Therapeutics, Inc.
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; the potential therapeutic and clinical benefits of the
Company’s product candidates including GT-02287; and the execution
of potential business development transactions and the effects
thereof on the financial position of the Company. 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.
Contacts:
CORE IR(516)
222-2560 ir@gaintherapeutics.com
Gain Therapeutics (NASDAQ:GANX)
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