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 the publication of preclinical data identifying a novel
class of small molecule allosteric regulators that demonstrate
therapeutic potential for galactosidase beta 1 (GLB1)-related
lysosomal storage disorders (LSDs), including GM1 gangliosidosis.
The study, “Validation of a highly sensitive HaloTag-based assay to
evaluate the potency of a novel class of allosteric β-Galactosidase
correctors,” was published in PLOS ONE.
GM1-gangliosidosis is an inherited, progressive
disorder characterized by the degeneration of brain and spinal cord
cells, leading to muscle weakness, skeletal abnormalities,
dystonia, and vision problems. Mutations in the GLB1 gene
significantly reduce the activity and function of the lysosomal
hydrolase enzyme β-galactosidase (β-Gal) due to protein
misfolding.
Depending on the age of onset, which can occur
during infancy and up into adulthood, life expectancy of patients
can be significantly shortened. Additionally, there are no
available disease-modifying treatments for GM1 gangliosidosis, with
available therapies only focused on reducing symptoms and improving
quality of life. To enhance patient outcomes, there is a
significant need to bring safe and effective treatments into the
clinic that can alter progression of the disorder.
“Lysosomal storage disorders are difficult to
treat with traditional pharmacological interventions because most
approaches cause an inhibitory effect on the lysosomal enzyme by
acting on the active site. The small allosteric regulators that we
are characterizing correct protein misfolding by binding the
mutated proteins outside the active site, without interfering with
enzymatic activity,” said Maurizio Molinari, PhD, Protein Folding
and Quality Control Group Leader, Institute for Research in
Biomedicine in Switzerland and senior author on the paper. “This
impressive data demonstrates the potential of allosteric modulators
as a therapeutic option for hard-to-treat, genetic diseases where
there are no effective approaches for disease modification.”
In the PLOS ONE study, scientists from Gain used
Gain’s proprietary Site-directed Enzyme Enhancement Therapy
(SEE-Tx) technology followed by molecular interaction and ligand
binding studies to identify and characterize two structurally
targeted allosteric regulators of β-Gal. A biochemical HaloTag
cleavage assay was developed by the researchers from the Institute
for Research in Biomedicine and utilized to measure lysosomal
delivery capabilities of β-Gal. Results showed that the two
compounds (GT-00513 and GT-00413) stabilized, enhanced and restored
the key biological lysosomal transport functions of β-Gal and
reduced the levels of the intracellular toxic substrate, GM1
ganglioside, in GM1 gangliosidosis patient-derived cells.
“Restoring the function of misfolded proteins is
a therapeutic approach that is accessible only through allosteric
small molecule regulators that can be efficiently discovered with
our pioneering computational drug discovery platform,” said Manolo
Bellotto, Chief Strategy Officer and General Manager of Gain
Therapeutics and co-author on the paper. “This approach has been
validated with our clinical-stage lead drug candidate GT-02287,
which restores the function of glucocerebrosidase for the treatment
of Parkinson’s disease and repairs the disease cascade triggered by
protein misfolding.”
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.
About Institute for Research in
Biomedicine (IRB, Bellinzona), an institute affiliated to
Università della Svizzera italiana (USI)
Founded in 2000 in Bellinzona, Switzerland, the
Institute for Research in Biomedicine (IRB) hosts 13 research
groups studying infectious, inflammatory, tumors and rare diseases
in order to identify new therapeutic strategies. As a result of its
activities, the IRB has gained considerable international
recognition in the fields of immunology, cell and structural
biology and DNA repair. Molinari’s laboratory investigates the
molecular mechanisms regulating chaperone-assisted protein folding
and the quality control processes determining whether a polypeptide
should be transported at the intra- or extra-cellular site of
activity, or if it should be selected for degradation. Emphasis is
given on establishing causes of diseases resulting from misfolding
of mutant gene products such as α1-antitrypsin deficiency and
lysosomal storage disorders and to explore novel therapeutic
approaches to alleviate disease phenotypes. IRB offers training
opportunities for young researchers at all levels. This is made
possible through collaborations with Swiss and foreign
universities. Since its opening, IRB has trained more than 1210 PhD
students who have gone on to careers in academia or industry both
in Switzerland and abroad. In 2021, IRB and IOR (Institute of
Oncology Research) formed the Bios+ (Bellinzona Institutes of
Science) association, with the mission to create new synergies and
to promote and coordinate the scientific research and teaching
activities of the two institutes. The vision of Bios+ is to promote
the growth of a biomedical research center in Italian-speaking
Switzerland. For more information, please visit www.irb.usi.ch.
About the research group of Prof.
Molinari (IRB, Bellinzona)
The Protein Folding and Quality Control group
lead by Prof. Molinari was established at the IRB in 2000. The
group is dissecting the mechanisms that regulate the
chaperone-assisted production of correctly folded proteins in the
Endoplasmic Reticulum (ER), and the quality control events that
select misfolded proteins for proteasome-driven ER-Associated
Degradation (ERAD), or for lysosome-driven
ER-to-Lysosome-Associated Degradation (ERLAD). Studies are ongoing
to characterize the unfolded protein responses (UPR) and the
ER-phagy pathways that operate in mammalian cells to ensure the
adaptation of ER size and activity to the cellular needs.
A thorough knowledge of the processes that
maintain ER function is instrumental to identify drug targets and
to design and assess therapies for human diseases elicited by
pathogens or linked to aberrant ER homeostasis resulting from
expression of defective gene products (e.g., rare genetic disorders
such as alpha1-antitrypsin deficiency or lysosomal storage
disorders). For more information, please visit Lab Molinari, X and
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
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