Gain Therapeutics Awarded CHF 2.5 million Innosuisse Grant to Advance Lead Program in GBA1 Parkinson’s Disease
May 03 2023 - 9:15AM
Gain Therapeutics, Inc. (Nasdaq: GANX), a biotechnology company
leading the discovery and development of allosteric small molecule
therapies, today announced that Innosuisse, the Swiss Innovation
Agency, supports GT Gain Therapeutics SA with the amount of CHF 2.5
million (~$2.8 million) to develop GT-02287, Gain’s lead program
for GBA1 Parkinson’s disease.
The Company’s Swiss Accelerator innovation
project supported by Innosuisse entitled “Harnessing Structurally
Targeted Allosteric Regulators to treat Parkinson’s Disease”
includes clinical pharmacology and preclinical studies with the
Company’s drug candidate GT-02287, which will be conducted as the
lead program progresses through Phase 1 and Phase 2 clinical
studies in GBA1 Parkinson’s disease.
GBA1 mutations are the major genetic risk factor
for Parkinson’s disease and are associated with earlier onset of
the disease, faster disease progression and increased rates of
cognitive decline. Mutations of the GBA1 gene cause misfolding and
dysfunction of the enzyme beta-glucocerebrosidase (GCase).
Preclinical models of GT-02287 have shown the ability of this
orally bioavailable and brain-penetrant molecule to target and bind
to GCase, prevent its degradation and allow its transport to the
lysosomes where the enzyme can carry out its biological function.
Furthermore, the data from preclinical models demonstrate that
enhancement of lysosomal GCase activity by GT-02287 protects
against key pathological features of Parkinson’s disease, including
alpha-synuclein related pathology and an increase in the survival
of dopaminergic neurons. These findings support the potential of
GT-02287 to slow or halt disease progression in GBA1 Parkinson’s
disease.
“We are grateful for the continued support from
Innosuisse, which speaks to the wealth of promising data we have
generated to date and the disease-modifying potential of our lead
program for patients suffering from GBA1 Parkinson’s disease and
other neurodegenerative disorders,” said Matthias Alder, Chief
Executive Officer of Gain Therapeutics. “This funding provides
significant financial support for the further development of
GT-02287 as we remain on track to submit the dossier for the start
of the Phase 1 clinical study in mid-2023.”
Dr. Manolo Bellotto, Chief Strategy Officer and
General Manager at Gain Therapeutics added: “The Swiss Accelerator
program under which we are supported was a highly competitive
process, and we are pleased with the validation of our science and
data that is reflected in the decision of Innosuisse to support the
program. This latest funding adds to a CHF 1.5 million funding
received from Innosuisse in 2021 for earlier research of allosteric
modulators for GBA1-related diseases, and we look forward to
continuing to build on this work and advance the development of a
treatment for this devastating disease.”
Innosuisse launched a call for projects for the
Swiss Accelerator in 2022 to provide funding to Swiss companies
with novel products and significant innovation potential. Three
independent experts evaluated 752 projects, of which 53 were
selected after a three-stage assessment process. Support per
project amounts to a maximum of CHF 2.5 million and covers up to 70
per cent of project costs.
About GT-02287GT-02287 is an
orally administered, brain-penetrant allosteric small molecule
modulator of beta-glucocerebrosidase (GCase), an enzyme expressed
by the GBA1 gene and responsible for degrading toxic
glycosphingolipid substrates glucosylceramide (GlcCer) and
glucosylsphingosine (GlcSph) in the lysosome. Mutations in the GBA1
gene cause GCase to be misfolded and dysfunctional, which results
in lysosomal build-up of its glycosphingolipid substrates. This in
turn causes the accumulation of aggregated α-synuclein, the
pathological hallmark of Parkinson’s disease, in dopaminergic and
other neuronal types in vulnerable brain regions. GT-02287 binds to
GCase, allowing it to fold correctly, and restores enzyme function.
We have shown in vitro and in vivo that GT-02287 increases GCase
levels, activity and its transport to the lysosomes, where it is
able to eliminate toxic lipid substrates and prevent the build-up
of toxic forms of alpha-synuclein in dopaminergic neurons. In
animal models of Parkinson’s disease, GT-02287 additionally showed
a reduction in neuroinflammation and improved the survival of
dopaminergic neurons in relevant brain regions, leading to an
increase in dopamine levels and an improvement of locomotor
function.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is leading the discovery and development of
allosteric small molecule therapies. The ability to identify
never-seen-before allosteric targets on proteins involved in
diseases across the full spectrum of therapeutic areas provides
opportunities for a range of drug-protein interactions, including
protein stabilization, protein destabilization, targeted protein
degradation, allosteric inhibition, and allosteric activation.
Gain’s pipeline spans neurodegenerative diseases, lysosomal storage
disorders, metabolic disorders, as well as other diseases that can
be targeted through protein degradation, such as oncology. Gain’s
lead program in Parkinson’s disease has been awarded funding
support from The Michael J. Fox Foundation for Parkinson’s Research
(MJFF) and The Silverstein Foundation for Parkinson’s with GBA and
it is a Swiss Accelerator innovation project supported by
Innosuisse. For more information, please visit
https://www.gaintherapeutics.com.
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for reporting data from ongoing preclinical studies or the
initiation of future clinical trials, including the timing for
completion of IND-enabling toxicology studies and submission of the
dossier requirement for commencement of a Phase 1 clinical program
for GT-02287 for GBA1 Parkinson’s disease and the potential
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Investor & Media Contact:Argot PartnersNoor
Pahlavi(212) 600-1902Gain@argotpartners.com
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