Full Press Release Details
Gain Therapeutics Presents Additional Clinical
and Biomarker Data from Phase 1b Clinical Study of GT-02287 and Preclinical Data from Novel Chemical Series at AD/PD 2026
Additional biomarker analysis reveals higher
CSF levels of DOPA decarboxylase (DDC - an emerging PD biomarker) at baseline with a reduction following 90 days of dosing with
GT-02287 in individuals with high baseline CSF glucosylsphingosine
Additional MDS-UPDRS data demonstrate continued
durability at day 150 of GT-02287 administration
Novel chemical series, headlined by advanced
lead GT-04686, is ready for IND-enabling studies for the treatment of Parkinson's disease and other neurological disorders
MD, March 18, 2026 -- Gain Therapeutics, Inc. (Nasdaq: GANX) ("Gain", or the "Company"),
a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies,
today announced the presentation of an oral presentation and poster at AD/PD 2026 International Conference on Alzheimer's
and Parkinson's Disease and Related Neurological Disorders, being held March 17-21, 2026, in Copenhagen, Denmark. The oral
presentation outlined new clinical and biomarker data from the Phase 1b clinical study of GT-02287 further supporting disease-modifying
potential in both idiopathic and GBA1 Parkinson's disease. The Company also presented a poster outlining preclinical data from a structurally
distinct chemical series of allosteric glucocerebrosidase (GCase) modulators, which are ready for IND-enabling studies for
the treatment of Parkinson's disease (PD) and other neurological disorders.
AD/PD Oral Presentation
The oral presentation, entitled, "An Open-Label Phase 1b
Study of GT-02287 in Parkinson's Disease," was delivered on-site by the Company's Chief Medical Officer, Jonas Hannestad,
M.D., Ph.D. Data on safety, tolerability, biomarkers, and clinical scores from the completed Part 1 of the study support continued
development of GT-02287 for PD. Of the 19 participants who completed dosing in Part 1, 16 chose to continue in the ongoing nine-month
extension (Part 2), further supporting the tolerability of GT-02287. A Data Monitoring Committee meeting on March 5, 2026, concluded
that the study should continue without any changes.
As previously presented in January 2026, in all participants with
elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased substantially after 90 days of
treatment with GT-02287. Elevated GluSph, a result of GCase dysfunction, has been shown to increase the aggregation of alpha synuclein
and to impair mitochondrial and lysosomal function in neurons.
As of March 10, 2026, of the 16 participants enrolled in the ongoing
nine-month extension, 14 had completed dosing for five months (Day 150). MDS-UPDRS scores remained stable over 150 days of dosing. Preliminary
data (subject to quality assurance) suggests that participants with elevated baseline levels of GluSph in CSF continued to benefit more
than those with low baseline levels of GluSph in CSF, with a difference of 6.7 points in the sum of MDS-UPDRS Part II and Part III
scores between the two groups at Day 150.
Additionally, in individuals with high levels of CSF GluSph at baseline,
levels of DOPA decarboxylase (DDC) decreased following 90 days of treatment with GT-02287. DDC is responsible for converting levodopa
into dopamine in the brain and peripheral nervous system and is elevated in the CSF of people with Parkinson's - likely due
to dopaminergic neuron dysfunction.
Gene Mack, President and CEO of Gain Therapeutics, commented on the
results, saying, "We are encouraged by the evolving biomarker evidence in the Phase 1b clinical study of GT-02287 in Parkinson's
disease that suggests GT-02287 is targeting the causative biology of PD. Building on our previously reported reduction of GluSph in CSF,
this additional data shows a correlation between decreased levels of GluSph, decreased levels of DDC, and improvements in MDS-UPDRS scores
associated with treatment with GT-02287." He continued, "Importantly, MDS-UPDRS scores remained stable and durable across
the overall study population after 150 days of treatment with GT-02287, which we view as an encouraging signal in this progressive neurological
disorder. The totality of the data continues to support the potential of GT-02287 in both idiopathic and GBA1 Parkinson's disease
and we hope to one day shift the treatment paradigm away from symptomatic relief and to disease modification."
Jonas Hannestad, Chief Medical Officer of Gain Therapeutics, stated,
"We are continuing to follow patients in the Phase 1b nine-month extension study that is expected to complete in September 2026
and look forward to presenting further data at scientific conferences throughout the balance of the year."
AD/PD Poster Presentation
The poster, titled, "Novel Allosteric GCase Modulators, Different
From the Clinical Stage GT-02287, for the Treatment of Parkinson's Disease," was presented on-site by the Company's
Head of Research Ana Maria Garcia-Collazo, Ph.D. The new, orally available, brain penetrant chemical series - led by GT-04686 -
demonstrates potential for future clinical development, as evidenced by restoration of key biological activities that are impaired in
Parkinson's disease.
These findings together support advancement of this novel chemical
series towards the clinic and the potential to be developed for PD and other indications where GCase deficiency is implicated.
"Our proprietary Magellan drug discovery platform has
yielded novel GCase allosteric modulators that have generated promising preclinical data in Parkinson's disease models. We look
forward to further development of these novel, structurally distinct compounds and continued deployment of Magellan to identify
leads targeting other neurodegenerative and rare diseases," said Joanne Taylor, Ph.D., Senior Vice President of Research at Gain.
PDF of the poster presented at AD/PD 2026 is available on the Science and Technology section of the Company's website at https://gaintherapeutics.com/science-and-technology/posters.
Gain Therapeutics' lead drug candidate, GT-02287, is in clinical
development for the treatment of Parkinson's disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant
small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes
misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related
In preclinical models of PD, GT-02287 restored GCase enzymatic function,
reduced ER stress, lysosomal and mitochondrial pathology, aggregated -synuclein, neuroinflammation and neuronal death, as well
as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD,
GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as
nesting. Compelling data in these models, demonstrating a disease-modifying effect of GT-02287, suggest that the drug candidate may have
the potential to slow or stop the progression of Parkinson's disease.
Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated
favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with an increase
in GCase activity among those receiving GT-02287 at clinically relevant doses.
GT-02287 is currently being evaluated in a Phase 1b clinical trial
for the treatment of Parkinson's disease with or without a GBA1 mutation. The primary endpoint of the trial, which enrolled participants
across seven sites in Australia, is to evaluate the safety and tolerability of GT-02287 after three months of dosing in people with Parkinson's
disease. The recently commenced Phase 1b study extension allows participants to continue to be treated with GT-02287 for up to a total
Initial results from the Phase 1b clinical trial in people with Parkinson's
disease demonstrated central nervous system target engagement, a reduction to baseline levels in the prespecified endpoint glucosylsphingosine
(GluSph), and improvement or stabilization in MDS-UPDRS scores.
Gain's lead program in Parkinson's disease has been awarded
funding support early in its development from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein
Foundation for Parkinson's with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon
2020 research and Innosuisse - Swiss Innovation Agency.
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 is currently being evaluated for the
treatment of Parkinson's disease with or without a GBA1 mutation in a Phase 1b clinical trial. GT-02287 has further potential in
Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. Gain has multiple undisclosed preclinical assets targeting
lysosomal storage disorders, metabolic diseases, and solid tumors.
Gain's unique approach enables the discovery of novel, allosteric
small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan 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.
This release contains "forward-looking statements" made pursuant to the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as "believes," "expects,"
"anticipates," "intends," "will," "may," "should," or similar expressions.
These forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future
performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance