Gain Therapeutics Progresses Krabbe Disease Program and Provides Scientific Update

Source:  Gain Therapeutics, Inc. 12/7/2021

Data Show Statistically Significant Increases in GALC Enzyme and Depletion of Toxic Substrate Psychosine

Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced a scientific update on the company’s Krabbe disease program. Gain is developing allosteric regulators to stabilize the galactosylceramidase (GALC) enzyme and reduce psychosine disease causing toxic substrate, potentially providing the first treatment option to patients with this devastating disease.

“These data provide additional validation of our approach in treating lysosomal storage diseases,” said Eric Richman, Chief Executive Officer of Gain. “We are encouraged by these early results and look forward to further studies and moving the program forward for Krabbe disease patients who currently have no or very limited therapeutic options.”

Krabbe disease, also known as globoid cell leukodystrophy, is a genetic disease that affects the central and peripheral nervous systems. It is caused by mutations in GALC, the gene that encodes the GALC enzyme necessary for the breakdown of several galactosyl-sphingolipids, particularly galactosylceramide and psychosine. These mutations cause misfolding and dysfunction of the GALC enzyme resulting in an accumulation of psychosine, which is highly toxic to all cell types and leads to loss of myelin (demyelination) and progressive damage to nerves in both the brain and peripheral tissues. GALC mutations are also implicated in numerous neurodegenerative disorders and is a dominant risk factor for multiple sclerosis.

In collaboration with Dr. Ernesto R. Bongarzone, Ph.D., Professor in Neuroscience at the College of Medicine at the University of Illinois, Chicago, initial compounds from this program were tested in mouse glial cultures and cell lines with relevant GALC mutations to measure their effect on GALC enzymatic activity and psychosine levels.

“The tested compounds showed significant reduction in psychosine levels in glial cultures,” said Dr. Bongarzone, Ph.D. “This potential to reduce psychosine is an exciting finding that warrants further research.”

The next phase of Gain’s program for Krabbe disease will include further evaluation of compounds in induced pluripotent stem cells, and additional evaluation of GALC activity and intracellular psychosine levels in more complex in vitro models.

About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It 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, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse.