Gain Therapeutics, Inc.
- Market Cap (Float-Adj) (Float-adjusted market cap computed by the low-float screener — matches the screener row. May differ from the standard cap (price × all shares outstanding) reported elsewhere.)
- $72.9M
- Price
- $1.67
- % Change (Daily % change from the live per-ticker quote feed.)
- 0.00%
- Volume
- 373.9K
About
Gain Therapeutics, Inc. is a biotechnology firm specializing in the creation of advanced treatments for conditions originating from improperly folded proteins. The company primarily directs its efforts towards rare genetic disorders and neurological ailments. It employs its unique Site-Directed Enzyme Enhancement Therapy (SEE-Tx) platform to pinpoint specific allosteric sites on malformed proteins. Subsequently, Gain Therapeutics identifies proprietary small molecules designed to bind to these locations, thereby restoring correct protein conformation and alleviating the disease. Their current development pipeline includes several structurally targeted allosteric regulator candidates aimed at combating a range of diseases, such as Morquio B, GM1 gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1. Established in 2017, the company is headquartered in Bethesda, Maryland.
Quick stats
Shares outstanding43.6M
Computed float42.7M
Float / shares97.8%
Key info
SectorHealthcare
IndustryBiotechnology
CIK (Central Index Key — the unique ID the SEC assigns to every filer. Use it to look up the company's filings on EDGAR.)0001819411
AI Summary
AI-generated1/17
standardbullishclinical trial
Gain Therapeutics Presents Positive Phase 1b Open-Label Extension Data For Rexaceract Demonstrating Further Evidence of Disease-Modifying Activity Through 12 Months Administration
Gain Therapeutics presented Phase 1b open-label extension data showing MDS-UPDRS motor scores remained stable over 12 months of rexaceract dosing in all 12 evaluable participants, with no clinically meaningful disease progression in Parkinson's disease.
No new safety signals were observed, and treatment-emergent adverse events declined in the 9-month extension versus the initial 3-month period; participants in both high and low CSF GluSph baseline groups showed stabilization.
The company will submit a new Phase 2 protocol to the FDA in the coming weeks and expects to begin the Phase 2 trial in 1Q27, a slight delay informed by Phase 1b learnings and discussions with potential partners.
Red flags
- open-label, uncontrolled data with small sample size (n=12) -- placebo effect cannot be excluded
- Phase 2 start moved to 1Q27 with a new protocol submission, indicating trial design changes after discussions with 'potential partners'