Gain Therapeutics Presents Positive Phase 1b Open-Label Extension Data For Rexaceract Demonstrating Further Evidence of Disease-Modifying Activity Through 12 Months Administration
Globe Newswire•01/10/2026•09:28 ET
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Key Highlights
- ➤12 participants completed 12 months; MDS-UPDRS motor scores remained stable to baseline
- ➤No clinically meaningful motor-symptom progression observed across 12 months of rexaceract dosing
- ➤Gain Therapeutics (GANX) expects FDA Phase 2 protocol submission in coming weeks
- ➤Phase 2 rexaceract trial expected to begin in 1Q27
- ➤No new safety signals observed; rexaceract generally well tolerated over 12 months
Expert Statements
Gene Mack, President and CEO of Gain Therapeutics
“Seeing MDS-UPDRS scores remain stable after a full year of dosing is an important clinical milestone for rexaceract. This data gives us greater confidence in the durability of the clinical profile we are seeing and strengthens our conviction as we advance rexaceract into the next stage of development. We have learned enough new information from the Phase 1b that we want to address in our Phase 2 planning to move the start of the study to 1Q27 and take the prudent step of submitting a new protocol to the FDA.”
Gene Mack, President and CEO of Gain Therapeutics
“We believe rexaceract has the potential to shift the treatment paradigm beyond symptom management by addressing the underlying biology of Parkinson’s disease, and we expect rexaceract to be the backbone of the next generation of treatment for Parkinson’s disease.”
Joanne Taylor, Chief Scientific Officer of Gain Therapeutics
“The stabilization of MDS-UPDRS scores over one year of treatment with rexaceract regardless of biomarker stratification is particularly encouraging. The consistency in durability across both low and high GluSph groups provides encouraging support for the continued investigation of the relationship between GluSph biology, glucocerebrosidase target engagement, and clinical outcomes in Parkinson’s disease.”
MDS-UPDRS motor scores demonstrated stability to baseline recorded at study entry with no disease progression among the 12 evaluable participants who completed 12 months of daily oral administration of rexaceract by August 31, 2026
Participants with both high and low baseline levels of GluSph experienced stabilization of MDS-UPDRS motor scores by 12 months
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