A new update has just arrived from Gain Therapeutics regarding their Phase 1b Parkinson’s disease trials: Gain Therapeutics CEO Gene Mack Issues Letter to Shareholders and Provides Operational Update - Perspectives from Gain Therapeutics.
“The results – both from our Phase 1b study of rexaceract and open-label extension – continue to support our hypothesis for the drug candidate and its mechanism of action and we continue to observe safety and tolerability through five months of treatment. We also continue to see strong engagement in the open-label extension. All 16 participants who entered the ongoing Phase 1b extension study remained on study at Day 150, and following the most recent review on July 6, 2026, an independent Data Monitoring Committee recommended the study continue without modification.”
"While longer term outcomes in this dosing extension are anticipated to read out in October 2026, we have already released encouraging data from Part 1 and Part 2 of the study which includes the first 90 days (3 months) administration of rexaceract, during which reduction in cerebrospinal fluid (CSF) levels of glucosylsphingosine (GluSph) in patients with elevated levels at baseline correlated with an early clinical benefit. That response is largely maintained after at least five months of administration, while the entire cohort of 16 patients can be categorized as non-progressors at both three and five months regardless of baseline characteristics. We look forward to the next updates at 270 days and 360 days, which will occur over the summer and at the conclusion of the study in October 2026. "
This is perhaps the best peer company for Herantis that I have found. The results are similar so far, meaning good tolerability for the drug candidate in Phase 1 trials and indications of the drug’s biological effect. Otherwise, the results are still too early to interpret efficacy.
That Gain drug candidate is a β-glucocerebrosidase activator and its mechanism likely also affects the same pathways influencing proteostasis via lysosomal function as HER-096. However, I am not entirely sure if all Parkinson’s patients would potentially benefit from that Gain treatment, or only patients who have deficiencies in that β-glucocerebrosidase function.