
Newly named drug leads to stable motor symptoms in Parkinson's
Nice to see the industry recognizing Rexaceract.

Nice to see the industry recognizing Rexaceract.
This is the long term Phase 1b data in Parkinson’s patients. These patients started in a 90 day safety trial and then transitioned to an additional 9 months on the drug. The majority of the patients transitioned to the year long study and the company has said none of the patients have shown any progression on the UPDRS scale. Typically after a year patients on average increase 4-6 points on the UPDRS scale. The data will be shared with big pharma to see if they want to own/partner on the worlds first drug that can actually change the trajectory of Parkinson’s. It also should have applications in Gaucher, Lewy Body Dementia and Alzheimers.
Looks like this was this morning. Great session for Gain to be part of.
While the actual presentation is Wednesday October 7th the data will be off embargo on October 1st. The data we are waiting for is the 360 day UPDRS scores for the Phase 1b data for Rexaceract. As told by our CEO none of the patients have shown progression after over a year of being on the drug. Seeing the actual data is what big pharma has been waiting for as it shows long term safety and efficacy. For previous conferences once the data is off embargo the company can then put out a pr of the data.
CEO says data release is reaching crezendo in terms of efficacy. None of the patients have shown any Parkinson’s progression after over 270 days in the trial. There will be CSF data taken after 360 days to get additional biomarker data that will confirm mechanism of disease modifying drug, Rexaceract.
Company and clinicians are happy with the UPDRS results. Plan to put out data at the Internation Congress of Parkinson’s Disease and Movement Disorders conference October 4-8, 2026.
Good video explaining how the two drugs work and what their short term prospects are. Both have readouts coming up. Gain's is in the next 3 weeks and Coya's is in Q1 of 2027.
Watch "Two Tiny Biotech Stocks Big Pharma Is About to Fight Over" on YouTube
Here is a recent interview. Nothing new but its a good reminder that this is a process.
Watch "This CEO is Fighting Parkinson’s & Other Neuro-Degenerative Diseases. Gene Mack - Gain Therapeutics" on YouTube
ATM usage has been low at only 300K shares in first 5 weeks of the 3rd quarter. Works out to be roughly 10K shares/day and the runway goes out into 2027. No rush to make a deal with full year data at major October conference. Short interest is quite high so look for them to cover in next few weeks. We know data as reported today. No Parkinson’s progression at a year.
Today's Q2 report indicates that Gains Parkinson’s drug, Rexaceract, has stopped Parkinson's progression in all 16 patients with 7 completing the year long trial and the rest finishing before reporting the data at the October International Congress of Parkinson’s Disease in Korea.
Participants with elevated baseline levels of glucosylsphingoshine (GluSph) in cerebrospinal fluid (CSF) continued to benefit more than those with low baseline levels of GluSph in CSF after 150 days of dosing with rexaceract, with a difference of 4.8 points in the sum of MDS-UPDRS Part II and Part III scores between the two groups at Day 150.
Participants provided unsolicited descriptions of perceived benefit after 90 days of dosing with rexaceract. Perceived benefits most commonly described included four instances of improved sense of smell and taste, four instances of improved balance or gait, and three instances of improved sleep.
Recent podcast explains how GT-02287 works.
Moving From Symptom Management to Disease Modification in Parkinson’s Research: Q&A with Dr. Jan Stoehr | PharmExec
Key Takeaways
Upstream strategies seek to slow Parkinson’s by interrupting misfolded-protein aggregation and brain-wide propagation, complementing symptomatic therapies that may wane as neurodegeneration advances.
Multimodal biomarkers can reveal pre-symptomatic disease biology, improve trial eligibility and stratification, quantify progression, and support pharmacodynamic evidence of target engagement.
SHOW MORE
Dr. Stoehr, neuroscience researcher at AbbVie discusses how biomarkers are opening a new window into Parkinson’s disease.
This recent article points out the area Gain's drug Rexaceract is able to improve by preventing the build up of toxic lysosomal metabolites. This article is on aging and is an indicator this drug may be applicable to much more than just neurological diseases such as Parkinsons, Alzheimers and Lewy Body Dementia.
“Zombie Cells” Reveal a Hidden Weakness That Could Help Fight Aging
https://scitechdaily.com/zombie-cells-reveal-a-hidden-weakness-that-could-help-fight-aging/
Some had speculated there was some reason the CMO left. Seems like they have probably been working together for years and made him an offer he couldn't refuse. At least we now one big pharma who is well aware of Gain and must think pretty highly of the work he did on GT-02287. Nobody poaches someone who isn't successful.
In a just released Shareholder letter the company confirmed all 16 patients in their Phase 1b study have showed no Parkinsons progression. They have also said the treatment is stable and durable as the trial progresses through the 9 month extension. The first patients will be completing their year on the drug and 270 day readout is coming according to this interview.
*https://youtu.be/AF6T\\\_MEZ-mQ?is=wcY-7czVzqD2-Ai1\*
*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*.https://gaintherapeutics.com/newsroom/press-releases/press-releases-2026/gain-therapeutics-ceo-gene-mack-issues-letter-to-shareholders-and-providesrational-update-2/https://gaintherapeutics.com/newsroom/press-releases/press-releases-2026/gain-therapeutics-ceo-gene-mack-issues-letter-to-shareholders-and-provides-operational-update-2/
In a just released Shareholder letter the company confirmed all 16 patients in their Phase 1b study have showed no Parkinsons progression. They have also said the treatment is stable and durable as the trial progresses through the 9 month extension. The first patients will be completing their year on the drug and 270 day readout is coming according to this interview.
https://youtu.be/AF6T\_MEZ-mQ?is=wcY-7czVzqD2-Ai1
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.https://gaintherapeutics.com/newsroom/press-releases/press-releases-2026/gain-therapeutics-ceo-gene-mack-issues-letter-to-shareholders-and-providesrational-update-2/https://gaintherapeutics.com/newsroom/press-releases/press-releases-2026/gain-therapeutics-ceo-gene-mack-issues-letter-to-shareholders-and-provides-operational-update-2/
Watch "Parkinson’s Breakthrough? GANX CEO Gene Mack on 9-Month Data, Bial’s Failure & What’s Next" on YouTube