Clinical Trial Updates
March 2026
AMT-130 Gene Therapy for Huntington's Disease
AMT-130 is a signal dose therapy that requires surgery to deliver. It is an adeno-associated virus (AAV), which are effective at delivering genes without causing disease or infection. This AAV encodes a micro RNA that lowers the levels of huntingtin protien. Initial phase I/II results reported in September 2025 showed that high doses of ATM-130 were generally well tolerated and that there were significant improvements in biomarkers (neurofilament light chain) and more importantly in disease progression as measured using two clinical tests. However, in March 2026 the FDA said that the data from this trial was not sufficient to prove effectiveness and asked for another trial to be done.
May 2026
VHB937 TREM2 Agonist for Alzheimer's Disease
VHB937 is a TREM2 agonist that works by stabilizing the protein with the hope that the immune cells that express it (microglia) will be more active against amyloid plaques. This phase II clinical trial is to assess the safety and efficacy of VHB937. Hopefully, the patients who recieve it will have improved memory, be more functional day-today, and have better biomarkers than the placebo group. The trial is now recruiting, click here to see if you qualify.
December 2025
Tofersen for SOD1 Induced ALS
Tofersen is an antisense oligonucleotide therapy that reduces the amount of toxic SOD1 produced in patients with SOD1 mutations. In 2022 it was found that, although it reduced biomarkers like neurofilament light chain and SOD1, it did not improve clinical end points. However, it was still approved in 2023 by the FDA. In December 2025, trial data from longer term use of Tofersen found that it significantly lowered risk of death, especially at earlier treatment points.
https://jamanetwork.com/journals/jamaneurology/fullarticle/2843130
https://www.nejm.org/doi/full/10.1056/NEJMoa2204705
https://clinicaltrials.gov/study/NCT02623699
September 2025
SARM1 Inhibitor LY3873862 for ALS
Massachussetts General Hospital announced that its Healey Center for ALS would be including Eli Lilly's oral SARM1 inhibitor LY3873862 in their ALS Platform Trial to see if it is safe and if it prevents or delays ALS progression. If the trial shows good results, it would represent a significant step forward in disease-modifying therapies for ALS. It began enrollment in late 2025.
https://www.massgeneral.org/neurology/als/news/new-regimen-for-healey-als-platform-trial-with-lilly