Clinical Trial Updates
August 2026
Bezisterim for Parkinson's
Bezisterim is an oral drug being developed to reduce inflammation without broadly suppressing the immune system. Because chronic inflammation in the brain may contribute to neurodegeneration in Parkinson's disease, the SUNRISE-PD phase II trial tested bezisterim in people with early Parkinson's who had not yet started levodopa.
After 12 weeks, patients receiving bezisterim showed improvements compared with placebo across measures of motor symptoms, non-motor symptoms, and daily activities. The treatment also lowered several markers of inflammation and nerve cell injury, including neurofilament light chain. The study was small, with 57 participants, and many of these analyses were exploratory, so larger and longer trials will be needed to determine whether bezisterim actually slows Parkinson's disease progression.
July 2026
Diranersen Tau-Lowering Therapy for Alzheimer's
Diranersen is an antisense oligonucleotide that lowers production of tau by targeting the RNA used to make the protein. Tau normally helps support neurons, but in Alzheimer's disease abnormal tau accumulates inside brain cells and forms tangles associated with neurodegeneration.
In the phase II CELIA trial, the lowest dose of diranersen slowed worsening by 26% on the CDR-SB, a measure of cognition and daily function, compared with placebo after 18 months. It also slowed decline by 42% on one cognitive test and 50% on another, while reducing tau in spinal fluid by about 50–65%. However, the study did not meet its primary endpoint because higher doses did not produce greater benefits than the lower dose. Biogen plans to move diranersen into phase III testing.
JUne 2026
SKY-0515 for Huntington's Disease
SKY-0515 is an oral drug that changes RNA splicing to reduce production of mutant huntingtin, the toxic protein that causes Huntington's disease. It also lowers PMS1, a protein involved in the expansion of the abnormal CAG repeat that may contribute to disease progression.
After twelve months of treatment, the highest dose lowered mutant huntingtin in the blood by up to 69% and lowered PMS1 RNA by up to 26%. Patients receiving SKY-0515 also had an average improvement of 0.38 points on the cUHDRS, a measure of Huntington's disease progression, compared with an expected decline of 0.92 points based on previous untreated patients. These results are promising, but only 15 patients contributed to the twelve-month clinical comparison and it used historical data rather than a simultaneous placebo group. A larger phase II/III trial is now underway and is recruiting.
June 2026
AP-101 Antibody for ALS
AP-101 is an antibody designed to target misfolded forms of SOD1, a protein that can become toxic and contribute to motor neuron degeneration in ALS. Unlike tofersen, which lowers production of SOD1 in people with SOD1 mutations, AP-101 is designed to remove misfolded SOD1 and is being studied in both genetic SOD1-ALS and more common sporadic ALS.
Updated phase II results found that AP-101 was generally well tolerated and reduced biomarkers of nerve damage. An exploratory analysis also found that patients who began AP-101 earlier had longer survival and delayed need for breathing support compared with those who initially received placebo. These results are encouraging, but the survival findings were exploratory and will need to be confirmed in the planned phase III trial.
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.
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.