Novartis Drops ALS Drug
Novartis discontinues experimental ALS drug development after phase two trial misses primary endpoints.
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Key Points
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The ASTRALS Phase 2 trial enrolled 251 people with early-stage ALS and compared VHB937 with placebo.
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The trial’s primary endpoint was a composite measure of survival without mechanical ventilation and changes in the ALS Functional Rating Scale–Revised, which assesses limb, respiratory and bulbar function.
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Novartis disclosed the discontinuation in a letter dated Aug. 25 to the European Organization for Professionals and People with ALS; detailed results are scheduled for an oral presentation at the 37th International Symposium on ALS/MND in December.
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The setback adds to broader difficulties for TREM2-based therapies: Alector and AbbVie’s TREM2 activator missed its primary endpoint in an Alzheimer’s Phase 2 trial in 2024, while Vigil Neuroscience later reported failure of its own TREM2-activating antibody.
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The ALS failure was described as Novartis’ fourth clinical setback in less than a month, following failures involving the cardiovascular drug pelacarsen and the myotonic-dystrophy therapy del-desiran, as well as the suspension of multiple immunology and neurology studies after three deaths.
Novartis has stopped developing VHB937, an experimental ALS drug, after it failed to beat placebo in a mid-stage trial. The Swiss drugmaker tested the therapy in 251 early-stage ALS patients but missed all primary and secondary goals. Yahoo Finance reports this marks Novartis' fourth major clinical setback in less than a month.
How VHB937 Was Supposed to Work
VHB937, also called lifonebart, targeted TREM2, a protein on immune cells. The drug was designed to activate these cells to protect motor neurons from damage in ALS. The approach made biological sense but failed to show real benefits in patients.
What the ASTRALS Trial Measured
The study tracked three key measures: survival without needing a ventilator, changes in the ALS Functional Rating Scale (which tests limb, breathing and swallowing function), and neurofilament light levels (a marker of nerve damage). VHB937 did not outperform placebo on any of these measures. Yahoo Finance states the trial enrolled 251 people with early-stage ALS.
Other TREM2 Drugs Also Failing
VHB937 is not alone. Alector and AbbVie's TREM2 activator missed its Alzheimer's trial goal in 2024. Vigil Neuroscience reported similar failure with its own TREM2-activating antibody. The repeated setbacks suggest the TREM2 approach may not work as scientists hoped.
Novartis Facing a Troubled Quarter
VHB937's failure is the fourth clinical blow in under one month. The company also reported setbacks with pelacarsen (a cardiovascular drug) and del-desiran (for myotonic dystrophy). Novartis also suspended multiple immunology and neurology studies after three patient deaths. Detailed ALS results will be presented in December at an international ALS conference.
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