Darwin Biotech Presented Randomized Phase 1 ALS Study Data of Cell-Free ALT001 and U.S. Phase 1/2 Study Design at 2026 NEALS Annual Meeting
ALT001 was well tolerated in randomized Phase 1 ALS study, with early signals on Function and NfL
No treatment-related serious adverse events at any of three doses
ALS function scores and plasma neurofilament light chain favored ALT001 over placebo at Day 31
U.S. Phase 1/2a study planned to test higher doses
BEIJING & CLEARWATER, Fla., October 30, 2026--Darwin Biotech ("Darwin"), a clinical-stage company developing treatments for neurological diseases, today announced that data from its randomized, double-blind, placebo-controlled Phase 1 study of ALT001 in amyotrophic lateral sclerosis (ALS), including 12-week results from an ongoing open-label extension (OLE), were presented in a poster at the 2026 Network of Excellence for ALS (NEALS) Annual Meeting, taking place October 27-30 in Clearwater, Florida.
ALT001 is a novel, cell-free protein complex derived from human mesenchymal stem cells. The Phase 1 study, led by Principal Investigator Prof. Yilong Wang and team at Beijing Tiantan Hospital, Capital Medical University, enrolled 36 participants with ALS who were randomized 3:1 to ALT001 at 1.0, 2.0 or 4.0 μg/kg or placebo (27 ALT001; 9 placebo). Primary endpoints were safety, tolerability, maximum tolerated dose and recommended dose. Secondary endpoints included the ALS Functional Rating Scale–Revised (ALSFRS-R), the ALS Assessment Questionnaire-40 (ALSAQ-40), the Modified Norris Scale, forced vital capacity, muscle function and biomarkers including plasma neurofilament light chain (NfL).
Key Phase 1 findings include:
Safety: ALT001 was generally well tolerated across all dose levels, with no treatment-related serious adverse events (SAEs) or significant intolerance events (SIEs) reported and no dose-limiting toxicities identified.
ALSFRS-R: At Day 31, mean ALSFRS-R scores increased from baseline by 1.22, 0.78 and 0.44 points in the 1.0, 2.0 and 4.0 μg/kg groups, respectively, compared with a mean decline of 0.78 points with placebo.
Plasma NfL: At Day 31, mean plasma NfL decreased from baseline by 5.02%, 17.16% and 17.05% in the 1.0, 2.0 and 4.0 μg/kg groups, respectively, compared with 2.23% with placebo.
Other measures: ALSAQ-40 scores showed favorable trends, Modified Norris Scale scores were generally stable, and grip strength was better preserved at 1.0 and 2.0 μg/kg than with placebo.
Efficacy measures were secondary endpoints, and these results are preliminary given the study's size and 31-day treatment period.
Following the Phase 1 study, 29 of 36 participants entered a 12-week OLE (11, 8 and 10 participants in the 1.0, 2.0 and 4.0 μg/kg cohorts, respectively), in which participants originally assigned to placebo received ALT001 at their cohort's dose. Among participants originally treated with ALT001, mean ALSFRS-R change from OLE baseline to Week 12 was −1.5, −3.4 and −1.3 points in the 1.0, 2.0 and 4.0 μg/kg cohorts, respectively. ALSFRS-R trajectories suggested slower functional decline in OLE participants than in Phase 1 participants who did not enter the extension. These findings are exploratory and are based on small, non-randomized groups with different follow-up timelines.