AB Science Achieves 42% Five-Year ALS Survival Rate with Masitinib in Phase 2b/3 Trial Data

Survival data from AB Science's masitinib ALS programme now show a five-year rate of 42.3% from symptom onset, roughly double the published historical benchmarks of 7%–27.8%, with a weighted mean near 24%. The figures, drawn from the Phase 2b/3 trial AB10015, are scheduled for presentation at the ENCALS Annual Meeting in Madrid on 25 June 2026, a congress whose abstracts are selected on scientific merit by an independent programme committee.

For development and regulatory teams tracking rare neurological assets, the dataset carries early filing signals. Masitinib, a tyrosine kinase inhibitor administered at 4.5 mg/kg/day in combination with riluzole, produced a five-year survival rate of 50% in the primary analysis population, patients with slower disease progression (ALSFRS-R ΔFS <1.1 points/month) at baseline. In the subset who had not yet experienced total loss of function at enrolment, the rate reached 53%. Critically, 49% of long-term survivors maintained functional independence without mechanical assistance, including permanent ventilation, gastrostomy, tracheotomy, or wheelchair dependence.

Professor Albert Ludolph, MD, PhD, of Ulm University Hospital, who will deliver the poster presentation (Reference No. 401), noted that the long-term survivor analysis appears to indicate a clinically meaningful survival benefit compared with historical controls, while cautioning that confirmatory evidence remains necessary. That qualification is material: any future NDA or MAA submission for an orphan neurological indication will require the agency to weigh single-trial survival data against the unmet-need threshold, particularly under 21 CFR Part 312 accelerated pathway criteria or EMA's conditional marketing authorisation framework.

Manufacturing and supply-chain planning for a tyrosine kinase inhibitor targeting a rare disease cohort involves its own regulatory layer. Process validation packages under ICH Q10 and commercial-scale GMP readiness will need to align with the orphan drug designation timelines AB Science holds in both the US and EU. Sterility assurance and analytical method transfer documentation would need to be in place well ahead of any rolling submission strategy.

The ENCALS presentation on 25 June will be the first public disclosure of the long-term outcome analysis from AB10015, and the dataset's reception by the neurology and regulatory science community will likely shape the timeline for any formal submission package.

Source: AB Science via GlobeNewswire, 9 June 2026.