NeuroSense Therapeutics has completed enrolment of a Phase IIb trial in amyotrophic lateral sclerosis (ALS), marking an important milestone in a busy year of ALS drug development.
The Phase IIb PARADIGM study (NCT05357950) has enroled 69 patients with ALS in Israel, Italy, and Canada, NeuroSense announced Monday. The six-month study, which investigates PrimeC (celecoxib/ciprofloxacin), has topline results expected Q4 2023.
PrimeC is an oral, extended-release formulation of celecoxib and ciprofloxacin, both of which have approval from the US Food and Drug Administration (FDA). The ALS candidate has orphan drug designation from both the FDA and the European Medical Association (EMA).
As primary endpoints, the placebo-controlled PARADIGM study is measuring two ALS blood biomarkers and drug safety. Secondary endpoints include the ALS functional rating scale – revised (ALSFRS-R) and survival, which the FDA has indicated is a preferred efficacy endpoint for ALS.
NeuroSense received the regulatory go-ahead from the FDA to enrol patients with ALS in PARADIGM last November.
ALS drug development
The NeuroSense enrolment update comes amid a hectic year in ALS drug development.
On April 26, the FDA granted Biogen’s Qalsody (tofersen) accelerated approval to treat ALS driven by a specific gene mutation following strong support from an FDA Advisory Committee (AdCom). Last September, the FDA approved Amylyx’s Relyvrio (sodium phenylbutyrate and taurursodiol) despite an AdCom narrowly voting against approval.
However, recent ALS drug development has not been all roses. In April, Cytokinetics announced the termination of its Phase III ALS program as the company refocuses on cardiomyopathy.