Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
The FDA has approved inebilizumab (Uplizna; Amgen) for the treatment of generalized myasthenia gravis (gMG) in adults who are antiacetylcholine receptor (AChR) and antimuscle specific tyrosine kinase (MuSK) antibody positive, administered twice yearly after 2 initial loading doses. This marks the third FDA-approved indication for inebilizumab, which is also approved for antiaquaporin-4 antibody positive neuromyelitis optica spectrum disorder and immunoglobulin G4–related disease. The approval of inebilizumab for gMG is supported by data from the phase 3 MINT trial (NCT04524273). In the trial, researchers 238 patients with gMG were randomly assigned 1:1 to either intravenous ineblizumab 300 mg (n = 119) or placebo (n = 119) for a 12-month period. Change from baseline in Myasthenia Gravis Activities of Daily Living (MD-ADL), the study’s primary end point, was statistically significant for inebilizumab-treated patients, with scores of –4.2 compared with –2.2 for those on placebo (P <.0001). The therapy showed a safe and well tolerated profile, consistent with prior safety findings.
According to a new announcement, the FDA has cleared the initiation of a pivotal, 500-patient, randomized, placebo-controlled phase 3 trial dubbed PREVAiLS, which will assess the efficacy and safety of pridopidine (Prilenia Therapeutics/Ferrer) in patients with early-stage, rapidly progressive amyotrophic lateral sclerosis (ALS). The company noted that recruitment at the first clinical trial sites in the United States is planned to begin in early 2026, with international sites expected to follow pending regulatory approval. PREVAiLS will enroll adults diagnosed with definite or probable ALS who are within 18 months of symptom onset across up to 60 ALS treatment centers worldwide. The trial design consists of a 48-week double-blind, placebo-controlled phase, randomized 3:2 to receive either pridopidine or placebo, followed by a 48-week open-label extension. The primary end point is change from baseline in the ALS Functional Rating Scale–Revised (ALSFRS-R), adjusted for mortality, at 48 weeks while secondary and exploratory end points include measures of speech, respiratory and bulbar function, quality of life, survival, patient-reported outcomes, and plasma biomarkers.
According to a company update, Sanofi’s tolebrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, did not meet its end points in the phase 3 PERSEUS study (NCT04458051) of patients with primary progressive multiple sclerosis (PPMS), and a regulatory submission is therefore not expected. In addition, it’s been reported that the FDA has pushed back its review of the agent as a treatment for non-relapsing secondary progressive MS (nrSPMS). More detailed results from the global, double-blind, randomized trial are expected to be presented at an upcoming meeting. In the release, it was noted that tolebrutinib did not meet its primary end point of delaying time of onset of 6-month composite confirmed disability progression (CDP) compared with placebo in the study cohort, which included those aged 18-55 with an Expanded Disability Status Scale (EDSS) of between 2.0 and 6.5.
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