Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
Newly presented data from a phase 1/2 trial showed that bemdaneprocel (BlueRock Therapeutics), an investigational cell therapy, maintained a favorable safety profile at 36 months, with stable motor outcomes and continued positive efficacy trends from baseline in patients with Parkinson disease (PD). The agent continues to be studied in an ongoing phase 3 registrational trial, dubbed exPDite-2, that will have data read out in 2027. Presented at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) Congress, held October 5-10, in Hawaii, the phase 1/2 trial included 12 patients with PD who received surgical transplantation of 1 of 2 different doses of bemdaneprocel cells to the post-commissural putamen bilaterally, along with immunosuppression regimen for 1 year. Within the high-dose cohort (n = 7), findings revealed a mean reduction of 17.9 points in MDS-Unified Parkinson Disease Rating Scale (UPDRS)-Part III at 36 months. The lower dose cohort experienced slightly less decreases (13.5 points), although both were considered to be clinically meaningful.
New long-term extension data from the phase 3 CHAMPION-NMOSD (NCT04201262) trial showed that patients with antiaquaporin (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD) treated with ravulizumab-cwvz (Ultomiris; Alexion) experienced no adjudicated on-trial relapses and most displayed stable or improvement in disability. Among 58 patients with NMOSD enrolled, 56 entered and 55 completed the long-term extension, with a median follow-up of 170.3 weeks (range, 11–243) totaling 189.7 patient-years. Presented at the 2025 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, held September 24-26, in Barcelona, Spain, researchers reported that throughout the primary treatment period and long-term extension, no patient who received ravulizumab-cwvz had an adjudicated on-trial relapse. Notably, ravulizumab-cwvz-treated patients maintained a 98.9% relative reduction in relapse risk compared with placebo (95% CI, 91.8-100; P <.0001).
According to a recent announcement, the FDA granted fast track designation to Bristol Myers Squibb’s investigational anti-microtube binding region-tau (anti-MBTR-tau) antibody, BMS-986446, for the treatment of patients with early-stage Alzheimer disease (AD). The drug is currently being studied in a phase 2 trial, dubbed TargetTau-1, which tests efficacy, safety, and tolerability of multiple doses in those with early AD. Anti-MTBR-tau antibodies like BMS-986446 are designed to target the MTBR of tau protein, a critical domain that drives tau aggregation into neurofibrillary tangles in AD. Unlike full-length tau, MTBR fragments are highly aggregation-prone and appear early in disease progression, making them an attractive therapeutic target.
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