Ravulizumab Meets Primary End Point in NMOSD, Dosing Initiated in TRANQUILITY II Study, NIH Launches EBV Vaccine Trial


Neurology News Network for the week ending May 14, 2022. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

AstraZeneca has announced findings from its phase 3 CHAMPION-NMOSD trial, which showed that ravulizumab-cwvz (Ultomiris) met its primary end point of time to first on-trial relapse, with no relapses observed in 58 patients with anti-aquaporin (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD) over a median treatment duration of 73 weeks.Less than 2 weeks after receiving FDA approval to treat generalized myasthenia gravis (gMG), these data on ravulizumab, a long-acting C5 complement inhibitor, show its benefit in adults with anti-AQP4 antibody-positive NMOSD. The therapeutic demonstrated statistically significant and clinically meaningful reductions in the risk of relapse compared with those on placebo from the external PREVENT trial a phase 3 study that evaluated eculizumab (Soliris; Alexion), which is FDA-approved for NMOSD treatment. The company noted that the safety profile of ravulizumab was consistent with what had previously been observed. In total, 56 of the original 58 patient cohort completed the primary treatment period and opted to continue in the long-term extension, which is ongoing.

According to a new announcement, patient dosing has begun in the TRANQUILITY II study, a pivotal phase 3 trial evaluating the efficacy and safety of BXCL501 (BioXcel Therapeutics), an orally dissolving thin film formulation of dexmedetomidine for the acute treatment of agitation in Alzheimer disease (AD).1 In April 2022, BXCL501 gained FDA approval for agitation associated with schizophrenia or bipolar I or II disorder in adults.Initiated in December 2021, the trial is part of a larger phase 3 program that also includes TRANQUILITY III, a randomized, placebo-controlled, adaptive study that is parallel in design to TRANQUILITY II. Both studies are expected to enroll approximately 150 patients with dementia older than the age of 65 years with change from baseline in Positive and Negative Syndrome Scale-Excitatory Component (PEC) as the primary end point. PEC scores will be recorded at 2 hours after initial dose and after subsequent doses as well. Following completion of the phase 3 trials, participants will be eligible to enroll in a 52-week, open-label safety study to evaluate the efficacy of continued use of BXCL501.

Months after data further confirmed the link between multiple sclerosis (MS) and Epstein-Barr virus (EBV), the National Institute of Allergy and Infectious Disease (NIAID), part of the National Institutes of Health (NIH), announced a new phase 1 study evaluating an investigational preventive vaccine for the virus, also known as herpesvirus 4. Participants will be followed for 18 to 30 months, and the trial is expected to last 4 years.Led by Jessica Durkee-Shock, MD, assistant research physician, Laboratory of Infectious Diseases, NIAID, the study will assess safety and immune response of a gp350-Ferritin nanoparticle vaccine with a saponin-based Matrix-M adjuvant. The vaccine, developed by the Laboratory of Infectious Diseases in collaboration with NIAID’s Vaccine Research Center, is designed to target EBV glycoprotein gp350, which is found on the surface of the virus and virus-infected cells. Anthony S. Fauci, MD, director, NIAID, said in a statement, "A vaccine that could prevent or reduce the severity of infection with the Epstein-Barr virus could reduce the incidence of infectious mononucleosis and might also reduce the incidence of EBV-associated malignancies and autoimmune diseases."

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