BXCL501 Study in Alzheimer Begins, Ublituximab BLA Accepted, Sublingual Apomorphine Shows Dominance


Neurology News Network for the week ending December 18, 2021. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

A phase 3 program studying BXCL501, an orally dissolving thin film formulation of dex-medetomidine, has been initiated to study the safety and efficacy of the drug in the acute treatment of agitation in patients with Alzheimer disease (AD). The program consists of 2 studies, TRANQUILITY 2 and TRANQULITY 3, BioXcel Therapeutics announced.In March 2021, BXCL501 received breakthrough designation from the FDA for the acute treatment of agitation associated with dementia. The designation was supported by positive topline data from the phase 1b/2 TRANQULITY study, with the treatment demonstrating significant reductions in agitation measures at both dose levels, 30 mcg and 60 mcg, at 2 hours post-dose. The parallel phase 3 pivotal trials will be randomized, placebo-controlled, and adaptive, enrolling approximately 150 patients with dementia older than the age of 65 years in each study. When agitation occurs, patients will self-administer a 40-mcg or 60-mcg dose of BXCL501, or placebo over a 3-month period.

A biologics license application (BLA) for ublituximab, an investigational glycoengineering anti-CD20 monoclonal antibody, was accepted by the FDA for treatment of relapsing forms of multiple sclerosis (RMS), TG Therapeutics has announced. Currently, the FDA does not plan to hold an advisory committee meeting to discuss the application and has set a prescription drug user fee goal date of September 28, 2022.Following positive results from the ULTIMATE 1 and 2 trials, identical phase 3 studies that evaluated ublituximab compared to teriflunomide (Aubagio; Sanofi) in patients with RMS, the BLA was submitted in September 2021. Both trials were conducted under a special protocol assessment established with the FDA.

Using a cost-effectiveness model, recently conducted research showed that apomorphine sublingual film, an FDA-approved medication to treat OFF episodes in patients with Parkinson disease (PD), is less costly and more effective on average compared with apomorphine hydrochloride and levodopa inhalation powder. After receiving US approval in 2020, investigators set out to understand how apomorphine sublingual film compared to other on-demand treatments from a US payer perspective over a 10-year period. Best case analysis used an annual discount rate of 3% for costs and utilities. Over a 10-year time horizon, on-demand treatment costs were estimated at $42,095 for apomorphine sublingual film, $276,320 for apomorphine hydrochloride, and $69,577 for levodopa inhalation powder. This resulted in treatment cost differences of $234,225 between the sublingual film vs apomorphine hydrochloride, and $27,482 between apomorphine sublingual film and the inhalation powder.

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