New Sponsors, Potential Development in NMOSD: Bruce Cree, MD, PhD, MAS, FAAN

Disease Spotlight | <b>Disease Spotlight: NMOSD</b>

Following the approval of 3 treatments for the disease, the clinical research director of the UCSF Multiple Sclerosis Center commented on the implications for the clinical development pipeline. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

“I think with these 3 drugs that are so effective—and are also, generally speaking, quite safe—they are creating a barrier to entry of new for new products because now you can't do a placebo-controlled trial. Anything you do is going to have to be based on a superiority study design, which necessitates a larger sample size and additional costs associated with drug development. Plus, when something is 78% to 92% effective, to get beyond that, you really have to be hitting the ball out of the park.”

The approval of 3 new therapeutic treatments for neuromyelitis optica spectrum disorder (NMOSD) creates the potential for these drugs not only to take over the market for this patient population, but also to create a barrier to entry for other treatments. The drugs in question, eculizumab (Soliris; Alexion), satralizumab (Enspryng; Genentech), and inebilizumab (Uplizna; Horizon), have changed the game for patients with NMOSD since 2019, as the condition previously had no proven therapies.

In conversation with NeurologyLive, Bruce Cree, MD, PhD, MAS, FAAN, clinical research director of the UCSF Multiple Sclerosis Center, and professor of clinical neurology, UCSF Weill Institute for Neurosciences, discussed this issue, as well as the potential for self-tolerizing strategies in NMOSD. Cree noted that NMOSD is an acquired disease, not inherited, with experts believing they may eventually be able to correct the breach in self-tolerance for patients who are no longer tolerant of aquaporin-4 and unable to develop an antibody response.