Dive Brief:

• A pill developed by Novartis succeeded in a pair of Phase 3 trials in people with a common form of multiple sclerosis, bolstering confidence in the drug’s prospects and sending company shares up by over 4% on Tuesday.
• The Swiss pharmaceutical giant didn’t provide detailed study results. Still, Novartis revealed the therapy, remibrutinib, demonstrated “superiority” over the drug Aubagio in reducing yearly relapse rates and inflammatory brain lesions in study participants. Treatment was associated with “clinically meaningful” effects on disability progress and, notably, didn’t impact liver safety — an issue for multiple drugs in remibrutinib’s class. 
• The findings could position remibrutinib, which is already approved as Rhapsido for chronic skin hives, to become a bigger sales contributor in the future. Novartis is looking to remibrutinib and other late-stage prospects to offset revenue losses from multiple patent expirations. It intends to share the MS study results with health regulators. 

Dive Insight:

Remibrutinib is what’s known as a BTK inhibitor, a kind of medicine best known for its impact on cancer but that drugmakers have more recently sought to turn against autoimmune disorders. Multiple companies have zeroed in on MS in particular, hoping that newer BTK blockers that can get into the brain might become new treatment options.

That endeavour has been riddled with trial failures and safety setbacks. One candidate from Merck & Co. stumbled in testing, while another from Sanofi fell short against relapsing MS but appeared more impactful against a less common form of the disease. That drug was rejected in the U.S., but approved in Europe in June. Testing of both therapies was slowed by the detection of liver-related side effects.

Roche, meanwhile, has claimed success in studies evaluating its own BTK inhibitor against relapsing MS. But analysts have questioned whether regulators will approve that treatment because of effects on the liver. 

Going forward safety concerns will “remain a focal point for investors” and the details Novartis shares will be “crucial to understanding [remibrutinib’s] potential placement in the treatment paradigm,” wrote William Blair analyst Myles Minter. 

In a separate note, Jefferies analyst Michael Leuchten wrote that the findings could unlock more than $3 billion in peak annual sales for Novartis in MS alone. Novartis is also testing the treatment on other neurological indications and inflammatory conditions. Should it succeed in ongoing studies in hidradenitis suppurativa and food allergies, remibrutinib could “become a franchise with high single-digit billion-dollar sales potential.”