Novartis sets sights on new oral MS drug after phase 3 wins

Novartis' oral BTK inhibitor remibrutinib has shown efficacy in a pair of phase 3 trials in relapsing multiple sclerosis (RMS), setting up marketing applications and boosting the drug's blockbuster credentials.

Data from the REMODEL-1 and REMODEL-2 trials showed that remibrutinib was significantly more effective than Novartis' older oral MS therapy Aubagio (teriflunomide) in reducing the annualised relapse rate (ARR), the primary endpoint of the studies, as well as "all key secondary endpoints," said the Swiss pharma group.

The company said it now plans to file for approval of remibrutinib for RMS with regulatory authorities around the world.

Remibrutinib has already been approved in the US, EU, and Japan for chronic spontaneous urticaria (CSU) under the Rhapsido brand name, and is being developed for a host of other immune-mediated and neurological conditions, including MS, chronic inducible urticaria (CINDU), hidradenitis suppurativa (HS), food allergies, and generalised myasthenia gravis (gMG).

It generated a little over $100 million in sales for Novartis in the first half of this year, but has been tipped by analysts as a big earner for the company, with peak annual sales forecasts ranging from $3 billion to as much as $9 billion. It is one of three major pipeline hopes along with Lp(a)-targeting drug pelacarsen for cardiovascular diseases and Duchenne muscular dystrophy (DMD) therapy delpacibart etedesiran (del-desiran).

Along with the reduction in ARR versus Aubagio – a former $2.3 billion blockbuster that lost patent protection in the US and Europe in 2023 – the REMODEL-1 and 2 trials also showed a significant reduction in new MRI lesions with remibrutinib and a "clinically meaningful" delay in disability progression, according to Novartis. The safety profile of the drug was also consistent with that seen in other clinical trials.

"Despite advances in treatment, an unmet need remains for oral therapies that can deliver robust relapse prevention [and] slow disability progression, while maintaining a favourable safety profile," said Shreeram Aradhye, Novartis' chief medical officer.

"The positive REMODEL results underscore the potential of remibrutinib as a high-efficacy oral therapy for people living with RMS with a differentiated benefit-risk profile."

The results sparked a rise in Novartis' share price, which had fallen earlier in the day after news emerged of three fatalities in one of its CAR-T programmes for immunological diseases.