Ocrelizumab

Frankfurt: Roche 's experimental multiple sclerosis drug achieved its main goal in one of two key late-stage trials testing it against the relapsing form of the disease, the Swiss drugmaker said on Monday, sending its shares up nearly 2%. The drug, fenebrutinib , was shown to significantly reduce the annualised relapse rate when compared with another drug teriflunomide , over a period of at least 96 weeks of treatment, the company said in a statement. Teriflunomide is sold under the brand name Aubagio by French drugmaker Sanofi. If successful, the drug could provide MS patients a new treatment option and bring in over $1 billion in annual peak sales for Roche , according to Leerink analysts. In a separate third study, Roche's drug candidate was shown to be at least as effective as its own Ocrevus drug in delaying the progression of another form of the disease known as primary progressive multiple sclerosis , the company said. SECOND LATE-STAGE STUDY DATA DUE IN FIRST HALF OF 2026 Roche said data from the second late-stage study against relapsing MS is expected by the first half of 2026. In late 2023, the U.S. FDA had stopped enrollment of new patients in this study, citing cases of liver injury. Roche said on Monday that liver safety was consistent with previous trials. It plans to share detailed data from both the studies at an upcoming medical meeting and will submit data from all three studies to regulatory authorities. Jefferies analysts said that the success of Roche's regulatory submissions for the drug are critically dependant on full safety and efficacy data from the three studies. Fenebrutinib belongs to a class of compounds known as Bruton's tyrosine kinase (BTK) inhibitors, where Sanofi is also a contestant in a development race that has seen trial setbacks by other drugmakers. The class of drugs is designed to selectively block the harmful autoimmune reaction behind MS for a more targeted approach than standard immunosuppressant therapy. (Reporting by Ludwig Burger and Bhanvi Satija in London; Editing by Christian Schmollinger, Jamie Freed and Conor Humphries)

Roche’s BTK inhibitor has cleared two registrational studies in different types of multiple sclerosis, a surprise turn for a drug class that’s seen several trial failures and safety setbacks. The Swiss drugmaker said Monday that fenebrutinib met the primary endpoint of both Phase 3 studies that separately tested it in people with relapsing multiple sclerosis and primary progressive multiple sclerosis (PPMS). The trials together enrolled more than 1,700 patients, according to a company release . CMO Levi Garraway described the wins as “unprecedented” and said they could pave the way for fenebrutinib to become a “best-in-disease medicine.” The relapsing MS success will be seen as a “positive surprise,” Jefferies analysts said in a note to investors. But the analysts said they weren’t sure if a hit in the primary endpoint chosen for the PPMS trial will be enough for a filing with regulators. Roche didn’t share detailed results from either study, but plans to do so at future medical meetings. The company said it will wait for data from a second Phase 3 RMS trial before finalizing regulatory filing plans. Those results are expected in the first half of 2026. In the first RMS trial, called FENhance 2, fenebrutinib significantly cut the annualized relapse rate versus Sanofi’s Aubagio over a treatment period of at least 96 weeks. Aubagio is an inhibitor of the DHODH enzyme that’s commonly used to treat relapsing MS. The other study, called FENtrepid, looked at fenebrutinib in patients with PPMS. The drug was no worse than Roche’s Ocrevus at slowing disability progression over a treatment period of at least 120 weeks. Ocrevus, which was approved for PPMS in 2017, pulled in just over $2 billion in third-quarter sales this year. Fenebrutinib showed a “numerical benefit” over Ocrevus as early as 24 weeks, according to Roche. It hasn’t been smooth sailing for fenebrutinib, or for other BTK inhibitors for MS. In 2023, the FDA placed a partial clinical hold on fenebrutinib because of drug-related liver injuries in the Phase 3 RMS trials, specifically elevated levels of liver enzymes and bilirubin. The FENhance 2 and FENtrepid trials were already fully enrolled at that point, so they weren’t significantly impacted while Roche investigated two cases of elevated liver biomarkers. Those levels returned to normal after the patients stopped taking the drug. Roche said safety results from the latest studies are consistent with previous trials. Other large drugmakers have also faced liver-related holds for their candidates from the same class, including Merck KGaA and Sanofi . Merck KGaA decided to end development of evobrutinib in March 2024, several months after a Phase 3 failure in RMS. In September, the FDA delayed an approval decision for Sanofi’s tolebrutinib in non-relapsing, secondary progressive multiple sclerosis, citing a “major amendment” to the company’s NDA. The agency is now expected to make a decision by Dec. 28.