Celgene reported Monday that an experimental pill for the main type of multiple sclerosis met its main goals in a two-year study. By the end of the year, the Summit, NJ, drugmaker will ask the FDA to consider the drug, ozanimod, for approval.
The Celgene (NASDAQ: CELG) drug is being tested in patients with relapsing multiple sclerosis (MS), which accounts for about 85 percent of all initial U.S. cases, according to the National Multiple Sclerosis Society. Relapsing MS is characterized by neurological attacks that flare up and go into remission.
If ozanimod is approved, it would mark the second major MS approval in as many years.
This March, the FDA approved ocrelizumab (Ocrevus), which was developed by Roche subsidiary Genentech. The approval covered primary progressive MS–the first-ever approval for patients whose conditions progresses, or worsens, without remission–as well as the relapsing form of the disease.
The Novartis drug, fingolimod (Gilenya), goes off patent in 2019; analysts are waiting for more data from Celgene later this year to determine if Celgene’s drug can be competitive.
The clinical trial results for Celgene’s MS drug weren’t entirely surprising; earlier this year, the drug developer released positive data from a separate Phase 3 study. The Celgene drug reduced the relapse rate in MS patients in both trials, beating a Biogen treatment, the injectable interferon beta-1a (Avonex).
The current trial, dubbed RADIANCE, and other studies for ozanimod, also evaluated how long the drug can stave off a patient’s decline into disability. Those results weren’t enough to distance the Celgene pill from treatment with interferon. In a research note, Leerink Partners analyst Geoffrey Porges called the lack of a disability benefit “disappointing” but did not think it would blunt the drug’s chances for approval.
“However, it does limit the eventual differentiation… against competitor Gilenya and its generic, which includes a disability benefit against placebo in its label,” wrote Porges.
RADIANCE tested two doses of the Celgene pill, 0.5 mg and 1 mg, in patients with the relapsing form of MS. The two-year trial, which enrolled 1,313 patients, compared the Celgene drug to treatment with interferon. Celgene says that both doses of its drug hit the goals of reducing the annualized relapse rate of MS compared to treatment with interferon. The company says its drug also hit secondary goals of reducing the lesions observed on the brain, both the number of total number of lesions, as well as the number of inflammatory lesions that are associated with the relapsing form of MS.
Celgene also says that both doses of its drug showed statistically significant reductions in brain atrophy compared to interferon. But the Celgene drug failed to show statistically significant improvement in disability across all three treatment groups.
In a research note, Evercore ISI analyst Umer Raffat wrote that expectations around the disability endpoint of the Celgene drug were misplaced. Novartis’ fingolimod never demonstrated a statistically significant disability benefit compared to an already available treatment like interferon, he wrote.
Barclays analyst Brittany Berliner views the ability of the Celgene drug to differentiate itself on safety and tolerability from fingolimod, or any generic versions of the Novartis drug, as key to its success. In relapsing MS, “the possibility of another oral option should be generally well received by neurologists compared to an injectable like Avonex, but we anticipate greater initial uptake from currently relapsed patients who are candidates for a switch and more gradual conversion from otherwise stable patients,” Berliner wrote.
Celgene added ozanimad to its drug pipeline through its 2015 acquisition of San Diego-based Receptos in a deal valued at up to $7.2 billion. Celgene was eager to branch out beyond blood cancer therapies and establish itself in inflammatory and autoimmune disorders. In 2014, Celgene received FDA approval for apremilast (Otezla), a drug developed to treat psoriatic arthritis. Celgene has also reported promising early-stage clinical trial results for a Crohn’s disease drug candidate acquired from Nogra Pharma.