- Celgene's experimental multiple sclerosis drug met its main objective in the second of two large Phase 3 study, notching a statistically significant reduction in annualized relapse rates compared to Biogen's commonly used Avonex (interferon β-1a).
- But the drug, known as ozanimod, failed to slow disability progression any better than Avonex, according to a pooled analysis of the two studies. As event rates were low in both the treatment and comparator arms, a statistically significant improvement would have been a powerful finding in favor of ozanimod.
- Celgene plans to submit ozanimod for U.S. approval by the end of this year and expects the drug could become a future blockbuster across multiple indications. Mid- to late-stage studies testing ozanimod in ulcerative colitis and Crohn's disease are currently underway.
Success with ozanimod would go a long ways to validating Celgene's $7.2 billion acquisition of Receptos, Inc. back in 2015, which gave Celgene access to the drug.
While Celgene did not reveal detailed data, ozanimod met both its primary and key secondary endpoints in the RADIANCE study — results which were in line with what was reported from the earlier, also positive SUNBEAM study.
Both trials compared two oral doses of ozanimod to intramuscular injections of Biogen's Avonex. The 0.5 mg and 1 mg doses each reduced annualized relapse rates as well as new or enlarging T2 MRI brain lesions and gadolinium-enhancing MRI lesions over two years of treatment.
RADIANCE enrolled 1,313 patients with relapsing multiple sclerosis, the most common type of the debilitating autoimmune disease.
Success in both studies bodes well for ozanimod's chances for winning U.S. approval, although failure to show a slowing of disability progression represents a missed opportunity for differentiation in a crowded therapeutic field.
Still, Celgene executives had primed analysts not to expect a hit on that measure.
"What we would expect here is not necessarily a p-value [showing statistical significance] here but clinically meaningful changes in that disability index," said Scott A. Smith, chief operating chief at Celgene, on a April 27 earnings call. "All the competitors have the disability data in their inserts, whether or not [the result] was significant or not."
If approved, ozanimod would compete with the likes of Novartis' Gilenya (fingolimod), Teva's Copaxone (glatiramer acetate) and Biogen's Tysabri (natalizumab).
Celgene executives believe ozanimod will be differentiated from Gilenya on an improved safety profile across a range of measures, including cardiac and liver toxicity.
Yet Celgene will also face a changing market. Generic copies to Gilenya, which modulates a similar pathway as ozanimod, could enter the market as soon as 2019, and copycat versions of high-dose Copaxone are also looming. Cheaper versions of the well-known drugs may pose challenges to uptake for ozanimod, if approved.
At the same time, Roche recently won approval for its closely watched Ocrevus (ocrelizumab), which is the first drug approved to treat the more severe, primary progressive form of MS, as well as the relapsing type. Priced at a competitive $65,000 annual list price, the drug could prove threatening to ozanimod sales as well.