Celgene Corporation (NASDAQ:CELG) announced  on 4/24/18 additional phase III data analyses evaluating the efficacy and safety of ozanimod, a novel, oral, selective sphingosine 1-phosphate 1 (S1P1) and 5 (S1P5) receptor modulator, versus interferon beta-1a (IFN β-1a) (Avonex®) in patients with relapsing multiple sclerosis (RMS).

The phase III SUNBEAM and RADIANCE Part B studies evaluated two doses of oral ozanimod (1 mg and 0.5 mg ozanimod HCl) compared with IFN β-1a in patients with RMS. In the new analyses, several pre-specified subgroups, including disability severity at baseline (EDSS ≤3.5 vs. EDSS >3.5), presence of gadolinium-enhanced lesions at baseline and prior treatment with disease-modifying therapies, were assessed. Data from these analyses, to be presented in an oral session on April 25, show dose-dependent effects of ozanimod on annualized relapse rates (ARR) versus IFN β-1a across these subgroups that were consistent with the primary endpoint of both SUNBEAM and RADIANCE Part B.

A pair of poster presentations on April 24 examined reductions in brain volume loss, a measure associated with multiple sclerosis (MS) disease progression, for ozanimod compared with IFN β-1a. As previously reported for SUNBEAM at one year and for RADIANCE Part B at two years, whole brain volume loss was reduced relative to IFN β-1a for both the 1 mg dose of ozanimod and the 0.5 mg dose. For both doses, all comparisons were nominally significant.

Additional data in the presentations from exploratory endpoints examined volume loss of specific segments of the brain. Increasing evidence suggests that disease-related damage to grey matter is of major importance in MS. The data analyses to be presented show reductions in cortical grey matter loss and thalamic volume loss are consistent with the reductions in whole brain volume loss seen in SUNBEAM at one year for ozanimod compared with IFN β-1a. Likewise, reductions in cortical grey matter loss and thalamic volume loss are consistent with the reductions in whole brain volume loss seen in RADIANCE Part B at two years.

The FDA rejected Celgene's application for ozanimod in February of 2018 due to insufficient data.  If approved, ozanimod will likely launch sometime in late 2019.

Ozanimod is a novel, oral, selective, sphingosine 1-phosphate 1 (S1P1) and 5 (S1P5) receptor modulator in development for immune-inflammatory indications including relapsing multiple sclerosis, ulcerative colitis and Crohn's disease.

Selective binding with S1P1 is believed to inhibit a specific subset of activated lymphocytes from migrating to sites of inflammation. The result is a reduction of circulating T and B lymphocytes that leads to anti-inflammatory activity. Importantly, immune surveillance is maintained.

Selective binding with S1P5 is thought to activate specific cells within the central nervous system (CNS). This has the potential to enhance remyelination (when the body is able to repair damaged myelin after inflammation is reduced) and prevent synaptic defects. Ultimately, neurological damage may be prevented.