- Celgene intends to file ozanimod for approval during the first quarter of 2019, a timeline for submission that generated mixed reactions among investors worried about the highly anticipated multiple sclerosis drug.
- Analysts saw ozanimod as a potential blockbuster, but expectations wavered after the Food and Drug Administration decided it wouldn't review the drug's approval application without more nonclinical and clinical pharmacology data. On a Friday call, Celgene's Chief Medical Officer Jay Backstrom confirmed the agency based its decision on a key metabolite.
- The ozanimod updates were tucked in with Celgene's first quarter earnings presentation. Revenue ticked up 19% year over year to $3.54 billion as all four of the biotech's biggest brands — Revlimid, Otezla, Pomalyst and Abraxane — beat consensus sales estimates.
A resubmission early next year isn't the worst outcome for ozanimod. Drug applications that receive a Refuse to File letter can experience years of delays before gaining the FDA's stamp of approval. And given recent guidance from the agency, Celgene could have been tasked with collecting a lot more pre-clinical data than regulators now appear to want.
In an investor note, Mizuho analyst Salim Syed acknowledge the expected filing time "is in-line with the average in our buyside survey, and way better than a 2-3 year delay which is what some Street observers were anticipating."
That doesn't mean Friday's announcement is without its downsides.
The timing setbacks mean that even with a thumbs up from regulators, ozanimod almost surely won't come to market until 2020 — a development that could have lasting repercussions for Celgene's value. Investment bank Leerink has a price target of $123 per Celgene share, but that hinges on a quicker ozanimod launch.
"This new timeline is three quarters behind what we have forecast and may put the commercial launch in some uncertainty given the expected entry of generic Gilenya," Leerink analyst Geoffrey Porges wrote in a May 4 note.
Celgene's new plan follows a Type A meeting, which focuses on stalled drug development or safety issues, it had with the FDA in April. The biotech anticipates conducting bridging studies to fulfill the agency's data requirements, and aims to have pre-New Drug Application meetings later this year.
"Where we are currently is we're making sure that we have the opportunity to demonstrate that we have adequate exposure through that bridging ... so that at the end the non-clinical reviewer feels that they have sufficient data," Backstrom said.
The intense focus on ozanimod stems from Celgene's dependence on its blood cancer drug Revlimid (lenalidomide), which accounts for more than 60% of revenue.
As such, the biotech has been under significant pressure to expand and diversify its portfolio. Recent acquisitions of Juno Therapeutics and Impact Biosciences added a slate of drugs to Celgene's pipeline, but most won't have the chance to enter the market for some time.
The stalled progress of ozanimod isn't hampering larger neuroscience interest, however. Just two months ago, Celgene dropped $100 million in a preclinical-stage deal with Prothena that aims to develop novel neuroscience and orphan disease therapeutics.
"In MS, we have a full commitment to optimize ozanimod, and then beyond that we're being very very thoughtful and programmatic in how we understand a broader, deeper approach to neurosciences," CEO Mark Alles said on the call.