Business, Drug Development

Celgene works to calm down anxious investors, promising to re-file ozanimod at the FDA in early 2019

For weeks now, analysts have been fretting over the future of Celgene’s $CELG multiple sclerosis drug ozanimod. Turned away at the FDA’s front door with a refuse-to-file letter, their attention focused on an active metabolite of the drug and speculation over whether or not the big biotech would be forced to run a new human study to satisfy regulators that would set them back years.

Mark Alles

This morning, Celgene execs went to some pains to explain that the delay would stretch out only to Q1 2019 as researchers undertook some bridging non-clinical studies of the drug. No lengthy human studies would be needed, the company added. And they would hustle along a European application alongside the FDA pitch in the first few months of next year.

The reason for the delay: “Ozanimod is metabolized in humans to form one major active metabolite (CC-112273) and other minor active metabolites,” the company reported. And that metabolite has a long half life of 10 to 13 days.

Evercore ISI analyst Umer Raffat sees this as a positive. He notes:

If this metabolite was a small % of overall ozanimod AUC, that would be a reasonable conclusion.  However, we learned that this metabolite is ~90% of AUC in humans… that’s key … because what that means to me is that this metabolite is effectively the drug as we know it.  Said another way, all the Ph 2/Ph 3 studies we saw on ozanimod was effectively this metabolite.  For that reason, it seems that human studies should not be needed and that’s what CELG is guiding.

Of course, if you were looking for a quicker turnaround, the glass at Celgene could look more half empty right now. Noted Geoffrey Porges:

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. Celgene announced that the ozanimod resubmission plan will include bridging non-clinical studies and additional analysis of existing PK/PD data, but emphasized that additional human clinical efficacy and safety studies are not needed.

The drug is a central part of CEO Mark Alles’ case that the company has a bright future ahead of it, capable of earning $4 billion to $6 billion. Alles has been racking up a series of new deals, buying Juno for $9 billion, and revamping the company’s command structure as a series of embarrassing snafus raised questions about a company that has long been admired for its ability to execute quickly and efficiently.

Celgene has little margin for error now. Any new slips will likely be severely punished.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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Flatiron Health New York City or San Francisco

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