FDA: Rejects NDA filing: “clinical and non-clinical pharmacology sections of the application were not sufficient to complete a review”: Celgene’s Relapsing Multiple Sclerosis Drug – Ozanimod
Reporter: Aviva Lev-Ari, PhD, RN
Celgene Provides Regulatory Update on Ozanimod for the Treatment of Relapsing Multiple Sclerosis
Conference call scheduled for today at 5:30 p.m. ET
SUMMIT, N.J.–(BUSINESS WIRE)– Celgene Corporation (NASDAQ:CELG) today announced that it has received a Refusal to File letter from the United States Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for ozanimod in development for the treatment of patients with relapsing forms of multiple sclerosis. Ozanimod is a novel, oral, selective sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator.
Upon its preliminary review, the FDA determined that the nonclinical and clinical pharmacology sections in the NDA were insufficient to permit a complete review. Celgene intends to seek immediate guidance, including requesting a Type A meeting with the FDA, to ascertain what additional information will be required to resubmit the NDA.
“We remain confident in ozanimod’s clinical profile demonstrated in the pivotal program in relapsing forms of multiple sclerosis,” said Jay Backstrom, M.D., Chief Medical Officer and Head of Global Regulatory Affairs for Celgene. “We will work with the FDA to expeditiously address all outstanding items and bring this important medicine to patients.”
Conference Call Information
Celgene will hold a conference call to discuss this update today at 5:30 p.m. ET. The conference call may be accessed by dialing 1-866-428-9517 for U.S.callers and 1-224-357-2194 for international callers. The passcode for the call is 9179457. The call can also be accessed via an audio webcast in the Investor Relations section of the company website at www.celgene.com. An audio replay will be available through March 6, 2018 by calling 1-855-859-2056 or 1-404-537-3406 and entering access code 9179457.
About Ozanimod
Ozanimod is a novel, oral, selective, sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator in development for immune-inflammatory indications, including relapsing multiple sclerosis, ulcerative colitis and Crohn’s disease. Selective binding with S1PR1 is believed to inhibit a specific sub set 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 S1PR5 is thought to activate specific cells within the central nervous system (CNS). This has the potential to enhance remyelination and prevent synaptic defects. Ultimately, neurological damage may be prevented.
Ozanimod is an investigational compound that is not approved for any use in any country.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next‐generation solutions in protein homeostasis, immuno‐oncology, epigenetics, immunology and neuro‐inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, Facebook and YouTube.
Forward-Looking Statements
This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the U.S. Securities and Exchange Commission.
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Celgene
Investors:
Patrick E. Flanigan III, 908-673-9969
Corporate Vice President, Investor Relations
or
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Catherine Cantone, 908-897-4256
Senior Director, Corporate Communications
Source: Celgene Corporation
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DIVE INTERPRETATION
Celgene’s ozanimod hit with Refusal to File in latest
Dive Brief:
- Celgene Corp. disappointed investors Tuesday night when the big biotech unexpectedly disclosed a Refusal to File letter from the Food and Drug Administration for its New Drug Application (NDA) for the multiple sclerosis drug ozanimod.
- The FDA said the clinical and non-clinical pharmacology sections of the application were not sufficient to complete a review. Celgene intends to request a type A meeting with the agency to better understand what is required for filing.
- While the company did not give exact guidance, analysts expect this could push back launch timelines of ozanimod by as much as a year, shifting the drug launch from 2018 to 2019.
Ozanimod, an oral, selective sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator being studied in multiple sclerosis, ulcerative colitis and Crohn’s disease, was meant to be a key driver for Celgene in the post-2020 time period. Some analysts had previously projected peak sales above $2.75 billion for the drug, but Mizuho analyst Salem Syed speculates this could jeopardize as much as $1 billion in sales.
The big biotech has been struggling to diversify its pipeline and has been in desperate need of a win. It already reported an expensive failure in October 2017 for the Crohn’s disease drug that it acquired for $710 million in 2014. And the patent situation around its blockbuster Revlimid (lenalidomide) has been shaky at best, threatening nearly $8.1 billion in revenues. Celgene made two major acquisitions in the first weeks of the year, picking up Impact Biomedicines for $7 billion and then Juno Therapeutics Inc. for $9 billion — but neither acquisition seems enough to stop the bleeding.
Investors were not pleased with the news, with the stock dropping nearly 8% in morning trading on Wednesday to about $89 per share. When Mark Alles took over as CEO in January 2016, the stock was trading above $100 apiece.
“This is yet another blow to investor (and analyst) confidence in this management team and their stewardship of the company’s key operational activities,” wrote Leerink Partners Geoffrey Porges in a note to clients.
Alles said on a Tuesday evening call with analysts said the recent acquisition and near-term catalysts from Juno’s lead drug JCAR17 would give the company the buffer it needs through 2020, and that Celgene is reaffirming its sales guidance for the 2020 time period.
Yet, the company wasn’t able to give much clarity on how this will impact the ozanimod filing in MS and said it will likely create delays with its European filing as well. Analysts worried that the filings of ozanimod for ulcerative colitis and Crohn’s could be delayed — although Alles noted the ulcerative colitis program would not be affected at this time.
“We certainly appreciate the events of the last eight months have caused investors a lot of concern and that is reflected in the value of the company,” said Alles on the call. “The complexity and some of the disappointments make it difficult to be predicting our future,” he added, but emphasized the company would bring in $19–20 billion in topline revenues in 2020.
Refusal to File letters were not a commonly used tool by the FDA, but the new administration under Scott Gottlieb seems to be tapping the tool more often of late.
Several other companies have gotten a no-go on applications recently as well, including Acorda Therapeutics for its Parkinson’s disease drug, Allergan for schizophrenia drug Vraylar (cariprazine), and SteadyMed Ltd. for a blood pressure medication. While the letters are not considered a rejection, they often delay applications and have derailed other drugs in the past.
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