FDA Advisors Back Regeneron, Sanofi's New Heart Drug With Limits

Published: Jun 11, 2015

DA Advisors Back Regeneron , Sanofi's New Heart Drug With Limits
June 10, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

Tarrytown, N.Y.-based Regeneron Pharmaceuticals, Inc. fell almost 17 percent in trading Wednesday, the day after the U.S. Food and Drug Administration (FDA) recommended a limited approval for the company’s application for cholesterol lowering drug Praluent (alirocumab).

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met yesterday to discuss the company's Biologics License Application. The Committee subsequently voted 13 to three (with no abstentions) that Regeneron and Sanofi “had sufficiently established that the low-density lipoprotein cholesterol (LDL-C, or bad cholesterol) lowering benefit of Praluent exceeds its risks to support approval in one or more patient populations.”

Praulent has been developed in partnership with Paris-based Sanofi. It was named one of five “summer blockbuster” drugs likely to come out this year.

The company said in a statement emailed to BioSpace late Tuesday that it was excited about Praulent’s future going forward.

"We are pleased with the Committee's recommendation to approve Praluent. Our clinical trial program focused on patients with high unmet need in which Praluent delivered significant reductions in LDL-C on top of statins and other lipid-lowering therapies," said Elias Zerhouni, president of Global R&D, Sanofi. "Our Phase III Praluent development program investigated both a 75 mg and 150 mg dose, providing flexible dosing regimens that can be tailored to individual patient cholesterol lowering needs."

The drug has also been accepted for review via a Marketing Authorization Application in the European Union. The drug is a monoclonal antibody that targets proprotein convertase subtilisin/kexin type I (PCSK9) and was studied in more than 5,000 patients, including 10 Phase III ODYSSEY trials.

Analysts were sanguine about the news, saying in notes to investors late Tuesday and this morning that what the panel said was just as important as how it said it.

“Despite lively discussion on LDL lowering as a surrogate endpoint for outcomes for this new class of drug, the panel felt ultimately that the bar for drug approval (LDL lowering) should not be changed at this time, that the genetics support the anti PCSK9 LDL lowering mechanism, and that the FH population and those with high CV risk represent an unmet medical need,” wrote Mark Schoenebaum, an analyst with ISI Evercore.

“On safety the panelists agreed that, despite no major safety signals, available data are not adequate to resolve potential concerns regarding adverse events (e.g. diabetes, neurocog, immunogenicity) and the safety of achieving very low LDL levels,” said Schoenebaum. “Therefore, the panelists acknowledged that the ongoing 18,000 patient outcomes trial will be important for addressing these outstanding questions.”

Geoffrey Porges, a closely watched analyst with Sanford Bernstein, said in a note titled “Curb Your Enthusiasm - AdCom Takes Cautious Approach to Scope of Initial Indication, Limited Downside” that the FDA will likely approve the drug but possibly in narrowed circumstances.

The AdCom proved much more conservative on safety and on the acceptability of LDL as a surrogate endpoint, with 50 percent of members proposing the drug either should not be approved or should be approved only in HeFH,” said Porges.

“We ultimately expect approval beyond HeFH, in the high risk not-at-goal population, which would result in limited changes to our current market model. A narrower label would dent forecasts, however, and the overhang will undermine the stock near term.”

When Will Pfizer's Breakup Happen?
Speculation that the revamping of Pfizer Inc. ’s internal business structure could happen as soon as this year has biotech wondering just when this Big Pharma company could see changes.

Last week an analyst with J.P. Morgan said he thinks there will be a much faster timeline than most of Wall Street had predicted for Pfizer’s stated mission to refocus its efforts on new medicines.

Pfizer initially announced in 2012 that it would be shedding units that were non-essential to that goal. It then promptly sold its nutrition silo to Nestle for $11.85 billion, which was rapidly accompanied by a public spin-off of its animal health business for $2.2 billion.

“While a Pfizer break-up would likely be a 2017 event, we see potential catalysts in 2015-2016," said Chris Schott, an analyst at J.P. Morgan. "Three years of audited financial statements (2014-2016) are required before any part of Pfizer can be spun off, and we also see 2017 as an attractive time for action as investors see Pfizer’s innovative pipeline clearly contributing to growth and the established business having transitioned to a more stable profile."

BioSpace wants to know what you think: Will Pfizer be a changed company by the end of 2015?

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