Voucher leapfrog

Sanofi, Regeneron could leapfrog Amgen in PCSK9 race with Priority Review voucher

Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) are hoping to use a newly acquired Priority Review voucher to jump ahead of Amgen Inc. (NASDAQ:AMGN) in the race to be first to market with a PCSK9 program.

Last Wednesday, rare disease company BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) sold a Priority Review voucher to Regeneron and Sanofi for $67.5 million, which the partners said they would use to obtain Priority Review for a BLA for alirocumab, a human mAb targeting proprotein convertase subtilisin/kexin type 9, to treat primary hypercholesterolemia. According to Sanofi spokesperson Elizabeth Baxter, it is the first ever purchase of a Priority Review voucher.

BioMarin gained the voucher earlier this year upon FDA's approval of Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). BioMarin was the first - and so far only - company to receive a voucher under the agency's Rare Pediatric Disease Priority Review Voucher program, which was created by PDUFA V to encourage the development of new drugs and biologics to prevent and treat rare pediatric diseases (see BioCentury, Oct. 12, 2012).

Also last Wednesday, Sanofi and Regeneron announced top-line data showing that alirocumab met the primary endpoint in nine Phase III trials. But the partners might be behind in terms of a U.S. regulatory submission. Amgen plans to submit a BLA for evolocumab in 3Q14, while Sanofi has only said it plans to submit its BLA before year end.

Thus a worst case scenario for Sanofi and Regeneron would see Amgen submit its BLA in early August and the partners submit their BLA just before year end, giving Amgen about a five-month head start. If that occurred, the Priority Review voucher would close the gap to about one month.

The best-case scenario would see Sanofi and Regeneron leapfrog Amgen. If Amgen submits the evolocumab BLA at the end of 3Q14, and Sanofi and Regeneron submit in early 4Q14, the partners could have an almost four-month head start.

Although Sanofi wouldn't disclose how it arrived at the voucher valuation, the partners must calculate that earlier revenues plus the potential first-mover advantage is worth more than $67.5 million.

The pediatric Priority Review voucher is a pilot program that sunsets one year after the third voucher is awarded.

Separately, FDA approved Regeneron's Eylea aflibercept to treat diabetic macular edema (DME).

Regeneron rose $21.22 to $324.31 last week. BioMarin added $1.35 to $61.95.

Tables

Earnings on Deck
Company Date Pre/post mkt 2Q14 EPS est 2Q13 EPS Expected chg
Ligand Pharmaceuticals Inc. (NASDAQ:LGND) 8/4 Pre $0.14 $0.07 100%
Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) 8/5 Pre $2.28 $0.79 189%
Bio-Rad Laboratories Inc. (NYSE:BIO) 8/5 Post $0.95 $1.20 -21%
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) 8/5 Post $1.92 $1.43 34%
Bruker Corp. (NASDAQ:BRKR)

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