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BMRN: BioMarin to Acquire Rights to Phenylketonuria (PKU) Franchise From Merck Serono
http://investors.bmrn.com/releasedetail.cfm?ReleaseID=934634

CC Link 8:30am ET: http://edge.media-server.com/m/p/4suk47qn

Strengthens and Expands BioMarin's Leadership Position Serving PKU Patients with Rights to Both Kuvan® (sapropterin dihydrochloride) and Pegvaliase Beyond North America

2016 Full-year Guidance for Kuvan Expected to be Between $320M-$350M including $70M-$80M in New Revenue from ROW Territories

Starting January 1, 2016 BioMarin will Commercialize and Record Sales of Kuvan in an Additional 55 Countries Already Served by BioMarin

Investor Call to be Held Today, October 1 at 8:30am ET/5:30am PT

SAN RAFAEL, Calif., Oct. 1, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), today announced that it has acquired all global rights to Kuvan® (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Under the terms of the agreement, BioMarin will provide Merck with an upfront payment of €340 million. An additional €60 million in milestones will be paid to Merck if combined sales of Kuvan and pegvaliase reach undisclosed cumulative sales thresholds. In addition, €125 million will be paid to Merck for regulatory milestones related to pegvaliase. Previously, BioMarin had exclusive rights to Kuvan in the United States and Canada and to pegvaliase in the United States and Japan. Under the terms of the transaction, BioMarin will now have exclusive worldwide rights to Kuvan and pegvaliase with the exception of Kuvan in Japan. Approved in 2007 in the U.S., Kuvan is a commercialized product for the treatment of patients with phenylketonuria (PKU). Pegvaliase is currently in registration-enabling pivotal studies as a potential therapeutic option for adult patients with phenylketonuria. With the potential approval of pegvaliase, the two products combined will expand and globalize BioMarin's leadership position by offering a wider range of treatment options to patients worldwide with PKU.

In 2005, Merck acquired from BioMarin the exclusive rights to Kuvan and the option to develop pegvaliase in markets outside of the U.S. and Japan. By regaining these rights to both products, BioMarin has the opportunity to expand its commercial efforts across the Company's global territories. Kuvan is currently sold by Merck in many countries where BioMarin has a commercial presence for both Naglazyme® (galsulfase) and Vimizim® (elosulfase alfa). Kuvan has Orphan Drug exclusivity in Europe until 2020. Upon closing of the transaction in January 2016, BioMarin will record all sales and profits of Kuvan.

"This is an excellent transaction for BioMarin as it provides numerous operational and strategic synergies for the Company," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We will leverage our established worldwide infrastructure and strong relationships within the PKU community to ensure that patients globally have access to Kuvan, and potentially pegvaliase upon approval. We look forward to expanding our PKU franchise beyond the US and Canadian markets and into our existing commercial footprint of about 60 countries where Kuvan is currently sold."

2016 Full-year Kuvan Guidance

Starting in January 2016, BioMarin will begin commercializing and recording revenue from all global sales of Kuvan, with the exception of Japan. The addition of new Kuvan revenue in 2016 from territories previously held by Merck will result in increased full-year revenue for BioMarin. Revenue from new ROW territories outside of the US is expected to be between $70 million to $80 million in 2016. Total Kuvan revenue to BioMarin for full-year 2016 is expected to be between $320 million and $350 million, including revenue from new ROW territories. BioMarin expects to transition commercialization activities from Merck over the next 6-12 months, and will evaluate opportunities to leverage its global commercial infrastructure to drive further growth of Kuvan in these new territories. The transaction is expected to be accretive to non-GAAP earnings beginning in 2016 and accretive to GAAP earnings in 2018.

Conference Call Details

BioMarin will host a conference call and webcast to discuss the acquisition of the global PKU franchise from Merck Serono on October 1 at 8:30am ET/5:30am PT. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.

U.S. / Canada Dial-in Number: 877.303.6313
International Dial-in Number: 631.813.4734
Conference ID: 50222632

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 50222632
About Phenylketonuria (PKU)

Phenylketonuria (PKU) is an autosomal recessive genetic disorder caused by a defect or a deficiency of the enzyme phenylalanine hydroxylase (PAH) or its cofactor tetrahydrobiopterin (BH4). PAH is required for the metabolism of phenylalanine, an essential amino acid found in all protein-containing foods. The prevalence of PKU in BioMarin global commercial territories is estimated to be approximately 50,000. If PKU patients are not treated with a phenylalanine-restricted diet, phenylalanine will accumulate in the blood and brain to abnormally high levels, thereby resulting in a variety of complications including mental retardation and brain damage, mental illness, seizures and tremors, and clinically significant cognitive problems. Universal systematic newborn screening programs were developed in the 1960s and early 1970s to enable diagnosis of all patients with PKU patients at birth.