BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program

BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program

NOVATO, Calif. and OXFORD, England, July 22 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) and Summit Corporation plc (AIM: SUMM) announced today that they have entered into an exclusive worldwide licensing agreement for Summit's novel preclinical candidate SMT C1100 and all follow-on molecules, which are being developed to treat the fatal genetic disorder Duchenne muscular dystrophy (DMD).

Under the terms of the licensing agreement, Summit will receive an upfront payment of $7 million in the form of an equity investment in Summit shares, future development and regulatory milestones totaling $51 million, tiered royalties rising to the low teens, depending on sales and product sales milestones, giving a total deal value of up to $143 million. The total expense to BioMarin in 2008 is expected to be approximately $1.8 million.

Summit will be responsible for completing the preclinical development of SMT C1100. BioMarin will be responsible for the clinical development, regulatory filing and commercialization of the product candidate.

"We are pleased to work with Summit on the Duchenne muscular dystrophy program. SMT C1100, an oral small molecule utrophin upregulator, has shown promise in animal models of DMD and may have the potential for treating the entire spectrum of DMD patients, not just those with a particular type of mutation," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "The DMD indication aligns well with our growing product development pipeline as it is a genetic disorder with no approved treatments. IND-enabling studies with SMT C1100 are underway, and we plan on entering the clinic in 2009. By leveraging our expertise in rapidly developing and commercializing products for focused patient populations, we hope to soon provide a new treatment option for all DMD patients."

Steven Lee Ph.D., Chief Executive of Summit said, "We are very pleased that BioMarin has become our partner for the DMD program. BioMarin has an unparalleled track record in developing orphan drugs to market and has developed and launched successfully three such drugs in record time. The expertise and commitment of the BioMarin team gives me great confidence that they are an excellent partner for this program. I believe they will help to deliver SMT C1100 into a medicine in the shortest timeframe possible for the benefit of all DMD patients.

"For Summit, this deal is important as it is the first of many that we anticipate signing from our broad pipeline of assets including two clinical and two preclinical programs with future research driven by our world leadership in two innovative technology platforms. Our business strategy is focused on out-licensing or partnering candidates at a preclinical or early clinical stage, where there is a strong demand from pharma and biotech companies looking to enhance their own pipelines, and this deal provides important validation of this strategy."

The equity investment of $7.0 million (pounds Sterling 3.54 million) will be made at 69 pence per share, calculated from the 60-day trailing share price at the time issue, upon which BioMarin will hold approximately 9.16% of the enlarged share capital of Summit.

About Duchenne muscular dystrophy (DMD) and SMT C1100

Duchenne muscular dystrophy is a fatal neuromuscular disorder that affects 1 in 3,500 boys with an estimated patient population of over 40,000 in the developed world.

DMD is caused by a genetic defect meaning DMD patients lack an important protein called dystrophin, which is crucial to maintaining muscle integrity and function. The absence of dystrophin results in extensive muscle wasting in all voluntary muscles as well as the heart and breathing muscles and causes severe restriction in the mobility of DMD patients by their early teens and is ultimately fatal, generally in their twenties. Currently there is no cure for DMD; corticosteroid treatment is the only frontline therapy and acts to only delay the progression of the disease.

Summit has identified SMT C1100, a proprietary, orally available small molecule with a novel mechanism of action for DMD. SMT C1100 acts to modify the progression of DMD by replacing dystrophin with an endogenous, functionally similar protein called utrophin. Summit believes the primary advantage of SMT C1100 is that it offers the potential to treat the entire DMD patient population. Summit recently presented important preclinical data for SMT C1100 demonstrating significantly improvements in the strength and function of muscles in in vivo models.

Due to the low patient population and high unmet medical need, DMD is designated as an orphan indication by the regulatory agencies. Orphan products can expect to receive additional regulatory support and accelerated approval in addition to seven and ten years of market exclusivity in the US and EU respectively upon designation by the FDA and the EMEA.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 1 clinical development for the treatment of PKU. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

About Summit plc

Summit plc is a leading UK biotechnology company with a broad preclinical and clinical pipeline, two world-leading technology platforms and an innovative business model that is expected to generate sustainable value for investors. Summit is developing many drug programmes that target unmet medical needs from which it intends to generate value by out-licensing attractive late preclinical or early clinical stage programmes in return for upfront, milestone and royalty payments. Summit uses its scientific expertise to target orphan indications, neuro-disorders and infectious diseases. Summit's drug pipeline is supported by its world leadership in two innovative technology platforms: carbohydrate chemistry and zebrafish biology. These platforms support existing programmes and also will be the source of future programmes to refuel Summit's drug pipeline. These platform technologies also form the basis of the Company's revenue generating service business. The company listed on the alternative investment market (AIM) of the London Stock Exchange in October 2004 -- symbol: SUMM. Further information about the company is available at

Forward-Looking Statements


This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expected development of SMT C1100, the continued clinical development and commercialization of BioMarin's products and other product candidates and actions by regulatory authorities. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results of planned and ongoing preclinical and clinical trials, including preclinical trials of SMT C1100; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2007 Annual Report on Form 10-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

Summit plc

This press release contains "forward-looking statements" within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as "anticipates", "intends", "plans", "seeks", "believes", "estimates", "expects" and similar references to future periods, or by the inclusion of forecasts or projections.

Forward-looking statements are based on Summit Corporation plc's current expectations and assumptions regarding our business, the economy and other future conditions. Because forward-looking statements relate to the future, by their nature, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Summit's actual results may differ materially from those contemplated by the forward-looking statements. Summit cautions you therefore that you should not rely on any of these forward-looking statements as statements of historical fact or as guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include regional, national, global political, economic, business, competitive, market and regulatory conditions.

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