BioMarin Pharmaceutical Inc. announced that it has entered into a stock purchase agreement to acquire LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers.

"The acquisition of LEAD Therapeutics will augment our development pipeline of orphan therapeutics. With LT-673 we see a tremendous opportunity to apply our expertise in developing therapeutics for genetic diseases to the field of oncology by targeting cancers with defined genetic mutations that make them susceptible to treatment with agents such as LT-673. There are attractive opportunities to treat rare cancers with PARP-sensitive mutations, as a single agent and in combination with other DNA damaging agents, and to improve on PARP inhibitors currently in development for more common tumour types," said Jean-Jacques Bienaime, chief executive officer of BioMarin. "We remain centrally focused on our core commercial business, the launch of amifampridine for LEMS in Europe and our phase 2 PEG-PAL and GALNS development programmes. We can now also look forward to an IND filing for LT-673 by the end of 2010."

Hank Fuchs, M.D., executive vice president and chief medical officer of BioMarin added, "PARP inhibitors have shown impressive survival benefits in cancer patients with tumours that have defects in DNA repair or in combination with DNA damaging agents. There are many opportunities to achieve selective lethality with PARP inhibitors in both rare and more common cancers. LT-673 has been proven to be highly active in mouse xenograft models of human cancer and appears to have favourable properties, including potency, selectivity, and bioavailability."

Under the terms of the stock purchase agreement, BioMarin will pay to the stockholders of LEAD $18 million upfront and will pay an additional $11 million upon acceptance of the IND filing (filing expected by the end of 2010), and up to $68 million for development and launch milestones for LT-673. As a result of this acquisition, BioMarin expects to incur approximately $11.0 to $13.0 million in operating expenses and acquisition related charges in 2010. Subject to customary closing conditions, the acquisition is expected to be completed by mid-February 2010.

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates.

LEAD Therapeutics began operations in April 2007 as a drug discovery company. LEAD has offices in San Bruno, California, and in Shanghai, China. Drug discovery programmes are conducted through an extensive network of collaborations with contract research organizations in China and North America, including a strategic relationship with ChemPartner in Shanghai.