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Summit Therapeutics plc

Drug Names(s): BMN 195, SMT C1100

Description: BMN 195 is an orally available small molecule inducer of utrophin gene expression, a naturally occurring homologue gene of dystrophin that is normally only expressed in fetal muscle. The goal of therapy would be to preserve muscle function and prevent the inexorable decline in strength seen in Duchenne muscular dystrophy (DMD) patients.

Deal Structure: BioMarin and Summit
In July 2008, BioMarin Pharmaceutical and Summit announced that they have entered into an exclusive worldwide licensing agreement for SMT C1100 and all follow-on molecules, which are being developed to treat 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. 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.

In August 2008, BioMarin decided to discontinue development of BMN 195 for DMD. Following BioMarin's decision, all intellectual property and programme rights to BMN 195 have been...See full deal structure in Biomedtracker

Partners: Sarepta Therapeutics, Inc.

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