OXFORD, U.K.—Summit Corp. plc and California-based BioMarin Pharmaceutical Inc. announced late last month they the companies inked 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, while 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," says Jean-Jacques Bienaimé, CEO of BioMarin. "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. 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."
DMD is a fatal neuromuscular disorder that affects 1 in 3,500 boys with an estimated developed world patient population of more than 40,000. DMD is caused by a genetic defect whereby 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.
Steven Lee Ph.D., CEO of Summit notes, "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," Lee says. DDN