The last weekday of June saw a pair of high-valuedeals, with Inhibrx LLC announcing a worldwide option and license agreementwith Celgene Corp. for an antibody program against an undisclosed target, whileBiogen Idec Inc. and Isis Pharmaceuticals Inc. revealed that they have enteredinto collaboration under which they will develop and commercialize a novelantisense drug for the treatment of myotonic dystrophy type 1 (DM1), which isalso known as Steinert disease.
The total deal value for the Inhibrx-Celgene deal potential could eventually exceed $500 millionafter all the upfront, clinical and regulatory milestone payments are tallied,with Inhibrx also eligible to receive royalties on commercial sales.
However, the companies have been very mum on otherdetails. Dr. Tom Daniel, president of research and early development forCelgene, says simply, "Inhibrx has developed an antibody with strong preclinicalstudy results on a highly validated target with very promising therapeuticpotential."
From the Inhibrx side, the company's president, MarkLappe, says only, "We are pleased to have a company of Celgene's caliber as thelicensee of this program. On a global basis, Celgene has the science, clinical,regulatory and commercial expertise as well as the commitment to patients thatcontinue to deliver disease-altering solutions to patients in need as quicklyas possible."
Meanwhile, with a potential deal value of as muchas $271 million, Biogen Idec and Isis said they have signed an exclusive,worldwide option and collaboration agreement to come up with a treatment for DM1,the most common form of muscular dystrophy in adults.
The genetic neuromuscular disease characterized byprogressive muscle atrophy, weakness and disabling muscle spasms. It is causedby a genetic defect in the dystrophia myotonica-protein kinase (DMPK) gene inwhich a sequence of three nucleotides repeats extensively, creating anabnormally long toxic RNA, which accumulates in the cell and prevents theproduction of proteins needed for normal cellular function. Isis' DM1 antisenseprogram is being developed to correct the underlying genetic defect that causesDM1.
Isis will receive an upfront payment of $12million and is responsible for the discovery of a lead antisense drug candidatetargeting DMPK for the treatment of DM1. Isis is eligible to receive up to $59million in milestone payments associated with the clinical development of theDMPK-targeting drug prior to licensing. Biogen Idec has the option to licensethe drug from Isis up through the completion of the Phase II trial. Isis couldreceive up to another $200 million in a license fee and regulatory milestonepayments. In addition, Isis will receive double-digit royalties on sales of thedrug.
Isis will be responsible for global development ofthe drug through the completion of Phase II clinical trials, with Biogen Idecproviding advice on the clinical trial design and regulatory strategy. IfBiogen Idec exercises its option, it will assume global development, regulatoryand commercialization responsibilities.
"Myotonic dystrophy is a debilitatingneuromuscular disease that often affects entire families," said Steven H.Holtzman, executive vice president of corporate development at Biogen Idec."The unmet need is great, and there are currently no therapies to slow orstop progression of the disease. Myotonic dystrophy has an identifiable geneticcause, the program fits with our mission to bring innovative therapies topatients with serious neurologic diseases, and Isis' antisense compound has thepotential to make a real difference. This collaboration, which is our secondwith Isis, reflects the tremendous respect we have for their scientificleadership and expertise in antisense technology."
"Biogen Idec is a world leader inneurodegenerative diseases," added B. Lynne Parshall, chief operatingofficer, chief financial officer and secretary for Isis. "This collaborationallows us to expand our pipeline of drugs for rare and severe diseases withBiogen Idec's additional resources and support. It also complements our newalliance with Biogen Idec for our Phase 1 program in spinal muscular atrophy,or SMA. As with SMA, we are using our antisense technology in a unique mannerto treat another devastating disease. Biogen Idec is an ideal partner for theseprograms with its expertise in neurodegenerative disease and global reach tohelp bring these therapies successfully to patients who have no treatmentoptions."
