Biogen Idec ($BIIB) started the year with a $299 million deal to develop a new antisense drug for rare instances of spinal muscular dystrophy with Isis ($ISIS). And now the big biotech is coming back from the deal table with a new pact that pairs them with Isis once again on another rare disease program, this time for myotonic dystrophy Type 1, a genetic neuromuscular ailment sometimes called Steinert disease.
Isis gets a check for $12 million for the new partnership along with $59 million in clinical milestones and up to $200 million for a license fee and regulatory milestones. Biogen Idec gets the option to license the program through Phase II. Isis is responsible for the clinical program while Biogen Idec godfathers the work, with the right to step in and take over if it pulls the trigger on the licensing deal.
DM1 is an inherited disease, and each generation it's passed down increases the severity of the disease. It starts with a defect in the DMPK gene, which initiates a cascade of reactions that shuts down the production of proteins which causes severe muscle spasms, muscle wasting and weakness. Using its antisense know-how, Isis believes it can interrupt the process at a critical stage, stopping the cascade that triggers severe symptoms among 150,000 patients in the U.S., Europe and Japan.
"This collaboration allows us to expand our pipeline of drugs for rare and severe diseases with Biogen Idec's additional resources and support," says B. Lynne Parshall, Isis' COO and CFO. "It also complements our new alliance 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 manner to treat another devastating disease. "
This type of genetically focused drug development program could also be a relatively quick effort. In their first pact, Isis noted that the dire need for a new treatment raised the prospect of filing for approval based on its proof-of-concept study. Rare diseases also don't require the big studies with thousands of patients needed to demonstrate safety in a large population. Any treatments for these diseases can also fetch six-figure prices. And that helps explain why a number of Big Pharma companies have been beefing up their work in the rare disease arena.
- here's the press release
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Biogen Idec strikes $299M deal for Isis antisense drug