A couple days ago Biogen Idec and Isis announced they woudl be partnering on another deal to apply Isis antisense technology to type 1 myotonic dystrophy. Under the deal, Isis gets a smallish $12 M upfront payment with back loaded milestones of $259 M possible.
Isis scientists are wagering they can fight myotonic dystrophy type 1 now that they have identified a gene that contains three individual DNA bases that repeat extensively, which leads to the production of long, toxic RNA molecules that accumulate in cells and cause the muscle problems. By creating an antisense molecule, Isis and Biogen are hoping to target and get rid of the toxic RNA. The condition affects about 150,000 patients.
“Myotonic dystrophy has an identifiable genetic cause, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis’ antisense compound has the potential to make a real difference,” said Steve Holtzman, Biogen’s executive vice president of corporate development.
This type of genetically focused drug development program could also be a relatively quick effort. Dire need for a new treatments in rare conditions can raise the prospect of filing for approval based on proof-of-concept studies. 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.
It's been awhile since antisense therapies have been getting positive press. Hopefully this deal will work out for both companies and the affected patients. It would also be good to see the technology successfully deployed for at least some conditions. See Xconomy and Fierce Biotech.
Posted by Bruce Lehr Jun 30th 2012
Completely agree, these monogenic diseases are perfect for antisense/RNAi attempts. If only ISIS had gone in this direction sooner I think patients and shareholders would have been better off
Posted by: BioDueDiligence | 06/30/2012 at 07:03 PM