Companies To Develop Antisense Drug for Myotonic Dystrophy

Isis and Biogen Idec are collaborating to develop antisense drugs for neurologic disorders; a trial of ISIS-DMPKRx for MMD1 (DM1) is expected in 2014

Article Highlights:
  • Biotechnology companies Isis Pharmaceuticals and Biogen Idec expect to move experimental antisense drug ISIS-DMPKRx into a phase 1 clinical trial in type 1 myotonic dystrophy (MMD1; also known as DM1) in 2014.
  • MDA-supported laboratory research has provided a foundation for development of antisense for MMD1.
  • MDA's Myotonic Dystrophy Clinical Research Network and a recently funded "natural history" study of congenital-onset MMD1 will supply important data for the design of the ISIS-DMPKRx trial.
by Margaret Wahl on September 10, 2013 - 4:55pm

Update (Oct. 16, 2013): Isis announced in an Oct. 16 press release that it has earned a $10 million "milestone" payment from Biogen Idec related to the selection and advancement of ISIS-DMPKRx to treat type 1 myotonic dystrophy.

Biotechnology companies Isis Pharmaceuticals and Biogen Idec have entered into a multiyear collaboration to advance antisense technology for the treatment of neurological diseases — including type 1 myotonic dystrophy (MMD1, or DM1).

Antisense technology uses molecules that block or alter the production of proteins from genetic instructions known as RNA. It can be used to coax cells to make a slightly different protein or to stop making a protein.

Isis (Carlsbad, Calif.) and Biogen Idec (Weston, Mass.) announced their plans in a Sept. 9, 2013, press release. The companies are currently collaborating to develop antisense compounds for two other neuromuscular diseases in MDA’s program:

The newly identified compound for MMD1 is ISIS-DMPKRx, the company announced in a Sept. 9 conference call. (A webcast of the approximately 45-minute call is available on the Isis website.)

The MMD drug, which will target genetic instructions for the DMPK protein, is expected to be tested in a phase 1 clinical trial in 2014.

The design of the anticipated clinical trial of ISIS-DMPKRx will rely in part on data gathered through MDA’s Myotonic Dystrophy Clinical Research Network, established in January 2013; and on a new MDA-funded "natural history" study of congenital-onset MMD1.

Several years of MDA-supported laboratory research related to MMD1 treatment will add to the foundation on which the drug developers will build.

"Myotonic dystrophy research has been moving forward rapidly in the last few years, and we have several potential therapies on the horizon," said Jane Larkindale, MDA's vice president of research. "In preparation for when these compounds move forward, MDA has been setting the stage for successful trials — funding projects like Nicholas Johnson's natural history study, which will teach us about the disease and allow effective trials to be developed. 

"We are happy to see Biogen and Isis continuing this collaboration, as so far the work of these companies in SMA and ALS has been moving both disease fields forward. We're also excited to see this happen in myotonic dystrophy."

Your rating: None Average: 4.5 (10 votes)
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email See comment policy