US-based Isis Pharmaceuticals has started Phase I/II trial of ISIS-DMPK to treat patients with myotonic dystrophy type 1 (DM1), a rare genetic neuromuscular disease.
DM1 is caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells.
It is primarily characterised by progressive muscle atrophy, weakness and myotonia, and is the most common form of muscular dystrophy in adults affecting around 150,000 patients in the US, Europe and Japan.
The company said that it has specifically designed ISIS-DMPK to reduce toxic DMPK RNA.
The six-week randomised, placebo-controlled, dose-escalation trial will evaluate the safety and tolerability of multiple ascending doses of ISIS-DMPKRx in about 36 patients with DM1.
Myotonic Dystrophy Foundation executive director Molly White said: "Myotonic Dystrophy, the most common form of muscular dystrophy, is a devastating disease with no therapeutic option.
"Myotonic dystrophy research has accelerated significantly in the last ten years, helping bring about the innovative science behind ISIS-DMPKRx, a drug that specifically targets the genetic defect that causes myotonic dystrophy type 1."
Isis chief operating officer Lynne Parshall said: "We have an innovative and productive partnership with Biogen Idec in developing drugs to treat severe and rare diseases, like DM1.
"In just under two and a half years, we have been able to discover and complete early development on ISIS-DMPKRx, which includes completing a Phase I single ascending-dose study in healthy volunteers."
Isis Pharmaceuticals and Biogen Idec have established four collaborations focused on using antisense technology to advance the treatment of neurological and neuromuscular disorders.
The partnership combines Isis expertise in antisense technology to evaluate potential neurological targets and discover antisense drugs with Biogen’s capability to develop therapies for neurological disorders.
Under the deal, Isis Pharmaceuticals is primarily responsible for drug discovery and early development of antisense therapies and Biogen has the option to licence each antisense programme at a particular stage in development.