Isis Pharmaceuticals, Inc., announced that it has earned a $2.8 million milestone payment from Biogen related to the advancement of the ongoing Phase 1/2a study of ISIS-DMPK-2.5Rx in patients with myotonic dystrophy type I (DM1).
“Our collaboration with Biogen has been very productive. ISIS-DMPK-2.5Rx has rapidly advanced into patients with DM1, and we continue to make progress across the board in our drug discovery programs with Biogen. Progress like this advances our severe and rare disease pipeline for neurological disorders, which may, as our programs advance, translate into the potential for significant revenue. Together with Biogen, we have advanced four drugs into the pipeline and have other targets in late-stage research. To date and including the $2.8 million earned today, we have generated nearly $27 million from Biogen related to the advancement of ISIS-DMPK-2.5Rx,” said B. Lynne Parshall, chief operating officer at Isis Pharmaceuticals.
ISIS-DMPK-2.5Rx is designed to reduce the production of the toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, for the potential treatment of DM1. ISIS-DMPK-2.5Rx is being evaluated in a randomized, placebo-controlled, dose-escalation Phase 1/2a study in patients with DM1.
There are currently no disease-modifying therapies for DM1. ISIS-DMPK-2.5Rx is the only program in clinical testing that is designed to address the underlying genetic defect that causes DM1.