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Ionis Pharmaceuticals, Inc.

Partner : Biogen Idec, Inc.

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Isis Pharmaceuticals Earns $2.8M for Advancing ISIS-DMPK-2.5 Rx in Patients with Myotonic Dystrophy Type I
CARLSBAD, Calif., Nov. 10, 2015 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today 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).

Isis Pharmaceuticals, Inc.
"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. For further study information, please visit www.clinicaltrials.gov and search for ISIS-DMPKRx or by the study number: NCT02312011.

DM1 is a debilitating rare genetic neuromuscular disease characterized by progressive muscle atrophy painful muscle spasms, cataracts, heart conduction defects, endocrine abnormalities and myotonia. DM1, the most common form of muscular dystrophy in adults, affects approximately 150,000 patients in the United States, Europe and Japan. There are currently no disease-modifying therapies. ISIS-DMPK-2.5Rx is the only program in clinical testing that is designed to address the underlying genetic defect that causes DM1. ISIS-DMPK-2.5Rx is a product of Isis' antisense technology, a unique drug discovery and development platform that can directly address the genetic cause of diseases, like DM1, that have been largely untreatable with current therapeutic approaches.

Patients with DM1 have a genetic defect in their DMPK gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long toxic RNA that accumulates in the nucleus of cells and prevents the production of proteins needed for normal cellular function. The number of triplet repeats can increase from one generation to the next, with the devastating consequences of more severe disease in each subsequent generation.

ABOUT ISIS and BIOGEN
Isis and Biogen have a broad strategic alliance focused on leveraging antisense technology to advance the treatment of neurological and neuromuscular disorders. This alliance 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. Isis is primarily responsible for drug discovery and early development of antisense therapies. Biogen has the option to license each antisense program at a particular stage in development. Current development-stage programs include antisense drugs to treat patients with spinal muscular atrophy (SMA), nusinersen, myotonic dystrophy type 1 (DM1), ISIS-DMPK-2.5Rx, and two undisclosed neurodegenerative diseases, ISIS-BIIB3Rx, and ISIS-BIIB4Rx. In addition to these four drugs, Isis and Biogen have numerous opportunities to evaluate additional targets for the development of drugs to treat neurological disorders.
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Source: press release, 11/10/15. http://ir.isispharm.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID=211...

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A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients With Myotonic Dystrophy Type 1
Estimated Enrollment: 80
Study Start Date: December 2014
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
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Source: clinical trials.gov. https://clinicaltrials.gov/ct2/show/NCT02312011?term=isis+pharmaceutical...

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
IONIS-DMPK-2.5RxMedical GeneticsMyotonic Dystrophy Type 1Antisense pharmaceuticalRNA

Mechanism of action: IONIS-DMPK-2.5Rx is an antisense drug designed to treat Myotonic Dystrophy Type 1 (DM1), a genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and disabling muscle spasms. DM1 is unusual in that the disease is not caused by a missing or malfunctioning protein, but rather is due to a genetic defect that causes a toxic RNA to accumulate in the cell which prevents the production of a number of proteins needed for normal cellular function. ISIS-DMPK-2.5Rx is designed to target the toxic RNA and reduce its accumulation thereby restoring normal cellular function.

Phase of Development: II

Event Type: Data: Phase II trial results

Dates: 2016-12-01 - 2016-12-31

Results: Pending