Isis to Showcase SMA Investigational Therapy at Investor Conferences

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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Phase 2 study of nusinersen

isis pharmaceuticalsIsis Pharmaceuticals, Inc., a drug development company using RNA-targeted technology to develop novel drugs for the treatment of cardiovascular, metabolic, severe and rare diseases such as spinal muscular atrophy (SMA), has been invited to present its pipeline of therapeutic products at three conferences within the next few months, according to a press release from the company.

Isis along with several of its partners will participate in the 2015 Leerink Global Healthcare Conference on Wednesday, February 11, in New York. In addition, during March the company will also be present at the Cowen and Company 35th Annual Health Care Conference on March 4 in Boston, as well as at the 27th Annual ROTH Conference five days later in Laguna Niguel.

The company’s pipeline includes 33 drug therapies targeting cardiovascular, metabolic, severe and rare diseases, neurological diseases and cancer. Isis is currently investigating ISIS-SMNRx for the treatment of SMA in a phase 3 study called CHERISH that includes about 120 non-ambulatory children suffering from the severe and rare genetic neuromuscular disease.

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The company initiated dosing of the patients in the CHERISH study last December, which granted Isis a $27 million milestone payment from its development partner Biogen Idec. In addition, Isis is also assessing ISIS-SMNRx in a different phase 3 study with infant SMA patients, called ENDEAR. The two studies are being conducted as part of an agreement with the U.S. Food and Drug Administration (FDA) for special protocol assessments (SPA).

Isis and Biogen Idec are working in partnership on the development and commercialization of ISIS-SMNRx under an agreement established by the two companies in 2012. While Isis is responsible for the global development of ISIS-SMNRx, Biogen Idec has the option to license the compound upon the first successful completion of the Phase 2 / 3 trial or of two Phase 2 / 3 studies. In January, Biogen granted another $7 million milestone payment to support the ongoing open-label extension clinical study.

The conferences will give Isis the opportunity to present its products, investigations and findings to researchers and potential investors. The company will provide a live webcast of the presentations in the “Investors & Media” section of the Isis website, which will be replayed 48 hours later and will be archived for a limited time.