Biogen Announces NURTURE, a New Phase 2 Clinical Trial

Biogen, in collaboration with Isis Pharmaceuticals, has announced that they will soon open recruiting for NURTURE. This is a multi-center, Phase 2 clinical study evaluating the efficacy of the investigational drug, ISIS-SMNRx (ISIS 396443), in pre-symptomatic newborns that have a genetic diagnosis of Spinal Muscular Atrophy (SMA).

The purpose of the NURTURE study is to evaluate the efficacy of the drug (whether early treatment with ISIS-SMNRx, before the signs of the disease are evident, could delay or prevent the development of the disease and its symptoms) and to further investigate the safety and tolerability of ISIS-SMNRx.

The NURTURE study will last about 2.5 years and is expected to enroll up to 25 infants from multiple participating clinical centers throughout the world. In the NURTURE study, all participants in the trial will receive the investigational drug ISIS-SMNRx.

The study will be conducted at multiple clinical centers worldwide (United States, Europe, The Middle East, South America and Asia Pacific). A complete list of study related criteria for this trial can also be found at (NCT02386553).

Ongoing Clinical Trials

In addition to this Phase 2 NUTURE study, Isis has two ongoing Phase 3 clinical trials testing ISIS-SMNRx.

  • ENDEAR is testing the use of ISIS-SMNRx in infants who have SMA type I and are already showing symptoms.
  • CHERISH is testing the use of ISIS-SMNRx in children ages 2-12 who have SMA type II.

A fourth study, EMBRACE, is planned. This will be a Phase 2 clinical study evaluating safety and exploratory efficacy of ISIS-SMNRx in approximately 20 patients with infantile or childhood-onset SMA. This study will provide data from a small subset of patients that do not meet the age and other criteria of the current Phase 3 studies ENDEAR and CHERISH. This study is expected to begin in the first half of 2015.

For More Information

To see a complete list of sites and criteria for any of these studies, please visit or download FAQs on ENDEAR/CHERISH and on NURTURE.

To learn more about Cure SMA’s involvement with ISIS-SMNRx, please visit our Therapeutic Approaches page.

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