Biogen and Ionis Pharmaceuticals recently provided an update on the clinical development of nusinersen (IONIS-SMNRx), which is currently being tested in Phase 3 clinical trials:

Dear members of the SMA community,

As we begin 2016, we wanted to provide an update on the nusinersen (IONIS-SMNRX) program to you and the many families around the world who are touched by SMA. Since our last community post, we have made important progress in moving the nusinersen development program forward, and we remain resolute to our goal to bring an approved therapy to the SMA community as quickly as possible.

Our regular meetings with regulators around the world have been productive and collaborative as we work to define the most expeditious path toward regulatory review and potential approval. It is through this partnership with regulatory agencies that we continue to explore all accelerated pathways – such as Accelerated Approval, Breakthrough Therapy and Priority Review – to hopefully achieve our ultimate goal of bringing a potential therapy to people with SMA in the shortest period of time.

Over the last few months, we have achieved multiple milestones that move us closer to this goal:

  • Enrollment in our clinical studies continues to progress, which means we are making excellent progress toward gathering the necessary data required for review and approval. Our pivotal, Phase 3 studies, are still on track to complete in the first half of 2017. Of importance:
    • Our childhood Phase 3 study, CHERISH, has completed our target enrollment.
    • We anticipate that our infant Phase 3 study, ENDEAR, will fully enroll by the first half of 2016.
    • The EMBRACE study has fully enrolled in the U.S.
    • The NURTURE study, which is enrolling pre-symptomatic newborns that have a genetic diagnosis of SMA, has enrolled more than 33% of participants. This study may allow us to discover if treatment with nusinersen before signs of the disease are evident could delay or prevent the development of SMA.
  • The SHINE study has started, which is the open-label extension for eligible patients who have completed ENDEAR and CHERISH, ensuring all children who have completed one of these studies have the opportunity to receive active therapy.

In addition to the above, Ionis Pharmaceuticals recently presented new data at the JP Morgan Conference in San Francisco from the patients continuing in the open-label Phase 2 studies. We continue to be optimistic about the data from these open-label studies. However, it is important to remember that our well-controlled Phase 3 pivotal studies were designed to definitively prove the safety and efficacy of nusinersen and thus support review and potential approval of the candidate. At every point along the development path of nusinersen, we have asked ourselves and regulators what possibilities might be available to shorten this timeline, and we continue to explore all regulatory options.

The consideration of appropriate timing for early access for patients in need is deeply important to us. Our intention is to open an early access program for patients once we are confident that doing so will not delay the clinical trials or compromise the data necessary for regulators to make a determination about the safety and efficacy of nusinersen, and after we are able to end the sham-controlled study arm.

As we move into the year to come, we will continue to work tirelessly to enroll and conduct our Phase 3 studies in the most expeditious way possible and are committed to updating the SMA community as often as we can. Most importantly, we remain steadfast to our goal to bring an approved therapy to the SMA community.

Biogen & Ionis Pharmaceuticals