Företagen Biogen och Ionis Pharmaceuticals har nyligen publicerat en uppdatering gällande de kliniska prövningar som är aktuella för SMA. NSMA väljer att publicera detta då det ger en bra sammanfattning om några viktiga kliniska studier som pågår i världen.

Biogen & Ionis release update on the development of Nusinersen

Dear members of the SMA community,

In response to your requests for information, we want to provide an update on the status of our investigational program and its direction 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 compound development program forward.

  • Enrollment is now completed in both of our Phase 3 studies, CHERISH (childhood-onset) and ENDEAR (infant-onset), as well as our Phase 2 EMBRACE study. These are important milestones for the nusinersen program and both Phase 3 studies remain on track to complete in the first half of 2017.
  • The open-label SHINE study remains open for patients who have completed participation in ENDEAR and CHERISH, as well as patients who have completed the open-label Phase 2 study in childhood-onset SMA patients.
  • Ionis recently presented additional data from the open-label Phase 2 study in infants at the American Academy of Neurology Meeting . While we continue to be optimistic about these data, our well-controlled Phase 3 studies (CHERISH and ENDEAR) are designed to provide the data necessary for regulatory review and approval.
  • For more information on the clinical trial studies please see the Clinicaltrials.gov website.

Additionally, the primary and secondary endpoints in our ENDEAR clinical trial protocol were recently updated. The decision to change the endpoints was carefully made based on additional insight from our open-label studies and discussions with regulators and experts in the field over the last year. The ENDEAR study was initially designed with a primary endpoint of permanent ventilation-free survival, meaning we would observe patients treated with 13 months of our investigational compound compared to untreated patients and evaluate the number of patients who survive without the need for permanent ventilation between the two groups. It became increasingly clear that measuring motor milestones, endpoints of Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (“CHOP INTEND”) and Compound Muscle Action Potential (CMAP), could be useful indicators of our investigational compound’s potential efficacy in the ENDEAR study. Therefore, study’s primary endpoint has now been changed to add a second primary endpoint: motor milestones.

We can assure you our number one goal remains advancing our investigational compound toward a potential approval as quickly as possible for as many possible. Based on conversations with regulators, data from our well-controlled Phase 3 studies will be required to support regulatory review and subsequent marketing approval. At every point we continue to ask, both ourselves and regulators, what viable options exist to shorten the timeline to approval. We must balance the urgency for a treatment option with the risk of unnecessary delay caused by filing without the needed data.

Behind the scenes, we are working diligently to expedite our investigational compound’s path to patients by preparing required elements of the regulatory filing ahead of Phase 3 study completion as appropriate. As soon as we have the necessary data to support a successful filing, we will communicate our plan to file to the SMA community.

Another top priority for us is expanded access to our investigational compound for eligible patients. Our intention is to open an expanded access program (EAP) for patients once we are confident doing so will not impact the clinical trials or compromise the data necessary for regulators to make a determination about the safety and efficacy of our investigational compound. What this means is that we will not be able to open an early access program until we are able to end the sham-controlled arm in the Phase 3 studies. In preparing for a potential EAP, we have been meeting with doctors and patient advocacy organizations in countries around the world, identifying treatment facilities, and ensuring manufacturing capacity. In addition, we have been discussing inclusion criteria with experts and bioethicists to ensure we are ready to enable access as quickly as possible in an ethical and equitable way.

Please know we understand every day counts for patients with SMA and their families. Each member of our team is working tirelessly and exploring every option to speed the development, and hopefully, approval of nusinersen.


The Biogen and Ionis SMA Team

Uppdateringen finns också att läsa på: Biogen & Ionis release update on the development of Nusinersen