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:
As we approach the upcoming Cure SMA conference in Anaheim, we want to provide an update on the status of the nusinersen program and its direction.
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.
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 nusinersen 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 nusinersen’s potential efficacy in the ENDEAR study. Therefore, study’s primary endpoint has now been changed to add a second primary endpoint: motor milestones.
Full text: http://www.curesma.org/news/biogen-ionis-spring-2016-update.html
As we approach the upcoming Cure SMA conference in Anaheim, we want to provide an update on the status of the nusinersen program and its direction.
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.
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 nusinersen 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 nusinersen’s potential efficacy in the ENDEAR study. Therefore, study’s primary endpoint has now been changed to add a second primary endpoint: motor milestones.
Full text: http://www.curesma.org/news/biogen-ionis-spring-2016-update.html
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