June 21, 2023
Roche Clinical Trial of Rugonersen in Angelman Syndrome
Roche has made the difficult decision not to move forward with a new clinical trial for rugonersen and has initiated the search for an external partner to take over the development. The conclusion of this trial undoubtedly brings feelings of disappointment, frustration, and sadness for many of us who have hoped and advocated for a breakthrough in the treatment of Angelman syndrome. It is a disappointment we share, and in this moment, it is crucial that we come together as a community to support one another. First and foremost, to those brave families that participated in the trial and made so many sacrifices for this community, we are in debt to you and your amazing individual with AS. The many days of traveling, assessments, fear and so much more makes you a true hero and we are here to support you in any way.
The primary objective of the ongoing Phase 1 TANGELO study is to evaluate the safety and tolerability of rugonersen. During the interim analysis (looking at the data at a pre-planned point while the study is still ongoing) rugonersen has demonstrated an acceptable safety profile. Although some encouraging effects were observed in the patients’ EEG, the observed level of clinical improvements in the TANGELO dosing regimens and as compared to natural history data (the usual course of development of individuals with AS in the absence of treatment) did not meet Roche’s internal criteria to move into the next phase of clinical development. They will still be moving forward with their alogabat trial and we will continue to partner with them to get this to the finish line.
This setback does not diminish the incredible progress we have made together, nor should it diminish the hope for possible treatments in the future. This landscape can be very hard to navigate, but there are still so many promising trials in process and in the pipeline that should give this community so much hope.
Shannon Barlow, ASF counselor, is standing by to meet with those who were in the trial and those in the community that need support to navigate through this. You can reach her by calling (773) 259-4200.
By standing united, we can find solace and empower one another to keep pushing forward. Remember, we are not alone. Researchers, scientists, and organizations around the world continue their dedicated work to understand Angelman syndrome better. Although the Roche trial may not have yielded the desired outcome, it is essential to recognize that progress in science is often marked by setbacks and obstacles. Our unwavering spirit will persevere, and we will seize every opportunity that comes our way.
Together, we will continue to raise our voices, advocate for increased funding and research, and explore alternative paths towards effective treatments for Angelman syndrome. Our community is resilient, and our love for our children, family members, and friends with Angelman syndrome knows no bounds. As we move forward, let us hold onto hope and the belief that one day, our collective efforts will result in breakthroughs that will change lives.
You can read the Roche community letter here.