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An update on Roche’s trials of rugonersen (RO7248824) in Angelman syndrome

We are sad to report that Roche has decided they will not initiate any new trials of rugonersen (RO7248824) in Angelman syndrome. If you are currently participating in the study, you should be hearing from the Investigator or their team at the site where your child is enrolled regarding important information about next steps for your child. 

Roche made this tough decision after looking at the current Phase 1 study results. The good news is that this was not based on any safety concerns. Roche stated that they set a hurdle for efficacy to be able to continue to invest in the development of this ASO. The results from their analysis showed “encouraging effects on EEG” (a biomarker of the drug’s effect on AS), but unfortunately the other effects they compared in treated patients versus the natural history did not meet their internal criteria to keep the rugonersen program going. So Roche has decided to try to find another company that would be willing to take charge of the rugonersen program.

So what does this really mean? The early safety results from the Phase 1 trial were acceptable, but the early effects were less than they hoped to see.

We know this is disappointing, first and most importantly for the families who have been in this trial. Before anything else, we want to acknowledge that this news is harder for you than anyone else, and we all, as a community, owe you our deepest gratitude. Your sacrifices and time spent were on behalf not just of your own loved ones but all of ours. Every study provides valuable insights and new knowledge about AS, and this one is no exception. 

Because the data is encouraging but not definitive, Roche has offered to allow an open-label extension for patients wanting to continue on it, with an estimated end date of February 2024. By then, Roche hopes it will have found a partner to take up the investigational therapy and move it forward. We hope that this in fact happens and rugonersen can be fully investigated to assess its longer term effects in patients living with AS.

It is also important to note what else this doesn’t mean. Roche will not be leaving the AS space, as they will continue their clinical study of alogabat and are in the process of activating sites for that study. 

At FAST, we constantly underscore the chutes-and-ladders aspect of drug development, according to which treatments can rise, or fall, based on science but also based on lots of other factors--including the regulatory environment, the markets, internal affairs at a specific company, shifts in company priorities, and more. 

We will continue to keep the entire community abreast of any new learnings about the results of this trial and the future of rugonersen. We are in close contact with the Roche team that was working on this trial.