Roche Discontinues Clinical Development of Rugonersen (RO7248824), an Antisense Oligonucleotide Therapy for Angelman Syndrome

On June 23, according to FirstWord, Roche will terminate the clinical development of antisense oligonucleotide therapy rugonersen for Angelman syndrome. Roche stated that the therapy was well tolerated, and this decision was not related to safety issues.

Angelman syndrome is a neurodevelopmental disorder caused by abnormal expression or functional defects of the maternal ubiquitin-protein ligase E3 (UBE3A) gene.

Rugonersen (RO7248824) is a selective antisense oligonucleotide (ASO) therapy with the potential to increase UBE3A mRNA and protein expression levels.

Phase I TANGELO study shows that although some "encouraging effects" were observed on EEG patterns (biomarkers of hereditary diseases), the level of clinical improvement in the interim analysis did not meet the predefined criteria for continuation. Therefore, Roche plans to seek external partners to take over the development of rugonersen.

The decision to halt the development of rugonersen does not mean that Roche is giving up on the Angelman syndrome field. Roche added that the ongoing Phase II study of alogabat for the treatment of Angelman syndrome will not be affected. The small molecule is a GABRA5 positive allosteric modulator.

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