- Rugonersen, a potentially best-in-class antisense oligonucleotide (ASO) therapy in development for Angelman syndrome, advanced into the pivotal Phase 3 BEACON clinical trial
- Angelman syndrome affects approximately 30,000 diagnosed individuals in the US and EU5 and has no approved treatments
- Topline data expected in early 2029
Cambridge (United States), July 28, 2026 – OHB Pediatrics Ltd. d/b/a Oak Hill Bio, a clinical-stage rare disease therapeutics company, today announced that the first participant has been dosed in BEACON, a pivotal Phase 3 clinical trial evaluating rugonersen, an investigational ASO for the treatment of Angelman syndrome.
“The dosing of the first participant in the BEACON (NCT07605429) trial marks an important milestone for the rugonersen development program and for the Angelman syndrome community,” said Dr. Brenda Vincenzi, Chief Medical Officer of Oak Hill Bio. “It is a privilege to once again work closely with individuals living with Angelman syndrome, their families, clinicians, and patient advocacy organizations as we advance this pivotal Phase 3 trial. Building on encouraging preclinical findings and the Phase 1 TANGELO trial, which demonstrated promising safety, target engagement, and signals of clinical activity, BEACON is designed to further evaluate the efficacy and safety of rugonersen. We are hopeful that this trial will bring us one step closer to delivering a meaningful treatment option for people living with Angelman syndrome, who continue to face significant unmet medical needs.”
Angelman syndrome is a neurodevelopmental disorder affecting approximately 15,000 individuals in each of the US and the EU5, caused by disruptions in the UBE3A gene leading to cellular damage within neurons. Rugonersen was originally developed by Roche to address the underlying disease biology of Angelman syndrome by restoring UBE3A expression.
Rugonersen’s previous Phase 1 TANGELO trial was conducted by Roche in pediatric Angelman syndrome participants aged 1-12 years and showed that treatment with rugonersen led to a dose-dependent partial normalization of pathological brain activity on a pharmacodynamic biomarker of brain function, electroencephalogram (EEG) delta power. Participants also exhibited improvements as compared to expectations based on natural history on multiple exploratory endpoints measuring the core symptoms of Angelman syndrome. Rugonersen showed a generally appropriate safety and tolerability profile in TANGELO. The data from TANGELO are extensively covered in Hipp et al, The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a Phase 1 trial. Nature Medicine (2025).
The Phase 3 BEACON trial (NCT07605429) has been designed as a global, randomized, double-blind,sham-controlled, multi-center trial that will enroll up to 165 participants with Angelman syndrome aged 1-50 years. Participants will be randomized on a 1:1 basis to receive either sham procedure or 120 mg rugonersen by intrathecal injection once every 12 weeks. The primary endpoint, defined as improvement from baseline in Bayley Scale of Infant and Toddler Development, Fourth Edition (BSID-4) raw score in cognition and/or expressive communication, will be measured after 56 weeks. All participants completing 56 weeks will be eligible to participate in an open-label extension, which will administer rugonersen for an additional 2 years and allow for a long-term evaluation of rugonersen’s safety and efficacy. Oak Hill Bio expects to report topline results in early 2029.