$32.5M to advance rugonersen into late-stage testing for Angelman
Oak Hill secures funding, adds board members for clinical trial program
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Oak Hill Bio has secured $32.5 million in financing to advance rugonersen, an experimental treatment for Angelman syndrome once in the pipeline at Roche, into late-stage clinical testing.
The planned Phase 3 trial was dubbed pivotal by the developer in a company press release, which called rugonersen a “potentially best-in-class” treatment.
The rare disease therapeutics company also announced that it has added new members to its board of directors, all with expertise to help guide the clinical trial program for rugonersen.
“We are thrilled to welcome these exceptional investors and company-builders to our team. We really appreciate that they’ve recognized the strong science and data behind rugonersen and the tremendous need for a disease-modifying therapy in the Angelman syndrome community,” said Josh Distler, Oak Hill’s CEO.
“With the financing and additions to our board, we are well-positioned to advance rugonersen into a Phase 3 study in the middle of 2026,” Distler said.
Angelman is caused by mutations in the UBE3A gene. Everyone inherits two copies of this gene — one from each biological parent — but in some brain cells, only the copy inherited from the mother is normally active. A rare disease, Angelman develops when the gene’s maternal copy is dysfunctional or missing, basically leaving cells without any functional version of the gene.
Rugonersen aims to turn on gene copy from father
Rugonersen, also known as RO7248824 or OHB-724, is designed to treat Angelman by reactivating the paternal copy of the UBE3A gene that’s normally turned off in brain cells. The therapy contains an antisense oligonucleotide — a small piece of genetic material — designed to block the activity of UBE3A-ATS, an RNA molecule that normally helps inactivate the paternal gene copy.
By blocking this RNA molecule, the therapy aims to essentially release the brakes and allow the paternal UBE3A gene copy to be active, giving cells a functional version of the gene.
Rugonersen was originally developed by Roche, which conducted preclinical testing and subsequently launched a Phase 1 clinical trial (NCT04428281) testing the therapy in children with Angelman.
Results from that study indicated that rugonersen had an acceptable safety profile. There were some signs indicating the treatment led to clinical improvements that would not be expected in Angelman patients without treatment. However, the effectiveness data fell short of the criteria Roche said were necessary to justify further investment in the program. In 2023, the company announced it would not be developing rugonersen any further.
Following that decision, the rights to rugonersen were obtained by Oak Hill, a company that specializes in acquiring and developing rare disease treatments that have been deprioritized by big pharma. Several former members of the rugonersen development team at Roche have also joined Oak Hill to help lead the therapy’s further development.
We believe rugonersen possesses best-in-class, disease-modifying potential.
Oak Hill said it expects the Phase 3 trial to start later this year.
Doug Fambrough, founder and portfolio manager of the KCap Biotechnology Fund, is one of the new members of Oak Hill’s board of directors.
“We are proud to support Oak Hill Bio in its mission to address a critical need in Angelman syndrome,” Fambrough said. “We were particularly impressed by rugonersen’s promising preclinical and Phase 1 data, which underscore rugonersen’s differentiation and potency. We believe rugonersen possesses best-in-class, disease-modifying potential and clearly demonstrates Oak Hill Bio’s ability to identify and acquire assets with significant therapeutic and commercial prospects.”
