Roche is back to square one for finding a partner to carry forward its culled Angelman syndrome drug.
Last June, the Swiss pharma had started a search for a partner after stopping clinical testing of the experimental medicine, called rugonersen. It had told patients and their families that early Phase 1 data on the antisense oligonucleotide didn’t meet its “internal criteria to move into the next phase of clinical development.”
On Thursday, Roche told Endpoints News that it had reached “advanced negotiations with respect to out-licensing rugonersen to a potential partner.” But that deal won’t move forward, the company said through a spokesperson.
The spokesperson declined to disclose the name of the other company, which they described as having “significant drug development experience in the field of genetically determined neurodevelopmental disorders.”
“Unfortunately, despite many efforts to reach an agreement, this potential partner informed us that they have decided to withdraw from the negotiations,” the spokesperson continued.
Roche will now look at finding other partners “and remain committed to finding the best solution for people living with Angelman syndrome,” the spokesperson said. The company has previously said the drug would be kept available to study participants until February 2024.
Angelman is a rare, genetic neurodevelopmental condition that disrupts speech and balance, among other daily functions. Patients have few options, and many drugmakers have backed out. At last year’s American Society of Gene & Cell Therapy meeting, Foundation for Angelman Syndrome Therapeutics Chief Science Officer Allyson Berent said 10 Angelman programs had been cut in recent years. All were preclinical.
With Roche out, Ultragenyx and Ionis are the only two companies in the clinic with antisense oligonucleotides for Angelman syndrome.
Both of those biotechs have recently released positive clinical results. Ionis said Thursday it plans to test its candidate ION582 in a pivotal trial after topline Phase 1/2a results showed it “demonstrated consistent improvements across multiple functional domains.” Its partner Biogen decided not to license and lead development of the candidate, the Boston drugmaker said Thursday.
Ultragenyx, meanwhile, plans to start a pivotal study this year for its candidate GTX-102, it said last month at the American Academy of Neurology annual meeting.