Endpoints News: Edging toward a pivotal readout, Biogen picks a new discovery partner for ophthalmic gene therapy

Almost two years after Biogen swooped again into the ophthalmic gene therapy field with an $800 million buyout of eye-focused Nightstar Therapeutics, it’s turning to a young player for a new program.

Germany’s ViGeneron will be deploying its next-generation AAV vectors to generate candidates for a target — Biogen isn’t disclosing which exactly — that would treat inherited eye disease. The big biotech partner has another target in mind, for which it has the option to add within two years.

Although the Alzheimer’s drug aducanumab has been shoved to a central, almost existential position at Biogen, the company has been placing measured bets on ophthalmology, which CEO Michel Vounatsos has called an emerging area and part of the “second wave of growth” to follow aducanumab in the mid-2020s.

With the new deal, it’s buying into a platform that boasts of “superior transduction efficiency” in retinal cells and intravitreal administration.

Once it’s come up with the AAV capsids, ViGeneron will play a role in the in vivo proof of concept, but leave it all to Biogen after that point. Biogen is paying an undisclosed upfront and footing the R&D bill.

Caroline Man Xu
“This collaboration exemplifies our strategy to develop in-house programs for selected retinal targets, while maximizing our proprietary technology platforms with additional collaboration programs for other targets in ophthalmology and further indications,” said Caroline Man Xu, co-founder and CEO of ViGeneron.

There’s no word yet on when the candidates might materialize, but they will likely come long after Biogen reads out the Phase III study of BIIB111 (timrepigene emparvovec), the lead asset from the Nightstar buyout. Designed to replace the gene that’s mutated in patients with choroideremia, the program “represents our next pivotal readout and our next potential commercial product after aducanumab,” Vounatsos said back in July.

The data are expected to come in the first half of 2021.

Aside from that and a second Nightstar experimental treatment for retinitis pigmentosa caused by mutations in the RPGR gene, Biogen has also picked up a preclinical gene therapy from Massachusetts Eye and Ear for inherited retinal degeneration due to mutations in the PRPF31 gene.

It all marks a second try at developing one-shot cures for rare eye diseases, after pulling out from a deal with Applied Genetic Technologies following a failed trial.