Biogen reveals gene therapy trial failure as investors remain laser-focused on aducanumab drama

Biogen biologics plant in Denmark
Biogen had hoped the gene therapy would become a one-time treatment for patients with X-linked retinitis pigmentosa, who currently have no other treatment options. (Biogen)

Biogen just can’t catch a break. The company revealed today that a gene therapy failed to improve vision in patients with X-linked retinitis pigmentosa, a disease area that has become ripe for the company’s rivals.

Cambridge, Massachusetts-based Biogen was testing the gene therapy cotoretigene toliparvovec, or BIIB112, in patients with the rare, inherited eye disease that causes progressive vision loss. The company had hoped the gene therapy would become a one-time treatment for these patients, who currently have no other options.

But the phase 2/3 trial called Xirius, which compared cotoretigene toliparvovec to placebo, missed its primary goal of improving retinal sensitivity at 12 months post-treatment. Biogen said the trial wasn’t a complete bust, however, as some improvement in vision in low light conditions was detected.

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Analysis of the trial is ongoing, and Biogen will release further details at a future scientific forum. Biogen was noncommittal on what will happen with the gene therapy now, only saying the data crunch would need to be completed before they determined the program’s fate.

That therapy came out of a $877 million deal for British biotech Nightstar Therapeutics, which spun out of the University of Oxford back in 2013. The retinitis pigmentosa gene therapy was in earlier-phase development and behind the leading candidate NSR-REP1, which is in later stage, pivotal trials for choroideremia, an as yet untreatable form of progressive vision loss.

Still, this is a blow for Biogen and in part for the buyout, and may shake confidence in its gene therapy approach.

The company has plenty of rivals in the ocular disease market. Janssen has partnered with private biotech Hemera Biosciences on a gene therapy for age-related macular degeneration. The pharma giant also dropped $100 million in 2019 to develop MeiraGTx’s clinical-stage gene therapies for retinal diseases, including X-linked retinitis pigmentosa. Allergan’s RST-001 is another contender.

This is not the first time Biogen has seen its hopes dashed in retinal diseases. In December 2018, a pact with Applied Genetic Technologies crumbled after the lead candidate of the program failed in a phase 1/2 trial.

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And of course, there’s aducanumab, Biogen’s Alzheimer’s disease hopeful currently sitting before FDA regulators that the pharma world—and patients—is eagerly awaiting. The drug experienced a high profile late-stage failure, which Biogen later deemed successful enough to send on to the FDA.

The Xirius trial failure may end up being just a slight blip on the radar of Biogen investors, who remain laser-focused on the aducanumab drama. Cotoretigene toliparvovec was not brought up on the company’s first-quarter earnings call in April and was mentioned just in passing in recent analyst commentary. Biogen shares were trading fairly steadily as the market opened today.