- Another one of the gene therapies that Biogen acquired through its $800 million purchase of Nightstar Therapeutics has failed a key clinical trial, the company announced Monday.
- The late-stage study tested whether Nightstar's therapy, now known as BIIB111, could improve eye sight in people with choroideremia, a rare, inherited disease that causes vision loss. To be considered a success, the study needed to show enough treated participants achieved a certain level of visual improvement one year later. That goal was not met, according to Biogen.
- The company said it will evaluate all the data from the trial before deciding what's next for BIIB111. Monday's news comes just one month after a different Nightstar gene therapy failed to significantly improve vision for people with X-linked retinitis pigmentosa, or XLRP, an uncommon disease that leads to blindness.
Drug developers have grown increasingly invested in gene therapy and its potential to effectively cure diseases with a single treatment. Large companies like Novartis, Roche and Bayer each spent billions of dollars over the last few years to establish themselves in the field. And with money flowing and the technology getting refined, the Food and Drug Administration has said it expects to review and approve 10 to 20 cell and gene therapies annually by 2025.
For Biogen, the acquisition of Nightstar, which was the largest in the company's history, offered a stronger foothold in both gene therapy work and ophthalmology. At the time, Biogen had just exited a collaboration with the gene therapy developer AGTC, after one of its experimental treatments generated disappointing results in a trial of XLRP patients. The company was also awaiting crucial data from studies of aducanumab, its closely watched drug for Alzheimer's disease.
Though the deal handed Biogen two gene therapy programs in late-stage testing — one for choroideremia, the other for XLRP — it deal didn't sit well with some Biogen shareholders, who expressed doubts about the likelihood that Nightstar's therapies would succeed and that the acquisition would deliver a worthwhile return on investment.
Two years later, those doubts have more merit.
In May, Biogen disclosed the XLRP study found no significant difference between patients who were given Nightstar's therapy and those who were not, as measured by how much their retinal sensitivity improved over the course of testing. Biogen said there were some "positive trends" seen in the study, but it's waiting to communicate next steps for the program until the full dataset is analyzed.
Similarly, the results announced Monday appear to put the choroideremia therapy's future in limbo, though some don't see much of a path forward. In a note to clients, Cantor Fitzgerald analyst Alethia Young wrote that while her team had viewed this therapy as one of Biogen's "most promising pipeline programs," the "limited efficacy" seen on the trial's various measures leads them to believe that the company won't develop it further.
Biogen said detailed results of the study would be made available at a future scientific forum.
In addition to the choroideremia announcement, Biogen's partner Sage Therapeutics unveiled on Tuesday mixed results from a study of their depression drug.
Despite the negative news, Biogen's share price was largely unaffected in Tuesday morning trading. That likely has to do with last week's FDA approval of aducanumab, which will be sold by Biogen as Aduhelm. Analysts suspect that, even with controversy swirling over whether it works, Aduhelm will be a multibillion-dollar product.
"With the Aduhelm approval, we believe this event will not result in as significant a loss in revenues for [Biogen], although it would have likely had a much larger impact if Aduhelm was not approved," Young wrote, referring to the negative readout for BIIB111.