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Biogen (BIIB) Eye Disease Drug Misses Primary Goal in Study

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Biogen, Inc. (BIIB - Free Report) announced that a phase II/III study evaluating cotoretigene toliparvovec (BIIB112), a gene therapy for treating patients with X-linked retinitis pigmentosa (XLRP), a rare, inherited retinal disease, failed to meet the primary endpoint.

The study failed to demonstrate a statistically significant improvement in the proportion of treated study eyes with ≥7 dB improvement from baseline at ≥5 of the 16 central loci of the 10-2 grid assessed by Macular Integrity Assessment (MAIA) microperimetry. 

The company said that though the study did not meet the endpoint, positive trends were observed in other pre-specified clinically relevant endpoints, such as a measure of visual acuity under low light conditions.

Biogen will decide on the next steps of the program after analyzing the final data set.

Biogen’s stock has risen 14.4% this year so far against a decrease of 5.6% for the industry



Please note that cotoretigene toliparvovec was added to Biogen’s portfolio with the June 2019 acquisition of gene therapy maker, Nightstar Therapeutics. The acquisition also added timrepigene emparvovec (BIIB111), a gene therapy candidate for the one-time treatment of choroideremia, also a rare inherited retinal disease. 

Biogen has an impressive portfolio of gene therapy candidates in its pipeline to address significant unmet medical needs, most of them acquired through in-licensing deals. In the four years from 2017-2020, Biogen executed 20 business development transactions including some gene therapy deals, which significantly boosted its pipeline.

Among some recent deals, Biogen signed a collaboration with Sangamo Therapeutics (SGMO - Free Report) in 2020 to make gene regulation therapies for a range of neurological indications including Alzheimer's disease. It also collaborated with Denali Therapeutics (DNLI - Free Report) in August 2020 to co-develop and co-commercialize the latter’s small molecule LRRK2 inhibitor program, DNL151, for Parkinson’s disease, which will enter late-stage development in 2021.In November 2020, Biogen signed a collaboration with Sage Therapeutics (SAGE - Free Report) , which added a late-stage program (zuranolone) in depression and movement disorders.

Biogen has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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