Biogen, Inc. ( BIIB Quick Quote BIIB - Free Report) announced that a phase III study evaluating timrepigene emparvovec (BIIB111), a gene therapy candidate for the one-time treatment of choroideremia, a rare inherited retinal disease failed to meet its primary or key secondary endpoints.
The primary endpoint of the phase III STAR study was the proportion of patients with an improvement of at least 15 letters from baseline in best corrected visual acuity (BCVA) at 12 months post-treatment as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. The study also failed to prove efficacy on the key secondary endpoints
Biogen will decide on the next steps of the program after analyzing the final data set, which is expected to be presented at a future medical meeting.
Biogen’s stock has risen 65.9% this year so far compared with an increase of 1.1% for the
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Please note that timrepigene emparvovec was added to Biogen’s portfolio with the June 2019 acquisition of gene therapy maker, Nightstar Therapeutics. The acquisition also added cotoretigene toliparvovec (BIIB112), a gene therapy for treating patients with X-linked retinitis pigmentosa (XLRP), also a rare, inherited retinal disease, to Biogen’s portfolio. Last month, Biogen announced that cotoretigene toliparvovec also failed to meet the primary endpoint in a phase II/III study.
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.
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 Quick Quote 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 Quick Quote 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 Quick Quote SAGE - Free Report) , which added a late-stage program (zuranolone) in depression and movement disorders.
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