Timrepigene emparvovec is under clinical development by Biogen and currently in Phase III for Retinitis Pigmentosa (Retinitis). According to GlobalData, Phase III drugs for Retinitis Pigmentosa (Retinitis) have a 20% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Timrepigene emparvovec’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Timrepigene emparvovec overview
Timrepigene emparvovec (BIIB-111) is under development for the treatment of spinal muscular atrophy, retinitis pigmentosa. It is administered through subretinal injection. It consists of an adeno-associated virus vector serotype 2 (AAV2) containing codon-optimized cDNA that is designed to produce full-length, fully functional human REP-1 protein. It was also under development for the treatment of choroideremia.
Biogen is a biopharmaceutical company that discovers, develops, and delivers drugs and biosimilars for the treatment of various neurological and neurodegenerative diseases. The company’s marketed products include Avonex (interferon beta-1a), Vumerity (diroximel fumarate), Tysabri (natalizumab), Tecfidera (dimethyl fumarate), Fampyra (prolonged-release fampridine tablets), and Plegridy (peginterferon beta-1a) for the treatment of multiple sclerosis (MS); Spinraza (nusinersen) for spinal muscular atrophy (SMA); Aduhelm (aducanumab-avwa) for alzheimer’s disease, and Fumaderm (fumaric acid esters) for severe plaque psoriasis. It has several product candidates targeting various indications such as MS, Parkinson’s disease, Lupus, Depression, CNS and neuromuscular disorders, Amyotrophic Lateral Sclerosis, and Alzheimer’s disease. The company sells its products through direct sales force, marketing groups, and distributors in the Americas, Europe, Asia, and other territories. Biogen is headquartered in Cambridge, Massachusetts, the US.
For a complete picture of Timrepigene emparvovec’s drug-specific PTSR and LoA scores, buy the report here.