Timrepigene emparvovec is a Gene Therapy owned by Biogen, and is involved in 6 clinical trials, of which 5 were completed, and 1 is ongoing. Timrepigene emparvovec (BIIB-111) is an adeno-associated viral vector (AAV2) encoding rab-escort protein 1 (REP1). Rab proteins are modified by geranyl-geranyl moieties necessary for membrane association and target-protein recognition. Geranyl-geranyl groups are transferred to Rab proteins by geranyl-geranyl transferase 2 (GGTase2). GGTase2 requires Rab escort protein 1 (REP1) to bind Rab proteins. REP1 null mutations underlie an X-linked retinal degeneration called choroideremia (CHM). Gene therapy is a technique for correcting defective genes responsible for disease development. Thus, rAAV2.REP-1 can be useful for the treatment of blindness. 

The revenue for Timrepigene emparvovec is expected to reach a total of $2bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Timrepigene emparvovec NPV Report.

Timrepigene emparvovec was originated by University of Oxford and is currently owned by Biogen.

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.

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Biogen Overview

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), 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); and Fumaderm (fumaric acid esters) for severe plaque psoriasis. It has several product candidates targeting various indications such as MS, Parkinson’s disease, CNS and neuromuscular disorders, Alzheimer’s disease, and idiopathic pulmonary fibrosis and stroke. 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.

The company reported revenues of (US Dollars) US$10,981.7 million for the fiscal year ended December 2021 (FY2021), a decrease of 18.3% over FY2020. In FY2021, the company’s operating margin was 25.9%, compared to an operating margin of 33.8% in FY2020. In FY2021, the company recorded a net margin of 14.2%, compared to a net margin of 29.8% in FY2020. The company reported revenues of US$2,508.5 million for the third quarter ended September 2022, a decrease of 3.1% over the previous quarter.

Quick View – Timrepigene emparvovec

Report Segments
  • Innovator
Drug Name
  • Timrepigene emparvovec
Administration Pathway
  • Intraocular
Therapeutic Areas
  • Central Nervous System
  • Ophthalmology
Key Companies
Highest Development Stage
  • Phase III

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.