Tralesinidase alfa is a Recombinant Enzyme owned by Allievex, and is involved in 3 clinical trials, of which 1 was completed, 1 is ongoing, and 1 is planned.

Tralesinidase alfa (BMN-250) is an enzyme replacement therapy using recombinant alpha-N-acetyglucosaminidase (NAGLU) with an insulin-like growth factor 2 (IGF2), or Glycosylation Independent Lysosomal Targeting (GILT) tag. The autosomal recessive defects in alpha-N-acetylglucosaminidase (NaGlu) are implicated in Sanfilippo B syndrome. NaGlu is a lysosomal enzyme which is involved in the degradation of the glycosaminoglycan (GAG) heparan sulphate, and a deficiency results in the accumulation of partially degraded GAGs inside lysosomes. Thus, the drug candidate could be effective in the treatment of Sanfilippo B syndrome.

The revenue for Tralesinidase alfa is expected to reach a total of $384m 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 Tralesinidase alfa NPV Report.

Tralesinidase alfa was originated by BioMarin Pharmaceutical and is currently owned by Allievex. BioMarin Pharmaceutical is the other company associated in development or marketing of Tralesinidase alfa.

Tralesinidase alfa Overview

Tralesinidase alfa (BMN-250) is under development for the treatment of Sanfilippo B syndrome (mucopolysaccharidosis type IIIB). It is administered through intracerebroventricular route. The therapeutic candidate is an enzyme replacement therapy using recombinant human alpha-N-acetyglucosaminidase (NAGLU) fused with a peptide derived from insulin-like growth factor 2 (IGF2) or glycosylation independent lysosomal targeting (GILT) tag. It is developed based on Biomarin technology.

BioMarin Pharmaceutical Overview

BioMarin Pharmaceutical (BioMarin) develops and commercializes innovative medicines for the treatment of rare genetic diseases and medical conditions such as Duchenne muscular dystrophy (DMD), phenylketonuria (PKU), achondroplasia, late infantile neuronal ceroid lipofuscinosis (CLN2) and hemophilia A. The company focuses on developing breakthrough treatments for debilitating and life-threatening diseases. Its product category consists of multiple clinical and preclinical offerings which include Naglazyme-Mucopolysaccharidosis Type VI (MPS VI), Aldurazyme-MPS I and others. The company operates through its manufacturing facilities in Novato, California. It conducts business operations in the US, Europe, Latin America, the Middle East, and Asia-Pacific. BioMarin is headquartered in San Rafael, California, the US.

The company reported revenues of (US Dollars) US$1,846.3 million for the fiscal year ended December 2021 (FY2021), a decrease of 0.8% over FY2020. The operating loss of the company was US$82.3 million in FY2021, compared to an operating loss of US$36.8 million in FY2020. The net loss of the company was US$64.1 million in FY2021, compared to a net profit of US$859.1 million in FY2020. The company reported revenues of US$505.3 million for the third quarter ended September 2022, a decrease of 5.3% over the previous quarter.

Quick View – Tralesinidase alfa

Report Segments
  • Innovator
Drug Name
  • Tralesinidase alfa
Administration Pathway
  • Intrathecal
Therapeutic Areas
  • Genetic Disorders
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.