OTL-201 is a gene-modified cell therapy commercialized by Orchard Therapeutics, with a leading Phase II program in Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ). According to Globaldata, it is involved in 1 clinical trial, which is ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of OTL-201’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for OTL-201 is expected to reach an annual total of $6 mn by 2039 globally based off GlobalData’s Expiry Model. The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). Applying the drug’s phase transition success rate to remaining R&D costs and likelihood of approval (LoA) to sales related costs provides a risk-adjusted NPV model (rNPV). The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress.

OTL-201 Overview

OTL-201 is under development for the treatment of mucopolysaccharidosis-IIIA (Sanfilippo Syndrome Type A). The therapeutic candidate is developed based on ex-vivo lentiviral gene therapy. It is an autologous MPSIIIA HSCs transduced with either LV-PGK-coSGSH or LV-CD11b-coSGSH vectors expressing codon-optimized SGSH. The therapeutic candidate acts by targeting N-sulfoglucosamine sulfohydrolase (SGSH) enzyme.

Orchard Therapeutics Overview

Orchard Therapeutics (Orchard) is a biotechnology company that develops gene therapies for the treatment of serious and life-threatening orphan diseases. Its pipeline products include Strimvelis, an autologous ex vivo gammaretroviral gene therapy for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID); OTL-103 for Wiskott-Aldrich syndrome (WAS); OTL-102 for X-linked chronic granulomatous disease (X-CGD); OTL-300 for transfusion-dependent beta-thalassemia; and OTL-200 for metachromatic leukodystrophy (MLD), among others. It collaborates with institutions that specialize in gene and cell therapy such as The University of Manchester, Boston Children’s Hospital, and The San Raffaele Telethon Institute for Gene Therapy. It operates in France, Germany, Italy, Switzerland, the Netherlands, the US and the UK. Orchard is headquartered in London, Greater London, the UK.

The company reported revenues of (US Dollars) US$22.7 million for the fiscal year ended December 2022 (FY2022), compared to a revenue of US$1.7 million in FY2021. The operating loss of the company was US$127.1 million in FY2022, compared to an operating loss of US$140.4 million in FY2021. The net loss of the company was US$150.7 million in FY2022, compared to a net loss of US$144.6 million in FY2021.

For a complete picture of OTL-201’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 17 November 2023

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To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, drug margins and company expenses. 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 valuation, 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.