NTLA-2001 is a gene therapy commercialized by Intellia Therapeutics, with a leading Phase III program in Familial Amyloid Cardiomyopathy. According to Globaldata, it is involved in 3 clinical trials, of which 2 are ongoing, and 1 is planned. GlobalData uses proprietary data and analytics to provide a complete picture of NTLA-2001’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for NTLA-2001 is expected to reach an annual total of $983 mn by 2038 in the US 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.

NTLA-2001 Overview

NTLA-2001 is under development for the treatment of patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRV-PN) and patients with transthyretin amyloidosis-related cardiomyopathy (ATTR-CM) The therapeutic candidate is administered through intravenous as an injection. It constitutes CRISPR/Cas9 guide RNAs directed to transthyretin (TTR) gene, packed in a lipid nanoparticle. It is developed based on CRISPR/Cas9 genome-editing technology.

Intellia Therapeutics Overview

Intellia Therapeutics is a biotechnology company. The company developing curative therapeutics using Clustered, Regularly Interspaced Short Palindromic Repeats and CRISPR associated 9 (CRISPR and Cas9) technology. Its pipeline products include NTLA-2001, NTLA-2002, NTLA-2003 and NTLA-3001. It develops in vivo programs for the treatment of liver diseases such as alpha-1 antitrypsin deficiency, and primary hyperoxaluria Type 1. Intellia Therapeutics also develops ex vivo programs such as hematopoietic stem cells (HSCs) and chimeric antigen receptor T cells (CAR-T cells). The company conducts genome editing in the fields of immuno-oncology and autoimmune and inflammatory diseases. Intellia Therapeutics is headquartered in Cambridge, Massachusetts, the US.
The company reported revenues of (US Dollars) US$36.3 million for the fiscal year ended December 2023 (FY2023), a decrease of 30.4% over FY2022. The operating loss of the company was US$515.3 million in FY2023, compared to an operating loss of US$458.2 million in FY2022. The net loss of the company was US$481.2 million in FY2023, compared to a net loss of US$474.2 million in FY2022.

For a complete picture of NTLA-2001’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 10 June 2024

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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.