Trofinetide is a small molecule commercialized by Acadia Pharmaceuticals, with a leading Pre-Registration program in Rett Syndrome. According to Globaldata, it is involved in 15 clinical trials, of which 12 were completed, 2 are ongoing, and 1 was terminated. GlobalData uses proprietary data and analytics to provide a complete picture of Trofinetide’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for Trofinetide is expected to reach an annual total of $346 mn by 2032 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.

Trofinetide Overview

NNZ-2566 (trofinetide) is under development for the treatment of Fragile X Syndrome, Rett Syndrome. It is administered through oral and intravenous route. The drug candidate is a synthetic analogue of a molecule derived from IGF-1. It was under development for the treatment of concussion and traumatic brain injury.

Acadia Pharmaceuticals Overview

Acadia Pharmaceuticals (Acadia) discovers, develops, and commercializes small molecule drugs for the treatment of central nervous system (CNS) disorders. The company’s marketed product Nuplazid (pimavanserin) is an FDA-approved drug for the treatment of Parkinson’s disease psychosis. The company’s pipeline includes pimavanserin for the treatment of various indications which include Schizophrenia, Major Depressive Disorder (MDD), dementia-related psychosis (DRP), and trofinetide for the treatment of Rett Syndrome. The company markets its product in the US. It has operational presence in Denmark, Switzerland, and the UK. Acadia is headquartered in San Diego, California, the US.

The company reported revenues of (US Dollars) US$484.1 million for the fiscal year ended December 2021 (FY2021), an increase of 9.6% over FY2020. The operating loss of the company was US$170.4 million in FY2021, compared to an operating loss of US$286.6 million in FY2020. The net loss of the company was US$167.9 million in FY2021, compared to a net loss of US$281.6 million in FY2020. The company reported revenues of US$130.7 million for the third quarter ended September 2022, a decrease of 2.9% over the previous quarter.

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


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, 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.