Arimoclomol is a small molecule commercialized by Zevra Therapeutics, with a leading Pre-Registration program in Niemann-Pick Disease Type C. According to Globaldata, it is involved in 11 clinical trials, of which 7 were completed, 1 is ongoing, and 3 were terminated. GlobalData uses proprietary data and analytics to provide a complete picture of Arimoclomol’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

Smarter leaders trust GlobalData

The revenue for Arimoclomol is expected to reach an annual total of $160 mn by 2033 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.

Arimoclomol Overview

Arimoclomol citrate (BRX-220) is under development for the treatment of Niemann-Pick disease type C. The drug candidate is a new molecular entity which is administered through oral route in the form of extended-release capsule. It acts by targeting heat shock protein 70.

The drug candidate was also under development for the treatment of stroke, peripheral neuropathy, insulin resistance, GCase-Parkinson’s disease sporadic inclusion body myositis (s-IBM), amyotrophic lateral sclerosis (ALS), sporadic inclusion body myositis and Gaucher disease Type 1 and Type 3.

Zevra Therapeutics Overview

Zevra Therapeutics (Zevra) formerly KemPharm Inc, s a rare disease therapeutics company that offers discovery and development of proprietary new molecular entity prodrugs. Its product portfolio include Arimoclomol, KP1077IH, KP1077N and Azstarys. Arimoclomol is an orally delivered, investigational product candidate being developed for Niemann-Pick disease type C (NPC). KP1077 is the Company’s lead clinical development product candidate, which is being developed as a treatment for idiopathic hypersomnia (IH) and narcolepsy. KP1077 is comprised solely of serdexmethylphenidate (SDX), the Company’s prodrug of d-methylphenidate (d-MPH). AZSTARYS is a prodrug for the treatment of ADHD in patients aged six years or older. Zevra is headquartered in Florida, the US.
The company reported revenues of (US Dollars) US$10.5 million for the fiscal year ended December 2022 (FY2022), a decrease of 63.5% over FY2021. The operating loss of the company was US$42.5 million in FY2022, compared to an operating loss of US$8.4 million in FY2021. The net loss of the company was US$41.5 million in FY2022, compared to a net loss of US$8.6 million in FY2021. The company reported revenues of US$2.9 million for the third quarter ended September 2023, a decrease of 65.8% over the previous quarter.

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

This content was updated on 10 June 2024

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


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.