Arimoclomol citrate ER is under clinical development by Zevra Therapeutics and currently in Pre-Registration for Niemann-Pick Disease Type C. According to GlobalData, Pre-Registration drugs for Niemann-Pick Disease Type C does not have sufficient historical data to build an indication benchmark PTSR for Pre-Registration. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Arimoclomol citrate ER LoA Report. Buy the report here.
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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Arimoclomol citrate ER overview
Arimoclomol citrate (BRX-220) was under development for the treatment of amyotrophic lateral sclerosis (ALS), sporadic inclusion body myositis and Gaucher disease Type 1 and Type 3. 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 Niemann-Pick disease type C, stroke, peripheral neuropathy, insulin resistance, GCase-Parkinson’s disease sporadic inclusion body myositis (s-IBM) and amyotrophic lateral sclerosis.
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.
For a complete picture of Arimoclomol citrate ER’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
