ALXN-2220 is under clinical development by Alexion Pharmaceuticals and currently in Phase I for Familial Amyloid Cardiomyopathy. According to GlobalData, Phase I drugs for Familial Amyloid Cardiomyopathy have a 100% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how ALXN-2220’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

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.

ALXN-2220 overview

NI-301 is under development for the treatment of familial amyloid cardiomyopathy (ATTR-CM), ATTR amyloidosis and unspecified indication. It is administered through intravenous route. The drug candidate is a recombinant human monoclonal IgG1 which acts by targeting transthyretin (TTR) protein. It is developed using reverse translational medicine (RTM) technology.

It was under development for the treatment of familial amyloid polyneuropathy.

Alexion Pharmaceuticals overview

Alexion Pharmaceuticals (Alexion), a subsidiary of AstraZeneca Plc, discovers, develops and markets therapies for severe and life-threatening diseases. The company’s brands comprise Kanuma, Koselug, Soliris, StrensIQ and Ultomiris among others. It develops medicines to treat conditions such as atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), hypophosphatasia (HPP), lysosomal acid lipase de?ciency (LAL-D), neurofibromatosis type 1 plexiform neurofibromas (NF1 PN), neuromyelitis optica spectrum disorder (NMOSD) and paroxysmal nocturnal hemoglobinuria (PNH). Alexion’s therapeutic areas include hematology, nephrology, neurology, metabolics, cardiology and others. Alexion collaborates with various research organizations and pharmaceutical companies to develop treatments for rare and severe diseases. The company operates in Europe and the Middle East, North America, Latin America and Asia Pacific. Alexion is headquartered in Boston, Massachusetts, the US.

For a complete picture of ALXN-2220’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 22 April 2024

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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.