ALN-APP is under clinical development by Alnylam Pharmaceuticals and currently in Phase I for Alzheimer’s Disease. According to GlobalData, Phase I drugs for Alzheimer’s Disease have a 63% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how ALN-APP’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. 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.

ALN-APP overview

ALN-APP is under development for the treatment of  Alzheimer’s Disease, cerebral amyloid angiopathy and early-onset Alzheimer’s Disease (EOAD). It acts by targeting amyloid precursor protein (APP). It is administered through intrathecal route. It is being developed based on C16-siRNA conjugate platform.

Alnylam Pharmaceuticals overview

Alnylam Pharmaceuticals (Alnylam) is a biopharmaceutical company, which discovers, develops, and commercializes drugs based on RNAi interference. The company’s marketed RNAi therapies include Onpattro, which is used in the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis; Givlaari for acute hepatic porphyria, Amvuttra, for the treatment of ATTR amyloidosis with cardiomyopathy; and Oxlumo for primary hyperoxaluria type 1 (PH1). The company develops a broad pipeline of investigational RNAi drugs in the areas of genetic medicines, hepatic infectious, cardio-metabolic, and central nervous system (CNS) diseases. Its late-stage product candidates include vutrisiran for ATTR amyloidosis, inclisiran for hypercholesterolemia, fitusiran for hemophilia and rare bleeding disorders, and lumasiran for severe PH1. The company operates subsidiaries in North America, Europe and Asia-Pacific. Alnylam is headquartered in Cambridge, Massachusetts, the US.

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

This content was updated on 20 February 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.