NTLA-2001 is under clinical development by Regeneron Pharmaceuticals and currently in Phase III for Familial Amyloid Cardiomyopathy. According to GlobalData, Phase III drugs for Familial Amyloid Cardiomyopathy have a 75% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how NTLA-2001’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.
NTLA-2001 overview
NTLA-2001 is under development for the treatment of patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRV-PN) and patients with transthyretin amyloidosis-related cardiomyopathy (ATTR-CM) The therapeutic candidate is administered through intravenous as an injection. It constitutes CRISPR/Cas9 guide RNAs directed to transthyretin (TTR) gene, packed in a lipid nanoparticle. It is developed based on CRISPR/Cas9 genome-editing technology.
Regeneron Pharmaceuticals overview
Regeneron Pharmaceuticals (Regeneron) discovers, develops, manufactures, and markets medicines for the treatment of serious medical conditions. Its products are focused on helping patients suffering from cancer, allergic and inflammatory diseases, eye diseases, metabolic and cardiovascular diseases, infectious diseases, neuromuscular diseases, hematologic conditions, pain, and rare diseases. Regeneron develops products using its Trap and VelociSuite technology platforms, which include VelocImmune, VelociGene, VelociMouse, VelociMab, Veloci-Bi, VelociT, and other related technologies. The company operates manufacturing facilities in Rensselaer, New York, and Limerick. It also collaborates with strategic partners to develop, manufacture, and commercialize its products. It sells its products to specialty pharmacies and distributors. The company operates in the US, the Netherlands, Bermuda, Ireland, Spain, India, Japan and the UK. Regeneron is headquartered in Tarrytown, New York, the US.
For a complete picture of NTLA-2001’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.
