Sapablursen sodium is under clinical development by Ionis Pharmaceuticals and currently in Phase II for Polycythemia Vera. According to GlobalData, Phase II drugs for Polycythemia Vera have a 40% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Sapablursen sodium’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.

Sapablursen sodium overview

IONISTMPRSS-6LRx is under development for the treatment of phlebotomy dependent polycythemia vera. It is administered through subcutaneous route. The therapeutic candidate is a second generation antisense oligonucleotide that targets transmembrane protease serine 6 (TMPRSS6, also known as matriptase-2). It is developed based on ligand-conjugated antisense (LICA) technology.

It was under development for the treatment of beta-thalassemia intermedia.

Ionis Pharmaceuticals overview

Ionis Pharmaceuticals (Ionis), formerly Isis Pharmaceuticals, focuses on the discovery and development of RNA-targeted drugs. The company develops medicines to treat various diseases including cancer, pulmonary, cardiovascular diseases, neurological and infectious diseases. Its major products include Spinraza for spinal muscular atrophy, Tegsedi for neuropathy, Waylivra for familial chylomicronemia syndrome, Qalsody for superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS), and Wainua for polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN). Ionis also has a pipeline of products in different phases of clinical trials. It develops products based on its proprietary antisense technology. The company partnered with other pharmaceutical companies to develop a range of products in different therapeutic areas. The company sells its products in North America and Europe. Ionis is headquartered in Carlsbad, California, the US.

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

This content was updated on 4 December 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.