Ulefnersen sodium is under clinical development by Ionis Pharmaceuticals and currently in Phase III for Amyotrophic Lateral Sclerosis. According to GlobalData, Phase III drugs for Amyotrophic Lateral Sclerosis have a 32% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Ulefnersen sodium’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.
Ulefnersen sodium overview
ION363 is under development for the treatment of amyotrophic lateral sclerosis (ALS) in patients with mutations in the Fused in Sarcoma (FUS) gene. It is a generation 2 antisense drug that prevents the production of the FUS protein. It is administered through intrathecal bolus route.
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 and Waylivra for familial chylomicronemia syndrome. 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 Ulefnersen sodium’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.
