Sapablursen sodium is under clinical development by Ionis Pharmaceuticals and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.
According to GlobalData, the latest event to affect Sapablursen sodium’s likelihood of approval (LoA) and phase transition for Beta Thalassaemia took place on 28 Dec 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.
GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Sapablursen sodium Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.
Sapablursen sodium overview
IONISTMPRSS-6LRx is under development for the treatment of beta-thalassemia intermedia and 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.
Ionis Pharmaceuticals overview
Ionis Pharmaceuticals (Ionis), formerly ISIS Pharmaceuticals, Inc., 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.
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