RPL-301 is under clinical development by Rocket Pharmaceuticals and currently in Phase I for Anemia. According to GlobalData, Phase I drugs for Anemia have an 82% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how RPL-301’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.

RPL-301 overview

RPL-301 is under development for the treatment of pyruvate kinase deficiency. The therapeutic candidate constitutes patient-derived immature bone marrow cells (called CD34+) genetically modified with a lentivirus to express pyruvate kinase (PKLR) enzyme. It is administered through intravenous route.

Rocket Pharmaceuticals overview

Rocket Pharmaceuticals (Rocket Pharma), formerly Inotek Pharmaceuticals Corp is a clinical-stage biopharmaceutical company. It focuses on

For a complete picture of RPL-301’s drug-specific PTSR and LoA scores, buy the report here.


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 its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.