ACTUS-101 is under clinical development by Asklepios BioPharmaceutical and currently in Phase II for Pompe Disease. According to GlobalData, Phase II drugs for Pompe Disease have a 63% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how ACTUS-101’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.
ACTUS-101 overview
ACTUS-101 is under development for the treatment of Pompe disease. The drug candidate is a non-replicating recombinant adeno-associated virus (AAV 2/8), which is administered through intravenous route. It acts by targeting the enzyme acid alpha-glucosidase.
Asklepios BioPharmaceutical overview
Asklepios BioPharmaceutical (AskBio), a subsidiary of Bayer AG, is a clinical-stage gene therapy company. It focuses on advancing genetic technology and developing life-saving adeno-associated virus (AAV) gene therapies. The company’s main activities include research and development of gene therapies targeting a range of disorders, including central nervous system, neuromuscular, metabolic, and cardiovascular diseases. AskBio’s product pipeline includes therapies for the treatment of neuromuscular, central nervous system, heart-related diseases including pompe, limb-girdle 2i, huntington’s, multiple system atrophy, parkinson’s, and congestive heart failure (nanocor). Its gene therapy solutions are utilized by patients and healthcare providers to address genetic disorders, with applications spanning various medical fields. The company operates in the US, Scotland, Germany, Spain, and France. AskBio is headquartered in Research Triangle Park, North Carolina, the US.
For a complete picture of ACTUS-101’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.
