ACTUS-101 is under clinical development by Asklepios BioPharmaceutical 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 ACTUS-101’s likelihood of approval (LoA) and phase transition for Pompe Disease took place on 24 Dec 2020, 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 ACTUS-101 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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) is AAV gene therapy development company that develops and markets novel protein and cellular based therapies. Its product pipeline includes therapies for the treatment of neuromuscular, central nervous system, heart related diseases including pompe, limb-girdle 2i, huntington’s, epilepsy, parkinson’s, and congestive heart failure (nanocor). AskBio has developed library of AAV biological nano particles that provides unsurpassed transduction. It serves pharmaceutical and biotechnology companies. AskBio is headquartered in Durham, North Carolina, the US.

Quick View ACTUS-101 LOA Data

Report Segments
  • Innovator
Drug Name
  • ACTUS-101
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Metabolic Disorders
Key Developers
Highest Development Stage
  • Phase II

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.