DO-1 is under clinical development by Dorphan and currently in the Phase I 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 DO-1’s likelihood of approval (LoA) and phase transition for GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) took place on 13 May 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their DO-1 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

DO-1 overview

DO-1 is under development for the treatment of MPSIVB (mucopolysaccharidosis IV type B) and GM1 gangliosidosis. The drug candidate is administered orally. The drug candidate is an iminosugar. The drug candidate targets mutated form of beta galactosidases.

Dorphan overview

Dorphan is a preclinical-stage drug discovery and development company that develops therapeutics for the treatment of devastating lysosomal storage diseases and mucopolysaccharidoses. The company’s pipeline product includes DO-1, a preclinical chaperone drug candidate for the treatment of GM1-gangliosidosis and mucopolysaccharidosis type IVB. It develops treatments for lysosomal storage diseases through small molecule-based pharmacological chaperone therapies. Dorphan also develops molecules for restoring the activity of mutated enzymes in mucopolysaccharidoses and lysosomal storage diseases. Dorphan is headquartered in Epalinges, Lausanne, Switzerland.

Quick View DO-1 LOA Data

Report Segments
  • Innovator
Drug Name
  • DO-1
Administration Pathway
  • Oral
Therapeutic Areas
  • Genetic Disorders
Key Developers
Highest Development Stage
  • Phase I

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.