Avicursen, a drug developed by Percheron Therapeutics, is currently undergoing Phase II clinical trials as a potential treatment for Duchenne Muscular Dystrophy (DMD). The drug's progress is being closely monitored, especially considering the historical success rate of DMD drugs transitioning from Phase II to Phase III trials.
Avicursen (ATL-1102): Mechanism and Development
Avicursen, also known as ATL-1102, is a second-generation antisense inhibitor targeting CD49d, a subunit of VLA-4 (very late antigen-4). It is designed to be administered either subcutaneously or via inhalation. The drug is formulated as a solution. Percheron Therapeutics is leveraging antisense technology in the development of Avicursen.
Percheron Therapeutics: Focus on Antisense Therapeutics
Percheron Therapeutics, formerly known as Antisense Therapeutics, is a biopharmaceutical company focused on the discovery, development, and commercialization of second-generation antisense pharmaceuticals. The company's pipeline includes treatments for immunology disorders and rare diseases, such as multiple sclerosis (MS) and DMD. Besides Avicursen, Percheron is also developing ATL1103 for growth hormone disorders like Acromegaly and ATL1102 (injection) for multiple sclerosis, which aims to reduce brain lesions.
Phase Transition Success Rate
According to GlobalData, Phase II drugs for Duchenne Muscular Dystrophy have a 61% phase transition success rate (PTSR) benchmark for progressing into Phase III. GlobalData assesses Avicursen's drug-specific PTSR and Likelihood of Approval (LoA) scores compared to these established benchmarks, considering attributes of the drug, the company, and its clinical trials.
