Arrowhead Research Corporation has been cleared to start its modified multiple-dose study of ARC-520 in patients who suffer from chronic hepatitis B infection by the U.S. Food and Drug Administration (FDA). The company is dedicated to the creation of targeted RNAi therapies, was notified verbally by the FDA through a preliminary call of a partial clinical hold.
Despite the approval of the ARC-520 study, the FDA requested the study to include 1 mg/kg dosages, instead of 2 and 4 mg/kg, as proposed in the design of the study. In addition, the agency also asked the company for additional information, regarding a final study report about the ongoing phase 2a trial in patients being administrated with 1-4 mg/kg ARC-520, as well as a final study report about an ongoing multiple-dose non-clinical study.
“Over the next 30 days, Arrowhead will begin preparations for the multiple-dose Phase 2b study,” stated the Arrowhead president and CEO, Christopher Anzalone. “We will work closely with the FDA throughout this process while we continue to seek approval to proceed with other planned studies in Asia and Europe,” he added, while the agency has committed to deliver a letter with the details on the requests within the next 30 days.
ARC-520 is an RNAi-based candidate for the treatment of chronic HBV infection, which is being studied to understand its ability to promote a functional cure for the disease. This implicates an immune clearant state characterized by hepatitis B s-antigen negative serum with or without sero-conversion. The small interfering RNAs (siRNAs) in ARC-520 acts at the mRNA level, upstream of the reverse transcription process, which is where the current standard of care nucleotide and nucleoside analogues take place.
Arrowhead is currently analyzing ARC-520 in a single-dose phase 2a study, which includes patients who are being treated with 1-4 mg/kg of the compound, and the preliminary results demonstrated to date report no serious adverse events or evidence of end organ toxicity. In addition, the multiple-dose, non-clinical study also currently underway demonstrated that the drug is well tolerated. The company is now planning on filing for additional phase 2b studies with both Asian and European agencies within the next weeks.
The studies of ARC-520 are receiving great attention from the medical community and public, as it can represent a potential cure for chronic HBV infection, a disease that currently affects about 350 to 400 million people all over the world. The chronic condition can cause cirrhosis of the liver and is responsible for 80% of primary liver cancers.