A drug to treat monkeypox from Ascletis Pharma has had its investigational new drug (IND) application approved by the U.S Food and Drug Administration (FDA).
The FDA made its decision based on the available data, which was 800 mg ASC10 taken twice a day, and Ascletis can now go ahead with a phase 1b clinical trial to evaluate the safety, tolerability, efficacy and pharmacokinetics of the tablets in patients with monkeypox virus disease.
ASC10 is an oral double prodrug. After oral administration, both ASC10 and single prodrug molnupiravir are rapidly and completely converted in vivo into the same active metabolite ASC10-A, also known as β-D-N4-hydroxycytidine (NHC) or EIDD-1931.
Ascletis’ research: monkeypox similar to smallpox
Preclinical studies show that ASC10-A has broad spectrum antiviral activities including potent activities against both monkeypox and SARS-CoV-2 viruses. Ascletis has filed multiple patent applications globally for ASC10 and its use in viral diseases including monkeypox virus infection.
In its research, Ascletis through the World Health Organisation (WHO) found that monkeypox virus is an orthopoxvirus that causes a disease with symptoms similar to smallpox.
As of yesterday (November 15), more than 79,000 confirmed cases have been reported globally and monkeypox virus has spread in 110 countries. In particular, a total of 28,947 confirmed monkeypox cases have been reported in the U.S. WHO assesses the risk of monkeypox virus in the region of the Americas as high.
FDA further validates Ascletis further
Data from in vitro antiviral cellular assay with infectious monkeypox virus, a study sponsored by Ascletis and conducted at IIT Research Institute (IITRI) of Illinois Institute of Technology in Chicago, U.S., showed that ASC10-A has potent antiviral activity against monkeypox virus, suggesting that ASC10 has the potential to be an effective treatment of monkeypox virus infection.
Researchers from National Institute of Infectious Diseases, Tokyo, Japan, tested 132 drugs and the results showed that molnupiravir (active metabolite ASC10-A) and other two drugs have potent cellular antiviral activity in the infectious monkeypox virus assay. The rest of 129 drugs such as remdesivir, favipiravir, sofosbuvir and ribavirin, do not have antiviral activities against monkeypox virus, Ascletis reported.
Jinzi J. Wu, founder, chairman and CEO of Ascletis, said: “It is urgently needed to develop a safe, effective and affordable solution for monkeypox as there is no approved treatment in the world. This IND approval of ASC10 for monkeypox from U.S. FDA further validates Ascletis’ in-house R&D capabilities on viral diseases, and it will accelerate our efforts to better address the global challenge imposed by the widespread monkeypox.”