Ascletis announces that the clinical trial application for ASC10 monkeypox indication has been approved by the US FDA

Based on available data, the U.S. Food and Drug Administration (FDA) has approved ASC10 for a Phase Ib study in monkeypox virus patients at a dose of 800 mg twice daily

Preclinical studies have shown that ASC10-A, the active metabolite of ASC10, has potent antiviral activity against monkeypox virus and novel coronavirus

Ascletis Pharmaceutical's WeChat public account announced today (November 16) morning that the company's board of directors announced that the US Food and Drug Administration (FDA) has approved the ASC10 monkeypox indication new drug clinical trial (IND) application. Based on available data, the US FDA has approved a randomized, double-blind, placebo-controlled Phase Ib study of ASC10 at a dose of 800 mg twice daily to evaluate the safety, tolerability, efficacy, and pharmacokinetics of ASC10 tablets in monkeypox virus patients.

ASC10 is an oral double prodrug. When administered orally, both ASC10 and the single prodrug monopiravir (molnupiravir) are rapidly and completely converted in vivo to the same active metabolite ASC10-A, also known as β-D-N4-hydroxycytidine (NHC) or EIDD-1931.

Preclinical studies have shown that ASC10-A has broad-spectrum antiviral activity, including monkeypox virus and novel coronavirus. Ascletis has filed several patent applications worldwide for ASC10 and its use in the treatment of viral diseases, including the treatment of monkeypox virus infection.

Monkeypox virus belongs to the genus Orthopoxvirus and causes illness similar to smallpox symptoms. According to the World Health Organization (WHO), as of 15 November 2022, there have been more than 79,000 confirmed cases worldwide and monkeypox virus has been circulating in 110 countries. Of these, a total of 28,947 confirmed cases of monkeypox virus have been reported from the US. The WHO assesses the Americas as a high-risk area for monkeypox virus.

Experimental data in vitro monkeypox virus infection cell models show that ASC10-A has potent antiviral activity against monkeypox virus, indicating that ASC10 has the potential to be an effective therapy for monkeypox virus infection, which was commissioned by Ascletis to be conducted by the IIT Institute (IITRI) of the Illinois Institute of Technology in Chicago, USA. Researchers at the National Institute of Infectious Diseases in Tokyo, Japan, tested 132 drugs and showed that monoravir (active metabolite ASC10-A) and two other drugs had potent cellular antiviral activity in monkeypox virus infection models. The remaining 129 drugs, such as remdesivir, favipiravir, sofosbuvir, and ribavirin, have no anti-monkeypox virus activity.

Dr. Jinzi Wu, Founder, Chairman of the Board and Chief Executive Officer of Ascletis:

No treatment for monkeypox virus infection has been approved globally, so it is imperative to develop a safe, effective and affordable monkeypox drug. The approval of ASC10 monkeypox indication IND by the US FDA further demonstrates Ascletis in the field of viral diseases and will accelerate our efforts to better address the global challenges posed by the widespread spread of monkeypox."