Shanghai, China, Dec. 9th, 2022 — Shanghai Ark Biopharmaceutical Co., Ltd. (Ark Biopharmaceutical), a biopharma company dedicated to the discovery and development of drugs for pediatric and respiratory diseases, today announced that the China’s National Medical Products Administration (NMPA) has formally accepted and granted Priority Review of its New Drug Application (NDA) for ziresovir (AK0529) for treatment of respiratory syncytial virus (RSV) infection.
A novel small-molecule RSV fusion (F) protein inhibitor, ziresovir is the first anti-RSV drug that has successfully completed phase 3 clinical study globally. Notably, as the first innovative pediatric drug discovered and developed in China before expanding to the rest of the world, it has received the first Breakthrough Therapy designation ever granted by NMPA to non-oncology investigational products.
The NDA submission was based on study results of the phase 3 registration trial (AirFLO): ziresovir demonstrated a rapid-onset of clinical effect, achieving 30% placebo-adjusted reduction in S&S score (p=0.002) on day 3 of treatment. The effect was most pronounced in young patients under 6 months. In this group, who are at high risk for severe RSV infection, a 55% placebo-adjusted reduction in S&S score was achieved (p<0.001). Moreover, in patients admitted to an intensive care unit (ICU), the length of ICU stay was significantly shorter in the ziresovir group than the placebo group (median 3.0 vs. 8.0 days; p=0.05). Ziresovir also showed a significant anti-viral effect: the ziresovir group showed 77% greater effect in viral load reduction on day 5 compared with the placebo group (p=0.006); and statistically significant viral load reduction in the ziresovir group throughout the treatment period. Follow-up data are available for up to 6 months, during which the incidence rate of recurrent wheezing was significantly lower in children under 6-month-old who were treated with ziresovir compared to the placebo group (9.0% vs. 26.3%; p=0.02). The AirFLO study showed good safety and tolerability profile for ziresovir in infants.
Dr. Jim Wu, CEO of Ark Biopharmaceutical commented: "This is our first NDA submission for a Class I New Drug accepted for review by China NMPA, and it represents an important business milestone for Ark Biopharmaceutical to become a biopharmaceutical company with both R&D and commercialization capabilities. With regulatory guidance and advice from NMPA, we will engage closely with the medical community to bring ziresovir to market and benefit Chinese RSV patients soon."
RSV is a highly prevalent airborne RNA virus which enters the trachea and lungs through oral and nasal inhalation, invades epithelial cells and causes damage in respiratory tract, which can lead to excessive airway mucus, lumen blockage, and dyspnea. The common symptoms of RSV infection are runny nose, fever, cough, and wheeze. Under serious conditions, respiratory distress and complete airway blockage may occur, leading to respiratory failure and death.
RSV infection is the leading cause of hospitalization and death in children under five years of age worldwide. According to The Lancet, there were 33 million RSV-related acute lower respiratory tract infections among children under 5 years of age worldwide in 2019, resulting in 3.6 million hospital admissions and 100,000 deaths due to RSV. Even in children with mild symptoms, untreated RSV infection may develop into chronic respiratory diseases, such as wheeze and asthma, and affect a child’s long-term growth and development.
The populations most susceptible to RSV infection are children, the elderly, and adults with impaired immune functions. Over 90% of children are infected with RSV before 2 years of age. At present, there are neither effective vaccine nor treatments available that specifically target RSV.
Ziresovir is a novel small-molecule RSV fusion (F) protein inhibitor. It binds to the F protein of the virus and prevents viral entry into human cells. Ziresovir can also suppress virus transmission by blocking cell fusion through the formation of “syncytia”, a characteristic event of RSV infection of host cells.
Ziresovir is the first oral anti-RSV drug that has successfully completed a phase 3 pivotal clinical study globally. It is the first innovative pediatric drug discovered and developed in China before expanding to the rest of the world. Ziresovir is also the first non-oncology investigational product ever granted Breakthrough Therapy designation by the Chinese National Medical Products Administration (NMPA).
About Ark Biopharmaceutical
Ark Biopharmaceutical is a biopharmaceutical company focusing on discovering and developing novel drugs for unmet medical needs, especially in pediatric and respiratory diseases. Since its inception in 2014, the company has established core technology platforms and discovered drug candidates with global patent protection. Through in-house R&D efforts and external collaborations, it has developed a promising and highly differentiated product pipeline. The core product, ziresovir, is the leading RSV antiviral drug that received a Breakthrough Therapy designation from NMPA. Another near-commercial asset is an FDA-approved ADHD drug AZSTARYS. Ark Biopharmaceutical owns the rights for development and marketing AZSTARYS in Greater China. The pipeline also includes a battery of clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global market.
Ark Biopharmaceutical has a track record of successful collaboration and partnership with multinational pharma companies such as Roche/Genentech, world-leading academic institutions such as The Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and global biotech companies, as well as multinational investment institutions.