Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") announced the successful completion of the Phase III clinical study of AK0901, a novel drug for the treatment of attention deficit hyperactivity disorder (ADHD). This milestone represents a substantial step forward for ArkBio in the research and development of drugs for pediatric psychiatric disorders, potentially offering new treatment options and improved therapeutic outcomes for children with ADHD in China.

ADHD is a chronic neurodevelopmental disorder characterized by age-inappropriate attention deficits and/or hyperactivity-impulsivity, often persisting into adulthood. Globally, approximately 7.2% of children are affected, with 60%–80% continuing to exhibit symptoms during adolescence and 50.9% into adulthood. About 65% of patients present with one or more comorbidities. ADHD impairs learning and causes multidimensional, lifelong functional challenges. In China, the prevalence of ADHD among children and adolescents is 6.4%, affecting over 23 million individuals. However, current treatment options remain limited, with existing drugs often falling short in terms of efficacy, safety, or convenience of drug administration, leading to poor patient compliance.

AK0901, a novel methylphenidate-based drug, is the first and only drug combining two active components of immediate-release dexmethylphenidate (d-MPH) and prodrug serdexmethylphenidate (SDX). It works by inhibiting the reuptake of dopamine and norepinephrine in the synaptic cleft, enhancing neurotransmitter efficiency to alleviate ADHD symptoms. AK0901 (Azstarys?) received U.S. FDA approval in March 2021 as the new-generation methylphenidate drug in nearly two decades, demonstrating superior safety and clinical efficacy, and a potential as best-in-class therapeutic drug for the treatment of pediatric ADHD.

The Phase III clinical study of AK0901 in China was led jointly by Beijing Anding Hospital affiliated with Capital Medical University and Peking University Sixth Hospital, with the participation of eight clinical centers nationwide. The study aimed to evaluate the efficacy, safety, and tolerability of oral AK0901 in children with ADHD aged 6 to 12. The study results met both primary and key secondary endpoints and reached statistical significance in all the visiting time points compared to placebo, which provides a strong scientific basis for future treatment of Chinese ADHD patients. 

Professor Zheng Yi, the chief expert of the pediatric team at Beijing Anding Hospital affiliated with Capital Medical University, stated,"The Phase III study of AK0901 has systematically validated its therapeutic value in Chinese children with ADHD through a rigorous multi-center randomized controlled clinical study. As a new generation methylphenidate drug, the synergistic effect of its immediate-release and prodrug components not only enables rapid symptom control but also reduces the compliance issues associated with current methylphenidate drugs due to the fluctuation of blood drug concentration. This should serve better the stepwise needs of school-aged children for 'classroom concentration + after-school behavior management', making disease management more feasible and effective. The research data provide high-quality evidence to support the establishment of a comprehensive management system for ADHD that suits the characteristics of Chinese children. We look forward to its approval and launch in China to upgrade the treatment option that benefits patients and families."

Professor Liu Jing, Director of the Center for Child and Adolescent Mental Health of Peking University, stated, "This study marks a breakthrough in confirmation of the efficacy and safety of AK0901 in Chinese ADHD children. Its unique dual component formulation of immediate-release and prodrug methylphenidate significantly reduces the peak-trough fluctuations of traditional methylphenidate drugs, maintaining stable symptom control while reducing the risk of drug abuse. This is of significant clinical value for ADHD patients who require years of continuous intervention. This achievement not only addresses the core concern of clinical safety for long-term medication in children but also provides a new solution for balancing the efficacy needs with long-term safety of ADHD children. We look forward to the market approval and launch of AK0901 for clinical use in China, allowing more Chinese children with ADHD to benefit and grow up healthily."

Dr. Jim Wu, CEO of ArkBio, stated, "The completion of AK0901 Phase III study marks a critical step forward in addressing unmet needs in ADHD treatment in China. We extend our sincere gratitude to all participants, the physician team and partners involved in this study for their efforts and contributions. This milestone will drive our continued commitment to advancing scientific innovation and drug development of ADHD therapeutics. "

About ArkBio

ArkBio is a global biotech company focusing on discovery and development of innovative drugs for unmet medical needs, especially in the therapeutic areas of respiratory, infectious, and pediatric diseases. Since its inception in 2014, the company has established core technology platforms and built an innovative and highly differentiated R&D pipeline through in-house R&D efforts and external collaborations. Our strong R&D pipeline includes innovative clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global clinical development. As one of the core assets, ziresovir is the first direct-acting RSV antiviral drug that has demonstrated positive results from a pivotal phase III study globally. Another near-commercialization product is AK0901, an FDA-approved novel therapy for pediatric ADHD, with ArkBio holding development and commercialization rights in Greater China.

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology, Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture investment institutions.