Shanghai Ark Biopharmaceutical Co., Ltd. (ArkBio) announced today the successful completion of Phase III clinical trials for AK0901, a novel methylphenidate-based drug developed for treating attention deficit hyperactivity disorder (ADHD). The study results represent a significant advancement in addressing the substantial unmet treatment needs for the estimated 23 million children and adolescents affected by ADHD in China.
The multi-center clinical trial, jointly led by Beijing Anding Hospital and Peking University Sixth Hospital, evaluated the efficacy, safety, and tolerability of AK0901 in children with ADHD aged 6 to 12 years. Results demonstrated that the drug met both primary and key secondary endpoints with statistical significance at all assessment timepoints compared to placebo.
A Novel Dual-Component Approach to ADHD Treatment
AK0901 stands out as the first and only ADHD medication combining two active components: immediate-release dexmethylphenidate (d-MPH) and prodrug serdexmethylphenidate (SDX). This innovative formulation works by inhibiting the reuptake of dopamine and norepinephrine in the synaptic cleft, enhancing neurotransmitter efficiency to alleviate core ADHD symptoms.
The drug, marketed as Azstarys® in the United States, received FDA approval in March 2021 as the first new-generation methylphenidate drug in nearly two decades. ArkBio holds the development and commercialization rights for the medication in Greater China.
Addressing a Significant Public Health Challenge
ADHD represents a substantial burden in China, with a prevalence rate of 6.4% among children and adolescents. The neurodevelopmental disorder is characterized by age-inappropriate attention deficits and/or hyperactivity-impulsivity that often persist into adulthood. Globally, approximately 7.2% of children are affected, with 60%-80% continuing to exhibit symptoms during adolescence and 50.9% into adulthood.
Professor Zheng Yi, chief expert of the pediatric team at Beijing Anding Hospital affiliated with Capital Medical University, highlighted the drug's clinical advantages: "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 dual-action mechanism addresses a key challenge in ADHD management by providing both immediate symptom relief and sustained control throughout the day. Professor Zheng noted that this approach better serves "the stepwise needs of school-aged children for 'classroom concentration + after-school behavior management,' making disease management more feasible and effective."
Improved Safety Profile and Reduced Abuse Potential
A significant advantage of AK0901 is its potential for reduced abuse risk compared to traditional stimulant medications. Professor Liu Jing, Director of the Center for Child and Adolescent Mental Health of Peking University, emphasized this benefit: "Its unique dual component formulation significantly reduces the peak-trough fluctuations of traditional methylphenidate drugs, maintaining stable symptom control while reducing the risk of drug abuse."
This characteristic is particularly valuable for ADHD patients who typically require years of continuous medication. "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," Professor Liu added.
Clinical Trial Design and Results
The Phase III study was conducted across eight clinical centers throughout China. While specific efficacy metrics were not disclosed in detail, the announcement confirmed that the trial achieved statistical significance across all primary and secondary endpoints compared to placebo.
The positive results provide a strong scientific foundation for the potential approval and clinical use of AK0901 in China, where current treatment options for ADHD remain limited. Existing medications often fall short in terms of efficacy, safety, or convenience of administration, leading to poor patient compliance.
Future Outlook and Market Potential
Dr. Jim Wu, CEO of ArkBio, expressed optimism about the drug's potential impact: "The completion of AK0901 Phase III study marks a critical step forward in addressing unmet needs in ADHD treatment in China. This milestone will drive our continued commitment to advancing scientific innovation and drug development of ADHD therapeutics."
AK0901 represents one of ArkBio's core assets approaching commercialization. The company, founded in 2014, focuses on developing innovative drugs for unmet medical needs in respiratory, infectious, and pediatric diseases. Its pipeline includes several innovative clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global development.
With the successful completion of the Phase III trial, ArkBio is positioned to pursue regulatory approval for AK0901 in China, potentially offering a significant advancement in the treatment landscape for Chinese children with ADHD. The company has not yet announced a timeline for regulatory submission or anticipated market availability.
