Recently, Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as "Vitalgen" or "the Company") has completed patient enrollment for its self-developed VGN-R09b in the Phase I/III registrational clinical trial (VGN-R09b-102) of severe Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD).
VGN-R09b is the first and only gene therapy product in China indicated for the treatment of AADCD, addressing a critical gap in the domestic therapeutic landscape. VGN-R09b-102 is a multicenter, single-arm, open-label Phase I/III clinical trial (CTR20241839) jointly conducted by two leading pediatric institutions: Shanghai Children's Medical Center affiliated to Shanghai Jiao Tong University School of Medicine and Children's Hospital of Fudan University. Current safety data indicate that VGN-R09b is well-tolerated, with no serious adverse reactions related to the investigational product or administration process observed. Efficacy data demonstrate a rapid onset of action for VGN-R09b, with patients beginning to show improvements in motor function attributable to the pharmacological effect as early as two weeks post-administration. Among early-enrolled patients followed for more than six months, 100% achieved head control (the first motor milestone), and some patients have achieved the ability to sit independently (the second motor milestone). Current data suggest that VGN-R09b provides substantial clinical benefit, with the potential for all treated patients to achieve improved motor function within six months following administration. The observed efficacy profile compares favorably with products approved for the same indication by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).
Dr. Zhao Xiaoping, CEO of Vitalgen, stated:
"As the company's first pediatric rare disease drug to enter the pivotal clinical stage, the successful completion of enrollment in the registrational trial for VGN-R09b not only marks a breakthrough in addressing the significant unmet medical need in AADCD but also fully validates the company's integrated independent innovation capabilities spanning from target discovery to clinical development. We remain steadfast in our commitment to addressing the urgent medical needs of children with rare diseases and their families. We will continue to advance subsequent development with full dedication, looking forward to bringing VGN-R09b to children with AADCD worldwide as soon as possible."
Dr. Lyu Hua, Senior Vice President of Registration and Clinical Development at Vitalgen, stated:
"Conducting clinical studies in pediatric rare diseases is fraught with challenges. The successful completion of patient enrollment in this registrational clinical study of VGN-R09b in children with severe AADCD is a significant achievement. We extend our gratitude to each participating family for their profound trust and full cooperation, as well as to the expert teams from Shanghai Children's Medical Center and Children's Hospital of Fudan University for their professional dedication and hard work." Our focus moving forward is to ensure high-quality data collection and analysis, thereby establishing a robust evidence-based medicine foundation to validate the clinical value of VGN-R09b in children with severe AADCD and providing critical support for subsequent regulatory submissions."
About AADCD
AADCD is caused by pathogenic mutations in the DDC gene encoding the AADC enzyme. AADC is the decarboxylase responsible for the conversion of L-DOPA (levodopa) and 5-HTP (5-hydroxytryptophan) into the neurotransmitters dopamine and serotonin, respectively. Deficiency in AADC disrupts the synthesis of key neurotransmitters that regulate the sympathetic nervous system, mood, cognition, and motor coordination. This leads to clinical manifestations including motor dysfunction, intellectual disability, reduced alertness, and sleep disturbances. Affected infants present with a floppy baby phenotype and fail to achieve critical motor milestones such as head control, sitting independently, standing, and walking. There is a risk of premature death within the first decade of life. Currently, no formal epidemiological studies on this disease have been conducted in mainland China. A newborn screening program for AADCD implemented in Taiwan reported an approximate birth incidence of 1 in 32,000 for AADCD.
About VGN-R09b
VGN-R09b is a gene therapy product independently developed by Vitalgen. It utilizes a recombinant adeno-associated virus (rAAV) as the gene therapy vector to enhance dopamine synthesis in the brain while promoting neuroprotection, thereby exerting its potential therapeutic effects. Administered via localized striatal injection, the product enables direct targeting of the affected area, reducing the required systemic dose and consequently minimizing associated adverse reactions such as immune responses. Theoretically, a single administration of gene therapy has the potential to provide long-term therapeutic efficacy. VGN-R09b previously received Clinical Trial Approval from the National Medical Products Administration (NMPA) on April 19, 2024, for the treatment of Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD) and idiopathic Parkinson's Disease (PD).