SHANGHAI, CHINA, March 19, 2025--Shanghai Vitalgen Biopharma Co., Ltd. (hereinafter referred to as "Vitalgen") announces the successful completion of patient enrollment in its Phase III pivotal clinical trial (VGR-R01-301) of VGR-R01, an investigational gene therapy for the treatment of Bietti crystalline dystrophy (BCD).
The completion of patient enrollment in the VGR-R01-301 trial makes a significant milestone in the clinical development of VGR-R01 for BCD. This is a multi-center, randomized, controlled clinical study (CTR20244386) designed to evaluate the efficacy and safety of VGR-R01 in individuals diagnosed with BCD. Participating in the study are the following five esteemed clinical institutions, Beijing Tongren Hospital affiliated to Capital Medical University, Shanghai First People's Hospital, The First Affiliated Hospital of Army Medical University, Wenzhou Medical University Eye Hospital, and Zhongshan Ophthalmic Center of Sun Yat-sen University. A total of 45 patients were successfully enrolled.
"VGR-R01 represents a global first in mouse model development, indication selection, target identification, and technological innovation.” said Dr. Xiaoping Zhao, CEO of Vitalgen. “We are pleased to announce the rapid completion of patient enrollment in this Phase III clinical trial, a testament to our commitment in putting patients first and fostering collaborative efforts with our partners and clinical experts in the community. Given that BCD is a severe ophthalmic condition prevalent among Asian populations with an unmet medical need, we are working tirelessly to ensure smooth progression of the clinical development of VGR-R01 in order to speedily bring an effective treatment to benefit the many BCD patients that have been underserved."
"VGR-R01 has the potential to become the 1st-in-class treatment for BCD, a degenerative eye disease without any adequate treatment. We are advancing this project with full speed and looking forward to obtaining relevant data as soon as possible." Said Dr. Hua Lv, Senior Vice President of Registration and Clinical Development of Vitalgen,
About BCD
Bietti crystalline dystrophy (BCD) is a recessive inherited retinal disorder characterized by the presence of tiny crystalline deposits in the retina (and sometimes the cornea), along with progressive atrophy of the retina and choroid. This condition is caused by mutations in the CYP4V2 gene, which is a critical lipid metabolism enzyme in the retinal pigment epithelium (RPE). Defective CYP4V2 led to abnormal retinal lipid deposits, resulting in progressive vision loss, night blindness, and constricted visual fields over time. Currently, treatment is largely supportive supplements’, such as vasodilators, vitamins, and traditional Chinese medicines that have no well-validated clinical benefits. Therefore, there is an urgent need to develop an effective therapy for treating and alleviating the sufferings of BCD patients.
About VGR-R01
VGR-R01 is a recombinant adeno-associated virus-based gene therapy that has been independently developed by Vitalgen. It is based on the strategy of replacing the defective CYP4v2 gene by sub-retinally delivering a wild-type CYP4V2 transgene to express functional CYP4v2 protein in the RPE. Restoration of the normal CYP4v2 enzymatic function would normalize the fatty acid metabolism in the RPE to alleviate the structural and functional damages of the retina. Therefore, VGR-R01 has the potential to become an effective therapy to slow or halt the progression of retinal degeneration of the affected BCD patients.
VGR-R01 received approval from the National Medical Products Administration (NMPA) to initiate clinical trials for the treatment of BCD, the first therapeutic agent for this indication to enter the registration clinical trial phase globally. VGR-R01 was incorporated into the Phase C of the "Care Plan" by the Center for Drug Evaluation (CDE) of the NMPA as announced in December 2024. In addition, it was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration in October 2024. Furthermore, it was approved for the Breakthrough Therapy Designation Program from CDE in August 2024.