Boston, MA, November 11, 2024 –Frontera is pleased to announce that the U.S. Food and Drug Administration (FDA) has cleared its Phase 2 Investigational New Drug (IND) application for FT-003 Injection, an innovative recombinant adeno-associated virus (rAAV) gene therapy product, for the treatment of neovascular age-related macular degeneration (nAMD). This clearance reflects the FDA’s recognition of Frontera’s early clinical and Chemistry, Manufacturing, and Controls (CMC) data from clinical trials conducted in China, marking a pivotal step in Frontera’s efforts to bring advanced gene therapies for chronic diseases to the US market.
“Currently, nAMD treatment requires frequent intravitreal injections of anti-VEGF antibodies every 2-4 months, imposing a lifelong burden on patients. The reported annual direct medical cost exceeds $10K/patient and indirect medical cost approaches $4K/ patients. These ongoing treatments are not only costly but can also result in compliance challenges that increase the risk of long-term vision loss,” said Dr. Li Xinyan, co-founder and CEO of Frontera. “By introducing therapeutic genes into retinal cells, FT-003 aims to transform these cells into continuous producers of functional proteins, providing sustained therapeutic effects with just a single dose. This stable therapeutic level could help prevent the disease progression associated with fluctuating drug concentrations.”
Dr. Li added, “With the recent FDA clearance of FT-003 for Phase 2 trials in the U.S., following the successful IND clearance for FT-002 in treating X-linked retinitis pigmentosa (XLRP), Frontera is achieving critical regulatory milestones in the U.S. This progress underscores the FDA’s confidence in Frontera’s innovative technologies, strong CMC platform, and promising clinical data. We are committed to advancing new treatments that can combat blindness and significantly improve patients’ quality of life.”
Neovascular Age-Related Macular Degeneration (nAMD)
Age-Related Macular Degeneration (AMD) is a leading cause of irreversible vision loss worldwide, particularly affecting older adults. Neovascular AMD (nAMD) affects 10-20% of AMD patients and is marked by rapid loss of central vision, which can occur over weeks to months. nAMD is characterized by abnormal blood vessel growth, leakage, hemorrhage, and edema in the eye, ultimately leading to scarring and vision impairment. The current nAMD patient number is around 500K in the US and is expected to increase to 600K in 2030 with the aging population. The annual direct medical cost of total nAMD population was estimated to exceeded $5B and indirect medical cost around $1.9B in the US in 2022.
About FT-003 Injection
FT-003 Injection is an rAAV-based gene therapy developed to target retinal cells, enabling them to produce a humanized recombinant fusion protein homologous to aflibercept. This protein inhibits vascular endothelial cell proliferation and reduces vascular permeability, delivering therapeutic benefits. Preclinical data suggest FT-003 could provide long-lasting effects with a single administration. In addition to the U.S. clearance, FT-003 has been approved for Phase 2 trials for both nAMD and diabetic macular edema (DME) in China, having demonstrated safety and efficacy in Phase 1 studies.
About Frontera
Frontera is a global gene therapy company dedicated to creating accessible, high-quality rAAV-based treatments for ophthalmology and cardiovascular diseases. The company has advanced capabilities in AAV capsid engineering, gene expression, GMP manufacturing, and quality control. Frontera’s production system, the Sf9 insect cell-baculovirus expression vector system (Sf9 BEVS), supports large-scale, high-quality manufacturing with enhanced safety and efficacy.
Frontera’s diverse pipeline includes “first-in-class” and “best-in-class” gene therapies targeting both genetic and chronic diseases. FT-003, designed for convenient intravitreal administration, meets the needs of millions with nAMD and DME and holds significant market potential. Beyond ophthalmology, Frontera is advancing treatments for hereditary cardiomyopathy, with upcoming IND filings in both China and the U.S.