Gene Therapy Trial Doses First Patient With Wet AMD in China

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The phase 1 clinical trial is the second of Frontera’s trials to begin dosing in 2023.

Frontera Therapeutics has dosed the first patient with wet age-related macular degeneration (AMD) in a phase 1 clinical trial (NCT05611424) evaluating its adeno-associated virus (AAV) vector gene therapy FT-003 at the Ophthalmology Hospital of Tianjin Medical University in Tianjin, China.1

“I am excited by the momentum the Frontera team has achieved so far this year with the dosing of the first patients in two gene therapy Phase 1 clinical trials during the month of January for FT-001 and FT-003,” Yong Dai, PhD, founder and chief executive officer, Frontera, said in a statement.1 “FT-003 is designed as a one-time treatment for patients with wet-AMD, a leading cause of blindness globally in people over 60 years old. We remain committed to validating our novel AAV technology platform and to making further breakthroughs in bringing gene therapies to patients with unmet medical needs, and expect to have initial clinical results for both FT-001 and FT-003 later this year.”

The trial will begin with a phase 1, 3x3 dose-escalation scheme in up to 18 enrolled participants of at least 45 years of age. The primary outcome is safety and tolerability of intraocular injection of FT-003 as measured by the severity and incidence of adverse events (AEs) and the secondary outcome is change in best-corrected visual acuity (BCVA) of the studied eye from baseline. FT-003 is designed to provide durable expression of therapeutic levels of intraocular protein to maintain vision and reduce the current treatment burden with frequent anti-vascular endothelial growth factors therapy which often results in undertreatment and vision loss in patients with wet AMD in real-world experience.

2023 has seen Frontera’s foray into clinical trials, with a phase 1 clinical trial of another AAV gene therapy, FT-001, also having dosed its first patient in China with Leber congenital amaurosis-2 (LCA-2) in January.2 The therapy also received investigational new drug (IND) clearance for a US trial in late 2022.3

WATCH NOW: Arshad Khanani, MD, on Restoring Aflibercept Expression in Wet AMD With Gene Therapy

“This is an exciting achievement for Frontera Therapeutics as we begin our first clinical trial and take a significant step forward as a company,” Dai said in a statement at that time.2

Also in Frontera’s pipeline are additional gene therapies in IND-enabling studies including FT-002 for “rare” ophthalmic diseases, FT-004 for hemophilia B, and FT-005 for another hematologicalindication. The company is also researching undisclosed gene therapy programs in ophthalmologic, hematologic, central nervous system, cardiovascular, and metabolic diseases. 

“Since Frontera’s founding, we have rapidly progressed a significant number of programs into IND enabling studies,” Dai said in another statement.3 “This year, we have advanced our lead program, FT-001, and have received both US FDA and China CDE IND clearances. Building on our product development momentum, we intend to advance a number of programs into clinical studies in the next 6 to 9 months. We will also leverage our APEX Technology & Manufacturing platform to create new innovative programs targeting large global markets such as cardiovascular, metabolic, and central nervous system diseases.”

REFERENCES
1. Frontera Therapeutics doses first patient in a clinical trial of FT-003 gene therapy for the treatment of wet AMD. News release. Frontera Therapeutics. February 2, 2023. https://finance.yahoo.com/news/frontera-therapeutics-doses-first-patient-140000649.html
2. Frontera Therapeutics doses first patient in phase 1 clinical trial for gene therapy FT-001 for the treatment of leber congenital amaurosis-2. News release. Frontera Therapeutics. January 6, 2023. https://www.fronteratherapeutics.com/frontera-therapeutics-doses-first-patient-in-phase-1-clinical-trial-for-gene-therapy-ft-001-for-the-treatment-of-leber-congenital-amaurosis-2/
3. Frontera Therapeutics receives additional IND clearance for its lead program FT-001. News release. Frontera Therapeutics. September 21, 2022. https://www.fronteratherapeutics.com/frontera-therapeutics-receives-additional-ind-clearance-for-its-lead-program-ft-001/
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