NEW YORK – Avirmax Biopharma on Friday said it anticipates submitting an investigational new drug (IND) application for ABI-201 in the fourth quarter of this year, seeking permission from US regulators to begin testing the gene therapy in patients with dry age-related macular degeneration (AMD).
If the US Food and Drug Administration clears its IND application, Avirmax said it will launch a Phase I/IIa trial in patients with dry AMD soon thereafter.
"ABI-201 has the potential to revolutionize the treatment for dry AMD and many other retinal disorders," Avirmax CEO Shengjiang Shawn Liu said in a statement. In ongoing IND-enabling studies, the Hayward, California-based company noted it is exploring ABI-201's safety, tolerability, vector biodistribution, transgene expression, and pharmacokinetics in monkeys.
Dry AMD is an eye disorder characterized by blurred or reduced vision due to the breakdown of the macula, a part of the retina. With ABI-201, Avirmax aims to preserve vision, prevent retinal pathogenesis, and avoid patients' progression from dry AMD to the more severe wet AMD.
The gene therapy is designed to use an adeno-associated virus vector, AAV.N54, to deliver three transgenes to the macula. The company has not disclosed the three transgenes but is hoping ABI-201 has anti-inflammatory properties as well as the ability to correct dysregulation of complement activation, protect retinal pigment epithelia and photoreceptors, and block retinal neovascularization.
Avirmax did not immediately respond to a question about which transgenes are involved in ABI-201.
AAV.N54 is the same capsid Avirmax is using in ABI-110, a gene therapy it is evaluating in a Phase I/IIa trial for wet AMD.