Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases, announced long-term preclinical efficacy data on ADVM-022 in a non-human primate model of wet age-related macular degeneration (wAMD). In this preclinical study, the efficacy of ADVM-022 at 13 months post-administration was consistent with earlier reported data, demonstrating that a single intravitreal injection of ADVM-022 was found to be safe and statistically significant (p<0.0001) in preventing the development of Grade IV lesions compared to the vehicle control group. ADVM-022 induced long-term efficacy that was comparable to aflibercept, an anti-Vascular Endothelial Growth Factor (VEGF) standard-of-care therapy. ADVM-022 was well tolerated, with no serious adverse events.

“We continue to be encouraged by the efficacy and sustained protein levels we are seeing a year post a single intravitreal injection of ADVM-022,” said Mehdi Gasmi, Ph.D, chief science and technology officer of Adverum Biotechnologies. “These durable efficacy data support our plans to advance ADVM-022 into the clinic and we remain on track to submit an Investigational New Drug (IND) Application for ADVM-022 in the second half of this year. We are excited to be working on a gene therapy candidate that has demonstrated the potential to alleviate the treatment burden of frequent injections for patients living with wet AMD.”

ADVM-022 utilizes a proprietary vector capsid (AAV.7m8) carrying an aflibercept coding sequence under the control of a proprietary expression cassette and is administered as a single intravitreal injection. VEGF overexpression can lead to wAMD progression and vision loss.  Treatment with ADVM-022 is designed to minimize the burden of frequent anti-VEGF injections, the current standard-of-care treatment for wAMD.