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Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today provided a program update on ADVM-022, a novel, single-administration gene therapy being evaluated in the OPTIC Phase 1 clinical trial in patients with wet age-related macular degeneration (wet AMD).
The Company has completed enrollment and dosing of patients (n=6) in the first cohort in the OPTIC trial. The independent data monitoring committee (DMC) determined that enrollment and dosing of patients in the second cohort could proceed. This was based on a review of the preliminary safety data from the first cohort of patients, which has shown no serious adverse events (SAEs) or dose-limiting toxicities (DLTs) following a single intravitreal injection of ADVM-022 at the initial trial dose of 6 x 10^11 vg/eye.
Adverum has received a notification from the U.S. Food and Drug Administration (FDA) in early April requesting additional CMC information and requirements on the ADVM-022 manufacturing process. The FDA has placed the Company’s IND on clinical hold until the agency has reviewed Adverum’s response, which was submitted last week. The Company is working closely with the agency during its review. The Company expects to resume dosing of patients once the FDA review is completed and the clinical hold is lifted.
“We are working with the FDA to resolve this matter as quickly as possible,” said Leone Patterson, chief executive officer of Adverum Biotechnologies. “We are deeply committed to the development of our novel gene therapy ADVM-022 for patients with wet AMD. In the OPTIC trial, the DMC reviewed the safety data and unanimously agreed that we could proceed to dosing the second cohort. No patient has experienced an SAE, with a follow up period of up to five months. We look forward to sharing 24-week primary and secondary outcomes from the first cohort of patients at a scientific meeting in the second half of this year.”