Adverum Biotechnologies has made significant progress in its lead clinical program for intravitreal gene therapy for retinal diseases, Leone Patterson, CEO and president, said at the J.P. Morgan Healthcare Conference.
Real-world anti-VEGF patient outcomes highlight the need for a long-term solution, such as intravitreal gene therapy with ADVM-022, to benefit patients and clinicians.
ADVM-022 is designed to target serious ocular diseases, such as age-related macular degeneration and diabetic retinopathy, with a long-lasting single injection administered in an outpatient setting.
“What we got here is a proprietary vector, AAV.7m8, which really was designed for one-time treatment and delivers long-term VEGF inhibition, enabling efficient intravitreal delivery and also increasing transduction of retinal cells along with increasing protein expression,” Patterson said.
In the OPTIC dose-ranging trials evaluating the safety and efficacy of ADVM-022 intravitreal injection, patients were undergoing active treatment for wet AMD, had a minimum of two anti-VEGF injections within the last 4 months, had a meaningful response to aflibercept, and had best corrected visual acuity between 20/32 and 20/320. Results showed patients maintained their vision, and ADVM-022 was safe and well tolerated with no serious adverse events.
“For the OPTIC trial, we were starting to enroll patients for cohort 3 and we plan to start enrollment for cohort 4 in Q1. We’ll have data across all four cohorts. The first half of the year, we’ll have data on cohorts 1 and 2, and in the second half of the year, we’ll have data on all four cohorts,” Patterson said. “And in diabetic retinopathy ... we plan to submit an IND in the first half of this year and begin enrollment in the second half. So, we’ll have two clinical programs addressing two large markets. We believe we’ll really change the treatment paradigm for patients living with these diseases with a one-time intravitreal injection.” – by Erin T. Welsh
Patterson L. Adverum Biotechnologies corporate update. Presented at the J.P. Morgan Healthcare Conference; Jan 13-16, 2020; San Francisco.
Disclosure: Patterson reports she is CEO and president of Adverum Biotechnologies.