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Adverum Biotechnologies Announces OPTIC Phase 1 Trial Progressing With Plans to Complete Patient Dosing in Cohort Three, Begin Enrollment in Cohort Four in Q1 2020

Adverum Biotechnologies, Inc. (Nasdaq: ADVM) today reviewed recent business and development progress and provided an outlook for 2020.

Benzinga · -

Adverum Biotechnologies, Inc. (Nasdaq: ADVM) today reviewed recent business and development progress and provided an outlook for 2020.

"In 2019, we made significant progress advancing our lead gene therapy candidate ADVM-022 in the ongoing OPTIC phase 1 dose-ranging clinical trial in patients with wet age-related macular degeneration (AMD)," said Leone Patterson, president and chief executive officer of Adverum Biotechnologies. "Patients with wet AMD typically require frequent ocular anti-VEGF injections to maintain their vision. Yesterday, additional data were presented from the first cohort of patients in OPTIC, which demonstrated zero anti-VEGF rescue injections required following a single intravitreal injection of ADVM-022 and with median follow up of 44 weeks, ADVM-022 treatment was safe and well-tolerated. This year, we look forward to presenting additional data from all four cohorts of patients in OPTIC. Additionally, we plan to submit an IND for ADVM-022 in a second indication, diabetic retinopathy, and begin enrolling patients in a new clinical trial. We believe ADVM-022 has the potential to be a paradigm-changing treatment for patients with wet AMD and for patients with diabetic retinopathy."

Recent Data Presented for First Cohort of OPTIC Phase 1 Clinical Trial (n=6)
On January 11, 2020, Adverum presented median 44-week (range of 40-52 weeks) data at the Atlantic Coast Retina Club and Macula 20/20. These data demonstrated that patients in the first cohort of OPTIC achieved vision maintenance and improvements in retinal anatomy with zero anti-VEGF rescue injections required for any patient. Additionally, the first patient treated in OPTIC has reached 52 weeks post ADVM-022 administration. ADVM-022 has been safe and well-tolerated, with no dose-limiting toxicities through the latest time point at December 1, 2019 and inflammation has been manageable with topical eye drops.

OPTIC Phase 1 Trial Execution
After completing patient enrollment in the first and second cohorts, Adverum dosed the first patient in the third cohort (n=9, dose of 2x10^11 vg/eye) in October 2019. In the third and fourth cohorts, Adverum plans to use prophylactic steroid eye drops instead of prophylactic oral steroids to manage inflammation.

2020 Outlook

First quarter of 2020:

Move to new corporate headquarters in Redwood City, CA, allowing for the expansion of the company's in-house process development capabilities to the 1,000-liter production scale
Complete patient dosing in the third cohort, begin enrollment in the fourth cohort, and determine if additional cohorts are needed in the OPTIC trial
First half of 2020:

Present longer-term data from the first cohort of patients in OPTIC
Present 24-week data from the second cohort of patients in OPTIC
Submit an investigational new drug application for ADVM-022 in diabetic retinopathy, a key VEGF-driven cause of vision loss among working-age adults
Second half of 2020:

Present longer-term data from the first cohort and second cohorts of patients in OPTIC trial
Present clinical data from the third and fourth cohorts of patients in the OPTIC trial
Begin enrolling patients in a planned phase 1/2 clinical trial for ADVM-022 in diabetic retinopathy to expand Adverum's clinical development pipeline
Upcoming Events
Adverum plans to participate in the following upcoming conferences:

J.P. Morgan's 38th Annual Healthcare Conference in San Francisco on January 15, 2020 at 2:30 pm PST
Angiogenesis, Exudation, and Degeneration 2020 in Miami on February 8, 2020 at 2:44 pm EST. Dr. David Boyer will present the 24-week data from the second cohort for the first time as well as an update from the first cohort of patients in the OPTIC trial. A KOL event and simultaneous webcast to discuss the data presented with management and an expert panel of retina specialists will take place on February 9, 2020 at 10:00 am EST
SVB Leerink's 9th Annual Global Healthcare Conference in New York on February 25, 2020 at 9 am EST
Cowen's 40th Annual Health Care Conference in Boston from March 2-4, 2020