Adverum Biotechnologies, Inc. (NASDAQ: ADVM) today announced clinical data for the first cohort of patients (n=6) in the OPTIC phase 1 clinical trial of ADVM-022, the company's intravitreal injection gene therapy, in treatment-experienced patients with wet age-related macular degeneration (wet AMD). The data are being presented today by Charles C. Wykoff M.D., Ph.D., director of research, Retina Consultants of Houston, at the Atlantic Coast Retina Club Macula 20/20 Annual Meeting inNew York, NY.
A copy of the presentation is available on the Adverum corporate website under Events and Presentations in the Investors section, available here.
In October 2019, Adverum presented data from the first cohort in OPTIC at a median 34-week time point (28-44 week range). Today, additional data for the first cohort are being presented, including efficacy and safety data, with a median follow up of 44 weeks at a range of 40-52 weeks, and included:
Zero rescue injections for any patient. Additionally, the first patient treated in OPTIC has reached 52 weeks post ADVM-022 administration.
Vision was maintained as demonstrated by stable mean Best Corrected Visual Acuity (BCVA) compared to baseline.
Anatomical improvements were maintained as assessed by optical coherence tomography (OCT) and central subfield thickness (CST) measurements, compared to baseline.
As of December 1, 2019, ADVM-022 continues to be well-tolerated in the first cohort with no drug-related or procedure-related serious adverse events (SAEs), no drug-related systemic adverse events and no adverse events meeting the criteria for dose-limiting toxicities (DLTs). Low-grade inflammation was reported in all six patients and was generally mild to moderate and responsive to steroid eye drops. One ocular SAE, a retinal detachment, that was not related to ADVM-022 or the administration procedure was reported.
OPTIC Phase 1 Clinical Trial Data from Cohort 1 (n=6)
Results Following a Single ADVM-022 Dose:
Median follow-up (weeks) 44
Minimum/Maximum follow-up (weeks) 40-52
Follow-up cutoff date December 1, 2019
Number of patients requiring anti-VEGF rescue injections 0 patients
Mean number of anti-VEGF rescue injections 0 injections
Change in BCVA1:
Mean (ETDRS letters)3 -1.0
Minimum/Maximum (ETDRS letters) -7.0 / +7.0
Change in CRT2:
Mean (mm)3 -25.5
Minimum/Maximum (mm)3 -117 / +32
Dose-limiting toxicities (DLTs) 0
Serious adverse events (SAEs)4 1
1 Best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) (i.e., sight charts)
2 Central retinal thickness (CRT), also referred to as central subfield thickness (CST) assessed using Optical Coherence Tomography (OCT) imaging and measured by an independent Central Reading Center
3 BCVA and CST values for patient with retinal detachment (unrelated to study treatment) used last observations prior to detachment
4 This event was deemed unrelated to ADVM-022 or any study procedure
"These longer-term follow-up data demonstrate that patients in this first cohort of OPTIC are achieving sustained benefits from ADVM-022, a one-time intravitreal therapy, and have not required any anti-VEGF rescue injections through a median of 44 weeks while demonstrating impressive anatomic improvements," said Charles C. Wykoff M.D., Ph.D., director of research, Retina Consultants of Houston and associate professor of clinical ophthalmology, Blanton Eye Institute, Houston Methodist Hospital and Weill Cornell Medical College, Houston Texas. "With a median follow-up period of 44 weeks, ADVM‑022 continues to control wet AMD disease activity in all 6 patients and the low-grade intraocular inflammation appears manageable with steroid eyedrops. Based on the data to date, ADVM-022 has the potential to be a meaningful and potentially transformative treatment for patients with wet AMD."
Aaron Osborne, MBBS, chief medical officer of Adverum, added, "These new clinical data are promising as they continue to support the safety, efficacy, and durable clinical profile of ADVM-022 and this therapy's potential to change the treatment paradigm for patients with wet AMD. Anti-VEGF injections, the current standard of care, carry a significant treatment burden and real-world outcomes data suggest that vision outcomes are suboptimal due to undertreatment. In the first cohort of OPTIC, we continue to see stable vision and anatomical improvements being maintained out to a median of 44 weeks after a single ADVM-022 injection in these difficult-to-treat patients who previously required frequent anti-VEGF injections. We look forward to presenting longer-term data from the first cohort and 24-week data from the second cohort of OPTIC on February 8 at the Angiogenesis, Exudation, and Degeneration 2020 symposium."