Adverum Biotechnologies Reports Additional Clinical Data from First Cohort of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD at the Atlantic Coast Retina Club Macula 20/20 Annual Meeting
--44 week median follow up for patients (n=6)--
--Zero anti-VEGF rescue injections required following intravitreal ADVM-022; First patient has reached 52-weeks post treatment--
--Vision remains stable and anatomical improvements maintained--
A copy of the presentation is available on the Adverum corporate website under Events and Presentations in the Investors section, available here.
- 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.
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:|
|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
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
About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMD
The multi-center, open-label, Phase 1, dose-escalation trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. In the first cohort, patients (n=6) received ADVM-022 at a dose of 6 x 10^11 vg/eye and in the second cohort, patients (n=6) received ADVM-022 at a dose of 2 x 10^11 vg/eye. In the third cohort (n=9), patients also are receiving a dose of 2 x 10^11 vg/eye and in the fourth cohort (n=9), patients will receive a dose of 6x10^11 vg/eye. Patients in the third and fourth cohorts will receive prophylactic steroid eye drops instead of oral steroids which were used in the first and second cohorts. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as mean number of anti-VEGF rescue injections and percentage of patients needing anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.
Eight leading retinal centers across the United States (U.S.) are participating in the OPTIC Phase 1 trial for ADVM-022. For more information on the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD, please visit https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene Therapy
ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD and diabetic retinopathy patients.
In recognition of the need for new treatment options for wet AMD, the
Adverum is currently evaluating ADVM-022 in the OPTIC Study, a Phase 1 clinical trial in patients 50 years and older with wet AMD. Additionally, Adverum plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the
About Wet Age-related Macular Degeneration (Wet AMD)
Age-related macular degeneration (AMD) is a progressive disease affecting the macula, the region of the retina at the back of the eye responsible for central vision. In patients with wet AMD, an aggressive form of AMD, abnormal blood vessels grow underneath and into the retina. These abnormal blood vessels leak fluid and blood into and beneath the retina, causing vision loss.
Wet AMD is a leading cause of vision loss in patients over 60 years of age, with a prevalence of approximately 1.2 million individuals in the U.S. and 3 million worldwide. The incidence of new cases of wet AMD in the U.S. is approximately 150,000 to 200,000 annually, and this number is expected to grow significantly as the country’s population ages.
The current standard-of-care therapy for wet AMD is anti-VEGF intravitreal injections. These are effective but typically require eye injections every 4-12 weeks in order to maintain vision. Compliance with this regimen can be difficult for patients, caregivers, and healthcare systems, leading to undertreatment and resulting in loss of vision.
Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements regarding: Adverum’s plans to report additional clinical data for ADVM-022 from the OPTIC trial and to advance ADVM-022, including Adverum’s plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the
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Source: Adverum Biotechnologies, Inc.