Adverum Biotechnologies Reports Positive Interim Data from Cohorts 1-3 of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
-- Robust efficacy and evidence of a dose response from single intravitreal injection (IVT) of ADVM-022 --
-- Long-term durability beyond 1 year from single IVT injection of ADVM-022 with zero rescue injections in Cohort 1 --
-- Encouraging early data from Cohort 3 --
-- Company to host a conference call and webcast with key opinion leaders today at
For the first time, interim data are being presented from patients in Cohort 31, 2 and updated data are being presented from Cohorts 1 and 2 following treatment with a single IVT injection of ADVM-022.
- ADVM-022 continues to show robust efficacy
- Long-term durability beyond 1 year from a single IVT injection with zero rescue injections in Cohort 1
- Further evidence of a dose response:
-- 6x1011 vg/eye: 6/6 patients rescue injection free
-- 2x1011 vg/eye: 8/113 patients rescue injection free
- ADVM-022 continues to be well tolerated with a favorable safety profile in all 3 cohorts:
-- ADVM-022 related ocular AEs mild (69%) to moderate (31%)
-- No ADVM-022 related SAEs or non-ocular adverse events
-- No evidence of vasculitis, retinitis, or choroiditis
-- Ocular inflammation, when observed, has been responsive to steroid eye drops
- Early evidence from Cohort 3 suggests that a 6-week prophylactic regimen of steroid eye drops results in fewer adverse events and less inflammation, compared to a 13-day prophylactic regimen of oral steroids as used in Cohorts 1 and 2. In all 9 patients in Cohort 3:
-- No cellular inflammation graded at a score above 1+ in any patient
-- No patients have required more than steroid eye drops
-- Only 8 ADVM-022-related AEs in 4 patients have been observed, all mild or moderate
- Early anatomic and vision improvements observed in Cohort 3, with first 5 patients with 20 weeks follow-up showing:
-- Mean CRT4 reduction (-137.8 mm)
-- Mean BCVA5 gain (+6.8 letters)
OPTIC Phase 1 Clinical Trial Data:
|Results Following a Single ADVM-022 Dose:||Cohort 1||Cohort 2||Cohort 31|
|Dose ADVM-022||Higher Dose
6 x 10^11 vg/eye
2 x 10^11 vg/eye
2 x 10^11 vg/eye
|Follow-up (median)||60 weeks||36 weeks||20 weeks|
|Prophylactic steroid regimen||13-day oral||13-day oral||6-week eye drops2|
|Number of patients requiring anti-VEGF rescue injections||0/6 patients||2/6 patients||1/5 for first 5 patients with 20 weeks follow-up|
|Total anti-VEGF rescue injections||0 injections||8 injections||2 injections|
|Follow-up BCVA5 and CRT4:||52-64 weeks
|First 5 patients with 20 weeks follow-up|
100% (6/6) Rescue Free
||Rescue Free Patients
|All Patients||Rescue Free Patients
|BCVA mean change from baseline (letters)||-2.7||-2.8||+2.3||+6.8||+8.8|
|CRT mean change from baseline (mm)||-26.2||-40.8||-30.0||-137.8||-149.8|
1 The first 5 patients had 20 weeks of follow-up as of
2 In Cohort 3, patients received 6-week prophylactic topical steroid regimen in place of the 13-day prophylactic oral steroid regimen used in Cohorts 1 and 2.
3 4/6 patients from Cohort 2 and 4/5 patients from Cohort 3 with 20 weeks follow up
4 Central retinal thickness (CRT)
5 Best corrected visual acuity (BCVA)
- Adverum reports
$297 millionin cash, cash equivalents and short-term investments as of March 31, 2020, compared to $166 millionas of December 31, 2019. In February 2020, Adverum raised approximately $140.8 millionin net proceeds from an underwritten public offering. The Company expects this quarter-end cash position to fund operations into 2022.
- Due to COVID-19, and the shelter-in-place mandated in the
State of California, Adverum implemented a mandatory work-from-home policy for all non-essential activities.
-- To minimize the chance of community infection, the company has limited on-site activities to only the most time-critical or necessary operational activities.
-- Pursuant to the SEC’s recent order under section 36 of the Securities Exchange Act of 1934, Adverum now expects to file its Form 10-Q for the first quarter of 2020 with the
SECon May 28, 2020.
- Submit an investigational new drug application for ADVM-022 in diabetic retinopathy, a key VEGF-driven cause of vision loss among working-age adults
- Present data from all four cohorts of 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
Conference Call and Webcast Today:
In addition, Adverum will host a conference call and webcast with expert retinal specialists to discuss the new OPTIC data and the potential opportunity for ADVM-022. The discussion will be held on
About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMD
The multi-center, open-label, Phase 1, dose-ranging 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 Cohort 1, patients (n=6) received ADVM-022 at a higher dose of 6 x 10^11 vg/eye and in Cohort 2, patients (n=6) received ADVM-022 at a lower dose of 2 x 10^11 vg/eye. In Cohort 3, patients (n=9) also received a dose of 2 x 10^11 vg/eye and in Cohort 4, patients (n=9) are receiving a dose of 6 x 10^11 vg/eye. Patients in Cohorts 3 and 4 receive prophylactic steroid eye drops instead of oral steroids which were used in Cohorts 1 and 2. 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 the need for anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.
Ten leading retinal centers across the United States (
About ADVM-022 Gene Therapy
ADVM-022 utilizes a proprietary 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 initiate a planned Phase 1/2 clinical trial of ADVM-022 for the treatment of diabetic retinopathy in the second half of 2020.
About Wet Age-related Macular Degeneration (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
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: the potential for ADVM-022 in treating patients with wet AMD expressed in Dr. Khanani’s and Dr. Osborne’s quotes; Adverum’s expectations as to the timing of reporting additional data in the second half of 2020, and as to its plans to advance ADVM-022 in diabetic retinopathy by filing an investigational drug application in the first half of 2020 and begin enrolling patients in a planned Phase 1/2 clinical trial in the second half of 2020; all of which are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC trial and vector production; the effects of the COVID-19 pandemic on the company’s operations; and ability to fund operations through completion of the OPTIC trial and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverum’s Form 10-K filed with the
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Source: Adverum Biotechnologies, Inc.