Adverum presents 2-year OPTIC data demonstrating sustained durability and a promising safety profile from a single intravitreal injection of ADVM-022 in patients with wet AMD who previously required frequent injections of anti-VEGF | DNA RNA and cells

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Adverum presents 2-year OPTIC data demonstrating sustained durability and a promising safety profile from a single intravitreal injection of ADVM-022 in patients with wet AMD who previously required frequent injections of anti -VEGF

– Results presented in a presentation today at the Retina Society’s 54e Annual Scientific Meeting –

– Robust expression of the aflibercept protein sustained for 2 years –

– More than 80% reduction in the annualized frequency of anti-VEGF injections at 2 x 10 ^ 11 yd / eye –

– More than half of patients without injection at a median follow-up of 1.7 years to 2 x 10 ^ 11 yd / eye –

REDWOOD CITY, CA, USA I October 01, 2021 I Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in eye and rare diseases, today announced new long-term data from the OPTIC clinical trial of the injection gene Single-office intravitreal (IVT) ADVM-022 in patients requiring frequent anti-VEGF injections for their age-related neovascular or wet macular degeneration (wet AMD). Safety and efficacy data from patients followed for 2 years after injection are presented at the 54th Retina Society Conference.e Annual Scientific Meeting in Chicago, Illinois. The presentation is available on the Publications in the Pipeline section of the Advum website.

“We have now followed all wet AMD patients in the OPTIC trial for at least one year, and in some cases beyond two years, after treatment with a single office intravitreal injection of ADVM-022. We are very pleased to report that the promising safety profile and durability of treatment effect continue to be maintained in this patient population, ”said Laurent Fischer, MD, President and CEO of Adverum Biotechnologies. “We are delighted to see that ADVM-022 has the potential to extend treatment benefits from weeks to years. “

Dr Fischer said: “We continue to analyze all data from patients receiving ADVM-022 in OPTIC and our INFINITY study in diabetic macular edema. We will present the latter’s data on October 9 at the American Society of Retina Specialists Annual Scientific Meeting, followed by an OPTIC data recall presentation on October 11. We look forward to completing our data analysis from our ADVM-022 program to meet with investigators and regulators as we define our way forward in wet AMD. We are planning a Phase 2 trial to evaluate doses of ADVM-022 at 2 x 10 ^ 11 and below with improved prophylaxis, with the goal of further improving the safety profile of ADVM-022 as we aim to provide this new therapy to patients. “

Key efficiency data (July 16, 2021, deadline) – 2 x 10 ^ 11 dose vg / eye (n = 15), whose evaluation is planned in a future phase 2 trial:

  • > 80% reduction in annualized anti-VEGF injection frequency1 in patients who previously required frequent injections
  • > 50% of patients (8/152) after a median follow-up of 1.7 years, remained completely free from any additional injection of anti-VEGF
  • Robust aflibercept expression levels continue to be maintained for 2 years after a single injection of ADVM-022
  • Vision (average MAVC3 from baseline) maintained over time
  • Anatomy of the retina (CST mean4 from baseline) maintained to improved

Key safety data (deadline July 16, 2021) – 2 doses of 10 ^ 11 yd / eye (n = 15) and 6 x 10 ^ 11 yd / eye (n = 15):

  • All ocular adverse events (AEs) related to ADVM-022 were mild (81%) to moderate (19%) in all patients with wet AMD in OPTIC, and patients achieved a follow-up period between 1 and 2 years after treatment
    • No dose-limiting toxicity
    • No clinically relevant decrease in intraocular pressure
    • No vasculitis, retinitis, choroiditis, vascular occlusion or endophthalmitis
  • At 2 x 10 ^ 11 vg / eye, eye inflammation was minimal and sensitive to steroidal eye drops

1 Annualized rate (Before) = (number of IVTs in the 12 months preceding the ADVM-022) / (days of the first IVT in the last 12 months in the ADVM-022 / 365.25)
Annualized rate (After) = (number of aflibercept IVTs since ADVM-022) / (days from ADVM-022 to last study follow-up / 365.25)
2 All patients in Cohort 2 (n = 6) and Cohort 3 (n = 9) treated with the dose of 2 x 10 ^ 11 vg / eye
3 Better corrected visual acuity (BCVA)
4 Central subfield thickness (CST)

“As OPTIC’s largest recruiter, I have observed the potential of ADVM-022, a novel intravitreal gene therapy, to dramatically reduce the treatment burden for my patients with age-related neovascular macular degeneration. . In the latest data from the OPTIC trial, we found a manageable safety profile, robust expression of aflibercept, and sustained anatomical improvements 21 months after a single 2 x 10 ^ 11 dose of ADVM-022 ” said Arshad M. Khanani, MD, MA, managing partner and director of clinical research, Sierra Eye Associates; Associate Clinical Professor, University of Nevada, Reno School of Medicine and member of the Scientific Advisory Board of Advum. “I look forward to continuing to contribute to future development plans for ADVM-022 in patients with neovascular AMD. “

Milestones planned for ADVM-022:

  • Plan to present the INFINITY data on Diabetic Macular Edema (DME) at the 39th Annual Scientific Meeting of the American Society of Retina Specialists (ASRS) on Saturday, October 9, 2021 at 10:28 a.m. CT (11:28 a.m. ET). As previously stated, the company no longer plans to develop ADVM-022 in DME.
  • Plan to present an OPTIC data reminder to ASRS on Monday, October 11, 2021 at 8:50 am CT (9:50 am ET)
  • Plan to complete data analysis by YE21 to develop a protocol and solicit feedback from researchers and regulatory authorities on a Phase 2 clinical trial in wet AMD to assess low doses (2 x 10 ^ 11 vg / eye and less) of ADVM-022 and improved prophylaxis
  • Plan to present additional long-term data from the OPTIC and OPTIC Extension studies in wet AMD

About the OPTIC trial of ADVM-022 in wet AMD
This multi-center, open-label, dose-determining trial is designed to assess the safety and tolerability of a single intravitreal administration (IVT) of ADVM-022 in patients with wet AMD. Patients on OPTIC are difficult to treat and had previously received frequent anti-vascular endothelial growth factor (VEGF) therapy. Patients received the 6 x 10 ^ 11 vg / eye dose of ADVM-022 in cohort 1 (n = 6) and cohort 4 (n = 9) and patients received the 2 x 10 ^ dose 11 vg / eye in cohort 2 (n = 6) and cohort 3 (n = 9). Patients in cohorts 3 and 4 received six weeks of prophylactic steroid eye drops instead of 13 days of oral prophylactic steroids which were used in cohorts 1 and 2. The primary endpoint of the trial is safety and tolerability of ADVM-022 after a single IVT. administration. Secondary endpoints include changes in best corrected visual acuity (BCVA), central retinal thickness (CRT) measurement, as well as the need for additional anti-VEGF injections. Each recruited patient will be followed for a total of two years.

For more information, please visit https://clinicaltrials.gov/ct2/show/NCT03748784 or the OPTIC test or https://clinicaltrials.gov/ct2/show/NCT04645212 for the OPTIC Extension trial.

About ADVM-022 gene therapy
ADVM-022 uses Advum’s proprietary vector capsid, AAV.7m8, carrying a codon-optimized aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a single intravitreal injection (IVT), designed to provide long-term efficacy and reduce the burden of frequent anti-VEGF injections, and improve real-world vision outcomes for patients. patients with age-related wet macular degeneration. (Wet AMD).

Recognizing the need for new treatment options for wet AMD, the United States Food and Drug Administration has granted ADVM-022 Fast Track designation for the treatment of wet AMD.

About 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 develop under and in the retina. These abnormal blood vessels leak fluid and blood into and under the retina, causing vision loss.

Wet AMD is a leading cause of vision loss in patients over the age of 60, with a prevalence of approximately 1.2 million people in the United States and 3 million worldwide.1. The incidence of new cases of wet AMD in the United States is approximately 150,000 to 200,000 per year, and that number is expected to increase dramatically as the country’s population ages.2.3.

The current gold standard treatments for wet AMD are anti-VEGF proteins. These therapies can be cumbersome, as patients typically require a chronic intravitreal injection (IVT) of anti-VEGF protein every 4 to 12 weeks. Adherence to this regimen can be difficult for patients and their caregivers, resulting in deficiencies in adherence and vision loss due to underdosing. These standard brand anti-VEGF therapies used for the treatment of wet AMD, DR, retinal venous occlusion and other eye diseases are estimated to have generated more than $ 11 billion in sales worldwide as of today. 20204.

1 Ophthalmol Arc. 2004; 122 (4): 564-572. doi: 10.1001 / archopht.122.4.564.
2 Brown GC, Brown MM, Sharma S, et al. The burden of age-related macular degeneration: an analysis of value-based medicine. Transactions of the American Ophthalmological Society. 2005.
3 Californian retina consultants. Advances in Wet AMD. Available at: https://www.californiaretina.com/advances-in-wet-amd/
4 Regeneron, Roche and Novartis 2020 year-end financial statements.

About Advum Biotechnologies
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in severe and rare eye diseases. Adverum is advancing the clinical development of its new gene therapy candidate, ADVM-022, as a unique intravitreal injection for the treatment of patients with age-related wet macular degeneration. For more information, please visit www.adverum.com.

THE SOURCE: Adverum Biotechnologies


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