Adverum Presents Clinical Data on ADVM-022 at the 39th Annual Scientific Meeting of the American Society of Retina Specialists | DNA RNA and cells

Adverum presents clinical data on ADVM-022 at the 39th Annual Scientific Meeting of the American Society of Retina Specialists

– The company continues to focus on the progression of low-dose ADVM-022 in patients with wet AMD –

REDWOOD CITY, CA, USA I October 09, 2021 I Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in eye and rare diseases, today presents data from the INFINITY clinical trial of ADVM-022 in patients with d Diabetic Macular Edema (DME) from the American Society of Retinal Specialists (ASRS) 39e Annual Scientific Meeting and will again present a presentation of long-term data from the OPTIC trial of ADVM-022 for wet AMD on Monday, October 11, 2021 at 8:50 a.m. CT (9:50 a.m. ET). Adverum anticipates the clinical development of ADVM-022 at low doses (2 x 10 ^ 11 vg / eye and below) in patients with age-related wet macular degeneration (wet AMD).

“While we do not anticipate any further development of ADVM-022 in EMR, we are committed to sharing data from the INFINITY study transparently at this scientific meeting to inform the future development of gene therapy. We are truly grateful to the patients and researchers who participated in this study, our scientific advisors and the Data and Safety Oversight Committee, who continue to support us by sharing their expertise, ”said Laurent Fischer, MD, President and CEO of Adverum Biotechnologies. “With new long-term data from the OPTIC study presented at the Retina Society meeting last weekend and again this Monday at ASRS, it has become clear that anti-VEGFs have different profiles. in different indications and that the safety profile of our new gene treatment was markedly different in DME patients with severe vascular and renal comorbidities at our dose of 6 x 10 ^ 11. These results further strengthen our strategy to focus the development of ADVM-022 on wet AMD, where we continue to observe a promising safety profile and durability of treatment effect that has been maintained in patients. at a median of 1.7 years after treatment with the 2 x 10 ^ 11 OPTICAL dose. We look forward to completing the review of our extensive data from the ADVM-022 program and seeking input from researchers and regulatory agencies as we plan a Phase 2 trial with low doses and improved prophylaxis. “

“These collective INFINITY results underscore the importance of the underlying disease pathology in addition to patient characteristics, dose selection and optimal steroid prophylaxis to determine the therapeutic window that balances efficacy and the safety of new gene therapies, “said Charles C. Wykoff, MD, Ph.D., research director, Retina Consultants of Texas; President of Research and Clinical Trials, Retina Consultants of America and member of the Scientific Advisory Board of Advum. “Focusing the future development of ADVM-022 on patients with wet AMD who require multiple anti-VEGF injections per year and evaluating low doses with improved prophylaxis is a cautious approach that maintains patient safety at first. plan while working to meet the unmet need to reduce the treatment burden of frequent anti-VEGF injections.

INFINITY trial in patients with DME
INFINITY was a masked study in diabetic patients with DME initiated in May 2020, with cohorts evaluating two dose levels of ADVM-022 compared to a cohort with a single dose level of aflibercept. In early May 2021, the study was immediately unmasked to address patient safety and better clinical management following an unexpected serious adverse event in a high dose DME patient with hypotonia , a rapid and clinically relevant decrease in intraocular pressure (IOP) with steroid refractory inflammation and requiring surgery. To date, two additional patients have required surgery and a total of five of twelve patients treated with the high dose have experienced clinically relevant IOP events requiring additional immunosuppressive agents. All of these patients had a history of severe vascular and / or kidney disease, and all events occurred 16 to 36 weeks after treatment. All INFINITY patients have now been followed for at least 36 weeks and no similar event has been observed in low dose DME patients or in wet AMD patients treated at high or low dose. weak.

A photo accompanying this announcement is available on: https://www.globenewswire.com/NewsRoom/AttachmentNg/86364e7b-e506-4140-9f9f-ea97d03c80ba

Data from the INFINITY trial in DME patients presented to ASRS
In data presented from the INFINITY trial (June 22, 2021, cut-off date, n = 34) at ASRS, patients with DME were randomized to receive either a single IVT injection of a low dose of 2x 10 ^ 11 vg / eye of ADVM -022, high dose 6 x 10 ^ 11 vg / eye of ADVM-022, or aflibercept.

  • Key learnings: The dose of ADVM-022 and the state of the disease appear to have an impact on the determination of the therapeutic window which balances efficacy and safety:
    • The dose-dependent inflammation observed in DME patients with dose-limiting toxicity at 6 x 10 ^ 11 vg / eye may be partly related to the study population which included patients with vascular disease and severe renal
    • No clinically relevant reduction in IOP was observed in the 2 x 10 ^ 11 vg / eye group
    • Further development of ADVM-022 will focus on wet AMD and low doses (2 x 10 ^ 11 yd / eye and less)

The presentation of the data is displayed on the Publications Pipeline section page of the company website.

OPTIC data in patients with wet AMD 1-2 years after treatment with ADVM-022
Last weekend, new long-term data from the OPTIC trial of ADVM-022 for wet AMD were presented at Retina Society 54e Annual Scientific Meeting on October 1, 2021. An additional presentation of the OPTIC data is scheduled for 8:50 am CT (9:50 am ET) on Monday, October 11, 2021 at ASRS and Adverum plans to release the data presentation on the Publications Company website Pipeline page at or before the start of the presentation.

  • Key learnings: Data from the OPTIC trial (July 16, 2021, deadline, n = 30) demonstrated in heavily treated patients that a single IVT injection of ADVM-022 provides sustained durability, a reduction of over 80% need for anti-VEGF and a promising safety profile.
    • Robust expression of aflibercept and sustained efficacy ensuring the stabilization of the observed CST
    • Weak eye side effects related to ADVM-022 occurred at 2 x 10 ^ 11 vg / eye
    • No clinically relevant low IOP event was observed at either dose
    • These results support further studies of ADVM-022 in wet AMD, including evaluation of low doses (2 x 10 ^ 11 vg / eye and less) and improved prophylaxis.
    • Long-term data from OPTIC and future studies may validate the importance of ADVM-022 in minimizing long-term fluctuations in CST

Milestones planned for ADVM-022:

  • Plan to complete data analysis by the end of 2021 to inform a protocol and solicit input from researchers and regulatory authorities on a Phase 2 clinical trial in wet AMD
  • Plan to present additional long-term data from the OPTIC and OPTIC Extension studies in wet AMD

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 office 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. wet age patients – related 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 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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