Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that the European Medicines Agency (EMA) has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment of wet age-related macular degeneration (wet AMD).
REDWOOD CITY, Calif., June 24, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that the European Medicines Agency (EMA) has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment of wet age-related macular degeneration (wet AMD). Adverum’s lead gene therapy candidate, ADVM-022 is a one-time, intravitreal (IVT) injection for the treatment of patients with wet AMD.
PRIME is a program launched by the EMA to enhance support for research on and development of medicines that target a significant unmet medical need. This regulatory program offers developers of promising medicines enhanced interaction and early dialogue and is designed to optimize development plans and speed evaluation ensuring these medicines reach patients as early as possible. According to the EMA, developers of medicines that are eligible for PRIME can expect additional opportunities for scientific advice and be eligible for accelerated assessment at the time of application for a marketing authorization. Currently, only three ophthalmology therapies to date have been granted PRIME designation out of 14 submissions.
“We are extremely pleased with EMA’s decision to grant ADVM-022 PRIME designation. Following the encouraging results seen in the OPTIC trial in wet AMD, our PRIME designation recognizes the broad potential of ADVM-022 and is based in part on our trial subjects now having three-year sustained aflibercept expression with stable disease and maintained long-term central subfield thickness and visual acuity after a single IVT injection,” said Laurent Fischer, M.D., president and chief executive officer at Adverum Biotechnologies. “The current standard of care in wet AMD, a highly prevalent disease, requires frequent anti-vascular endothelial growth factor (anti-VEGF) injections in the eye and is a lifelong burden for many patients and their caregivers. We believe ADVM-022 has the potential to provide a durable, safe and cost-effective in-office treatment option that addresses the needs of these patients, and their families, as well as retina specialists and health systems worldwide. We look forward to taking advantage of the benefits provided to us through PRIME as we continue the development of ADVM-022 in areas of high unmet need.”
A Phase 2 study in wet AMD is planned to investigate the 2x10^11 vg/eye dose and a new, lower 6x10^10 vg/eye dose of ADVM-022, as well as new enhanced prophylactic steroid regimens, including local steroids and a combination of local and systemic steroids. Adverum anticipates dosing the first patient in the third quarter of 2022.
About Wet Age-Related Macular Degeneration
Wet AMD, also known as neovascular AMD or nAMD, is an advanced form of AMD, affecting approximately 10% of patients living with AMD. Wet AMD is a leading cause of blindness in patients over 65 years of age, with a prevalence of approximately 20 million individuals worldwide living with this condition. The incidence of new cases of wet AMD is expected to grow significantly worldwide as populations age. AMD is projected to impact 288 million people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases.
About ADVM-022 in Wet AMD
ADVM-022 is Adverum’s clinical-stage gene therapy product candidate being developed for the treatment of wet AMD. ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), ADVM-022 has the advantage of being administered as a one-time, IVT injection in the office and is designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and improve vision outcomes for patients with wet AMD.
About Adverum Biotechnologies
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with neovascular or wet age-related macular degeneration. For more information, please visit www.adverum.com.
Forward-looking Statements
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 initiate a Phase 2 study in wet AMD to investigate the 2x10^11 vg/eye dose and a new, lower 6x10^10 vg/eye dose of ADVM-022, as well as new enhanced prophylactic steroid regimens, including local steroids and a combination of local and systemic steroids, planned for the third quarter of 2022. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; and the potential for future complications or side effects in connection with use of ADVM-022. Additional risks and uncertainties facing Adverum are set forth under the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reports, including Adverum’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 filed with the SEC on May 12, 2022. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
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