Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ophthalmology and rare diseases, today announced the publication of preclinical long-term expression and efficacy data in Molecular Therapy, the leading international journal for research on the development of molecular and cellular therapeutics to correct genetic and acquired diseases.
The publication entitled, "Preclinical evaluation of ADVM-022, a novel gene therapy approach to treating wet age-related macular degeneration," reported the following:
A single intravitreal administration of ADVM-022 in non-human primates (NHPs) provided stable and robust intraocular expression of aflibercept and resulted in high levels of aflibercept within the retina and choroid where wet age-related macular degeneration (wet AMD) occurs Robust aflibercept expression in retinal and choroidal tissues obtained at 16 months post-administration demonstrated that cells transduced with ADVM-022 may continue to generate aflibercept for an extended period of time
In a laser-induced choroidal neovascularization model in NHPs, a single intravitreal administration of ADVM-022 13 months before lasering prevented the occurrence of clinically relevant choroidal neovascularization lesions, similar to animals that received a bolus of intravitreal aflibercept (standard-of-care) at the time of lesioning
A single intravitreal administration of ADVM-022 may provide a safe and effective long-term treatment option for wet AMD
The full online publication can be accessed at the following link: https://doi.org/10.1016/j.ymthe.2018.11.003
Adverum recently received Fast Track Designation from the U.S. Food and Drug Administration (FDA) and announced initiation of a phase 1 trial evaluating ADVM-022 in patients with wet AMD.
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. At least six leading retinal centers across the United States are expected to participate in the phase 1 trial. The trial is expected to enroll 18 patients and evaluate three doses of ADVM-022; first dose: 6 x 10^11 vg/eye, second dose: 2 x 10^12 vg/eye, and third dose: 6 x 10^12 vg/eye. Patients will be administered a tapering prophylactic corticosteroid regimen. The primary endpoint of the trial is the safety and tolerability of ADVM-022 at 24 weeks 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 rescue anti-VEGF injections and percentage of patients needing rescue anti-VEGF injections. Each patient enrolled is expected to be followed for a total of two years. For further details about the trial, enrollment and eligibility please contact adverumopticstudy@adverum.com or visit https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene Therapy Candidate in Wet AMD
Adverum's gene therapy candidate, ADVM-022, utilizes a proprietary vector capsid (AAV.7m8) carrying an aflibercept coding sequence under the control of a proprietary expression cassette and is administered as a single intravitreal administration. AAV.7m8 was discovered by directed evolution and is a variant of AAV2. The vector was designed with the purpose of improved transduction efficiency to the retina when administered intravitreally. ADVM-022 is designed to provide potentially sustained therapeutic levels of aflibercept and to minimize the burden of frequent anti-VEGF injections.
About Wet Age-related Macular Degeneration (Wet AMD)
Age-related macular degeneration (AMD) is a progressive disease affecting the retinal cells in the macula, the region of the eye responsible for central vision. Disease progression results in the death of retinal cells and the gradual loss of vision. Approximately 10% of patients living with AMD have an advance form of the disease called wet AMD, in...
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