Adverum Biotechnologies Presents Additional Long-term Preclinical Data on ADVM-022 in Wet AMD at ASGCT 21st Annual Meeting
Long-term Protection in Nonhuman Primate Model of Wet AMD --
“It is exciting to see that a single administration of ADVM-022 has the potential to provide long-term protection against choroidal neovascularization, which is associated with vision loss in wAMD,” said
Preclinical data from the ADVM-022 poster presentation at ASGCT include:
- After 13 months, a single intravitreal injection of ADVM-022 was found to be safe and statistically significant (p<0.0001) in preventing the development of Grade IV lesions compared to the vehicle control group. The efficacy at 13 months was consistent with earlier-reported data, demonstrating that ADVM-022 induced long-term efficacy that was comparable to aflibercept, an anti-Vascular Endothelial Growth Factor (VEGF) standard-of-care therapy. Additionally, mean CNV complex areas were significantly smaller (p<0.0001) in the ADVM-022 and aflibercept groups, compared with vehicle, when analyzed by spectral domain optical coherence tomography (SD-OCT).
- ADVM-022 induced sustained intraocular expression of aflibercept for up to 16 months following a single intravitreal injection. Robust levels of aflibercept protein were detected up to 16 months in aqueous and vitreous humor and, more importantly, in retina and choroid tissues, where neovascularization occurs in wAMD.
- A separate pharmacokinetics study revealed that levels of vector-derived aflibercept measured in the vitreous and the retina 56 days post ADVM-022 injection match the levels of aflibercept recombinant protein 3-4 weeks post bolus of protein injection, which is well within the therapeutic window of the standard of care. In addition, the levels of vector-derived aflibercept at 56 days were consistent with those observed in the long-term efficacy study at 16 months. Taken together, these data suggest that a single injection of ADVM-022 administered intravitreally can generate stable levels of aflibercept found to be within the therapeutic window of the standard-of-care recombinant protein.
- ADVM-022 was well tolerated, with no serious adverse events. ADVM-022 had mild effects on aqueous and vitreous cell infiltrates, keratic precipitates, and intraocular pressure (IOP). Treatment-related side effects were limited to mild inflammatory responses and related transient IOP decrease. Longitudinal OCT studies, fundoscopy, and fluorescein angiography did not identify changes in retinal morphology, optic nerve head and vascular integrity.
About ADVM-022 Gene Therapy for wAMD
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 injection. Vascular endothelial growth factor (VEGF) activity is associated with wAMD progression and vision loss. Anti-VEGF standard-of-care therapies administered every 4-8 weeks have shown the potential to prevent disease progression and preserve or even improve patients’ vision. Treatment with ADVM-022 is designed to minimize the burden of frequent anti-VEGF injections, the current standard-of-care treatment for wAMD.
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with
Adverum’s Forward-Looking Statements
Statements contained in this press release regarding Adverum’s intention to file an IND application for ADVM-022 in the second half of 2018 and potential for further development of ADVM-022 are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. 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 described in Adverum’s periodic reports filed with the
Contact for Adverum:
Leone PattersonInterim President and Chief Executive Officer 650-665-7222 firstname.lastname@example.org
Source: Adverum Biotechnologies, Inc.