Adverum Biotechnologies Announces Long-term Preclinical Efficacy Data on ADVM-022 Gene Therapy in Wet AMD
-- 13-Month Data Show Efficacy and Durability of Protein Expression following a
-- Data to be Presented in a Poster Session on
The data will be presented in a poster presentation on
“We continue to be encouraged by the efficacy and sustained protein levels we are seeing a year post a single intravitreal injection of ADVM-022,” said
“ADVM-022 represents a novel approach to treating wAMD with a gene therapy administered as a single intravitreal injection,” said Szilard Kiss, M.D., director of clinical research in the
About the ADVM-022 Preclinical Study
The long-term efficacy of ADVM-022 was evaluated in the industry-standard laser-induced choroidal neovascularization (CNV) model in non-human primates (NHPs). NHPs received a single intravitreal injection of either ADVM-022 (n=4, 100 µL, ~2x1012 vg/eye, bilaterally) or vehicle (n=4, 100µL, bilaterally) 12.5 months prior to lasering of the macular region of the retina to induce VEGF upregulation and CNV. As a positive control, a separate group of animals received bilateral intravitreal injections of aflibercept recombinant protein (n=4, 30 µL, 1.2 mg/eye), an anti-VEGF standard-of-care therapy, at the time of lasering. Clinically-relevant Grade IV lesions were evaluated at two and four weeks post lesioning and results were as follows:
| Efficacy Data Incidence of Grade IV Lesion |
|||
| ADVM-022 (13 Months) (n=4) |
Aflibercept (at Lasering) (n=4) |
Vehicle Control (13 months) (n=4) |
|
| 2 Weeks Post Lesioning | 0%1,2 | 2.8%1,2 | 42.8%1 |
| 4 Weeks Post Lesioning | 6.3%1,2 | 4.5%1,2 | 40.3%1 |
| 1p<0.0001 vs vehicle | |||
| 2 p=0.4 and 0.7 between ADVM-022 and aflibercept groups at 2 and 4 weeks, respectively | |||
Three additional animals that did not undergo laser treatment and that received the same intravitreal ADVM-022 injection showed stable vitreous levels of aflibercept at approximately 3 µg/mL 13 months post vector administration.
ASGCT Poster Session
Poster Title: AAV.7m8-aflibercept Provides Long-term Protection in a Non-human Primate Model of
Session Title: Neurologic Diseases (Including Ophthalmic and Auditory Diseases) II
Time:
Location: Hilton Chicago, Stevens Salon C & D
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. VEGF overexpression can lead to wAMD progression and vision loss. Treatment with ADVM-022 is designed to minimize the burden of frequent anti-VEGF injections, the current standard-of-care treatment for wAMD.
About
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:
Chief Financial Officer
650-665-7222
lpatterson@adverum.com
Source: Adverum Biotechnologies, Inc.