Adverum Biotechnologies Presents Updated Preclinic

MENLO PARK, Calif., Sept. 27, 2017 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM ), a leading gene therapy company advancing novel medicines to address unmet needs in serious rare and ocular diseases, today will present updated preclinical data on ADVM-022 in non-human primate models of wet AMD (wAMD). Vascular endothelial growth factor (VEGF) overexpression plays a key role in the development and progression of wAMD as well as other retinal conditions.  ADVM-022 utilizes a novel vector designed to allow for intravitreal delivery of gene sequences encoding an inhibitor of VEGF activity. Intravitreal delivery is the route of administration for current standard of care anti-VEGF therapies, and is preferred over invasive surgical subretinal delivery of gene sequences. 

Mehdi Gasmi, Ph.D., Chief Science and Technology Officer at Adverum will present updated data today at 2 p.m. ET at the Targeting Ocular Disorders conference taking place in Boston, MA September 27–28, 2017.

Previously reported data using the industry standard, laser-induced choroidal neovascularization (CNV) model in a non-human primate study demonstrated that a single intravitreal administration of ADVM-022 had comparable efficacy in reducing grade IV CNV lesions to an intravitreal injection of standard-of-care anti-VEGF therapies. In addition, data showed durable anti-VEGF protein expression with therapeutic protein levels at least 20 weeks after a single intravitreal administration of ADVM-022. 

Today, additional pharmacokinetic data are being presented, demonstrating sustained expression for 52 weeks, with only mild ocular inflammation following a single intravitreal administration of ADVM-022 in one non-human primate. In a separate study, sustained expression for at least seven months has been observed in several non-human primates. Adverum will continue to monitor these non-human primates to assess long-term durability of sustained expression of anti-VEGF protein after a single administration of ADVM-022.

“These additional long-term data continue to demonstrate sustained expression of anti-VEGF protein and that our novel vector enables intravitreal delivery,” said Amber Salzman, Ph.D., president and chief executive officer of Adverum Biotechnologies. “Today, patients living with wAMD undergo frequent intravitreal injections, which can increase the burden of managing this disease and poor compliance with treatment can lead to vision loss. We believe the potential of a single intravitreal injection will be of great benefit to patients and clinicians.”

About Adverum Biotechnologies, Inc. Adverum is a gene therapy company advancing novel medicines that may offer life-changing benefits to patients living with 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, the Company generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development. For more information please visit www.adverum.com.

Adverum Contact: Charles Butler Vice President, Investor Relations & Corporate Communications 650-272-6260