Benzinga | Feb 2, 2021 09:06AM EST

Adverum Biotech Highlights Publication Of Preclinical Long-Term Safety Data On ADVM-022 IVT Gene Therapy

Adverum Biotechnologies, Inc. (NASDAQ:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the publication of preclinical data on ADVM-022 intravitreal (IVT) gene therapy in Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO). ADVM-022 is in clinical trials for wet AMD and DME, and this preclinical study in NHPs is the longest safety and expression study to date, with measurements out 30 months following a single IVT injection.

"There is a growing body of both clinical and preclinical data demonstrating durable efficacy and favorable safety profile following a single IVT injection of ADVM-022," said Laurent Fischer, M.D., chief executive officer at Adverum Biotechnologies. "In this preclinical study, we saw long-term, sustained aflibercept expression out to 30 months following ADVM-022. The levels of aflibercept were sustained at therapeutic levels, with no measurable adverse effects on normal retinal structure and function. We are excited to work on developing ADVM-022 as a potential "one and done" IVT injection therapy that may dramatically reduce the treatment burden for patients living with wet AMD and DME."

Szil?rd Kiss, M.D., academic retina specialist, added, "Currently, patients with wet AMD are treated with frequent anti-VEGF intravitreal injections to maintain their vision. One of the highest priorities in research today is to develop therapies that extend the duration of efficacy following treatment, enabling patients to preserve sight for months or years following treatment. The preclinical data on ADVM-022 demonstrate long-term safety and aflibercept expression following a single intravitreal injection of this novel IVT injection gene therapy. We are excited to continue to assess ADVM-022 as it demonstrates the potential to improve real-world visual outcomes over intermittent anti-VEGF injections for patients living with wet AMD."

The publication, titled "Long-Term Safety Evaluation of Continuous Intraocular Delivery of Aflibercept by the Intravitreal Gene Therapy Candidate ADVM-022 in Nonhuman Primates," reported the following:

* A single IVT injection of ADVM-022 (2 x 10^12 vg/eye in this study) appears to be safe and well tolerated and resulted in sustained expression of aflibercept with no detectable adverse effects on normal retinal structure or function measured out to 30 months.

* Sustained therapeutic levels of aflibercept expression post IVT injection of ADVM-022 were confirmed from serial anterior chamber and vitreous humor taps

* Overall ocular health was comprehensively assessed by longitudinal anterior and posterior segment analysis by slit-lamp, fundus examination, optical coherence tomography (OCT), blue light fundus autofluorescence (FAF), as well as terminal histological evaluation; the effect on retinal function was determined by electroretinography (ERG).

* The integrity of retinal pigment epithelium (RPE) was maintained throughout the study with no histological abnormalities observed.

* Mild-to-moderate inflammatory responses were observed that did not require steroid treatment.

The full online publication can be accessed from the TVST website.