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Adverum Biotechnologies, Inc. ADVM announced data from cohort 1 of the phase I study — OPTIC — evaluating its gene therapy candidate, ADVM-022, in patients with wet age-related macular degeneration (“AMD”).
The cohort 1 of the study was evaluating a one-time intravitreal 6 x 10^11 vg/eye dose of ADVM-022 to observe patients for vision maintenance and improvements in retinal anatomy over a period of 24 weeks. Data from the study showed that the therapy was safe and well tolerated. Meanwhile, patients treated with ADVM-022 achieved sustained improvements in retinal anatomy and did not require anti-VEGF rescue injections. The patients enrolled in the study had previously required frequent anti-VEGF injections to avoid loss of vision.
Moreover, ADVM-022 achieved a mean change of -2 letters in best corrected visual acuity and a mean reduction of 52.7 micrometer in central retinal thickness.
Although data suggests that treatment with ADVM-022 was generally positive, it also resulted in mild adverse events in all patients and different types of inflammation was observed in most of them.
Shares of Adverum plunged more than 50% on Sep 12, following study data readout. However, the company’s shares are up 89.3% so far this year against the industry’s decrease of 1.3%.
We note that the company is evaluating a lower dose of 2 x 10^11 vg/eye in cohort 2 of the OPTIC study. Data from the study will likely demonstrate whether a lower dosage of ADVM-022 will lead to a reduction in inflammation events in patients.
We remind investors that the candidate was granted Fast Track designation by the FDA for wet AMD in September 2018.
The development of this candidate is crucial for the company as it discontinued the development of ADVM 043, a gene-therapy candidate for the treatment of alpha-1 antitrypsin (A1AT) deficiency, in November 2018.
The company is planning to develop ADVM-022 in diabetic retinopathy. It also has collaboration agreements with Editas Medicine EDIT and Regeneron Pharmaceuticals REGN for other candidates.
The market potential of wet AMD is significant as it is a leading cause of vision loss in patients above 60 years of age. There are approximately 1.2 million individuals in the United States and 3 million globally affected by wet AMD. Approximately 150,000 to 200,000 people are estimated to be affected by wet AMD in the United States every year.
We note that Novartis NVS is also developing a therapy targeting wet AMD. In April, the FDA has granted priority review to Novartis’ Biologics License Application seeking approval for brolucizumab as a treatment for wet AMD.