Adverum Biotechnologies(US:ADVM)2020-03-16 19:08ADVM-022 for Wet AMD - ADVM-022 is a novel intravitreal gene therapy candidate for VEGF-driven retinal diseases, designed for long-lasting efficacy with a single injection[5] - Wet AMD affects approximately 12 million patients in the U S, and ADVM-022 has received FDA Fast Track designation for this indication[6] - Global sales for approved anti-VEGF therapies reached $11 billion in 2019[18] - OPTIC Phase 1 trial data shows a robust efficacy signal and dose response, with Cohort 1 (6x10^11 vg/eye) showing 6/6 patients rescue injection free and Cohort 2 (2x10^11 vg/eye) showing 4/6 patients rescue free[69] - In the OPTIC trial Cohort 1, the mean BCVA change from baseline was -1.0 ETDRS letters, and the mean CST change from baseline was -25.5 µm[50] ADVM-022 for Diabetic Retinopathy - Approximately 8 million people in the U S have diabetic retinopathy[6], and about 1 million receive treatment[75] - The company plans to submit an IND application for ADVM-022 in diabetic retinopathy in the first half of 2020 and initiate a Phase 1/2 clinical trial in the second half of 2020[6] Platform and Capabilities - The company utilizes an AAV 7m8 vector optimized for IVT delivery and is developing next-generation vectors through a proprietary directed evolution platform[7] - The company is leveraging in-house expertise and external GMP capabilities for scalable manufacturing[80] Financial Position - The company was well capitalized with $166 million in cash as of December 31, 2019, plus $141 million net proceeds from February 2020 offering[8]