Adverum Biotechnologies reports additional data from Phase 1 trial of ADVM-022
Adverum Biotechnologies (ADVM) announced additional clinical data for the first cohort of patients in the ongoing OPTIC phase 1 clinical trial of ADVM-022, the company’s intravitreal injection gene therapy, in treatment-experienced patients with wet age-related macular degeneration.
Adverum also announced enrollment plans for the third and fourth cohorts in the ongoing OPTIC trial.
The third cohort has been initiated and patients will be treated with ADVM-022 at a dose of 2 x 10^11 vg/eye, the same dose used in the second cohort.
Subsequently, patients in the fourth cohort will be treated with ADVM-022 at a dose of 6×10^11 vg/eye, the same dose used in the first cohort.
Since inflammation has generally been mild and responsive to steroid eye drops, patients in the third and fourth cohorts will receive prophylactic steroid eye drops instead of prophylactic oral steroids.
Previously, on September 12, 2019, Adverum presented data from the first cohort in the ongoing OPTIC trial at a pre-specified 24-week time point.
The additional data for this cohort includes key outcomes with a median follow-up of 34 weeks.
In treatment-experienced patients previously requiring frequent anti-VEGF injections to maintain vision, the data continue to demonstrate that the single ADVM-022 injection in this cohort was sufficient to maintain vision, with zero rescue injections required for any of the six patients.
Aaron Osborne, MBBS, chief medical officer of Adverum, added, “The clinical profile of ADVM-022 demonstrates this gene therapy’s potential to be a significant advance for patients with wet AMD. It is very encouraging that there continues to be zero rescue injections in this cohort of treatment-experienced patients with more than 6 months follow-up on all patients. We are expanding the development of ADVM-022 and are excited to share that enrollment is now open for the third cohort in OPTIC.
We look forward to being able to deliver this novel intravitreal gene therapy candidate as soon as possible to patients with wet AMD and diabetic retinopathy, our second indication for ADVM-022. We are grateful for all of the investigators, patients, and caregivers who continue to participate in the OPTIC trial.”
Adverum plans to begin dosing patients in the third cohort of the OPTIC trial in the fourth quarter of 2019 and plans to begin enrollment in the fourth cohort in the first quarter of 2020.
Adverum plans to present 52-week data from the first cohort of patients in the OPTIC trial as well as 24-week data from the second cohort of patients in the first half of 2020.
Adverum plans to submit an investigational new drug application for the treatment of ADVM-022 in diabetic retinopathy in the first half of 2020.
Commenting on Adverum Biotechnologies update at AAO, Piper Jaffray analyst Tyler Van Buren notes that zero rescue injections have been required and disease activity by OCT and visual acuity has been maintained with 6 ADVM-022 patients now out to a median of 34 weeks.
Importantly, despite the intense criticism over inflammation and visual acuity fluctuations during the initial data disclosure last month, numerous KOLs at the conference were unconcerned and believe that ADVM-022 looks like a potential treatment option so far, he contends.
Overall, the analyst argues that safety continues to be clean in these initial patients and looks forward to the 52-week update in first half of 2020.
Van Buren reiterates an Overweight rating and $14 price target on the shares as ADVM-022 continues to look like a viable gene therapy candidate for wet AMD, which has $1B-plus potential.
ADVM closed at $5.29.
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