Adverum Biotechnologies Inc. (NASDAQ:ADVM) develops gene therapy product candidates designed to offer sustained recovery to patients suffering from ocular and rare diseases. The company has been working on Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy. Interim data from Cohorts 1-4 of the OPTIC have, for the first time, demonstrated the transformative potential of ADVM-022.
Some of the key highlights from the therapy included improved mean CRT2 and maintained BCVA1. There was also a notable reduction in annualized anti-VEGF injection rate and tolerance of ADVM-022 plus a favorable safety profile in all 4 cohorts.
Nonetheless, there were mild and moderate adverse events of 78% and 22%, respectively. The overall observation was that ADVM-022 could greatly reduce the treatment burden for patients with wet age-related macular degeneration (AMD).
Moving the Potentially Transformative Therapy into Pivotal Trials
The patients enrolled in OPTIC were difficult to treat, according to Arshad M. Khanani, the managing partner of Sierra Eye Associates. They had previously required repeated injections to uphold their vision. Hence, the treatment response of ADVM-022 can be described as unprecedented and remarkable. From the safety profile, the prophylactic steroid eye drop regimen was effective in controlling early ocular inflammation.
From all these, Khanani says they are at the point where they could push the transformative therapy into pivotal levels. The aim is to improve the real-world results for patients living with wet AMD. The company has completed dosing 30 patients in OPTIC and is looking forward to having the data ready by the end of the year. It is also seeking to indulge the U.S. and international regulatory authorities in its pivotal clinical trial initiative in mid-2021.
Expansion of Business Capabilities and Operations
The CEO of Adverum, Laurent Fischer, says the company has what it takes to help millions of patients with wet AMD and DME worldwide.
The current therapies for these conditions can be burdensome, and their administration’s compliance is difficult for patients and their caregivers. Compliance deficiencies mean loss of vision.
However, the biotech is anticipating to expand its business capabilities and operations to accommodate or resolve these and other related challenges.