by: Alex Young & Anthony DeFino
Adverum Biotechnologies reported positive 52-week data from the phase 2 LUNA trial as well as long-term follow-up results from the OPTIC trial of Ixo-vec in patients with wet age-related macular degeneration.
“Today’s clinical updates underscore Ixo-vec’s potential best-in-class product profile in wet AMD and highlight that we have essentially de-risked our phase 3 pivotal program,” Laurent Fischer, CEO of Adverum, told Healio. “We’ve consistently demonstrated best-in-industry clinical activity, and with our phase 2 LUNA study, we’ve shown to continue to outperform our target of more than 50% of patients injection-free and more than 80% treatment burden reduction in a hard-to-treat population.”
LUNA is an ongoing phase 2 trial of 60 patients with wet AMD who were randomly assigned to receive 6E10 vg/eye or 2E11 vg/eye of Ixo-vec (ixoberogene soroparvovec) intravitreal gene therapy. The trial was designed to help determine the dose and prophylactic regimen for phase 3 registrational trials, according to a press release.
While both doses of the therapy helped patients maintain visual and anatomic endpoints, reduced treatment burden, and were well tolerated, the company chose the 6E10 vg/eye dose with topical eye drops for the registrational trials.
“For Ixo-vec, the reliable safety profile demonstrated throughout the totality of this data is equally important,” Fischer said. “Notably, no 6E10 LUNA patients, the dose we are taking forward into our pivotal program, had inflammation at 52 weeks or any subsequent visit.”
In the OPTIC trial, 30 patients who received 2E11 vg/eye or 6E11 vg/eye of Ixo-vec experienced an 86% reduction in annualized anti-VEGF injections through year 4, and about 50% of patients were injection-free through 4 years of treatment.
“Inadequate durability and the burden of frequent lifelong injections are significant challenges that have a profound impact on patients, their families, and their care teams,” Fischer said. “As a result, patients are chronically undertreated, and this ultimately leads to poor vision outcomes in the real world, both for those who receive bolus anti-VEGF therapy that’s currently the standard of care and for the many patients who stop treatment — up to 40% of patients stop therapy within 2 years and more than 50% after 5 years — and therefore are not benefiting from anti-VEGF.”
