A US biotechnology company, Advernum Biotechnologies Inc., (NASDAQ:ADVM), has announced the enrolment of a Phase 2 study for the treatment of diabetic macular edema (DME), using a potential “one-time intravitreal injection gene therapy for VEGF-driven retinal diseases”. ADVM-022 is a gene therapy (AAV.7m8-aflibercept gene therapy) for the “INFINITY” study, registered at clinicaltrials.gov (NCT04418427). According to the sponsor, INFINITY is aimed to recruit 33 eligible subjects randomly assigned to receive one of two doses of ADVM-022, or, assigned to the control arm to receive a sham ocular injection with a preceding aflibercept injection. Participants who are assigned to receive ADVM-022 will be further randomized to receive a preceding aflibercept or sham ocular injection.
Obviously, the “holy grail” for anti-VEGF treatment without necessary repeat injections would be an enormous benefit for industry, patients and clinicians. To pursue this objective, a previous Phase I study (OPTIC, for wet AMD), was carried out by Advernum and reported that patients were administered a single intravitreal injection of ADVM-022 at 6×10^11 vg/eye. The study showed no serious adverse events, no dose limiting toxicities and no non-ocular adverse events related to ADVM-022. Importantly, “patients with nAMD treated with a single injection of ADVM-022 were able to maintain vision and improve anatomical outcomes without requiring any rescue anti-VEGF injections through a median of 34 weeks”.
According to the sponsor for the INFINITY study, patient enrolment was progressed during the COVID-19 pandemic, and completed within six months. Commenting on the clinical trial activity, Dr. Arshad M. Khanani, M.D., M.A., a principal investigator in the INFINITY trial, stated, “patients with DME require frequent, long-term anti-VEGF intravitreal injections, which during COVID-19 is even more challenging and in many parts of the world actually impossible. ADVM-022 is a single in-office intravitreal injection therapy that has shown promising efficacy and safety for over two years in patients with wet AMD in the OPTIC trial. In INFINITY, we are excited to study ADVM-022 in our patients with DME, who are often of working age and need a more durable treatment over the long course of their disease”. According to the sponsor, ADVM-022 may be a potential “transformative treatment” for patients with DME.