Early results of AMD gene therapy have been positive, giving drugs for 15 months at a time without the need for anti-VEGF rescue injections!

Introduction: Clinical phase gene therapy company Adverum Biotechnologies recently released new positive interim data for the glass in-cavity injection (IVT) gene therapy ADVM-022 treatment wet age-related macular degeneration (wet-AMD) phase I OPTIC study queue 1-4.
, a clinical-stage gene therapy company, recently released new positive interim data for the glass in-cavity injection (IVT) gene therapy ADVM-022 treatment of wet age-related macular degeneration (wet-AMD) Phase I OPTIC research queue 1-4.
the study, conducted in patients requiring frequent injections of antivascular endothormic growth factor (VEGF), showed that low-dose and high-dose ADVM-022 single treatment significantly reduced the frequency of annualized anti-VEGF injections.
, in particular, patients with queue 1 who received high doses of single treatment maintained the efficacy of zero anti-VEGF resuscitation injections for more than 15 months.
ADVM-022 is a new gene therapy that is being developed for the treatment of wet AMD and diabetic macular edema (DME), which can continue to provide aflibercept( Abersip), the active pharmaceutical ingredient of The Bayer/Regenerative Meta-Heavy Eye Drug Eylea.
Eylea requires regular injections, and ADVM-022 has the potential to become a "once and for all" IVT injection therapy that promises to significantly reduce the treatment burden on millions of wet AMD and DME patients worldwide.
OPTIC is a multi-center, open-label, dose-range Phase I study designed to assess the safety and toadability of single-injection ADVM-022 in a single glass in wet AMD patients who respond to VEGF therapy.
patients with queue 1 (n=6) and queue 4 (n=9) received ADVM-022 treatment at high doses (6 x 10 x 11vg/eye), and patients with queue 2 (n=6) and queue 3 (n=9) received ADVM-022 treatment at low doses (2x10^11vg/eye).
13-day preventive oral steroids used in groups 1 and 2, and patients in groups 3 and 4 received preventive steroid eye drops for 6 weeks.
end point of the study was the safety and toadability of ADVM-022 after a single IVT dosing.
secondary endpoints include changes in optimal corrective vision (BCVA), measurement of central retina thickness (CRT), and the need for resuscitation injections against VEGF.
this is the first time it has published interim data for all four queues (as of July 23, 2020), including preliminary security data.
These data further confirm the potential of ADVM-022 to significantly reduce the treatment burden on wet AMD patients: - ADVM-022 continues to show strong treatment response: average BCVA continues to maintain, average CRT continues to improve;
- Further evidence of dose effects: 100% of patients in the high-dose group (queue 1:6/6) did not require resaltic injection therapy, and 67% of patients in the low-dose group (queue 2 and 3:10/15) did not require resaltic injection therapy.
- After ADVM-022 was given, the annualized anti-VEGF injection rate decreased significantly: 100% in the high-dose group and 87% in the low-dose group.
- ADVM-022 continued to show good tolerance, with four queues showing good safety characteristics: all ADVM-022-related eye adverse events were mild (78%) to moderate (22%), and 1 patient with moderate relapsed staphylocoditis thought to be associated with ADVM-022 responded to steroid tricoocytes (queue 1).
eye inflammation observed in the united States responded to steroid eye drops.
there is no clinical or fluorescent evidence of rear inflammation, no vasculitis, retinitis, vasculitis, vascular closure, or intraoculitis.
- Early evidence from Queue 4 is consistent with Queue 3, suggesting that steroid eye drops can reduce adverse events and inflammation compared to the oral steroid prevention programmes used in Queue 1 and Queue 2. Arshad M. Khanani, a top registered researcher in the
OPTIC study and an associate clinical professor of ophthalmology at the University of Nevada, said the long-lasting anatomical response shown after a single-dose glass injection of ADVM-022 was unprecedented, given that patients in the group were difficult to treat and previously required frequent injections to maintain vision.
safety analysis to date shows that ADVM-022 has good tolerance and that preventive steroid eye drops are effective in limiting early eye inflammation.
source: Adverum's gene therapy shows encouraged action in wet AMD study.