Arshad M. Khanani
It has been a busy year, full of medical meetings with friends and colleagues from around the globe. One thing is for certain, the future has never looked brighter for our field. It has been my distinct honor to be a part of new developments and present these advancements at recent meetings, which will bring new treatment options for our patients.
A big theme in treatment of vascular retinal diseases has been durability and sustained delivery of therapy. Despite recent approvals of longer-acting therapies, our patients with neovascular age-related macular degeneration (nAMD) still often require a life-long and burdensome schedule of treatment with intravitreal injections that is difficult to maintain and results in suboptimal vision outcomes in the real world. I am excited by the multiple next-generation approaches in clinical development such as gene therapy and extended release tyrosine-kinase inhibitors (TKIs) that have shown promising results to address this. At EURETINA this year, I presented such results for a promising gene therapy, 4D-150, from 4D Molecular Therapeutics (4DMT) and the PRISM study in nAMD.
4D-150 is a single intravitreal injection that provides continuous, local and steady-state expression of anti-VEGF (aflibercept and VEGF-C RNAi) with the goal of maintaining disease control with minimal to no supplemental treatment, which is expected to result in long term vision preservation. The data from the PRISM phase I/II study I presented at EURETINA showed robust and durable clinical activity through up to 2.5 years in a broad range of patient populations. The phase IIb results showed 70 percent of patients remained injection free and 80 percent required 0-1 injections after a single injection of 4D-150. In recently diagnosed patients that most resemble the potential phase III population, 87 percent of patients were injection-free and 13 percent only required one injection through 52 weeks. Importantly, 4D-150's safety and tolerability data continue to be in line with approved anti-VEGF agents and with a simple, predictable topical steroid prophylaxis. Based on the data to date, 4D-150 has the potential to provide a significant reduction in treatment burden and long-term vision preservation for our patients.
I was pleased to see the retina community energized by the 4D-150 results from PRISM and I am excited to participate in the 4FRONT phase III program starting early next year.
I look forward to seeing many of you at upcoming medical meetings and discussing the many exciting advancements in our field. Thank you in advance for joining me in supporting development of promising therapies to provide the best possible care for our patients.
