US retinal specialists identify 4D Molecular Therapeutics’ 4D-150, Adverum Biotechnologies’ Ixo-vec, and Johnson & Johnson’s JNJ-1887 as most likely pipeline candidates for prescription if approved.
Exton, PA., July 24, 2024 – The American Society of Retinal Specialists (ASRS) annual meeting held last week in Stockholm, Sweden, showcased groundbreaking advancements in gene therapy for retinal disorders. The promising data presented during the event has generated excitement among ophthalmologists and suggests a significant transformation in the treatment landscape for retinal diseases. Among the notable presentations, 4D Molecular Therapeutics’ 4D-150 and Adverum Biotechnologies’ Ixo-vec stood out with compelling phase 2 data. These therapies demonstrated efficacy, safety and a reduction in treatment burden, setting a new precedent for gene therapies that maintain or improve visual acuity while significantly reducing treatment burden along with a favorable safety profile.
These advancements signal a new era in retinal treatments, moving away from traditional therapies. Spherix Global Insights surveyed 77 US retinal specialists in the Special Topix™: Gene Therapy (US) study on the evolution of the retinal landscape. As one member of Spherix Global Insights’ Ophthalmology Community remarked:
“I think that in five years from now, we’re going to be having a completely different conversation. I think that all these genetic agents that are coming out, these intraocular factory agents, these Adverum and RegenxBio agents, this is going to be the way of the future. The eye will become its own factory for aflibercept, and we won’t be using Lucentis, Eylea, and Vabysmo anymore. They’ll all fall by the wayside.”
Furthermore, retinal specialists identified 4D-150, Ixo-vec, and Johnson & Johnson’s JNJ-1887 as the most likely candidates for prescription. Meanwhile, AbbVie/RegenxBio’s ABBV-RGX-314 generated less interest due to its invasive route of administration (subretinal or suprachoroidal injections).
The study analysis also reveals that the highest interest in gene therapy uptake will be for retinitis pigmentosa (RP), Stargardt disease, and geographic atrophy (GA)—conditions with significant unmet needs for effective treatments. With regards to age-related and diabetic retinal disease, 84% of ophthalmologists overwhelmingly agree that a gene therapy with a durability of two years would improve treatment outcomes, compared to a 49% agreement for therapies with a six-month durability. Moreover, specialists show a preference for therapies that improve visual acuity over those that maintain it.
Despite the enthusiasm, safety concerns remain a primary barrier to adoption. Retinal specialists are cautiously optimistic, closely monitoring trial results for long-term consequences, administration methods, and costs. “I’m open to gene therapy but cautious about long-term consequences,” noted one specialist, while another added, “I am excited about gene therapy, but I am very nervous about potential side effects and blindness.” These statements illustrate that while gene therapies hold revolutionary potential, patient safety remains the top priority for ophthalmologists.
Spherix will continue to closely monitor the market to help navigate this evolving landscape and support informed decision-making in retinal disorder treatments.
Spherix’s recent gene therapy study answers key business questions regarding the future potential of gene therapies, including indications and endpoints of utmost interest to retinal specialists, their attitudes regarding the application timing and location for gene therapy, and the educational requirements they foresee needing from manufacturers.
Special Topix™ is an independent service that includes access to a report or series of reports based on current events or topics of interest in specialty markets covered by Spherix.
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Spherix Global Insights Contacts
Blaine Cloud, Ophthalmology Franchise Head
blaine.cloud@spherixglobalinsights.com
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