Executives from 4D Molecular Therapeutics (NASDAQ:FDMT) said the company is positioning its lead retinal gene therapy candidate, 4D-150, as a potential “backbone” treatment for wet age-related macular degeneration and diabetic macular edema, aiming to reduce the need for repeated anti-VEGF injections.
Speaking at RBC Capital Markets’ 2026 Global Healthcare Conference, Chris Simms, chief commercial and business officer, said the company is trying to change the current treatment model in retinal disease from periodic bolus injections to an “always onboard” therapy that can be supplemented when needed.
Regulatory Discussions and DME Trial Plans
Simms said 4D Molecular Therapeutics has not seen any company-specific issues in its communications with regulators despite broader volatility at the FDA. He described the company’s Phase 3 program in wet AMD as “very traditionally designed,” comparing its structure to prior pivotal programs for therapies such as Vabysmo and Eylea.
The company plans to begin a pivotal DME trial in the third quarter. Simms said 4D Molecular Therapeutics has regulatory alignment from the FDA, EMA and Japan for a single global DME trial, which will be conducted in partnership with Otsuka in Asia-Pacific.
While the company has not yet disclosed the full DME trial design, Simms said investors should expect a “standard and traditional” approach similar to the company’s 4FRONT-1 and 4FRONT-2 studies in wet AMD. He also said the DME study is likely to include rescue criteria and a prophylactic steroid regimen, though details have not yet been shared.
Wet AMD Opportunity and Competitive Landscape
Simms said the current anti-VEGF treatment paradigm, which has relied on intravitreal injections for roughly two decades, has created substantial treatment burden for patients and physicians. He said real-world data suggest patients often lose the initial vision gains achieved after starting therapy, in part because of undertreatment or lapses in care.
He said continuous VEGF suppression has shown the potential to preserve vision over time, citing monthly Lucentis dosing and Genentech’s Susvimo implant as examples of approaches that provide sustained disease control. Simms said 4D-150 is intended to provide a similar long-term outcome through a biological approach rather than an implant.
Asked about long-acting tyrosine kinase inhibitors and other gene therapy programs in development, Simms said those therapies may have a role if successful, but he argued that 4D-150 is differentiated because it is not merely trying to extend durability by a week or two. Instead, he said the goal is to allow many patients to avoid further bolus injections for months or years.
Simms also highlighted the intravitreal delivery of 4D-150 as a potential advantage over surgical or subretinal gene therapy approaches, saying it could integrate more easily into retina practices.
Market Potential and Pricing
RBC analyst Lisa Walter noted that the branded wet AMD market is worth about $10 billion and the DME market about $4 billion. Simms said 4D Molecular Therapeutics projects the global anti-VEGF market could reach as much as $20 billion around the company’s expected launch horizon as the population ages.
Simms said the company does not view 4D-150 as a niche product. Based on physician feedback, he said doctors may initially avoid using the therapy in patients who are well controlled with only two or three injections per year, a group he estimated at about 10% of the overall AMD population. For the broader patient population, he said physicians have indicated they would consider 4D-150 if the Phase 3 data support the profile seen to date.
On pricing, Simms said 4D-150 would not follow the multi-million-dollar pricing model associated with some rare disease gene therapies. He said he would be surprised if the price were above $100,000, though the final decision will depend on Phase 3 data, market dynamics and other factors. He also said the company believes its manufacturing expertise and low dose could support a cost of goods profile of less than $1,000.
Clinical Endpoints and Upcoming Data
The company’s two pivotal wet AMD studies, 4FRONT-1 and 4FRONT-2, are non-inferiority trials that include rescue criteria for supplemental anti-VEGF injections. Simms said the company believes it has derisked the program as much as possible while maintaining clinical relevance, but added that outcomes cannot be guaranteed until data are available.
Simms said treatment burden reduction will be an important secondary endpoint. He pointed to Phase 2 data showing treatment-free rates of 50% to 70%, depending on the population, and overall treatment burden reductions in the 80% to 90% range. He said results around 50% treatment-free and more than 80% treatment burden reduction at week 52 would be viewed by the company as a major success.
Simms said investors should expect two-year follow-up data from the PRISM study in broad and recently diagnosed patient populations to show continued treatment burden reduction and safety follow-up. He also confirmed that an update from the DME SPECTRA trial is expected in the second half of the year.
Cash Position
Kristian Humer, chief financial officer, said 4D Molecular Therapeutics ended the first quarter with $558 million in cash. He said the company is guiding to a cash runway into the second half of 2028, though that runway does not include the commercial build-out that would be needed after 4FRONT-1 data.
About 4D Molecular Therapeutics (NASDAQ:FDMT)
4D Molecular Therapeutics, founded in 2015 and headquartered in Emeryville, California, is a clinical-stage biotechnology company focused on the development of targeted gene therapies for rare diseases. The company employs its proprietary Gene Expression AAV (GEA) platform to engineer novel adeno-associated virus (AAV) capsids with enhanced tissue selectivity and transduction efficiency. This platform aims to improve the precision and durability of gene delivery compared to traditional AAV approaches.
4D’s pipeline includes both preclinical and clinical-stage programs across multiple therapeutic areas.
