- Regeneron Pharmaceuticals on Thursday said a high-dose form of its top-selling drug Eylea met the goals of two Phase 3 trials, positioning the company and its partner Bayer to seek approval from regulators.
- Study results show the higher dose version was non-inferior to Eylea on measures of visual acuity in patients with either age-related macular degeneration or diabetic macular edema. A majority of patients achieved those results despite less-frequent dosing than Eylea, which is administered via injection once every two months.
- The findings include some caveats, including evidence of decreased effectiveness with extended dosing. Still, the results beat the expectations of Wall Street analysts, many of whom now argue Regeneron will hold onto market share despite rising competition.
For years, the market for the “wet” form of age-related macular degeneration (AMD) and diabetic macular edema (DME), two common types of vision loss, has been dominated by Eylea.
The drug is consistently the top-selling medicine despite other available options, including Roche’s Lucentis and off-label use of the cancer drug Avastin. It’s also fended off newer threats, like Novartis’s Beovu, and seen would-be competitors fall short in clinical testing. In 2021, ten years after its initial approval, Eylea generated nearly $6 billion in U.S. sales and more than $9 billion globally, making it one of the world’s best-selling pharmaceutical products.
Yet Eylea’s run could be nearing its end. The Food and Drug Administration has approved two biosimilar versions of Lucentis this year. One of them, developed by Coherus BioSciences, was given an “interchangeable” designation by the agency, meaning pharmacists can substitute it for the branded version.
Treatment regimens requiring fewer injections than Eylea are making inroads, too. Roche has two options: a refillable implant called Susvimo and an injectible antibody drug known as Vabysmo. Analysts have been paying particularly close attention to the latter, which has dug into Eylea’s market share in recent months. Gene therapies from RegenXBio and others, designed to provide long-lasting benefit with a single treatment, are also advancing.
Some ophthalmologists additionally use a “treat-and-extend” approach to reduce the injection burden on patients.
Regeneron’s answer is a higher-dose form of Eylea. The company has been developing an 8 mg dose, four times higher than Eylea. It’s trying to prove the new version is as effective and safe as its counterpart with just three or four injections per year.
The study results disclosed Thursday show Regeneron appears to have achieved that goal. Regeneron enrolled patients with DME in one trial and AMD in the other and gave them either a typical Eylea regimen, or a high-dose version once every three or four months. The trials tested whether the new version could match Eylea at helping patients see more letters on an eye chart.
In the DME trial, patients who received Eylea saw an average of 9.2 more letters after 48 weeks, compared to 8.8 for patients given the high-dose formulation every three months and 7.9 for those receiving it every four months. Likewise, patients given Eylea in the AMD study saw 7.6 more letters on the chart at the end of the period, compared to 6.7 and 6.2 for the other two groups, respectively.
The numerically weaker results with extended dosing “may impact overall perceptions and potential market conversion,” wrote RBC Capital Markets analyst Brian Abrahams in a note to clients. Nonetheless, they met the study’s criteria for non-inferiority and “likely support approvability,” he added.
No new safety signals emerged, Regeneron said.
Nearly 90% of DME patients and 77% of wet AMD patients were able to stay on either a three- or four-dose per year regimen for 48 weeks without needing further treatment. Though cross-trial comparisons can be complicated, those results topped the 51% and 45% numbers reported in tests of Vabysmo, wrote SVB Securities analyst David Risinger.
“We think these data are game changing, providing not just an answer to the Vabysmo threat, but putting the Eylea franchise back on the offensive, in a position to gain even more share,” wrote Piper Sandler analyst Christopher Raymond.
Regeneron plans to present detailed results at a future medical meeting. The results sent shares climbing nearly 20% Thursday, adding more than $12 billion in market value.