A new drug for patients who have a hard time controlling their asthma could be nearing an FDA review.
Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi this morning are touting results this morning from a 1,902-patient Phase 3 study called Liberty Asthma Quest. In it, their injectable antibody drug dupilumab (Dupixent), already approved for adults with certain forms of eczema, has shown an ability to reduce severe asthma attacks and increase lung function in adolescent and adult patients when taken with standard treatments. As a result, Regeneron and Sanofi plan to file for approval of dupilumab in asthma in the U.S. by the end of the year.
It should be noted that two companies only unveiled top-line results on Monday. The full details will be disclosed at a future medical meeting, and they will give a better indication of the magnitude of dupilumab’s benefit. A second Phase 3 study testing the drug’s ability to help asthma patients cut their use of oral corticosteroids should produce results before the end of 2017 as well. But for now, here are the headline numbers from Liberty Asthma Quest:
—A high (300 mg) dose of dupilumab, given once every two weeks to patients whose disease couldn’t be controlled by standard asthma treatments, helped reduce patients’ lung function by 9 percent after 12 weeks of treatment and lower the number of asthma attacks by 46 percent after 52 weeks. Both results, the two main goals of the study, were statistically significant.
—A lower (200 mg) dose of dupilumab performed “generally comparable” to the higher dose in affecting asthma attacks and lung function, according to the statement, but Regeneron didn’t provide specifics.
—The drug performed better in patients with higher counts of eosinophils—a type of white blood cell more commonly seen in patients who have a harder time controlling their asthma, Regeneron said. Patients with 150 eosinophilic cells per microliter of blood or greater saw a 60 percent reduction in severe asthma attacks and an 11 percent increase in lung function. Those numbers jumped to a 67 percent reduction in attacks and an 18 percent boost in lung function in people with 300 eosinophilic cells/microliter. Roughly half the patients in Liberty Asthma Quest had more than 300 eosinophilic cells/microliter or more, and 70 percent in the trial had at least 150 eosinophilic cells/microliter.
—Side effects were comparable between dupilumab and placebo. Injection site reactions were the most common side effects, affecting 17 percent of patients on Regeneron’s drug compared to 8 percent on placebo.
Despite the results, Regeneron shares fell about 3.45 percent in pre-market trading on Monday. Leerink Partners analyst Geoffrey Porges wrote in a research note that while the data should support a broad label for dupilumab that differentiates it from rival drugs, the benefit seen in Liberty Asthma Quest wasn’t as strong as the one dupilumab produced in a mid-stage study, the results of which were published in the Lancet in 2016.
About 10 to 20 percent of the people with asthma—some 1 million people in the U.S., Regeneron chief scientific officer George Yancopoulos said in the statement—can’t control their disease with existing inhalable treatments that help open up the airways. Regeneron is trying to show dupilumab is an option for these patients, who might otherwise have to make more expensive trips to the hospital (the company didn’t disclose its impact on hospital visits this morning). But the study is important for other reasons as well.
Regeneron is best known for aflibercept (Eylea), a drug for a form of age-related vision loss that is its primary revenue driver. The drug generated $919 million in the last quarter alone, more than 62 percent of the company’s total revenue. But as part of a wide-ranging alliance with Sanofi, the company has developed several other additional antibody drugs now either on the market or close to it—like dupilumab, the cholesterol lowering PCSK9 blocker alirocumab (Praluent), and the arthritis drug sarilumab (Kevzara). A cancer immunotherapy drug, cemiplimab, is also advancing forward in clinical testing.
Dupilumab, however, is seen as the drug with perhaps the broadest commercial potential. So far the drug, approved in March for adults with atopic dermatitis, a form of eczema, generated $29 million in sales over its first quarter on the market. But analysts have been predicting much more for dupilumab over the years because of its potential as a treatment not just for eczema, but other diseases as well, among them asthma. Regeneron believes the drug can help treat a range of allergic diseases, and has trials underway for eosinophilic esophagitis, children with atopic dermatitis, children with asthma, and nasal polyps. Liberty Asthma Quest is a key proof point in its dupilumab hypothesis.