Regeneron's Skin-Clearing Drug Hits Goals in Late Trialsby
Dupilumab reaches targets for effectiveness, disease severity
About 1.6 million U.S. patients with unmet treatment need
Regeneron Pharmaceuticals Inc. and Sanofi said their drug to treat a serious skin condition met its goals in two final-stage trials, clearing the path toward regulatory approval in a potentially lucrative field.
More than a third of patients with a severe type of eczema known as atopic dermatitis saw their skin clear or come close in each trial of the drug, called dupilumab, the companies said Friday in a statement. Ten percent or fewer patients on placebo got the same results. Regeneron rose 5.5 percent to $380.28 at 10:34 a.m. in New York.
“It’s very exciting to have something else in our arsenal for patients who have been recalcitrant to so many things,” Joseph Merola, a dermatologist at Brigham and Women’s Hospital in Boston who worked on part of the trial, said by telephone. He said that while the drug worked well, he expects it initially will be used mostly in more severe cases.
Regeneron is betting on the drug to help it break into the territory of treatments aimed at the immune system and expand beyond the flagship eye-disease drug Eylea, which last year accounted for 65 percent of company sales. Dupilumab works by blocking two cellular signaling proteins that trigger inflammation, a key cause of symptoms in diseases like atopic dermatitis. Regeneron has also started a final-stage trial looking at the drug for patients whose asthma isn’t treated well by current therapies.
There are about 1.6 million patients with moderate to severe atopic dermatitis in the U.S. who don’t respond well to existing treatments, according to the companies. The skin condition is sometimes debilitating, and analysts from Cowen & Co. and RBC Capital Markets have described the market as lucrative.
Sanofi’s management said dupilumab could be a blockbuster drug with multiple uses, according to a note to clients by Seamus Fernandez of Leerink Partners. The top-selling specialty drug in the U.S. is AbbVie Inc.’s Humira, which also treats inflammatory conditions, according to prescription benefit manager Express Scripts Holding Co.
The two trials, called SOLO 1 and SOLO 2, followed a combined 1,379 patients with moderate to severe atopic dermatitis who were randomly divided into groups getting different doses of dupilumab or a placebo. In SOLO 1, 37 percent of patients who got weekly drug injections and 38 percent who got them biweekly had clear or almost clear skin by the end of the trial. In SOLO 2, 36 percent of patients got the same result with weekly or biweekly treatments.
Patients on the highest dose of the drug saw their eczema decrease on a severity index by 72 percent in SOLO 1 and 69 percent in SOLO 2. Those results compared with improvements of 38 percent and 31 percent in corresponding placebo groups. Both the skin-clearing and severity results exceeded the minimum expected by Geoffrey Porges, an analyst at Leerink who wrote about the studies in a note to clients on March 18.
Atopic dermatitis heavily affects young children, according to Brigham’s Merola, and they weren’t studied in the trials. Sixty percent of those who get the disease have it in their first year of life, he said, and parents of young children may be averse to accepting the drug until it had been shown to be safe in that population. Regeneron is now conducting an early study of the drug in children and hopes to move into final-stage trials, Bola Akinlade, Regeneron’s head of clinical sciences, said in a phone interview.
Patients in the trial who got dupilumab had few serious side effects, the companies said. Seven percent to 12 percent of people on the drug got pink eye, compared to 2 percent in the placebo group. Except for injection-site reactions, side effects didn’t track based on the amount of drug taken, Akinlade said.
The U.S. Food and Drug Administration has granted dupilumab breakthrough therapy status, which may give treatments priority review. The companies plan to file for regulatory approval in the third quarter of 2016.