ARS Pharmaceuticals reports neffy meets primary endpoints in Phase 2 study

ARS Pharmaceuticals announced efficacy results in its phase 2 inpatient chronic spontaneous urticaria study with neffy, an investigational new drug. The trial met its primary endpoints with both 1 mg and 2 mg neffy demonstrating statistically significant and clinically meaningful changes from baseline in itch, hives, urticaria and erythema scores as early as 5 minutes after dosing. Urticaria is a skin disorder that causes itchy hives and/or angioedema; 50% of chronic urticaria cases1 are non-responsive to first-line antihistamine therapy. These data for neffy are being presented in an oral presentation today, February 26, at 1:10 pm ET at the 2024 American Academy of Allergy, Asthma and Immunology in Washington, D.C. in Room 207B of the Convention Center. The EPI-U01 study was a randomized, placebo-controlled, cross-over study evaluating the safety and efficacy of epinephrine nasal spray in patients with chronic spontaneous urticaria treated with chronic medications who were still experiencing flares. This oral presentation includes data analysis from 18 adult patients as of the cut-off date who enrolled in this study and returned to the clinical site while experiencing a flare, where they were randomized to receive either a single treatment of 1 mg or 2 mg neffy or placebo in a crossover design. All subjects had flares with pruritus and hives scores greater than or equal to 2 on a 3 point severity scale, despite all patients having been treated with antihistamines or Xolair. There was no meaningful difference in efficacy on patient-reported itch severity score, patient-reported hives severity score, investigator-related extent of urticaria, or investigator-related erythema score between 1 mg and 2 mg neffy doses, indicating that the 1 mg dose may be sufficient to activate the beta-2 adrenergic receptors responsible for stopping the mast cell degranulation and allergic mediator release that leads to an urticaria flare. neffy was well-tolerated, with adverse events reported in 8 subjects, which were all mild or moderate in severity. The most common adverse event reported was nasal discomfort in 5 subjects. There were no serious adverse events.