ARS Pharmaceuticals plans to resubmit its NDA for Neffy, a needle-free epinephrine nasal spray, following successful clinical trials, with hopes of gaining approval later in the year.

ARS Pharmaceuticals Inc announced clinical data on Neffy, an epinephrine nasal spray designed to treat anaphylaxis and chronic urticaria, showcasing a 100% response rate in pediatric anaphylaxis induced by oral food challenges.

The data will be presented during the 2024 American Academy of Allergy, Asthma and Immunology. The meeting will be held Feb 23-26 in Washington, DC.

One late-breaking presentation reports efficacy outcomes for the first time with neffy in pediatric patients who developed anaphylaxis symptoms following oral food challenge. The efficacy data demonstrated that 100% of patients responded to a single dose of neffy with a 16-minute median time to complete resolution of anaphylaxis symptoms.

“I believe the robust and rapid clinical responses observed with neffy during anaphylaxis due to oral food challenge demonstrate that neffy is at least as effective as injection,” says Motohiro Ebisawa, MD, PhD, director of Clinical Research Center for Allergy and Rheumatology at Sagamihara National Hospital, past-president of the World Allergy Organization, and principal investigator of the study, in a release. “My allergy colleagues and I are eagerly awaiting regulatory approval of neffy, so that we can prescribe this needle-free and easy-to-carry treatment option to our severe allergy patients, many of whom do not carry, do not administer or delay use of epinephrine autoinjectors.”

A second presentation details the statistically significant and clinically meaningful reductions in urticaria symptoms achieved by neffy across all endpoints in its randomized, controlled proof-of-concept study.

“Epinephrine is the first-line treatment and only medication for anaphylaxis that reduces risk of hospitalization and death,” says Sarina Tanimoto, MD, PhD, chief medical officer and co-founder of ARS Pharma, in a release. “The data from these studies to be presented at AAAAI in anaphylaxis and urticaria patients demonstrated that neffy worked as well and as fast as delivering epinephrine by injection, but without the need for a needle and its accompanying limitations.”

Additional neffy data to be presented include pharmacokinetic/ pharmacodynamic (PK/PD) effects in pediatric subjects, improved temperature stability versus injection products, improvements in patient quality of life, and increased device carriage, use and time to use rates.

Neffy Efficacy Study in Oral Food Challenge-Induced Anaphylaxis

This study assessed the safety and efficacy of a single dose of neffy given to pediatric patients with anaphylaxis symptoms induced by an oral food challenge.

Two mg (30 kg+ body weight) 1 mg (15-30 kg body weight) doses of neffy were administered when patients exhibited gastrointestinal, respiratory, or circulatory symptoms that were grade two or higher on the three-grade scale in the Severity Classification of Organ Symptoms by the Japanese Society of Allergology Anaphylaxis Guidelines 2022.

A total of 15 patients aged 6 to 17 were enrolled; six subjects (15-30 kg body weight) were dosed with 1 mg neffy, and nine subjects (30 kg+ body weight) were dosed with 2 mg neffy.

Treatment guidelines indicate a second dose of epinephrine should be given if clinical response is not observed within the first 15 minutes.

  • 100% of the patients responded to the single dose of neffy.
  • After dosing neffy, the median time to complete resolution of anaphylaxis symptoms was 16 minutes.
  • One of 15 patients (6.7%) experienced a biphasic reaction two hours and 45 minutes following complete resolution of symptoms with the single dose of neffy and required additional epinephrine treatment. For epinephrine injection products, biphasic reactions are reported to occur at a frequency of 12.8% in children with food-induced anaphylaxis (Gupta et al. JACI: In Practice, 2021).
  • For epinephrine injection products, two or more doses of epinephrine are required for 10.8% of oral food challenges treated with epinephrine (n = 2,436 events) (Patel et al. JACI, 2021).
  • Clinical data in this neffy trial is therefore in line with historical clinical response data for injection products.

The trial demonstrated safety results similar to the previously reported safety profile of neffy and approved epinephrine products with adverse events reported in seven subjects dosed with neffy, all of which were mild or moderate and most of which resolved quickly.

ARS Pharma remains on track to announce topline results from its repeat dose study of neffy under nasal allergen challenge conditions in the first quarter of 2024 and resubmit its New Drug Application mid-first half of 2024 with an expected Prescription Drug User Fee Act action date and US launch, if approved, in the mid-second half of 2024.