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US FDA: Aimmune peanut allergy drug effective in children; adult benefit unclear

The U.S. Food and Drug Administration noted the potential of Aimmune Therapeutics Inc.'s AR101 to reduce peanut-related allergic reactions, though the agency posed some questions in its review of the company's application for approval ahead of a Sept. 13 meeting.

In particular, the regulator pointed to a lack of robust evidence on the product's efficacy in adults and an apparent increase in allergic reactions during increased dosing of AR101.

Aimmune's stock was trading up approximately 11.7% to $24.65 as of 12:34 p.m. ET.

"The FDA did strike a relatively positive tone," Stifel analyst Derek Archila wrote, following the briefing documents' release on Sept. 11.

AR101, expected to receive a decision from the FDA in late January 2020, will be evaluated by the FDA's Allergenic Products Advisory Committee at the Sept. 13 meeting. The experimental drug, which saw delays due to the U.S. government's shutdown earlier this year, was granted both fast track and breakthrough therapy designations.

Archila forecast a 90% probability of approval in his Sept. 11 note.

AR101 is an oral immunotherapy, a peanut protein powder applied to food and used to slowly build up and maintain a certain level of peanut tolerance. AR101 would be administered with initial dose escalation under the supervision of a qualified healthcare professional in a healthcare setting, and be increased in dosing every two weeks until 300 milligrams is reached. Patients would then be treated with 300 milligrams of daily maintenance treatment, Aimmune said in its briefing document.

According to Aimmune's late-stage trial, treatment with AR101 allowed peanut-allergic patients to tolerate a certain amount of peanut protein. However, during dose escalation, AR101-treated patients saw more frequent systemic allergic reactions, defined by effects on at least two body systems, than placebo-treated participants, the FDA noted in its report.

Unmet need in peanut allergies

Peanut allergy accounts for the most fatal food-induced allergic reaction, called anaphylaxis, in the U.S. Peanut allergy affects approximately 2% of children in the U.S., most of whom do not outgrow their allergies. Allergic responses can be triggered by less than one-sixth of a peanut kernel, or less than 50 milligrams of peanut protein, Aimmune said.

Patients' quality of life can be affected due to fear of accidental exposure and the burden of avoiding allergenic foods. Accidental exposures occur at an annual incidence of between 12.4% and 14.3% in peanut-allergic children, the FDA reported.

The current standard of care for peanut allergy is prevention via a strict avoidance diet, and accidental exposure is treated with immediate injection of epinephrine for more severe reactions or with antihistamines for milder symptoms.

Aimmune said peanut allergy prevalence is increasing, denoting a greater need for an immunotherapy that could desensitize patients.

Clinical data

Aimmune's application for approval is based on two phase 3 studies, ARC003 and ARC007, and follow-on extension studies providing longer-term safety and efficacy evidence.

The studies were composed mainly of participants aged 4 to 17, although 55 adult participants were evaluated in ARC003.

While children treated with AR101 met main and secondary goals in ARC003, demonstrating tolerance of peanut protein upwards of 1,000 milligrams, adult patients did not see a significant treatment difference, the FDA pointed out. Adult participants also posted more substantial discontinuation rates, as 54% stopped treatment due to protocol-mandated dose titration failures.

However, Aimmune said 85% of adults who did complete treatment met the primary goal of peanut protein tolerance with no more than mild symptoms, compared with 15% of placebo-treated adults.

Overall, during oral food challenges that tested tolerance of single doses of 3, 10, 30, 100, 300, 600 and then 1,000 milligrams of peanut protein powder given at 20- to 30-minute intervals, AR101-treated participants reported less severe symptoms compared to the placebo group. Self-reported incidences of accidental peanut exposure during trials were also less severe in AR101-treated patients than in placebo recipients, Aimmune said.

In addition, patients' immunologic changes seemed to demonstrate an ongoing process of desensitization.

But during dose escalation portions of AR101 treatment, which totaled one year in the ARC003 study, patients receiving AR101 saw more frequent adverse events than placebo recipients. Overall, 89.1% of AR101-treated patients experienced treatment-related adverse events versus 57.5% of placebo patients.

Adverse events decreased over time once patients proceeded to the maintenance phase of treatment, the FDA noted. However, 13 serious adverse events were reported, 10 of which stemmed from AR101. Common adverse events included abdominal pain, throat irritation, itching, vomiting, cough and sneezing.

According to Aimmune's document, "mild and moderate symptoms of systemic allergic reaction are common" during treatment with any form of immunotherapy.

During trials, 12 participants were diagnosed with eosinophilic esophagitis, a chronic immune-mediated inflammatory disease of the esophagus. All 12 were taking AR101, and 10 cases were considered treatment-related.

Aimmune said whether these cases were exacerbated or caused by AR101, or associated with symptoms prior to treatment remains unclear.

Aimmune said it will provide a medication guide for AR101 treatment if the drug is approved. In addition, availability of auto-injectable epinephrine and training on proper use will be required for patients receiving AR101, Aimmune said.

A long-term safety study, ARC008, is underway and will conclude at three years of AR101 maintenance treatment.

Long-term treatment evidence was an issue brought up by the Institute for Clinical and Economic Review, the U.S. pricing watchdog. Aimmune emphasized that the ARC008 trial provides an opportunity to collect data after approval.

"Combining strict avoidance practices with AR101 therapy to increase tolerance to peanut protein will reduce the incidence and severity of allergic reactions ... and provide patients with peanut allergy a sense of control over the unpredictable risk, ultimately allowing them and their families to improve the quality of their lives," Aimmune concluded.