Aimmune Therapeutics $AIMT has detailed the results of its Phase III study of its peanut allergy therapy known as AR101 in the New England Journal of Medicine in an article — and independent assessment — that will likely stoke its supporters as much as the skeptics in the field.
And once it is marketed, if it gets that far, you can expect a severe blowback on the price, considering this is a natural byproduct that also has been used at the same dose that costs virtually nothing to make — but wouldn’t have the same stamp of approval that a product OK’d by the FDA would have.
On the bright side, the article in the NEJM underscored that a dusting of carefully controlled 12% defatted peanut flour used in the treatment did help a broad swathe of 4- to 17-year-old allergic patients in the study — two thirds to be exact — eventually tolerate a dose of up to 4 peanuts without the severe and possibly life threatening reaction that they could normally expect. That was true of only 4% of placebo group in the study.
A. Wesley Burks, the senior author, said that the therapy “could change practice for peanut allergy.” And they’re just weeks away from filing for an approval at the FDA, with the EMA to follow next year.
Looked at in terms of efficacy alone, their data will get you through to an approval and some high fives all around. But it’s not as simple as that, with some big concerns about the nature of this product and the price tag that could make it a blockbuster. Critics didn’t overlook the fact that 11.6% of the patients on AR101 dropped out of the study due to side effects, compared to 2.4% in the placebo arm.
“This is not something to start at home,” notes Michael Perkin, one of the leading specialists in children’s allergies, in an independent review of the NEJM article. “Epinephrine was used by 14.0% of the participants in the active-drug group as a result of reactions to treatment. The longer-term side effects of sustained consumption of an allergen to which the body has produced IgE antibodies remain unknown. Current thinking has focused on eosinophilic disease, such as eosinophilic esophagitis, but surveillance and follow-up will be crucial.”
“The potential market for these products is believed to be billions of dollars,” adds Perkin. “It is perhaps salutary to consider that in the study conducted by the Cambridge group (which used a less precise but equivalent 12% defatted peanut flour), children underwent desensitization with a bag of peanut flour costing peanuts.”
The details, and even the criticism, won’t help Paris-based DBV Technologies make its case for their rival allergy treatment, which failed a Phase III trial but is still bound to regulators.
Just days ago Nestlé Health Science upped its bet on Aimmune Therapeutics by $98 million, with its now $276 million stake in the company giving it 19% of the biotech’s equity.
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