Aim­mune's peanut al­ler­gy treat­ment could 'change prac­tice' — with safe­ty chal­lenges and a price any­thing but peanuts

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

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.

Michael Perkin

“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.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



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