New peanut al­ler­gy ther­a­pies aren’t as ef­fec­tive — or near­ly as in­ex­pen­sive — as ab­sti­nence, says ICER

While peanut al­ler­gy ther­a­pies from Aim­mune and DBV Tech­nolo­gies are locked in a race to the fin­ish line, in­flu­en­tial cost-ef­fec­tive­ness watch­dog ICER has de­ter­mined that nei­ther ther­a­py of­fers su­pe­ri­or net health ben­e­fit com­pared to strict peanut avoid­ance, in a fi­nal re­port pub­lished on Wednes­day.

Tra­di­tion­al­ly, peanut al­ler­gies are man­aged by avoid­ance, but the threat of ac­ci­den­tal ex­po­sure can­not be nul­li­fied.

Some physi­cians have al­so been dos­ing pa­tients with peanut pow­der oral­ly, al­beit off la­bel. Aim­mune’s $AIMT AR101 and DBV’s $DB­VT Vi­askin Peanut are set to be the pi­o­neer­ing peanut al­ler­gy treat­ments ap­proved by the FDA. AR101 is ef­fec­tive­ly peanut flour, which must be mixed in­to pud­ding, ap­ple­sauce, or oth­er foods. Dos­ing is es­ca­lat­ed grad­u­al­ly, and the ther­a­py must be con­tin­ued dai­ly to main­tain de­sen­si­ti­za­tion. Vi­askin Peanut is a patch ap­plied dai­ly to the up­per back that de­liv­ers peanut anti­gen to de­sen­si­tize the pa­tient. The patch must be worn for longer pe­ri­ods as time pass­es and used every day to main­tain de­sen­si­ti­za­tion.

While the ICER pan­el ac­knowl­edged that de­sen­si­ti­za­tion as a sur­ro­gate out­come is promis­ing, they were look­ing for da­ta to demon­strate that de­sen­si­ti­za­tion is linked to im­proved qual­i­ty of life and re­duced re­ac­tions to ac­ci­den­tal ex­po­sure to peanuts.

Based on sur­ro­gate out­comes — oral food chal­lenges — AR101 ap­pears to be more ef­fec­tive than Vi­askin Peanut, but car­ries more ad­verse ef­fects. In ad­di­tion, in stud­ies of AR101, Vi­askin Peanut, and off-la­bel ther­a­py (OIT), ep­i­neph­rine use and sys­temic al­ler­gic re­ac­tions in­creased, ICER found.

Over­all, the body of ev­i­dence is not strong enough to sug­gest that AR101 or Vi­askin Peanut of­fer a su­pe­ri­or net health ben­e­fit ver­sus strict peanut avoid­ance, the pan­el said, adding that there is al­so un­cer­tain­ty about the long-term ef­fects of ei­ther ther­a­py.

“There is hope that the rates of sys­temic al­ler­gic re­ac­tions, ep­i­neph­rine use, and re­ac­tions to ac­ci­den­tal ex­po­sure will de­crease with con­tin­ued ther­a­py, but this re­mains to be demon­strat­ed. The po­ten­tial need for life­long ther­a­py rais­es is­sues about long-term ad­her­ence to treat­ment, par­tic­u­lar­ly dur­ing ado­les­cence and young adult­hood,” ICER said.

ICER did ac­knowl­edge, as it did in its draft re­port, that one of the lim­i­ta­tions of its analy­sis is that it as­sumed the util­i­ty of the two peanut al­ler­gy ther­a­pies on the ba­sis of ex­ist­ing da­ta on food al­ler­gies, but not specif­i­cal­ly the peanut al­ler­gy pa­tient pop­u­la­tion, due to “the pauci­ty of pref­er­ence-weight­ed health-re­lat­ed qual­i­ty of life es­ti­mates in food al­ler­gy pa­tients and their care­givers.”

“With both AR101 and Vi­askin, pa­tients still need to spend the rest of their life avoid­ing peanuts and still wor­ried about ac­ci­den­tal ex­po­sure and won­der­ing if their peanut treat­ment will pro­tect them from ana­phy­lax­is,” said James Wallen, who owns AMW Con­sul­tants, which is fo­cused on as­sist­ing for­eign biotech­nol­o­gy com­pa­nies un­der­stand the US al­ler­gy im­munother­a­py mar­ket.

“With OIT (off-la­bel oral im­munother­a­py), the pa­tient is eat­ing peanuts dai­ly so they no longer have that fear of avoid­ing ac­ci­den­tal ex­po­sure. When you are deal­ing with chil­dren (since both ther­a­pies on­ly looked at or “suc­ceed­ed” in kids), the QOL (qual­i­ty of life) dif­fer­ences al­so have to con­sid­er the par­ent’s lev­el of com­fort with their child’s de­sen­si­ti­za­tion suc­cess. This is where OIT is com­plete­ly dif­fer­ent and vast­ly su­pe­ri­or from both AR 101 and Vi­askin in my opin­ion,” he told End­points News.

Nei­ther com­pa­ny has di­vulged its pric­ing plans, so ICER used an­a­lyst pro­jec­tions to eval­u­ate each ther­a­py’s long-term cost-ef­fec­tive­ness: AR101 at $4,200/year and Vi­askin Peanut at $6,500/year.

One of the ways ICER makes these cal­cu­la­tions is on qual­i­ty-ad­just­ed-life-years (QALYs), a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life.

Treat­ment with AR101 re­sult­ed in 0.75 in­cre­men­tal QALYs, while treat­ment with Vi­askin Peanut came up rel­a­tive­ly short, re­sult­ing in 0.26 in­cre­men­tal QALYs — when both were com­pared to no im­munother­a­py treat­ment over a life­time, ICER’s analy­sis sug­gest­ed.

“These ben­e­fits are due to im­proved sub­jec­tive qual­i­ty of life de­spite the rel­a­tive rar­i­ty with which se­ri­ous events oc­cur. The ul­ti­mate val­ue of these prod­ucts will be de­ter­mined by the prices that are set by the man­u­fac­tur­ers and their long-term ef­fec­tive­ness,” ICER con­clud­ed.

ICER’s analy­sis al­so in­di­cat­ed on­ly 41% of el­i­gi­ble pa­tients could be treat­ed with AR101 and 71% of el­i­gi­ble pa­tients could be treat­ed with Vi­askin Peanut in a giv­en year with­out ex­ceed­ing ICER’s bud­get im­pact thresh­old of $991 mil­lion.

Both Aim­mune and DBV said ICER’s con­clu­sions were pre­ma­ture.

The fi­nal re­port is gen­er­al­ly bi­ased against im­munother­a­py — and fails to cap­ture the full val­ue of AR101, Aim­mune said, un­der­scor­ing that ICER did not in­clude in its analy­sis the long-term ef­fi­ca­cy and qual­i­ty-of-life da­ta from an open-la­bel fol­low-on study, as well as clin­i­cal out­comes da­ta from the com­pa­ny’s Eu­ro­pean late-stage tri­al.

“We be­lieve this fi­nal re­port rais­es more ques­tions than it an­swers and should be viewed as an ear­ly start­ing point for fu­ture con­ver­sa­tions—not the fi­nal word—about the val­ue of AR101,” Aim­mune chief Jayson Dal­las said in a state­ment.

Mean­while, DBV voiced sim­i­lar con­cerns, sug­gest­ing it dis­agreed with ICER’s method­ol­o­gy as well as the tim­ing of the re­port.

Among oth­er points of con­tention, DBV took is­sue with ICER’s in­clu­sion of a Lancet study on oral im­munother­a­py (OIT), which did not in­clude Vi­askin Peanut clin­i­cal tri­als. “DBV has con­cerns with group­ing Vi­askin Peanut’s rat­ing along with this sys­temic OIT analy­sis,” a spokesper­son told End­points News.

Aim­mune ef­fec­tive­ly leapfrogged DBV when the lat­ter re­scind­ed an ap­pli­ca­tion to mar­ket Vi­askin Peanut patch last year in re­sponse to FDA con­cerns about the state of man­u­fac­tur­ing and qual­i­ty con­trol da­ta sub­mit­ted.

An FDA de­ci­sion for AR101 is ex­pect­ed in Jan­u­ary 2020, while DBV is ex­pect­ed to sub­mit its mar­ket­ing ap­pli­ca­tion lat­er in 2019. The so far un­tapped mar­ket is ex­pect­ed to grow to $4.5 bil­lion in 2027 glob­al­ly, ac­cord­ing to Glob­al­Da­ta.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.