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.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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