Com­pared to avoid­ance, Aim­mune's peanut al­ler­gy treat­ment an im­prove­ment over DB­V's prod­uct — ICER

Peanut al­ler­gy treat­ments from spar­ring drug­mak­ers — Aim­mune and DBV Tech­nolo­gies — are in­cre­men­tal­ly ben­e­fi­cial, but their long-term cost-ef­fec­tive­ness will be de­ter­mined by the price at which they are even­tu­al­ly mar­ket­ed, ICER said in a draft re­port pub­lished on Tues­day, which con­clud­ed us­ing as­sumed prices that the lat­ter’s prod­uct, Vi­askin Peanut, was far less of an im­prove­ment over peanut avoid­ance, com­pared to AR101.

Akin to NICE in the UK, ICER is an in­de­pen­dent body that an­a­lyzes the cost-ef­fec­tive­ness of drugs and oth­er med­ical ser­vices in the Unit­ed States. Un­like NICE, though, ICER is not gov­ern­ment-af­fil­i­at­ed, but its de­ter­mi­na­tions are in­creas­ing­ly be­com­ing in­flu­en­tial with pay­ers.

For now, peanut al­ler­gies are man­aged by avoid­ance, but the threat of ac­ci­den­tal ex­po­sure can­not be nul­li­fied. Aim­mune’s AR101 and DBV’s Vi­askin Peanut are set to be the pi­o­neer­ing peanut al­ler­gy treat­ments ap­proved by the FDA, but there is “sig­nif­i­cant un­cer­tain­ty about the long-term risks and ben­e­fits” for both ther­a­pies, as each has been stud­ied in place­bo-con­trolled one-year clin­i­cal tri­als, ICER said, not­ing that da­ta from the ex­ten­sion tri­als are sparse.

An FDA ap­proval 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.

Aim­mune $AIMT ef­fec­tive­ly leapfrogged DBV $DB­VT 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.

Since nei­ther ther­a­py has been ap­proved yet, ICER con­duct­ed its analy­ses us­ing price as­sump­tions, based on an­a­lyst mod­els, which project AR101 will cost be­tween $5,000 and $10,000 for the first six months of use, and $300 to $400 per month af­ter and that Vi­askin Peanut will cost more than $6,000 for a year’s sup­ply.

Based on these es­ti­mates, ICER as­sumed a place­hold­er cost for AR101 at $350 per month ($6,595 for months 1-6 in­clud­ing clin­i­cal vis­its for dose es­ca­la­tion; $4,200 per year there­after). For Vi­askin Peanut, the in­sti­tute as­sumed a place­hold­er cost of $6,500 per year. That works out to AR101 cost­ing $84,000 over a life­time, and Vi­askin Peanut cost­ing $56,000 over a life­times, ICER said.

“Rel­a­tive to our AR101 pric­ing as­sump­tions based on our con­ver­sa­tions with Aim­mune, we had con­ser­v­a­tive­ly as­sumed the low­er end of the com­pa­ny’s com­mu­ni­cat­ed pric­ing range of $5,000 for the up-dos­ing phase and $5,000 per year there­after ($415/month) – so $7,500 for the first year. All-in, the first year costs ICER us­es for its analy­sis are high­er by about 16%,” Stifel’s Derek Archi­la wrote in a note.

For Vi­askin Peanut, ICER’s as­sumed an­nu­al cost of ther­a­py is 20% high­er than Stifel’s $5,000 as­sump­tion, he added.

ICER based its cost-ef­fec­tive­ness cal­cu­la­tions 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 0.63 in­cre­men­tal QALYs, while treat­ment with Vi­askin Peanut came up rel­a­tive­ly short, re­sult­ing in 0.22 in­cre­men­tal QALYs — when 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.

While in­for­ma­tive, the re­port is not con­clu­sive, part­ly be­cause a one-year time­frame may not re­flect the en­tire ben­e­fit of a ther­a­py that pro­vides in­creas­ing ef­fi­ca­cy over time such as AR101 or Vi­askin peanut, and no qual­i­ty of life da­ta (good or bad) was fac­tored in­to this analy­sis, Archi­la said.

ICER did ac­knowl­edge 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.”

ICER’s as­sess­ment is “pre­ma­ture” and the in­sti­tute’s frame­work does not take in­to ac­count the bur­den care­givers car­ry, in terms of mak­ing ther­a­peu­tic de­ci­sions or mak­ing out-of-pock­et costs for treat­ment, a DBV spokesper­son wrote in an email to End­points News.

“We dis­agree with ICER on many as­pects of its over­all method­ol­o­gy, as well as the tim­ing of this re­port. Be­cause the ICER mod­el re­lies on health eco­nom­ic mea­sure­ments…for which there are no pub­lished da­ta/ev­i­dence for peanut-al­ler­gic pa­tients [as there are no ap­proved FDA-ap­proved treat­ments], we be­lieve the draft re­port find­ings were dri­ven by in­ac­cu­rate clin­i­cal out­comes and cost in­puts…We be­lieve the lack of FDA-ap­proved ther­a­peu­tic op­tions for peanut al­ler­gy and as­so­ci­at­ed ab­sence of health state util­i­ty and long-term treat­ment da­ta, com­bined with pa­tient het­ero­gene­ity, pre­cludes an ac­cu­rate and re­li­able cost-ef­fec­tive­ness as­sess­ment, in­clud­ing by ICER.”

End­points has al­so con­tact­ed Aim­mune for com­ment.


Im­age: Shut­ter­stock

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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Boehringer In­gel­heim ties the knot with Num­ab on new an­ti­bod­ies; Ca­balet­ta inks pact with Ar­ti­san

→ Switzerland’s Numab Therapeutics has added Boehringer Ingelheim to its roster of collaborators. And they will start with two projects aiming at developing new drugs for difficult-to-treat lung and gastrointestinal cancers and patients with geographic atrophy. “Numab’s technology platform fits well with our internal antibody discovery and engineering capabilities and will enhance our efforts to deliver transformative antibody-based therapeutics to patients,” said Paige Mahaney, an SVP at Boehringer Ingelheim.

David Hallal

AlloVir tests how much an an­tivi­ral biotech can reap in a pan­dem­ic stock mar­ket

The pandemic stock market has proven fruitful for virtually any type of biotech. Now a 7-year-old cell therapy startup will see how much it can yield for a company that specializes in fighting viruses.

AlloVir, a company that until 2019 largely lived off grant money, has filed for a $100 million IPO to back its line of off-the-shelf, virus-fighting T cells. Although in normal circumstances, $100 million could be a solid return for a biotech that got its first major round of funding only last year, we’ll have to wait to see how much the company ultimately earns. As Covid-19 has sent investor money scurrying to almost anyone in drug development, every single biotech to go public this year has prized above their midpoint or upsized their offering, according to Renaissance Capital, sometimes dramatically so.

Take­da’s post-merg­er deals con­tin­ue as OTC sub­sidiary is pre­pared for sale

Takeda has been burdened under mountains of debt since acquiring Shire for $62 billion 18 months ago, but one of the company’s biggest moves yet to relieve the stress could be on the horizon.

The Japanese pharma is preparing a bidding war for its $3.7 billion over-the-counter subsidiary, a sale that would dwarf previous spinoffs. Any transaction would be the latest in what’s been a long string of sell-offs, as Takeda marches unflinchingly toward its ultimate goal of shedding $10 billion in assets.

In­vestors give ail­ing Unum a lease on life and a whole new suite of ex­per­i­men­tal can­cer drugs

Investors, it seems, are willing to give Unum Therapeutics one last shot — or at least one last shot to a company of that name.

The ailing cancer biotech, beset by a series of clinical holds and multiple failed lead programs, announced today that they’ve acquired Kiq LLC and that investors are putting in $104 million to advance Kiq’s pipeline of kinase inhibitors. Unum shareholders will now own only 16.2% of the company and CEO Chuck Wilson indicated that the cell therapies the biotech has worked on since its founding may be on their way out, saying Unum will “explore strategic options” for those products.