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

On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Cor­re­vio is putting it­self up on the auc­tion block af­ter FDA re­view pan­el points to an­oth­er re­jec­tion

For 13 years, the Canadian biotech Correvio tried to get the FDA to accept a heart drug since abandoned by Merck and Astellas. Yesterday, the agency’s outside experts voted 11-2 against approval, all but assuring another rejection for the atrial fibrillation compound vernakalant.

And today Correvio announced that Correvio may soon be no more. The company said it is looking to sell itself as its stock plummets into penny-stock territory $CORV and its potential moneymaker sputters once more.

Psilocybin mushrooms (via The Denver Post)

In a key step for psy­che­del­ic re­search, mag­ic mush­room com­pound clears first clin­i­cal safe­ty hur­dle

Exasperated with the often-ineffective existing slate of antidepressants, COMPASS Pathways set up shop in London 2016 — and made a beeline for psilocybin, the psychoactive ingredient in magic mushrooms.

On Wednesday, the startup said its man-made version of the chemical — which is illegal across geographies in its natural fungi form — had been well-tolerated in an early-stage, placebo-controlled trial in 89 healthy volunteers.

Al­pham­ab On­col­o­gy rounds out HKEX's sec­ond biotech IPO year with $230M raise and high lo­cal in­ter­est

Alphamab Oncology has inspired a surge of local interest in what will likely be the Hong Kong Stock Exchange’s last biotech run of the year, pricing its IPO on the high end of the range and raising over $230 million (HK$1.83 billion).

After rejigging the offering structure and making up to 50% available for enthusiastic local investors, the biotech sold 179.4 million shares at $1.31 (HK$10.2) and saw its stock rise to $1.77 ($13.8) on the first day of trading.

For sale: Long-act­ing PhI­II GLP-1 di­a­betes drug that’s way be­hind ri­vals, now spurned by Sanofi

Almost exactly 4 years ago Sanofi came to the bargaining table with South Korea’s Hanmi bearing $434 million dollars in cash and offering about $4 billion in milestones to in-license their once-weekly GLP-1 injectable. The pact was intended to revive their ailing diabetes division. Instead, it turned into a very expensive grave to mark the end of Sanofi’s R&D ambitions in the field.

Sanofi CEO Paul Hudson used efpeglenatide’s demise — while committing to paying hundreds of millions of more dollars to push it through 5 late-stage studies — as a marker of the company’s determination to stay focused on first and best-in-class drugs.

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Parkin­son's trans­plants emerge as stem cell pi­o­neer Jeanne Lor­ing joins R&D race

Jeanne Loring hadn’t studied Parkinson’s in 22 years when she got an email from a local neurologist.

The neurologist, Melissa Houser, didn’t know Loring had ever published on the disease. She was just looking for a stem cell researcher who might hear her out. 

“I think I was just picked out of a hat,” Loring told Endpoints News. 

At a meeting in Loring’s Scripps Research office, Houser and a Parkinson’s nurse practitioner, Sherrie Gould, asked her why there was so much research done in stem cell transplants for other neurodegenerative diseases but not Parkinson’s. They wanted to know if she would work on one. 

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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