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

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

George Scangos / Credit: Cornell University

ARCH, Soft­Bank-backed Vir Biotech­nol­o­gy un­der­whelms with $143 mil­lion IPO

George Scangos went back to Wall Street, and came back 700 million pennies short.

Scangos’ vaunted startup Vir Biotechnology raised $143 million in an IPO they hoped would earn $150 million. Shares were priced at $20, the low-end of the $20-$22 target.

Launched with backing from ARCH Venture’s Robert Nelsen, Masayoshi Son’s SoftBank Vision Fund, and the Bill & Melinda Gates Foundation, the infectious disease startup was one of a new wave of well-resourced biotechs that emerged with deep enough coffers to pursue a full R&D line rather than slowly build their case by picking off a single lead program.

Ex-Ab­b­Vie part­ner Prin­cip­ia posts en­cour­ag­ing PhII re­sults for its BTK-in­hibitor

Months after their breakup with high-profile partner AbbVie, Principia announced positive preliminary results from the second half of a Phase II trial on their lead drug.

The San Francisco biotech announced data from part B of its Phase II open-label trial testing the BTK inhibitor PRN1008 on patients with pemphigus vulgaris, a rare autoimmune disease affecting the skin and mucous membranes. Of 15 enrolled patients, 6 achieved complete responses and 4 remain on the therapy.

Roche vice-chair: Let's re­pair the dam­age that short-term prof­it dri­ve has done to the plan­et

In his latest push for environmental advocacy, the vice chairman of Roche’s board of directors has told a group of business executives that “short-term profit maximization has destroyed the planet, environmentally and socially.”

Andre Hoffman
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In re­ver­sal, NICE backs Rubra­ca af­ter Clo­vis agrees to a price cut

NICE has changed its mind, agreeing to cautiously endorse Clovis Oncology’s Rubraca after the drugmaker agreed to cut its price — about two months after the UK cost-effectiveness agency’s initial rejection.

Rubraca, known chemically as rucaparib, is approved for use in the EU as monotherapy for the maintenance treatment of adult patients with high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who have relapsed after platinum-based chemotherapy.

Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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