Aim­mune's peanut al­ler­gy treat­ment could 'change prac­tice' — with safe­ty chal­lenges and a price any­thing but peanuts

Aim­mune Ther­a­peu­tics $AIMT has de­tailed the re­sults of its Phase III study of its peanut al­ler­gy ther­a­py known as AR101 in the New Eng­land Jour­nal of Med­i­cine in an ar­ti­cle — and in­de­pen­dent as­sess­ment — that will like­ly stoke its sup­port­ers as much as the skep­tics in the field. 

And once it is mar­ket­ed, if it gets that far, you can ex­pect a se­vere blow­back on the price, con­sid­er­ing this is a nat­ur­al byprod­uct that al­so has been used at the same dose that costs vir­tu­al­ly noth­ing to make — but wouldn’t have the same stamp of ap­proval that a prod­uct OK’d by the FDA would have.

On the bright side, the ar­ti­cle in the NE­JM un­der­scored that a dust­ing of care­ful­ly con­trolled 12% de­fat­ted peanut flour used in the treat­ment did help a broad swathe of 4- to 17-year-old al­ler­gic pa­tients in the study — two thirds to be ex­act — even­tu­al­ly tol­er­ate a dose of up to 4 peanuts with­out the se­vere and pos­si­bly life threat­en­ing re­ac­tion that they could nor­mal­ly ex­pect. That was true of on­ly 4% of place­bo group in the study.

A. Wes­ley Burks

A. Wes­ley Burks, the se­nior au­thor, said that the ther­a­py “could change prac­tice for peanut al­ler­gy.” And they’re just weeks away from fil­ing for an ap­proval at the FDA, with the EMA to fol­low next year.

Looked at in terms of ef­fi­ca­cy alone, their da­ta will get you through to an ap­proval and some high fives all around. But it’s not as sim­ple as that, with some big con­cerns about the na­ture of this prod­uct and the price tag that could make it a block­buster. Crit­ics didn’t over­look the fact that 11.6% of the pa­tients on AR101 dropped out of the study due to side ef­fects, com­pared to 2.4% in the place­bo arm.

Michael Perkin

“This is not some­thing to start at home,” notes Michael Perkin, one of the lead­ing spe­cial­ists in chil­dren’s al­ler­gies, in an in­de­pen­dent re­view of the NE­JM ar­ti­cle. “Ep­i­neph­rine was used by 14.0% of the par­tic­i­pants in the ac­tive-drug group as a re­sult of re­ac­tions to treat­ment. The longer-term side ef­fects of sus­tained con­sump­tion of an al­ler­gen to which the body has pro­duced IgE an­ti­bod­ies re­main un­known. Cur­rent think­ing has fo­cused on eosinophilic dis­ease, such as eosinophilic esophagi­tis, but sur­veil­lance and fol­low-up will be cru­cial.”

“The po­ten­tial mar­ket for these prod­ucts is be­lieved to be bil­lions of dol­lars,” adds Perkin. “It is per­haps salu­tary to con­sid­er that in the study con­duct­ed by the Cam­bridge group (which used a less pre­cise but equiv­a­lent 12% de­fat­ted peanut flour), chil­dren un­der­went de­sen­si­ti­za­tion with a bag of peanut flour cost­ing peanuts.”

The de­tails, and even the crit­i­cism, won’t help Paris-based DBV Tech­nolo­gies make its case for their ri­val al­ler­gy treat­ment, which failed a Phase III tri­al but is still bound to reg­u­la­tors.

Just days ago Nestlé Health Sci­ence upped its bet on Aim­mune Ther­a­peu­tics by $98 mil­lion, with its now $276 mil­lion stake in the com­pa­ny giv­ing it 19% of the biotech’s eq­ui­ty.

Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

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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Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

It’s a race to the Phase III finish line now for the 2 leading mRNA vaccines in the pipeline for Covid-19.

BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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