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.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Oc­u­lar Ther­a­peu­tix ham­mered by a PhII fail­ure in dry eye dis­ease — shares tank

Ocular Therapeutix $OCUL has had its ups and downs in the 7 years since it went public. Friday was one of those down days.

The Bedford, MA-based biotech reported that its lead experimental eye drug, OTX-CSI (cyclosporine intracanalicular insert), failed a Phase II trial for dry eye disease. And the stock experienced one of its periodic meltdowns, dropping more than 30% ahead of the bell.

The therapy flat failed the primary endpoint: increased tear production at 12 weeks as measured by the Schirmer’s Test compared to the vehicle control group. And while investigators called out an improvement from baseline in “signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert,” it wasn’t statistically significant.

Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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