Aim­mune wins ap­proval for peanut im­munother­a­py, charges 10k+

The FDA has ap­proved the first treat­ment for peanut al­ler­gy, al­though it comes with some con­tro­ver­sy.

Un­like most of the con­tro­ver­sial drugs that come be­fore the FDA, the bulk of ques­tions don’t cen­ter around this drug’s ef­fec­tive­ness or side ef­fects.

Pal­forzia, de­vel­oped by Aim­mune, has been shown to pre­vent the most se­vere re­ac­tions from ex­po­sure to small amounts of peanuts. In a Phase III tri­al, chil­dren giv­en es­ca­lat­ing dos­es of the drug were then giv­en 600 mg peanut pro­tein — about 2 peanuts — had a 67% chance of avoid­ing a se­vere re­ac­tion. For the place­bo pa­tients, the fig­ure was 4%.

Jayson Dal­las Aim­mune

“The goal — step 1 in terms of ther­a­py — is to be able to treat a child to the point you can say, if that child goes in­to the com­mu­ni­ty and ac­ci­den­tal­ly gets ex­posed to a peanut some­where, they’re not go­ing to have a life-threat­en­ing event,” Aim­mune CEO Jayson Dal­las told End­points News in an in­ter­view at the JP Mor­gan con­fer­ence ear­li­er this month.

“They may have a no re­ac­tion at all, they may have a lit­tle tin­gling, but they’re not go­ing to be at risk.”

The FDA ad­vi­so­ry com­mit­tee point­ed out that this wasn’t ide­al — you can’t eat a peanut but­ter sand­wich and a sig­nif­i­cant num­ber of the pa­tients couldn’t even eat the 2 peanuts  — and not­ed the ad­verse re­ac­tions some had to the pill. But large­ly, they agreed it was a sub­stan­tial step for­ward for a con­di­tion that has seen vir­tu­al­ly no in­no­va­tion.

Crit­ics, though, have point­ed to what’s in the drug: peanuts. Pal­forzia is a form of im­munother­a­py, by which pro­longed ex­po­sure to an al­ler­gen — in this case, peanut pow­der, most­ly but not en­tire­ly equiv­a­lent to what you can find on a su­per­mar­ket shelf — is used to build up re­sis­tance. Some doc­tors have long done this with­out any pre­scribed drug and to some suc­cess. In a 2018 study, re­searchers start­ed 270 pa­tients on 1/2,500th of a peanut and worked 80% of them up to 12 peanuts.

Pal­forzia, op­po­nents say, would take this off-la­bel treat­ment and charge thou­sands of dol­lars for it. An­a­lysts had pro­ject­ed a lit­tle over $4,000 for the price and over $1 bil­lion in peak sales. The ac­tu­al an­nu­al price, Aim­mune an­nounced to­day, will be $10,680.

The In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view al­so point­ed to some of the ad­verse ef­fects pa­tients ex­pe­ri­enced from re­ceiv­ing the drug, and said the long-term ben­e­fit wasn’t clear enough to jus­ti­fy the cost and those ad­verse ef­fects.

Aim­mune has said those risks are to be ex­pect­ed when ex­pos­ing pa­tients to an al­ler­gen. They ar­gued that while doc­tors can pre­scribe pa­tients off-la­bel peanut pow­der, they are ex­posed to law­suits for any re­ac­tion a kid could have to the pow­der. By bring­ing the drug in­to the med­ical sys­tem, they say they give an op­tion for doc­tors to pre­scribe pa­tients with­out mak­ing them­selves li­able.

The drug is ap­proved for chil­dren ages 4 to 17. Aim­mune said they plan to hold pre­sen­ta­tions and work­shops to train 1,300 al­ler­gists on how to ad­min­is­ter the drug.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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No­var­tis to pay near­ly $178M in law­suit over BRAF drug — and will be on the hook for roy­al­ty

After a four-year battle over a cancer drug patent, Novartis has been ordered by a California judge to pay a Daiichi Sankyo subsidiary $177.8 million.

Plexxikon filed a lawsuit against the pharma giant in 2017, alledging that Tafinlar, a rival to its melanoma drug Zelboraf that was brought to market in collaboration with Roche, has stepped on its intellectual property. The jury ruled in its favor, adding that the infringement is in fact willful.

Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

Bio­gen de­fends slow roll­out of new Alzheimer's drug, crit­i­cizes neg­a­tive me­dia at­ten­tion

As Biogen execs bemoaned the negative media coverage around Aduhelm’s approval a month ago, the biotech isn’t gaining much traction yet in using its new drug, largely due to a lack of insurance coverage, according to an earnings call Thursday.

Management indicated that of the nearly 900 sites that were prepped and ready following Aduhelm’s approval, 325 of those, or about 35%, have completed a positive pharmacy and therapeutics (P&T) review or won’t require one. The review is a step some hospitals or health systems take prior to using a new drug. Some major sites, however, have said they won’t participate.