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.

Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

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Eli Lil­ly re-ups di­ver­si­ty pledge, pitch­ing in $30M to ven­ture fund for mi­nor­i­ty-owned health­care firms

The fight against racial injustice spurred by a series of high-profile shootings of Black men by police earlier this year put Big Pharma and healthcare — industries targeted for their lack of diversity — in the hot seat. Eli Lilly made an early pledge to change its ways and put more back into the community, and now it’s continuing to make good on that commitment.

Lilly will infuse $30 million into the Unseen Capital Health Fund, a venture fund looking to invest in early-stage minority-owned healthcare companies that have been historically “unseen” by the investment community, the pharma said Friday.

CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

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David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

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UP­DAT­ED: Am­gen tops cost watch­dog's price gougers list based on 'un­sup­port­ed' in­creas­es for En­brel with­out mean­ing­ful da­ta

In a top 10 ranking of the most egregious price gougers from 2019, Amgen’s Enbrel topped US cost watchdog ICER’s naughty list with “unsupported” markups that added as much as $403 million to the nation’s drug spend during that time.

Price increases for some of pharma’s most popular drugs have long been a focus of consumer ire, but the industry has argued those increases are routine and meant to cover the cost of R&D innovation. Without meaningful guidance at the state or federal level, ICER looked to connect how much a drug had progressed in the clinic compared with its increase in both wholesale and net price in 2019.

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Albert Bourla, Pfizer CEO (AP Images)

#JPM21: Al­bert Bourla pre­pares Pfiz­er to set­tle in to next phase post-Up­john, but that does­n't mean he's rul­ing out deals

One of the iconic brands in biopharma, Pfizer took a big gamble on the strength of its in-house science when it decided to offload its flagging Upjohn generics business last year. Now, a more agile Pfizer is looking to cement its identity for the future, but one thing will stay the same: M&A is still very much a part of the game plan.

With its Upjohn generics business off the books, Pfizer is looking to double down on its branded medicines with the goal of hitting 6% annual growth each year — previously unheard-of at old Pfizer — while continuing to develop its blockbuster pipeline, CEO Albert Bourla said at a JP Morgan fireside chat Tuesday.

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Stefan Oelrich (Bayer)

What can a 157-year-old phar­ma gi­ant bring to the ta­ble of cell and gene ther­a­pies? Quite a bit, Bay­er says

By now, Bayer has sketched out in bold strokes some grand plans for cell and gene therapy, cemented by big-dollar acquisitions of platform companies.

But just how do you stitch together a new unit bursting with the newest ideas within a storied pharma?

Stefan Oelrich, the head of Bayer’s pharma division, briefly lifted the curtain and spotlighted three key factors as he took the stage on a virtual media day, flanked by Emile Nuwaysir and Sheila Mikhail, the chiefs of BlueRock and AskBio, who each introduced their work in a way you’d expect from a biotech CEO.