As­traZeneca chief is bet­ting the com­pa­ny’s fu­ture on a sin­gle roll of the dice

Bioreg­num
The view from End­points
As­traZeneca CEO Pas­cal So­ri­ot

Near­ly five years in­to his stint at the helm of As­traZeneca, CEO Pas­cal So­ri­ot has now reached his year of liv­ing dan­ger­ous­ly.

This morn­ing the phar­ma chief out­lined a steadi­ly de­clin­ing rate of rev­enue. Core EPS is ex­pect­ed to slide in the low to mid teens as gener­ics con­tin­ue to chop away at their Crestor fran­chise. All their old block­busters are be­ing dis­en­fran­chised, in a man­ner of speak­ing.

So­ri­ot, not known to wa­ver in times of a cri­sis, wasn’t about to break down now. He an­nounced: “It is an ex­cit­ing time as we rapid­ly ap­proach the in­flec­tion point for our an­tic­i­pat­ed re­turn to long-term growth, built on the sol­id foun­da­tions of a sci­ence-led pipeline.”

In­vestors weren’t very ex­cit­ed by the num­bers, though, send­ing the com­pa­ny’s shares down by close to 3% this morn­ing.

Af­ter a whole slate of clin­i­cal set­backs in 2016, the fi­nan­cial ero­sion at As­traZeneca has fo­cused an un­wa­ver­ing spot­light on the com­pa­ny’s com­bi­na­tion study for the PD-L1 check­point dur­val­um­ab and treme­li­mum­ab, a CT­LA-4, dubbed MYS­TIC.

A late­com­er to the check­point in­hi­bi­tion field, As­traZeneca is mak­ing a high-risk at­tempt at cut­ting in­to the line of heavy­weights com­pet­ing on lung can­cer, look­ing for ev­i­dence that they have a com­bo that can ri­val the ad­vanc­ing for­tunes of Mer­ck’s Keytru­da/chemo com­bi­na­tion, now un­der re­view.

As­traZeneca re­cent­ly tweaked MYS­TIC, adding months to the time­line as the phar­ma com­pa­ny awaits pro­gres­sion-free sur­vival da­ta this year and over­all sur­vival da­ta in 2018. And the move was wide­ly viewed as sig­nal­ing some un­der­ly­ing con­cerns about how the fi­nal da­ta read­out will look.

De­lays of any kind, though, are poi­son to As­traZeneca, as faster com­pa­nies con­tin­ue to cap­i­tal­ize on the first wave of check­points and more com­pa­nies mus­cle in along­side them. Pfiz­er, al­lied with Mer­ck KGaA, al­so has a check­point now un­der re­view at the FDA. Mer­ck, Bris­tol-My­ers and Roche are al­ready well in­to the mar­ket, with their own fol­low-up plans. And Bris­tol-My­ers’ de­ci­sion not to pur­sue an ac­cel­er­at­ed ap­proval for Op­di­vo (PD-1)/Yer­voy (CT­LA-4) in lung can­cer raised even more doubts about the ap­proach at As­traZeneca.

The stakes for As­traZeneca keep get­ting high­er, cre­at­ing a re­mark­able high-wire act for a Big Phar­ma play­er.

Not on­ly does As­traZeneca have to gain an ap­proval now, the com­pa­ny al­so has to be rec­og­nized as a leader in the field, po­si­tioned to grab bil­lions of dol­lars in new sales. Just falling short of ex­pec­ta­tions will be enough to quash hopes for the near-term turn­around that As­traZeneca needs.

“Rarely has a sin­gle tri­al re­sult been so cru­cial to a com­pa­ny the size of As­traZeneca,” said Mick Coop­er of Trin­i­ty Delta.

And how.

Rev­enue in 2016 hit $23 bil­lion in 2016, down from $24.7 bil­lion. But So­ri­ot promised $45 bil­lion in rev­enue by 2023, now just six years away. The com­pa­ny has al­ready start­ed back­ing away from that num­ber, blam­ing cur­ren­cy val­u­a­tions. But even their new goals will rapid­ly evap­o­rate with­out a big score on the I/O front.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Dan O'Day (AP Images)

A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisitions, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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Vlad Coric (Photo Credit: Andrew Venditti)

Bio­haven scores CGRP OK for acute mi­graine — can the com­mer­cial team catch up with Al­ler­gan on its de­but?

Seven years after spinning out of Yale, Biohaven has entered the ranks of commercial-stage biotechs.

The FDA handed down an OK for its CGRP drug, rimegepant, as an acute treatment. Dubbed Nurtec, the orally dissolving pill will join Allergan’s (soon to be AbbVie’s) Ubrelvy and Lilly’s Reyvow on the market amid a new wave of migraine therapies reshaping the disease space.

In a pivotal Phase III trial, Nurtec hit the co-primary endpoints on pain freedom and freedom from most bothersome symptoms at two hours post dose, proving superior to placebo.