The 10 most pop­u­lar sto­ries in End­points News — so far in 2017

John Car­roll

Since Ar­salan Arif and I ramped up the main edi­tion of End­points News 16 months ago, we’ve pub­lished more than a mil­lion words on bio­phar­ma R&D.

Tol­stoy, eat your heart out.

This year we’ve in­creased web traf­fic, track­ing close to an av­er­age of 20,000 read­ers per day — with more than 22,000 email sub­scribers — which is con­sid­er­ably high­er than where we start­ed in the mid­dle of 2016.

So what are bio­phar­ma peo­ple read­ing in End­points?

Every Black Fri­day we mark the hol­i­day with our top 10 list of our most pop­u­lar re­ports (here’s last year’s ver­sion). Read­ers vot­ed with their fin­gers, and here are their top 10 sto­ries.

1 Mer­ck was hit hard by a vi­cious cy­ber­at­tack yes­ter­day. Here is what we know.

Pub­lish­er Ar­salan Arif wrote the best-read re­port of the year at End­points News af­ter hear­ing about a glob­al cy­ber­at­tack which blast­ed Mer­ck, caus­ing chaos at the phar­ma gi­ant. Over time, we would hear about hun­dreds of mil­lions in dam­ages, which are still rack­ing up at the com­pa­ny. Mer­ck says it did what it could to pro­tect it­self, but this virus hit hard. Chances are, cy­ber­se­cu­ri­ty has tak­en on a whole new mean­ing at Mer­ck.

2 Top 20 block­buster drugs in the late-stage pipeline — Eval­u­atePhar­ma

If there’s one thing 15 years in the trench­es of biotech re­port­ing has taught me, it’s that in­dus­try read­ers like some in­sights on the big drugs in the late-stage pipeline. It tells us who’s do­ing the best work, which com­pa­nies have the best prospects and where the big bets are be­ing made. There’s a lot more to this than peak sales, but the drugs picked by Eval­u­atePhar­ma rep­re­sent a ver­i­ta­ble trea­sure trove of block­busters worth po­ten­tial­ly more than $30 bil­lion. Check it out.

3 The top 15 spenders in the glob­al drug R&D busi­ness: 2017

Those com­pa­nies that dom­i­nate the late-stage pipeline are al­so typ­i­cal­ly the ones that foot the li­ons’s share of the bill for drug de­vel­op­ment. Last year, the big bill due for the top 15 com­pa­nies’ re­search bud­gets re­sult­ed in a wimpy pay­out, with a big drop in new drug ap­provals, but that’s be­ing sand­wiched be­tween two sol­id years of suc­cess­es at the FDA. I’ve been fol­low­ing this R&D num­ber for a long while now. And while the fo­cus may change and peo­ple are hired and fired, the en­gine of drug de­vel­op­ment over­all re­mains in high gear. It’s al­so true that high gear is not good enough, and that longterm pro­duc­tiv­i­ty is­sue con­tin­ues to haunt the in­dus­try’s fu­ture.

4 British bil­lion­aire Jim Mel­lon and high-pro­file part­ners roll the dice on an an­ti-ag­ing up­start

We are on the cusp of a tidal shift in biotech R&D, with more and more com­pa­nies look­ing to de­vel­op ther­a­pies that can add bet­ter life to your years, and grad­u­al­ly more years to your life. British bil­lion­aire Jim Mel­lon wrote a book on the top­ic, and now he and some close col­leagues are div­ing in di­rect­ly to raise cash and start work. These trends take years to play out, so don’t look for any overnight rev­o­lu­tions in drug R&D. Still, the wheels are turn­ing, and read­ers are pay­ing close at­ten­tion.

5 In stag­ger­ing set­back, tox­ic re­ac­tion kills Cel­lec­tis’ first CAR-T pa­tient, forc­ing tri­al halt

CAR-T has a safe­ty is­sue, as any­one fa­mil­iar with the field knows all too well. Cel­lec­tis is out to make one of these new can­cer ther­a­pies — now craft­ed from pa­tient cells — so they can be made in bulk and tak­en down from the shelf as need­ed. But an ear­ly death helped raise the red flag for this new kind of CAR-T as well. The FDA lift­ed this hold re­cent­ly, al­low­ing Cel­lec­tis to go back in­to the clin­ic, but on­ly af­ter the French biotech came up with a host of new safe­ty mea­sures. Rev­o­lu­tions are nev­er easy. Or safe. This was a time­ly re­minder that those fac­tors re­al­ly nev­er change.

6 The scoop: Marathon’s R&D pro­gram for Duchenne MD drug like­ly came in at a bar­gain base­ment price

This is one of my fa­vorite sto­ries of the year. I’m glad it was one of yours, too. Marathon Phar­ma was try­ing to pull a fast one when it slipped through an ap­pli­ca­tion on an old cheap steroid as an or­phan ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy. Quite a few par­ents pre­ferred de­flaza­cort over a re­al­ly cheap gener­ic al­ter­na­tive, pay­ing around $1,000 a year to get it from a Lon­don sup­pli­er. Ac­cord­ing to Marathon, it had to charge $84,000 a year for its new ther­a­py — point­ing to the big clin­i­cal pro­gram need­ed for the ap­proval. We blew that the­o­ry sky high with some pro­fes­sion­al es­ti­mates of what it re­al­ly cost, and how the com­pa­ny looked to make a killing. The sto­ry blew up, law­mak­ers got in­volved and Marathon de­cid­ed to sell it off and take the mon­ey and run. Now PTC is the proud own­er, and its new price runs along a slid­ing scale based on weight. It’s not re­al­ly cheap­er at all, but the con­tro­ver­sy has blown over — for now.

7 Ter­ri­ble op­tics: As­traZeneca shares take a hit fol­low­ing re­port that CEO So­ri­ot is mov­ing to Te­va

Months lat­er, this head­line still sur­pris­es me. And I wrote it. Pas­cal So­ri­ot had been fight­ing for a turn­around at As­traZeneca for the past 5 years. To re­port­ed­ly be in­ter­est­ed in tak­ing the Te­va job would be an­oth­er way of say­ing ‘so long, suck­ers’ to every­one he man­aged at As­traZeneca. So­ri­ot even­tu­al­ly made it clear that he wasn’t leav­ing As­traZeneca, and even­tu­al­ly Te­va an­nounced it had signed a new CEO. But So­ri­ot nev­er de­nied he dis­cussed the open­ing. And this sto­ry al­ways re­mains in the back of every­one’s heads as As­traZeneca makes a halt­ing jour­ney back from the val­ley of death it’s been in.

8 King can­cer: The top 10 ther­a­peu­tic ar­eas in bio­phar­ma R&D

There’s no doubt that 2017 is the year of can­cer R&D. New projects are peak­ing, break­throughs are gain­ing land­mark ap­provals and more mon­ey than ever has flowed in­to the field. As a re­sult, as we point­ed out in this break­down of R&D projects by ther­a­peu­tic field, there’s now can­cer R&D and every­thing else that is go­ing on. We’ve fol­lowed up with more da­ta points that un­der­score this over­whelm­ing trend. And you can ex­pect to hear a lot more about this as com­pa­nies com­pete over a fi­nite num­ber of pa­tients and mar­ket share. Some trends have con­se­quences, and this is one of them.

9 Al­ny­lam achieves break­through RNAi suc­cess as PhI­II patisir­an study hits all goals, shares soar

This re­al­ly was a re­mark­able year for ma­jor break­throughs. Al­ny­lam’s suc­cess with the Phase III for patisir­an sets up its first ever FDA ap­pli­ca­tion, af­ter 15 years of hard work. This com­pa­ny stayed true to the RNAi faith while big phar­ma com­pa­nies bailed and doubts grew that any­one could ever make a com­mer­cial suc­cess out of it. But the crew at Alnlyam made it hap­pen. That kind of suc­cess de­serves to be rec­og­nized, and the in­dus­try clear­ly didn’t over­look the im­pli­ca­tions.

10 In a stun­ning set­back, Roche says its top can­cer drug Tecen­triq failed a key PhI­II study

Iron­i­cal­ly, I’m writ­ing this up on the same day that Roche’s stock jumped 6%, large­ly be­cause its triple com­bo of Tecen­triq, Avastin and chemo proved ef­fec­tive in in­creas­ing pro­gres­sion-free sur­vival for first-line lung can­cer. Back in May, though, its fail­ure for a Phase III blad­der can­cer tri­al, af­ter an ac­cel­er­at­ed ap­proval, un­der­scored just how frag­ile suc­cess can be in the PD-1/L1 check­point are­na. One com­pa­ny grabs the lead, the next day they blow up in the clin­ic, or a ri­val does bet­ter in its cho­sen are­na. Now sev­er­al years in­to the check­point rev­o­lu­tion, the sur­pris­es keep on com­ing with amaz­ing reg­u­lar­i­ty, dri­ving on more com­pe­ti­tion for what is rapid­ly be­com­ing a wild­ly over­crowd­ed mar­ket niche. This was just one chap­ter of a sto­ry that shows no signs of com­ing to a close — but change is com­ing.

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

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Dan O'Day (AP Images)

UP­DAT­ED: 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 acquisition, 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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Vlad Coric (Photo Credit: Andrew Venditti)

UP­DAT­ED: 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.