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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

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Vas Narasimhan, Novartis CEO (Gian Ehrenzeller/Keystone via AP)

No­var­tis pulls the plug on UK-based car­dio­vas­cu­lar study

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.