The 10 most pop­u­lar re­ports in End­points News — so far

A lit­tle more than 5 months ago, Ar­salan Arif and I launched the main edi­tion of End­points News. We hit the ground run­ning and gun­ning for a dai­ly in­dus­try re­port that would pro­vide some re­al in­sights on the deals, da­ta and dra­ma flow­ing around the bio­phar­ma world every day.

We’re all about the lat­est news in con­text, guid­ed by 13+ years of dai­ly cov­er­age. And we re­cent­ly passed is­sue #100, with about a quar­ter mil­lion words of search­able con­tent.

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We’re us­ing the Thanks­giv­ing break to high­light what you, our read­ers, have vot­ed as the most pop­u­lar con­tent we’ve put up in that time. The top 10 links, ranked by web traf­fic, starts with a scoop and ends with two sto­ries about back-to-back dis­as­ters that oc­curred ear­li­er this week and im­me­di­ate­ly went se­mi-vi­ral.

So with­out more ado, here’s the most clicked sto­ries of 2016, so far.

1. Scoop: No­var­tis dis­bands its pi­o­neer­ing cell and gene ther­a­py unit

We got a tip ear­ly on Au­gust 31 that No­var­tis was dis­solv­ing its 400-per­son cell and stem cell unit and lay­ing off a bunch of the staffers. When I first heard it, I thought it had to be wrong. Why now? Just as the first cell ther­a­pies in CAR-T were head­ing for reg­u­la­tors? And with com­pe­ti­tion breath­ing down its neck? It didn’t make any sense. The com­pa­ny, though, con­firmed the sto­ry, and our news break trig­gered a seis­mic re­ac­tion in the in­dus­try, which al­so wasn’t ex­pect­ing it. The moral of this sto­ry is that we are all ears. If you send us a good tip, we’ll check it out. (You can se­cure­ly send us things us­ing PGP.)

This is one of sev­er­al big changes we’ve tracked at No­var­tis in re­cent months. Al­so among our top sto­ries: No­var­tis un­veils a new glob­al R&D struc­ture, cre­at­ing cen­ters in Cam­bridge, MA and Basel. And there was this: No­var­tis’ re­treat on CAR-T in­cludes ax­ing most of its se­nior ex­ecs on the team.

 

2. Where the mon­ey is: The top 100(+) VCs in­vest­ing in the US

There are few in­dus­tries where da­ta is more high­ly re­gard­ed than in biotech. So it’s no sur­prise that a de­tailed list of the top VC out­fits op­er­at­ing in the US — ranked by the num­bers — was a hit just as we start­ed the new on­line news source. And it con­tin­ues to at­tract a steady stream of traf­fic from an in­dus­try which is fu­eled by bil­lions in ven­ture cash each year. Cal­i­for­nia, with a big Bay Area clus­ter and a siz­able hub in San Diego, clear­ly at­tract­ed most of the cash. And Boston/Cam­bridge is clear­ly the sec­ond big US hub. Keep an eye on this site: We’ll be back in 2017 with more de­tailed num­bers.

 

3. The 15 top R&D spenders in the glob­al bio­phar­ma busi­ness

More num­bers, this time break­ing our the top 15 re­search spenders in the in­dus­try. This is the life blood of in­no­va­tion, as we know it. And the con­tin­ued in­vest­ment in R&D is at the heart of the grow­ing de­bate over drug pric­ing in Amer­i­ca. The top 15 spenders ac­count for the li­on’s share of re­search spend­ing in bio­phar­ma, and I’ve been fol­low­ing the chang­ing struc­tures and strate­gies be­hind each of these com­pa­nies for more than a decade. Each year we see more R&D group over­hauls, but un­der­ly­ing every­thing has been a big com­mit­ment to seek­ing out and part­ner­ing with ex­ter­nal col­lab­o­ra­tors. That’s been re­ward­ed with mixed re­sults.

 

4. Top 10 pipeline blowups, set­backs and sna­fus in H1 2016

It’s al­ways a bit of a strug­gle to come up with my lat­est set of top pipeline blowups. Not be­cause they’re rare. Quite the op­po­site. Lim­it­ing my­self to 10 can be dif­fi­cult. At the top of this first list for End­points is Clo­vis, which has nev­er ful­ly ex­plained the mys­te­ri­ous switch-up in the da­ta it pre­sent­ed on rocile­tinib. The com­pa­ny stopped re­spond­ing to my queries months ago as it hun­kered down to weath­er a tsuna­mi of law­suits and a fed­er­al in­ves­ti­ga­tion. Mean­while, it’s been fo­cused on its PARP in­hibitor ru­ca­parib, which looks to be squar­ing off against some tough com­pe­ti­tion. Some of the top 10 are the un­for­tu­nate con­se­quence of a sim­ple bi­o­log­ic mis­take. Some are cau­tion­ary tales that of­fer a glimpse of how not to go about drug de­vel­op­ment. Some­times, that can be hard to dis­tin­guish. But it’s al­ways in­ter­est­ing.

 

5. Mer­ck trig­gers a new round of lay­offs in R&D re­or­ga­ni­za­tion, push­ing more jobs in­to Cam­bridge, San Fran­cis­co

One of the biggest sin­gle trends over the past few years has been the mi­gra­tion of bio­phar­ma R&D in­to the big hubs. And Mer­ck demon­strat­ed its com­mit­ment to that hub strat­e­gy back in Ju­ly as it set off a new round of lay­offs while mov­ing more of its re­search op­er­a­tions in­to Boston and the Bay Area, the two gi­ant clus­ters in the US. In just the last few months we’ve seen new over­hauls — both big and small — at No­var­tis, As­traZeneca, Pfiz­er and Glax­o­SmithK­line as well, con­tin­u­ing a years-long process. It may nev­er end. Con­stant change is the rule in R&D, whether you push it or it push­es you.

 

6.  Two more pa­tients die as Juno’s lead CAR-T turns lethal again; tri­al halt­ed

This is not your typ­i­cal pipeline cat­a­stro­phe. Juno al­ready ex­pe­ri­enced an ex­tra­or­di­nar­i­ly short clin­i­cal hold for its lead CAR-T back in the sum­mer, fol­low­ing the deaths of 4 pa­tients in two stud­ies. At the time, they man­aged to con­vince the FDA that they were quite cer­tain that drop­ping one of the drugs used to con­di­tion pa­tients would solve that nasty neu­ro­tox­i­c­i­ty is­sue. The FDA agreed in a mat­ter of days, and now two more pa­tients have been killed. I sus­pect that Juno’s next move will be to drop JCAR015 and in­stant­ly shift fo­cus to JCAR017, their next top pipeline hope­ful. But we need a much, much bet­ter ac­count­ing of what hap­pened here, from the com­pa­ny as well as the FDA. A call with an­a­lysts by the com­pa­ny and a long-wind­ed ‘no com­ment’ from the FDA isn’t go­ing to cut it.

 

7. Don­ald Trump sends a vague­ly word­ed love let­ter to bio­phar­ma as stock ral­ly con­tin­ues

I’ll ad­mit that we don’t have a whole lot of de­tailed in­sight in­to what Don­ald Trump is think­ing about when it comes to bio­phar­ma. Maybe that’s one rea­son why this sto­ry about his en­thu­si­as­tic, though com­plete­ly un­ex­plained, po­si­tion sup­port­ing a re­formed FDA and faster drug ap­provals at­tract­ed so much at­ten­tion. We want to know what he’s think­ing, but for now we’ll have to set­tle for what we can get. Based on sev­er­al sur­veys we did dur­ing the cam­paign, it’s clear that the big ma­jor­i­ty of ex­ecs in the in­dus­try were op­posed to a Trump pres­i­den­cy. But a stock ral­ly af­ter the elec­tion and an up­beat at­ti­tude about a sup­port­ive pol­i­cy — with­out any new hints about Medicare ne­go­ti­a­tions on drug prices or reim­por­ta­tion — has start­ed to turn the tide in his fa­vor. Bio­phar­ma may nev­er love Trump, but the in­dus­try is start­ing to like what lit­tle it’s seen so far.

 

8. Al­ny­lam shares crater af­ter tri­al deaths force in­ves­ti­ga­tors to scrap PhI­II RNAi drug

Any­time a promi­nent com­pa­ny gets hit with an un­ex­pect­ed dis­as­ter, like this one at Al­ny­lam, it tends to raise big­ger ques­tions. Did the fail­ure of one drug have im­pli­ca­tions for the pipeline? How about ri­vals? How did it get this far? Al­ny­lam tried might­i­ly to put the col­lapse of its late-stage ef­fort in­to a more com­fort­ing con­text, but the de­ci­sion to halt the pro­gram clear­ly rat­tled in­vestors. Now any new hint of trou­ble is like­ly to get the spot­light of care­ful at­ten­tion, along with a bliz­zard of mixed com­ments on Twit­ter.

 

9. It’s over: Eli Lil­ly shares tank af­ter its huge gam­ble on Alzheimer’s drug solanezum­ab ends in fail­ure

Give Eli Lil­ly points for try­ing. But be sure to deduct a few for re­fus­ing to ad­mit de­feat. When you run a huge Phase III study and your drug flops, maybe it’s time to walk away. For Eli Lil­ly, though, there was enough clin­i­cal ev­i­dence of suc­cess to war­rant an­oth­er mon­u­men­tal­ly ex­pen­sive ef­fort. CEO John Lech­leit­er has al­ready timed his de­par­ture, so you can’t say he re­signed over this mess. But this is one black eye that will cause more ex­ecs to be more care­ful about how they spend their in­vestors’ mon­ey. So­la at best may have turned in­to a weak but mar­ketable drug. Its loss shouldn’t be mourned. We’re bet­ter off look­ing at bet­ter al­ter­na­tives in the pipeline. And maybe now com­pa­nies will stop tout­ing their prospects to the press. Pa­tients don’t need to be giv­en false hope. This is a ter­ri­ble, tough dis­ease. And for the past decade, all in­ves­ti­ga­tors have ex­pe­ri­enced when it comes to ac­tu­al­ly slow­ing this dis­ease is de­feat.

 

10. Clin­ton cam­paign staff: “We have start­ed the war with phar­ma!!”

Cred­it where it’s due. End­points News‘ Shehla Shakoor re­al­ly got in­to search­ing the Wik­iLeaks col­lec­tion of emails from the Clin­ton cam­paign, and she found a re­al eye-open­er in this one. It turns out that Clin­ton’s care­ful­ly aimed barb at Mar­tin Shkre­li was part of a care­ful­ly cal­i­brat­ed plan to spot­light her pledge to rein in drug prices and bad ac­tors like Shkre­li. And se­nior staffers were ready to do a high-five as the mar­kets made quite a to-do over the Twit­ter af­fair. In­ter­est­ing­ly, ab­solute­ly noth­ing came out of the hub­bub. No one reined in Tur­ing or its big price hike. Shkre­li was in­dict­ed on un­re­lat­ed charges and Clin­ton lost. But the po­lit­i­cal war with phar­ma goes on.


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Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.