Thumbs Up/Thumbs Down: Am­gen's si­lence is deaf­en­ing; The drug pric­ing mob is back

 

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

 

Am­gen bugs out, and they’re mak­ing every­one in bio­phar­ma look bad.

Just about every­one en­gaged in drug de­vel­op­ment loves to talk about pos­i­tive da­ta and how the most im­por­tant thing about suc­cess is al­ways the pa­tient who ben­e­fits. But when the light turns red on a set­back, the death star men­tal­i­ty dom­i­nates and ooz­ing com­pas­sion dis­ap­pears as they clamp down and hun­ker down in si­lence. I’ve seen it a thou­sand times. Am­gen, though, just took the bug-out shuf­fle to a whole new ex­treme. Their kid­ney dis­ease drug Parsabiv (etel­cal­ce­tide) was cheered through a big Phase III pro­gram, leav­ing every­one wait­ing for the in­evitable ap­proval. But this week we learned that it was re­ject­ed by the FDA. Why? Am­gen won’t say, or even hint at it. Those pa­tients they re­cruit­ed to test the drug for the good of all (and to pro­tect a key rev­enue source for Am­gen)? Well, they don’t need to know ei­ther. Frankly, the whole thing dis­gusts me. Want to know why Big Bio­phar­ma has such a seedy rep? It’s shady events like this that put it in­to per­spec­tive. To Am­gen: At least try to hold your­selves ac­count­able and ex­plain what hap­pened here hon­est­ly. Shame on Am­gen CEO Robert Brad­way and R&D di­rec­tor Sean Harp­er for al­low­ing it to hap­pen. You need to do bet­ter. Much, much bet­ter.  There are pub­lic re­spon­si­bil­i­ties in­volved in drug de­vel­op­ment, and you’re shirk­ing them.

The mob is back, will the in­dus­try ever ef­fec­tive­ly tack­le the drug pric­ing con­tro­ver­sy?

It’s quite clear now that if there’s one is­sue that is guar­an­teed to trig­ger a fu­ri­ous pub­lic out­cry, it’s price goug­ing on old prod­ucts. Mar­tin Shkre­li, Valeant, now My­lan have all proved that. Buy­ing old prod­ucts and then march­ing up the price for a wind­fall prof­it can in­cite a dig­i­tal lynch mob. And one day the pitch­forks won’t stay fo­cused on just one com­pa­ny. They’ll come for every­one, and it will not be ig­nored by the po­lit­i­cal elite. Be­cause the fact is that the ma­jors have gen­er­al­ly adopt­ed this strat­e­gy. The price in­creas­es may be a more mod­est 100% to 200%, but the con­tro­ver­sy they’re invit­ing is clear. I’ve said it be­fore, and I’ll say it again, high prices on new, sig­nif­i­cant ther­a­pies are jus­ti­fied and need­ed to sus­tain biotech and pro­vide the next wave of treat­ments. It’s time, as some­one I know al­lud­ed to re­cent­ly, for the lead­ers in the in­dus­try to stop fight­ing fires and fig­ure out how to get rid of the match­es. Or it may be your biotech house that burns next. And biotech isn’t the bad guy in this dra­ma.

 The $14B Medi­va­tion buy­out of­fers an in­dus­try-wide lift af­ter a tough H1.

It wasn’t As­traZeneca or Al­ler­gan, but Pfiz­er did fi­nal­ly get a buy­out done with the Medi­va­tion ac­qui­si­tion. Yes, they prob­a­bly paid too much. But Medi­va­tion is the kind of com­pa­ny that makes a good tar­get for a deal: Small pipeline, mar­ket­ed prod­uct, easy to bolt on with­out dis­rupt­ing big, es­tab­lished R&D groups. It was good to see that there are buy­ers will­ing to pay a pre­mi­um for buy­outs like this. Score one for biotech — and star deal­mak­er David Hung — af­ter an ug­ly first half of the year. Frankly, we all ben­e­fit by a big score like this.

Sanofi comes up short, again.

Sanofi knew it need­ed the Medi­va­tion buy­out when CEO Olivi­er Brandi­court de­cid­ed to go af­ter it as his first big growth move. It failed, which isn’t all that sur­pris­ing giv­en its track record of the past few years. Where Sanofi suc­ceeds is through its col­lab­o­ra­tions. In­ter­nal R&D re­mains a dis­ap­point­ment. As a top 10 glob­al R&D op­er­a­tion, Sanofi has to start prov­ing that it can ac­quire late-stage as­sets and get them over the goal line alone. Maybe next time they won’t be left at the al­tar. There’s a lot rid­ing on a turn­around.

What a great week for in­no­va­tion lovers in biotech.

I start­ed off the week with a piece on Ax­el Bou­chon, the point man at Bay­er for start­ing a whole port­fo­lio of am­bi­tious new biotech ven­tures. Case­bia will help de­fine that new group with its 5-year, $335 mil­lion re­search bud­get and a new home on Kendall Square. Then there were sto­ries on Kr­ish­na Yesh­want’s strat­e­gy at GV (Google Ven­tures) and a new ven­ture from some young en­tre­pre­neurs fresh out of Brown. To help wrap the week, De­nali’s ex­ec­u­tive crew took some time to re­view Chap­ter Two at the neu­rode­gen­er­a­tion start­up, which has now raised close to $350 mil­lion as it preps its first clin­i­cal tri­al. I love the for­ward-look­ing think­ing. A mix of big and lit­tle play­ers al­so of­fers some per­spec­tive on where we are now in biotech; a no-longer-young in­dus­try in con­stant need of new ideas and new ven­tures. And they keep com­ing.

Ike the mouse is ag­ing R&D’s new mas­cot.

Or, he would be if he wasn’t dead. Ike lived to the ripe old age of 1,400 days, 140 years in hu­man longevi­ty. Matt Kae­ber­lein and his team of in­ves­ti­ga­tors in Seat­tle ac­com­plished this with ra­pamycin, a dan­ger­ous drug that no one needs to think of as the spring of youth. But there are path­ways and mech­a­nisms in­volved here that will help in­spire the next round of re­search in­to hu­man longevi­ty and healthy liv­ing. I trig­gered quite a dust-up on Twit­ter with com­ments like that. The FDA has to get in line (no easy task!)  and there’s more to think about than ex­tend­ing our lives by hun­dreds of years. Get­ting 90 good years would be a great start. Make no mis­take, ag­ing R&D is on its way to be­com­ing a big field in biotech. Things take time in this world, though. So don’t look for any overnight rev­o­lu­tions.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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