Bris­tol-My­ers makes a de­ci­sive move to fix its R&D prob­lem; Let's not un­der­es­ti­mate Trump now

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

Bris­tol-My­ers steps up to fix a de­railed R&D or­ga­ni­za­tion. But ques­tions linger

No big bio­phar­ma has suf­fered a big­ger set­back than Bris­tol-My­ers Squibb’s back­ward march on Op­di­vo. Over­reach­ing in lung can­cer, they have for­feit­ed their lead role on one of the biggest new drug fran­chis­es in the in­dus­try. In a busi­ness where not screw­ing up is half the game, they blew it. And this is a com­pa­ny that has had one of the most in­no­v­a­tive and pro­duc­tive R&D op­er­a­tions of the past decade.

Com­pa­ny ex­ecs have been keep­ing a stiff up­per lip about it all, but heads had to roll, and chief sci­en­tist Fran­cis Cuss was a nat­ur­al choice for the chop­ping block. In his place, the com­pa­ny turned to a very dis­tin­guished gen­tle­man on the board, Tom Lynch, a Mass Gen­er­al vet­er­an.

Lynch has had an ex­tra­or­di­nary ca­reer, but turn­ing to a board mem­ber with such a long re­sume has to raise ques­tions about just how long he plans to stay and what he hopes to ac­com­plish. Bris­tol-My­ers now has to fight back at a time the R&D group — a top-15 group that con­sumes $5 bil­lion a year – is un­der­go­ing a re­vamp.

Wrestling with Bris­tol-My­ers’ R&D chal­lenges is a mighty task. The stakes are enor­mous. Let’s hope Lynch is up to the task.

This is not the time to stop tak­ing Trump se­ri­ous­ly

Bloomberg’s Max Nisen to­day writes that “Trump’s tweets are still dan­ger­ous, but it ap­pears in­creas­ing­ly like­ly he’s just cry­ing wolf.” And he fol­lows up with the lat­est, more mut­ed re­sponse by in­vestors to Trump’s lat­est war cry for drug price re­form.

It’s too ear­ly, though, to pooh-pooh what Trump has in mind. Rep. Eli­jah Cum­mings came out of his meet­ing with Trump this week clear­ly pumped that the pres­i­dent had cheered his bill for a fed­er­al for­mu­la­ry for Medicare. And Trump ap­par­ent­ly sug­gest­ed that he was fine with diss­ing many Re­pub­li­cans in the process, look­ing to sat­is­fy the base with an open­ly pop­ulist po­si­tion.

These next few months are go­ing to be ab­solute­ly crit­i­cal for bio­phar­ma. I was chat­ting with a biotech CEO a few weeks ago who was clear­ly steamed at the idea that the full force of the fed­er­al gov­ern­ment could be brought to bear on drug prices. A for­mu­la­ry, threat­en­ing to leave out drugs in or­der to lever­age low­er prices, was un­ac­cept­able to him.

At the time, I dis­count­ed the no­tion that Medicare price ne­go­ti­a­tions would be dra­con­ian, es­pe­cial­ly with a Re­pub­li­can in the White House. But Trump is push­ing hard to de­liv­er on a fre­quent­ly re­peat­ed promise now. And dra­con­ian is ex­act­ly how he sounds. Com­bined with a promise to dereg­u­late drug de­vel­op­ment, what­ev­er that means, Trump does not in­spire con­fi­dence or clar­i­ty in what’s in store for the in­dus­try.

It’s time to tread care­ful­ly on these is­sues. Writ­ing Trump off now as a blowhard would be a ter­ri­ble mis­cal­cu­la­tion. In Wash­ing­ton DC, the dev­il is al­ways in the de­tails of new leg­is­la­tion. Let’s pay care­ful at­ten­tion to what law­mak­ers are ham­mer­ing out.

A new re­view com­par­ing R&D bud­gets to US pre­mi­um pric­ing stokes an un­end­ing ar­gu­ment

Some­times, you know ear­ly on when you’ve hit a nerve with a sto­ry. And so it was with my piece on a new study about the dis­con­nect be­tween R&D bud­gets and the pre­mi­um prices that Amer­i­cans pay for drugs.

An­a­lysts at Memo­r­i­al Sloan-Ket­ter­ing took a look at just how big US pre­mi­ums are com­pared to the rest of the de­vel­oped world and de­ter­mined that they more than paid for the top com­pa­nies’ to­tal R&D bud­gets. Then they called the in­dus­try out for its pi­ous protests about need­ing high prices to pay for in­no­va­tion.

Quite a few of the peo­ple in the Twit­ter­verse ob­ject­ed, for the ob­vi­ous rea­son that these com­pa­nies are in busi­ness to make a prof­it, not cov­er costs. And then both sides were off and run­ning, snip­ing back and forth about what drug com­pa­nies are about.

The de­bate — a healthy one, I think — sim­ply un­der­scores the in­her­ent con­flict with­in Big Phar­ma. These com­pa­nies con­stant­ly in­sist to the pub­lic that their chief mis­sion is to help pa­tients. And then they turn around and cheer big prof­its to Wall Street.

As one an­a­lyst point­ed out to me in an email ex­change, Ap­ple has a much big­ger prof­it mar­gin than Big Phar­mas. Ap­ple sells a prod­uct, though, which con­sumers want but don’t have to have, so no one chal­lenges what they charge or its re­la­tion to the R&D bud­get. The bet­ter the drug, the more it’s ab­solute­ly re­quired, and the more it costs.

At some point the bio­phar­ma busi­ness may fig­ure out how to re­solve the con­flict, but I doubt it.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.