Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

Ken Fra­zier Mer­ck

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

“Un­der­neath these pro­grams, un­der­neath these prod­ucts […] un­der­neath what Ken and Rob told you about the fu­ture of the com­pa­ny, there is this enor­mous­ly sta­ble plat­form and what you will hear this morn­ing is first of all our abil­i­ty to ex­e­cute clin­i­cal­ly and com­mer­cial­ly to bring these prod­ucts to life,” he said of the Mer­ck lega­cy be­fore turn­ing to Roy Baynes and Mike Nal­ly for a more de­tailed look on R&D.

Perl­mut­ter’s com­ments echo CEO Ken Fra­zier’s in­sis­tence that the team here can build a fran­chise like Keytru­da not just once or twice but re­peat­ed­ly, and come as the board is re­port­ed­ly scout­ing their suc­ces­sors.

Fra­zier ad­dressed the in­evitable tran­si­tion ques­tion dur­ing Q&A by say­ing this:

Every­thing we need to dri­ve the growth we talked about […] is al­ready in our hands and so for us it’s re­al­ly fo­cus­ing on ex­e­cut­ing in the mar­ket­place and fo­cus­ing in the de­vel­op­ment space to make sure we main­tain that lead­er­ship. As it re­lates to suc­ces­sion, the board is very much fo­cused on that is­sue. What I can say is I’m ex­treme­ly pleased by the breadth of the lead­er­ship tal­ent in the com­pa­ny.

Rob Davis Mer­ck

The take-home mes­sage: Mer­ck, now at a 52-week high mar­ket cap of $221 bil­lion, is go­ing to do just fine.

On the BD front, the com­pa­ny is stick­ing to bolt-on M&A deals and strate­gic col­lab­o­ra­tions, though CFO Rob Davis em­pha­sizes that “we have the ca­pa­bil­i­ty to pret­ty much do what we want.” He al­so promised a top-down, com­pa­ny-wide ef­fort to cre­ate a lean­er struc­ture, with an eye to be­gin cut­ting R&D ex­pens­es as a per­cent­age of sales — a stan­dard met­ric in phar­ma — be­gin­ning in 2021.

Roy Baynes Mer­ck

As much as ex­ecs hyped up the growth po­ten­tial across the en­tire pipeline, Keytru­da re­mains the foun­da­tion­al pro­gram as an un­prece­dent­ed, ex­treme­ly large prod­uct that’s “on­ly go­ing to get big­ger” ac­cord­ing to Fra­zier.

“I re­al­ly be­lieve we’re still in the ear­ly stages of the op­por­tu­ni­ty we have with Keytru­da,” added Frank Clyburn, chief com­mer­cial of­fi­cer. And if you take Lyn­parza and Lenvi­ma in­to ac­count, “we be­lieve over the next five years I want you to think about the op­por­tu­ni­ty for more than 50 ad­di­tion­al in­di­ca­tions.”

Source: Mer­ck

Click on the im­age to see the full-sized ver­sion

Frank Clyburn Mer­ck

The mas­sive net­work of tri­als cen­tered around Keytru­da has pushed over the 1,000 mark, Baynes, the head of clin­i­cal de­vel­op­ment, said, with more than 100 in ear­li­er lines of ther­a­py (be­fore or right af­ter surgery), up­wards of 600 com­bi­na­tions, and 75 reg­is­tra­tional stud­ies un­der­way.

Breast and prostate can­cers loom large among them. As monother­a­py ap­proach­es have proven weak, Mer­ck is look­ing at new com­bos and ear­li­er lines for po­ten­tial first-to-mar­ket op­por­tu­ni­ties. The PARP in­hibitor Lyn­parza might al­so play a big role in tack­ling those two mar­kets, while Mer­ck and part­ner As­traZeneca ex­plore 19 oth­er in­di­ca­tions for the drug.

Mike Nal­ly Mer­ck

As for the rest of the pipeline out­side on­col­o­gy — vac­cines and hos­pi­tal care be­ing the main pil­lars — the com­pa­ny is look­ing to both grow ex­ist­ing prod­ucts while steadi­ly push­ing a “rich pipeline.”

Chief mar­ket­ing of­fi­cer Mike Nal­ly high­light­ed two drugs in par­tic­u­lar: MK-8591, which “has a true po­ten­tial to be a game chang­er for pa­tients with HIV”; and gefapix­ant, a P2X3 and P2X2/3 re­cep­tor an­tag­o­nist first po­si­tioned for chron­ic cough.

A glob­al vi­sion will be cru­cial to achiev­ing all of this. In his pre­sen­ta­tion, CFO Davis de­scribed Mer­ck has the fastest-grow­ing multi­na­tion­al phar­ma in Chi­na, swelling 43% over the last 18 months when the av­er­age was 11% — thanks in part to prod­uct ap­provals from Keytru­da to the HPV vac­cine Gar­dasil.

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.