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.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.