Mer­ck’s glo­ri­ous vic­to­ry in lung can­cer marks a Russ­ian win­ter for Bris­tol-My­ers

Sun­day was check­point in­hibitor day at ES­MO, with the three big play­ers now in the mar­ket out­lin­ing how they’re lin­ing up in the block­buster field of lung can­cer ther­a­py. Mer­ck was left in the en­vi­able po­si­tion of dri­ving home some im­pres­sive re­sults from its clin­i­cal study while Bris­tol-My­ers Squibb had to try to put its mis­er­able out­comes in a some­what pos­i­tive con­text and a de­ter­mined Roche/Genen­tech team an­gled in on its niche.

As­traZeneca CEO Pas­cal So­ri­ot, mean­while, had to stand on the side­lines de­vout­ly wish­ing he was in the game of block­busters.

Bris­tol-My­ers Squibb $BMY made a Napoleon­ic mis­cal­cu­la­tion when it de­cid­ed to see if Op­di­vo could beat chemother­a­py as a front­line treat­ment for a broad pa­tient pop­u­la­tion suf­fer­ing from non-small cell lung can­cer. We al­ready knew from the top-line an­nounce­ment that the tri­al failed — trig­ger­ing some­thing of a Russ­ian win­ter for Bris­tol stock. Now we know that it failed re­al­ly, re­al­ly bad­ly, and in­vestors weren’t hap­py, send­ing its shares down sharply Mon­day morn­ing.

Low­er­ing the bar to pa­tients with 5% or more of their can­cer cells ex­press­ing PD-L1, Bris­tol’s Op­di­vo de­layed tu­mor pro­gres­sion 4.2 months. Chemo? 5.9 months.

“We thought Op­di­vo could beat chemother­a­py, and we have an­swered the ques­tion — for the broad pop­u­la­tion it is not enough,” Fouad Namouni, on­col­o­gy de­vel­op­ment head at Bris­tol-My­ers, told David Crow at the Fi­nan­cial Times.

That’s ac­tu­al­ly a very valu­able point and in the sci­ence world it would and should be ap­plaud­ed. But even in sub­group analy­ses, Bris­tol’s in­ves­ti­ga­tors could find new ev­i­dence of suc­cess. To in­vestors ex­pect­ing Bris­tol-My­ers to con­tin­ue their dom­i­na­tion of the sec­tor, though, it all looks like an epic mis­cal­cu­la­tion and set­back with­out a sin­gle re­deem­ing fea­ture.

Mer­ck R&D chief Roger Perl­mut­ter $MRK made no such mis­take in mov­ing on the front­line mar­ket front. Play­ing Welling­ton in this R&D Wa­ter­loo, Perl­mut­ter fo­cused on pa­tients with a 50%-plus PD-L1 ex­pres­sion rate and round­ed up a 50% re­duc­tion in the risk of dis­ease pro­gres­sion and 40% plunge in risk of death com­pared to chemo in pre­vi­ous­ly un­treat­ed pa­tients.

This is what a new stan­dard of care for a seg­ment of front-line lung can­cer cas­es looks like. This drug is al­ready be­ing sold and you can bet that the phar­ma gi­ant will move as fast as it can to cap­i­tal­ize on this mar­ket. That’s a glo­ri­ous vic­to­ry by any bio­phar­ma stan­dard.

Over re­cent months Bris­tol-My­ers stock has re­treat­ed sig­nif­i­cant­ly while Mer­ck has ad­vanced. That’s how you can score the war. And Mon­day morn­ing Mer­ck stock bub­bled up an­oth­er 3% while Bris­tol-My­ers shares plunged 8%.

But Mer­ck won’t re­main un­chal­lenged. Steal­ing some of the thun­der at ES­MO is Genen­tech’s Tecen­triq. Roche’s big check­point, the third to be ap­proved and start spread­ing its wings, achieved a sol­id suc­cess in sec­ond-line NSCLC. Their 13.8 month me­di­an sur­vival rate com­pared fa­vor­ably with the 9.6 months record­ed for the chemo arm.

Dan Chen, who’s head­ed up can­cer im­munother­a­py de­vel­op­ment for Genen­tech, told Fier­cePhar­ma’s Car­ly Helfand that the da­ta were “es­sen­tial­ly un­prece­dent­ed.” But when you sin­gled out the high PD-L1 ex­pressers, the da­ta weighed even more heav­i­ly on Tecen­triq’s side: 20.5 months me­di­an sur­vival com­pared to 8.9 months in the chemo arm.

Now Roche $ROG can break out from its unique blad­der can­cer ap­proval and start to tear up the sec­ond-line lung can­cer mar­ket, look­ing for an ad­van­tage with all the high PD-L1 ex­pressers. A front­line pitch can’t be far off.

While Mer­ck, Bris­tol-My­ers and Roche are divvy­ing up the mar­ket, As­traZeneca is still side­lined af­ter its ef­forts fell well be­hind its ri­vals. But who was that stand­ing on the edge of the bat­tle­field?

“Sud­den­ly, this tri­al news opens quite some op­por­tu­ni­ties for us, in both monother­a­py and com­bi­na­tion ther­a­py,” a sun­ny As­traZeneca Chief Ex­ec­u­tive Pas­cal So­ri­ot told Reuters’ Ben Hirschler, siz­ing up the fast-chang­ing lung can­cer mar­ket.

As­traZeneca’s check­point pro­gram $AZN has been a lag­gard and will ar­rive to the fray very late. For So­ri­ot, though, it’s all char­ac­ter­ized as an ad­van­tage; a chance to see how oth­ers have done their stud­ies so they can match the best work. But af­ter a se­ries of clin­i­cal set­backs, As­traZeneca looks like its re­ced­ing from So­ri­ot’s promis­es of great ad­vances on the rev­enue front, 4 years af­ter he got the top job. He needs a piece of the check­point mar­ket—and fast.

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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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.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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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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