Four years af­ter EU ap­proval, NICE fi­nal­ly backs Revlim­id use in cer­tain first-line mul­ti­ple myelo­ma pa­tients

Four years af­ter the Eu­ro­pean Com­mis­sion sanc­tioned the use of Cel­gene’s flag­ship Revlim­id to treat adults with pre­vi­ous­ly-un­treat­ed mul­ti­ple myelo­ma who are not el­i­gi­ble for trans­plant, Eng­land’s Na­tion­al Health Ser­vice (NHS) will adopt its use as the first or sec­ond line of de­fense — in cer­tain pa­tients. Al­though the block­buster drug is rou­tine­ly used as a back­bone treat­ment in much of the de­vel­oped world, un­til now, the drug was on­ly cleared for third-line use by NICE.

Revlim­id, which gen­er­at­ed rough­ly $9.7 bil­lion in glob­al 2018 sales, has long been Cel­gene’s key­stone treat­ment, but its use in Eng­land had been re­strained due to pric­ing dis­agree­ments. The drug is al­so cru­cial to Bris­tol-My­er’s $BMY $74 bil­lion bet on the big biotech (al­though the loss of patent pro­tec­tion is loom­ing).

Mul­ti­ple myelo­ma is a life-threat­en­ing form of blood can­cer, which orig­i­nates in the bone mar­row, and af­fects rough­ly 17,500 peo­ple in the UK at any giv­en time, ac­cord­ing to char­i­ty Myelo­ma UK, which es­ti­mates each year that about 5,700 new pa­tients are di­ag­nosed with the dis­ease.

NICE pub­lished two pieces of fi­nal draft guid­ance on Fri­day. In the first re­port, NICE rec­om­mend­ed the use of Revlim­id — known chem­i­cal­ly as lenalido­mide — in com­bi­na­tion with the cor­ti­cos­teroid dex­am­etha­sone as an op­tion for treat­ment-naive adults with mul­ti­ple myelo­ma who are not el­i­gi­ble for a stem cell trans­plant and can­not take thalido­mide. This new op­tion could ben­e­fit about 2,100 pa­tients, NICE said.

Thalido­mide (or borte­zomib, or Take­da’s $TAK Vel­cade, for those who can’t take thalido­mide) re­mains the first line of de­fense, al­though NICE ac­knowl­edged that se­vere side-ef­fects as­so­ci­at­ed with its use can rule out its adop­tion. How­ev­er, “(l)enalido­mide plus dex­am­etha­sone can­not be rec­om­mend­ed for un­treat­ed mul­ti­ple myelo­ma in peo­ple who could take thalido­mide be­cause this would not be cost ef­fec­tive,” NICE un­der­scored.

Lenalido­mide is avail­able as a 21‑cap­sule pack, and its cost varies per dose, up to £4,368 for the high­est 25 mg dose/pack. But, Cel­gene $CELG has now agreed to a (con­fi­den­tial) dis­count that has sat­is­fied NICE. 

In the sec­ond re­port, NICE backed the use of lenalido­mide as an op­tion for treat­ing mul­ti­ple myelo­ma in adults if they have had on­ly one pre­vi­ous ther­a­py, in­clud­ing borte­zomib. Par­tic­u­lar­ly for those that have been first treat­ed with borte­zomib, the on­ly next treat­ment in the doc­tor’s tool­box is chemother­a­py — how­ev­er, ev­i­dence in­di­cates that the lenalido­mide+dex­am­etha­sone is more ef­fec­tive than cy­to­tox­ic chemother­a­py. In one clin­i­cal study, pa­tients tak­ing lenalido­mide lived on av­er­age 7 months longer, NICE said.

About 320 pa­tients are ex­pect­ed to ben­e­fit, it added.

The most ‘plau­si­ble’ cost-ef­fec­tive­ness es­ti­mate for lenalido­mide plus dex­am­etha­sone may be above the range that NICE nor­mal­ly con­sid­ers be­ing a cost-ef­fec­tive use of NHS re­sources, re­searchers high­light­ed in the re­port, but con­ced­ed that since lenalido­mide has been rec­om­mend­ed for use as a first treat­ment (for which it is cost-ef­fec­tive) — the need for lenalido­mide as a sec­ond treat­ment will like­ly de­crease be­cause peo­ple are more like­ly to have it as a first treat­ment in the fu­ture.

Last month, NICE al­so backed the use of J&J’s $JNJ Darza­lex in com­bi­na­tion with Vel­cade as a sec­ond-line treat­ment for mul­ti­ple myelo­ma via the can­cer drugs fund.

Cel­gene, mean­while, is ad­vanc­ing the use of triplet mul­ti­ple myelo­ma ther­a­pies. On Thurs­day, the EU ap­proved 1) Revlim­id in com­bi­na­tion with Vel­cade and dex­am­etha­sone for adult pa­tients with pre­vi­ous­ly un­treat­ed mul­ti­ple myelo­ma who are not el­i­gi­ble for trans­plant; 2) Im­novid in com­bi­na­tion with Vel­cade and dex­am­etha­sone in adult pa­tients with mul­ti­ple myelo­ma, who have re­ceived at least one pri­or treat­ment reg­i­men in­clud­ing Revlim­id.


Im­age Source: As­so­ci­at­ed Press

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Am­gen puts its foot down in shiny new South San Fran­cis­co hub as it re­or­ga­nizes R&D ops

Amgen has signed up to be AbbVie’s neighbor in South San Francisco as it moves into a nine-story R&D facility in the booming biotech hub.

The arrangement gives Amgen 240,000 square feet of space on the Gateway of Pacific Campus, just a few minutes drive from its current digs at Oyster Point. The new hub will open in 2022 and house the big biotech’s Bay Area employees working on cardiometabolic, inflammation and oncology research.

Ab­b­Vie, Scripps ex­pand part­ner­ship, for­ti­fy fo­cus on can­cer drugs

Scripps and AbbVie go way back. Research conducted in the lab of Scripps scientist Richard Lerner led to the discovery of Humira. The antibody, approved by the FDA in 2002 and sold by AbbVie, went on to become the world’s bestselling treatment. In 2018, the drugmaker and the non-profit organization signed a pact focused on developing cancer treatments — and now, the scope of that partnership has broadened to encompass a range of diseases, including immunological and neurological conditions.

South Ko­rea jails 3 Sam­sung ex­ecs for de­stroy­ing ev­i­dence in Bi­o­Log­ics probe

Three Samsung executives in Korea are going to jail.

The convictions came in what prosecutors had billed as “biggest crime of evidence destruction in the history of South Korea”: a case of alleged corporate intrigue that was thrown open when investigators found what was hidden beneath the floor of a Samsung BioLogics plant. Eight employees in total were found guilty of evidence tampering and the three executives were each sentenced to up to two years in prison.

Nick Plugis, Avak Kahvejian, Cristina Rondinone, Milind Kamkolkar and Chad Nusbaum. (Cellarity)

Cel­lar­i­ty, Flag­ship's $50M bet on net­work bi­ol­o­gy, mar­ries ma­chine learn­ing and sin­gle-cell tech for drug dis­cov­ery

Cellarity started with a simple — but far from easy — idea that Avak Kahvejian and his team were floating around at Flagship Pioneering: to digitally encode a cell.

As he and his senior associate Nick Plugis dug deeper into the concept, they found that most of the models others have developed take a bottom-up approach, where they assemble the molecules inside cells and the connections between them from scratch. What if they opt for a top-down approach, aided by single-cell transcriptomics and machine learning, to gauge the behavior of the entire cellular network?

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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