Puma wins big FDA OK for ner­a­tinib af­ter run­ning the gamut with in­vestors

Short in­vestors loved to hate Puma Biotech­nol­o­gy, tak­ing the com­pa­ny $PBYI to task for the side ef­fects that ham­pered its lead can­cer drug ner­a­tinib. But the FDA had no trou­ble sanc­tion­ing the prod­uct for breast can­cer, ap­prov­ing it to­day for use in pre­vent­ing re­cur­rence of the dis­ease af­ter the biotech gained a lop­sided vote in its fa­vor among out­side agency ex­perts.

The FDA ap­proved ner­a­tinib — to be sold as Ner­l­ynx — for pre­vent­ing the re­turn of breast can­cer af­ter ther­a­py in­clud­ing trastuzum­ab. And the em­pha­sis in the OK was on adding op­tions for doc­tors and pa­tients.

“HER2-pos­i­tive breast can­cers are ag­gres­sive tu­mors and can spread to oth­er parts of the body, mak­ing ad­ju­vant ther­a­py an im­por­tant part of the treat­ment plan,” said Richard Paz­dur, M.D., di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence and act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “Now, these pa­tients have an op­tion af­ter ini­tial treat­ment that may help keep the can­cer from com­ing back.”

There are some caveats in the la­bel. Con­cerned about the high rate of Grade 2 and Grade 3 di­ar­rhea in the clin­i­cal stud­ies, reg­u­la­tors are telling physi­cians to stop us­ing the drug if their pa­tient ex­pe­ri­ences Grade 4 di­ar­rhea or Grade 2 af­ter a max­i­mum dose re­duc­tion. But Michael Schmidt at Leerink sees the plus­es as well in a broad la­bel:

The prod­uct la­bel looks broad, not re­strict­ing use to spe­cif­ic pa­tient pop­u­la­tions (e.g., hor­mone-re­cep­tor [HR] pos­i­tive pa­tients) and there is no black box warn­ing. The pre­scrib­ing in­for­ma­tion con­tains fair­ly de­tailed in­for­ma­tion about the use of an­tidiar­rheal pro­phy­lax­is mea­sures, pro­vid­ing spe­cif­ic in­for­ma­tion on how to man­age the drug’s main side ef­fect.

Puma says it isn’t re­leas­ing the price yet and has no time­line on that. Its stock price still had some up­side left af­ter the OK, though, with shares surg­ing 7.5% on a com­pa­ny with a $3.2 bil­lion mar­ket cap. Now that the ap­proval has come through, look for lots more chat­ter about Puma as a po­ten­tial takeover tar­get — even though M&A has large­ly been MIA this year in biotech.

Alan Auer­bach

The FDA ex­perts raised a va­ri­ety of con­cerns in their re­view of ner­a­tinib, mak­ing some points that could hin­der the drug’s mar­ket po­ten­tial now that Puma CEO Alan Auer­bach has a green light to sell it. Crit­i­cal to its suc­cess is a bet­ter un­der­stand­ing of its prop­er role in treat­ing breast can­cer, and the lim­its of its ef­fec­tive­ness.

To get here, Puma had to over­come reg­u­la­tors’ ob­jec­tions in the spring of 2016 to fil­ing the drug with the da­ta that were avail­able, cit­ing a prob­lem­at­ic ap­proach to de­ci­pher­ing the re­sults. The shorts were al­so fix­at­ed on a high rate of grade 3 di­ar­rhea in the tri­als, but ex­perts hard­ly paid any at­ten­tion to that, hap­py to trade off the po­ten­tial for man­age­able side ef­fects for a chance of adding a new ar­row to their quiver of ther­a­pies.

“There’s no ex­cla­ma­tion point af­ter my ‘yes’,” said An­drew Sei­d­man, an on­col­o­gist at Memo­r­i­al Sloan Ket­ter­ing af­ter the FDA vote. “It’s just a yes.” He added that he was hap­py that the drug looks durable and the sta­tis­ti­cal analy­ses ap­plied to the da­ta giv­en the sig­nif­i­cant changes to the study de­sign. “I do think that physi­cians will se­lect pa­tients very se­lec­tive­ly for us­ing this,” he not­ed, echo­ing a con­cern that the ap­proval the com­pa­ny was seek­ing was too broad giv­en the da­ta avail­able.

Puma’s po­si­tion has been height­ened af­ter an­a­lysts fa­vor­ably com­pared ner­a­tinib’s 34% re­duc­tion in the risk of re­cur­rence or death against a mar­gin­al im­prove­ment seen in a Roche com­bi­na­tion study of Per­je­ta and Her­ceptin. It takes a leap to do these kinds of cross-tri­al analy­ses that dri­ve drug de­vel­op­ers a tad crazy now and then, but Wall Street an­a­lysts were quick to give Puma a thumbs up, par­tic­u­lar­ly af­ter it had just hand­i­ly won the FDA pan­el vote.

Eval­u­atePhar­ma pegged 2022 sales at $1.25 bil­lion, mak­ing it one of the top 15 drugs in the in­dus­try pipeline at the be­gin­ning of this year.

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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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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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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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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