FDA stamps fast OK on Astel­las' pi­o­neer­ing FLT3 AML drug gilter­i­tinib, ex­pand­ing on a record year for new ap­provals

Astel­las has picked up boast­ing rights to gain­ing the first FDA ap­proval of a FLT3 in­hibitor for a spe­cif­ic group of FLT3 pos­i­tive acute myeloid leukemia pa­tients — which ac­counts for close to a third of the pop­u­la­tion.

Back in May the Japan­ese phar­ma re­port­ed that the agency had of­fered a snap 6-month re­view on its fast-track pro­gram for gilter­i­tinib, some­thing that’s be­come quite com­mon in the on­col­o­gy group.

That’s good news for the 30% or so of the 19,000 new­ly di­ag­nosed AML cas­es each year that will be linked to the ge­net­ic mu­ta­tion. The drug has been in a piv­otal study where in­ves­ti­ga­tors have been study­ing it’s abil­i­ty to cut re­laps­es in the pa­tient pop­u­la­tion.

The drug will be sold as Xospa­ta, and Astel­las set the whole­sale price at $22,500 for 30 days, which would trans­late to $270,000 for a year of ther­a­py — though pay­ers ne­go­ti­ate down from that fig­ure. In a state­ment, the com­pa­ny said that their in­ter­im analy­sis of the piv­otal pro­gram tracked a me­di­an du­ra­tion of re­sponse of 4.6 months.

Alexan­der Ed­ward Perl

This OK de­liv­ers a “new, high­ly-ef­fec­tive, and well-tol­er­at­ed treat­ment op­tion to the clin­ic for a group of tru­ly high-risk pa­tients who, un­til to­day, had no spe­cif­ic ther­a­pies avail­able be­yond chemother­a­py to treat their dis­ease,” says Alexan­der Perl, an as­so­ciate pro­fes­sor of Hema­tol­ogy-On­col­o­gy at the Uni­ver­si­ty of Penn­syl­va­nia. Perl has been lead­ing the clin­i­cal tri­al pro­gram for this drug.

“Al­though we’re wait­ing for the fi­nal analy­sis of (the Phase III study) AD­MI­RAL, the avail­able da­ta with gilter­i­tinib show few­er and milder side ef­fects than typ­i­cal­ly is seen with tra­di­tion­al chemother­a­py,” Perl added, not­ing that they al­so tracked a sig­nif­i­cant­ly high­er rate of com­plete re­spons­es.

The ap­proval marks the FDA’s 54th OK of the year as it goes deep­er in­to record ter­ri­to­ry af­ter pass­ing the old record set in 1996.


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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Otello Stampacchia. Omega Funds

Af­ter sev­er­al high pro­file start­up launch­es, om­niv­o­rous Omega Funds clos­es $438M fund to pur­sue more

Omega Funds likes to work backwards. It invests with the end game — denoted by the Greek letter in its name — in mind, and it keeps tabs on the number of marketed medical products that culminate from its ventures: 37 in 16 years.

So when founder and managing director Otello Stampacchia declares it’s the most exciting time to be investing in life sciences in a generation, it’s perhaps only natural that Omega has closed its largest fund to date. With $438 million in total commitments for Fund VI, the firm will continue injecting capital into a broad swath of companies across the US and Europe.

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Janet Woodcock (Credit: CQ Roll Call/AP)

For­eign drug in­spec­tions de­cline as FDA hir­ing strug­gles con­tin­ue

The US reliance on imported pharmaceuticals and ingredients is rising as foreign drug facility inspections decreased by about 10% from 2016 to 2018. Part of the reason for the decline: The US Food and Drug Administration (FDA) said it’s still struggling to hire new inspectors.

Testifying before a House subcommittee on Tuesday, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, defended the agency’s approach and discussed some of the vulnerabilities of its foreign inspection program. She said FDA is looking to hire 50 new inspectors, but there are difficulties.

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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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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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.