As­traZeneca, Mer­ck win red car­pet treat­ment at the FDA as a 3-time los­er seeks speedy OK for rare tu­mors

As­traZeneca’s R&D group doesn’t take fail­ure ly­ing down.

Hit with piv­otal set­backs for their MEK 1/2 in­hibitor selume­tinib in sev­er­al can­cer stud­ies, they re­grouped and in­clud­ed it in their $8.4 bil­lion al­liance with Mer­ck as the ther­a­py was steered in­to a Phase II for rare cas­es of neu­rofi­bro­mato­sis type 1, or NF1.

That da­ta gave the phar­ma gi­ant a break­through drug des­ig­na­tion in the spring, and now they are on track for a Q2 2020 PDU­FA af­ter the FDA added a pri­or­i­ty re­view to the gift pack­age to­day.

By in­hibit­ing the MEK en­zyme in the RAS/MAPK path­way in­ves­ti­ga­tors have demon­strat­ed that the drug can con­trol er­rant sig­nal­ing that con­trols cell growth — at least rel­e­vant to NF1.

Back in 2018, when Ned Sharp­less was run­ning the Na­tion­al Can­cer In­sti­tute — and where he’s head­ed back af­ter a stint as act­ing di­rec­tor of the FDA — he held up the re­search work on selume­tinib to a House com­mit­tee as one of the most im­por­tant projects in their can­cer moon­shots pro­gram, cit­ing the be­fore-and-af­ter pic­tures from par­ents with kids suf­fer­ing from in­op­er­a­ble tu­mors sparked by the dis­ease.

Ac­cord­ing to a re­port from the Chil­dren’s Tu­mor Foun­da­tion, which in­vest­ed in the pre­clin­i­cal work, in­ves­ti­ga­tors tracked a 70% re­sponse rate, with tu­mors shrink­ing at least 20%.

Pas­cal So­ri­ot As­traZeneca

Five years ago, as As­traZeneca CEO Pas­cal So­ri­ot pledged to make As­traZeneca in­to a roar­ing suc­cess with $45 bil­lion in an­nu­al sales while fend­ing off a takeover at­tempt by Pfiz­er, selume­tinib loomed large among the ex­per­i­men­tal drugs he tagged for a block­buster fu­ture. Then the fail­ures start­ed to mount.

As­traZeneca was forced to con­cede 3 years ago that the drug flopped in KRAS-pos­i­tive non-small cell lung can­cer. Selume­tinib whiffed on pro­gres­sion-free sur­vival and failed to score on over­all sur­vival as well. And As­traZeneca had to walk away from the first Phase III for selume­tinib in 2015 af­ter the drug — in-li­censed from Ar­ray — failed for an eye can­cer called uveal melanoma. Most re­cent­ly the drug failed a Phase III study for dif­fer­en­ti­at­ed thy­roid can­cer in the sum­mer of 2018.

A win here would be sweet for the resur­gent on­col­o­gy team at As­traZeneca, which has fu­eled As­traZeneca’s long-await­ed come­back over the past year with big suc­cess­es for Lyn­parza, Tagris­so and to a more lim­it­ed ex­tent for the PD-L1 Imfinzi.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.