Cel­gene ex­ec jumps to head blue­bird bio ops in Eu­rope, where its $1.8M gene ther­a­py Zyn­te­glo is now avail­able

Days af­ter shak­ing hands with Ger­man reg­u­la­tors over the launch and cov­er­age of its be­ta-tha­lassemia gene ther­a­py, blue­bird bio has wooed a Cel­gene ex­ec to lead its Eu­ro­pean op­er­a­tions.

Nico­la Hef­fron

Nico­la Hef­fron, a bio­phar­ma vet with stints across Eli Lil­ly, GSK and Shire, jumps from a brief tenure over­see­ing mar­ket­ing for Cel­gene’s myeloid port­fo­lio in Sum­mit, NJ. She will now be based in Zug, Switzer­land.

She’s re­plac­ing An­drew Oben­shain as he joins CEO Nick Leschly and the lead­er­ship team in Boston, ac­cord­ing to Bloomberg, which first re­port­ed the news. Oben­shain’s new ti­tle is “chief of wings.”

On Mon­day blue­bird an­nounced that Ger­many will be the first coun­try to com­mer­cial­ly of­fer Zyn­te­glo, their pro­ce­dure en­cod­ing βA-T87Q-glo­bin gene in CD34+ cells ex­tract­ed from pa­tients. Un­der their val­ue-based pay­ment scheme, the $1.8 mil­lion price is di­vid­ed in­to five in­stall­ments. Af­ter an ini­tial pay­ment is made at the time of in­fu­sion, the pay­ers wait and see and on­ly pay if the pa­tients con­tin­ue to be trans­fu­sion-free.

“Mul­ti­ple statu­to­ry health in­sur­ances” have signed on­to the plan, blue­bird said, and Uni­ver­si­ty Hos­pi­tal of Hei­del­berg will host the first qual­i­fied treat­ment cen­ter.

The biotech has been busy sort­ing out man­u­fac­tur­ing specs and talk­ing to in­di­vid­ual coun­tries since the EU is­sued an his­toric OK last June. It’s sanc­tioned for a spe­cif­ic group of be­ta-tha­lassemia pa­tients — those who are 12 years and old­er, trans­fu­sion de­pen­dent, do not have a β0/β0 geno­type and for whom hematopoi­et­ic stem cell trans­plan­ta­tion is ap­pro­pri­ate but a donor is not avail­able.

“For pa­tients with TDT, life­long chron­ic blood trans­fu­sions are re­quired in or­der to sur­vive,” blue­bird chief com­mer­cial of­fi­cer Al­i­son Fin­ger em­pha­sized in a state­ment. Their one-time in­fu­sion promis­es to do away with the trans­fu­sions for good.

A rolling BLA sub­mis­sion to the FDA has be­gun, blue­bird added.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA have vowed not to let politics get in the way of science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped health agencies under his purview — including the FDA — of their rulemaking ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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Eli Lilly CSO Dan Skovronsky (file photo)

#ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

#ES­MO20: It’s not just Keytru­da any­more — Mer­ck spot­lights 3 top ear­ly-stage can­cer drugs

Any $12 billion megablockbuster in the portfolio tends to overshadow everything else in the pipeline. Which is something Merck can tell you a little bit about.

Keytruda not only dominates the PD-(L)1 field, it looms over everything Merck does, to the point some analysts wonder if Merck is a one-trick pony.

There’s no shortage of Keytruda data on display at ESMO this weekend, but now the focus is shifting to the future role of new drugs and combos in maintaining that lead position for years to come. And the pharma giant has a special focus for 3 early-stage efforts where Roger Perlmutter’s oncology team is placing some big bets.

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Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

Sean Bo­hen's break from bio­phar­ma is over. The ex-As­traZeneca CMO has re­tired his Big Phar­ma jer­sey and is now — hap­pi­ly — run­ning a lit­tle biotech

The last I had heard about Sean Bohen, he had stepped out of his high-profile job as chief medical officer at AstraZeneca at the beginning of 2019 as CEO Pascal Soriot triggered a broad-ranging R&D shakeup. And then, earlier this week, I got a chance to catch up.

It turns out that Bohen decided at the time that he would not just jump into a new job in the booming biopharma business. As an oncologist, he had worked on the big programs at AstraZeneca, and before that he was at Genentech. That was good for a ticket to just about anyplace in the big biopharma world. But he felt it was time to stop and think things through.

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