Monica Bertagnolli (PR Newswire/Natera)

Biden to tap Har­vard sur­geon, clin­i­cal tri­als ex­pert as NCI di­rec­tor

Mon­i­ca Bertag­nol­li, an em­i­nent sur­gi­cal on­col­o­gist and physi­cian-sci­en­tist, is set to suc­ceed Ned Sharp­less as di­rec­tor of the Na­tion­al Can­cer In­sti­tute.

Ned Sharp­less

Al­though the White House has yet to con­firm the ap­point­ment, mul­ti­ple can­cer re­search or­ga­ni­za­tions and pa­tient ad­vo­ca­cy groups ap­plaud­ed the choice, in­clud­ing AS­CO, where Bertag­nol­li pre­vi­ous­ly served as pres­i­dent. Cur­rent­ly a pro­fes­sor of surgery at Har­vard Med­ical School, and the chief of the Di­vi­sion of Sur­gi­cal On­col­o­gy at Brigham and Women’s Hos­pi­tal and Dana-Far­ber Can­cer In­sti­tute, Bertag­nol­li would be the first woman to lead the NCI.

As the Biden ad­min­is­tra­tion’s sci­en­tif­ic lead­er­ship re­mains in flux, Bertag­nol­li would fill a key void left be­hind af­ter Sharp­less stepped down in April, cap­ping a five-year tenure.

Dou­glas Lowy, prin­ci­pal deputy di­rec­tor, has been head­ing the agency on an act­ing ba­sis.

Out of 27 in­sti­tutes that form the NIH, NCI is the old­est and largest, com­mand­ing a bud­get of $7 bil­lion to fund can­cer re­search.

As a sur­geon, Bertag­nol­li spe­cial­izes in gas­troin­testi­nal tu­mors. In her lab, she stud­ies a gene named APC (ade­no­ma­tous poly­po­sis coli), how its mu­ta­tion or loss ini­ti­ates tu­mor trans­for­ma­tion and whether drugs that work on this path­way can treat col­orec­tal can­cer.

“An ac­com­plished can­cer sur­geon and re­searcher with deep ex­per­tise in com­mu­ni­ty-based can­cer re­search, Dr. Bertag­nol­li has the knowl­edge, pas­sion, and skillset to suc­cess­ful­ly lead the na­tion’s top fed­er­al can­cer re­search agency and the wider U.S. can­cer re­search en­ter­prise,” reads a state­ment from AS­CO CEO Clif­ford Hud­is.

Among Bertag­nol­li’s nu­mer­ous oth­er roles are VP of Coali­tion of Can­cer Co­op­er­a­tive Groups, a non­prof­it aimed at rais­ing aware­ness about clin­i­cal tri­als; chair of Al­liance for Clin­i­cal Tri­als in On­col­o­gy; CEO of Al­liance Foun­da­tion Tri­als; and mem­ber of the Na­tion­al Acad­e­my of Med­i­cine’s Na­tion­al Can­cer Pol­i­cy Fo­rum.

Com­mend­ing Bertag­nol­li’s re­port­ed ap­point­ment, the As­so­ci­a­tion of Amer­i­can Can­cer In­sti­tutes high­light­ed that she’s a “long­time ad­vo­cate of in­creas­ing di­ver­si­ty in can­cer re­search as it ap­plies to physi­cians, re­searchers, and pa­tients en­rolled in clin­i­cal tri­als.”

Dur­ing her AS­CO tenure, she made “(c)ar­ing for every pa­tient, learn­ing from every pa­tient” her pres­i­den­tial theme and trav­eled with fel­low lead­ers to un­der­served com­mu­ni­ties, host­ing town halls to hear from pa­tients, sur­vivors and care­givers.

“Con­sid­er­able work re­mains to give every pa­tient the op­por­tu­ni­ty to par­tic­i­pate in, and ben­e­fit from, can­cer clin­i­cal tri­als, but it is a task that all on­col­o­gists must em­brace,” she wrote in a 2019 ed­i­to­r­i­al in the Can­cer Let­ter. “We owe it to our pa­tients and our field to give every­one a chance to con­tribute to re­search that in­creas­es our un­der­stand­ing of and leads to bet­ter treat­ments for can­cer.”

Sharp­less sim­i­lar­ly told STAT that Bertag­nol­li is a “ter­rif­ic choice”:

She’s a mar­velous sur­geon and na­tion­al leader in can­cer clin­i­cal tri­als. She’s just the right per­son to lead Pres­i­dent Biden’s ‘Can­cer Moon­shot.’

Hav­ing spelled out some am­bi­tious sci­en­tif­ic and bio­med­ical projects, Biden is still scram­bling to re­cruit ex­perts who can man­age them. There’s still no per­ma­nent di­rec­tor of ARPA-H, the new agency set up to fund high-risk, high-re­ward re­search. More than four months af­ter Er­ic Lan­der re­signed from the top sci­ence spot at the White House amid a ha­rass­ment scan­dal, Biden re­cent­ly nom­i­nat­ed Arati Prab­hakar, a renowned ap­plied physi­cist, to head up the Of­fice of Sci­ence and Tech­nol­o­gy Pol­i­cy. He has yet to name a suc­ces­sor to Fran­cis Collins, the for­mer NIH di­rec­tor called out of re­tire­ment to be act­ing OSTP di­rec­tor.

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

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Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

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Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

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George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

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David Hallal (L) and George Daley (Hallal photo: Bertrand Guay/AFP via Getty Images)

David Hal­lal's El­e­vate­Bio launch­es new com­pa­ny to 'dis­rup­t' off-the-shelf cell ther­a­py, but pro­vides few oth­er de­tails

David Hallal’s ElevateBio is launching a new company Thursday as it looks to continue making its mark in the cell and gene therapy spaces. But Hallal is also keeping his cards close to the vest, preferring to toe the line between bombast and mystique rather than going all-in in one direction.

The new company comes out of a partnership with Boston Children’s Hospital and research from George Daley, the dean of Harvard Medical School. The triumvirate claims to have found a way to design better off-the-shelf cell therapies using new methods discovered in Daley’s Boston Children’s Hospital lab (Harvard is not involved in the collaboration).

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(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.

Vlad Coric, Biohaven CEO

Bio­haven touts surge in Nurtec sales ahead of Pfiz­er takeover

Forget buyer’s remorse, Pfizer is likely feeling pretty good about its $11.6 billion Biohaven takeover deal following reports of a 57% sales boost for migraine med Nurtec.

Biohaven reported in Q2 results on Friday that it’s cleared the necessary antitrust hurdles to move forward with the sale of its calcitonin gene-related peptide (CGRP) assets to Pfizer. However, because the company is “focused on workstreams related to the closing” of the deal, it did not host a call with analysts and investors.