Peter Marks (Jim Lo Scalzo/Pool via AP Images)

UP­DAT­ED: FDA's Pe­ter Marks to Con­gress: Youngest kids vac­cine won't need to hit 50% ef­fi­ca­cy mark

The FDA’s top vac­cine leader told a con­gres­sion­al com­mit­tee on Fri­day af­ter­noon that al­though the adult vac­cines had to meet a 50% thresh­old for ef­fi­ca­cy against Covid-19 in­fec­tions, that same stan­dard will not need to be met for the vac­cines for the youngest group of chil­dren, for which a vac­cine is not yet avail­able.

The agency is cur­rent­ly re­view­ing da­ta from Mod­er­na’s two-shot vac­cine for this youngest group as it awaits fur­ther da­ta from Pfiz­er on its po­ten­tial three-dose shot. Mod­er­na has said its vac­cine is 51% ef­fec­tive in chil­dren 6 months to 2 years of age and 37% ef­fec­tive in 2- to 5-year-olds. The agency al­so pre­vi­ous­ly sched­uled and then can­celed an ad­comm to re­view da­ta on two dos­es of Pfiz­er’s vac­cine for chil­dren un­der the age of 5.

Ac­cord­ing to a read­out of the meet­ing from the House se­lect sub­com­mit­tee on the coro­n­avirus cri­sis, Marks ex­plained that the FDA would not with­hold au­tho­riza­tion — de­spite pre­vi­ous guid­ance — for a pe­di­atric vac­cine sole­ly be­cause it did not reach a 50% ef­fi­ca­cy thresh­old at block­ing symp­to­matic in­fec­tions. All of the oth­er adult and chil­dren’s vac­cines cur­rent­ly au­tho­rized in the US have lost sig­nif­i­cant amounts of ef­fi­ca­cy due to the Omi­cron vari­ant, but they still re­main ef­fec­tive at re­duc­ing the risk of se­vere dis­ease, hos­pi­tal­iza­tion and death.

“If these vac­cines seem to be mir­ror­ing ef­fi­ca­cy in adults and just seem to be less ef­fec­tive against Omi­cron like they are for adults, we will prob­a­bly still au­tho­rize,” Marks said.

Jim Clyburn

Mean­while, Marks di­rect­ly ad­dressed con­cerns that the agency might slow-play the Mod­er­na ap­pli­ca­tion and wait to re­view both ap­pli­ca­tions to­geth­er, say­ing that the VRB­PAC meet­ings set for next month will be moved up if nec­es­sary.

“Ob­vi­ous­ly if we get through re­views faster, then we will send them to com­mit­tees soon­er,” Marks said, ac­cord­ing to Rep. Jim Clyburn’s ac­count of the meet­ing. Marks al­so told Clyburn and his staff that VRB­PAC mem­bers have re­served ear­li­er dates, en­abling FDA to po­ten­tial­ly “move dates up even by a week for any of these re­views.”

But he al­so ex­plained that re­view­ing these vac­cines for the youngest chil­dren in­volves more “com­plex­i­ty” than the re­view of adult vac­cines, and the agency’s re­view process may re­quire more time.

He al­so said that the emer­gence of new vari­ants made the process of re­view­ing vac­cines more com­plex, ex­plain­ing it “was a lit­tle sim­pler for the orig­i­nal sub­mis­sions in late 2020 when we didn’t have the vari­ants and tons of im­muno­genic­i­ty da­ta to go through.

“At the end of the day, we want peo­ple to have con­fi­dence in get­ting vac­ci­nat­ed,” he said, ac­cord­ing to Clyburn. Marks al­so re­it­er­at­ed the im­por­tance of vac­ci­na­tions to pro­tect chil­dren against the coro­n­avirus, stat­ing: “We need to get more kids vac­ci­nat­ed, not just in the younger than 5 age range, but al­so old­er than 5.”

Since mak­ing these re­marks to Clyburn, Marks told a me­dia brief­ing ear­li­er this week that he will main­tain the stan­dard for 50% ef­fi­ca­cy for this shot for the youngest kids group, but that “it’s pos­si­ble in sub­pop­u­la­tions they might be a lit­tle less ef­fec­tive than 50%.”

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with Marks’ lat­est com­ment and to note Mod­er­na’s stat­ed ef­fi­ca­cy for its vac­cine for these two groups of the un­der 6 pop­u­la­tion.

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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Sen. Kyrsten Sinema (D-AZ) (Tom Williams/CQ Roll Call via AP Images)

De­moc­rats se­cure sup­port from key sen­a­tor ahead of po­ten­tial drug pric­ing vote

Senate Democrats may have all the votes they need to pass major drug pricing reform, after Sen. Kyrsten Sinema (D-AZ) reportedly pledged her support on Thursday — but will they fit it in before recess?

Sinema said she has agreed to “move forward” with the reconciliation bill with some stipulations, including the removal of a carried tax provision, according to recent reports. The bill is still expected to reduce the deficit by $300 billion, and Sen. Chuck Schumer (D-NY) said that he now anticipates “support from the entire Senate Democratic conference,” the Washington Post reported. 

CDC, NIH, FDA lead­ers call for US-based clin­i­cal tri­al of small­pox drug in treat­ing mon­key­pox

With the rising number of monkeypox cases, leading researchers at the CDC, FDA and NIH are calling on a randomized clinical trial to see if an approved smallpox drug is effective at treating monkeypox.

No monkeypox treatments are approved in the US, so patients looking to get relief for their lesions and other symptoms from the virus must go through a set of hurdles to get the smallpox drug through a government expanded access program. Approved for smallpox in 2018, the drug is marketed as TPOXX by the biotech SIGA. The European Union approved it for monkeypox in addition to smallpox earlier this year and the UK followed suit in July.

CDER director Patrizia Cavazzoni, FDA commissioner Robert Califf and OPDP acting director Catherine Gray

Drug pro­mo­tion en­force­ment so far sim­i­lar un­der new FDA chief, with just 4 let­ters in first half of 2022

Will a trio of new bosses at the FDA lead to an increase in drug advertising enforcement? It’s a good question, but with few letters sent out so far, there’s just not enough information to tell, says one longtime FDA regulatory watchdog.

“I don’t think there is anything to indicate the Administration is leaning one way or another,” Mark Senak, a lawyer at FleishmanHillard who tracks FDA actions on his Eye on FDA blog, said in an email. “It is worth noting that we have a new FDA Commissioner, new head of CDER and a new head of OPDP which opens up the possibility of change, but there has not yet been enough enforcement activity to identify real change.”

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