FDA post­pones ad­comm for Pfiz­er's Covid-19 vac­cine for the youngest age group

The FDA on Fri­day af­ter­noon said it’s no longer mov­ing for­ward with its plan to au­tho­rize two dos­es (while wait­ing for da­ta on a third dose) of Pfiz­er’s Covid-19 vac­cine for the youngest group of chil­dren, from 6 months through 4 years of age.

Pe­ter Marks FDA

“Based on the agency’s pre­lim­i­nary as­sess­ment, and to al­low more time to eval­u­ate ad­di­tion­al da­ta, we be­lieve ad­di­tion­al in­for­ma­tion re­gard­ing the on­go­ing eval­u­a­tion of a third dose should be con­sid­ered as part of our de­ci­sion-mak­ing for po­ten­tial au­tho­riza­tion,” FDA’s top vac­cine of­fi­cial Pe­ter Marks and act­ing FDA com­mis­sion­er Janet Wood­cock said in a state­ment while an­nounc­ing that next week’s VRB­PAC meet­ing would be de­layed.

Marks said the da­ta, some of which was “late-break­ing,” are com­ing in very rapid­ly be­cause of the large num­ber of cas­es of Omi­cron and “at this time it makes sense to wait for the third dose be­fore tak­ing ac­tion.”

Janet Wood­cock

“The da­ta we saw made us re­al­ize we need to see a third dose in or­der to pro­ceed with an au­tho­riza­tion,” he added.

The ex­ten­sion al­lows the FDA time to re­ceive up­dat­ed da­ta on the two and three-dose reg­i­men, Pfiz­er said, while con­duct­ing a thor­ough eval­u­a­tion of it.

The com­pa­ny had ini­tial­ly said in De­cem­ber that it would test a third dose of its Covid-19 vac­cine in the tri­al af­ter an­nounc­ing that non-in­fe­ri­or­i­ty was not met for chil­dren be­tween the ages of two and five when com­pared to old­er teenagers in the cur­rent tri­al. But for the youngest group, from 6 months to 2-years-old, non-in­fe­ri­or­i­ty was met.

But Marks said it was a “safe as­sump­tion” that the ef­fi­ca­cy de­ci­sion would be based on clin­i­cal da­ta from Pfiz­er.

Pfiz­er said in a state­ment that in these chil­dren, da­ta on the first two 3 µg dos­es in this age group are be­ing shared with the FDA on an on­go­ing ba­sis. Cas­es con­tin­ue to ac­cu­mu­late ac­cord­ing to the study pro­to­col and more da­ta are be­ing gen­er­at­ed be­cause rates of in­fec­tion and ill­ness re­main high in chil­dren of this age, es­pe­cial­ly due to the re­cent Omi­cron surge.

“The in­de­pen­dent Da­ta Mon­i­tor­ing Com­mit­tee (DMC) for the study sup­ports the con­tin­u­a­tion of the tri­al ac­cord­ing to the pro­to­col and be­lieve that the da­ta col­lect­ed to date in­di­cate the vac­cine is well tol­er­at­ed and sup­port a po­ten­tial three-dose reg­i­men,” Pfiz­er added.

Some pub­lic health ex­perts said they thought FDA was cor­rect in press­ing the pause but­ton.

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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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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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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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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(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.

Al Sandrock, Voyager Therapeutics CEO

Al San­drock prunes his post-Bio­gen voy­age with sim­i­lar fo­cus on ALS

Al Sandrock is narrowing the focus of Voyager Therapeutics, concentrating on CNS diseases that were the hallmark of his time leading R&D at Biogen, including an emphasis on a familial form of ALS for which his former employer is getting a speedy review at the FDA.

Less than six months into his journey as CEO at Voyager, Sandrock is focusing the preclinical pipeline on Alzheimer’s disease, GBA1 Parkinson’s disease and SOD1 amyotrophic lateral sclerosis, the rare form of ALS for which the FDA will decide whether to approve Biogen’s tofersen by Jan. 25, 2023.

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Pfiz­er ter­mi­nates PhI­II study of rare car­dio­vas­cu­lar drug picked up in $11.4B Ar­ray ac­qui­si­tion

While Pfizer’s $11.4 billion acquisition of Array BioPharma in the summer of 2019 was mainly focused on oncology, namely Braftovi and Mektovi, there were a few non-cancer assets, including a Phase III drug being tested in a rare cardiovascular disease.

The late-stage trial is now being axed, alongside any further development of the oral small molecule, the pharma giant disclosed after the closing bell on Wednesday. Based on an interim futility analysis of the global Phase III REALM-DCM trial, Pfizer determined a path forward was not in its best interest. Pfizer no longer expected the study would meet its primary goal.

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