Özlem Türeci, BioNTech co-founder and Uğur Şahin, BioNTech CEO

Third dose bumps up ef­fi­ca­cy of Pfiz­er-BioN­Tech's Covid-19 vac­cine in youngest group of chil­dren to 80%

Pfiz­er and BioN­Tech said Mon­day that they’re ready to ap­proach the FDA this week with ear­ly da­ta for their boost­er shot for Covid-19 vac­cine in the youngest age group (6 months to un­der 5 years), which showed 80.3% ef­fi­ca­cy based on 10 symp­to­matic Covid cas­es iden­ti­fied be­gin­ning sev­en days af­ter the third dose.

“The study sug­gests that a low 3-ug dose of our vac­cine, care­ful­ly se­lect­ed based on tol­er­a­bil­i­ty da­ta, pro­vides young chil­dren with a high lev­el of pro­tec­tion against the re­cent COVID-19 strains,” Uğur Şahin, CEO and co-founder of BioN­Tech said in a state­ment. “We are prepar­ing the rel­e­vant doc­u­ments and ex­pect com­plet­ing the sub­mis­sion process to the FDA this week, with sub­mis­sions to EMA and oth­er reg­u­la­to­ry agen­cies to fol­low with­in the com­ing weeks.”

The vac­cine al­so proved to be as safe as place­bo, Pfiz­er said.

The FDA is like­ly to of­fer a swift EUA to both Pfiz­er’s vac­cine and Mod­er­na’s two-dose Covid-19 vac­cine for this youngest group of chil­dren with­in the next month or so, bar­ring any last-minute con­cerns by FDA staff. Af­ter re­view­ing the ap­pli­ca­tions, the agency said it will hold a joint ad­vi­so­ry com­mit­tee meet­ing for the two vac­cines on June 15. And June 14 will be the new meet­ing date for the FDA and its ad­vi­so­ry com­mit­tee of ex­ter­nal ex­perts to dis­cuss Mod­er­na’s EUA re­quest for 6 years through 17 years of age.

Once the apps are ful­ly sub­mit­ted and the meet­ing dates are fi­nal­ized, the FDA will re­lease its own analy­ses of the Pfiz­er and Mod­er­na da­ta no lat­er than two busi­ness days be­fore each meet­ing.

Late last month, Mod­er­na com­plet­ed the fil­ing of its EUA ap­pli­ca­tion, pre­vi­ous­ly an­nounc­ing that its vac­cine is 51% ef­fec­tive in chil­dren 6 months to 2 years of age and 37% ef­fec­tive in 2- to 5-year-olds. And while that sub-50% ef­fi­ca­cy num­ber may raise eye­brows, FDA’s Pe­ter Marks has said that it’s OK as long as the ef­fi­ca­cy is above 50% for the en­tire range of the ap­pli­ca­tion sub­mit­ted (6 months to 17 year-olds).

Mean­while, Pfiz­er and BioN­Tech’s tri­al is still wrap­ping up, with the pro­to­col spec­i­fy­ing a for­mal analy­sis will be per­formed when at least 21 cas­es have ac­crued from sev­en days af­ter the third dose. Fi­nal vac­cine ef­fi­ca­cy da­ta will be shared once avail­able, Pfiz­er said.

In to­tal, the part­ners’ Phase II/III tri­al in­clud­ed 1,678 chil­dren re­ceiv­ing a third dose of the 3-µg for­mu­la­tion at least two months af­ter the sec­ond dose at a time when Omi­cron was the pre­dom­i­nant vari­ant.

The im­muno­genic­i­ty analy­sis of geo­met­ric mean titer ra­tio and sero-re­sponse rate was con­duct­ed on a sub­set of study par­tic­i­pants one month fol­low­ing the third dose in chil­dren 6 months to un­der 5 years of age, com­pared to the sec­ond dose in the 16- to 25-year-old pop­u­la­tion. Pfiz­er said non-in­fe­ri­or­i­ty was met for both the 6- to 24-month-old pop­u­la­tion and the 2- to un­der 5-year-old pop­u­la­tion for both co-pri­ma­ry end­points.

Three 3-µg dos­es of the Pfiz­er-BioN­Tech vac­cine were well-tol­er­at­ed in this age group, Pfiz­er added, not­ing that the ma­jor­i­ty of ad­verse events were mild or mod­er­ate.

The chil­dren who par­tic­i­pat­ed in the tri­al and re­ceived place­bo are now be­gin­ning to re­ceive their vac­cines.

Ed­i­tor’s note: Up­dat­ed with more in­for­ma­tion on the new FDA ad­vi­so­ry com­mit­tee meet­ing times.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Photo: Julia Weeks/AP Images

FDA ax­es re­quire­ment for pos­i­tive Covid test be­fore Paxlovid use

FDA announced today that doctors and pharmacists can now prescribe Paxlovid to patients without a positive test for Covid-19.

CDER Director Patrizia Cavazzoni reissued Paxlovid’s authorization letter Wednesday, saying it has revised the authorization to “no longer require positive results of direct SARS-CoV-2 viral testing.” The EUA now requires instead that adults and kids 12 years of age and older have a “current diagnosis of mild-to-moderate COVID-19.”

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Giovanni Caforio, Bristol Myers Squibb CEO (Nicolas Messyasz/Sipa via AP Images)

Bris­tol My­ers turns at­ten­tion to new prod­ucts in wake of Revlim­id patent loss

Bristol Myers Squibb CEO Giovanni Caforio is shifting his focus to newer products as generic sales continue to gnaw at the company’s blockbuster myeloma drug Revlimid.

Both Revlimid and Abraxane sales took a dive last year thanks to generic rivals, BMS reported in its Q4 and full-year results on Thursday. As a result, Q4 sales dipped 5% and full-year sales remained flat. However, Caforio sees a silver lining — or rather, two of them.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP)

FTC makes an ex­am­ple of GoodRx, bans dis­counter from shar­ing pri­vate health da­ta with ad­ver­tis­ers

Prescription drug discount provider GoodRx will no longer be allowed to share its users’ sensitive health data with advertisers after the Federal Trade Commission charged the online coupon provider with failing to notify consumers of such disclosures to Facebook, Google, and other companies.

GoodRx agreed to pay a $1.5 million civil penalty for violating the FTC’s Health Breach Notification Rule after the FTC said it repeatedly violated a 2017 promise to not share sensitive personal health information. The FTC alleged that the company shared users’ prescription medications and personal health conditions with third party advertisers and platforms like Facebook, Google, Criteo, Branch and Twilio.