FDA's ad­comm votes unan­i­mous­ly in fa­vor of Mod­er­na and Pfiz­er's Covid-19 vac­cines for the youngest chil­dren

The FDA’s VRB­PAC met Wednes­day in a tense meet­ing to de­cide the fate of both Pfiz­er/BioN­Tech and Mod­er­na’s Covid-19 vac­cines for the youngest groups of chil­dren, from ages 6 months to 6 years. Ul­ti­mate­ly, the com­mit­tee of 21 out­side ex­perts vot­ed unan­i­mous­ly in fa­vor of Mod­er­na’s two-shot vac­cine, and Pfiz­er’s three-shot vac­cine (both 21-0).

The ex­perts all ex­pressed sup­port for vac­ci­nat­ing chil­dren in this youngest pop­u­la­tion to bet­ter pre­vent against Covid-re­lat­ed hos­pi­tal­iza­tions and deaths that have risen in this group dur­ing the lat­est wave of the Omi­cron vari­ant.

Ad­comm pan­elist Hay­ley Alt­man-Gans of Stan­ford Uni­ver­si­ty Med­ical Cen­ter praised the pos­i­tive vote for Mod­er­na’s vac­cine, say­ing the sci­ence around this de­ci­sion has been tak­en “very se­ri­ous­ly” and that, “I’m very glad for this op­tion for peo­ple in the sci­en­tif­ic com­mu­ni­ty who take care of chil­dren and for fam­i­lies.”

For Pfiz­er’s vac­cine, pan­elists of­fered a few reser­va­tions giv­en the two-dose da­ta seemed to show lit­tle ef­fi­ca­cy, but the group sup­port­ed the over­all risk-ben­e­fit ra­tio.

Pan­elist Paul Of­fit, di­rec­tor of the vac­cine ed­u­ca­tion cen­ter at the Chil­dren’s Hos­pi­tal of Philadel­phia, said of the Pfiz­er shot that he was con­cerned of the low lev­els of ef­fi­ca­cy af­ter the sec­ond vac­cine dose, and said this may end up be­ing a four-dose vac­cine.

“I just won­der whether par­ents will un­der­stand that” re­gard­ing the sit­u­a­tion af­ter two dos­es, Of­fit said. But he al­so stressed that he would sup­port it.

CBER di­rec­tor Pe­ter Marks urged the com­mit­tee at the out­set to “be care­ful that we don’t be­come numb to the num­ber of pe­di­atric deaths be­cause of the over­whelm­ing num­ber of old­er deaths here. Every life is im­por­tant.” He al­so called on Pfiz­er just be­fore that vote to ex­plain more around the ef­fi­ca­cy of its vac­cine af­ter just two dos­es.

For Mod­er­na’s two-shot se­ries, FDA’s analy­sis from ear­li­er this week con­firmed what Mod­er­na an­nounced, that ef­fi­ca­cy was 36.8% (95% CI 12.5, 54.0) for the 2 to 5 age group, and 50.6%(95% CI 21.4, 68.6) for par­tic­i­pants 6 to 23 months of age. But un­like oth­er age groups, those re­spons­es oc­curred when Omi­cron was pre­dom­i­nant.

Those rates are based on the 2,594 chil­dren en­rolled in the 2 to 5 age group of Mod­er­na’s tri­al and re­ceiv­ing the vac­cine, with 4.6% end­ing up with CDC-de­fined Covid-19, 14 days af­ter the sec­ond dose, and 7.1% of the 858 who were on place­bo got Covid-19. For the younger group, 6 to 36 months, of the 1,511 chil­dren who re­ceived the Mod­er­na shot, 5.4% caught Covid, while 8.8% of the 513 caught it in the place­bo arm.

Over­all, though, vac­cine ef­fec­tive­ness for all four pe­di­atric age co­horts for which Mod­er­na is ap­ply­ing (Pfiz­er al­ready has an EUA for those over 5) was in­ferred by im­muno­bridg­ing based on a com­par­i­son of SARS-CoV-2 neu­tral­iz­ing an­ti­body (nAb) re­spons­es one month af­ter the sec­ond dose to the nAb re­spons­es gen­er­at­ed by those 18 to 25 years of age.

For Pfiz­er’s 3-shot se­ries, ef­fi­ca­cy was de­ter­mined by the 376 kids who re­ceived the vac­cine and 179 who re­ceived place­bo in the 6 to 23 months age range, with one Covid-19 case oc­cur­ring in the vac­cine arm and two in the place­bo arm. For the 589 who re­ceived the Pfiz­er vac­cine and the 271 place­bo re­cip­i­ents in the 2 to 4 years of age range, two cas­es oc­curred in the vac­cine group and five in the place­bo group.

But as with the Mod­er­na shot, Pfiz­er’s vac­cine ef­fec­tive­ness was in­ferred by im­muno­bridg­ing, based on a com­par­i­son of neu­tral­iz­ing an­ti­body re­spons­es with a com­para­tor group of par­tic­i­pants 16 to 25 years of age.

The FDA brief­ing doc­u­ments al­so said that es­ti­mates of vac­cine ef­fi­ca­cy among par­tic­i­pants six months through five years ap­pear con­sis­tent with the VE es­ti­mates ob­served in adults dur­ing Omi­cron, based on ob­ser­va­tion­al stud­ies and that de­scrip­tive analy­ses of ef­fi­ca­cy “fur­ther sup­port­ed and pro­vid­ed di­rect ev­i­dence of vac­cine ef­fec­tive­ness.”

The same com­mit­tee of out­side vac­cine and in­fec­tious dis­ease ex­perts al­so met yes­ter­day and vot­ed in fa­vor of ex­pand­ing the use of Mod­er­na’s Covid vac­cine to teenagers and oth­er chil­dren over the age of 6 who pre­vi­ous­ly on­ly had ac­cess to the Pfiz­er vac­cine.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.