Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a po­ten­tial EUA ap­pli­ca­tion, Mod­er­na an­nounced they have com­plet­ed en­roll­ment in their 30,000-per­son Phase III Covid-19 vac­cine tri­al, with over a third of vol­un­teers non-white and a quar­ter over the age of 65.

The an­nounce­ment caps what has been the most close­ly-watched re­cruit­ment race in the his­to­ry of drug de­vel­op­ment, as Pfiz­er and Mod­er­na rushed to get enough vol­un­teers to prove whether or not ex­per­i­men­tal vac­cines could ac­tu­al­ly pro­tect peo­ple from con­tract­ing Covid-19. Pfiz­er reached that mark on Sept. 15. Mod­er­na said around the same time that they would slow down en­roll­ment to en­sure they en­rolled enough par­tic­i­pants from mi­nor­i­ty and at-risk groups.

The race has cooled some­what since then, as new FDA guid­ance de­mand­ing more safe­ty da­ta threw cold wa­ter on a po­ten­tial Oc­to­ber ap­proval. Still, Mod­er­na says it’s re­mained in­tense­ly fo­cused, giv­ing a rare shoutout to their CRO PPD in a press re­lease for stay­ing on top of things as they steered the Phase III in a new di­rec­tion.

“The at­ten­tion on this tri­al has been un­like any­thing any­one has ever been through be­fore,” Mod­er­na pres­i­dent Stephen Hoge told End­points News. “The amount of re­port­ing be­fore we even dosed a pa­tient was amaz­ing and al­most dai­ly since.”

The com­plet­ed en­roll­ment comes on the same day that a much-an­tic­i­pat­ed FDA ad­vi­so­ry com­mit­tee sits down to dis­cuss the var­i­ous vac­cine tri­als, al­though they won’t dive in­to any spe­cif­ic can­di­dates.

Since mid-Sep­tem­ber, Mod­er­na has al­most ex­clu­sive­ly en­rolled non-white par­tic­i­pants, slides re­leased to­day show. Their num­bers now com­pare fa­vor­ably with Pfiz­er, whose study was 75% white when it crossed the 29,000-per­son mark, al­though the Big Phar­ma has since blue­print­ed plans to en­roll an­oth­er 14,000 vol­un­teers, par­tic­u­lar­ly from pop­u­la­tions un­der-rep­re­sent­ed in their ini­tial co­horts.

With­out of­fer­ing spe­cif­ic bench­marks, the FDA has em­pha­sized the need for this kind of di­ver­si­ty for months, not­ing the dis­pro­por­tion­ate toll the virus has tak­en on Black and Lati­no Amer­i­cans and those who are old­er and have co-mor­bidi­ties.

With en­roll­ment com­plet­ed, the fu­ture of Mod­er­na’s vac­cine can­di­date now lies in the hands of the FDA ad­comm, the agency it­self, and the vi­cis­si­tudes of epi­demi­ol­o­gy. Hoge said that the com­pa­ny ex­pects to have both an in­ter­im ef­fi­ca­cy read­out and enough safe­ty fol­low-up to sat­is­fy the FDA by mid-No­vem­ber, a time­line that would put them al­most neck-and-neck with Pfiz­er.

Among the main ques­tions fac­ing the ad­comm to­day is what hap­pens to the tri­als if the vac­cines look ef­fec­tive at that point. Pfiz­er asked the FDA to al­low them to vac­ci­nate those in the place­bo.  Hoge ex­pressed a sim­i­lar sen­ti­ment, though he ac­knowl­edged that raised ques­tions about how oth­er com­pa­nies will be able to con­duct tri­als for much-need­ed vac­cines.

“It re­al­ly de­pends on what the scope of an EUA or ap­proval is,” he said. “Clear­ly, if there’s a vol­un­teer in our study or any study and a vac­cine be­comes avail­able to them, whether it’s un­der ap­proval or EUA, eth­i­cal­ly they have every right to go get that vac­cine. And what that means is you al­so want them to know whether they got the vac­cine or not. You don’t want them to go get dou­ble-vac­ci­nat­ed, which cre­ates oth­er risks.”

“One of the things we all have to wres­tle with here — I don’t think it’s a com­pa­ny health mat­ter, it’s a reg­u­la­to­ry mat­ter, it’s a pub­lic health mat­ter — is how do we deal with that,” he added. “It’s com­pli­cat­ed.”

Hoge of­fered that, if their vac­cine was au­tho­rized on­ly for those over 75, then oth­er com­pa­nies could test their vac­cine in peo­ple un­der the age of 75 and ex­trap­o­late from there. He al­so spec­u­lat­ed about syn­thet­ic con­trols where, rather than ran­dom­iz­ing vol­un­teers, vac­ci­nat­ed in­di­vid­u­als are com­pared epi­demi­o­log­i­cal­ly to “sim­i­lar” un­vac­ci­nat­ed peo­ple.

Huge mar­ket ques­tions al­so loom for both Pfiz­er and Mod­er­na. SVB Leerink’s Ge­of­frey Porges spec­u­lat­ed yes­ter­day that Pfiz­er could gain a mul­ti-bil­lion dol­lar mar­ket ad­van­tage by hav­ing the first vac­cine out of the gate. Hoge, though, re­ject­ed that idea, point­ing out that vac­cines will be in short sup­ply for con­sid­er­able time af­ter the first few are au­tho­rized, mak­ing them dif­fer­ent than vir­tu­al­ly any oth­er new drug in re­cent mem­o­ry.

“The re­al­i­ty is if you don’t have enough sup­ply to sat­is­fy all that de­mand, it’s re­al­ly just gonna be main­tain­ing that sup­ply and sat­is­fy­ing as much as you can,” he said. “Frankly, I think if we on­ly get two out there — Pfiz­er and Mod­er­na — in the near term, I think that’s a big con­cern.”

Longer term, an­a­lysts have tried to parse how large the mar­ket is for boost­er shots and re­peat vac­ci­na­tions, while pub­lic health ex­perts have puz­zled over just how long im­mu­ni­ty will last. Hoge of­fered a mid­dle ground, ar­gu­ing that the fu­ture for Covid may look sim­i­lar to the fu­ture for RSV, where on­ly the el­der­ly need re­peat­ed boost­er shots.  He ar­gued the cur­rent da­ta sug­gest young peo­ple wouldn’t need more than a sin­gle boost­er over a long-term pe­ri­od.

“Whether there’s go­ing to be val­ue for a boost­er or an en­dem­ic mar­ket down be­low the age of 55 or out­side of those co-mor­bid conid­tions — my hope is no,” he said. “That would sug­gest that we re­al­ly don’t have con­trol over this virus.”

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Paul Hudson, Sanofi CEO (via Getty)

Sanofi's $20B buy­out of Gen­zyme pays off again with Eu­ro­pean OK for first Nie­mann-Pick drug

Sanofi CEO Paul Hudson has made clear his intention to develop new rare disease drugs and broaden his company’s offerings. That effort leaped forward on Friday with the EMA’s signing off on the company’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease.

The enzyme replacement therapy, developed to replace patients’ deficient or defective enzyme, known as acid sphingomyelinase, was first developed by Genzyme, which Sanofi acquired for more than $20 billion in 2011. That acquisition has also helped Sanofi pull in sales in the field of MS.

Proac­tive­ly pre­vent­ing short­ages: New FDA guid­ance spells out which drugs re­quire risk man­age­ment plans

As the majority of drug shortages are still associated with manufacturing-related quality issues, the FDA on Thursday published new draft guidance spelling out how to proactively assess risks to manufacturing processes and supply chains, while understanding the market’s vulnerabilities.

While drug shortages peaked in 2011, the FDA says in its new 18-page draft guidance that the number of new drug shortages “has declined significantly since” that peak, reaching a low in 2015 and 2016, thanks in part to a new law’s enactment, known as FDASIA, which helped the agency better prevent or mitigate drug supply disruptions and shortages, and clarified cGMP requirements.

Janet Woodcock (Sipa via AP Images)

FDA com­mis­sion­er an­nounces new role for Janet Wood­cock, down­shift­ing away from drugs

New FDA commissioner Rob Califf told staff in a memo yesterday evening that longtime drug center director Janet Woodcock, who’s now serving as Califf’s principal deputy, would take on more of a role as “a strategic thought partner, with decision-making authorities” at several different offices within FDA. Curiously, the list of offices and centers does not include the Office of New Drugs or the Office of Generic Drugs.

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Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.