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.

Pascal Soriot (AP Images)

As­traZeneca, Ox­ford her­ald 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine, tout­ing eas­i­er dis­tri­b­u­tion, low­er price

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic.

With an average efficacy of 70%, the headline number may appear less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Simeon George, SR One CEO (SR One)

Scoop: SR One crew com­pletes a com­pli­cat­ed spin­out from Glax­o­SmithK­line. And now they have a $500M fund to in­vest on their own

It’s taken close to 2 years, but Simeon George and his team at SR One have completed their spinout from GlaxoSmithKline, ending a saga as one of the longest running venture arms of Big Pharma as they go out on their own to forge the next chapter with a new and independent $500 million fund.

GSK is sticking with the spinout, this time as a minority investor — though a big one. And I’m told that the R&D group at GSK will remain involved in evaluating their new plays, helping with the scientific due diligence involved in scouting the world for new opportunities during a period of explosive growth in biotech investing.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

It's of­fi­cial: Pfiz­er and BioN­Tech have sub­mit­ted their Covid-19 vac­cine to the FDA -- and the agency cir­cled a date for the ad­comm

Pfizer and BioNTech’s request for emergency authorization of its Covid-19 vaccine is now in the FDA’s hands. Regulators caught the application Friday afternoon in an open field dash to the goal line.

The agency immediately marked Dec 10 on the calendar for an advisory committee meeting to discuss the request. Pfizer and BioNTech first announced the submission was coming on Friday morning, and Pfizer CEO Albert Bourla confirmed the filing in a video statement later that afternoon. The “historic day,” as Bourla called it, came just 248 days after the pharma first announced plans to develop the candidate with BioNTech — a process which typically takes years.

CEO Matthew Kane (Precision BioSciences)

In an ap­par­ent R&D about-face, Eli Lil­ly part­ners with Pre­ci­sion Bio­Sciences on genome edit­ing in a deal worth up to near­ly $2.7B

As a large multinational corporation, Eli Lilly has their hands in boundless projects, from cancer and immuno-oncology to diabetes, psoriasis and Crohn’s disease. But Friday they signaled a shift in their R&D focus toward genome editing, leaping into a cutting-edge field CEO Dave Ricks had shied away from as recently as January 2019.

The big pharma is ponying up $100 million upfront to partner with Precision BioSciences, focusing initially on Duchenne muscular dystrophy and two other undisclosed in vivo targets. Lilly is also acquiring $35 million worth of the biotech’s stock, and has the option to develop three additional in vivo therapies.

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

Pfiz­er and BioN­Tech are send­ing the first Covid-19 vac­cine to the FDA to­day

The first Covid-19 vaccine is heading to regulators.

Pfizer and BioNTech announced Friday morning that they would ask today for the FDA to issue an emergency use authorization for their Covid-19 candidate.

The submission, a major milestone in the fight against the pandemic and in history’s most intensive and closely-watched drug development race, will put the ball in the agency’s court, where regulators will pour over all the data the companies have generated since winter and solicit recommendations from outside advisors before making a decision.

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The pri­ma­ry failed. The key sec­ondary failed. But this biotech still be­lieves it can win an FDA OK in ALS

Two years after the executive team at BrainStorm Cell Therapeutics decided to back off a controversial attempt to sell their stem cell therapy for ALS under the new ‘Right to Try’ legislation, the biotech is back with the top-line data from Phase III. And the data aren’t good.

Researchers say the drug — with a once-proposed price of $300,000 — failed the primary endpoint as well as the key secondaries on disease progression. But the executive crew still thinks it’s approvable. And in fact, the biotech also insists the FDA is eager to review it.

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Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er, BioN­Tech say vac­cine is 95% ef­fec­tive, EUA sub­mis­sion 'with­in days'

Pfizer and BioNTech have done the final efficacy analysis for their Phase III Covid-19 trial, and the results confirm the headline-grabbing release from last week: Their vaccine is 95% effective at preventing Covid-19.

They also said the vaccine was 94.5% effective in older adults, mitigating concerns that the first Covid-19 vaccines might not work as well in one of the populations most susceptible to severe disease.

Up­dat­ed: FDA gives Cel­lec­tis all clear af­ter pa­tient death halt­ed off-the-shelf CAR-T tri­al

Four months after a patient death forced Cellectis to halt one of their off-the-shelf CAR-T trials, the FDA has given them the OK to start dosing patients again.

The Paris-based biotech said they changed the study’s protocol to accommodate the agency’s concerns, although they will still have to work with investigators to obtain local approval to restart the trial and start recruiting patients again. Prior its halt, the Phase I had sites in New York, New Jersey and Texas.