Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for Cure­Vac $CVAC is com­ing up with a loom­ing ear­ly-stage read­out on their mR­NA Covid-19 vac­cine in the clin­ic. But for now they’ll make do with an up­beat as­sess­ment on the pre­clin­i­cal an­i­mal da­ta they used to get in­to the clin­ic.

Re­searchers for the Ger­man biotech say they got the high an­ti­body titers and T cell ac­ti­va­tion they were look­ing for, lin­ing up a ham­ster chal­lenge to demon­strate — in a sim­ple mod­el — that the vac­cine could pro­tect the fur­ry crea­tures. Like the oth­er mR­NA vac­cines, the drug sends in­struc­tions to spur cells to dec­o­rate them­selves with the dis­tinc­tive spike on the virus to elic­it an im­mune re­sponse.

Their sum­ma­ry:

Neu­tral­iz­ing an­ti­bod­ies start­ed to de­vel­op 3 weeks af­ter the first vac­ci­na­tion and strong­ly in­creased af­ter the sec­ond vac­ci­na­tion. The im­pact of dif­fer­ent dos­ing sched­ules for the two-in­jec­tion (prime and boost) reg­i­men was in­ves­ti­gat­ed at 2µg with 1, 2, 3, or 4 weeks be­tween the first and sec­ond vac­ci­na­tion. The da­ta showed in­duc­tion of IgG1 and IgG2a bind­ing an­ti­bod­ies 7 days af­ter the first vac­ci­na­tion. Titers in­creased af­ter the sec­ond vac­ci­na­tion, show­ing high­er re­sponse lev­els with longer in­jec­tion in­ter­vals.

The biotech post­ed the study in pre-print on bioRx­iv, with a planned pub­li­ca­tion in a peer-re­viewed jour­nal, so there’s no ob­jec­tive re­view of what they found. In it, re­searchers note:

By ter­mi­na­tion on day 49, all sera 96 de­rived from an­i­mals vac­ci­nat­ed on d0/28 were able to neu­tralise 50% of in­fect­ing virus (100 TCID50) in 97 cell cul­ture at a di­lu­tion of 1:5120.

Even at this stage of the game, though, with the Pfiz­er/BioN­Tech and Mod­er­na vac­cines be­ing test­ed in large piv­otal stud­ies, an­i­mal da­ta can still get a rise out of the in­vestor class. Cure­Vac took ad­van­tage of its new list­ing on Nas­daq to add an 8% bump to its share price, en­hanc­ing their rough­ly $9 bil­lion mar­ket cap.

Cure­Vac made the leap to Nas­daq over the sum­mer as the ex­ec­u­tive crew mapped out a short path that they be­lieve puts them in con­tention for rolling out a vac­cine in the near term to fight the pan­dem­ic. Their fundrais­ing ef­forts quick­ly pieced to­geth­er a $1 bil­lion raise, with an ini­tial $16 price on the stock. It’s now more than $50 a share.

For­tunes will be made — and lost — in the months to come. But for to­day, Cure­Vac is count­ing it­self among the win­ners. — John Car­roll

Phase­Bio shuts down Covid-19 tri­al due to ‘evolv­ing’ land­scape, poor in­ter­im read­out

A biotech con­duct­ing a clin­i­cal-stage tri­al look­ing to study its ex­per­i­men­tal drug in Covid-19-re­lat­ed ARDS was shut down Fri­day morn­ing.

Phase­Bio had been re­search­ing whether or not its pemzivip­tadil pro­gram, pri­mar­i­ly be­ing stud­ied in pul­monary ar­te­r­i­al hy­per­ten­sion, could have an ef­fect on hos­pi­tal­ized Covid-19 pa­tients. But the com­pa­ny said that due to the “evolv­ing Covid-19 treat­ment land­scape,” as well as FDA guid­ance and a less-than-stel­lar in­ter­im read­out, it will be end­ing the tri­al.

The com­pa­ny said it had re­cent­ly sub­mit­ted a new tri­al pro­to­col for Covid-19 in re­sponse to that chang­ing land­scape, but reg­u­la­tors re­turned with ques­tions about the like­ly need for ad­di­tion­al tri­als with enough pa­tients to ad­e­quate­ly as­sess the mor­tal­i­ty risk.

In­vestors did not ap­pear pleased with the news as the mar­ket opened Fri­day, with Phase­Bio shares $PHAS down about 6% at the open­ing bell.

Phase­Bio had planned for an en­roll­ment of 70 in each of its treat­ment arms. The to­tal was de­ter­mined to be un­like­ly to be big enough, and the need for an en­tire­ly new tri­al mea­sur­ing mor­tal­i­ty rates al­so proved too dif­fi­cult to en­roll.

Fur­ther­more, the in­ter­im read­out was ac­cel­er­at­ed to in­clude on­ly the first 25 pa­tients treat­ed with pemzivip­tadil. In both the 40 mg and 100 mg dos­es, Phase­Bio did not see any­thing in­di­cat­ing that the pro­gram would meet its pri­ma­ry end­point, though it not­ed that a larg­er sam­ple size would be need­ed to draw a de­fin­i­tive con­clu­sion. Nev­er­the­less, the tri­al was halt­ed.

Phase­Bio’s Phase IIb study in pul­monary ar­te­r­i­al hy­per­ten­sion will re­sume en­roll­ment, the com­pa­ny added. It had been paused due to Covid-19 com­pli­ca­tions as well as Phase­Bio steer­ing re­sources to­ward its ARDS study.

In re­sponse to the Covid-19 pan­dem­ic, vast amounts of time and en­er­gy have been spent try­ing to find ef­fec­tive treat­ments and vac­cines for the dis­ease. There are near­ly 4,000 tri­als list­ed on the US gov­ern­ment’s web­site, and thus far on­ly 24 have been ter­mi­nat­ed — less than 1%.

Just Thurs­day, Gilead’s Vek­lury be­came the first ful­ly ap­proved Covid-19 treat­ment and on­ly three ther­a­pies have even re­ceived emer­gency use au­tho­riza­tion: Vek­lury, con­va­les­cent plas­ma and hy­drox­y­chloro­quine. The lat­ter’s EUA has since been re­voked. — Max Gel­man

Biotech ex­ecs ral­ly around An­tho­ny Fau­ci as Trump bites back 

Days af­ter Pres­i­dent Don­ald Trump waged a ver­bal at­tack on An­tho­ny Fau­ci, call­ing him a “dis­as­ter” and an “id­iot,” 9 in­dus­try lead­ers pub­lished a let­ter in sup­port of the NI­AID di­rec­tor and agen­cies such as the CDC and FDA.

The au­thors of the let­ter, pub­lished in Na­ture, in­clude: Ron Co­hen, pres­i­dent and CEO of Acor­da Ther­a­peu­tics; Cedric Fran­cois, pres­i­dent and CEO of Apel­lis Phar­ma­ceu­ti­cals; John Crow­ley, chair­man and CEO of Am­i­cus Ther­a­peu­tics; Paul Hast­ings, pres­i­dent and CEO of Nkar­ta Ther­a­peu­tics; Rachel King, CEO of Gly­coMimet­ics; Ted Love, pres­i­dent and CEO of Glob­al Blood Ther­a­peu­tics; John Maraganore, CEO of Al­ny­lam Phar­ma­ceu­ti­cals; Michelle Mc­Mur­ry-Heath, pres­i­dent and CEO of the Biotech­nol­o­gy In­no­va­tion Or­ga­ni­za­tion; and Je­re­my Levin, chair­man and CEO of Ovid Ther­a­peu­tics and chair­man of the Biotech­nol­o­gy In­no­va­tion Or­ga­ni­za­tion.

They called mem­bers of the CDC, FDA and NIH the coun­try’s “‘Unit­ed Armed Forces’ against COVID-19,” and Fau­ci an “Amer­i­can hero.”

“How­ev­er, we are con­cerned that var­i­ous par­ties in this crit­i­cal coali­tion have come un­der po­lit­i­cal­ly mo­ti­vat­ed at­tacks,” they wrote.

On Sun­day, Fau­ci said in an in­ter­view with 60 Min­utes that the White House has lim­it­ed his me­dia ap­pear­ances, and called the Trump cam­paign’s fram­ing of his com­ments in an ad “out­ra­geous.”

In re­sponse, Trump said: “Peo­ple are tired of hear­ing Fau­ci and all these id­iots, all these peo­ple that have got­ten it wrong.” He claimed, with­out ev­i­dence, that more peo­ple would’ve died if he had fol­lowed Fau­ci’s ad­vice.

The pres­i­dent has pre­vi­ous­ly ac­cused a “deep state” at the FDA of in­ten­tion­al­ly de­lay­ing a vac­cine and ther­a­peu­tics, and fought the agency on stricter guid­ance for Covid-19 vac­cine EUAs.

“Not on­ly are these at­tacks com­plete­ly un­jus­ti­fied, but they al­so risk in­tim­i­dat­ing and de­mor­al­iz­ing the very peo­ple we all are re­ly­ing on to help end the COVID-19 night­mare. As such, they are ir­re­spon­si­ble and pose dan­ger to us all,” in­dus­try lead­ers wrote in their let­ter pub­lished Thurs­day.

Ear­li­er this week, the in­de­pen­dent Gov­ern­ment Ac­count­abil­i­ty Of­fice launched an in­ves­ti­ga­tion in­to the White House’s in­flu­ence on the CDC and FDA. The news came a week af­ter De­mo­c­ra­t­ic Sen­a­tors Pat­ty Mur­ray, Gary Pe­ters and Eliz­a­beth War­ren wrote to the GAO, cit­ing in­ci­dents in which they ac­cused the Trump ad­min­is­tra­tion of pres­sur­ing the agen­cies.

“Many of our col­leagues and friends are work­ing as part of Op­er­a­tion Warp Speed; we stand unit­ed with them, with the many good peo­ple at FDA, CDC and NIH, and with Dr. Fau­ci, in up­hold­ing the high sci­en­tif­ic, med­ical and eth­i­cal prin­ci­ples that will al­low us to de­feat COVID-19,” the Na­ture let­ter con­cludes. — Nicole De­Feud­is 

Pe­ru PM says coun­try will not sign vac­cine deal with As­traZeneca

Phar­ma com­pa­nies have been mak­ing deals with gov­ern­ments all over the world to sup­ply their po­ten­tial Covid-19 vac­cines, but one coun­try has de­cid­ed to re­ject an agree­ment with one big vac­cine de­vel­op­er.

Pe­ru’s prime min­is­ter Wal­ter Mar­tos an­nounced in a news con­fer­ence Thurs­day that his gov­ern­ment would not sign a vac­cine pur­chase con­tract with As­traZeneca, per a Reuters re­port. Mar­tos said the British drug­mak­er did not pro­vide enough da­ta from its stud­ies and of­fered few­er dos­es than oth­er com­pa­nies de­vel­op­ing vac­cines.

“The oth­er labs have caught up with us ac­cord­ing­ly, how­ev­er As­traZeneca has not,” Mar­tos said, per Reuters. “They were of­fer­ing us a very low quan­ti­ty of vac­cines com­pared with oth­er lab­o­ra­to­ries that are of­fer­ing us large quan­ti­ties at low­er costs,” he said.

As­traZeneca has been one of the fron­trun­ners of the Covid-19 vac­cine race, cur­rent­ly plac­ing 3rd in End­points News’ track­er. How­ev­er, their Phase III tri­al came to a glob­al halt in ear­ly Sep­tem­ber af­ter a British vol­un­teer de­vel­oped what was thought to be a rare spinal in­flam­ma­tion dis­or­der.

Stud­ies have since re­sumed in the UK, South Africa, Brazil, In­dia and Japan, but re­main on hold in the US. On Tues­day, Reuters re­port­ed that the US por­tion of the tri­al could re­sume soon, per­haps by the end of the week.

Al­so ear­li­er this week, a healthy vol­un­teer died in As­traZeneca’s Brazil study, the coun­try’s health agency said. The in­di­vid­ual was re­port­ed­ly in the place­bo group of the tri­al and test­ing will con­tin­ue un­en­cum­bered. Ox­ford Uni­ver­si­ty, work­ing with the firm to cre­ate the vac­cine, said in a state­ment “there have been no con­cerns about safe­ty of the clin­i­cal tri­al.” — Max Gel­man

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

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (Getty)

As­traZeneca CEO So­ri­ot plans new study to test that con­tro­ver­sial 90% ef­fi­ca­cy fig­ure, wait­ing for US da­ta be­fore go­ing to FDA

Pascal Soriot spent the long Thanksgiving weekend digging AstraZeneca out of a hole, promising to put an end to the questions around its interim Phase III vaccine data by conducting a new study while going to regulators with a large part of what it already has.

AstraZeneca and its partners at Oxford had initially touted high-level results from two studies conducted in the UK and Brazil as positive. But the enthusiasm was soon shadowed by confusion as observers probed into how the highest, 90% efficacy was seen in a dosing regimen given to a small group of volunteers due to an error. Among a larger cohort given the intended shots, the vaccine was only 62% effective, a rate that would’ve been respectable had Pfizer/BioNTech and Moderna not posted efficacy rates of 94%, 95% for their mRNA candidates. And many weren’t sure what to make of the average 70% number that AstraZeneca ran in headlines.

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Scoop: Google’s GV spear­heads the Spot­light syn­di­cate — back­ing an up­start biotech aimed at ‘de­moc­ra­tiz­ing’ gene edit­ing

CRISPR had no sooner started to shake the very foundations of drug development before its limitations began to loom large. Gene editing could change the world — if only you could get around the hurdles that threatened to trip up every program.

So it’s only natural to see CRISPR 2.0 taking shape before the pioneers can get the lead therapies through development. And who better than Google’s GV venture arm to take the lead spot in a small syndicate backing some scientists with their own unique twist on a solution?

Sev­er­al weeks af­ter get­ting hit with an RTF, Y-mAbs lands ap­proval for its oth­er neu­rob­las­toma can­di­date

Nearly two months after handing Y-mAbs a refusal to file letter for one of its main neuroblastoma candidates, the FDA gave the biotech an accelerated OK for the other — but with a box warning.

Y-mAbs, which flew mostly under the radar until a few years ago, snagged approval for naxitamab-gqgk as a second-line treatment in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF). Patients older than 1 year old can take the drug for relapsed or refractory high-risk neuroblastoma in the bone or bone marrow. The good news cushioned last month’s blow, sending the company’s stock $YMAB — which sank more than 18% upon news of the RTF — up 10.15% as of Monday morning.

Stephané Bancel (Endpoints at JPM20)

Mod­er­na cal­i­brates fi­nal Covid-19 vac­cine ef­fi­ca­cy at 94.1% — and to­day it's gun­ning for the EUA

Nearly a year ago, as the coronavirus emerged in China, the NIH and four major companies bet on an unproven genetic technology as the best tool for developing a vaccine to stem the outbreak. Today, a second such vaccine is heading to the FDA.

Moderna said Monday that they will request an emergency use authorization from the FDA after a final analysis showed their mRNA vaccine was 94.1% effective at preventing symptomatic Covid-19. The data confirm the results from an interim analysis and matches efficacy Pfizer and BioNTech showed in a Phase III study, setting the biotech up to potentially nab one of the first two Covid-19 vaccine OKs.

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FDA gives Rhythm the green light for set­melan­otide, a drug aimed at re­duc­ing obe­si­ty in cer­tain ge­net­ic dis­or­ders

A little over a year after completing successful pivotal trials, Rhythm Pharmaceuticals $RYTM has its first drug approval on its hands.

The Boston-based biotech announced Friday that the FDA gave setmelanotide the thumbs-up for three rare genetic disorders that result in obesity in patients six and older. It’s the agency’s first such approval, Rhythm said, with the indicated deficiencies being the POMC, PCSK1 and LEPR genes. Rhythm will market the drug as Imcivree, and plans to have it on the shelves in the first quarter of 2021.

Robert Clarke (Kinaset)

Ki­naset launch­es with $40M and a JAK in­hibitor from Vec­tura's old pipeline

Kinaset Therapeutics is joining the search for a better severe asthma treatment, picking up where Vectura left off when it decided to clear house last year.

UK-based Vectura — which took a big hit when its most advanced candidate flopped in a Phase III asthma trial back in 2018 — recently shifted to a CDMO model, offloading all of its R&D programs. Robert Clarke, who’s worked on inhalable therapeutics for 21-plus years, had close contacts at the company and took a look at what they were offering. After doing some research, he was attracted by VR475, a pan-JAK inhibitor.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.