Covid-19 roundup: Pfiz­er hus­tling up mil­lions of dos­es of vac­cine — de­tails $748M BioN­Tech deal; CDC tweaks stance on con­tro­ver­sial malar­ia drugs

Pfiz­er and BioN­Tech are ready to out­line the de­tails of their am­bi­tious al­liance on a Covid-19 mR­NA vac­cine for the world.

Their plan now is to start hu­man tri­als in a mat­ter of days, while ramp­ing up man­u­fac­tur­ing for a glob­al mar­ket well ahead of the first read­out. And the Ger­man mR­NA com­pa­ny — large­ly owned by the bil­lion­aire Stru­eng­mann broth­ers — is get­ting a big up­front to go with the col­lab­o­ra­tion.

The phar­ma gi­ant is pro­vid­ing the biotech $185 mil­lion in cash, in­clud­ing an eq­ui­ty in­vest­ment of $113 mil­lion. Then there’s $563 mil­lion in mile­stones. They plan to joint­ly com­mer­cial­ize the vac­cine around the plan­et, with Fo­s­un tak­ing Chi­na per their sep­a­rate pact with BioN­Tech.

The clin­i­cal tri­al sup­plies of the vac­cine will be pro­vid­ed by BioN­Tech’s ex­ist­ing man­u­fac­tur­ing fa­cil­i­ty, leav­ing the part­ners to start im­me­di­ate­ly build­ing out enough man­u­fac­tur­ing ca­pac­i­ty to pro­vide “mil­lions” of dos­es be­fore the end of this year and “hun­dreds of mil­lions” by next year. All of that in­vest­ment is “at risk” — a bet that their jab can end the out­break.

Pfiz­er, mean­while, is al­so be­gin­ning test­ing soon on a new Covid-19 drug for mild to mod­er­ate cas­es. The com­pa­ny tells the Wall Street Jour­nal that the ther­a­py has the po­ten­tial to stop the virus from repli­cat­ing. And they al­so plan to study their rheuma­toid arthri­tis drug Xel­janz against the virus, part of a mul­ti-prong ef­fort to com­bat Covid-19.

First up, though, is mR­NA.

A group of mR­NA com­pa­nies are tak­ing a shot at pro­duc­ing new vac­cines in record time. That group in­cludes BioN­Tech, Cure­Vac and Mod­er­na in the US. Their tech­nol­o­gy, while yet to pro­duce an ap­proved prod­uct, holds the promise of swift iden­ti­fi­ca­tion of the anti­gens need­ed to come up with an ef­fec­tive way to spur im­mune re­sis­tance to the coro­n­avirus.

If it works, it will rev­o­lu­tion­ize pan­dem­ic pre­pared­ness ef­forts. The whole world is watch­ing what hap­pens next. — John Car­roll

CDC tweaks guid­ance on con­tro­ver­sial coro­n­avirus meds — re­port

The Eu­ro­pean Med­i­cines Agency has cau­tioned against us­ing the pair of malar­ia drugs — chloro­quine and hy­drox­y­chloro­quine — out­side of clin­i­cal tri­als or na­tion­al emer­gency use pro­grams for coro­n­avirus pa­tients, cit­ing the po­ten­tial for se­ri­ous side-ef­fects at high dos­es or when used in com­bi­na­tion with oth­er drugs.

Mean­while, in the mid­dle of March, Pres­i­dent Trump re­port­ed­ly per­son­al­ly pressed fed­er­al health of­fi­cials to make the malar­ia drugs avail­able to treat the new coro­n­avirus. Soon af­ter, the FDA cit­ed lim­it­ed in-vit­ro and anec­do­tal clin­i­cal da­ta to en­dorse the emer­gency use of the two ther­a­pies to treat malar­ia and lu­pus among oth­er dis­or­ders for Covid-19 when clin­i­cal tri­als are not avail­able, or par­tic­i­pa­tion is not fea­si­ble.

Now, the CDC has walked in­to the con­tro­ver­sy. Ac­cord­ing to a re­port by Reuters, the agency hasti­ly re­moved from its web­site atyp­i­cal guid­ance in­form­ing doc­tors on how to pre­scribe hy­drox­y­chloro­quine and chloro­quine. Ini­tial­ly, the CDC web­page, ti­tled In­for­ma­tion for Clin­i­cians on Ther­a­peu­tic Op­tions for Pa­tients with COVID-19, said: “Al­though op­ti­mal dos­ing and du­ra­tion of hy­drox­y­chloro­quine for treat­ment of COVID-19 are un­known, some U.S. clin­i­cians have re­port­ed anec­do­tal­ly” sev­er­al ways to pre­scribe the drugs to treat Covid-19.

A few tiny tri­als out of France and Chi­na have so far yield­ed in­con­clu­sive ev­i­dence on the use of the drugs in pa­tients with Covid-19. The stud­ies and the da­ta that em­anat­ed from them is not peer-re­viewed.

“Why would CDC be pub­lish­ing anec­dotes?” asked Lynn Gold­man, dean of the Milken In­sti­tute School of Pub­lic Health at George Wash­ing­ton Uni­ver­si­ty, in con­ver­sa­tion with the wire agency.

On Wednes­day, the CDC web­site read: “There are no drugs or oth­er ther­a­peu­tics ap­proved by the US Food and Drug Ad­min­is­tra­tion to pre­vent or treat COVID-19.” The trun­cat­ed guid­ance al­so said “hy­drox­y­chloro­quine and chloro­quine are un­der in­ves­ti­ga­tion in clin­i­cal tri­als” for use in coro­n­avirus pa­tients.

Trump has been tout­ing the pair of drugs as Covid-19 treat­ments for weeks now, de­spite cau­tion from his own sci­en­tif­ic ad­vi­sors, in­clud­ing in­fec­tious dis­ease ex­pert and NI­AID chief An­tho­ny Fau­ci who has in­sist­ed that there is no strong sci­en­tif­ic proof back­ing their ef­fi­ca­cy against Covid-19.

Cowen an­a­lyst Yaron Wer­ber, in a note on Wednes­day, sug­gest­ed that giv­en the ex­ist­ing (lim­it­ed) da­ta on hy­drox­y­chloro­quine (HCQ), the po­ten­tial for its use will like­ly be as a pro­phy­lac­tic agent.

“(B)ased on all avail­able da­ta, HCQ is un­like­ly to sig­nif­i­cant­ly im­prove clin­i­cal out­comes for hos­pi­tal­ized pa­tients with COVID-19…we would like­ly al­ready be aware if HCQ was hav­ing a ro­bust ef­fect giv­en its wide use through­out Chi­na, Italy and the U.S. thus far,” he wrote. “The role for HCQ in post-ex­po­sure pro­phy­lax­is ap­pears more promis­ing with many clin­i­cal tri­als cur­rent­ly in­ves­ti­gat­ing this con­cept. The drug has shown ef­fec­tive­ness in vi­ral clear­ance in vit­ro and may pro­vide ben­e­fit if vi­ral repli­ca­tion is im­paired in the very ear­ly stages of ill­ness, be­fore the sys­temic in­flam­ma­to­ry cas­cade takes hold.” — Na­tal­ie Grover

Boston hos­pi­tals as­sem­ble a small Covid-19 study in the front lines of the Covid-19 war

A flu an­tivi­ral from Japan that was rushed in­to use in Chi­na to help fight the first out­break of Covid-19 will now be fea­tured in a small US study — right in one of the cen­tral fronts of the out­break.

Brigham and Women’s Hos­pi­tal, Mass­a­chu­setts Gen­er­al Hos­pi­tal and the Uni­ver­si­ty of Mass­a­chu­setts Med­ical School have be­gun col­lab­o­rat­ing on a Phase II study of favipi­ravir that will re­cruit 50 pa­tients suf­fer­ing from coro­n­avirus, ac­cord­ing to its man­u­fac­tur­er, Fu­ji­film.

This small tri­al comes a lit­tle more than a week af­ter Fu­ji­film launched a more am­bi­tious Phase III in Japan.

Favipi­ravir — sold as Avi­gan in Japan since its ini­tial ap­proval in 2014 — at­tract­ed a lot of at­ten­tion in Chi­na dur­ing the peak of its fight against the new virus. The Chi­nese adopt­ed it af­ter a pair of small stud­ies. And it’s helped shed some ad­di­tion­al lus­ter on Gilead’s remde­sivir, which many be­lieve will work bet­ter us­ing the same mech­a­nism of ac­tion.

As far as the da­ta we’ve seen so far, though, it’s all still a crap­shoot.

The drug in­hibits RNA poly­merase nec­es­sary for in­fluen­za virus repli­ca­tion, which could be use­ful in the Covid-19 war. The ju­ry, though, is still very much out over how ef­fec­tive it may be or whether it can be more than a mar­gin­al treat­ment against a fast-spread­ing virus. — John Car­roll

Biotechs broad­en the spec­trum of an­ti-in­flam­ma­to­ry drugs thrown against Covid-19

With the FDA sig­nalling a will­ing­ness to wave a broad spec­trum of Covid-19 ther­a­pies in­to the clin­ic, two biotechs are pitch­ing in their pipeline stars to tame the in­flam­ma­tion of­ten re­spon­si­ble for dev­as­tat­ing pa­tients.

Bio­haven has the green light to be­gin test­ing in­tranasal vazegepant — its CGRP re­cep­tor an­tag­o­nist de­signed for acute mi­graines — in pul­monary com­pli­ca­tions of the vi­ral in­fec­tion. “This pan­dem­ic is a call to ac­tion for our en­tire in­dus­try,” CEO Vlad Coric said, and Bio­haven with re­searchers at Thomas Jef­fer­son Uni­ver­si­ty to de­vel­op the pro­to­col.

Sim­i­lar­ly, San Diego-based Cal­ciMed­ica has per­mis­sion to study whether its small mol­e­cule drug — which in­hibits cal­ci­um re­lease-ac­ti­vat­ed cal­ci­um (CRAC) chan­nels — can keep pa­tients with se­vere pneu­mo­nia from go­ing on ven­ti­la­tors.

Once they pro­vid­ed da­ta from their Phase II pan­cre­ati­tis tri­al in­volv­ing pa­tients with sys­temic in­flam­ma­tion and hy­pox­ia, as well as an­i­mal da­ta sug­gest­ing CM4620-IE has pro­tec­tive ef­fects on lung in­jury, the FDA ap­proved their IND in less than 10 days, CMO Su­dar­shan Heb­bar told End­points News.

The tri­al will start small with plans to re­cruit 60 pa­tients be­tween Re­gions Hos­pi­tal in St. Paul, Min­neso­ta and Hen­ry Ford Hos­pi­tal in De­troit. Pa­tients will be ran­dom­ized to re­ceive drug or place­bo in a 2:1 ra­tio. — Am­ber Tong

Ra­maswamy’s Data­vant kicks off ef­fort to cre­ate a da­ta repos­i­to­ry for US coro­n­avirus pa­tients

For­get Zoom, health care and soft­ware com­pa­nies are re­port­ed­ly look­ing to col­late da­ta from coro­n­avirus-in­fect­ed pa­tients to study dis­ease trans­mis­sion, iden­ti­fy the most vul­ner­a­ble groups of the pop­u­la­tion and eval­u­ate how ef­fec­tive in­ves­ti­ga­tion­al drugs are.

Vivek Ra­maswamy’s Data­vant — which ag­gre­gates and an­a­lyzes re­al-world med­ical da­ta from a suite of sources to as­sist bio­phar­ma com­pa­nies, providers and pay­ers — be­gan to spear­head one such ef­fort in March, ac­cord­ing to the Wall Street Jour­nal, which cit­ed a per­son fa­mil­iar with the mat­ter.

Health care tech com­pa­nies — Allscripts Health­care So­lu­tions and Change Health­care — have com­mit­ted to do­nat­ing da­ta for the ef­fort, while health in­sur­ance com­pa­ny An­them has been ap­proached to con­tribute med­ical claims da­ta, the re­port said.

Data­vant, which calls the big CRO Parex­el a part­ner, aims to in­clude da­ta about every Covid-19 pos­i­tive pa­tient (ex­clud­ing names or iden­ti­fy­ing de­tails) draw­ing on in­for­ma­tion that em­anates from hos­pi­tals, phar­ma­cies and health in­sur­ers. The reg­istry is be­ing po­si­tioned as a free re­source for gov­ern­ment and aca­d­e­m­ic re­searchers, the WSJ re­port­ed.

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

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Tillman Gerngross (Adagio)

Till­man Gern­gross' Covid-19 an­ti­body moon­shot scores $336M with the help of new ace CFO. Is an IPO next?

Less than a year into its existence, serial biotech entrepreneur Tillman Gerngross’ antibody play Adagio has raced ahead into a pivotal trial for its lead drug for Covid-19 on the back of some very promising preclinical data. Now, crossover investors led by Peter Kolchinsky at RA are rolling up the Brinks truck — and that could spell an IPO in the offing for Adagio.

Adagio has bagged $336 million as part of a Series C round led by RA Capital to advance lead single-shot antibody ADG20 through a pivotal Phase I/II/III trial for the treatment of mild to moderate Covid-19 patients at high risk of infection, the biotech said Monday.

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UP­DAT­ED: New Kaiser analy­sis shows how lim­it­ing price ne­go­ti­a­tions to tar­get­ed drugs may bet­ter fo­cus up­com­ing leg­is­la­tion

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

Covid-19 roundup: Sup­ply chain short­ages lead to half the Mod­er­na dos­es de­liv­ered to Cana­da; J&J shots paused out of con­cern clots would be in­ap­pro­pri­ate­ly treat­ed — re­port

The number of doses of Moderna’s vaccine expected to be delivered to Canada by the end of April has nearly been cut in half, according to the country’s procurement minister Anita Anand.

Between 1 million and 2 million doses of the 12.3 million expected to be delivered in time for the second quarter will be delayed until the third, as Moderna said Friday that shipments to Canada and the UK are behind schedule after a supply chain shortage will delay deliveries.

When is a drug re­al­ly a de­vice? Court knocks down FDA ap­peal in try­ing to sort that grey area

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.

Q1: A flood of in­vestor cash drove biotech's num­bers to new record highs, and the tor­rent of cash is mov­ing up­stream fast

If you thought biotech was booming last year, wait until you get a load of the numbers from Q1 2021.

On virtually every level, with one exception, the money engine was working around the clock in the first 3 months of this year. Venture capital has reached such a fever peak that the average B round now weighs in at an average mega-weight value of $100 million. The money flow is also finding its way to the mouth of the R&D river, where discovery work now merits the big bucks instead of cautionary seed funds.

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Jami Rubin (EQRx)

Ja­mi Ru­bin, once fa­bled for grilling bio­phar­ma ex­ecs, de­camps to head fi­nance at drug pric­ing dis­rupter

As Goldman Sachs’ top pharmaceutical analyst, Jami Rubin was known for asking the tough questions. Now, as she takes the lead on EQRx’s mission to rewrite the rules of drug pricing, we’ll see how good her answers are.

Rubin made the jump to biotech on April 5, becoming EQRx’s new CFO, the company said Monday. She’s coming from PJT Partners, where she’s been a partner providing strategic guidance for biotech and pharmaceutical companies for the last couple years. With EQRx’s recent $500 million Series B round in the books, it wouldn’t be a surprise if she was already lining up a public debut.

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