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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Petro Terblanche, Afrigen Biologics managing director (Kristin Palitza/picture-alliance/dpa/AP Images)

WHO-backed Afrigen plans a charge to­ward the clin­ic with Africa's first Covid-19 vac­cine

NEW YORK — When vaccines from high-income countries did not arrive in Africa, a local WHO-backed company decided to take the matter into its own hands.

The South Africa-based company Afrigen Biologics and Vaccines has developed the continent’s first mRNA Covid-19 vaccine that will enter clinical trials in early 2023. Afrigen developed the shot by copying publicly available sequencing information about Moderna’s shot after the biotech and Pfizer refused assistance.

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