George Painter in his Atlanta Lab (DRIVE)

Cre­ative or un­eth­i­cal? Re­searchers’ Covid-19 Go­FundMe cam­paign spurs crit­ics

It had been a busi­ness pro­fes­sor’s idea, back be­fore Christ­mas, when the most in­fec­tious threat fac­ing the US was a po­ten­tial­ly harsh flu sea­son. DRI­VE In­no­va­tions, a non-prof­it biotech found­ed by a pi­o­neer­ing HIV re­searcher at Emory Uni­ver­si­ty, was look­ing for new ways of rais­ing mon­ey and draw­ing press. Charles Goetz, who stud­ied en­tre­pre­neur­ship, sug­gest­ed Go­FundMe.

What ap­peared on­line three months lat­er had the smooth­ness of a VC pitch and the com­mand­ing lilt of a call to arms. That was the week the coro­n­avirus moved from far-flung threat to loom­ing men­ace for many Amer­i­cans, when Vice Pres­i­dent Pence was named to head the White House task­force, the stock mar­ket suf­fered its worst fall since 2008 and the first cas­es of un­known ori­gin ap­peared on the West Coast — a sign the virus was spread­ing with­in the US. Go­FundMe put DRI­VE’s cam­paign as the “Ur­gent Cause” at the cen­ter of its home­page. They promised a tan­ta­liz­ing an­ti­dote.

“What if you could help fund a cure for coro­n­avirus?” DRI­VE asked.

Their page ex­plained they had de­vel­oped EI­DD-2801, the “most promis­ing po­ten­tial an­tivi­ral ther­a­peu­tic for coro­n­avirus that can be tak­en as a pill,” but need­ed fund­ing to get to a hu­man tri­al. A three-minute video weaved archival video to stitch a thread be­tween the 1918 Span­ish Flu, the 2014–2016 Ebo­la out­break, and Covid-19. It ex­plained more pan­demics are com­ing, pan­demics their drug could shield against. The fundrais­ing goal was $5 mil­lion.

“We’re un­der at­tack,” a deep male voice said. “We need a weapon.”

The un­usu­al cam­paign, its or­ga­niz­ers told End­points News, point­ed to the dif­fi­cul­ty re­searchers have find­ing fund­ing in a cri­sis, a con­cern echoed by oth­er sci­en­tists who turned to crowd­fund­ing or in­di­vid­ual donors for their coro­n­avirus re­search.

It would al­so baf­fle some bioethi­cists, who saw a non-prof­it try­ing to prof­it off mass pan­ic and toss­ing around the word “cure” for a drug that had nev­er been put in hu­mans.

Art Ca­plan

“I think it’s very un­eth­i­cal to go out to peo­ple who are wor­ried and pan­icked, who are worked up, and say how would you like to put mon­ey in our kit­ty to pur­sue a re­search idea that is hy­per un­like­ly ever to work,” Art Ca­plan, found­ing head of New York Uni­ver­si­ty School of Med­i­cine’s Di­vi­sion of Med­ical Ethics, told End­points.

“And it al­so gives a sug­ges­tion that if the biotech found an an­swer — by some mir­a­cle — they know how to man­u­fac­ture it in big dos­es, which they wouldn’t,” he added. “They would have to sell it to some­one else and they would have to get it ap­proved and make mil­lions or bil­lions of dos­es. There’s noth­ing com­ing soon.”

But just how un­like­ly would EI­DD-2801 work? The drug emerged from some of the re­searchers that de­vel­oped Gilead’s remde­sivir, the first an­tivi­ral to cap­ture the world’s hopes. And last week, af­ter Sci­ence Trans­la­tion­al Med­i­cine pub­lished DRI­VE’s pre­clin­i­cal work on SARS-CoV-2, the virus that caus­es Covid-19, the FDA ap­proved a Phase I clin­i­cal tri­al test­ing their pill in in­fect­ed pa­tients. As new da­ta cloud remde­sivir’s fu­ture, it could emerge as one of the most promis­ing an­tivi­rals weapons against Covid-19 — if it can make it in time.

“EI­DD-2801 is a great ad­vance,” Vi­neet Men­ach­ery, a coro­n­avirus re­searcher not af­fil­i­at­ed with the drug, told End­points in an email fol­low­ing the Sci­ence study. “The dis­ad­van­tage of [EI­DD-2801] is that it has not gone in­to hu­man tri­al.”

A plan to save the world

In the 10 years since its found­ing, Go­FundMe has emerged as both Amer­i­can life­line and punch­line. Its plat­form let des­per­ate pa­tients raise thou­sands for treat­ments and med­ical bills, but to some, it al­so ar­tic­u­lat­ed a kind of lit­er­ary dystopia, a world in which health­care went to who­ev­er told the best sto­ry. “Health ex­perts wor­ry coro­n­avirus will over­whelm Amer­i­ca’s Go­FundMe sys­tem,” The Onion cracked in March.

Oc­ca­sion­al­ly that med­ical care meant ba­sic re­search or an aca­d­e­m­ic’s tri­al. Al­most nev­er, though, has a biotech used the plat­form. In­dus­try costs are too high and oth­er fund­ing sources too ap­par­ent.

DRI­VE In­no­va­tions was an atyp­i­cal biotech. It was found­ed in 2013 by Den­nis Li­ot­ta, a pro­fes­sor of or­gan­ic chem­istry at Emory Uni­ver­si­ty who in the ear­ly ‘90s in­vent­ed emtric­itabine, a chem­i­cal that blocks HIV from copy­ing it­self and is now used in sev­er­al med­ica­tions, in­clud­ing Gilead’s best-sell­ing De­scovy and Tru­va­da. In the ear­ly years of the AIDS cri­sis, Li­ot­ta ex­plained in a 2016 Tedx Talk, drug­mak­ers had ig­nored the virus, in part be­cause it killed pa­tients so quick­ly com­pa­nies didn’t think they could turn a prof­it. A re­lat­ed dy­nam­ic held true for virus­es like dengue, Zi­ka, and chikun­gun­ya — dev­as­tat­ing dis­eases that re­ceived lit­tle at­ten­tion from drug­mak­ers be­cause they af­fect­ed poor­er re­gions where com­pa­nies were un­like­ly to re­coup the cost of re­search and de­vel­op­ment.

The new ven­ture, seed­ed with $10 mil­lion in HIV drug roy­al­ties, would re­search com­pounds for sin­gle-strand­ed RNA virus­es, a broad cat­e­go­ry of de­cep­tive­ly sim­ple pathogens that, by their es­ti­mate, ac­count­ed for 80% of the world’s “vi­ral bur­den.” Ex­cept in­stead of stop­ping at lab test­ing and then li­cens­ing it — as most aca­d­e­mics do — DRI­VE would take it through a proof-of-con­cept tri­al or fur­ther, de-risk­ing the in­vest­ment for a larg­er biotech or phar­ma com­pa­ny, who could then bring it to mar­ket.

“We’d bring it to what we called a val­ue in­flec­tion point,” Li­ot­ta told End­points. “We were try­ing to be ahead of the curve, try­ing to an­tic­i­pate that one of these virus­es might cre­ate a prob­lem.”

Li­ot­ta tapped for­mer Glax­co Well­come vi­rol­o­gy ex­ec­u­tive George Painter as CEO and se­r­i­al biotech ex­ec­u­tive David Per­ry­man as COO. The idea filled a blindspot those around biotech had long rec­og­nized but of­ten felt pow­er­less to ad­dress.

Alex Kar­nal

“I think the non-prof­it ap­proach is great,” Alex Kar­nal, man­ag­ing di­rec­tor of the health­care VC firm Deer­field Part­ners, told End­points. “One of the prob­lems for our busi­ness is that we re­quire our rate-of-re­turn. To the ex­tent those dol­lars can be do­nat­ed, and they don’t re­quire that same re­turn, then the hur­dle for mov­ing it for­ward has been low­ered.”

The com­pa­ny be­gan with dis­eases that would draw gov­ern­ment con­tracts. That meant Venezue­lan Equine En­cephali­tis Virus, or VEEV, a po­ten­tial­ly fa­tal in­fec­tion that the So­vi­et Union tried to weaponize dur­ing the Cold War and that the gov­ern­ment was par­tic­u­lar­ly in­ter­est­ed in hav­ing a de­fense against. They found a promis­ing com­pound in a drug Li­ot­ta’s long­time col­lab­o­ra­tor, Ray­mond Schi­nazi, once test­ed on he­pati­tis called β-D-N4-hy­drox­y­cy­ti­dine, or NHC. (A sec­ond ear­ly com­pound, for he­pati­tis B, was spun out in­to its own biotech, called An­tios).

The holy grail, though, was al­ways a drug that worked on 3, 4 or more virus­es. That would give it com­mer­cial po­ten­tial and, in the­o­ry, make it a bet­ter de­fense against nov­el in­fec­tions. They sent NHC to the NIH and else­where to screen against a mul­ti­tude of virus­es. Last Oc­to­ber, they pub­lished the re­sults for flu in Sci­ence Trans­la­tion­al Med­i­cine.

In fer­rets, NHC killed the flu by a process they called “er­ror cat­a­stro­phe” and didn’t con­fer re­sis­tance — qual­i­ties that gave it promise against the flu strains that hu­mans have lit­tle im­mu­ni­ty to and that can thus cause pan­demics like the 2009 h1n1 out­break. The same year, the NIH grant­ed up-to $15.9 mil­lion to run a hu­man tri­al.

David Per­ry­man

Still, that was promised mon­ey, not pock­et mon­ey. And six years in, Per­ry­man found him­self fac­ing a flaw im­plic­it in DRI­VE’s mod­el. Gov­ern­ment grants could be plen­ti­ful, but they didn’t pay for all the nuts-and-bolts op­er­a­tions of a biotech — they didn’t pay for his job or patents or oth­er mem­bers of his staff. That’s when, in De­cem­ber, Charles Goetz sug­gest­ed they try Go­FundMe, a way of get­ting funds di­rect­ly and po­ten­tial­ly at­tract­ing larg­er donors.

“They pay for sci­ence, they don’t pay for peo­ple run­ning things,” Per­ry­man said of gov­ern­ment con­tracts. “We want­ed to try some­thing that was po­ten­tial­ly help­ful, new and worst case sce­nario raised the aware­ness of what we’re try­ing to do.”

The cam­paign would not on­ly be about flu but about their broad­er mis­sion of tack­ling ne­glect­ed in­fec­tious dis­eases. Per­ry­man re­mem­bers sit­ting at a meet­ing lat­er that month to set up the page and tick­ing off the virus­es the com­pound has worked against: Ebo­la, the flu, SARS.

“The peo­ple in the room looked at me and went, what is SARS?” Per­ry­man said. “I said, ‘You don’t re­mem­ber what SARS was? My God, are you kid­ding me?”

Den­nis Li­ot­ta giv­ing a Tedx Talk (Tedx via Youtube)

Click on the im­age to see the full-sized ver­sion

“The US gov­ern­ment is left scram­bling”

SARS-CoV-2, epi­demi­ol­o­gists now be­lieve, had al­ready be­gun spread­ing when that meet­ing took place. In ear­ly Feb­ru­ary, when the mag­ni­tude of the threat be­came clear, DRI­VE said they would be­gin test­ing NHC against the new virus. It was in some ways the pan­dem­ic they had been prepar­ing for.

“Most an­tivi­rals are what we call one-bug, one-drug com­pounds, so this one was pret­ty unique,” Li­ot­ta told End­points. “Maybe we’re in the right place at the right time.”

DRI­VE was buoyed by the same ev­i­dence that buoyed many of the ear­ly drug and vac­cine ef­forts: Da­ta on oth­er coro­n­avirus­es. A month af­ter their flu study, they pub­lished a study in Vi­rol­o­gy that showed the drug was po­tent against MERs and SARs. “The emer­gence of coro­n­avirus­es (CoVs) in­to hu­man pop­u­la­tions from an­i­mal reser­voirs has demon­strat­ed their epi­dem­ic ca­pa­bil­i­ty, pan­dem­ic po­ten­tial, and abil­i­ty to cause se­vere dis­ease,” the au­thors wrote. “How­ev­er, no an­tivi­rals have been ap­proved to treat these in­fec­tions.”

Un­like Gilead or Mod­er­na, though, DRI­VE said they didn’t have cash run­ways to piv­ot to a new dis­ease. Their grants were ear­marked for flu; use it for oth­er re­search — lose the grant. The NIH soon opened an “ur­gent award” sys­tem where grantees could ap­ply to redi­rect their funds to Covid-19, and the Bio­med­ical Ad­vance Re­search Agency (BAR­DA) called for ap­pli­ca­tions. DRI­VE said they were ap­ply­ing for gov­ern­ment funds but that process funds was in­her­ent­ly slow.

In an out­break, “the US gov­ern­ment is left scram­bling: where’s the mon­ey go­ing to come from?” Per­ry­man said. “What bud­get? What al­lo­ca­tions? There’s a gap of time.”

The Go­FundMe, Per­ry­man said, could fill that gap. The cam­paign went live on Feb­ru­ary 27 and quick­ly raised over $10,000, bol­stered by a $5,000 per­son­al do­na­tion from Li­ot­ta. The cam­paign, though, was con­ceived in De­cem­ber. Asked if the switch to coro­n­avirus-fo­cused mar­ket­ing had been an ef­fort to draw peo­ple in­to a cam­paign that had been planned be­fore any­one knew a nov­el coro­n­avirus ex­ist­ed, Per­ry­man said, “Yeah, maybe. But it’s more about this com­pound and the op­por­tu­ni­ties that came out of it.”

Maria Bot­tazzi

Yet oth­er re­searchers have echoed their con­cerns. Pe­ter Hotez, found­ing dean of Bay­lor’s Na­tion­al School for Trop­i­cal Med­i­cine, built a tri­al-ready SARS vac­cine in 2016 be­fore NIH fund­ing went dry. As he strug­gled to find mon­ey to test it on the new coro­n­avirus, he told End­points he was sug­gest­ed to start a Go­FundMe. “A shame though that you have to go to that re­course be­cause there is an in­abil­i­ty of se­cur­ing funds by oth­er means,” Maria Bot­tazzi, Hotez’s col­lab­o­ra­tor, told End­points in an email last month about DRI­VE’s cam­paign. “We still have not been suc­cess­ful in get­ting fund­ing to move our vac­cine out of the freez­er.”

David Boul­ware, a trop­i­cal dis­ease re­searcher at the Uni­ver­si­ty of Min­neso­ta, raised over $15,000 on Go­FundMe for a hy­drox­y­chloro­quine tri­al in March, be­fore the uni­ver­si­ty stepped in, agree­ing to fund the tri­al and re­im­burse the donors.

“We sub­mit­ted for an NIH grant, but how long would that take?” he told End­points. “It was un­clear.”

Mitchell Kro­nen­berg

Mitchell Kro­nen­berg, CSO of the La Jol­la In­sti­tute for Imm­nunol­o­gy, didn’t com­ment on the Go­FundMe cam­paign or DRI­VE but told End­points his re­search cen­ter had to look for new ways of rais­ing mon­ey as they switched most of their op­er­a­tions to fo­cus on Covid-19.

“It just takes longer to get fed­er­al mon­ey,” Kro­nen­berg said. “They’re do­ing things to speed things up, but it’s still — the NIH is a $40 bil­lion agency with thou­sands and thou­sands of em­ploy­ees … they can’t move the way an in­di­vid­ual donor would.”

But was Go­FundMe the right way to move? Some pub­lic health ex­perts and ethi­cists were skep­ti­cal, if not out­right fu­ri­ous. While agree­ing the fed­er­al re­sponse could be slow and im­per­fect, they ar­gued that us­ing Go­FundMe — an in­creas­ing­ly com­mon phe­nom­e­non among aca­d­e­mics, if not biotechs — pri­or­i­tized the best sto­ry over the best sci­ence. At its worst, the cam­paign was a moral night­mare, a grab for funds from civil­ians afraid of a mys­te­ri­ous virus and pos­si­bly un­aware of the high risks of biotech. There was no men­tion on the page that na­ture of drug de­vel­op­ment meant the drug would like­ly fail, as for-prof­it biotech’s IPO doc­u­ments might.

“It down­grades the de­tailed un­der­stand­ing of sci­ence that has been such a main­stay of Amer­i­can sci­ence,” Jairam Lin­gap­pa, an epi­demi­ol­o­gist at the Uni­ver­si­ty of Wash­ing­ton who worked at the CDC dur­ing the SARS epi­dem­ic, told End­points.

Ca­plan took is­sue with DRI­VE’s use of the word “cure,” when they on­ly had done an­i­mal stud­ies. He said they were about as close to a cure for pan­demics as some­one who cleared a tu­mor in a mouse is to cur­ing can­cer.

Leigh Turn­er

“They don’t do a great job telling po­ten­tial donors that there’s not a good chance there’s go­ing to be an ef­fec­tive ther­a­py that emerges from this,” Leigh Turn­er, a bioethi­cist at the Uni­ver­si­ty of Min­neso­ta whose pub­lished on pa­tient-run Go­FundMe cam­paigns, told End­points. “It’s a com­mon rhetor­i­cal route on crowd­fund­ing. This is not a place where you go for risks and ben­e­fits and low like­li­hood.”

Per­ry­man said he took the op­po­site view. He com­pared DRI­VE to the Red Cross, pro­vid­ing both aid in a cri­sis and a way for peo­ple who might oth­er­wise feel help­less to give back. His view was bol­stered in part by donors, many of whom were con­nect­ed with the Uni­ver­si­ty, which sent out alum­ni emails and reached out to spe­cif­ic groups about the cam­paign, and who told End­points they gave be­cause they trust­ed Emory or Per­ry­man and they didn’t care if the drug ul­ti­mate­ly worked or not. “I didn’t give be­cause I thought this group might have an ef­fec­tive treat­ment for Covid, I be­lieve in what they’re do­ing,” Jody Coo­ley, a Geor­gia lawyer and a friend of Per­ry­man’s, who gave $100 told End­points.

Lau­rie Zoloth, a Uni­ver­si­ty of Chica­go ethi­cist, called the cam­paign a “clever” in­no­va­tion amid a fed­er­al sys­tem that failed to ad­dress the cri­sis, but one that had to be ful­ly test­ed. Aaron Kessel­heim, a Har­vard bioethi­cist who spe­cial­izies in phar­ma, backed Emory. He said it’s peo­ple’s choice what char­i­ties they want to do­nate to, and Li­ot­ta and the uni­ver­si­ty’s his­to­ry with in­fec­tious dis­eases gave the cam­paign le­git­i­ma­cy. He took lit­tle is­sue with the word­ing.

“I feel like the phar­ma­ceu­ti­cal in­dus­try us­es this kind of lan­guage a lot,” Kessel­heim told End­points. “If there’s any­body out there — this guy’s got a fan­tas­tic track record.”

The mol­e­c­u­lar struc­ture for EI­DD-2801. If a virus us­es it to build its genome, it can kill the virus by “er­ror cat­a­stro­phe”

Click on the im­age to see the full-sized ver­sion

“Er­ror Cat­a­stro­phe”

Just how like­ly or un­like­ly is it that EI­DD-2801 will work?

For most of mod­ern med­ical his­to­ry, our best treat­ment for virus­es has been to avoid them. Vac­ci­na­tion knocked out rav­aging pathogens from small­pox to po­lio, while en­vi­ron­men­tal in­ter­ven­tions end­ed malar­ia in much of the de­vel­oped world. Over the years, tai­lored drugs were de­vel­oped for HIV and he­pati­tis but what’s re­mained elu­sive have been broad-spec­trum an­tivi­rals: drugs that can treat a host of new and old virus­es the way many an­tibi­otics can treat bac­te­r­i­al in­fec­tions.

That’s the gap DRI­VE tried to fill and when Covid-19 broke out in De­cem­ber, there were es­sen­tial­ly two ex­per­i­men­tal drugs that fit the bill: remde­sivir, which af­ter some ear­ly anec­do­tal ev­i­dence caught the hopes of much of the world, and EI­DD–2801.

Stan­ley Perl­man

“This drug seems to work well,” Stan­ley Perl­man, who has stud­ied coro­n­avirus­es for 38 years at the Uni­ver­si­ty of Iowa and who was not in­volved in any EI­DD-2801 study, told End­points.

The list of coro­n­avirus re­searchers is short. EI­DD-2801 was de­vel­oped for coro­n­avirus­es at the same Van­der­bilt and Uni­ver­si­ty of North Car­oli­na labs that, ear­li­er in the decade, iden­ti­fied remde­sivir. And it op­er­ates by a sim­i­lar mech­a­nism. Both are nu­cle­o­side ana­logues – ba­si­cal­ly de­coy ver­sions of the mol­e­cules RNA virus­es use to write their genome as they repli­cate. Remde­sivir acts like a cap; the virus grabs it and sud­den­ly can’t add any more mol­e­cules, its genome sealed shut. EI­DD-2801 is like gunk in the works; the virus can keep adding new bases but the de­coy mol­e­cule forces ram­pant mu­ta­tions down the genome un­til the virus reach­es what re­searchers call “er­ror cat­a­stro­phe.” It blows a gas­ket.

Mark Deni­son

“Those mu­ta­tions dam­age the RNA, they dam­age the vi­ral pro­tein, they dam­age the virus’ abil­i­ty to in­fect and to grow,” Mark Deni­son, the Van­der­bilt coro­n­avirus re­searcher who helped de­vel­op both drugs, told End­points. “This is def­i­nite­ly com­pa­ra­ble. It has sim­i­lar po­ten­cy and ef­fi­ca­cy against mul­ti­ple coro­n­avirus­es.”

Be­cause the en­zyme that writes the genome, RNA poly­merase, is sim­i­lar across many virus­es, both drugs held po­ten­tial to treat a long list of in­fec­tions.

What has Deni­son, Perl­man and oth­ers par­tic­u­lar­ly ex­cit­ed is the pack­ag­ing: NHC comes as a pill. That means it could be giv­en ear­li­er than remde­sivir, which is giv­en as a hos­pi­tal in­fu­sion, and to more peo­ple, po­ten­tial­ly even as a pro­phy­lac­tic for health­care work­ers and those at risk. It al­so seems to get around the virus’ “proof­read­ing” abil­i­ty to spot and pre­vent copy­ing of de­coy mol­e­cules. Last­ly, it showed ac­tiv­i­ty against coro­n­avirus­es re­sis­tant to remde­sivir, open­ing up the po­ten­tial for com­bi­na­tion treat­ments.

There are no mouse mod­els yet for nov­el coro­n­avirus. To show pre­clin­i­cal da­ta that could bur­nish a clin­i­cal tri­al, DRI­VE, UNC and Van­der­bilt re­searchers test­ed NHC against SARS and MERS in mice, and against SARS-CoV-2, SARS, MERS and zoonot­ic coro­n­avirus­es in an­tivi­ral as­says and hu­man lung cell cul­tures. They post­ed their re­sults in a preprint on March 20, and Sci­ence Trans­la­tion­al Med­i­cine pub­lished the study on April 6 – the first the jour­nal has pub­lished on a po­ten­tial Covid-19 drug. Im­mu­nol­o­gists raved.

“I im­plore them to con­tin­ue their ef­forts and we need new ap­proach­es that are clin­i­cal­ly vi­able (pill form),” Gene Olinger, an im­mu­nol­o­gist who has worked on coro­n­avirus­es and Ebo­la and prin­ci­ple ad­vi­sor for MRIGlob­al, told End­points in an email. “We need more an­tivi­rals in our ar­se­nal against virus­es and broad spec­trum are more valu­able.”

The DRI­VE Lab (Emory)

Where’d the mon­ey go?

By the time Sci­ence Trans­la­tion­al Med­i­cine pub­lished the study, DRI­VE had li­censed NHC to Ridge­back Bio­ther­a­peu­tics, the tiny hus­band-and-wife biotech that in 2018 li­censed mAb114 from the NIH. Last Au­gust, that drug was one of two that beat out remde­sivir in a four-arm tri­al against Ebo­la, the in­fec­tion the Gilead an­tivi­ral had orig­i­nal­ly been built to treat.

At the time, DRI­VE told End­points they would con­tin­ue the crowd­fund­ing cam­paign as they still need­ed the re­sources. But af­ter the FDA ap­proved a tri­al on April 7, Ridge­back CEO Wendy Hol­man told End­points she “had no knowl­edge” of a Go­FundMe cam­paign and that Ridge­back “is fund­ing the Phase I tri­als com­plete­ly on our own.” DRI­VE did not re­spond to sub­se­quent re­quests for com­ment.

For all of im­mu­nol­o­gists’ ex­cite­ment over NHC, many be­lieve it could prove more ef­fec­tive against fu­ture out­breaks than this one. Both remde­sivir and NHC suf­fer from a sim­i­lar flaw: They’re more ef­fec­tive the ear­li­er they’re giv­en. That means by the time a pa­tient is in se­vere con­di­tion – the stage at which remde­sivir has been giv­en so far – it may be too late for the an­tivi­ral to help. A pill would be eas­i­er to give ear­ly in the dis­ease, but that ap­proach pos­es prob­lems for an in­fec­tion that most peo­ple re­cov­er from and for a drug that has no pre­built man­u­fac­tur­ing base.

“You’d be treat­ing 85 out of 100 peo­ple who are go­ing to re­cov­er by them­selves with­out any par­tic­u­lar ther­a­py,” Perl­man said. “That be­comes a huge – not waste, but a huge use of re­sources.”

Vi­neet Men­ach­ery

Remde­sivir, Men­ach­ery point­ed out, had al­ready cleared safe­ty stud­ies be­fore the out­break. Al­though the pre­clin­i­cal work showed no safe­ty is­sues, NHC is a mu­ta­gen – a drug that caus­es ge­net­ic mu­ta­tions – and thus could have off-tar­get ef­fects, Men­ach­ery said. Olinger echoed those con­cerns. Al­though Li­ot­ta said the drug “has a very good safe­ty pro­file,” there’s no time­line on when NHC might clear such a tri­al. Mean­while oth­er drugs tar­get­ed for Covid-19, such as an­ti­bod­ies from Re­gen­eron and Vir, are ex­pect­ed to be in the clin­ic by ear­ly sum­mer and emer­gency vac­cines could be avail­able for health­care work­ers by Fall.

“For fu­ture out­breaks, NHC may be an ide­al drug to stock­pile,” Men­ach­ery said. “How­ev­er, with the re­main­ing bar­ri­ers, it is not clear if it will be use­ful in time to deal with Covid-19.”

It’s un­clear but doubt­ful that the Go­FundMe helped NHC reach this point. DRI­VE raised on­ly a tiny frac­tion of the $5 mil­lion goal and at­tract­ed no ma­jor cor­po­rate donors – al­though Dol­ly Par­ton did do­nate $1 mil­lion to Van­der­bilt, where Deni­son hous­es his lab and vac­cine work is un­der­way. In ear­ly March, Roy More­jon, founder of the crowd­fund­ing con­sult­ing agency En­ven­tys Part­ners, told End­points that DRI­VE’s cam­paign nev­er looked promis­ing. Its video tried to rouse or scare, but lacked the hu­man in­ter­est – the sick pa­tient fac­ing ru­inous bills – that have be­come the hall­mark of a suc­cess­ful Go­FundMe.

Otel­lo Stam­pac­chia

Otel­lo Stam­pac­chia, founder of the VC Omega Funds, took a more op­ti­mistic view for the cam­paign at the time, say­ing he had nev­er seen any­thing like it, and that with the im­pact the virus had al­ready had on places like his home coun­try of Italy, a cam­paign could strike a chord.

“I think it’s quite pos­si­ble that some­one could raise tens and tens of mil­lions on Go­FundMe just be­cause its su­per-top­i­cal right now,” he said.

The count, as of this writ­ing, was $38,136 – or 00.7% of their $5 mil­lion goal. Do­na­tions have slowed to a trick­le. The last one was from Anony­mous for $500, adding to over $1,500 raised since they li­censed the drug away on March 23.

“May God bless your ef­forts to speed­i­ly de­vel­op a cure,” Geral­dine White wrote in a do­na­tion note two days lat­er. She gave $100.

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

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Like the flu vac­cine every year, the FDA could move quick­ly on a vari­ant-tar­get­ed Covid vac­cine

In the same way that the FDA signs off on flu vaccines every year without requiring large clinical trials to measure their efficacy, the FDA may employ a similar strategy in authorizing variant-focused versions of the mRNA vaccines.

As the world braces for more data on the latest variant Omicron, which may reduce vaccine efficacy, top vaccine developers like Moderna and Pfizer-BioNTech have promised they can pull together a new vaccine targeted against a specific Covid variant in about 100 days. Since Omicron emerged last week, Pfizer-BioNTech, Moderna and J&J have all said they’ve begun work on Omicron-specific vaccines, if needed.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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More man­u­fac­tur­ing is­sues: Fen­nec preps for sec­ond CRL for po­ten­tial hear­ing loss drug

Shares of Fennec Pharmaceuticals stock were cut almost in half early Monday as the company said manufacturing issues caused another FDA rejection of its reformulated version of sodium thiosulfate, which is intended to help kids who lose hearing due to chemo treatment.

The biotech had resubmitted an NDA for the drug to treat platinum-based, chemo-related ototoxicity in young children earlier this year. The first NDA was denied by the FDA last year, with the agency citing manufacturing issues with the biotech’s supplier.

Róbert Wessman, Alvogen CEO

Biotech bil­lion­aire Róbert Wess­man en­gi­neers $450M deal for Alvo­gen sub­sidiaries

Alvogen is handing off two of its subsidiaries to CEO Róbert Wessman and his healthcare investment firm Aztiq — who has now tied the two together in a massive biobucks deal.

In an alliance with Thailand’s PTT Group subsidiary Innobic, the two companies signed an agreement last week to buy a 100% stake in Alvogen Emerging Market Holdings Limited (AEMH) for $475 million from Alvogen, buying out shareholders such as CVC Capital Partners and Singapore’s Temasek. And now, the group is the majority shareholder of Alvogen’s former Asian subsidiary Lotus Pharmaceuticals and the only shareholder of Alvogen Malta, the owner of B2B pharma Adalvo.

Shankar Musunuri, Ocugen CEO

Ocu­gen hits an­oth­er snag in bring­ing its Bharat Biotech-part­nered Covid-19 vac­cine to the US

Back in July, Ocugen and Bharat Biotech unveiled some Phase III data suggesting their Covid-19 vaccine, Covaxin, was 77.8% effective at preventing overall disease in India. They’re now looking to launch an immuno-bridging study to see if those numbers hold up in the US — but on Friday, the FDA said not so fast.

Regulators have placed a clinical hold on the partners’ IND, Ocugen shared on Friday. Ocugen’s stock $OCGN sank 9% on the news Friday, but was up almost 7% in pre-market trading on Monday.

Covid-19 roundup: As Omi­cron spreads, African biotech re­port­ed­ly close to re­pro­duc­ing Mod­er­na's vac­cine, while WHO cre­ates pan­dem­ic pre­ven­tion body

The emergence of the Omicron variant over the holiday has reignited the focus on vaccine equity, and in its efforts to bring more shots to Africa, one South African biotech is reportedly close to reproducing Moderna’s mRNA shot.

Afrigen Biologics and Vaccines is speeding toward its pivotal trials, the Washington Post reported Sunday, though it’s doing so without Moderna’s recipe. The biotech reportedly has finished sequencing the Moderna vaccine and plans to soon compare its own recreation to Moderna’s jab.