A trop­i­cal dis­ease doc­tor turns his sights on the coro­n­avirus

If all goes well, hun­dreds of health­care work­ers across the coun­try will soon get a pack­age at their door con­tain­ing a pack­et of 65-year-old pills and a set of in­struc­tions.

The pills con­tain ei­ther an an­ti-malar­i­al med­ica­tion called hy­drox­y­chloro­quine or place­bo, and the in­struc­tions con­tain the pro­to­col for the Unit­ed States’ fourth coro­n­avirus clin­i­cal tri­al. The brain child of a trop­i­cal dis­ease re­searcher well ac­quaint­ed with malar­ia, the study could soon tell us if there’s a way to help pro­tect those most ex­posed to the virus from falling ill.

David Boul­ware

“This is not what I do on a day-to-day ba­sis,” Uni­ver­si­ty of Min­neso­ta pro­fes­sor David Boul­ware, who nor­mal­ly works on HIV and tu­ber­cu­lo­sis menin­gi­tis, told End­points News. “But this is such a pub­lic health cri­sis that some­one had to do some­thing.”

Boul­ware be­gan plan­ning the tri­al last week as the US out­break be­gan. Hy­drox­y­chloro­quine and its ana­log, chloro­quine, have been used for decades to treat malar­ia — turn­ing up the pH in cells and in­ter­fer­ing with the pro­teins the virus us­es for in­fec­tions. Chi­nese doc­tors turned to it ear­ly in the coro­n­avirus out­break in their scram­ble for po­ten­tial ther­a­pies, and in the last few days, as the glob­al need for treat­ments has sharp­ened and as enough time has passed for patch­es of da­ta to come out, the drug has emerged as a lead­ing can­di­date to treat Covid-19.

The World Health Or­ga­ni­za­tion said yes­ter­day it would be one of four drugs they test in a glob­al, mul­ti-arm study. Ear­ly da­ta from a 24-per­son non-ran­dom­ized study in France sug­gest­ed it might be vast­ly su­pe­ri­or to stan­dard of care in clear­ing the virus (the full da­ta com­pli­cat­ed mat­ters). It’s gar­nered pro­po­nents both in Na­ture and on Twit­ter.

Boul­ware’s study, though, takes a dif­fer­ent ap­proach than most of the hand­ful of hy­drox­y­chloro­quine clin­i­cal tri­als un­der­way world­wide — and a dif­fer­ent de­sign, he said, than he or the FDA had ever seen.

“We had a call with FDA this morn­ing and they were like, ‘yeah this is kind of in­ter­est­ing, yeah we’ve nev­er seen this be­fore,’” Boul­ware said.

Rather than treat­ing pa­tients who are al­ready sick and hos­pi­tal­ized, the study is test­ing the drug as post-ex­po­sure pro­phy­lax­is, i.e. try­ing to pre­vent in­fec­tion in those who have been ex­posed but have not yet shown symp­toms. It could po­ten­tial­ly pro­vide a method to help pro­tect the most vul­ner­a­ble, par­tic­u­lar­ly health­care work­ers. An Ox­ford team is try­ing a sim­i­lar idea, al­though not all coro­n­avirus ex­perts see po­ten­tial.

“I think that this drug is not use­ful,” Stan­ley Perl­man, who has stud­ied coro­n­avirus­es for 38 years at the Uni­ver­si­ty of Iowa, wrote to End­points in an email.

To get the drug to places in the US where the out­break is on­go­ing and ex­po­sure is high­est, Boul­ware chose to run the study es­sen­tial­ly on­line and through FedEx.

There is no tri­al site. Health­care work­ers and oth­ers who have been ex­posed in the last 3 days can sign up on­line. They’ll re­ceive the pack­age at their door by 10:30 am the next day. The reg­i­men lasts 5 days, at which point there’s an on­line sur­vey. An­oth­er sur­vey is tak­en at the end of 14 days. If you’re symp­tom-free, you’re done. If you have symp­toms, the re­searchers send an­oth­er sur­vey ask­ing if you’ve been hos­pi­tal­ized and, if so, the de­tails of that hos­pi­tal stay, and if you have a pend­ing lab test.

Boul­ware pub­li­cized the treat­ment on Twit­ter and oth­er so­cial me­dia. As of this morn­ing, 70 vol­un­teers had en­rolled from more than a dozen states. If they en­roll­ment is quick and the drug is very ef­fec­tive, they could have an an­swer in a cou­ple weeks, Boul­ware said.

“We’re try­ing to re­al­ly en­roll peo­ple at very high risk of trans­mis­sion, so the tri­al can be done quick­ly with the small­est num­ber of peo­ple en­rolled,” Boul­ware said. “If it works, great and every­one can use this. Or, if it doesn’t work, then we need to have a dif­fer­ent strat­e­gy. That’s the con­cern: We don’t want to be try­ing ran­dom med­i­cines and have no idea what works.”

Al­ready, larg­er looks at some an­tivi­rals have mud­died a once-promis­ing pic­ture. A New Eng­land Jour­nal of Med­i­cine study pub­lished yes­ter­day test­ing the HIV drug com­bo lopinavir–ri­ton­avir showed no ben­e­fit over stan­dard-of-care for coro­n­avirus pa­tients. The French chloro­quine study re­leased full re­sults to­day, re­veal­ing they had ex­clud­ed 3 pa­tients who had re­ceived chloro­quine, in­clud­ing one who lat­er died. Remde­sivir, the Gilead an­tivi­ral that’s emerged as per­haps the lead­ing can­di­date to treat coro­n­avirus, looked ini­tial­ly promis­ing when de­ployed against Ebo­la in 2014, on­ly to lat­er per­form lit­tle bet­ter than stan­dard-of-care when test­ed in a ran­dom­ized tri­al.

Boul­ware isn’t bank­ing on the drug, but he said it may be more ef­fec­tive in pre­vent­ing in­fec­tion than in treat­ing pa­tients who are al­ready in the hos­pi­tal.

“At that point, they’re re­al­ly re­al­ly sick and it may not help the pa­tient much,” he said.

If the drug works, though, that won’t be the end of the sto­ry. When he de­cid­ed to launch his study last week, Boul­ware was able to or­der enough pills for an en­tire course of treat­ment for $12. But he warned that if the drug is shown to work, de­mand will quick­ly far out­strip man­u­fac­tur­ers’ abil­i­ty to sup­ply it.

Yes­ter­day, Britain banned both ex­port and hoard­ing of chloro­quine.

“They’re go­ing to sell a lot of it,” Boul­ware said of the hand­ful of com­pa­nies who make the drug. ‘There’s go­ing to be a short­age of sup­ply.”

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

Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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UP­DAT­ED: A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

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And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

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Aaron Royston, venBio

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Paris-based Servier announced Friday that it has struck a deal to buy out the antibody expert. The acquisition comes 2 years after Servier acquired Shire’s cancer business for $2.4 billion. They’ve been working with Symphogen on a slate of programs, including some favs – PD-1, LAG3 and TIM3 — where they are looking to differentiate themselves from the more prominent drugs in these niches.

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Developing new drugs is incredibly hard. That’s why, despite superhuman efforts from the industry, we’re still looking at 12-18 months minimum before we can realistically hope for a vaccine for Covid-19, and probably months before there’s a proven viable drug treatment.

But our increasing ability to begin to industrialize the drug discovery and development process through an engineering approach means that we have more hope for speeding up this process than ever before — and not just to defeat coronavirus, but to benefit the development of all new medicines in the future.

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