Covid-19 roundup: Count­ing 1,087 tri­als for coro­n­avirus, R&D ex­perts say much of it is a com­plete waste; WHO sci­en­tist por­tends it will be 'years' be­fore Covid-19 threat will be con­tained

There are good ex­am­ples of R&D pro­grams for Covid-19 drugs and vac­cines, such as CEPI’s plan to ad­vance a slate of new pro­grams. But if you pull back and look at a land­scape of more than 1,000 clin­i­cal tri­als now un­der­way try­ing to make a mark, the field is a mess.

That, in blunt trans­la­tion, is the point of a new op-ed piece in the BMJ penned by Paul Glasziou, di­rec­tor of the In­sti­tute for Ev­i­dence Based Health­care, and col­leagues.

Ti­tling their piece “Waste in covid-19 re­search,” the team notes:

Be­fore the pan­dem­ic, it was es­ti­mat­ed that up to 85% of re­search was wast­ed be­cause of poor ques­tions, poor study de­sign, in­ef­fi­cien­cy of reg­u­la­tion and con­duct, and non or poor re­port­ing of re­sults. Many of these prob­lems are am­pli­fied in covid-19 re­search, with time pres­sures and in­ad­e­quate re­search in­fra­struc­ture con­tribut­ing.

The au­thors count­ed 1,087 clin­i­cal tri­als for Covid-19 in­ter­ven­tions on clin­i­cal­tri­, “and though some will pro­vide use­ful in­for­ma­tion, many are too small and poor­ly de­signed to be help­ful, mere­ly adding to the covid-19 noise. Of the 145 reg­is­tered tri­als of hy­drox­y­chloro­quine, for ex­am­ple, 32 have a planned sam­ple size of ≤100, 10 have no con­trol group, and 12 are com­par­a­tive but non-ran­domised. Out­come mea­sures vary wide­ly, and on­ly 50 seem to be mul­ti­cen­tre. Strik­ing­ly, on­ly one pro­vides a pro­to­col, and even lim­it­ed reg­istry de­tails re­veal un­jus­ti­fied out­come switch­ing.”

One of the oth­er prob­lems with the fran­tic rush in­to Covid-19, they add, is that non-drug in­ter­ven­tions, such as the use of masks in pre­vent­ing the spread of the virus, are be­ing ig­nored. — John Car­roll

WHO sci­en­tist por­tends it will be ‘years’ be­fore Covid-19 threat will be con­tained

Paint­ing a grim pic­ture at the Fi­nan­cial Times’ Glob­al Board­room dig­i­tal con­fer­ence, the World Health Or­ga­ni­za­tion’s chief sci­en­tist pre­dict­ed it will take some four to five years be­fore Covid-19 is con­trolled.

Apart from ther­a­peu­tics, there is a fever­ish ef­fort to de­vel­op a vac­cine glob­al­ly, with some sci­en­tists hop­ing to have an ear­ly taste of da­ta by the end of this year or ear­ly 2020. Pret­ty much all re­searchers agree that a safe and ef­fec­tive vac­cine is the on­ly way out of the cri­sis, but pro­duc­tion and eq­ui­table dis­tri­b­u­tion hur­dles re­main.

On Wednes­day, the WHO’s Soumya Swami­nathan in­di­cat­ed a myr­i­ad of fac­tors would de­ter­mine how long the threat of the coro­n­avirus will re­main in play, in­clud­ing whether it mu­tates, the con­tain­ment mea­sures im­posed and whether a vac­cine is de­vel­oped.

Pe­ter Pi­ot, pro­fes­sor of glob­al health at the Lon­don School of Hy­giene and Trop­i­cal Med­i­cine who was al­so speak­ing on the FT pan­el and is him­self re­cov­er­ing from the vi­ral in­fec­tion, said coun­tries should be think­ing about the sit­u­a­tion in terms of years, not months, and find ways to change the par­a­digm from lock­downs to more “gran­u­lar, tar­get­ed types of in­ter­ven­tions,” ac­cord­ing to the FT re­port.

He al­so stressed the im­por­tance of test­ing, in line with the WHO’s rec­om­men­da­tions. There is “no op­tion but to in­vest more in test­ing,” Pi­ot said. — Na­tal­ie Grover

Swiss drug­mak­er No­var­tis thinks a re­al­is­tic vac­cine time­line is “one and a half to two years”

Vac­cines are hard to make. On av­er­age, in the pre-pan­dem­ic era, a decade was a rea­son­able time­frame to de­vel­op a vac­cine, al­though some virus­es such as HIV have proved elu­sive so far to the sci­en­tif­ic tool­box. The record for the fastest-ever vac­cine is for Ebo­la, which rav­aged West­ern Africa be­tween 2014 and 2016 — and that time­line is five years.

But the glob­al im­pact of Covid-19 has trig­gered a gar­gan­tu­an ef­fort from sci­en­tists the world over, with some re­searchers promis­ing their first batch of sol­id da­ta by the end of this year — for ex­am­ple, the group at Ox­ford hopes to have vi­able da­ta by the fall.

Vas Narasimhan, chief of No­var­tis — a com­pa­ny that is not de­vel­op­ing a vac­cine — said that a vac­cine will like­ly on­ly be avail­able in “one and a half to two years” in line with a cau­tious­ly op­ti­mistic view of the phar­ma­ceu­ti­cal in­dus­try, Bloomberg re­port­ed, cit­ing an opin­ion piece pub­lished by the CEO in a Swiss pub­li­ca­tion.

While health reg­u­la­to­ry bod­ies in Eu­rope and the Unit­ed States have gen­er­al­ly agreed to weak­en the ev­i­den­tiary stan­dards for ear­ly vac­cine stud­ies giv­en the ex­panse of the coro­n­avirus cri­sis, sci­en­tists and ex­perts have preached cau­tion against these am­bi­tious time­lines.

Bill Gates, who was elect­ed to pour sub­stan­tial re­sources as part of his foun­da­tion to fight Covid-19, has said it could take be­tween 9 months to two years to de­liv­er a vac­cine. The vac­cine part­ner­ship be­tween Ox­ford, As­traZeneca and Vac­citech — which aims to make 100 mil­lion dos­es by the end of 2020 — in­cludes pro­vi­sions to dis­trib­ute the vac­cine to low and medi­um-in­come coun­tries, al­though the de­tails are sparse. Pres­i­dent Don­ald Trump’s re­cent­ly-an­nounced “Op­er­a­tion Warp Speed,” seeks to pro­duce 300 mil­lion im­mu­niza­tions by the end of this year. — Na­tal­ie Grover

France’s Abi­vax kicks off big Covid-19 tri­al with drug that is en­gi­neered to have a three-pronged at­tack on Covid-19: an­tivi­ral, an­ti-in­flam­ma­to­ry and tis­sue re­pair

With the French drugs and ethics agen­cies sign­ing off on a 1,034-pa­tient place­bo-con­trolled phase IIb/III tri­al, called miR-AGE, of the com­pa­ny’s ex­per­i­men­tal drug, ABX464, Abi­vax is hop­ing the drug will help pre­vent se­vere in­flam­ma­tion that leads to acute res­pi­ra­to­ry dis­tress syn­drome in el­der­ly or high-risk pa­tients suf­fer­ing from Covid-19.

The Parisian biotech’s oral ther­a­py, which is al­so be­ing eval­u­at­ed as a treat­ment for ul­cer­a­tive col­i­tis, has been shown to up­reg­u­late miR-124, a “phys­i­o­log­i­cal brake” of in­flam­ma­tion, as well as tamp down cy­tokines in­volved in the Covid-19 cy­tokine storm. Sep­a­rate­ly, in vi­vo da­ta sug­gest ABX464 in­hibits repli­ca­tion of SARS-CoV-2, the virus be­hind Covid-19, the com­pa­ny said.

“Un­for­tu­nate­ly, no pro­phy­lac­tic or ther­a­peu­tic treat­ment has shown much ef­fi­ca­cy in any rig­or­ous tri­al to treat the se­vere form of COVID-19; help­ing clin­i­cians pre­vent res­pi­ra­to­ry dis­tress and death in COVID-19 pa­tients and lim­it longer-term pul­monary dam­age is of para­mount ne­ces­si­ty,” said Abi­vax chief Hart­mut Ehrlich in a state­ment. “The ro­bust, rig­or­ous de­sign of the miR-AGE ABX464 tri­al en­sures we will draw valid sci­en­tif­ic and med­ical con­clu­sions …We al­ready have ABX464 cap­sules in stock to treat app. 50,000 pa­tients and could scale-up ABX464 man­u­fac­tur­ing for over one mil­lion pa­tients with­in months.”

Gilead’s an­tivi­ral remde­sivir is the on­ly drug that has been grant­ed emer­gency use au­tho­riza­tion in the Unit­ed States (it is al­so un­der re­view at the EMA) on the ba­sis of place­bo-con­trolled clin­i­cal tri­al da­ta. How­ev­er, on­ly the top-line num­bers have been re­leased; the de­tailed da­ta are still to come. Mean­while, dis­tri­b­u­tion and ac­cess are fraught, with its mak­er do­nat­ing its ini­tial sup­ply of dos­es for rough­ly 140,000 pa­tients to the fed­er­al gov­ern­ment, be­cause there is no por­tal for hos­pi­tals to ap­ply for ac­cess to the med­i­cine or out­lined cri­te­ria for how the drug will be dis­trib­uted and to whom, caus­ing con­ster­na­tion and de­spair for doc­tors and pa­tients alike. — Na­tal­ie Grover

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

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust of the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Covid-19 roundup: Op­er­a­tion Warp Speed's 7th vac­cine is live at­ten­u­at­ed; Small biotech touts big suc­cess where gi­ants have failed

Operation Warp Speed is stacking its vaccine portfolio with a “TBD” new candidate: a live attenuated vaccine that can be administered in a single dose, potentially as an oral formulation rather than an injection.

Sound familiar?

That could be because the unannounced candidate appears to match the profile of an inoculation being developed by Merck, according to Bloomberg, which first reported the development based on a presentation by Moncef Slaoui.

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CDC’s Robert Redfield, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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On­ly five months af­ter a Se­ries A launch, Taysha goes pub­lic with $157M IPO

As has been the trend in 2020, Taysha Gene Therapies has become the latest biotech to make a quick ascent from a small, privately-funded company to enjoying its very own Nasdaq ticker.

The Dallas-based biotech raised $157 million for its IPO after pricing shares at $20 apiece Thursday, the high-point of its expected range. Initially pegging $100 million in financing, Taysha offered a little less than 8 million shares and will trade under the $TSHA symbol.

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Chair of FDA's vac­cine ad­comm — who's al­so a lead in­ves­ti­ga­tor of Mod­er­na's vac­cine — re­cus­es her­self from Covid-19 talks

When the FDA’s Vaccines and Related Biological Products Advisory Committee meets next month to discuss the development and authorization of Covid-19 vaccines, the chairwoman won’t be there.

Hana El Sahly has recused herself from the expert panel’s review of the topic, citing her role as a lead investigator in Moderna’s Phase III trial, Reuters reported. An associate professor of virology and microbiology at Baylor College of Medicine in Houston, El Sahly was appointed the chairwoman last year.