The race to de­vel­op Covid-19 drugs and vac­cines is on — here’s what’s hap­pen­ing in the UK

Weeks away from the re­sults of on­go­ing US and Chi­na tri­als test­ing its ex­per­i­men­tal an­tivi­ral remde­sivir, Gilead is go­ing to tri­al the failed Ebo­la drug in a small group of coro­n­avirus pa­tients in Eng­land and Scot­land. The Unit­ed King­dom is al­so home to a range of oth­er ther­a­peu­tic ef­forts, as the pan­dem­ic rages on across the globe.

On Tues­day, Southamp­ton, UK-based start­up Synair­gen kicked off a mid-stage place­bo-con­trolled study test­ing its ex­per­i­men­tal drug, SNG001 — an in­haled for­mu­la­tion of in­ter­fer­on-be­ta-1a — that has pre­vi­ous­ly shown to be safe and ef­fec­tive in im­prov­ing lung func­tion in asth­ma pa­tients with a res­pi­ra­to­ry vi­ral in­fec­tion in a pair of Phase II tri­als.

In­ter­fer­ons, a fam­i­ly of nat­u­ral­ly oc­cur­ring pro­teins se­cret­ed by the im­mune sys­tem, typ­i­cal­ly boost the body’s im­mune re­sponse to un­in­vit­ed guests such as virus­es, bac­te­ria and can­cer.

Richard Mars­den Synair­gen

“When we’ve col­lect­ed cells from pa­tients with COPD and asth­ma and old­er peo­ple…we find that their lung cells don’t re­spond very well to virus­es,” CEO Richard Mars­den said in an in­ter­view. “We have al­so along the way al­ways rec­og­nized that with an emerg­ing virus, the drug could be used.”

As Covid-19 start­ed to gath­er steam in Chi­na, Synair­gen tried to get things start­ed, but to no avail. Italy was the next plan. “We had some re­al­ly good in­ter­ac­tion there,” said Mars­den. “But they went from, you know, just busy to very busy to ex­treme­ly busy to un­able-to-com­mu­ni­cate busy.”

Even­tu­al­ly, they de­cid­ed their home ground — the UK, where they have an on­go­ing COPD tri­al — would be the best place to kick off a Covid-19 study. Ini­tial­ly, the pi­lot phase of the tri­al will have 100 pa­tients (50 will get a place­bo, and 50 will get SNG001). If all goes well, a piv­otal study will be con­duct­ed.

This ap­proach is one of many, as com­pa­nies race to de­sign and de­vel­op di­ag­nos­tics, drugs and vac­cines to stem the tide of the pan­dem­ic. “(W)e need high qual­i­ty clin­i­cal re­search to work out what is work­ing, what isn’t work­ing; we be­lieve place­bo-con­trolled tri­als are the way to do that,” Mars­den said.

Last week, the UK gov­ern­ment is­sued a state­ment con­firm­ing that the two decades-old malar­ia drugs: chloro­quine and hy­drox­y­chloro­quine, which have been tout­ed as po­ten­tial treat­ments for pa­tients in­fect­ed with the coro­n­avirus, have not been sanc­tioned for use against the virus in the UK.

Al­though clin­i­cal tri­als are on­go­ing, no con­clu­sions have been reached on the safe­ty and ef­fec­tive­ness of these med­i­cines, not­ed the Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency. In stark con­trast, in the Unit­ed States, the FDA on Sun­day is­sued emer­gency au­tho­riza­tion for the pair of drugs that Pres­i­dent Don­ald Trump has re­peat­ed­ly backed, on the ba­sis of anec­do­tal re­ports.

Mar­tin Lan­dray Ox­ford

In the UK, sci­en­tists at Ox­ford Uni­ver­si­ty are al­so look­ing at re­pur­pos­ing oth­er drugs for use against Covid-19. Last week, re­searchers an­nounced they would be test­ing lopinavir-ri­ton­avir, ap­proved used to treat HIV, and the steroid dex­am­etha­sone, in con­sent­ing adults that have test­ed pos­i­tive for Covid-19 in NHS hos­pi­tals. The project, in which pa­tients will ei­ther get one of the two drugs, or place­bo in ad­di­tion to stan­dard-of-care treat­ment, has won £10.5 mil­lion in gov­ern­ment fund­ing.

“The stream­lined de­sign of this clin­i­cal tri­al al­lows con­sent­ing pa­tients to be en­rolled in large num­bers eas­i­ly and with­out com­pro­mis­ing pa­tient safe­ty or adding sig­nif­i­cant­ly to the work­load of busy hos­pi­tals and their staff,” said the tri­al’s deputy chief in­ves­ti­ga­tor Mar­tin Lan­dray, who al­so serves as a pro­fes­sor of med­i­cine and epi­demi­ol­o­gy Uni­ver­si­ty of Ox­ford, in a state­ment.

Vac­cines in the works

Ox­ford re­searchers al­so have a vac­cine can­di­date in place.

On Jan­u­ary 10 — long be­fore the coro­n­avirus in­fec­tion was named Covid-19 or as­sumed pan­dem­ic pro­por­tions — a team of Ox­ford re­searchers led by Pro­fes­sors Sarah Gilbert, An­drew Pol­lard, Adri­an Hill and Dr. Sandy Dou­glas had be­gun their search for a vac­cine. On March 18, they honed in on a can­di­date: a chim­panzee ade­n­ovirus vac­cine vec­tor (ChA­dOx1).

Chim­panzee ade­n­ovi­ral vec­tors are well stud­ied, hav­ing been used in vac­cines tar­get­ing over 10 dif­fer­ent dis­eases. The Ox­ford vac­cine con­tains the ge­net­ic se­quence of the sur­face spike pro­tein found on SARS-CoV-2 — the virus be­hind Covid-19 — in­side the ChA­dOx1 con­struct. If the project is suc­cess­ful, vac­ci­na­tion with this prod­uct will pro­duce the sur­face spike pro­tein of the coro­n­avirus, prim­ing the im­mune sys­tem to at­tack the coro­n­avirus if it lat­er in­fects the body.

The re­searchers — who have pre­vi­ous­ly de­vel­oped a vac­cine for MERS that showed promise in ear­ly clin­i­cal tri­al — said last week they would start screen­ing peo­ple for a clin­i­cal tri­al, al­though the vac­cine is still weeks away from be­ing ready for hu­man test­ing. The en­roll­ment goal is to hit 510 vol­un­teers, and work is be­ing done to scale up man­u­fac­tur­ing in haste.

About a two-hour dri­ve away, re­searchers at the Uni­ver­si­ty of Cam­bridge al­so have a Covid-19 vac­cine in the works.

Pro­fes­sor Jonathan Heeney, head of the lab­o­ra­to­ry of vi­ral zoonotics and chief of spin­off com­pa­ny DIOSyn­Vax, has spear­head­ed re­search, aid­ed by com­put­er mod­el­ing of the virus’ struc­ture.

By putting the ge­net­ics of the virus un­der a mi­cro­scope, the com­pa­ny has iden­ti­fied a key part of the ge­net­ic code that the virus us­es to pro­duce the es­sen­tial part of its coat: the spikes, which is what the vac­cine is en­gi­neered to tar­get.

“A vac­cine strat­e­gy needs to be laser spe­cif­ic, tar­get­ing those do­mains of the virus’ struc­ture that are ab­solute­ly crit­i­cal for dock­ing with a cell, while avoid­ing the parts that could make things worse,” he said in a state­ment. “Our tech­nol­o­gy does just that.”

Pre­clin­i­cal tri­als are yet to be con­duct­ed, but he ex­pects the vac­cine can­di­date could be ready for hu­man tri­als by June. Fund­ing, how­ev­er, is re­quired.

“We need a ‘Big Phar­ma’ part­ner to help us scale up our ac­tiv­i­ties,” he said.

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

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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WIB22: Chas­ing af­ter ever-evolv­ing sci­ence takes a drug hunter across the pond

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Like many scientists, Fiona Marshall would tell you that she loved the natural world growing up — going to look at crabs running around the beach near her childhood home, pondering about the tides. But one thing about biology, in particular, stood out: It was constantly changing, and changing very quickly.

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Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.