Covid-19 roundup: Alex­ion's C5 in­hibitor Ul­tomiris miss­es the PhI­II bar; Lon­za in wait­ing game to use in­gre­di­ents in Mod­er­na vac­cine

C5 in­hi­bi­tion isn’t the an­swer to the search for treat­ments for se­vere Covid-19 af­ter all.

Alex­ion is paus­ing en­roll­ment to a Phase III study test­ing Ul­tomiris, the long-act­ing fol­low-on to Soliris, among pa­tients re­quir­ing me­chan­i­cal ven­ti­la­tion af­ter the in­de­pen­dent da­ta mon­i­tor­ing com­mit­tee raised a lack of ef­fi­ca­cy in an in­ter­im analy­sis. Among 122 pa­tients (out of a planned en­roll­ment of 270), there was no mean­ing­ful dif­fer­ence in sur­vival at Day 29.

The biotech stopped short of shelv­ing the pro­gram right away, say­ing in­stead it will con­duct fur­ther analy­sis to de­ter­mine next steps — with an eye to iden­ti­fy­ing any sub­groups that might ben­e­fit.

Ap­proved for rare blood dis­or­ders, Ul­tomiris is one of the key grow­ing fran­chise drugs fea­tured in As­traZeneca’s sur­prise $39 bil­lion buy­out of Alex­ion. Like the cash cow Soliris, it works by block­ing the com­ple­ment C5, and the ini­tial hy­poth­e­sis was that the drug could low­er cy­tokine and chemokine lev­els, there­by re­duc­ing lung in­flam­ma­tion.

John Orloff

“While ini­tial anec­do­tal re­ports from com­pas­sion­ate use cas­es were promis­ing, these re­sults demon­strate the im­por­tance of con­duct­ing con­trolled clin­i­cal tri­als to ful­ly eval­u­ate the po­ten­tial of new treat­ment ap­proach­es and gen­er­ate the nec­es­sary ev­i­dence to make in­formed de­ci­sions,” John Orloff, Alex­ion’s head of R&D, said in a state­ment.

More da­ta are com­ing, the com­pa­ny not­ed. A team of re­searchers at Cam­bridge Uni­ver­si­ty Hos­pi­tals NHS Foun­da­tion Trust is lead­ing a plat­form study to in­ves­ti­gate the po­ten­tial of giv­ing im­mune mod­u­la­tors ear­li­er — be­fore hos­pi­tal­ized pa­tients need me­chan­i­cal ven­ti­la­tion — to pre­vent pro­gres­sion of the virus.

Re­pur­pos­ing com­mer­cial drugs to treat Covid-19, es­pe­cial­ly at the se­vere stage, has proven an ar­du­ous, wind­ing jour­ney for many big play­ers. Roche and Re­gen­eron/Sanofi’s bet on the IL-6 class large­ly came up emp­ty (al­though new da­ta sug­gest there might yet be legs to the ear­ly hope), and No­var­tis didn’t have bet­ter luck with Jakafi, its In­cyte-part­nered JAK in­hibitor.

Eli Lil­ly, though, did man­age to se­cure an emer­gency use au­tho­riza­tion for its own JAK drug Olu­mi­ant — in com­bi­na­tion to Gilead’s ap­proved an­tivi­ral, remde­sivir. Mean­while, Op­er­a­tion Warp Speed is promis­ing up to $354 mil­lion to mass man­u­fac­ture an ex­per­i­men­tal drug Mer­ck ob­tained via the ac­qui­si­tion of On­coIm­mune, which was shown to re­duce the risk of res­pi­ra­to­ry fail­ure or death by 50% in a tri­al of 203 se­vere and crit­i­cal pa­tients.—Am­ber Tong

Lon­za await­ing Swiss li­cense at plant man­u­fac­tur­ing ac­tive in­gre­di­ents in Mod­er­na’s vac­cine

Lon­za, the Basel-head­quar­tered man­u­fac­tur­ing gi­ant, is cur­rent­ly in wait­ing mode at a new plant in Visp, Switzer­land, where it’s start­ed man­u­fac­tur­ing ac­tive in­gre­di­ents in Mod­er­na’s Covid-19 vac­cine — they just can’t be used yet.

Reuters re­port­ed Thurs­day that the man­u­fac­tur­er is still await­ing a nec­es­sary li­cense from the coun­try’s drug reg­u­la­tor Swissmedic, but that the first batch was ex­pect­ed to be ready this month. Lon­za can pro­duce and ship the drug in­gre­di­ents be­fore the fa­cil­i­ty is grant­ed a li­cense; they just can’t be re­leased be­fore that ap­proval comes.

Af­ter com­ple­tion of the ini­tial pro­duc­tion line, Lon­za is prepar­ing for two more Visp fa­cil­i­ties to be com­mis­sioned, Reuters re­port­ed. The lines cost rough­ly $79 mil­lion, re­quire be­tween 60 and 70 em­ploy­ees to op­er­ate, and can each pro­duce enough in­gre­di­ents for 300 mil­lion an­nu­al dos­es of the mR­NA vac­cine.

Once pro­duced, Reuters said Lon­za would ship the in­gre­di­ents to Lab­o­ra­to­rios Far­ma­ceu­ti­cos ROVI in Madrid for fi­nal fill­ing.

It’s un­clear when ex­act­ly Lon­za could gain the Swissmedic li­cense to use the in­gre­di­ents, but a spokesper­son told Reuters they ex­pect­ed a vis­it from state in­spec­tors soon. — Con­ner Mitchell

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

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Jean-Christophe-Hyvert, Lonza

Lon­za look­ing to build on 'd­if­fer­en­ti­at­ed ad­van­tage' in Covid-19, CD­MO mar­ket­place in 2021

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.

IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.