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.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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Mi­rati’s drug sitra­va­tinib flops PhI­II in com­bo with Op­di­vo for cer­tain lung can­cer

Mirati Therapeutics’ path to a second drug approval will likely have to wait. The San Diego biotech company said Wednesday that its investigational lung cancer drug failed a Phase III trial testing it in combination with Bristol Myers Squibb’s Opdivo.

The drug, sitravatinib, and Opdivo weren’t better than the chemo drug docetaxel at keeping patients alive, Mirati said in a press release. The spectrum-selective kinase inhibitor missed the primary goal of overall survival in patients with second- or third-line advanced non-squamous, non-small cell lung cancer.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Teresa Bitetti, Takeda's president of the global oncology business unit

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

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