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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Andrew Crockett, KalVista CEO

KalVista ends a PhII study ear­ly af­ter pa­tients suf­fer se­vere and life-threat­en­ing side ef­fects

KalVista took a beating Tuesday after announcing it would scrap a Phase II trial for one of its experimental drugs.

The biotech said in an early morning press release that it is terminating the study for KVD824 after multiple patients in every treatment group saw unsafe, elevated levels of certain liver enzymes. By ending the trial now, KalVista hopes to save some money and funnel it toward another study for its lead program, CEO Andrew Crockett said in a statement.

Pen­ny stock play­er to re­view all op­tions to try stay­ing afloat af­ter clin­i­cal tri­al fail

Adamis Pharmaceuticals is slowly tumbling down, and the biotech is looking at all its options.

After a Phase II/III trial failure last month that sent the penny stock player down an additional 50% to just 15 cents a share, the company said Monday that it is examining options to get the best value for its investors. A statement from Adamis indicates that alternatives include anything from a partnership to a sale of Adamis’ two commercial products, Zimhi and Symjepi.