As­traZeneca, Alex­ion close mas­sive $39B merg­er; blue­bird bio earns EU nod for Lenti-D as big split ap­proach­es

With the fi­nal hur­dle re­cent­ly cleared, As­traZeneca and Alex­ion can fi­nal­ly cel­e­brate their $39 bil­lion merg­er.

The drug­mak­ers closed their ma­jor M&A pact Wednes­day, with Alex­ion for­mal­ly tak­ing its place as the British drug gi­ant’s rare dis­ease unit. The UK’s Com­pe­ti­tion & Mar­kets Au­thor­i­ty last week gave its stamp of ap­proval to the deal, mark­ing the last sig­nif­i­cant reg­u­la­tor to sign off.

Alex­ion will bring aboard block­buster drug Soliris as well as its suc­ces­sor drug Ul­tomiris, the fo­cus of a ma­jor pa­tient switch­ing cam­paign as the old­er best­seller ap­proach­es the first of its patent cliffs in the com­ing years.

It’s not all gravy for ei­ther drug, though. Up­start com­peti­tors like Apel­lis are hunt­ing for both drugs’ biggest pa­tient pop­u­la­tions and have rea­son to think their da­ta could be bet­ter.

Blue­bird bio earns ap­proval for Lenti-D in Eu­rope

With a big split be­tween its rare dis­ease and on­col­o­gy units com­ing, blue­bird bio has earned an ap­proval in Eu­rope for gene ther­a­py Lenti-D for chil­dren with ear­ly cere­bral adrenoleukody­s­tro­phy (CALD) and an ABCD1 ge­net­ic mu­ta­tion with­out a matched sib­ling donor, the com­pa­ny said Wednes­day.

The ther­a­py will be mar­ket­ed as Skysona in the EU and be­come the on­ly drug ap­proved there for CALD, an ul­tra-rare neu­ro­log­i­cal dis­or­der that caus­es a break­down in myelin, which func­tions as a pro­tec­tive sheath around nerve cells, and is of­ten fa­tal.

Avail­able stan­dard of care for CALD is a stem cell trans­plant, which comes with a risk of se­vere com­pli­ca­tions, and rough­ly 80% of pa­tients do not have a matched donor avail­able.

Lenti-D’s ap­proval comes as a breath of fresh air for blue­bird as it looks to split in two af­ter years of tur­moil around its gene ther­a­py busi­ness, once the dar­ling of biotech’s rare dis­ease space.

Ear­li­er this month, the drug­mak­er lift­ed a vol­un­tary EU mar­ket­ing hold on an­oth­er gene ther­a­py, Zyn­te­glo for be­ta tha­lassemia, af­ter a pa­tient death in one of the drug­mak­er’s oth­er prod­ucts, Lenti­Glo­bin, halt­ed clin­i­cal stud­ies. Blue­bird de­ter­mined that death was high­ly un­like­ly to have been caused by the de­liv­ery virus used in both Zyn­te­glo and Lenti­Glo­bin, eas­ing in­vestors’ con­cerns some­what.

Bio­gen snags neu­rol­o­gy R&D pact with RNAi spe­cial­ist

With a con­tro­ver­sial Aduhelm ap­proval in hand, Bio­gen has found its next for­ay in­to neu­rol­o­gy.

Bio­gen and New York-based Mir­imus have signed a de­vel­op­ment deal for a slate of RNAi-based ther­a­pies tar­get­ing undis­closed neu­rol­o­gy tar­gets, the part­ners said Wednes­day. Terms of the deal weren’t dis­closed.

Mir­imus, an 11-year-old biotech look­ing to de­vel­op RNAi and CRISPR-based ther­a­peu­tics, said the pact would help the com­pa­ny ad­vance its pre­clin­i­cal work in­to hu­man tests.

“This col­lab­o­ra­tion marks an im­por­tant ad­vance for Mir­imus as we seek to ex­tend our ex­per­tise in RNAi an­i­mal mod­els to the de­vel­op­ment of RNAi-based bio­ther­a­peu­tics,” CEO Prem Prem­srirut said in a state­ment.

AI start­up Ex­sci­en­tia kicks off CDK-fo­cused JV with Chi­nese biotech

Shang­hai-based on­col­o­gy up­start GT Ape­iron and AI wun­derkind Ex­sci­en­tia have been hard at work on a CDK7 dis­cov­ery project that has re­cent­ly been bear­ing fruit. Now, the part­ners are tak­ing the next step and launch­ing a joint ven­ture to seek out mol­e­cules across the en­tire CDK space.

The JV will lever­age Ex­sci­en­tia’s AI and ma­chine learn­ing dis­cov­ery plat­form to help in­fuse GT’s ear­ly pipeline with new can­di­dates. Fi­nan­cial terms of the deal were not dis­closed, but the part­ners said they would split own­er­ship of any en­su­ing mol­e­cules with Ex­sci­en­tia tak­ing an eq­ui­ty stake in GT.

“Based on what they have al­ready achieved, Ex­sci­en­tia is clear­ly the leader in AI-dri­ven drug dis­cov­ery, and we have wit­nessed this first-hand in our col­lab­o­ra­tion so far,” GT Ape­iron pres­i­dent Mingxi Li said in a state­ment.

Ex­sci­en­tia, one of a group of drug­mak­ers who have claimed the first “AI-dis­cov­ered” drug, is com­ing off a mas­sive, $500 mil­lion mega-round in April and a dis­cov­ery col­lab­o­ra­tion with drug pric­ing dis­rup­tor EQRx that the pair hopes will prove to be a desta­bi­liz­ing force on high-list prices for brand­ed drugs.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.