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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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FTC pulls re­main­ing case against Ab­b­Vie; New EU clin­i­cal tri­als sys­tem com­ing in 2022; Abing­worth bets big on CymaBay

The Federal Trade Commission on Friday withdrew its remaining case against AbbVie after the Supreme Court declined to review a lower court’s ruling.

The punt by SCOTUS means that while the Illinois pharma company illegally blocked patients’ access to lower-cost alternatives to its testosterone drug AndroGel, the FTC will no longer be able to return about $500 million directly to AndroGel consumers.

UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; 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.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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Alan Hirzel, Abcam

Drug sup­pli­er Ab­cam brings a long­time col­lab­o­ra­tor in house as part of $340M buy­out pact

BioVision has supplied Abcam with research tools since 1999, and now the two are making it official as part of a merger unveiled Monday.

Abcam will buyout BioVision as part of a $340 million acquisition deal to bring aboard the supplier’s biochemical and cell-based assays for biological research, as well as recombinant proteins, antibodies and enzymes.

The deal will give Abcam control of BioVision’s portfolio and allow for both the expansion of research existing areas of focus such as oncology, neuroscience and epigenetics and preparation to expand into new products. As a part of the deal, Abcam will develop and supply products and services to NKY, the previous owner of BioVision and receive support for ongoing development and commercialization of in vitro diagnostic products.