As­traZeneca's Covid-19 vac­cine woes, Mon­cef Slaoui re­signs over sex­u­al ha­rass­ment claims, FDA acts tough, and more

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You know the week has been over­flow­ing with news when sto­ries that you thought were huge on Mon­day get large­ly buried by Wednes­day and full news cy­cles get wrapped in­to one re­port. It may be a good idea to sit down for this edi­tion — it’s go­ing to take a while.

José Basel­ga’s shock­ing death and lega­cy

The sud­den death at 61 of José Basel­ga, the bril­liant on­col­o­gy R&D chief at As­traZeneca and a tow­er­ing fig­ure in can­cer drug de­vel­op­ment who had ear­li­er been chief med­ical of­fi­cer at Memo­r­i­al Sloan Ket­ter­ing, shook the in­dus­try be­fore the week even be­gan. The out­pour­ing of trib­utes and farewells from col­leagues and for­mer col­lab­o­ra­tors re­flect­ed the breadth of his lega­cy. Basel­ga suc­cumbed to Creutzfeldt-Jakob dis­ease, a rapid­ly pro­gres­sive and lethal but ex­treme­ly rare neu­rode­gen­er­a­tive ail­ment. His daugh­ter launched a fundrais­er to sup­port re­search in­to the con­di­tion, which you can see here.

As­traZeneca tries to put out fire

As­traZeneca con­fi­dent­ly de­clared a 79% ef­fi­ca­cy fig­ure for its Covid-19 vac­cine in a US tri­al, as­sum­ing it had a straight path to­ward an EUA. Lit­tle did it know it was steer­ing straight in­to a storm, as the NIH is­sued a mid­dle-of-the-night state­ment just hours lat­er rais­ing con­cerns about the da­ta they chose to dis­close. As it turned out, the Da­ta and Safe­ty Mon­i­tor­ing Board over­see­ing the tri­al blast­ed As­traZeneca for cher­ry-pick­ing the most fa­vor­able num­ber rather than more com­plete and re­cent re­sults — an act that they said would erode pub­lic trust. The com­pa­ny tried to put out the fire with a pri­ma­ry cut of the more up-to-date num­bers, sug­gest­ing the vac­cine is 76% ef­fec­tive — al­though the fullest pic­ture may not emerge un­til the FDA re­views the full dataset.

Mon­cef Slaoui ac­cused of sex­u­al ha­rass­ment

Mon­cef Slaoui — whom Glax­o­SmithK­line ter­mi­nat­ed as the board chair at its bio­elec­tron­ics joint ven­ture Gal­vani fol­low­ing al­le­ga­tions of sex­u­al ha­rass­ment and in­ap­pro­pri­ate con­duct from an em­ploy­ee that GSK said was “sub­stan­ti­at­ed” in an in­ves­ti­ga­tion — has of­fi­cial­ly cut all pro­fes­sion­al ties with his res­ig­na­tion from the VC firm Medicxi. The long­time GSK ex­ec had been im­mers­ing back in­to bio­phar­ma af­ter wrap­ping a stint as the vac­cine czar of Op­er­a­tion Warp Speed. Rep­ri­mand­ed by Em­ma Walm­s­ley in a com­pa­ny-wide let­ter, Slaoui had of­fered an “un­re­served” apol­o­gy to the em­ploy­ee in­volved and stepped down from two biotech boards.

FDA takes Pfiz­er to task over tanezum­ab

The fu­ture isn’t look­ing bright for Pfiz­er and Eli Lil­ly’s an­ti-NGF os­teoarthri­tis drug tanezum­ab. Fol­low­ing an in-depth re­view from the FDA that raised a litany of con­cerns about sig­nif­i­cant safe­ty risks, an ad­vi­so­ry com­mit­tee vot­ed al­most unan­i­mous­ly to rec­om­mend against its ap­proval. Per the reg­u­la­tors’ as­sess­ment, the 41 tri­als Pfiz­er has run over 15 years of­fered “sub­stan­tial ev­i­dence of ef­fec­tive­ness” but the pro­posed risk eval­u­a­tion and mit­i­ga­tion strat­e­gy may not be enough to as­suage fears of joint-re­lat­ed prob­lems. Out­side ex­perts agreed, with many ask­ing for long-term fol­lowup stud­ies and at least one go­ing fur­ther to say the drug didn’t seem any bet­ter than as­pirin or ibupro­fen, or even place­bo.

Bio­phar­ma’s di­ver­si­ty prob­lem

Among the many is­sues that the Covid-19 pan­dem­ic has ex­posed is the trou­bling un­der­rep­re­sen­ta­tion of mi­nor­i­ty groups in clin­i­cal tri­als. But how did the in­dus­try get here, and what are some ways to break those struc­tur­al bar­ri­ers to in­clu­sion? In­vest­ing in com­mu­ni­ties, build­ing trust and fos­ter­ing di­ver­si­ty from the top down are go­ing to be cru­cial, Nicole De­Feud­is writes. And if Mod­er­na’s move in its vac­cine tri­al is any sig­nal of what’s to come, the in­dus­try could be head­ed for a tip­ping point.

Can bet­ter la­bel­ing avert a dis­as­ter?

A con­sor­tium of phar­ma and tech com­pa­nies is com­ing to­geth­er to solve a seem­ing­ly es­o­teric but po­ten­tial­ly dead­ly prob­lem: how drug­mak­ers ac­tu­al­ly keep track of where a pa­tients’ cells are go­ing. “We are beg­ging for stan­dard­iza­tion wher­ev­er pos­si­ble,” an ex­pert told Kyle Blanken­ship, and la­bel­ing and iden­ti­ty of cells are one of the most im­por­tant ar­eas for that. Oth­er­wise, “some­one is go­ing to make a ma­jor mis­take, and we are rac­ing against time,” said Amy DuRoss, whose com­pa­ny, Vineti, is of­fer­ing an open source track­ing sys­tem.


PRE­MI­UM

Lon­nie Moul­der aims glob­al 

You haven’t heard much from him, but Lon­nie Moul­der hasn’t been idle in the two years since he closed the $5 bil­lion sale of Tesaro to Glax­o­SmithK­line. And he’s fi­nal­ly ready to take the lime­light as in­ter­im CEO of Zenas Bio­Phar­ma, a biotech start­up with roots in Chi­na and the US for which he has as­sem­bled a pipeline with sev­en drugs. Eu­rope will soon be added to the frame as he pur­sues glob­al deals that will set him up to pur­sue all three of the world’s top phar­ma mar­kets. This time, though, the can­cer-ori­ent­ed Moul­der is jump­ing in­to new are­nas, au­toim­mune con­di­tions and rare dis­eases.

Bridge­Bio clears ear­ly hur­dle with NIH help

Mike Collins, an NIH re­searcher and di­vi­sion chief who spe­cial­izes in bone and min­er­al me­tab­o­lism, gave Bridge­Bio’s Cal­ci­lytix team the idea to treat a rare ge­net­ic dis­ease called ADH1 with a cal­ci­um sens­ing re­cep­tor (CaSR) an­tag­o­nist. Two years lat­er, the biotech — with the help of a prin­ci­pal in­ves­ti­ga­tor from the NIHhas laid out a clear set of da­ta from a tiny, 6-per­son Phase II study show­ing how the drug they se­lect­ed did ex­act­ly what they want­ed it to: rais­ing the lev­el of cal­ci­um in the blood to nor­mal lev­els while whit­tling back the over­abun­dance of cal­ci­um in urine. The big­ger test, though, is still ahead.


PEO­PLE

  • Long­time Gilead R&D chief William Lee is hang­ing up his cleats, and Dan O’Day has re­cruit­ed Flav­ius Mar­tin from Am­gen to run the game. Mar­tin, who led Am­gen’s re­search for the on­col­o­gy, in­flam­ma­tion and car­diometa­bol­ic units out of South San Fran­cis­co, is ex­pect­ed the car­ry the torch in im­muno-on­col­o­gy.
  • Di­ana Brainard, one of the mas­ter­minds be­hind Gilead’s Covid-19 drug remde­sivir, is jump­ing ship to take up the CEO post at an­tivi­ral T cell play­er AlloVir. She takes over from David Hal­lal, who has re­cruit­ed the biotech to his man­u­fac­tur­ing base­camp.
  • In the lat­est re­fresh for Mer­ck’s C-suite, Car­o­line Litch­field is get­ting pro­mot­ed to CFO, re­plac­ing Robert Davis as he moves to the top job va­cat­ed by Ken Fra­zier. The cur­rent trea­sur­er, Litch­field is a vet­er­an at the com­pa­ny for more than three decades.
  • Gio­van­ni Caforio had a rel­a­tive­ly tame year com­pared to the oth­er Big Phar­ma CEOs throw­ing them­selves in­to the Covid-19 fight, but he’s still re­ward­ed with a $20 mil­lion pay pack­age for over­see­ing the big Cel­gene in­te­gra­tion and con­fronting a par­al­lel cri­sis in health dis­par­i­ties.

FDA+

  • Amy Aber­nethy, the FDA’s prin­ci­pal deputy com­mis­sion­er of food and drugs and act­ing chief in­for­ma­tion of­fi­cer, is mak­ing a sur­prise ex­it from the agency af­ter a lit­tle more than two years — dur­ing which she spear­head­ed a ma­jor re­struc­tur­ing of IT and es­tab­lished stan­dards for re­al world ev­i­dence. Her de­par­ture couldn’t come at a more crit­i­cal time.
  • Should the in­dus­try brace it­self for a tougher reg­u­la­to­ry en­vi­ron­ment? SVB Leerink an­a­lyst Ge­of­frey Porges sparked a de­bate with new pre­dic­tions about re­stric­tions on drug pric­ing, chal­leng­ing FDA drug re­views and greater scruti­ny of M&A. And while some FDA watch­ers are wary of call­ing it a shift so soon, a Sen­ate com­mit­tee has kicked off talks on drug pric­ing re­forms with a Re­pub­li­can call­ing the cur­rent sys­tem “a mess across the board.”
  • The FDA needs to be hav­ing bet­ter and clear­er sci­en­tif­ic di­a­logues to fos­ter the de­vel­op­ment of com­plex gener­ics, Te­va COO Chris­tine Baed­er ar­gued in an in­ter­view with Zach Bren­nan, as the spot­light shines on the next it­er­a­tion of the Gener­ic Drug User Fee Amend­ments.
  • The HHS in­spec­tor gen­er­al has cleared a No­var­tis pro­gram that pays for low-in­come pa­tients to ac­cess its $475,000 CAR-T ther­a­py Kym­ri­ah, prais­ing its ef­fec­tive­ness while rul­ing the risk of overuti­liza­tion is low.

R&D

  • In a first for the LAG-3 class, Bris­tol My­ers Squibb says a com­bi­na­tion of re­latlimab and its PD-1 Op­di­vo sig­nif­i­cant­ly ex­tend­ed pro­gres­sion-free sur­vival in a Phase III study for melanoma. It’s a big win for both Bris­tol and the so-far lag­gard path­way, which has drawn a pack of phar­ma and biotech play­ers.
  • Roche’s R&D team was hand­ed a mixed bag this week. On one hand, it staked a toe­hold in the $12 bil­lion race to push check­point ther­a­pies in­to ear­li­er stages of can­cer, as Phase III da­ta sug­gest­ed ad­ju­vant use of its PD-1 block­er Tecen­triq helped ear­ly-stage lung can­cer pa­tients live longer than chemother­a­py did. On the oth­er, the phar­ma gi­ant has halt­ed dos­ing for its late-stage study of tomin­ersen, an an­ti­sense drug it li­censed from Io­n­is in hopes of tack­ling Hunt­ing­ton’s dis­ease.
  • A failed Phase IIa study left lit­tle to sal­vage for Fre­quen­cy Ther­a­peu­tics’ four-shot treat­ment reg­i­men for hear­ing loss. But the ex­ecs, who in­sist the tri­al de­sign is to blame, still sees a way for­ward with a sin­gle dose.
  • No­var­tis says a $2 bil­lion drug in its ra­dio­phar­ma­ceu­ti­cals port­fo­lio has de­liv­ered “ground­break­ing” da­ta for prostate can­cer, hit­ting both co-pri­ma­ry end­points on over­all sur­vival and ra­di­ograph­ic pro­gres­sion-free sur­vival while set­ting them up for a po­ten­tial block­buster fu­ture.

DIS­COV­ERY

  • A pair of vet­er­ans of George Church’s lab tracked the poop of 200 wild an­i­mals over the last four years. Now, they say they’ve gath­ered a vast trove of ge­net­ic in­for­ma­tion that could be mined for new drugs on a long list of dis­eases — with back­ing from an Is­raeli bil­lion­aire.
  • Can a “su­per-sea­son­al” flu shot of­fer­ing pro­tec­tion for three to five years bridge the gap be­tween cur­rent and uni­ver­sal vac­cines? The NIH and the Uni­ver­si­ty of Wash­ing­ton are tak­ing a shot in­to the clin­ic this April af­ter pub­lish­ing an­i­mal da­ta sug­gest­ing broad re­sponse to their nanopar­ti­cle tech­nol­o­gy.

REG­U­LA­TO­RY

  • No­vo Nordisk has been slammed with a refuse-to-file let­ter from the FDA re­gard­ing a once-week­ly semaglu­tide in­jec­tion at the 2 mg dose lev­el, a rare re­buke for the Dan­ish di­a­betes drug mak­er’s most high-pro­file pro­gram.
  • Safe­ty sig­nals ob­served in a post­mar­ket­ing study have spurred the FDA to add a new warn­ing to the la­bel of Glax­o­SmithK­line’s shin­gles vac­cine Shin­grix, not­ing it was as­so­ci­at­ed with an in­creased risk of a rare but de­bil­i­tat­ing dis­or­der known as Guil­lain-Bar­ré syn­drome.
  • About two and a half years af­ter sell­ing off roy­al­ties on a dis­ap­point­ing Sanofi-part­nered di­a­betes drug, Zealand Phar­ma has a new in-house prod­uct to boast about. The in­jectable dasiglucagon will be sold as Ze­ga­logue to treat se­vere hy­po­glycemia.
  • Mer­ck may be on the fir­ing line for ac­cel­er­at­ed ap­provals of Keytru­da, but that hasn’t stopped it from adding a new ap­proval for the PD-1 in­hibitor as a com­bi­na­tion ther­a­py with chemo to treat front­line metasta­t­ic esophageal can­cer or gas­troe­sophageal junc­tion car­ci­no­ma.

CORO­N­AVIRUS

  • The US will no longer dis­trib­ute Eli Lil­ly’s bam­lanivimab alone due to the in­creas­ing spread of coro­n­avirus vari­ants, al­though hos­pi­tals can still or­der the an­ti­body as part of a com­bo. The coun­try­wide halt fol­lows the gov­ern­ment’s de­ci­sion to stop de­liv­er­ing the monother­a­py to three states where vari­ants ran ram­pant.
  • Glax­o­SmithK­line and Vir are sub­mit­ting their mon­o­clon­al an­ti­body, VIR-7831, for an emer­gency use au­tho­riza­tion in mild-to-mod­er­ate Covid-19 pa­tients, pur­su­ing a sim­i­lar in­di­ca­tion as cur­rent­ly au­tho­rized an­ti­bod­ies based on re­cent­ly an­nounced Phase III da­ta.
  • Al­bert Bourla de­clared that Pfiz­er is ready to go so­lo, if nec­es­sary, on fu­ture mR­NA en­deav­ors, hav­ing ac­crued decades worth of ex­pe­ri­ence over the past year work­ing with BioN­Tech on the ul­tra-fast de­vel­op­ment of a Covid-19 vac­cine. The cur­rent pipeline, though, still ap­pears to lean heavy on the Ger­man part­ner.
  • Re­gen­eron says it will seek an EUA for a low­er dose of its an­ti­body cock­tail, which was shown to re­duce hos­pi­tal­iza­tion or death by 70% in a new Phase III study. The 1,200 mg dose ap­peared just as good as the au­tho­rized 2,400 mg dose, with some ev­i­dence in­di­cat­ing that even small­er dos­es, as low as 300 mg, might al­ready be enough.

START­UP

  • A group be­hind the tech­nol­o­gy lead­ing to J&J’s Covid-19 vac­cine has scored some $47 mil­lion to test out a new kind of pre­ven­ta­tive prod­uct: a nasal spray that can pro­tect peo­ple from broad virus fam­i­lies tem­porar­i­ly. It’s ear­ly days, but a look un­der the hood was ev­i­dent­ly enough to get GV, F-Prime and some sea­soned an­tivi­ral ex­perts ex­cit­ed.
  • RA Cap­i­tal’s low-pro­file small mol­e­cule in­cu­ba­tor has spawned a neu­ro up­start, with $80 mil­lion to go af­ter hy­per­ex­citabil­i­ty dis­or­ders from chron­ic pain to epilep­sy to de­pres­sion. “It’s a lot about ex­e­cu­tion,” CEO Bob Azel­by said, which is where the ex­pe­ri­enced C-suite comes in.
  • Mi­crosoft and Sam Alt­man are back­ing a self-pro­fessed AI en­thu­si­ast’s take at a com­pu­ta­tion­al drug dis­cov­ery plat­form, with am­bi­tions span­ning oph­thal­mol­o­gy, in­fec­tious dis­ease, neu­rol­o­gy, im­munol­o­gy and ag­ing.

PHAR­MA

  • No­var­tis’ top ranks are ea­ger to sep­a­rate the com­pa­ny from its trou­bled past — but le­gal records are keep­ing them ground­ed. In an un­usu­al state­ment, the Swiss drug­mak­er pushed back on com­ments from chair­man Jo­erg Rein­hardt claim­ing they nev­er bribed doc­tors, call­ing it “in­ac­cu­rate and in­con­sis­tent” with what they ad­mit­ted in court.
  • Thanks to a $150 mil­lion en­dow­ment from Er­ic and Wendy Schmidt, the Broad In­sti­tute is open­ing a new cen­ter to ex­plore what di­rec­tor Todd Gol­ub calls the dual rev­o­lu­tions of bi­ol­o­gy and ma­chine learn­ing. A few phar­ma part­ners have al­ready gath­ered at its wings.
  • Ab­b­Vie is on the of­fen­sive over Hu­mi­ra two years be­fore the first biosim­i­lars to the megablock­buster are ex­pect­ed to hit the mar­ket, tak­ing one Ice­landic de­vel­op­er to court over al­le­ga­tions it stole trade se­crets to cre­ate its copy­cat ver­sion.
  • Con­tin­u­ing its fight against a Trump-era rule to al­low drug im­ports from Cana­da, in­dus­try group PhRMA has now pe­ti­tioned the FDA to re­ject New Mex­i­co’s im­port plan be­cause of miss­ing de­tails that may make the im­port­ed drugs un­safe and not cost-ef­fec­tive.

FI­NANC­ING

  • Biotech VC bets con­tin­ue to run the gamut as Gy­ro­scope grabbed $148 mil­lion on the heels of pos­i­tive da­ta on its dry AMD gene ther­a­py, Brii capped a tu­mul­tuous month with $155 mil­lion in fresh fund­ing for an in­fec­tious dis­ease pipeline, while the pro­tein print­ing shop Ab­Sci closed a $125 mil­lion crossover round. Oth­er ven­ture ideas re­load­ing in cash this week re­volve around bis­pecifics, eye can­cer, IL-2s, CTPS1 and gene ther­a­pies for coro­nary heart dis­ease.
  • The erup­tion of biotech IPOs pro­pelled La­va Ther­a­peu­tics and its gam­ma delta T cell-tar­get­ing bis­pecifics to a $100.5 mil­lion raise to start the week. Edge­wise and De­sign Ther­a­peu­tics, Ike­na On­col­o­gy and Uni­verse Phar­ma­ceu­ti­cals fol­lowed, grab­bing a col­lec­tive $566 mil­lion for drugs tar­get­ing rare mus­cle ail­ments, de­gen­er­a­tive dis­or­ders, meta­bol­ic path­ways and the el­der­ly.
  • Things were qui­eter on the fil­ing side, though both of the new S-1s are no­table: Re­cur­sion is look­ing to take its AI plat­form pub­lic af­ter a hefty Se­ries D, while Zymer­gen would pave the way for syn­thet­ic bi­ol­o­gy com­pa­nies to de­but on Nas­daq. Chardan brought the lat­est SPAC deal, pulling Ren­o­va­cor on­to Wall Street to ad­vance an AAV‑based gene ther­a­py that would fix a rare heart mu­ta­tion.
  • With an eye on ear­ly-stage biotech, River­Vest Ven­ture Part­ners has closed its largest fund yet at $275 mil­lion. The goal would be to “par­tic­i­pate more ful­ly” in lat­er rounds and dou­ble down on its quick ex­it strat­e­gy.

MAN­U­FAC­TUR­ING

  • To rise up to the chal­lenge of mass-pro­duc­ing mR­NA vac­cines — at a cost and scale pre­vi­ous­ly un­thought of — the head of de­sign at Gink­go Bioworks drew in­spi­ra­tion from the project he was work­ing on be­fore the pan­dem­ic: mak­ing syn­thet­ic meat.
  • With sales of Zol­gens­ma flag­ging, No­var­tis is shut­ter­ing the Long­mont, CO man­u­fac­tur­ing plant it leased from As­traZeneca just a year ago. The de­par­ture leaves 400 em­ploy­ees with­out jobs, as the phar­ma gi­ant con­cen­trates pro­duc­tion of cur­rent and fu­ture gene ther­a­pies in two oth­er sites and con­tract net­works.
  • Set out to be a cen­tral play­er in the cell ther­a­py 2.0 move­ment, Lyell is prepar­ing to open a $65 mil­lion man­u­fac­tur­ing cen­ter in the Seat­tle area — one de­signed in a way that they “wouldn’t be fit­ting a square peg in­to a round hole in the fu­ture.”

DON’T MISS

  • More than two years af­ter emerg­ing from stealth, Maze Ther­a­peu­tics has opened up its $191 mil­lion pipeline for a first look. The pipeline fea­tures three pro­grams, in­clud­ing a new small mol­e­cule ap­proach for the rare lyso­so­mal dis­or­der Pompe dis­ease, a chron­ic kid­ney dis­ease tar­get that will put them in square com­pe­ti­tion with Ver­tex, and an ALS gene ther­a­py that the­o­ret­i­cal­ly could be mar­ket­ed for the en­tire pa­tient pop­u­la­tion.
  • 2021 was sup­posed to be the year Odonate filed an NDA for its metasta­t­ic breast can­cer drug. In­stead, the biotech is scrap­ping those plans and wind­ing down op­er­a­tions in one fell sweep af­ter de­ter­min­ing the da­ta pack­age is un­like­ly to sup­port ap­proval of its oral chemo. At last count, 153 staffers were on board.
  • Look­ing to repli­cate Roche’s suc­cess with Evrys­di, Take­da has racked up an­oth­er ear­ly-stage dis­cov­ery deal with Evotec to de­vel­op RNA tar­gets ripe for small mol­e­cule ther­a­peu­tics. The pact builds on oth­er col­lab­o­ra­tions with the Ger­man play­er.
  • UK soft­ware provider In­sight­ful Sci­ence is ac­quir­ing Dot­mat­ics, a cloud-based sci­en­tif­ic R&D da­ta man­age­ment plat­form found­ed by two ex-Mer­ck sci­en­tists and based in San Diego, to beef up its cloud re­search plat­form.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, 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.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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