Leg­endary biotech leader pass­es away, re­newed ten­sion around ad­u­canum­ab, Mer­ck­'s rare de­feat, and more

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Leg­endary Gilead CEO dies

John C. Mar­tin, who led Gilead to great­ness in de­vel­op­ing a huge­ly prof­itable HIV drug fran­chise, has died at the age of 69. Mar­tin helmed the com­pa­ny for a 20-year stretch just six years af­ter join­ing as VP of R&D back in 1990, dur­ing which Gilead built a rep as a fear­some com­peti­tor in HIV, field­ing drug cock­tails that kept the dead­ly AIDS at bay for mil­lions — while al­so be­ing tar­get­ed by crit­ics for be­ing tone deaf on pric­ing con­tro­ver­sies. Daniel O’Day, who took over as Mar­tin and his close col­league and suc­ces­sor John Mil­li­gan both left a cou­ple of years ago, not­ed his lega­cy “will be felt for gen­er­a­tions to come.”

Ad­u­canum­ab dra­ma es­ca­lates

The re­view of Bio­gen’s Alzheimer’s drug ad­u­canum­ab has been any­thing but typ­i­cal. And the ten­sion is grow­ing even more as three mem­bers of the FDA’s ad­vi­so­ry com­mit­tee — who have al­ready of­fered their per­spec­tive in a meet­ing — laid out their case for why the agency should re­ject ad­u­canum­ab in an op-ed in JA­MA. The ed­i­to­r­i­al brings the com­mit­tee’s con­cerns back to the fore two months be­fore the dead­line for reg­u­la­tors to make a de­ci­sion. “The FDA needs to get the sci­ence right,” an au­thor told End­points News in an in­ter­view. “They usu­al­ly do and I be­lieve in this in­stance they will as well.”

Rare snub for Mer­ck

Af­ter get­ting a unan­i­mous thumbs down from the ad­comm, Mer­ck has been hand­ed an un­sur­pris­ing CRL over its ap­pli­ca­tion to mar­ket Keytru­da for high-risk triple-neg­a­tive breast can­cer. The ex­pert pan­el had vot­ed no af­ter the FDA can­cer czar, Richard Paz­dur, per­son­al­ly took the stage to ad­mon­ish Mer­ck for rush­ing the fil­ing be­fore the da­ta were there to sup­port it — a rare and sting­ing re­buke for a phar­ma gi­ant used to cruis­ing its PD-1 to­ward new des­ti­na­tions with grace.

At long last, a BC­MA CAR-T

It took much longer than Bris­tol My­ers Squibb had hoped, but it’s fi­nal­ly clinched an ap­proval for ide-cel, the BC­MA CAR-T ther­a­py for mul­ti­ple myelo­ma it got from Cel­gene. The drug, now dubbed Abec­ma, will sport a list price of $419,500. Once a close­ly-watched event be­cause of its weight in a $6 bil­lion CVR, the ap­proval in mul­ti­ple myelo­ma re­ceived a rel­a­tive­ly mut­ed wel­come as it was large­ly ex­pect­ed, even though cer­tain de­tails on the la­bel still sur­prised some an­a­lysts. While Bris­tol My­ers can now claim the hon­or as the first com­pa­ny to have two ap­proved CAR-Ts in its fold, ri­vals like J&J may be catch­ing up soon.

Bris­tol My­ers Squibb’s tax trou­ble

Bris­tol My­ers Squibb could be on the hook for $1.4 bil­lion in back tax­es ac­cord­ing to an ac­ci­den­tal dis­clo­sure from the IRS, New York Times re­port­ed. The US gov­ern­ment has bat­tled Big Phar­ma for years over off­shore patent trans­fer schemes. At the cen­ter of this par­tic­u­lar case is Bris­tol My­ers’ ef­fort in 2012 to es­tab­lish an Irish sub­sidiary as a ve­hi­cle to shut­tle US drug patent rights over to a coun­try with low­er cor­po­rate tax rates. For its part, the com­pa­ny says it’s in com­pli­ance with ap­plic­a­ble reg­u­la­tions and is work­ing to re­solve the mat­ter.

Gene edit­ing run-in

On the sur­face, In­tel­lia was giv­ing a pret­ty rou­tine — if ex­cit­ing — pre­sen­ta­tion at a Cold Spring Har­bor Lab con­fer­ence about a new ap­proach to gene edit­ing. There was just one prob­lem: They didn’t cred­it or ac­knowl­edge the sci­en­tists be­hind the ear­ly pa­pers de­tail­ing the first base edit­ing sys­tems, from which the CRISPR biotech said they have dif­fer­en­ti­at­ed. It seemed “there was a cal­cu­lat­ed in­tent to present these as new,” said an at­tendee. Ja­son Mast spoke with David Liu and Alex­is Ko­mor, two key fig­ures in the pi­o­neer­ing work, to ex­plore why the pair feared that In­tel­lia was rewrit­ing his­to­ry in a way that could be detri­men­tal to young re­searchers.

Lil­ly sex­u­al ha­rass­ment

A month af­ter oust­ing CFO Josh Smi­ley for in­ap­pro­pri­ate con­duct, Eli Lil­ly is fac­ing new al­le­ga­tions that an ex­ec­u­tive and a se­nior man­ag­er at its Wash­ing­ton, DC of­fice sex­u­al­ly dis­crim­i­nat­ed against and ha­rassed mul­ti­ple women. Sonya Elling, a Lil­ly lob­by­ist of 16 years, ac­cused her su­per­vi­sor Leigh Ann Pusey of cre­at­ing a hos­tile en­vi­ron­ment and be­ing a “work­place bul­ly.” Shawn O’Neail, an­oth­er su­per­vi­sor hired by Pusey, al­leged­ly made “sex­u­al­ly ag­gres­sive ges­tures” to­ward the em­ploy­ees.


PRE­MI­UM

Bet­ter-own­er hy­poth­e­sis

Bridge­Bio is wrap­ping a busy, buzzy month with a $2.4 bil­lion al­liance to roll out their next drug in the US — in­fi­gra­tinib — which looks to be in the la­bel-writ­ing stage at the FDA as reg­u­la­tors set up a chip shot on a near-term ap­proval. Team­ing up with a well-con­nect­ed and in­flu­en­tial Swiss com­pa­ny, Neil Ku­mar is giv­ing away some com­mer­cial­iza­tion rights in ex­change for Helsinn shoul­der­ing a chunk of glob­al de­vel­op­ment costs as they look to score a se­ries of down­stream ap­provals. “It’s the bet­ter-own­er hy­poth­e­sis,” he tells John Car­roll.

Chim­ing in

As the FDA out­lines plans to up­date the dis­ease pro­gres­sion mod­els and how they’re used in drug de­vel­op­ment, No­var­tis, Genen­tech as well as BIO spelled out sug­ges­tions on what to fo­cus on and raised ques­tions on how the agency might use these mod­els to in­form the agency’s de­ci­sion mak­ing.


DEALS

  • With the clock tick­ing on Am­gen’s patents on block­buster im­munol­o­gy meds, Bob Brad­way is tak­ing a far-look­ing bet on a new path­way to treat im­mune sys­tem dis­eases. Seat­tle-based pri­vate biotech Rodeo Ther­a­peu­tics is get­ting $55 mil­lion up­front and $666 mil­lion in fu­ture mile­stones hinged large­ly on the suc­cess of small mol­e­cule drugs tar­get­ing an en­zyme known as 15-PGDH.
  • Al­ready a ma­jor play­er in ra­dio­phar­ma­ceu­ti­cals, No­var­tis is dou­bling down by ac­quir­ing rights to two new ther­a­pies from Switzer­land’s iTher­a­nos­tics. The drugs are de­signed to bind to fi­brob­last ac­ti­va­tion pro­teins on the sur­face of tu­mor cells and bring a ra­dioac­tive par­ti­cle there — shin­ing a light on a new tar­get just days af­ter de­clar­ing a prostate can­cer win.
  • Mer­ck is lin­ing up a buy­out deal to get its women’s health spin­out start­ed, set­ting aside up to $240 mil­lion to ac­quire Aly­dia Health, a med­ical de­vice com­pa­ny work­ing on tech­nol­o­gy to con­trol and treat ab­nor­mal post­par­tum uter­ine bleed­ing or he­m­or­rhage.
  • On a spree for dis­cov­ery deals, Take­da is tak­ing a chance on a low-pro­file play­er that promis­es to de­ploy its co­va­lent small mol­e­cule “fish­ing” plat­form against dif­fi­cult tar­gets, de­vel­oped by a pro­tégé of KRAS ex­pert Ke­van Shokat. The up­front and biobucks add up to $500 mil­lion over the next two years.
  • In a con­tin­ued cam­paign to crack down on big bio­phar­ma M&A, the FTC is try­ing to block Il­lu­mi­na’s pro­posed ac­qui­si­tion of Grail and its can­cer-de­tect­ing blood test, for which the se­quenc­ing gi­ant had agreed to pay $8 bil­lion. Il­lu­mi­na vowed to “vig­or­ous­ly de­fend” its buy­out — al­though it’s al­ready lost to the an­titrust watch­dog once.
  • Eli Cas­din and Kei­th Meis­ter have swooped in with a merg­er for their sec­ond SPAC, send­ing pro­teomics play­er So­ma­Log­ic to Nas­daq with a $1.23 bil­lion val­u­a­tion. Com­ing just three months af­ter the com­pa­ny’s Se­ries A ex­ten­sion, the com­pa­ny says the deal would al­low it to dou­ble down on its suite of test­ing tools.

FDA+

  • As Pres­i­dent Joe Biden un­veiled his $2 tril­lion in­fra­struc­ture plan, the White House al­so de­tailed mea­sures to make it much hard­er for large multi­na­tion­als — bio­phar­ma in­clud­ed — to avoid or evade tax­es by us­ing tax havens abroad.
  • The Biden ad­min­is­tra­tion is in no rush to nom­i­nate a per­ma­nent com­mis­sion­er, per­fect­ly hap­py for Janet Wood­cock to run the show in the act­ing role, Zach Bren­nan re­ports. But for­mer FDA of­fi­cials and com­mis­sion­ers are push­ing the Biden ad­min­is­tra­tion to nom­i­nate some­one quick­ly for the role. Here’s why.
  • For­mer FDA com­mis­sion­er Rob Califf called out the agency’s “un­rec­og­nized abil­i­ty to bad­ger spon­sors” on mat­ters of clin­i­cal tri­al di­ver­si­ty as a pan­el of ex­perts agreed they could play a more promi­nent role, list­ing pos­si­ble fol­low-up ac­tions to en­sure stud­ies are rep­re­sen­ta­tive.
  • House De­moc­rats have kicked off a process to of­fi­cial­ly over­turn the Trump ad­min­is­tra­tion’s last-minute at­tempt to tear down more than 95% of the FDA’s reg­u­la­tions, call­ing it a “ma­li­cious rule” that would cause chaos. Mean­while, the lat­est se­mi-an­nu­al reg­u­la­to­ry agen­da for HHS is con­spic­u­ous­ly light on bio­phar­ma-re­lat­ed rules, ei­ther pro­posed or fi­nal, for the FDA.
  • In an ef­fort to ease the process around pe­di­atric can­cer drug de­vel­op­ment, the FDA and EMA are of­fer­ing a new tem­plate to pro­vide com­pa­nies with an eas­i­er way to seek sci­en­tif­ic ad­vice from both agen­cies, fea­tur­ing more close­ly aligned time­lines for sub­mis­sions.
  • The FDA is putting its foot down on Mak­e­na, the pre-term birth drug mar­ket­ed by AM­AG Phar­ma­ceu­ti­cals (re­cent­ly ac­quired by Co­vis Phar­ma). A re­cent­ly pub­lished meta-analy­sis is not chang­ing their de­ci­sion to pull the ac­cel­er­at­ed ap­proval, the agency said in a pub­lic re­buke, af­ter the con­fir­ma­to­ry study failed to show a ben­e­fit.

CORO­N­AVIRUS

  • Vault­ing ahead on a post-Mar­tin Shkre­li turn­around, Hu­mani­gen an­nounced that its an­ti­body helped keep pa­tients with Covid-19 off ven­ti­la­tion in a Phase III tri­al. De­spite a da­ta dis­crep­an­cy, the biotech says it’s fil­ing a EUA as soon as pos­si­ble, po­ten­tial­ly giv­ing doc­tors just their sec­ond proven tool for help­ing pa­tients with se­vere dis­ease.
  • BioN­Tech, Pfiz­er’s rel­a­tive­ly qui­et Ger­man biotech part­ner for its Covid-19 vac­cine, made noise with its earn­ings re­port as it pro­ject­ed about €10 bil­lion ($11.5 bil­lion) in vac­cine rev­enue. The two com­pa­nies have been rack­ing up sup­ply con­tracts, just as a new re­al-world study from the CDC con­firms the high ef­fi­ca­cy of mR­NA vac­cines from them and Mod­er­na.
  • As vari­ants thwart the so­lo use of Eli Lil­ly’s Covid-19 an­ti­body bam­lanivimab, the phar­ma gi­ant has de­clared in­ter­im suc­cess at pair­ing it with a ri­val treat­ment from Glax­o­SmithK­line and Vir. Just what the re­sults on vi­ral loads mean on the re­spec­tive an­ti­bod­ies, though, re­main up for in­ter­pre­ta­tion.
  • The Gov­ern­ment Ac­count­abil­i­ty Of­fice con­clud­ed in a re­port that the US fed­er­al gov­ern­ment’s con­tri­bu­tion to re­search on Gilead’s an­tivi­ral remde­sivir did not re­sult in any patents, set­tling a long­stand­ing dis­pute over whether the gov­ern­ment can low­er the drug’s price by claim­ing co-in­ven­tor rights.
  • Al­though As­traZeneca’s Covid-19 vac­cine is safe and ef­fec­tive, new da­ta show that younger women are more fre­quent­ly see­ing the very rare cas­es of some­times fa­tal blood clots when com­pared to the gen­er­al pop­u­la­tion, the EMA says. More women re­ceived the vac­cine than men, and reg­u­la­tors warn it’s pre­ma­ture to draw any con­clu­sions.

FI­NANC­ING

  • On­col­o­gy dom­i­nat­ed the ven­ture rounds an­nounced this week, with a range of fla­vors on the menu: Genen­tech vets go­ing head-to-head with Mer­ck in I/O ($73 mil­lion); tar­get­ed ther­a­pies that don’t give in eas­i­ly to tu­mor re­sis­tance ($106 mil­lion); a Flag­ship start­up un­leash­ing its epige­nom­ic con­trollers against c-myc ($126 mil­lion); Pfiz­er’s ADC plat­form in the hands of an alum ($152 mil­lion), and so on. But neu­ro­mus­cu­lar play­er En­tra­da al­so squeezed in a $116 mil­lion crossover round, while Jen­nifer Doud­na’s newest CRISPR start­up nabbed $100 mil­lion and a vo­cal in­vestor.
  • The Jen­ner In­sti­tute, the Ox­ford group be­hind As­traZeneca’s Covid-19 vac­cine, just got fund­ing from CARB-X to pur­sue a dif­fer­ent kind of vac­cine for an in­fec­tion that’s not quite as press­ing, but nev­er­the­less on the rise: gon­or­rhea.

PEO­PLE

  • We now know where Mike Nal­ly is head­ed af­ter leav­ing the chief mar­ket­ing post at Mer­ck. Re­port­ed­ly passed up for the top job at Mer­ck, Nal­ly will be­come Flag­ship Pi­o­neer­ing’s lat­est CEO-part­ner and take the helm at ma­chine learn­ing-fo­cused Gen­er­ate Bio­med­i­cines.
  • Long­time Koch In­sti­tute di­rec­tor Tyler Jacks can fi­nal­ly wrap his 19-year tenure af­ter lock­ing in Matthew Van­der Hei­den, an MIT bi­ol­o­gy pro­fes­sor and can­cer cell me­tab­o­lism re­searcher, as his suc­ces­sor. Jacks’ next move? Re­turn “full time to the ex­cite­ment of the lab” while Van­der Hei­den steers the cen­ter to­ward ma­chine learn­ing and AI.
  • Bri­an Kas­par is ready to leave the Zol­gens­ma da­ta ma­nip­u­la­tion scan­dal be­hind. The found­ing CSO of AveX­is is tak­ing a board role at End­sulin, a small gene ther­a­py play­er, with an old bud­dy from the pi­o­neer­ing biotech. The goal? To take a shot at cur­ing type 1 di­a­betes.

CELL/GENE TX

  • Since the FDA slapped a clin­i­cal hold on its lead gene ther­a­py in De­cem­ber, uniQure has been work­ing with ex­perts to sort out what led to the sin­gle liv­er can­cer case caus­ing con­cern. The biotech now says it’s “high­ly un­like­ly” that its he­mo­phil­ia B drug was to blame — with “mul­ti­ple analy­ses” they will now take to reg­u­la­tors.
  • Over­flow­ing with ideas to shift the cell ther­a­py par­a­digm, Rick Klaus­ner’s Lyell has spun out some of its pro­tein en­gi­neer­ing re­search in­to a new biotech called Out­pace Bio, with a goal to “make step change in how we can pro­gram cells to be more ef­fi­ca­cious,” Max Gel­man re­ports.

R&D

  • Wave’s close­ly-watched an­ti­sense oligonu­cleotide pro­gram be­came the lat­est ca­su­al­ty in the cru­sade against Hunt­ing­ton’s dis­ease, forc­ing the biotech to scrap two drug can­di­dates and turn to a back-up ap­proach — a sec­ond time.
  • Dan­ish biotech Or­p­hazyme’s lead drug ari­mo­clo­mol — a sup­pos­ed­ly “pipeline in a prod­uct” mol­e­cule — has whiffed in a sec­ond Phase III rare dis­ease tri­al. It couldn’t pre­vent pro­gres­sion of in­clu­sion body myosi­tis in a 150-per­son study. An FDA de­ci­sion, though, still awaits for a dif­fer­ent in­di­ca­tion based on a sec­ondary end­point.
  • Io­n­is says one of the mid-stage, whol­ly-owned an­ti­sense drugs for rare, non-neu­ro­log­i­cal dis­eases that CEO Brett Mo­nia boast­ed about at JP Mor­gan ear­li­er this year has de­liv­ered a sol­id slate of pos­i­tive Phase II re­sults, po­ten­tial­ly set­ting it up for a block­buster show­down with Take­da.

PHAR­MA

  • Sanofi has tak­en home a sec­ond ap­proval for Sar­clisa, the key CD38 drug that it’s tak­ing in­to a ma­jor ri­val­ry with J&J. It’s now OK’d for use in com­bi­na­tion with Am­gen’s Kypro­lis and the steroid dex­am­etha­sone for adults with re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma who have re­ceived be­tween one and three pre­vi­ous lines of treat­ment.
  • In search of cash to fi­nance the spin­out of its Pfiz­er-part­nered con­sumer health di­vi­sion, Glax­o­SmithK­line has made the un­ortho­dox call to sell its roy­al­ty rights for Ex­elix­is drugs Cabome­tyx and Cometriq for $342 up­front — and promis­es of up to $50 mil­lion more from Roy­al­ty Phar­ma.
  • A boom­ing mar­ket for in­flam­ma­to­ry bow­el dis­ease drugs could be just the tick­et for Ab­b­Vie’s new im­munol­o­gy launch­es to fill Hu­mi­ra’s shoes, ac­cord­ing to SVB Leerink an­a­lyst Ge­of­frey Porges. But there’s plen­ty of meat left on the $32 bil­lion bone.
  • Putting a cap to one of the coun­try’s biggest health scan­dals, a French court found phar­ma com­pa­ny Servi­er guilty of de­cep­tion and manslaugh­ter around its di­a­betes drug Me­di­a­tor. France’s health med­i­cines agency was fined along­side Servi­er.

MAN­U­FAC­TUR­ING

  • The US Of­fice of Spe­cial Coun­sel has un­veiled the de­tails of a whistle­blow­er com­plaint from a for­mer FDA in­spec­tor who al­leged nu­mer­ous vi­o­la­tions at a Mer­ck man­u­fac­tur­ing plant in North Car­oli­na that’s now be­ing read­ied to make the J&J Covid-19 vac­cine. Among the al­le­ga­tions: the agency had learned of these is­sues as ear­ly as 2018 but failed to act on them.
  • When — not if — a flu pan­dem­ic comes around, Sanofi wants to be pre­pared for it. The French drug­mak­er, which pro­duces the world’s best­selling flu vac­cine, is drop­ping more than $703 mil­lion to build a new man­u­fac­tur­ing fa­cil­i­ty and in­crease the sup­ply of Flu­zone.
  • With its own vac­cine ef­forts lag­ging be­hind, Glax­o­SmithK­line has signed an­oth­er deal with No­vavax pledg­ing fill-fin­ish ca­pac­i­ty for 60 mil­lion dos­es of the re­com­bi­nant nanopar­ti­cle shots des­ig­nat­ed for the UK.

DON’T MISS

  • The­o­ries abound on why vir­tu­al­ly all Alzheimer’s drugs of the past two decades have failed. Stu­art Lip­ton, a lead­ing de­men­tia ex­pert who helped de­vel­op the last FDA-ap­proved Alzheimer’s drug, has pro­posed an al­ter­na­tive ex­pla­na­tion: Per­haps the amy­loid-clear­ing an­ti­bod­ies were “para­dox­i­cal­ly in­duc­ing more in­flam­ma­tion in the brain.” His lab is work­ing on a way to fix that.
  • With the bar­ri­ers of en­try to tra­di­tion­al biotech hubs like Boston and San Fran­cis­co get­ting ever high, is it time for com­pa­nies to take R&D homes else­where? A new re­port by JLL sug­gests so, with Seat­tle top­ping the list for growth in life sci­ences em­ploy­ment.
  • With plen­ty of gi­ants romp­ing and stomp­ing in the AI drug dis­cov­ery land­scape these days, star­tups must find a niche to break in. RA Cap­i­tal is seed­ing one such biotech, us­ing AI to learn physics and re­do­ing al­go­rithms in a way that founder Vir­ginia Burg­er says will open up the no­to­ri­ous class of in­trin­si­cal­ly dis­or­dered pro­teins.

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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