A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

2022-08-06
Endpts
IPO
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A $5B Pfiz­er buy­out?
Pfiz­er CEO Al­bert Bourla has vowed to leave no stone un­turned in the search for new biotech deals, and the BD team is not let­ting him down. Ru­mor has it (or rather, the Wall Street Jour­nal re­port­ed) that Pfiz­er is in the fi­nal stages of ac­quir­ing Glob­al Blood Ther­a­peu­tics for $5 bil­lion. Ac­cord­ing to the Jour­nal re­port, though, Pfiz­er is not the on­ly buy­er at the deal ta­ble and while the phar­ma gi­ant may be close to clinch­ing it, there are no guar­an­tees it will con­tin­ue.
M&A Thurs­day
On the same day Am­gen CEO Bob Brad­way bel­lied up to the M&A ta­ble to scoop up the new­ly anoint­ed com­mer­cial biotech Chemo­Cen­tryx and its re­cent­ly ap­proved rare dis­ease drug for $3.7 bil­lion out of the cash stock­pile, Gilead en­gi­neered its own buy­out to cush­ion its im­mune check­point pipeline. The $405 mil­lion deal for Miro­Bio adds an an­ti­body dis­cov­ery plat­form for po­ten­tial treat­ments of in­flam­ma­to­ry dis­eases.
Al­ny­lam’s AT­TR sweep
In a land­mark win, Al­ny­lam laid claim to a ma­jor suc­cess in Phase III, with its RNAi drug patisir­an hit­ting the pri­ma­ry end­point in its APOL­LO-B study for AT­TR amy­loi­do­sis with car­diomy­opa­thy. One of the most im­por­tant piv­otal tri­als to read out this year, the study re­sults sent Al­ny­lam’s shares soar­ing as it cel­e­brates a topline hit with the 6-Minute Walk Test. And that sets the stage for a quick march to the FDA in search of a 2023 mar­ket launch.
An­drew Lo’s rare dis­ease quest
A decade ago, An­drew Lo des­per­ate­ly sought out a ther­a­py for his moth­er’s lung can­cer that had de­vel­oped re­sis­tance to ex­ist­ing drugs. What he learned in that process set him on a quest to change the way rare dis­ease drugs are de­vel­oped and paid for. Now, the MIT fi­nance pro­fes­sor’s pro­pos­als are rip­pling through biotech and get­ting test­ed in the re­al world. Jared Whit­lock’s pro­file takes us deep­er in­to his sto­ry.
The search for male con­tra­cep­tives
Years ago, Big Phar­ma aban­doned male con­tra­cep­tive de­vel­op­ment, putting to bed one clin­i­cal-stage hor­mon­al con­tra­cep­tive, lay­ing off teams and evap­o­rat­ing a li­brary of a cou­ple of hun­dred pos­si­ble tar­gets. A new crop of star­tups has emerged to build up the field — de­spite a dearth of in­dus­try sup­port — to launch mul­ti­ple clin­i­cal tri­als in the com­ing years to con­vince fun­ders and reg­u­la­tors that they can halt sperm from reach­ing the egg. And the over­turn­ing of Roe v. Wade is shed­ding more light on their quest, Kyle LaHu­cik re­ports.
PRE­MI­UM
Re­gen­eron plots I/O move
Two of the most out­spo­ken — and suc­cess­ful — drug de­vel­op­ers in biotech say they’ve col­lect­ed ear­ly-stage clin­i­cal da­ta that are point­ing them down the trail to the holy grail in can­cer im­munother­a­py R&D. Re­gen­eron chief sci­en­tist George Yan­copou­los and CEO Len Schleifer used the Q2 call to spot­light their ear­ly suc­cess with a com­bi­na­tion of the “home­grown” PS­MAx­CD28 cos­tim­u­la­to­ry bis­pe­cif­ic an­ti­body REGN5678 in com­bi­na­tion with their PD-1 check­point Lib­tayo, which is now ful­ly theirs af­ter re­gain­ing rights from Sanofi.
Gran­flu­encers take up room in phar­ma
Cathy Chester’s lat­est In­sta­gram post is about her ex­pe­ri­ence be­ing preg­nant while hav­ing mul­ti­ple scle­ro­sis — in 1992. Chester is one of the faces of a new gen­er­a­tion of old­er so­cial in­flu­encers build­ing big au­di­ences and com­mu­ni­ties on­line. Of­ten called Gran­flu­encers or Boom­flu­encers — the 55+ group of ag­ing adults with ma­jor sway in­clude fash­ion­istas, re­la­tion­ship ad­vice gu­rus, fit­ness buffs and much more — they’ve be­come health con­di­tion lead­ing voic­es like Chester, Beth Bu­lik writes.
Q2 EARN­INGS
“Chill­ing,” “oner­ous” and “his­toric mis­take”: These were some of the words on tape as Big Phar­ma CEO took their turns dur­ing Q2 calls with an­a­lysts blast­ing the Sen­ate De­moc­rats’ drug pric­ing re­form. Ac­cord­ing to them, en­abling Medicare ne­go­ti­a­tions would stymie in­no­va­tion — al­though some were will­ing to ac­knowl­edge that re­form is need­ed.
As Mod­er­na pre­pares two bi­va­lent vari­ant-spe­cif­ic Covid-19 boost­ers for de­liv­ery in the fall and looks in­to a po­ten­tial mon­key­pox pro­gram, CEO Stéphane Ban­cel stressed on the Q2 call that “now is not the time to slow down.” For now, the com­pa­ny ex­pects pub­lic health au­thor­i­ties to be the key pur­chasers — al­though it’s al­so look­ing at po­ten­tial pri­vate com­mer­cial mar­kets.
Eli Lil­ly had some drea­ry Q2 num­bers to re­port. Ac­cord­ing to Lil­ly, that was due to both low­er prices for some prod­ucts — in­sulin was the stand­out — as well as a for­eign ex­change hit as a strong dol­lar bites and a drop of 13% on Covid-19 rev­enue. Look­ing ahead at drug pric­ing re­forms, CEO David Ricks warns it could force his com­pa­ny to move away from small mol­e­cule re­search and fo­cus on oth­er ar­eas.
Speak­ing with an­a­lysts af­ter his shiny new ac­qui­si­tion, Am­gen CEO Bob Brad­way added his voice to the cho­rus of phar­ma CEOs lament­ing the pend­ing drug pric­ing leg­is­la­tion but didn’t linger for long. Rather, he want­ed to talk about the “ex­cit­ing” ad­di­tions to the au­toim­mune/in­flam­ma­to­ry pipeline and up­com­ing biosim­i­lar launch­es.
For­get buy­er’s re­morse, Pfiz­er is like­ly feel­ing pret­ty good about its $11.6 bil­lion Bio­haven takeover deal fol­low­ing re­ports of a 57% sales boost for mi­graine med Nurtec. Bio­haven al­so re­port­ed in Q2 re­sults that it’s cleared the nec­es­sary an­titrust hur­dles to move for­ward with the sale of its CGRP pro­grams as­sets to Pfiz­er.
Bay­er CEO Wern­er Bau­mann high­light­ed “strong op­er­a­tional per­for­mance” while rais­ing the full-year guid­ance. But be­fore div­ing in­to the de­tails, he sum­ma­rized Bay­er’s plan to set­tle a slate of on­go­ing lit­i­ga­tion re­lat­ing to the ac­tive in­gre­di­ent of its weed­killer Roundup, ex­press­ing con­fi­dence that the ma­jor cas­es will soon be be­hind it.
With pro­duc­tion fi­nal­ly up and run­ning for its weight loss drug We­govy, No­vo Nordisk said it plans to make all dose strengths avail­able in the US by the end of the year. How­ev­er, CEO Lars Fruer­gaard Jør­gensen al­so not­ed “a bit low­er ramp-up ver­sus planned” as it gets back on track fol­low­ing a short­age stem­ming from is­sues with a con­tract man­u­fac­tur­er.
Boehringer In­gel­heim’s phar­ma di­vi­sion sales led com­pa­ny earn­ings for the first half of 2022, notch­ing a sales in­crease of al­most 12%, with the di­a­betes brand Jar­diance lead­ing the way at €2.5 bil­lion and id­io­path­ic pul­monary fi­bro­sis drug Ofev com­ing at €1.5 bil­lion in the first half. Thanks to ex­pan­sions in­to the car­dio­vas­cu­lar space, Jar­diance’s sales jumped by more than 74% com­pared to the first half of 2021 sales.
Gilead CEO Dan O’Day un­corked some sol­id Q2 re­sults, tout­ing a surge in Yescar­ta and Trodelvy sales and an uptick in de­mand for HIV med Bik­tarvy, de­spite sink­ing sales of the Covid-19 an­tivi­ral Vek­lury. Com­ment­ing on the $2.7 bil­lion im­pair­ment charge they put on Trodelvy, ex­ecs said they took a “con­ser­v­a­tive ap­proach” and will re­view the val­ue lat­er.
One of Hori­zon Ther­a­peu­tics’ top sell­ers isn’t per­form­ing as well as ex­pect­ed, CEO Tim Wal­bert re­vealed on his Q2 call, lead­ing the phar­ma com­pa­ny to switch up its strat­e­gy. Wal­bert told in­vestors that get­ting the thy­roid eye dis­ease drug Te­pez­za and its rep­re­sen­ta­tives in­to oph­thal­mol­o­gists’ and en­docri­nol­o­gists’ of­fices is of ut­most im­por­tance.
STAR­TUPS
Alex­is Borisy and Ben Aus­pitz want to be in­tel­li­gent with their lat­est biotech, not “stu­pid.” That’s in the words of Borisy, the re­peat biotech en­tre­pre­neur be­hind the likes of Blue­print Med­i­cines and EQRx. The duo wants to bring to­geth­er “in­tel­li­gent­ly de­signed” can­cer drugs, hence their start­up’s name: IDRx. With $122 mil­lion in hand, their first pro­gram will be a com­bo of two com­pounds from Mer­ck KGaA and Blue­print.
Five years af­ter form­ing and with more than $100 mil­lion from mar­quee back­ers, a biotech from BeiGene co-founder Xi­aodong Wang is back with an­oth­er whop­ping in­vest­ment, this time at $200 mil­lion. Siron­ax said the new funds will bankroll stud­ies of drugs for age-re­lat­ed de­gen­er­a­tive dis­eases, in­clud­ing two RIPK1 in­hibitors al­ready in the clin­ic.
Look­ing to ap­ply plant-based sup­ply to the man­u­fac­tur­ing of cell ther­a­pies, French biotech Core Bio­gen­e­sis se­cured $10.5 mil­lion in a Se­ries A to push its tech for­ward. The com­pa­ny us­es cameli­na, oth­er­wise known as wild flax tra­di­tion­al­ly used in gas­tron­o­my, to man­u­fac­ture pro­teins from the seeds of the plant.
Tak­ing its next big step in­to the al­lo­gene­ic CAR-T field, Roche is ty­ing up along­side Po­sei­da Ther­a­peu­tics with $220 mil­lion in up­front and quick cash in a trade­mark on­col­o­gy al­liance that comes with a moth­er lode of mile­stones. The deal of­fers the phar­ma gi­ant dibs on a slate of off-the-shelf CAR-Ts for hema­to­log­ic ma­lig­nan­cies, in­clud­ing a BC­MA drug for mul­ti­ple myelo­ma.
Mean­while, Genen­tech is spin­ning its own deal to jump in­to fi­bro­sis with a pro­gram from Kiniksa. Genen­tech shelled out $80 mil­lion in up­front cash and an­oth­er $20 mil­lion in near-term pay­ments to li­cense Kiniksa’s vixare­limab, for­mer­ly known as KPL-716, and shep­herd it in­to new clin­i­cal stud­ies.
Sanofi found a high-pro­file part­ner in Chi­na to take two of its top can­cer drug prospects through the clin­ic and on­to the big Asian mar­ket: In­novent. Paris-based Sanofi spot­light­ed a deal with In­novent to ac­quire €300 mil­lion in the Chi­nese phar­ma’s shares, at a 20% markup, with an op­tion on €300 mil­lion more at a lat­er date. The drugs in­volved are an an­ti­body-drug con­ju­gate tar­get­ing CEA­CAM5 and a mid-stage IL-2 ther­a­py.
Ipsen con­tin­ued its deal­mak­ing ways to kick off the new month, shelling out a hand­some $45 mil­lion up­front to Maren­go Ther­a­peu­tics, a TCR up­start led by for­mer Mer­ck KGaA can­cer chief Zhen Su. The deal in­volves two ex­per­i­men­tal drugs out of Maren­go’s bis­pe­cif­ic an­ti­body plat­form. And the pay­off could be huge.
It took a cou­ple of ex­tra months to get all the reg­u­la­to­ry sign-offs, but CSL said it’s lined up all the nec­es­sary ap­provals to get its $11.7 bil­lion Vi­for buy­out done. Once the M&A deal is com­plete, cur­rent Vi­for com­mer­cial chief Hervé Gis­serot will move up to the gen­er­al man­ag­er’s role at the sub and re­port to CSL COO Paul McKen­zie.
Wa­ter­town, MA-based For­ma Ther­a­peu­tics has long made it clear that olu­tasi­denib, its oral in­hibitor of mu­tant isoc­i­trate de­hy­dro­ge­nase 1, is a non-core pro­gram. So per­haps it’s lit­tle sur­prise that right as the FDA ac­cepts its NDA, For­ma’s li­cens­ing the drug to Rigel Phar­ma­ceu­ti­cals in a $235 mil­lion deal.
Hav­ing turned to lay­offs ear­li­er this year to tight­en its fi­nan­cial belt, mi­cro­cap biotech Ava­lo Ther­a­peu­tics added some much-need­ed cash to the bank. The Rockville, MD-based biotech is sell­ing AVTX-007, the IL-18 an­ti­body orig­i­nal­ly from As­traZeneca’s now-de­funct Med­Im­mune, to the UK’s Apol­lo Ther­a­peu­tics, for $15 mil­lion up­front and $74 mil­lion in mile­stones.
Ab­b­Vie has got more work for So­sei Hep­tares. A year and a half af­ter break­ing off a lega­cy Al­ler­gan pact in­volv­ing a slate of So­sei neu­rol­o­gy can­di­dates, Ab­b­Vie drew up a whole slew of neu­ro tar­gets it wants to di­rect So­sei’s G pro­tein-cou­pled re­cep­tor ex­per­tise and struc­ture-based drug de­sign savvi­ness to­ward. The heav­i­ly back-end­ed deal starts with a $40 mil­lion up­front.
R&D
Four years af­ter promis­ing ear­ly da­ta in head and neck can­cer drew As­traZeneca to a big-mon­ey deal with In­nate Phar­ma around its next-gen­er­a­tion check­point in­hibitor mon­al­izum­ab, dis­ap­point­ing Phase III re­sults pushed As­traZeneca to shut­ter a study. The drug flunked a fu­til­i­ty in­ter­im analy­sis. The part­ners, though, will keep test­ing it in lung can­cer.
In the first few days of 2020, In­cyte re­port­ed that its JAK1 in­hibitor failed a Phase III study in first-line, acute graft vs host dis­ease, trig­ger­ing a stock melt­down. This time around, In­cyte isn’t even com­plet­ing a study of the drug, known as itac­i­tinib, be­cause ef­fi­ca­cy da­ta from part of a Phase II/III study give the bio­phar­ma no signs of hope for a piv­otal suc­cess in treat­ment-naïve chron­ic GVHD.
While Pfiz­er’s $11.4 bil­lion ac­qui­si­tion of Ar­ray Bio­Phar­ma in the sum­mer of 2019 was main­ly fo­cused on on­col­o­gy, name­ly Braftovi and Mek­tovi, there were a few non-can­cer as­sets, in­clud­ing a Phase III drug be­ing test­ed in a rare car­dio­vas­cu­lar dis­ease. That late-stage tri­al is now be­ing axed, along­side any fur­ther de­vel­op­ment of the oral small mol­e­cule, fol­low­ing an in­ter­im fu­til­i­ty analy­sis.
Am­gen is tak­ing its foot off the ped­al just a few weeks af­ter be­gin­ning a late-stage tri­al of a Ky­owa Kirin drug for which it dished out $400 mil­lion last year in ex­change for ex-Japan rights, paus­ing a Phase III tri­al to look at new dos­es. At the same time, it al­so swept a BC­MA BiTE out of the pipeline in a cleanup, while drop­ping a PS­MA can­di­date in fa­vor of an­oth­er.
While Neu­ro­crine Bio­sciences con­tin­ues to grow the sales of its tar­dive dysk­i­ne­sia drug for pa­tients who ex­pe­ri­ence un­com­mon side ef­fects from men­tal health drugs, it is giv­ing up on an es­sen­tial tremor drug that failed a Phase II study. Cit­ing “neg­a­tive” topline ef­fi­ca­cy re­sults, the com­pa­ny won’t study the in­di­ca­tion any fur­ther — al­though an epilep­sy study is on­go­ing.
As Pieris Phar­ma­ceu­ti­cals fa­vors an As­traZeneca-part­nered pro­gram in asth­ma, and be­cause the mid-stage study is tak­ing longer than ex­pect­ed, the biotech is de­cid­ing to end work on a HER2 drug. De­spite a cou­ple of clin­i­cal part­ner­ships, the biotech de­cid­ed to stop test­ing cin­re­ba­fusp al­fa (PRS-343) in gas­tric can­cer.
Less than six months in­to his jour­ney as CEO at Voy­ager, Al San­drock is nar­row­ing the fo­cus, con­cen­trat­ing on CNS dis­eases that were the hall­mark of his time lead­ing R&D at Bio­gen, in­clud­ing an em­pha­sis on a fa­mil­ial form of ALS, Alzheimer’s dis­ease and Parkin­son’s dis­ease. For the rest, the biotech will ex­plore po­ten­tial deals.
When you’ve got an al­liance as hefty as the one Ar­cus and Gilead have, every up­date counts. That’s why the com­pa­nies found an­a­lysts frown­ing on the lat­est dis­clo­sure around quem­li­clu­s­tat, their small mol­e­cule in­hibitor of CD73, sug­gest­ing a triplet com­bo looked sim­i­lar to chemother­a­py alone and they will need to wait for “more ma­ture” sur­vival da­ta. An­a­lysts didn’t hide their dis­ap­point­ment.
In April, Eliem Ther­a­peu­tics’ lead can­di­date, ETX-810, failed a Phase II study for di­a­bet­ic nerve pain, buck­ling the Seat­tle-based biotech’s stock by over half. Now, an­oth­er shot with the drug — a sec­ond Phase II tri­al for a form of back pain — al­so failed. The com­pa­ny did not sug­ar­coat the news, say­ing it will drop the drug al­to­geth­er.
Lit­tle Kazia Ther­a­peu­tics hit a ma­jor hur­dle de­vel­op­ing the Genen­tech castoff pax­al­is­ib for glioblas­toma. In a plat­form tri­al or­ga­nized by the Glob­al Coali­tion for Adap­tive Re­search, the PI3K in­hibitor failed to meet pre-de­fined cri­te­ria for con­tin­u­ing to a sec­ond stage — up­end­ing Kazia’s reg­u­la­to­ry plans, es­pe­cial­ly in Chi­na.
Ipsen’s grand plan to open up new mar­kets for the can­cer drug Onivyde hit a snag as its Phase III tri­al test­ing the ther­a­py in small cell lung can­cer failed the pri­ma­ry end­point on over­all sur­vival, cast­ing a shad­ow on its reg­u­la­to­ry prospects. But Ipsen isn’t giv­ing up on the drug, which it paid top dol­lar to li­cense from Mer­ri­mack, and oth­er tri­als are still on­go­ing.
The US-Dan­ish biotech Al­lar­i­ty Ther­a­peu­tics is hav­ing to piv­ot its pipeline strat­e­gy af­ter the FDA forced it to go back to the draw­ing board for an on­col­o­gy ther­a­py. For the com­pa­ny, the agency’s re­quest for a new dos­ing study means its for­mer lead drug is no longer com­mer­cial­ly vi­able. It will now re­fo­cus its strat­e­gy around com­bi­na­tion ther­a­pies.
What hap­pens if you try a sec­ond time and again don’t suc­ceed? Tra­vere Ther­a­peu­tics will be ask­ing it­self that ques­tion af­ter a sec­ond at­tempt to gun for ac­cel­er­at­ed ap­proval was re­ject­ed by the FDA. The for­mer Mar­tin Shkre­li biotech had at­tempt­ed to push through its lead drug, sparsen­tan, to a speedy OK in a rare kid­ney dis­ease, but reg­u­la­tors pre­fer the more me­thod­i­cal, full ap­proval route.
Con­cert Phar­ma­ceu­ti­cals now has the fi­nal batch of Phase III da­ta it needs to take its hair loss drug to the FDA. Af­ter sell­ing off the bulk of its pipeline, the biotech un­veiled topline re­sults from its sec­ond piv­otal tri­al of CTP-543 in adult pa­tients with mod­er­ate to se­vere alope­cia area­ta sug­gest­ing the JAK1/JAK2 in­hibitor could re­duce alope­cia sever­i­ty.
Last week, Oton­o­my thought it was head­ed to­ward a Phase III tri­al in the first half of next year for its drug to re­duce ring­ing and buzzing in the ear. But “un­ex­pect­ed” re­sults show­ing its OTO-313 did not beat place­bo in the con­di­tion known as tin­ni­tus is forc­ing it to switch course, ax­ing the pro­gram and shift­ing pri­or­i­ties to the on­ly oth­er clin­i­cal-stage as­set in its pipeline.
PEO­PLE
In the af­ter­math of a se­ries of FDA clin­i­cal holds, David Hung’s Nu­va­tion Bio de­cid­ed to dis­con­tin­ue de­vel­op­ment on one of its ear­ly-stage clin­i­cal pro­grams that was the sub­ject of the hold. The CDK in­hibitor was linked to cas­es of in­flam­ma­tion in the eye. On top of this, Nu­va­tion plans to lay off 30 em­ploy­ees, rep­re­sent­ing about 35% of its work­force.
Near­ly two years af­ter his ar­rest as part of a smug­gling in­ves­ti­ga­tion, Frank Zhang is back in the game. Zhang, the founder and for­mer CEO of Gen­Script Biotech, has been ap­point­ed as a di­rec­tor of Leg­end Biotech, the CAR-T spe­cial­ist still ma­jor­i­ty owned by Gen­Script. Charges against Zhang were dropped ear­li­er this year, when he al­so re­turned to a non-ex­ec­u­tive di­rec­tor role at Gen­Script.
Nav­i­gat­ing the chop­py biotech wa­ters af­ter rais­ing a mod­est IPO last year, Evax­ion Biotech, a Dan­ish start­up that us­es AI to de­vel­op per­son­al­ized im­muno-on­col­o­gy ther­a­pies, is shak­ing up its C-suite. Lars Staal Weg­n­er has de­cid­ed to ex­it as the com­pa­ny’s CEO. Per Nor­lén, who has over 20 years in the in­dus­try, will take over as the new chief in six months.
The FDA slapped a hold on Beam Ther­a­peu­tics’ pre­clin­i­cal off-the-shelf CAR-T ther­a­py, staving off its ap­pli­ca­tion to be­gin clin­i­cal tri­als in pa­tients with re­lapsed/re­frac­to­ry T-cell acute lym­phoblas­tic leukemia and lym­phoma. Beam, a pi­o­neer in base edit­ing co-found­ed by David Liu, has yet to pro­vide any in­sight in­to the rea­son be­hind the hold.
Gene ther­a­py biotech MeiraGTx is look­ing to get some quick cash and is putting up its man­u­fac­tur­ing fa­cil­i­ties to do it. In an un­usu­al arrange­ment, the com­pa­ny clinched a loan with af­fil­i­ates of Per­cep­tive Ad­vi­sors for up to $100 mil­lion. The mon­ey should ex­tend the com­pa­ny’s cash run­way for the next two years and in­to Q4 of 2024.
David Hal­lal’s El­e­vate­Bio is launch­ing a new com­pa­ny with Boston Chil­dren’s Hos­pi­tal fea­tur­ing re­search from George Da­ley, the dean of Har­vard Med­ical School. Oth­er than the fact that they’re work­ing on off-the-shelf cell ther­a­pies, Hal­lal is keep­ing his cards close to the vest, pre­fer­ring to toe the line be­tween bom­bast and mys­tique rather than go­ing all-in in one di­rec­tion.
As the cell ther­a­py field ma­tures, two CD­MOs are com­ing to­geth­er in a merg­er to ex­pand their reach. Lykan Bio­science, based in Mass­a­chu­setts, and RoslinCT, based in Ed­in­burgh, UK, will be­come a sin­gle ad­vanced ther­a­pies CD­MO of­fer­ing process de­vel­op­ment ex­per­tise as well as cGMP man­u­fac­tur­ing for a broad range of au­tol­o­gous and al­lo­gene­ic cell ther­a­pies.
FI­NANC­ING
As the for­mer glob­al brand leader of Mer­ck’s an­ti­fun­gal busi­ness, Francesco Maria Lavi­no knows his facts: There are on­ly three class­es of an­ti­fun­gals. But F2G, the biotech where he’s now CEO, is con­fi­dent that it can soon bring a fourth an­ti­fun­gal class to the mar­ket. And it’s closed $70 mil­lion in new fi­nanc­ing to get there.
Amid a bear mar­ket, pub­lic com­pa­nies have raced to in­vestors pri­mar­i­ly on­ly af­ter show­ing im­pres­sive clin­i­cal da­ta or reach­ing oth­er promi­nent mile­stones. Ar­cutis Bio­ther­a­peu­tics fol­lowed that path: Days af­ter the FDA ap­proved its pso­ri­a­sis drug, the com­pa­ny looked to raise $150 mil­lion in a pub­lic of­fer­ing.
The lead­ers of Blue Wa­ter Vac­cines are join­ing the wave of blank check op­er­a­tors ditch­ing plans to dock their boat on Wall Street. While the Blue Wa­ter duo of Joseph Her­nan­dez and Jon Garfield was suc­cess­ful in get­ting their first SPAC to merge with a biotech, they have de­cid­ed to back out of the SPAC mar­ket, fol­low­ing the suit of many oth­ers.
CORO­N­AVIRUS
Re­gen­eron ter­mi­nat­ed four stud­ies of its mon­o­clon­al an­ti­body com­bi­na­tion, known as RE­GEN-COV, months af­ter the FDA halt­ed use of the drug for which the fed­er­al gov­ern­ment had doled out bil­lions of dol­lars. As the EUA was rolled back and a BLA re­view has gone be­yond the ex­tend­ed de­ci­sion date.
As Covid-19 cas­es mount once again and mil­lions of peo­ple ex­pe­ri­ence lin­ger­ing symp­toms months af­ter di­ag­no­sis, Ax­cel­la Ther­a­peu­tics wants to treat long Covid, but like oth­ers in the past, it failed to meet the pri­ma­ry goal of a mid-stage study. The biotech isn’t giv­ing up on the pan­dem­ic dis­ease’s long-term ef­fects, though, and will at­tempt to per­suade reg­u­la­tors in the US and UK with fa­tigue scores from the Phase IIa in dis­cus­sions about a piv­otal tri­al.
For as long as it’s ex­ist­ed, Ake­bia has al­ways billed it­self as a kid­ney dis­ease spe­cial­ist. But as it grap­ples with a cost­ly FDA re­jec­tion of its lead ane­mia drug, the biotech is do­ing what­ev­er it can to stay afloat — and that could mean hold­ing on­to the drug’s po­ten­tial in treat­ing acute res­pi­ra­to­ry dis­tress syn­drome even af­ter fail­ing a tri­al.
It was late last year when the biotech Green­Light Bio­sciences inked a deal with CD­MO gi­ant Sam­sung Bi­o­log­ics to man­u­fac­ture its mR­NA Covid-19 vac­cine. Now that Sam­sung com­plet­ed the first com­mer­cial-scale en­gi­neer­ing run for Green­Light’s Covid-19 vac­cine boost­er can­di­date, Green­Light took the oc­ca­sion to cel­e­brate and tout its mR­NA plat­form.
PHAR­MA
Roche is look­ing to score a class win in the hy­per­com­pet­i­tive PD-(L)1 field. The phar­ma gi­ant put out word that its sub­cu­ta­neous ver­sion of Tecen­triq looked equiv­a­lent to the IV in a piv­otal phar­ma­co­ki­net­ic study, putting the com­pa­ny on track to jump ahead with an eas­i­er-to-ad­min­is­ter for­mu­la­tion that could prove dis­tinct­ly at­trac­tive to pa­tients.
A week af­ter ad­vo­cates called up­on Vi­iV Health­care to low­er the price of its long-act­ing HIV in­jectable Apre­tude, the com­pa­ny struck a deal with the Med­i­cines Patent Pool to make the drug more ac­ces­si­ble in low- and mid­dle-in­come coun­tries. The li­cens­ing agree­ment will al­low gener­ic man­u­fac­tur­ers in 90 coun­tries to de­vel­op, man­u­fac­ture and sup­ply their own ver­sions of Apre­tude, pend­ing ap­proval.
Mark­ing some set­backs in its grand plans around the PD-1 cash cow, Mer­ck re­port­ed Keytru­da has missed two in­di­ca­tions — as a first-line ther­a­py in com­bi­na­tion with Ei­sai’s Lenvi­ma for liv­er can­cer, and al­so as a lat­er-line treat­ment for metasta­t­ic prostate can­cer. The drug brought in $5 bil­lion in the last quar­ter.
Near­ly a decade af­ter J&J’s Ste­lara was ap­proved to treat adults with pso­ri­at­ic arthri­tis, the phar­ma gi­ant is ex­pand­ing the la­bel to in­clude the small per­cent­age of chil­dren who suf­fer from the au­toim­mune dis­ease. Be­cause of a lim­it­ed avail­abil­i­ty to pe­di­atric PsA pa­tients, reg­u­la­tors based their ap­proval on safe­ty and ef­fi­ca­cy da­ta ex­trap­o­lat­ed from mul­ti­ple Phase III stud­ies in adult and pe­di­atric pa­tients with plaque pso­ri­a­sis and PsA.
LAW
Pros­e­cu­tors lost an ap­peal to length­en the sen­tences of two for­mer GSK sci­en­tists who plead­ed guilty to steal­ing trade se­crets. While they sought to put the re­searchers be­hind bars for years, the court stuck with the orig­i­nal sen­tence be­cause their new com­pa­ny nev­er prof­it­ed off the stolen in­for­ma­tion and pros­e­cu­tion didn’t prove they in­tend­ed to harm GSK.
In a case brought by wel­fare-ben­e­fit plans, a fed­er­al ap­peals court ruled in Ab­b­Vie’s fa­vor, up­hold­ing an Illi­nois court’s de­ci­sion to dis­miss al­le­ga­tions that the phar­ma gi­ant cre­at­ed an un­law­ful “patent thick­et” around its block­buster drug Hu­mi­ra. “But what’s wrong with hav­ing lots of patents?” the judge wrote in his opin­ion.
Less than a week af­ter Te­va inked a $4.25 bil­lion deal to set­tle opi­oid-re­lat­ed lit­i­ga­tion in sev­er­al states, many of the same states are now tout­ing a sim­i­lar deal with Ab­b­Vie. Ab­b­Vie’s Al­ler­gan unit agreed to pay up to $2.37 bil­lion (in­clud­ing set­tle­ments al­ready reached with in­di­vid­ual states) to state and lo­cal gov­ern­ments af­fect­ed by the opi­oid cri­sis.
Bausch Health will con­tin­ue lit­i­gat­ing over Xi­fax­an, the an­tibi­ot­ic used to treat ir­ri­ta­ble bow­el syn­drome with di­ar­rhea and one oth­er con­di­tion, threat­en­ing to ap­peal all the way up to the US Court of Ap­peals for the Fed­er­al Cir­cuit. Go­ing up against Nor­wich — now part of gener­ics mak­er Alvo­gen — Bausch and its Sal­ix unit are look­ing to re­tain the patent.
MAR­KET­INGRX
Want a pre­scrip­tion drug with that so­da? While not di­rect­ly pos­si­ble, ads for phar­ma brands now run­ning on bev­er­age and snack cool­er screens at phar­ma­cy re­tail­ers may at least in­spire cus­tomers to think about it. Op­ti­miz­eRx is hook­ing up with Cool­er Screens me­dia com­pa­ny to bring pre­scrip­tion drug ad­ver­tis­ing to re­frig­er­a­tor front doors at phar­ma­cies in­clud­ing Wal­greens, CVS and Kroger.
Add se­niors’ ad­vo­cate AARP to the list of groups push­ing hard for pro­posed drug pric­ing leg­is­la­tion. The group’s most re­cent tar­get­ed TV ad cam­paign push­es con­gres­sion­al rep­re­sen­ta­tives to act. Its spe­cif­ic fo­cus? “Put mon­ey back in the pock­ets of se­niors” through the part of the bill that would al­low Medicare to ne­go­ti­ate drug prices.
Bris­tol My­ers Squibb’s lat­est mul­ti­ple scle­ro­sis ini­tia­tive is go­ing to work — lit­er­al­ly. Its new “MS Work Space” pro­gram tar­gets em­ploy­ers with a suite of on­line ed­u­ca­tion and ad­vice to help them sup­port pa­tients with MS. Com­pa­nies can use the web­site to tap the re­sources and can get even more help or in­for­ma­tion from BMS.
Bio­gen’s short film about spinal mus­cu­lar at­ro­phy talks about the be­hind-the-scenes care giv­en to SMA pa­tients. The “In­vis­i­bles” work, cre­at­ed by VM­LY&R Health Spain, was launched ear­li­er this sum­mer to raise aware­ness about SMA in gen­er­al as ef­forts to add new­born screen­ing for SMA across Eu­rope are in­creas­ing.
A new study from Bio­haven and the Amer­i­can Mi­graine Foun­da­tion is link­ing mi­graine to men­tal health; Eye­care brand Bausch + Lomb is ask­ing peo­ple to dance with their eyes in a new Tik­Tok chal­lenge for its Lu­mi­fy OTC brand to treat eye red­ness; Kaiser Health and Politi­co dis­put­ed claims in a new po­lit­i­cal ad about drug pric­ing leg­is­la­tion; and oth­er snip­pets await in this week’s Mar­ket­ingRx roundup.
FDA+
Will a trio of new boss­es at the FDA — a new agency com­mis­sion­er, head of CDER and head of OPDP — lead to an in­crease in drug ad­ver­tis­ing en­force­ment? With few let­ters sent out so far, there’s just not enough in­for­ma­tion to tell, says one long­time FDA reg­u­la­to­ry watch­dog who doesn’t see the ad­min­is­tra­tion lean­ing one way or an­oth­er yet.
De­spite win­ning an ap­proval back in 2016, Aca­dia’s Nu­plazid has not had the eas­i­est of paths in its jour­ney to ex­pand in­to new ar­eas. It just hit an­oth­er road­block as the FDA re­ject­ed Aca­dia’s ap­pli­ca­tion to ex­pand Nu­plazid use in­to Alzheimer’s-re­lat­ed psy­chosis. In the re­jec­tion let­ter, the FDA al­so rec­om­mend­ed that Aca­dia con­duct an ad­di­tion­al tri­al in ADP.
Four months ago, the FDA placed a clin­i­cal hold on Celyad’s CAR-T tri­al for col­orec­tal can­cer af­ter two pa­tients died dur­ing the study. Reg­u­la­tors have now lift­ed the hold, al­low­ing the Bel­gian com­pa­ny’s tri­al to set sail once again. Celyad was test­ing its ther­a­py in com­bi­na­tion with Mer­ck’s Keytru­da and not­ed that both pa­tients “pre­sent­ed with sim­i­lar pul­monary find­ings.”
In a new guid­ance sur­round­ing changes re­gard­ing dis­pos­able ma­te­ri­als, the FDA stat­ed that any changes to dis­pos­able ma­te­ri­als used pri­mar­i­ly in drug and bi­o­log­i­cal prod­uct man­u­fac­tur­ing, in­clud­ing at con­tract man­u­fac­tur­ing sites, must “be avail­able up­on re­quest dur­ing an FDA in­spec­tion.”
Move over, Lu­cen­tis: The FDA ap­proved the first in­ter­change­able biosim­i­lar to Roche’s block­buster an­ti-VEGF drug, name­ly Co­herus’ Cimer­li. The copy­cat ver­sion is ex­pect­ed to hit the mar­ket in Oc­to­ber af­ter get­ting the FDA’s thumbs-up in five reti­nal in­di­ca­tions. Cimer­li isn’t the first biosim­i­lar to ri­val Roche’s eye drug — but it is the first to win in­ter­change­able sta­tus, mean­ing it can be sub­sti­tut­ed at the phar­ma­cy in some cas­es.
MAN­U­FAC­TUR­ING
Quite lit­er­al­ly reach­ing for the stars to launch new man­u­fac­tur­ing op­er­a­tions, Eli Lil­ly is part­ner­ing with a Flori­da-based space in­fra­struc­ture man­u­fac­tur­er named Red­wire to part­ner on de­vel­op­ing space tech­nol­o­gy for drug man­u­fac­tur­ing. The first step will be “to con­duct crit­i­cal test­ing dur­ing the ini­tial flight mis­sions” in low Earth or­bit on its first ex­per­i­ments.
Ther­mo Fish­er com­plet­ed a $76 mil­lion ex­pan­sion of one of its man­u­fac­tur­ing fa­cil­i­ties in New York, which spe­cial­izes in dry pow­der me­dia man­u­fac­tur­ing. Part of a broad­er plan to beef up ca­pa­bil­i­ties glob­al­ly, it will ex­tend its ca­pa­bil­i­ties for ma­te­ri­als used in vac­cines and bi­o­log­ic ther­a­py de­vel­op­ment and man­u­fac­tur­ing.
While its name brings to mind sticky notes and pack­ag­ing tape, 3M does al­so man­u­fac­ture a range of con­sumer and com­mer­cial prod­ucts. It is now plan­ning to spin out its health­care prod­ucts di­vi­sion in­to a sep­a­rate pub­licly trad­ed com­pa­ny, in­cor­po­rat­ing prod­ucts used in the man­u­fac­tur­ing of vac­cines and re­com­bi­nant pro­teins.
Fresh off an ex­pan­sion a year ago, CD­MO Cu­ria is scal­ing up man­u­fac­tur­ing for a cannabi­noid can­di­date from Aus­tralia’s In­can­nex Health­care. Cu­ria is tasked with de­vel­op­ing and pro­duc­ing a GMP-grade in­haled drug prod­uct for the treat­ment of con­cus­sion and trau­mat­ic brain in­jury . The com­pa­ny al­so tapped a new pres­i­dent in Niall Con­don, as Steve Lichter re­tires.
Ger­man glass­mak­er Schott — which has been man­u­fac­tur­ing glass for a wide range of us­es for 138 years and has built up a ro­bust health­care di­vi­sion — will es­tab­lish a stand­alone com­pa­ny specif­i­cal­ly for its phar­ma busi­ness. As part of the carve-out, the phar­ma side will op­er­ate un­der the new name of Schott Phar­ma AG & Co. KGaA.
So­ci­etal CD­MO scored ser­vice con­tracts from three new cus­tomers; Chime Bi­o­log­ics, which net­ted in­vestor cash in 2021 for a 15x man­u­fac­tur­ing ex­pan­sion, re­ceived a com­mer­cial man­u­fac­tur­ing li­cense in Chi­na; Te­va hit a sup­ply snag with its pop­u­lar AD­HD drug Adder­all; learn more in the Man­u­fac­tur­ing roundup.
DON’T MISS
Ris­ing mon­key­pox cas­es have put the US on high alert as it an­nounces a na­tion­al health emer­gency, which grants the gov­ern­ment more pow­er in its re­sponse. The news comes as Bavar­i­an Nordic con­tin­ues to fill or­ders for its Jyn­neos vac­cine and oth­er com­pa­nies — in­clud­ing Mod­er­na — con­sid­er jump­ing in­to the vac­cine race.
Re­searchers spent close to a decade look­ing for signs that the failed Alzheimer’s drug crenezum­ab might work in a study that re­strict­ed it­self to a group of fam­i­lies with a rare ge­net­ic mu­ta­tion that left them sus­cep­ti­ble to ear­ly-on­set dis­ease. While it failed, AC Im­mune still put a pos­i­tive spin on the dataset as it presents the num­bers at a con­fer­ence.
With the ris­ing num­ber of mon­key­pox cas­es, lead­ing re­searchers at the CDC, FDA and NIH are call­ing on a ran­dom­ized clin­i­cal tri­al to see if an ap­proved small­pox drug is ef­fec­tive at treat­ing mon­key­pox. No mon­key­pox treat­ments are ap­proved in the US, so pa­tients look­ing to get re­lief for their le­sions and oth­er symp­toms must go through a set of hur­dles to ac­cess a stock­pile of drugs un­der an ex­pand­ed ac­cess pro­gram.
Sen­ate De­moc­rats may have all the votes they need to pass ma­jor drug pric­ing re­form, af­ter Sen. Kyrsten Sine­ma (D-AZ) re­port­ed­ly pledged her sup­port — but the ques­tion is now whether they can fit it in be­fore re­cess.
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