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An­oth­er round of ma­jor med­ical con­fer­ences is up­on us, and Kyle LaHu­cik is in Barcelona, bring­ing us live cov­er­age from the Eu­ro­pean So­ci­ety of Car­di­ol­o­gy Con­gress. We’ll be post­ing up­dates on our web­site through­out the week­end.

Mod­er­na goes on the patent of­fen­sive

Mod­er­na said in March it would be­gin en­forc­ing its Covid-19 vac­cine patents in wealthy coun­tries. In an ag­gres­sive move, it is su­ing Pfiz­er and BioN­Tech over their mR­NA shot, al­leg­ing that their ri­val Covid-19 vac­cine copied parts of Mod­er­na’s vac­cine tech­nol­o­gy that it had patent­ed be­tween 2010 and 2016, when it was de­vel­op­ing an mR­NA vac­cine for MERS. Con­sid­er Pfiz­er “sur­prised” — while a le­gal schol­ar be­lieves that Mod­er­na’s pre­vi­ous pledge about patent en­force­ment could be key here.

Roy­al­ty treat­ment 

In­vestors have pulled back from the biotech in­dus­try in re­cent months. But not Pablo Legor­re­ta, who qui­et­ly pi­o­neered a drug de­vel­op­ment niche that’s in de­mand. His com­pa­ny, Roy­al­ty Phar­ma, is dou­bling down on its spe­cial­ty in­vest­ment strat­e­gy with plans to de­ploy $10 bil­lion to $12 bil­lion in cap­i­tal in the next five years, up from a $7 bil­lion fore­cast two years ago. Legor­re­ta, who keeps a low pro­file and rarely grants in­ter­views, told End­points fea­tures ed­i­tor Jared Whit­lock that these beefed-up plans stem from the sec­tor’s large cap­i­tal needs both in the im­me­di­ate and dis­tant fu­ture.

Is a biotech recov­ery on the way?

The Covid im­pact — es­ca­lat­ing the biotech in­dus­try’s mis­sion to bring new treat­ments and vac­cines to the world in record time — led to a “sug­ar high,” and the sub­se­quent crash has been weigh­ing on dozens of drug de­vel­op­ers. But signs of a re­cov­ery are on the way, Kyle LaHu­cik re­ports, thanks to a se­ries of pos­i­tive clin­i­cal tri­al read­outs, ac­qui­si­tions, drug ap­provals and the pop­u­lar da­ta-to-fi­nanc­ing train. In a some­what brave move, Third Har­mon­ic, the At­las-backed start­up led by Na­tal­ie Holles, is test­ing the IPO wa­ters.

The mon­key­pox pipeline

Two years af­ter biotech and phar­ma sprung to ac­tion to cre­ate vac­cines and ther­a­pies in record time for the rapid­ly cir­cu­lat­ing Covid-19 pan­dem­ic, a hand­ful of drug de­vel­op­ers and vac­cine mak­ers are con­sid­er­ing their role in pre­vent­ing and treat­ing fu­ture cas­es of the WHO’s lat­est pub­lic health emer­gency: mon­key­pox. They in­clude Vir, Mod­er­na, Gilead — all play­ers in the Covid-19 race — and oth­ers like NightHawk Bio­sciences, Blue Wa­ter Vac­cines and Tonix Phar­ma­ceu­ti­cals. In the mean­time, reg­u­la­tors are work­ing with Bavar­i­an Nordic to as­sess whether some ex­pired dos­es of its Jyn­neos vac­cine can still be used.

Gene ther­a­pies and how to pay for them

As the Eu­ro­pean Com­mis­sion’s ap­proval of Bio­Marin’s he­mo­phil­ia A gene ther­a­py, Roc­ta­vian, marks the sec­ond OK for ex­pen­sive gene ther­a­pies for blood dis­or­ders in re­cent days (af­ter blue­bird’s Zyn­te­glo), their price tags are shin­ing a spot­light on out­comes-based pric­ing deals as both com­pa­nies look to lever­age the strong ef­fi­ca­cy and dura­bil­i­ty of their ther­a­pies. But ex­perts point out that such deals are of­ten se­cre­tive. Roc­ta­vian will cost $1.5 mil­lion while Zyn­te­glo car­ries a $2.8 mil­lion stick­er.

PRE­MI­UM

Nav­i­gat­ing vac­ci­na­tion slumps

It’s been a tough cou­ple of years for tra­di­tion­al vac­cine mak­ers. While the Covid-19 pan­dem­ic spurred a flur­ry of in­no­va­tion and turned a white-hot spot­light on the footrace for Covid vac­cine ap­provals, rou­tine vac­ci­na­tion rates plum­met­ed. Judy Stew­art, GSK’s head of vac­cines in the US, has weath­ered vac­cine in­dus­try ups and downs long be­fore the Covid-19 pan­dem­ic. She re­cent­ly spoke with End­points Mar­ket­ingRx ed­i­tor Beth Sny­der Bu­lik about GSK’s ef­forts be­fore and dur­ing Covid as well as phar­ma’s lat­est dri­ve for da­ta and in­no­va­tions through part­ner­ships and ac­qui­si­tions to help keep vac­ci­na­tions at the top of the mind.

DEALS

• Pfiz­er wasn’t the first — or on­ly — com­pa­ny that want­ed to buy Glob­al Blood Ther­a­peu­tics. In fact, Pfiz­er el­bowed a ri­val bid­der through­out two months of in­tense ne­go­ti­a­tions al­most right down to the last minute, ac­cord­ing to de­tails re­vealed in a new SEC fil­ing. But ul­ti­mate­ly, af­ter four pro­pos­als and a phone call from Pfiz­er CEO Al­bert Bourla to GBT chief Ted Love, it was Pfiz­er that won the bid­ding war, seal­ing the deal at $68.5 per share — or $5.4 bil­lion.
• Two Mon­days have come and gone with­out Mer­ck’s hot­ly-ru­mored takeover of Seagen, keep­ing ob­servers wait­ing. And ac­cord­ing to a new Bloomberg re­port, that’s be­cause the two sides have yet to agree on a price, stalling talks. While pre­vi­ous re­ports say Mer­ck could be look­ing at a $40 bil­lion bid, a re­cent loss in its patent dis­pute with Dai­ichi Sankyo could af­fect Seagen’s val­u­a­tion.
• Af­ter spend­ing five years on Finch Ther­a­peu­tics’s mi­cro­bio­me plat­form and a part­ner­ship around drugs for in­flam­ma­to­ry bow­el dis­ease, Take­da is now walk­ing away from it all. Finch, which has re­ceived a to­tal of $44 mil­lion from the pact, will al­so get all the da­ta and IP gen­er­at­ed dur­ing the col­lab­o­ra­tion.
• Af­ter pick­ing up two drugs from a Bob Langer spin­out in a deal ear­li­er this year, Al­con is stay­ing busy. The for­mer No­var­tis sub­sidiary fo­cused on eye dis­eases is ac­quir­ing Aerie Phar­ma­ceu­ti­cals in a $770 mil­lion buy­out to bag two ap­proved eye drugs, Rock­la­tan and Rho­pres­sa, as well as an ad­vanced pro­gram.

CELL/GENE TX

• CAR-T ther­a­pies tra­di­tion­al­ly have been made us­ing virus vec­tors. While CRISPR/Cas9 gene edit­ing promised to over­come some of its lim­i­ta­tions, com­bin­ing the two tech­nolo­gies proved chal­leng­ing. But in a new pa­per, re­searchers from UCSF de­scribe a way to im­prove the ef­fi­cien­cy of a less tox­ic non­vi­ral CAR-T man­u­fac­tur­ing method.
• Jeff Blue­stone’s Sono­ma Bio­ther­a­peu­tics is putting its cash re­serves to use and en­ter­ing a lease to de­vel­op an 83,000-square-foot man­u­fac­tur­ing and R&D cen­ter in Seat­tle. The com­pa­ny’s gene mod­i­fied Treg ther­a­pies, born out of the R&D lab in South San Fran­cis­co, will be made at the site.
• Armed with a $100 mil­lion grant, David Hal­lal’s El­e­vate­Bio will part­ner with the Uni­ver­si­ty of Pitts­burgh to man­u­fac­ture cell and gene ther­a­pies in Pitts­burgh. The 30-year deal will have El­e­vate­Bio lo­cate its Base­Camp and GMP man­u­fac­tur­ing fa­cil­i­ties in the city, with the site even­tu­al­ly run­ning gene edit­ing, in­duced pluripo­tent stem cell (iP­SC) and cell, vec­tor and pro­tein en­gi­neer­ing ef­forts.
• With its first base edit­ing clin­i­cal tri­al un­der­way for het­erozy­gous fa­mil­ial hy­per­c­ho­les­terolemia, Verve is ready to re­veal its sec­ond base edit­ing pro­gram: one go­ing af­ter ho­mozy­gous FH, a rar­er type of heart dis­ease in which pa­tients in­her­it two gene mu­ta­tions that im­pair their liv­er’s abil­i­ty to re­move bad cho­les­terol. The ther­a­py would turn off the ANGPTL3 gene per­ma­nent­ly.

STAR­TUPS

• Search­ing for new TCR tar­gets as well as bet­ter ways to en­gage them, an im­munother­a­py start­up emerged with tech­nol­o­gy out of Stan­ford and $40 mil­lion in Se­ries A fund­ing led by West­lake Vil­lage BioPart­ners, with par­tic­i­pa­tion from Light­speed Ven­ture Part­ners. In ad­di­tion to cell ther­a­pies, 3T Bio­sciences be­lieves its tar­gets can lead to bis­pecifics and pep­tide vac­cines.
• As com­pa­nies small and large be­gin to test the wa­ters and go main­stream with a se­ries of dif­fer­ent psy­che­delics, Free­dom Bio­sciences — a clin­i­cal-stage com­pa­ny de­vel­op­ing ther­a­peu­tics us­ing ke­t­a­mine and oth­er psy­che­delics — net­ted it­self $10.5 mil­lion in seed fi­nanc­ing. Found­ed by a long­time ex­pert in the field, Free­dom says its can­di­date is dif­fer­ent from oth­er ke­t­a­mine-based prod­ucts on the mar­ket sim­ply due to the ef­fi­ca­cy du­ra­tion.
• Al­ler­gan nev­er steered the de­pres­sion drug it scored from a $560 mil­lion ac­qui­si­tion of Nau­rex to ap­proval, in­stead tak­ing a $2.5 bil­lion write­down fol­low­ing suc­ces­sive Phase III fail­ures. But the founders and crew at Nau­rex are con­vinced this class of drugs — not the same one but next-gen­er­a­tion ren­di­tions — still has po­ten­tial, grab­bing it from Ab­b­Vie’s shelves. And they’re com­ing out of stealth with the start­up, Gate Neu­ro­sciences, now.
• A trio of No­var­tis vets is com­ing out of stealth with their idea of lever­ag­ing Akt in­hibitors, typ­i­cal­ly used to treat can­cers, for rare dis­eases that cause blood ves­sel mal­for­ma­tions. Vaderis Ther­a­peu­tics, their Medicxi-backed start­up, is ready to take a lead drug in­to the clin­ic for an 80-pa­tient proof-of-con­cept study.

FI­NANC­ING

• As mR­NA takes a more promi­nent perch, Eu­ro­pean biotech eTh­eR­NA raised $39 mil­lion to fur­ther de­vel­op its plat­form. The Se­ries B2 fea­tures two big-name in­vestors: Mod­er­na co-founder Ken­neth Chien and ex-Bay­er and Ther­mo Fish­er CEO Mar­i­jn Dekkers. Both of them are join­ing the biotech’s board of di­rec­tors.
• Rid­ing on the slow­ly bub­bling field of tar­get­ed ra­di­a­tion ther­a­py and go­ing af­ter a broad­er metasta­t­ic prostate can­cer in­di­ca­tion, Aus­tralian biotech Ad­van­Cell hopes to ad­dress a key prob­lem in tar­get­ed ra­di­a­tion ther­a­py — ra­dioiso­tope sup­ply — and now it has an ad­di­tion­al $12.4 mil­lion to back its idea.
• In the field of ra­di­oli­gand ther­a­py, PS­MA, an anti­gen over­ex­pressed in prostate can­cer cells, has loomed large. But Ak­tis On­col­o­gy is stay­ing away from it while try­ing to de­vel­op drugs against a myr­i­ad of oth­er can­cers. And now, the MPM-backed biotech has an ad­di­tion­al $84 mil­lion to de­vel­op that port­fo­lio and build out its ra­dioiso­tope sup­ply.
• UK-based biotech Mereo Bio­Phar­ma is fac­ing some heat from one of its prin­ci­pal share­hold­ers who are look­ing to make sev­er­al changes to the biotech’s board amid some re­cent road­blocks for the com­pa­ny. Rubric Cap­i­tal Man­age­ment, a New York-based cap­i­tal firm, wants to re­place four of the cur­rent board di­rec­tors. But Mereo isn’t oblig­ing eas­i­ly.

PEO­PLE

• Af­ter rais­ing heaps of cap­i­tal dur­ing the first two years of the Covid-19 pan­dem­ic to help bio­phar­mas shift to vir­tu­al clin­i­cal stud­ies, a few de­cen­tral­ized clin­i­cal tri­als providers have con­duct­ed lay­offs in re­cent months as they ad­just busi­ness pri­or­i­ties, End­points learned. That in­cludes pri­vate­ly held Med­able, which dis­closed a $304 mil­lion Se­ries D last Oc­to­ber, and Sci­ence 37, which al­so be­gan trad­ing last Oc­to­ber af­ter a SPAC merg­er.
• Fol­low­ing more than 50 years of gov­ern­ment ser­vice, An­tho­ny Fau­ci will step down in De­cem­ber as chief med­ical ad­vi­sor to Pres­i­dent Joe Biden, di­rec­tor of the NIH’s Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases and chief of his NI­AID lab on im­munoreg­u­la­tion. But he al­so em­pha­sized that he’s not re­tir­ing.
• Af­ter some repo­si­tion­ing — and a $126 mil­lion fi­nanc­ing round to take it down that di­rec­tion — Sin­ga­pore’s Tes­sa Ther­a­peu­tics has found a new cap­tain to steer the ship. Thomas Willem­sen is jump­ing from Take­da, where he was most re­cent­ly SVP, Asia-Pa­cif­ic, to spear­head Tes­sa’s dri­ve to de­vel­op new CAR-T ther­a­pies, in­clud­ing both au­tol­o­gous and off-the-shelf ones.
• Austin, TX-based Ae­glea Bio­Ther­a­peu­tics an­nounced it was un­der­tak­ing a cor­po­rate re­struc­tur­ing and re­duc­ing head­count in an at­tempt to fo­cus on now-lead can­di­date AGLE-177, for a rare ge­net­ic dis­ease. Amid the changes, pres­i­dent and CEO An­tho­ny Quinn stepped down from all po­si­tions, leav­ing gen­er­al coun­sel Jim Kas­ten­may­er in charge in the in­ter­im.
• Ker­ry Blan­chard re­signed as Ever­est Med­i­cines’ CEO and a mem­ber of the board of di­rec­tors, ef­fec­tive im­me­di­ate­ly. Af­ter spend­ing near­ly 17 years at Eli Lil­ly where he rose to the rank of VP, Blan­chard spent three years as a top ex­ec­u­tive for oth­er Chi­nese phar­mas be­fore head­ing up Ever­est. He led the com­pa­ny’s IPO, deal­mak­ing and de­vel­op­ment strat­e­gy, re­cent­ly cul­mi­nat­ing in the sale of Trodelvy re­gion­al rights back to Gilead for $280 mil­lion up­front.
• Just two days af­ter se­cur­ing FDA ap­proval for the first gene ther­a­py for a chron­ic con­di­tion, blue­bird bio’s chief reg­u­la­to­ry of­fi­cer was on to her next jour­ney. Af­ter an 11-year ca­reer at blue­bird, Anne-Vir­ginie Eggi­mann moved over to Tessera Ther­a­peu­tics, the Flag­ship-in­cu­bat­ed biotech that re­cent­ly at­tract­ed for­mer Ab­b­Vie vice chair Michael Sev­eri­no as CEO.
• Bay Area biotech Atara Bio­ther­a­peu­tics sub­mit­ted WARN no­tices to Cal­i­for­nia that it plans to lay off 77 em­ploy­ees at three dif­fer­ent sites with­in the state as part of a slim­ming plan that the biotech dis­cussed at its Q2 earn­ings call ear­li­er this month. The cell ther­a­py play­er says by re­duc­ing the cash burn, it hopes to be­come a lean­er or­ga­ni­za­tion cen­tered on R&D.

PHAR­MA

• Af­ter wrestling with the San­doz prob­lem for sev­er­al years, No­var­tis CEO Vas Narasimhan fi­nal­ly de­cid­ed to go with a spin­out of the gener­ics gi­ant as a sep­a­rate com­pa­ny. The phar­ma gi­ant says it will set up San­doz as an in­de­pen­dent com­pa­ny in H2 2023 af­ter nav­i­gat­ing the sep­a­ra­tion through reg­u­la­tors. But de­spite a large port­fo­lio and a pipeline of 15 biosim­i­lars, its weak fi­nan­cial per­for­mance means San­doz will have an un­cer­tain re­cep­tion on its own.
• Take­da scored a land­mark ap­proval for its dengue vac­cine, the first step in crack­ing open a po­ten­tial block­buster mar­ket and prov­ing it can side­step the dis­as­ter Sanofi ex­pe­ri­enced with Deng­vax­ia. In­done­sia ap­proved the vac­cine, for­mer­ly known as TAK-003 and now brand­ed Qden­ga, for the pre­ven­tion of dengue dis­ease re­gard­less of pri­or dengue ex­po­sure.
• As J&J and Ab­b­Vie duke it out with As­traZeneca and oth­er BTK com­peti­tors in chron­ic lym­pho­cyt­ic leukemia, the part­ners are cel­e­brat­ing a first for Im­bru­vi­ca in a pe­di­atric pop­u­la­tion. The drug was ap­proved as a sec­ond-line treat­ment for chron­ic graft ver­sus host dis­ease in chil­dren one year and old­er.
• In­cyte nabbed its sec­ond ap­proval from fed­er­al reg­u­la­tors for can­cer drug pemi­ga­tinib, brand­ed as Pe­mazyre — as the po­ten­tial for even more in­di­ca­tions awaits. The FDA ap­proved Pe­mazyre un­der pri­or­i­ty re­view for re­lapsed or re­frac­to­ry myeloid/lym­phoid neo­plasms with FGFR1 re­arrange­ment, an ex­treme­ly rare can­cer sub­type.

R&D

• No­var­tis sus­pend­ed dos­ing in a Hunt­ing­ton’s dis­ease study af­ter safe­ty sig­nals emerged, spark­ing con­cerns that the drug, branaplam, may be caus­ing pe­riph­er­al neu­ropa­thy. The wor­ry­ing find­ings in­clude changes in a bio­mark­er sug­gest­ing neu­ronal dam­age and neu­ro­log­i­cal ex­ams, as well as numb­ness or tick­ling sen­sa­tions re­port­ed by pa­tients.
• The 50-year race to de­vel­op an RSV vac­cine turned a cor­ner with Pfiz­er un­veil­ing promis­ing piv­otal da­ta for its bi­va­lent RSV vac­cine can­di­date, promis­ing to take the da­ta to the FDA ahead of sev­er­al ri­vals in a mar­ket that could stretch to $10 bil­lion by 2030. Pfiz­er said the can­di­date demon­strat­ed vac­cine ef­fi­ca­cy of 66.7% in a Phase III tri­al.
• Two months af­ter re­veal­ing the topline da­ta — and quick­ly go­ing to the mar­ket for a cap­i­tal in­fu­sion of $95 mil­lion — 89bio is now pre­sent­ing the mid-stage re­sults of its high-triglyc­eride drug at the Eu­ro­pean So­ci­ety of Car­di­ol­o­gy Con­gress. In pa­tients with se­vere hy­per­triglyc­eridemia, 89bio’s drug per­formed bet­ter than place­bo. It will en­ter Phase III next year.
• No­vo Nordisk is one step clos­er to un­lock­ing a new semaglu­tide fran­chise — one it hopes can spur even bet­ter re­sults for peo­ple with type 2 di­a­betes. It post­ed pos­i­tive Phase II topline re­sults with Ca­griSe­ma, a sub­cu­ta­neous com­bi­na­tion of semaglu­tide and an ex­per­i­men­tal amylin ana­logue dubbed ca­gril­in­tide, sug­gest­ing “nu­mer­i­cal­ly high­er” re­duc­tions in blood sug­ar lev­els and body weight than ei­ther drug alone.
• Just a month af­ter bring­ing out the bud­get axe, PACT Phar­ma is now show­ing more of what it has planned next. The com­pa­ny’s sole clin­i­cal tri­al has now been sus­pend­ed. Ac­cord­ing to clin­i­cal­tri­als.gov, the pause is due to a “busi­ness de­ci­sion,” with CEO Scott Gar­land not­ing via email that the com­pa­ny is adding a new fo­cus on tar­get­ing shared neoanti­gens, plan­ning to put a pro­gram in the clin­ic next year.
• The UK-based Ox­ford Uni­ver­si­ty re­searchers be­hind the suc­cess­ful Covid-19 treat­ment tri­al RE­COV­ERY are back again, this time cob­bling to­geth­er a ran­dom­ized con­trolled tri­al to see if Siga Tech­nolo­gies’ small­pox treat­ment Tpoxx (tecovir­i­mat) is safe and ef­fec­tive for mon­key­pox. The NIH plans to launch a sim­i­lar tri­al in the US next week.

LAW

• GSK called on the US Supreme Court not to re­vive its case against Te­va Phar­ma­ceu­ti­cals re­gard­ing so-called “skin­ny” gener­ic drug la­bels, where cer­tain in­di­ca­tions are carved out so gener­ics can come to mar­ket more quick­ly. The case at hand in­volves GSK’s win over Te­va in a long-run­ning bat­tle over the gener­ic gi­ant’s skin­ny la­bel for GSK’s be­ta-block­er Coreg.
• A long­time Kad­mon con­sul­tant plead­ed guilty to a sin­gle count of se­cu­ri­ties fraud in con­nec­tion to more than $360,000 in prof­its he made trad­ing on in­sid­er knowl­edge of the com­pa­ny’s im­pend­ing $1.9 bil­lion Sanofi buy­out. Frank Glass­ner was ar­rest­ed back in May.
• A Cal­i­for­nia fed­er­al ap­peals court let Ab­b­Vie off the hook from a whistle­blow­er law­suit al­leg­ing the com­pa­ny held fraud­u­lent patents on two Alzheimer’s drugs, there­fore over­charg­ing Medicare. The case traces back to a com­plaint filed by patent lawyer Zachary Sil­ber­sh­er back in 2018 against Al­ler­gan, which was lat­er ac­quired by Ab­b­Vie.
• A cou­ple months af­ter Bris­tol My­ers Squibb brought its patent fight with Gilead’s Kite unit to the Supreme Court, Gilead is now mak­ing the ar­gu­ment that the pe­ti­tion should be de­nied be­cause it chal­lenges more than 50 years of prece­dent. The dis­pute dates back to 2017, be­fore Juno was ac­quired by Cel­gene and then got swal­lowed by Bris­tol My­ers Squibb.
• The man­u­fac­tur­er of a gener­ic tablet used in med­ica­tion abor­tions with­drew its case against Mis­sis­sip­pi state re­quire­ments that re­strict­ed ac­cess to abor­tions. Gen­Bio­Pro, which filed the orig­i­nal suit in 2020, at­trib­uted the de­ci­sion to “the changed na­tion­al land­scape.”
• Weeks af­ter snap­ping up the chron­ic kid­ney dis­ease drug Velphoro in its $11.7 bil­lion Vi­for buy­out, CSL pulled in a court win that gives it at least a few more years on the mar­ket with­out com­pe­ti­tion from Te­va. A judge rude that Te­va’s gener­ic can­di­date for Velphoro — which has yet to be ap­proved — would in­fringe on a patent held by what is now part of CSL Vi­for.
• In­di­v­ior isn’t get­ting out of its class ac­tion Sub­ox­one law­suit with­out a tri­al, a fed­er­al judge ruled. Its two mo­tions for a sum­ma­ry judg­ment were dis­missed in a case al­leg­ing the com­pa­ny com­mit­ted a “prod­uct hop” scheme to pre­vent gener­ic com­pe­ti­tion to its opi­oid ad­dic­tion treat­ment Sub­ox­one by switch­ing to the sub­lin­gual film and with­draw­ing pills.

CORO­N­AVIRUS

• A House sub­com­mit­tee fo­cused on the pan­dem­ic re­leased an­oth­er scathing re­port de­tail­ing the ex­tent to which the White House in­ject­ed pol­i­tics in­to a time-sen­si­tive sit­u­a­tion. The 69-page re­port found that the Trump White House “ex­ert­ed ex­treme and in­ap­pro­pri­ate pres­sure” on FDA and for­mer FDA com­mis­sion­er Stephen Hahn to not on­ly reau­tho­rize hy­drox­y­chloro­quine’s EUA, but de­liv­er mis­lead­ing­ly pos­i­tive news about con­va­les­cent plas­ma.
• Us­age of Pfiz­er’s Covid-19 treat­ment Paxlovid has spiked across the US in re­cent months, with more than 2 mil­lion cours­es ad­min­is­tered dur­ing this lat­est Omi­cron wave since June. But a new ob­ser­va­tion­al study pub­lished in NE­JM found that Paxlovid on­ly worked to avoid hos­pi­tal­iza­tion among pa­tients 65 years of age or old­er, with no ev­i­dence of ben­e­fit found in younger adults.
• Three months af­ter bring­ing on a new CEO, In­ovio reached a set­tle­ment with in­vestors over ac­cu­sa­tions that it ex­ag­ger­at­ed progress on its Covid-19 vac­cine can­di­date. The biotech, once claim­ing to be at the heart of Op­er­a­tion Warp Speed, agreed to shell out $44 mil­lion in a mix of cash and stock to end a class ac­tion law­suit.

MARK­ERT­IN­GRX

• Mod­er­na wants peo­ple to know it’s not a typ­i­cal phar­ma com­pa­ny, and its newest cam­paign hits that point home with a leg­endary sports and so­cial change agent. Bil­lie Jean King kicks off Mod­er­na’s “Here’s to the Change Mak­ers” cam­paign and its in­au­gur­al spon­sor­ship of the US Open that in­cludes a TV ad fea­tur­ing ten­nis footage clips from the ’70s and ’80s, with a mes­sage from her to­day.
• How should phar­ma com­pa­nies use the lim­it­ed char­ac­ter space in a pro­mo­tion­al tweet? While in­clud­ing risk in­for­ma­tion in the body of a post made pa­tients more like­ly to rec­og­nize po­ten­tial dan­gers, new FDA-led re­search sug­gests it al­so made them less like­ly to pur­sue more in­for­ma­tion. The re­searchers note the study re­sults sup­port FDA guid­ance on this top­ic.
• Ab­b­Vie is en­list­ing re­al peo­ple — in­clud­ing pa­tients along­side its own em­ploy­ees and sci­en­tists — in un­script­ed so­cial me­dia ads un­der its cor­po­rate cam­paign “Here. Now.” The so­cial and dig­i­tal on­line video ads fea­ture the peo­ple talk­ing about Ab­b­Vie sci­ence and med­i­cines and the per­son­al con­nec­tions for each.
• When col­lege stu­dent Amalia de­vel­oped de­bil­i­tat­ing stom­ach pain and even­tu­al­ly an in­abil­i­ty to eat or drink, she turned to art and paint­ing to ex­press her pain and hope­less feel­ings. She was even­tu­al­ly di­ag­nosed with the rare ge­net­ic dis­ease acute he­pat­ic por­phyr­ia — and now both Amalia and her art are fea­tured in Al­ny­lam Phar­ma­ceu­ti­cals’ lat­est cam­paign for its med Givlaari, ap­proved in 2019 to treat AHP in adults.
• No­var­tis’ first-ever pa­tient DTC ad for metasta­t­ic breast can­cer drug Kisqali points up the broad­er and more re­cent pro­lif­er­a­tion of mBC treat­ment op­tions in gen­er­al. The 15-sec­ond TV com­mer­cial fea­tures mBC pa­tient and Kisqali user Lau­ren, who is a wife and moth­er of three girls, and talks about be­ing the “first gen­er­a­tion” of peo­ple who will change what it means to live with metasta­t­ic breast can­cer.
• It’s “lights, cam­era, ac­tion” at Klick Health these days with the of­fi­cial open­ing of its own in-house pro­duc­tion stu­dio. Called Stu­dio K, the full-ser­vice pro­duc­tion set­up in­cludes a sound stage, ed­it suite, record­ing booth and green room — and it’s al­ready booked up. The in-house stu­dio is one of three new Klick busi­ness prac­tices, the oth­er two bring a new cor­po­rate brand­ing group, Klick Brands, and a so­cial me­dia in­flu­encer prac­tice called Klick In­flu­ence.
• Mon­i­ca Ocha­pa is in her third year pur­su­ing a doc­tor­ate of pub­lic health at the his­tor­i­cal­ly black uni­ver­si­ty Mor­gan State — and one of two first-ever re­cip­i­ents of a new Pfiz­er vac­cine fel­low­ship there. The two-year pro­gram aims to cre­ate a path for more mi­nor­i­ty prac­ti­tion­ers as lead­ers in vac­cine med­ical de­vel­op­ment and trans­late re­search and analy­sis in­to re­al-world health so­lu­tions.
• Pfiz­er and BioN­Tech are pro­mot­ing their Covid-19 vac­cine Comir­naty with a fo­cus on teens in a new na­tion­al TV ad; Idor­sia’s cam­paign for sleep med Qu­viviq in part­ner­ship with world-cham­pi­on ski­er Lind­sey Vonn now in­cludes a TV com­mer­cial; Phar­ma ad spend drops slight­ly year over year, amid dou­ble-dig­it de­clines in oth­er in­dus­tries; and more news in brief fea­ture in Mar­ket­ingRx roundup.

FDA+

• It’s been a lit­tle more than six months since FDA com­mis­sion­er Rob Califf won Sen­ate con­fir­ma­tion, and while he high­light­ed some of his key goals ear­ly on, he of­fered the agency’s lat­est ar­ray of top pri­or­i­ties in a JA­MA view­point. At the top of the list, even as gen­er­al fa­tigue has tak­en hold among the pub­lic’s per­cep­tion to­ward the pan­dem­ic, is Covid-19, and the on­go­ing opi­oid ad­dic­tion cri­sis, which took more than 100,000 lives in 2021.
• Al­though their num­bers have dwin­dled in re­cent years, FDA ad­vi­so­ry com­mit­tees have been in the spot­light, and some ex­perts are now seek­ing re­forms to make them more use­ful, in­clud­ing a trio of lead­ers from Har­vard. Writ­ing in the NE­JM, they call on the FDA to pro­vide more clar­i­ty and con­sis­ten­cy as to when it would con­vene an ad­comm.
• Of the near­ly 40 biosim­i­lars ap­proved to date by the FDA, on­ly three have won the cov­et­ed in­ter­change­abil­i­ty des­ig­na­tion so far, in part be­cause it car­ries ad­di­tion­al re­quire­ments such as switch­ing stud­ies. But the FDA is open­ing the door for in­ter­change­able biosim­i­lars to be ap­proved with­out con­duct­ing these switch­ing stud­ies first.
• In an­oth­er key Eu­ro­pean OK, Gilead se­cured the first ap­proval for lenaca­pavir to treat mul­tidrug re­sis­tant HIV. The drug, brand­ed as Sun­len­ca on the con­ti­nent, is de­signed to be ad­min­is­tered once every six months, giv­ing Gilead a long-act­ing op­tion to take on Vi­iV. A cap­sid in­hibitor, it’s al­so been billed as the next big HIV play giv­en its po­ten­tial to both treat and pre­vent in­fec­tion.
• One of the more an­tic­i­pat­ed H2 events will oc­cur al­most im­me­di­ate­ly af­ter La­bor Day — the un­usu­al sec­ond ad­comm for Amy­lyx Phar­ma­ceu­ti­cals’ ex­per­i­men­tal ALS drug. At least one an­a­lyst is try­ing to read the tea leaves, and he gives the drug a 50% chance at good news at the ad­comm.
• Foghorn Ther­a­peu­tics’ flag­ship pro­gram took an­oth­er hit as the FDA con­vert­ed its par­tial clin­i­cal hold on a blood can­cer drug to a full hold. The Phase I dose es­ca­la­tion study of FHD-286, an in­hibitor of BRG1/BRM, was halt­ed due to sus­pect­ed cas­es of fa­tal dif­fer­en­ti­a­tion syn­drome crop­ping up, and a new da­ta sub­mis­sion trig­gered the move.
• Up­dat­ing guid­ance from 2016 on how com­pa­nies can charge for in­ves­ti­ga­tion­al drugs un­der an IND, the FDA now tells spon­sors work­ing un­der ex­pand­ed ac­cess that they can spread out their man­u­fac­tur­ing, ad­min­is­tra­tive or mon­i­tor­ing costs from the first year over the ex­pect­ed du­ra­tion of the ex­pand­ed IND or pro­to­col, po­ten­tial­ly eas­ing the bur­den for small­er com­pa­nies.
• French biotech Ery­tech Phar­ma is knock­ing off its plan to get FDA ap­proval for its drug Gras­pa in hy­per­sen­si­tive acute lym­phoblas­tic leukemia, fol­low­ing feed­back from the reg­u­la­tors. De­spite what it sees as pos­i­tive Phase II da­ta, the chang­ing com­pet­i­tive land­scape and the FDA’s re­quest for more clin­i­cal da­ta made it too dif­fi­cult for Ery­tech.
• The FDA has placed a hold on the clin­i­cal tri­als for Swiss biotech Phar­varis’ oral drug PHA121, which aims at treat­ing hered­i­tary an­gioede­ma, a rare ge­net­ic con­di­tion. The reg­u­la­tors placed the hold af­ter re­view­ing the com­pa­ny’s non­clin­i­cal da­ta
• Ipsen’s palo­varotene, the oft-ma­ligned rare bone dis­ease pro­gram it ac­quired from Clemen­tia, is go­ing to get a clos­er look from reg­u­la­tors on Hal­loween. The FDA sched­uled an ad­vi­so­ry com­mittee to ex­am­ine the drug on Oct. 31, set­ting up a show­down for a drug that once failed a Phase III test. Its de­ci­sion date is slat­ed for Dec. 29.

MAN­U­FAC­TUR­ING

• Ther­mo Fish­er opened the doors to two sites with­in days as part of a $650 mil­lion in­vest­ment to build sites across the US, UK and Chi­na this year. The first was a $105 mil­lion, 400,000 square foot fa­cil­i­ty in Lebanon, TN, sev­er­al miles east of Nashville that will pro­duce cus­tomiz­able sin­gle-use bio­pro­cess­ing con­tain­ers and flu­id trans­fer as­sem­bly sys­tems for bi­o­log­ics, vac­cine and cell and gene ther­a­py man­u­fac­tur­ing. The sec­ond, clos­er to its Mass­a­chu­setts home, is a new vi­ral vec­tor man­u­fac­tur­ing plant in Plainville, MA that cost about $180 mil­lion.
• Ear­li­er this year, FDA in­ves­ti­ga­tors found is­sues at Te­va Phar­ma­ceu­ti­cals’ plant in Irvine, CA, forc­ing the com­pa­ny to pad­lock the doors. It ul­ti­mate­ly de­cid­ed to pull the plug on re­open­ing it. In a WARN no­tice, Te­va re­vealed that it is ax­ing 305 jobs at the site, which has had a mul­ti­tude of is­sues, and per­ma­nent­ly clos­ing it.
• The FDA’s post­mar­ket reg­u­la­tions re­quire that drug man­u­fac­tur­ers no­ti­fy the agency about any sig­nif­i­cant prod­uct qual­i­ty de­fects in mar­ket­ed prod­ucts with­in three work­ing days. But a new re­port from the agency found that of the 1,143 man­u­fac­tur­ing sites that were el­i­gi­ble to sub­mit a FAR from 2018 to 2021, al­most half (49%) of the sites did not sub­mit a re­port.
• Amid a scram­ble for more mon­key­pox vac­cines, Grand Rapids, Michi­gan-based CD­MO Grand Riv­er Asep­tic Man­u­fac­tur­ing land­ed a deal with Bavar­i­an Nordic, to fill and fin­ish its Jyn­neos vac­cine. Its CEO spoke with End­points Man­u­fac­tur­ing ed­i­tor Tyler Patchen to ex­plain how a rel­a­tive­ly new play­er in the CD­MO world land­ed the con­tract.
• As Re­silience keeps up the mo­men­tum post-Se­ries D, the bio­man­u­fac­tur­er reeled in an­oth­er part­ner­ship, this time in the Gold­en State. It was named by the Cal­i­for­nia In­sti­tute for Re­gen­er­a­tive Med­i­cine as one of its ini­tial in­dus­try re­source part­ners to sup­port trans­la­tion­al and clin­i­cal phase grant ap­pli­ca­tions.
• In­di­an gener­ics man­u­fac­tur­er Mor­ton Grove Phar­ma­ceu­ti­cals is shut­ter­ing its Illi­nois site due to man­u­fac­tur­ing is­sues that have built up over sev­er­al years. The De­part­ment of Jus­tice ac­cused it of sell­ing adul­ter­at­ed drugs in a law­suit al­leg­ing the com­pa­ny’s syrups and nasal sprays made at the fa­cil­i­ty were not made in com­pli­ance with cGMP stan­dards.
• Build­ing off a pre­vi­ous re­vi­sion in 2007, the Eu­ro­pean Com­mis­sion re­leased its lat­est up­date to its guide­line on man­u­fac­tur­ing ster­ile drugs. The doc­u­ment pro­vides tech­ni­cal guid­ance around the in­tri­ca­cies of each step, and it fol­lows the rec­om­men­da­tion of the GMP/GDP In­spec­tors Work­ing Group and the PIC/S com­mit­tee to re­flect changes in both reg­u­la­to­ry and man­u­fac­tur­ing.
• Chi­nese CD­MO Por­ton Ad­vanced So­lu­tions man­aged to gar­ner more than $80 mil­lion to con­tin­ue a busi­ness ex­pan­sion; Lon­za ex­pand­ed the range of en­gi­neer­ing ser­vices at its Mon­teg­gio fa­cil­i­ty; ABEC, a provider of en­gi­neered so­lu­tions and ser­vices for biotech man­u­fac­tur­ing, will in­vest $11 mil­lion to ex­pand its sin­gle-use dis­pos­able con­tain­er man­u­fac­tur­ing ca­pac­i­ty; check out the rest in our Man­u­fac­tur­ing roundup.

DON’T MISS

• Quick­ly con­firm­ing re­ports, the US’ Pub­lic Com­pa­ny Ac­count­ing Over­sight Board re­vealed it’s struck an agree­ment with Chi­nese reg­u­la­to­ry au­thor­i­ties that would al­low the in­spec­tion of au­dit re­ports for US-list­ed Chi­nese com­pa­nies, which would save them from delist­ing. Now, ac­cord­ing to SEC chair Gary Gensler, “The proof will be in the pud­ding.”
• A few months af­ter a lead­er­ship shake­up, the White House’s Of­fice of Sci­ence and Tech­nol­o­gy Pol­i­cy is launch­ing one of its most am­bi­tious pol­i­cy changes to date by mak­ing all tax­pay­er-fund­ed re­search more ac­ces­si­ble to the pub­lic. Agen­cies will have un­til the end of 2025 to make pub­li­ca­tions and re­search paid for with tax­pay­er dol­lars pub­licly ac­ces­si­ble, for free, with­out any re­stric­tion.

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.