May 21, 2022

Amber Tong Senior Editor 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 . It’s al­ways a bit­ter­sweet mo­ment say­ing good­bye, but as Josh Sul­li­van goes off to new ad­ven­tures we are grate­ful for the way he’s built up the End­points Man­u­fac­tur­ing sec­tion — which the rest of the team will now car­ry for­ward. If you’re not al­ready, this may be a good time to sign up for your week­ly dose of drug man­u­fac­tur­ing news. Thank you for read­ing and wish you a rest­ful week­end. Amid mon­key­pox fears, biotechs spring to ac­tion With a rapid rise of mon­key­pox cas­es caus­ing con­cerns around the world, vac­cine pro­cure­ment is weigh­ing on the mind of gov­ern­ment of­fi­cials. Bavar­i­an Nordic land­ed con­tracts with both the US’ BAR­DA and an un­named Eu­ro­pean coun­try to sup­ply its small­pox vac­cine, Jyn­neos. While the BAR­DA’s $119 mil­lion op­tion has a longer term time­frame, the Eu­rope pur­chase high­lights an “ emer­gency sit­u­a­tion .” At the same time, Emer­gent BioSo­lu­tions ac­quired Tem­bexa, a small­pox treat­ment, from Chimerix for $225 mil­lion in cash. Ex­perts reck­on that small­pox vac­cines should of­fer some pro­tec­tion against mon­key­pox, and pre­vi­ous vac­ci­na­tions, cou­pled with the right pub­lic health re­sponse, should stem the cur­rent out­break. Craft­ing the right pro­teins For decades, pro­tein de­sign was a bi­o­log­i­cal bor­der­land. But in the last few years, a se­ries of break­throughs has sud­den­ly turned an aca­d­e­m­ic cu­rios­i­ty in­to a vi­able way of de­vel­op­ing new drugs, in­clud­ing ones that are more ac­cu­rate, more com­plex, or can be de­vel­oped faster than med­i­cines made through oth­er meth­ods. In his fi­nal End­points In Fo­cus, Ja­son Mast takes us in­side the lab of David Bak­er — once among the field’s long rangers — at the Uni­ver­si­ty of Wash­ing­ton’s In­sti­tute for Pro­tein De­sign to take a peek at how de­sign-based ap­proach­es are trans­form­ing drug de­vel­op­ment. Cuts, cuts every­where Mauled by the bear mar­ket, a mul­ti­tude of biotechs con­tin­ue to feel the heat — and the list might sur­prise you. Agios is shak­ing things up as 50 ex­plorato­ry re­search em­ploy­ees will be let go and CSO Bruce Car will de­part the com­pa­ny at the end of Ju­ly. With piv­otal da­ta still years away, Schol­ar Rock, the dark­horse SMA play­er, plans to lay off 39 peo­ple while CMO Yung Chyung steps down. French biotech Nanobi­otix, which IPO’d dur­ing the pan­dem­ic boom, is chop­ping pre­clin­i­cal re­search , amend­ing some on­go­ing stud­ies and mov­ing some em­ploy­ees ful­ly re­mote to stay afloat. San Fran­cis­co’s Ap­plied Mol­e­c­u­lar Trans­port will cut its work­force by 40% on top rolling back pro­grams, and among those leav­ing is its CSO. Small cap biotech Aque­s­tive, mean­while, didn’t pro­vide a rea­son for why long­time CEO Kei­th Kendall is hand­ing the ba­ton to COO Daniel Bar­ber. Janet Wood­cock has a new role Long­time CDER di­rec­tor Janet Wood­cock, who’s now serv­ing as FDA com­mis­sion­er Rob Califf’s prin­ci­pal deputy, would take on more of a role as “a strate­gic thought part­ner, with de­ci­sion-mak­ing au­thor­i­ties” at sev­er­al dif­fer­ent of­fices with­in the agency, ac­cord­ing to a new memo. In­sid­ers said the an­nounce­ment ef­fec­tive­ly means she won’t be re­turn­ing to or step­ping on the toes of any­one serv­ing in the var­i­ous roles she held pre­vi­ous­ly. Mod­er­na’s CFO trou­ble Days af­ter Mod­er­na sent new CFO Jorge Gomez pack­ing — af­ter just one day on the job — over an in­ves­ti­ga­tion launched by his for­mer em­ploy­er, the mR­NA biotech said it was blind­sided by the probe. Al­though den­tal equip­ment com­pa­ny Dentsply be­gan in­ves­ti­gat­ing Gomez in ear­ly March, Mod­er­na claimed it was un­aware un­til the pub­lic dis­clo­sure on May 10. It de­cid­ed to part ways with Gomez that same day. And Flag­ship’s Noubar Afeyan, who’s al­so chair of the Mod­er­na board, de­fend­ed the vet­ting and hir­ing process. To­ward more di­verse clin­i­cal tri­als While calls to di­ver­si­fy clin­i­cal tri­als have grown loud­er in re­cent years — gain­ing sup­port from fed­er­al agen­cies such as the FDA and NIH — progress has large­ly stalled , ac­cord­ing to a new re­port that sug­gests swaths of pa­tients in mi­nor­i­ty groups con­tin­ue to be left out. Pfiz­er is delv­ing in­to the is­sue through a new part­ner­ship with Head­lands Re­search that aims to ini­ti­ate re­search sites in ar­eas with di­verse pop­u­la­tions, while J&J launched its “ De­pres­sion Looks Like Me ” cam­paign in hopes of nor­mal­iz­ing the dis­cus­sion around de­pres­sion — es­pe­cial­ly for peo­ple in the LGBTQ+ com­mu­ni­ty. DEALS Two months af­ter Atara Bio­ther­a­peu­tics hit the hold but­ton on its lead CAR-T 2.0 ther­a­py fol­low­ing a pa­tient death, its Big Phar­ma part­ners at Bay­er are bow­ing out of a $670 mil­lion glob­al al­liance. And the move is forc­ing a re­vamp of Atara’s pipeline plans, even as re­search ex­ecs vow to con­tin­ue work on the two drugs al­lied with Bay­er 18 months ago. Ab­b­Vie is join­ing its fel­low Big Phar­ma com­peti­tors and jump­ing in­to the Treg space. In a new col­lab­o­ra­tion, it will be team­ing up with pri­vate biotech Cu­gene to ad­vance a sin­gle Treg pro­gram tar­get­ing IL-2. Ab­b­Vie is shelling out $48.5 mil­lion up­front to col­lab­o­rate and will pay an undis­closed sum of mile­stones and roy­al­ties. As Big Phar­ma slow­ly but sure­ly warms up to the po­ten­tial of dig­i­tal ther­a­peu­tics, Pfiz­er took an­oth­er step in its part­ner­ship with Ice­landic biotech Side­kick Health to launch a prod­uct specif­i­cal­ly geared for atopic der­mati­tis. The two have co-launched oth­er prod­ucts be­fore and will work to­geth­er to fig­ure out how best to of­fer pa­tients “per­son­al­ized sup­port around care and man­age­ment of symp­toms.” All Blue’s bid to take over a bad­ly bat­tered Zymeworks — bol­stered by its ap­point­ment of cel­e­brat­ed on­col­o­gist, Pulitzer prize-win­ning writer and biotech ex­ec Sid­dhartha Mukher­jee to the pro­posed board — of­fi­cial­ly turned hos­tile as Zymeworks’ board re­ject­ed the un­so­licit­ed, $733 mil­lion of­fer. Six years af­ter Ot­su­ka first signed a li­cens­ing and co-de­vel­op­ment agree­ment for vadadu­s­tat, Ake­bia’s drug can­di­date for re­nal ane­mia re­lat­ed to chron­ic kid­ney dis­ease, the whole deal is off. Ot­su­ka had orig­i­nal­ly nabbed co-com­mer­cial­iza­tion rights in the US in ad­di­tion to oth­er large mar­kets but de­cid­ed to pull out in the wake of a com­plete re­sponse let­ter. Drawn in by a cell pouch de­vice, Evotec is tak­ing a mi­nor­i­ty stake as it strikes a part­ner­ship with Ser­no­va in hopes of cre­at­ing a func­tion­al cure for di­a­betes . While Ser­no­va’s cur­rent ther­a­py makes use of donor cells, it can now use Evotec’s stem cell-de­rived, in­sulin-pro­duc­ing be­ta cells to make a cell re­place­ment ther­a­py. Sh­iono­gi, an ac­tive play­er in the arid field of an­timi­cro­bials, has now set its eye on the ad­ja­cent sphere of an­ti­fun­gals. The Japan-based phar­ma is pay­ing $100 mil­lion up­front to part­ner with F2G to push olo­rofim through clin­i­cal tri­als and reg­u­la­to­ry ac­tion in Eu­rope and Asia. The drug is be­ing test­ed to treat a rare mold in­fec­tion that can im­pact im­muno-sup­p­resed peo­ple. Two months ago, Or­p­hazyme en­list­ed the help of the Dan­ish courts to help over­see a re­struc­tur­ing in the wake of tri­al fail­ures, C-suite shake­ups and reg­u­la­to­ry re­jec­tions. The res­o­lu­tion of the court-run ef­forts are com­ing in­to view now as Kem­Pharm agrees to ac­quire Or­p­hazyme and its once-hyped “pipeline-in-a-prod­uct” for $12.8 mil­lion in cash, while al­so as­sum­ing some li­a­bil­i­ties. CELL & GENE TX Even Pe­ter Marks, the di­rec­tor of the FDA’s CBER, is wor­ried about the com­mer­cial vi­a­bil­i­ty of cell and gene ther­a­pies. In a rare, di­rect ad­dress to the “800 pound go­ril­la in the room” that is re­im­burse­ment, he talked at a con­fer­ence about spon­sors are start­ing to see in­di­ca­tions where there may on­ly be sev­er­al dozen pa­tients world­wide as non­vi­able. Pfiz­er, Sarep­ta, Sol­id Bio­sciences and Genethon be­lieve they have found a rea­son for the safe­ty is­sues that have plagued gene ther­a­pies for Duchenne mus­cu­lar dy­s­tro­phy. The four com­pa­nies sug­gest­ed there may be a “ class ef­fect, ” and that more re­search is need­ed to find out the root cause. Ahead of the FDA, the EMA is giv­ing its thumbs up for PTC Ther­a­peu­tics’ gene ther­a­py for aro­mat­ic amino acid de­car­boxy­lase de­fi­cien­cy, an ul­tra-rare ge­net­ic dis­ease that leaves pa­tients un­able to pro­duce cer­tain hor­mones in the brain, usu­al­ly lead­ing to de­vel­op­men­tal de­lays, weak mus­cle tone and in­abil­i­ty to con­trol the move­ment of the limbs. SQZ Biotech show­cased new re­search sug­gest­ing that its un­ortho­dox, pro­to­type point-of-care cell ther­a­py man­u­fac­tur­ing tech could lead to com­pa­ra­ble or even bet­ter per­for­mance than a con­ven­tion­al clean-room-based man­u­fac­tur­ing process. The first gen­er­a­tion is ex­pect­ed to en­ter the clin­ic in 2023. Dark Horse Con­sult­ing walked away from the FDA’s two-day ad­comm on gene ther­a­py last Sep­tem­ber feel­ing it was a “missed op­por­tu­ni­ty.” Now, the firm is step­ping to the plate with its own rec­om­men­da­tions for AAVs — sub­mit­ting those ideas in the form of a draft guid­ance . The 10-page guid­ance hones in on the role that emp­ty AAV vec­tor cap­sids can play in prod­uct im­pu­ri­ty, and looks to es­tab­lish re­lease cri­te­ri­on for full to emp­ty cap­sid ra­tios. Far­go-based DNA and mR­NA pro­duc­er Alde­vron has li­censed the rights to man­u­fac­ture and com­mer­cial­ize the Eu­re­ca-V Nu­cle­ase , a wild-type MAD7 CRISPR Type-V nu­cle­ase, from In­scrip­ta. The part­ners will aim to of­fer this nu­cle­ase as a stan­dard re­search-grade and GMP cat­a­log item. CHI­NA Mer­ck qui­et­ly inked a deal to pick up a cer­tain “ large mol­e­cule ” can­cer drug can­di­date from Chi­na’s Kelun Phar­ma­ceu­ti­cal. Cov­er­ing all ter­ri­to­ries out­side main­land Chi­na, Tai­wan, Hong Kong and Macau, the deal starts at $47 mil­lion up­front but mile­stones can add up to $1.3 bil­lion. Green Val­ley Phar­ma, the Shang­hai drug­mak­er that scored a stun­ning ap­proval to mar­ket an Alzheimer’s drug in Chi­na two years ago, is shut­ter­ing a Phase III tri­al that would’ve paved its way to the FDA. A lack of fund­ing had forced its hand, ac­cord­ing to the biotech, which said a re­cent Covid-19 out­break put it in a cash crunch and it wasn’t able to raise fund­ing in time. In an un­usu­al arrange­ment, Chi­nese on­col­o­gy gi­ant Jiang­su Hen­grui launched Luzsana Biotech­nol­o­gy to serve as an arm to de­vel­op and de­liv­er its ther­a­peu­tics out­side of the Chi­nese mar­ket . The biotech al­ready has lo­ca­tions in Tokyo, Basel, Switzer­land and Prince­ton, New Jer­sey, with more than 120 em­ploy­ees. CEO Scott Filosi was a top com­mer­cial ex­ec at Mer­ck KGaA. Suzhou, Chi­na-based Star­na Ther­a­peu­tics emerged with about $24 mil­lion to take on two dif­fi­cult ar­eas in biotech R&D: RSV and id­io­path­ic pul­monary fi­bro­sis. The Se­ries A will sup­port pre­clin­i­cal work for its mR­NA vac­cine can­di­date in RSV, which is based on tech from its co-founder, a PI at Peking Uni­ver­si­ty. R&D As the US tran­si­tions away from Covid-19 vac­cine sci­ence in­to more ther­a­peu­tic work, the NIH award­ed $577 mil­lion to nine aca­d­e­m­ic groups in an ef­fort to dri­ve an­tivi­ral de­vel­op­ment for Covid and a slew of oth­er virus­es. The grants will cre­ate nine an­tivi­ral drug dis­cov­ery (AViDD) cen­ters, whose main goals are to do the pre­clin­i­cal dis­cov­ery work for drugs against virus­es with pan­dem­ic po­ten­tial. Two years af­ter a bit­ter set­back at the FDA, with its cash re­serves ebbing away, Tri­ci­da is ex­e­cut­ing a high-stakes gam­ble on win­ning a swift ap­proval at the FDA by putting an “ ad­min­is­tra­tive stop ” to a piv­otal tri­al. A win would put them back in play with a new drug to com­mer­cial­ize. A loss, as one Wall Street an­a­lyst re­cent­ly as­sessed, would take the stock to ze­ro. Ather­sys’ stem cell ther­a­py has failed yet again. In a 206-per­son tri­al con­duct­ed in Japan, Ather­sys’ ex­per­i­men­tal treat­ment for stroke failed its pri­ma­ry end­point of “ex­cel­lent out­come,” a com­bined mea­sure of three stroke re­cov­ery scores. Then, as it’s done in the past, the com­pa­ny piv­ot­ed to a post hoc analy­sis , fo­cus­ing on a younger sub­set of pa­tients. A Phase IIb flop is cast­ing a shad­ow on Enan­ta Phar­ma­ceu­ti­cals’ ef­forts to take on RSV, a no­to­ri­ous­ly tough field strewn with Big Phar­ma ri­vals. Al­though its an­tivi­ral failed to re­duce symp­toms for oth­er­wise healthy adults, the biotech is hold­ing out hope for a nar­row­er group of pa­tients who would need a treat­ment the most. Ab­b­Vie is ap­proach­ing the FDA with a new ther­a­py to po­ten­tial­ly treat Parkin­son’s dis­ease, us­ing pro­drugs of two med­ica­tions com­mon­ly used for the con­di­tion. Its NDA was for AB­BV-951, a so­lu­tion of lev­odopa and car­bidopa pro­drugs ad­min­is­tered through a 24-hour in­fu­sion. It’s de­signed for ad­vanced Parkin­son’s pa­tients who don’t re­spond well to oral ther­a­py. Rock­et Phar­ma­ceu­ti­cals’ lat­est cut of gene ther­a­py da­ta has at least one an­a­lyst won­der­ing about the cu­ra­tive po­ten­tial. Re­port­ing topline re­sults from a Phase II piv­otal tri­al in­volv­ing pa­tients with se­vere leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-1), Rock­et said among nine par­tic­i­pants, the over­all sur­vival at one year is 100% — and there were al­so im­prove­ments in symp­toms, paving the way for a reg­u­la­to­ry fil­ing ear­ly next year. When Ze­ga­logue, al­so known as dasiglucagon, got ap­proved by the FDA ear­ly last year for se­vere hy­po­glycemia in di­a­betes pa­tients, Zealand Phar­ma said that wasn’t the end of its vi­sion for the drug. It’s now de­liv­ered on a Phase III tri­al for a new in­di­ca­tion — a ge­net­ic con­di­tion called con­gen­i­tal hy­per­in­sulin­ism — with pos­i­tive re­sults that could pave the way for a fil­ing. Mod­er­na and non­prof­it re­search group IAVI (In­ter­na­tion­al AIDS Vac­cine Ini­tia­tive) are jump­ing in­to the next stage of their col­lab­o­ra­tion around an mR­NA an­ti-HIV vac­cine. IAVI will spon­sor a new Phase I tri­al dubbed IAVI G003, which will take place in Rwan­da and South Africa to see if ear­li­er re­sults in the US will trans­late in­to a dif­fer­ent pop­u­la­tion. Months af­ter tout­ing some fuzzy topline read­outs, As­traZeneca and its biotech part­ner Avil­lion de­tailed the da­ta they’re tak­ing to reg­u­la­tors for their asth­ma drug, PT027. The in­haled, fixed-dose res­cue med­ica­tion, which com­bines two com­mon­ly used drugs, cut the risk of se­vere ex­ac­er­ba­tion by 27% (p<0.001) com­pared to al­buterol res­cue alone. PEO­PLE While Seagen is still in­ves­ti­gat­ing claims of do­mes­tic vi­o­lence against Clay Sie­gall, the com­pa­ny and its long­time CEO agreed that it’s bet­ter he re­sign now . Since the biotech re­vealed that Sie­gall was on leave, more de­tails have emerged, in­clud­ing the fact that he was ar­rest­ed and placed un­der a tem­po­rary re­strain­ing or­der from his wife. He de­nied the al­le­ga­tions. Mod­er­na’s rapid fire de­vel­op­ment of its huge­ly suc­cess­ful mR­NA vac­cine for Covid saved lives, changed the vac­cine in­dus­try for­ev­er and made CEO Stéphane Ban­cel a bil­lion­aire. But per­haps the sweet­est re­ward came this week, when Ban­cel was named a Cheva­lier — ba­si­cal­ly knight­ed — by the pres­i­dent of France. Ready to trans­form it­self in­to a se­ri­ous mid- to late-stage can­cer play­er that could take on ri­vals as big as As­traZeneca, Pfiz­er-part­nered biotech Zen­tal­is ap­point­ed Eli Lil­ly vet Kim Black­well to suc­ceed An­tho­ny Sun as CEO. To fu­el the de­vel­op­ment of its two lead can­di­dates, Zen­tal­is raised an ad­di­tion­al $200 mil­lion from an of­fer­ing. Richard “Ric” Kayne, the bil­lion­aire who mount­ed an ac­tivist at­tack at Alzheimer’s-fo­cused Athi­ra af­ter it oust­ed founder Leen Kawas, is sus­pend­ing his cam­paign to in­sert him­self to the com­pa­ny’s board of di­rec­tors. Though Kayne will no longer be em­pha­siz­ing the need for change, Athi­ra share­hold­ers are still al­lowed to vote for his slate of nom­i­na­tions. STAR­TUPS Flag­ship craft­ed a new start­up out of pieces from a pair of fledg­lings in the VC’s nest. And as Inzen and Cyg­nal merge in­to Sonata Ther­a­peu­tics, Roche vet and Cyg­nal CEO Pearl Huang will de­part in pur­suit of new op­por­tu­ni­ties. Where­as Inzen was fo­cused on the cel­lu­lar mi­croen­vi­ron­ment, Cyg­nal ze­roed in on the pe­riph­er­al ner­vous sys­tem. We’ve seen plen­ty of new com­pa­nies fo­cus­ing on CAR-Ts — or CAR-NKs and CAR-Ms. But In­cep­tor Bio wants to at­tempt all three at once . With $37 mil­lion, it hopes to flesh out its on­col­o­gy pro­grams while al­so build­ing out its own man­u­fac­tur­ing fa­cil­i­ty in Gainesville, FL. While ac­knowl­edg­ing the chal­lenges, the biotech hopes the ap­proach yields more shots on goal. New York-based Cam­bri­an Bio­phar­ma spun out a new start­up with the idea of ex­tend­ing women’s “healthspans.” Fo­cused on ovaries , Ovi­va is armed with $11.5 mil­lion from Cam­bri­an and the sci­en­tif­ic knowhow of two re­searchers at Mass Gen­er­al. FI­NANC­ING Pri­vate eq­ui­ty is mak­ing more big in­roads in­to the biotech scene. And An­toine Pa­piernik and the crew at Sofinno­va Part­ners are get­ting a sub­stan­tial boost in the mon­ey they have avail­able to in­vest. The $32 bil­lion pri­vate eq­ui­ty play­er Apol­lo Glob­al Man­age­ment has stepped up to take a mi­nor­i­ty stake in the life sci play­er, of­fer­ing €1 bil­lion of man­aged cap­i­tal to its funds. Jeff Aronin’s cell and gene ther­a­py biotech Cas­tle Creek Bio­sciences has raised $112 mil­lion in eq­ui­ty, End­points News learned. Af­ter pulling IPO am­bi­tions last De­cem­ber, the com­pa­ny se­cured mon­ey from 54 pri­vate in­vestors to fund late-stage work on its ex vi­vo gene ther­a­py for chron­ic wounds due to re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa. Just a few short weeks af­ter Swan­Bio laid off a quar­ter of its 60-per­son work­force, it has re­turned with $56 mil­lion in new fi­nanc­ing . While scut­tled fundrais­ing plans ap­par­ent­ly trig­gered the lay­offs, at least two found­ing in­vestors re­main stead­fast and con­fi­dent in its plan to head to­ward the clin­ic. Bask­ing in the spot­light of a $1 bil­lion deal with J&J, Remix Ther­a­peu­tics is back with $70 mil­lion in VC mon­ey as it con­tin­ues to try and drug RNA pro­cess­ing. While the plat­form could be used for drug dis­cov­ery across fields, Remix has pri­mar­i­ly been fo­cus­ing on “high val­ue tar­gets in on­col­o­gy and neu­ro­science right now.” Shankar Ra­maswamy doesn’t in­tend to re­peat the Roivant play­book that his broth­er, Vivek, start­ed eight years ago. In­stead, he wants to con­sol­i­date the gene ther­a­py process and be the go-to shop in a field that has rid­den a rocky road for many years. With that vi­sion for a “con­sol­i­dat­ed en­gine,” he and his 160-em­ploy­ee biotech have reeled in a $270 mil­lion Se­ries C. Three years af­ter Clover Health tapped ex-Pfiz­er ex­ec Cheng Zhang to lever­age the health in­sur­er’s da­ta in un­earthing bio­mark­ers and drug tar­gets, Clover Ther­a­peu­tics got a facelift . Now named Char­ac­ter Bio­sciences, it raised $18 mil­lion to car­ry on with the same goal, which is to dis­cov­er po­ten­tial ther­a­pies for dis­eases of ag­ing. Work­ing out of North Car­oli­na’s re­search tri­an­gle, pre­ci­sion an­tibac­te­r­i­al drug­mak­er Lo­cus Bio­sciences closed a $35 mil­lion Se­ries B fea­tur­ing some no­table VC names. The BAR­DA-backed biotech’s lead can­di­date is a phage ther­a­py tar­get­ing E. coli caus­ing uri­nary tract in­fec­tions. An Iowa seed sup­pli­er led a $29.5 mil­lion in­fu­sion in­to tiny, Rochester, MN-based biotech Vyr­i­ad, which is de­vel­op­ing an on­colyt­ic virus de­rived from the Vesic­u­lar stom­ati­tis virus. The round is an ex­ten­sion to a Se­ries B led by Re­gen­eron, which is col­lab­o­rat­ing on a com­bo with the PD-1 drug Lib­tayo. LAW Cap­ping a years-long trade­mark case, Roche’s Spark Ther­a­peu­tics and blue­bird bio filed a joint mo­tion to dis­miss a trade­mark in­fringe­ment suit over blue­bird’s “Be the Spark” sick­le cell cam­paign — which Roche claimed in­fringed on its Spark trade­marks. But the ac­tu­al set­tle­ment agree­ment re­mains con­fi­den­tial. Two years ago, No­var­tis got slammed with hun­dreds of mil­lions in fines from the US De­part­ment of Jus­tice for en­gag­ing in bribery and mis­con­duct in or­der to boost sales in dif­fer­ent coun­tries. Now, one of those coun­tries, Greece, is on the of­fen­sive — is­su­ing a state­ment that the gov­ern­ment is com­mit­ted to claim­ing com­pen­sa­tion from the Swiss mega-phar­ma. Sanofi has faced a slew of al­le­ga­tions over the last sev­er­al years that its epilep­sy drug De­pakine caus­es birth de­fects when tak­en dur­ing preg­nan­cy. In a set­back, a court in Nan­terre — a city sev­er­al miles out­side of Paris — ruled that Sanofi must pay up to €450,000 in dam­ages to a fam­i­ly whose child suf­fered a form of autism af­ter ex­po­sure to De­pakine. Hen­ri­et­ta Lacks’ fam­i­ly’s fight for jus­tice faces an un­cer­tain fate as a Bal­ti­more fed­er­al judge con­sid­ers toss­ing the case. Judge Deb­o­rah Board­man is weigh­ing Ther­mo Fish­er’s mo­tion to dis­miss the claims against it on the grounds that the statute of lim­i­ta­tions has passed, and the con­tin­u­ing harm doc­trine does not ap­ply. Pune, In­dia-based gener­ic drug man­u­fac­tur­er Em­cure Phar­ma­ceu­ti­cals is press­ing a court in Wash­ing­ton to toss out a suit brought on by HDT Bio over a vac­cine to pre­vent Covid-19. Call­ing its for­mer part­ner’s de­mand for $950 mil­lion in dam­ages “ bloat­ed ,” Em­cure slams the suit as “a made-for-head­lines hit piece.” CORO­N­AVIRUS As­traZeneca is dou­bling down on de­vel­op­ing an­ti­bod­ies for Covid-19. Build­ing on the long-act­ing an­ti­body com­bo now known as Evusheld, As­traZeneca this time is bet­ting on new­ly-launched RQ Biotech­nol­o­gy, a UK-based out­fit with roots at the Uni­ver­si­ty of Ox­ford and med­ical re­search char­i­ty LifeArc. The $157 mil­lion deal brings in a suite of ear­ly-stage an­ti­bod­ies tar­get­ing SARS-CoV-2. Amid ris­ing con­cerns of po­ten­tial de­vel­op­ment of re­sis­tance to Pfiz­er’s Covid-19 pill Paxlovid, the com­pa­ny re­port­ed­ly de­clined to sup­ply re­searchers with the an­tivi­ral for use in com­bi­na­tion stud­ies. For its part, Pfiz­er said it’s eval­u­at­ing a “mul­ti-pronged strat­e­gy” that in­cludes think­ing ahead to sec­ond-gen­er­a­tion an­tivi­rals and com­bi­na­tion ther­a­pies, but it hasn’t be­gun any tri­als. The lat­est wave of the pan­dem­ic — marked by Omi­cron and its sub-vari­ants — has seen high­er hos­pi­tal­iza­tion rates for young chil­dren, health agen­cies have ob­served. That’s part of the rea­son why the FDA is au­tho­riz­ing a sin­gle boost­er shot of Pfiz­er and BioN­Tech’s mR­NA vac­cine for kids be­tween 5 and 11 years old. The FDA re­ject­ed Uni­ver­si­ty of Min­neso­ta ID doc David Boul­ware’s EUA to use flu­vox­am­ine, a gener­ic SS­RI al­ready ap­proved to treat OCD, as an out­pa­tient treat­ment for those test­ing pos­i­tive for Covid-19. Ac­cord­ing to reg­u­la­tors, sub­mit­ted da­ta on “clin­i­cal­ly mean­ing­ful out­comes” were “not per­sua­sive.” PHAR­MA With atopic der­mati­tis ri­vals breath­ing down Dupix­ent’s neck, Sanofi and Re­gen­eron se­cured a first win in new ter­ri­to­ry in what Sanofi’s head of im­munol­o­gy and in­flam­ma­tion Naimish Pa­tel called the fastest ap­proval he’s ever seen. The FDA ap­proved the drug to treat pa­tients 12 years and old­er with eosinophilic esophagi­tis (EoE), an in­flam­ma­to­ry con­di­tion that caus­es swelling and scar­ring of the esoph­a­gus, which Pa­tel called the first in a “sec­ond wave” of in­di­ca­tions. Ver­tex Phar­ma­ceu­ti­cals has been one of the buzzi­er names in the bustling Boston biotech scene, but now the com­pa­ny is look­ing to vault to num­ber one sta­tus — at least in terms of phys­i­cal foot­print. It plans to build a $595 mil­lion cen­ter in the Sea­port area to ac­com­mo­date grow­ing R&D as well as man­u­fac­tur­ing needs, es­pe­cial­ly in its cell and gene ther­a­pies pro­gram. The Eu­ro­pean Med­i­cines Agency is rec­om­mend­ing the sus­pen­sion of about 100 drugs af­ter find­ing da­ta in­tegri­ty is­sues at an In­dia-based CRO that ran tests for the gener­ic drugs. The CRO in ques­tion, Syn­chron Re­search Ser­vices, isn’t the first to come un­der the mi­cro­scope for da­ta fail­ings. As with those cas­es, reg­u­la­tors are re­quir­ing the drug spon­sors to re­peat their bioe­quiv­a­lence/bioavail­abil­i­ty stud­ies at an ac­cept­able al­ter­nate study site. In­dus­try lob­by­ing group PhRMA lodged a win against com­mer­cial in­sur­ers tak­ing ad­van­tage of fi­nan­cial as­sis­tance pro­grams de­signed to shoul­der the high costs of drugs. A new court rul­ing sided with PhRMA , which sued the fed­er­al gov­ern­ment over a 2020 rule that would’ve re­quired drug man­u­fac­tur­ers to en­sure their as­sis­tance went to pa­tients. A team of re­searchers dug through all the patent and reg­u­la­to­ry ex­clu­siv­i­ties grant­ed to in­halers ap­proved by the FDA be­tween 1986 and 2020 and found that they of­ten re­ward­ed in­cre­men­tal ad­just­ments to old­er prod­ucts, es­pe­cial­ly on the de­liv­ery de­vice. The new pa­per, pub­lished in Health Af­fairs, ar­gues that this sys­tem en­abled com­pa­nies to skirt around cheap­er com­pe­ti­tion. You prob­a­bly al­ready call it this way al­ready, but Glax­o­SmithK­line fi­nal­ly of­fi­cial­ly changed its name to GSK. Fold­ing the his­to­ry of Glaxo Well­come and SmithK­line Beecham in­to three sim­ple let­ters, the name change comes weeks ahead of GSK’s an­tic­i­pat­ed con­sumer health­care spin­out. The Sen­ate vot­ed along par­ty lines, 51-50, with Vice Pres­i­dent Ka­mala Har­ris cast­ing the tie-break­er, to make Pres­i­dent Biden’s ap­pointee Al­varo Bedoya the de­cid­ing vote on a split 2-2 Fed­er­al Trade Com­mis­sion. His ap­point­ment could not on­ly spell more trou­ble for bio­phar­ma M&A ac­tiv­i­ty, but it may al­so mean that FTC Chair Lina Khan has what she needs to move for­ward on a study around the phar­ma mid­dle­men known as phar­ma­cy ben­e­fit man­agers. And a bi­par­ti­san trio of sen­a­tors didn’t waste any time call­ing on Khan to move for­ward with the in­ves­ti­ga­tion. The so­cial part of Roche’s en­vi­ron­men­tal, so­cial and gov­er­nance (ESG) strat­e­gy is cen­tered on ac­cess to its drugs and di­ag­nos­tics. The Roche in­au­gur­al Ac­cess to Health­care in­vestor call fol­lows a gen­er­al phar­ma in­dus­try trend in high­light­ing ESG, and dur­ing the call ex­ecs out­lined their tar­gets while al­so point­ing out what they have ac­com­plished. Stay­ing in the back­seat to Mer­ck’s Keytru­da, Bris­tol My­ers Squibb re­port­ed an­oth­er piv­otal set­back try­ing to move in­to a big­ger po­si­tion in front­line ther­a­py. Its Op­di­vo/Yer­voy com­bo flopped in a Phase III study of first-line ther­a­py for pre­vi­ous­ly un­treat­ed pa­tients with the most com­mon form of blad­der can­cer, fail­ing to hit over­all sur­vival. It looks like War­ren Buf­fett is re­think­ing his health­care in­vest­ments. Berk­shire Hath­away backed out of two ma­jor hold­ings in the phar­ma in­dus­try, in­clud­ing a $410 mil­lion in­vest­ment in Ab­b­Vie and a $324.4 mil­lion stake in Bris­tol My­ers Squibb. The move comes af­ter Berk­shire aban­doned its Te­va shares just last quar­ter. MAR­KET­INGRX In phar­ma pub­lic re­la­tions, every day feels like it might be a cri­sis. In fact, 76% of phar­ma com­mu­ni­ca­tions lead­ers ex­pect to face three or more crises this year — com­pared to less than half (49%) of their peers in oth­er in­dus­tries, ac­cord­ing to a new sur­vey. End­points Mar­ket­ingRx ed­i­tor Beth Bu­lik dives in­to the sen­ti­ment , how the Covid-19 pan­dem­ic am­pli­fied it and what PR ex­perts are do­ing to pre­pare. What do physi­cians think about the emerg­ing mar­ket for re­cur­rent C. diff? That de­pends on which spe­cial­ists you talk to. Fe­cal mi­cro­bio­ta trans­plants, or FMT, are fa­vored by gas­troen­terol­o­gists fa­mil­iar with them, while in­fec­tious dis­ease doc­tors pre­fer tra­di­tion­al small mol­e­cule ther­a­pies, ac­cord­ing to a re­cent fu­ture mar­ket eval­u­a­tion by Spher­ix Glob­al In­sights. Phar­ma ad spend­ing is con­cen­trat­ed at the top. A hand­ful of health con­di­tions — along with a hand­ful of brands in­side each con­di­tion — dom­i­nate in­dus­try ad­ver­tis­ing, ac­cord­ing to deep dive re­search from Me­di­aRadar. Its lat­est re­port finds that five ther­a­py ar­eas ac­count­ed for al­most half (43%) of all phar­ma DTC spend­ing last year, with di­a­betes com­ing up on top, fol­lowed by pso­ri­a­sis, arthri­tis, HIV/AIDs and breast can­cer. GSK en­list­ed ’80s child star turned di­rec­tor, writer and mom Soleil Moon Frye, known for her “Punky Brew­ster” char­ac­ter, dur­ing the pan­dem­ic last year to spot­light menin­gi­tis B vac­ci­na­tions for teens. At the time, the al­ready low rate for MenB vac­ci­na­tions was dip­ping even low­er. Now the “ Ask2BSure ” cam­paign is re-up­ping with Frye and oth­er par­ent in­flu­encers to con­tin­ue to raise aware­ness and with the ad­di­tion­al goal to em­pha­size the im­por­tance of par­ents’ shared de­ci­sion-mak­ing with doc­tors. Ab­b­Vie’s Al­ler­gan Aes­thet­ics is en­list­ing re­al peo­ple for the first time in a new cam­paign for CoolSculpt­ing. Its lat­est push for the body fat-freez­ing sys­tem in­cludes TV com­mer­cials, along with dig­i­tal and so­cial me­dia, where peo­ple talk about hav­ing chil­dren or ag­ing and their bod­ies chang­ing. Ho­log­ic en­lists long­time spokesper­son and breast can­cer sur­vivor Sh­eryl Crow to talk about an­nu­al mam­mo­grams; Three-time Olympic medal win­ner Gail De­v­ers stars in Hori­zon’s lat­est dis­ease aware­ness cam­paign; J&J cen­ters pub­lic health threats in its newest Quick­Fire chal­lenge; catch up on these and snip­pets in this week’s Mar­ket­ingRx roundup . FDA+ Af­ter a pa­tient died in an ear­ly-stage study, the FDA is ap­ping the brakes on a Flag­ship-backed biotech’s R&D ef­forts to de­vel­op a new treat­ment for blood can­cers. Foghorn’s par­tial clin­i­cal hold fol­lows a sim­i­lar in­ci­dent at Ku­ra On­col­o­gy, which was sim­i­lar­ly con­duct­ing a study in acute myeloid leukemia. Foghorn said dif­fer­en­ti­a­tion syn­drome may be to blame. The FDA is dip­ping its toe in­to the wild world of con­gres­sion­al pol­i­tics as it at­tempts to fix a ma­jor court de­ci­sion that could have a chill­ing ef­fect on rare dis­ease R&D. In a rare state­ment that ob­servers say amounts to lob­by­ing, the FDA wrote that the rul­ing that it nev­er should’ve ap­proved a rare dis­ease drug due to or­phan drug ex­clu­siv­i­ty could have cat­a­stroph­ic con­se­quences. The first ver­sion of the Sen­ate health com­mit­tee’s bill to reau­tho­rize all the dif­fer­ent FDA user fees con­tains on­ly a few con­tro­ver­sial items and does not ad­dress ei­ther ac­cel­er­at­ed ap­proval re­forms or clin­i­cal tri­al di­ver­si­ty. The House En­er­gy and Com­merce Com­mit­tee, on the oth­er hand, found bi­par­ti­san sup­port for a bill that fea­tures ac­cel­er­at­ed ap­proval re­forms, man­u­fac­tur­ing in­spec­tion tweaks at the FDA, and oth­ers. Sanofi CEO Paul Hud­son’s ef­fort to de­vel­op new rare dis­ease drugs and broad­en his com­pa­ny’s of­fer­ings leaped for­ward with the EMA’s sign­ing off on Sanofi’s — and the EU’s — first drug to treat the non-cen­tral ner­vous sys­tem man­i­fes­ta­tions of the rare and de­bil­i­tat­ing Nie­mann-Pick dis­ease. The en­zyme re­place­ment ther­a­py was first de­vel­oped by Gen­zyme, and it’s not yet ap­proved in the US. As the ma­jor­i­ty of drug short­ages are still as­so­ci­at­ed with man­u­fac­tur­ing-re­lat­ed qual­i­ty is­sues, the FDA pub­lished new draft guid­ance spelling out how to proac­tive­ly as­sess risks to man­u­fac­tur­ing process­es and sup­ply chains, while un­der­stand­ing the mar­ket’s vul­ner­a­bil­i­ties. Ac­cord­ing to the agency, three types of drugs will re­quire risk man­age­ment plans. An FDA-fund­ed pi­lot pro­gram found that on­ly about half of a se­lect group of clin­i­cal tri­als could be well-em­u­lat­ed with avail­able re­al world ev­i­dence. The pro­gram sought to em­u­late 30 ran­dom­ized clin­i­cal tri­als us­ing clin­i­cal prac­tice da­ta, which they were able to for 50% of them. But for oth­ers, sub­stan­tive em­u­la­tion chal­lenges led to di­ver­gent re­sults. Eli Lil­ly has clinched a key OK for tirzepatide, draw­ing the cur­tain on what it hopes will be a ma­jor car­diometa­bol­ic fran­chise and a se­ri­ous ri­val to a megablock­buster from No­vo Nordisk. With the FDA ap­proval , Lil­ly will start mar­ket­ing tirzepatide in­jec­tion as Moun­jaro to adults with type 2 di­a­betes, the first such treat­ment that’s al­so a dual GIP/GLP-1 re­cep­tor ag­o­nist. Back in late De­cem­ber, the FDA flagged con­cerns about the glass vials Gilead used to con­tain its ex­per­i­men­tal HIV ther­a­py lenaca­pavir, cit­ing risks of form­ing sub-vis­i­ble glass par­ti­cles in the drug so­lu­tion. A com­plete re­sponse let­ter lat­er, Gilead said it’s fi­nal­ly found an al­ter­na­tive glass vial that sat­is­fies the FDA, which is lift­ing the clin­i­cal hold. The path Cy­to­ki­net­ics has plot­ted for its lead heart drug will face one more po­ten­tial ob­sta­cle as it ap­proach­es its PDU­FA date this fall: an ad­comm . The biotech was in­formed that the FDA plans to con­vene its pan­el of ex­perts to dis­cuss the ap­pli­ca­tion for ome­cam­tiv mecar­bil in heart fail­ure, set­ting up an­oth­er pos­si­ble speed bump fol­low­ing dis­ap­point­ing da­ta and deal breakups. MAN­U­FAC­TUR­ING Re­silience, the bio­man­u­fac­tur­ing shop co-found­ed by Bob Nelsen, found its next part­ner with­in the ARCH port­fo­lio. In a col­lab­o­ra­tion built for the long haul, it will make Be Bio’s B cell ther­a­pies for rare dis­eases, shoul­der­ing the ini­tial man­u­fac­tur­ing costs in ex­change for mile­stones and roy­al­ties down the road. Con­tin­u­ing its ex­pan­sion spree, Fu­ji­film Irvine Sci­en­tif­ic has opened its new In­no­va­tion and Col­lab­o­ra­tion Cen­ter in Suzhou New Dis­trict, Chi­na. Ac­cord­ing to Fu­ji­film, the 12,000-square-foot site will be re­spon­si­ble for the com­pa­ny’s cell cul­ture me­dia op­ti­miza­tion, analy­sis and de­sign ser­vices. Even af­ter Utah-based Clene failed to hit its pri­ma­ry end­points for its ALS drug last year, the state of Mary­land is putting its mon­ey at least be­hind Clene’s man­u­fac­tur­ing fa­cil­i­ty. The Mary­land Board of Pub­lic Works fi­nal­ized a $3 mil­lion, 60-month loan fa­cil­i­ty with Clene that will go to­ward equip­ment pur­chas­es and sup­port the ex­pan­sion of Clene’s op­er­a­tions. Just a week af­ter Tai­wan-based CD­MO Bo­ra Phar­ma­ceu­ti­cals ac­quired Eden Bi­o­log­ics, it es­tab­lished Bo­ra Bi­o­log­ics, ex­pand­ing in­to the bio­phar­ma­ceu­ti­cal mar­ket. With the buy­out, it gained pro­duc­tion ca­pac­i­ty of cell lines as well as im­proved qual­i­ty con­trol and in­spec­tion spec­i­fi­ca­tions. Four years af­ter No­var­tis first an­nounced plans to close a site in Grims­by, UK — and fol­low­ing sev­er­al de­lays — it fi­nal­ly sold the fa­cil­i­ty. As a re­sult of the sale to Hum­ber In­dus­tri­als, more than 200 work­ers will face im­mi­nent lay­offs . As Catal­ent ven­tures fur­ther in­to the world of cell and gene ther­a­py man­u­fac­tur­ing, the New Jer­sey-based CD­MO has re­vealed its new ade­no-as­so­ci­at­ed vi­ral vec­tor plat­form . The Up­Tem­po Vir­tu­oso plat­form can stream­line the AAV man­u­fac­tur­ing process and it’s de­signed to pro­duce prod­ucts for the clin­ic in just nine months. DON’T MISS Biotech is one of the smartest, best ed­u­cat­ed in­dus­tries on the plan­et. And in these times, savvy, ex­pe­ri­enced lead­ers run­ning biotech star­tups are be­ing test­ed as nev­er be­fore, as even those with strong prospects are be­ing cooked in the same ket­tle as the bad. So how do you re­spond to these ex­tra­or­di­nary times? That’s what End­points’ John Car­roll will be ex­plor­ing dur­ing a live pan­el dis­cus­sion at BIO in San Diego. He in­tro­duces his guests here . Mar­tin Shkre­li, the in­fa­mous biotech CEO who made head­lines for his jeer­ing as­sault on a le­gion of crit­ics in and out of Con­gress, is back on the streets af­ter four years in­side fed­er­al prison. As his lawyer con­firmed, the “phar­ma bro” had been trans­ferred to a halfway house in New York with a few more months to go un­der fed­er­al cus­tody, slat­ed to end Sep­tem­ber 14. Gilead Sci­ences’ $100 mil­lion pledge to fight the HIV epi­dem­ic in the south­ern US states vault­ed it to the top of an an­nu­al phil­an­thropic giv­ing list , ahead of the Bill & Melin­da Gates Foun­da­tion for the first time since the Fun­ders Con­cerned About AIDS be­gan count­ing. AUTHOR John Carroll Editor & Founder Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront. Keep reading Endpoints with a free subscription Unlock this story instantly and join 142,500+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Tyler Patchen News Reporter A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand. Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development. According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now. Read More John Carroll Editor & Founder Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts. This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share. Keep reading Endpoints with a free subscription Unlock this story instantly and join 142,500+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Zachary Brennan Senior Editor Sanofi CEO Paul Hudson has made clear his intention to develop new rare disease drugs and broaden his company’s offerings. That effort leaped forward on Friday with the EMA’s signing off on the company’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease. The enzyme replacement therapy, developed to replace patients’ deficient or defective enzyme, known as acid sphingomyelinase, was first developed by Genzyme, which Sanofi acquired for more than $20 billion in 2011. That acquisition has also helped Sanofi pull in sales in the field of MS. Read More Zachary Brennan Senior Editor As the majority of drug shortages are still associated with manufacturing-related quality issues, the FDA on Thursday published new draft guidance spelling out how to proactively assess risks to manufacturing processes and supply chains, while understanding the market’s vulnerabilities. While drug shortages peaked in 2011, the FDA says in its new 18-page draft guidance that the number of new drug shortages “has declined significantly since” that peak, reaching a low in 2015 and 2016, thanks in part to a new law’s enactment, known as FDASIA, which helped the agency better prevent or mitigate drug supply disruptions and shortages, and clarified cGMP requirements. Read More