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.

With AS­CO be­hind us and BIO just ahead, we’re feel­ing the heat of the biotech sum­mer. Many of my col­leagues will be in San Diego next week. Safe trav­els to every­one mak­ing the trip there.

#BIO22 spe­cial

End­points News will be host­ing our first in-per­son events at BIO in two years, and we have a full slate of pan­els lined up to dis­cuss bio­phar­ma at the cross­roads. Set­ting the stage, End­points founder John Car­roll chat­ted with BIO chair Paul Hast­ings, polled the E100 group of biotech CEOs for their sen­ti­ments as well as views on hot but­ton is­sues, and asked Deal­For­ma chief Chris Doko­ma­ji­lar to crunch the num­bers a lit­tle bit ear­ly on H1 for a look at the trends in play. The spe­cial pre­view al­so fea­tures a look in­to M&A for the sec­ond half of the year, ac­cord­ing to sea­soned play­ers, and per­spec­tives from re­al es­tate de­vel­op­ers. Here’s the full line­up with more de­tails.

#AS­CO22: View from the ground

Over the past week­end, tens of thou­sands of physi­cians, clin­i­cal tri­al in­ves­ti­ga­tors, aca­d­e­mics, R&D sci­en­tists and bio­phar­ma lead­ers de­scend­ed up­on Chica­go for the first in-per­son AS­CO meet­ing since the pan­dem­ic on­set. And as Kyle LaHu­cik ob­served on the side­lines of the con­fer­ence, even as a suite of promi­nent drugs swept the head­lines with da­ta up­dates, min­gling face-to-face stole the spot­light as ex­ecs re­unit­ed with “50,000 of (their) clos­est friends.” Check out our full cov­er­age here.

Smooth flight for blue­bird? 

Ahead of what may be make-or-break meet­ings for blue­bird bio, the FDA re­leased brief­ing doc­u­ments on two of its gene ther­a­pies sig­nal­ing a like­ly green light for one to treat a rare blood dis­or­der and ques­tions for the oth­er — an ex­per­i­men­tal treat­ment for cere­bral adrenoleukody­s­tro­phy — due to un­cer­tain­ty in both ben­e­fit and risk. Sur­pris­ing­ly, though, the ad­comm gave a big thumbs up to both the CALD and be­ta-tha­lassemia ther­a­pies de­spite those con­cerns.

ARCH’s man­u­fac­tur­ing play racks up cash, part­ners

Re­silience, the CD­MO with am­bi­tions to be­come the Fox­conn of bio­man­u­fac­tur­ing, had quite the week. It got a boost in the form of a $625 mil­lion Se­ries D, which will be used to con­tin­ue in­vest­ing in its in­fra­struc­ture through more deals, po­ten­tial ac­qui­si­tions and R&D ex­pan­sion so that it can do it all — and be ready for any­thing. Then it cracked open two part­ner­ships, first with the Uni­ver­si­ty of Texas MD An­der­son Can­cer Cen­ter then with the Park­er In­sti­tute for Can­cer Im­munother­a­py, around cell and gene ther­a­pies for can­cer.

PRE­MI­UM

GV plucks up No­var­tis vet for lat­est en­tre­pre­neur-in-res­i­dence

David Schenkein has added a new mem­ber to GV’s still quite ex­clu­sive club of biotech CEOs-in-wait­ing. And this time the Google Ven­tures play­er is turn­ing to some­one he’d worked with for years at Agios, the com­pa­ny he built from a white sheet of pa­per. Schenkein re­cruit­ed Kevin Marks out of NI­BR, where he’s been work­ing as head of the on­col­o­gy dis­cov­ery group, a move that he says rep­re­sents the “growth mode” at GV life sci­ences. While he’s not rush­ing to join a start­up, Marks not­ed drug re­sis­tance is a key are­na he’s fo­cused on.

#AS­CO22

• Fol­low­ing back-to-back Phase III TIG­IT flops, Roche spelled out the da­ta on the first failed tri­al, dubbed SKY­SCRAPER-02. Com­pared to an av­er­age over­all sur­vival of 12.9 months with on­ly Tecen­triq and chemother­a­py, the ad­di­tion of tiragolum­ab failed to move the nee­dle at all, with an over­all sur­vival of 13.1 months — an ad­mit­ted­ly dis­ap­point­ing re­sult.
• In the lat­est in a se­ries of big clin­i­cal set­backs, Pfiz­er de­tailed how its close­ly watched PALO­MA-2 Phase III study of Ibrance com­bined with the chemo hor­mone ther­a­py Fe­mara (letro­zole) failed to sig­nif­i­cant­ly move the di­al on sur­vival for first-line cas­es of metasta­t­ic breast can­cer com­pared to Fe­mara alone, with a mar­gin­al haz­ard ra­tio of 0.956. The phar­ma gi­ant was left de­fend­ing the CDK4/6 fran­chise.
• Mi­rati Ther­a­peu­tics is pulling out all the stops to beef up its case for ada­gra­sib as it plays catch-up to Am­gen’s Lumakras. In 19 evalu­able pa­tients with non-small cell lung can­cer who have ac­tive and un­treat­ed CNS metas­tases, the biotech said its drug led to six ob­jec­tive in­tracra­nial re­spons­es, a first in CNS-spe­cif­ic ac­tiv­i­ty.
• Breast can­cer is of­ten cat­e­go­rized by well-de­fined sub­groups, but with their lat­est En­her­tu da­ta, As­traZeneca and Dai­ichi Sankyo be­lieve they may just ce­ment a new one: HER2-low. Among a group of pa­tients who pre­vi­ous­ly would’ve been con­sid­ered HER2-neg­a­tive, En­her­tu beat out chemother­a­py in staving off the can­cer and ex­tend­ing over­all sur­vival.
• Months af­ter draw­ing ire and invit­ing skep­ti­cism from an­a­lysts for re­leas­ing an up­beat Q&A with­out any hard da­ta for its lat­est Trodelvy tri­al, Gilead re­leased the full da­ta at AS­CO, show­ing that the an­ti­body-drug con­ju­gate slight­ly out­did stan­dard care in a Phase III study in­volv­ing pa­tients with HR+/HER2- metasta­t­ic breast can­cer.
• Leg­end and J&J’s Janssen said Carvyk­ti, the sec­ond FDA-ap­proved BC­MA CAR-T, has shown it can stay ef­fec­tive at al­most 28 months of me­di­an fol­low-up. The ther­a­py, which first took AS­CO by sur­prise five years ago, saw its over­all re­sponse rate re­main­ing con­sis­tent at 98% among the 97 pa­tients treat­ed, with 70% of the pa­tients still alive.
• Here’s an­oth­er slate of up­beat da­ta com­ing from a low-pro­file Chi­nese biotech: Shang­hai-based Ori­Cell, a pri­vate play­er, high­light­ed promis­ing Phase I re­sults for its per­son­al­ized GPRC5D-di­rect­ed CAR-T OriCAR-017. Di­rect­ed at a small group of treat­ment-re­sis­tant pa­tients suf­fer­ing from mul­ti­ple myelo­ma, the biotech re­port­ed a 100% over­all re­sponse rate with 60% strin­gent CRs. The tri­al al­so re­cruit­ed pa­tients who failed a BC­MA-di­rect­ed CAR-T.
• Look­ing to build on its break­out Covid suc­cess and put to­geth­er an on­col­o­gy fran­chise, BioN­Tech is back with more da­ta on its in­di­vid­u­al­ized mR­NA can­cer jab known as au­to­gene ce­vumer­an in the most com­mon form of pan­cre­at­ic can­cer. Specif­i­cal­ly, it shared more about a sin­gle-site Phase I study among a sub­set of pa­tients with poor prog­no­sis.
• Adicet gave a de­tailed break­down of its buoy­ant ear­ly da­ta for its off-the-shelf gam­ma delta T cell play, re­veal­ing that at the da­ta cut­off, six of the eight pa­tients en­rolled record­ed a com­plete re­sponse. That’s like­ly to sharp­en the fo­cus on the gam­ma delta crowd — which is hop­ing to see en­hanced ef­fi­ca­cy — but dura­bil­i­ty re­mains to be seen.
• Mis­match re­pair-de­fi­cient, a rare bio­mark­er, was the spot­light of a small Phase I study where every sin­gle pa­tient treat­ed with GSK’s PD-1, Jem­per­li (dostar­limab), saw their rec­tal can­cer com­plete­ly dis­ap­pear. None of them need­ed chemother­a­py, ra­di­a­tion, or surgery — the com­bi­na­tion that makes up the stan­dard of care for this dis­ease.
• As it dodges a takeover bid, Zymeworks wants to fol­low Dai­ichi Sankyo’s foot­steps in find­ing a Big Phar­ma part­ner to turn its HER2 bis­pe­cif­ic, zanidatam­ab, in­to a world­wide drug. And it said it has the da­ta to sup­port it, re­port­ing 1 com­plete re­sponse and 18 par­tial re­spons­es in a small study — trans­lat­ing to a con­firmed ORR of 90.5%.
• Merus wants to tack­le a rare ge­nom­ic fu­sion in on­col­o­gy that im­pacts about 5,000 new pa­tients in the US each year, and the biotech thinks its in­ter­im ef­fi­ca­cy da­ta — sport­ing a 34% over­all re­sponse rate — can lead its bis­pe­cif­ic zeno­cu­tuzum­ab to be­come the first ap­proved med for those pa­tients. Mean­while, chief com­peti­tor El­e­va­tion On­col­o­gy dis­closed its own da­ta up­date.
• CG On­col­o­gy built on some head-turn­ing AACR da­ta on a com­bi­na­tion of its on­colyt­ic virus with Mer­ck’s Keytru­da for blad­der can­cer; EQRx con­ced­ed that, even though the PD-L1 in­hibitor it li­censed from CStone de­liv­ered pos­i­tive Phase III, it plans to file for ap­proval out­side of US first. Here’s the best of the rest from the AS­CO week­end.

PHAR­MA

• Add Cana­da to the list of coun­tries where Bio­gen’s con­tro­ver­sial Alzheimer’s drug Aduhelm isn’t want­ed by health of­fi­cials. The biotech is pulling ad­u­canum­ab’s new drug sub­mis­sion in Cana­da af­ter the coun­try’s health agency in­di­cat­ed that “the da­ta pro­vid­ed would not be suf­fi­cient to sup­port a mar­ket­ing au­tho­riza­tion.”
• Bris­tol My­ers Squibb laid out some big plans over the last cou­ple of years around po­ten­tial la­bel ex­pan­sions for its Cel­gene-ac­quired ane­mia drug Re­blozyl. But one of those will no longer be in the cards as BMS pulled the plug on an ane­mia pro­gram as reg­u­la­tors call in­to ques­tion the drug’s risk-ben­e­fit pro­file in a rare blood dis­or­der.
• No­var­tis is the lat­est com­pa­ny to fall vic­tim to a cy­ber­at­tack. Spe­cial­ized IT web­site Bleep­ing Com­put­er first re­port­ed that the Swiss gi­ant got hacked late last week, af­ter hack­ing group In­dus­tri­al Spy of­fered up da­ta on its “ex­tor­tion mar­ket­place” that was ap­par­ent­ly stolen from No­var­tis labs. The com­pa­ny said lit­tle oth­er than that it’s aware of the mat­ter and no sen­si­tive da­ta have been com­pro­mised.
• Amid in­creased con­cerns about the lack of clin­i­cal tri­al di­ver­si­ty, new re­search sug­gests that many Black pa­tients aren’t made aware of tri­als in the first place. Of 102 pa­tients who self-iden­ti­fied as Black in a re­cent Metasta­t­ic Breast Can­cer Al­liance sur­vey, 40% said that no one on their care team had dis­cussed clin­i­cal tri­als, even though more than 90% said they would be in­ter­est­ed in learn­ing about them.
• Hav­ing racked up ap­provals in more than 100 coun­tries, GSK is bring­ing one of its wide­ly-li­censed vac­cines to the US, giv­ing pe­di­a­tri­cians a new op­tion for a com­mon child­hood shot for the first time in more than 50 years. The FDA ap­proved GSK’s measles, mumps and rubel­la vac­cine for in­di­vid­u­als 12 months and old­er, which is brand­ed as Pri­or­ix.
• More than four years af­ter Roche bought Igny­ta, ac­quir­ing a drug — en­trec­tinib — that scored FDA ap­proval about 18 months lat­er, Roche now has a com­pan­ion di­ag­nos­tic to pair with that drug and ex­pand­ed treat­ment op­tions. The agency ap­proved a com­pan­ion di­ag­nos­tic from Foun­da­tion Med­i­cine for en­trec­tinib, which is cur­rent­ly be­ing mar­ket­ed as Ro­z­lytrek.
• A let­ter au­thored by a trio of Har­vard re­searchers — in­clud­ing med­ical pro­fes­sors Ben Rome and Aaron Kessel­heim — sought to add clar­i­ty to how the launch price for new­ly-FDA-ap­proved drugs has been steadi­ly in­creas­ing. Be­tween 2008 and 2021, they wrote, the launch prices for pre­scrip­tion drugs sky­rock­et­ed al­most 90x.
  
• As it rounds out its first decade as an in­de­pen­dent com­pa­ny on Wall Street, Ab­b­Vie joined some of its phar­ma brethren in lay­ing out an En­vi­ron­ment, So­cial & Gov­er­nance strat­e­gy. Amid a big sus­tain­abil­i­ty push in the in­dus­try, Ab­b­Vie has been able to re­duce its car­bon emis­sions by 25% since 2015. By 2035, the com­pa­ny hopes to reach 50% — along­side oth­er goals.
• Sen­a­tor Raphael Warnock, De­mo­c­rat of Geor­gia, urged Sen­ate Ma­jor­i­ty Leader Chuck Schumer to fol­low through on his promise to bring to vote a bill that would cap month­ly in­sulin costs at $35 for those with in­sur­ance. In a let­ter, he said “Geor­gians are hurt­ing” and that he’s deeply dis­ap­point­ed.

R&D

• GSK an­nounced that its adult RSV vac­cine cleared a Phase III tri­al of­fer­ing “ex­cep­tion­al pro­tec­tion” to 25,000 en­rollees over the age of 60, set­ting up a planned roll­out with reg­u­la­tors. But while GSK cheered this as a clear suc­cess, and a land­mark first, it’s keep­ing the key fig­ure on ef­fi­ca­cy in pre­vent­ing se­vere in­fec­tions un­der wraps, for now, leav­ing the next big ques­tion of how this will look to reg­u­la­tors and in­dus­try ri­vals still unan­swered.
• Dis­ap­point­ing Phase II/III re­sults for Prax­is’ lead de­pres­sion drug sent the biotech’s shares on a nose­dive, forc­ing a sub­stan­tial pipeline re­or­ga­ni­za­tion and lay­offs. A can­di­date that once in­spired high hopes, the GA­BA pos­i­tive al­losteric mod­u­la­tor has had a bumpy road. It missed the pri­ma­ry end­point as well as all of its sec­ondary end­points.
• In­novent Bi­o­log­ics burst on­to the obe­si­ty scene, crash­ing the big ri­val­ry be­tween Eli Lil­ly and No­vo Nordisk, with Phase II da­ta show­ing its GLP-1 re­cep­tor/glucagon re­cep­tor dual ag­o­nist re­duced pa­tients’ body weights by more than 12% at the high­est dose com­pared to place­bo. The tri­al was con­duct­ed in Chi­na.
• A Cal­i­for­nia biotech fa­mil­iar with speed bumps ran in­to an­oth­er ear­ly Wednes­day as it tries to push its on­ly ap­proved drug in­to an­oth­er in­di­ca­tion. Ac­cord­ing to Rigel Phar­ma­ceu­ti­cals, a sur­pris­ing place­bo re­sponse spurred the Phase III fail­ure for its bleed­ing dis­or­der drug fos­ta­ma­tinib. But it’s no stranger to head­winds.
• Blue­print is forg­ing ahead on its slow crawl to broad­en its mar­ket for Ay­vak­it. For the sec­ond part of a Phase II tri­al in the non-ad­vanced form of sys­temic mas­to­cy­to­sis, the biotech is swap­ping out the pri­ma­ry end­point at the FDA’s rec­om­men­da­tion so that it can look at a broad­er group of pa­tients
• Al­ny­lam is show­ing off some new ef­fi­ca­cy da­ta on a drug at the heart of its $1 bil­lion-plus Re­gen­eron col­lab­o­ra­tion ce­ment­ed three years ago. The pos­i­tive news cen­ters on a Phase II de­scrip­tive tri­al for cem­disir­an, an RNAi drug tar­get­ing the C5 com­po­nent of the path­way that the part­ners are work­ing on in the kid­ney dis­ease known as IgAN.
• As it looks to bring le­brik­izum­ab to mar­ket and chal­lenge Dupix­ent, Eli Lil­ly shared ear­ly long0term da­ta for 10 pa­tients on its atopic der­mati­tis drug. Of the 10 pa­tients who re­spond­ed to le­brik­izum­ab at 16 weeks, eight main­tained skin clear­ance at one year on the two-week or four-week reg­i­men, Lil­ly re­port­ed fol­low­ing pos­i­tive Phase III re­sults.
• Aim­ing to jump ahead of Big Phar­ma heavy­weights in the set­back-laden C. dif­fi­cile race, Seres is bar­rel­ing for­ward with its own mi­cro­bio­me treat­ment af­ter stak­ing a late-stage win. Re­sults from an open-la­bel ex­ten­sion study sug­gest­ed its drug spurred a sus­tained clin­i­cal re­sponse in 91.3% of pa­tients at eight weeks and 86% at 24 weeks, con­firm­ing da­ta from an ear­li­er tri­al.

PEO­PLE

• A year af­ter a Supreme Court opin­ion put the fi­nal nail in the cof­fin for Amarin’s fish-oil heart drug, the biotech is mak­ing some changes to keep its doors open. Now that those block­buster dreams are quashed, it will be cut­ting 65% of its US com­mer­cial staff, while al­so chop­ping over­all ex­pens­es and “re­fin­ing” its R&D strat­e­gy. Its CFO is among the ones to go. All of this will save $100 mil­lion over the next year and keep Vas­cepa alive, most­ly in Eu­rope. And the UK’s NICE fu­eled those dreams by agree­ing to cov­er the drug
• Sanofi is fac­ing down a strike threat in Ko­rea amid an in­creas­ing num­ber of con­fronta­tions be­tween Big Phar­ma and their la­bor unions over pay de­mands. A Ko­re­an pub­li­ca­tion cites la­bor of­fi­cials who have been un­hap­py over the com­pa­ny’s of­fer of a 1.5% in­crease in pay, cit­ing in­fla­tion as well as ris­ing sales dur­ing the pan­dem­ic.
• Fol­low­ing a dis­ap­point­ing da­ta re­veal and sub­se­quent stock drop late last year, Ra­dius Health is now fac­ing an ac­tivist at­tack from two mi­nor­i­ty in­vestors aim­ing to right the ship. Ve­lan Cap­i­tal In­vest­ment Man­age­ment and Reper­toire Part­ners filed their proxy state­ments in a bid to nom­i­nate a slate of three new board mem­bers at Ra­dius’ an­nu­al share­hold­er meet­ing next month.
• The biotech bear mar­ket con­tin­ues to put pres­sure on small and mid-cap com­pa­nies, with can­cer-fo­cused Vin­cerx now un­der­go­ing a shift. Just a lit­tle less than two years af­ter it went pub­lic via a SPAC, and af­ter shares fell more than 80% in 2022, Vin­cerx said it would lay off 33% of its staff as part of an ef­fort to get its lead pro­gram through two Phase I stud­ies.

DEALS

• Beef­ing up its neu­ro pipeline in the shad­ow of Aduhelm, Bio­gen struck a deal with Cana­di­an biotech Alec­tos Ther­a­peu­tics to de­vel­op a drug aimed at restor­ing lyso­some func­tion, adding an­oth­er can­di­date to its Parkin­son’s ef­forts. The deal, which cen­ters around GBA2, in­volves $15 mil­lion up­front and hun­dreds of mil­lions more in po­ten­tial mile­stones.
• Suf­fer­ing biotech Yu­man­i­ty has found that “strate­gic al­ter­na­tive” it start­ed search­ing for ear­ly this year as its share price shriv­eled, forc­ing deep staff cuts. It will sell off most of its pipeline — in­clud­ing a Phase I Parkin­son’s drug and dis­cov­ery-stage neu­ro­science can­di­dates — to J&J for $26 mil­lion in cash. As for the rest of the com­pa­ny, it’s re­verse merg­ing with the pri­vate biotech Kine­ta, which will wind up with the pub­lic list­ing and one Mer­ck-part­nered pro­gram.

AI

• For Alex Zha­voronkov, the re­cent biotech mar­ket down­turn is, in fact, a good thing be­cause it’s crys­tal­iz­ing the win­ners in the AI drug dis­cov­ery world — his com­pa­ny, In­sil­i­co, be­ing one of them in his view. Proud as ever about the $60 mil­lion round he just raised, the CEO is adding ro­bot­ic labs to his vi­sion for the fu­ture of how drugs are made.
• Sev­er­al months af­ter ty­ing the bow with Sanofi, French-US AI play­er Owkin an­nounced its next deal with Big Phar­ma, col­lab­o­rat­ing with Bris­tol My­ers Squibb in car­dio­vas­cu­lar dis­ease and us­ing its fed­er­at­ed learn­ing plat­form to im­prove the phar­ma’s clin­i­cal tri­al op­ti­miza­tion and de­sign. It’s grab­bing $80 mil­lion to kick things off, mon­ey that will be chan­neled in­to da­ta gen­er­a­tion.
• Hav­ing been in­volved in a num­ber of at­tri­tion task force ef­forts dur­ing his tenure as Eu­ro­pean R&D chief at Pfiz­er, David Rob­lin reck­ons there’s a dif­fer­ent way to go through the R&D process — with ma­chine learn­ing and new tech­nolo­gies like sin­gle-cell tran­scrip­tomics. That’s why he’s jump­ing on as CEO of Re­la­tion Ther­a­peu­tics, which closed a $25 mil­lion seed round to build a “sec­ond to none” plat­form of in­te­gra­tion.

CELL & GENE TX

• Tes­sa Ther­a­peu­tics in Sin­ga­pore has been through mul­ti­ple rais­es over the last decade for its cell ther­a­py R&D work. But with US-based VC Po­laris step­ping in to lead the new $126 mil­lion round, the biotech is call­ing this new one a Se­ries A. “I think there’s no way to sug­ar­coat this,” the CEO says. “We are repo­si­tion­ing the com­pa­ny.”
• En route to meet­ing with the FDA lat­er this year on po­ten­tial reg­is­tra­tion strat­e­gy for its al­lo­gene­ic CAR-T, Pre­ci­sion Bio­sciences is out with new in­ter­im Phase I/IIa clin­i­cal da­ta on the lead pro­gram sport­ing a high rate of com­plete re­spons­es. But the biotech will have to shore up safe­ty con­cerns of its CD-19 di­rect­ed drug as four deaths were re­port­ed in a Phase I/IIa tri­al.
• Weeks af­ter shar­ing im­pres­sive ear­ly da­ta on its first tri­al for an off-the-shelf CAR-T ther­a­py, Cari­bou Bio­sciences is fol­low­ing up with re­sults from a sixth pa­tient who had an ini­tial com­plete re­sponse but saw their can­cer progress three months lat­er. And it’s es­ca­lat­ing the dose to see if it can make the treat­ment more durable.
• Amid a push for more in-house ca­pac­i­ty, gene ther­a­py play­er Re­genxbio opened a new fa­cil­i­ty at its cam­pus in the Wash­ing­ton DC sub­urb of Rockville, MD. Built in a year, the $65 mil­lion, 132,000-square-foot GMP fa­cil­i­ty will en­able the com­pa­ny to boost its man­u­fac­tur­ing of NAV Tech­nol­o­gy-based ade­no-as­so­ci­at­ed virus, or AAV, vec­tors at scales up to 2,000 liters.

STAR­TUPS

• The fa­ther-son duo be­hind Teneo­bio is back with an­oth­er biotech, com­pris­ing three bis­pe­cif­ic an­ti­bod­ies that Am­gen didn’t want when dish­ing out $900 mil­lion up­front to buy it out last year.  Tech­ni­cal­ly, An­co­ra Biotech is three shell com­pa­nies, one for each of its an­ti­bod­ies or “Teneo ba­bies,” that could ei­ther go pub­lic or be sold off in­di­vid­u­al­ly. Light­speed and SR One are back­ing An­co­ra with $60 mil­lion.
• David Bak­er, one of the biggest names in the pro­tein de­sign space, is throw­ing his weight be­hind an­oth­er biotech — one that seeks to take his famed work in pro­tein fold­ing and ex­tend it fur­ther in­to drug dis­cov­ery and de­vel­op­ment. CHARM Ther­a­peu­tics, based out of the UK, launched with $50 mil­lion and a pro­tein and lig­and pre­dic­tion plat­form dubbed Drag­on­Fold.
• Look­ing to cir­cum­vent the ob­sta­cles trip­ping up the vi­ral vec­tors that car­ried the first gen­er­a­tion of gene ther­a­pies in­to the clin­ic by us­ing non-vi­ral de­liv­ery, Code Bio­ther­a­peu­tics raised $75 mil­lion, cour­tesy of a group of blue-chip in­vestors, to push two pro­grams in­to IND-en­abling stud­ies. Led by GSK BD vet Bri­an McVeigh, Code Bio has a Take­da deal un­der its belt.
• San Fran­cis­co Bay’s An­a­genex emerged from “se­mi-stealth,” seek­ing to cre­ate a closed feed­back loop on small mol­e­cule drug de­vel­op­ment and se­cur­ing $30 mil­lion via a Catalio-led Se­ries A. CEO Nico­las Tilmans added that it seeks to take a sort of di­rect­ed evo­lu­tion ap­proach, look­ing to re­fine its li­brary of com­pounds for a spe­cif­ic tar­get over and over again and im­prove, al­lud­ing to evo­lu­tion­ary bi­ol­o­gist Charles Dar­win’s con­cept known as Dar­win’s finch­es.
• Look­ing to catch the ra­dio­phar­ma wave, Berlin-based Ariceum Ther­a­peu­tics se­cured $26.7 mil­lion as it starts with a team and as­sets from Ipsen. The lead pro­gram is a pep­tide de­riv­a­tive, and ex­ecs ar­gue that work­ing with a Phase II clin­i­cal com­pound gives them an edge over oth­ers.
• De­gron Ther­a­peu­tics, a transpa­cif­ic biotech fo­cused on mol­e­c­u­lar glues, scored $22 mil­lion in Se­ries A funds as it works to vie for Big Phar­ma’s at­ten­tion. Found­ed just last year out of Shang­hai Tech Uni­ver­si­ty, the biotech boasts of a li­brary of about 6,000 po­ten­tial mol­e­c­u­lar glues.

FI­NANC­ING

• De­spite the bear mar­ket cre­at­ing on­go­ing strug­gles in the biotech sec­tor, Tim Lu’s Sen­ti Bio is charg­ing ahead with the com­ple­tion of its SPAC deal. But it’s cross­ing the fin­ish line with a much low­er cash haul than orig­i­nal­ly planned as SPAC in­vestors chose to cash out.
• Psy­che­delics up­start Eleu­sis, on the oth­er hand, axed its SPAC deal with Sil­ver Spike, cit­ing “cur­rent un­fa­vor­able mar­ket con­di­tions.” The deal would have giv­en the nine-year-old biotech about $288 mil­lion to bankroll hu­man tri­als of a psilo­cy­bin-based IV treat­ment and fu­el the ex­pan­sion of Eleu­sis’ sub­sidiary over­see­ing psy­che­del­ic drug ther­a­py clin­ics.
• Amid a strug­gling pub­lic mar­ket for biotechs, Eu­ro­pean ven­ture cap­i­tal firm For­bion drummed up an ini­tial close of €470 mil­lion ($504 mil­lion) for its sec­ond growth fund meant to boost late clin­i­cal-stage biotechs. This time around, it will be look­ing be­yond phar­ma spin­outs and pri­vate rounds to scout “pub­lic op­por­tu­ni­ties or un­der­val­ued as­sets.”
• Michelle Dipp and Jen­nifer Lum want to fo­cus on the things in biotech that are of­ten side­lined: man­u­fac­tur­ing, di­ag­nos­tic tests, soft­ware — the tools need­ed to pro­duce new drugs. Their first fund at Biospring Part­ners net­ted $245 mil­lion, and they’ve al­ready in­vest­ed in a cou­ple of com­pa­nies.
• On­ly a hand­ful of biotechs are go­ing af­ter LIL­RBs, com­po­nents of the in­nate im­mune sys­tem. But af­ter an ini­tial crop of da­ta out of Mer­ck in au­tumn 2020, more in­ter­est sprung up in the area, and a for­mer Mer­ck ex­ec has se­cured $74 mil­lion to take his Zurich-area biotech Im­munOs Ther­a­peu­tics’ lead pro­gram in­to a Phase Ia study.
• With the help of RA Cap­i­tal, Philly biotech Min­eralys Ther­a­peu­tics got its sec­ond VC in­fu­sion, worth $118 mil­lion cour­tesy of a Se­ries B af­ter break­ing out of stealth with $40 mil­lion just over a year ago. With a con­sor­tium of oth­er in­vestors tag­ging along for the ride, the biotech is cur­rent­ly fo­cus­ing on one as­set for hy­per­ten­sion — and putting its nose to the grind to ac­cel­er­ate R&D ef­forts.
• One of the ba­sic lessons in sci­ence class is that mi­to­chon­dria act as the pow­er­house of the cell. But LU­CA Sci­ence, a Japan­ese biotech, is look­ing to take it a step fur­ther, har­ness­ing the pow­er of mi­to­chon­dria to even­tu­al­ly cre­ate ther­a­pies for or­gan and tis­sue re­pair. CEO Rick Tsai, a Mer­ck and Al­ler­gan vet, just closed a $30.3 mil­lion Se­ries B.

CORO­N­AVIRUS

• A pan­el of FDA’s out­side ex­perts rec­om­mend­ed 21 to 0, with one mem­ber ab­stain­ing, that the agency give emer­gency use au­tho­riza­tion for No­vavax’s two-dose Covid-19 vac­cine for peo­ple aged 18 years and old­er. The ad­vi­sors’ vote of con­fi­dence was de­liv­ered days af­ter the FDA’s sci­en­tists de­ter­mined the vac­cine to be ef­fec­tive but with the caveat of po­ten­tial rare heart in­flam­ma­tion. But un­like its peers, which all re­ceived FDA clear­ance a day af­ter the ad­comm, No­vavax will have to wait for the agency’s re­view of man­u­fac­tur­ing up­dates.
• Mod­er­na says the lead can­di­date for the fall 2022 Covid boost­er shot has emerged as ear­ly one-month da­ta sug­gest that a bi­va­lent boost­er vac­cine tar­get­ing both Omi­cron and the orig­i­nal strain is more ef­fec­tive than its cur­rent shot in a Phase II/III tri­al with over 700 par­tic­i­pants.
• Af­ter months up­on months of ne­go­ti­a­tions with­in the World Trade Or­ga­ni­za­tion on a Covid-19 vac­cine IP waiv­er, or­ga­ni­za­tion lead­er­ship says that a deal may be in place, soon. But the crit­ics keep pil­ing up. A hand­ful of phar­ma ad­vo­ca­cy and lob­by­ing groups de­cried an IP waiv­er deal, ar­gu­ing that it’s un­nec­es­sary and threat­ens to quash in­no­va­tion.
• J&J’s trou­bles with Emer­gent BioSo­lu­tions are far from over. The em­bat­tled con­tract man­u­fac­tur­er, which trashed tens of mil­lions of J&J’s Covid-19 vac­cine dos­es be­cause of its con­t­a­m­i­nat­ed Bal­ti­more plant, is now telling J&J that it will have to pay some­where in the range of $125 mil­lion to $420 mil­lion for try­ing to ter­mi­nate their man­u­fac­tur­ing con­tract.
• While Mod­er­na and Pfiz­er have tak­en much of the lime­light when it comes to Covid-19 vac­cines, As­traZeneca’s boss is stand­ing by his com­pa­ny’s de­vel­op­ment ef­forts. In an in­ter­view with the BBC, Pas­cal So­ri­ot claimed his com­pa­ny’s Covid-19 vac­cine has man­aged to save a mil­lion lives de­spite fac­ing set­backs and said, “I don’t think I would do any­thing dif­fer­ent­ly from what we did.”

LAW

• With­out ad­mit­ting to any wrong­do­ing, Bris­tol My­ers Squibb agreed to pay al­most $11 mil­lion to set­tle al­le­ga­tions filed in a Cal­i­for­nia dis­trict court that it took part in an un­law­ful scheme with oth­er phar­ma com­pa­nies like Gilead and Janssen to pro­tect their HIV drug prof­its while slow­ing gener­ic com­pe­ti­tion.
• No­vo Nordisk is in fed­er­al court, thanks to the Equal Em­ploy­ment Op­por­tu­ni­ty Com­mis­sion. 62-year-old Deb­o­rah Hunter, an obe­si­ty care spe­cial­ist em­ployed at the phar­ma, ac­cused No­vo of age dis­crim­i­na­tion when she orig­i­nal­ly got passed over for a lat­er­al trans­fer in 2018. While No­vo Nordisk found that a man­ag­er vi­o­lat­ed com­pa­ny pol­i­cy, Hunter was nev­er of­fered the trans­fer.
• Three Re­pub­li­can and three De­mo­c­rat sen­a­tors called on the US Patent and Trade­mark Of­fice to at­tack phar­ma patent thick­ets be­fore they start. The let­ter points to these patent thick­ets, Specif­i­cal­ly, the law­mak­ers ask that the PTO con­sid­er changes to its reg­u­la­tions and prac­tices “to ad­dress these prob­lems where they start, dur­ing ex­am­i­na­tion” in the pre-is­suance stage.

MAR­KET­INGRX

• The new GSK — which, for the first time in its 300-year his­to­ry, is no longer in the con­sumer health­care busi­ness — has a new look to go with its new strat­e­gy and brand iden­ti­ty. End­points Mar­ket­ingRx ed­i­tor Beth Bu­lik goes be­hind the scenes to shed light on how the com­pa­ny went to its em­ploy­ees to de­fine its new cul­ture.
• The FDA’s Of­fice of Pre­scrip­tion Drug Pro­mo­tion slapped its third un­ti­tled let­ter of the year on­to New Jer­sey-based Al­thera Phar­ma­ceu­ti­cals, say­ing the biotech’s DTC ad — which came in the form of a pam­phlet — cre­at­ed a mis­lead­ing im­pres­sion about the safe­ty pro­file of its cho­les­terol drug Roszet (ro­su­vas­tatin and eze­tim­ibe) and com­bined study re­sults in an un­ortho­dox way.
• More than 3,000 Lati­nos in the US are liv­ing with cys­tic fi­bro­sis, but many of them don’t fit the mold of a typ­i­cal pa­tient. Roche’s Genen­tech is hop­ing to change that and dri­ve aware­ness in the Lati­no com­mu­ni­ty with its new “So­mos La Gran Fa­mil­ia FQ” cam­paign. Work­ing with the Bon­nell Foun­da­tion, Genen­tech cre­at­ed a CF hub web­site specif­i­cal­ly for Lati­no fam­i­lies, pa­tients and care­givers.
• J&J’s Janssen am­bas­sadors liv­ing with HIV are the “Pos­i­tive­ly Fear­less” cam­paign ad­vo­cates the com­pa­ny hopes will lead the way for oth­ers. As part of the next it­er­a­tion, Janssen is plan­ning a me­dia tour with two of the am­bas­sadors, Daniel Garza and Ka­maria Laf­frey, talk­ing to TV and ra­dio sta­tions across the coun­try about the cam­paign and their lived ex­pe­ri­ences with HIV.
• CSL Behring is chron­i­cling the sto­ry of he­mo­phil­ia with pa­tients, care­givers, doc­tors and re­searchers through pho­tog­ra­phy — and the lens of renowned UK pho­tog­ra­ph­er Rankin. Best known for his dis­tinc­tive por­traits of fa­mous faces and fash­ion fig­ures, Rankin’s “Por­traits of Progress” se­ries fea­tures every­day peo­ple of the he­mo­phil­ia com­mu­ni­ty.
• As Ak­ili In­ter­ac­tive preps its AD­HD video game ther­a­py for launch, it’s tap­ping com­mer­cial vet­er­an Matt Franklin to lead the way. Franklin, whose most re­cent work is with di­ag­nos­tics com­pa­nies, will join Ak­ili as pres­i­dent and chief op­er­at­ing of­fi­cer with a fo­cus on the launch and mar­ket­ing of En­deav­or­Rx, its FDA-ap­proved cog­ni­tive ther­a­py for at­ten­tion deficit hy­per­ac­tiv­i­ty dis­or­der.
• Roche is throw­ing its sup­port be­hind blad­der can­cer aware­ness with a so­cial me­dia cam­paign to help ad­dress the stig­ma around the dis­ease; Su­per­nus high­lights how kids go from dis­tract­ed to fo­cused in its first TV ad for Qel­bree non-stim­u­lant AD­HD med; Big Phar­mas land on the an­nu­al list of best places to work for re­cent grad­u­ates; and more tid­bits await in the Mar­ket­ingRx roundup.

FDA+

• The House over­whelm­ing­ly passed a pack­age of leg­is­la­tion that will reau­tho­rize FDA user fee pro­grams — fund­ing agency re­views of drugs, med­ical de­vices, gener­ics and biosim­i­lars — as well as oth­er at­tached re­forms, set­ting up what is ex­pect­ed to be some heavy ne­go­ti­at­ing with the Sen­ate as the two sides have sev­er­al ma­jor dif­fer­ences.
• It’s been al­most 10 months since the FDA grant­ed Co­vis Phar­ma a hear­ing to re­view its con­tro­ver­sial preterm birth drug Mak­e­na, which won an ac­cel­er­at­ed ap­proval but failed its con­fir­ma­to­ry tri­al, and still the com­pa­ny and FDA are wran­gling over the de­tails of that hear­ing. In a new let­ter, Co­vis lawyers are now ob­ject­ing to the in­clu­sion of an un­fa­vor­able study.
• The EMA re­leased a new list of crit­i­cal med­i­cines to bat­tle the on­go­ing pan­dem­ic, which will put the mar­ket au­tho­riza­tion hold­ers for these Covid-19 vac­cines and drugs on the hook for reg­u­lar­ly up­dat­ing the EMA with da­ta on po­ten­tial or ac­tu­al short­ages and avail­able stocks, fore­casts of sup­ply and de­mand. Mem­ber states will al­so have to pro­vide reg­u­lar re­ports on es­ti­mat­ed de­mand on a na­tion­al lev­el.
• Mis­souri-based Nos­trum Phar­ma­ceu­ti­cals, once at the cen­ter of a Shkre­li-es­que gener­ic drug pric­ing scan­dal, just lost its chance to chal­lenge the FDA’s com­plete re­sponse let­ter for its asth­ma drug as a court ruled that CRLs aren’t tech­ni­cal­ly fi­nal re­jec­tions, cre­at­ing a “statu­to­ry bar­ri­er” to hear­ing Nos­trum’s pe­ti­tion.

MAN­U­FAC­TUR­ING

• As Pfiz­er is con­tin­u­ing to dou­ble down on its Paxlovid ef­forts, more room at its fa­cil­i­ty in Michi­gan will now be re­quired. The phar­ma will be com­mit­ting $120 mil­lion to its 1,300-acre man­u­fac­tur­ing fa­cil­i­ty in Kala­ma­zoo, MI to en­able US-based pro­duc­tion in sup­port of its Covid-19 oral an­tivi­ral, with an ex­pan­sion in the pro­duc­tion of API and RSM.
• Astel­las is the lat­est to pitch its tent in the small city of San­ford, NC. The com­pa­ny’s gene ther­a­py branch, which came out of the ac­qui­si­tion of Au­dentes in 2019, opened a new man­u­fac­tur­ing fa­cil­i­ty in the city, com­pris­ing 135,000 square feet and cost­ing about $100 mil­lion.
• CSL’s flu vac­cine-fo­cused sub­sidiary Se­qirus com­plet­ed a new fa­cil­i­ty ex­pan­sion at its site in Hol­ly Springs, NC, a sub­urb of Raleigh. The ex­pan­sion, which start­ed in 2018, cost the com­pa­ny $156 mil­lion and will add around 80 jobs to the area. It is de­signed to sup­port the for­mu­la­tion and fill-fin­ish of its cell-based flu vac­cines in pre-filled sy­ringes for glob­al dis­tri­b­u­tion.
• As it dou­bles down on man­u­fac­tur­ing, Sam­sung Bi­o­log­ics land­ed a con­tract with No­var­tis worth $81 mil­lion, ac­cord­ing to a reg­u­la­to­ry fil­ing. While the com­pa­nies are keep­ing most of the de­tails un­der wraps, the deal marks a siz­able por­tion of Sam­sung sales.
• Eli Lil­ly is be­ing brought to court in New Jer­sey by a for­mer em­ploy­ee who is seek­ing dam­ages af­ter act­ing as a whistle­blow­er. Am­rit Mu­la, a for­mer as­so­ciate di­rec­tor of em­ploy­ee re­la­tions at Lil­ly’s plant in Branch­burg, NJ — which has drawn the ire of the FDA and the DOJ — al­leged as she in­ves­ti­gat­ed com­plaints of vi­o­la­tions, Lil­ly kept telling her to stop and ha­rass­ing her.
• EQT’s pri­vate eq­ui­ty fund, EQT X, and Mubadala In­vest­ment Com­pa­ny, the in­vest­ment branch of the Unit­ed Arab Emi­rates, are mak­ing a big in­vest­ment in cold chain man­age­ment through their $3 bil­lion deal to buy En­vi­ro­tain­er, a tem­per­a­ture-con­trolled sup­ply chain provider for the trans­porta­tion of bio­phar­ma­ceu­ti­cals, from Cin­ven and No­vo Hold­ings.
• A Form 483 is shin­ing a light on what ex­act­ly got In­di­an drug man­u­fac­tur­er Au­robindo Phar­ma in trou­ble with the FDA. Dur­ing an in­spec­tion at its for­mu­la­tion plant, in­ves­ti­ga­tors found an un­ex­plained dis­crep­an­cy and a fail­ure of a batch of its com­po­nents to meet any of its spec­i­fi­ca­tions.
• The FDA’s in­spec­tion of Sun Phar­ma’s fa­cil­i­ty in Halol, In­dia, mean­while, raised sev­er­al con­cerns around san­i­ta­tion and im­prop­er con­trols, among oth­er in­frac­tions, ac­cord­ing to its Form 483. It’s not the first time Sun, or this par­tic­u­lar site, has run in­to prob­lems.
• Swiss man­u­fac­tur­er Lon­za ex­pand­ed its pres­ence in Bend, OR, an area more known for its out­door ameni­ties and the last re­main­ing Block­buster Video store. The ex­pan­sion adds to its small mol­e­cules fa­cil­i­ty, which al­so serves as the com­pa­ny’s cen­ter of ex­cel­lence for bioavail­abil­i­ty en­hance­ment and in­haled de­liv­ery for its small mol­e­cules busi­ness unit.

DON’T MISS

• Mod­er­na’s Cin­derel­la sto­ry — chang­ing the world, sav­ing lives while get­ting re­ward­ed with a gi­gan­tic val­u­a­tion with its Covid-19 vac­cine — is get­ting a scary rewrite on Wall Street. Fear­ing faster than ex­pect­ed rev­enue drop, a top an­a­lyst is point­ing to a “trans­for­ma­tive” BD deal as the way to go.
• The FTC is turn­ing up the heat on the drug in­dus­try’s mid­dle­men, launch­ing an in­ves­ti­ga­tion in­to phar­ma­cy ben­e­fit man­ag­er prac­tices and the ef­fect on drug prices. The move comes just a few months af­ter a dif­fer­ent study pro­pos­al failed to gain enough sup­port among the FTC com­mis­sion­ers. But with a re­vamped study and a new ap­pointee, the lat­est vote was unan­i­mous.
• When Pres­i­dent Joe Biden reignit­ed the Oba­ma-era Can­cer Moon­shot ini­tia­tive four months ago, sci­en­tists won­dered where the mon­ey would come from. The CDC is now dol­ing out the first in­stall­ment of a five-year, $1.1 bil­lion grant to get things go­ing, chan­nel the fund­ing through three na­tion­al pro­grams.
• About a month af­ter the mon­key­pox out­break emerged in Eu­rope, Bavar­i­an Nordic says it’s see­ing “over­whelm­ing” in­ter­est in its small­pox vac­cine Jyn­neos, in­clud­ing a new con­tract with Cana­da worth $56 mil­lion and an ex­pand­ed deal with the US. As it con­tin­ues talks with oth­er coun­tries, the Dan­ish com­pa­ny al­so up­dat­ed its 2022 sales fig­ures again.

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.