Astel­las taps sea­soned CMO amid shift to on­col­o­gy; Doug Man­ion steps in­to a new Are­na fol­low­ing $6.7B buy­out

Bern­hardt Zei­her

As Bern­hardt Zei­her re­tires from Astel­las’ chief med­ical of­fi­cer po­si­tion, the se­lec­tion of his suc­ces­sor says a lot about the changes he’s seen at the com­pa­ny over his 12-year tenure.

Tadaa­ki Taniguchi, the new CMO, is a sur­geon in on­col­o­gy by train­ing who’s led de­vel­op­ment for Mer­ck, Bris­tol My­ers Squibb and As­traZeneca in Japan be­fore mov­ing to the US, where he will be based. His most re­cent role at As­traZeneca was SVP, head of on­col­o­gy busi­ness unit Asia strat­e­gy.

Look­ing back on his time, Zei­her told End­points News that he be­lieves the suc­cess­es his group — span­ning al­most 2,700 peo­ple — has had with the cur­rent pipeline sets the com­pa­ny up for the next wave of can­cer ther­a­pies, be it bi­func­tion­al an­ti­bod­ies, cell ther­a­pies or on­colyt­ic virus­es. On top of notch­ing la­bel ex­pan­sions for the Pfiz­er-part­nered prostate Xtan­di, in re­cent years Astel­las al­so saw the ap­proval of Xospa­ta in acute myeloid leukemia and Seagen’s Pad­cev in urothe­lial can­cer.

“Prob­a­bly the biggest thing we’ve done as an or­ga­ni­za­tion is re­al­ly grow our on­col­o­gy port­fo­lio, ca­pa­bil­i­ties and re­al­ly es­tab­lished a strong pres­ence there,” he said.

When he first joined Astel­las from Pfiz­er — leav­ing as the in­flam­ma­to­ry dis­ease area lead — Zei­her re­called the com­pa­ny be­ing a “very re­gion­al com­pa­ny” with head­quar­ters in Japan and of­fices in Eu­rope and the US. His tenure, he would say, was al­so char­ac­ter­ized by a “great deal of glob­al­iza­tion,” which meant form­ing more glob­al struc­tures, har­mo­niz­ing pro­ce­dures and im­prov­ing ef­fi­cien­cies.

The com­pa­ny is al­so mov­ing in­to ear­li­er-stage projects and cut­ting-edge tech­nolo­gies such as gene ther­a­py. Ever since Astel­las ac­quired Au­dentes for $3 bil­lion, it’s been un­cov­er­ing safe­ty con­cerns with AAV gene ther­a­pies, with pa­tient deaths rock­ing the en­tire com­mu­ni­ty. Un­der Zei­her, re­searchers are still work­ing to find out the ex­act is­sues and how to re­solve them — some­thing Taniguchi will al­so have to pick up once he starts in Oc­to­ber.

“It’s been very much a roller coast­er,” Zei­her said, but added, “we re­main in­cred­i­bly com­mit­ted to gene ther­a­py.”

Am­ber Tong


Doug Man­ion

→ Ex-Kleo Phar­ma­ceu­ti­cals chief Doug Man­ion has turned up as pres­i­dent and COO of Aclaris, just a month af­ter the Wayne, PA im­muno-in­flam­ma­to­ry dis­ease biotech ap­point­ed In­ter­cept and Pfiz­er alum Gail Cawk­well as CMO. Man­ion was a Bris­tol My­ers ex­ec for 11 years be­fore tak­ing the Kleo job, and he led R&D at Are­na Phar­ma­ceu­ti­cals un­til Pfiz­er’s big $6.7 bil­lion pur­chase in De­cem­ber 2021. With Man­ion’s ar­rival, Neal Walk­er has re­lin­quished the ti­tle of pres­i­dent but will re­tain his CEO du­ties.

Olivi­er Bogillot

→ Strik­ing a €300 mil­lion on­col­o­gy deal on Thurs­day with In­novent to ex­pand its reach in Chi­na, Sanofi has made sev­er­al hires, start­ing with the ap­point­ment of Olivi­er Bogillot as head of US gen­er­al med­i­cines. Bogillot came to the French phar­ma from Bris­tol My­ers in 2015 and will fin­ish out his time as pres­i­dent of Sanofi France be­fore start­ing his new gig on Sept. 1.

Deb­o­rah Glass­er

Next, Deb­o­rah Glass­er be­came head of vac­cines, North Amer­i­ca at the multi­na­tion­al on June 20, break­ing away from Bio­gen af­ter al­most three years as US SVP of the Alzheimer’s fran­chise and 13 years over­all. Be­sides her work with the in­fa­mous ad­u­canum­ab, Glass­er was glob­al VP and as­set lead for Bio­gen’s SMA (Spin­raza) port­fo­lio. Fi­nal­ly, Kel­logg and Keurig Dr. Pep­per vet An­drew Loucks — the ex-CEO of Sen­so­ry Cloud — will be Sanofi’s head of North Amer­i­ca con­sumer health­care ef­fec­tive Aug. 8.

Per Nor­lén

Lars Staal Weg­n­er is out as CEO of Copen­hagen-based Evax­ion Biotech, and the im­munother­a­py play­er has wel­comed Per Nor­lén as his suc­ces­sor. Nor­lén left As­traZeneca in 2010 to be­come CMO of Al­li­ga­tor Bio­science and was el­e­vat­ed to CEO at the Swedish biotech sev­en years lat­er. He re­cent­ly jug­gled the roles of CMO at Xin­tela and CEO of its sub­sidiary Targin­ta. Weg­n­er’s res­ig­na­tion doesn’t mean he’s left the com­pa­ny for good: He’ll still be an ad­vi­sor at Evax­ion, part of the IPO class of 2021 whose shares $EVAX have tanked along with every­one else in the in­dus­try this year.

Mic­ah Mack­i­son

→ Dutch CNS biotech Vi­co Ther­a­peu­tics has poached Mic­ah Mack­i­son from Lo­can­abio, nam­ing him CEO af­ter his two-and-a-half-year tenure as CBO and EVP of strat­e­gy and cor­po­rate de­vel­op­ment. From 2015-20, he was SVP, cor­po­rate de­vel­op­ment and strat­e­gy at As­sem­bly Bio­sciences and he’s al­so led cor­po­rate strat­e­gy at Lund­beck. Ear­ly in his ca­reer, Mack­i­son held fi­nan­cial posts at Pfiz­er and Eli Lil­ly. Vi­co is tar­get­ing spin­ocere­bel­lar atax­ia types 3 and 1 and Hunt­ing­ton’s with its lead as­set, an an­ti­sense oligonu­cleotide named VO659.

John North­cott

→ When Nek­tar’s drug be­m­peg crum­bled in mul­ti­ple late-stage tri­als in com­bi­na­tion with Bris­tol My­ers’ Op­di­vo, it opened the flood­gates for lay­offs, as near­ly 70% of the work­force lost their jobs. Chief com­mer­cial of­fi­cer John North­cott ex­it­ed the com­pa­ny in June and has now resur­faced at Arv­inas in the same ca­pac­i­ty. North­cott, a Roche/Genen­tech vet as­signed to Avastin, has al­so been com­mer­cial chief with Lex­i­con Phar­ma­ceu­ti­cals and Phar­ma­cyclics be­fore mak­ing his way to Nek­tar in De­cem­ber 2019. In Ju­ly 20221, John Hous­ton-led Arv­inas inked an­oth­er pro­tein degra­da­tion deal with Pfiz­er worth $1 bil­lion in cash and up to $1.4 bil­lion in mile­stones.

Joanne Protano

Launch­ing last No­vem­ber with $50 mil­lion in fi­nanc­ing from Flag­ship, tR­NA biotech All­tr­na has se­lect­ed Joanne Protano as CFO. This marks an­oth­er ap­point­ment with­in Noubar Afeyan’s fam­i­ly of com­pa­nies for Protano, who was Flag­ship’s SVP of fi­nance, new ven­tures from 2015-17. Since then, she’s been VP of fi­nance and SVP of fi­nance and op­er­a­tions for Ru­bius Ther­a­peu­tics. Lo­visa Afzelius hand­ed the keys to Michelle Wern­er at All­tr­na in April, part of a wave of CEO-part­ner ap­point­ments at Flag­ship that in­clud­ed Mar­go Geor­giadis and Michael Sev­eri­no.

Wendy Li

Gra­cell Biotech­nolo­gies has made its sec­ond Peer Re­view ap­pear­ance in three weeks with Wendy Li as CMO. Li worked in on­col­o­gy de­vel­op­ment at Genen­tech, Sanofi and Pfiz­er, then held CMO posts at Si­huan Phar­ma­ceu­ti­cal and Ex­u­ma Biotech. Samuel Zhang, a Big Phar­ma vet in his own right, joined as CBO in Ju­ly at the Chi­nese CAR-T play­er, which opened 2021 by rais­ing a hefty $209 mil­lion in the good old days of IPOs.

Shuan­glian Li

→ With its lead drug tale­trec­tinib in Phase II stud­ies for ROS1 fu­sion-pos­i­tive NSCLC, US-Chi­na biotech An­Heart Ther­a­peu­tics has named Shuan­glian Li as CMO (US). Pri­or to lead­ing clin­i­cal de­vel­op­ment at An­sun Bio­phar­ma and Sky­line Ther­a­peu­tics, Li was glob­al clin­i­cal lead for the EGFR in­hibitor mobo­cer­tinib at Take­da and had been a med­ical di­rec­tor at Ari­ad Phar­ma­ceu­ti­cals un­til the Japan­ese phar­ma snapped it up five years ago in a $5.2 bil­lion deal.

→ Led by ex-No­var­tis Gene Ther­a­pies busi­ness chief Lisa De­schamps and notch­ing an $80 mil­lion Se­ries A last De­cem­ber, gene ther­a­py out­fit Avi­ado­Bio has tapped David Coop­er as CMO. Coop­er, a 14-year No­vo Nordisk vet in med­ical af­fairs, had filled the role of VP, clin­i­cal de­vel­op­ment, CNS since 2019 at uniQure and worked on such drugs as Hunt­ing­ton’s can­di­date AMT 130, which re­vealed ear­ly da­ta in late June that “pleas­ant­ly sur­prised” CEO Matt Ka­pus­ta.

Lih-Ling Lin

→ Mass­a­chu­setts-based Phar­maEssen­tia has brought aboard Lih-Ling Lin as CSO. Lin joins the com­pa­ny from Sanofi, where she served as head of check­point im­munol­o­gy clus­ter in the im­munol­o­gy and in­flam­ma­tion re­search area. Pri­or to that, Lin was with Pfiz­er for more than 21 years, round­ing out her tenure with the drug gi­ant as se­nior di­rec­tor, in­flam­ma­tion and im­munol­o­gy.

Lee Ann Mar­chion­na

→ Over at Feng Zhang’s Ar­bor Biotech­nolo­gies, Lee Ann Mar­chion­na has signed on as chief peo­ple of­fi­cer. Dur­ing her time as a prin­ci­pal at Vi­tal­i­ty HR, Mar­chion­na was ei­ther an in­ter­im chief peo­ple of­fi­cer or a frac­tion­al chief hu­man re­sources of­fi­cer for an ex­ten­sive list of com­pa­nies, in­clud­ing Al­bireo, Spero Ther­a­peu­tics, En­tra­da Ther­a­peu­tics, Ike­na On­col­o­gy and Geno­cea Bio­sciences. Ear­li­er, she was VP of hu­man re­sources at Ther­mo Fish­er. Now she fo­cus­es her at­ten­tion on a CRISPR biotech that gath­ered up a $215 mil­lion Se­ries B round in No­vem­ber 2021.

Er­ic Schlezinger

→ San Fran­cis­co pro­tein degra­da­tion shop Nurix Ther­a­peu­tics has locked in Er­ic Schlezinger as chief peo­ple of­fi­cer. Af­ter lead­ing hu­man re­sources at Vir Biotech­nol­o­gy from 2017-19, Schlezinger held the same role at Adamas Phar­ma­ceu­ti­cals, a Parkin­son’s biotech that Su­per­nus bought for $400 mil­lion in Oc­to­ber 2021. Nurix cashed in with a $120 mil­lion round in March 2020, then bol­stered its al­liance with Sanofi as the French phar­ma dished out $22 mil­lion near­ly 10 months lat­er on top of the $77 mil­lion from the orig­i­nal deal in 2019.

Kasey Rosa­do

→ Swing­ing and miss­ing again with its stem cell ther­a­py for stroke in June, Ather­sys has named Kasey Rosa­do as in­ter­im CFO short­ly af­ter share­hold­ers at the cash-strapped com­pa­ny signed off on a re­verse stock split. Rosa­do is a Price­wa­ter­house­C­oop­ers vet who was se­nior man­ag­ing di­rec­tor at Anku­ra be­fore join­ing Ather­sys, which sev­ered ties with As­pire Cap­i­tal Fund a month ago, rip­ping up a $100 mil­lion agree­ment.

Ali Siam

→ Sun­ny­vale, CA-based an­ti-ag­ing com­pa­ny Rube­do Life Sci­ences has plugged in Ali Siam as CBO and Ofir Moreno as SVP of drug dis­cov­ery. A J&J strate­gic mar­ket­ing vet who al­so had a three-year stay at Ab­b­Vie, Siam comes to Rube­do af­ter his stint as busi­ness chief of Cel­matix. Moreno has end­ed a near­ly 12-year as­so­ci­a­tion with MEI Phar­ma, where he was pro­mot­ed to SVP of phar­ma­ceu­ti­cal sci­ences in Ju­ly 2019.

Sin­gu­lar Ge­nomics Sys­tems is bring­ing in two new faces, both from 10x Ge­nomics, to its ex­ec team with the ap­point­ments of Sam Ropp as CCO and Jeff Bullard as head of sales for North Amer­i­ca. Dur­ing his tenure at 10x, Ropp was SVP of glob­al sales. Ropp al­so has ex­pe­ri­ence from Bec­ton Dick­in­son and Bio-Rad Lab­o­ra­to­ries un­der his belt.

Mean­while, Bullard was se­nior di­rec­tor of sales — Amer­i­ca. Pri­or to his decade-long stint with 10x, Bullard was with Ther­mo Fish­er, Life Tech­nolo­gies, Ion Tor­rent and Ap­plied Biosys­tems/Am­bion.

Ken Kuhn

→ Two Il­lu­mi­na vets have joined Seat­tle pro­teomics com­pa­ny Nau­tilus Biotech­nol­o­gy: Er­ic Spence (VP of in­stru­ment en­gi­neer­ing) spent four years with Il­lu­mi­na, then hopped over to Ag­i­lent Tech­nolo­gies be­fore tak­ing on a string of roles at Genap­sys, leav­ing the ge­nom­ic se­quenc­ing play­er as se­nior di­rec­tor, in­stru­ment de­vel­op­ment. Ken Kuhn (VP of reagent and plat­form de­vel­op­ment) was se­nior di­rec­tor of prod­uct de­vel­op­ment dur­ing a 16-year ca­reer at Il­lu­mi­na, and he’s re­cent­ly been VP of sys­tems in­te­gra­tion, prod­uct de­vel­op­ment and op­er­a­tions at En­co­dia.

→ Af­ter los­ing its CEO in Feb­ru­ary, biotech re­search group ReNeu­ron has locked in its CFO Cather­ine Ist­ed to the chief ex­ec role. Ist­ed joined the UK-based com­pa­ny in Oc­to­ber 2021 af­ter a stint at Ox­ford Bio­med­ica. Ist­ed will of­fi­cial­ly take the man­tle on Sept. 1.

Rolan­do Braw­er

Rolan­do Braw­er has tak­en on the role of EVP, strat­e­gy & cor­po­rate de­vel­op­ment for dig­i­tal cell bi­ol­o­gy spe­cial­ist Berke­ley Lights. Braw­er — a for­mer Ge­nom­ic Health/Ex­act Sci­ences ex­ec — re­cent­ly served as VP, sci­ence & tech­nol­o­gy, al­liances & ven­tures at Dana­her Cor­po­ra­tion, and from 2014-16, he was head of li­cens­ing at Ther­mo Fish­er.

Michael Drake

→ Right be­fore Am­gen agreed to buy Chemo­cen­tryx for $3.7 bil­lion, the Big Phar­ma gave Michael Drake a seat on the board of di­rec­tors. Since Au­gust 2020, Drake has been pres­i­dent of the Uni­ver­si­ty of Cal­i­for­nia and was pres­i­dent of Ohio State from 2014-20.

Gary Nabel and Joseph Pa­pa lead a group of new board mem­bers at Paul Pe­ter Tak’s Can­del Ther­a­peu­tics that will re­place Alan Smith, Shaan Ghan­di and Udi Meirav on Aug. 8. Nabel, the chief in­no­va­tion of­fi­cer at OP­KO and ex-Sanofi CSO, along with Pa­pa — the out­go­ing Bausch Health CEO — will be joined by Eko De­vices CFO Re­nee Gae­ta on the board.

Roshawn Blunt

→ Chaired by Mike Grey, mi­to­chon­dria-fo­cused Re­neo Phar­ma­ceu­ti­cals has elect­ed Roshawn Blunt to the board of di­rec­tors. Blunt, who joined the board at Kro­nos Bio in No­vem­ber 2021, is an Am­gen and J&J vet who is the man­ag­ing di­rec­tor of 1798 Con­sul­tants.

→ Af­ter VBL Ther­a­peu­tics’ ovar­i­an can­cer drug ofran­er­gene obade­n­ovec whiffed in Phase III a cou­ple weeks ago, the biotech chopped its staff by 35%, while Ron Co­hen, Ben­nett Shapiro and Al­i­son Fin­ger have all re­signed this week from the board of di­rec­tors. VBL says it has enough cash avail­able to keep go­ing with a small­er crew for the next year.

Lau­ra Bessen

An­drew Hirsch-led pro­tein degra­da­tion biotech C4 Ther­a­peu­tics has added Lau­ra Bessen and Don­na Gro­gan to the board of di­rec­tors. At the tail end of her 15 years with Bris­tol My­ers (2001-2016), Bessen was VP, head of US med­ical. Gro­gan was CMO of Clemen­tia un­til the com­pa­ny was sold to Ipsen, which has had a dev­il of a time with the cen­ter­piece of the deal, the rare bone dis­ease drug palo­varotene.

Rad­hi­ka Tripu­ra­neni is head­ed to the board of di­rec­tors at Eli Lil­ly’s NASH col­lab­o­ra­tor Terns Phar­ma­ceu­ti­cals. Tripu­ra­neni, the chief de­vel­op­ment of­fi­cer with Prothena, is the ex-VP of med­ical af­fairs for MyoKar­dia and Alex­ion.

Sap­na Sri­vas­ta­va

Fore­site Labs in­cu­ba­tor Alu­mis has brought on Sap­na Sri­vas­ta­va, the for­mer chief fi­nan­cial and strat­e­gy of­fi­cer of In­tel­lia Ther­a­peu­tics, to its board of di­rec­tors. Along with her stint at In­tel­lia, Sri­vas­ta­va is the ex-chief fi­nan­cial and strat­e­gy of­fi­cer for Abide Ther­a­peu­tics (ac­quired by Lund­beck).

→ New Jer­sey-based Am­neal Phar­ma­ceu­ti­cals has pulled up a chair for for­mer FDA deputy com­mis­sion­er Deb Au­tor on the board of di­rec­tors. Au­tor spent half of her six years at My­lan as head of glob­al qual­i­ty and the oth­er half as head of strate­gic glob­al qual­i­ty and reg­u­la­to­ry pol­i­cy. She was al­so As­traZeneca’s glob­al head of reg­u­la­to­ry ex­cel­lence from 2019-21.

Alessan­dra Ce­sano

Puma Biotech­nol­o­gy — which ex­pe­ri­enced a drop in its stock in 2019 af­ter sales of its drug Ner­l­ynx fell short of Wall Street es­ti­mates — has re­cruit­ed Alessan­dra Ce­sano to its board of di­rec­tors. Ce­sano serves as CMO of ES­SA Phar­ma­ceu­ti­cals and is the for­mer CMO of NanoS­tring. Ce­sano has al­so pre­vi­ous stints at Cleave Bio­sciences, Nodal­i­ty, Am­gen, Bio­gen and SmithK­line Beecham.

→ Cana­di­an bio­phar­ma Find Ther­a­peu­tics — backed by in­vestors CTI Life Sci­ences and ad­Mare BioIn­no­va­tions, among oth­ers — has named Paul Truex as chair­man of the board, tak­ing over for Lau­rence Rul­leau, who will re­main on the board. Truex cur­rent­ly serves as chair­man and CEO of Thryv Ther­a­peu­tics. Truex al­so has ex­pe­ri­ence from stints at Mile­stone, Trius, An­thera, Penin­su­la and Ver­si­cor.

Rally­bio, run by a crew of Alex­ion alums who picked up an old Kymab ane­mia drug from Sanofi in May, has plucked up Wendy Chung for its board of di­rec­tors. Chung is a pro­fes­sor of pe­di­atrics and med­i­cine at Co­lum­bia Uni­ver­si­ty Irv­ing Med­ical Cen­ter and al­so serves on the board of Prime Med­i­cine. Ad­di­tion­al­ly, Chung sits on the sci­en­tif­ic ad­vi­so­ry boards of Sage Bionet­works, Taysha, He­lix and Re­gen­eron Ge­net­ics Cen­ter.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.

BREAK­ING: Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck 5 years ago, where the soft spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.

No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.

FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Bernhardt Zeiher, outgoing Astellas CMO (Astellas)

Q&A: Astel­las' re­tir­ing head of de­vel­op­ment re­flects on gene ther­a­py deaths

For anyone who’s been following discussions about the safety alarms surrounding the adeno-associated viruses (AAV) commonly used to deliver gene therapy, Astellas should be a familiar name.

The Japanese pharma — which bought out Audentes Therapeutics near the end of 2019 and later built a gene therapy unit around the acquisition — rocked the field when it reported three patient deaths in a trial testing AT132, the lead program from Audentes designed to treat a rare muscle disease called X-linked myotubular myopathy (XLMTM).

When the company restarted the trial, it adjusted the dose and instituted a battery of other measures to try to prevent the same thing from happening again. But tragically, the first patient to receive the new regimen died just weeks after administration. The therapy remains under clinical hold, and just weeks ago, Astellas flagged another safety-related hold for a separate gene therapy candidate. In the process of investigating the deaths, the company has also taken flak about the way it disclosed information.

Big questions remain — questions that can have big implications about the future of AAV gene therapies.

Bernhardt Zeiher did not imagine any of it when he first joined Astellas as the therapeutic area leader in inflammation, immunology and infectious diseases. But his ascent to chief medical officer and head of development coincided almost exactly with Astellas’ big move into gene therapy, putting him often in the driver’s seat to grapple with the setbacks.

As Zeiher prepares to retire next month after a 12-year tenure — leaving the unfinished tasks to his successor, a seasoned cancer drug developer — he chatted with Endpoints News, in part, to discuss the effort to understand what happened, lessons learned and the criticism along the way.

The transcript has been lightly edited for length and clarity.

Endpoints: I want to also ask you a bit about the gene therapy efforts you’ve been working on. Astellas has really been at the forefront of discovering the safety concerns associated with AAV gene therapy. What’s that been like for you?

Zeiher: Well, I have to admit, it’s been a bit of a roller coaster. We acquired Audentes. Huge amount of enthusiasm. What we saw with AT132 — that was the lead program in XLMTM — was just remarkable efficacy. I mean, kids who went from being on ventilators, not able to eat for themselves, sit up, do things like that, to off ventilators, walking, you know, really — one investigator called it this Lazarus-like effect. It was just really dramatic efficacy. And then to have the safety events that occurred. So they actually occurred within that first year of the acquisition. So we had the three patient deaths. Me and my organization became very, very much involved. In fact, Ed Conner, who had been the chief medical officer, he left after some of the deaths, but I stepped in as the kind of acting chief medical officer, we had another chief medical officer who was involved, and then we had a fourth death, and I became acting again for a period of time.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.