WIB22: Hear from our 2022 Women in Bio­phar­ma R&D hon­orees, and catch our pan­el on em­pow­er­ing women

If we’ve learned any­thing from our an­nu­al Women in Bio­phar­ma R&D re­port over the last four years, it’s that there’s much to cel­e­brate.

We re­ceived more than 500 nom­i­na­tions this year high­light­ing ex­tra­or­di­nary women who are blaz­ing trails in drug de­vel­op­ment. While se­lect­ing just 20 hon­orees has be­come in­creas­ing­ly dif­fi­cult, we were thrilled to de­but this year’s group with you last week. They’re sci­en­tists, CEOs, re­searchers and more who have de­vel­oped new drugs for rare and life-threat­en­ing dis­eases, changed the way we think about com­mon con­di­tions such as mi­graine, and bro­ken bound­aries.

Our team spent time with each hon­oree, craft­ing pro­files which you’ll find in our spe­cial re­port pub­lished last week. You can al­so catch a glimpse of those con­ver­sa­tions in the video clips be­low. For the third year, we in­vit­ed a pan­el of ex­perts to dis­cuss the lim­i­ta­tions of ex­ist­ing gen­der di­ver­si­ty ef­forts, and how the in­dus­try can do bet­ter. If you didn’t get a chance to see it live, you can re­play this year’s en­tire show here:

We ap­plaud this year’s hon­orees, and thank our read­ers for help­ing us con­tin­ue the tra­di­tion. We’ll see you next year.

Am­ber Tong and Nicole De­Feud­is

Lo­visa Afzelius: Raised in Swe­den, Lo­visa Afzelius be­gan her ca­reer as a com­pu­ta­tion­al chemist at As­traZeneca. In those ear­ly days, she re­mem­bers writ­ing her own al­go­rithms, run­ning her own ex­per­i­ments and de­vel­op­ing the in­ter­faces to vi­su­al­ize the re­sults — an ex­pe­ri­ence that in­grained in her the im­por­tance of un­der­stand­ing bi­ol­o­gy in or­der to de­sign the best com­pu­ta­tion­al tools for mak­ing drugs. Af­ter be­ing re­cruit­ed to lead im­munol­o­gy at Pfiz­er, Afzelius made the leap to the start­up world. She is now the orig­i­na­tion part­ner at Flag­ship Pi­o­neer­ing, launch­ing and lead­ing com­pa­nies push­ing for­ward da­ta-dri­ven drug dis­cov­ery.

Sheena Au­ro­ra: When Sheena Au­ro­ra was young, mi­graines were con­sid­ered a dis­or­der suf­fered by hys­ter­i­cal women. But she was among a group of re­searchers to first cap­ture and show that painful episodes of mi­graine with au­ra hap­pen be­cause of an un­der­ly­ing phys­i­o­log­i­cal event, a sem­i­nal find­ing. She went on to lead key tri­als that es­tab­lished Botox as a treat­ment for mi­graine, help launch Eli Lil­ly’s CGRP in­hibitor Em­gal­i­ty and, in her cur­rent role, lead Im­pel Phar­ma­ceu­ti­cals’ nasal­ly ad­min­is­tered Trud­he­sa. And she’s still work­ing to des­tig­ma­tize the dis­ease.

Sue Dil­lon: Sue Dil­lon, the for­mer im­munol­o­gy chief at John­son & John­son’s Janssen, helped run one of the biggest shows in im­munol­o­gy, work­ing on such well-known drugs as Rem­i­cade, Sim­poni, Ste­lara and Trem­fya. But af­ter more than 16 sto­ried years at the phar­ma gi­ant, Dil­lon joined up with a for­mer col­league, Karyn O’Neil, to launch Aro Bio­ther­a­peu­tics around a new tech­nol­o­gy for ge­net­ic med­i­cine. De­scribed by col­leagues as a pur­pose-dri­ven leader, Dil­lion cred­its her ac­com­plish­ments to the phe­nom­e­nal teams she’s built along the way, from dis­cov­ery to de­vel­op­ment and back.

Ruxan­dra Draghia-Ak­li: Through­out her ca­reer, Ruxan­dra Draghia-Ak­li has been fo­cused on chang­ing the sys­tem. Af­ter her stud­ies of ge­net­ic sci­ence took her from Ro­ma­nia to France to the US, she was in­spired to join the Eu­ro­pean Com­mis­sion, where she helped launch a pub­lic-pri­vate part­ner­ship to kick­start new re­search. Then in­dus­try came call­ing and she joined Mer­ck as vice pres­i­dent of glob­al vac­cines, be­fore jump­ing to J&J to be­come its head of glob­al pub­lic health R&D, launch­ing re­search cen­ters around the world to ad­dress big chal­lenges.

Ruth Gi­meno: Across her 26-year ca­reer, meta­bol­ic dis­ease has been the dom­i­nant theme for Ruth Gi­meno, cur­rent­ly group vice pres­i­dent of di­a­betes, obe­si­ty and car­diometa­bol­ic re­search at Eli Lil­ly. She ini­tial­ly fell in love with re­search as an un­der­grad­u­ate stu­dent in the UK, then moved to the US from her na­tive Ger­many for a post­doc and joined Mil­len­ni­um in her first in­dus­try job, fol­lowed by years at Pfiz­er be­fore mov­ing to Lil­ly. The type 2 di­a­betes drug Moun­jaro, grad­u­at­ed out of Gi­meno’s purview, is now in Phase III for obe­si­ty, but Gi­meno and her team are al­ready look­ing ahead to how they can do even bet­ter.

Jen­nifer Gor­don: Jen­nifer Gor­don’s first in­ter­view af­ter she grad­u­at­ed from col­lege in the ear­ly ‘90s was the on­ly one she need­ed to set her on a decades-long mis­sion of bring­ing one of the first CRISPR-based treat­ments in­to the clin­ic for HIV. Af­ter meet­ing with the sci­en­tif­ic founder of Ex­ci­sion in that fate­ful in­ter­view, she spent 30 years in acad­e­mia as an NIH-fund­ed in­ves­ti­ga­tor at Tem­ple Uni­ver­si­ty. About two years ago, she made the big leap to be­come se­nior vice pres­i­dent of R&D at Ex­ci­sion, which re­cent­ly be­gan a hu­man tri­al.

Ai­da Habtezion: Ai­da Habtezion’s path to one of the top jobs in all of bio­phar­ma was not a tra­di­tion­al one. From a young age, she had want­ed to be a pro­fes­sor. She re­al­ized her dream and earned tenure at Stan­ford teach­ing med­i­cine, but al­so wore mul­ti­ple hats, run­ning both a trans­la­tion­al lab and clin­i­cal prac­tice. While she had con­tem­plat­ed a ca­reer change for a while, a call in the mid­dle of the Covid-19 pan­dem­ic, when Pfiz­er was still test­ing its vac­cine, was what ul­ti­mate­ly drew her to phar­ma. From her C-suite perch as Pfiz­er’s chief med­ical of­fi­cer, Habtezion is mak­ing health eq­ui­ty a key part of her mis­sion.

Jian Irish: Grow­ing up dur­ing the Cul­tur­al Rev­o­lu­tion in Chi­na, Jian Irish re­mem­bered the per­son­al re­la­tion­ships her dad, a sur­geon, de­vel­oped with the pa­tients he treat­ed. While she end­ed up tak­ing a dif­fer­ent ca­reer path, her love for med­i­cine did not abate. Af­ter over­see­ing on­col­o­gy op­er­a­tions and man­u­fac­tur­ing at Am­gen, Irish held a stint at Sanofi be­fore get­ting re­cruit­ed to run Kite Phar­ma’s sup­ply chain and man­u­fac­tur­ing as the cell ther­a­py pi­o­neer pre­pared to launch the CAR-T Yescar­ta. As pres­i­dent and COO at Metageno­mi, she is push­ing new bound­aries.

Louise Kir­man: From No­var­tis to start­up biotech, Louise Kir­man is look­ing to bring a “par­a­digm shift” to dif­fi­cult can­cer tar­gets that some would call un­drug­gable. Join­ing the Swiss phar­ma gi­ant as a med­i­c­i­nal chemist straight out of school, she worked on a range of projects, start­ing with PI3K in­hibitors at a time she was told ki­nase in­hibitors would nev­er get to mar­ket. And al­though not hav­ing a PhD has at times been a chal­lenge, she did not let it stop her from land­ing a di­rec­tor post at Cedil­la Ther­a­peu­tics, a pre­ci­sion on­col­o­gy com­pa­ny, and lead­ing its top breast can­cer pro­gram.

Suma Kr­ish­nan: Af­ter spend­ing time with pa­tients who were di­ag­nosed with what some call “the worst dis­ease you’ve nev­er heard of,” Suma Kr­ish­nan ded­i­cat­ed her­self to find­ing a treat­ment. Kr­ish­nan, who is the co-founder and chief op­er­at­ing of­fi­cer of Krys­tal Biotech, spear­head­ed a gene ther­a­py gel that showed promise in a late-stage clin­i­cal tri­al for a rare ge­net­ic skin dis­or­der called dy­s­troph­ic epi­der­mol­y­sis bul­losa. The FDA is re­view­ing whether to ap­prove the ther­a­py and will de­cide by Feb­ru­ary.

Fiona Mar­shall: Like many sci­en­tists, Fiona Mar­shall would tell you that she loved the nat­ur­al world grow­ing up. But bi­ol­o­gy stood out be­cause the sci­ence was chang­ing even as she learned it. A world-renowned ex­pert in G pro­tein-cou­pled re­cep­tors who spent for­ma­tive years at GSK, Mar­shall co-found­ed Hep­tares Ther­a­peu­tics to dis­cov­er GPCR drugs and went on to take up the big re­spon­si­bil­i­ty of re­build­ing Mer­ck’s re­search pres­ence in the UK. And she is still hop­ing to stay ahead of the tech­nol­o­gy as the new pres­i­dent of the No­var­tis In­sti­tutes for Bio­Med­ical Re­search.

Sab­ri­na Mar­tuc­ci John­son: Sab­ri­na Mar­tuc­ci John­son is out to build a new kind of women’s health com­pa­ny. While it was ini­tial­ly chal­leng­ing to con­vince in­vestors, her start­up, Daré Bio­science, played a ma­jor part in at­tract­ing more in­dus­try at­ten­tion and dol­lars to women’s health. The port­fo­lio mod­el aims to take prod­ucts from in­no­va­tion to de­vel­op­ment, in con­trast to what John­son saw as frag­men­ta­tion in the mar­ket. Al­though fi­nan­cial chal­lenges re­main, her ef­forts are be­gin­ning to trans­late in­to ap­proved med­i­cines.

Chris­tel Menet: De­spite be­ing one of the most pop­u­lar tar­gets in drug de­vel­op­ment, G pro­tein-cou­pled re­cep­tors re­main rather per­plex­ing. Chris­tel Menet was hooked on these re­cep­tors at Faust Phar­ma­ceu­ti­cals, where she got her first ex­pe­ri­ence de­sign­ing drugs, and lat­er joined Gala­pa­gos as one of its first chemists. It was un­der her lead­er­ship that the JAK in­hibitor fil­go­tinib, now ap­proved in the UK and EU, en­tered pre­clin­i­cal tri­als. As chief sci­en­tif­ic of­fi­cer at Con­fo Ther­a­peu­tics, she’s re­turn­ing to GPCR re­search, with the new chal­lenge of find­ing un­chart­ed tar­gets.

Jane Parnes: Jane Parnes had a big de­ci­sion to make in the sum­mer of 2007. She could re­turn to the pro­fes­sor job she had held for 24 years, or she could ex­tend her stay at Am­gen, where she had tak­en on a role of ex­ec­u­tive med­ical di­rec­tor of ear­ly de­vel­op­ment. Fas­ci­nat­ed with the trans­la­tion­al work she was do­ing, she made the leap to in­dus­try. Over the next 15 years, she de­signed ear­ly stud­ies and played a piv­otal role in the de­vel­op­ment of an an­ti­body that was re­cent­ly ap­proved by the FDA for se­vere asth­ma.

Katy Rez­vani: In a small but wide­ly-cit­ed 11-per­son study, sev­en pa­tients saw signs of their can­cer com­plete­ly go away af­ter get­ting a new ther­a­py made from nat­ur­al killer cells, some of the ear­li­est clin­i­cal proof for this nascent tech­nol­o­gy. Katy Rez­vani, whose lab at MD An­der­son de­vel­oped that ther­a­py and lat­er li­censed it to Take­da, pushed through ear­ly skep­ti­cism to val­i­date the ap­proach. Up­root­ed at a young age when her fam­i­ly fled Iran, she plant­ed roots in the UK and now Texas, men­tor­ing the next gen­er­a­tion of sci­en­tists along the way.

Am­ber Salz­man: A for­mer IT ex­ec at GSK, Am­ber Salz­man dove in­to gene ther­a­py de­vel­op­ment af­ter her son was di­ag­nosed with a rare and of­ten fa­tal ge­net­ic con­di­tion. Af­ter years of co­or­di­nat­ing re­search ef­forts and clear­ing hur­dles, the re­sult­ing gene ther­a­py snagged an ac­cel­er­at­ed ap­proval. In par­al­lel with be­ing a par­ent and ad­vo­cate, Salz­man is a sea­soned ex­ec­u­tive who led mul­ti­ple biotechs, in­clud­ing epi­ge­net­ic-fo­cused Epic Bio, where she is now CEO.

Lau­ra Sepp-Loren­zi­no: Lau­ra Sepp-Loren­zi­no’s med­ical ed­u­ca­tion start­ed in her moth­er’s phar­ma­cy in 1960s Buenos Aires. Af­ter ex­per­i­ment­ing with every­thing from small mol­e­cule can­cer drugs to vac­cines, from RNA in­ter­fer­ence to nu­cle­ic acid ther­a­pies, she ar­rived at CRISPR gene edit­ing biotech In­tel­lia, head­ing up sci­ence strat­e­gy and re­search and ear­ly de­vel­op­ment.

Effie Toz­zo: Born in Greece, ed­u­cat­ed in France and a fol­low­er of the US Big Phar­ma-to-biotech ex­ec­u­tive path, Effie Toz­zo chan­nels her in­stincts as a sailor in her sci­en­tif­ic lead­er­ship at Avi­lar Ther­a­peu­tics, which bets that it can take pro­tein degra­da­tion to the next lev­el. Crit­i­cal to both Avi­lar and sail­ing is the art of com­mu­ni­ca­tion, es­pe­cial­ly as drug R&D amounts to a se­ries of set­backs that re­quire solv­ing a prob­lem to get to the next stage. Through­out her ca­reer, Toz­zo has ini­ti­at­ed and steered dozens of drug dis­cov­ery pro­grams to the clin­ic

Bar­bara Wirostko: An oph­thal­mol­o­gist by train­ing, Bar­bara Wirostko was in­spired at a young age by her fa­ther, al­so an oph­thal­mol­o­gist, who worked from home. She went to Cor­nell and stud­ied mi­cro­bi­ol­o­gy be­fore at­tend­ing Co­lum­bia’s med­ical school. While Wirostko’s re­sume boasts po­si­tions at a hand­ful of phar­ma and biotech com­pa­nies, in­clud­ing Pfiz­er, Al­theos, Jade Ther­a­peu­tics and Eye­Gate Phar­ma­ceu­ti­cals, she’s cur­rent­ly chief med­ical of­fi­cer at Qlaris Bio, a com­pa­ny she co-found­ed to de­vel­op nov­el ther­a­pies for se­ri­ous eye dis­eases.

Lili Yang: Our fi­nal hon­oree is Lili Yang. As a child of en­gi­neers, Yang’s love for sci­ence is root­ed in her back­ground. As a co-founder of Ap­pia Bio, which spun out of her UCLA lab, Yang and a team of in­dus­try ex­perts are look­ing to re-en­gi­neer a rare type of im­mune cell to con­quer ma­lig­nant tu­mors. The team in­cludes some big names, such as No­bel Lau­re­ate David Bal­ti­more, who’s worked with Yang since her PhD days back in 1999. If she could give ad­vice to young women en­ter­ing the field, she would tell them not to lim­it them­selves, and to think big.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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