Dorothee Kern’s path from bas­ket­ball star to founder of bil­lion-dol­lar biotech(s?); Five Prime Ther­a­peu­tics hands the reins to Genen­tech vet

I as­sure you that I did not plan to do this. I want­ed to write a lit­tle sto­ry about pro­teins and can­cer, nu­clear mag­net­ic imag­ing and a sci­en­tist turned en­tre­peneur of bil­lion-dol­lar biotechs. Maybe a touch on com­mu­nist East Ger­many. A sprin­kle on her (what I as­sumed had been) past life as a world-class ath­lete. I did not — would not — plan on writ­ing a sto­ry about some­one suc­ceed­ing “on-the-court and off-the-court,” a sto­ry held to­geth­er by what I as­sumed would have to be forced par­al­lels be­tween bas­ket­ball and sci­ence.

And then I spoke to Dorothee Kern, and the first thing she told me to ex­plain her work was a bas­ket­ball anal­o­gy, one about her own game. And when I fol­lowed up, ask­ing about any con­nec­tion be­tween bas­ket­ball and why she got in­to biotech, her sec­ond re­sponse is: “I want to win and cure pa­tients … there’s this com­pet­i­tive spir­it, but you want to win the cham­pi­onship, you have to get drugs on the mar­ket.”

The first re­sponse was about an anal­o­gy be­tween the pro­teins she watch­es be­ing in mo­tion and she her­self be­ing in con­stant mo­tion. “Shoot­ing for the win” was al­so men­tioned.

“There’s a rea­son they call me the pro­tein dy­nam­ics la­dy!” she said.

Bas­ket­ball is in­deed about as fun­da­men­tal to Kern as her sci­ence. She calls the sport “her ATP” — and she has a lot of ATP, her frame built like an ath­lete and her words spo­ken with the fre­net­ic en­er­gy be­fit­ting of some­one who goes by “Doro.”

The com­pe­ti­tion, I learned, nev­er ac­tu­al­ly be­came a thing of the past. This sum­mer she had planned to re­turn with the Ger­man team to the FIM­BA Max­ibas­ket­ball Eu­ro­pean Cham­pi­onship to de­fend their ti­tle. Covid-19 end­ed that, so for ex­cite­ment, Kern, a pro­fes­sor of bio­chem­istry at Bran­deis Uni­ver­si­ty, will have to set­tle for the launch of her sec­ond biotech: MO­MA Ther­a­peu­tics, which emerged this week out of Third Rock with an $86 mil­lion Se­ries A and a fo­cus on a ver­sa­tile class of pro­teins called mol­e­c­u­lar ma­chines. Her first biotech, found­ed in 2016, had emerged with a $57 mil­lion Se­ries A and lat­er at­tract­ed a $400 mil­lion Soft­bank-led Se­ries C.

The new one, she said, will be even big­ger. Or at least the sci­ence will be.

“I com­pare these mol­e­c­u­lar ma­chines and MO­MA to a pow­er of 3 to what has been done so far,” Kern said. “No­body has done this. It’s on a whole dif­fer­ent scale.”

Dorothee Kern dri­ving up the court

Kern grew up in com­mu­nist East Ger­many, the daugh­ter of two high-mind­ed bio­chemists who con­sid­ered bio­chem­istry a din­ner-ta­ble top­ic of choice and who re­fused to co­op­er­ate with the com­mu­nists or their se­cret po­lice, the Stasi. That rec­ti­tude would cost her fa­ther a pro­mo­tion and her moth­er her job and, with high school re­served for par­ty loy­al­ists, could have cost Kern her ed­u­ca­tion. Ex­cept that Kern had just made the ju­nior na­tion­al bas­ket­ball team. Her par­ents con­vinced the gov­ern­ment that was a pub­lic ser­vice, one wor­thy of an ex­cep­tion.

Bas­ket­ball pro­vid­ed a way in­to the sys­tem. Sci­ence, Kern said, pro­vid­ed a refuge from that sys­tem, from a dic­ta­to­r­i­al gov­ern­ment that wrote the text­books to suit what­ev­er ver­sion of events they thought ex­pe­di­ent. For a nat­u­ral­ly cu­ri­ous stu­dent, it was the on­ly field that looked like it could pro­vide re­al an­swers.

“Every­thing was twist­ed, there was no truth be­ing told in all the oth­er sub­jects: his­to­ry, eco­nom­ics and busi­ness, the hu­man­i­ties,” Kern said. “The one thing they couldn’t twist was 2+2=4.”

One day in 1987, fu­ture No­ble Prize win­ner Kurt Wüthrich gave a lec­ture at the Leopold­ina, a cen­turies-year old learned so­ci­ety where East Ger­man sci­en­tists could still hear vis­it­ing lec­tures from the West. At the time, X-ray crys­tal­log­ra­phy — the same tech­nique that led to the dis­cov­ery of the dou­ble he­lix — had be­come the hottest thing in bio­chem­istry, with jour­nal af­ter jour­nal plas­ter­ing their front pages with new im­ages of mol­e­cules, black-and-white still lifes of the chem­i­cal world. This was ex­cit­ing but it al­so struck Kern as fun­da­men­tal­ly lack­ing.

“The whole beau­ty of en­zymes is that they ac­tu­al­ly move,” Kern said. “And if they don’t move they are dead. There’s a rea­son life doesn’t ex­ist be­low 180 kelvin.”

In a brief aside, Wüthrich men­tioned nu­clear mag­net­ic res­o­nance spec­troscopy, a dif­fer­ent tech­nique he had de­vel­oped to take pic­tures of atoms. Sit­ting in the au­di­ence, Kern re­al­ized she could use the same tech­niques to take pic­tures of mol­e­cules and specif­i­cal­ly pro­teins in mo­tions, what she calls “re­al-time movies.”

East Ger­many, though, lacked the tools need­ed for these ex­per­i­ments. They were like kids’ toys, she said, in com­par­i­son to West­ern equip­ment, and ear­ly on she could on­ly study sub­strates, lit­tle shards off the over­all whole. Then the Berlin Wall fell. She be­came a fel­low in Swe­den, a post-doc in Berke­ley. She be­gan to make her movies, il­lu­mi­nat­ing in­vis­i­ble par­ti­cles that moved on mil­lisec­ond scales.

“It was a dream come true,” she said. She com­pared it to go­ing from pick­up bas­ket­ball to the world cham­pi­onships.

(Kern, mean­while, had nev­er giv­en up bas­ket­ball, play­ing on the Ger­man na­tion­al team un­til she left and con­tin­u­ing to school guys half her age in Boston five days a week. I asked her if she ever had thought about choos­ing one or the oth­er, point­ing out that the time com­mit­ments of be­ing a grad­u­ate stu­dent or sci­en­tist and a na­tion­al bas­ket­ball play­er. “Ab­solute­ly not,” she said. “I get bored quick­ly. I love mul­ti-task­ing.”)

Her mo­tion work would bring ac­co­lades and a pro­fes­sor­ship at Bran­deis. Even­tu­al­ly, be­tween 2012 and 2014, she start­ed to learn how to link those pro­teins’ move­ment with their func­tion. She be­gan to study al­losteric sites — spots of a pro­tein away from the bind­ing site (where most drugs are tar­get­ed) that can nonethe­less af­fect the over­all func­tion of the mol­e­cule. It was the kind of how-things-work un­der­stand­ing her en­tire ca­reer had been build­ing to.

It was al­so the kind of in­for­ma­tion that could be used to build drugs. Kern, who had al­ways been too con­cerned with dis­cov­er­ing how things work to go in­to biotech, booked a meet­ing with Third Rock Ven­tures.

Be­fore a dozen ex­ec­u­tives, she pulled out her lap­top and played La­dy Gaga’s “Just Dance” while the lit­tle dots in a pro­tein di­a­gram pulsed to the beat. She of­fered no fi­nan­cial or rev­enue pro­jec­tions. Nev­er­the­less, she pro­posed a biotech that would chan­nel her 30 years of re­search on pro­tein mo­tion in­to treat­ments for can­cer and neu­rode­gen­er­a­tive dis­eases. “Dance,” she called it, af­ter her ‘danc­ing’ pro­teins.

“Yes,” Third Rock said. And then, with Justin Tim­ber­lake-from-The Social Net­work flair: “Just change the name.”

Re­lay Ther­a­peu­tics now says they are on the verge of the clin­ic, al­though they have not re­vealed any tar­gets or drug can­di­dates and no “pipeline” is list­ed on their site. Work­ing there changed Kern. She still cared deeply about fun­da­men­tal dis­cov­er­ies, she said, but the po­ten­tial to help pa­tients was “eye-open­ing.”

The newest ven­ture, MO­MA, was a Third Rock idea. Kern ac­knowl­edges that of the found­ing team of her, Timur Yuzusafi, Eva No­gales and Jo­hannes Wal­ter, she has the least di­rect ex­pe­ri­ence with the sci­ence at the biotech’s cen­ter, but in many ways it builds off work she has done for decades. The new biotech fo­cus­es on mol­e­c­u­lar ma­chines — pro­teins that are unique in their size and their de­gree of move­ment, shape-shift­ing over and over again to car­ry out their tasks.

That size and nim­ble­ness al­so makes them dif­fi­cult to study. Nev­er mind crys­tal­log­ra­phy, that old tech­nique that came in vogue as Kern was an un­der­grad. Many of these pro­teins don’t crys­tal­lize. How to build, screen and in­hib­it them are still open ques­tions.

Those as­pects are al­so what make them so ex­cit­ing. They do so much, and that opens up a world of po­ten­tial drugs.

“Where they are in­volved is just mind-bog­gling,” Kern said. “You could build a large phar­ma com­pa­ny around this go­ing for­ward.”

Ja­son Mast


→ Af­ter re­cent­ly strik­ing a $350 mil­lion deal with Bio­gen to work on Alzheimer’s and with their first prod­uct can­di­date soon en­ter­ing a Phase III tri­al, ge­nom­ic med­i­cine com­pa­ny Sang­amo Ther­a­peu­tics has wooed GSK vet D. Mark Mc­Clung as EVP and CBO. Pri­or to hop­ping aboard Sang­amo, Mc­Clung served as VP and gen­er­al man­ag­er of glob­al on­col­o­gy com­mer­cial at Am­gen. Mc­Clung joined Am­gen from Onyx Phar­ma­ceu­ti­cals, where he was CCO. Dur­ing his two decades at GSK, Mc­Clung served in var­i­ous roles in­clud­ing VP and head of glob­al com­mer­cial for GSK On­col­o­gy.

In ad­di­tion, Stephane Bois­sel, the com­pa­ny’s EVP of cor­po­rate strat­e­gy, will be hit­ting the ex­it at the end of Ju­ly, re­turn­ing to an en­tre­pre­neur­ial project. Bois­sel joined the com­pa­ny in 2018 af­ter serv­ing as CEO of Tx­Cell (now Sang­amo France).

Tom Civik

Five Prime Ther­a­peu­tics, which has strug­gled through a pe­ri­od of tu­mult be­cause of lay­offs and set­backs with its drug cabi­ral­izum­ab, has brought on Tom Civik to be its CEO. He re­places William Ringo, who was named in­ter­im CEO in Sep­tem­ber af­ter the res­ig­na­tion of Aron Knicker­bock­er. An in­dus­try vet­er­an of more than a quar­ter-cen­tu­ry, Civik ar­rives at the Bay Area on­col­o­gy biotech from Foun­da­tion Med­i­cine, where he was CCO. Dur­ing his 17 years at Genen­tech, Civik com­mer­cial­ized such ther­a­pies as Avastin, Tarce­va, Tecen­triq and Ale­cen­sa as VP and fran­chise head.

Robert Dug­gan

As re­port­ed ear­li­er in the week, Bob is back. Bob Dug­gan will be CEO at Sum­mit Ther­a­peu­tics, ef­fec­tive im­me­di­ate­ly, while re­main­ing chair­man of the an­tibi­otics-fo­cused British biotech’s board. Glyn Ed­wards’ res­ig­na­tion paves the way for Dug­gan to take over, and as Sum­mit’s lead­ing share­hold­er, the bil­lion­aire en­tre­pre­neur won’t re­ceive com­pen­sa­tion. Dug­gan was pre­vi­ous­ly CEO of Phar­ma­cyclics, which he sold to Ab­b­Vie in 2015 for $21 bil­lion, and be­fore that was CEO at Com­put­er Mo­tion.

→ Af­ter fin­ish­ing up his fi­nal lap at Adap­ti­m­mune, James No­ble has hopped aboard the board of di­rec­tors as chair­man at Orexo AB. No­ble found­ed Adap­ti­m­mune in 2014 and fi­nal­ly hand­ed the reins over to GSK vet Adri­an Raw­cliffe last sum­mer. Pre­vi­ous­ly, No­ble served as co-CEO of Im­muno­core.

In­go Chakravar­ty

→ Boston biotech Car­du­ri­on Phar­ma­ceu­ti­cals, which fo­cus­es on treat­ments for car­dio­vas­cu­lar dis­eases, has ap­point­ed Pe­ter Lawrence as pres­i­dent and CEO. For 14 years, Lawrence had been pres­i­dent and COO of Ar­Qule, which was sold to Mer­ck in Jan­u­ary. Be­fore Ar­Qule, he was the founder and gen­er­al part­ner of Pod Ven­ture Part­ners. Lawrence suc­ceeds in­ter­im CEO Michael Mendel­sohn, who will stay on as chair­man of Car­du­ri­on’s board of di­rec­tors.

→ In March, Mesa Biotech got emer­gency au­tho­riza­tion from the FDA to do rapid coro­n­avirus test­ing. This week, the San Diego start­up has tapped In­go Chakravar­ty as pres­i­dent and CEO, suc­ceed­ing co-founder Hong Cai. Be­fore be­gin­ning his tenure at Mesa Biotech, Chakravar­ty was pres­i­dent and CEO of NAV­I­CAN Ge­nomics and al­so had an ar­ray of lead­er­ship po­si­tions at Gen­Mark Di­ag­nos­tics, Gen-Probe, Roche and Ven­tana Med­ical Sys­tems.

Ax­el Hoos

Ax­el Hoos, the SVP, R&D gov­er­nance chair and ther­a­peu­tic area head for on­col­o­gy at GSK, is join­ing the board of di­rec­tors at im­munother­a­py biotech TCR2 Ther­a­peu­tics. Hoos has al­so been glob­al med­ical lead in im­munol­o­gy and on­col­o­gy at Bris­tol My­ers Squibb.

Jean-Pierre Wery has been ap­point­ed ex­ec­u­tive chair­man of the board at Crown­Bio, a pre­clin­i­cal on­col­o­gy CRO out of San Diego. Wery will al­so be chief tech­nol­o­gy of­fi­cer for JSR Life Sci­ences, which ac­quired Crown­Bio in 2018. Wery was Crown­Bio’s CEO for four years, and his suc­ces­sor, Armin Spu­ra, as­sumed the role April 1. Spu­ra takes the reins af­ter briefly serv­ing as VP, Asia Pa­cif­ic & Greater Chi­na for CareDx. He al­so took on lead­er­ship roles at Life Tech­nolo­gies and Ther­mo Fish­er Sci­en­tif­ic.

Ohana Bio­sciences has en­list­ed for­mer GSK ex­ec Ramiro Cas­tro-San­ta­maria as CMO. Dur­ing his 14 year stint at GSK, Cas­tro-San­ta­maria most re­cent­ly served as VP of clin­i­cal sci­ences and head of rheuma­tol­ogy. Pri­or to that, he served at Boehringer In­gel­heim and Phar­ma­cia (lat­er Pfiz­er).

Edgar­do Barac­chi­ni

→ As clin­i­cal pro­grams progress with LSD-1 in­hibitor bomedem­stat, San Fran­cis­co-based Ima­go Bio­sciences, which tar­gets bone mar­row dis­eases, has cho­sen Edgar­do Barac­chi­ni as CBO. Barac­chi­ni held the same post at Xen­cor for eight years. He was al­so head of busi­ness de­vel­op­ment at Metaba­sis Ther­a­peu­tics from 2002 un­til 2009, when it merged with Lig­and Phar­ma­ceu­ti­cals.

→ Hav­ing closed its $33 mil­lion Se­ries B and brought on CMO and head of R&D Christo­pher Haqq in Oc­to­ber, Eli­cio Ther­a­peu­tics has named Es­ther Welkowsky VP, clin­i­cal op­er­a­tions. Pri­or to her ar­rival at Eli­cio, which cen­ters on am­phiphile im­munother­a­pies, Welkowsky served as ex­ec­u­tive di­rec­tor of clin­i­cal op­er­a­tions at Al­lo­gene. She was al­so a con­trib­u­tor to such NDA fil­ings as Zyti­ga, Yon­delis, Avastin, Rap­ti­va and Lu­cen­tis.

Adam Salt­man

Adam Salt­man has stepped away from the FDA to be­come the CMO at Eko, which works in car­diac and pul­monary care. While at the FDA, Salt­man was a med­ical of­fi­cer at the Cen­ter for De­vices and Ra­di­o­log­i­cal Health. He was al­so a car­dio­tho­racic sur­geon at Mount Sinai Beth Is­rael in New York and the Uni­ver­si­ty of Mass­a­chu­setts.

Cel­lia Habi­ta has made the leap to Im­mod­u­lon Ther­a­peu­tics as CMO. Be­fore mov­ing over to the British im­muno-on­col­o­gy com­pa­ny, Habi­ta was pres­i­dent and CEO of Ar­i­anne Clin­i­cal Re­search. Oth­er roles be­fore her time with Ar­i­anne in­clude di­rec­tor of prod­uct de­vel­op­ment at Im­mu­sol and VP and SVP of clin­i­cal and med­ical af­fairs at Ad­ven­trx.

Cheryl Co­hen

As re­port­ed this week, Mei Phar­ma an­nounced a $682.5 mil­lion part­ner­ship, in­clud­ing $100 mil­lion up­front, with Ky­owa Kirin tar­get­ing PI3K. Al­so this week, Cheryl Co­hen has joined Mei’s board of di­rec­tors. Co­hen is the founder and pres­i­dent of CLC Con­sult­ing and was al­so CCO of Medi­va­tion. She’s al­so held posts at J&J com­pa­nies Cen­to­cor and Health Care Sys­tems, Inc.

→ Af­ter boost­ing their cof­fers with an amend­ed CAR-T deal with Servi­er in Feb­ru­ary, Cel­lec­tis has tapped for­mer Cel­gene ex­ec Car­rie Brown­stein as CMO. Dur­ing her time at Cel­gene, Brown­stein most re­cent­ly served as VP, glob­al clin­i­cal re­search and de­vel­op­ment, ther­a­peu­tic area head for myeloid dis­eases. Pri­or to her role at Cel­gene, Brown­stein held a post at Roche.

Neil Gra­ham has signed on at Evelo Bio­sciences as the Cam­bridge, MA biotech’s chief de­vel­op­ment of­fi­cer. Pri­or to his move to Evelo, which fo­cus­es on mon­o­clon­al mi­cro­bials to fight can­cer, Gra­ham was VP, strate­gic pro­gram di­rec­tion, im­munol­o­gy and in­flam­ma­tion at Re­gen­eron for the last decade. He was al­so SVP, pro­gram and port­fo­lio man­age­ment at Ver­tex.

Er­ic Pauwels

PTC Ther­a­peu­tics, which now has to wait a lit­tle longer for the FDA’s re­view of ris­diplam for SMA, has made two changes to its lead­er­ship team, with Er­ic Pauwels and Matthew Klein step­ping in as CBO and chief de­vel­op­ment of­fi­cer, re­spec­tive­ly. In 2015, Pauwels joined PTC as SVP and GM of the Amer­i­c­as. Klein was pre­vi­ous­ly CEO and CMO of Bio­Elec­tron Tech­nol­o­gy Cor­po­ra­tion.

Cy­to­Dyn, de­vel­op­ing CCR5 an­tag­o­nist leron­limab (PRO 140), has named Scott Kel­ly as CMO and head of busi­ness de­vel­op­ment. Kel­ly joined Cy­to­Dyn in April 2017 as a di­rec­tor and has served as chair­man of the board since De­cem­ber 2018. Kel­ly will con­tin­ue to serve his du­ties as chair­man. Pri­or to join­ing the com­pa­ny, Kel­ly served at Resur­gens Or­thopaedics.

Jonathan To­bin

Ar­ix Bio­science has been oc­cu­py­ing its share of this space late­ly, and this week is no dif­fer­ent. Jonathan To­bin, cur­rent­ly the in­vest­ment di­rec­tor at Ar­ix in Lon­don, has climbed in­to the role of man­ag­ing di­rec­tor, ef­fec­tive im­me­di­ate­ly. Changes have al­so been made to the in­vest­ment team, in­clud­ing the pro­mo­tion of in­vest­ment as­so­ciate John Cas­sidy to prin­ci­pal.

TRA­CON Phar­ma­ceu­ti­cals has wel­comed Sha­he Garabe­di­an to its team as SVP of qual­i­ty as­sur­ance. Be­fore join­ing the San Diego bio­phar­ma, Garabe­di­an built his qual­i­ty as­sur­ance bona fides at Are­na Phar­ma­ceu­ti­cals (VP of qual­i­ty), In­vit­ro­gen (VP, qual­i­ty as­sur­ance and GxP com­pli­ance) and SUG­EN (ex­ec­u­tive di­rec­tor and head of qual­i­ty as­sur­ance).

Thomas Bre­gen­z­er

Cy­tel, which pro­vides clin­i­cal re­search ser­vices and soft­ware so­lu­tions for the de­sign and analy­sis of clin­i­cal tri­als, has named Thomas Bre­gen­z­er glob­al head of bio­sta­tis­tics. Bre­gen­z­er was at Parex­el for more than 21 years, most re­cent­ly as se­nior di­rec­tor, EU head of bio­sta­tis­tics and sta­tis­ti­cal pro­gram­ming.

GNS Health­care has in­tro­duced Earl Stein­berg as chief of its health plan di­vi­sion to “ad­vance the com­pa­ny’s health plan of­fer­ings and dri­ve clin­i­cal an­a­lyt­ics ef­forts across GNS, par­tic­u­lar­ly with­in bio­phar­ma mar­ket-ac­cess and val­ue-based drug col­lab­o­ra­tions be­tween bio­phar­ma and pay­ers.” Stein­berg comes to GNS af­ter be­ing co-founder and CEO of xG Health So­lu­tions.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Image: Shutterstock

Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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No­vavax CFO Greg Covi­no makes a quick ex­it as lead­er­ship carousel keeps turn­ing; Kite ex­ecs fly away to a cell ther­a­py up­start team­ing up with Lyell

→ If it seems like Covid-19 vaccine bridesmaid Novavax is shuttling staffers in and out, you’re not alone. This week, Greg Covino announced he’s leaving the biotech after holding the CFO job for a blink-and-you-miss-it tenure. An advisory role within the company awaits for Covino, who came to Novavax just five months ago from Bristol Myers Squibb, while CCO and CBO John Trizzini juggles even more executive responsibility by taking on the interim CFO title.

J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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