Endpoints@JPM: (left to right) Steve Pearson, Nick Leschly, Bari Talente, Stephen Ubl, John Carroll

#JPM20: 'The NPV is al­ways wrong.' Take­da preps an­oth­er spin­out — this time on psych

Ed­i­tor’s Note: End­points News is re­port­ing live from #JPM20 af­ter kick­ing things off with an ac­tion-packed event, which you can re­play here. What fol­lows is a stream of tid­bits we have col­lect­ed while wan­der­ing around Union Square in San Fran­cis­co. Check back in through­out the week for up­dates by John Car­roll and Ja­son Mast.

Christophe We­ber and Andy Plump, Take­da

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SAN FRAN­CIS­CO — A year ago Take­da CEO Christophe We­ber and R&D chief Andy Plump ar­rived at JP Mor­gan right on the heels of clos­ing their big Shire buy­out. Now they’re back af­ter shak­ing up the port­fo­lio, boost­ing R&D spend­ing by about 50% to $4.5 bil­lion and ad­just­ing the pipeline — a task which isn’t quite fin­ished yet.

In an in­ter­view on Tues­day, Plump told me that Take­da is prepar­ing the lat­est in a long string of spin­outs af­ter set­ting aside a pack­age of psych drugs that would be bet­ter suit­ed to the hands of some spe­cial­ists. Like a lot of the ma­jor R&D out­fits, it’s not one of their core fields of ex­per­tise.

These Take­da drugs — in­clud­ing ther­a­pies for de­pres­sion and schiz­o­phre­nia — are “very in­ter­est­ing but still dif­fi­cult,” in­volv­ing 3 clin­i­cal-stage pro­grams and a “hand­ful” of pre­clin­i­cal ef­forts, which Plump is quick to con­cede of­fer plen­ty of chal­lenges to de­vel­op­ers.

Spin­outs are some­thing that Take­da is good at. They’ve been do­ing these deals for sev­er­al years now  — in the US, Eu­rope and Asia — cre­at­ing up to 25 com­pa­nies where they typ­i­cal­ly hand off drugs to en­tre­pre­neurs and re­tain a chunk of eq­ui­ty of around 25% to 30% of the biotech in­volved.

Their de­ci­sions on what to keep and what to deal out, adds Plump and We­ber, have noth­ing to do with pro­ject­ed rev­enue.

The key as­pect is in­no­va­tion, says We­ber. Are they in­no­v­a­tive ther­a­pies? And it’s not be­cause you shouldn’t do it on pro­ject­ed rev­enue so much as you can’t do it that way.

You can’t do it on NPV, adds Plump, be­cause then “NPV be­comes the dri­ver of the de­ci­sion and the NPV is al­ways wrong.”

The two Take­da ex­ecs spent con­sid­er­able time and ef­fort in re­vamp­ing Take­da in­to a glob­al, top 15 play­er with scale.

“We were lack­ing scale to sus­tain our R&D in­vest­ment,” says the CEO. The Shire buy­out “gave us scale that al­lows us to com­pete with any­one.” — John Car­roll

#JPM20: Catal­ent CEO John Chimin­s­ki isn’t go­ing any­where im­por­tant with­out his lucky boots

End­points@JPM: John Car­roll with Catal­ent CEO John Chimin­s­ki at a fire­side chat.

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In my one-on-one with Catal­ent CEO John Chimin­s­ki I was drawn to the cow­boy boots he was wear­ing — not ex­act­ly the kind of stan­dard Brooks Broth­ers at­tire you of­ten see at JP Mor­gan — and asked him how many miles he had put on the boots as he trav­eled the world man­ag­ing a glob­al CD­MO.

Here’s part of the re­sponse, and it’s price­less:

In ad­vance of the IPO…our own­ers, Black­stone, went to the in­vest­ment bankers and they said, ‘Hey, be­fore Chimin­s­ki goes out on the road show, he’s got to shave his beard and he can’t wear the boots.’ So I told the in­vestors — and I would just say, I used a cou­ple of hand ges­tures — and I said, look, you might as well shave my head, be­cause I’ll have about that much con­fi­dence. And I will tell you right now, if I didn’t show up with my boots, which is a lit­tle hard­er nowa­days cause I’ve got arthrit­ic hips, if I didn’t show up with my boots, I’d had some in­vestors that are sell­ing. So these boots have about a mil­lion miles and 165 mil­lion shares of Catal­ent were sold with these ba­bies…

Boots aren’t go­ing any­where guys. — John Car­roll

#JPM20 Paul Hud­son con­tin­ues to re­brand Sanofi, promis­es more ac­qui­si­tions and megablock­buster in Dupix­ent 

Paul Hus­don (GET­TY)

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Paul Hud­son wants you to know that Sanofi’s rep­u­ta­tion as a timid com­pa­ny isn’t true. Or at least it’s no longer true.

“An ac­cu­sa­tion la­beled or lev­eled at us the last few years is that we’ve been a lit­tle too in­to the late-stage, a lit­tle too risk-averse and per­haps we changed the pro­file of the com­pa­ny to be a lit­tle bit con­ser­v­a­tive,” Hud­son said at Sanofi’s JP Mor­gan pre­sen­ta­tion. “I can tell you from the peo­ple I’m work­ing along­side now it’s not the case.”

Hud­son used his first JPM pre­sen­ta­tion as Sanofi CEO to dou­ble down on the vi­sion he’s hint­ed at since he was first tapped for the job in June and laid out firm­ly last month. That in­cludes less di­a­betes, more vac­cines, more on­col­o­gy and a clean break from car­dio­vas­cu­lar. It al­so, he guar­an­teed, in­cludes ac­qui­si­tions.

“We will en­rich our pipeline ex­ter­nal­ly,” Hud­son said.

Of the as­sets they have, Dupix­ent is the cen­ter­piece. Hud­son again said Sanofi could make the asth­ma and rash drug a megablock­buster, with over $10 bil­lion in peak sales as it reach­es more pa­tients. That would be about 5 times its cur­rent an­nu­al­ized sales and, along with mid-to-high sin­gle-dig­it growth in vac­cines, a key part of hit­ting Hud­son’s goal of a 32% busi­ness mar­gin. (Part of the Dupix­ent prof­its go to Re­gen­eron, who co-de­vel­oped the drug).

Not part of that plan is push­ing di­a­betes drugs in the US, where Hud­son said per­sis­tent price pres­sure had cut in­to prof­its, de­spite the fact they’re sell­ing more of their prod­ucts, which in­clude in­sulin and a GLP-1 ag­o­nist. Sanofi end­ed di­a­betes and car­dio­vas­cu­lar re­search in the De­cem­ber re­struc­ture.

“It is what it is,” Hud­son said, adding that there were more prof­itable di­a­betes op­por­tu­ni­ties abroad, in­clud­ing in Chi­na.

In that De­cem­ber re­struc­ture, Sanofi re­worked the agree­ment with Re­gen­eron and said they would fo­cus on six drugs: a pre­ven­ta­tive treat­ment for RSV in in­fants, two new he­mo­phil­ia drugs, a lyso­so­mal dis­or­der drug, a se­lec­tive es­tro­gen in­hibitor for breast can­cer and a BTK in­hibitor for MS.

He went through each at JPM, and promised there would be a read­out on the MS drug in days or weeks, which they’ll act on im­me­di­ate­ly.

“We are ready to go with four Phase III tri­als,” Hud­son said.  “We will not wait and will not hes­i­tate.”

Skep­ti­cal they can pull it off? Hud­son won’t ar­gue, but he said in Sanofi’s case, there’s not much else to do.

“There may be skep­ti­cism,” he said, “but frankly there’s been more skep­ti­cism for longer about what we’ve failed to de­liv­er be­fore.” — Ja­son Mast

#JPM20: Con­sid­ered New Pfiz­er yet? The CEO says he’s whip­ping it in­to shape and get­ting more fo­cused in R&D

Al­bert Bourla (AP Im­ages)

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SAN FRAN­CIS­CO — Al­bert Bourla isn’t a fan of the Old Pfiz­er. Or of the an­a­lysts who are ig­nor­ing the New Pfiz­er.

Bourla, giv­ing his sec­ond JP Mor­gan pre­sen­ta­tion as Pfiz­er CEO af­ter over 25 years at the US drug-mak­ing gi­ant, con­tin­ued the re­brand he had been tapped to lead: Po­si­tion­ing Pfiz­er as a com­pa­ny that had been bloat­ed for years but was now set for a stream­lined fu­ture.

“Phase II suc­cess rates – the in­dus­try is at 30%. Pfiz­er for many years was at 15%,” Bourla told JP Mor­gan an­a­lyst Christo­pher Scott on stage. “We were tak­ing an ap­proach of very lit­tle rig­or.”

Bourla said Pfiz­er would fur­ther pare down on non-R&D ex­pens­es, con­tin­u­ing a trend they be­gan by spin­ning off their Up­john di­vi­sion and com­bin­ing their con­sumer health busi­ness in­to a joint ven­ture with Glax­o­SmithK­line. The GSK ven­ture will move to an IPO with­in 3 or 4 years, he said, echo­ing pre­vi­ous com­ments from the British phar­ma.

With­in re­search, Bourla said, they would fo­cus on 6 ar­eas, rather than the 12 they had long de­vel­oped. Asked which parts of the pipeline were ex­cit­ing but over­looked, Bourla dove for a minute through the R&D as­sets he thought Wall Street had ig­nored, in­clud­ing their vac­cines, gene ther­a­pies, and in­flam­ma­tion drugs.

“We do have five dif­fer­ent JAKS that we are start­ing in more than 10 dif­fer­ent in­di­ca­tions, and on­ly one of them I have seen mi­nor pro­jec­tions for,” he said.  “I can go on and on.”

Bourla said they would fo­cus on li­cens­ing small add-ons to the pipeline in those 6 ar­eas and wouldn’t move to­ward a larg­er merg­er or ac­qui­si­tion. It was a dis­cour­ag­ing com­ment for a JP Mor­gan crowd watch­ing for the next ma­jor deal, but al­so one Pfiz­er has made in the past — months be­fore ac­quir­ing Ar­ray in an $11.4 bil­lion deal.

“We nev­er say nev­er,” Bourla said. — Ja­son Mast

#JPM20: Con­sum­mate deal­mak­er Vivek Ra­maswamy bags a record re­gion­al up­front in lat­est deal — this time fo­cused on Japan

Vivek Ra­maswamy at the US-Chi­na Bio­phar­ma In­no­va­tion and In­vest­ment Sum­mit in Shang­hai on Oc­to­ber 23, 2018; Cred­it: End­points News, Pharm­Cube

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SAN FRAN­CIS­CO — At the start of my pan­el on deal­mak­ing on Mon­day I joked that con­sum­mate ne­go­tia­tor Vivek Ra­maswamy was prob­a­bly mak­ing a deal as we spoke, tex­ting terms on the mo­bile.

To­day he tells me I wasn’t far off the mark. A deal was cook­ing right off stage.

On Day Three of JP Mor­gan Ra­maswamy has com­plet­ed what his team is call­ing “the largest ever re­gion­al li­cens­ing deal for a derm prod­uct pri­or to Phase III da­ta and one of the largest up­front pay­ments ever for a Japan­ese li­cens­ing deal pri­or to Phase III da­ta across any in­di­ca­tion.”

And this one was signed late Tues­day night af­ter they put the fin­ish­ing touch­es to the con­tract.

“I al­most laughed when you said that,” Ra­maswamy tells me about the pan­el com­ment. “If I had not put my phone on flight mode that would have been true.”

Todd Za­vod­nick

His der­ma­tol­ogy play Der­ma­vant has li­censed out ex­clu­sive rights to de­vel­op, reg­is­ter and mar­ket tap­inarof in Japan to Japan To­bac­co, which is pass­ing on the li­cense to its sub­sidiary, Torii Phar­ma­ceu­ti­cal. The Japan­ese com­pa­ny is pay­ing $60 mil­lion and up to $53 mil­lion in de­vel­op­ment mile­stones for tap­inarof, in de­vel­op­ment for pso­ri­a­sis and atopic der­mati­tis.

Der­ma­vant CEO Todd Za­vod­nick is clear­ly stoked about the num­bers for his drug, and the shot of work­ing on this with JT’s com­pa­ny.

“It’s not just a li­cens­ing deal,” he says, “it’s the right part­ner.”

Ra­maswamy bought the drug from GSK for up to $324 mil­lion. The phar­ma gi­ant had been re­vamp­ing its pipeline as R&D chief Hal Bar­ron took aim at a come­back. The non­s­teroidal an­ti-in­flam­ma­to­ry top­i­cal cream — which ac­ti­vates the aryl hy­dro­car­bon re­cep­tor — hasn’t been a high-pro­file agent. But re­searchers have high­light­ed promis­ing mid-stage da­ta to un­der­score its po­ten­tial. Now it’s in Phase III for pso­ri­a­sis, with plans to tee up atopic der­mati­tis.

Not sur­pris­ing­ly, any men­tion of AD im­me­di­ate­ly spark com­par­isons to the mar­ket­ing jug­ger­naut Re­gen­eron and Sanofi have cre­at­ed for Dupix­ent. But Der­ma­vant’s drug is a top­i­cal, and Za­vod­nick says that in AD, pa­tients typ­i­cal­ly get an in­jectable and a top­i­cal. So he be­lieves that pa­tients will start with the top­i­cal and then move to the com­bi­na­tion, leav­ing plen­ty of room for his drug in the mar­ket.

Ra­maswamy al­so notes that it’s rare to see two sig­nif­i­cant der­ma­tol­ogy deals at one JP Mor­gan. Eli Lil­ly got the par­ty start­ed with their $1.1 bil­lion Der­mi­ra buy­out, which gave them le­brik­izum­ab. Der­ma­vant’s deal makes for a nice book end to that pack­age of news.

Says Ra­maswamy: “Der­ma­tol­ogy is back.” — John Car­roll

#JPM20 Two biotech leg­ends talk about gene ther­a­py’s past and fu­ture

Jim Wil­son (WUXI)

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SAN FRAN­CIS­CO — I had a chance on Tues­day to mod­er­ate a dis­cus­sion on gene ther­a­py R&D at the huge WuXi con­fer­ence at the Hilton with Fra­zier’s Tachi Ya­ma­da — ex-CSO at GSK and Take­da — and James Wil­son from Penn, two big fig­ures in the field who start­ed a new biotech a lit­tle un­der a year ago to fo­cus on mono­genic dis­eases of the CNS. I did a bow to both of them for every­thing they’ve pi­o­neered in the last 2 decades, which spurred Ya­ma­da to give Wil­son cred­it for the sci­ence work. As for Wil­son, he got the biggest re­sponse from the au­di­ence with a re­mark that Tachi is al­ways just called by his first name, mak­ing him the Be­y­once of biotech. — John Car­roll

#JPM20: Bio­gen CEO Michel Vounatsos of­fers a ‘smile of con­fi­dence’ as he presents ad­u­canam­ab

Michel Vounatsos (GET­TY)

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SAN FRAN­CIS­CO – Bio­gen isn’t soft­en­ing its rhetoric.

Two months af­ter re­search chief Al San­drock all-but dared the FDA not to ap­prove its Alzheimer’s drug, CEO Michel Vounatsos told an over­flow­ing ball­room at JP Mor­gan that the suc­cess of a drug that op­er­ates like ad­u­canum­ab is a “dream.”

“Some of you were at CTAD and saw the da­ta,” Vountsos said, re­fer­ring to the con­tro­ver­sial sub­group analy­sis they pre­sent­ed their ad­u­canum­ab tri­al at the Clin­i­cal Tri­als for Alzheimer’s Dis­ease con­fer­ence.  “Who in the room doesn’t know some­one who has been de­ment­ed?”

That analy­sis, com­ing months af­ter the com­pa­ny first an­nounced the drug had failed, re­lied on be­liev­ing how late changes – called ‘pro­to­col amend­ments’ – to one of two iden­ti­cal tri­als made one suc­ceed and the oth­er fail.

“With­out all the pro­to­col amend­ments, I would not be here, to­day, stand­ing with a smile of con­fi­dence on the way for­ward,” Vounatsos said, not quite smil­ing.

Still, Bio­gen has heard the crit­i­cism from in­vestors, most no­tably Baird’s Bri­an Sko­r­ney, that their big am­bi­tions have left them with few as­sets in their pipeline like­ly to raise rev­enue soon. Vounatsos walked through the oth­er parts of their pipeline, in­clud­ing a lu­pus drug, an ALS drug, a new MS drug and, of course, an­oth­er new Alzheimer’s drug with a dif­fer­ent ap­proach: Tau. — Ja­son Mast

End­points@JPM: Arie Bellde­grun, Josh Bilenker, Stéphane Ban­cel, Vivek Ra­maswamy, Mar­i­anne De Backer dis­cuss pric­ing with John Car­roll.

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#JPM20: Gilead launch­es new T cell en­gi­neer­ing play­er — with help from fa­mil­iar play­ers at Vi­da and West­lake

SAN FRAN­CIS­CO — Af­ter mak­ing its mark in can­cer cell ther­a­pies with the Kite buy­out, Gilead has al­lied it­self with a pair of ven­ture in­vestors to hunt for new ther­a­pies that can ad­dress the flip side of that ther­a­peu­tic coin. And they’re chip­ping in­to the launch round while adding more cash and mile­stones for a re­search col­lab­o­ra­tion to get things un­der­way.

The new biotech they’re show­ing off around JP Mor­gan this week is Kyver­na Ther­a­peu­tics, which is be­ing gift­ed with some cut­ting-edge T cell en­gi­neer­ing tech as well as a $25 mil­lion Se­ries A to fund work on the dis­cov­ery of new drugs for au­toim­mune dis­ease — brak­ing the im­mune sys­tem in­stead of or­ga­niz­ing an at­tack. Gilead signed off on a col­lab­o­ra­tion that starts with a $17.5 mil­lion up­front and a full slate of re­search and com­mer­cial mile­stones that can go up to $570 mil­lion.

The start­up brings to­geth­er some high-pro­file fig­ures and tech­nol­o­gy.

Do­minic Borie, a Genen­tech vet and the for­mer head of ex­ter­nal re­search for Hori­zon Ther­a­peu­tics, is head­ing the new ven­ture as CEO. While at Stan­ford, Borie was cred­it­ed with play­ing a part in val­i­dat­ing JAK in­hi­bi­tion for rheuma­toid arthri­tis — a break­through tech that a bevy of drug de­vel­op­ers would like to leapfrog with new and bet­ter drugs. Jef­frey Greve, for­mer­ly at Delinia ahead of their Cel­gene buy­out, is CSO.

They’re be­ing backed by Fred Co­hen at Vi­da Ven­tures and Beth Sei­den­berg, who found­ed West­lake Vil­lage BioPart­ners in LA af­ter jump­ing from Klein­er Perkins, where she spe­cial­ized in biotech for years. Co­hen kick­start­ed Vi­da along with a group of ex­pe­ri­enced ven­ture play­ers, with a big role for Arie Bellde­grun, who sold Kite to Gilead and re­tains some A list con­tacts at the big biotech.

The up­start will be us­ing syn­Notch tech­nol­o­gy from Kite, which picked it up in Gilead’s ac­qui­si­tion of Wen­dell Lim’s Cell De­sign Labs a cou­ple of years ago, which Bellde­grun had backed as well, sit­ting on the board with Sei­den­berg.

These are the re­la­tion­ships that breed trust and com­pa­nies. — John Car­roll

#JPM20: Bio­Marin chief Bi­en­aime is build­ing a gene ther­a­py pipeline, and the man­u­fac­tur­ing need­ed to con­trol mar­kets

JJ Bi­en­aimé

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SAN FRAN­CIS­CO — JJ Bi­en­aimé and his team aren’t just run­ning as fast as pos­si­ble to stay in the lead in the race to the first mar­ket­ing OK for a gene ther­a­py for he­mo­phil­ia A. They’re build­ing a pipeline, and the man­u­fac­tur­ing ca­pac­i­ty need­ed to dom­i­nate their mar­kets.

In a chat just ahead of the fir­ing gun at JP Mor­gan, the Bio­Marin CEO told me that the biotech had ob­tained of­fi­cial sanc­tions in the US and UK to launch a Phase I/II study of BMN 307 this quar­ter for phenylke­tonuria (PKU). Now they will set out to see if a sin­gle dose of their gene ther­a­py can re­store nat­ur­al Phe me­tab­o­lism, nor­mal­ize plas­ma Phe lev­els and en­able a nor­mal di­et in pa­tients with PKU.

Next up in the gene ther­a­py port­fo­lio, he tells me, is HAE.

What’s more, he adds, Bio­Marin has com­plet­ed work at a fa­cil­i­ty in No­va­to that dou­bles their gene ther­a­py ca­pac­i­ty — jump­ing from 5,000 to 10,000 pa­tients a year. That could han­dle the he­mo­phil­ia A mar­ket and the rest of the mar­kets to come for some time. It al­so gets them around the bot­tle­neck of gene ther­a­py pro­duc­tion that has been hold­ing back some of the new play­ers in the field.

“It’s not our in­tent to turn in­to a pure gene ther­a­py com­pa­ny, but it will play an im­por­tant role in Bio­Marin,” says Bi­en­aimé.

For now, the sto­ry around gene ther­a­py at Bio­Marin is cen­tered on a sim­ple theme: Bio­Marin has the first shot at an up­com­ing ap­proval in he­mo­phil­ia A, but a wan­ing lev­el of re­sponse leaves a few an­a­lysts won­der­ing whether Sang­amo, in par­tic­u­lar, can catch up with a bet­ter ther­a­py. The rea­son­ing is built around the as­sump­tion that pa­tients would pre­fer to wait for a bet­ter gene ther­a­py, as they can on­ly be dosed with an AAV prod­uct once.

Bi­en­aimé, though, doesn’t be­lieve that’s true. He points to new work on next-gen gene ther­a­pies skirt­ing AAV that can be re­peat­ed­ly dosed, and that opens the door to a re-up down the road if their prod­uct stops work­ing. Pa­tients will take a prospec­tive cure, he says, with a 4-year up­date com­ing up that he be­lieves will show a con­tin­ued pro­tec­tion against bleeds.

One oth­er point: The CEO be­lieves that giv­en the high cost of treat­ing he­mo­phil­ia pa­tients now, pay­ers will ac­cept a drug that costs $2 mil­lion to $3 mil­lion. (Not that that is the price they ex­pect to charge. That de­ci­sion comes lat­er.)

But will they want some as­sur­ances that it will work long enough to make it less ex­pen­sive in the long run? — John Car­roll

#JPM20: Ver­tex lays out post-CF am­bi­tions as Gilead de­murs on ac­qui­si­tion

Jeff Lei­den (via YouTube)

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SAN FRAN­CIS­CO – Ver­tex laid out a vi­sion for a post-CF and a post-Jef­frey Lei­den fu­ture at their in­vestor pre­sen­ta­tion Mon­day morn­ing. It looks a lot like the past.

That fu­ture, the out­go­ing CEO Lei­den said, will cen­ter on oth­er fields like cys­tic fi­bro­sis: Spe­cif­ic nich­es where few life-al­ter­ing drugs ex­ist, in­clud­ing sick­le cell and a rare lung dis­ease. Those are al­so fields where Ver­tex could stand to charge the high prices they’re be­com­ing known for.

“You will nev­er see a me-too drug from us,” Lei­den said, a day af­ter Alex­is Borisy sparked wide­spread buzz with a new biotech aimed at low­er­ing drug prices through me-too drugs. “We think trans­for­ma­tive med­i­cines are of the high­est val­ue for pa­tients.”

This year was all but des­tined to mark a new chap­ter for Ver­tex. Near­ly 20 years af­ter start­ing their CF pro­gram, the com­pa­ny in­tro­duced their cap­stone last year in Trikaf­ta, a drug that cov­ers 90% of pa­tients. Lei­den cel­e­brat­ed the news by leav­ing the CEO spot to be­come ex­ec­u­tive chair­man.

Lei­den may or may not have been re­fer­ring to Borisy’s star­tups with his com­ments, but the ref­er­ence is fit­ting. Ver­tex’s drugs have made cys­tic fi­bro­sis a liv­able dis­ease, but it comes at a price tag – over $300,000 for Trikaf­ta – pay­ers like the NHS have balked at.

Resh­ma Ke­wal­ra­mani

With the Ver­tex changes and Gilead sit­ting on both a wealth of cash and a shaky pipeline, ru­mors swirled the big Cal­i­for­nia biotech could pur­sue a mas­sive buy­out. But ear­ly Mon­day morn­ing, Gilead CEO Daniel O’Day threw cold wa­ter on that, telling in­vestors that the com­pa­ny was look­ing to ex­pand but any changes would be “bolt-on ac­qui­si­tions.”

In what was billed as a “fire­side chat,” in­com­ing Ver­tex CEO Resh­ma Ke­wal­ra­mani de­tailed the pipeline she would lead. Those as­sets in­clude mar­ket­ing an ex­pand­ed in­di­ca­tion for Trikaf­ta (it’s not yet ap­proved for chil­dren un­der 12), a drug for al­pha-1 an­tit­rypsin de­fi­cien­cy – a lung dis­or­der sim­i­lar to cys­tic fi­bro­sis – their gene ther­a­py pro­gram for sick­le cell and be­ta tha­las­samia, and the po­ten­tial cell ther­a­py cure for di­a­betes they ac­quired with the $950 mil­lion Sem­ma buy­out. 

The wa­ter-cool­er has al­so buzzed with talk that Ver­tex could ac­quire CRISPR Ther­a­peu­tics, their part­ner on the gene ther­a­py pro­gram.

Ke­wal­ra­mani didn’t ad­dress that. In­stead, she of­fered a tar­get for Ver­tex’s most re­cent ac­qui­si­tion: bring­ing the cell ther­a­py in­to the clin­ic.

“We’ve set an am­bi­tious goal for our­selves with Sem­ma,” Ke­wal­ra­mani said. — Ja­son Mast

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Drug­mak­ers 'inch­ing ahead' in in­creas­ing ac­cess to drugs world­wide, with Glax­o­SmithK­line lead­ing the pack

Top drug developers are “inching ahead” in improving access to much-needed drugs around the world — an issue that has been underscored by the Covid-19 pandemic. But there’s still more work to do, Access to Medicine Foundation executive director Jayasree Iyer said.

Every two years, the Access to Medicines Index ranks the top 20 biotechs leading the push for better access to medicines in low- and middle-income countries. This year’s report, published Tuesday, looks at drug access in 106 countries.

Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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David Marek, Myovant

My­ovant beefs up da­ta pack­age in NDA #3, boost­ing its case for longterm dos­ing of Pfiz­er-part­nered re­l­u­golix

When Pfizer handed over $650 million in cash to partner on Myovant’s relugolix, the pharma giant made clear that the deal — valued at $4.2 billion total — was just as much about the approved indication of prostate cancer as the two women’s health conditions the drug could treat.

A month later, the two companies are offering another glimpse of the therapy’s longterm potential in endometriosis.

Look­ing to win over some skep­ti­cal an­a­lysts, Rhythm beats the drum on in­ter­im da­ta in PhII bas­ket study for ad­di­tion­al in­di­ca­tions

Rhythm Pharmaceuticals has been working toward expanding the FDA approval they received just two months ago for three rare genetic disorders that result in obesity. In December, their Phase III cut of data saw mixed reactions from analysts, but new interim results released Tuesday may provide more excitement.

In an ongoing Phase II study for setmelanotide across individuals with one of three distinct rare genetic diseases of obesity, 65 patients had reached the Dec. 17 cutoff date for evaluation. Among patients who met the primary endpoint of at least 5% weight loss over three months, Rhythm saw an average reduction of no less than 7.1% in any of the groups.