Scathing short at­tack from an ob­scure source ac­cus­es Nan­jing Leg­end of ma­nip­u­lat­ing CAR-T da­ta — Gen­script shares nose­dive

Gen­script, the Chi­nese com­pa­ny be­hind overnight CAR-T star Nan­jing Leg­end Biotech, has seen its stock nose­dive af­ter a damn­ing short re­port ques­tioned its da­ta in­tegri­ty, R&D ex­per­tise, safe­ty record and man­u­fac­tur­ing ca­pa­bil­i­ty.

The stock dipped as much as 47% on the Hong Kong Stock Ex­change be­fore trad­ing was sus­pend­ed at around a 26% loss — or $1 bil­lion.

While Gen­script has yet to is­sue an of­fi­cial re­sponse, the com­pa­ny told Na­tion­al Busi­ness Dai­ly that the short re­port “con­tains mul­ti­ple flaws that are in­ac­cu­rate, and the com­pa­ny will pub­lish a clar­i­fi­ca­tion state­ment as soon as pos­si­ble.”

Xi­ao­hu (Frank) Fan

Once a vir­tu­al un­known con­tract re­search or­ga­ni­za­tion, Gen­script came in­to the glob­al spot­light when its sub­sidiary Leg­end pre­sent­ed some jaw-drop­ping re­sults for its BC­MA-tar­get­ing CAR-T at 2017 AS­CO. Then J&J of­fered a high pro­file en­dorse­ment through its Janssen unit by hand­ing $350 mil­lion up­front to part­ner on LCAR-B38M for mul­ti­ple myelo­ma.

But ac­cord­ing to the re­port writ­ten in Chi­nese and at­trib­uted to Flam­ing Re­search — an ob­scure firm whose web­site lists on­ly one re­port on Ti­bet Wa­ter — Leg­end like­ly forged the da­ta, and Janssen missed out on key due dili­gence in haste to catch up with Cel­gene as the two gi­ants fight for dom­i­nance in mul­ti­ple myelo­ma.

Here’s how they got to that con­clu­sion: Leg­end’s CAR-T da­ta came from three “well rec­og­nized hema­tol­ogy hos­pi­tals” in Shang­hai and Jiang­su, and one mediocre hos­pi­tal in Xi’an. But it was the re­searchers in Xi’an who of­fered the most com­pelling da­ta, where­as the da­ta from the oth­er sites didn’t hold up.

“Giv­en that Xi’an da­ta were over­all bet­ter than Shang­hai da­ta, choos­ing to se­lec­tive­ly dis­close Xi’an da­ta is clear­ly fraud­u­lent be­hav­ior,” the re­port reads. “Like­wise, doc­tors in Shang­hai have doubts about the Xi’an da­ta: On one hand Leg­end was more con­cerned with treat­ment ef­fects than safe­ty, on the oth­er the Xi’an hos­pi­tal didn’t have suf­fi­cient record on safe­ty da­ta.”

The in­ci­dent, wide­ly re­port­ed by Chi­nese me­dia, al­so rais­es ques­tions about the volatil­i­ty of the HKEX at a crit­i­cal time as non-rev­enue biotechs start go­ing pub­lic on the ex­change.

Gen­script the CRO was first list­ed in 2015 but got its big break this year in the wake of Leg­end’s stel­lar CAR-T re­sults, surg­ing as much as 500%. Now that its stock is close­ly tied to the per­for­mance of its biotech sub­sidiary, what­ev­er hap­pens here could serve as a cau­tion­ary tale for the pre-rev­enue biotechs that have on­ly re­cent­ly been al­lowed on­to the city’s ex­change.

The 14-page re­port starts with the the­sis that it’s worth less than a quar­ter of its cur­rent price. Aside from is­sues with the da­ta, it high­lights a pa­tient death that oc­curred at a Shang­hai site in 2017, which Gen­script dis­closed but hasn’t fol­lowed up on, as an il­lus­tra­tion of the ther­a­py’s po­ten­tial risk. It fur­ther at­tacks Frank (Xi­ao­hu) Fan, Leg­end’s R&D chief, for his lack of cre­den­tials in drug de­vel­op­ment and the “se­ri­ous chal­lenges” he’s faced in ap­ply­ing for patents — in ad­di­tion to the gen­er­al lack of ex­per­tise in his team.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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Which top 10 big phar­mas have the most to gain — or lose — over the next 5 years?

When Evaluate Pharma crunched the likely drug sales numbers for the big 10, 2 stood out. 

Takeda, with its big Shire buyout under its belt, is set to almost double its worldwide sales record for 2018 over 5 years, putting it in the big 10 — the 9th spot, to be exact — which is exactly where CEO Christophe Weber wants to be. 

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Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Maze in­ter­im chief Charles Hom­cy pass­es ba­ton to Ja­son Colo­ma; William Col­lier tapped as CEO of Ar­bu­tus

→ Four months in­to Maze Ther­a­peu­tics’ launch, Charles Hom­cy is pass­ing the start­up’s reins to an­oth­er Third Rock col­league. Ja­son Colo­ma, the cur­rent COO, is leav­ing his role at the VC firm to be­come full-time CEO on a mis­sion to trans­late ge­net­ic in­sights in­to new med­i­cines. Equipped with a whop­ping $191 mil­lion with sup­port from Arch, Maze’s big idea is to find and tar­get ge­net­ic mod­i­fiers that play a role in Mendelian dis­eases caused by an­oth­er gene. Colo­ma, a Roche/Genen­tech vet sea­soned in busi­ness de­vel­op­ment, was CBO at Corvus Phar­ma­ceu­ti­cals be­fore join­ing Third Rock in 2017.

Re­gen­eron/Sanofi's an­ti­body un­der­whelms in asth­ma study — shares of ri­val Anap­tys­Bio pay the price

Al­though ex­pec­ta­tions were mut­ed, Re­gen­eron $REGN and Sanofi’s $SNY ex­per­i­men­tal IL-33 an­ti­body has un­der­whelmed in a proof-of-con­cept mid-stage asth­ma tri­al. Al­though the drug sig­nif­i­cant­ly im­proved the loss of asth­ma con­trol and lung func­tion as a monother­a­py com­pared to a place­bo, its ef­fect was nei­ther su­pe­ri­or to the es­tab­lished Dupix­ent, nor of val­ue when com­bined with the IL-4/IL-13 treat­ment.