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.

Jim Mellon [via YouTube]

Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

On Monday, Juvenescence completed its $100 million series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017.  Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer and Amarin.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.