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.

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.