Frank Zhang (AP Images)

CAR-T fil­ing in sight, Frank Zhang grabs full con­trol of J&J-part­nered Leg­end Biotech, steps down from Gen­Script

Two months af­ter Yuan Xu steered Leg­end Biotech to a $424 mil­lion pub­lic de­but on the Nas­daq, founder and chair­man Frank Zhang is grab­bing the reins as CEO.

In con­junc­tion with the move, Zhang is al­so step­ping down from the helm of Gen­Script — a po­si­tion he’s held for 18 years. Gen­Script, a Hong Kong-list­ed CRO, hatched Leg­end as a sub­sidiary in 2015 be­fore spin­ning it out, and re­mains a ma­jor­i­ty share­hold­er.

“This shows how much com­mit­ment I have for Leg­end,” he said in a con­fer­ence call. “Over the past years, Leg­end has al­ways been un­der my watch. And I’m very in­ter­est­ed in see­ing how it will grow in the next few years.”

Now is the nat­ur­al time for a tran­si­tion as Leg­end and its part­ners at J&J gear up to ini­ti­ate a BLA fil­ing of their BC­MA-tar­get­ed CAR-T ther­a­py by the end of the year, with an EMA fil­ing to fol­low in ear­ly 2021.

“I can as­sure you that this CEO tran­si­tion has noth­ing to do with any change in the planned ac­tiv­i­ties for the JNJ-4528 pro­gram, in­clud­ing clin­i­cal de­vel­op­ment and al­so reg­u­la­to­ry plans,” CFO Ying Huang said in the call.

Hav­ing built up a re­sume fea­tur­ing a who’s who in Big Phar­ma — span­ning Genen­tech, Glax­o­SmithK­line, No­var­tis, Am­gen, Gilead and Mer­ck — Xu took the helm in 2018 and has been run­ning Leg­end out of New York. She stepped down for “per­son­al rea­sons,” ac­cord­ing to a press re­lease.

Zhang promised that the dai­ly op­er­a­tions and re­la­tion­ship with J&J should re­main in­tact.

Jef­feries an­a­lyst Biren Amin was con­vinced. While the EMA time­line marks a slight de­lay from the orig­i­nal plan, he’s not both­ered ei­ther.

“We think Leg­end pur­pose­ly takes ex­tra time to pre­pare a strong CMC pack­age, to stay on the safe side,” he said.

The com­pa­ny might have tak­en a page from ri­vals’ re­cent trou­bles, he added:

Re­call that BMS and BLUE re­ceived Re­fusal-To-File (RTF) let­ter by FDA for the BLA sub­mis­sion of ide-cel/bb2121 in Mar ’20 and re­sub­mit­ted at the end of Ju­ly, which caused a 4-mos de­lay in reg­u­la­to­ry path. FDA re­quired a sup­ple­men­tal doc­u­men­ta­tion of CMC to pro­vide de­tails on val­i­da­tion con­trol process, and we think Leg­end takes ac­tions to min­i­mize any po­ten­tial reg­u­la­to­ry risks.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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News brief­ing: PureTech plans Nas­daq de­but with sec­ondary list­ing; GoodRx prices $725M IPO

London Stock Exchange-listed PureTech Health announced Wednesday that it’s looking to extend to Nasdaq. But due to its “strong cash position,” the biotech says it isn’t issuing any new shares in the potential secondary listing.

The company’s shares closed at £256.50 Tuesday on the London Stock Exchange. Its candidate LYT-100 is currently in Phase I development for various indications, including lymphatic flow disorders and fibrotic and inflammatory disorders. PureTech is expecting a Phase Ib readout in lymphatic flow disorders later this year, and is also planning to launch a Phase II study for the drug to treat respiratory conditions experienced after Covid-19.