Yescar­ta goes to Chi­na as Fo­s­un Kite nabs his­toric first CAR-T OK

The CAR-T era has of­fi­cial­ly ar­rived in Chi­na.

Kite Phar­ma — which had lost out to No­var­tis in the bat­tle for first FDA ap­proval by just two months — can now claim a his­toric first at the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion, where its joint ven­ture with Fo­s­un steered Yescar­ta to a land­mark OK.

First ac­cept­ed for pri­or­i­ty re­view last March, the CD19-tar­get­ing ther­a­py is in­di­cat­ed for pa­tients with re­lapsed or re­frac­to­ry large B-cell lym­phoma who have re­ceived two or more lines of pri­or treat­ment, ac­cord­ing to an NPMA state­ment. That in­cludes dif­fuse large B-cell lym­phoma and pri­ma­ry me­di­asti­nal large B cell lym­phoma, among oth­ers, they added.

It’s been a long way com­ing. The cre­ation of Fo­s­un Kite pre­dat­ed Kite’s first FDA ap­proval in 2017, as Arie Bellde­grun and David Chang pon­dered their prospec­tive leap in­to key Asian mar­kets.

By tap­ping in­to Fo­s­un’s clin­i­cal net­work, man­u­fac­tur­ing ca­pac­i­ty and over­all strong on­col­o­gy pres­ence in Chi­na, Kite promised to bring its CAR-Ts — not just Yescar­ta but the oth­ers to come — to the coun­try faster and go deep­er.

The $40 mil­lion up­front cash and $20 mil­lion ini­tial fund­ing didn’t hurt, ei­ther.

“At the time the deal was done, the Phase II pro­gram was un­der­way,” Chang re­called to End­points News re­cent­ly. The ex­pe­ri­ence there, he added, drove home the les­son that “every­thing re­al­ly had to be start­ed very quick­ly, man­u­fac­tur­ing and all that, so the lead time re­quire­ment was pret­ty high.”

Ri­vals fol­lowed with a sim­i­lar mod­el. Juno en­list­ed WuXi to start JW Ther­a­peu­tics, now a HKEX-list­ed com­pa­ny with its own liso-cel in­spired CD19 CAR-T un­der re­view; Sin­ga­pore’s Tes­sa launched its own JV but kept a larg­er chunk of eq­ui­ty for it­self.

For their next-gen, off-the-shelf CAR-T play­er Al­lo­gene, Chang and Bellde­grun went with Hill­house-backed start­up Over­land — there­by al­ly­ing them­selves with a group of sea­soned and well-con­nect­ed biotech fig­ures in Chi­na.

Kite, which is now a sub­sidiary of Gilead, has a sec­ond CD19-di­rect­ed CAR-T on the US mar­ket dubbed Tecar­tus. But Fo­s­un Kite is keep­ing the rest of its pipeline some­what opaque for now.

With JW and J&J-backed Leg­end near­ing the fin­ish line while new­er en­trants like CARs­gen and Gra­cell load­ing up on cash to vault to the front, you can ex­pect much more to come in the space.

So­cial im­age cred­it: Fo­s­un

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Rare dis­ease drug­mak­ers to Con­gress: Don't gut the ac­cel­er­at­ed ap­proval path­way

The controversy over the FDA’s accelerated approval pathway is heating up.

Last week, the FDA’s top oncology official Rick Pazdur said the pathway is “under attack,” largely due to the agency’s recent accelerated approval of Biogen’s controversial Alzheimer’s drug and the surrogate endpoint used in that decision. In the meantime, three accelerated approval indications have been pulled since July 1 (two from Bristol Myers Squibb and one from Merck in recent weeks), even as Pazdur called on critics of the pathway to not miss the more positive, big picture, with some cancer drugs proving to be enormously helpful and approved years before their confirmatory trials were completed.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Janet Woodcock (Bill Clark/CQ Roll Call via AP Images)

HHS ex­tends Aduhelm in­ves­ti­ga­tion in­to the ac­cel­er­at­ed ap­proval path­way, wad­ing in­to a brew­ing con­tro­ver­sy

The government investigation into how the FDA approved Aduhelm appears to point well beyond the agency’s ties with Biogen in the leadup to its approval of their controversial Alzheimer’s drug Aduhelm.

The HHS Office of Inspector General posted a notice Wednesday that officials will review the accelerated approval pathway, the regulatory mechanism the agency used to approve the drug in the face of conflicting data over whether it could actually slow Alzheimer’s patients’ mental decline. Now the Aduhelm OK is just one branch of an investigation called for last month by acting FDA commissioner Janet Woodcock.

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Samantha Du, Zai Lab CEO (Zai Lab)

Saman­tha Du's Zai Lab inks sur­pris­ing re­search col­lab with Schrödinger for DNA dam­age drug

Headed by Samantha Du, Chinese oncology specialist Zai Lab has made no qualms about its aggressive in-licensing strategy to drive Western drugs into regional markets. That strategy has been profitable so far, but that doesn’t mean Du’s team isn’t willing to try something new.

In a surprising volte-face, Zai Lab has signed its name to a research collaboration with physics-based discovery outfit Schrödinger looking for an oncology candidate targeting the DNA damage repair pathway, the partners said Wednesday.

FDA slaps back small-cap's bid to re­pur­pose 50-year-old chemother­a­py

When the German drugmaker Medac handed off a decades-old cancer compound to their US subsidiary in February, it thought it would be smooth sailing to market. After all, the drug had recently been approved in Europe and was available in Canada.

On Tuesday, though, Medac and that publicly traded subsidiary, Medexus, announced the FDA had rejected the compound, telling the company they would need to get new data and reanalyze their Phase III trial before marketing it in the US.