Samantha Du, Zai Lab CEO

Chi­na's Zai Lab spins 2 more li­cens­ing deals, in­clud­ing a sud­den dive in­to neu­ro­science

Chi­na can­cer spe­cial­ist Zai Lab has made its name in-li­cens­ing West­ern drugs and us­ing its deep knowl­edge of the East Asian mar­ket to churn out re­sults. The biotech has now signed two more li­cens­ing deals for a trio of drugs, in­clud­ing a very sud­den jump in­to the neu­ro­science space.

First, Zai Lab inked a deal Tues­day with Karuna Ther­a­peu­tics for rights in main­land Chi­na, Hong Kong, Macau and Tai­wan to KarXT, an in­ves­ti­ga­tion­al M1/M4-pre­fer­ring mus­carinic ag­o­nist be­ing test­ed to treat a va­ri­ety of psy­chi­atric con­di­tions.

Zai Lab will shell out $35 mil­lion up­front in this one with the po­ten­tial for an ad­di­tion­al $80 mil­lion in de­vel­op­ment and reg­u­la­to­ry mile­stones and $72 mil­lion in sales mile­stones on top of roy­al­ties.

Karuna is eval­u­at­ing KarXT in a slate of late-stage tri­als to treat schiz­o­phre­nia and psy­chosis in Alzheimer’s pa­tients with two Phase III stud­ies for the schiz­o­phre­nia pro­gram ex­pect­ed to read­out in 2022. Mean­while, the com­pa­ny is set to be­gin an ad­di­tion­al Phase III tri­al look­ing at KarXT as an ad­junct ther­a­py for pre­vi­ous­ly treat­ed adults by the end of the year.

Mean­while, an up­com­ing Phase III study for KarXT in psy­chosis in Alzheimer’s is set to kick off by mid-2022.

The deal is sig­nif­i­cant in that it rep­re­sents Zai Lab’s first for­ay in­to neu­ro­science, an­oth­er branch­ing out for the can­cer spe­cial­ist. In a state­ment, CEO Saman­tha Du had this to say about why her team de­cid­ed to make the leap:

Our col­lab­o­ra­tion with Karuna is a sig­nif­i­cant mile­stone for Zai Lab, mark­ing the ex­pan­sion and di­ver­si­fi­ca­tion of our de­vel­op­ment and com­mer­cial port­fo­lio in­to neu­ro­science, our fourth ther­a­peu­tic area. KarXT is well po­si­tioned to serve as the an­chor as­set in our new neu­ro­science fran­chise. Zai Lab’s mis­sion is to de­liv­er in­no­v­a­tive med­i­cines to ad­dress un­met med­ical needs of pa­tients, and we look for­ward to work­ing with Karuna to bring KarXT to pa­tients in need in Greater Chi­na as soon as pos­si­ble.

In a sec­ond pact signed Tues­day, Zai Lab will pay $25 mil­lion up­front and a po­ten­tial ad­di­tion­al $590 mil­lion in down­stream mile­stones for rights to Blue­print Med­i­cines’ in­ves­ti­ga­tion­al EGFR TKI in­hibitors BLU-945 and BLU-701 in Chi­na, Hong Kong, Macau and Tai­wan, the com­pa­nies said Tues­day.

Both drugs are be­ing de­vel­oped specif­i­cal­ly to tar­get drug re­sis­tant mu­ta­tions in EGFR-pos­i­tive lung can­cer pa­tients who do not re­spond to oth­er EGFR in­hibitors. BLU-945 specif­i­cal­ly tar­gets the T790M and C797S and mu­ta­tions while BLU-701 tar­gets C797S with pre­clin­i­cal mod­els show­ing a high de­gree of CNS pen­e­tra­tion, po­ten­tial­ly mak­ing it a good op­tion for pa­tients with brain metas­tases.

Zai Lab sees these drugs as po­ten­tial first-in-class op­tions in Chi­na for EGFR-pos­i­tive NSCLC pa­tients whose dis­ease pro­gress­es with oth­er EGFR in­hibitors, and Blue­print is us­ing the deal as part of a glob­al clin­i­cal ef­fort to see both drugs reach ap­proval in Chi­na and else­where.

If ei­ther drug does get ap­proved in Chi­na, Blue­print will be due tiered roy­al­ties in the “low teens to mid-teens.”

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

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For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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