Rachel Haurwitz (Caribou)

Ab­b­Vie taps Jen­nifer Doud­na start­up for li­cens­ing deal worth up to $300M for 2 'off-the-shelf' CRISPR-edit­ed CAR-Ts

“Off-the-shelf” CAR-Ts have emerged as a hot-tick­et tar­get in next-gen on­col­o­gy R&D, with Big Phar­mas plac­ing big down pay­ments on nov­el tech. Now, a Cal­i­for­nia biotech play­er found­ed by one of the CRISPR move­ment’s most promi­nent mouth­pieces has added ma­jor drug­mak­er Ab­b­Vie as a team­mate.

Ab­b­Vie will pay Cari­bou Bio­sciences $40 mil­lion in up­front cash and $300 mil­lion in biobucks to de­vel­op two of the biotech’s al­lo­gene­ic CAR-T ther­a­pies, the part­ners said Wednes­day. The biotech will al­so be due roy­al­ty pay­ments for any fu­ture com­mer­cial as­sets.

Jen­nifer Doud­na

Cari­bou, co-found­ed by CRISPR maven Jen­nifer Doud­na and head­ed by one of Doud­na’s acolytes, Rachel Hau­r­witz, us­es hy­brid DNA/RNA edit­ing tech­nol­o­gy dubbed chRD­NA — pro­nounced “chardon­nay” for the wine drinkers — the biotech be­lieves can stop off-tar­get edit­ing com­mon to cur­rent CRISPR ap­pli­ca­tions.

Ab­b­Vie will have the op­tion to pay an ex­tra fee for ac­cess to two ad­di­tion­al CAR-T pro­grams, Cari­bou said.

On a call with End­points News, Hau­r­witz de­clined to com­ment on what drew Ab­b­Vie to Cari­bou’s tech­nol­o­gy or what ther­a­peu­tic ar­eas the drug­mak­er want­ed to tar­get with the po­ten­tial CAR-Ts. That’s not un­usu­al: Ab­b­Vie is usu­al­ly cagey with its ear­ly de­vel­op­ment deals.

In­stead, the young CEO — just 35 and one of the lead­ing voic­es in the CRISPR move­ment — tout­ed her plat­form’s po­ten­tial to re­work cur­rent lim­i­ta­tions on al­lo­gene­ic CAR-Ts, in­clud­ing pa­tients’ im­mune sys­tems re­ject­ing donor cells. The ef­fects could be most ap­par­ent, how­ev­er, in man­u­fac­tur­ing, a chron­i­cal­ly ex­pen­sive and time con­sum­ing process for CAR-Ts.

Un­like tra­di­tion­al CAR-Ts, which re­quire com­pa­nies to re-en­gi­neer pa­tients cells that are of­ten dam­aged af­ter chemother­a­py and past ther­a­pies, al­lo­gene­ic ther­a­pies can use donor cells that are health­i­er and faster to ad­min­is­ter.

“It re­al­ly has tremen­dous ad­van­tages from a tim­ing per­spec­tive,” Hau­r­witz said. “There are of course re­al­ly nice ad­van­tages in terms of qual­i­ty and cost of goods.”

Al­lo­gene­ic cell ther­a­pies are among the hottest fields in next-gen on­col­o­gy, and Ab­b­Vie — one of the four largest drug­mak­ers on earth — is seek­ing to take a mod­est-sized bet out on Cari­bou’s stout rep­u­ta­tion and clin­i­cal po­ten­tial.

The biotech has two lead pro­grams: CB-010, an an­ti-CD19 CAR-T that deletes PD-1 cur­rent­ly in a Phase I tri­al, and CB-011, a pre­clin­i­cal as­set that hopes to uti­lize “im­mune cloak­ing” to avoid the hu­man im­mune sys­tem, Hau­r­witz said.

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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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