Cel­gene joins the PD-1 check­point club, bag­ging BeiGene drug with $413M down and $1B on the ta­ble

Cel­gene is join­ing the fast grow­ing PD-1 club.

John Oyler, BeiGene

The big biotech $CELG has tak­en a lead po­si­tion in the sec­ond wave of PD-1 check­points in the clin­ic, bag­ging rights to BeiGene’s $BGNE can­di­date for sol­id tu­mors in a $1.4 bil­lion deal, with $263 mil­lion of that paid up­front in a li­cens­ing fee, $150 mil­lion for an eq­ui­ty stake and $980 mil­lion more in mile­stones.

It’s a rich deal for this stage of the game, aimed at the rich­est health­care mar­kets on the plan­et. Cel­gene gets the US and Eu­ro­pean mar­kets and every­thing else out­side of Asia, which the Chi­nese com­pa­ny is keep­ing for it­self, along with glob­al de­vel­op­ment rights on hema­to­log­i­cal ma­lig­nan­cies. Two years ago, Cel­gene paid As­traZeneca $450 mil­lion up front to part­ner on their PD-L1 for heme.

The move was an­nounced the same day as Mer­ck’s com­bi­na­tion stud­ies match­ing its PD-1 drug Keytru­da with Cel­gene’s Revlim­id and Po­m­a­lyst were put on hold by the FDA — a month af­ter in­ves­ti­ga­tors flagged a high­er rate of death in the check­point arm. This was one of the rare in­stances where a check­point was linked to a po­ten­tial­ly lethal safe­ty is­sue. So far, their abil­i­ty to spur an im­mune sys­tem at­tack on can­cer cells has been carv­ing out bil­lions in new rev­enue, with hun­dreds of com­bi­na­tion stud­ies un­der­way to match the im­muno-on­col­o­gy ap­proach with tar­get­ed ther­a­pies.

BeiGene, which is in late-stage stud­ies in Chi­na, is al­so get­ting Cel­gene’s com­mer­cial op­er­a­tions in the mas­sive Asian mar­ket, with ex­clu­sive rights to com­mer­cial­ize Revlim­id, Abrax­ane and Vi­daza. BeiGene al­so gets rights to Cel­gene’s C-122, a next-gen CelMOD cur­rent­ly in de­vel­op­ment for lym­phoma and he­pa­to­cel­lu­lar car­ci­no­ma. And BeiGene is com­mit­ted to beef­ing up its man­u­fac­tur­ing op­er­a­tions to sup­port the part­ner­ship in Asia.

The PD-(L)1 crew al­ready has 5 ther­a­pies on the US mar­ket. Mer­ck and Bris­tol-My­ers Squibb led the way, fol­lowed by Roche, Pfiz­er/Mer­ck KGaA and As­traZeneca. But they keep com­ing. Cel­gene’s check­point will start a piv­otal glob­al pro­gram next year, mak­ing it a dis­tinct­ly late en­try in the field.

While the ba­sic bi­ol­o­gy of the PD-1 drugs is well un­der­stood at this point, mak­ing the clin­i­cal risk rel­a­tive­ly mod­est, there have still been plen­ty of sur­pris­es as new pro­grams push ahead. Bris­tol-My­ers found that out with its mis­step in lung can­cer, which has cost the com­pa­ny dear­ly. There are some big de­bates go­ing on now on whether PD-1 is in­her­ent­ly more po­tent than PD-L1 in dis­man­tling a can­cer cell’s stealth mech­a­nism. That dis­cus­sion was sharp­ened af­ter Roche’s PD-L1 Tecen­triq failed a cru­cial late-stage study, but it’s nowhere close to be­ing re­solved.

For its part, BeiGene says that its PD-1 just might be bet­ter than the rest, point­ing to some added en­gi­neer­ing in the Fc re­gion of the an­ti­body. And Chi­na once again ben­e­fits from news that points to the boom­ing coun­try as a hot spot in drug de­vel­op­ment.

Cel­gene is bring­ing in the PD-1 as the com­pa­ny con­tin­ues one of the most am­bi­tious pipeline cam­paigns in the in­dus­try, beef­ing up its R&D group with a deal spree that has now con­tin­ued for years. For BeiGene, Cel­gene’s quest is their op­por­tu­ni­ty to part­ner up with one of the most ag­gres­sive biotechs in the busi­ness.

“This strate­gic part­ner­ship with Cel­gene is a trans­for­ma­tion­al event for BeiGene, tran­si­tion­ing us in­to a com­mer­cial-stage com­pa­ny and prepar­ing us well for the fu­ture po­ten­tial launch of our in­ter­nal­ly de­vel­oped com­pounds, some of which are al­ready in piv­otal tri­als in Chi­na,” said John Oyler, CEO and co-founder of BeiGene.

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.

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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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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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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‘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.

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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UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

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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