Re­gen­eron/Sanofi cir­cle Oc­to­ber 28 on the cal­en­dar as the FDA be­gins a speedy re­view of the world’s 6th PD-1/L1 check­point

A Re­gen­eron/Sanofi team has a tar­get date for launch­ing the world’s 6th PD-1/L1 check­point in­hibitor. And while they may be late to the grow­ing league of ri­vals — dom­i­nat­ed by Bris­tol-My­ers Squibb and Mer­ck — they plan to en­ter with a mar­ket splash, with con­sid­er­able as­sis­tance from FDA reg­u­la­tors who are steel keen to ex­pand the field.

The FDA has agreed to give the PD-1 cemi­plimab a pri­or­i­ty re­view, say the bio­phar­ma part­ners, of­fer­ing a PDU­FA date of Oc­to­ber 28. That comes on top of the break­through ther­a­py des­ig­na­tion from last fall, un­der­scor­ing that reg­u­la­tors will move fast here. The de­vel­op­ers are push­ing hard for an ap­proval to use the drug as the first of the PD-1/L1 ther­a­pies for metasta­t­ic cu­ta­neous squa­mous cell car­ci­no­ma, a non-melanoma skin can­cer, who are not el­i­gi­ble for surgery.

That dead­line gives the Re­gen­eron/Sanofi team time to see if they can make a splash at AS­CO in a few weeks with da­ta from their sin­gle-arm Phase II tri­al. The EMA start­ed their re­view ear­li­er this month.

As­sum­ing they get a quick OK — and giv­en the ex­pe­ri­ence reg­u­la­tors have with PD-1/L1s and the rep these de­vel­op­ers are bring­ing to the ta­ble, that’s easy to do —  you can ex­pect the team to start look­ing to make up for lost time as they ex­pand the use of cemi­plimab. Both Re­gen­eron and Sanofi still have high hopes for the over­all mar­ket op­por­tu­ni­ty, as a line­up of ma­jors like Roche, As­traZeneca and Pfiz­er/Mer­ck KGaA grap­ple with the two top play­ers for a slice of a block­buster mar­ket.

John Reed

At the be­gin­ning of the year Re­gen­eron and Sanofi com­mit­ted an ex­tra bil­lion dol­lars to their war bud­get for cemi­plimab. It rep­re­sents the lat­est in a se­ries of big pro­grams from the part­ners, who are wind­ing down their an­ti­body al­liance. And it will like­ly line up as one of John Reed’s first wins as he re­places Elias Zer­houni at the helm of Sanofi’s glob­al R&D team.

Be­hind them is a grow­ing tsuna­mi of PD-1/L1 can­cer check­points, with a host of new com­bos look­ing to ex­pand their use and widen their im­pact. The Can­cer Re­search In­sti­tute ran a study that found 164 PD-1/L1s in the pipeline, from pre­clin­i­cal through mar­ket­ing stages. No­var­tis has one in de­vel­op­ment sole­ly for in-house use. And we may find a few thrown in as low-pried com­modi­ties, mak­ing check­point 2.0 more im­por­tant than ever for the lead­ers.

Re­gen­eron/Sanofi plan to be on the in­side, look­ing out.

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.

What lured Hal Bar­ron away?; Top FDA minds on ac­cel­er­at­ed ap­proval re­forms; ‘Dead wrong’ Aduhelm ad blitz; 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.

Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.

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Mer­ck wins le­gal bat­tle over in­sur­ance cov­er­age af­ter ran­somware at­tack

Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.

The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.

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Crit­ics push back on Alzheimer’s As­so­ci­a­tion ad blitz to get Medicare to change its Aduhelm rul­ing: 'Dead wrong'

The latest Alzheimer’s Association advertising campaign encourages people to fight.

Not against the disease or for more research or treatments, but against the Centers for Medicare and Medicaid Services. More specifically, CMS’ recent reimbursement decision to only pay for Biogen and Eisai’s controversial Alzheimer’s drug Aduhelm for patients in clinical trials.

With CMS’ preliminary decision now in a 30-day comment period, patient advocates’ goal is to convince CMS to reverse its decision with a marketing blitz and public pressure.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Fail­ing to con­firm clin­i­cal ben­e­fit, Gilead pulls 2 ac­cel­er­at­ed ap­proval in­di­ca­tions for can­cer drug

Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.

“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.

Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

An­oth­er day, an­oth­er xeno­trans­plant, as Unit­ed Ther­a­peu­tics looks to beat com­peti­tors to sci-fi-es­que break­through

Xenotransplantation is having a moment.

Last October, a team from NYU successfully transplanted a kidney from a pig into a brain-dead patient, although observers cast doubt on the importance of the experiment. Then, earlier this month, surgeons at the University of Maryland transplanted a pig heart into a dying human, who appears to still be stable.

Now, another group is planting a flag in the xenotransplantation field. Surgeons at the University of Alabama at Birmingham said Thursday they have achieved the first kidney transplant from a pig to a brain-dead patient, publishing their peer-reviewed findings online. The team, aiming to differentiate itself from the others through the genetic modifications used, is hoping there’s now enough research to soon begin clinical xenotransplantation studies.

Richard Pazdur (via AACR)

Time lim­its on ac­cel­er­at­ed ap­provals? FDA's on­col­o­gy chief Rick Paz­dur eyes po­ten­tial re­forms via in­ter­na­tion­al ap­proach­es

The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.

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