Two people believed to be FDA inspectors leave the Juno facility in Bothell, MA, as captured by a spotter hired by an investor. (via Mizuho analyst Salim Syed)

Bris­tol My­ers hints at some prob­lems with an FDA in­spec­tion need­ed for the liso-cel OK, and the Cel­gene CVR in­vestors are pan­ick­ing

The nail-bit­ing among the in­vestors who held on to their con­tin­gent val­ue rights $BMYRT from the Cel­gene buy­out is giv­ing way to pan­ic to­day.

Dur­ing their Q3 call with an­a­lysts, ex­ecs said that the FDA had in­spect­ed the Both­ell, WA plant be­ing used for liso-cel (JCAR017), the CAR-T Bris­tol My­ers picked up in the deal. That’s a plus.

But there was a hitch.

They went on to say that no in­spec­tion had yet even been sched­uled for the Texas CMO in­volved. And with that, the slow trick­le of in­vestors seep­ing out of the CVR group turned in­to an in­stant rout. The stock plunged about 80% from the $3.40 still left for the CVR at the end of the day yes­ter­day. That took it down in­to pen­ny stock ter­ri­to­ry.

Why the stam­pede?

Sal­im Syed

Re­mem­ber that dur­ing last-minute buy­out ne­go­ti­a­tions, Bris­tol My­ers CEO Gio­van­ni Caforio in­sist­ed that they re­place cash on the ta­ble with a $9 CVR tied to the ap­proval of 3 drugs by cer­tain dates. Ozan­i­mod made the dead­line, but the fate of liso-cel (JCAR017) and ide-cel are hang­ing on FDA ac­tions. Liso-cel was de­layed and ide-cel got slapped with a refuse-to-file. That drug has un­til the end of Q1 next year to make good.

As Bris­tol My­ers notes in its Q3 pre­sen­ta­tion to­day, there’s a Nov. 16 PDU­FA date loom­ing for liso-cel, and with­out a clean bill of health on CMC, reg­u­la­tors aren’t like­ly to come through with an OK for a once close­ly-watched ther­a­py that earned break­through sta­tus at the FDA. That could spell an­oth­er sig­nif­i­cant de­lay.

Sal­im Syed at Mizuho, who’s been track­ing every tiny de­tail around the CMC work — in­clud­ing pri­vate eye re­ports com­plete with tele­pho­to lens­es on the Both­ell in­spec­tion — summed it up here:

The way it was couched on the call was that this is due to COVID-19 and the FDA want­i­ng to keep its em­ploy­ees safe. How­ev­er, in­vestors were ex­pect­ing, and what seemed to make most log­i­cal sense, is to in­spect the plants to­geth­er close­ly in se­quence, Both­ell and Texas.

Syed, though, is urg­ing calm in the melee. While the PDU­FA is dat­ed Nov. 16, the dead­line on the CVR is the end of this year — still some weeks out.

Re­mem­ber, the FDA on­ly needs here about ~3 weeks (by rea­son­able stan­dards and our KOL call) post-in­spec­tion to fin­ish up all the pa­per­work and get a drug to the fin­ish line.

Be­sides, he adds, this is a BTD pro­gram, so the FDA should be will­ing to hur­ry up. The ex­ecs at Bris­tol My­ers nev­er said if there could be a vir­tu­al in­spec­tion and be­sides, the com­pa­ny al­ready said that if they learned that they would miss the PDU­FA, they’d say so prompt­ly.

So far, so good on that score. The PDU­FA re­mains in place.

But with the dead­line draw­ing very, very close, a lot of the in­vestors are bolt­ing.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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FDA can­cels ODAC meet­ing this week to re­view two more dan­gling ac­cel­er­at­ed ap­provals — but won't ex­plain why

The FDA’s Oncologic Drugs Advisory Committee has decided to cancel a planned meeting on Thursday to discuss two cancer drugs that previously won accelerated approvals but failed to confirm clinical benefit in required follow-up trials or have taken a long time to finish those trials.

The FDA said in a statement that the meeting “is no longer needed” but did not offer further detail on why exactly it was canceled, telling Endpoints News to contact the companies. Attempts to contact both Secura Bio and Acrotech went unreturned. The companies may have decided to pull these treatments from the market, or they’ve come to new agreements with the agency on their confirmatory trials.