Fight­ing a gener­ic on­slaught, No­var­tis adds CAR-T to its grow­ing list of late-stage block­busters

One of the rea­sons why No­var­tis isn’t be­ing forced to bid in­to the stratos­phere for late-stage drugs is that its in­dus­try-top­ping R&D bud­get — $9 bil­lion last year — has al­ready fund­ed one of the biggest late-stage pipelines in the in­dus­try. The phar­ma gi­ant un­der­scored that to­day as it out­lined 13 prospec­tive block­busters with up­com­ing piv­otal read­outs in the com­ing months and years, a group that now in­cludes its lead CAR-T ther­a­py for the very first time. And it added more de­tails on its im­muno-on­col­o­gy strat­e­gy, which has lagged be­hind lead­ers like Mer­ck, Bris­tol-My­ers Squibb and Roche.

The CDK 4/6 in­hibitor LEE011 is its most ad­vanced ther­a­py in Phase III. But No­var­tis is plan­ning an am­bi­tious roll­out of da­ta and reg­u­la­to­ry fil­ings as it looks to push past some pun­ish­ing gener­ic com­pe­ti­tion that has blunt­ed its rev­enue growth. It’s al­so look­ing at a slate of sec­ond-gen­er­a­tion im­muno-on­col­o­gy pro­grams, with some 20 clin­i­cal stud­ies in the field un­der­way in ear­ly 2017.

As we al­ready knew, its CAR-T CTL019 will be filed for pe­di­atric ALL some­time in the very near fu­ture, with piv­otal tri­al da­ta wait­ing in Q2. An­oth­er fil­ing for DL­B­CL is com­ing up in the sec­ond half of the year – with a ma­jor read­out from JULI­ET – and No­var­tis is step­ping up its game by bull­ish­ly as­sert­ing its $1 bil­lion-plus an­nu­al sales po­ten­tial.

Jef­feries’ Pe­ter Welford not­ed this morn­ing that the CAR-T up­date is par­tic­u­lar­ly sig­nif­i­cant.

“No­var­tis has, for the first time, in­clud­ed CTL019 on its pipeline of pos­si­ble block­busters, un­der­scor­ing its con­fi­dence in the prod­uct’s com­mer­cial po­ten­tial. De­spite lo­gis­ti­cal chal­lenges, we fore­cast at least $1bn WW peak sales for £65-75m in­come to OXB (Ox­ford Bio­Med­ica) p.a. pro­vid­ing an im­por­tant sus­tain­able rev­enue stream for c.5p/share NPV at 80% prob­a­bil­i­ty, more than un­der­pin­ning the cur­rent share price.”

No­var­tis’ bull­ish fore­cast fol­lows its con­tro­ver­sial move to shut­ter its in­de­pen­dent cell and gene ther­a­py unit, ax­ing about 120 staffers in the process. The phar­ma gi­ant, though, spec­i­fied that it not on­ly hasn’t aban­doned its CAR-T pipeline as it en­gi­neered its lat­est glob­al re­or­ga­ni­za­tion of R&D, it’s up­ping its game.

That could present some new pres­sure on Kite, which is hus­tling along its own rolling sub­mis­sion for a pi­o­neer­ing CAR-T, while Juno has yet to de­cide what it will do with its trou­bled lead CAR-T, now stymied by a num­ber of pa­tient deaths.

No­var­tis al­so has a cat­a­lyst rich year planned, with sub­mis­sions on AMG334 for mi­graine, part­nered with Am­gen, as well as BAF312 for sec­ondary pro­gres­sive MS. You can get the full pic­ture here, on pages 57 and 58.

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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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.

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.

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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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.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.