No­var­tis takes a $200M hit to write off a flop as ex­ecs spell out a cau­tious R&D strat­e­gy

With its top line locked in­to a hold­ing pat­tern as its sales teams strug­gle to ex­pand fran­chise rev­enue, No­var­tis has out­lined its near-term R&D strat­e­gy, tak­ing a $200 mil­lion hit to write off the dis­ap­point­ing heart drug sere­lax­in (RLX030) while look­ing be­yond a de­ci­sion lat­er in the year on its pi­o­neer­ing CAR-T drug as it shoots for new FDA fil­ings.

Joe Jimenez. No­var­tis

The phar­ma gi­ant said re­cent­ly that it has wrapped its ini­tial FDA ap­pli­ca­tion for CTL019. And in a new note out from Biren Amin the Jef­feries an­a­lyst pre­dicts that we’ll be see­ing the close­ly-watched fol­lowup da­ta from the JULI­ET study at the In­ter­na­tion­al Con­fer­ence on Ma­lig­nant Lym­phoma on June 14 in Lugano, Switzer­land.

Fol­low­ing a hoped-for ap­proval in CAR-T, No­var­tis re­vealed this morn­ing that its dis­cus­sions with reg­u­la­tors is en­cour­ag­ing the com­pa­ny to aim for a 2018 fil­ing for SEG101 (crizan­l­izum­ab) for sick­le cell pain crises af­ter it wraps the PK/PD com­pa­ra­bil­i­ty study to fi­nal man­u­fac­tur­ing process. Right along­side the phar­ma gi­ant’s R&D team will an­gle for an FDA ap­proval of BAF312 (sipon­i­mod) for re­laps­ing mul­ti­ple scle­ro­sis.

What you shouldn’t look for: Any big M&A pacts. Like a num­ber of its ma­jor league ri­vals, No­var­tis ex­ecs say that val­u­a­tions in biotech have reached a for­bid­ding high. No­var­tis CEO Joe Jimenez adds that the com­pa­ny’s BD team plans to move up­stream, to ear­li­er-stage as­sets and deals, as the com­pa­ny stays fo­cused on what you can get in a $2 bil­lion to $5 bil­lion bolt-on.

“The price of some of these as­sets has in­creased to the point that we don’t feel like we can cre­ate val­ue for No­var­tis share­hold­ers,’’ Chief Ex­ec­u­tive Of­fi­cer Joe Jimenez said on a con­fer­ence call Tues­day, ac­cord­ing to a re­port from Bloomberg.

Net in­come for No­var­tis slid 15% in the first quar­ter, which the com­pa­ny large­ly at­trib­uted to the $200 mil­lion charge on sere­lax­in. The write off for its one-time block­buster hope can’t come as any kind of sur­prise. A month ago No­var­tis said that its 4-year study of the heart drug flubbed the pri­ma­ry end­point, fail­ing to sig­nif­i­cant­ly cut the rate of car­dio­vas­cu­lar death or re­duce wors­en­ing heart fail­ure among pa­tients with acute heart fail­ure.

No­var­tis is left mak­ing slow progress with En­tresto, which has been grap­pling with wide­spread pay­er re­sis­tance. In the mean­time, its big flag­ship drug Gleevec is be­ing ham­mered by gener­ic com­pe­ti­tion.

No­var­tis gained an edge with the March ap­proval of Kisqali (ri­bo­ci­clib), a CDK 4/6 drug which looks like it will have to square off against Pfiz­er as well as Eli Lil­ly. And it re­worked its com­mer­cial­iza­tion pact with Am­gen on their late-stage mi­graine drug. But it’s al­so suf­fered oth­er set­backs in late-stage re­search, in­clud­ing the Phase III fail­ure last fall of Fo­vista for wet AMD, which has crimped its pipeline plans.

No­var­tis is in an awk­ward po­si­tion, un­will­ing or un­able to gam­ble big on new ef­forts and main­tain­ing a very care­ful ap­proach to the top line with­out a megablock­buster able to cap­ture grow­ing sales to fu­el its growth. The com­pa­ny is be­com­ing one of the most pre­dictable play­ers in Big Phar­ma, and that’s not nec­es­sar­i­ly a good thing.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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