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.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Ahead of strate­gic up­date, new Sanofi CEO mulls op­tions for con­sumer health­care arm — re­ports

Big pharma has made moves to sharpen its focus on developing new medicines, while slow-growing consumer health divisions fall by the wayside. Looks like another large drugmaker is considering a similar move. On Thursday, reports citing sources indicated that Sanofi is reportedly mulling a joint venture, sale, or a public listing of its consumer health arm.

The French group is in discussions for options that could value the division at $30 billion, Bloomberg and Reuters reported, citing sources familiar with the matter.

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The triple crown in biotech: An all-or-noth­ing bet on an FDA ap­proval of 3 drugs over 16 months starts to­day

Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.

The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.

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Genap­sys fi­nal­ly un­veils vaunt­ed se­quencer, but can it dent Il­lu­mi­na?

Hesaam Esfandyarpour holds what looks like a mini-cooler up to the computer screen in his California office.

Esfandyarpour is in his late-30s, with crows feet creeping up against a youthful face. He wears a gray polo and the device in his hand — with its hard plastic-looking shell, blue-and-white pattern, and a white plastic paddle resembling a handle jutting out the front — might contain diced strawberries and peanut-butter sandwiches to meet mom and the kids at a SoCal park. Instead, Esfandyarpour tells me it’s going to change medicine and biopharma research.

Brii Bio backs in­fec­tious dis­ease start­up while ink­ing deal for its lead TB drug, dou­bling down on an­tibi­otics

Almost two years after leaving GSK to launch Brii Bio with a whopping $260 million in funding, Zhi Hong is seeing the trans-Pacific infectious disease specialist he set out to build take shape.

“Our pipeline is coming together,” he told Endpoints News, with 12 partnered assets plus some internal programs.

As its latest partner, AN2 Therapeutics, comes into the limelight for the first time with a $12 million seed round, so is Brii’s plans in the antibiotics space. Brii has obtained China rights to AN2’s antibacterial targeting mycobacterium tuberculosis for multi-drug resistant TB, which it says is in the clinical stage.

UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Nearly a century passed between the first proto-immunotherapy attempts in cancer — crude and obscure but nonetheless with some scientific basis — and Jim Allison’s first T cell paper. Thirty-plus years flipped between the discovery of CTLA-4 as an off-switch and the approval of Yervoy. Twenty-two rolled between PD-1’s isolation and Opdiva and Keytruda. 

Longwood co-founder Lea Hachigian is betting she can hasten that. It’s a bet on newly established single-cell genomic analysis tech and the ability to crunch endless troves of data at a rate few others can, and investors including Leaps by Bayer and Novartis Venture Fund just put $39 million behind it. They call it Immunitas.