No­var­tis lays out block­buster launch­es for 2018-2020, with sur­pris­ing in­clu­sion of Kym­ri­ah

As No­var­tis shakes off its Al­con eye busi­ness and the loss of two high-pro­file ex­ec­u­tives, chief Vas Narasimhan has out­lined the Swiss drug­mak­er’s ros­ter of 14 block­buster launch­es, a bulk of which are ex­pect­ed to win ap­proval be­tween this year and 2020. The drugs in­clud­ed in the list are large­ly fa­mil­iar, but the in­clu­sion of the com­pa­ny’s pi­o­neer­ing CAR-T ther­a­py Kym­ri­ah, whose adop­tion has been wob­bly due to man­u­fac­tur­ing woes, will raise some eye­brows.

In 2017, No­var­tis spent a hefty $9 bil­lion on R&D, mak­ing it one of the top spenders in glob­al bio­phar­ma. In­vestors ex­pect to see the com­pa­ny to now get bang for its buck. Last No­vem­ber, the com­pa­ny used it R&D re­view to high­light 26 block­buster con­tenders in its ar­se­nal and of­fer a rosy pic­ture of things to come af­ter suf­fer­ing a few knocks — the FDA re­ject­ing its heart drug, and the com­pa­ny aban­don­ing its at­tempt at a Rit­ux­an copy­cat.

Since then, the com­pa­ny has seen good days and bad. On the pos­i­tive front, its gene ther­a­py for SMA se­cured the FDA’s pri­or­i­ty re­view, and its sick­le cell drug won break­through ther­a­py sta­tus. The drug­mak­er al­so swal­lowed a cell and gene ther­a­py man­u­fac­tur­er in an at­tempt to un­clog the com­mer­cial roll­out for Kym­ri­ah. In a first for a multi­na­tion­al phar­ma­ceu­ti­cal com­pa­ny, No­var­tis’ San­doz unit tied up with Cana­di­an med­ical cannabis pro­duc­er Tilray, a land­mark en­dorse­ment of the con­tro­ver­sial plant. On Wednes­day, as part of its full-year 2018 re­sults, the drug­mak­er said four new­er prod­ucts — in­clud­ing Cosen­tyx and En­tresto — in its ros­ter of med­i­cines achieved block­buster sta­tus in 2018.

On the oth­er end of the scale, No­var­tis has al­so suf­fered a num­ber of set­backs in the last quar­ter of 2018. For in­stance, its mi­graine drug Aimovig — billed as a block­buster and part­nered with Am­gen — was re­fused en­dorse­ment by the UK’s NICE, months af­ter the agency de­clined to give the nod for Kym­ri­ah. Two top ex­ec­u­tives — CAR-T chief David Leb­wohl and on­col­o­gy head Liz Bar­rett — al­so left the drug­mak­er to take top po­si­tions in small biotechs. And J&J dealt No­var­tis a blow when its pso­ri­a­sis drug Trem­fya beat the dom­i­nant Cosen­tyx in a head-to-head study.

2018

  • AIMOVIG — The mi­graine drug forms part of a new crop of bi­o­log­ics tar­get­ing the CGRP pro­tein that trans­mits pain sig­nals in­to the brain. The drug was the first to win ap­proval in 2018, and with­in months ri­val treat­ments from Lil­ly and Te­va were al­so giv­en reg­u­la­to­ry nods. All three have demon­strat­ed sim­i­lar ef­fi­ca­cy and safe­ty in tri­als. Like its ri­vals, the drug is priced at $575 per month and has been pegged as a po­ten­tial block­buster. Fol­low­ing the NICE set­back, re­ports sug­gest­ed Aimovig was ex­clud­ed from phar­ma­cy ben­e­fits man­ag­er CVS’ for­mu­la­ry in the Unit­ed States. This will be a blow, but there are oth­er PBMs on the block.
  • KYM­RI­AH — The pi­o­neer­ing CAR-T drug was FDA ap­proved amidst great fan­fare in 2017, and since then the FDA has ex­pand­ed its use in the Unit­ed States and the EC al­so ap­proved the drug last Au­gust. NICE re­fused to en­dorse the pricey drug. Mean­while, ri­vals such as Gilead’s Yescar­ta have emerged, and Kym­ri­ah sales have suf­fered due to man­u­fac­tur­ing is­sues. In the fourth quar­ter, the drug gen­er­at­ed a pal­try $28 mil­lion. How­ev­er, No­var­tis is do­ing its best to shore up man­u­fac­tur­ing, hav­ing bought cell and gene ther­a­py man­u­fac­tur­er Cell­for­Cure.
  • LU­TATHERA — The can­cer drug, which came with No­var­tis’ $4 bil­lion ac­qui­si­tion of Ad­vanced Ac­cel­er­a­tor Ap­pli­ca­tions in 2017, paid off hand­some­ly in ear­ly 2018 af­ter the FDA gave it a quick OK. No­var­tis re­port­ed an 11% jump in on­col­o­gy rev­enue in the fourth quar­ter, dri­ven part­ly by Lu­tathera sales of $81 mil­lion.

2019

  • BYL719 — Oth­er­wise known as alpelis­ib, the PI3K in­hibitor near­ly dou­bled pro­gres­sion-free sur­vival in a third of pa­tients with a hard-to treat form of breast can­cer last Oc­to­ber. Oth­er drugs in this class, in­clud­ing Roche’s taselis­ib, have large­ly crashed and burned due to safe­ty con­cerns.
  • MAYZENT — Ex­pect­ed to launch this year, the MS drug al­so known as sipon­i­mod is crit­i­cal for the com­pa­ny as one of its top sell­ers Gilenya has gener­ic com­pe­ti­tion loom­ing. Com­pa­ny of­fi­cials have pre­vi­ous­ly de­scribed the drug’s da­ta in glow­ing terms, and an­a­lysts have con­curred, of­fer­ing a $3 bil­lion peak sales pro­jec­tion for the treat­ment.
  • RTH258 — The eye drug has been built up as a ri­val to Re­gen­eron’s flag­ship block­buster in­jec­tion Eylea and CEO Narasimhan has gone so far as to say RTH258 is “con­sis­tent­ly su­pe­ri­or” to Eylea, af­ter con­duct­ing ret­ro­spec­tive analy­ses of late-stage da­ta last year. Eylea has been un­touch­able for more than a decade, so it is up to No­var­tis to prove oth­er­wise.
  • ZOL­GENS­MA — The gene ther­a­py is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion in May. In its re­view post­ed in De­cem­ber, ICER sug­gest­ed the Zol­gens­ma — with a list price of $2 mil­lion — would of­fer more cost-ef­fec­tive ben­e­fit in the long run ver­sus ri­val Bio­gen’s Spin­raza. How­ev­er, the Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the cu­ra­tive ther­a­py, which it ac­quired via a $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

2020

  • COSEN­TYX — The pso­ri­a­sis drug that be­came a block­buster in 2016 is one of No­var­tis’ key drugs, and the drug­mak­er is work­ing on ex­pand­ing the IL-17A in­hibitor’s mar­ket by in­clud­ing pa­tients with pso­ri­at­ic arthri­tis, anky­los­ing spondyli­tis and non-ra­di­ograph­ic ax­i­al spondy­larthri­tis (nrAxS­pA). Piv­otal da­ta are ex­pect­ed lat­er this year and, if pos­i­tive, will be fol­lowed by a mar­ket­ing ap­pli­ca­tion be­fore the end of this year.
  • EN­TRESTO — The med­i­cine is an­oth­er top sell­er for No­var­tis and is cur­rent­ly ap­proved for HFrEF (for­mer­ly known as sys­tolic heart fail­ure) — in these pa­tients the heart mus­cle does not con­tract ef­fec­tive­ly, re­duc­ing the lev­el of oxy­gen-rich blood pumped in­to to the body. Ini­tial­ly a slow mov­ing prod­uct ham­pered by physi­cians re­luc­tant to adopt a new ther­a­py and in­sur­ers who balked at its price, En­tresto is now com­fort­ably a block­buster prod­uct. The drug is now un­der eval­u­a­tion for HF­pEF (al­so re­ferred to as di­as­tolic heart fail­ure) — a now larg­er group of pa­tients whose heart mus­cle con­tract nor­mal­ly but ven­tri­cles do not re­lax as they should dur­ing ven­tric­u­lar fill­ing. Da­ta from the tri­al PARAGON-HF are ex­pect­ed in the third quar­ter of 2019.
  • INC280 — Oth­er­wise known as cap­ma­tinib, the MET in­hibitor was li­censed to No­var­tis from In­cyte in 2009. Mid-stage da­ta on the drug in cer­tain pa­tients with the most com­mon form of lung can­cer (NSCLC), pre­sent­ed last Oc­to­ber, showed INC280 in­duced an ORR of 72% in treat­ment-naive pa­tients and about 39% in pre­vi­ous­ly treat­ed pa­tients. No­var­tis is ex­pect­ed to sub­mit a mar­ket­ing ap­pli­ca­tion lat­er this year.
  • OMB157 — Oth­er than the ap­proved Gilenya and Mayzent — which is cur­rent­ly un­der re­view — ofa­tu­mum­ab (OMB157) is an­oth­er drug No­var­tis is hop­ing to use in MS. The drug, al­ready sold as Arz­er­ra, has caused a num­ber of prob­lems for No­var­tis as a treat­ment for leukemia — the drug­mak­er was forced to pull it off the mar­ket out­side the US as com­pe­ti­tion heat­ed up. No­var­tis thinks it can re­pur­pose the drug for MS, an in­di­ca­tion for which it is cur­rent­ly be­ing in­ves­ti­gat­ed in two Phase III stud­ies.
  • PDR001 COM­BO — PDR001, al­so called spar­tal­izum­ab, is No­var­tis’ PD-1 in­hibitor and is be­ing test­ed in a num­ber of can­cers. A late-stage study test­ing the drug in com­bi­na­tion with No­var­tis’ ap­proved can­cer drugs — Tafin­lar and Mekin­ist — in pa­tients with metasta­t­ic melanoma is on­go­ing.
  • QVM149 — The asth­ma drug is cur­rent­ly be­ing test­ed against stan­dard triple com­bi­na­tion ther­a­py in a late-stage study in un­con­trolled asth­mat­ics — the tri­al is ex­pect­ed to be com­plet­ed this year.
  • SEG101 — Ex­pect­ed to launch in 2020, the sick­le cell dis­ease drug has se­cured the FDA’s break­through ther­a­py des­ig­na­tion ear­li­er this month. Af­ter a de­lay in 2018, a mar­ket­ing ap­pli­ca­tion for the drug is ex­pect­ed this year.

Im­age: NO­VAR­TIS

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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