No­var­tis promis­es a speedy CAR-T pitch, boasts about its slate of block­busters-to-be

No­var­tis sent out a loud and clear mes­sage this morn­ing: De­spite fold­ing its stand­alone cell ther­a­py unit, the phar­ma gi­ant says it re­mains on track to de­liv­er an ap­pli­ca­tion for its CAR-T CTL-019 for pe­di­atric acute lym­phoblas­tic leukemia “in ear­ly 2017.” And an ap­pli­ca­tion for dif­fuse large B cell lym­phoma is due in the sec­ond half of next year, which could put it well be­hind ri­val Kite Phar­ma. At the same time, the phar­ma gi­ant — now un­der as­sault from gener­ic com­pe­ti­tion — boast­ed about a dozen late-stage pro­grams with block­buster po­ten­tial to earn more than a bil­lion dol­lars a year.

James Brad­ner, Pres­i­dent, No­var­tis In­sti­tutes for Bio­Med­ical Re­search (NI­BR)

The lat­est CAR-T up­date is on one of the slides on of­fer this morn­ing for the Q3 re­view, as No­var­tis looks to re­as­sure in­vestors that a re­cent de­ci­sion to in­te­grate the cell and gene ther­a­py unit in its re­search or­ga­ni­za­tion — elim­i­nat­ing about 120 po­si­tions — has not de­railed its de­vel­op­ment ef­fort on the CAR-T front, where T cells are reengi­neered to tar­get can­cer cells.

No­var­tis had ea­ger­ly high­light­ed the biotech-like agili­ty of its in­de­pen­dent unit when it was es­tab­lished. But ques­tions have been grow­ing about just how well the pipeline of cell ther­a­pies has been ad­vanc­ing, es­pe­cial­ly af­ter ex­ecs or­dered the unit to be dis­solved. That move was quick­ly fol­lowed by No­var­tis’ de­ci­sion to cull the ranks of its R&D op­er­a­tion as it con­cen­trat­ed ef­forts in a few key hubs, search­ing for cost sav­ings by stream­lin­ing the or­ga­ni­za­tion.

Kite wants to file the first ap­pli­ca­tion for a CAR-T ap­proval in DL­B­CL be­fore the end of this year, pro­vid­ed the FDA al­lows it. Crit­ics, though, have won­dered if de­clin­ing re­sponse rates and a de­mand for more ma­ture da­ta will slow or scut­tle Kite’s play. Kite CEO Arie Bellde­grun re­cent­ly told me that even if the FDA wants to wait to see 6-month re­sults, they’ll have that in Feb­ru­ary. And he’s con­fi­dent that Kite can gain the first-mover ad­van­tage in the field.

Juno Ther­a­peu­tics, mean­while, had to post­pone its plans for an ap­pli­ca­tion this year af­ter its CAR-T drugs killed 4 peo­ple in two dif­fer­ent stud­ies, forc­ing re­searchers to drop flu­dara­bine from the drug reg­i­men used to prep pa­tients to pre­vent a lethal­ly tox­ic re­ac­tion to the ther­a­py. Now lag­ging be­hind in third place, the one­time con­tender has had to ad­just its po­si­tion on the im­por­tance of be­ing in the lead. In a re­cent in­ter­view with the Econ­o­mist, Juno CEO Hans Bish­op said be­ing first isn’t im­por­tant.

Both Kite and Juno al­so know that these pi­o­neer CAR-T ther­a­pies are deeply flawed and will quick­ly be over­tak­en by new tech­nolo­gies that amp up ef­fi­ca­cy and damp down safe­ty threats. Even­tu­al­ly, they al­so are look­ing to ad­vance off-the-shelf ther­a­pies that can re­place the per­son­al­ized meds that re­ly on pa­tient’s T cells.

An­a­lysts will be look­ing for some signs to­day that No­var­tis will be able to per­form in the sec­ond and third waves to come, when its cut­ting edge treat­ment will start to dull in com­par­i­son. With cell ther­a­py leader Oz Azam and oth­ers from the old unit now leav­ing No­var­tis, that will take some con­vinc­ing on their part.

Here’s its hit list of block­buster con­tenders and the time­line for hus­tling them to reg­u­la­tors:

  • There’s the CDK4/6 breast can­cer drug LEE011 (ri­bo­ci­clib), which has wrapped Phase III.
  • BAF312 (sipon­i­mod) for sec­ondary pro­gres­sive mul­ti­ple scle­ro­sis has al­so com­plet­ed late-stage de­vel­op­ment.
  • Next up is Fo­vista, an ap­tamer an­ti-PDGF in-li­censed from Oph­thotech that will read out in the piv­otal tri­al for neo­vas­cu­lar AMD dur­ing this quar­ter.
  • AMG 334 (part­nered with Am­gen), a CGRP re­cep­tor ag­o­nist for mi­graine, al­so reads out this quar­ter.
  • RLX030 (sere­lax­in) is on track for an H1 2017 read­out for heart fail­ure, a big fo­cus at No­var­tis.
  • RTH258 (brolu­cizum­ab) for neo­vas­cu­lar AMD al­so reads out in H1.
  • ACZ885 (canakinum­ab, Ilarus) should see piv­otal da­ta for CV risk re­duc­tion next year.
  • Cosen­tyx (AIN457) is on track to read out in 2018 for non-ra­di­ograph­ic ax­i­al SpA.
  • QVM149 should wrap up in asth­ma in 2018.
  • En­tresto will read out in a new in­di­ca­tion — heart fail­ure with pre­served EF — in 2019.
  • QAW039  (fe­vip­iprant) com­pletes on asth­ma in 2019.
  • And OMB157 (ofa­tu­mum­ab) rounds out the 2019 pro­jec­tion for re­laps­ing mul­ti­ple scle­ro­sis.

 

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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