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.

 

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.