Un­fazed by re­cent car­dio set­backs, No­var­tis takes an­oth­er PhI­II chal­lenge head-on with Akcea drug

In the lat­est move of its long wres­tle with the gi­ant car­dio mar­ket, No­var­tis is mount­ing a late-stage cam­paign for a lipopro­tein(a)-low­er­ing drug de­vel­oped by Akcea.

The phar­ma gi­ant is go­ing for an op­tion to take over Phase III de­vel­op­ment three months af­ter the close­ly held Io­n­is af­fil­i­ate un­veiled Phase II da­ta that showed its drug, then called AKCEA-APO(a)-LRx, could slash Lp(a) lev­els sig­nif­i­cant­ly in at-risk car­dio pa­tients.

There’s no men­tion of any fi­nan­cials in the press re­lease, but ac­cord­ing to the terms spelled out in their 2017 pact, Akcea stands to pock­et $150 mil­lion for the li­cense fee.

No­var­tis is now pro­ject­ing big things for the as­set, which it has re­named TQJ230.

John Tsai

“No treat­ments are cur­rent­ly avail­able to sub­stan­tial­ly low­er Lp(a). Peo­ple with this in­her­it­ed risk fac­tor are fac­ing car­dio­vas­cu­lar risks that can­not be ad­dressed ef­fec­tive­ly with lifestyle changes,” said John Tsai, head of glob­al drug de­vel­op­ment. “We’re ex­cit­ed about the nov­el, RNA-tar­get­ing ap­proach that could be a game-chang­er for peo­ple with el­e­vat­ed Lp(a). If our Phase 3 tri­al suc­ceeds, we ex­pect that TQJ230 will be­come the lead­ing treat­ment op­tion and an­oth­er pil­lar of our long­stand­ing com­mit­ment to re-imag­in­ing car­dio­vas­cu­lar med­i­cine.”

This will mark an­oth­er ma­jor car­dio ef­fort af­ter canakinum­ab, a key an­ti-in­flam­ma­to­ry drug that Tsai’s pre­de­ces­sor (and cur­rent CEO) Vas Narasimhan tout­ed as a block­buster in the mak­ing, on­ly to piv­ot quick­ly to can­cer when the FDA bat­ted the ap­pli­ca­tion back. While No­var­tis sup­plied ev­i­dence that its drug re­duced the like­li­hood of ma­jor car­dio­vas­cu­lar events like heart at­tack or stroke in a large sub­group of pa­tients, it didn’t quite make the cut as a tar­get­ed ther­a­py.

And that, in turn, fol­lowed the col­lapse of its at­tempt to get its oth­er car­dio drug sere­lax­in ap­proved.

For a po­ten­tial mass mar­ket in­di­ca­tion like el­e­vat­ed Lp(a), hav­ing a fair­ly clean safe­ty pro­file is crit­i­cal, Stifel an­a­lysts wrote in a note call­ing the opt-in a “val­i­dat­ing event” for Io­n­is/Akcea’s plat­form tech, which is de­signed to aug­ment the po­ten­cy of an­ti­sense drugs.

From No­var­tis’ van­tage point, we’d imag­ine that ahead of ex­e­cut­ing the Akcea deal, they were al­ready com­fort­able with the po­ten­tial ben­e­fits of low­er­ing Lp(a) – thus the ph2, and the de­ci­sion to opt-in, was like­ly large­ly about whether or not a clean-enough pro­file was es­tab­lished. (…) while the ph2 da­ta weren’t pris­tine, elim­i­na­tion of throm­bo­cy­tope­nia and re­nal func­tion is­sues sug­gests that LI­CA can go a long way in widen­ing the ther­a­peu­tic in­dex of an­ti­sense drugs.

TQJ230 will now test the the­o­ry sur­round­ing Lp(a) as a bio­mark­er for car­dio risk — and No­var­tis’ abil­i­ty to turn hype in­to re­al com­mer­cial re­sults.

At the Amer­i­can Heart As­so­ci­a­tion Sci­en­tif­ic Ses­sions last No­vem­ber, Akcea re­port­ed that around 98% of pa­tients in the 20mg week­ly co­hort and 81% in the 60mg every four weeks group achieved “clin­i­cal­ly sig­nif­i­cant re­duc­tions in Lp(a) lev­els bring­ing them be­low the rec­om­mend­ed thresh­old of risk for CVD events (<50 mg/dL).”

Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Eli Casdin, Casdin Capital

Eli Cas­din backs Codex­is' plat­form tech with $50M eq­ui­ty buy

About a month af­ter Codex­is notched a deal with No­var­tis $NVS, the Cal­i­for­nia com­pa­ny $CDXS on Thurs­day said long-time in­vestor Cas­din Cap­i­tal is putting up $50 mil­lion in a pri­vate place­ment, which puts the New York-based in­vest­ment firm in con­trol of more than 5% of the pro­tein en­gi­neer­ing play­er’s stock.

Eli Cas­din start­ed his epony­mous in­vest­ment firm in 2012 and dates his re­la­tion­ship with Codex­is back to at least a decade. About three years ago, Cas­din Cap­i­tal be­gan in­vest­ing in the in­dus­tri­al biotech com­pa­ny, af­ter it piv­ot­ed its fo­cus to the life sci­ences — un­der the aus­pices of new chief John Nicols — away from the en­er­gy in­dus­try.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

In­vestors pony up $476M for the lat­est round of biotech IPOs to hit the Street

Three biotechs — and a genome se­quenc­ing play­er — have caught the lat­est tide to the Gold Coast of IPOs, round­ing out the first half of 2019 with 23 new drug de­vel­op­ers mak­ing it on Nas­daq.

Most of these com­pa­nies filed their IPOs al­most si­mul­ta­ne­ous­ly, though we’re still wait­ing on word of fel­low class­mate Bridge­Bio’s pric­ing af­ter CEO Neil Ku­mar set the terms at $14 to $16 a share on Mon­day in search of a $240 mil­lion (or so) wind­fall. If he’s suc­cess­ful, that would take the one-week haul past the $700 mil­lion mark, a fresh sign that in­vestors’ en­thu­si­asm for new­ly coined pub­lic biotechs hasn’t cooled.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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Robert Forrester, Verastem

Ve­rastem CEO For­rester steps to the ex­it as the board hunts com­mer­cial-savvy ex­ec for the be­lea­guered biotech

Robert For­rester is step­ping down as CEO of Ve­rastem On­col­o­gy $VSTM just 8 months af­ter the com­pa­ny nabbed an ap­proval for du­velis­ib, a PI3K drug with a sto­ried past — and what ap­pears as not much of a fu­ture.

The biotech put out word this morn­ing that For­rester will take an ad­vi­so­ry role with Ve­rastem while COO Dan Pa­ter­son steps up to take charge of the lead­er­ship team and the board looks around for a new CEO.