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).”

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.