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).”

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

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David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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