Watch out Pfiz­er, No­var­tis’ LEE011 bags quick OK for front­line use against breast can­cer

Bruno St­rig­i­ni, CEO No­var­tis On­col­o­gy

No­var­tis picked up a key reg­u­la­to­ry win to­day, gain­ing the FDA’s quick OK for LEE011, its CDK 4/6 drug now ap­proved as a front­line ther­a­py for post­menopausal women with hor­mone-re­cep­tor pos­i­tive, hu­man epi­der­mal growth fac­tor re­cep­tor-2 neg­a­tive ad­vanced or metasta­t­ic breast can­cer in com­bi­na­tion with any aro­matase in­hibitor.

The drug, al­so known as ri­bo­ci­clib, hits the mar­ket as Kisqali with peak sales es­ti­mates of around $2.5 bil­lion a year, giv­ing the phar­ma gi­ant a clear shot at a new block­buster fran­chise.

The phar­ma gi­ant has kept to the in­side track at the FDA right through the re­view. The OK now puts No­var­tis in di­rect com­pe­ti­tion with Pfiz­er’s Ibrance (pal­bo­ci­clib), the pi­o­neer in this field, with Eli Lil­ly com­ing up from be­hind with abe­maci­clib.

The head­line fig­ure from ES­MO, based on a study that was wrapped ear­ly due to its suc­cess, was that ri­bo­ci­clib com­bined with letro­zole re­duced the risk of death or pro­gres­sion by 44% among first-line pa­tients com­pared to letro­zole alone.

Re­searchers al­so pegged me­di­an PFS of 25.3 months for Kisqali plus letro­zole com­pared to 16 months for letro­zole alone.

Bill Hin­shaw, head of No­var­tis US On­col­o­gy, told re­porters this af­ter­noon that they’ve priced this drug whole­sale at $10,950 for the 600 mg dose, $8,760 for their 400 mg dose and $4,380 at 200 mg for a 28-day pack­age with 21 pills, to al­low for flex­i­ble dos­ing and pric­ing. Pa­tients can al­so get the drug at no up­front cost as they’re work­ing out de­tails with their in­sur­er.

Sea­mus Fer­nan­dez at Leerink is go­ing with Pfiz­er in this show­down, not­ing that No­var­tis’ price is an 18% to 20% dis­count over Ibrance, which should help it bore its way on­to pay­ers’ for­mu­la­ries. Be he al­so as­serts that safe­ty is­sues with Kisqali will lim­it its mar­ket pen­e­tra­tion, fa­vor­ing Ibrance.

The close­ly-watched da­ta have kept this drug square­ly in the sights of an­a­lysts cov­er­ing this mar­ket. It’s a top-10 con­tender this year, in the front ranks of late-stage block­busters, with an EMA de­ci­sion ex­pect­ed in the sec­ond half of this year. There was no im­me­di­ate word on how much No­var­tis plans to charge for the drug.

Dr. Gabriel N. Hor­to­bagyi, MD An­der­son

“Kisqali is em­blem­at­ic of the in­no­va­tion that No­var­tis con­tin­ues to bring for­ward for peo­ple with HR+/HER2- metasta­t­ic breast can­cer,” said Bruno St­rig­i­ni, CEO, No­var­tis On­col­o­gy. “We at No­var­tis are proud of the com­pre­hen­sive clin­i­cal pro­gram for Kisqali that has led to to­day’s ap­proval and the new hope this med­i­cine rep­re­sents for pa­tients and their fam­i­lies.”

The UK’s As­tex is al­so chuffed. As­tex and No­var­tis be­gan col­lab­o­rat­ing on dis­cov­ery back in 2005, start­ing them down a path that led to this new drug.

Gabriel N. Hor­to­bagyi, MD, Pro­fes­sor of Med­i­cine, De­part­ment of Breast Med­ical On­col­o­gy, The Uni­ver­si­ty of Texas MD An­der­son Can­cer Cen­ter and MONALEESA-2 Prin­ci­pal In­ves­ti­ga­tor, not­ed:

These re­sults af­firm that com­bi­na­tion ther­a­py with a CDK4/6 in­hibitor like ri­bo­ci­clib and an aro­matase in­hibitor should be a new stan­dard of care for ini­tial treat­ment of HR+ ad­vanced breast can­cer.

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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UP­DAT­ED: Im­munomedics spells out PFS ben­e­fit of Trodelvy in mTNBC, hunt­ing a full OK just weeks af­ter ac­cel­er­at­ed ap­proval

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Sec­ond PhI­II study for Idor­si­a's sleep drug re­turns pos­i­tive re­sults, but al­so rais­es new ques­tions

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.

Douglas Love, Annexon CEO (Annexon)

IPO bound? Ac­tu­al­ly, An­nex­on was al­ready prepped and primed to toss its S-1 to Wall Street as in­vestors ral­lied

The Wall Street IPO shuffle generally calls for a little distance between the crossover ante and the Wall Street double, but with the window on the street wide open and biotech sizzling hot, who’s waiting?

The crew at Annexon didn’t leave anyone in suspense for long about their IPO plans. A day after the Bay Area biotech with clinical plans to target neurodegeneration quietly unveiled a $100 million raise, they were back with an S-1 outlining a pitch to double that — or more.

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Pfiz­er re-ups on Mis­sion Ther­a­peu­tics col­lab­o­ra­tion, lead­ing a $15M round and grab­bing first dibs on DUBs

Seven years after Pfizer first invested in Mission Therapeutics, a biotech that researches selectively inhibiting deubiquitylating enzymes (DUBs), the pharma giant is re-upping its commitment to the company in another sign of confidence in the field of protein degradation.

Pfizer’s VC arm is heading up a $15 million round, announced Monday morning, and increasing its overall stake in Mission. Pfizer is also entering into a licensing agreement that would give it first dibs at negotiating exclusivity after accessing certain DUB inhibitors and screening them for their potential as drugs.