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.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.