Kite catch­es the in­side track on the last leg of a race to the fin­ish line with No­var­tis on a CAR-T first

David Chang, Kite

The race be­tween No­var­tis $NVS and Kite $KITE to win the first FDA ap­proval of a CAR-T for can­cer is now in the fi­nal stretch, and Kite just short­ened its dis­tance to the fin­ish line.

The biotech an­nounced this morn­ing that the FDA is pro­vid­ing a pri­or­i­ty re­view for their drug KTE-C19 (axi­cab­ta­gene ciloleu­cel), which will cut the time it takes for reg­u­la­tors to com­plete their ex­am­i­na­tion from 10 months down to 6. The FDA’s PDU­FA date is No­vem­ber 29.

Kite’s big ri­val No­var­tis won a pri­or­i­ty re­view two months ago, which should pro­vide a com­fort­able lead for the first de­ci­sion on its land­mark drug. Both rep­re­sent the cut­ting edge of a new class of drug that ex­tracts cells from pa­tients and then reengi­neers them in­to an at­tack weapon tar­get­ing can­cer cells.

Both KTE-C19 and No­var­tis’ CTL019 are tapped as two of the top block­busters in the in­dus­try pipeline. They’re go­ing af­ter dif­fer­ent in­di­ca­tions on the first round, with Kite ini­tial­ly tak­ing on drug-re­sis­tant non-Hodgkin lym­phoma. But No­var­tis — tak­ing its first shot at chil­dren with acute lym­phoblas­tic leukemia — will switch fo­cus to its JULI­ET study for some head-to-head com­par­isons on dif­fuse large B-cell lym­phoma that will at­tract plen­ty of in­tense at­ten­tion, as well as spec­u­la­tion over the im­pact it will have on Kite’s stock price.

That new da­ta — which al­ready earned a spe­cial break­through ti­tle at the FDA — should ar­rive next month.

In the mean­time, reg­u­la­tors will be fol­low­ing through on re­views for two des­ig­nat­ed ‘break­through’ drugs, mak­ing it at least pos­si­ble for the two drugs to reach an ex­pert pan­el at about the same time.

Kite had man­aged to get through its reg­is­tra­tion pro­gram with­out any ex­cep­tion­al safe­ty is­sues — right un­til it was hit a few weeks ago with the death of a pa­tient due to cere­bral ede­ma. The biotech went to some pains to note just how sick this pa­tient was when he died, but its stock slid as in­vestors con­sid­ered the im­pli­ca­tions. Juno’s lead CAR-T was knocked out of the race when 5 pa­tients died of cere­bral ede­mas, or brain swelling, in­clud­ing 2 who died af­ter the FDA de­cid­ed to lift a clin­i­cal hold on the pro­gram af­ter just a few days.

Those deaths and all the safe­ty is­sues fac­ing CAR-T ther­a­pies will now get care­ful scruti­ny at the FDA.

“Pa­tients with re­frac­to­ry ag­gres­sive NHL face a dire prog­no­sis with on­ly a 50 per­cent chance of sur­viv­ing six months. This un­der­scores the ur­gent med­ical need for these pa­tients and why every day mat­ters, from de­vel­op­ment to man­u­fac­tur­ing to clin­i­cal ex­pe­ri­ence,” said David Chang, ex­ec­u­tive vice pres­i­dent of re­search and de­vel­op­ment and CMO of Kite. “We firm­ly be­lieve in the po­ten­tial for axi­cab­ta­gene ciloleu­cel to ad­dress this need and forge a new path for the fu­ture of cell ther­a­py.”

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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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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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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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.