Prep­ping for a rapid-fire launch, Kite Phar­ma gets a pass from FDA on axi-cel pan­el re­view

The FDA has ev­i­dent­ly learned all it wants at this point from its out­side on­col­o­gy ex­perts on CAR-T. Kite Phar­ma’s close­ly-watched CAR-T drug axi-cel is get­ting a pass on an ad­comm meet­ing, which may sig­nal a quick thumbs up from reg­u­la­tors.

Kite $KITE ex­ecs think so, say­ing to­day that they will be ful­ly launch-ready by Sep­tem­ber in case an ear­ly OK comes through. And that bull­ish stance earned a 7% boost to its share price to­day.

David Chang, Kite

In a Q2 call with an­a­lysts Tues­day morn­ing, Kite R&D chief David Chang told an­a­lysts the FDA had no­ti­fied the biotech that reg­u­la­tors will not be or­ga­niz­ing a pan­el re­view with out­side ex­perts for axi-cel. No­var­tis was first up in Ju­ly, gain­ing unan­i­mous back­ing for CTL019 from an FDA pan­el in the lead­up to an ear­ly-Oc­to­ber PDU­FA date.

No­var­tis had been seen as the clear front-run­ner in the race to get on the mar­ket first, but with the FDA fin­ish line blur­ring, they may now both launch al­most si­mul­ta­ne­ous­ly.

“We are ex­treme­ly en­cour­aged by the re­cent ad­vi­so­ry com­mit­tee meet­ing” for No­var­tis, Chang said, which he sees as a like­ly har­bin­ger for Kite. “The FDA has in­formed us that they will not sched­ule an ad­vi­so­ry com­mit­tee meet­ing” for axi-cel. This fol­lows an FDA in­spec­tion of its man­u­fac­tur­ing fa­cil­i­ty and its treat­ment cen­ters in the lead-up to an ac­cel­er­at­ed re­view and fi­nal de­ci­sion.

Kite faces a No­vem­ber 29 dead­line for its mar­ket­ing de­ci­sion from the FDA, but the com­pa­ny says it’s ready to start mak­ing the per­son­al­ized ther­a­py and start ship­ping al­most im­me­di­ate­ly.

These pi­o­neer­ing CAR-T drugs of­fer a prover­bial game-chang­ing ap­proach in treat­ing blood can­cers. T cells are ex­tract­ed from pa­tients and armed with chimeric anti­gen re­cep­tors, turn­ing them in­to can­cer fight­ers that are pumped in­to a po­tent mix that is then in­fused in­to the pa­tient.

To say that Kite has been an­tic­i­pat­ing the launch for sev­er­al years now is an un­der­state­ment. Axi-cel is wide­ly viewed as a block­buster in the mak­ing, and Kite has been build­ing man­u­fac­tur­ing op­er­a­tions and a com­mer­cial group with plans to hit the ground run­ning, hit­ting a vein-to-vein turn­around time on this ther­a­py of 17 days. In the mean­time, its R&D group to­day sig­naled that they have filed for an IND to start Phase I work on KITE-585, its next-gen ap­proach that tar­gets BC­MA.

“With the an­tic­i­pat­ed events on the hori­zon for the re­main­der of 2017,” said CEO Arie Bellde­grun, “the po­ten­tial for CAR-T to be­come one of the most pow­er­ful an­ti-can­cer agents for cer­tain pa­tients may fi­nal­ly be re­al­ized.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Robert Gould, Fulcrum Therapeutics CEO

Ful­crum stum­bles in PhII of old GSK drug, send­ing shares tum­bling

Investors are selling off shares of Fulcrum Therapeutics $FULC after their lead drug failed in a Phase II trial.

The company, founded three years ago on new research techniques such as CRISPR screening, isolated a gene called DUX4 they believed to have a central role in facioscapulohumeral muscular dystrophy, where patients’ muscle dies and is replaced by fat. And to target it, they licensed a GlaxoSmithKline drug that had failed as a cardio drug.

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Eisai moves to 200 Metro Blvd. by late 2021 (ON3)

Ei­sai is cre­at­ing a new US cor­po­rate, R&D HQ in Roche’s old Nut­ley, NJ cam­pus

Eight years after Roche pulled up stakes from Nutley, NJ in a major R&D reorganization, Japan’s Eisai is moving its US corporate and research hub into their old campus.

Now the ON3 property, Eisai — a longtime Biogen partner focused on neurodegenerative disorders like Alzheimer’s — will bring together a staff of up to 1,200 employees. And execs are pitching the move to the New Jersey campus as a cultural game-changer.

Lig­and scoops up Pfenex for up to $516M, adding pro­teins to their an­ti­body chick­ens and de­liv­ery tech

The technology hunting folks over at Ligand Pharmaceuticals have picked up a new one from across town, for a significant price.

Ligand has acquired fellow San Diego-based biotech Pfenex and their protein expression platform for $438 million cash, plus $78 million in contingent value agreements should an undisclosed milestone be hit before the end of next year.  The deal pays $12 per share, or $4.34 more than what Pfenex had been trading at before the announcement.