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.”

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

UP­DAT­ED: Ac­celeron res­ur­rects block­buster hopes for so­tater­cept with pos­i­tive PhII — and shares rock­et up

Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

“We’re thrilled to report such positive topline results from the PULSAR trial,” Acceleron CEO Habib Dable said in a statement. The company said in a conference call they plan on discussing a Phase III trial design with regulators.

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Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”

Pfiz­er ax­es 6 ear­ly to late-stage can­cer stud­ies from the pipeline — with one oth­er cut for sick­le cell dis­ease

Pfizer trimmed a group of 3 R&D programs using their PD-L1 Bavencio — partnered with Merck KGaA — in their latest pipeline cull.

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UP­DAT­ED: In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

Incyte’s efforts to breathe a second life into ruxolitinib — its JAK inhibitor sold in pill form as Jakafi — has been greeted with clear, if preliminary and unsurprising, Phase III success.

Topline data from the TRuE-AD2 cements ruxolitinib’s foundational importance for Incyte, and gives analysts hope that there might yet be room for growth in a pipeline that’s suffered multiple R&D setbacks.

Stephen Hahn, AP

The FDA un­veils a new reg­u­la­to­ry frame­work to speed along gene ther­a­pies, re­ward­ing the lead­ing play­ers

Bioregnum Opinion Column by John Carroll

The emphasis at the FDA over the past 5 years or so has been on assisting drug developers as much as they can to speed up regulatory reviews and push more drugs into the market. And they are now crafting a final set of regulations aimed at flagging through a whole new generation of gene therapies in clinical testing at a rapid clip.

In a set of 6 prospective guidances posted on the FDA web site Tuesday morning, FDA commissioner Stephen Hahn committed the agency to staying flexible in handing out designations that are critical to gaining early approvals for drugs that claim to be once-and-done but don’t have anything close to the data needed to prove it.

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