Deaths de­rail Juno's launch count­down, giv­ing Kite and No­var­tis the lead

The FDA’s hold on Juno Ther­a­peu­tics’ lead CAR-T pro­gram, JCAR015, last­ed on­ly six days. But the de­rail­ment was se­ri­ous enough to push its ex­pect­ed ap­proval date from 2017 back in­to 2018, leav­ing Kite Phar­ma and No­var­tis an­gling for the first ap­provals in the field in 2017.

Juno CEO Hans Bish­op

“Re­gard­ing the ROCK­ET tri­al,” Juno CEO Hans Bish­op told an­a­lysts Thurs­day evening, “the process of get­ting IRB ap­proval across mul­ti­ple sites along with the gat­ed en­roll­ment for the next six pa­tients leaves us to now es­ti­mat­ing ap­proval as ear­ly as the first half of 2018.”

The new H1 2018 pro­jec­tion marks a set­back for Juno, which had been seen as run­ning neck and neck with Kite in the race to get the land­mark ap­proval for a new can­cer ther­a­py that takes cells from pa­tients and reengi­neers them in­to a can­cer cell at­tack ve­hi­cle.

The hold last month, fol­low­ing the death of sev­er­al pa­tients from lethal cas­es of cere­bral ede­ma, stunned long­time ob­servers of Juno. The biotech quick­ly and suc­cess­ful­ly ap­pealed to the agency to lift the hold, say­ing they be­lieved that adding flu­dara­bine to its pre­con­di­tion­ing reg­i­men for pa­tients — prep­ping them to bet­ter re­spond to their CAR-T — had cre­at­ed a tox­ic com­bi­na­tion with the ther­a­py, killing 4 pa­tients. Reg­u­la­tors, who had al­so placed the same ther­a­py briefly on hold af­ter cy­tokine re­lease syn­drome al­so killed some pa­tients ear­ly on, were quick to re­spond af­fir­ma­tive­ly.

The de­lay leaves JCAR015 poised to en­ter the mar­ket just a year ahead of JCAR017. Bish­op de­scribes the sec­ond drug as “the back­bone of our CD19 fran­chise,” which is aimed at NHL, pe­di­atric and adult ALL and CLL.

Juno, though, is al­so mov­ing a va­ri­ety of pro­grams across a broad R&D front, and is now go­ing af­ter mul­ti­ple myelo­ma in a new pact that ropes in long­time col­lab­o­ra­tors at Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter.

In a sep­a­rate press re­lease, the Seat­tle-based biotech an­nounced Thurs­day evening that it had struck a deal with MSK and Eu­re­ka Ther­a­peu­tics for IP on bind­ing do­mains need­ed to com­mer­cial­ize a CAR-T for mul­ti­ple myelo­ma. These bind­ing do­mains were de­vel­oped in a pact that Eu­re­ka and MSK had struck ear­li­er.

That deal is par­tic­u­lar­ly sig­nif­i­cant, as it in­volves a ful­ly-hu­man bind­ing do­main tar­get­ing B-cell mat­u­ra­tion anti­gen (BC­MA), along with bind­ing do­mains against two ad­di­tion­al undis­closed mul­ti­ple myelo­ma tar­gets. The BC­MA pact puts Juno on a po­ten­tial col­li­sion course with blue­bird bio, which al­so chose that tar­get in a col­lab­o­ra­tion its pur­su­ing with Cel­gene. Those two play­ers re­struc­tured their deal last year to con­cen­trate on BC­MA and Cel­gene lat­er signed a ma­jor pact with Juno as well.

Pfiz­er and Cel­lec­tis are al­so aim­ing at BC­MA in their col­lab­o­ra­tion, but they’re con­cen­trat­ing on a sec­ond-gen, off-the-shelf prod­uct.

Juno CSO Hy Lev­it­sky

“We ex­pect the BC­MA CAR to en­ter hu­man test­ing as ear­ly as the first half 2017,” Juno CSO Hy Lev­it­sky told an­a­lysts. “We’re op­ti­mistic the CAR T cell ther­a­py can be an im­por­tant com­po­nent in treat­ing pa­tients with mul­ti­ple myelo­ma. And we are pleased to bring ad­di­tion­al ful­ly hu­man body do­main against BC­MA and oth­er tar­gets in­to our pro­gram. We be­lieve that a mul­ti-pronged ap­proach may be nec­es­sary to treat this dis­ease and hence the im­por­tance of ac­cess to sev­er­al hu­man con­structs spe­cif­ic for more than one tar­get.”

This isn’t the first such pact that Juno and Eu­re­ka have struck. Back at the be­gin­ning of the year the two com­pa­nies struck a deal on a ful­ly hu­man bind­ing do­main that tar­gets MUC16. These bind­ing do­mains play a key role in im­prov­ing cell per­sis­tence, amp­ing up their ef­fect on pa­tients. And the in­tense ri­val­ry to dom­i­nate the first wave of com­mer­cial CAR-Ts to hit the mar­ket has spurred an arms race for the best tech with the most po­ten­tial.

Novartis CEO Vas Narasimhan (via AP Images)

Go­ing 0-for-4 on a big PhI­II pro­gram, No­var­tis scraps their top asth­ma prospect and sig­nal big trou­ble for a lit­tle biotech

Novartis this morning reported that their asthma drug fevipiprant flunked two more Phase III studies, crushing their hopes in what had been a star H2 development program at the pharma giant.

Back in October Novartis buried the news that fevipiprant had failed the first 2 Phase III studies in their Q3 report, noting a lack of success in scoring an improvement in FEV1 among moderate patients. But the pharma giant immediately shifted focus to LUSTER 1 and 2 in moderate to severe patients, noting that they were the core studies needed for registration and a blockbuster future.

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Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary: about $752 million in total.  The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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