An­oth­er can­cer vac­cine play out of Gene­va shoots for 'hard end­point' in PhII head and neck can­cer test

Af­ter qui­et­ly plow­ing the arid can­cer vac­cine field for a decade, a low pro­file Swiss biotech is throw­ing it­self in the ring as a play­er to watch in the fre­net­ic race to ex­tend the ben­e­fits of im­munother­a­pies to sol­id tu­mors.

Bernard Mach

Co-found­ed by biotech vet Bernard Mach — who was in­volved in the cre­ation of Bio­gen and Novim­mune — and his son Nico­las, Max­i­VAX is laser-fo­cused on de­vel­op­ing a two-part can­cer vac­cine that ac­ti­vates the im­mune sys­tem to at­tack can­cer. And it has been award­ed a €2.785 mil­lion ($3.07 mil­lion) grant from the Eu­ro­pean Com­mis­sion — on top of CHF 5 mil­lion ($5.01 mil­lion) from a Se­ries B2 round — to push through a Phase II study in head and neck can­cer in Switzer­land and kick off a US study for an undis­closed rare tu­mor.

Much has been made of can­cer vac­cines’ po­ten­tial to com­ple­ment im­mune check­point in­hibitors, which can be in­cred­i­bly ef­fec­tive in sub­sets of pa­tients, some­times as few as 20%. But the first gen­er­a­tion of can­cer vac­cines, large­ly made of pep­tides de­rived from can­cer cells, has failed to in­duce the im­mune ef­fects need­ed to make a dif­fer­ence on their own.

Dim­itrios Goundis

A key is­sue, Max­i­VAX CEO Dim­itrios Goundis said, is that pep­tides car­ry mu­ta­tions that vary from pa­tient to pa­tient. Their so­lu­tion: Iso­late the whole tu­mor cells, con­tain­ing the com­plete anti­genic reper­toire for the im­mune cells to pro­file and tar­get, in­ac­ti­vate them, then im­plant them sub­cu­ta­neous­ly next to an ad­ju­vant meant to sus­tain the im­mune re­sponse.

“So our ther­a­py ba­si­cal­ly is a vac­cine, which is pa­tient spe­cif­ic, and an im­mune boost­ing agent (GM-CSF) which is de­liv­ered by en­cap­su­lat­ed ge­net­i­cal­ly mod­i­fied cells, which al­low con­tin­ued de­liv­ery of GM-CSF over sev­er­al days at the site of vac­ci­na­tion,” he told End­points News. “And with that we ad­dress we think all the weak­ness­es that we’ve iden­ti­fied over the course of the last 20, 30 years when peo­ple start to look at vac­cines against can­cers in a more sys­tem­at­ic way.”

Nico­las Mach

GM-CSF, or gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor, is a nat­u­ral­ly oc­cur­ring im­mune mod­u­la­tor that has a rel­a­tive­ly short half life, nor­mal­ly dis­ap­pear­ing with­in a cou­ple hours in the blood­stream. To keep it flow­ing, Max­i­VAX is in­sert­ing a gene in­to hun­dreds of thou­sands of cells to pro­duce a steady sup­ply of GM-CSF such as im­mune cells that can stay en­er­gized as they get fa­mil­iar­ized with ir­ra­di­at­ed tu­mor cells.

The im­prove­ment should be clear cut, he said. While check­points have ex­tend­ed me­di­an sur­vival in ad­vanced and re­frac­to­ry head and neck can­cer from three to six months, that still means half of the pa­tients don’t live that long. For the on­go­ing Phase II, for which they are re­cruit­ing around 40 pa­tients across six sites, Max­i­VAX will be mon­i­tor­ing the num­ber of pa­tients who pass the 6-month sur­vival thresh­old as the main ef­fi­ca­cy mea­sure.

“It’s a hard end­point,” he said. “So we’re not on­ly look­ing at whether or not the tu­mor is re­duced in size or dis­ap­pears, but al­so what is the ef­fect of that in pro­long­ing the life of the pa­tients.”

He ex­pects the fi­nal re­sults from the open-la­bel tri­al to be in around 2021. Be­fore that, Max­i­VAX’s small team of 10 will al­so ini­ti­ate a Phase II for which they have al­ready ob­tained an IND in the US.

The new funds from pri­vate in­vestors — Max­i­VAX has man­aged to stay away from ven­ture cap­i­tal­ists in rais­ing over $15 mil­lion so far — will al­so go to­ward build­ing out man­u­fac­tur­ing.

Right now the biotech re­lies on the Gene­va Hos­pi­tal, where Nico­las Mach is head of on­col­o­gy, to iso­late the tu­mor cells and to gen­er­ate and fill the cap­sules with GM-CSF pro­duc­ing cells. Its of­fices are lo­cat­ed in the neigh­bor­hood, which al­so hous­es AMAL Ther­a­peu­tics, an­oth­er next-gen can­cer vac­cine play­er re­cent­ly bought out by Boehringer In­gel­heim.

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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Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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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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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. 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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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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