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.

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

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

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.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but 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.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.