Ker­ris­dale 2-0 this week: Bavar­i­an Nordic's can­cer vac­cine is a bust in PhI­II

An­oth­er can­cer vac­cine has bit­ten the dust.

Six long years af­ter start­ing their Phase III tri­al for the prostate can­cer vac­cine Prost­vac, an in­de­pen­dent mon­i­tor­ing com­mit­tee says it’s time to sim­ply scrap Bavar­i­an Nordic’s {$BA­VA.CO} study af­ter con­clud­ing it wasn’t work­ing at a fi­nal in­ter­im analy­sis stage.

In­vestors who had stuck with the Dan­ish biotech weren’t pleased. Its shares lost half their val­ue, and one an­a­lyst, Pe­ter Welford at Jef­feries, says the flop could read through to a fol­low up pro­gram. He wrote:

We note BA­VA al­so has CV-301 in Phase II for blad­der can­cer and en­ter­ing Phase Ib/II for lung can­cer (NSCLC) +Keytru­da, plus Phase II for urothe­lial car­ci­no­ma +Tecen­triq. CV-301 com­pris­es a sim­i­lar back­bone and TRI­COM co-stim­u­la­to­ry mol­e­cules to PROST­VAC with dif­fer­ent anti­gens. Hence, giv­en the sig­nif­i­cant set­back for PROST­VAC, we ex­pect the mar­ket to now more heav­i­ly dis­count CV-301, with our NPV c.DKK30/share (9%).

Paul Chap­lin, Bavar­i­an Nordic

Bris­tol-My­ers Squibb paid $60 mil­lion up front to li­cense the can­cer vac­cine, with $80 mil­lion lined up for the op­tion on Phase III da­ta, with hun­dreds of mil­lions in prospec­tive mile­stones.

That all just went poof.

The late-stage fail­ure fol­lows a short at­tack from Sahm Ad­ran­gi’s Ker­ris­dale Cap­i­tal, who called it two years ago as a se­ries of can­cer vac­cines like Stimu­vax fell short of the mark.

“The his­to­ry of ther­a­peu­tic can­cer vac­cines is two decades of un­mit­i­gat­ed fail­ure,” Ad­ran­gi wrote in 2015. “We ex­pect noth­ing dif­fer­ent from Bavar­i­an Nordic.”

The set­back al­so fol­lows mul­ti­ple de­lays, which Chap­lin had cheered as an in­di­ca­tion that tak­ing more time to reach the num­ber of events need­ed for the read­out was an in­di­ca­tion of suc­cess.

That the­o­ry, which has been used by North­west Bio and oth­ers, has been proven wrong time and again.

Ker­ris­dale Cap­i­tal has been proven right twice this week, with Sage’s ug­ly mis­fire on SRSE prov­ing that the drug was no bet­ter than a place­bo in get­ting a re­sponse. But in that case, be­ing right didn’t mean win­ning a cash re­ward, as Sage’s stock went up af­ter the Phase III fail­ure.

As far as the stock mar­ket is con­cerned now, these are spe­cial times, when in­vestor re­ac­tions can be marked­ly dif­fer­ent than the norm.

I asked Ad­ran­gi what he thought about the fail­ure. His re­sponse:

“Prost­vac’s fail­ure is what any ob­serv­er should have ex­pect­ed giv­en the un­ex­cep­tion­al ear­li­er stage treat­ment arm sur­vival. The vac­cine’s un­so­phis­ti­cat­ed de­sign was doomed for fail­ure — sim­ply ad­min­is­ter­ing tu­mor-as­so­ci­at­ed anti­gens and hop­ing for an ef­fec­tive im­mune re­sponse al­ways seemed to us a dead end.”
“We are ex­treme­ly dis­ap­point­ed for pa­tients that this study of Prost­vac as monother­a­py was not suc­cess­ful,” said Bavar­i­an Nordic CEO Paul Chap­lin. “While this is cer­tain­ly not the de­sired out­come, we re­main stead­fast be­liev­ers in the pow­er of com­bi­na­tion treat­ments, in­clud­ing im­munother­a­pies, to trans­form the fu­ture of can­cer ther­a­pies.”

An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Can an FDA hus­tle up on trastuzum­ab be far be­hind?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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