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.”

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.