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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Jean-Jacques Bienaime, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Lynn Seely, Myovant CEO

My­ovant’s re­l­u­golix wins a piv­otal prostate can­cer show­down with an old stan­dard — com­ing down to the wire on ap­provals

Myovant $MYOV has rounded the final turn in its development race to get relugolix down to the regulatory wire at the FDA. And the biotech joined the virtual crowd at ASCO with the kind of data needed to keep the investor crowd’s attention.

Much of the attention on the drug has been focused on uterine fibroids, where AbbVie just scored a regulatory win for their rival drug Oriahnn (elagolix) as the biotech posted results in prostate cancer at the ASCO meeting.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

File photo (Endpoints News)

In a sting­ing set­back, Pfiz­er’s can­cer block­buster Ibrance flops in key ad­ju­vant set­ting

One of Pfizer’s top, long-running R&D catalysts has gone up in smoke, and it took an $11 billion bite of their market cap in the process.

The monitoring committee determined that Pfizer’s adjuvant study using Ibrance combined with standard endocrine therapy in an adjuvant setting for early-stage breast cancer has officially failed to make the cut. The combo failed to beat the standard alone, tripping over the futility analysis. And the Pfizer team will now wrap the study early after pumping up hopes that their blockbuster cancer therapy could find billions more by proving its efficacy for disease-free survival in a major area — something AstraZeneca just accomplished with Tagrisso to great fanfare.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.