Cure­Vac’s lead mR­NA pro­gram flunks a crit­i­cal PhI­Ib study, rais­ing ques­tions for high-pro­file biotech uni­corns

In­g­mar Ho­err

Cure­Vac’s lead­ing mR­NA pro­gram CV9104, a prostate can­cer vac­cine, has failed a crit­i­cal Phase IIb study. And that sting­ing set­back for the most ad­vanced clin­i­cal study in the field will raise fresh ques­tions about a com­plex new tech­nol­o­gy that has at­tract­ed bil­lions of dol­lars in in­vest­ment cash.

The Ger­man biotech re­port­ed that not on­ly did CV9104 fail the pri­ma­ry end­point on im­prov­ing over­all sur­vival, there was al­so no im­prove­ment on pro­gres­sion-free sur­vival over a place­bo.

Cure­Vac’s can­cer vac­cine is a 6-mR­NA con­coc­tion aimed at whip­ping up an im­mune sys­tem at­tack by en­cod­ing for 6 anti­gens over ex­pressed in prostate can­cer.

The Ger­man biotech – run by CEO In­g­mar Ho­err – has raised $360 mil­lion for its work, pri­mar­i­ly backed by Ger­man bil­lion­aire Di­et­mar Hopp along with ad­di­tion­al fund­ing from Bill Gates’ foun­da­tion, both in­trigued by the rev­o­lu­tion­ary po­ten­tial of in­struct­ing cells to pro­duce ther­a­peu­tics.

The most promi­nent com­pa­ny in the mes­sen­ger RNA space is Mod­er­na, a Boston-based biotech which has raised $1.9 bil­lion and just this week de­tailed a full pipeline of 5 clin­i­cal-stage ther­a­pies and an­oth­er 7 pre­clin­i­cal projects. Mod­er­na CEO Stephane Ban­cel is al­so work­ing on a can­cer vac­cine with Mer­ck, but he’s al­ready sought to dis­tin­guish their ap­proach to an in­di­vid­u­al­ly tai­lored ther­a­py with the shared-anti­gen ap­proach that Cure­Vac has.

I asked Ban­cel about the Cure­Vac set­back, and he re­spond­ed:

“Can­cer vac­cines con­tin­ues to be a high risk en­deav­or, which is why we part­nered with Mer­ck, a leader in im­muno-on­col­o­gy.”

Whether that kind of dis­tinc­tion will help Mod­er­na avoid get­ting dinged by Cure­Vac’s fail­ure, though, has yet to be seen. Mod­er­na has come un­der re­peat­ed at­tack by STAT’s Dami­an Garde, who has fo­cused on ques­tions re­gard­ing the tech­nol­o­gy and Mod­er­na’s cau­tious ap­proach in tack­ling vac­cines first, with a lead­ing ef­fort on pan­dem­ic flu. But that’s the long game that Ban­cel is de­ter­mined to play as he lines up his own first round of da­ta on those pro­grams deemed most like­ly to suc­ceed.

The lat­est fail­ure at Cure­Vac al­so fol­lows a wave of clin­i­cal flops for can­cer vac­cines in gen­er­al, which proved far too weak to di­rect a sig­nif­i­cant im­mune sys­tem at­tack on can­cer. That first-wave fail­ure was fol­lowed by check­point in­hibitors, which ef­fec­tive­ly take the brake off the im­mune sys­tem, as well as per­son­al­ized vac­cines that seek out the in­di­vid­ual anti­gens that will make the most com­pelling dif­fer­ence for pa­tients. But that is al­so still very ear­ly in the de­vel­op­ment process.

Cure­Vac’s next step will like­ly fo­cus on a com­bo ap­proach, match­ing their mR­NA tech with check­points, chemo and so on, which is al­ready part of the plan.

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

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

Top Har­vard chemist caught up in FBI’s 'T­hou­sand Tal­ents' drag­net, ac­cused of ly­ing about Chi­nese con­nec­tions, pay

The FBI’s probe into the alleged theft of R&D secrets by Chinese authorities has drawn Harvard’s top chemist into its net.

The agency accused Charles M. Lieber, who chairs the university’s chemistry and chemical biology department, with lying about his involvement in China’s Thousand Talents campaign, which was established as a way of drawing in innovators from around the world. And the scientist, 60, was charged with making false statements about his ties to China.

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Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Passage Bio — Jim Wilson’s self-described “legacy company” — has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”

Pfiz­er ax­es 6 ear­ly to late-stage can­cer stud­ies from the pipeline — with one oth­er cut for sick­le cell dis­ease

Pfizer trimmed a group of 3 R&D programs using their PD-L1 Bavencio — partnered with Merck KGaA — in their latest pipeline cull.

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UP­DAT­ED: In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

Incyte’s efforts to breathe a second life into ruxolitinib — its JAK inhibitor sold in pill form as Jakafi — has been greeted with clear, if preliminary and unsurprising, Phase III success.

Topline data from the TRuE-AD2 cements ruxolitinib’s foundational importance for Incyte, and gives analysts hope that there might yet be room for growth in a pipeline that’s suffered multiple R&D setbacks.

Stephen Hahn, AP

The FDA un­veils a new reg­u­la­to­ry frame­work to speed along gene ther­a­pies, re­ward­ing the lead­ing play­ers

Bioregnum Opinion Column by John Carroll

The emphasis at the FDA over the past 5 years or so has been on assisting drug developers as much as they can to speed up regulatory reviews and push more drugs into the market. And they are now crafting a final set of regulations aimed at flagging through a whole new generation of gene therapies in clinical testing at a rapid clip.

In a set of 6 prospective guidances posted on the FDA web site Tuesday morning, FDA commissioner Stephen Hahn committed the agency to staying flexible in handing out designations that are critical to gaining early approvals for drugs that claim to be once-and-done but don’t have anything close to the data needed to prove it.

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