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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

John Leonard, Intellia CEO

In­tel­li­a's CRISPR pro­gram that ed­its genes di­rect­ly in pa­tients shows dura­bil­i­ty in AT­TR amy­loi­do­sis

The first in vivo CRISPR/Cas9 gene editing program has some new durability data showing sustained reduction of a toxic protein in ATTR amyloidosis at all four dose levels in a small 15-patient study.

Intellia Therapeutics presented the much-anticipated data for its Regeneron-collaborated NTLA-2001 Friday morning, adding to the initial Phase I results it first delivered almost a year ago to the day.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”