A can­cer vac­cine that might work? Mod­er­na posts 'promis­ing' ear­ly snap­shot for Mer­ck-part­nered pro­gram and raids Bris­tol My­ers for a new can­cer R&D chief

Re­mem­ber that per­son­al­ized can­cer vac­cine Mod­er­na was de­vel­op­ing?

As the world awaits da­ta from its late-stage Covid-19 can­di­date, the mR­NA play­er is in­ter­rupt­ing its most in­ten­sive­ly watched pro­gram with an up­beat ear­ly snap­shot of da­ta from its mes­sen­ger RNA can­cer vac­cine mR­NA-4157. And the Mod­er­na team is us­ing the oc­ca­sion to in­tro­duce a new chief for their on­col­o­gy unit, raid­ed from Bris­tol My­ers Squibb’s can­cer group.

When com­bined with Mer­ck’s Keytru­da in the Phase I KEYNOTE-603 tri­al, re­searchers re­port, the vac­cine ap­pears some­what ef­fec­tive in shrink­ing tu­mors. Among 10 pa­tients with HPV-neg­a­tive head and neck squa­mous cell car­ci­no­ma, the over­all re­sponse rate is 50%: 2 com­plete re­spons­es and 3 par­tial re­spons­es. And it is trans­lat­ing to con­sid­er­able ben­e­fit over the PD-1 in­hibitor alone:

Me­di­an pro­gres­sion free sur­vival (mPFS) is 9.8 months, which com­pares fa­vor­ably to the pub­lished ORR and mPFS of 14.6% and 2.0 months re­spec­tive­ly, for Keytru­da monother­a­py. In­clud­ing four pa­tients with sta­ble dis­ease, the Dis­ease Con­trol Rate (DCR) is 90% (9/10). Me­di­an du­ra­tion of re­sponse has not been reached.

“The da­ta are pre­lim­i­nary and the sam­ple size is small, but it is promis­ing,” Uni­ver­si­ty of Ari­zona pro­fes­sor Julie Bau­man, who’s pre­sent­ing the in­ter­im da­ta at The So­ci­ety for Im­munother­a­py of Can­cer’s An­nu­al Meet­ing, said in a state­ment. “A phase I tri­al is about safe­ty first and fore­most, and we now know this treat­ment is safe and tol­er­a­ble. But, we al­so have a strong sig­nal to point us to fur­ther study this in head and neck can­cer.”

Jef­feries an­a­lyst Michael Yee ob­served that the re­sults com­pare fa­vor­ably even with the Keytru­da/chemo com­bo, the stan­dard of care in first-line treat­ment, which yields an ORR of 36% and mPFS of 4.9 months.

“While 4/5 re­spon­ders achieved PR af­ter 2 dos­es of Keytru­da pri­or to mR­NA-4157, all 4 re­spons­es deep­ened af­ter mR­NA-4157 ad­min­is­tra­tion w/ 2 PRs con­vert­ing to CRs,” he wrote “Add’l, one pt pro­gressed on Keytru­da, but start­ed to re­spond af­ter mR­NA-4157.”

In the mi­crosatel­lite sta­ble col­orec­tal car­ci­no­ma group, though, no re­spons­es were ob­served out of 17 pa­tients.

With a clean tol­er­a­bil­i­ty so far, Mod­er­na now plans to ex­pand the HN­SCC co­hort to 40 pa­tients. For each one, the com­pa­ny tai­lor makes a vac­cine with mes­sen­ger RNA that en­codes up to 20 neoepi­topes that its al­go­rithms pre­dict would stir up the strongest im­mune re­sponse.

It will be do­ing so un­der the lead­er­ship of Praveen Aa­nur, who’s jump­ing from Bris­tol My­ers Squibb to be­come Mod­er­na’s ther­a­peu­tic area head for on­col­o­gy de­vel­op­ment. Aa­nur spent the last 7 years work­ing on Bris­tol My­ers’ im­muno-on­col­o­gy pipeline. He re­ceived an MBBS from Ban­ga­lore Uni­ver­si­ty, an MPH from the Uni­ver­si­ty of Al­aba­ma and an MBA from Co­lum­bia Uni­ver­si­ty School of Busi­ness.

Can­cer vac­cines are gain­ing trac­tion among hefty bio­phar­ma play­ers again as new biotechs spring up fol­low­ing a se­ries of clin­i­cal dis­as­ters among the gen-1 play­ers. Re­gen­eron is al­lied with BioN­Tech — now a house­hold name thanks to its Pfiz­er-part­nered Covid-19 ef­fort — on a Phase II test­ing a 4-anti­gen can­di­date, while Roche re­cent­ly shelled out $200 mil­lion for a neoanti­gen pro­gram.

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.”

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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FDA un­veils new draft guid­ance to help with oligonu­cleotide ther­a­peu­tics de­vel­op­ment

While oligonucleotides, a wide variety of synthetically modified RNA or RNA/DNA hybrids that bind to a target RNA sequence to alter RNA and/or protein expression, have been winning approvals in recent years (e.g. Novartis’ cholesterol drug Leqvio), the regulatory agency is offering new draft guidance for those looking to follow a similar path.

The non-binding guidance, titled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance for Industry” deals with pharmacokinetic, pharmacodynamic, and safety assessments required as part of oligonucleotide therapeutics R&D.

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