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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Mene Pangalos (AstraZeneca via YouTube)

As­traZeneca shuts the PhI­II door for Ion­is' PC­SK9 drug de­spite pos­i­tive PhI­Ib

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

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Up­dat­ed: Bio­gen throws it­self back in­to mud­dled da­ta ar­gu­ments with more de­tails on its an­ti­sense ALS drug

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.