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.

Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slapdown, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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Cedric Francois, Apellis CEO (Apellis)

Apel­lis joins the grow­ing num­ber of bio­phar­mas scrap­ping a failed Covid-19 pro­gram af­ter an ear­ly flop

The global pandemic set off a frenzy of R&D activity as biotechs around the world scrambled to see if they could come up with a new medication or vaccine to help fight back. But even as the mRNA standouts are highlighting the market El Dorado open to successful teams, the failures are starting to pile up.

Thursday afternoon it was Apellis’ $APLS turn to deep-six a new drug.

The biotech reports that their C3 therapy APL-9 had failed to move the needle on mortality when combined with standard of care, as compared to SOC alone.

Norbert Bischofberger (Kronos)

Kro­nos seals pact with reg­u­la­tors to hunt AML with pre­vi­ous­ly off-lim­its bio­mark­er end­point

As head of R&D at Gilead, Norbert Bischofberger shelved the SYK inhibitor entospletinib after it proved to need too lengthy a development cycle to win approval. The FDA heard those concerns and will now give entospletinib, once again under Bischofberger’s watchful eye at Kronos, a faster shot on goal.

Kronos has reached an agreement with the FDA to conduct a Phase III trial with a unique primary endpoint, the company announced Thursday, one that it hopes will accelerate entospletinib’s path forward in a certain type of acute myeloid leukemia. The endpoint is measurable residual disease (MRD) negativity, which Kronos says can paint a clearer picture when it comes to the study’s complete response rate.