The top win­ners and losers on AS­CO ab­stract night: Loxo, Blue­print, Jounce, Mer­ck KGaA and more

Let the joust­ing be­gin.

The big ab­stract drop ahead of AS­CO — the an­nu­al Olympics of can­cer R&D — pro­vid­ed some ear­ly, quick snap­shots that helped dri­ve stocks up or down, or sim­ply pro­vid­ed a chance to tout some po­ten­tial in a hot­ly con­test­ed field.

As more and more bio­phar­ma mon­ey has been in­vest­ed in the on­col­o­gy field in re­cent years, AS­CO has been at­tract­ing a big­ger range of en­trants, and ab­stract night will help de­ter­mine who comes out on top dur­ing the melee ahead. I’ve picked out a few of the most no­tice­able ab­stracts, which you can see be­low.

Loxo takes an­oth­er round in its bruis­ing, toe-to-toe fight with Blue­print

Josh Bilenker

Loxo On­col­o­gy $LOXO was the big win­ner Wednes­day night as in­vestors be­gan to pick through a pile of AS­CO ab­stracts to see what gems could be culled from the num­bers.

The biotech’s stock soared 18% overnight as in­vestors spot­light­ed a 69% over­all re­sponse rate among 32 evalu­able RET-fu­sion pos­i­tive pa­tients tak­ing LOXO-292. Loxo’s claim to fame is that it de­vel­ops can­cer drugs that tar­get small, ge­net­i­cal­ly de­fined pa­tient groups with an ag­nos­tic ap­proach to tu­mor types. Reg­u­la­tors at the FDA have been en­thu­si­as­tic about this emerg­ing field, which bodes well for Loxo. And they backed that en­thu­si­asm up with da­ta demon­strat­ing a 65% re­sponse rate in NSCLC and 83% for pap­il­lary thy­roid can­cer. 84% (27/32) of the pa­tients had ra­di­ograph­ic tu­mor re­duc­tion rang­ing from 19% to 67%.

That’s good, but it may well get bet­ter. Loxo CEO Josh Bilenker has flagged that since the Jan­u­ary cut­off date for the ab­stract the da­ta are even bet­ter now, which we’ll see at AS­CO. Loxo helped stoke the en­thu­si­asm with a note high­light­ing that LOXO-292 has been se­lect­ed for best of show at AS­CO, which will keep the com­pa­ny in the spot­light.

Can­cer R&D, though, is the ul­ti­mate blood sport in biotech. And when some­thing goes up, it’s of­ten at the ex­pense of a ri­val. In this case, that’s Blue­print Med­i­cines — again — which has al­ready felt the sting of a neg­a­tive com­par­i­son with Loxo.

Blue­print Med­i­cines $BPMC has been ad­vanc­ing BLU-667, which has been at­tract­ing warm re­views by an­a­lysts — un­less they start com­par­ing it to the ri­val. That side-by-side com­par­i­son knocked their stock back at AACR, and it did it again last night as the num­bers once again fa­vored Loxo. Shares are down about 8% in pre-mar­ket trad­ing Thurs­day.

No­var­tis vs Gilead/Kite: Is Kym­ri­ah bet­ter and safer than Yescar­ta?

Few ri­val­ries have been as in­tense as the show­down be­tween these two pi­o­neers in the CAR-T field. No­var­tis’ Kym­ri­ah $NVS still has to over­come a nag­ging is­sue with one-time man­u­fac­tur­ing is­sues, but Gilead’s Yescar­ta $GILD is now be­ing com­pared with its ri­val, and at first blush may have some ex­plain­ing to do. 

A group in Bei­jing ran a small com­par­i­son study of the two types of CAR-Ts — which use the 4-1BB and CD28 co-stim­u­la­to­ry sig­nal­ing do­mains — for CD19-pos­i­tive B-cell acute lym­phoblas­tic leukemia and found that the Kym­ri­ah/4-1BB ap­proach ap­pears to have a dis­tinct set of ad­van­tages. 

In that 4-1BB arm there was a 100% over­all ob­jec­tive re­sponse rate, com­pared to 89% in the CD28 arm. In ad­di­tion, and more im­por­tant­ly, all 5 of the pa­tients suf­fer­ing from se­ri­ous Grade 3 or 4 cy­tokine re­lease syn­drome were in the CD28/Yescar­ta group. This ar­gu­ment has a long way to run, and Gilead won’t con­cede an inch of the race. But the com­par­isons have just be­gun.

On Mon­day, Gilead con­tact­ed us to of­fer this state­ment:

It is im­por­tant to note that Yescar­ta was not eval­u­at­ed in this study. The ab­stract dis­cuss­es da­ta from a study eval­u­at­ing oth­er CAR T prod­ucts us­ing 4-1BB and CD28 co-stim­u­la­to­ry sig­nal­ing do­mains, re­spec­tive­ly. Im­por­tant­ly, Yescar­ta is en­gi­neered us­ing Kite’s man­u­fac­tur­ing process. The CD28 CAR T eval­u­at­ed in this tri­al was not man­u­fac­tured by Kite and there have been no head-to-head stud­ies of Yescar­ta com­pared to ti­s­agen­le­cleu­cel.

Ever­core ISI an­a­lyst Umer Raf­fat this morn­ing called the re­sults of this study provoca­tive, but wants to see the de­tails. So do I.

Jounce shares plunge on the lat­est da­ta cut for JTX-2011

Eliz­a­beth Tre­hu

The biggest los­er overnight was Jounce Ther­a­peu­tics $JNCE, which took a nasty hit af­ter post­ing their up­date on their lead ther­a­py — JTX-2011. As a monother­a­py, 1 out of 7 pa­tients with gas­tric can­cer re­spond­ed, com­pared to 2 out of 19 who got the com­bo with Op­di­vo — an 11% re­sponse rate. The rate wasn’t much bet­ter in triple-neg­a­tive breast can­cer. 

In a re­lease, re­searchers hit the theme that these were heav­i­ly pre­treat­ed pa­tients, but on­look­ers were in a can­tan­ker­ous mood and didn’t like the un­der­whelm­ing num­bers. Shares plunged 26% and Wells Far­go down­grad­ed the stock.

Cel­gene struck a ma­jor deal to col­lab­o­rate with Jounce on this drug, and that wasn’t ig­nored this morn­ing.

“The pre­lim­i­nary da­ta from pa­tients across mul­ti­ple sol­id tu­mor types en­rolled in the ICON­IC tri­al show that JTX-2011 is well-tol­er­at­ed alone and in com­bi­na­tion with nivolum­ab and has demon­strat­ed ev­i­dence of bi­o­log­ic ac­tiv­i­ty and tu­mor re­duc­tions in heav­i­ly pre-treat­ed pa­tients who have failed all avail­able ther­a­pies. In ad­di­tion, a po­ten­tial sur­ro­gate bio­mark­er of re­sponse has been iden­ti­fied that may help to guide JTX-2011 de­vel­op­ment,” said Eliz­a­beth Tre­hu, chief med­ical of­fi­cer of Jounce Ther­a­peu­tics.

Nek­tar sees a big ero­sion in re­sponse rates for close­ly-watched I/O star NK­TR-214

Nek­tar $NK­TR scored one of the biggest deals in bio­phar­ma so far this year when Bris­tol-My­ers came in with a $3.6 bil­lion deal to part­ner on NK­TR-214. That part­ner­ship was an­nounced in the wake of the first glimpse of how ef­fec­tive a pair­ing of their drug could be with Op­di­vo, with 63% of a small group of ad­vanced melanoma pa­tients re­spond­ing to first-line ther­a­py. But in Nek­tar’s up­date this week re­searchers note that the re­sponse rate in the bas­ket study showed a re­duced melanoma im­pact, with a 52% re­sponse rate.

Re­nal cell car­ci­no­ma al­so dropped, falling to 54%, down from 71% re­port­ed in the com­pa­ny’s Q4 call in ear­ly March.

That’s by no means the kiss of death. Re­sponse rates tend to de­cline over time. But an­a­lysts will be watch­ing these num­bers close­ly to see just how far they drop for a drug that is now front and cen­ter in the late-stage on­col­o­gy pipeline. The stock is down 3% in pre-mar­ket trad­ing, with the ju­ry still out on this promis­ing ther­a­py.

Mer­ck KGaA plans to shine a light on its can­cer pipeline at AS­CO — with Pfiz­er jump­ing in

Mer­ck KGaA will be back at AS­CO look­ing to earn some re­spect for its can­cer drug pipeline. So far the bulk of the at­ten­tion has gone to Baven­cio, its PD-L1 check­point in­hibitor part­nered with Pfiz­er, which is fight­ing an up­hill bat­tle to gain mar­ket share against the lead­ers in the field. But the Ger­man Mer­ck has a pipeline in on­col­o­gy, and they will do their best to high­light their chances on a range of ther­a­pies in Chica­go.

Its c-Met re­cep­tor ty­ro­sine ki­nase in­hibitor tepo­tinib has earned some ku­dos from Bern­stein. And re­searchers post­ed da­ta on 15 pa­tients with ad­vanced non-small cell lung can­cer har­bor­ing MET ex­on 14 skip­ping mu­ta­tions, with 60% demon­strat­ing a con­firmed par­tial re­sponse. An­a­lysts be­lieve this drug could hit $650 mil­lion in sales by 2030 — not a block­buster but a sol­id suc­cess, which the com­pa­ny bad­ly needs af­ter a long drought in clin­i­cal de­vel­op­ment suc­cess­es.

On the com­bo front, where all the PD-1/PD-L1 play­ers are fo­cus­ing on in a va­ri­ety of ways, Mer­ck KGaA tout­ed their M7824, a TGF-ß trap/an­ti-PD-L1 bi-func­tion­al im­munother­a­py fu­sion pro­tein. High PD-L1 ex­press­ing pa­tients ex­hib­it­ed an ORR of 71.4%.

The next big step on Baven­cio lies in com­bo ther­a­pies, and there Mer­ck KGaA says it gained some ear­ly-stage ev­i­dence to back up a com­bi­na­tion of the check­point with lor­la­tinib in non-small cell lung can­cer — a key com­pet­i­tive front for these play­ers. And their com­bo came out way ahead in the JAVELIN Lung 101 study, which com­pared their check­point with Xalko­ri (crizo­tinib) and the lor­la­tinib match-up. Lor­la­tinib — a drug Pfiz­er has high hopes for — came out way ahead. From the ab­stract:

The con­firmed ob­jec­tive re­sponse rate with A+C in ALK− pts was 16.7% (95% CI, 2.1-48.4; par­tial re­sponse [PR] in 2 pts), and with A+L in ALK+ pts was 46.4% (95% CI, 27.5-66.1; PR in 12 pts; com­plete re­sponse in 1 pt).


Im­age: Poster ses­sion at AS­CO 2017. AS­CO

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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(Credit: Shutterstock)

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Saqib Islam, SpringWorks CEO

Pfiz­er spin­out Spring­Works will ship its first drug to the FDA be­fore year’s end with PhI­II win

SpringWorks Therapeutics thinks it has cemented the backbone for its first “pipeline-in-a-product” oncology treatment and will send it to the FDA before the clock strikes 2023 with a Phase III win on Tuesday.

The oral gamma secretase inhibitor, dubbed nirogacestat, beat placebo on the primary goal of progression-free survival in adults with progressing desmoid tumors.

The soft-tissue tumors can lead to long-lasting pain, disfigurement and amputation, and there are currently no approved meds for the rare oncology indication. The tumors typically impact patients aged 20 to 44 years old and disproportionately affect women at rates 2 to 3 times higher, with up to a total of 1,650 new cases diagnosed in the US annually, according to SpringWorks.

Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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