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

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

Special report

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Maryland’s Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesn’t vanish but becomes a recurrent virus like the flu?

“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regeneron’s Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly – an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his “Regeneron Infectious Diseases”-minted espresso glass – and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Jim Scholefield via PR Newswire

Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Bank­rupt an­tibi­otics mak­er Ar­a­digm turns to old part­ner/in­vestor for fi­nal $3M fire sale

Grifols once paid Aradigm $26 million for a stake in its inhaled antibiotics. But with Aradigm now in bankruptcy, the Spanish drugmaker is dishing out a final $3.2 million to buy it all.

The fire sale — which comes one year after Aradigm filed for Chapter 11 following a regulatory trifecta for disaster — will see Grifols obtain assets and IP to Apulmiq (formerly Pulmaquin and Linhaliq in Europe), Lipoquin and free ciprofloxacin. In addition to waiving its claims in the bankruptcy case, Grifols also agreed to milestone payments up to $3 million more upon any regulatory approvals.