Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Char­lie Sil­ver wants to look re­al­ly, re­al­ly close­ly at a lot of your cells. And he just got a lot of mon­ey to do so.

Sil­ver’s start­up, Mis­sion Bio, raised $70 mil­lion in a Se­ries C round Thurs­day led by No­vo Hold­ings. The mon­ey, which brings Mis­sion Bio to $120 mil­lion raised since its 2012 found­ing, will be used to ad­vance the sin­gle-cell se­quenc­ing plat­form they built to de­tect ear­ly re­sponse or re­sis­tance to new can­cer ther­a­pies.

“We want this every­where,” Sil­ver told End­points News. “Every­where where ther­a­py re­sis­tance is im­por­tant — and it’s im­por­tant for most of on­col­o­gy.”

In the last few years, sin­gle-cell se­quenc­ing tech­nol­o­gy has be­come ubiq­ui­tous in bio­med­ical re­search. It’s al­lowed re­searchers to zoom in­to what’s un­fold­ing in an in­di­vid­ual cell, as op­posed to blend­ing mul­ti­ple cells to­geth­er and get­ting an av­er­age, as pre­vi­ous se­quenc­ing meth­ods have re­quired.

Much of that work, though, has been through RNA se­quenc­ing, al­so known as RNA-Seq. A quick PubMed for RNA-Seq search turns up over 10,000 pa­pers since the start of 2019. This year alone, it’s been used to pro­file the hu­man an­ti­body re­sponse, com­pare mouse and hu­man brains, and even to see if you can get a por­trait of some­one’s mi­cro­bio­me out of their sperm.

Much of the work, though, has come in can­cer, where cell het­ero­gene­ity — or the va­ri­ety of dif­fer­ent cells and mu­ta­tions — can of­fer key clues about how can­cer aris­es, de­vel­ops, re­sponds to and re­sists ther­a­py. There, Sil­ver said, Mis­sion Bio has an ad­van­tage by fo­cus­ing on DNA rather than RNA. They’re not the on­ly DNA-Seq plat­form, but he claims “we are the on­ly ones that do sin­gle-cell DNA at every scale, from sin­gle mu­ta­tion, copy num­ber through the whole chro­mo­some” and the on­ly ones that can link that DNA snap­shot to the pro­teins on the cells.

“We took our plat­form and ba­si­cal­ly pur­pose-built it for phar­ma,” Sil­ver said. “The com­bi­na­tion of DNA and pro­tein to­geth­er tends to be ex­act­ly what phar­ma needs for drug de­vel­op­ment, be­cause you can link to­geth­er the mu­ta­tion you’re try­ing to drug, along with the path­way that you can now link to­geth­er with pro­tein.”

So far, Mis­sion Bio has test­ed the plat­form with a hand­ful of small biotechs, such as Agios and Ag­i­lent Tech­nolo­gies, and can­cer cen­ters such as MD An­der­son. They claim to have un­named part­ner­ships with Big Phar­ma as well.

The idea, Sil­ver said, is to give re­searchers tools to see ear­li­er whether a pa­tient is re­spond­ing to a ther­a­py or evolv­ing re­sis­tance to a ther­a­py. That could in the­o­ry then short­en de­vel­op­ment time, al­low­ing com­pa­nies to abort doomed tri­als or weed peo­ple with the wrong mol­e­c­u­lar pro­file out, mak­ing sure on­ly those most like­ly to re­spond to the ther­a­py are stud­ied.

In a pa­per in Blood in March, MD An­der­son re­searchers used the plat­form to dis­cov­er tiny pock­ets of can­cer cells with rare mu­ta­tions that lim­it­ed pa­tients’ re­sponse to the acute myeloid leukemia drug Ven­clex­ta. In May, in Blood Ad­vances, re­searchers at Agios used it to find new re­sis­tance mech­a­nisms to their AML drug Tib­so­vo.

“Thor­ough cat­a­loging of re­sis­tance mech­a­nisms to tar­get­ed ther­a­pies has proven in­valu­able in the de­vel­op­ment of next-gen­er­a­tion ther­a­pies, such as sec­ond- and third-gen­er­a­tion in­hibitors of BCR-ABL, EGFR, and ALK,” the re­searchers not­ed, “and in the de­vel­op­ment of ef­fi­ca­cious com­bi­na­tion ther­a­pies such as BRAF-MEK dual in­hi­bi­tion in melanoma.”

In ad­di­tion to find­ing new can­cer part­ners for their plat­form, Sil­ver said they were al­so go­ing to use the Se­ries C fund­ing to push in­to gene and cell ther­a­py. Mis­sion’s plat­form, he said, could help char­ac­ter­ize how suc­cess­ful­ly cells have been edit­ed.

“We’re re­al­ly ex­pand­ing,” he said.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA have vowed not to let politics get in the way of science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped health agencies under his purview — including the FDA — of their rulemaking ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Eli Lilly CSO Dan Skovronsky (file photo)

#ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

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AstraZeneca has unveiled the final, mature overall survival data that cemented Lynparza’s first approval in prostate cancer approval — touting its lead against rivals with the only PARP inhibitor to have demonstrated such benefit.

But getting the Merck-partnered drug to the right patients remains a challenge, something the companies are hoping to change with the new data cut.

The OS numbers on the subgroup with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer are similar to the first look on offer when the FDA expanded the label in May: Lynparza reduced the risk of death by 31% versus Xtandi and Zytiga. Patients on Lynparza lived a median of 19.1 months, compared to 14.7 months for the anti-androgen therapies (p = 0.0175).

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

Dan Skovronsky, Eli Lilly CSO

An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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#ES­MO20: It’s not just Keytru­da any­more — Mer­ck spot­lights 3 top ear­ly-stage can­cer drugs

Any $12 billion megablockbuster in the portfolio tends to overshadow everything else in the pipeline. Which is something Merck can tell you a little bit about.

Keytruda not only dominates the PD-(L)1 field, it looms over everything Merck does, to the point some analysts wonder if Merck is a one-trick pony.

There’s no shortage of Keytruda data on display at ESMO this weekend, but now the focus is shifting to the future role of new drugs and combos in maintaining that lead position for years to come. And the pharma giant has a special focus for 3 early-stage efforts where Roger Perlmutter’s oncology team is placing some big bets.

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