With Sanofi en­ter­ing the mul­ti­ple myelo­ma mar­ket, J&J flash­es da­ta on new rare dis­ease

Since its 2015 ap­proval, Darza­lex has earned J&J bil­lions as one of the most po­tent drugs avail­able for mul­ti­ple myelo­ma, a com­mon can­cer that for years had few good ther­a­pies. Now, as Sanofi en­ters with a sim­i­lar drug, the com­pa­ny is edg­ing clos­er to ex­pand­ing its us­es.

Most no­tably, they have been test­ing the drug on AL amy­loi­do­sis, a rare and po­ten­tial­ly fa­tal dis­ease in which B cells pro­duce mis­shapen an­ti­bod­ies. These ab­nor­mal an­ti­bod­ies scrunch to­geth­er to form clumps called amy­loid (sim­i­lar to the plaques im­pli­cat­ed in Alzheimer’s but with dif­fer­ent pro­teins), which can build up in a num­ber of or­gans and pre­vent them from func­tion­ing prop­er­ly. Pa­tients live a me­di­an of 6 months to 3 years af­ter di­ag­no­sis. There are no specif­i­cal­ly ap­proved ther­a­pies, but some pa­tients al­so have mul­ti­ple myelo­ma — al­so a B cell ma­lig­nan­cy — and doc­tors have tried us­ing drugs de­vel­oped for the can­cer as a treat­ment.

In a piv­otal Phase III tri­al pre­sent­ed this week­end at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion vir­tu­al meet­ing, J&J test­ed three med­ica­tions of­ten used to treat AL amy­loi­do­sis against those three med­ica­tions plus Darza­lex. Al­though there are no da­ta yet on over­all sur­vival, the J&J com­bo cleared tu­mors at a far high­er clip than the con­trol, which in­clud­ed the mul­ti­ple myelo­ma drug Vel­cade and two chemother­a­pies. The com­plete re­sponse rate was 53% com­pared with 18%. The drug al­so showed 42% high­er pro­gres­sion free sur­vival and proved bet­ter at pre­vent­ing heart or kid­ney de­te­ri­o­ra­tion.

The com­pa­ny plans to file ap­proval in the US and the EU lat­er this year, ac­cord­ing to a J&J pre­sen­ta­tion from April. The tri­al used the sub­cu­ta­neous form of the drug. Ap­proved ear­li­er this year for mul­ti­ple myelo­ma, it re­duced the time it takes to ad­min­is­ter the drug from 7 hours to 5 min­utes.

Ray­mond Comen­zo

In a state­ment, lead in­ves­ti­ga­tor Ray­mond Comen­zo point­ed to the long-run­ning chal­lenges in treat­ing AL amy­loi­do­sis. The dis­ease can fes­ter for years be­fore ma­jor symp­toms ap­peared, and then they are of­ten con­fused with more com­mon ail­ments.

“Pa­tients with AL amy­loi­do­sis of­ten ex­pe­ri­ence poor out­comes be­cause their symp­toms are con­fused with more com­mon con­di­tions, caus­ing de­lays in di­ag­no­sis,” Comen­zo said. “The new­ly di­ag­nosed pa­tient pop­u­la­tion in AL amy­loi­do­sis is the most chal­leng­ing to treat, and many do not reach sec­ond-line ther­a­py.

“The high­er rate of hema­to­log­ic com­plete re­sponse,” he added, be­fore us­ing the chem­i­cal name for the drug, “as well as the no­table sus­tain­ment of ma­jor or­gan func­tion in those re­ceiv­ing sub­cu­ta­neous dara­tu­mum­ab in the AN­DROM­E­DA study, sug­gest that sub­cu­ta­neous dara­tu­mum­ab may be a promis­ing treat­ment for new­ly di­ag­nosed pa­tients with AL amy­loi­do­sis who have been, for some time, in ur­gent need of new ther­a­peu­tic op­tions.”

Darza­lex earned J&J a hair un­der $3 bil­lion last year, but they will face com­pe­ti­tion go­ing for­ward. Sanofi land­ed their first in-house can­cer ap­proval in a decade in March for Sar­clisa, a mul­ti­ple myelo­ma drug that works by the same mech­a­nism as Darza­lex, block­ing a re­cep­tor that’s over­ex­pressed on mul­ti­ple myelo­ma cells, called CD38.

Oth­er tri­als test­ing mul­ti­ple myelo­ma drugs in amy­loi­do­sis are un­der­way, in­clud­ing one — run by South­west On­col­o­gy and the Na­tion­al Can­cer In­sti­tute — on Sar­clisa.

So­cial: Kristof­fer Trip­plaar, Sipa via AP Im­ages

#ES­MO20: As­traZeneca aims to spur PRO­found shift in prostate can­cer treat­ment with Lyn­parza OS da­ta

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).

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.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: 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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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.

#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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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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#ES­MO20: Trodelvy da­ta show that Gilead­'s $21B buy­out may have been worth the big pre­mi­um

Gilead CEO Dan O’Day has been on a shopping spree. And while some analysts gawked at the biotech’s recent $21 billion Immunomedics buyout, new data released at virtual ESMO 2020 suggest the acquisition may have been worth the hefty price.

The deal, announced last weekend, will give California-based Gilead $GILD Trodelvy, which was recently approved for metastatic triple-negative breast cancer (mTNBC).

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