Lil­ly's ap­proved can­cer drug Lartru­vo fails con­fir­ma­to­ry study, set­ting the stage for with­draw­al of reg­u­la­to­ry en­dorse­ment

Back in 2016, Lil­ly won FDA ac­cel­er­at­ed ap­proval for its soft tis­sue drug olara­tum­ab on the ba­sis of en­cour­ag­ing da­ta from a small 133-pa­tient mid-stage study, but on Fri­day a larg­er, con­fir­ma­to­ry tri­al meant to ce­ment the ap­proval showed that the treat­ment failed to help pa­tients live longer, which means the US health reg­u­la­tor can re­scind its en­dorse­ment of the drug.

The drug, sold as Lartru­vo, had won the FDA nod in com­bi­na­tion with the chemother­a­py dox­oru­bicin as a first-line treat­ment for a sub­set of pa­tients with the dis­ease, which had seen no new ap­provals in decades. By the third quar­ter of 2018, Lil­ly had raked in $221.2 mil­lion in Lartru­vo sales last year.

The con­fir­ma­to­ry Phase III tri­al — AN­NOUNCE — test­ed Lartru­vo in com­bi­na­tion with dox­oru­bicin, ver­sus dox­oru­bicin monother­a­py in pa­tients with ad­vanced or metasta­t­ic soft tis­sue sar­co­ma. The study did not meet the main goal of im­prov­ing over­all sur­vival in the pa­tient pop­u­la­tion, in­clud­ing the sub­set of leiomyosar­co­ma pa­tients — there was no dif­fer­ence in sur­vival be­tween the study arms for ei­ther pop­u­la­tion, the com­pa­ny said.

Anne White

Da­ta from the pre­vi­ous, pos­i­tive Phase II tri­al had helped Lil­ly se­cure ac­cel­er­at­ed ap­proval in the Unit­ed States, and con­di­tion­al ap­proval in Eu­rope. But those ap­provals were con­tin­gent on fa­vor­able AN­NOUNCE re­sults con­firm­ing its ben­e­fit, and now the com­pa­ny will talk to reg­u­la­tors to de­ter­mine the next steps for the drug.

For now, the drug­mak­er has sus­pend­ed Lartru­vo pro­mo­tion, and ex­pects to in­cur a pre-tax charge in the range of $70 mil­lion to $90 mil­lion (or $0.10 per share, af­ter tax) in the first quar­ter of 2019.

“Lil­ly was sur­prised and dis­ap­point­ed that LARTRU­VO did not im­prove sur­vival for pa­tients with ad­vanced soft tis­sue sar­co­ma in this study,” said Anne White, pres­i­dent of Lil­ly On­col­o­gy in a state­ment. Lil­ly’s $LLY shares slipped about 3% pre-mar­ket.

The drug is cur­rent­ly al­so be­ing test­ed in com­bi­na­tion with gem­c­itabine and do­c­etax­el in a sep­a­rate Phase II tri­al in ad­vanced soft tis­sue sar­co­ma pa­tients.

The fail­ure fol­lows Lil­ly’s an­nounce­ment this month to swal­low Loxo On­col­o­gy for $8 bil­lion. Ac­cord­ing to a SEC fil­ing post­ed on Thurs­day, Lil­ly ex­ec­u­tives were look­ing for a swift deal when they sat down with Loxo CEO Josh Bilenker five days ahead of Christ­mas. They were will­ing to fork out $230 a share for the small­er com­pa­ny, in a hur­ry to get the deal done in time for the JP­Mor­gan ex­trav­a­gan­za. There were no oth­ers bid­ders for the com­pa­ny, but in or­der to has­ten the buy­out, Lil­ly agreed even­tu­al­ly to pay $235 per Loxo share, al­low­ing for the ac­qui­si­tion to be re­vealed on Jan­u­ary 7.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.