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.

An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Can an FDA hus­tle up on trastuzum­ab be far be­hind?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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