Shat­ter­ing an old record, FDA quick­ly OKs pi­o­neer­ing, site-ag­nos­tic can­cer drug larotrec­tinib from Loxo and Bay­er

The FDA has hus­tled up a quick OK for larotrec­tinib, of­fer­ing their stamp of ap­proval for a site-ag­nos­tic can­cer drug from Loxo On­col­o­gy and their part­ners at Bay­er, a prime ex­am­ple of a new wave of on­col­o­gy drugs hit­ting the field.

The la­bel for their drug, now called Vi­t­rakvi, was the first thing to hit Twit­ter af­ter the mar­ket closed, which you can see here.

Stam­ford, CT-based Loxo $LOXO — run by CEO Josh Bilenker — is one of the trail­blaz­ers in per­son­al­ized can­cer ther­a­pies where pa­tients are grouped not by the site of tu­mor de­vel­op­ment but by ge­net­ics — an ap­proach that calls for broad­er se­quenc­ing to ID the ge­net­ic mu­ta­tions un­der­pin­ning each pa­tients’ can­cer. That re­quires lots of work to iden­ti­fy pa­tients. But they have da­ta from a small piv­otal study to back up their break­through.


Specif­i­cal­ly there were 50 pa­tients — 43 adults and 12 chil­dren and adults with TRK fu­sion can­cer — with 12% com­plete and 64% par­tial re­spon­ders. That’s a high rate of suc­cess and it qual­i­fied for a pri­or­i­ty re­view at an agency that likes to press the met­al on new drug OKs — par­tic­u­lar­ly for can­cer.

Now comes the re­al­ly hard part. To find the few thou­sand US pa­tients that could ben­e­fit will re­quire can­cer pa­tients to get their DNA se­quenced, which is un­com­mon. Then theres the price. Bay­er set the whole­sale price at just un­der $400,000 a year, putting the oral drug in the most ex­pen­sive drug cat­e­go­ry. Then there’s a liq­uid for­mu­la­tion avail­able for cer­tain chil­dren at $132,000 a year.

Bay­er is al­so of­fer­ing a mon­ey-back guar­an­tee to pay­ers with as­sur­ances that pa­tient’s out-of-pock­et ex­po­sure will usu­al­ly be lim­it­ed to small sums.

An­drew Berens at Leerink ex­pects to see the num­bers add up over the years.

We see peak Vi­t­rakvi rev­enues in the US of ~$700mn by 2030, with an ad­di­tion­al ~$375mn op­por­tu­ni­ty avail­able for next-gen­er­a­tion TRK in­hibitor LOXO-195, de­signed specif­i­cal­ly to ad­dress re­sis­tance mu­ta­tions that emerge with larotrec­tinib use.

“Its ap­proval re­flects ad­vances in the use of bio­mark­ers to guide drug de­vel­op­ment and the more tar­get­ed de­liv­ery of med­i­cine,” not­ed FDA com­mis­sion­er Scott Got­tlieb, who likes to step in when the agency breaks new ground. “We now have the abil­i­ty to make sure that the right pa­tients get the right treat­ment at the right time. This type of drug de­vel­op­ment pro­gram, which en­rolled pa­tients with dif­fer­ent tu­mors but a com­mon gene mu­ta­tion, wouldn’t have been pos­si­ble a decade ago be­cause we knew a lot less about such can­cer mu­ta­tions.”

A few months af­ter the da­ta ar­rived Bay­er bought in, hand­ing over a $400 mil­lion up­front, with $450 mil­lion in mile­stones for the de­vel­op­ment and first sale of larotrec­tinib, with an­oth­er $200 mil­lion on the ta­ble for LOXO-195. There’s al­so $500 mil­lion on the books for com­mer­cial goals.

Mer­ck was the first to score in this site-ag­nos­tic field, win­ning an FDA OK last year to use Keytru­da against tu­mors that were mi­crosatel­lite in­sta­bil­i­ty-high or char­ac­ter­ized by mis­match re­pair de­fi­cien­cy.

This is the 53rd new drug ap­proval for the FDA’s CDER, which with sev­er­al new bi­o­log­ic OKs this year has shat­tered the record to­tal of 53 drugs ap­proved in 1996. With 5 weeks to go in the year, the agency has plen­ty of time to set a brand new bar for pro­duc­tiv­i­ty in bio­phar­ma R&D.


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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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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

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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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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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.