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.


Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Top biotech an­a­lyst projects a gloomy out­look for Pfiz­er's JAK port­fo­lio

Many in the pharma world are hoping — better yet, expecting — JAK inhibitors to provide one of the next big boons for the industry. Few have invested as heavily in this area as Pfizer, which boasts a portfolio including Xeljanz and at least five mid-to-late stage candidates in the pipeline.

But a top Wall Street analyst is pumping the brakes on just how much good fortune is in store for the Big Pharma.

Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

It’s a race to the Phase III finish line now for the 2 leading mRNA vaccines in the pipeline for Covid-19.

BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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