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.


Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Bank­rupt an­tibi­otics mak­er Ar­a­digm turns to old part­ner/in­vestor for fi­nal $3M fire sale

Grifols once paid Aradigm $26 million for a stake in its inhaled antibiotics. But with Aradigm now in bankruptcy, the Spanish drugmaker is dishing out a final $3.2 million to buy it all.

The fire sale — which comes one year after Aradigm filed for Chapter 11 following a regulatory trifecta for disaster — will see Grifols obtain assets and IP to Apulmiq (formerly Pulmaquin and Linhaliq in Europe), Lipoquin and free ciprofloxacin. In addition to waiving its claims in the bankruptcy case, Grifols also agreed to milestone payments up to $3 million more upon any regulatory approvals.

DB­V's peanut pre­ven­tion patch ap­proach­es key stage of ap­proval process

Almost a year and a half after DBV Technologies pulled its peanut allergy immunotherapy patch from FDA review, the biotech will get their day in court. The FDA has scheduled an advisory committee hearing for May 15.

In the two-horse race to develop the first immunotherapy for peanut allergy, DBV had the early lead, filing an NDA for their patch in 2018. But on December 20 of that year, the company withdrew their application after, they said, meeting with regulators and determining they had not submitted “sufficient detail regarding data on manufacturing procedures and quality controls.” Aimmune filed their BLA 3 days later and won approval as the first immunotherapy for peanuts this month.

An­to­nio Gual­ber­to starts post-Ku­ra ca­reer at Ei­sai sub­sidiary H3; eF­FEC­TOR co-founder Siegfried Re­ich jumps to Turn­ing Point

→ Days after Kura Oncology announced the departure of co-founder Antonio Gualberto, we finally know where he wound up. Eisai subsidiary H3 Biomedicine has recruited him as CMO to finding the right patients to its four clinical-stage small molecule assets hitting genomic drivers of cancer.

“Challenges of these and many other precision medicine approaches are on one hand technical — a need for robust and precise diagnostics — and on the other hand derived by the challenge to alter standard clinical practice in settings where patient screening, e.g. by tumor DNA sequencing, is not standard practice,” he wrote to Endpoints News on his way back to Boston from Eisai’s Tokyo offices. “Only compelling clinical activity can drive clinicians and pathologists to modify standard clinical practice.”