FDA OKs Mer­ck’s heavy­weight Keytru­da for can­cer niche — trig­ger­ing ti­tle fight with the street brawlers at Re­gen­eron

George Yan­copou­los

When Re­gen­eron won its first OK for their PD-1 Lib­tayo as a treat­ment for re­cur­rent or metasta­t­ic cu­ta­neous squa­mous cell car­ci­no­ma, the sec­ond most com­mon form of skin can­cer, com­pa­ny pres­i­dent George Yan­copou­los crowed about their abil­i­ty to grab a ma­jor niche mar­ket left wide open by the 2 big pow­er­hous­es in the field, Mer­ck and Bris­tol My­ers.

To­day, though, Re­gen­eron and their part­ners at Sanofi no longer en­joy a mo­nop­oly in that par­tic­u­lar field. Mer­ck grabbed an OK for their top-sell­ing PD-1 Keytru­da in the same group of pa­tients. But don’t ex­pect Re­gen­eron — still strug­gling to boost sales — to let go of their best-in-class claims.

Keytru­da gar­nered the FDA ap­proval based on an “ob­jec­tive re­sponse rate (ORR) of 34% (95% CI, 25-44), in­clud­ing a com­plete re­sponse rate of 4% and a par­tial re­sponse rate of 31%. Among re­spond­ing pa­tients, 69% had on­go­ing re­spons­es of six months or longer. Af­ter a me­di­an fol­low-up time of 9.5 months, the me­di­an du­ra­tion of re­sponse (DOR) had not been reached (range, 2.7 to 13.1+ months).”

When Re­gen­eron scored their way in­to the mar­ket, they count­ed an ORR of “47% (95% CI: 38, 57), with 4% com­plete and 44% par­tial re­sponse rates … The me­di­an re­sponse du­ra­tion was not reached (range: 1.0 to 15.2+ months), and 61% of re­spons­es were durable for 6 months or longer.”

Cross-tri­al com­par­isons are al­ways dicey, but Re­gen­eron is a pu­gna­cious play­er, like­ly to spot­light any ad­van­tage. When I talked to Yan­copou­los about it at JP­Mor­gan in ear­ly 2019, he asked why Mer­ck and oth­ers were run­ning “thou­sands” of check­point tri­als, but no one went af­ter CSCC ahead of Re­gen­eron.

His an­swer: Be­cause they aren’t near­ly as smart.

It’s so mind­less they missed this huge op­por­tu­ni­ty,” he added em­phat­i­cal­ly, with­out try­ing to hide his con­tempt.

He al­so de­scribed pre­scrib­ing a ri­val PD-L1 for any­thing as “med­ical mal­prac­tice” as he went on to trash Baven­cio from Pfiz­er and Mer­ck KGaA.

The op­por­tu­ni­ty Yan­copou­los re­ferred to is still fair­ly small pota­toes com­pared to Keytru­da’s $12 bil­lion reign as the world’s lead­ing PD-1 check­point. Lib­tayo earned on­ly $193 mil­lion last year, un­der­scor­ing just how hard it is for the world’s 6th PD-(L)1 to carve out a fran­chise.

Re­gen­eron, though, isn’t afraid to go toe-to-toe with Mer­ck. They un­der­scored that with pos­i­tive da­ta in April for first-line pa­tients with non-small cell lung can­cer that test­ed pos­i­tive for PD-(L)1.

Re­gen­eron — which has an out­stand­ing rep in R&D — nev­er backs down from a fight un­less it’s been forced to, as with PC­SK9. In the check­point drug com­mu­ni­ty, no one ex­pects them to wa­ver now.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Brid­get Martell on tak­ing Yale spin­out 'to adult­hood'; Cul­li­nan On­col­o­gy CEO an­nounces res­ig­na­tion as Bris­tol My­ers hema­tol­ogy ex­ec is wel­comed aboard

Bridget Martell’s first clinical trial was an unorthodox one.

Then a resident in internal medicine at Yale, she was given an award to explore a career in clinical research — which was how she wound up leading a Phase I study for a therapeutic cocaine vaccine designed to help overcome addiction. She ended up overseeing the trial from Patient 1 to Patient 110 and, in the end, the early trial was positive.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty


I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Pascal Soriot, AstraZeneca CEO (via Getty images)

UP­DAT­ED: FDA slaps As­traZeneca's MCL-1 can­cer drug with a hold af­ter safe­ty is­sue — 2 years af­ter Am­gen axed a trou­bled ri­val

There are new questions being posed about a class of cancer drugs in the wake of the second FDA-enforced clinical hold in the field.

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca says that regulators hit them with a hold on their rival therapy of the same class.

The pharma giant noted on clinicaltrials.gov that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.”

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Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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