Hervé Hoppenot, Incyte CEO

In­cyte se­cures sec­ond FDA ap­proval for bile duct can­cer drug in mu­tat­ed can­cer type

In­cyte nabbed its sec­ond ap­proval from fed­er­al reg­u­la­tors for can­cer drug pemi­ga­tinib, brand­ed as Pe­mazyre — as the po­ten­tial for even more in­di­ca­tions await.

The com­pa­ny, head­ed by CEO Hervé Hop­penot, put out word be­fore the mar­ket opened on Fri­day that the FDA ap­proved Pe­mazyre un­der pri­or­i­ty re­view for re­lapsed or re­frac­to­ry myeloid/lym­phoid neo­plasms (MLNs) with FGFR1 re­arrange­ment. The drug, an FGFR1/2/3 in­hibitor, was orig­i­nal­ly ap­proved back in 2020 for cholan­gio­car­ci­no­ma, al­so known as bile duct can­cer.

In­cyte VP of on­col­o­gy drug de­vel­op­ment Pe­ter Lang­muir told End­points News that the drug had been in the clin­ic around sev­en years, start­ing with clin­i­cal tri­als in 2015. In study­ing cholan­gio­car­ci­no­ma, In­cyte found a sub­set of pa­tients who had mu­ta­tions in the FGFR2 gene, which Pe­mazyre tar­get­ed and help­ing se­cure its even­tu­al ap­proval in the in­di­ca­tion.

“And while we were pur­su­ing the cholan­gio­car­ci­no­ma in­di­ca­tion, we re­al­ized that there was a sub­set of pa­tients with hema­to­log­ic ma­lig­nan­cies that had al­ter­ations in the FGFR1 gene,” Lang­muir not­ed.

That dis­or­der, re­lapsed or re­frac­to­ry MLNs with FGFR1 re­arrange­ment, was once known as 8p11 myelo­pro­lif­er­a­tive syn­drome.

Lang­muir added that while the dis­ease is ex­treme­ly rare (In­cyte said in a state­ment that the dis­ease im­pacts less than 1 in 100,000 peo­ple in the US), it presents in dif­fer­ent ways, such as lym­phoma or leukemia. But all of these pre­sen­ta­tions have what Lang­muir called a “chro­mo­so­mal translo­ca­tion,” where a chro­mo­some breaks and reat­tach­es to dif­fer­ent chro­mo­somes, at spe­cif­ic chro­mo­some 8p11.

The FDA ap­proval was based on da­ta from In­cyte’s Phase II FIGHT-203 study, which test­ed safe­ty and ef­fi­ca­cy in 28 pa­tients with re­lapsed or re­frac­to­ry MLNs with FGFR1 re­arrange­ment in a sin­gle-arm tri­al. How­ev­er, the tri­al con­tin­ued en­roll­ment and ul­ti­mate­ly fin­ished with 47 pa­tients. Lang­muir said that In­cyte will be pre­sent­ing da­ta on the en­tire set of pa­tients at a lat­er date.

Ac­cord­ing to the state­ment, at least 17 pa­tients achieved a com­plete re­sponse across mul­ti­ple co­horts, with the time to re­sponse rang­ing from as short as 44 days to well over a year, in one case. Me­di­an time to re­sponse was 104 days in one sub­set of pa­tients.

The most com­mon ad­verse re­ac­tions in pa­tients, In­cyte said, in­clud­ed hy­per­phos­phatemia (ab­nor­mal­ly high serum phos­phate lev­els), nail tox­i­c­i­ty, alope­cia and stom­ati­tis.

As for fu­ture in­di­ca­tions, Lang­muir added that Phase II stud­ies are on­go­ing and test­ing Pe­mazyre in glioblas­toma and lung can­cer. How­ev­er, In­cyte will wait on the da­ta read­outs be­fore con­firm­ing that those in­di­ca­tions are what the com­pa­ny will pur­sue next.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.