J&J and Ab­b­Vie's block­buster BTK in­hibitor scoops a pe­di­atric in­di­ca­tion in graft-ver­sus-host dis­ease

As J&J and Ab­b­Vie duke it out with As­traZeneca and oth­er BTK com­peti­tors in chron­ic lym­pho­cyt­ic leukemia (CLL), the part­ners are cel­e­brat­ing a first for Im­bru­vi­ca in a pe­di­atric pop­u­la­tion.

Im­bru­vi­ca won ap­proval on Wednes­day as a sec­ond-line treat­ment for chron­ic graft ver­sus host dis­ease (cGVHD) in chil­dren one year and old­er, a con­di­tion in which donor cells at­tack the re­cip­i­ent af­ter a stem cell or bone mar­row trans­plant. It af­fects be­tween 52% and 65% of all chil­dren who re­ceive al­lo­gene­ic trans­plants, ac­cord­ing to Janssen and Ab­b­Vie — and un­til now, there were no ap­proved treat­ments for that age group.

“Imag­ine go­ing through a trans­plant and then be­ing told you have a mod­er­ate to se­vere chron­ic dis­ease that can some­times al­so be life-threat­en­ing,” prin­ci­pal in­ves­ti­ga­tor Paul Car­pen­ter said in a news re­lease. “If these chil­dren were be­tween one and 12 and didn’t re­spond to steroid treat­ment, we didn’t have any rig­or­ous­ly stud­ied treat­ment op­tions — un­til now.”

The ap­proval was based on da­ta from the Phase I/II iMAG­INE tri­al in pa­tients 1 to 22 years old, which showed an over­all re­sponse rate of 60% at week 25 and a me­di­an du­ra­tion of re­sponse of 5.3 months, ac­cord­ing to the FDA.

The most com­mon ad­verse re­ac­tions in­clud­ed ane­mia, mus­cu­loskele­tal pain, pyrex­ia, di­ar­rhea, pneu­mo­nia, ab­dom­i­nal pain, stom­ati­tis, throm­bo­cy­tope­nia and headache.

Craig Tendler

“The pe­di­atric cGVHD com­mu­ni­ty is a prime ex­am­ple of an un­der­served pa­tient pop­u­la­tion with high un­met med­ical needs for whom Janssen is com­mit­ted to de­vel­op­ing life-sav­ing ther­a­pies,” said Craig Tendler, glob­al head of late-stage de­vel­op­ment, di­ag­nos­tics and med­ical af­fairs in Janssen R&D’s hema­tol­ogy and on­col­o­gy units, in a state­ment.

Im­bru­vi­ca was first ap­proved to treat cGVHD in the sec­ond-line adult pop­u­la­tion back in 2017. But be­fore that, it snagged an ac­cel­er­at­ed ap­proval in sec­ond-line MCL in 2013 and has since racked up a range of oth­er in­di­ca­tions.

Janssen and Ab­b­Vie are fac­ing heat from As­traZeneca in CLL, where the ri­val phar­ma post­ed some long-term da­ta from a head-to-head tri­al com­par­ing Im­bru­vi­ca to its own BTK in­hibitor Calquence at AS­CO last year. BeiGene is al­so po­si­tion­ing its Brukin­sa in CLL, and Eli Lil­ly is re­cruit­ing for a head-to-head tri­al test­ing its LOXO-305 against Im­bru­vi­ca, Calquence and Brukin­sa in MCL.

Last sum­mer, a Delaware dis­trict court up­held J&J and Ab­b­Vie’s patent ex­clu­siv­i­ty for Im­bru­vi­ca, like­ly de­lay­ing gener­ic com­pe­ti­tion un­til 2032.

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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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.

Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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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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Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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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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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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