This roundup of stories from the ASH 2024 convention first appeared in STAT’s “ASH in 30” e-newsletter. To get future editions, join right here.
Welcome again, ASH followers. Here’s your rundown of Monday’s information.
By the way in which, watching large Navy ships depart port simply behind the conference middle will all the time be a powerful sight.
With AQUILA information, Johnson & Johnson seeks first drug approval for remedy of smoldering myeloma
Treatment with daratumumab or J&J’s Darzalex alone appeared to enhance outcomes for sufferers with high-risk smoldering myeloma, a situation that precedes a number of myeloma, within the major outcomes of the randomized Phase 3 AQUILA trial. Those outcomes are a part of functions that J&J despatched to the FDA and EMA for approval for Darzalex monotherapy to deal with high-risk smoldering myeloma, and specialists anticipate that regulatory businesses will give the appliance a “thumbs up,” mentioned Ola Landgren, a myeloma doctor and researcher on the University of Miami who didn’t work on the examine.
The examine randomized 390 smoldering myeloma sufferers to both obtain Darzalex or obtain energetic monitoring. After a median of 65.2 months, sufferers who acquired Darzalex had a 51% lowered danger of illness development or dying, and progression-free survival at 5 years was 63.1% amongst sufferers who acquired the drug and 40.8% amongst sufferers who didn’t. Seventeen sufferers within the Darzalex group additionally achieved an entire response or higher, based on the outcomes printed within the New England Journal of Medicine on Monday.
Treating sufferers with smoldering myeloma has lengthy been a hotly debated subject amongst myeloma physicians, partially as a result of clinicians weren’t sure that early remedy benefitted sufferers; it’s additionally typically unclear which smoldering sufferers will go on to develop energetic myeloma. Those sufferers who’re fated to develop a number of myeloma might have stronger remedy than Darzalex alone, argued Hearn Jay Cho, the CMO of the Multiple Myeloma Research Foundation, who wasn’t concerned with the work.
“If you attempt to do one thing mild, attenuated, extra tolerable, you undertreat the people who find themselves on the cusp of a number of myeloma. This is the dilemma,” he mentioned. The information are thrilling, Cho mentioned, however he felt there are nonetheless challenges earlier than adopting this as commonplace remedy for smoldering myeloma.
Other physicians strongly disagreed. “You can’t argue anymore that there isn’t any profit from early remedy,” mentioned Irene Ghobrial, a myeloma doctor and researcher on the Dana-Farber Cancer Institute who didn’t work on the examine. She mentioned she plans to make use of Darzalex in a few of her smoldering sufferers, although she added she wasn’t certain if everybody within the myeloma neighborhood will.
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Blenrep’s comeback story retains rolling on
A mixture remedy with the GSK drug Blenrep lower the chance of dying by 42% amongst sufferers with relapsed or refractory a number of myeloma, based on interim information from the DREAMM-7 trial offered Monday. Based on the information thus far, GSK projected that the median general survival interval will attain 84 months for the Blenrep arm in comparison with 51 months for the comparator.
As readers in all probability bear in mind, Blenrep received accelerated approval from the FDA in 2020 as a sophisticated myeloma remedy. But after it failed in a confirmatory examine, GSK withdrew it from the market.
GSK stored up this system, nevertheless, and with the success in DREAMM-7, in addition to one other examine known as DREAMM-8, it not too long ago refiled Blenrep with the FDA. An approval resolution is anticipated by subsequent summer time. The firm has lofty ambitions for shifting the drug into earlier traces of remedy as properly.
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Emerging efforts to ease ‘conditioning’ earlier than genetic remedies
The approval one 12 months in the past of two genetic medicines for sickle cell illness, together with the primary remedy powered by CRISPR gene modifying, was a milestone for folks dwelling with a debilitating blood illness lengthy uncared for by the medical institution. But entry to the near-curative remedies has been slowed by their excessive price and the difficult procedures required to manage them.
New analysis offered right here highlighted rising efforts to take away one of the crucial regarding obstacles for sufferers: a “conditioning” course of a extremely poisonous chemotherapy known as busulfan required to get rid of diseased cells from the bone marrow and make room for the genetically modified cells.
“Busulfan conditioning is a really difficult a part of the process that may maintain a variety of sufferers again from getting remedy, and it ought to,” mentioned John Evans, CEO of Beam Therapeutics, which is growing a CRISPR-based remedy for sickle cell, and a softer conditioning routine, additionally powered by CRISPR, that eliminates the necessity for busulfan.
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Kura Oncology touts encouraging early-stage trial outcomes for menin inhibitor in AML
Kura Oncology, using excessive on promising outcomes of a small molecule drug for acute myeloid leukemia, believes its remedy has blockbuster potential — despite the fact that it’s going through stiff competitors that features a pharma big and a biotech that has beat it in a race for regulatory approval.
The San Diego biotech is growing a molecule that inhibits menin, a protein that regulates the manufacturing of different proteins, together with people who drive most cancers cells to develop and unfold uncontrollably. During ASH, Kura introduced Phase 1 trial outcomes displaying that 100% of newly recognized AML sufferers with mutations within the gene NPM1 had full responses to its oral drug, ziftomenib, mixed with an intensive chemotherapy routine, whereas 83% of sufferers with rearrangements of the gene KMT2A had full responses to this routine and Kura’s drug.
Ziftomenib was properly tolerated in any respect doses studied, and 50 out of 51 sufferers taking ziftomenib and intensive chemo (cytarabine and daunorubicin) have been nonetheless alive as of the Oct. 1 information cutoff.
“This actually has the potential to be game-changing,” Troy Wilson, Kura’s CEO, advised STAT’s Jonathan Wosen. “You are turning acute leukemia right into a power, manageable situation like hypertension. That can be the purpose.”
Market analysts appreciated the information, with analysts for Cantor writing that this was a “best-case information replace.”
Johnson & Johnson is growing its personal menin inhibitor. And Kura is going through competitors from Syndax, a Massachusetts biotech that received approval final month for a menin inhibitor in relapsed or refractory AML sufferers with mutations in KMT2A. But Wilson argues that the best market alternative is within the first-line setting, and he provides that Kura’s drug doesn’t have an effect on coronary heart rhythms or blood cell counts in the way in which that Syndax’s drug does. Wilson believes ziftomenib may someday usher in $5 billion in U.S. gross sales, although that’ll rely partially on the outcomes of two international Phase 3 research the corporate and its growth associate Kyowa Kirin, will launch subsequent 12 months.
Information on Oxbryta’s withdrawal nonetheless scant
Pfizer representatives attended however didn’t communicate throughout a particular assembly Monday set as much as talk about the latest withdrawal of Oxbryta, its drug for sickle cell illness attributable to security issues. The lack of recent data on what, precisely, occurred with the drug left session attendees asking questions that couldn’t be answered.
There’s a “lengthy listing of questions” that Pfizer and regulators want to deal with, mentioned John Strouse, a sickle cell illness professional from Duke University who chaired Monday’s assembly.
Alexis Thompson, a pediatric hematologist on the Children’s Hospital of Philadelphia, mentioned well timed disclosures of scientific investigations is critical, however “clearly didn’t occur right here.”
A report from the European Medicines Agency is anticipated to be launched later this month, though an FDA inquiry will doubtless take longer, Strouse added.
