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. Right here’s your rundown of Monday’s information.
By the best way, watching large Navy ships depart port simply behind the conference middle will at all times be a formidable sight.
With AQUILA information, Johnson & Johnson seeks first drug approval for therapy of smoldering myeloma
Therapy 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 Section 3 AQUILA trial. These 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 count on that regulatory companies will give the applying a “thumbs up,” stated Ola Landgren, a myeloma doctor and researcher on the College of Miami who didn’t work on the research.
The research randomized 390 smoldering myeloma sufferers to both obtain Darzalex or obtain energetic monitoring. After a median of 65.2 months, sufferers who obtained Darzalex had a 51% diminished danger of illness development or dying, and progression-free survival at 5 years was 63.1% amongst sufferers who obtained the drug and 40.8% amongst sufferers who didn’t. Seventeen sufferers within the Darzalex group additionally achieved a whole response or higher, in accordance with the outcomes revealed within the New England Journal of Drugs on Monday.
Treating sufferers with smoldering myeloma has lengthy been a hotly debated matter amongst myeloma physicians, partially as a result of clinicians weren’t sure that early therapy benefitted sufferers; it’s additionally typically unclear which smoldering sufferers will go on to develop energetic myeloma. These sufferers who’re fated to develop a number of myeloma may have stronger remedy than Darzalex alone, argued Hearn Jay Cho, the CMO of the A number of Myeloma Analysis Basis, who wasn’t concerned with the work.
“When 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. That is the dilemma,” he stated. The info are thrilling, Cho stated, however he felt there are nonetheless challenges earlier than adopting this as customary therapy for smoldering myeloma.
Different physicians strongly disagreed. “You may’t argue anymore that there isn’t any profit from early therapy,” stated Irene Ghobrial, a myeloma doctor and researcher on the Dana-Farber Most cancers Institute who didn’t work on the research. She stated she plans to make use of Darzalex in a few of her smoldering sufferers, although she added she wasn’t positive if everybody within the myeloma neighborhood will.
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Blenrep’s comeback story retains rolling on
A mix therapy with the GSK drug Blenrep reduce the danger of dying by 42% amongst sufferers with relapsed or refractory a number of myeloma, in accordance with interim information from the DREAMM-7 trial introduced Monday. Primarily based on the information to this point, GSK projected that the median total survival interval will attain 84 months for the Blenrep arm in comparison with 51 months for the comparator.
As readers most likely bear in mind, Blenrep gained accelerated approval from the FDA in 2020 as a complicated myeloma therapy. However after it failed in a confirmatory research, GSK withdrew it from the market.
GSK saved up this system, nonetheless, and with the success in DREAMM-7, in addition to one other research known as DREAMM-8, it lately refiled Blenrep with the FDA. An approval choice is predicted by subsequent summer season. The corporate has lofty ambitions for transferring the drug into earlier strains of therapy as nicely.
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Rising efforts to ease ‘conditioning’ earlier than genetic therapies
The approval one yr 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 residing with a debilitating blood illness lengthy uncared for by the medical institution. However entry to the near-curative therapies has been slowed by their excessive price and the difficult procedures required to manage them.
New analysis introduced right here highlighted rising efforts to take away one of the vital regarding obstacles for sufferers: a “conditioning” course of a extremely poisonous chemotherapy known as busulfan required to eradicate 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 can maintain a whole lot of sufferers again from getting therapy, and it ought to,” stated 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, driving excessive on promising outcomes of a small molecule drug for acute myeloid leukemia, believes its remedy has blockbuster potential — though it’s going through stiff competitors that features a pharma large 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. Throughout ASH, Kura introduced Section 1 trial outcomes displaying that 100% of newly identified 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 nicely 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 really has the potential to be game-changing,” Troy Wilson, Kura’s CEO, advised STAT’s Jonathan Wosen. “You might be turning acute leukemia right into a persistent, manageable situation like hypertension. That might be the purpose.”
Market analysts favored 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 gained approval final month for a menin inhibitor in relapsed or refractory AML sufferers with mutations in KMT2A. However 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 best way 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 Section 3 research the corporate and its improvement accomplice Kyowa Kirin, will launch subsequent yr.
Info on Oxbryta’s withdrawal nonetheless scant
Pfizer representatives attended however didn’t communicate throughout a particular assembly Monday set as much as focus on the current withdrawal of Oxbryta, its drug for sickle cell illness resulting from security issues. The shortage of recent info on what, precisely, occurred with the drug left session attendees asking questions that couldn’t be answered.
There’s a “lengthy record of questions” that Pfizer and regulators want to deal with, stated John Strouse, a sickle cell illness skilled from Duke College who chaired Monday’s assembly.
Alexis Thompson, a pediatric hematologist on the Youngsters’s Hospital of Philadelphia, stated well timed disclosures of scientific investigations is critical, however “clearly didn’t occur right here.”
A report from the European Medicines Company is predicted to be launched later this month, though an FDA inquiry will possible take longer, Strouse added.
