Latest news with #NPM1
National Post
4 days ago
- Health
- National Post
LabPMM® Receives New York State Approval for the NPM1 MRD Assay - Informing Therapy and Accelerating Targeted Trials
Article content SAN DIEGO — Invivoscribe is happy to announce that its wholly owned subsidiary, the Laboratory for Personalized Molecular Medicine ® (LabPMM) has received approval from New York State (NYS) for the NPM1 MRD Assay. This approval comes just two months after gaining NYS approval for our FLT3 ITD MRD Assay. Together these tests represent a critical tool for patients with acute myeloid leukemia (AML), clinicians and pharmaceutical companies. This new approval underscores Invivoscribe's ongoing commitment to providing the most accurate, standardized measurable residual disease (MRD) testing solutions worldwide. Article content The NPM1 MRD Assay is a pivotal development in the fight against AML, offering an ultra-sensitive DNA sequencing method to accurately measure trace levels of residual leukemia cells in patients with the NPM1 mutation variants. NPM1 mutations are considered an ideal target for MRD assessment because they are present in ~30% of adult AML cases, 1 stable over time, 2 and, if present in blood at allele fractions ≥0.01%, are associated with increased relapse and worse overall survival. 3 Recent studies show emerging evidence that pre-transplant MRD testing for NPM1 and FLT3 -ITD identifies AML patients in remission who are most likely to relapse or experience poor survival. 3,4,5 With this approval, LabPMM is helping to transform the landscape of AML research, treatment and drug development. By using MRD as a surrogate endpoint in clinical trials, instead of relying solely on overall survival (OS), pharmaceutical companies can accelerate their drug development timelines. This is particularly valuable in acute disease, where time is of the essence, and earlier intervention can dramatically improve patient outcomes. Article content 'We are proud to receive New York State approval for our NPM1 MRD Assay by NGS, marking our second assay approved by New York State this year,' said Jordan Thornes, V.P., Global Clinical Laboratory Operations at LabPMM. 'This milestone reflects our continued dedication to advancing precision diagnostics in cancer care. With this latest approval, we're further empowering clinicians with sensitive, reliable tools to detect residual disease and guide treatment decisions with confidence.' Article content LabPMM's NPM1 and FLT3 ITD MRD Assays are standardized next generation sequencing (NGS) tests that complement the LeukoStrat ® CDx FLT3 Mutation Assay, which is used to guide treatment selection for patients with AML. These services are offered in the U.S., European Union, and across Asia to ensure patients around the world have access to high-quality, standardized testing and to support the development of cutting-edge cancer treatments. LabPMM remains committed to advancing precision medicine and improving outcomes for patients worldwide. For more information about the NPM1 MRD Assay and LabPMM's full test menu, please visit or contact us at inquiry@ and follow us on LinkedIn. Article content About Invivoscribe Article content Invivoscribe ® is a global, vertically integrated biotechnology company dedicated to Improving Lives with Precision Diagnostics ®. For thirty years, Invivoscribe has improved the quality of healthcare worldwide by providing high quality standardized reagents, tests, and bioinformatics tools to advance the field of precision medicine. Invivoscribe has a successful track record of partnerships with pharmaceutical companies interested in clinical trial testing via our global lab network located in the U.S., Germany, Japan and China, and in developing and commercializing companion diagnostics, with our rigorous expertise in both regulatory and laboratory services. Providing distributable kits, as well as clinical trial services through its globally located clinical lab subsidiaries (LabPMM ®), Invivoscribe is an ideal partner from diagnostic development, through clinical trials, regulatory submissions, and commercialization. Article content Article content Article content Article content Article content Article content
Business Wire
4 days ago
- Business
- Business Wire
LabPMM ® Receives New York State Approval for the NPM1 MRD Assay - Informing Therapy and Accelerating Targeted Trials
SAN DIEGO--(BUSINESS WIRE)--Invivoscribe is happy to announce that its wholly owned subsidiary, the Laboratory for Personalized Molecular Medicine ® (LabPMM) has received approval from New York State (NYS) for the NPM1 MRD Assay. This approval comes just two months after gaining NYS approval for our FLT3 ITD MRD Assay. Together these tests represent a critical tool for patients with acute myeloid leukemia (AML), clinicians and pharmaceutical companies. This new approval underscores Invivoscribe's ongoing commitment to providing the most accurate, standardized measurable residual disease (MRD) testing solutions worldwide. The NPM1 MRD Assay is a pivotal development in the fight against AML, offering an ultra-sensitive DNA sequencing method to accurately measure trace levels of residual leukemia cells in patients with the NPM1 mutation variants. NPM1 mutations are considered an ideal target for MRD assessment because they are present in ~30% of adult AML cases, 1 stable over time, 2 and, if present in blood at allele fractions ≥0.01%, are associated with increased relapse and worse overall survival. 3 Recent studies show emerging evidence that pre-transplant MRD testing for NPM1 and FLT3 -ITD identifies AML patients in remission who are most likely to relapse or experience poor survival. 3,4,5 With this approval, LabPMM is helping to transform the landscape of AML research, treatment and drug development. By using MRD as a surrogate endpoint in clinical trials, instead of relying solely on overall survival (OS), pharmaceutical companies can accelerate their drug development timelines. This is particularly valuable in acute disease, where time is of the essence, and earlier intervention can dramatically improve patient outcomes. 'We are proud to receive New York State approval for our NPM1 MRD Assay by NGS, marking our second assay approved by New York State this year,' said Jordan Thornes, V.P., Global Clinical Laboratory Operations at LabPMM. 'This milestone reflects our continued dedication to advancing precision diagnostics in cancer care. With this latest approval, we're further empowering clinicians with sensitive, reliable tools to detect residual disease and guide treatment decisions with confidence.' LabPMM's NPM1 and FLT3 ITD MRD Assays are standardized next generation sequencing (NGS) tests that complement the LeukoStrat ® CDx FLT3 Mutation Assay, which is used to guide treatment selection for patients with AML. These services are offered in the U.S., European Union, and across Asia to ensure patients around the world have access to high-quality, standardized testing and to support the development of cutting-edge cancer treatments. LabPMM remains committed to advancing precision medicine and improving outcomes for patients worldwide. For more information about the NPM1 MRD Assay and LabPMM's full test menu, please visit or contact us at inquiry@ and follow us on LinkedIn. About Invivoscribe Invivoscribe ® is a global, vertically integrated biotechnology company dedicated to Improving Lives with Precision Diagnostics ®. For thirty years, Invivoscribe has improved the quality of healthcare worldwide by providing high quality standardized reagents, tests, and bioinformatics tools to advance the field of precision medicine. Invivoscribe has a successful track record of partnerships with pharmaceutical companies interested in clinical trial testing via our global lab network located in the U.S., Germany, Japan and China, and in developing and commercializing companion diagnostics, with our rigorous expertise in both regulatory and laboratory services. Providing distributable kits, as well as clinical trial services through its globally located clinical lab subsidiaries (LabPMM ®), Invivoscribe is an ideal partner from diagnostic development, through clinical trials, regulatory submissions, and commercialization.
Yahoo
13-06-2025
- Health
- Yahoo
Syndax reports publication of revumenib data from BEAT AML trial
Syndax (SNDX) Pharmaceuticals announced that data from the BEAT AML trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mutant NPM1 and KMT2A-rearranged acute myeloid leukemia patients were published in the Journal of Clinical Oncology and simultaneously presented in an oral session at the 30th European Hematology Association Annual Congress Meeting being held June 12-15, 2025, in Milan, Italy and virtually. The publication and EHA presentation report updated results from the Phase 1b BEAT AML trial evaluating the safety and clinical activity of revumenib in combination with venetoclax/azacitidine in newly diagnosed older adults with mNPM1 or KMT2Ar AML. The trial is being conducted as part of The Leukemia & Lymphoma Society's Beat AML Master Clinical Trial. As of September 2024, 43 patients were enrolled and treated in BEAT AML across two dose levels of revumenib in combination with venetoclax and azacitidine. Overall, 79% of patients had mNPM1 AML and 21% had KMT2Ar AML. Revumenib was generally well tolerated at both dose levels in combination with venetoclax and azacitidine without a maximal tolerated dose identified. The most common overall non-hematologic treatment-emergent adverse events (TEAEs) of any grade were nausea (60%), constipation (53%), QTc prolongation (44%), hypokalemia (44%), and vomiting (42%). Overall Grade greater than or equal to3 non-hematologic AEs were rare and similar between both dose levels. In the intent-to-treat population, the observed rate of complete remission was 67%, composite complete remission was 81%, and the overall response rate was 88%. Among 37 patients with measurable residual disease response assessment, 100% were MRD negative by centralized flow cytometry testing. The median duration of CRc was 12.0 months. 23% of patients had proceeded to hematopoietic stem cell transplantation as of the February 2025 data cut off. In an early analysis of survival from this single-arm trial, the median overall survival observed was 15.5 months. Subset analysis showed a CRc rate of 77% and an observed median OS of 15.5 months in mNPM1 patients with intermediate risk by ELN 2024, and a CRc rate of 89% and observed median OS of 18.0 months in KMT2Ar patients. In contrast, historical data from newly diagnosed mNPM1 patients with intermediate risk treated with venetoclax and azacitidine show a CRc of 57% and median OS of 9.9 months.1 In newly diagnosed KMT2Ar AML patients treated with venetoclax and hypomethylating agent therapy, a CRc rate of 43% and median OS of 2.5 months was observed in a retrospective analysis. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on SNDX: Disclaimer & DisclosureReport an Issue Syndax announces new data from AUGMENT-101 trial of Revuforj Syndax Pharmaceuticals: Promising 2025 Outlook and Strategic Product Launches Drive Buy Rating Closing Bell Movers: slips 3% after results despite topping estimates Syndax exec buys $105.8K, director buys $91.1K in common stock Syndax Pharmaceuticals Appoints New Head of R&D Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Insider
03-06-2025
- Business
- Business Insider
Kura Oncology says ‘positive' results from KOMET-001 Phase 2 trial of ziftomenib
Kura Oncology (KURA) announced the presentation of positive pivotal results from the KOMET-001 Phase 2 registration-directed trial of ziftomenib, a once-daily, oral investigational menin inhibitor, in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia in an oral session at the 2025 American Society of Clinical Oncology Annual Meeting being held in Chicago, IL from May 30 – June 3, 2025. 'We are delighted to announce positive pivotal data from the KOMET-001 trial in R/R NPM1-mutated AML patients treated with ziftomenib,' said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. 'NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this patient population. With these encouraging results and a PDUFA target action date of November 30, 2025, we and our partners at Kyowa Kirin look forward to supporting FDA with its review of the ziftomenib New Drug Application and are well-positioned to meaningfully impact relapsed or refractory patients with NPM1 mutations.' The KOMET-001 Phase 2 population included 92 adult patients with R/R NPM1-m AML. The median age was 69. Patients were heavily pretreated, with 33% having received three or more prior lines of therapy (median prior lines: 2) and 59% having been previously treated with venetoclax. A complete remission plus CR with partial hematological recovery rate of 23% was observed among patients with R/R NPM1-m AML in the Phase 2 portion of the KOMET-001 trial. Among those 21 patients who achieved CR/CRh, 13 had a CR and 8 had a CRh. The median duration of CR/CRh responses was 3.7 months and the restricted mean duration of response was 4.3 months at the time of the data cutoff. MRD status was assessed in 19 of 21 patients who achieved CR/CRh, and 63% of these patients were MRD-negative. Comparable CR/CRh rates were observed across pre-specified subgroups, regardless of prior HSCT, prior venetoclax or FLT3/IDH co-mutations. Additional patient benefit beyond CR/CRh was observed with a rate of transfusion conversion of 21% and a rate of maintenance of transfusion independence of 20%. A median OS of 16.4 months was observed for responders and a median overall survival of 3.5 months was observed among non-responders. The safety population included 112 adult patients with R/R NPM1-m AML from the pooled Phase 1b and Phase 2 portions of the KOMET-001 trial. The safety profile observed with ziftomenib in this population was consistent with previously reported data. Treatment-related adverse events led to treatment discontinuations in 3% of patients. TRAEs of Grade greater than or equal to3 which occurred in more than 10% of patients were limited to differentiation syndrome, which was well managed by protocol-specified mitigation strategies and no Grade 4/5 treatment-related DS was observed. Although QTc prolongation was reported in three patients per investigator assessment, all three patients were on concomitant medications associated with QTc prolongation, two had electrolyte abnormalities and one had a prior diagnosis of atrial fibrillation.
The Star
13-05-2025
- Health
- The Star
Acute myeloid leukaemia: New test improves survival time
A highly sensitive test that detects traces of disease in the bone marrow of patients with a rare and aggressive type of blood cancer could help double their chances of survival, a first-of-its-kind trial has found. The 10-minute procedure – which involves an injection in the hip bone every three months – identified signs of acute myeloid leukaemia (AML) returning in patients before it showed in blood tests. This gives medics a 'window of opportunity' to treat people while they are still well, researchers said. Experts hope the test could become routine care for patients with AML. Detecting low levels AML is a cancer that causes the bone marrow to produce a large number of abnormal blood cells, with tens of thousands of people diagnosed with it around the world each year. The risk of developing the disease increases with age and it is most common in people over 75. After treatment, patients are usually seen every few weeks for blood tests. Trial chief investigator and Britain's Guy's and St Thomas' NHS Foundation Trust honorary consultant haematologist Professor Dr Nigel Russell said: 'There's a significant risk for these patients that the leukaemia is going to come back again, and that's normally detected by a deterioration in the blood tests. 'Sometimes it can happen very quickly, and sometimes it can happen over a period of weeks. 'So normally, the patient comes along, has a blood test done, the blood test is okay, they're sent off again. 'That's the procedure that's been the case for many years. 'But the trial included these extra tests in the bone marrow every three months to detect if there's any evidence of what we call residual leukaemia; it's such a low level that the blood tests are normal, but the disease can still be present at very low levels in the bone marrow.' The bone marrow test takes around 10 minutes and is performed under local anaesthetic, with a hollow needle inserted into the patient's hip bone to take the sample. 'It detects the RNA present in the leukaemia cells, but not present in normal cells,' Prof Russell added. Starting treatment while well For the study, led by King's College London and published in The Lancet Haematology journal, patients were screened for mutations in the NPM1 and FLT3 genes, which are common in younger people with AML. The trial involved 637 people who were in remission from AML and had, over three years, either received standard monitoring or standard monitoring with additional bone marrow tests every three months. Prof Russell said: 'It was about one-third of patients that really benefited from this approach that had a doubling of their survival, which was a massive improvement in outcome for these patients. 'And really, it's quite a simple extra procedure to have done. 'Since the results of the trial have become available, this sort of technology is now part of what we call the standard of care. 'The important thing is that it gives you a window of opportunity to treat the patient before they become unwell. 'Because if the patient relapses, they can become quite unwell very quickly and their blood counts are very abnormal. 'They have to come into hospital and they have to have further treatment of chemotherapy, and then possibly go on to a bone marrow transplant. 'The advantage of detecting it early is the patients can have these treatments when they're still well – even potentially as an outpatient, because their blood tests are normal. 'They don't need to come into hospital as an emergency and they're starting with a fresh start really, rather than being ill again.' Giving more time Jane Leahy, 51, from Wimbledon, London, is one of the patients who took part in the trial. She was diagnosed with AML in December 2014 at the age of 41, and after chemotherapy, went into remission the following April. The mother-of-two had her bone marrow tested as part of the trial, which picked up abnormalities a few months later. After restarting treatment, the bone marrow test results also showed the chemotherapy was not working, with a stem cell transplant her only chance of going into remission again. 'Without testing, I wouldn't have gone on to have my transplant, because my blood showed I was still in remission,' she said. Nine years after her bone marrow transplant – which was donated by her sister – Leahy is still in remission. She added: 'It's pretty scary really, when I think about potentially what could have happened. 'And there were so many things throughout my treatment journey, it felt like the stars were aligning. 'I feel incredibly lucky.' Leahy said bone marrow testing gives doctors 'more time', adding: 'The time is always critical, because it's so acute and so aggressive. 'And a lot of people die from AML before they get to treatment, because it's just diagnosed so late. 'So it just gives more time, more opportunities to try different treatment approaches and pick it up earlier, before it becomes too late to do anything about.' – PA Media/dpa
