Waldenstrom Macroglobulinemia

Ongoing international oncology collaboration works to empower health care workers and save lives across diverse resource environments. Accra, GHANA, Oct. 29, 2024 /PRNewswire/ -- International oncology experts are gathering in Accra, Ghana for a series of meetings beginning today, to update cancer treatment recommendations in the NCCN Harmonized Guidelines™ for Sub-Saharan Africa. This is the latest event from a longstanding collaboration between the African Cancer Coalition (ACC), American Cancer Society (ACS), and National Comprehensive Cancer Network® (NCCN®), and the Clinton Health Access Initiative (CHAI) that collectively form the group Allied Against Cancer. Together, the organizations are focused on providing context-appropriate, evidence-based cancer care strategies that address the unique challenges faced across the region. Continue Reading NCCN Harmonized Guidelines for Sub-Saharan Africa available for free at NCCN.org/harmonized. The NCCN Harmonized Guidelines™ for Sub-Saharan Africa are free resources for optimizing and standardizing cancer care across the region. They are available at NCCN.org/harmonized or via the Virtual Library of NCCN Guidelines® App. "The Sub-Saharan Africa region is dealing with a rising rate of cancer incidences. Standardizing care based on the latest evidence-based expert consensus-driven recommendations helps ensure consistency in treatment and enables better planning for how to use available resources," said Dr. Henry Ddungu, Senior Consultant, Hematology/Oncology; African Cancer Coalition. "We can ensure people with cancer receive the most effective treatment by harnessing local expertise combined with global best practices." During the meeting, the updates to the NCCN Harmonized Guidelines™ for Sub-Saharan Africa will focus on caring for blood malignancies and children's cancers, including: Acute Lymphoblastic Leukemia Acute Myeloid Leukemia B-cell Lymphomas Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Chronic Myeloid Leukemia Hodgkin Lymphoma Multiple Myeloma Pediatric Acute Lymphoblastic Leukemia Pediatric Aggressive B-Cell Lymphomas Pediatric Central Nervous System Cancers Waldenstrom's Macroglobulinemia/Lymphoplasmacytic Lymphoma "Since the launch of Allied Against Cancer and publication of the first NCCN Harmonized Guidelines™ for Sub-Saharan Africa in 2017, we have grown to include guidelines covering more than 90% of adult cancer incidences, and most pediatric cancers," said Anu Agrawal, MD, Vice President, Global Cancer Support, American Cancer Society (ACS). "We are proud of the work we've accomplished collaboratively and excited to see continued progress in cancer care across the region." "As we strive for health equity across the globe, it is vital that health care providers are equipped with the best tools and resources to deliver optimal care for a variety of different settings," said Crystal S. Denlinger, MD, Chief Executive Officer, NCCN. "These guidelines are a critical step for helping cancer care professionals stay current on the latest advancements in treatment. They provide a roadmap for ideal options while also taking into account the challenges that exist when delivering care in resource limited settings." Dr. Denlinger was joined by NCCN's Chief Medical Officer, Wui-Jin Koh, MD, along with the following NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Panel leadership: Ranjana H. Advani, MD, Stanford Cancer Institute Jessica K. Altman, MD, Robert H. Lurie Comprehensive Cancer Center of Northwestern University Hiroto Inaba, MD, PhD, St. Jude Children's Research Hospital/The University of Tennessee Health Science Center The NCCN Harmonized Guidelines™ offer color-coded tiers of treatment recommendations based on resource availability. They cover more than 40 different cancer types, plus screening, supportive care, and specific population concerns. They have been utilized nearly 70,000 times since 2017 and have been endorsed by the Federal Ministries of Health and leading cancer centers in Ethiopia, Liberia, Malawi, Nigeria, Tanzania, Uganda, and Zambia. Learn more about Allied Against Cancer's collaborative work to improve cancer care in Sub-Saharan Africa at alliedagainstcancer.org. Visit NCCN.org/global for other free international cancer prevention and treatment resources. About the National Comprehensive Cancer Network The National Comprehensive Cancer Network® (NCCN ®) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, equitable, and accessible cancer care so all patients can live better lives. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines ®) provide transparent, evidence-based, expert consensus recommendations for cancer treatment, prevention, and supportive services; they are the recognized standard for clinical direction and policy in cancer management and the most thorough and frequently-updated clinical practice guidelines available in any area of medicine. The NCCN Guidelines for Patients® provide expert cancer treatment information to inform and empower patients and caregivers, through support from the NCCN Foundation®. NCCN also advances continuing education, global initiatives, policy, and research collaboration and publication in oncology. Visit NCCN.org for more information. Media Contact: Rachel Darwin 267-622-6624 [email protected] Photo - Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NX-5948 demonstrated robust clinical activity with objective responses observed in 7 of 9 (77.8%) evaluable Waldenstrom’s patients in the ongoing Phase 1a/1b clinical trial Responses are durable and deepen over time with two patients on treatment for greater than one year Data were presented at the 12th International Workshop on Waldenstrom’s Macroglobulinemia (IWWM-12) Oct. 19, 2024 -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced the presentation of clinical data from its ongoing Phase 1a/1b clinical trial of NX-5948, an orally bioavailable, brain penetrant degrader of Burton’s tyrosine kinase (BTK), in patients with relapsed/refractory Waldenstrom’s macroglobulinemia (WM) at the 12th International Workshop on Waldenstrom’s Macroglobulinemia (IWWM-12) which is being held in Prague, Czech Republic October 17–19, 2024. “We are encouraged by the emerging positive data from NX-5948 in patients with Waldenstrom’s macroglobulinemia, which add to the previously disclosed robust clinical activity observed in patients with chronic lymphocytic leukemia,” said Paula G. O’Connor, M.D., chief medical officer of Nurix. “These data support our decision to advance NX-5948 into the ongoing Phase 1b expansion cohort in patients who have previously received at least one prior line of therapy including a BTK inhibitor and patients presenting with Bing-Neel syndrome, a rare form of WM with central nervous system involvement where NX-5948’s ability to penetrate the brain may offer a distinct advantage.” The data presented at IWWM-12 included previously reported safety findings for all patients in the Phase 1a dose escalation study treated with NX-5948 at doses ranging from 50 mg to 600 mg once daily by oral administration regardless of diagnosis (n=79) based on an April 17, 2024 data cut. NX-5948 demonstrated a tolerable safety profile, and the safety profile for patients with WM was consistent with the safety profile for the overall population (WM patient safety data not shown separately). New data from an October 10, 2024 data cut include the baseline characteristics of the first 13 patients with WM enrolled across both the Phase 1a and Phase 1b portions of the trial, clinical response assessments in 9 response-evaluable patients, and duration on study for all 13 patients. Among the 13 WM patients, the median age was 74 years and the median number of prior lines of therapy was 3. All 13 patients previously had been treated with both BTK inhibitors (BTKi) and chemotherapy/chemo-immunotherapy. Three patients (23.1%) had received prior treatment with the non-covalent BTKi pirtobrutinib, and one patient (7.7%) had received prior treatment with a BCL2 inhibitor. Baseline mutation status in MYD88 and CXCR4 was captured from patient records, and eight patients (61.5%) had mutations in MYD88, and two patients (15.4%) had mutations in CXCR4. Among the nine patients who were evaluable for response, seven patients (77.8%) had an objective response and two patients experienced stable disease (22.2%). All seven responses were observed at the first assessment at 8 weeks, and five remain on treatment with two patients on treatment for longer than one year. Responses were observed in patients regardless of their baseline mutations in MYD88 and CXCR4. Two illustrative cases studies of patients treated with NX-5948 were presented. The first case study is a patient with baseline MYD88 and CXCR4 mutations and four prior lines of therapy, including autologous bone marrow transplantation and ibrutinib, who demonstrated a rapid response observed at the first assessment and remained on study at the time of the October 10, 2024 data cut with greater than one year of treatment (currently in cycle 16; 28 days per cycle). NX-5948 treatment resulted in deepening of response over time as measured by reduction in serum IgM levels, a key biomarker of clinical response in WM patients. The second case study is a patient with baseline MYD88 mutation and three prior lines of treatment, having most recently progressed while on zanubrutinib. This patient also experienced a rapid response at the first assessment with decreasing IgM through treatment which was ongoing in cycle 15 at the time of the October 10, 2024 data cut. The IWWM-1 presentation is available in the Scientific Resources section of Nurix website in the Posters and Presentations section. NX-5948 is an investigational, orally bioavailable, brain penetrant, small molecule degrader of BTK. NX-5948 is currently being evaluated in a Phase 1 clinical trial in patients with relapsed or refractory B cell malignancies. Nurix has previously reported that NX-5948 is highly potent against a range of tumor cell lines that are resistant to current BTK inhibitor therapies, an important consideration in heavily pretreated CLL/SLL patient populations. Additional information on the ongoing clinical trial can be accessed at clinicaltrials.gov (NCT05131022). WM is a rare, slow growing type of non-Hodgkin’s lymphoma that is characterized by the replacement of normal bone marrow cells by malignant lymphocytic cells that produce monoclonal IgM. This replacement leads to anemia, bleeding, and impaired immune function, while the elevated IgM levels may cause neurologic symptoms. In the United States the annual incidence rate is approximately 3 per million or between 1,000 to 1,500 newly diagnosed patients per year. Recommended first-line treatments including chemoimmunotherapy and BTK inhibitor therapy. There are no therapies approved to treat patients after BTKi. Additional therapeutic options are needed. Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative small molecules and antibody therapies based on the modulation of cellular protein levels as a novel treatment approach for cancer, inflammatory conditions, and other challenging diseases. Leveraging extensive expertise in E3 ligases together with proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform, to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin-proteasome system to selectively decrease or increase cellular protein levels. Nurix’s wholly owned, clinical stage pipeline includes targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates activation of multiple immune cell types including T cell and NK cells. Nurix is headquartered in San Francisco, California. For additional information visit http://www.nurixtx.com. The content above comes from the network. if any infringement, please contact us to modify.

Nurix Therapeutics’ BTK degrader has elicited responses in a small group of patients with certain cancers, supporting the biotech’s claim that the drug could potentially offer a better alternative to BTK inhibitors. The Phase 1a/1b trial tested Nurix’s candidate, NX-5948, in 13 people with Waldenstrom’s macroglobulinemia, nine of whom were evaluable for response at data cutoff. Seven patients had an objective response within the first eight weeks, and the remaining two had stable disease. The responses were seen regardless of mutation status, Nurix said. All 13 patients had an average three prior lines of treatment, including both BTK inhibitors and chemotherapy or chemo-immunotherapy, according to results presented Saturday at the International Workshop on Waldenstrom’s Macroglobulinemia in Prague. Five patients are still taking NX-5948, including two who have remained on the treatment for more than a year. Although the evaluable patients had a limited duration of follow-up, the data trend “further advances the impressive durability of response narrative building around NX-5948,” Stifel analysts said Monday. BTK inhibitors, such as BeiGene’s Brukinsa and AstraZeneca’s Calquence, made more than $10 billion in sales last year combined. Nurix said the data support its decision to advance NX-5948 into an ongoing Phase 1b expansion cohort in Waldenstrom’s patients who have had at least one prior line of therapy, and those with a rare form of the disease called Bing-Neel syndrome that affects the central nervous system. The Waldenstrom’s results come several months after the drug achieved a 69% objective response rate in 26 patients with relapsed or refractory chronic lymphocytic leukemia in the same study, including those who had become resistant to treatment with BTK inhibitors. NX-5948 is a small-molecule protein degrader, meaning it could have higher potency than BTK inhibitors, according to Nurix. Degraders could also be favorable due to their potentially “broad coverage of resistance mutations,” Jefferies analysts said.