Latest news with #BPDCN
Medscape
3 days ago
- Business
- Medscape
PVEK: New Standard of Care in Rare, Deadly Blood Cancer?
CHICAGO — The antibody-drug conjugate (ADC) pivekimab sunirine (PVEK) yielded high response rates with good durability in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) in the phase 1/2 CADENZA trial. 'We conclude that PVEK represents a potentially practice-changing paradigm for patients with the rare disease BPDCN,' said study author Naveen Pemmaraju, MD, of MD Anderson Cancer Center in Houston, Texas, during an oral abstract session at the American Society of Clinical Oncology (ASCO) 2025 annual meeting. 'One half of first-line patients treated with PVEK subsequently received stem cell transplant. PVEK treatment had a manageable safety profile, and infusion-related reactions and peripheral edema were resolved with supportive care. No capillary-leak syndrome was observed.' CADENZA enrolled 84 adult patients with CD123-positive BPDCN. Of these patients, 33 were newly diagnosed, and 51 patients had relapsed or refractory (R/R) BPDCN with one, two, or three prior lines of therapy. In the first-line group, 22 patients had de novo disease and 11 had a prior or concomitant hematologic malignancy (PCHM). CADENZA's primary endpoint was the composite rate of complete response (CR) plus CR with minimal skin abnormality (CRc) in the first-line de novo patients. The authors found that 70% of patients achieved the composite CR, while 14% of the patients with R/R disease did. Regarding the overall response rate (ORR), 85% of first-line patients responded to PVEK, while 35% of patients with R/R disease responded. Responses among patients with R/R disease were early and durable (median, 1.4 months and 9.2 months, respectively). PCHM status did not affect overall survival (OS) in first-line patients: Median OS was 16.6 months in both the de novo and PCHM subgroups. The discussant for this study, Raajit Rampal, MD, PhD, of Memorial Sloan Kettering Cancer Center in New York City, told Medscape Medical News that the CADENZA trial supports the use of CD123 as a therapeutic target in BPDCN. He also noted that the current standard of care often causes potentially life-threatening capillary leak syndrome (CLS): 'There were no cases of CLS in this dataset, which means we may have an opportunity to deliver anti-CD123 therapies, with no CLS as a potential side effect.' Rampal also cited PVEK's apparent ability to enable many patients to bridge to stem cell transplant, 'the fundamental prospect in terms of long-term survival provisions with BPDCN.' Following this presentation, Pemmaraju sat down with Medscape Medical News . The following interview has been edited for clarity. Please give us some context on BPDCN and why it is difficult to treat. BPDCN is a rare, aggressive hematologic malignancy primarily involving skin, bone marrow, lymph nodes and the central nervous system. We think there are only 500 BPDCN diagnoses per year in the United States. Current treatment regimens are limited to chemotherapy regimens adapted from AML, ALL, lymphoma, and myeloma. The median age at diagnosis is around 65 years, so many patients are not eligible for intensive chemo or stem cell transplant. CD123 is overexpressed on all BPDCN blasts, making it an ideal target for immunochemotherapy. What is the top takeaway from your presentation? BPDCN has an overall poor prognosis with significant unmet need. Currently, tagraxofusp-erzs is the standard of care and the only approved treatment for BPDCN. PVEK is a new CD123-targeting agent. This novel monotherapy, given once every 3 weeks, had high response rates for frontline patients with BPDCN and was highly durable. Even for patients with R/R disease, whose prognosis is only a few months, PVEK showed some promising activity both in response and durability. Patients with BPDCN are in need of improved frontline therapies to treat their disease, so we're very excited to see this phase 1/2 trial going extremely well in terms of safety and efficacy. The responses we have observed make PVEK a strong candidate as a standard-of-care treatment. What is another important thing you want your colleagues to know about this study? The CADENZA trial enrolled 84 patients, which is a huge number in a rare disease field like ours. I'm proud we were able to achieve a real-world demographic split. That means we had older patients, just like I see in the clinic, and half the patients had skin involvement along with bone marrow. In the relapsed group, the prior therapy had been quite intense. Half of these patients had received chemo, half had received tagraxofusp, and a third had received a transplant. This may be the best study I've ever done in terms of enrolling patients who are truly representative with all the comorbidities, and all the difficulties, you see in clinical practice. You mentioned that PVEK is more patient-friendly than the standard of care. Why is that? PVEK is given once every 3 weeks via an infusion that takes only 30 minutes or less. Many of our other drugs, of course, require a much more intensive schedule that can be difficult for patients, especially when they must be given on an inpatient basis. PVEK can be given outpatient even from the beginning, which is much more convenient for patients. You referred several times to patients' skin lesions. Is that part of the BPDCN disease process or is that a treatment reaction? I want to emphasize this is a BPDCN trait not related to treatment. BPDCN has a predilection for the skin. The vast majority of patients with BPDCN, anywhere from 70% to 90%, will present with skin findings. It's very common for my patients to present with 'just' a skin lesion that turns out to be anything but. Unlike other skin-containing malignancies, BPDCN is a bone marrow blood cancer that can go to an acute leukemia and become acutely life threatening. Let's differentiate that from the cutaneous toxicity very commonly seen in chemo-based regimens. What's ahead in PVEK research? We will keep presenting our data, including at the European Hematological Association and as an ASCO Encore presentation. We hope to submit PVEK to regulatory agencies in the near future. Ultimately, can we combine PVEK with other agents active in the AML and BPDCN space to create a next-generation therapy? Eventually, once we have two active agents for BPDCN, we will need to determine the sequencing of these drugs. What's the natural history of these treated patients? Are they living longer? I think answers to all those questions will come out in the coming year or two. The study was funded by AbbVie. Pemmaraju disclosed having relationships with AbbVie, Aplastic Anemia and MDS International Foundation, Aptitude Health, Astellas Pharma, Blueprint Medicines, Bristol Myers Squibb, CancerNet, CareDx, Celgene, Cimeio Therapeutics, ClearView Healthcare Partners, CTI BioPharma Corp, Curio Science, Dava Oncology, EUSA Pharma, Harborside Press, Imedex, ImmunoGen, Intellisphere, Magdalen Medical Publishing, Medscape, Menarini Group, Neopharm, Novartis, OncLive, Pacylex, Patient Power, Peerview, PharmaEssentia, and Physicians' Education Resource. Rampal reported having ties with AbbVie, Blueprint Medicines, Bristol Myers Squibb/Celgene, Constellation Pharmaceuticals, CTI BioPharma Corp, Disc Medicine, Galecto, Incyte, Karyopharm Therapeutics, Novartis, Pharmaessentia, Promedior, SERVIER, Sierra Oncology, Stemline Therapeutics, Sumitomo Dainippon, and Zentalis.
Globe and Mail
15-04-2025
- Health
- Globe and Mail
BPDCN Market Set for Robust Growth Through 2032 Driven by Targeted Therapies and Rising Disease Awareness
The key BPDCN companies in the market include - AbbVie, ImmunoGen, Mustang Bio, Genentech, Stemline Therapeutics, Jazz Pharmaceuticals, Menarini Group, Cellex Patient Treatment GmbH, and Xencor, are actively engaged in the BPDCN market. The Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) market across the 7MM is poised for significant growth through 2032. This growth is driven by advancements in targeted therapies, increased awareness of the disease among healthcare professionals, and ongoing research and development efforts. Major pharmaceutical and biotech companies, including AbbVie, ImmunoGen, Mustang Bio, Genentech, Stemline Therapeutics, Jazz Pharmaceuticals, Menarini Group, Cellex Patient Treatment GmbH, and Xencor, are actively engaged in the BPDCN market. A recent report titled, ' Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) - Market Insight, Epidemiology And Market Forecast - 2032 ' by DelveInsight, provides a comprehensive analysis of BPDCN epidemiology, treatment landscape, and market trends across the 7MM, which includes the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan. The US leads the market with the largest annual patient population diagnosed with BPDCN, primarily due to increased awareness campaigns and improved diagnostic tools that enhance early detection rates. BPDCN is a rare and aggressive hematologic cancer that originates from plasmacytoid dendritic cells. It accounts for less than 1% of all hematologic malignancies and is characterized by rapid progression and a poor prognosis. The disease primarily affects older adults, with a median age of diagnosis around 66 years. Additionally, BPDCN has a higher prevalence among males, and individuals of Caucasian descent are slightly more affected than those from other demographics. Historically, BPDCN treatment relied on chemotherapy regimens adapted from leukemia or lymphoma protocols. However, recent breakthroughs have transformed the treatment paradigm. ELZONRIS (Stemline Therapeutics/Menarini Group), a targeted cytotoxin, was approved by the FDA in December 2018 as the first and only targeted therapy for BPDCN. Although treatment options for BPDCN are currently limited, several promising experimental therapies are under investigation. Several anti-CD123 CAR-T therapies are emerging as potential breakthroughs, utilizing genetically engineered T cells to target CD123, owing to the success of ELZONRIS. Dive deeper into the evolving BPDCN treatment landscape and uncover key innovations from targeted cytotoxins to next-gen CD123 CAR-T therapies transforming patient outcomes Combination therapies are also gaining traction, such as CD123-targeted agents combined with hypomethylating drugs like azacitidine and BCL-2 inhibitors like venetoclax, which have shown the potential to enhance antitumor responses. Furthermore, advancements in allogeneic hematopoietic stem cell transplantation (allo-HCT) are refining patient selection criteria and conditioning regimens to improve outcomes for patients who achieve remission. In March 2025, Menarini Group Announces Collaboration with VisualDx to Aid in Identifying People Who May Have BPDCN. Stay ahead with the latest breakthroughs, FDA approvals, and eco-friendly innovations shaping the future of the BPDCN treatment landscape. Visit BPDCN Recent Developments Looking ahead, the BPDCN market in the 7MM is projected to experience significant growth over the next decade as stakeholders address unmet needs through innovation and collaboration. With ongoing advancements in targeted therapies and increasing awareness among clinicians and patients alike, the market landscape for this rare malignancy is expected to expand. Table of Contents 1. Key Insights 2. Executive Summary of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) 3. Competitive Intelligence Analysis for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) 4. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Market Overview at a Glance 5. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Disease Background and Overview 6. Patient Journey 7. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Epidemiology and Patient Population 8. Treatment Algorithm, Current Treatment, and Medical Practices 9. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Unmet Needs 10. Key Endpoints of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Treatment 11. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Marketed Products 12. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Emerging Therapies 13. Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Seven Major Market Analysis 14. Attribute analysis 15. 7MM: Market Outlook 16. Access and Reimbursement Overview of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) 17. KOL Views 18. BPDCN Market Drivers 19. BPDCN Market Barriers 20. Appendix 21. DelveInsight Capabilities 22. Disclaimer 23. About DelveInsight Related Reports Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Malignancy Pipeline Insight Blastic Plasmacytoid Dendritic Cell Neoplasm Pipeline Insight provides comprehensive insights about the BPDCN pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the BPDCN manufacturers, including Genentech/AbbVie, Sanofi (NASDAQ: SAN), and ImmunoGen (NASDAQ: IMGN), among others. About DelveInsight DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Arpit Anand Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
Yahoo
20-03-2025
- Health
- Yahoo
Menarini Group Announces Collaboration with VisualDx to Aid in Identifying People Who May Have Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
BPDCN is an aggressive hematologic malignancy with a historically poor prognosis VisualDx is a clinical decision support system used by more than 2,300 hospitals, clinics, and medical schools around the world VisualDx's system seeks to enhance identification of people who may have BPDCN, leveraging actual images of BPDCN skin lesions and artificial intelligence/machine learning (AI/ML) technologies incorporated into the VisualDx platform FLORENCE, Italy and NEW YORK, March 20, 2025 /CNW/ -- The Menarini Group ("Menarini"), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. ("Stemline"), a wholly-owned subsidiary of the Menarini Group focused on bringing transformational oncology treatments to cancer patients, today announced that they are collaborating with VisualDx to enhance identification of people who may have BPDCN. This is an example of innovative companies working together to bring artificial intelligence/machine learning (AI/ML) tools to help identify BPDCN as a possible early differential diagnosis. The project includes leveraging actual images of BPDCN skin lesions and AI/ML technologies incorporated into the VisualDx platform. The AI model is now live within VisualDx. VisualDx is a physician-led company committed to improving medical decision-making, medical education and research. The VisualDx tool is a clinical decision support system used by more than 2,300 hospitals, clinics, and medical schools around the world. The software includes a comprehensive database of clinical images, submitted through partnerships with learning institutions and others, and vetted by clinicians, to assist healthcare professionals to identify skin concerns across all skin types. Through the AI image search, clinicians can better understand different skin conditions with the goal of supporting patients to get more accurate diagnoses throughout their care journeys. BPDCN is an aggressive orphan hematologic malignancy with a historically poor prognosis (approximately 8.7 to 14 months post diagnosis[1]) that typically presents skin lesions and can also involve the bone marrow, blood, central nervous system, lymph nodes and viscera. Dermatologists may be the first to recognize the signs of BPDCN and biopsy suspicious lesions. Pathologists can then test for BPDCN by testing for certain biomarkers which are highly expressed on BPDCN cells. Tagraxofusp-erzs is the only approved treatment for patients with BPDCN, and the first and only approved CD123-targeted therapy, in the United States, Europe and other global regions. "BPDCN is a rare and clinically aggressive hematologic malignancy, often presenting with cutaneous lesions. Due to its aggressive nature and the immature cell involvement, the prognosis can be poor if not treated promptly," said Marina Konopleva, MD, Phd, Professor, Molecular Pharmacology, Director, Leukemia Program and Co-Director, Blood Cancer Institute, at Montefiore Einstein. "Early diagnosis plays a critical role in improving patient outcomes, and emerging AI/ML technologies may offer valuable support in the differential diagnosis and early identification of BPDCN." "BPDCN often first presents as a skin lesion and usually has a poor prognosis. There is a dire need for early diagnosis so that patients may access appropriate treatment options," said Elcin Barker Ergun, CEO of the Menarini Group. "We are delighted to collaborate with VisualDx to provide healthcare teams with a tool using the latest artificial intelligence/machine learning (AI/ML) technology to help interpret challenging skin lesions." VisualDx does not store any images uploaded by clinicians taking a picture of their patients' skin exams. This helps maintain the patient's privacy. About BPDCNBPDCN, formerly blastic NK-cell lymphoma, is an aggressive, orphan hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN typically presents skin lesions and may also involve bone marrow, blood, central nervous system, lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. The World Health Organization (WHO) termed this disease "BPDCN" in 2008; previous names included blastic NK cell lymphoma and CD4+/CD56+ hematodermic neoplasm. About ELZONRIS® (Tagraxofusp-erzs)U.S. Indication: ELZONRIS is a prescription medicine used to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients 2 years and older. Full prescribing information for the U.S. can be found at IMPORTANT SAFETY INFORMATION, ELZONRIS®Boxed WARNING: CAPILLARY LEAK SYNDROME Capillary Leak Syndrome (CLS) which may be life-threatening or fatal, can occur in patients receiving ELZONRIS. Monitor for signs and symptoms of CLS and take actions as recommended. Warnings and Precautions Capillary Leak SyndromeCapillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range - 1 to 46 days), and all but 5 patients experienced an event in Cycle 1. Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability. Hypersensitivity ReactionsELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur. HepatotoxicityTreatment with ELZONRIS was associated with elevations in liver enzymes. In patients receiving ELZONRIS in clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption. Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Withhold ELZONRIS temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved. Adverse ReactionsMost common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST. Please see full Prescribing Information, including Boxed report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or About The Menarini GroupThe Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of over $4.7 billion and over 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for cardiology, oncology, pneumology, gastroenterology, infectious diseases, diabetology, inflammation, and analgesia. With 18 production sites and 9 Research and Development centers, Menarini's products are available in 140 countries worldwide. For further information, please visit About Stemline Therapeutics Therapeutics, Inc. ("Stemline"), a wholly-owned subsidiary of the Menarini Group, is a commercial-stage biopharmaceutical company focused on bringing transformational oncology treatments to patients. Stemline commercializes elacestrant, an oral endocrine therapy indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy, in the U.S., Europe, and other global regions. Stemline also commercializes tagraxofusp-erzs, a novel targeted therapy directed to CD123, for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic cancer, in the United States, Europe, and other global regions. In addition, Stemline commercializes selinexor, an XPO1 inhibitor for multiple myeloma, in Europe. The company is also conducting multiple label-expansion studies with elacestrant and tagraxofusp in breast and hematologic cancer indications, respectively, and has an extensive clinical pipeline of additional drug candidates in various stages of development for a host of solid and hematologic cancers. About VisualDxVisualDx is a company dedicated to improving medical decisions through augmented thinking and timely visualization. It is committed to reducing disparities in medicine and believes technology can bridge gaps in knowledge to bring about more equitable care. VisualDx has become the standard professional resource at more than 2,300 hospitals, clinics and medical schools worldwide by combining problem oriented clinical search with the world's best curated medical image library, plus medical knowledge from experts and sophisticated machine learning algorithms to help with differential diagnosis, variation, treatment, and patient communication. Learn more at [1] Pagano L, et al Haematological. 2013;98 (2): 239-246 and Pemmaraju N Curr Hematol Malig Rep. 2017; 12(6): 510-512 Logo - View original content to download multimedia: SOURCE Menarini Industrie Farmaceutiche Riunite View original content to download multimedia:
