Latest news with #EuropeanHematologyAssociation
Yahoo
07-08-2025
- Business
- Yahoo
Disc Medicine Reports Second Quarter 2025 Financial Results and Provides Business Update
Company on track to submit NDA for bitopertin in erythropoietic protoporphyria (EPP) under accelerated approval pathway in October 2025, supported by successful pre-NDA meeting Expect initial data from Phase 2 study of DISC-0974 in patients with anemia of myelofibrosis (MF) and multiple dose data from Phase 1b study of DISC-0974 in patients with anemia of non-dialysis-dependent chronic kidney disease (NDD-CKD) in Q4 2025 Initiated Phase 2 study of DISC-3405 in polycythemia vera (PV) with initial data expected in 2026 Presented positive clinical data updates across the portfolio at the European Hematology Association (EHA) Annual Congress, including longer term efficacy and safety data from the HELIOS trial of bitopertin in EPP, durability data from Phase 1b study of DISC-0974 in MF anemia, and healthy volunteer data from DISC-3405 studies Strong financial position ending Q2 with $650.0 million in cash, cash equivalents, and marketable securities; expected to fund operations into 2028 WATERTOWN, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today reported financial results for the second quarter ended June 30, 2025, and provided a recap of recent program and corporate developments. 'We are pleased with the continued momentum this past quarter, highlighted by clinical data presentations that support further advancement across our programs and continued progress toward our first NDA submission. Following positive feedback from our pre-NDA meeting, we are on track to submit an NDA for bitopertin in EPP under the accelerated approval pathway in October,' said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. 'We have also made great progress across the rest of our pipeline, initiating a Phase 2 study of DISC-3405 in polycythemia vera, and looking ahead to data from the Phase 2 trial of DISC-0974 in MF anemia and Phase 1b trial in NDD-CKD anemia in the second half of the year. Our team's commitment to execution, supported by a strong balance sheet that provides cash runway into 2028, has positioned Disc to prepare for the potential commercialization of bitopertin and advance pipeline development as we enter the next phase of growth.' Recent Highlights and Anticipated Milestones:Presented data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers Progressing confirmatory Phase 3 APOLLO clinical trial of bitopertin in adults and adolescents with EPP Completed positive pre-NDA meeting with FDA, confirming alignment with the agency on the expected timing, format, and content of planned NDA submission for bitopertin in EPP Expect to submit an NDA in October 2025 under the FDA's accelerated approval pathway based on Disc's existing data package Publication of the results from a preclinical study conducted in collaboration with Boston Children's Hospital showing that, in mice, bitopertin may help prevent liver disease in EPP, in addition to ameliorating blood PPIX levels. The paper, 'The GLYT1 inhibitor bitopertin mitigates erythroid PPIX production and liver disease in erythroid protoporphyria,' was published in the Journal of Clinical Investigation (corresponding authors Sarah Ducamp and Paul Schmidt)Hosted a virtual MF Anemia KOL event on May 9, 2025, discussing DISC-0974 and its potential to play a significant role in the treatment of anemia in patients with MF A replay of the webcast is available on the Events & Presentations page on the investor relations portion of the Company website Presented clinical data from the continuation phase of the Phase 1b trial of DISC-0974 in MF anemia demonstrating durable hematologic response at EHA 2025 Progressing RALLY-MF Phase 2 study of DISC-0974 in patients with anemia of MF with initial data expected in Q4 2025 Exploratory cohort for patients on concomitant momelotinib or pacritinib fully enrolled, and trial protocol updated to allow patients on these therapies into the main study cohorts Progressing Phase 1b study of DISC-0974 in patients with anemia of NDD-CKD with multiple-dose data expected in Q4 2025Presented updated SAD/MAD data from the Phase 1 trial of DISC-3405 in healthy volunteers providing proof of mechanism to support advancement of the program at EHA 2025 Initiated a Phase 2 study of DISC-3405 in patients with PV with initial data expected in 2026 Corporate: Appointed Nadim Ahmed, President and CEO of Cullinan Therapeutics, to the Company's Board of Directors in July, bringing to the Company over 25 years of development and commercial leadership experience including multiple product launches in the hematology space Second Quarter 2025 Financial Results: Cash Position: Cash, cash equivalents, and marketable securities were $650.0 million as of June 30, 2025, which are expected to fund operational plans into 2028. Research and Development Expenses: R&D expenses were $46.3 million for the three months ended June 30, 2025, as compared to $23.5 million for the three months ended June 30, 2024. The increase in R&D expenses was primarily driven by the progression of Disc's portfolio, including bitopertin's clinical studies and drug manufacturing, the advancement of the DISC-0974 program, and increased headcount, as well as a payment of a $10 million milestone upon initiation of the APOLLO study. Selling, General and Administrative Expenses: SG&A expenses were $15.1 million for the three months ended June 30, 2025, as compared to $7.4 million for the three months ended June 30, 2024. The increase in SG&A expenses was primarily due to increased headcount including establishing infrastructure to support potential commercialization. Net Loss: Net loss was $55.2 million for the three months ended June 30, 2025, as compared to $26.4 million for the three months ended June 30, 2024. The increase was primarily due to higher operating costs in the current period to support the continued advancement of our pipeline. About Disc Medicine Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit Available Information Disc announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts and the investor relations section of the Company website at in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD. Disc Cautionary Statement Regarding Forward-Looking Statements This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding: Disc's expectations with respect to the next stages of its development programs for bitopertin, DISC-0974 and DISC-3405, including projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; the registrational pathway for bitopertin, including the potential for accelerated approval and the projected timeline for an NDA submission; and the strength of its financial position and its anticipated cash runway. The use of words such as, but not limited to, 'believe,' 'expect,' 'estimate,' 'project,' 'intend,' 'future,' 'potential,' 'continue,' 'may,' 'might,' 'plan,' 'will,' 'should,' 'seek,' 'anticipate,' or 'could' or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc's current beliefs, expectations and assumptions regarding the future of Disc's business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc's capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc's product candidates; Disc's plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc's planned preclinical studies and clinical trials; the timing of the availability of data from Disc's clinical trials; Disc's ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc's preclinical studies and clinical trials and the risk that the results of Disc's preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc's filings with the SEC, including in the 'Risk Factors' section of Disc's Annual Report on Form 10-K for the year ended December 31, 2024, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. DISC MEDICINE, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In thousands, except share and per share amounts) (Unaudited) Three months ended June 30, Six months ended June 30, 2025 2024 2025 2024 Operating expenses: Research and development $ 46,319 $ 23,485 $ 74,082 $ 47,189 Selling, general and administrative 15,091 7,367 27,274 15,125 Total operating expenses 61,410 30,852 101,356 62,314 Loss from operations (61,410 ) (30,852 ) (101,356 ) (62,314 ) Other income (expense), net 6,215 4,560 12,195 9,078 Income tax expense (52 ) (60 ) (171 ) (65 ) Net loss $ (55,247 ) $ (26,352 ) $ (89,332 ) $ (53,301 ) Net loss per share, basic and diluted $ (1.58 ) $ (1.03 ) $ (2.61 ) $ (2.11 ) Weighted-average common shares outstanding, basic and diluted 35,024,592 25,649,043 34,179,364 25,229,456 DISC MEDICINE, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) June 30, December 31, 2025 2024 (Unaudited) Assets Cash, cash equivalents, and marketable securities $ 649,973 $ 489,881 Other current assets 11,619 3,734 Total current assets 661,592 493,615 Non-current assets 3,469 3,158 Total assets $ 665,061 $ 496,773 Liabilities and Stockholders' Equity Current liabilities $ 20,606 $ 23,316 Non-current liabilities 30,290 29,870 Total liabilities 50,896 53,186 Total stockholders' equity 614,165 443,587 Total liabilities and stockholders' equity $ 665,061 $ 496,773 Media Contact Peg RusconiDeerfield Investor Relations Contact Christina TartagliaPrecision AQ
Cision Canada
03-07-2025
- Business
- Cision Canada
Vertex Presents Longer-Term Data at the 2025 European Hematology Association (EHA) Congress Demonstrating Durability of CASGEVY® and Provides Update on Expanding Global Access to CASGEVY Français
- Data from longer-term follow-up of patients in ongoing clinical trials further demonstrate durability of the clinical benefits of CASGEVY ® - - Multiple reimbursement agreements secured, expanding access to CASGEVY to more patients around the world - TORONTO, July 3, 2025 /CNW/ - Vertex Pharmaceuticals (Nasdaq: VRTX) recently announced positive longer-term data for Pr CASGEVY ® (exagamglogene autotemcel) from global ongoing pivotal clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the European Hematology Association (EHA) Congress, continue to demonstrate the durable clinical benefits of CASGEVY. The longest follow up in SCD patients now extends more than 5.5 years and in TDT patients more than 6 years, with a mean of 39.4 months and 43.5 months, respectively. CASGEVY is the first authorized CRISPR/Cas9 gene-edited therapy. "This longer-term data reinforces CASGEVY's durable clinical benefits for eligible people living with sickle cell disease or transfusion-dependent beta thalassemia," said Kevin Kuo, M.D., Hematologist and Associate Professor in the Division of Hematology, University of Toronto, Clinician Investigator in the Red Blood Cell Disorders Clinic at University Health Network, and Principal Investigator for the CLIMB-131 clinical program. "These results are a reminder of what science can achieve, especially for patients and communities with significant unmet need." New longer-term follow-up data presented from the CASGEVY trials In SCD, 43/45 (95.6%) evaluable patients (those with at least 16 months of follow-up) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 84.9%, 99.5%). The mean duration of VOC-free was 35.0 months (range 14.4 to 66.2 months). All evaluable patients (45/45 [100%]) achieved freedom from in-patient hospitalization for severe VOCs for at least 12 consecutive months (HF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 92.1%, 100%), with a mean hospitalization-free of 36.1 months (range 14.5 to 66.2 months). In TDT, 54/55 (98.2%) evaluable patients (those with at least 16 months of follow-up) achieved transfusion-independence for at least 12 consecutive months with a weighted average hemoglobin (Hb) of at least 9 g/dL (TI12) in CLIMB-111 and CLIMB-131 combined (95% CI: 90.3%, 100%). The mean duration of transfusion independence was 40.5 months (range 13.6 to 70.8 months). The one evaluable patient who did not achieve TI12 has been transfusion free for 14.8 months. Iron removal therapy has been stopped for more than 6 months in 39/56 (69.6%) treated patients following infusion with CASGEVY, with sustained improvement in ferritin and liver iron content, suggesting that CASGEVY has the potential to correct ineffective erythropoiesis. Patients continue to demonstrate stable levels of fetal hemoglobin (HbF) and allelic editing. The safety profile of CASGEVY continues to be generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. Progress in bringing CASGEVY to patients Through reimbursement agreements, Vertex has secured access for eligible SCD or TDT patients in multiple countries including Austria, Bahrain, England, Denmark, the Kingdom of Saudi Arabia, Northern Ireland, Scotland, the United Arab Emirates, the United States and Wales. In Canada, CASGEVY received positive recommendations for reimbursement from both Canadian health technology agencies between December 2024 and January 2025; however, a Letter of Engagement from the pan-Canadian Pharmaceutical Alliance (pCPA) is pending. Vertex is continuing to work with government and reimbursement authorities globally to secure sustainable access for additional eligible patients. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or "sickled" red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person's life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. About Pr CASGEVY ® (exagamglogene autotemcel) Pr CASGEVY ® is an autologous genome edited hematopoietic stem cell-based therapy for eligible patients with SCD or TDT, in which a patient's own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CLIMB Trials The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD and recurrent VOCs. The trials are closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion in these trials. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients who received CASGEVY, including those in other CLIMB trials. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies in select regions for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Kevin Kuo, M.D., in this press release, and statements regarding expectations for the anticipated durable clinical benefits of CASGEVY, expectations for the safety profile of CASGEVY, expectations for the Letter of Engagement from the pCPA, plans to continue working with government and reimbursement authorities globally to secure sustainable access for patients, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that eligible patient access to CASGEVY may not be achieved on the anticipated timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. Vertex Pharmaceuticals Incorporated Media: [email protected] orCanada: +1 647-790-1600orU.S.: 617-341-6992 SOURCE Vertex Pharmaceuticals (Canada) Inc.
Yahoo
01-07-2025
- Automotive
- Yahoo
Autolus Therapeutics (AUTL) Presents Positive Long-Term Obe-cel Data at EHA 2025
Autolus Therapeutics plc (NASDAQ:AUTL) is one of the 10 best healthcare penny stocks to buy according to analysts. On June 12, the company presented long-term follow-up data from its FELIX study at the 2025 European Hematology Association (EHA) Congress. The presentation highlighted the potential of obecabtagene autoleucel (obe-cel; brand name Aucatzyl) for long-term remission in adult patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia (r/r B-ALL). A scientist in a lab coat holding a petri dish, the dish containing a 3D multicellular tissue. FELIX is a phase 1b/2, open-label, multicenter, single-arm study evaluating obe-cel in adults with r/r CD19-positive B-ALL. The study investigated obe-cel (formerly AUTO1), an autologous CD19 CAR T-cell therapy designed for rapid CAR T cell expansion and a favorable safety profile. According to the presented data, the estimated 3-year overall survival rate was 55.4%. The data also demonstrated sustained remission rates, with 40% of patients remaining in molecular remission at 36 months. Commenting on the development, Dr. Christian Itin, Autolus CEO, said: 'Obe-cel's durability of response without any subsequent therapy in two out of every five responders is a key factor leading the transformation of therapy for adult r/r B-ALL patients. At a median follow-up of 33 months, we are encouraged to see a continuation of the long-term plateau we observed at the last data cut.' Autolus Therapeutics plc (NASDAQ:AUTL) is a clinical-stage biopharmaceutical company. It develops programmed T cell therapies to treat cancer and autoimmune diseases. Its lead candidate is obe-cel, a CD19-targeting treatment for adult acute lymphoblastic leukemia (ALL). Other pipeline programs include AUTO1/22 (pediatric ALL), AUTO4 (T-cell lymphoma), AUTO6NG (neuroblastoma), and AUTO8 (multiple myeloma). While we acknowledge the potential of AUTL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: Goldman Sachs Energy Stocks: 10 Stocks to Buy and 10 Best AI Stocks to Buy According to Billionaire David Tepper. Disclosure: None. 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 Wire
15-06-2025
- Health
- Business Wire
Genmab Announces Epcoritamab Investigational Combination Therapy Demonstrates High Response Rates in Patients with Relapsed or Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) Eligible for Autologous Stem Cell Transplantation (ASCT)
COPENHAGEN, Denmark--(BUSINESS WIRE)-- Genmab A/S (Nasdaq: GMAB) today announced new results from the Phase 1b/2 EPCORE ® NHL-2 trial Arm 10 (NCT04663347), evaluating epcoritamab, a T-cell engaging bispecific antibody administered subcutaneously, in combination with rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) in adult patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) who are eligible for autologous stem cell transplantation (ASCT). Results demonstrated an overall response rate (ORR) of 87 percent, a complete response (CR) rate of 65 percent and a partial response (PR) of 23 percent. The majority of patients (65 percent) proceeded to ASCT. At six months, an estimated 81 percent of responses were ongoing, 74 percent of patients were progression free, and 100 percent of patients were alive. These results were shared today during an oral presentation at the 30 th European Hematology Association (EHA) 2025 Congress. The safety profile of this combination therapy showed cytokine release syndrome (CRS) being low grade and no discontinuations due to treatment-emergent adverse events (TEAEs). The most common TEAEs were neutropenia (74 percent), anemia (68 percent), and thrombocytopenia (68 percent). CRS occurred in 52 percent; all were low grade (1/2) and resolved. One patient had immune effector cell-associated neurotoxicity syndrome (ICANS; grade 1), which resolved. No clinical tumor lysis syndrome was observed. Infections occurred in 18 patients (58 percent); five (16 percent) had serious infections. There were no Grade 5 TEAEs. 'These results are particularly encouraging because many of the patients in this study had high-risk disease, having progressed rapidly after initial treatment,' said Raul Cordoba, MD, PhD, Head of the Lymphoma Unit at the Fundacion Jimenez Diaz University Hospital, Madrid, Spain. 'This combination therapy of epcoritamab plus rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) offers a potential new treatment option for patients with relapsed/refractory diffuse large B-cell lymphoma, providing high response rates and a bridge to potentially curative autologous stem cell transplantation." Among patients in the study who progressed within 12 months after first-line treatment (n=20), epcoritamab in combination with R-ICE demonstrated an 85 percent ORR and 55 percent CR. Patients in the study who progressed after 12 months from first-line therapy experienced a 91 percent ORR and 82 percent CR. Additionally, patients with one prior line of therapy experienced an 88 percent ORR and 68 percent CR, and patients who were treated with more than one prior line of therapy experienced an 83 percent ORR and 50 percent CR. 'The results from this trial highlight the potential of this investigational epcoritamab containing regimen, especially in patients who progress quickly after initial treatment, and reinforce our joint efforts with AbbVie to develop epcoritamab as a core therapy for B-cell lymphomas, especially as we develop epcoritamab in earlier lines of therapy and a broader patient population,' said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. 'Our comprehensive EPCORE clinical trial program is dedicated to advancing epcoritamab as both monotherapy and in combination to address the significant unmet need in relapsed/refractory diffuse large B-cell lymphoma and other hematologic malignancies." Use of epcoritamab + R-ICE in patients with R/R DLBCL eligible for ASCT is not approved and the safety and efficacy of epcoritamab for use as a combination therapy in DLBCL have not been established. About Diffuse Large B-Cell Lymphoma DLBCL is the most common type of non-Hodgkin's lymphoma (NHL) worldwide, accounting for approximately 25-30 percent of all NHL cases. In the U.S., there are approximately 25,000 new cases of DLBCL diagnosed each year. DLBCL can arise in lymph nodes as well as in organs outside of the lymphatic system, occurs more commonly in the elderly and is slightly more prevalent in men. DLBCL is a fast-growing type of NHL, a cancer that develops in the lymphatic system and affects B-cell lymphocytes, a type of white blood cell. For many people living with DLBCL, their cancer either relapses, which means it may return after treatment, or becomes refractory, meaning it does not respond to treatment. Although new therapies have become available, treatment management can remain a challenge. About the EPCORE ® NHL-2 Trial EPCORE NHL-2 is a Phase 1b/2 open-label interventional trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics/biomarkers, immunogenicity, and preliminary efficacy of epcoritamab as a monotherapy and in combination with other standard of care agents in patients with B-cell non-Hodgkin's lymphoma (B-NHL). The trial consists of two parts: Part 1 (Dose Escalation) and Part 2 (Dose Expansion). The primary objective of Part 1 is safety, and the primary goal of Part 2 is preliminary efficacy. The primary endpoint was overall response rate (ORR) based on best overall response per Lugano criteria. MRD negativity was assessed as a secondary endpoint. Arm 10 of the EPCORE NHL-2 study enrolled 31 patients with R/R DLBCL, who were eligible for R-ICE and ASCT, and had received ≥1 prior line of treatment. At the time of data cutoff (December 18, 2024), median follow-up was 11 months (range, 6−15). Among the 31 patients treated with epcoritamab 48 mg + R-ICE, 61 percent were Ann Arbor stage III/IV, 42 percent had bulky disease ≥7 cm, 81 percent had one prior LOT (range, 1−3), and 65 percent had progressed within 12 months of first-line treatment. More information on this trial can be found at (NCT: 04663347). About Epcoritamab Epcoritamab is an IgG1-bispecific antibody created using Genmab's proprietary DuoBody ® technology and administered subcutaneously. Genmab's DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells. i Epcoritamab (approved under the brand name EPKINLY ® in the U.S. and Japan, and TEPKINLY ® in the EU) has received regulatory approval in certain lymphoma indications in several territories. Where approved, epcoritamab is available as a readily accessible therapy without the need for reducing tumor burden ('debulking'). Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies' oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication. Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes five ongoing Phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) in patients with R/R FL (NCT05409066), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with R2 compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit for more information. EPKINLY ® (epcoritamab-bysp) U.S. INDICATIONS & IMPORTANT SAFETY INFORMATION What is EPKINLY? EPKINLY is a prescription medicine used to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, or follicular lymphoma (FL) that has come back or that did not respond to previous treatment after receiving 2 or more treatments. EPKINLY is approved based on patient response data. Studies are ongoing to confirm the clinical benefit of EPKINLY. It is not known if EPKINLY is safe and effective in children. Important Warnings—EPKINLY can cause serious side effects, including: Cytokine release syndrome (CRS), which is common during treatment with EPKINLY and can be serious or life-threatening. To help reduce your risk of CRS, you will receive EPKINLY on a step-up dosing schedule (when you receive 2 or 3 smaller step-up doses of EPKINLY before your first full dose during your first cycle of treatment), and you may also receive other medicines before and for 3 days after receiving EPKINLY. If your dose of EPKINLY is delayed for any reason, you may need to repeat the step-up dosing schedule. Neurologic problems that can be life-threatening and lead to death. Neurologic problems may happen days or weeks after you receive EPKINLY. People with DLBCL or high-grade B-cell lymphoma should be hospitalized for 24 hours after receiving their first full dose of EPKINLY on day 15 of cycle 1 due to the risk of CRS and neurologic problems. Tell your healthcare provider or get medical help right away if you develop a fever of 100.4°F (38°C) or higher; dizziness or lightheadedness; trouble breathing; chills; fast heartbeat; feeling anxious; headache; confusion; shaking (tremors); problems with balance and movement, such as trouble walking; trouble speaking or writing; confusion and disorientation; drowsiness, tiredness or lack of energy; muscle weakness; seizures; or memory loss. These may be symptoms of CRS or neurologic problems. If you have any symptoms that impair consciousness, do not drive or use heavy machinery or do other dangerous activities until your symptoms go away. EPKINLY can cause other serious side effects, including: Infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment and treat you as needed if you develop an infection. You should receive medicines from your healthcare provider before you start treatment to help prevent infection. Tell your healthcare provider right away if you develop any symptoms of infection during treatment, including fever of 100.4°F (38°C) or higher, cough, chest pain, tiredness, shortness of breath, painful rash, sore throat, pain during urination, or feeling weak or generally unwell. Low blood cell counts, which can be serious or severe. Your healthcare provider will check your blood cell counts during treatment. EPKINLY may cause low blood cell counts, including low white blood cells (neutropenia), which can increase your risk for infection; low red blood cells (anemia), which can cause tiredness and shortness of breath; and low platelets (thrombocytopenia), which can cause bruising or bleeding problems. Your healthcare provider will monitor you for symptoms of CRS, neurologic problems, infections, and low blood cell counts during treatment with EPKINLY. Your healthcare provider may temporarily stop or completely stop treatment with EPKINLY if you develop certain side effects. Before you receive EPKINLY, tell your healthcare provider about all your medical conditions, including if you have an infection, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. If you receive EPKINLY while pregnant, it may harm your unborn baby. If you are a female who can become pregnant, your healthcare provider should do a pregnancy test before you start treatment with EPKINLY and you should use effective birth control (contraception) during treatment and for 4 months after your last dose of EPKINLY. Tell your healthcare provider if you become pregnant or think that you may be pregnant during treatment with EPKINLY. Do not breastfeed during treatment with EPKINLY and for 4 months after your last dose of EPKINLY. In DLBCL or high-grade B-cell lymphoma, the most common side effects of EPKINLY include CRS, tiredness, muscle and bone pain, injection site reactions, fever, stomach-area (abdominal) pain, nausea, and diarrhea. The most common severe abnormal laboratory test results include decreased white blood cells, decreased red blood cells, and decreased platelets. In follicular lymphoma the most common side effects of EPKINLY include injection site reactions, CRS, COVID-19, tiredness, upper respiratory tract infections, muscle and bone pain, rash, diarrhea, fever, cough, and headache. The most common severe abnormal laboratory test results include decreased white blood cells and decreased red blood cells. These are not all of the possible side effects of EPKINLY. Call your doctor for medical advice about side effects. You are encouraged to report side effects to the FDA at (800) FDA-1088 or or to Genmab US, Inc. at 1-855-4GENMAB (1-855-443-6622). Please see Full Prescribing Information and Medication Guide, including Important Warnings. Globally, prescribing information varies; refer to the individual country product label for complete information. About Genmab Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab's vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO ®) antibody medicines. Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit and follow us on LinkedIn and X. This Media Release contains forward looking statements. The words 'believe,' 'expect,' 'anticipate,' 'intend' and 'plan' and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with preclinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmab's most recent financial reports, which are available on and the risk factors included in Genmab's most recent Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at Genmab does not undertake any obligation to update or revise forward looking statements in this Media Release nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law. Genmab A/S and/or its subsidiaries own the following trademarks: Genmab ®; the Y-shaped Genmab logo ®; Genmab in combination with the Y-shaped Genmab logo ®; HuMax ®; DuoBody ®; HexaBody ®; DuoHexaBody ®, HexElect ® and KYSO™. EPCORE ®, EPKINLY ®, TEPKINLY ® and their designs are trademarks of AbbVie Biotechnology Ltd. i Engelberts PJ, et al. DuoBody-CD3xCD20 Induces Potent T-Cell-Mediated Killing of Malignant B Cells in Preclinical Models and Provides Opportunities for Subcutaneous Dosing. EBioMedicine
Yahoo
13-06-2025
- Health
- Yahoo
Novartis reports positive results from Phase IIIB study of iptacopan for PNH
Novartis has reported positive outcomes from the multi-centre Phase IIIB APPULSE-PNH study of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal haemoglobinuria (PNH). These findings are set to be presented at the European Hematology Association (EHA) Congress in 2025. The single-arm, open-label, multinational trial assessed the oral therapy in PNH subjects with haemoglobin (Hb) levels of ≥10g/dL, who transitioned from anti-C5 therapies (eculizumab or ravulizumab). It enrolled 52 subjects and aimed to expand the clinical evidence for the therapy by including patients with higher baseline Hb levels than those in the pivotal Phase III programme. Following 24 weeks of treatment with the therapy, subjects experienced an average increase in Hb levels of 2.01g/dL, with a significant number achieving normal or near-normal levels. During the trial, none of the participants needed blood transfusions, and 92.7% of them reached Hb levels of at least 12g/dL. In addition, subjects reported improvements in fatigue, with Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores aligning with those of the general population by day 168. Novartis noted that Fabhalta-treated subjects also maintained intravascular haemolysis control and resolved extravascular haemolysis control, indicated by lactate dehydrogenase levels remaining below 1.5 times the upper limit of normal and a decrease in absolute reticulocyte count. Along with these findings, the company will also present longer-term data from the APPLY-PNH and APPOINT-PNH Phase III trials at the EHA Congress. Novartis development president and chief medical officer Shreeram Aradhye said: 'New data from APPULSE-PNH, combined with findings from the Phase III roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforce the efficacy and safety profile of Fabhalta in delivering real benefits to patients.' Fabhalta was discovered by Novartis and acts as a Factor B inhibitor of the alternative complement pathway. It also offers a new treatment option for the rare and life-threatening complement-mediated blood disorder, PNH. The company recently shared topline outcomes from the Phase III PSMAddition trial's pre-specified interim analysis, in which Pluvicto plus standard of care (SoC) treatment demonstrated a benefit in treating prostate-specific membrane antigen (PSMA)-positive metastatic hormone-sensitive prostate cancer (mHSPC). "Novartis reports positive results from Phase IIIB study of iptacopan for PNH" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
