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ROCKVILLE, Md. and SUZHOU, China, Dec. 10, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing, and commercializing therapies to address global unmet medical needs primarily for malignancies, today announced that Prof. Weiming Li, of Wuhan Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, has presented the latest clinical data of its novel drug candidate, olverembatinib (HQP1351), as a second-line therapy in patients with chronic-phase chronic myeloid leukemia (CP-CML), in an Oral Report at the 66th American Society of Hematology (ASH) Annual Meeting, taking place in San Diego, CA, the United States. The ASH Annual Meeting is one of the largest gatherings of the international hematology community, bringing together the most cutting-edge scientific research and latest data of investigational therapies that represent leading scientific and clinical advances in the global hematology field. Garnering growing interest from the global research community, results from multiple clinical and preclinical studies on four of Ascentage Pharma's drug candidates (olverembatinib, lisaftoclax, APG-2449, and APG-5918) have been selected for presentations, including two Oral Reports, at this year's ASH Annual Meeting. Furthermore, this is the seventh consecutive year for studies of olverembatinib to be selected for Oral Reports at the meeting. Results released through the Oral Report are the first batch of data of olverembatinib as a second-line therapy in patients with non-T315I-mutant CP-CML, which indicated that the drug may offer a safe and effective second-line therapy to patients with CP-CML, especially those who had failed on second-generation TKIs in the first-line setting. These data showed that, in patients with CP-CML who were resistant/intolerant to one prior line of TKIs, olverembatinib demonstrated a complete cytogenetic response (CCyR) rate of 74.1%, a major molecular response (MMR) rate of 40.6%; and in patients who had been treated with second-generation TKIs in the first-line setting, olverembatinib demonstrated a CCyR rate of 78.9% and an MMR rate of 43.5%, with efficacy improved over time. In terms of safety, olverembatinib showed a profile similar to that was previously reported. The study did not observe any new safety signals or report any arterial occlusive events (AOEs) or venous thromboembolisms (VTEs). As the first approved third-generation BCR-ABL inhibitor in China, olverembatinib has already been approved for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant CP-CML or accelerated-phase (AP-) CML harboring the T315I mutation; and adult patients with CP-CML resistant to and/or intolerant of first- and second-generation TKIs. Olverembatinib is being jointly commercialized in China by Ascentage Pharma and Innovent Biologics. Prof. Weiming Li, the principal investigator of the study, commented, "Among patients with CP-CML, it is a common situation in which patients need to switch to another therapy after failing the first-line treatment with TKIs, and the efficacy of first- and second-generation TKIs often falls short of the desired outcome. Therefore, patients have the desperate need for more second-line therapies that can offer higher efficacy. This is what propelled us to design such a study to evaluate the efficacy and safety of olverembatinib as a second-line therapy in patients with non-T315I-mutant CP-CML. At the meeting, we released the first dataset of olverembatinib as a second-line therapy for patients with non-T315I-mutant CP-CML. These data showed that, olverembatinib as a second-line treatment offers promising responses and potential clinical benefits, as well as favorable safety and tolerability, especially in patients who had received second-generation TKIs in the first-line setting, thus suggesting that olverembatinib can potentially provide a new strategy for the second-line treatment of patients with CML. In the future, we hope to release more clinical data that can support the adoption of olverembatinib for the second-line treatment and guideline inclusions. We will strive to bring a better new treatment option to patients and their physicians." Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, said, "When the very first batch of clinical data of olverembatinib came out in 2018, it was selected for Oral Report at the ASH Annual Meeting. Since then, clinical studies of the drug have been featured in Oral Reports at the meeting for seven consecutive years. For studies of a particular drug (olverembatinib) in a certain indication (CML) to be selected for Oral Reports for seven years in a row, it reflects the strong interest from the international hematology community. The data reported through the Oral Report this year reaffirmed the strong potential of olverembatinib in CML. Fulfilling our mission of addressing unmet clinical needs in China and around the world, we will expedite the global clinical development of our key drug candidates such as olverembatinib to bring more safe and effective therapies to patients as soon as possible." Highlights of the data this study reported at ASH 2024 are as below: Olverembatinib as Second-Line (2L) Therapy in Patients (pts) with Chronic Phase-Chronic Myeloid Leukemia (CP-CML)Format: Oral PresentationAbstract#: 480Session: 632. Chronic Myeloid Leukemia: Novel Molecules in Clinical Practice Highlights: Background: Olverembatinib, a third-generation tyrosine kinase inhibitor (TKI), has demonstrated remarkable efficacy and a favorable safety profile in patients with CML resistant and/or intolerant to at least 2 TKIs or with the T315I mutation. The aim of this study was to assess the efficacy and safety of olverembatinib as a second-line treatment for patients with CP-CML without the T315I mutation. Introduction: This is a single-arm, multicenter, open-label study designed to evaluate the efficacy, safety, and patients' quality of life of orally administered olverembatinib (40 mg QOD) in patients with CP-CML who were resistant/intolerant to one prior line of TKIs (including imatinib, flumatinib, nilotinib, and dasatinib) without the T315I mutation. Enrolled Patients and Study Methods: As of November 15, 2024, the study enrolled a total of 43 patients with non–T315I-mutant CML-CP. These patients received orally administered olverembatinib 40 mg every other day (QOD) in 28-day cycles. Efficacy Results: As of November 15, 2024, 33 (78.6%) patients had at least one efficacy assessment, 28 (66.7%) had at least two, 23 (54.8%) had at least three. Three patients had not yet undergone their first efficacy assessment. At data cutoff, 74.1% (20/27) of patients achieved a complete cytogenetic response (CCyR) and 40.6% (13/32) achieved a major molecular response (MMR). The CCyR and MMR rates evaluated at the end of Cycles 6, 9, 12, and 18 were 45.5% and 28.7%, 57.2% and 32.0%, 61.1% and 32.0%, and 68.9% and 40.7%, respectively, suggesting that efficacy improved over time. In 33 efficacy-evaluable patients, 24 were pretreated with second-generation TKIs as first-line treatment, of whom 78.9% (15/19) achieved CCyR, and 43.5% (10/23) achieved MMR. In 9 patients who were pretreated with imatinib, 50.0% achieved CCyR (4/8) and 33.3% achieved MMR (3/9). Safety Results: The median (range) treatment duration was 16.0 (2-18) months. A total of 41 (95.3%) patients experienced any-grade treatment-related adverse events (TRAEs), of whom 21 (48.8%) had grade ≥ 3 treatment-related adverse events (TRAEs) and 6 (14.0%) had olverembatinib-related serious AEs (SAEs). Nonhematologic TRAEs included skin hyperpigmentation (51.2%), hyperuricemia (30.2%), and creatine phosphokinase increased (25.6%). Most of these TRAEs were grade 1 or 2. Grade ≥ 3 hematologic toxicities included platelet count decreased (46.5%), neutropenia (25.6%), and anemia (9.3%). Only one patient experience grade 1 hypertension that was possibly olverembatinib-related, and no AOEs or VTEs were reported during the study. Olverembatinib-related SAEs included platelet count decreased (7.0%), anemia and elevated ALT (2.3% each). No deaths were reported. Conclusions: Olverembatinib may provide an effective and safe second-line treatment option for patients with CP-CML, especially those who have failed on second-generation TKIs in the first-line setting. * Olverembatinib is an investigational drug that has not been approved for any indication outside China. About Ascentage Pharma Ascentage Pharma (6855.HK) is a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily in malignancies. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK. The company has built a rich pipeline of innovative drug candidates that includes novel, highly potent Bcl-2 and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation TKIs. Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company has conducted more than 40 clinical trials in the US, Australia, Europe, and China, including 13 registrational studies (completed/ ongoing/planned). Olverembatinib, the company's first lead asset developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company's first approved product in China, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). To date, the drug had been included into the China National Reimbursement Drug List (NRDL). Furthermore, olverembatinib has been granted Orphan Drug Designations (ODDs) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU. To date, Ascentage Pharma has obtained a total of 16 ODDs from the US FDA and 1 Orphan Designation from the EMA of the EU for 4 of the company's investigational drug candidates. Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships and other relationships with numerous leading biotechnology and pharmaceutical companies such as Takeda, AstraZeneca, Merck, Pfizer and Innovent; and research and development relationships with leading research institutions such as Dana-Farber Cancer Institute, Mayo Clinic, National Cancer Institute and the University of Michigan. The company has built a talented team with a wealth of global experience in the discovery and development of innovative drugs and fully functional commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients. Forward-Looking Statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, Ascentage Pharma undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events, or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions and expectations or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions and expectations may alter in light of future development.
ROCKVILLE, Md. and SUZHOU, China, Dec. 10, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing, and commercializing therapies to address global unmet medical needs primarily for malignancies, today announced that it has released the latest clinical data from a global study of its novel drug candidate, olverembatinib (HQP1351), in patients with heavily pretreated chronic myeloid leukemia (CML), in a Poster Presentation at the 66th American Society of Hematology (ASH) Annual Meeting, taking place in San Diego, CA, the United States. Prof. Hagop Kantarjian, MD, and Prof. Elias Jabbour, MD, from the Department of Leukemia, The University of Texas MD Anderson Cancer Center, are the principal investigators of the study. The ASH Annual Meeting is one of the largest gatherings of the international hematology community, bringing together the most cutting-edge scientific research and latest data of investigational therapies that represent leading scientific and clinical advances in the global hematology field. Garnering growing interest from the global research community, results from multiple clinical and preclinical studies on four of Ascentage Pharma's drug candidates (olverembatinib, lisaftoclax, APG-2449, and APG-5918) have been selected for presentations, including two Oral Reports, at this year's ASH Annual Meeting. Furthermore, this is the seventh consecutive year for studies of olverembatinib to be selected for Oral Reports at the meeting. After releasing the preliminary results from the global study of olverembatinib in an Oral Report at ASH 2022 and the updated results from a larger patient sample at ASH 2023, this year Ascentage Pharma presented the 1.5-year follow-up data in patients with heavily pretreated CML-CP. In the latest data, olverembatinib showed strong, durable and consistent antileukemic activity, the ability to overcome resistance/intolerance to the third-generation tyrosine kinase inhibitor (TKI) ponatinib or the allosteric STAMP inhibitor asciminib, as well as favorable tolerability in patients with heavily pretreated CML-CP. As the first approved third-generation BCR-ABL inhibitor in China, olverembatinib has already been approved for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant CP-CML or accelerated-phase (AP-) CML harboring the T315I mutation; and adult patients with CP-CML resistant to and/or intolerant of first- and second-generation TKIs. In January 2024, olverembatinib was cleared by the US Food and Drug Administration (FDA) to enter a global registrational Phase III trial in previously treated adult patients with CML-CP. Prof. Elias Jabbour commented, "Olverembatinib is a very promising next-generation TKI, and existing data demonstrate its therapeutic ability in patients with resistant/intolerant to ≥2 TKIs CP-CML and above. The high response rate that Olverembatinib has shown in Ponatinib or Asciminib failed patients is encouraging. We continue to recruit for the global Phase III trial examining this novel therapy." Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, said, "The latest data from this global study further demonstrated olverembatinib's therapeutic potential in patients with drug-resistant CML, including those who had failed prior treatment with ponatinib and asciminib, and once again underscored the drug candidate's promise in addressing an unmet global clinical need in CML. At Present, a global registrational Phase III study of olverembatinib has been cleared by the US FDA and is already ongoing. We hope, in not too distant future, this innovative drug will become accessible to patients worldwide who are in desperate need of novel therapies. Fulfilling our founding mission of addressing unmet clinical needs in China and around the world, we will expedite the global clinical development of our key drug candidates such as olverembatinib to bring more safe and effective therapies to patients as soon as possible." Highlights of the data this study reported at ASH 2024 are as below: Olverembatinib (HQP1351) Overcomes Resistance/Intolerance to Asciminib and Ponatinib in Patients (pts) with Heavily Pretreated Chronic-Phase Chronic Myeloid Leukemia (CP CML): A 1.5-Year Follow-up Update with Comprehensive Exposure-Response (E-R) Analyses Format: Poster Presentation Abstract#: 3151 Session: 632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Poster II Highlights: Introduction: New treatment options are needed for patients with CP-CML resistant/intolerant to third-generation (3G) TKI ponatinib and/or asciminib, a specifically targeting the ABL myristoyl pocket (STAMP) inhibitor. Olverembatinib is a well-tolerated TKI with potential to overcome resistance. This update presents efficacy and safety data of olverembatinib in patients with heavily pretreated CP-CML. Enrolled Patients and Study Methods: Adults with CP-CML previously treated with ≥2 TKIs and/or a STAMP inhibitor, adequate organ function, and no major molecular response (MMR) were eligible. As of July 28, 2024, 67 pts with CP-CML were enrolled; median (range) follow-up was 74.3 (0.1-217.1) weeks; median (range) age, 50 (21-80) years; and 38 (56.7%) patients were male. Patients were randomly allocated to receive olverembatinib at doses of 30, 40, or 50 mg orally every other day (QOD) in 28-day cycles, with stratification based on T315I mutation status. Comprehensive E-R analyses were performed. Efficacy Results: No patient had efficacy at baseline. 35 of 60 (58.3%) evaluable patients achieved CCyR and 29/64 (45.3%) achieved MMR. At 12 months, the overall MMR rate was 61.4% (27/44). CCyR was achieved by 66.7% of patients with the T315I mutation vs 54.8% without it, and MMR was achieved by 50.0% vs 43.5%, respectively. Of 28 cytogenetic response-evaluable patients with ponatinib-failed CP-CML, 15 (53.6%) achieved CCyR. The CCyR rates in patients with prior ponatinib resistance and intolerance were 52.2% (12/23) and 75.0% (3/4), respectively. In the 30 molecular response-evaluable patients who were previously treated with ponatinib, 12 (40.0%) achieved MMR, including 47.8% (11/23) of those with prior resistance and 16.7% (1/6) with intolerance. No patient above had efficacy at baseline. In evaluable patients with asciminib treatment failure, 37.5% (6/16) achieved CCyR and 30% (6/20) achieved MMR, including a CCyR rate of 30.8% (4/13) and an MMR rate of 26.7% (4/15) in those with prior resistance, and a CCyR rate of 50.0% (1/2) and an MMR rate of 25.0% (1/4) in those with intolerance. No patient had efficacy at baseline. Safety Results: Among 66 subjects receiving olverembatinib, a total of 62 (93.9%) reported treatment-emergent adverse events (TEAEs) of any grade, with 44 (66.7%) experiencing grade ≥ 3 TEAEs. In addition, 60 (90.9%) patients reported TRAEs of any grade. Common TRAEs (≥20%) were elevated creatine phosphokinase (37.9%), thrombocytopenia (24.2%), and increased alanine aminotransferase (22.7%). Conclusions: Olverembatinib was well tolerated and showed strong and durable antileukemic activity in patients with heavily pretreated CP-CML. The registrational study is recruiting. * Olverembatinib is an investigational drug that has not been approved for any indication outside China. About Ascentage Pharma Ascentage Pharma (6855.HK) is a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily in malignancies. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK. The company has built a rich pipeline of innovative drug candidates that includes novel, highly potent Bcl-2 and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation TKIs. Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company has conducted more than 40 clinical trials in the US, Australia, Europe, and China, including 13 registrational studies (completed/ ongoing/planned). Olverembatinib, the company's first lead asset developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company's first approved product in China, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). To date, the drug had been included into the China National Reimbursement Drug List (NRDL). Furthermore, olverembatinib has been granted Orphan Drug Designations (ODDs) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU. To date, Ascentage Pharma has obtained a total of 16 ODDs from the US FDA and 1 Orphan Designation from the EMA of the EU for 4 of the company's investigational drug candidates. Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships and other relationships with numerous leading biotechnology and pharmaceutical companies such as Takeda, AstraZeneca, Merck, Pfizer and Innovent; and research and development relationships with leading research institutions such as Dana-Farber Cancer Institute, Mayo Clinic, National Cancer Institute and the University of Michigan. The company has built a talented team with a wealth of global experience in the discovery and development of innovative drugs and fully functional commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients. Forward-Looking Statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, Ascentage Pharma undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events, or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions and expectations or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions and expectations may alter in light of future development.
CAMBRIDGE, Mass., Dec. 10, 2024 /PRNewswire/ -- Nona Biosciences, a global biotechnology company providing a total solution from "Idea to IND" (I to ITM), ranging from target validation and antibody discovery through preclinical research, today announced a collaboration with Kodiak Sciences Inc. (Nasdaq: KOD). This partnership aims to advance the discovery of novel multi-target antibodies to treat ophthalmic diseases, leveraging Nona's proprietary Harbour Mice® fully human antibody platform. The Harbour Mice® platform generates fully human monoclonal antibodies in both the two heavy and two light chains (H2L2) format and the heavy chain-only (HCAb) format, eliminating the need for additional engineering or humanization. The HCAb Harbour Mice® platform, in particular, is transforming antibody development by producing unique, fully human heavy chain-only antibodies that are approximately half the size of conventional IgGs, offering significant advantages for next-generation antibody therapies. Under the agreement, Kodiak Sciences gains the right to use both the H2L2 and HCAb Harbour Mice® platforms in multiple programs for therapeutic antibody discovery and development. "We are pleased to collaborate with Kodiak Sciences to advance novel antibody therapies for ophthalmic diseases," said Jingsong Wang, M.D., Ph.D., Chairman of Nona Biosciences. "This agreement further validates our proprietary Harbour Mice® technology platform. With Nona's industry-leading technology and expertise, we look forward to supporting Kodiak Sciences in accelerating their next-generation therapeutic antibody development and bringing more innovative therapies to patients." "Retinal diseases involve a complex etiology, so advancing more effective therapies will require targeting multiple pathways at once," said Victor Perlroth, M.D., Chairman and CEO of Kodiak Sciences Inc. "We are thrilled to announce our partnership with Nona Biosciences, leveraging their advanced Harbour Mice® platforms and robust expertise in antibody discovery. This collaboration will accelerate our mission to create next generation biotherapeutics, combining our strengths to bring innovative treatments to patients in need." About Nona Biosciences Nona Biosciences is a global biotechnology company committed to cutting-edge technology innovations and providing a total solution from "Idea to IND" ("I to ITM"), ranging from target validation and antibody discovery through preclinical research. The integrated antibody and antibody-related discovery services with multiple modalities range from antigen preparation, animal immunization, single B cell screening, to antibody lead generation and engineering, developability assessment and pharmacological evaluation, leveraging advantages of Harbour Mice® platforms and the experienced therapeutic antibody discovery team. Harbour Mice® generates fully human monoclonal antibodies in classical two light and two heavy chain (H2L2) format, and heavy chain-only (HCAb) format. Integrating Harbour Mice® and a single B cell cloning platform, Nona Biosciences is focused on driving global inventions of transformative next-generation drugs. For more information, please visit: www.nonabio.com. About Kodiak Sciences Inc. Kodiak Sciences (Nasdaq: KOD) is a biopharmaceutical company committed to researching, developing, and commercializing transformative therapeutics to treat a broad spectrum of retinal diseases. We are focused on bringing new science to the design and manufacture of next generation retinal medicines to prevent and treat the leading causes of blindness globally. For more information, please visit www.kodiak.com.
SHANGHAI and NANJING, China and SAN JOSE, Calif., Dec. 10, 2024 /PRNewswire/ -- IASO Biotherapeutics ("IASO Bio"), a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies and biologics, today shared findings via a poster presentation of the results on the impact of CAR T-Cell Persistence on Clinical Outcomes in Relapsed/Refractory Multiple Myeloma(R/RMM) with Equecabtagene Autoleucel (Eque-cel, FUCASO) at the 66th American Society of Hematology (ASH) Annual Meeting. The results highlighted the importance of the efficacy-to-target ratio in the treatment of multiple myeloma, emphasizing the crucial role of sustained CAR T-cell persistence, unaffected by baseline soluble B-cell maturation antigen (sBCMA) levels, to achieve optimal disease control. Title: Impact of CAR T-Cell Persistence on Clinical Outcomes in Relapsed/Refractory Multiple Myeloma: Insights from the Phase 2 FUMANMA-1 StudyPublication Number: 4761Presentation Time: 6:00 PM -8:00 PM, Monday, December 9, 2024（San Diego） Conclusions The efficacy-to-target ratio is a crucial determinant of clinical outcomes in patients treated with Eque-cel, underscoring the importance of CAR T-cell persistence. This ratio may serve as a biomarker for future treatment planning, highlighting the need for sustained CAR T-cell persistence to achieve optimal disease control. Baseline sBCMA levels do not negatively affect the persistence and efficacy of Eque-cel. This post-hoc analysis of the Phase 2 study of FUMANBA-1 primarily investigated the relationship between CAR T-cell persistence and progression-free survival (PFS) as well as time to progression (TTP). It also examined the impact of sBCMA levels through the lens of the efficacy-to-target ratio, measured by the ratio of vector copy number (VCN) duration to baseline sBCMA levels. With a median follow-up of 24.67 months, 107 patients in 14 centers were treated with Eque-cel. At the data cutoff, the ongoing remission group showed a longer median VCN duration than those with progression or relapse（12.52m vs 9.03m）,as shown in Figure 1.The median efficacy-to- target ratio for the 107 subjects was 1.05 (days*mL/ng). As shown in Figure 2, those with a lower ratio had a significantly higher risk of disease progression, with a hazard ratio (HR) for TTP of 3.07 (95% CI: 1.51–6.24, p=0.0011) and for PFS of 2.3 (95% CI: 1.27–4.14, p=0.0045), both indicating a strong correlation between the efficacy-to-target ratio and the risk of disease progression. Among baseline characteristics—including ECOG score, R-ISS and ISS disease stages, tumor BCMA expression, tumor burden, baseline sBCMA level, previous autologous stem cell transplantation (ASCT), triple-class exposure, bridging therapy, prior CAR-T treatment, and lymphodepleting conditioning—only previous ASCT (n=30) and ADA as post-infusion factors were significantly associated with Eque-cel persistence, with hazard ratios (HR) 0.35 and 5.79, respectively, as shown in Table 1. Table 1：Influencing factors analysis for CAR-T cell persistence Among the 107 subjects, only 14 exhibited an aplastic neutrophil recovery phenotype, characterized by persistent neutrophil counts below 500/μl for 14days or more. This phenotype did not significantly affect VCN duration compared to patients without the condition, indicating that the long-term persistence of Eque-cel does not increase hematological toxicity. The principal investigator of this study, Professor Lugui Qiu, from the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, and Professor Chunrui Li, from Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, stated: "This post-hoc analysis explored the determinants of long-term efficacy after CAR-T treatment through the lens of the efficacy-to-target ratio (VCN persistence/sBCMA). It confirmed the significance of this ratio in achieving long-term control of multiple myeloma (PFS and TTP). The findings suggest that a CAR-T product with long-lasting efficacy, unaffected by the target, which means not affected by sBCMA levels, would be a more optimal choice in the treatment of MM. The result further highlighted the crucial role of sustained persistence of CAR-T cells in improving the prognosis of patients with R/RMM. In our 2023 ASH oral report, we previously confirmed a positive correlation between Eque-cel persistence and the maintenance of MRD negativity. The median duration time of CAR-T cells for Eque-cel was 419 days, which may be a key factor in achieving the high MRD negativity rate of 97.8% and the sustained MRD negativity rate of 81.7% at 12 months post-infusion. Notably, only 14 of 107 subjects exhibited aplastic neutrophil recovery lasting 14 days or more. This finding indicates that the long-term persistence of Eque-cel does not increase hematological toxicity, further validating the safety of this therapy." Dr. Jie Chen, Chief Medical Officer of IASO Bio, stated:The outcomes presented by IASO Bio at the ASH Annual Meeting highlighted the significant impact of the efficacy-to-target ratio on disease control in multiple myeloma. The data demonstrated that subjects with an efficacy-to-target ratio above the median value had a better prognosis in terms of PFS and TTP. These findings further confirmed the positive effect of long-term CAR-T cells persistence in achieving sustained remission. Eque-cel was deliberately selected in its early development to be a candidate molecule not influenced by sBCMA levels, offering patients deeper and more durable remission. Additionally, the study showed that only a small number of patients experienced neutropenia lasting more than 14 days after treatment with Eque-cel. The result affirmed that the long-term persistence of Eque-cel does not increase the hematological toxicity, further validating its safety profile. We are fully confident in the outstanding efficacy and safety of Eque-cel. Currently, We have initiated and are rapidly advancing a randomized controlled Phase 3 clinical study of this therapy for second- and third-line multiple myeloma. We look forward to bringing this advanced therapy to a broader population of multiple myeloma patients both domestically and internationally as soon as possible." About FUMANBA-1 StudyFUMANBA-1 is a single-arm, open-label phase 1b/2 registrational clinical study conducted in 14 Chinese centers to assess the efficacy and safety of the Equecabtagene Autoleucel in patients with R/RMM who have received ≥3 lines of prior therapies. The trial enrolled patients with RRMM who had previously received at least three lines of therapy, including proteasome inhibitors and immunomodulatory agents-based chemotherapy regimens, and had disease progression on their last line of therapy. Patients who had previously received BCMA CAR-T therapy were also eligible for enrollment. About IASO BioIASO Bio is a biopharmaceutical company focused on the discovery and development of innovative cell therapies and biologics for oncology and autoimmune diseases. IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercialization. Its pipeline includes a diversified portfolio of over 10 novel products, including Equecabtagene Autoleucel (a fully human BCMA CAR-T injection). Equecabtagene Autoleucel received Biologics License Application (BLA) approval from China's National Medical Products Administration (NMPA) in June 2023 and U.S. FDA IND approval for the treatment of R/RMM in December 2022. Leveraging its strong management team, innovative product pipeline, as well as integrated and high quality manufactural and clinical capabilities, IASO aims to deliver transformative, curable, and affordable therapies that fulfil unmet medical needs to patients in China and around the world. For more information, please visit http://www.iasobio.com or www.linkedin.com/company/iasobiotherapeutics.
SHANGHAI and HONG KONG, Dec. 10, 2024 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for hematologic malignancies and solid tumors, today announced that it presented the latest data from two clinical studies of selinexor in two posters at the 2024 American Society of Hematology Annual Meeting (ASH 2024). Details on the Posters: Title: Weekly Selinexor, Bortizomib and Dexamethasone (SVd) Versus Twice Weekly Bortizomib and Dexamethasone (Vd) in Chinese Patients with Relapsed and Refractory Multiple Myeloma (RRMM): Primary Analysis of Phase III Bench StudyPublication Number: 4748 Session: 654. Multiple Myeloma: Pharmacologic Therapies: Poster IIIDate: Monday, December 9, 2024Time: 6:00 PM - 8:00 PM (Pacific time) 10:00 AM - 12:00 PM, December 10, 2024 (Beijing time) The BENCH study is a Phase III randomized, open-label, multicenter clinical trial, aiming to evaluate the efficacy and safety of selinexor, bortezomib and dexamethasone (SVd) regimen against bortezomib and dexamethasone (Vd) regimen in Chinese adult patients with R/R MM who have received one to three prior lines of therapy. At present, a New Drug Application (NDA) based this study has already been submitted to and accepted by China's National Medical Products Administration (NMPA). As of May 9, 2024, a total of 154 Chinese R/R MM patients were randomized to SVd group (n=101) or Vd group (n=53) and 152 patients received at least one dose of study drug (safety population). Efficacy results showed that the median progression-free survival (mPFS) was 8.1 months with SVd group and 6.3 months with Vd group. The overall response rate (ORR) was higher in SVd group than in Vd group. SVd group had a significantly higher proportion of patients with a very good partial response (VGPR) or better responses. The median time to response and the median duration of response were 0.8 vs 1.4 months and 9.7 vs 7.2 months (SVd vs Vd), respectively. During the study, some subjects experienced treatment emergent adverse events (TEAEs). The most frequent Grade 3-4 adverse events (AEs) for SVd and Vd (≥10%) included thrombocytopenia, lymphocytopenia, and anemia. The incidence of Grade≥2 peripheral neuropathy (PN) was significantly lower in SVd than in Vd group. The BENCH study has met its primary and key secondary endpoints, with results consistent with the BOSTON study. These results showed that the SVd regimen decreased the risk of progression and obtained much more profound responses compared to standard Vd regimen in Chinese patients with R/R MM. The incidence of Grade ≥2 PN were significantly reduced. Title: Selinexor Combined with Tislelizumab in Patients with Relapsed or Refractory Extranodal NK/T-Cell Lymphoma (R/R ENKTL): Preliminary Results of Arm C, from a Multicenter, Single-Arm, Phase I/II Study, TouchPublication Number: 4448 Session: 625. T Cell, NK Cell, or NK/T Cell Lymphomas: Clinical and Epidemiological: Poster IIIDate: Monday, December 9, 2024Time: 6:00 PM - 8:00 PM (Pacific time) 10:00 AM - 12:00 PM, December 10, 2024 (Beijing time) The Phase I/II TOUCH study is investigating selinexor combined with different drugs in R/R ENKTL. Arm C of the study aims to evaluate the safety, tolerability and preliminary efficacy of selinexor in combination with anti-PD-1 antibody tislelizumab. As of 18 May 2024, 17 R/R ENKTL patients were enrolled in Arm C [Sel 40mg (n=3); Sel 60mg (n=14)]. Sixteen patients previously exposed to both L-asparaginase (L-Asp) and checkpoint inhibitors (CPIs) including 8 patients who had received prior Tislelizumab (Tis); eleven (64.7%) patients were refractory to their last-line therapy. Efficacy results showed that, of 16 CPIs exposed patients (one patient was efficacy non-evaluable), the ORR was 75% (12/16), including 7 CRs and 5 PRs; and the median PFS was 6.7 months (95% CI 1.5, NE). During the study, all patients experienced TEAEs. The most common TEAEs included anemia, neutropenia, asthenia, decreased appetite, weight loss, and thrombocytopenia. Ten patients (58.8%) experienced Grade≥3 TEAEs. Treatment emergent serious adverse events (TESAEs) occurred in 4 patients (23.5%), of which two were considered treatment related. No patient discontinued due to TEAEs. Most of toxicities were manageable by dose modification and supportive care. In the study, the chemo-free regimen, Sel plus Tis, showed a favorable response rate and manageable safety profile in R/R ENKTL. This approach may probably reverse drug resistance in ENKTL that has progressed after prior CPI treatment. About Antengene Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading commercial-stage R&D-driven global biopharmaceutical company focused on the discovery, development, manufacturing and commercialization of innovative first-in-class/best-in-class therapeutics for the treatment of hematologic malignancies and solid tumors, in realizing its vision of "Treating Patients Beyond Borders". Since 2017, Antengene has built a pipeline of 9 oncology assets at various stages going from clinical to commercial, including 6 with global rights, and 3 with rights for the APAC region. To date, Antengene has obtained 31 investigational new drug (IND) approvals in the U.S. and Asia, and submitted 10 new drug applications (NDAs) in multiple Asia Pacific markets, with the NDA for XPOVIO® (selinexor) already approved in Mainland of China, Taiwan China, Hong Kong China, Macau China, South Korea, Singapore, Malaysia, Thailand and Australia. Forward-looking statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. For a further discussion of these and other factors that could cause future results to differ materially from any forward-looking statement, please see the other risks and uncertainties described in the Company's Annual Report for the year ended December 31, 2023, and the documents subsequently submitted to the Hong Kong Stock Exchange. For more information, please contact: Investor Contacts: Donald LungE-mail: Donald.Lung@antengene.comMobile: +86 18420672158 PR Contacts:Peter QianE-mail: Peter.Qian@antengene.comMobile: +86 13062747000
Australian Human Research Ethics Committees approves SKY-0515 treatment for up to 12 weeks in the company's ongoing Phase 1 trial in patients with Huntington's Disease Volumetric MRI measurement is also approved as exploratory study endpoint BOSTON, Dec. 10, 2024 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company, today announced that the Australian Human Research Ethics Committees (HREC) have approved SKY-0515 treatment for up to 12 weeks in the Company's ongoing Phase 1 trial in patients with Huntington's Disease (HD). HREC also agreed to the inclusion of Volumetric MRI (vMRI) measurements as an early exploratory endpoint in the trial. vMRI measurement of the brain's regions affected in HD can provide additional data on SKY-0515's effect in HD patients. "We're excited to announce that after demonstrating 72% huntingtin (HTT) mRNA reduction with SKY-0515 in the healthy volunteer portion of this study, we have been approved to extend the duration of treatment in Huntington's patients," said Sergey Paushkin, Head of R&D, Skyhawk Therapeutics. "We're thankful to the HREC for their careful review of our data and approval of this extension, as well as their approval of our use of vMRI measurements as an exploratory endpoint. Both approvals broaden our opportunity to demonstrate the performance of SKY-0515 and may enable us to progress to pivotal trials faster than previously expected." SKY-0515 is Skyhawk's investigational, oral small molecule RNA splicing modifier developed through the company's novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein, a key driver of somatic CAG-repeat expansion and HD pathology. About SKY-0515 Phase 1 Clinical Study SKY-0515 is currently being evaluated in a Phase 1 clinical trial. The Phase 1 clinical trial is a first-in-human trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SKY-0515 in healthy volunteers and individuals with early-stage Huntington's disease (HD). The trial is separated into three parts. Topline pharmacodynamic results for Parts A (single-ascending dose) and B (multiple-ascending dose), which evaluated SKY-0515 in healthy volunteers, demonstrated a dose-dependent reduction of HTT mRNA and reached an average reduction of 72% at a daily oral dose of 9mg. SKY-0515 was generally well tolerated at all doses tested. Part C is a double-blind placebo-controlled parallel design study of two dose levels of SKY-0515 and placebo in individuals with early-stage HD, which aims to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamic parameters. Recruitment for Part C is ongoing, and topline data are expected in mid-2025. Additional information for the SKY-0515 Phase 1 study, including participating sites and eligibility criteria, can be found at the Australian New Zealand Clinical Trials Registry. About Skyhawk TherapeuticsSkyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk's novel platform applies to a broad range of disease areas, including neurodegenerative disease, metabolic disease, autoimmune disease, fibrosis and oncology. For more information visit www.skyhawktx.com. Skyhawk ContactKyle Dow, VP Corporate Developmentkyle.dow@skyhawktx.com Logo - https://mma.prnasia.com/media2/710814/Skyhawk_Therapeutics_Logo.jpg?p=medium600
Biotechnology
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