Latest news with #2025Congress
Yahoo
a day ago
- Business
- Yahoo
Biomea Fusion Presents Updated Preliminary Clinical Data for Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory Acute Leukemia at EHA 2025
New clinical results show sustained CRi, deep bone marrow responses, and encouraging survival in FLT3-mutant AML patients, including those previously treated with gilteritinib REDWOOD CITY, Calif., June 13, 2025 (GLOBE NEWSWIRE) -- Biomea Fusion, Inc. ('Biomea' or the 'Company') (Nasdaq: BMEA), a clinical-stage diabetes and obesity medicines company, today announced updated preliminary clinical data from the ongoing Phase I COVALENT-103 trial of BMF-500 in adults with relapsed or refractory ('R/R') acute leukemia ('AL'). The results will be presented in a poster presentation at the European Hematology Association ('EHA') 2025 Congress in Milan, Italy. The presentation by Dr. Farhad Ravandi of The University of Texas MD Anderson Cancer Center will highlight emerging safety, pharmacokinetics/pharmacodynamics, and early clinical activity data for BMF-500, a highly selective covalent FMS-like tyrosine kinase 3 ('FLT3') inhibitor, in heavily pretreated patients with R/R AL. Key Results from the EHA 2025 Poster Presentation27 patients were enrolled across two study arms, Arm A (no CYP3A4 inhibitor; n=10) and Arm B (CYP3A4 inhibitor; n=17). All patients had R/R AL, with a median of 4 prior lines of therapy. 18 of the 27 patients were FLT3 mutations ('FLT3m') while the other 9 patients had FLT3 wild-type AL. Frequent co-mutations included WT1, TP53, IDH1/2, and NRAS. All 18 FLT3m patients had failed gilteritinib in the R/R setting and 9 of the 18 had received at least two FLT3 inhibitors prior to study entry. 26 of 27 (96%) enrolled patients had also received and failed the BCL2 inhibitor venetoclax. Key findings include: Clinical Activity Observed: 9 of 11 efficacy-evaluable FLT3m patients, defined as all patients enrolled who received at least one dose and had at least one disease assessment, showed bone marrow (BM) blast reduction; 5 of 11 achieved >50% BM blast reduction. 1 FLT3m patient achieved complete remission with incomplete hematologic recovery (CRi), sustained for 6 cycles. 1 FLT3m patient achieved morphologic leukemia-free state (MLFS); response is ongoing. 1 FLT3m patient met all criteria for partial response (PR) except platelet recovery; categorized as near PR. 2 of 4 efficacy-evaluable FLT3 wild-type patients achieved durable disease control ≥120 days, with treatment ongoing for one patient. Additional clinical improvements include reductions in peripheral blasts, transfusion dependency, and frequency of hydroxyurea use. Pharmacokinetics/Pharmacodynamics: FLT3 inhibition correlated with BMF-500 systemic exposures. Bone marrow and plasma concentrations of BMF-500 and its metabolites were comparable, suggesting good compartmental penetration. Survival: Median overall survival (mOS) among all treated FLT3m patients (n=18) was 3.8 months (Arm A) and 3.5 months (Arm B) during dose escalation. For the efficacy-evaluable FLT3m patients (n=12), the mOS for Arms A and B was 3.8 and 3.6 months, respectively during dose escalation. These survival durations compare favorably to historical mOS of 2.1 months in patients with R/R FLT3m acute myeloid leukemia ('AML') post-failure with both gilteritinib and venetoclax. 1 Ongoing Dose Escalation: Dose escalation continues at 200 mg BID (Arm A) and 75 mg BID (Arm B). Based on observed activity and tolerability, further evaluation is underway to determine optimal biologic dose ('OBD') and recommended Phase II dose ('RP2D'). Safety and Tolerability BMF-500 was generally well-tolerated across dose levels. No dose-limiting toxicities (DLTs), QT prolongation, or discontinuations due to treatment-related adverse events were reported. Escalation is ongoing without safety restrictions. 'The updated COVALENT-103 results continue to support the potential of BMF-500 as a selective, covalent FLT3 inhibitor,' said Mick Hitchcock, Ph.D., Interim Chief Executive Officer of Biomea Fusion. 'We are encouraged by the depth of bone marrow responses, the achievement of MLFS and CRi, and the early survival benefit in heavily pretreated patients with FLT3 mutations who had progressed following prior FLT3 inhibitor therapy. These data speak to BMF-500's potential to meaningfully improve outcomes in a high-risk AML population with no currently available treatment options.' Following completion of the COVALENT-103 dose escalation phase in R/R AL patients with FLT3m, Biomea plans to conclude its internal development of BMF-500 in oncology and is actively exploring strategic partnerships to advance the program. Poster Presentation Details Date/Time: Saturday, June 14 (18:30-19:30 CEST) Title: Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory (R/R) Acute Leukemia (AL): Preliminary Phase 1 Data from the COVALENT-103 Study (NCT05918692) Poster Number: PS1520 Presenter: Farhad Ravandi, M.D., University of Texas MD Anderson Cancer Center About COVALENT-103 COVALENT-103 is a multicenter, open-label, non-randomized trial seeking to evaluate the safety and efficacy of BMF-500, a twice daily oral treatment, in adult patients with R/R AL with FLT3 wild-type or FLT3m. The Phase I COVALENT-103 study aims to evaluate the safety and tolerability of BMF-500, determine the optimal biologic dose and recommended Phase II dose, and identify initial efficacy signals. Additional information about the Phase I clinical trial of BMF-500 can be found at using the identifier, NCT05918692. About BMF-500 BMF-500 is an investigational, orally bioavailable, covalent small molecule inhibitor of FLT3, discovered in-house using Biomea's proprietary FUSION™ System. Designed to be highly potent and selective, BMF-500 has demonstrated encouraging potential in extensive preclinical studies. Its kinase inhibitory profile indicates strong target selectivity, which may translate to a reduced risk of off-target effects. About Biomea FusionBiomea Fusion is a clinical-stage diabetes and obesity medicines company focused on the development of its oral small molecules, icovamenib and BMF-650, both designed to significantly improve the lives of patients with diabetes, obesity, and other metabolic diseases. We aim to cure. Visit us at and follow us on LinkedIn, X, and Facebook. Forward-Looking Statements Statements we make in this press release may include statements which are not historical facts and are considered forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the 'Securities Act'), and Section 21E of the Securities Exchange Act of 1934, as amended (the 'Exchange Act'). These statements may be identified by words such as 'aims,' 'anticipates,' 'believes,' 'could,' 'estimates,' 'expects,' 'forecasts,' 'goal,' 'intends,' 'may,' 'plans,' 'possible,' 'potential,' 'seeks,' 'will,' and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact, including statements regarding the clinical and therapeutic potential of our product candidates and development programs, including BMF-500, the potential of BMF-500 as a treatment for patients with FLT3m R/R AL, our research, development, partnership and regulatory plans, and the timing of such events may be deemed to be forward-looking statements. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. Any forward-looking statements in this press release are based on our current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements, including the risk that preliminary or interim results of preclinical studies or clinical trials may not be predictive of future or final results in connection with future clinical trials and the risk that we may encounter delays in preclinical or clinical development, patient enrollment and in the initiation, conduct and completion of our ongoing and planned clinical trials and other research and development activities. These risks concerning Biomea's business and operations are described in additional detail in its periodic filings with the U.S. Securities and Exchange Commission (SEC), including its most recent periodic report filed with the SEC and subsequent filings thereafter. Biomea Fusion explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Reference: 1. Corley et al. (P1798) HemaSphere 2024;8(S1), 3339-3340. Contact: Meichiel Jennifer Weiss Sr. Director, Investor Relations and Corporate DevelopmentIR@
Yahoo
2 days ago
- Business
- Yahoo
Data of InnoCare's Robust Hemato-Oncology Pipelines Presented at the European Hematology Association (EHA) 2025 Congress
BEIJING, June 13, 2025--(BUSINESS WIRE)--Latest data of InnoCare's (HKEX: 09969; SSE: 688428) robust oncology pipelines were presented at the ongoing European Hematology Association (EHA) 2025 Congress. Poster Presentation: 1. First Presentation of Efficacy and Safety Data for First-Line Treatment of CLL/SLL with BCL2 Inhibitor Mesutoclax in Combination with BTK Inhibitor Orelabrutinib (Abstract No.: PS1567) The study showed that mesutoclax (100 and 125 mg) in combination with orelabrutinib was safe and well tolerated in patients with treatment naïve (TN) CLL/SLL. Substantial efficacy was observed, with early high undetectable MRD (uMRD) rate. 42 patients with TN CLL/SLL were enrolled (mesutoclax 100 mg, n=21; 125 mg, n=21). The fixed-duration treatment of mesutoclax in combination with orelabrutinib is expected to deliver deeper remissions for TN CLL/SLL patients. In all patients, the overall response rate (ORR) was 97.6%. At week 24 of the combination therapy, in the 125 mg dose cohort, the overall response rate (ORR) was 100%, the complete remission rate (CRR) was 23.8%, the CRR in target lesions was 57.1%, and the peripheral blood (PB) uMRD rate was 48%. In the 100 mg mesutoclax dose cohort, the ORR was 95.2%, the CRR was 19.0%, the CRR in target lesions was 42.9%, and the PB uMRD rate was 19%. With the extension of the duration of treatment, the therapeutic efficacy is expected to be further improved. All patients are still on treatment. No disease progression or death occurred, and no adverse events leading to discontinuation of treatment were reported. Due to its favorable efficacy and safety profile, a registrational Phase III clinical study of mesutoclax (125 mg once daily) in combination with orelabrutinib for the treatment of TN CLL/SLL patients has been initiated, with patient enrollment being accelerated. 2. Orelabrutinib Combined with Bendamustine-Rituximab or Obinutuzumab followed by Orelabrutinib Maintenance in Untreated Marginal Zone Lymphoma (OPTIMIZE): A Multicenter, Single-Arm, Phase II Study (Abstract No.: PF898) The absence of a standard first-line treatment for marginal zone lymphoma (MZL) have inspired the exploration of novel regimens. In recent years, Bruton's tyrosine kinase inhibitors (BTKis) have demonstrated durable responses with a favorable benefit-risk profile across all MZL subtypes in the relapsed/refractory setting. Orelabrutinib is a novel BTK inhibitor with higher selectivity and fewer off-target than other BTK inhibitors. Orelabrutinib combined with bendamustine-rituximab or obinutuzumab followed by orelabrutinib maintenance was effective and well-tolerated in untreated patients with MZL. The majority of patients presented with MALT lymphoma and had Ann Arbor stage II-IV disease. At the end of induction treatment, the overall response rate (ORR) was 100.0% among all enrolled patients. At the data cutoff, the median progression-free survival (PFS) and overall survival (OS) remained immature. No BTK inhibitor-related adverse events (AEs), such as atrial fibrillation or bleeding, were observed. 3. The Rationality, Efficacy and Safety of Orelabrutinib plus Obinutuzumab (O2) in Systemic Treatment-naïve Marginal Zone Lymphoma: A Prospective Cohort Study (Abstract No.: PS1902) The Orelabrutinib plus Obinutuzumab regimen had well rationality and demonstrated promising efficacy. Across the entire study and during the period of induction therapy, the best objective response rate (ORR) was 100%. 3 patients who achieved partial response (PR) after the induction therapy subsequently achieved complete response (CR) in the maintenance period. The 18-month progression-free survival (PFS) and overall survival (OS) rates were both 100%. 4. Ultra-low Dose Radiotherapy Combined with Orelabrutinib Improves the Complete Response Rate of Treatment for Localized Ocular Adnexal Extranodal Marginal Zone B-cell Lymphoma (Abstract No.: PS1901) The combination of ultra-low dose radiotherapy and orelabrutinib in the treatment of ocular adnexal extranodal marginal zone lymphoma (OA-EMZL) not only improves the effectiveness of treatment but also significantly reduces the toxic effects of radiotherapy, providing a new approach for the treatment of localized OA-EMZL. Of all the 17 patients who completed treatment, 16 patients achieved complete response (CR) and one patient achieved a partial response. Additionally, the lesions in the patients still undergoing treatment have shrunk compared to before treatment. 5. Orelabrutinib plus Bendamustine-Rituximab (OBR) versus Bendamustine-Rituximab (BR) in Transplant-ineligible, Intermediate- to High-risk Mantle Cell Lymphoma (MCL) (Abstract No.: PS1969) This open-label, randomized, multi-center study aims to compare the efficacy and safety of Orelabrutinib plus Bendamustine-Rituximab versus Bendamustine-Rituximab in transplant-ineligible, intermediate- to high-risk mantle cell lymphoma (MCL) patients. Early data suggest that Orelabrutinib plus Bendamustine-Rituximab could reduce disease progression events compared to Bendamustine-Rituximab in high-risk MCL. Updated outcomes on efficacy, safety, and biomarker analysis will be reported. 6. Pomalidomide, Rituximab, Orelabrutinib, and MiniCHOP-like (PRO-miniCHOP) in Elderly Patients with Newly Diagnosed Diffuse Large B-cell Lymphoma: Updated Results from a Phase II Study (Abstract No.: PF950) The results further support Pomalidomide, Rituximab, Orelabrutinib, and MiniCHOP-like (PRO-miniCHOP) as a potential treatment option for elderly patients with diffuse large B-cell lymphoma (DLBCL), demonstrating promising efficacy and acceptable safety, especially for those who responded to the Pomalidomide-Rituximab-Orelabrutinib (PRO) induction therapy. A total of 32 patients were enrolled in this study, of whom 26 patients completed ≥3 cycles of the PRO-miniCHOP, resulting in a complete response rate (CRR) of 65.4% and overall response rate (ORR) of 100.0%. Among the 21 patients who completed the full 6-cycle therapy with PRO-miniCHOP, both the CRR and ORR were 95.2%. At a median follow-up of 15.6 months, the median progression-free survival (PFS) and overall survival (OS) had not yet been reached, with the 2-year PFS and OS rates being 94.7% and 100.0%, respectively. 7. Orelabrutinib Addition to R-CHOP-like Regimen Adapted to Response in Treatment-Naïve Non-GCB DLBCL: Update Results of Orient Study (Abstract No.: PS1943) In non-germinal center B-cell-like (non-GCB) diffuse large B-cell lymphoma (DLBCL) patients who responded to orelabrutinib plus rituximab (OR) induction, orelabrutinib plus R-CHOP-like (OR-CHOP) exhibited favorable antitumor activity and manageable safety. Update results further support the orelabrutinib plus R-CHOP-like therapy as an option in this disease. At end of 6-cycle orelabrutinib plus R-CHOP-like, response (all 100%) was independent of double-expressing lymphoma (DEL), extranodal involvement (EI), and Lymphgen subtypes. No off-target-related cardiac toxicities occurred. 8. Primary Efficacy and Safety of First-line R-MTO Regimen (Rituximab, Methotrexate, Thiotepa, and Orelabrutinib) followed by Autologous Hematopoietic Stem Cell Transplantation in PCNSL (Abstract No.: PF966) The Rituximab, Methotrexate, Thiotepa, and Orelabrutinib (R-MTO) induction treatment has demonstrated notable efficacy in achieving higher response rate among patients with newly diagnosed primary central nervous system lymphoma (PCNSL), with a manageable safety profile. At the data cutoff, all patients had completed four cycles of induction therapy, and the complete response (CR) rate and overall response rate (ORR) were 93.34% and 96.67%, respectively. The 12-month progression-free survival (PFS) and overall survival (OS) rates were 82.26% and 85.33%, respectively. 9. Orelabrutinib, Rituximab Combined with High-Dose Methotrexate as Induction Therapy in Newly Diagnosed Primary Central Nervous System Lymphoma (Abstract No.: PS1917) This study demonstrated the promising efficacy of the Orelabrutinib, Rituximab Combined with High-Dose Methotrexate induction regimen in newly diagnosed primary central nervous system lymphoma (PCNSL), with encouraging response rates and durable progression-free survival (PFS). The regimen also showed promising overall survival (OS) trends, highlighting its potential as an effective treatment. Treatment-related adverse events (TRAEs) were manageable, and no severe BTK inhibitor-related off-target toxicities (e.g., atrial fibrillation/flutter) were observed. These findings suggest that the Orelabrutinib, Rituximab Combined with High-Dose Methotrexate regimen is a well-tolerated and effective therapeutic option for PCNSL. Except the above poster presentations, more than 10 studies were also selected as poster presentations or online publications at the meeting. For more detailed clinical data, please refer to EHA website. About InnoCare InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancers and autoimmune diseases with unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, Hong Kong, and the United States. InnoCare Forward-looking Statements This report contains the disclosure of some forward-looking statements. Except for statements of facts, all other statements can be regarded as forward-looking statements, that is, about our or our management's intentions, plans, beliefs, or expectations that will or may occur in the future. Such statements are assumptions and estimates made by our management based on its experience and knowledge of historical trends, current conditions, expected future development and other related factors. This forward-looking statement does not guarantee future performance, and actual results, development and business decisions may not match the expectations of the forward-looking statement. Our forward-looking statements are also subject to a large number of risks and uncertainties, which may affect our short-term and long-term performance. View source version on Contacts Media Chunhua Investors 86-10-66609999ir@ 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
2 days ago
- Health
- Business Wire
Data of InnoCare's Robust Hemato-Oncology Pipelines Presented at the European Hematology Association (EHA) 2025 Congress
BEIJING--(BUSINESS WIRE)--Latest data of InnoCare's (HKEX: 09969; SSE: 688428) robust oncology pipelines were presented at the ongoing European Hematology Association (EHA) 2025 Congress. First presentation of the efficacy and safety data for first-line treatment of CLL/SLL with BCL2 inhibitor Mesutoclax in combination with BTK inhibitor orelabrutinib Poster Presentation: 1. First Presentation of Efficacy and Safety Data for First-Line Treatment of CLL/SLL with BCL2 Inhibitor Mesutoclax in Combination with BTK Inhibitor Orelabrutinib (Abstract No.: PS1567) The study showed that mesutoclax (100 and 125 mg) in combination with orelabrutinib was safe and well tolerated in patients with treatment naïve (TN) CLL/SLL. Substantial efficacy was observed, with early high undetectable MRD (uMRD) rate. 42 patients with TN CLL/SLL were enrolled (mesutoclax 100 mg, n=21; 125 mg, n=21). The fixed-duration treatment of mesutoclax in combination with orelabrutinib is expected to deliver deeper remissions for TN CLL/SLL patients. In all patients, the overall response rate (ORR) was 97.6%. At week 24 of the combination therapy, in the 125 mg dose cohort, the overall response rate (ORR) was 100%, the complete remission rate (CRR) was 23.8%, the CRR in target lesions was 57.1%, and the peripheral blood (PB) uMRD rate was 48%. In the 100 mg mesutoclax dose cohort, the ORR was 95.2%, the CRR was 19.0%, the CRR in target lesions was 42.9%, and the PB uMRD rate was 19%. With the extension of the duration of treatment, the therapeutic efficacy is expected to be further improved. All patients are still on treatment. No disease progression or death occurred, and no adverse events leading to discontinuation of treatment were reported. Due to its favorable efficacy and safety profile, a registrational Phase III clinical study of mesutoclax (125 mg once daily) in combination with orelabrutinib for the treatment of TN CLL/SLL patients has been initiated, with patient enrollment being accelerated. 2. Orelabrutinib Combined with Bendamustine-Rituximab or Obinutuzumab followed by Orelabrutinib Maintenance in Untreated Marginal Zone Lymphoma (OPTIMIZE): A Multicenter, Single-Arm, Phase II Study (Abstract No.: PF898) The absence of a standard first-line treatment for marginal zone lymphoma (MZL) have inspired the exploration of novel regimens. In recent years, Bruton's tyrosine kinase inhibitors (BTKis) have demonstrated durable responses with a favorable benefit-risk profile across all MZL subtypes in the relapsed/refractory setting. Orelabrutinib is a novel BTK inhibitor with higher selectivity and fewer off-target than other BTK inhibitors. Orelabrutinib combined with bendamustine-rituximab or obinutuzumab followed by orelabrutinib maintenance was effective and well-tolerated in untreated patients with MZL. The majority of patients presented with MALT lymphoma and had Ann Arbor stage II-IV disease. At the end of induction treatment, the overall response rate (ORR) was 100.0% among all enrolled patients. At the data cutoff, the median progression-free survival (PFS) and overall survival (OS) remained immature. No BTK inhibitor-related adverse events (AEs), such as atrial fibrillation or bleeding, were observed. 3. The Rationality, Efficacy and Safety of Orelabrutinib plus Obinutuzumab (O2) in Systemic Treatment-naïve Marginal Zone Lymphoma: A Prospective Cohort Study (Abstract No.: PS1902) The Orelabrutinib plus Obinutuzumab regimen had well rationality and demonstrated promising efficacy. Across the entire study and during the period of induction therapy, the best objective response rate (ORR) was 100%. 3 patients who achieved partial response (PR) after the induction therapy subsequently achieved complete response (CR) in the maintenance period. The 18-month progression-free survival (PFS) and overall survival (OS) rates were both 100%. 4. Ultra-low Dose Radiotherapy Combined with Orelabrutinib Improves the Complete Response Rate of Treatment for Localized Ocular Adnexal Extranodal Marginal Zone B-cell Lymphoma (Abstract No.: PS1901) The combination of ultra-low dose radiotherapy and orelabrutinib in the treatment of ocular adnexal extranodal marginal zone lymphoma (OA-EMZL) not only improves the effectiveness of treatment but also significantly reduces the toxic effects of radiotherapy, providing a new approach for the treatment of localized OA-EMZL. Of all the 17 patients who completed treatment, 16 patients achieved complete response (CR) and one patient achieved a partial response. Additionally, the lesions in the patients still undergoing treatment have shrunk compared to before treatment. 5. Orelabrutinib plus Bendamustine-Rituximab (OBR) versus Bendamustine-Rituximab (BR) in Transplant-ineligible, Intermediate- to High-risk Mantle Cell Lymphoma (MCL) (Abstract No.: PS1969) This open-label, randomized, multi-center study aims to compare the efficacy and safety of Orelabrutinib plus Bendamustine-Rituximab versus Bendamustine-Rituximab in transplant-ineligible, intermediate- to high-risk mantle cell lymphoma (MCL) patients. Early data suggest that Orelabrutinib plus Bendamustine-Rituximab could reduce disease progression events compared to Bendamustine-Rituximab in high-risk MCL. Updated outcomes on efficacy, safety, and biomarker analysis will be reported. 6. Pomalidomide, Rituximab, Orelabrutinib, and MiniCHOP-like (PRO-miniCHOP) in Elderly Patients with Newly Diagnosed Diffuse Large B-cell Lymphoma: Updated Results from a Phase II Study (Abstract No.: PF950) The results further support Pomalidomide, Rituximab, Orelabrutinib, and MiniCHOP-like (PRO-miniCHOP) as a potential treatment option for elderly patients with diffuse large B-cell lymphoma (DLBCL), demonstrating promising efficacy and acceptable safety, especially for those who responded to the Pomalidomide-Rituximab-Orelabrutinib (PRO) induction therapy. A total of 32 patients were enrolled in this study, of whom 26 patients completed ≥3 cycles of the PRO-miniCHOP, resulting in a complete response rate (CRR) of 65.4% and overall response rate (ORR) of 100.0%. Among the 21 patients who completed the full 6-cycle therapy with PRO-miniCHOP, both the CRR and ORR were 95.2%. At a median follow-up of 15.6 months, the median progression-free survival (PFS) and overall survival (OS) had not yet been reached, with the 2-year PFS and OS rates being 94.7% and 100.0%, respectively. 7. Orelabrutinib Addition to R-CHOP-like Regimen Adapted to Response in Treatment-Naïve Non-GCB DLBCL: Update Results of Orient Study (Abstract No.: PS1943) In non-germinal center B-cell-like (non-GCB) diffuse large B-cell lymphoma (DLBCL) patients who responded to orelabrutinib plus rituximab (OR) induction, orelabrutinib plus R-CHOP-like (OR-CHOP) exhibited favorable antitumor activity and manageable safety. Update results further support the orelabrutinib plus R-CHOP-like therapy as an option in this disease. At end of 6-cycle orelabrutinib plus R-CHOP-like, response (all 100%) was independent of double-expressing lymphoma (DEL), extranodal involvement (EI), and Lymphgen subtypes. No off-target-related cardiac toxicities occurred. 8. Primary Efficacy and Safety of First-line R-MTO Regimen (Rituximab, Methotrexate, Thiotepa, and Orelabrutinib) followed by Autologous Hematopoietic Stem Cell Transplantation in PCNSL (Abstract No.: PF966) The Rituximab, Methotrexate, Thiotepa, and Orelabrutinib (R-MTO) induction treatment has demonstrated notable efficacy in achieving higher response rate among patients with newly diagnosed primary central nervous system lymphoma (PCNSL), with a manageable safety profile. At the data cutoff, all patients had completed four cycles of induction therapy, and the complete response (CR) rate and overall response rate (ORR) were 93.34% and 96.67%, respectively. The 12-month progression-free survival (PFS) and overall survival (OS) rates were 82.26% and 85.33%, respectively. 9. Orelabrutinib, Rituximab Combined with High-Dose Methotrexate as Induction Therapy in Newly Diagnosed Primary Central Nervous System Lymphoma (Abstract No.: PS1917) This study demonstrated the promising efficacy of the Orelabrutinib, Rituximab Combined with High-Dose Methotrexate induction regimen in newly diagnosed primary central nervous system lymphoma (PCNSL), with encouraging response rates and durable progression-free survival (PFS). The regimen also showed promising overall survival (OS) trends, highlighting its potential as an effective treatment. Treatment-related adverse events (TRAEs) were manageable, and no severe BTK inhibitor-related off-target toxicities (e.g., atrial fibrillation/flutter) were observed. These findings suggest that the Orelabrutinib, Rituximab Combined with High-Dose Methotrexate regimen is a well-tolerated and effective therapeutic option for PCNSL. Except the above poster presentations, more than 10 studies were also selected as poster presentations or online publications at the meeting. For more detailed clinical data, please refer to EHA website. About InnoCare InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancers and autoimmune diseases with unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, Hong Kong, and the United States. InnoCare Forward-looking Statements This report contains the disclosure of some forward-looking statements. Except for statements of facts, all other statements can be regarded as forward-looking statements, that is, about our or our management's intentions, plans, beliefs, or expectations that will or may occur in the future. Such statements are assumptions and estimates made by our management based on its experience and knowledge of historical trends, current conditions, expected future development and other related factors. This forward-looking statement does not guarantee future performance, and actual results, development and business decisions may not match the expectations of the forward-looking statement. Our forward-looking statements are also subject to a large number of risks and uncertainties, which may affect our short-term and long-term performance.
Business Upturn
2 days ago
- Business
- Business Upturn
Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June
Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy. Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia. CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in a poster session on Saturday, June 14th 6:30 – 7:30 p.m. CEST (12:30 – 1:30 p.m. EDT) at the European Hematology Association (EHA) 2025 Congress in Milan, Italy. In this study, the Company's proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSCs in NHPs. Latest data from this ongoing NHP study showed that at five months a single intravenous administration of Editas' tLNP resulted in mean on-target editing levels in the HBG1/2 promoter region of 58% in HSCs: well exceeding the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, the biodistribution data in NHPs with Editas' tLNP continue to show significant de-targeting of the liver in contrast to standard LNPs. 'These data from our in vivo HSC program confirm our ability to achieve high efficiency delivery, therapeutically relevant editing levels and favorable biodistribution in NHPs. These data validate the further development of Editas' proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia,' said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. Editas Medicine's in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in HbF and total hemoglobin (Hb) in clinical trials. The presentation details are listed below. Abstracts can be accessed on the EHA website , and the presentation will be posted on the Editas Medicine website during the conference. Poster Presentation Details: Title: Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for β-hemoglobinopathies Date/Time: Saturday, June 14, 2025, 6:30 – 7:30 p.m. CEST/ 12:30 – 1:30 p.m. EDT Location: Allianz MiCo, Milano Convention Centre Session: Poster Session 2 About Editas Medicine As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute's Cas12a patent estate and Broad Institute and Harvard University's Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit . Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
Yahoo
06-06-2025
- Health
- Yahoo
Rectify Pharma Presents Translational Data Demonstrating Improvements in Kidney Function and Reductions in Vascular Calcification at ERA 2025
Potent, selective ABCC6-targeting positive functional modulator (PFM) improved multiple markers of renal function in animal models of chronic kidney disease (CKD) PFM improved inorganic pyrophosphate (PPi) efflux via on-target engagement of the ABCC6 membrane transporter and reduced vascular calcification in animal models of CKD BOSTON, June 06, 2025 (GLOBE NEWSWIRE) -- Rectify Pharmaceuticals, Inc., ('Rectify') a biotechnology company pioneering positive functional modulators (PFMs) that restore and enhance membrane protein function, today showcased new data from its cardiorenal program with its ABCC6-targeting PFM, RTY-822. The results were presented in an oral presentation during the European Renal Association (ERA) 2025 Congress, taking place in Vienna, Austria, June 4 – 7, 2025. 'These translational results demonstrate that Rectify's PFM reduces vascular calcification and kidney injury and improves kidney function through enhancement of ABCC6 function,' said Rajesh Devraj, Ph.D., President and Chief Executive Officer of Rectify. 'We believe the strength of these data underscores the potential of our approach to offer a novel and differentiated therapeutic strategy to treat renal dysfunction and injury, as well as address vascular calcification driving cardiovascular disease. We are committed to continuing to advance meaningful therapies for patients with CKD and serious cardiovascular conditions.' Robert Hughes, Ph.D., Chief Scientific Officer of Rectify, added, 'Enhancing the function of ABCC6 holds the potential to address key drivers of vascular calcification and tubular dysfunction seen in CKD. With demonstrated on-target engagement, RTY-822 shows significant promise to reduce CKD-associated calcium deposits in arterial walls, a well-established contributor to cardiovascular morbidity and mortality seen in patients with CKD, and remarkably, also improves glomerular function and tubular injury. The translational readiness of this program makes it a compelling de-risked asset with the potential to address CKD and multiple cardiovascular indications.' Title: A Positive Functional Modulator of ABCC6 Decreases Vascular Calcification and Improves Kidney Function in a Rat Adenine Diet Model of Chronic Kidney DiseaseAbstract Number: 1723 Presenter: John Miller, Ph.D. Session: Chronic Kidney Disease, Focused Oral Room 4Date and Time: Friday, June 6, 2025, 8:51 a.m. – 8:57 a.m. CEST In CKD, reduced ABCC6 activity or expression may lead to lower PPi levels, contributing to pathological calcification in blood vessels and kidneys. Restoring ABCC6 function or enhancing its expression offers a new and compelling therapeutic strategy to prevent the progression of CKD towards end-stage renal disease in addition to reducing large vessel calcification, a major driver of cardiovascular complications in CKD. Key findings RTY-822, an orally administered PFM targeting ABCC6 increased both ABCC6 protein expression and plasma PPi levels in primary human hepatocytes, supporting its proposed mechanism of action In a rat model of CKD and vascular calcification induced by an adenine-enriched diet with calcitriol supplementation: RTY-822 treatment led to statistically significant reductions in vascular calcification in the aorta and the femoral arteries After a six-week treatment course, RTY-822 improved glomerular and tubular kidney functions, with statistically significant reductions in serum creatinine, urea, and cystatin C, and an improved estimated glomerular filtration rate (eGFR) Calcium accumulation in the kidney was also significantly reduced Kidney injury biomarkers were reduced, including a significant decrease in plasma KIM-1 and urinary Lipocalin-2 (NGAL) levels In rats fed an adenine-rich diet without calcitriol supplementation, treatment with RTY-822 resulted in: Improved glomerular and tubular kidney functions Reduced serum creatinine and cystatin C levels Increased eGFR Reduced urinary NGAL levels, a biomarker of acute kidney injury The presentation is available on the Rectify website at About Rectify Pharmaceuticals, Inc. ('Rectify') Rectify is advancing Positive Functional Modulators (PFMs), a novel class of oral, small molecules that restore and enhance membrane protein function to address the underlying cause of serious diseases. Rectify's PFMs have potential to modulate the activity of wild-type and mutated membrane-bound proteins, a historically difficult challenge with a small molecule approach. The Company's breakthrough product platform enables efficient and rapid discovery of first- and best-in-class small molecule therapies with the potential to address membrane protein dysfunction for treatment of rare and common diseases, including liver, cardio-renal-metabolic, and neurodegenerative diseases. Rectify was founded and seeded by Atlas Venture who co-led the $100M Series A round with Omega Funds and were joined by Forbion and Longwood Fund. For more information, please visit or follow us on X and LinkedIn. ContactMediaJonathan PappasLifeSci Communicationsjpappas@ 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
