Erythropoiesis

China Medical System: New Drug Application of Desidustat Tablets Accepted in China

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火曜日, 4月 23, 2024
CMS, Tablet, Desidustat, NMPA, NDA, National Medical Products Administration, Kidney failure, New Drug Application, Erythropoiesis, Anemia, Chronic kidney disease, CKD, Week, Patient, Dialysis, Prevalence, Marketing, Diagnosis, Advertising, EPO, Medical device

Desidustat Tablets are novel, oral HIF-PHI for treating anaemia in non-dialysis adult chronic kidney disease patients.

Key Points: 
  • Desidustat Tablets are novel, oral HIF-PHI for treating anaemia in non-dialysis adult chronic kidney disease patients.
  • The primary endpoint has indicated that Desidustat is more effective than placebo in increasing Hb level.
  • China Medical System Holdings Limited (“CMS” or the “Group”) is pleased to announce that on 22 April 2024, the New Drug Application (NDA) of Desidustat Tablets  (“Desidustat Tablets” or the “Product”) has been accepted by the National Medical Products Administration of China (NMPA).
  • Any treatment-related decisions made by healthcare professionals should be based on the patient’s specific circumstances and in accordance with the drug package insert.

Feinstein Institutes Secures $6.1M NIH Grant for Red Cell Disorder Research

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水曜日, 4月 3, 2024
Biotechnology, Health, Science, Pharmaceutical, Research, Diamond, Molecular medicine, Sickle cell disease, Erythropoiesis, Hematology, HMGB1, Cell cycle, Mortality, Doctor of Philosophy, Quality of life, NIH, Diamond–Blackfan anemia, DBA, Credit, Lung, SCD, Medical research, Anemia, Bangladesh Technical Education Board, Multimedia, Conditional sentence, Pharmaceutical industry

To further our understanding and potential treatments of these disorders, The Feinstein Institutes for Medical Research has been awarded a $6.1 million grant from the National Institutes of Health (NIH).

Key Points: 
  • To further our understanding and potential treatments of these disorders, The Feinstein Institutes for Medical Research has been awarded a $6.1 million grant from the National Institutes of Health (NIH).
  • View the full release here: https://www.businesswire.com/news/home/20240403646471/en/
    Dr. Lionel Blanc will lead the new NIH-funded red cell disorders research (Credit: Feinstein Institutes)
    Led by Lionel Blanc, PhD , professor in the Institute of Molecular Medicine at the Feinstein Institutes, the seven-year initiative aims to build off previous research and will shed light on DBA, SCD and anemia.
  • “Understanding red cell blood disorders, how they develop and progress, is essential given their global impact on millions,” said Dr. Blanc.
  • In 2019, he received a $2.5 million grant from the NIH to study treatment for erythropoietic disorders, including DBA.

Keros Therapeutics Presents Clinical Data from its KER-050 Program at the 65th American Society of Hematology Annual Meeting and Exposition

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月曜日, 12月 11, 2023
Patient, Quality of life, Erythropoietin, Protein, Exposition, Fever, Reticulocyte, Homeostasis, Bone marrow, MF, IWG, Haematopoiesis, Research, Erythropoiesis, Diarrhea, Anemia, KROS, Fatigue, Nausea, Headache, RBC, Iron overload, Society, MDS, Therapy, Weakness, Ruxolitinib, Thrombocytopenia, ASH, TI, Week, Periodic breathing, MBBS, Biomarker, Safety, Acute myeloid leukemia, HTB, Cytopenia, Disorder, Pharmaceutical industry

“Additionally, we are encouraged by the preliminary data from the lowest three dose cohorts from our ongoing Phase 2 clinical trial in MF.

Key Points: 
  • “Additionally, we are encouraged by the preliminary data from the lowest three dose cohorts from our ongoing Phase 2 clinical trial in MF.
  • Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these mITT24 patients.
  • All data presented from this trial is as of the data cut-off date.
  • 13 of those 18 patients (72.2%) achieved TI for at least 24 weeks over the first 48 weeks of treatment.

Agios Presents Positive Results from Phase 2 Portion of the RISE UP Pivotal Study in Sickle Cell Disease at 65th ASH Annual Meeting and Exposition

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日曜日, 12月 10, 2023
Momentum, Agios Pharmaceuticals, Erythropoiesis, Sickle cell disease, Agios, Exposition, Hemoglobinopathy, Arm, BID, Trial of the century, University of Texas System, Sickle cell trait, Pain, Female, Headache, Disease, Fatigue, Dysmenorrhea, Nausea, Anemia, Society, Influenza-like illness, Therapy, ASH, Death, Marker, Back pain, University, AGIO, Medicine, Arthralgia

CAMBRIDGE, Mass., Dec. 09, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today presented detailed results from the Phase 2 portion of the global RISE UP study of mitapivat in sickle cell disease in an oral presentation (abstract #271) at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, which is being hosted Dec. 9-12, 2023, in San Diego.

Key Points: 
  • The safety profile for mitapivat observed in the study was generally consistent with previously reported data in other studies of sickle cell disease and other hemolytic anemias.
  • Improvements were observed in annualized rates of sickle cell pain crises, and markers of hemolysis and erythropoiesis for both mitapivat treatment arms compared to placebo.
  • “Currently there are no approved oral therapies that address both sickle cell pain crises and chronic anemia, two of the hallmark symptoms of the disease.
  • Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients With Sickle Cell Disease: RISE UP Phase 2 Results
    Oral Abstract Session: 114.

Disc Medicine Reports Third Quarter 2023 Financial Results and Provides Business Update

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木曜日, 11月 9, 2023
FDA, Research, IRON, Glycine, Erythropoiesis, Food, EPP, PV, Sodium- and chloride-dependent glycine transporter 1, G&A, AURORA, Iron, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Ruxolitinib, Light, Porphyria, Inflammation, Poster, Sunlight, Society, ASH, Development, Photosensitivity, Hematology, Quality of life, Phase 10, Anemia, PPIX, Safety, MF, XLP, Pain, Patient, Administration, Polycythemia, Rare-earth element, Pharmaceutical industry, Vaccine, Disc

Third Quarter 2023 Financial Results:

Key Points: 
  • Third Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $370.5 million as of September 30, 2023, which are expected to fund our operational plans well into 2026.
  • Research and Development Expenses: R&D expenses were $14.4 million for the quarter ended September 30, 2023, as compared to $7.9 million for the quarter ended September 30, 2022.
  • General and Administrative Expenses: G&A expenses were $4.5 million for the quarter ended September 30, 2023, as compared to $2.6 million for the same period in 2022.
  • Net Loss: Net loss was $14.1 million for the quarter ended September 30, 2023, as compared to $16.2 million for the third quarter of 2022.

Ryvu Therapeutics Provides an Update on the Progress of RVU120 Phase I Studies in Patients with Solid Tumors and AML/HR-MDS, and Presents the Updated Development Plan for RVU120

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月曜日, 10月 23, 2023
Medulloblastoma, Bone marrow, Plasma, AUC, Acute myeloid leukemia, Cmax, ESMO, Erythropoiesis, Principal investigator, Aes, PD, Congress, MD, Adenoid cystic carcinoma, CEST, Ruxolitinib, Cell, Data, Syndrome, All-comers track meet, Phase, SD, Risk, Webcast, Pancreatic cancer, Venetoclax, NPM1, WSE, ADCC, Safety, European Society for Medical Oncology, Patient, AML, Sarcoma, Pharmaceutical industry, Medical imaging, Dietary supplement

The total budget for the Phase II clinical development of RVU120 aligns with the estimates initially announced in the Ryvu Development Plans for 2022-2024.

Key Points: 
  • The total budget for the Phase II clinical development of RVU120 aligns with the estimates initially announced in the Ryvu Development Plans for 2022-2024.
  • The presented RVU120 development plan aligns with the budget outlined in Ryvu Development Plans for 2022-2024 and targets multi-billion-dollar market potential.
  • At the ESMO Congress 2023, Ryvu announced updated clinical Phase I data from Phase I/II study of RVU120 in relapsed/refractory metastatic advanced solid tumors.
  • Ryvu will host a webinar today (Monday, October 23) at 9:00 am CEST to discuss further RVU120 development plans.

Disc Medicine Initiates a Phase 1 Study of DISC-3405 (anti-TMPRSS6 mAb) in Healthy Volunteers

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火曜日, 10月 3, 2023
IRON, Pharmacokinetics, Erythropoiesis, Polycythemia, Patient, Fast track (FDA), Iron, TMPRSS6, Safety, Pharmaceutical industry, Disc

DISC-3405 is a monoclonal antibody designed to target TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) to increase hepcidin and decrease iron.

Key Points: 
  • DISC-3405 is a monoclonal antibody designed to target TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) to increase hepcidin and decrease iron.
  • “The initiation of this trial marks the third program that Disc has brought into the clinic and strengthens our position as leaders in the field of hepcidin biology and iron homeostasis.
  • We look forward to pursuing those indications upon the completion of this trial,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
  • Following completion of this study, Disc plans to initiate a trial in polycythemia vera, for which DISC-3405 has received Fast Track Designation.

Disc Medicine Reports Second Quarter 2023 Financial Results and Provides Business Update

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金曜日, 8月 11, 2023
Sodium- and chloride-dependent glycine transporter 1, Ruxolitinib, Chronic kidney disease, European Hematology Association, EPP, AURORA, G&A, Porphyria, Inflammation, MF, Dialysis, Congress, IRON, Development, Erythropoiesis, Glycine, Patient, Administration, Anemia, EHA, Mab, XLP, Research, Polycythemia, Technology transfer, Safety, Pharmaceutical industry, Nursing, Cryptocurrency, Vaccine, Disc

Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023.

Key Points: 
  • Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023.
  • Second Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $377.6 million as of June 30, 2023, which are expected to fund our operational plans well into 2026.
  • Research and Development Expenses: R&D expenses were $12.1 million for the quarter ended June 30, 2023, as compared to $7.7 million for the quarter ended June 30, 2022.
  • Net Loss: Net loss was $15.9 million for the quarter ended June 30, 2023, as compared to $9.6 million for the second quarter of 2022.

Agios Announces Positive Results from Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease with Both Mitapivat Dose Arms Achieving Statistically Significant Hemoglobin Response

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月曜日, 6月 26, 2023
Partnership, Hemolysis, University of Texas System, Aes, AGIO, Comparative oncology, Erythropoiesis, Report, Agios, Patient, Data, BID, University, Anemia, Host, RISE, Sickle cell disease, Agios Pharmaceuticals, Rare, Fatigue, Disease, Dentistry, Pharmaceutical industry, Copper, University of Texas Health Science Center at Houston

CAMBRIDGE, Mass., June 26, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that the Phase 2 portion of the global RISE UP study of mitapivat in sickle cell disease met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms. The safety profile for mitapivat observed in the study was generally consistent with previously reported data in other studies of sickle cell disease and other hemolytic anemias. Improvements were observed in markers of hemolysis and erythropoiesis and annualized rates of sickle cell pain crises at both mitapivat doses compared to placebo. These results support proceeding with the Phase 3 portion of the study.

Key Points: 
  • Improvements were observed in markers of hemolysis and erythropoiesis and annualized rates of sickle cell pain crises at both mitapivat doses compared to placebo.
  • “Sickle cell disease is a complex and debilitating disease characterized by anemia, pain crises, fatigue, cognitive effects and more.
  • Given the promising data for both mitapivat dose arms, the company will continue to analyze the study data over the coming weeks to select a dose for the Phase 3 study.
  • Agios plans to present a full analysis of the RISE UP Phase 2 data at an upcoming medical meeting.

Keros Therapeutics Presents Clinical Trial and Preclinical Study Results from its KER-050 Program and Preclinical Data from its ALK2 Inhibitor Program at the 28th Annual Congress of the European Hematology Association

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金曜日, 6月 9, 2023
NT, Red blood cell, Cohort, Bone marrow, Patient, Iron, Male, Nosebleed, Transferrin, Fatigue, Diet, Myocardial infarction, Diarrhea, TI, HTB, Adenine, Research, Injection site reaction, Hematology, Heart, Chronic kidney disease, Solar cycle 1, COVID-19, Hematologic disease, Transfusion-dependent anemia, IWG, Biomarker, Acute myeloid leukemia, Erythropoiesis, Spleen, Anemia, Iron overload, ALK2, Therapy, Haematopoiesis, RBC, Ruxolitinib, European Hematology Association, LTB, Hematocrit, Part, Inflammation, MDS, Chronic obstructive pulmonary disease, EHA, RS, Nausea, Periodic breathing, Mouse, AI, CKD, KROS, Pharmaceutical industry, Dietary supplement, Medical device, Medical imaging, Silviculture, Residential care, Copper, Palladium, MF

In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.

Key Points: 
  • In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.
  • Of these patients, 37 had completed at least 24 weeks of treatment or discontinued as of the data cut-off date (the “evaluated RP2D patients”).
  • Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these evaluated RP2D patients.
  • By targeting ALK2 inhibition to suppress hepcidin, RKER-216 increased iron availability for erythropoiesis and partially rescued anemia in CKD mice.