Iron overload

Agios Presents Positive Results from Phase 3 ENERGIZE Study of Mitapivat in Non-Transfusion-Dependent Thalassemia in Plenary Session at the European Hematology Association 2024 Hybrid Congress

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星期六, 六月 15, 2024
Head, American University, Iron overload, Patient, Fatigue, Agios Pharmaceuticals, Thalassemia, NTD, Disease, University of Toronto, AGIO, Congress, Hemolysis, University, Master of Science, S104, Pyruvate kinase, Central European, Anemia, American University of Beirut, Arm, Quality of life, BID, FRCPC, Medical device

CAMBRIDGE, Mass., June 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today presented detailed results from the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia in a plenary session (abstract #S104) at the European Hematology Association 2024 (EHA2024) Hybrid Congress, which is being held June 13-16, 2024, in Madrid, Spain. In a related poster presentation (abstract #P1529), the company presented additional data from the ENERGIZE study highlighting clinically meaningful improvements in health-related quality of life measures and patient-reported outcomes among patients in the mitapivat arm compared to those in the placebo arm.

Key Points: 
  • The ENERGIZE study achieved its primary endpoint, with mitapivat demonstrating a statistically significant increase in hemoglobin response rate compared to placebo.
  • 122 (93.8%) in the mitapivat arm and 62 (96.9%) in the placebo arm completed the 24-week double-blind period of the study.
  • Baseline demographics and characteristics were balanced between mitapivat and placebo arms, and representative of a population of non-transfusion dependent thalassemia patients.
  • Agios will host a virtual investor breakout session tomorrow, June 16, 2024, at 10:00 a.m.

Agios Announces Phase 3 ENERGIZE-T Study of Mitapivat Met Primary Endpoint and All Key Secondary Endpoints in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

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星期一, 六月 3, 2024
Aes, Health, Gulf Cooperation Council, RBC, Patient, GCC, Mitapivat, Safety, Internal medicine, First, Host, TRR, Marketing, Conditional sentence, Incidence, Pyruvate kinase, Head, University of Milan, Red blood cell, Part, BID, Partnership, Agios Pharmaceuticals, University, Iron overload, AGIO, Pharmaceutical industry

Statistical significance was also achieved for all key secondary endpoints evaluating additional measures of reduction of transfusion burden compared to placebo.

Key Points: 
  • Statistical significance was also achieved for all key secondary endpoints evaluating additional measures of reduction of transfusion burden compared to placebo.
  • Treatment with mitapivat demonstrated a statistically significant reduction in transfusion burden compared to placebo, as measured by the TRR endpoint.
  • Agios plans to present a more detailed analysis of the Phase 3 ENERGIZE-T data at an upcoming medical meeting.
  • The event can be accessed under “Events & Presentations” in the Investors and Media section of the company's website at www.agios.com .

Agios Pharmaceuticals to Present Clinical and Translational Data in Rare Blood Disorders at European Hematology Association 2024 Hybrid Congress

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星期二, 五月 14, 2024
Sickle cell disease, Grady Memorial Hospital, EHA, Harvard Medical School, Hospital, Central European, Hereditary spherocytosis, Winship Cancer Institute, Abstract, University Medical Center, MMSc, All but dissertation, AGIO, American University of Beirut, Launch, Disease, Anemia, Thalassemia, Hematology, Utrecht University, University, Hydroxycarbamide, University Medical Center Utrecht, Oncology, Iron overload, American University, Pharmaceutical industry

CAMBRIDGE, Mass., May 14, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that data from its programs will be presented at the European Hematology Association 2024 (EHA2024) Hybrid Congress, to be held June 13-16, 2024, in Madrid, Spain.

Key Points: 
  • Additional presentations will highlight data across Agios’ pipeline in rare blood disorders, including sickle cell disease and pyruvate kinase deficiency.
  • In total, seven abstracts led by Agios and external collaborators will be presented or published.
  • The accepted abstracts are listed below and are available online on the EHA congress website at www.ehaweb.org.
  • Data from the global Phase 3 ENERGIZE study of mitapivat in alpha- or beta- non-transfusion-dependent thalassemia.

Rallybio Reports First Quarter 2024 Financial Results and Provides Business Updates

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星期四, 五月 9, 2024
Professional Services, Health, Finance, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Birth, Administration, DSM-IV codes, Corporation, Pregnancy, Risk, Safety, Pharmacokinetics, Publication, Iron overload, Johnson & Johnson, Gestational age, Data, Research and development, Patient, Marker beacon, Caregiver, Workforce, G&A, Pharmaceutical industry

Rallybio is on track to initiate a Phase 2 trial in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT in the second half of 2024.

Key Points: 
  • Rallybio is on track to initiate a Phase 2 trial in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT in the second half of 2024.
  • Rallybio expects to provide an update on this analysis and future development plans for RLYB116 in the second half of 2024.
  • Net Loss and Net Loss Per Common Share: Rallybio reported a net loss of $19.0 million, or $0.47 per common share, for the first quarter of 2024.
  • This compares to a net loss of $17.3 million, or $0.43 per common share, for the first quarter of 2023.

Inflammasome Therapeutics Announces First Patient Dosed in Geographic Atrophy (GA) Clinical Trial

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星期一, 四月 29, 2024
Health, Other Science, Clinical Trials, Research, Science, Optical, Biotechnology, AMD, Brain, University, Iron, Cell, Risk, Publication, Iron overload, Central nervous system, Inflammasome, K8, Disease, Nervous system, Patient, Eye, Multiple sclerosis, DSM-IV codes, ALS, Multimedia, Atrophy, Reactive oxygen species, Therapy, Ophthalmology, Science and nature, Pharmaceutical industry

Inflammasome Therapeutics ( https://www.inflam.com ), a private company developing a new class of inflammasome inhibitor drugs, Kamuvudines, as therapies for prevalent, degenerative diseases, announced the first patient has been dosed in a first-in-class clinical trial for dry AMD.

Key Points: 
  • Inflammasome Therapeutics ( https://www.inflam.com ), a private company developing a new class of inflammasome inhibitor drugs, Kamuvudines, as therapies for prevalent, degenerative diseases, announced the first patient has been dosed in a first-in-class clinical trial for dry AMD.
  • “This marks the second trial underway with our Kamuvudines in ophthalmology,” said Dr. Paul Ashton, President and CEO of Inflammasome Therapeutics, developers of both the compounds and delivery systems for administration.
  • There are 38 other interventional clinical trials for GA registered in clinicaltrials.gov., almost all of which target individual toxic substances, but not the underlying cause of the atrophy, inflammasome activation.
  • Inflammasome Therapeutics is expected to begin clinical trials in some of these diseases soon as well,” he affirmed.

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

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星期三, 一月 17, 2024
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

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星期三, 一月 17, 2024
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Bond Biosciences Announces Successful Pre-IND Meeting with the FDA to Advance the Development of BBI-001 for Treatment of Iron Overload Associated with Hereditary Hemochromatosis

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星期四, 一月 11, 2024
Research, General Health, Pharmaceutical, Biometrics, Clinical Trials, Science, Cardiology, Biotechnology, FDA, Other Science, Health, Diabetes, Nephrology, CMC, Patient, MD, HH, Bond, Chemistry, Division, IND, Food, Safety, Iron overload, Doctor of Philosophy, Hereditary haemochromatosis, Clinical trial, GI, Pharmaceutical industry

BBI-001 is a novel investigational new drug.

Key Points: 
  • BBI-001 is a novel investigational new drug.
  • It is a non-absorbed, oral therapeutic designed to rapidly form a strong and selective iron chelate in the digestive tract, thus inhibiting iron absorption in patients with symptomatic iron overload due to HH.
  • The structure and properties of BBI-001 have been designed to restrict it to the GI tract to inhibit systemic expose.
  • “FDA was very thoughtful in reviewing the Bond briefing package and replying in depth on the proposed non-clinical and clinical development program options, to facilitate the next Phase of clinical development” observed Curtis L. Scribner, MD, Bond’s Chief Medical Officer.

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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星期一, 十二月 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.

Alterity Therapeutics Announces Presentation of New Data Demonstrating Novel Mechanisms of ATH434

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星期四, 十一月 16, 2023
SUNY, Society, Buffalo, SAN, Therapy, State university system, Disease, ATH, Biochemistry, ASX, Oxidative stress, Political moderate, Cassava Sciences, Iron overload, Pharmaceutical industry, Alterity

The poster entitled, “Potent Antioxidant and Mitochondrial-protectant Effects of ATH434, a Novel Inhibitor of α-Synuclein Aggregation with Moderate Iron-binding Affinity,” presents new data indicating that ATH434 can preserve mitochondrial function after oxidative injury and exert direct anti-oxidant activity independent of its iron binding properties.

Key Points: 
  • The poster entitled, “Potent Antioxidant and Mitochondrial-protectant Effects of ATH434, a Novel Inhibitor of α-Synuclein Aggregation with Moderate Iron-binding Affinity,” presents new data indicating that ATH434 can preserve mitochondrial function after oxidative injury and exert direct anti-oxidant activity independent of its iron binding properties.
  • These features were not observed with another iron binding agent approved for treating iron overload that was also investigated.
  • David Stamler, M.D., Chief Executive Officer of Alterity, commented, “These exciting new data underscore the potential of ATH434 as a treatment for neurodegenerative diseases, including Parkinson’s disease and related disorders.
  • We have long known that ATH434 is able to reduce labile iron which, when elevated, can drive oxidative stress.