Thrombocytopenia

Patritumab Deruxtecan Granted Priority Review in the U.S. for Certain Patients with Previously Treated Locally Advanced or Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

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星期五, 十二月 22, 2023
Biotechnology, Pharmaceutical, Health, Oncology, MSD, Senior, Food, Leukopenia, Priority review, Hypokalemia, Daiichi Sankyo, FDA, Breakthrough therapy, PDUFA, EGFR, ILD, Anemia, Fatigue, HER3, Neutropenia, MD, Biologics license application, Recurrence, Patritumab, Weakness, Thrombocytopenia, Merck & Co., DXD, Patient, Safety, Medicine, MRK, ADC, NSCLC, BLA, Pharmaceutical industry

The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is June 26, 2024.

Key Points: 
  • The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is June 26, 2024.
  • The Priority Review follows receipt of Breakthrough Therapy Designation granted by the FDA in December 2021.
  • RTOR allows the FDA to review components of an application before submission of the complete application.
  • Twelve patients (5.3%) had confirmed treatment-related interstitial lung disease (ILD) as determined by an independent adjudication committee.

Ivy Brain Tumor Center Announces Promising Data for Niraparib in Phase 0/2 Glioblastoma Clinical Trial

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星期五, 十一月 17, 2023
Temozolomide, Glomus tumor, Barrow Neurological Institute, Brain, Safety, Annual general meeting, PST, Progression-free survival, Glioblastoma, Clinical trial, Patient, Thrombocytopenia, PFS, Radiation, Pakistan Society of Neuro-Oncology, GSK, PARP, Standard of care, MGMT, Niraparib, Survival, Society, SNO, HIV disease progression rates, Hedera, Pharmaceutical industry, Medical imaging, Oncology

Phoenix, AZ, Nov. 17, 2023 (GLOBE NEWSWIRE) -- The Ivy Brain Tumor Center at Barrow Neurological Institute announces promising results from a Phase 0/2 trial of niraparib in patients with newly diagnosed glioblastoma with unmethylated MGMT.

Key Points: 
  • Phoenix, AZ, Nov. 17, 2023 (GLOBE NEWSWIRE) -- The Ivy Brain Tumor Center at Barrow Neurological Institute announces promising results from a Phase 0/2 trial of niraparib in patients with newly diagnosed glioblastoma with unmethylated MGMT.
  • Glioblastoma is an incurable malignant brain tumor and MGMT-unmethylated glioblastoma is the most common and fastest-recurring variant, comprising more than 60 percent of all glioblastoma tumors.
  • In the Phase 0 component of the study, patients exposed to niraparib demonstrated high drug concentrations in brain tumor tissue.
  • These presentations represent a broader portfolio of pharmacodynamic and pharmacokinetic-driven clinical trials, utilizing industry-supported drug development to test new-in-class therapies for brain tumor patients.

Humacyte Announces Two Presentations at the VEITHsymposium® of Positive Clinical Results of the Human Acellular Vessel™ (HAV™) in the Treatment of Vascular Trauma

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星期五, 十一月 17, 2023
Sale, Efficacy, Food, Extremities, BLA, Ernest, Denver Health Medical Center, Risk, Surgeon, Clinical trial, Meta-analysis, Amputation, Constipation, Peripheral artery disease, HAV, Thrombocytopenia, Research, Death, FDA, FACS, Army, HUMA, Standard of care, Infection, File, University, Tachycardia, Vascular surgery, Doctor of Philosophy, Anemia, Evaluation, Common, Biologics license application, Saphenous, Injury, Trial of the century, Pharmaceutical industry, Medical imaging, Medical device, Fever, MD, Patient, Safety

DURHAM, N.C., Nov. 17, 2023 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, today announced the presentation of positive V005 Phase 2/3 clinical trial results of the investigational Human Acellular Vessel (HAV) in the treatment of vascular trauma, as well as results from the treatment of wartime injuries in Ukraine. The two presentations were made at the VEITHsymposium, a major vascular surgery conference in New York City. Results showed the HAV had higher rates of patency (blood flow), and lower rates of amputation and infection, compared to historic synthetic graft benchmarks. Humacyte plans to file a Biologics License Application (BLA) for the HAV for an indication in vascular trauma patients with the U.S. Food and Drug Administration (FDA) during the current quarter.

Key Points: 
  • The two presentations were made at the VEITHsymposium, a major vascular surgery conference in New York City.
  • Results showed the HAV had higher rates of patency (blood flow), and lower rates of amputation and infection, compared to historic synthetic graft benchmarks.
  • Fox, MD, FACS, Director of Vascular Surgery at the University of Maryland Capital Region, a clinical investigator in the V005 trial.
  • Common adverse events reported were anemia, vascular graft thrombosis, blood loss anemia, pyrexia, thrombocytopenia, constipation, nausea peripheral edema, and tachycardia.

NextCure Publishes Non-Clinical Data Defining the Mechanism of Action for NC525, a Novel LAIR-1 Antibody for AML

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星期三, 十一月 15, 2023
Safety, Health, Acute myeloid leukemia, Patient, Thrombocytopenia, Journal, SOC, Neutropenia, Cell death, Cancer, Survival, AML, Stem cell, Manuscript, Pharmaceutical industry

The data demonstrate that NC525 induces cell death in acute myeloid leukemia (AML) blast cells and leukemic stem cells (LSCs) through leukocyte-associated immunoglobin-like receptor-1 (LAIR-1) agonism by driving a unique apoptotic signaling pathway.

Key Points: 
  • The data demonstrate that NC525 induces cell death in acute myeloid leukemia (AML) blast cells and leukemic stem cells (LSCs) through leukocyte-associated immunoglobin-like receptor-1 (LAIR-1) agonism by driving a unique apoptotic signaling pathway.
  • NC525 is a humanized monoclonal antibody (mAb) that specifically binds to LAIR-1 and kills LSCs, while sparing healthy hematopoietic stem cells (HSCs).
  • The publication details a novel mechanism for the potential treatment of AML, as the expression level of the LAIR-1 receptor on leukemic cells acts as a key regulator.
  • Thus, NC525 holds great promise as an important and novel treatment for patients with resistant and refractory AML (R/R AML).

New Analyses Presented at ASH 2023 Support the Potential Long-Term Response and Safety of Kite’s Tecartus® in Patients With Aggressive Blood Cancers

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星期二, 十二月 12, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Hospitals, Clinical Trials, Hackensack University Medical Center, Safety, Cytokine release syndrome, Senior, City, ASH, Aes, CRS, Haematopoietic system, Sepsis, Patient, Abstract, Thrombocytopenia, Blinatumomab, The James Cancer Hospital, Division, SCT, Acute lymphoblastic leukemia, Neutropenia, ORR, Encephalopathy, B-ALL, Infection, Survival, Death, CD20, Doctor of Philosophy, Society, Abstract (summary), OS, Confidence interval, TP53, LD, Cytopenia, Pharmaceutical industry, Nursing, Hematology, Brexucabtagene autoleucel, Lymphoma, MD

“The clinical results and real-world evidence presented at ASH clearly support the potential for long-term response and safety of Tecartus in aggressive blood cancers for which patients have limited treatment options,” said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite.

Key Points: 
  • “The clinical results and real-world evidence presented at ASH clearly support the potential for long-term response and safety of Tecartus in aggressive blood cancers for which patients have limited treatment options,” said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite.
  • The median OS for patients with complete response (CR) (n=46) was 58.7 months.
  • Efficacy and safety outcomes for 23 patients with R/R MCL enrolled in ZUMA-18, a multicenter, open-label, expanded-access study of Tecartus, were also presented.
  • An analysis of a CIBMTR observational database of R/R MCL patients receiving Tecartus from 84 U.S. centers was presented.

Geron Announces IMerge Phase 3 Presentations at ASH Highlighting Significant Durability of Transfusion Independence and Breadth of Effect Across MDS Subgroups with Imetelstat in Lower Risk MDS

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星期一, 十二月 11, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Yale Cancer Center, Associate, International Prognostic Scoring System, Quality, IPSS, VAF, ASH, Acute myeloid leukemia, Cytopenia, Risk, Pros, Corporation, Yale school, Fatigue, Patient, Thrombocytopenia, Research, Periodic breathing, Imetelstat, Quality of life, Internal medicine, MDS, Neutropenia, MBBS, Yale School of Medicine, Bleeding, Survival, Pain, AML, Geron Corporation, Society, Iron overload, Mutation, RBC, Pharmaceutical industry, Dietary supplement, Lancet, Geron, SF3B1, Blood, Medicine, Hematology

The results showed that imetelstat consistently had higher RBC-TI response rates than placebo across these different risk subgroups.

Key Points: 
  • The results showed that imetelstat consistently had higher RBC-TI response rates than placebo across these different risk subgroups.
  • Overall, durable 24-week and 1-year RBC-TI responses were observed with imetelstat in all lower- and higher-risk subgroups.
  • This analysis suggests clinical benefit of imetelstat across different molecularly defined subgroups and independent of the underlying molecular mutation pattern.
  • This analysis indicates that achievement of RBC-TI was associated with improved survival, suggesting that transfusion dependence is a modifiable predictor of clinical outcomes in lower risk MDS.

Bristol Myers Squibb Announces Data at ASH 2023 from Diverse Multiple Myeloma Pipeline, Underscoring Range of Tailored Treatment Approaches to Address Unique Patient Needs

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星期二, 十二月 12, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Dexamethasone, ASH, CRS, Patient, Cell, Infection, Bortezomib, B-cell maturation antigen, GPRC5D, Carfilzomib, Therapy, Bristol Myers Squibb, ORR, Cancer, Diagnosis, Anemia, International Myeloma Foundation, CD3, Cytokine release syndrome, Haematopoietic system, Multiple myeloma, Immunomodulatory imide drug, Safety, Immune system, Incidence, Aes, DOR, Science, Hook effect, Public, Drug discovery, Society, Estate (law), Lenalidomide, Proteasome inhibitor, Thrombocytopenia, PFS, BMY, Daratumumab, Neutropenia, Partnership, Skin, Life, Mezi proudy, Research, NYSE, Disease, MRD, Translational Genomics Research Institute, TCE, Survival, BMS, BCMA, Biology, Pharmaceutical industry, Vaccine, Fine chemical, Cohort, RRMM

Bristol Myers Squibb (NYSE: BMY) today announced updated results from three key programs within its broad multiple myeloma research pipeline, highlighting its diverse targets and molecular approaches to address unique patient needs within the disease.

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) today announced updated results from three key programs within its broad multiple myeloma research pipeline, highlighting its diverse targets and molecular approaches to address unique patient needs within the disease.
  • These data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, California.
  • While multiple myeloma remains a relentless disease, we continue to transform the multiple myeloma treatment paradigm by dramatically improving outcomes for every patient.
  • As a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

Two Early Studies Evaluating Potential First-in-Class CELMoD™ Agent Golcadomide for the Treatment of Non-Hodgkin Lymphomas Presented at ASH 2023

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星期二, 十二月 12, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Bristol Myers Squibb, Safety, NYSE, Non-Hodgkin lymphoma, Pneumonia, ASH, Ikaros, Rituximab, Patient, Cell, Thrombocytopenia, BMY, Lymphoma, Death, CMR, DL, Neutropenia, ORR, Anemia, Society, NK, Febrile neutropenia, Pharmaceutical industry, Fever, DL-1, Hematology

Bristol Myers Squibb (NYSE: BMY) announced the results of two early studies evaluating combinations of potential first-in-class CELMoD™ agent golcadomide in non-Hodgkin lymphomas.

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) announced the results of two early studies evaluating combinations of potential first-in-class CELMoD™ agent golcadomide in non-Hodgkin lymphomas.
  • These data are being presented in separate posters ( #4459 , #4496 , #1631 ) at the 2023 American Society of Hematology (ASH) Annual Meeting from December 9-12.
  • “In the studies being presented at ASH 2023, golcadomide has shown potential warranting further evaluation in patients with first-line and previously treated large B-cell lymphomas.
  • There were 4 deaths on treatment during the study with one considered related to the study treatment.

MorphoSys’ Pelabresib Improves All Four Hallmarks of Myelofibrosis in Phase 3 MANIFEST-2 Study

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星期一, 十二月 11, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Cough, FACP, Spleen, PST, Food, European Medicines Agency, JAK, GMT, ASH, Headache, Plasma, Disease, Constipation, IL-6, Fatigue, MorphoSys, Patient, Thrombocytopenia, CET, Periodic breathing, Weakness, COVID-19, Prognosis, Diarrhea, Nausea, Survival, EST, Dysgeusia, TNF, Anemia, Fibrosis, Ruxolitinib, BET, Bone marrow, Dizziness, Vaccine, Dietary supplement, Medical imaging

Average hemoglobin levels were greater in patients receiving pelabresib and ruxolitinib than in those receiving placebo plus ruxolitinib, starting at week 9 and continuing to week 24.

Key Points: 
  • Average hemoglobin levels were greater in patients receiving pelabresib and ruxolitinib than in those receiving placebo plus ruxolitinib, starting at week 9 and continuing to week 24.
  • Increased cytokine levels are associated with all four disease hallmarks; increased IL-8 levels are also associated with worse survival outcomes.
  • Discontinuation rates due to adverse events were 10.7% with pelabresib and ruxolitinib and 6.5% with placebo plus ruxolitinib.
  • “The four hallmarks of myelofibrosis – enlarged spleen, anemia, bone marrow fibrosis and disease-associated symptoms – have a strong impact on a patient’s life.

bluebird bio Announces FDA Approval of LYFGENIA™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

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星期五, 十二月 8, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Red blood cell, Sickle cell disease, Patient, Anemia, Quality of life, Bangladesh Technical Education Board, Qualification, Webcast, Priority review, Lung, Vasculitis, Food, Incidence, Pain, Priapism, Acute myeloid leukemia, Bluebird, PIN, Genetic disorder, Regenerative medicine, Division, Ronas Voe, DSM-IV codes, Hematology, Vaso-occlusive crisis, Courage, Adolescence, Hemolytic anemia, Stomatitis, Cellular, Febrile neutropenia, History, Leukopenia, Thrombocytopenia, Neutropenia, University, QTC, Life, NHLBI, Bone marrow, Research, HBA, Bluebird bio, Telephone, Disease, Acute chest syndrome, Caregiver, NIH, MD, Blood transfusion, Pharmaceutical industry, Vaccine, Online shopping

LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Key Points: 
  • LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
  • “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events.
  • The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
  • A replay of the webcast will be available on the bluebird website for 90 days following the event.