Bone marrow

Edgewood Oncology Announces Positive Efficacy Data From Investigator-Sponsored Study of BTX-A51 in Preclinical Models of Liposarcoma

Retrieved on: 
Sunday, April 7, 2024
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Bone marrow, CDK7, MDM2, Apoptosis, BH3, CDK, AML, Dana–Farber Cancer Institute, Patient, LPS, Neoplasm, DNA, Cancer, Liposarcoma, Clinical trial, Azacitidine, CDK9, MCL1, Vaccine

The presentation (Abstract 604), “Targeting casein kinase 1 alpha (CK1alpha) and transcriptional CDKs (CDK7/9) in human liposarcomas,” highlighted findings for BTX-A51 in preclinical human models of LPS.

Key Points: 
  • The presentation (Abstract 604), “Targeting casein kinase 1 alpha (CK1alpha) and transcriptional CDKs (CDK7/9) in human liposarcomas,” highlighted findings for BTX-A51 in preclinical human models of LPS.
  • The data demonstrate that BTX-A51 has preclinical efficacy in treating patient-derived LPS in cell lines and human xenograft models and provides insight into the synergy gained by inhibiting both CK1α and CDK9.
  • “Dedifferentiated liposarcomas (DDLPS) are rare tumors derived from precursors of fat cells which can occur anywhere in the body.
  • Importantly, preliminary in vivo data in an LPS patient-derived xenograft model reveal that BTX-A51 is well-tolerated under conditions that inhibit tumor growth.

BrainStorm Cell Therapeutics Announces Agreement with FDA on a Special Protocol Assessment (SPA) for Phase 3b Trial in ALS

Retrieved on: 
Tuesday, April 9, 2024
Neurology, Neuroprotection, Element, Conference call, Institutional review board, Cerebrospinal fluid, Survival, Disease, ALS, SPA, Bone marrow, CSF, Data monitoring committee, Webcast, Patient, Protocol, Biomarker, Mortality, Microsoft Exchange Server, Stem cell, DMC, Screening, Agency, Safety, CAFS, Brainstorm, Therapy, Marketing, FDA, BLA, Neuroinflammation, Food, Pharmaceutical industry

NEW YORK, April 9, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it received written agreement from the U.S. Food and Drug Administration (FDA), under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn® in amyotrophic lateral sclerosis (ALS). 

Key Points: 
  • (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it received written agreement from the U.S. Food and Drug Administration (FDA), under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn® in amyotrophic lateral sclerosis (ALS).
  • The SPA agreement with the FDA validates the clinical trial protocol and statistical analysis of the planned Phase 3b trial of NurOwn, demonstrating their adequacy for addressing objectives that support a future BLA (Biologics License Application) in ALS.
  • We appreciate the Agency's engagement and guidance during the SPA process and look forward to moving forward with the study."
  • The Phase 3b trial (Study BCT-006-US) will be a two-part, multicenter, study designed to assess the efficacy and safety of NurOwn in patients with ALS.

CARVYKTI® is the First and Only BCMA-Targeted Treatment Approved by the U.S. FDA for Patients with Relapsed or Refractory Multiple Myeloma Who Have Received at Least One Prior Line of Therapy

Retrieved on: 
Saturday, April 6, 2024
Guillain–Barré syndrome, Bone marrow, Medical College of Wisconsin, Encephalopathy, Survival, Immunotherapy, BCMA, Patient, Hypotension, Mortality, B-cell maturation antigen, Edema, Periodic breathing, Associate professor, Dexamethasone, Certification, Neutropenia, Dizziness, Risk, Headache, Chills, Myelodysplastic syndrome, Coagulopathy, DPD, Johnson & Johnson, Immune system, Effect, Fatigue, Thrombocytopenia, Food, Disease, Drive, Appetite, Lymphocytopenia, JNJ, Constipation, Multiple myeloma, Acute myeloid leukemia, CRS, Death, Hypersensitivity, Cytopenia, Cytokine release syndrome, ASCO, Use, Hematology, FDA, PVD, Annual general meeting, T cell, DSM-IV codes, Incidence, Tachycardia, Bortezomib, Diarrhea, Viral, Hope, United, Cough, Daratumumab, Nausea, Society, Division, Physician, Pharmaceutical industry

HORSHAM, Pa., April 5, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today that the U.S. Food and Drug Administration (FDA) has approved CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide.1 With this approval, CARVYKTI® becomes the first and only B-cell maturation antigen (BCMA)-targeted therapy approved for the treatment of patients with multiple myeloma as early as first relapse.

Key Points: 
  • "This milestone underscores our commitment to improve outcomes for patients and transform the treatment of multiple myeloma with CARVYKTI," said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine.
  • CARVYKTI® is a cell therapy that works by harnessing a patient's immune system, or T cells, to fight the disease.
  • Treatment requires extensive training, preparation, and certification to ensure a positive experience for patients.
  • Since initial approval in February 2022, Johnson & Johnson has made significant advances in manufacturing to rapidly scale CARVYKTI® production.

Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin Treatment of cystinosis, 19/02/2021 Positive

Retrieved on: 
Tuesday, April 9, 2024
Community, Diagnosis, Civil service commission, Prevalence, Cell, Bone marrow, Disease, Novartis, Patient, Committee, RNA, Autotransplantation, EMA, IRIS, Cystinosis, Agency, European, COMP, Blood, European Commission, Marketing, Medicine, Pharmaceutical industry

All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

Key Points: 
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The medicine, also known as AVR-RD-O4, is a gene therapy, produced from immature blood (stem) cells collected from the patient.
  • After infusion into the patient, these cells permanently settle in the bone marrow, where they multiply to produce mature blood cells.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in ha[...]

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Graft-versus-host disease, Medicine, Cell, Bone marrow, Risk, Haematopoiesis, Patient, Committee, Knowledge, Regulation, HSCT, EMA, IRIS, European, Leukemia, COMP, Immune system, European Commission, Consultant, Safety, Haematopoietic system, Blood, Marketing, Umbilical cord, CATS, Pharmaceutical industry

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Key Points: 


Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, WHIM, WHIM syndrome, Myelokathexis, Pneumonia, Bone marrow, CXCR4, Risk, Patient, Committee, Knowledge, Hypogammaglobulinemia, Antibody, Regulation, Viral, EMA, IRIS, Wart, Otitis, Protein, Lymphocyte, European, Infection, COMP, Immune system, European Commission, Neutrophil, Blood, Safety, Consultant, Marketing, Movement, Pharmaceutical industry

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

Key Points: 


Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, B cell, Gaucher's disease, Cell, CD34, Bone marrow, Bleeding, Disease, Seizure, Bruise, Patient, Committee, Knowledge, Fracture, Spleen, Regulation, Fatigue, Autotransplantation, EMA, IRIS, Human, Life expectancy, Eye, Bone pain, Anemia, European, COMP, Sponsor, European Commission, Safety, PPD, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Fabry disease, Brain, Cell, CD34, Disease, Nervous system, Patient, Committee, Knowledge, Tissue, Stroke, Kidney failure, Fats, Regulation, Autotransplantation, EMA, IRIS, Human, Eye, Sugar, European, Skin, COMP, Heart, Sponsor, European Commission, Enzyme replacement therapy, Blood, PPD, Pain, Safety, Marketing, Bone marrow, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Invivyd Announces FDA Authorization for Emergency Use of PEMGARDA™ (Formerly VYD222) for Pre-exposure Prophylaxis (PrEP) of COVID-19

Retrieved on: 
Friday, March 22, 2024
Diphenhydramine, Anaphylaxis, Hook effect, Pre-exposure prophylaxis, Half-life, Risk, Bone marrow, COVID-19, Patient, Metoprolol, Adrenaline, Epitope, DSM-IV codes, SARS-CoV-2, Death, Adolescence, Bank statement, Post-exposure prophylaxis, Clinical trial, CDC, Antibody, Vaccination, Safety, Immunodeficiency, Vaccine, FDA, Food, Medicine

Recipients should not be currently infected with or have had a known recent exposure to an individual infected with SARS-CoV-2.

Key Points: 
  • Recipients should not be currently infected with or have had a known recent exposure to an individual infected with SARS-CoV-2.
  • “The PEMGARDA EUA marks a transformational moment for Invivyd and for the many moderately to severely immunocompromised people who are vulnerable to COVID-19 disease in the U.S.
  • It was developed using INVYMAB™, the company’s platform approach which combines state-of-the-art viral surveillance and predictive modeling with advanced antibody engineering.
  • The severity of the reactions was generally mild (17/27) or moderate (8/27), but two reactions were life-threatening.

Salarius Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Friday, March 22, 2024
Research, Topotecan, TC, LSD1, Survival, Bone marrow, Progression-free survival, DCR, Patient, Acquisition, Deuterium, NHL, IND, Myelodysplastic syndrome, Non-Hodgkin lymphoma, Clinical trial, Azacitidine, Chronic myelomonocytic leukemia, Probability, Cyclophosphamide, FDA, MD Anderson Cancer Center, Ewing sarcoma, Consultant, Food, Pharmaceutical industry

HOUSTON, March 22, 2024 (GLOBE NEWSWIRE) -- Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company using protein inhibition and protein degradation to develop cancer therapies for patients in need of new treatment options, today reported financial results for the three and 12 months ended December 31, 2023 and provided a business update.

Key Points: 
  • Cash and cash equivalents were $5.9 million as of December 31, 2023, compared with $12.1 million as of December 31, 2022.
  • In August 2023 Salarius announced a comprehensive review of strategic alternatives focused on maximizing shareholder value.
  • Research and development expenses were $0.06 million for the fourth quarter of 2023, compared with $4.7 million for the fourth quarter of 2022, reflecting the above-mentioned cost-savings plan.
  • Net cash used for operating activities during the fourth quarter of 2023 was $1.5 million, compared with $4.7 million during the same quarter in 2022, reflecting the above-mentioned cost-savings plan.