Thrombocytopenia

Viracta Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update

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Giovedì, Maggio 9, 2024
FDA, CRR, DLBCL, Investment, Safety, Food, Nausea, Research, PTCL, Peripheral T-cell lymphoma, Thrombocytopenia, Insurance, Weight loss, Appetite, PTLD, Data, Dor, Patient, Fatigue, Diarrhea, SDD, Therapy, PMDA, Valganciclovir, Food and Drug Administration, SAN, Anemia, Pharmaceutical industry

We look forward to sharing additional data from Stage 1, which continue to mature, as well as initial results from Stage 2 in the third quarter of 2024.

Key Points: 
  • We look forward to sharing additional data from Stage 1, which continue to mature, as well as initial results from Stage 2 in the third quarter of 2024.
  • As of the February 7, 2024 data cutoff date, Nana-val demonstrated greater efficacy than nanatinostat alone and was generally well-tolerated.
  • Present Stage 1 + Stage 2 data (n=21) in the R/R EBV+ PTCL cohort in patients treated with Nana-val in the third quarter of 2024.
  • Initiate a dose-optimization cohort to confirm the RP2D as part of the study’s Phase 2 expansion by year-end 2024.

Knight Therapeutics Reports First Quarter 2024 Results

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Giovedì, Maggio 9, 2024
Knight, DLBCL, News, ITP, Health maintenance organization, Hot, Vaginal discharge, Depreciation, Thrombocytopenia, Stomach, Inventory, Collection, Trial of the century, ASCT, Lymphoma, Patient, Acquisition, VVA, IAS, Menopause, Lenalidomide, Absorption, Blue balls, Bookkeeping

Inventories: As at March 31, 2024, inventories were at $95,400, an increase of $3,566 or 4%.

Key Points: 
  • Inventories: As at March 31, 2024, inventories were at $95,400, an increase of $3,566 or 4%.
  • Accounts payable and accrued liabilities: As at March 31, 2024, accounts payable and accrued liabilities were $94,711, an increase of $4,094 or 5%.
  • Cash, cash equivalents and marketable securities: As at March 31, 2024, Knight had $181,859 in cash, cash equivalents and marketable securities, an increase of $20,034 or 12% compared to December 31, 2023.
  • Knight will host a conference call and audio webcast to discuss its first quarter ended March 31, 2024, today at 8:30 am ET.

Foghorn Therapeutics Provides First Quarter 2024 Financial and Corporate Update

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Lunedì, Maggio 6, 2024
Chromatin, Protein, SMARCA4, BRG1, Security, Research, SMARCA2, IND, Prostate cancer, LDAC, Acetyltransferase, BRM, AACR, Lung cancer, Multiple myeloma, AML, CBP, ARID1B, Lilly, Complex, Merck, NSCLC, DLBCL, ARID1A, Survival, Thrombocytopenia, Therapy, Gene expression, Biology, Cancer, Prostate, Data, Patient, Selective, SWI/SNF, Neoplasm, MDS, Anemia, Pharmaceutical industry

In April 2024, Foghorn presented preclinical data highlighting pipeline progress from multiple potential first-in-class medicines, including the first presentation of preclinical data for FHD-909, at the 2024 American Association for Cancer Research (AACR) Annual Meeting held April 5-10, 2024.

Key Points: 
  • In April 2024, Foghorn presented preclinical data highlighting pipeline progress from multiple potential first-in-class medicines, including the first presentation of preclinical data for FHD-909, at the 2024 American Association for Cancer Research (AACR) Annual Meeting held April 5-10, 2024.
  • In April 2024, Foghorn hosted the first Future of Disease and Chromatin Regulation Summit at the Foghorn corporate headquarters in Cambridge, Massachusetts.
  • In April 2024, Foghorn appointed Kristian Humer as Chief Financial Officer.
  • In April, Foghorn presented new pharmacodynamic and pharmacokinetic preclinical data at the 2024 AACR Annual Meeting highlighting:
    No observed thrombocytopenia in vivo.

Press Release: Rilzabrutinib LUNA 3 phase 3 study met primary endpoint in immune thrombocytopenia

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Martedì, Aprile 23, 2024
FDA, DSM-IV codes, Prurigo nodularis, Fast track (FDA), Safety, Risk, ITP, Food, Thrombocytopenia, Orphan, Research and development, Development, Patient, Fatigue, Intracranial hemorrhage, LUNA, Asthma, Sanofi, Hematology, BTK, IgG4-related disease

Positive results from the LUNA 3 phase 3 study demonstrated that rilzabrutinib 400 mg twice daily orally achieved the primary endpoint of durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP).

Key Points: 
  • Positive results from the LUNA 3 phase 3 study demonstrated that rilzabrutinib 400 mg twice daily orally achieved the primary endpoint of durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP).
  • The safety profile of rilzabrutinib was consistent with that reported in previous studies.
  • LUNA 3 study met its primary endpoint demonstrating a significantly higher proportion of patients receiving rilzabrutinib achieved the primary endpoint of durable platelet response versus placebo.
  • This clinically and statistically significant result was achieved in a population of patients with primary ITP that had been refractory to prior therapy.

Viracta Therapeutics Announces Positive Topline Nana-val Results from Stage 1 of the NAVAL-1 Trial in Patients with Relapsed or Refractory Epstein-Barr Virus-Positive (EBV+) Peripheral T-Cell Lymphoma

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Lunedì, Aprile 15, 2024
FDA, Patient, Safety, CRR, Nausea, PTCL, Peripheral T-cell lymphoma, Thrombocytopenia, Weight loss, Appetite, Fatigue, Diarrhea, ITT, Therapy, Valganciclovir, SAN, Anemia, Pharmaceutical industry

SAN DIEGO, April 15, 2024 (GLOBE NEWSWIRE) -- Viracta Therapeutics, Inc. (Nasdaq: VIRX), a clinical-stage precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, today reported positive topline results from Stage 1 of the pivotal Phase 2 NAVAL-1 trial from both arms of the relapsed or refractory (R/R) Epstein-Barr virus-positive (EBV+) peripheral T-cell lymphoma (PTCL) cohort. Patients were randomized to either nanatinostat monotherapy (n=10) or to nanatinostat in combination with valganciclovir (Nana-val, n=10). These data were featured in an oral presentation during the 2024 Joint Annual Congress of Taiwan Society of Blood and Marrow Transplantation and The Hematology Society of Taiwan.

Key Points: 
  • Patients were randomized to either nanatinostat monotherapy (n=10) or to nanatinostat in combination with valganciclovir (Nana-val, n=10).
  • These data were featured in an oral presentation during the 2024 Joint Annual Congress of Taiwan Society of Blood and Marrow Transplantation and The Hematology Society of Taiwan.
  • Patients who did not respond to nanatinostat monotherapy after 6 weeks of treatment were offered the opportunity to cross over to receive Nana-val.
  • Five nanatinostat monotherapy patients crossed over to receive Nana-val, two of whom remain on Nana-val treatment with stable disease as of the data cutoff.

Global clinical development on Dengue antiviral candidate will start, Hyundai Bioscience strives to win emergency use authorization

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Venerdì, Aprile 19, 2024
Public, Viral, Risk, Safety, IC50, Bacteria, Death, Thrombocytopenia, Trial of the century, Dengue virus, DDS, Polymer, Viral load, Patient, Bleeding, Niclosamide, Immunity, Absorption, COVID-19, Infection, Vaccine

Against this backdrop, a host of countries in the Americas and Asia declared a public health emergency due to Dengue.

Key Points: 
  • Against this backdrop, a host of countries in the Americas and Asia declared a public health emergency due to Dengue.
  • Second, Dengue antiviral should be administered in the early stages to properly deal with the Dengue virus.
  • In the near future, Hyundai Bioscience plans to initiate clinical studies in America and Southeast Asia with the aim of emergency use authorization.
  • "The success of our future clinical study may lead to emergency use authorization as the world's first Dengue antiviral," Hyundai Bioscience USA CEO Kim Kyung-il said.

Foghorn Therapeutics Presents New Preclinical Data on Potential First-in-Class BRM Selective Inhibitor FHD-909 and Selective CBP and Selective EP300 Degrader Oncology Programs

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Martedì, Aprile 9, 2024
Well, BRM, Acetyltransferase, Gene expression, Survival, NSCLC, PD, SMARCA2, Neoplasm, Megakaryocyte, Cancer, Prostate, Lung cancer, Protein, IND, EP300, AACR, ATPase, Therapy, BRG1, Safety, CBP, Lilly, Anemia, ID, Thrombocytopenia, Annual general meeting, Pharmaceutical industry

CAMBRIDGE, Mass., April 09, 2024 (GLOBE NEWSWIRE) -- Foghorn® Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious diseases by correcting abnormal gene expression, today announced new preclinical data for potential first-in-class medicines including FHD-909, a BRM (SMARCA2) selective inhibitor, selective CBP degrader, and selective EP300 degrader programs at the 2024 American Association for Cancer Research (AACR) Annual Meeting. Foghorn management will hold a conference call and webcast today at 5 p.m. ET to review important pipeline updates.

Key Points: 
  • “Notably, our first-in-class BRM selective inhibitor FHD-909 has demonstrated favorable tolerability and encouraging dose-dependent single agent activity in preclinical models of BRG1 mutated tumors.
  • Additionally, we are applying our long-acting formulation capabilities to our degrader programs, which further enhances the clinical potential of these drug candidates.
  • However, the ATPase domains of BRM and BRG1are 92% identical which has made identifying a selective BRM inhibitor challenging.
  • Attempts to selectively inhibit CBP or EP300 individually have been challenging due to the high homology between the two proteins.

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

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Sabato, Aprile 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.

Nuvectis Pharma Announces Encouraging Preliminary Data from the NXP800 Phase 1b Clinical Trial in Platinum-Resistant ARID1a-Mutated Ovarian Cancer

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Giovedì, Marzo 14, 2024
Bevacizumab, Ovarian cancer, Disease, Patient, Malignancy, Clinical trial, Fourth grade, Platinum, Thrombocytopenia, Food, Pharmaceutical industry

Fort Lee, NJ, March 14, 2024 (GLOBE NEWSWIRE) -- Nuvectis Pharma, Inc. (NASDAQ: NVCT) today announced preliminary data from the ongoing Phase 1b clinical trial of NXP800 in patients with platinum resistant ARID1a-mutated ovarian cancer, a deadly disease of unmet medical need. The NXP800 development program in this disease was granted Fast Track Designation by the U.S. Food and Drug Administration (“FDA”). The Phase 1b clinical trial is being conducted in top clinical centers in the United States and the United Kingdom.

Key Points: 
  • Fort Lee, NJ, March 14, 2024 (GLOBE NEWSWIRE) -- Nuvectis Pharma, Inc. (NASDAQ: NVCT) today announced preliminary data from the ongoing Phase 1b clinical trial of NXP800 in patients with platinum resistant ARID1a-mutated ovarian cancer, a deadly disease of unmet medical need.
  • The NXP800 development program in this disease was granted Fast Track Designation by the U.S. Food and Drug Administration (“FDA”).
  • The Phase 1b clinical trial is being conducted in top clinical centers in the United States and the United Kingdom.
  • Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis, commented, “We are pleased to share the preliminary results from the NXP800 Phase 1b study in the target patient population of platinum resistant, ARID1a-mutated ovarian cancer patients.

Takeda Announces Approval of ADZYNMA® Intravenous Injection 1500 (apadamtase alfa /cinaxadamtase alfa) in Japan for Patients with Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

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Martedì, Marzo 26, 2024
Health, General Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Microangiopathic hemolytic anemia, Incidence, Headache, Patient, Thrombotic thrombocytopenic purpura, Constipation, Mortality, Head, TTP, Nausea, Toll, Heart, Safety, Itch, Thrombocytopenia, Hypertension, Pharmaceutical industry

Takeda ( TSE: 4502/NYSE:TAK ) today announced that the Japanese Ministry of Health, Labour and Welfare has approved the use of ADZYNMA (apadamtase alfa /cinaxadamtase alfa) for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) for individuals 12 years of age and older.1 ADZYNMA is the first and only approved recombinant ADAMTS13 protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.

Key Points: 
  • Takeda ( TSE: 4502/NYSE:TAK ) today announced that the Japanese Ministry of Health, Labour and Welfare has approved the use of ADZYNMA (apadamtase alfa /cinaxadamtase alfa) for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) for individuals 12 years of age and older.1 ADZYNMA is the first and only approved recombinant ADAMTS13 protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.
  • cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme.2 It is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, headache and abdominal pain.2,3,4 When left untreated, acute TTP events have a mortality rate of >90%.2,4
    “The approval of ADZYNMA is an important milestone for people living with cTTP in Japan, who had limited treatment options and now have the first treatment option specifically approved to treat this ultra-rare condition,” said Yasushi Kajii, Head, R&D Japan Region at Takeda.
  • “Developing innovative treatments that make a difference in the lives of patients is at the heart of what we do.
  • In Period 3, the incidence of TEAEs was 2.8% (1/36) in this drug group: nausea and headache (1 subject each).6
    This approval does not result in any changes to Takeda’s consolidated forecast for the fiscal year ending March 31, 2024 (FY2023).