Cytopenia

Sobi Receives US FDA Fast Track Designation for Emapalumab-lzsg being investigated in Macrophage Activation Syndrome

Retrieved on: 
fredag, maj 24, 2024
Patient, Infosys, Food, Emapalumab, Hemophagocytic lymphohistiocytosis, FDA, Fever, Condylar resorption, Swedish Orphan Biovitrum, Death, Rheumatology, Tocilizumab, Medicine, Fast Track, Ferritin, Hepatosplenomegaly, Interferon, Cytopenia, Liver, Macrophage activation syndrome, MAS, HLH, Pharmaceutical industry

WALTHAM, Mass., May 24, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to emapalumab-lzsg being investigated as a potential therapeutic option in patients with Macrophage Activation Syndrome (MAS).

Key Points: 
  • WALTHAM, Mass., May 24, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to emapalumab-lzsg being investigated as a potential therapeutic option in patients with Macrophage Activation Syndrome (MAS).
  • Emapalumab is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity.
  • Fast track designation is designed to facilitate the development and expedite the review of medicines to treat serious conditions that may fill an unmet medical need.
  • MAS is a severe complication of rheumatic diseases, most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA).

Nkarta Reports First Quarter 2024 Financial Results and Corporate Highlights

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torsdag, maj 9, 2024
NK, Investment, Lupus nephritis, Safety, Research, Cy, B cell, IND, Autoimmune disease, CD19, Cyclophosphamide, Patient, Cytopenia, NHL, SLE, Lymphoid leukemia, G&A, Investigational New Drug, Pharmaceutical industry

Nkarta expects to provide an update on first patient dosing for NKX019 in LN in the first half of 2024.

Key Points: 
  • Nkarta expects to provide an update on first patient dosing for NKX019 in LN in the first half of 2024.
  • In March 2024, Nkarta completed an underwritten offering of common stock and pre-funded warrants with gross proceeds of $240.1 million.
  • Nkarta had cash, cash equivalents, restricted cash, and investments in marketable securities of $450.0 million as of March 31, 2024.
  • Net loss was $29.5 million, or $0.58 per basic and diluted share, for the first quarter of 2024.

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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lördag, 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.

Nkarta Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Highlights

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torsdag, mars 21, 2024
Research, Conference, Cy, AML, Patient, SAN, Lymphoma, Cytopenia, Cytarabine, European Hematology Association, G&A, SLE, NHL, Clinical trial, Investment, Society, Safety, Depreciation, NK, Autoimmune disease, Pharmaceutical industry

Research and development (R&D) expenses were $96.8 million for the full year 2023 and $23.3 million for the fourth quarter of 2023.

Key Points: 
  • Research and development (R&D) expenses were $96.8 million for the full year 2023 and $23.3 million for the fourth quarter of 2023.
  • Non-cash stock-based compensation expense included in R&D expense was $8.0 million for the full year 2023 and $1.7 million for the fourth quarter of 2023.
  • General and administrative (G&A) expenses were $34.9 million for the full year 2023 and $7.9 million for the fourth quarter of 2023.
  • Net loss was $27.8 million, or $0.57 per basic and diluted share, for the fourth quarter of 2023.

Cellectar Biosciences Reports High Rate of Complete Remission in Investigator Initiated Phase I Study of Iopofosine in Combination with External Beam Radiotherapy in Recurrent Head and Neck Cancer

Retrieved on: 
måndag, mars 4, 2024
Head, Neck, Recurrence, Toxicity, Anemia, Completeness, Radiation, SPECT/CT, Chemoradiotherapy, EBRT, Macroglobulinemia, Lymphocytopenia, Thrombocytopenia, Patient, Neutropenia, Survival, Poster, Cytopenia, Cancer, Pharmaceutical industry

The twelve patients treated for locoregionally recurrent head and neck squamous cell carcinoma previously received chemoradiation alone (42%), surgery (58%) or surgery combined with radiation or chemoradiation (92%).

Key Points: 
  • The twelve patients treated for locoregionally recurrent head and neck squamous cell carcinoma previously received chemoradiation alone (42%), surgery (58%) or surgery combined with radiation or chemoradiation (92%).
  • The data were presented in a poster at the 2024 Multidisciplinary Head and Neck Cancers Symposium held February 29-March 2, 2024, in Phoenix, AZ.
  • Complete remission was achieved in 64% of patients, with an ORR of 73% (n=11).
  • Observed adverse events were consistent with the known toxicity profile of iopofosine I 131, with cytopenias being the most common with all patients recovering.

Keros Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

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onsdag, februari 28, 2024
Therapy, Research, Syndrome, Investment, Obesity, Technology transfer, Growth, KROS, PAH, Patient, Disorder, Protein, Ruxolitinib, Cytopenia, Orthostatic hypertension, Haematopoiesis, Keros, Pharmaceutical industry

Keros did not generate any revenue for the year ended December 31, 2022.

Key Points: 
  • Keros did not generate any revenue for the year ended December 31, 2022.
  • Research and development expenses were $37.5 million for the fourth quarter and $135.3 million for the year ended December 31, 2023, as compared to $24.9 million for the fourth quarter and $87.3 million for the year ended December 31, 2022.
  • General and administrative expenses were $9.1 million for the fourth quarter and $34.8 million for the year ended December 31, 2023, as compared to $7.1 million and $27.5 million for the fourth quarter and year ended December 31, 2022.
  • Keros’ cash and cash equivalents as of December 31, 2023 was $331.1 million compared to $279.0 million as of December 31, 2022.

Iovance’s AMTAGVI™ (lifileucel) Receives U.S. FDA Accelerated Approval for Advanced Melanoma

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fredag, februari 16, 2024
BRAF, SAN, Neoplasm, Cohort, Immune system, Kaplan–Meier estimator, Immunotherapy, TIL, Information, FDA, Melanoma, Food, News, Cytopenia, Infection, PD-1, AIM, National Cancer Institute, Respiratory failure, Travel, Epidemiology, Interim, Safety, Physician, BRAF V600, Patient, Journal, RECIST, ATC, Disease, Clinical trial, Kidney failure, MEK, Pharmaceutical industry

SAN CARLOS, Calif., Feb. 16, 2024 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) cell therapies for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has approved AMTAGVI™ (lifileucel) suspension for intravenous infusion. AMTAGVI is a tumor-derived autologous T cell immunotherapy indicated for the treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. This indication is approved under an accelerated approval based on overall response rate (ORR) and duration of response. Iovance is also conducting TILVANCE-301, a Phase 3 trial to confirm clinical benefit.

Key Points: 
  • This indication is approved under an accelerated approval based on overall response rate (ORR) and duration of response.
  • AMTAGVI is the first and the only one-time, individualized T cell therapy to receive FDA approval for a solid tumor cancer.
  • “Given the significant unmet needs in the advanced melanoma community, we are proud to offer a personalized, one-time therapeutic option for these patients.
  • Iovance will host a conference call and live audio webcast today to discuss the FDA approval of AMTAGVI.

Bristol Myers Squibb Receives Positive CHMP Opinion for CAR T Cell Therapy Abecma (idecabtagene vicleucel) in Earlier Lines of Therapy for Triple-Class Exposed Relapsed and Refractory Multiple Myeloma

Retrieved on: 
fredag, januari 26, 2024
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Bristol Myers Squibb, Death, Patient, Immunomodulatory imide drug, European, CHMP, Progression-free survival, Neurotoxicity, European Medicines Agency, Cytopenia, Civil service commission, European Commission, Society, EMA, Risk, Head, Cytokine release syndrome, CRS, Multiple myeloma, Food, PFS, FDA, ASH, Committee, BMY, Pharmaceutical industry

The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.

Key Points: 
  • The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.
  • “This positive CHMP opinion represents an important step toward bringing our potentially transformative first-in-class anti-BCMA CAR T cell therapy, Abecma, to more patients earlier in the multiple myeloma treatment paradigm to improve outcomes,” said Anne Kerber, M.D., senior vice president and head, Late Clinical Development, Hematology, Oncology, Cell Therapy (HOCT), Bristol Myers Squibb.
  • Treatment with Abecma exhibited a well-established safety profile, with mostly low-grade and transient occurrences of cytokine release syndrome (CRS) and neurotoxicity.
  • In the EU, the EC delivers its final decision approximately two months following receipt of the CHMP opinion.

Cellectar Biosciences Announces Positive Topline Data Achieving Primary Endpoint in Pivotal Clinical Study of Iopofosine I 131 in Waldenstrom’s Macroglobulinemia

Retrieved on: 
måndag, januari 8, 2024
Sequela, Quality of life, Febrile neutropenia, WM, Death, Anemia, International, IWMF, Disease, Neutropenia, NDA, WAM, News, Cancer, Thrombocytopenia, SCR, Macroglobulinemia, MCI, MRR, Mayo Clinic, Mitt, Patient, Safety, Medicine, Cytopenia, Immunoglobulin M, Pharmaceutical industry

The CLOVER WaM study met its primary endpoint with a major response rate (MRR) of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value

Key Points: 
  • The CLOVER WaM study met its primary endpoint with a major response rate (MRR) of 61% (95% confidence interval [44.50%, 75.80%, two-sided p value
  • Notably, iopofosine monotherapy achieved an 8% stringent complete remission (sCR) in this highly refractory WM population.
  • “The results from this pivotal study utilizing just four doses of iopofosine monotherapy in heavily pretreated patients are very compelling, demonstrating deep and durable remissions.
  • “Iopofosine’s high major response rate and achievement of the study’s primary endpoint in highly refractory, Waldenstrom’s macroglobulinemia patients exhibits its potentially practice-changing clinical profile.

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

Retrieved on: 
måndag, december 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.