Hypophosphatemia

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024
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BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

Human medicines European public assessment report (EPAR): Carvykti, ciltacabtagene autoleucel, Date of authorisation: 25/05/2022, Revision: 5, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
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Human medicines European public assessment report (EPAR): Carvykti, ciltacabtagene autoleucel, Date of authorisation: 25/05/2022, Revision: 5, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Carvykti, ciltacabtagene autoleucel, Date of authorisation: 25/05/2022, Revision: 5, Status: Authorised

ImmunoGen Presents Findings from Newly Diagnosed Acute Myeloid Leukemia Cohorts in Phase 1b/2 Study of Pivekimab Sunirine in Combination with Azacitidine and Venetoclax at ASH

Retrieved on: 
Sunday, December 10, 2023
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Molecular Pharmacology, Fever, Associate, Azacitidine, Hypotension, Safety, CCR, Spacecraft, Pneumonia, ASH, University, Cytopenia, Disease, Cellular, Constipation, Fatigue, Mortality, Penile ultrasonography, Patient, IMGN, DNA, MRD, Medical Affairs Bureau, Pharmacokinetics, Peripheral edema, Flow cytometry, Capillary leak syndrome, MD, Diarrhea, Cancer, Nausea, Survival, Drug resistance, Transplant, AML, Apoptosis, MD Anderson Cancer Center, Society, ImmunoGen, Hypokalemia, Hypophosphatemia, Venetoclax, Rare-earth element, Pharmaceutical industry, Birth control, Vaccine, Medicinal plants, Research

These findings will be presented in a poster session at the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, California.

Key Points: 
  • These findings will be presented in a poster session at the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, California.
  • “The MRD negativity rates, which are indicative of a deep remission, are particularly promising in the treated patient population.
  • Response rates and MRD negativity were numerically comparable between cohorts 1 and 2, despite differences in the venetoclax schedule.
  • We look forward to continuing to expand our cohort of newly diagnosed unfit patients to inform the development path for pivekimab in AML.”
    ImmunoGen is also presenting two preclinical posters at ASH.

American Regent Introduces Potassium Phosphates, USP; FDA-Approved and "AP" Rated¹

Retrieved on: 
Thursday, October 19, 2023
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In addition, inappropriate intravenous administration of undiluted or insufficiently diluted potassium phosphates as a rapid "IV push" has resulted in cardiac arrest, cardiac arrhythmias, hypotension, and death.

Key Points: 
  • In addition, inappropriate intravenous administration of undiluted or insufficiently diluted potassium phosphates as a rapid "IV push" has resulted in cardiac arrest, cardiac arrhythmias, hypotension, and death.
  • Vein Damage and Thrombosis: Potassium Phosphates Injection must be diluted and administered in intravenous fluids or used as an admixture in parenteral nutrition.
  • You are encouraged to report adverse drug events to American Regent, Inc. at 1-800-734-9236 or to the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.
  • You are encouraged to report adverse drug events (ADEs) to American Regent:
    ADEs may also be reported to the FDA:

Inozyme Pharma Announces Positive Interim Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE)

Retrieved on: 
Tuesday, September 26, 2023
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BOSTON, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive interim safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE, pseudoxanthoma elasticum).

Key Points: 
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.
  • Exploratory biomarker data were collected throughout the study to provide evidence of the potential for disease modification with ongoing treatment with INZ-701.
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • PK properties were consistent with those observed in the Phase 1/2 clinical trial in adults with ENPP1 Deficiency.

Day One Announces Updated FIREFLY-1 Data for Tovorafenib and Completion of Rolling NDA Submission to FDA for Relapsed or Progressive Pediatric Low-Grade Glioma (pLGG)

Retrieved on: 
Monday, September 11, 2023
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BRISBANE, Calif., Sept. 11, 2023 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced the recently completed submission of the rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tovorafenib as a monotherapy in relapsed or progressive pediatric low-grade glioma (pLGG). The Company anticipates the FDA will file the rolling NDA by mid-November 2023.

Key Points: 
  • For the majority of patients in the relapsed setting, there is no standard of care and no approved therapies.
  • An updated Clinical Study Report (CSR) was submitted to the FDA with an additional six months of safety and efficacy data through June 5, 2023.
  • The NDA submission also includes an exploratory analysis of ORR by Response Assessment for Neuro-Oncology Low-Grade Glioma (RANO-LGG).
  • Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2026.

Hyloris Pharmaceuticals Reports 2023 Half-Year Results and provides Business Outlook

Retrieved on: 
Wednesday, September 6, 2023
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A potential approval date for the US market was set for 17 October 2023 by the U.S. Food & Drug Administration.

Key Points: 
  • A potential approval date for the US market was set for 17 October 2023 by the U.S. Food & Drug Administration.
  • In January 2023, Hyloris in-licensed HY-088, a product candidate targeting hypophosphatemia, a serious condition causing patients to have low level of phosphate in the blood.
  • For Dofetilide IV, the results of the pivotal clinical study, allowing regulatory submission, are expected by the summer of 2024.
  • Hyloris aims to improve ease of administration and dosage control, and thus potentially improving clinical outcome.

VANFLYTA® Now Available in U.S. for Patients with Newly Diagnosed FLT3-ITD Positive AML

Retrieved on: 
Wednesday, August 9, 2023
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VANFLYTA was approved by the FDA on July 20, 2023 in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive as detected by an FDA-approved test.

Key Points: 
  • VANFLYTA was approved by the FDA on July 20, 2023 in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive as detected by an FDA-approved test.
  • The results of QuANTUM-First were published in The Lancet .1
    The safety of VANFLYTA was evaluated in 265 patients with newly diagnosed FLT3-ITD positive AML who received VANFLYTA once daily (35.4 mg with chemotherapy, 26.5 to 53 mg as maintenance) in the QuANTUM-First trial.
  • Daiichi Sankyo is committed to ensuring that patients in the U.S. who are prescribed VANFLYTA can access the medication and receive appropriate financial support.
  • Provider and patient support and information regarding distribution, access, and reimbursement are now available through Daiichi Sankyo Access Central by visiting www.DSIAccessCentral.com or calling 1-866-4-DSI-NOW (1-866-437-4669).

Day One Reports Second Quarter 2023 Financial Results and Corporate Progress

Retrieved on: 
Monday, August 7, 2023
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BRISBANE, Calif., Aug. 07, 2023 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its second quarter 2023 financial results and highlighted recent corporate achievements.

Key Points: 
  • R&D Expenses: Research and development expenses were $32.2 million for the second quarter of 2023 compared to $22.6 million for the second quarter of 2022.
  • G&A Expenses: General and administrative expenses were $17.1 million for the second quarter of 2023 compared to $14.2 million for the second quarter of 2022.
  • Net Loss: Net loss totaled $45.9 million for the second quarter of 2023 with non-cash stock compensation expense of $9.5 million, compared to $36.5 million for the second quarter of 2022 with non-cash stock compensation expense of $5.6 million.
  • 2023 Wedbush PacGrow Healthcare Conference, August 8-9, 2023
    Morgan Stanley 21st Annual Global Healthcare Conference, September 11-13, 2023

VANFLYTA® First FLT3 Inhibitor Approved in the U.S. Specifically for Patients with Newly Diagnosed FLT3-ITD Positive AML

Retrieved on: 
Thursday, July 20, 2023
Oncology, Health, FDA, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Magnesium, QT interval, Doctor of Philosophy, Alkaline phosphatase, Patient, Division, Risk, Daiichi Sankyo, HR, Leukemia, Nosebleed, Thrombocytopenia, REMS, Maintenance, Febrile neutropenia, Diarrhea, Pharmacy, Duke Cancer Institute, Sepsis, Vomiting, FLT3, Fungal infection, Lancet, Death, Headache, Safety, Neutropenia, Cardiac arrest, Indigestion, ECG, Abdominal pain, Acute myeloid leukemia, Insomnia, Lymphocytopenia, Anemia, CR, Mucositis, Hypermagnesemia, Hyperkalemia, Hypophosphatemia, Mitigation, Hypocalcemia, HSCT, QT, Bone marrow, AML, NE, Hypokalemia, Nausea, Hypernatremia, Upper respiratory tract infection, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, TSE, Pharmaceutical industry, Nursing, MD, Hypoalbuminemia

VANFLYTA will be available by prescription in the U.S. in the coming weeks.

Key Points: 
  • VANFLYTA will be available by prescription in the U.S. in the coming weeks.
  • VANFLYTA is approved with a Boxed WARNING for QT prolongation, torsades de pointes and cardiac arrest.
  • The FDA concurrently approved a companion diagnostic test to detect FLT3-ITD mutations in patients with newly diagnosed AML.
  • Daiichi Sankyo is committed to ensuring that patients in the U.S. who are prescribed VANFLYTA can access the medication and receive appropriate financial support.