Alkaline phosphatase

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

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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.

Surrozen Announces Safety, Pharmacodynamic and Liver Function Data for SZN-043

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Monday, April 1, 2024
MELD, Metabolism, Regeneration, Dicarboxylic acid, Survival, Liver disease, PD, Safety, Patient, History, Alkaline phosphatase, CYP1A2, Crosstalk (biology), ASGR1, Pharmacokinetics, Therapy, ALP, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., April 01, 2024 (GLOBE NEWSWIRE) -- Surrozen, Inc. (“Surrozen” or the “Company”) (Nasdaq: SRZN), a company pioneering targeted therapeutics that selectively activate the Wnt Pathway for tissue repair and regeneration, today provided an update on the Phase 1a clinical trial of SZN-043 in healthy volunteers and patients with cirrhosis.   The Phase 1a study was completed in February 2024. SZN-043 demonstrated acceptable safety and tolerability in all subjects, with evidence of target engagement, Wnt signal activation and effects on liver function.   The observed safety and pharmacodynamic activity were the basis for the Company’s previous announcement that it planned to initiate enrollment in the Phase 1b study in alcohol-associated hepatitis.

Key Points: 
  • SZN-043 demonstrated acceptable safety and tolerability in all subjects, with evidence of target engagement, Wnt signal activation and effects on liver function.
  • The observed safety and pharmacodynamic activity were the basis for the Company’s previous announcement that it planned to initiate enrollment in the Phase 1b study in alcohol-associated hepatitis.
  • The randomized, placebo-controlled Phase 1a trial enrolled a total of 48 subjects, including 40 healthy volunteers and 8 patients with cirrhosis and a history of liver disease.
  • Cirrhotic patients also showed evidence of liver function effects after treatment with SZN-043 as measured by HepQuant which is a test that measures cholate clearance, a liver specific function that quantifies liver function.

BeiGene Receives FDA Approval for TEVIMBRA® for the Treatment of Advanced or Metastatic Esophageal Squamous Cell Carcinoma After Prior Chemotherapy

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Thursday, March 14, 2024
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TEVIMBRA will be available in the U.S. in the second half of 2024.

Key Points: 
  • TEVIMBRA will be available in the U.S. in the second half of 2024.
  • The FDA is also reviewing Biologics License Applications (BLAs) for tislelizumab as a first-line treatment for patients with unresectable, recurrent, locally advanced, or metastatic ESCC and patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma.
  • BeiGene has launched more than 17 potentially registration-enabling trials with TEVIMBRA, of which 11 Phase 3 randomized trials and four Phase 2 trials have already had positive readouts.
  • More than 900,000 patients have been prescribed TEVIMBRA globally to date.

CymaBay Reports Fourth Quarter and Year Ended December 31, 2023 Financial Results and Provides Corporate Update

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Wednesday, February 28, 2024
Alkaline phosphatase, Acquisition, Liver, Child, Therapy, EMA, Ursodeoxycholic acid, New Drug Application, Gilead Sciences, Education, IDEAL, FDA, Science, Quality of life, Food, Marketing, U.S. FDA, Breakthrough, ALP, Hospital, PBC, Phase 3, Cirrhosis, Hepatology, Seladelpar, Safety, ULN, European Medicines Agency, Numerical, Patient, MHRA, UDCA, Itch, NDA, NRS, Pharmaceutical industry

NEWARK, Calif., Feb. 28, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, today announced corporate updates and financial results for the year and fourth quarter ended December 31, 2023.

Key Points: 
  • NEWARK, Calif., Feb. 28, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, today announced corporate updates and financial results for the year and fourth quarter ended December 31, 2023.
  • “2023 was a seminal year for CymaBay with critical achievements in the development of our investigational therapeutic, seladelpar.
  • Net loss for the year ended December 31, 2023 and 2022 was $105.4 million and $106.0 million, or ($0.99) and ($1.21) per share, respectively.
  • Net loss for the three months ended December 31, 2023 was higher than the three months ended December 31, 2022 primarily due to higher operating expenses.

Gilead Sciences Expands Liver Portfolio With Acquisition of CymaBay Therapeutics

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Monday, February 12, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Hospitals, Clinical Trials, Alkaline phosphatase, EMA, Woman, Gilead Sciences, Multimedia, Liver disease, Quality of life, Ascending cholangitis, PBC, Risk, Hepatology, Cirrhosis, Seladelpar, Therapy, Fatigue, PRIME, Primary biliary cholangitis, Patient, BofA Securities, Guggenheim Partners, Itch, Centerview Partners, Lazard, Pharmaceutical industry

Gilead Sciences, Inc. (Nasdaq: GILD) and CymaBay Therapeutics, Inc. (Nasdaq: CBAY) announced today a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion.

Key Points: 
  • Gilead Sciences, Inc. (Nasdaq: GILD) and CymaBay Therapeutics, Inc. (Nasdaq: CBAY) announced today a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion.
  • View the full release here: https://www.businesswire.com/news/home/20240211034242/en/
    “We are looking forward to advancing seladelpar by leveraging Gilead’s long-standing expertise in treating and curing liver diseases,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences.
  • Seladelpar is an investigational, oral, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, shown to regulate critical metabolic and liver disease pathways.
  • “Today’s agreement with Gilead is the culmination of years of focus and determination at CymaBay to advance seladelpar and bring new hope to people living with PBC and their families,” said Sujal Shah, President, and CEO at CymaBay Therapeutics.

CymaBay Announces FDA Acceptance of NDA and Priority Review for Seladelpar for the Treatment of Primary Biliary Cholangitis

Retrieved on: 
Monday, February 12, 2024
Alkaline phosphatase, Liver, Child, Ursodeoxycholic acid, New Drug Application, BTD, Cirrhosis, Inflammation, FDA, Quality of life, Food, Breakthrough, Chief, Conditional sentence, PDUFA, ALP, PBC, Seladelpar, Therapy, Fatigue, Patient, Itch, Mortality, NDA, Pharmaceutical industry

NEWARK, Calif., Feb. 12, 2024 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for seladelpar, an investigational treatment for the management of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act (PDUFA) target action date of August 14, 2024 and notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • PBC is a rare, chronic condition that can lead to inflammation and eventually liver cirrhosis.
  • People living with PBC can also experience symptoms that significantly impact their quality of life such as pruritus (itch) and fatigue.
  • Seladelpar is an investigational, potent, selective, orally active PPARδ agonist, or delpar, in development for PBC treatment.
  • "We are encouraged by the agency's decision to grant priority review for seladelpar, and its recognition of the significant need for new treatment options for people living with PBC.

Pliant Therapeutics Announces Positive Safety and Exploratory Efficacy Data from the INTEGRIS-PSC Phase 2a Trial of Bexotegrast 320 mg in Patients with Primary Sclerosing Cholangitis and Suspected Liver Fibrosis

Retrieved on: 
Sunday, February 4, 2024
ALP, AGAF, IBD, Ascending cholangitis, Liver failure, Pharmacokinetics, SAN, Washington State University, Inflammation, Fibrosis, Cirrhosis, Itch, PSC, Ursodeoxycholic acid, MRI, Telephone, Magnetic resonance imaging, Inflammatory bowel disease, Patient, Safety, FDA, EMA, FACP, Disease, IPF, SAE, FRCP, ELF, Week, Therapy, Conference call, Alkaline phosphatase, Interest, UDCA, FACG, University, Webcast, Liver, Primary sclerosing cholangitis, Plasma, Liver disease, Conference, Medical imaging

SOUTH SAN FRANCISCO, Calif., Feb. 04, 2024 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX) today announced 12-week interim data from the 320 mg dose group of INTEGRIS-PSC, a multinational, randomized, double-blind, placebo-controlled Phase 2a clinical trial of bexotegrast in patients with primary sclerosing cholangitis (PSC) and suspected moderate to severe liver fibrosis. The 320 mg group met its primary and secondary endpoints demonstrating that bexotegrast was well tolerated over a 12-week treatment period and its plasma concentrations increased with dose. There was no dose relationship for adverse events. Pruritus and cholangitis occurred less frequently on bexotegrast than on placebo.

Key Points: 
  • The trial’s exploratory efficacy endpoints assessed changes in the liver fibrosis markers, Enhanced Liver Fibrosis (ELF) score and PRO-C3 levels, as well as liver biochemistry and magnetic resonance imaging (MRI) of the liver.
  • In addition, MRI imaging continued to show evidence of improved hepatocyte function and bile flow with bexotegrast at the 320 mg dose relative to placebo.
  • INTEGRIS-PSC is a multinational, randomized, dose-ranging, double-blind, placebo-controlled Phase 2a trial evaluating bexotegrast at once-daily oral doses of 40 mg, 80 mg, 160 mg, 320 mg or placebo for 12 weeks in 121 patients with PSC.
  • The 320 mg group enrolled 27 patients in the active arm and added 9 new patients to the pooled placebo arm.

NGM Bio Announces New Clinical Data from Ongoing Trial of NGM707 in Advanced Solid Tumors and Outlines Evolved Strategy for Aldafermin and NGM120 to Focus on Rare Conditions with Significant Unmet Need

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Tuesday, January 9, 2024
Bile, Woman, Hyperemesis gravidarum, Research, Biology, Hospital, Weight loss, MSS, FRS, Hepatology, University, ILT3, Liver disease, FGF19, GFRAL, LAIR1, Liver, Doctor of Philosophy, NASH, CRC, Pembrolizumab, Trial of the century, Neoplasm, NGM, SAN, PRS, Public health, Genetic epidemiology, Preterm birth, Vomiting, Cirrhosis, DCR, Dehydration, AASLD, Immunotherapy, Place of birth, Medicine, Pregnancy, Pre-eclampsia, Therapy, ELF, Patient, Bile acid malabsorption, Fibrosis, Nausea, Alkaline phosphatase, PSC, Gastrointestinal disease, CRN, IBS-D, Malnutrition, FDA, Biomarker, Pharmaceutical industry

NGM Bio also outlined its strategy to evolve clinical development of its product candidates aldafermin and NGM120 to focus on rare conditions characterized by significant unmet need.

Key Points: 
  • NGM Bio also outlined its strategy to evolve clinical development of its product candidates aldafermin and NGM120 to focus on rare conditions characterized by significant unmet need.
  • Recent landmark genetic research confirmed the link between this rare, devastating condition experienced during pregnancy to higher levels of GDF15.
  • Given our deep expertise in GDF15 biology, we believe we are well positioned to potentially pursue this indication for NGM120.
  • NGM Bio previously reported data from a randomized, double-blind, placebo-controlled Phase 2 study of aldafermin for the treatment of PSC.

Human medicines European public assessment report (EPAR): Mycamine, micafungin, Date of authorisation: 25/04/2008, Revision: 18, Status: Authorised

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Tuesday, January 9, 2024
Fungal infection, Liver, Marketing, Chills, INN, Risk, Leukopenia, Hypotension, Fungus, Hypertension, Nausea, DNA, Inflammation, Liver disease, Anatomy, IIA, CHMP, Diarrhea, Fever, Infection, ATC, Hepatomegaly, European, Neutrophil, Neoplasm, HIV, Tachycardia, Anemia, Hypocalcemia, Hypokalemia, Patient, Medical Subject Headings, Caspofungin, European Commission, Echinocandin, Kidney failure, International, Bilirubin, Neutropenia, Blood, Language, Thrombocytopenia, Magnesium, Select, Medicine, Vomiting, Phlebitis, Amphotericin B, Allergy, Fluconazole, Headache, Rat, Haematopoiesis, Committee, Checklist, Alkaline phosphatase, Rash, Medical device

Human medicines European public assessment report (EPAR): Mycamine, micafungin, Date of authorisation: 25/04/2008, Revision: 18, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Mycamine, micafungin, Date of authorisation: 25/04/2008, Revision: 18, Status: Authorised

Human medicines European public assessment report (EPAR): Eladynos, abaloparatide, Date of authorisation: 12/12/2022, Revision: 3, Status: Authorised

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Tuesday, January 2, 2024
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Human medicines European public assessment report (EPAR): Eladynos, abaloparatide, Date of authorisation: 12/12/2022, Revision: 3, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Eladynos, abaloparatide, Date of authorisation: 12/12/2022, Revision: 3, Status: Authorised