Proteinuria

Eloxx Pharmaceuticals Reports Additional Confirmation that All Nonsense Mutation Alport Syndrome Patients Treated with ELX-02 in Phase 2 Study had Improvement in Kidney Morphology and Clinical Benefit of Reduction or Stabilization of Proteinuria

Retrieved on: 
Monday, October 9, 2023
Health, Transmission electron microscopy, RNA, Urine protein/creatinine ratio, Kidney, Proteinuria, Rare, Biopsy, FSD, UPCR, TEM, Patient, Medical imaging

WATERTOWN, Mass., Oct. 09, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported results from an assessment of patient biopsies by NIPOKA GmbH (Nipoka). They have developed a highly accurate method for the quantification of podocyte foot process morphology. These results confirm previously reported positive biopsy results from the proof-of-concept Phase 2 open-label clinical trial (NCT05448755) of ELX-02 for the treatment of Nonsense Mutation Alport syndrome patients. Analysis of formalin-fixed paraffin-embedded (FFPE) biopsy samples by Nipoka show ELX-02 treatment improved podocyte foot process morphology with lower effacement in all three patients at the end of the 8-week study period.

Key Points: 
  • They have developed a highly accurate method for the quantification of podocyte foot process morphology.
  • These results confirm previously reported positive biopsy results from the proof-of-concept Phase 2 open-label clinical trial ( NCT05448755 ) of ELX-02 for the treatment of Nonsense Mutation Alport syndrome patients.
  • “With this accurate analysis of the patient biopsies and quantification of changes, we now have unequivocal evidence of morphology and clinical improvement in all three Nonsense Mutation Alport patients treated with ELX-02.
  • Improvement in kidney morphology drives clinical benefit in this devastating rare disease,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.

Omeros Corporation Provides Update on Interim Analysis of ARTEMIS-IGAN Phase 3 Trial of Narsoplimab in IgA Nephropathy

Retrieved on: 
Monday, October 16, 2023
Health, Stem Cells, Managed Care, Clinical Trials, Research, Science, Pharmaceutical, Cancer, Biomarker, M.D, Placebo, PIN, Safety, UPE, FDA, Corporation, Immunoglobulin A, Proteinuria, Clinical trial, Patient, ARTEMIS, Pharmaceutical industry, Omeros, IgA nephropathy

Topline results show that narsoplimab did not achieve statistically significant improvement over placebo.

Key Points: 
  • Topline results show that narsoplimab did not achieve statistically significant improvement over placebo.
  • The UPE reduction in the placebo group was markedly greater than that reported in trials of other agents in IgA nephropathy.
  • Based on the absence of statistical significance and as previously agreed with FDA, Omeros will not submit an application for approval of narsoplimab in this indication and will discontinue the ARTEMIS-IGAN clinical trial.
  • “We want to thank all the patients and investigators who participated in the trial,” said Gregory A. Demopulos, M.D, chairman and chief executive officer of Omeros.

DaVita Statement on GLP-1 and Potential Implications on Chronic Kidney Disease

Retrieved on: 
Thursday, October 12, 2023
Cardiovascular disease, SELECT, Urine, Novo Nordisk, Death, Research, Risk, CKD, IA Financial Group, Mortality, SGLT2, Health, Type 2, FLOW, Type 2 diabetes, Kidney, Proteinuria, Chronic kidney disease, Clinical trial, DaVita Inc., Patient, C-peptide, GFR, News, Kidney disease, ACE, Pharmaceutical industry, Medical imaging

The following statement can be attributed to Dr. Jeff Giullian, chief medical officer for DaVita Inc.

Key Points: 
  • The following statement can be attributed to Dr. Jeff Giullian, chief medical officer for DaVita Inc.
    "We've been closely monitoring the developments related to GLP-1s, and are excited about the potential benefits this class of drugs could have on society and patients with kidney disease.
  • We will continue monitoring closely as further evidence becomes available to identify the potential benefit of GLP-1s to those afflicted with kidney disease.
  • In the meantime, DaVita remains focused on providing exceptional patient care and advancing the practice of kidney care."
  • The trial sought to demonstrate benefits for the composite endpoint comprised of five separate endpoints: death from kidney disease; death from cardiovascular disease; sustained reduction in GFR by at least 50%; progression to stage 5 CKD; and initiation of chronic kidney replacement therapy.

Mallinckrodt Announces Publication of Two Journal Manuscripts with Clinical and Economic Evidence for Acthar® Gel (Repository Corticotropin Injection)

Retrieved on: 
Tuesday, October 10, 2023
Narrative, RA, Adrenocorticotropic hormone, Health, Literature review, Food, Polymyositis, Dermatomyositis, MS, Abstract, Autoimmunity, Length of stay, Spasm, Clinical, Manuscript, Copyright in Historical Perspective, MOA, Nephrotic syndrome, Rheumatoid arthritis, FDA, Proteinuria, RCI, Patient, ClinicoEconomics and Outcomes Research, History, Literature, Lupus, Multiple sclerosis, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Sarcoidosis, Outcomes research, Inflammation, SLE, Pharmaceutical industry, Medical device, Mallinckrodt, Adrenocorticotropic hormone (medication)

DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Mallinckrodt plc (OTCMKTS: MNKTQ), a global specialty pharmaceutical company, today announced the publication of two journal manuscripts reinforcing the clinical and economic evidence supporting the efficacy, safety profile, continued use, and cost-effectiveness of Acthar Gel for appropriate patients across a range of FDA-approved autoimmune and inflammatory disease indications.1,2 

Key Points: 
  • Mallinckrodt's health economics manuscript titled "Acthar Gel (RCI): A Narrative Literature Review of Clinical and Economic Evidence" was published on June 24, 2023 in ClinicoEconomics and Outcomes Research.
  • Acthar is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides.
  • Acthar Gel is approved by the U.S. Food and Drug Administration (FDA) for the treatment of several autoimmune disorders and medical conditions known to cause inflammation.3
    Please see indications and Important Safety Information below.
  • Outcomes may be influenced by therapies not evaluated in the study and the clinical outcomes may not be solely attributable to Acthar.

Calliditas announces filing with UK MHRA for Kinpeygo in IgA nephropathy

Retrieved on: 
Tuesday, October 3, 2023
EEA, CET, Health care, EGFR, Medication, Glomerular filtration rate, Immunoglobulin A, Budesonide, Person, Proteinuria, Sustainability, European Economic Area, CALT, Risk, Kidney, United Kingdom, Stada Arzneimittel, Lancet, UPCR, MHRA, Disease, Pharmaceutical industry, IgA nephropathy

STOCKHOLM, Oct. 3, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (NASDAQ: CALT) (STO: CALTX) ("Calliditas") today announced that its partner STADA Arzneimittel AG ("STADA") has submitted a request to the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom to convert the conditional marketing authorization for Kinpeygo®, a treatment for primary IgA nephropathy (IgAN), to standard, or "full", marketing authorization.

Key Points: 
  • STOCKHOLM, Oct. 3, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (NASDAQ: CALT) (STO: CALTX) ("Calliditas") today announced that its partner STADA Arzneimittel AG ("STADA") has submitted a request to the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom to convert the conditional marketing authorization for Kinpeygo®, a treatment for primary IgA nephropathy (IgAN), to standard, or "full", marketing authorization.
  • Kinpeygo is an orphan medicinal product and the first and only treatment approved in the UK for IgAN, a rare, progressive autoimmune disease of the kidney with a high unmet need.
  • Kinpeygo is currently approved under conditional approval to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/gram.
  • : +46 76 403 35 43, email: [email protected]
    The information was sent for publication, through the agency of the contact persons set out above, on October 3, 2023 at 09:20 a.m. CET.

Calliditas announces filing with UK MHRA for Kinpeygo in IgA nephropathy

Retrieved on: 
Tuesday, October 3, 2023
EEA, CET, Health care, EGFR, Medication, Glomerular filtration rate, Immunoglobulin A, Budesonide, Person, Proteinuria, Sustainability, European Economic Area, CALT, Risk, Kidney, United Kingdom, Stada Arzneimittel, Lancet, UPCR, MHRA, Disease, Pharmaceutical industry, IgA nephropathy

STOCKHOLM, Oct. 3, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (NASDAQ: CALT) (STO: CALTX) ("Calliditas") today announced that its partner STADA Arzneimittel AG ("STADA") has submitted a request to the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom to convert the conditional marketing authorization for Kinpeygo®, a treatment for primary IgA nephropathy (IgAN), to standard, or "full", marketing authorization.

Key Points: 
  • STOCKHOLM, Oct. 3, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (NASDAQ: CALT) (STO: CALTX) ("Calliditas") today announced that its partner STADA Arzneimittel AG ("STADA") has submitted a request to the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom to convert the conditional marketing authorization for Kinpeygo®, a treatment for primary IgA nephropathy (IgAN), to standard, or "full", marketing authorization.
  • Kinpeygo is an orphan medicinal product and the first and only treatment approved in the UK for IgAN, a rare, progressive autoimmune disease of the kidney with a high unmet need.
  • Kinpeygo is currently approved under conditional approval to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/gram.
  • : +46 76 403 35 43, email: [email protected]
    The information was sent for publication, through the agency of the contact persons set out above, on October 3, 2023 at 09:20 a.m. CET.

Calliditas Therapeutics granted orphan drug designation by the FDA for the treatment of Alport syndrome with setanaxib

Retrieved on: 
Wednesday, September 27, 2023
Kidney disease, ESRD, US Foods, IR, Chronic kidney disease, PBC, ODD, Cochlea, SCCHN, Head, Alport syndrome, Neck, Trial of the century, Hypertension, Proteinuria, Internet, Primary biliary cholangitis, Sustainability, Genetic disorder, Patient, Huber loss, CALT, FDA, SA, IPF, CKD, Gene, Idiopathic pulmonary fibrosis, Food, Pharmaceutical industry, Satellite navigation device, GFR, Alport

STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib.

Key Points: 
  • STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib.
  • Based on supportive pre-clinical work, Calliditas plans to initiate a randomized, placebo-controlled phase 2 clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023.
  • "We are excited to start another clinical program in the renal space targeting an orphan indication where today there are no approved products," said CEO Renée Aguiar-Lucander.
  • Alport syndrome is a genetic disorder arising from the mutations in the genes that code for type 4 collagen.

Calliditas Therapeutics granted orphan drug designation by the FDA for the treatment of Alport syndrome with setanaxib

Retrieved on: 
Wednesday, September 27, 2023
Kidney disease, ESRD, US Foods, IR, Chronic kidney disease, PBC, ODD, Cochlea, SCCHN, Head, Alport syndrome, Neck, Trial of the century, Hypertension, Proteinuria, Internet, Primary biliary cholangitis, Sustainability, Genetic disorder, Patient, Huber loss, CALT, FDA, SA, IPF, CKD, Gene, Idiopathic pulmonary fibrosis, Food, Pharmaceutical industry, Satellite navigation device, GFR, Alport

STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib.

Key Points: 
  • STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib.
  • Based on supportive pre-clinical work, Calliditas plans to initiate a randomized, placebo-controlled phase 2 clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023.
  • "We are excited to start another clinical program in the renal space targeting an orphan indication where today there are no approved products," said CEO Renée Aguiar-Lucander.
  • Alport syndrome is a genetic disorder arising from the mutations in the genes that code for type 4 collagen.

Travere Therapeutics to Present Abstracts at the 17th International Symposium on IgA Nephropathy (IIGANN 2023)

Retrieved on: 
Tuesday, September 26, 2023
Kidney failure, Jay Traver, SPARTAN, IgA nephropathy, Immunoglobulin A, Proteinuria, International Symposium on Endovascular Therapy, Patient, SAN, Radar, Therapy, Safety, Pharmaceutical industry

SAN DIEGO, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company and its collaborators will present preliminary findings from the SPARTAN Study evaluating the potential effect of FILSPARI® (sparsentan) as a first-line therapy in patients with newly diagnosed IgA nephropathy (IgAN) in a late-breaking trial session at the 17th International Symposium on IgA Nephropathy (IIGANN2023) in Ryogoku, Tokyo, September 28-30, 2023.

Key Points: 
  • SAN DIEGO, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company and its collaborators will present preliminary findings from the SPARTAN Study evaluating the potential effect of FILSPARI® (sparsentan) as a first-line therapy in patients with newly diagnosed IgA nephropathy (IgAN) in a late-breaking trial session at the 17th International Symposium on IgA Nephropathy (IIGANN2023) in Ryogoku, Tokyo, September 28-30, 2023.
  • The Company will also present preclinical data supporting the nephroprotective mechanism of action of sparsentan, a dual endothelin angiotensin receptor antagonist, and an analysis of the UK National Registry of Rare Renal Diseases (RaDaR) which demonstrated that patients with IgAN traditionally considered ‘low-risk’, with proteinuria less than one gram per day, had high rates of kidney failure.
  • “Patients with IgA nephropathy need both early and effective treatment options capable of significant and sustained reduction of proteinuria to preserve kidney function,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics.
  • “We’re pleased to be presenting 24-week data on proteinuria reduction, blood pressure control, and safety from the ongoing SPARTAN Study evaluating the effect of sparsentan as a first-line treatment option in patients with newly diagnosed IgAN.”
    Sparsentan Treatment of Incident Patients With IgA Nephropathy as First-line Therapy: Interim Findings From the SPARTAN Trial

Travere Therapeutics Announces Confirmatory Data from the Phase 3 PROTECT Study of FILSPARI® Demonstrating Long-Term Kidney Function Preservation in IgA Nephropathy; Narrowly Missing eGFR Total Slope Endpoint versus Active Control, Irbesartan

Retrieved on: 
Thursday, September 21, 2023
Medicine, Kidney failure, International, Heart Protection Study, CMA, Jay Traver, European Medicines Agency, Clinical trial, EGFR, European Directive on Traditional Herbal Medicinal Products, CSL Vifor, Immunoglobulin A, Dialysis, Committee, EMA, Proteinuria, Conditional, Nephrology, ANCOVA, Shanda, Publication, Patient, Division, Risk, Irbesartan, Kidney transplantation, Webcast, Committee for Medicinal Products for Human Use, FDA, Therapy, PROTECT, Kidney disease, Safety, CHMP, Hydraulic machinery, Medical device, Pharmaceutical industry, EU, IgA nephropathy

This outcome is incredibly important for IgAN patients, who face the risk of progression to kidney failure in their lifetime.

Key Points: 
  • This outcome is incredibly important for IgAN patients, who face the risk of progression to kidney failure in their lifetime.
  • “Since our accelerated approval, we’ve continued to hear inspiring stories of the impact this medicine is having on people living with IgAN.
  • While eGFR total slope narrowly missed statistical significance, the overall evidence from PROTECT suggests potential long-term benefit of FILSPARI as a foundational treatment for patients with IgAN.
  • Following the live webcast, an archived version of the call will be available for 30 days on the Company’s website.