Autosomal dominant polycystic kidney disease

Mironid appoints Dr Catherine Kelleher as Chief Medical Officer

Retrieved on: 
Tuesday, October 24, 2023
University of Colorado School of Medicine, Cass, Therapy, Rare, ADPKD, Nephrology, Drug, Drug development, Senior, Amgen, University, Patient, Autosomal dominant polycystic kidney disease, Associate, Rheumatology, Pharmaceutical industry, Medicine

October 24 2023, Glasgow, Scotland – Mironid, a biopharmaceutical company developing small molecule therapeutics for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), a serious hereditary kidney disease, today announces the appointment of Dr Catherine (Cass) Kelleher as Chief Medical Officer.

Key Points: 
  • October 24 2023, Glasgow, Scotland – Mironid, a biopharmaceutical company developing small molecule therapeutics for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), a serious hereditary kidney disease, today announces the appointment of Dr Catherine (Cass) Kelleher as Chief Medical Officer.
  • Cass most recently served as Chief Medical Officer at Anteris Bio and previously as Senior Vice President of Clinical Development at ChemoCentryx.
  • “Mironid has the potential to improve the ADPKD disease space by offering a first-in-class, innovative molecule to treat patients with ADPKD and possibly other rare diseases,” commented Dr Cass Kelleher, Chief Medical Officer of Mironid.
  • “I look forward to helping make this a reality, working closely with the executive team at Mironid as we work to progress our treatment into the clinic.”
    “I am delighted to welcome Cass as Chief Medical Officer to the team here at Mironid,” commented Dr Neil Wilkie, Chief Executive Officer of Mironid.

Mironid extends Series A round raising £35 million to date for development of first-in-class small molecules to treat life-threatening hereditary kidney disease

Retrieved on: 
Wednesday, September 13, 2023
European Investment Fund, Camp, Kidney failure, Drug development, University, Therapy, AMP, Polyuria, Sofinnova, Cell proliferation, Autosomal dominant polycystic kidney disease, PKD2, Cyst, Patient, University of Strathclyde, PKD1, Series, AICA ribonucleotide, Kidney, Growth, ADPKD, Scottish Enterprise, IND, Disease, Pharmaceutical industry

The Company will use the proceeds to progress its lead discovery programme through IND-enabling studies and to extend its patent estate.

Key Points: 
  • The Company will use the proceeds to progress its lead discovery programme through IND-enabling studies and to extend its patent estate.
  • It affects over 12 million people worldwide, with 50% of patients developing kidney failure by the age of 60.
  • The disease is caused predominantly by mutations in the PKD1 or PKD2 gene and is characterized by uncontrolled growth of fluid-filled cysts in the kidney.
  • Robust in vitro and disease model data show significant efficacy across all disease endpoints, including a reduction in cyst number and kidney volume.

Poxel Reports Cash and Revenue for the First Quarter 2023 and Provides Corporate Update

Retrieved on: 
Wednesday, May 17, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Adrenoleukodystrophy, Prescription, Imeglimin, Adenosine, Plasma, Journal, JPY, Patient, Serono, Merck, POC, Sumitomo Group, Disease, Male, Business development, FY, Pharmacokinetics, Autosomal dominant polycystic kidney disease, American Association for the Study of Liver Diseases, USD, European Commission, Polycystic kidney disease, Kidney International, HIV disease progression rates, Fatty liver disease, ADPKD, FDA, IPF, Safety, Abstract, DPP4, ALD, ODD, JDS, Merck Serono, Biomarker, DSM-IV codes, AMN, Non-U.S. operators of the M60 Patton, IRIS, Sumitomo Pharma, SGLT2, VLCFA, Life Sciences (journal), Regulation of tobacco by the U.S. Food and Drug Administration, Food, Autosome, Pharmaceutical industry, Bank, Management, EUR, Euronext

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today provided a corporate update and announced its cash position and revenue for the first quarter ended March 31, 2023.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today provided a corporate update and announced its cash position and revenue for the first quarter ended March 31, 2023.
  • Thomas Kuhn, Chief Executive Officer of Poxel, stated: “This quarter we successfully restructured our debt obligations and increased our financial flexibility with a new equity-linked financing with IRIS.
  • For the Sumitomo FY 2023, as a conservative assumption in line with Sumitomo’s forecast, Poxel expects to receive 8% royalties on TWYMEEG net sales.
  • As of March 31, 2023, cash and cash equivalents were EUR 10.6 million (USD 11.6 million), as compared to EUR 13.1 million (USD 14.0 million) as of December 31, 2022.

Poxel Reports Financial Results for Full Year 2022 and Provides Corporate Update

Retrieved on: 
Thursday, March 23, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Journal of Hepatology, Prescription, Imeglimin, Adenosine, Plasma, Journal, Magnetic resonance imaging, JPY, Patient, Serono, Histology, Conditional sentence, Merck, POC, Pioglitazone, Sumitomo Group, Disease, Male, Pharmacokinetics, Autosomal dominant polycystic kidney disease, European Commission, Kidney International, HIV disease progression rates, Fatty liver disease, ADPKD, FDA, IPF, Safety, ALD, ODD, Merck Serono, Medicine, Biomarker, DSM-IV codes, Fibrosis, Human, Adrenoleukodystrophy, Acceleration, AMN, Workforce, Partnership, IRIS, Sumitomo Pharma, Edema, Clinical trial, VLCFA, EUR, Life Sciences (journal), Regulation of tobacco by the U.S. Food and Drug Administration, Food, TZD, Pharmaceutical industry, Management, Security (finance), Euronext

POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), a clinical stage biopharmaceutical company developing innovative treatments for serious chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its results for the year ended December 31, 2022 and provided a corporate update.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), a clinical stage biopharmaceutical company developing innovative treatments for serious chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its results for the year ended December 31, 2022 and provided a corporate update.
  • Based on recent sales trends, Sumitomo has increased its fiscal year 20223 forecast by 20% to JPY 1.8 billion6 (EUR 12.8 million)5.
  • For the Sumitomo fiscal year 2023 (ending March 31, 2024), as a conservative assumption, Poxel expects to receive 8% royalties on TWYMEEG net sales.
  • Beyond 2024, Poxel expects to receive escalating double-digit royalties as well as additional sales-based payments upon achievement of contractually based sales thresholds.

Poxel Announces Publication in Kidney International of PXL770 Preclinical Results in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Wednesday, March 1, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Medicine, Kidney International, MD, ADPKD, Disease, AMPK, DSM-IV codes, Fatty liver disease, Adenosine, Kidney, Life Sciences (journal), Autosomal dominant polycystic kidney disease, NASH, HIV disease progression rates, Conditional sentence, Euronext, Tolvaptan, Pharmaceutical industry

POXEL SA (Paris:POXEL) (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for serious chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, announced today the publication of preclinical results in autosomal dominant polycystic kidney disease (ADPKD) for PXL770, a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator.

Key Points: 
  • POXEL SA (Paris:POXEL) (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for serious chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, announced today the publication of preclinical results in autosomal dominant polycystic kidney disease (ADPKD) for PXL770, a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator.
  • To access the publication online in the life sciences journal, Kidney International, please use the following link: A novel direct adenosine monophosphate kinase activator ameliorates disease progression in preclinical models of Autosomal Dominant Polycystic Kidney Disease.
  • (kidney-international.org)
    “ADPKD remains a major cause of end stage renal disease and is associated with substantial additional unmet medical needs.
  • These results confirm the potential utility of AMPK activation for this disease and support the development of PXL770 in a Phase 2 clinical program for ADPKD.”

Poxel Reports Cash and Revenue for the Full Year 2022 and Provides Corporate Update

Retrieved on: 
Wednesday, February 15, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, NASH, Sumitomo Pharma, Biomarker, CFO, ALD, ODD, European Commission, Edema, Acceleration, ADPKD, Prescription, Safety, Human, Patient, Clinical trial, Workforce, Fibrosis, Autosomal dominant polycystic kidney disease, Merck Serono, Serono, Food, Adrenoleukodystrophy, Histology, EUR, POC, IRIS, Merck, IPF, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Male, USD, VLCFA, Plasma, Fatty liver disease, Disease, FDA, Magnetic resonance imaging, Pioglitazone, TZD, JPY, Imeglimin, DSM-IV codes, COVID-19, AMN, Pharmacokinetics, Pharmaceutical industry, Security (finance), Management, Sumitomo Group, Euronext

Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We are very pleased with the most recent figures of TWYMEEG sales in Japan.

Key Points: 
  • Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We are very pleased with the most recent figures of TWYMEEG sales in Japan.
  • For the Sumitomo fiscal year 2023 (ending March 31, 2024), as a conservative assumption Poxel expects to receive 8% royalties on TWYMEEG net sales.
  • In Q4 2022, Poxel initiated a corporate savings plan which includes a significant workforce reduction.
  • Full-Year Cash and Revenue ended December 31, 2022
    As of December 31, 2022, total cash and cash equivalents were EUR 13,1 million (USD 14 million)3, as compared to EUR 32,3 million at December 31, 2021 and EUR 17.1 million at September 30, 2022.

XORTX Announces Submission of Orphan Drug Designation Request for XRx-008 Program to Treat Autosomal Dominant Kidney Disease

Retrieved on: 
Wednesday, February 1, 2023
Marketing, Society, ADPKD, Food, Plasma protein binding, Kidney disease, PKD, Oxipurinol, Orphan Drug Act of 1983, Drug development, Cyst, Hyperuricemia, American Society of Nephrology, ODD, FDA, US Foods, Autosomal dominant polycystic kidney disease, Degenerative disease, Therapy, Pharmaceutical industry

CALGARY, Alberta, Feb. 01, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce it submitted an Orphan Drug Designation (“ODD”) Request to the US Food and Drug Administration (“FDA”) for the XRx-008 program for the treatment of Autosomal Dominant Polycystic Kidney Disease (“ADPKD”).

Key Points: 
  • CALGARY, Alberta, Feb. 01, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce it submitted an Orphan Drug Designation (“ODD”) Request to the US Food and Drug Administration (“FDA”) for the XRx-008 program for the treatment of Autosomal Dominant Polycystic Kidney Disease (“ADPKD”).
  • XORTX anticipates updates regarding the ODD application and will provide announcements as information becomes available.
  • XORTX intends to pursue these benefits as part of the drug development for XRx-008 for treatment of ADPKD, pending designation of the ODD Request.
  • A tax credit of 25% of the qualified clinical drug testing costs awarded upon drug approval is also possible.

Poxel Provides Corporate Update and Reports Cash and Revenue for the Third Quarter and Nine Months 2022

Retrieved on: 
Tuesday, November 8, 2022
Oncology, Health, Research, Pharmaceutical, Science, Biotechnology, Food, Plasma, Prescription, Adrenoleukodystrophy, VLCFA, TZD, ALD, Growth, Fibrosis, FDA, Patient, Association, Company, MD, Biomarker, Serono, NASH, Merck Serono, AMN, ODD, Clinical trial, Orphan, Institute of Liver and Biliary Sciences, American Association, ADPKD, Disease, Fatty liver disease, POC, IPF, American Association for the Study of Liver Diseases, Non-alcoholic fatty liver disease, USD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Histology, Merck, Autosomal dominant polycystic kidney disease, Magnetic resonance imaging, Human, JPY, Degenerative disease, Edema, Pioglitazone, EUR, IRIS, Safety, AASLD, Male, Pharmacokinetics, Type 2 diabetes, Pharmaceutical industry, Euronext, Imeglimin

The royalty stream from TWYMEEGs growth is poised to deliver significant value to Poxel, noted Thomas Kuhn, Chief Executive Officer of Poxel.

Key Points: 
  • The royalty stream from TWYMEEGs growth is poised to deliver significant value to Poxel, noted Thomas Kuhn, Chief Executive Officer of Poxel.
  • This is an important milestone for Poxel, but also more broadly for NASH, for which there is still no approved treatment.
  • Based on these results, we are prioritizing PXL065 in NASH and are actively looking for a partner to advance its development.
  • Third Quarter and Nine Months Ended September 30, 2022 Cash and Revenue
    As of September 30, 2022, total cash and cash equivalents were EUR 17.1 million (USD 16.6 million), as compared to EUR 16.1 million at June 30, 2022.

Global Autosomal Dominant Polycystic Kidney Disease Epidemiology Forecast Report to 2032 - ResearchAndMarkets.com

Retrieved on: 
Friday, October 7, 2022
Genetics, Pharmaceutical, Health, Arachnoid, Epidemiology, Cyst, Hypertension, Colin L. Masters, Doctor of Philosophy, ERM, PKD2, Growth, Population, Diagnosis, Mitral valve prolapse, Kidney failure, Patient, Urinary tract infection, Degenerative disease, Renal cysts and diabetes syndrome, Kidney, Diverticular disease, Polycystic kidney disease, Hernia, Stabat Mater, Hematuria, Adult, Liver, United Kingdom, Fibrosis, PKD1, Master's degree, Intracranial aneurysm, Autosome, Left ventricular hypertrophy, Chronic kidney disease, Pancreas, Autosomal dominant polycystic kidney disease, PKD, Vaccine, Pharmaceutical industry, Lithium, Medical device, Health insurance, Management

The "Autosomal Dominant Polycystic Kidney Disease - Epidemiology Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Autosomal Dominant Polycystic Kidney Disease - Epidemiology Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.
  • This report delivers an in-depth understanding of Autosomal Dominant Polycystic Kidney Disease, historical and forecasted epidemiology as well as Autosomal Dominant Polycystic Kidney Disease trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
  • The diagnosed prevalent cases of Autosomal Dominant Polycystic Kidney Disease are likely to change by 2032 in the forecast period 2022-2032.
  • The report provides the segmentation of the disease epidemiology for the 7MM, a total number of total Diagnosed Prevalent Cases of Autosomal Dominant Polycystic Kidney Disease, Age-specific cases of Autosomal Dominant Polycystic Kidney Disease, and Mutation-specific cases of Autosomal Dominant Polycystic Kidney Disease.

Poxel Announces PXL770 Granted Orphan Drug Designation from the U.S. FDA for Autosomal-Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Monday, October 3, 2022
Research, FDA, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Oncology, ODD, Inflammation, Degenerative disease, Congress, VP, Adenosine, Autosomal dominant polycystic kidney disease, Pharmacology, Scientific communication, Euronext Paris, Kidney failure, ALD, Food, AMPK, Lists of diseases, CEO, Chronic kidney disease, FDA, Polycystic kidney disease, R, NASH, Fatty liver disease, Partnership, Lipid metabolism, Kidney, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Autosome, Dialysis, Patient, Adrenoleukodystrophy, Forward-looking statement, Abdominal pain, Kidney disease, Therapy, Company, Senior, Pharmaceutical industry, Medical device, ADPKD, Imeglimin, Euronext

ADPKD is an inherited form of chronic kidney disease where the unmet need is high, with only one approved drug which is associated with significant safety-tolerability challenges.

Key Points: 
  • ADPKD is an inherited form of chronic kidney disease where the unmet need is high, with only one approved drug which is associated with significant safety-tolerability challenges.
  • The pathophysiology of ADPKD involves altered kidney metabolism and there is a strong rationale for AMPK activation as a therapeutic approach.
  • Autosomal dominant polycystic kidney disease (ADPKD) is the fourth leading cause of renal failure and the most common inherited cause of kidney disease.
  • 1 For more information on Orphan Drug Designation, see : https://www.fda.gov/industry/developing-products-rare-diseases-condition...
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