Polycystic kidney disease

XORTX Announces Publication of Key Research in ADPKD

Retrieved on: 
Monday, April 22, 2024
Kidney disease, University, Xanthine oxidase, Mouse, Kidney, Uric acid, XO, Prognosis, American Journal, Cyst, Research, Oxipurinol, SUA, Disease, Patient, Serum, Polycystic kidney disease, Therapy, PKD, Pharmaceutical industry

This study shows, for the first time, that chronically increased SUA can significantly increase cyst index and increase kidney size in ADPKD.

Key Points: 
  • This study shows, for the first time, that chronically increased SUA can significantly increase cyst index and increase kidney size in ADPKD.
  • Dr. Allen Davidoff, CEO of XORTX, stated, “We are pleased to have supported this pioneering research in polycystic kidney disease by Dr. Charles Edelstein of the University of Colorado.
  • In human ADPKD, kidney size and declining kidney filtering capacity are correlated and are key indicators of disease progression and prognosis.
  • These mechanistic studies are important for the planning of future clinical studies of xanthine oxidase inhibition in patients with ADPKD.”

Eloxx Pharmaceuticals Provides ELX-02 and ZKN-013 Program Updates

Retrieved on: 
Tuesday, April 16, 2024
FDA, Genetics, Drug discovery, ADPKD, Mutation, IPS, Food, Prevalence, Proteinuria, Organoid, OTC, Kidney, Gene, Orphan, RNA, Partnership, UPCR, Almirall, U.S. FDA, FAP, IND, RDEB, Disease, SAD, Patient, Biopsy, Clinical trial, Polycystic kidney disease, ODD, Food and Drug Administration, PKD, Pharmaceutical industry

WATERTOWN, Mass., April 16, 2024 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today provided program updates for ELX-02 and ZKN-013, including Orphan Drug Designation (ODD) for ELX-02.

Key Points: 
  • Eloxx has significantly advanced the development of ELX-02 for the treatment of Alport syndrome with Nonsense Mutations (NMAS).
  • “The recent ELX-02 program updates, including Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome highlights the significant unmet medical needs of Alport Syndrome patients with Nonsense Mutations,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • The designation was based on a review of the prevalence of NMAS and the data from the Proof-of-concept Phase 2 Study that Eloxx announced top-line results for in 2023.
  • In March 2024, Eloxx announced an exclusive license agreement with Almirall to develop and commercialize ZKN-013 in orphan indications including RDEB and FAP with nonsense mutations.

XORTX Announces New Clinical Advisory Board Member

Retrieved on: 
Wednesday, March 27, 2024
Sanofi, Database, Internal medicine, Critical Path Institute, XRX, TEMPO, Patient, Research, Biomarker, TAME, Polycystic kidney disease, National Institutes of Health, MD, Kidney disease, European Medicines Agency, Publication, PKD, Clinical trial, Boston Medical Center, Therapy, Grady Memorial Hospital, FDA, Food, Pharmaceutical industry

CALGARY, Alberta, March 27, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a biopharmaceutical company focused on developing innovative therapies to treat autosomal dominant polycystic kidney disease (“ADPKD”), is pleased to announce that Dr. Ronald Perrone has joined the Company’s Clinical Advisory Board.

Key Points: 
  • CALGARY, Alberta, March 27, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a biopharmaceutical company focused on developing innovative therapies to treat autosomal dominant polycystic kidney disease (“ADPKD”), is pleased to announce that Dr. Ronald Perrone has joined the Company’s Clinical Advisory Board.
  • Dr. Allen Davidoff stated, “We are excited that Dr. Ron Perrone has agreed to join XORTX’s Clinical Advisory Board.
  • Dr. Perrone brings substantial medical and professional experience as a thought leader, combined with clinical experience treating patients with ADPKD and kidney disease.
  • We are privileged to have Dr. Perrone join our esteemed Clinical Advisory Board alongside current members, Dr. Petter Bjornstad, Dr. Richard Johnson, Dr. Federico Maese, Dr. Anjay Rostogi and Dr. Charles Edelstein.

XORTX Highlights Achievements of 2023 and Preparation for Registration Clinical Trial

Retrieved on: 
Tuesday, March 19, 2024
Consultant, Nephrology, Provisional application, XRX, Partnership, Chemistry, Kidney disease, Tablet, Orphan, Bryant University, University, EMA, Polycystic kidney disease, Trial of the century, Oxipurinol, MBA, GMP, Diet, PKD, Treatment, Society, Therapy, Patent, Rensselaer Polytechnic Institute, FDA, Marketing, Diagnosis, American Society of Nephrology, Uric acid, Pharmaceutical industry

Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals.

Key Points: 
  • Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals.
  • Each of these milestones permit the next step in the Company’s clinical development plan, being a “registration” clinical trial – XRX-OXY-201 in pursuit of accelerated approval and support of the Company’s lead program XRx-008 program for ADPKD.
  • Chemistry and Manufacturing, Clinical and Pre-Clinical Highlights – Produced drug substance for oxypurinol production and produced GMP drug substance; confirmed XORLO™ formulation, produced enhanced bioavailability and produced clinical supply of tablets for clinical trials.
  • August 29, 2023 – XORTX submitted an Orphan Drug Designation application to the EMA for the treatment of ADPKD.

Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Thursday, March 21, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Clinical Trials, Death, Autosome, VRTX, Growth, Disease, PC1, Patient, Kidney failure, Kidney transplantation, Polycystic kidney disease, IND, Vertex Pharmaceuticals, PKD1, Vertex, ADPKD, Dialysis, Cystic fibrosis, Food, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
  • ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease.
  • ADPKD is a life-shortening genetic kidney disease characterized by the growth of numerous kidney-enlarging cysts that impair kidney function and can ultimately lead to kidney failure, requiring dialysis or kidney transplantation, and premature death.
  • The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein.

C-Path Appoints Dr. Klaus Romero as New CEO

Retrieved on: 
Tuesday, January 9, 2024
Arizona State University, Patient, Therapy, FCP, Associate, CSO, Critical Path Institute, Tuberculosis, University, Society, MBA, Polycystic kidney disease, Alzheimer's disease, Disease, Drug development, Clinical pharmacology, MPH, Duchenne muscular dystrophy, International Society, Parkinson's disease, American College, Pharmacology, Kidney transplantation, Pharmaceutical industry

TUCSON, Ariz., Jan. 9, 2024 /PRNewswire/ -- Critical Path Institute (C-Path) today announced that Klaus Romero, M.D., M.S., FCP, has been named its new Chief Executive Officer. Romero, a seasoned clinician scientist, has been vital in positioning C-Path as an essential partner in accelerating drug development worldwide, significantly shaping scientific and regulatory pathways to expedite the delivery of therapies for people in urgent need.

Key Points: 
  • TUCSON, Ariz., Jan. 9, 2024 /PRNewswire/ -- Critical Path Institute (C-Path) today announced that Klaus Romero, M.D., M.S., FCP, has been named its new Chief Executive Officer.
  • "Assuming the position of CEO at C-Path represents a profound honor, coupled with a tremendous sense of responsibility," said Romero.
  • His ascension to CEO is a significant milestone in our mission to bring transformative therapies to patients who desperately need them."
  • Romero will continue to serve a dual role as CEO and CSO until the CSO position is filled.

XORTX Submits a New Patent for the Treatment of Chronic Kidney Disease

Retrieved on: 
Wednesday, January 3, 2024
DN, Immunoglobulin A, IgA nephropathy, Kidney disease, Diabetic nephropathy, Uric acid, Trial of the century, Oxipurinol, Therapy, Autosome, Pharmacokinetics, Focal segmental glomerulosclerosis, Patent, Chronic kidney disease, ADPKD, Treating, Study, Polycystic kidney disease, XRX, Safety, Lupus nephritis, XOI, CKD, Pharmaceutical industry

CALGARY, Alberta, Jan. 03, 2024 (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, announces submission of a new patent for the treatment of chronic kidney disease (“CKD”).

Key Points: 
  • CALGARY, Alberta, Jan. 03, 2024 (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, announces submission of a new patent for the treatment of chronic kidney disease (“CKD”).
  • This patent is designed to protect new discoveries and strategies for the treatment of individuals with varied degrees of kidney function in the setting of CKD.
  • This patent application is intended to claim new opportunities to enhance how the xanthine oxidase inhibitor class of drugs may be dosed in the future.
  • Importantly, how to further improve the safe and effective administration of this class of drugs, including oxypurinol.”

Eastern Michigan's Brian Dooley Chosen For 2023 Disney Spirit Award

Retrieved on: 
Monday, December 4, 2023
Walt Disney, EMU, Sports complex, Walt Disney World, Student, Polycystic kidney disease, Eastern Michigan University, Multimedia, ESPN, Victory Day, University of Michigan, EST, Family, Football, Film industry, Boilermaker

LAKE BUENA VISTA, Fla., Dec. 4, 2023 /PRNewswire/ -- Helping one's teammates is part of being a college football player. But Eastern Michigan offensive lineman Brian Dooley took that to another level, donating his scholarship to a teammate, an incredibly selfless act that earned him the 2023 Disney Spirit Award.

Key Points: 
  • But Eastern Michigan offensive lineman Brian Dooley took that to another level, donating his scholarship to a teammate, an incredibly selfless act that earned him the 2023 Disney Spirit Award.
  • Eastern Michigan University offensive lineman Brian Dooley is the recipient of the 2023 Disney Spirit Award.
  • Since 1996 the Disney Spirit Award has been presented every year by Walt Disney World Resort to the most inspirational figures in college football.
  • Dooley is the 28th recipient of the Disney Spirit Award and the first from Eastern Michigan University.

XORTX Sponsored Study Presented at the American Society of Nephrology – Kidney Week 2023

Retrieved on: 
Thursday, November 2, 2023
Uric acid, Session, University of Colorado Colorado Springs, ADPKD, Xanthine oxidase, Polycystic kidney disease, Effect, Cytokine, Research, Society, American Society of Nephrology, Acute kidney injury, PKD, ASN, Hyperuricemia, Kidney disease, University, Diet, Nephrology, Therapy, Patient, Oxipurinol, Pharmaceutical industry, Phosphorus, Prognosis

CALGARY, Alberta, Nov. 02, 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 the presentation of new research findings at the American Society of Nephrology meeting being held November 3, 2023 in Philadelphia. The abstract entitled "The Effect of Lowering Uric Acid with a Xanthine Oxidase Inhibitor on PKD in Mice” was reviewed by the ASN review panel for scientific merit and novel discoveries. The studies were conducted at the University of Colorado in the independent laboratory of Dr. Charles Edelstein and were sponsored by XORTX. The studies will be presented at Kidney Week during the Session Title: Genetic Diseases: Cystic - Therapeutic Investigations and Prognosis, November 2, 2023.

Key Points: 
  • The studies were conducted at the University of Colorado in the independent laboratory of Dr. Charles Edelstein and were sponsored by XORTX.
  • The studies will be presented at Kidney Week during the Session Title: Genetic Diseases: Cystic - Therapeutic Investigations and Prognosis, November 2, 2023.
  • This poster reports amalgamated results of studies in either rat and mouse models of polycystic kidney disease (“PKD”) challenged with low to moderate circulating uric acid.
  • The effect of increased uric acid on cyst growth – kidney size - and kidney function, as well as inflammatory markers was measured.

Poxel Announces its Participation at Upcoming Scientific Conference

Retrieved on: 
Monday, October 2, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, NASH, Adrenoleukodystrophy, Scientific communication, ALD, AMPK, DSM-IV codes, Adenosine, Metabolism, Polycystic kidney disease, Fatty liver disease, R, ADPKD, Pharmacology, Autosome, Pharmaceutical industry, Vaccine, Euronext

POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), 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 announced that it will participate at the upcoming 12th International Meeting on AMPK on October 4th, 2023.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), 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 announced that it will participate at the upcoming 12th International Meeting on AMPK on October 4th, 2023.
  • Sophie Bozec, Executive Vice President, R&D Pharmacology and Scientific Communication at Poxel, will host a virtual presentation on “Direct AMPK Activation: From Preclinical to Clinical.
  • Perspectives in Rare Metabolic Diseases”.
  • The presentation will review Poxel’s molecule PXL770, which is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, focused on rare diseases treatment, including adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD).