PKD

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.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, PKD, Personalized medicine, EMA, Research, MAA, Investment, Marketing, Food, Arrhythmogenic cardiomyopathy, Circulation, BLA, PDUFA, Fanconi anemia, Conference, Safety, DCM, European Medicines Agency, Food and Drug Administration, Muscle weakness, Efgartigimod alfa, Pyruvate kinase deficiency, Patient, Disorder, Clinical trial, Medication, ACM, IND, AAV, CGMP, CMC, Priority review, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

PYC'S FOURTH DRUG CANDIDATE HAS DISEASE-MODIFYING POTENTIAL IN POLYCYSTIC KIDNEY DISEASE

Retrieved on: 
Monday, November 13, 2023
SAN, Food, Therapy, US Food Sovereignty Alliance, Kidney disease, Disease, PYC, IND, Patient, DSM-IV codes, Probability, FDA, Kidney, PKD, New Drug Application, Human, RNA, Swelling, 3D, Cyst, AEDST, Mutation, Drug development, Pharmaceutical industry, Cryptocurrency, Vaccine, Drug discovery

PERTH, Australia and SAN FRANCISCO, Nov. 13, 2023 /PRNewswire/ -- PYC Therapeutics today announces the results of a study conducted in human 3-dimensional models derived from patients with end-stage renal failure due to Autosomal Dominant Polycystic Kidney Disease (PKD). The results demonstrate that an investigational drug candidate designed by PYC (known as PYC-003) to address this disease at its root cause is effective. These 3D patient-derived cyst models represent the 'gold-standard' pre-clinical assay for evaluating drug candidates in this indication[8].

Key Points: 
  • The results demonstrate that an investigational drug candidate designed by PYC (known as PYC-003) to address this disease at its root cause is effective.
  • PYC-003 is the fourth program in the Company's development pipeline – joining three other first-in-class RNA drug candidates with disease-modifying potential currently progressing into or through human trials.
  • An Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) to enable the commencement of human trials for this drug candidate is planned for H2 2024.
  • A New Drug Application in support of this candidate could be submitted following two clinical trials rather than the conventional three[11].

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.

Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, November 6, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Consensus, Patient, Research, Busulfan, American College, CGMP, Genetic testing, Clinical trial, AAV, PDUFA, Incidence, IMPD, LV, Danon disease, Malignancy, Medication, Mortality, CMC, Conference, CTA, Acquisition, PKP2, Disease, JACC, Journal, Consensus decision-making, Priority review, Male, PKD, Natural history, MHRA, Disorder, EMA, Arrhythmia, Journal of the American College of Cardiology, LAMP2, Safety, Investment, Diagnosis, BLA, Pediatric Blood & Cancer, Pharmaceutical industry, Medical device, Security (finance), Fine chemical, G&A

Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.

Key Points: 
  • Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.
  • Highlights of the publication include a review of diagnosing Danon Disease emphasizing the importance of genetic testing upon clinical suspicion, natural history, management recommendations and recent advances in potential gene therapy treatment.
  • Rocket is working towards initiation of Phase 2 pivotal trial activities in Europe and the UK.
  • As of September 30, 2023, Rocket had cash, cash equivalents and investments of $437.2 million.

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

Retrieved on: 
Thursday, October 19, 2023
Research, Genetics, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Patient, PKP2, Medication, Danon disease, MBA, Arrhythmogenic cardiomyopathy, Pharming, PKD, Cell, Fanconi anemia, Disorder, Gene, ESGCT, European Society of Gene and Cell Therapy, Pyruvate kinase deficiency, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27.
  • Updated data will be presented from the Phase 1 clinical trial of RP-L301 for Pyruvate Kinase Deficiency (PKD).
  • Previously disclosed data will be presented from the Phase 2 pivotal trial of RP-L102 for Fanconi Anemia, Phase 1 trial of RP-A501 for Danon Disease and preclinical studies supporting the Phase 1 trial of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).
  • Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma, will also give an Invited Talk about the importance of collaboration with and commitment to patients in gene therapy development.

XORTX Sponsored Study Selected for Presentation at the American Society of Nephrology – Kidney Week 2023

Retrieved on: 
Thursday, September 28, 2023
Session, University of Colorado Colorado Springs, University, Xanthine oxidase, Effect, American Society of Nephrology, PKD, Research, ASN, Uric acid, Polycystic kidney disease, Patent, ADPKD, Prognosis, Therapy, Kidney, Society, Kidney disease, Pharmaceutical industry

The abstract outlines study results from a mouse model of polycystic kidney disease challenged with low to moderate circulating uric acid.

Key Points: 
  • The abstract outlines study results from a mouse model of polycystic kidney disease challenged with low to moderate circulating uric acid.
  • The study also identifies a novel inflammatory profile as a possible mechanism of injury inducing an increased cyst index and kidney enlargement.
  • XORTX will provide further details of the study at the time of the presentation during the first week of November at the ASN meeting scheduled for November 2, 2023.
  • XORTX granted patents and additional patent applications supporting the XRx-008 program are wholly owned by XORTX.”