UPCR

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.

Alpine Immune Sciences Shares Updated Clinical Data from Povetacicept in IgA Nephropathy

Retrieved on: 
Wednesday, April 10, 2024
Health, Other Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, FDA, Congress, Immunoglobulin G, ALPN, University, Glomerular filtration rate, Proteinuria, Survival, Cytokine, MD, Hematuria, UPCR, Webcast, Immunoglobulin A, EGFR, Disease, Nephrology, Patient, WCN, Alpine, Inflammation, Multimedia, Infection, Biomarker, Exhibition, University of Leicester, Autoimmunity, BAFF, Pharmaceutical industry

Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today shared updated clinical data for povetacicept in IgA nephropathy (IgAN) which will be presented as a late breaking poster at the World Congress of Nephrology (WCN) April 13-16, 2024 in Buenos Aires, Argentina.

Key Points: 
  • Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today shared updated clinical data for povetacicept in IgA nephropathy (IgAN) which will be presented as a late breaking poster at the World Congress of Nephrology (WCN) April 13-16, 2024 in Buenos Aires, Argentina.
  • RUBY-3 is a multiple ascending dose, multi-cohort, open label, phase 1b/2a study of povetacicept in autoimmune glomerulonephritis, including IgA nephropathy, where povetacicept is administered subcutaneously (SC) once every four weeks.
  • As of March 01, 2024, 41 patients with IgAN had received povetacicept 80 or 240 mg subcutaneously every 4 weeks.
  • These data strongly support the inhibition of APRIL/BAFF pathways by povetacicept and its efficacy in the treatment of IgAN as well as the need for further clinical development.

Calliditas Therapeutics Presents Additional Data Analyses from the Phase 3 NeflgArd trial of Nefecon in Primary IgA Nephropathy at the ISN World Congress of Nephrology 2024

Retrieved on: 
Thursday, April 18, 2024
Congress, CALT, UPCR, Immunoglobulin A, EGFR, Patient, Budesonide, ISN, Pharmaceutical industry

STOCKHOLM, April 18, 2024 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas"), today announced additional data analyses from the 2-year Phase 3 NeflgArd trial evaluating Nefecon (TARPEYO® (budesonide) delayed-release capsules/Kinpeygo®) in patients with IgA nephropathy (IgAN), were presented at the ISN World Congress of Nephrology in Buenos Aires, Argentina on April 13-16, 2024.

Key Points: 
  • STOCKHOLM, April 18, 2024 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas"), today announced additional data analyses from the 2-year Phase 3 NeflgArd trial evaluating Nefecon (TARPEYO® (budesonide) delayed-release capsules/Kinpeygo®) in patients with IgA nephropathy (IgAN), were presented at the ISN World Congress of Nephrology in Buenos Aires, Argentina on April 13-16, 2024.
  • "We were pleased to share additional analyses from the 2-year Phase 3 NeflgArd trial of Nefecon in IgAN at this year's World Congress of Nephrology," said Richard Philipson, Chief Medical Officer of Calliditas.
  • "These additional data further reinforce the impact of Nefecon across the entire study population, irrespective of baseline UPCR levels or patient's racial and ethnic backgrounds."
  • These findings strongly suggest preserved kidney function and provide support for Nefecon as a disease-modifying therapy in patients with IgAN.

Calliditas Therapeutics Presents Additional Data Analyses from the Phase 3 NeflgArd trial of Nefecon in Primary IgA Nephropathy at the ISN World Congress of Nephrology 2024

Retrieved on: 
Thursday, April 18, 2024
Congress, CALT, UPCR, Immunoglobulin A, EGFR, Patient, Budesonide, ISN, Pharmaceutical industry

STOCKHOLM, April 18, 2024 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas"), today announced additional data analyses from the 2-year Phase 3 NeflgArd trial evaluating Nefecon (TARPEYO® (budesonide) delayed-release capsules/Kinpeygo®) in patients with IgA nephropathy (IgAN), were presented at the ISN World Congress of Nephrology in Buenos Aires, Argentina on April 13-16, 2024.

Key Points: 
  • STOCKHOLM, April 18, 2024 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas"), today announced additional data analyses from the 2-year Phase 3 NeflgArd trial evaluating Nefecon (TARPEYO® (budesonide) delayed-release capsules/Kinpeygo®) in patients with IgA nephropathy (IgAN), were presented at the ISN World Congress of Nephrology in Buenos Aires, Argentina on April 13-16, 2024.
  • "We were pleased to share additional analyses from the 2-year Phase 3 NeflgArd trial of Nefecon in IgAN at this year's World Congress of Nephrology," said Richard Philipson, Chief Medical Officer of Calliditas.
  • "These additional data further reinforce the impact of Nefecon across the entire study population, irrespective of baseline UPCR levels or patient's racial and ethnic backgrounds."
  • These findings strongly suggest preserved kidney function and provide support for Nefecon as a disease-modifying therapy in patients with IgAN.

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Monday, April 8, 2024
Diabetes, Anaphylaxis, IgA nephropathy, Glaucoma, CYP3A4, Dermatitis, Patient, Parasitism, Indinavir, Budesonide, Ritonavir, Quality of life, Peripheral edema, UPCR, Drug interaction, Vaccine, Saquinavir, Erythromycin, Congress, White, Risk, Headache, Spasm, Ketoconazole, Immunoglobulin A, Liver, Adrenal insufficiency, Immune system, Prediabetes, Proteinuria, Hypertension, Contraindication, Arthralgia, Ciclosporin, International Society, Immunosuppression, KOL, Acne, Infection, EGFR, Measles, Miscarriage, ISN, Hypersensitivity, Pregnancy, HPA, Chickenpox, Fetus, Birth, Itraconazole, CALT, Upper respiratory tract infection, Osteoporosis, Kidney, Cataract, Tropical ulcer, Pharmaceutical industry

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Monday, April 8, 2024
Diabetes, Anaphylaxis, IgA nephropathy, Glaucoma, CYP3A4, Dermatitis, Patient, Parasitism, Indinavir, Budesonide, Ritonavir, Quality of life, Peripheral edema, UPCR, Drug interaction, Vaccine, Saquinavir, Erythromycin, Congress, White, Risk, Headache, Spasm, Ketoconazole, Immunoglobulin A, Liver, Adrenal insufficiency, Immune system, Prediabetes, Proteinuria, Hypertension, Contraindication, Arthralgia, Ciclosporin, International Society, Immunosuppression, KOL, Acne, Infection, EGFR, Measles, Miscarriage, ISN, Hypersensitivity, Pregnancy, HPA, Chickenpox, Fetus, Birth, Itraconazole, CALT, Upper respiratory tract infection, Osteoporosis, Kidney, Cataract, Tropical ulcer, Pharmaceutical industry

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

Vera Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 20, 2024
Quarter, Disease, Patient, UPCR, Clinical trial, Therapy, EGFR, Proteinuria, FDA, BLA, Hematuria, Pharmaceutical industry

“We believe atacicept 72-week data from the Phase 2b ORIGIN trial are consistent with a profile of true disease modification.

Key Points: 
  • “We believe atacicept 72-week data from the Phase 2b ORIGIN trial are consistent with a profile of true disease modification.
  • The ORIGIN 2b results also provide us with even greater confidence in the ongoing ORIGIN Phase 3 trial, which continues to be on track with regard to enrollment,” stated Robert Brenner, M.D., Chief Medical Officer of Vera Therapeutics.
  • During the year ended December 31, 2023, net cash used in operating activities was $92.2 million, compared to $67.6 million for the same period last year.
  • Vera reported $160.7 million in cash, cash equivalents, and marketable securities as of December 31, 2023.

IgA Nephropathy Pipeline Dynamics Signal Further Treatment Evolution on the Horizon

Retrieved on: 
Thursday, March 14, 2024
Nephrology, Indoor Living, Novartis, Patient, Alnylam Pharmaceuticals, Perception, SGLT2, Immunoglobulin A, Budesonide, NDA, Record, Breakthrough therapy, UPCR, Atrasentan, Kidney, EGFR, Alexion, FDA, CHMP, Physician, Health, Pharmaceutical industry

For several years, Spherix Global Insights has been closely tracking the IgAN market and recently released its fourth annual US patient chart audit study.

Key Points: 
  • For several years, Spherix Global Insights has been closely tracking the IgAN market and recently released its fourth annual US patient chart audit study.
  • Spherix is the leading authority on IgAN market dynamics and will continue to provide insight into how nephrologists are treating and approaching care for their IgAN patients.
  • Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits.
  • Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.

Vertex Advances Inaxaplin (VX-147) into Phase 3 Portion of Adaptive Phase 2/3 Clinical Trial for the Treatment of APOL1-Mediated Kidney Disease

Retrieved on: 
Monday, April 1, 2024
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, BTD, Orphan drug, APOL1, RPD, Disease, Patient, Apolipoprotein L1, FSGS, Standard of care, EMA, UPCR, European Medicines Agency, Vertex Pharmaceuticals, Clinical trial, Vertex, Proteinuria, Food, FDA, IDMC, VRTX, Pharmaceutical industry

The clinical trial is designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with proteinuric kidney disease mediated by two variants in the APOL1 gene, known as AMKD.

Key Points: 
  • The clinical trial is designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with proteinuric kidney disease mediated by two variants in the APOL1 gene, known as AMKD.
  • In addition, the trial has been expanded to include adolescents with AMKD ages 10 to 17 years.
  • The IDMC also recommended enrolling adolescents with AMKD ages 10 to 17 years in the Phase 3 portion of the study.
  • The European Medicines Agency (EMA) has also granted inaxaplin Priority Medicines (PRIME) and Orphan Drug designations for AMKD.

Alpine Immune Sciences Provides Corporate Update and Full Year 2023 Financial Results

Retrieved on: 
Monday, March 18, 2024
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Research program, Administration, Congress, Development, WT, Lupus, Patient, Immunoglobulin A, Glomerular filtration rate, SLE, Inflammation, MD, UPCR, Neonatal Fc receptor, American College, Investment, Clinical trial, Society, EGFR, Nephrology, BAFF, Proteinuria, Immunoglobulin G, Autoimmunity, ALPN, Pharmaceutical industry

Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today reported full year 2023 financial results and company highlights for the fourth quarter ended December 31, 2023.

Key Points: 
  • Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today reported full year 2023 financial results and company highlights for the fourth quarter ended December 31, 2023.
  • “2023 was a transformational year for Alpine, with initial IgA nephropathy (IgAN) data presented at the American Society of Nephrology Kidney Week 2023 suggesting a best-in-class profile for povetacicept, our next-generation dual BAFF/APRIL inhibitor.
  • In addition, povetacicept was well tolerated during subcutaneous administration, with no instances of IgG
  • Net Loss: Net loss for the year ended December 31, 2023, was $32.2 million compared to $57.8 million for the same period in 2022.