Alport syndrome

ZyVersa Therapeutics Announces IRB Approval of Phase 2a Clinical Trial Protocol to Evaluate Cholesterol Efflux Mediator™ VAR 200 in Patients with Diabetic Kidney Disease

Retrieved on: 
Monday, March 18, 2024
Alport syndrome, Diabetic nephropathy, VAR, Institutional review board, Lipid, Patient, Focal segmental glomerulosclerosis, Kidney disease, Fibrosis, Clinical trial, Kidney, Therapy, Chronic kidney disease, Safety, Dialysis, Cholesterol, IRB, Pharmaceutical industry

Cholesterol Efflux MediatorTM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression.

Key Points: 
  • Cholesterol Efflux MediatorTM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression.
  • WESTON, Fla., March 18, 2024 (GLOBE NEWSWIRE) -- ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for the treatment of renal and inflammatory diseases with high unmet medical needs, announces Institutional Review Board (IRB) approval of the Phase 2a clinical trial protocol to evaluate the efficacy and safety of Cholesterol Efflux Mediator VAR 200 in patients with diabetic kidney disease.
  • It is the first in human trial for VAR 200 intended to substantiate that the promising preclinical results demonstrated in three different animal models of kidney disease (diabetic kidney disease, focal segmental glomerulosclerosis, and Alport syndrome) translate to patients with kidney disease.
  • We are excited about the potential of Cholesterol Efflux MediatorTM VAR 200 to protect against kidney injury and reduce kidney disease progression.”

Calliditas Year-end report, January - December 2023

Retrieved on: 
Wednesday, February 21, 2024
Head, Sustainability, European Commission, Operating cost, Marketing, Food and Drug Administration, USD, National Medical Products Administration, FDA, Food, Risk, MAH, Patent, Patient, Alport syndrome, Trial of the century, NMPA

Operating income amounted to SEK 41.8 million and SEK 32.5 million for the three months ended December 31, 2023, and 2022, respectively.

Key Points: 
  • Operating income amounted to SEK 41.8 million and SEK 32.5 million for the three months ended December 31, 2023, and 2022, respectively.
  • Loss per share before and after dilution amounted to SEK 0.34 and SEK 0.07 for the three months ended December 31, 2023, and 2022, respectively.
  • Cash amounted to SEK 973.7 million and SEK 1,249.1 million as of December 31, 2023, and 2022, respectively.
  • Net sales amounted to SEK 1,206.9 million, of which TARPEYO net sales amounted to SEK 1,075.8 million, for the year ended December 31, 2023.

Calliditas Year-end report, January - December 2023

Retrieved on: 
Wednesday, February 21, 2024
Head, Sustainability, European Commission, Operating cost, Marketing, Food and Drug Administration, USD, National Medical Products Administration, FDA, Food, Risk, MAH, Patent, Patient, Alport syndrome, Trial of the century, NMPA

Operating income amounted to SEK 41.8 million and SEK 32.5 million for the three months ended December 31, 2023, and 2022, respectively.

Key Points: 
  • Operating income amounted to SEK 41.8 million and SEK 32.5 million for the three months ended December 31, 2023, and 2022, respectively.
  • Loss per share before and after dilution amounted to SEK 0.34 and SEK 0.07 for the three months ended December 31, 2023, and 2022, respectively.
  • Cash amounted to SEK 973.7 million and SEK 1,249.1 million as of December 31, 2023, and 2022, respectively.
  • Net sales amounted to SEK 1,206.9 million, of which TARPEYO net sales amounted to SEK 1,075.8 million, for the year ended December 31, 2023.

Orphan designation: 5-(3,4-Dichloro-phenyl)-N-((1R,2R)-2-hydroxy-cyclohexyl)-6-(2,2,2-trifluoro-ethoxy)-nicotinamide Treatment of Alport syndrome, 20/06/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Alport syndrome, Committee, Patient, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - 5-(3,4-Dichloro-phenyl)-N-((1R,2R)-2-hydroxy-cyclohexyl)-6-(2,2,2-trifluoro-ethoxy)-nicotinamide
    - Intended use
    - Treatment of Alport syndrome
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2791
    - Date of designation
    - Sponsor
    Veristat Spain S.L.
  • Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Orphan designation: vonafexor Treatment of Alport syndrome, 25/07/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Alport syndrome, Committee, EMA, Pharmaceutical industry

EU/3/23/2808 - orphan designation for treatment of Alport syndrome

Key Points: 
  • EU/3/23/2808 - orphan designation for treatment of Alport syndrome
    vonafexor
    OrphanHuman
    Enyo Pharma
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Orphan designation: setanaxib Treatment of Alport syndrome, 08/11/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Alport syndrome, Committee, Patient, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - setanaxib
    - Intended use
    - Treatment of Alport syndrome
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2850
    - Date of designation
    - Sponsor
    Calliditas Therapeutics AB
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

ZyVersa Therapeutics CEO, Stephen C. Glover, Issues Letter to Shareholders Providing Outlook for 2024

Retrieved on: 
Wednesday, January 3, 2024
Alport syndrome, Quality of life, Entrepreneurship, Inflammasome, NLRP1, Survival, IND, VAR, Letter, Kidney disease, Inflammation, FSGS, AIM2, Health, Human, Therapy, Clinical trial, Diabetic nephropathy, Focal segmental glomerulosclerosis, Kidney, Proteinuria, NLRP3, DKD, ACE, Optimism, SGLT2, Patient, Capital, Fibrosis, Dialysis, Environment, Pharmaceutical industry

Combined, our two proprietary product platforms target a global total addressable market of around $75 billion.

Key Points: 
  • Combined, our two proprietary product platforms target a global total addressable market of around $75 billion.
  • On the financial front, various market watchers are seeing signals that the biotech and pharma investment environment is set to trend positively in 2024.
  • Capital is again starting to flow to companies that are seeking to innovate through differentiated technologies or novel mechanisms of action.
  • We look forward to embarking on what we believe will be a productive 2024.

Eloxx Pharmaceuticals Reports Third Quarter 2023 Financial and Operating Results and Provides Business Update

Retrieved on: 
Monday, November 13, 2023
Sale, FSD, RDEB, ASN, Kidney, GBM, American Society of Nephrology, Patient, CFF, FDA, Glomerular basement membrane, G&A, Health, R, JEB, ATM, Society, TEM, OTC, RNA, Proteinuria, Biopsy, Pharmaceutical industry, Phosphorus, Microscope, Cryptocurrency, Alport syndrome

WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.

Key Points: 
  • WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.
  • Data from the Phase 2 study of ELX-02 for Alport Syndrome was included in two presentations at the American Society of Nephrology (ASN) Kidney Week 2023.
  • R&D expenses were $1.3 million for the three months ended September 30, 2023, which included $0.1 million in stock-based compensation.
  • Additionally, we sold shares of common stock through our ATM program during the quarter for gross proceeds of $1.8 million.

Calliditas Interim Report January - September 2023

Retrieved on: 
Tuesday, November 7, 2023
International Symposium on Endovascular Therapy, FDA, Operating cost, PDUFA, SEK, Nephrology, Magazine, ASN, NOX, USD, CMO, IgA nephropathy, Alport syndrome, Rare-earth element, Cryptocurrency, Pharmaceutical industry, Alport, EU, Lancet

Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.

Key Points: 
  • Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.
  • Significant Events in Q3 2023, in Summary
    On July 13 Calliditas announced supportive interim data from Phase 2 head and neck cancer trial with lead NOX inhibitor candidate, setanaxib.
  • Calliditas invites investors, analysts and press to a presentation of the Quarterly Report 2023 at 14:30 pm.
  • Calliditas' CEO Renee Aguiar-Lucander will present the report together with CFO Fredrik Johansson, CMO Richard Philipson and President North America Andrew Udell.

Calliditas Interim Report January - September 2023

Retrieved on: 
Tuesday, November 7, 2023
International Symposium on Endovascular Therapy, FDA, Operating cost, PDUFA, SEK, Nephrology, Magazine, ASN, NOX, USD, CMO, IgA nephropathy, Alport syndrome, Rare-earth element, Cryptocurrency, Pharmaceutical industry, Alport, EU, Lancet

Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.

Key Points: 
  • Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.
  • Significant Events in Q3 2023, in Summary
    On July 13 Calliditas announced supportive interim data from Phase 2 head and neck cancer trial with lead NOX inhibitor candidate, setanaxib.
  • Calliditas invites investors, analysts and press to a presentation of the Quarterly Report 2023 at 14:30 pm.
  • Calliditas' CEO Renee Aguiar-Lucander will present the report together with CFO Fredrik Johansson, CMO Richard Philipson and President North America Andrew Udell.