Ahus

Anti-Bb Antibody (NM8074) Receives US FDA Clearance to Start Clinical Trial in Naïve aHUS Patients (Rare Disease)

Retrieved on: 
Monday, January 30, 2023
Disease, AP, Food, Patient, BB, Efficacy, Acquisition, Thrombotic microangiopathy, Safety, CP, Phase, Complement, Hemolysis, Partnership, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Ahus, Anemia, C3b, Inflammation, Pharmaceutical industry, Eculizumab, Adult

CLEVELAND, Jan. 30, 2023 (GLOBE NEWSWIRE) -- NovelMed announced today that the U.S. Food and Drug Administration (FDA) cleared the Company’s investigational drug NM8074 to initiate an efficacy trial in patients with aHUS in coming months. NovelMed is advancing its anti-Bb antibody (NM8074) to treat patients with evidence of treatment-related resistance or complement-mediated relapses in aHUS, a rare disease with unmet need. NovelMed is a clinical-stage biopharma company focused on the development of humanized monoclonal antibody treatments for several complement-mediated disorders.

Key Points: 
  • NovelMed is advancing its anti-Bb antibody (NM8074) to treat patients with evidence of treatment-related resistance or complement-mediated relapses in aHUS, a rare disease with unmet need.
  • NovelMed is a clinical-stage biopharma company focused on the development of humanized monoclonal antibody treatments for several complement-mediated disorders.
  • NM8074 has recently completed a Phase I trial in healthy volunteers with no safety concerns.
  • NM8074 blocks the AP-mediated formation of C3b, an important molecule responsible for incurable anemia seen in both Soliris and Ultomiris treated patients.

4D Molecular Therapeutics Announces Updates on Clinical Pipeline and Additional Preclinical Programs

Retrieved on: 
Monday, January 9, 2023
IND, Disease, Partner (business rank), Echocardiography, PRISM, Clinical trial, Heart, Dialysis, A101, MD, GA, M.A, Degenerative disease, CF, Initiation, University of Nevada, Reno, Biopsy, Interim, NIH, QOL, University, IHC, Cystic fibrosis, Kidney, AMD, Toxic leukoencephalopathy, Intravitreal administration, RNA, Drug discovery, MRI, Choroideremia, Real-time, GMP, Stanford University School of Medicine, Development, Ahus, Fabry disease, Geographic atrophy, FDA, Retinitis pigmentosa, KCCQ, Addition, Atypical hemolytic uremic syndrome, Fabry, Principal, Ophthalmology, VG, Pulmonology, Enzyme replacement therapy, CFTR, DME, Clinical research, Safety, Diabetic retinopathy, ISH, Modulation, Pharmaceutical industry, Vaccine, 3D printing, Medical imaging, Patient

The doses to be evaluated in DME are expected to be similar to those used in the 4D-150 wet AMD clinical trial.

Key Points: 
  • The doses to be evaluated in DME are expected to be similar to those used in the 4D-150 wet AMD clinical trial.
  • It is estimated that there are over one million individuals with GA in the United States according to published data.
  • In addition, I’m excited by the potential of 4D-175, the new preclinical product candidate utilizing the same R100 vector.
  • Cardiac clinical endpoint data (MRI, echocardiography, cardiopulmonary exercise testing [CPET] and QOL assessment) from evaluations at baseline and 12 months after treatment were assessed.

Worldwide Rare Kidney Diseases Industry Report to 2035 - Key Market Trends and Forecasts - ResearchAndMarkets.com

Retrieved on: 
Monday, December 5, 2022
Pharmaceutical, Health, Other Health, Molecule, Lupus nephritis, Fabry disease, Dialysis, Disorder, Kidney, European Medicines Agency, Cystinosis, Renal tubular acidosis, Diagnosis, RKD, EMA, Patient, Prevalence, Biopsy, Food and Drug Administration, Research, Hospital, Atypical hemolytic uremic syndrome, LN, Diabetes, Incidence, COVID-19, Survival, Ahus, Administration, Mortality, USFDA, USD, Pharmaceutical industry, Cysteamine, IgA nephropathy

The "Rare Kidney Diseases Market by Target Indications, Type of Molecule, Route of Administration and Key Geographies: Industry Trends and Global Forecast, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Rare Kidney Diseases Market by Target Indications, Type of Molecule, Route of Administration and Key Geographies: Industry Trends and Global Forecast, 2022-2035" report has been added to ResearchAndMarkets.com's offering.
  • This report features an extensive study of the current market landscape and the likely future potential associated with the rare kidney diseases market, over the next decade.
  • The term rare kidney diseases (RKD) represents around 150 different indications.
  • It is worth highlighting that the prevalence rate of rare kidney diseases is estimated to be 60-80 per 100,000 cases / individuals in the US and Europe.

Omeros Corporation Reports Third Quarter 2022 Financial Results

Retrieved on: 
Wednesday, November 9, 2022
Oncology, Health, Infectious Diseases, Research, Science, Pharmaceutical, Biotechnology, IgA nephropathy, Review, Investment, Atypical hemolytic uremic syndrome, Adult, DRI, Center, Proteinuria, Literature, American Society of Hematology, OND, Society, Ahus, Ophthalmology, COVID-19, BLA, PNH, Immunoglobulin A, Cancer, CRL, Congress, News, Jean-Louis Vincent, Safety, Survival, Rayner, Inflammation, Probability, Centers for Medicare & Medicaid Services, Corporation, Medicare, Patient, CMS, Recurrence, FDA, Pharmaceutical industry, Hematology, Agency

Rayners U.S. net sales of OMIDRIA were $33.0 million for the third quarter of 2022.

Key Points: 
  • Rayners U.S. net sales of OMIDRIA were $33.0 million for the third quarter of 2022.
  • In September 2022, results of the narsoplimab treatment arm of the I-SPY COVID-19 adaptive platform trial sponsored by Quantum Leap Healthcare Collaborative were announced.
  • Total costs and expenses for the third quarter of 2022 were $50.8 million compared to $39.8 million for the third quarter of 2021.
  • The increase was primarily due to the manufacturing of narsoplimab drug substance in the third quarter of 2022 for future commercial and clinical use.

Omeros Corporation Reports Second Quarter 2022 Financial Results

Retrieved on: 
Tuesday, August 9, 2022
Research, Infectious Diseases, FDA, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Oncology, U.S. Securities and Exchange Commission, Disclosure, OND, Investment, CRL, Security (finance), Marketing, Patient, Rayner, CMS, Securities Act of 1933, Tardive dyskinesia, Lynch, Securities Exchange Act of 1934, Disease, Drug Quality and Security Act, Risk, Inflammation, Sale, Translational medicine, Proteinuria, Immunoglobulin A, Endpoint security, Corporation, Safety, Paroxysmal nocturnal hemoglobinuria, Publication, BLA, PDE7, IgA nephropathy, Ophthalmology, PNH, Death, Forward-looking statement, Growth, Atypical hemolytic uremic syndrome, Micronutrient deficiency, Cancer, Income, COVID-19, Mission statement, Program, Infection, Â, Time, FDA, Ahus, Government, Human, Pharmaceutical industry, Vaccine, Glomerulopathy, Omeros

Rayners reported net sales of $34.5 million for the second quarter of 2022, all of which were in the U.S., establish a new all-time high for quarterly OMIDRIA sales and represent a $5.7 million increase over net sales of $28.8 million reported by Omeros for the second quarter of last year.

Key Points: 
  • Rayners reported net sales of $34.5 million for the second quarter of 2022, all of which were in the U.S., establish a new all-time high for quarterly OMIDRIA sales and represent a $5.7 million increase over net sales of $28.8 million reported by Omeros for the second quarter of last year.
  • At June 30, 2022, Omeros had $122.6 million of cash, cash equivalents and short-term investments available for operations, which is a reduction of $19.7 million from March 31, 2022.
  • During the second quarter of 2022, Omeros earned royalties of $17.2 million on sales of OMIDRIA, which were recorded as a reduction from the OMIDRIA contract royalty asset.
  • Total costs and expenses for the second quarter of 2022 were $37.4 million compared to $45.6 million for the second quarter of 2021.

Samsung Bioepis Presents New Phase 3 Study of SB12 (Eculizumab), A Proposed Biosimilar to Soliris, at the European Hematology Association (EHA) Congress 2022

Retrieved on: 
Friday, June 10, 2022
Science, PNH, PK, Neuromyelitis optica spectrum disorder, ECU, Week, Hematology, LinkedIn, Pharmacokinetics, Ahus, European Hematology Association, Acetylcholine receptor, TS1, Ophthalmology, Endocrinology, Patient, GLOBE, LDH, DNA, TS2, EHA, Social media, Twitter, Paroxysmal nocturnal hemoglobinuria, Gastroenterology, PD, GMG, Microdeletion syndrome, Degenerative disease, Eculizumab, NMOSD, Safety, Generic drug, Pharmaceutical industry

The Phase 3 study results are presented as an e-poster at the 27th European Hematology Association 2022 (EHA2022) Hybrid Congress held in Vienna, Austria and virtually, from June 9 to 12, 2022.

Key Points: 
  • The Phase 3 study results are presented as an e-poster at the 27th European Hematology Association 2022 (EHA2022) Hybrid Congress held in Vienna, Austria and virtually, from June 9 to 12, 2022.
  • Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone.
  • Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world's leading biopharmaceutical company.
  • Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, hematology, endocrinology and gastroenterology.

2021 Atypical Hemolytic Uremic Syndrome Epidemiology Forecast to 2030 - ResearchAndMarkets.com

Retrieved on: 
Monday, November 8, 2021
Health, Other Health, Population, View, Atypical hemolytic uremic syndrome, Disease, Classification, Risk, Epidemiology, Master, Ahus, Doctor of Philosophy, That Total Age, Diagnosis, Atypical, United Kingdom, Total, Renewable energy, Agriculture, Residential care, Pharmaceutical industry, Medical device, Vaccine

The disease epidemiology covered in the report provides historical as well as forecasted Atypical Hemolytic Uremic Syndrome epidemiology [segmented as Total Prevalent Cases of Atypical hemolytic uremic syndrome, Total Diagnosed Cases of Atypical hemolytic uremic syndrome, Total Age group-specific Cases of Atypical hemolytic uremic syndrome, and Total Treated Cases of Atypical hemolytic uremic syndrome] in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2018 to 2030.

Key Points: 
  • The disease epidemiology covered in the report provides historical as well as forecasted Atypical Hemolytic Uremic Syndrome epidemiology [segmented as Total Prevalent Cases of Atypical hemolytic uremic syndrome, Total Diagnosed Cases of Atypical hemolytic uremic syndrome, Total Age group-specific Cases of Atypical hemolytic uremic syndrome, and Total Treated Cases of Atypical hemolytic uremic syndrome] in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2018 to 2030.
  • The Atypical Hemolytic Uremic Syndrome Report and Model provide an overview of the risk factors and global trends of Atypical Hemolytic Uremic Syndrome in the seven major markets (7MM: The United States, Germany, France, Italy, Spain, the United Kingdom, and Japan)
    The report provides insight about the historical and forecasted patient pool of Atypical Hemolytic Uremic Syndrome in seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
  • The report provides the segmentation of the Atypical Hemolytic Uremic Syndrome epidemiology by total age group-specific cases in the 7MM.
  • Quantify patient populations in the global Atypical Hemolytic Uremic Syndrome market to improve product design, pricing, and launch plans
    The Atypical Hemolytic Uremic Syndrome epidemiology report and model were written and developed by Masters and Ph.D. level epidemiologists
    The Atypical Hemolytic Uremic Syndrome Epidemiology Model developed by the publisher is easy to navigate, interactive with dashboards, and epidemiology based on transparent and consistent methodologies.

Omeros’ Narsoplimab Stabilized Kidney Function and Reduced Urinary Complement Proteins in a Patient with IgA Vasculitis

Retrieved on: 
Friday, November 5, 2021
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Society, Urine, Movement disorders, Security (finance), Securities Exchange Act of 1934, COVID-19, Treatment, Ahus, Cancer, Therapy, MBL, Drug Quality and Security Act, United Kingdom, European Medicines Agency, Hypertension, Phenylephrine, Infection, Research, Securities Act of 1933, Forward-looking statement, Vasculitis, U.S. Securities and Exchange Commission, Annual general meeting, Corporation, Patient, FDA, Nephron, HSCT, Inflammation, University of Copenhagen Faculty of Health and Medical Sciences, GPCR, Program, Immunoglobulin A, SEC, Proteinuria, Atypical hemolytic uremic syndrome, Kidney, Creatinine, University, Neurological disorder, Hematuria, BLA, IgA nephropathy, Dialysis, Kidney disease, PDE7, GPR174, All India Mahila Congress, Fibrosis, ASN, Pharmaceutical industry, Vaccine, Narsoplimab, Omeros Corporation, NARSOPLIMAB, OMEROS CORPORATION

The objective was to stabilize her rapidly deteriorating kidney function to allow for renal transplantation.

Key Points: 
  • The objective was to stabilize her rapidly deteriorating kidney function to allow for renal transplantation.
  • Diagnosed with IgA vasculitis, she developed rapidly worsening kidney function with highly elevated creatinine levels, hypertension, proteinuria, and hematuria despite treatment with systemic corticosteroids.
  • After one course of 12 weekly doses of narsoplimab, her kidney function stabilized and she was able to undergo successful renal transplantation without the need for dialysis.
  • As reported in yesterdays ASN Annual Meeting presentation, complement levels assessed in urinary samples collected during the patients clinical course demonstrated substantial reduction in local complement activation associated with narsoplimab treatment-related lectin-pathway inhibition and stabilization of kidney function.

Global Atypical Hemolytic Uremic Syndrome Market to 2030 - Insights, Epidemiology and Market Forecasts - ResearchAndMarkets.com

Retrieved on: 
Monday, October 25, 2021
Health, Pharmaceutical, Atypical hemolytic uremic syndrome, Ahus, United Kingdom, Customer insight, CAGR, Renewable energy, Toy

The report will help in developing business strategies by understanding trends shaping and driving the Atypical Hemolytic Uremic Syndrome market.

Key Points: 
  • The report will help in developing business strategies by understanding trends shaping and driving the Atypical Hemolytic Uremic Syndrome market.
  • To understand the future market competition in the Atypical Hemolytic Uremic Syndrome market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for the Atypical Hemolytic Uremic Syndrome market.
  • To understand the future market competition in the Atypical Hemolytic Uremic Syndrome market.

Alexion Announces Upcoming Data Presentations at the American Society of Nephrology’s Virtual Kidney Week 2020

Retrieved on: 
Friday, October 9, 2020
Biotechnology, General Health, Pharmaceutical, Health, Health, Medical specialties, Clinical medicine, Blood disorders, Orphan drugs, Alexion Pharmaceuticals, Atypical hemolytic uremic syndrome, Eculizumab, Ahus, Ravulizumab, Hemolysis, Hemolytic–uremic syndrome, Atypical Hemolytic Uremic Syndrome (aHUS), ULTOMIRIS® (ravulizumab‑cwvz), SOLIRIS® (eculizumab), Alexion

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that four abstracts have been accepted for presentation at the American Society of Nephrologys Kidney Week 2020, taking place virtually from October 22 to 25, 2020.

Key Points: 
  • Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that four abstracts have been accepted for presentation at the American Society of Nephrologys Kidney Week 2020, taking place virtually from October 22 to 25, 2020.
  • Alexion also plans to present on the comparative efficacy of ULTOMIRIS and SOLIRIS in the treatment of adults with aHUS.
  • Efficacy and Safety of Ravulizumab in Pediatric Patients with Atypical Hemolytic Uremic Syndrome Previously Treated with Eculizumab: 26week and 1year Data.
  • Comparative Efficacy of Ravulizumab and Eculizumab in the Treatment of Atypical Hemolytic Uremic Syndrome: An Indirect Comparison Using Clinical Trial Data.