LDH

The United States FDA Awards Orphan Drug Designation (ODD) to NM5072 for Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)

Retrieved on: 
Monday, April 15, 2024
FDA, Pain, Mortality, Nephrology, Inflammation, Monocyte, Cell, Anemia, Alternative complement pathway, Safety, Ophthalmology, Red blood cell, Food, Hematology, Chelsea Handler, Dermatology, Half-life, Atypical hemolytic uremic syndrome, Blood, AP, Hyperlipidemia, Hemoglobinuria, Disease, Patient, Neurology, Fatigue, Platelet, Diagnosis, Infection, Hemolysis, Immune system, PNH, ODD, Quality of life, Chronic pain, LDH, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.

Key Points: 
  • The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.
  • CLEVELAND, April 15, 2024 (GLOBE NEWSWIRE) -- NovelMed today announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States.
  • Among these, Paroxysmal Nocturnal Hemoglobinuria (PNH) emerges as a prominent indication for complement blockers heading towards FDA approval.

Ferring Presents Pivotal Phase 3 Efficacy and Safety Data for Investigational Treatment, SI-6603 (condoliase), in Lumbar Disc Herniation at ASIPP 2024

Retrieved on: 
Monday, April 8, 2024
Biotechnology, Men, Health, Consumer, Other Health, Pharmaceutical, Surgery, Other Science, Research, Medical Devices, Science, Women, Seniors, Clinical Trials, Week, Sciatica, LDH, Incidence, Ferring Pharmaceuticals, Patient, Spinal disc herniation, Șaeș, University, ESI, Quality of life, Manager, University of Illinois Urbana-Champaign, Society, Back pain, LSM, Pain, SAE

"Based on these findings, SI-6603 could provide another option where a single injection to the lumbar disc could offer patients significant relief from the unrelenting pain often associated with lumbar disc herniation."

Key Points: 
  • "Based on these findings, SI-6603 could provide another option where a single injection to the lumbar disc could offer patients significant relief from the unrelenting pain often associated with lumbar disc herniation."
  • An analysis assessing current treatment patterns and gaps in clinical management of patients with newly diagnosed LDH was also presented.
  • The analysis reviewed medical claims data of more than a million U.S. patients over a four-year period.
  • Among patients who received more than one ESI, one in five (22.4%) underwent surgery with 14.2% of those patients receiving repeated surgeries.

Antibody Solutions Secures Grant for Challenging Diagnostic Initiative

Retrieved on: 
Thursday, April 4, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Medical Devices, Infectious Diseases, Genetics, LDH, Infection, Bill, RDT, Risk, Public, NGS, Antibody Solutions, Doctor of Philosophy, LMIC, Biology, Malaria, Antibody, Melinda French Gates, Vaccine

Antibody Solutions, a leading provider of fit-for-purpose antibody discovery services, announced today that it received a 15-month grant for nearly $1.2 million from the Bill & Melinda Gates Foundation to develop reagents for highly-specified rapid diagnostic tests (RDT) to detect malaria.

Key Points: 
  • Antibody Solutions, a leading provider of fit-for-purpose antibody discovery services, announced today that it received a 15-month grant for nearly $1.2 million from the Bill & Melinda Gates Foundation to develop reagents for highly-specified rapid diagnostic tests (RDT) to detect malaria.
  • The end result is that those RDTs are unable to differentiate between different strains of malaria,” explained John Kenney, PhD, co-founder and president of Antibody Solutions.
  • ”
    Antibody Solutions has already begun work to identify these new antibody reagents by harnessing Cellestive, its antibody discovery platform, that includes multiple discovery pathways such as hybridoma library, primary B-cell screening, and guided next-generation sequencing (NGS).
  • The grant from the foundation will also allow Antibody Solutions to work with industry experts in both public health and the development and deployment of healthcare solutions in low- and middle-income (LMIC) settings, ensuring that these reagents will be widely available.

Omeros Corporation Reports Fourth Quarter and Year-End 2023 Financial Results

Retrieved on: 
Monday, April 1, 2024
Mental Health, Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, Science, Oncology, PIN, PNH, Patient, Sale, CUD, Graft-versus-host disease, Investment, Clinical trial, Hemolysis, Calcineurin, Emory University, ASH, Risk, L-DOPA, Thumbay Hospital, Behavior informatics, Trial of the century, Myelodysplastic syndrome, Cancer, Safety, Transplant, LID, DRI, BLA, Corporation, Paroxysmal nocturnal hemoglobinuria, Tardive dyskinesia, Parkinson's disease, Publication, DSM-IV codes, Retirement, FDA, PDE7, LDH, Hematology, Mortality, Hematopoietic stem cell transplantation, Bone marrow failure, Abstract, Society, Pharmacology, Pharmaceutical industry

Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.

Key Points: 
  • Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.
  • Total operating expenses for the fourth quarter of 2023 were $39.8 million compared to $48.2 million for the third quarter of 2023.
  • Interest expense during the fourth quarter of 2023 was $7.1 million compared to $7.9 million during the prior quarter.
  • During the fourth quarter of 2023, we earned $3.4 million in interest and other income compared to $4.4 million in the third quarter.

Pelage Presents Late-Breaking Data at AAD 2024 Meeting Demonstrating PP405 Activates Human Hair Follicle Stem Cells Ex Vivo and in Phase 1 Clinical Study

Retrieved on: 
Saturday, March 9, 2024
MPC, Woman, LDH, CMO, LOS, Hair loss, Blood, Pattern hair loss, Biopsy, Fur, Skin, Data, Annual general meeting, B cell, AAD, Patient, Clinical trial, Inhibitor, American Academy, Pharmaceutical industry

LOS ANGELES, March 9, 2024 /PRNewswire/ -- Pelage Pharmaceuticals, a clinical-stage regenerative medicine company pioneering a new generation of treatments for hair loss, today presented positive translational and Phase 1 clinical data demonstrating the company's novel small molecule therapy PP405 reactivates dormant hair follicle stem cells to trigger hair growth. Data were presented in a late-breaking oral session of the American Academy of Dermatology (AAD) 2024 Annual Meeting in San Diego, titled Inhibition of pyruvate oxidation activates human hair follicle stem cells ex vivo.

Key Points: 
  • Data were presented in a late-breaking oral session of the American Academy of Dermatology (AAD) 2024 Annual Meeting in San Diego, titled Inhibition of pyruvate oxidation activates human hair follicle stem cells ex vivo.
  • "The translational data derived from our ex vivo studies on human facelift skin have been further validated in a Phase 1 first-in-human clinical trial demonstrating PP405 effectively reactivates dormant hair follicle stem cells," said Daniel Gil, Ph.D., CEO of Pelage Pharmaceuticals.
  • The LDH activity corresponded with a significant increase in Ki67 signal in the hair bulge, indicating a proliferative response of the hair follicle stem cells.
  • PP405 may also have applications for other forms of alopecia, including telogen effluvium (stress-induced hair loss) and chemotherapy-induced hair loss.

ASTHO Appoints Joseph Kanter, MD, MPH, as New Chief Executive Officer

Retrieved on: 
Monday, March 4, 2024
LDH, Multimedia, Addiction, City, Naloxone, MBA, Sound, Health, COVID-19, History, Tulane University, Public, Policy, MPH, Medicine, Health equity, Homelessness, State, Infection, Environment, Partnership, Nursing

ARLINGTON, Va., March 4, 2024 /PRNewswire/ -- The Association of State and Territorial Health Officials (ASTHO) today announced that Joseph Kanter, MD, MPH, has been named as the organization's chief executive officer, effective April 8, 2024.

Key Points: 
  • ARLINGTON, Va., March 4, 2024 /PRNewswire/ -- The Association of State and Territorial Health Officials (ASTHO) today announced that Joseph Kanter, MD, MPH, has been named as the organization's chief executive officer, effective April 8, 2024.
  • Dr. Kanter, a board-certified emergency physician, brings a wealth of experience to ASTHO including prior public service as both a Louisiana state health officer and municipal health director.
  • Dr. Kanter joins ASTHO from the Louisiana Department of Health (LDH) where he served as state health officer and medical director from January 2021 - February 2024.
  • ASTHO members, the chief health officials of these jurisdictions, are dedicated to formulating and influencing sound public health policy and to ensuring excellence in public health practice.

Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment

Retrieved on: 
Monday, February 12, 2024
LDH, Infection, FDA, Food, Hemoglobinuria, Paroxysmal nocturnal hemoglobinuria, Alternative complement pathway, ODD, Safety, Atypical hemolytic uremic syndrome, ANCA, Death, Patient, Hyperlipidemia, AP, Diagnosis, AAV, PNH, Chelsea Handler, Hemolysis, Pharmaceutical industry

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.

Key Points: 
  • FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.
  • The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV).
  • CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) --  NovelMed today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition.

Human medicines European public assessment report (EPAR): Epysqli, Date of authorisation: 26/05/2023, Revision: 1, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Patient, Medical Subject Headings, Marketing, Protein, Risk, Immune system, Infection, ATC, Neisseria meningitidis, Paroxysmal nocturnal hemoglobinuria, Bleeding, PNH, Disease, Headache, Pharmacist, LDH, Language, Vaccination, Medicine, Blood, Laboratory, European Medicines Agency, C5, Select, Cell, Safety, Hemolysis, Bacteria, Anatomy, Paroxysmal attack, Anemia, Hematology, IIA, Skin, Medical device

Human medicines European public assessment report (EPAR): Epysqli, Date of authorisation: 26/05/2023, Revision: 1, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Epysqli, Date of authorisation: 26/05/2023, Revision: 1, Status: Authorised

EMPAVELI® (pegcetacoplan) Provided Long-Term Control of PNH in New Data Presented at ASH Annual Meeting

Retrieved on: 
Monday, December 11, 2023
PEGASUS, Risk, PNH, Eculizumab, Disease, APLS, LDH, Trial of the century, Society, Standard of care, Head, Duke University, ASH, Infection, PRINCE, Patient, Safety, Bacteria, Medicine, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting.

Key Points: 
  • The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting.
  • After starting treatment with EMPAVELI, key markers of disease rapidly improved and were sustained in both treatment-naïve patients and patients previously treated with eculizumab.
  • Improvements in hemoglobin reached normal or near-normal levels, and mean lactate dehydrogenase (LDH) was maintained below the upper limit of normal.
  • “By achieving and maintaining normal clinical measures over time, EMPAVELI has the potential to elevate the standard of care for all adults with PNH."

Kira Pharmaceuticals Presents Positive Results of KP104 Phase 2 Study in Complement-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) at the 2023 ASH Annual Meeting

Retrieved on: 
Thursday, December 14, 2023
Safety, Interim, ASH, Cohort, Headache, PD, PNH, Patient, ULN, IgA nephropathy, Week, Microangiopathy, Pharmacokinetics, Immunoglobulin A, COVID19, Common, Society, RBC, LDH, Medical device, Pharmaceutical industry, Fine chemical, Vaccine

The interim results support the advancement of KP104 to Phase 3 studies as an optimal and safe treatment for PNH patients to address currently unmet medical needs.

Key Points: 
  • The interim results support the advancement of KP104 to Phase 3 studies as an optimal and safe treatment for PNH patients to address currently unmet medical needs.
  • The presentation has also been selected to be featured in the 2024 Highlights of ASH.
  • Kira Pharmaceuticals is committed to advancing KP104 as an innovative therapy for patients with PNH and other complement-mediated diseases.
  • Title: KP104, a bifunctional C5 antibody/factor H fusion protein, effectively controls both intravascular and extravascular hemolysis: interim results from a phase 2 study in complement inhibitor-naïve PNH patients