C3a

NovelMed Phase I Clinical Trial Shows Inhibition of the Alternative Pathway and Preservation of the Classical Pathway – A Long-Acting Anti-Properdin Monoclonal Antibody NM3086 for PNH Patients

Retrieved on: 
Monday, June 5, 2023
C3b, PD, C5a, Serum, Receptor (biochemistry), IVH, Pathology, CP, Pharmacokinetics, AP, Intravascular hemolysis, EVH, C3a, Disorder, Trial of the century, MAC, Clinical trial, Paroxysmal nocturnal hemoglobinuria, Patient, Anemia, PNH, Complement factor B, FDA, Therapy, ADA, Safety, Vaccine, News, Hydraulic machinery, Pharmaceutical industry

Total AP inhibition was achieved through all cohorts and the duration of alternative pathway (AP) inhibition occurred in a dose-dependent manner.

Key Points: 
  • Total AP inhibition was achieved through all cohorts and the duration of alternative pathway (AP) inhibition occurred in a dose-dependent manner.
  • Correspondingly, the classical pathway (CP) remained unaffected, suggesting that NM3086 selectively blocks the AP while maintaining the host defense.
  • The duration of AP-C3b inhibition followed the AP inhibition profile very closely in a dose dependent manner.
  • CLEVELAND, June 05, 2023 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, Inc. announced today topline results from First-in-Human Phase I clinical trial of its complement blocker monoclonal anti-Properdin antibody, known as NM3086.

CANbridge Pharmaceuticals Granted Orphan Drug Designation for CAN 106 for the Treatment of Myasthenia Gravis

Retrieved on: 
Wednesday, November 16, 2022
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, FDA, Pompe, Weakness, Spinal muscular atrophy, Muscle contraction, Paroxysmal nocturnal hemoglobinuria, American Society of Gene and Cell Therapy, Acetylcholine, C5a, European Society for Medical Oncology, Glycogen storage disease, Ventilation, Glycogen storage disease type II, Neuromuscular disease, Degenerative disease, WuXi Biologics, University of Massachusetts, Patient, US, C3b, European Society of Gene and Cell Therapy, Muscle, Cell, C3a, SMA, Hospital, Glioblastoma, Research, MAC, Safety, Woman, Hunter syndrome, Drug, C3, Myasthenia gravis, Disease, Complement component 5, US FDA, Gene, C5, Congress, PNH, University, Haemophilia, Society, Fabry disease, Orphan, Pharmaceutical industry, Vaccine

Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.

Key Points: 
  • Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.
  • We continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for myasthenia gravis and other complement-mediated diseases.
  • CAN106 is a clinical-stage investigational novel, long-acting recombinant humanized monoclonal antibody that binds to and neutralizes C5, a key component of the complement system.
  • CAN106 acts downstream of C3 in the complement pathway, preserving the generation of C3a and C3b, which are important for innate immunity.

BioCryst Presents Data Demonstrating >99 Percent Suppression of Alternative Pathway Complement Activity with BCX9930 in C3G Patients

Retrieved on: 
Friday, August 26, 2022
PARK, Security (finance), COVID-19, AP, Pharmacokinetics, C3a, Kidney, Building, Disease, Time-invariant system, Serum, RESEARCH, BioCryst Pharmaceuticals, Poster, Galidesivir, U.S. Securities and Exchange Commission, CET, Marburg virus disease, Patient, GLOBE, Complement, Human Disease, Nasdaq, FDA, Yellow fever, Annual report, PMN, IgA nephropathy, Pharmaceutical industry, Medical device, Aluminium

The data, which are the first data with BCX9930 in patients with C3G, are being presented in a poster session at the 18th European Meeting on Complement in Human Disease, which is being held in Bern, Switzerland, from August 26-29, 2022.

Key Points: 
  • The data, which are the first data with BCX9930 in patients with C3G, are being presented in a poster session at the 18th European Meeting on Complement in Human Disease, which is being held in Bern, Switzerland, from August 26-29, 2022.
  • The analyses found that a single oral dose (600 mg) of BCX9930:
    Demonstrated >99 percent (median) suppression of the AP, and that >98 percent (median) suppression was maintained for 24 hours post-dosing, in C3G patients.
  • Achieved rapid (within two hours) and maximal (median >99 percent relative to pre-dose levels) suppression of the generation of C3a, a product of C3 convertase activity, in both healthy volunteers and C3G patients.
  • Currently, BioCryst is conducting the RENEW proof-of-concept basket study evaluating BCX9930 in C3G, immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN), all rare renal diseases caused by dysregulation of the alternative pathway.

First Patient Dosed in CAN106 Phase 1b/2 Trial for Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Retrieved on: 
Monday, March 28, 2022
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Kidney, Cell, Thrombosis, Heart, MPS II, University of Massachusetts Medical School, University, MAC, Death, 2-Bromo-LSD, Glioblastoma, Degenerative disease, Patient, Pulmonary hypertension, GBM, Peking Union Medical College Hospital, Research, WuXi Biologics, Incidence, C3a, PNH, Hemolysis, PHN, Paroxysmal nocturnal hemoglobinuria, Hunter syndrome, Administration, CEO, C3, Anemia, MPS, C5, Safety, C5a, Steroid, Diagnosis, Glycogen storage disease, C3b, University of Massachusetts, Disorder, CH50, Complement component 5, Lists of diseases, Paroxysmal attack, Medicine, Fatigue, Protein, Spiroplasma mirum, Ravulizumab, Haemophilia, Pharmaceutical industry, Vaccine, Fine chemical, Hematology, CAN106, PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH), CANBRIDGE PHARMACEUTICALS INC.

CAN106 was previously shown to be safe and well-tolerated, with dose-dependent and linear pharmacokinetic exposure, in a study of healthy volunteers in Singapore.

Key Points: 
  • CAN106 was previously shown to be safe and well-tolerated, with dose-dependent and linear pharmacokinetic exposure, in a study of healthy volunteers in Singapore.
  • Based on these results, Chinas National Medical Products Administration approved the CAN106 Phase 1b/2 trial for the treatment of patients with PNH.
  • CAN106 is a novel, long-acting recombinant human monoclonal antibody that binds to and neutralizes C5, a key component of the complement system.
  • Paroxysmal nocturnal hemoglobinuria (PNH) belongs to a group of fatal and rare disorders that occur when the complement system is dysregulated.

CANbridge Reports Positive Top-Line CAN106 Phase 1 Data

Retrieved on: 
Monday, February 7, 2022
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, PNH, Disorder, MPS II, Single, C5, Immune system, IND, MPS, Ravulizumab, Investigational New Drug, Safety, Anemia, 2-Bromo-LSD, Glioblastoma, Steroid, Hemolysis, Glycogen storage disease, Cell, Blood, SAD, WuXi Biologics, Complement component 5, Paroxysmal nocturnal hemoglobinuria, Research, C3, Șaeș, Haematopoietic system, Haemophilia, C3b, Pulmonary hypertension, Incidence, Paroxysmal attack, Patient, Kidney, Heart, University of Massachusetts, Diagnosis, Hunter syndrome, Protein, University, PHN, PK, GBM, Thrombosis, Lists of diseases, CEO, C5a, Spiroplasma mirum, Death, C3a, MAC, CH50, PD, University of Massachusetts Medical School, Partnership, Degenerative disease, Fatigue, Vaccine, Pharmaceutical industry, Palladium, Fine chemical, Pharmacokinetics, the Study, CAN106, Paroxysmal Nocturnal Hemoglobinuria (PNH), CANbridge Pharmaceuticals Inc., THE STUDY, CAN106, PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH), CANBRIDGE PHARMACEUTICALS INC.

CAN106 was shown to be safe and well-tolerated with mostly mild or moderate adverse events and no drug-related serious adverse events (SAEs).

Key Points: 
  • CAN106 was shown to be safe and well-tolerated with mostly mild or moderate adverse events and no drug-related serious adverse events (SAEs).
  • CAN106 showed dose-dependent and linear pharmacokinetic exposure with low inter-subject variability and a terminal elimination half-life of approximately 32 days.
  • These results suggest complete functional blockade of terminal complement activity, and importantly, provide the first human data validating CAN106 as a potential treatment for complement-mediated diseases.
  • The objectives were to assess the safety and tolerability of single escalating doses of CAN106, to characterize the PK and PD profile of CAN106, and to evaluate the immunogenicity of CAN106.

NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone

Retrieved on: 
Monday, January 31, 2022
AP, MAC, IND, Safety, Lists of diseases, Patient, Therapy, Pathology, Food, Infection, Conditional sentence, Hemolysis, BB, LDH, Thrombus, Inflammation, Properdin, CP, Pharmacokinetics, Serum, Disease, Complement component 5, C5a, Alternative-complement-pathway C3/C5 convertase, PNH, Paroxysmal nocturnal hemoglobinuria, C3b, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, C3a, Pharmaceutical industry, Vaccine, C5, C3, Pegcetacoplan, Eculizumab, Ravulizumab

Through its targeted specificity to the complement alternative pathway (AP), NM8074 is expected to improve upon current treatments such as Soliris, Ultomiris, and Empaveli, which are approved for treatment of a limited number of rare diseases.

Key Points: 
  • Through its targeted specificity to the complement alternative pathway (AP), NM8074 is expected to improve upon current treatments such as Soliris, Ultomiris, and Empaveli, which are approved for treatment of a limited number of rare diseases.
  • NM8074 selectively binds to complement alternative pathway specific protein Bb and inhibits the formation of C3a/C3b, C5a/C5b, and MAC, products that mediate inflammation and tissue damage in numerous diseases.
  • NM8074 carries a mechanistic advantage over current platforms because it targets the disease-specific complement alternative pathway (AP) without blocking the classical pathway (CP) required for host defense.
  • NovelMed is a clinical-stage biopharmaceutical company committed to innovating and developing biologics to treat rare diseases caused by the overactivation of the complement alternative pathway.

Gemini Therapeutics Reports Second Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 12, 2021
Biotechnology, General Health, Health, Pharmaceutical, Clinical Trials, SEC, Poster, CFH, Smoking, Annual general meeting, Ageing, Anetoderma, 40, Population, GA, PK, FDA, Association, Safety, Boat, Choroidal neovascularization, U.S. Securities and Exchange Commission, CNV, Reading, Food, Geographic atrophy, LinkedIn, Securities Act of 1933, Night vision, AMD, Drug Quality and Security Act, Company, A-DNA, Research, Risk, Inflammation, Biomarker, Severe cognitive impairment, Regulation of tobacco by the U.S. Food and Drug Administration, Eye, MA, Retina, File, Annual report, Forward-looking statement, NAMD, Twitter, COVID-19, Disease, ARVO, Therapy, Patient, Pharmacokinetics, Nasdaq, Western world, Social media, Visual acuity, C3a, Factor, Pharmaceutical industry, Vaccine, the Phase 2a ReGAtta Study, the Phase 2a Study of Repeat Intravitreal Injections of GEM103 in Neovascular Age-related Macular Degeneration (nAMD), GEM103, Dry Age-Related Macular Degeneration (AMD), Gemini Therapeutics, THE PHASE 2A REGATTA STUDY, THE PHASE 2A STUDY OF REPEAT INTRAVITREAL INJECTIONS OF GEM103 IN NEOVASCULAR AGE-RELATED MACULAR DEGENERATION (NAMD), GEM103, DRY AGE-RELATED MACULAR DEGENERATION (AMD), GEMINI THERAPEUTICS

Gemini Therapeutics, Inc. (Nasdaq: GMTX), a clinical stage precision medicine company developing innovative treatments for genetically-defined age-related macular degeneration (AMD), today reported its financial results for the second quarter ended June 30, 2021 and provided a business update.

Key Points: 
  • Gemini Therapeutics, Inc. (Nasdaq: GMTX), a clinical stage precision medicine company developing innovative treatments for genetically-defined age-related macular degeneration (AMD), today reported its financial results for the second quarter ended June 30, 2021 and provided a business update.
  • In May 2021, the Company completed enrollment in the Phase 2a safety and tolerability study.
  • At June 30, 2021, Gemini held $167.5 million in cash, $7.9 million of principal outstanding debt and 43.1 million shares outstanding.
  • Gemini Therapeutics is a clinical stage precision medicine company developing novel therapeutic compounds to treat genetically defined age-related macular degeneration (AMD).

Vaniam Group Announces Dr. Quaovi H. Sodji of Stanford University as the Inaugural Recipient of Conquer Cancer-Vaniam Group LLC Young Investigator Award

Retrieved on: 
Friday, June 18, 2021
C3a, Pancreatic cancer

CHICAGO, June 18, 2021 /PRNewswire/ --Vaniam Group announced today that the Conquer Cancer-Vaniam Group LLC inaugural Young Investigator Award (YIA) was bestowed upon Quaovi H. Sodji, MD, PhD, of Stanford University for his research in pancreatic cancer.

Key Points: 
  • CHICAGO, June 18, 2021 /PRNewswire/ --Vaniam Group announced today that the Conquer Cancer-Vaniam Group LLC inaugural Young Investigator Award (YIA) was bestowed upon Quaovi H. Sodji, MD, PhD, of Stanford University for his research in pancreatic cancer.
  • This annual award will provide research grants to Black, African, or African American researchers who specialize in oncology.
  • Dr. Sodji received the inaugural YIA for his research that explores targeting the complement C3a and C3a receptor as a novel immunotherapy approach against pancreatic cancer.
  • Founded in 2007 as a virtual-by-design organization, Vaniam Group harnesses the talents and expertise of team members around the world.

Apellis Announces New Data Demonstrating Correlation between Complement Activation and COVID-19 Severity

Retrieved on: 
Thursday, October 15, 2020
Medical specialties, Clinical medicine, Medicine, C-reactive protein, C3a, Lactate dehydrogenase, Complement component 3, Complement system, Complement membrane attack complex, Interleukin 6, C4A, Complement component 4

CRP, C-reactive protein; IL-6, interleukin 6; LDH, lactate dehydrogenase; TCC, terminal complement complex; ULN, upper limit of normal.

Key Points: 
  • CRP, C-reactive protein; IL-6, interleukin 6; LDH, lactate dehydrogenase; TCC, terminal complement complex; ULN, upper limit of normal.
  • Apellis also announced preliminary results from the first part of the interventional Phase 1/2 study of APL-9 in patients with severe COVID-19.
  • The observational exploratory study evaluated the relationship between markers of complement activation and disease severity in 41 patients with COVID-19.
  • The primary objective of the observational study was to quantify systemic concentrations of the complement activation products Bb, C3a, C4a and terminal complement complex (TCC) and correlate them to COVID-19 severity.