Complement system

Annexon Reports Significant Progress with its Priority Programs and Third Quarter 2023 Financial Results

Retrieved on: 
Montag, November 13, 2023
Molecule, Ophthalmology, Visual acuity, Investment, HD, EMA, Research, Kidney, Autoimmune disease, Driver, C3, Conference, R, Facial muscles, Inflammation, Treatment, Meta-analysis, American Academy, American Society of Nephrology, G&A, Guillain, Society, Letter, Paratriathlon, European Medicines Agency, American Academy of Ophthalmology, Professional, C4, AAO, System, Nephrology, AMD, Immunoglobulin therapy, PACA, Plasma, Disease, Complement component 1q, Medicine, Complement system, FDA, Geographic atrophy, GBS, ARCHER, C5, HIV disease progression rates, Risk management, Pharmaceutical industry, Vaccine, Cryptocurrency, Patient, LN, ALS

EMA orphan drug designation requires that a novel rare disease therapeutic demonstrates the potential for significant benefit over available therapies.

Key Points: 
  • EMA orphan drug designation requires that a novel rare disease therapeutic demonstrates the potential for significant benefit over available therapies.
  • Importantly, Annexon also announced that it has achieved target enrollment of 225 patients in the randomized, double-blind, placebo-controlled Phase 3 trial of ANX005 in patients with GBS.
  • Research and development (R&D) expenses: R&D expenses were $27.9 million for the quarter ended September 30, 2023, reflecting the advancement of the company’s priority programs, including GBS, GA and ANX1502, compared to $27.9 million for the quarter ended September 30, 2022.
  • General and administrative (G&A) expenses: G&A expenses were $6.9 million for the quarter ended September 30, 2023, compared to $8.2 million for the quarter ended September 30, 2022.

Calliditas announces poster presentations at ASN Digital Kidney Week 2021

Retrieved on: 
Mittwoch, November 3, 2021
Head, Chemokine, University, Nasdaq Stockholm, Goal, Inflammation, PDT, Person, CET, U.S. Securities and Exchange Commission, Private Securities Litigation Reform Act, Security (finance), FDA, Peyer's patch, ASN, Congenital nephrotic syndrome, CALT, Immunoglobulin A, American Society of Nephrology, Nasdaq, Risk, Complement system, TMA, NOX, TEL, Budesonide, Therapy, Society, Kidney, University of Leicester, CMO, Primary biliary cholangitis, Animal, Online gambling, Pharmaceutical industry

STOCKHOLM, Nov. 3, 2021 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced upcoming presentations concerning Nefecon, its lead product candidate, at the American Society of Nephrology (ASN) annual Kidney Week 2021 conference, which will be held virtually November 2-7, 2021.

Key Points: 
  • STOCKHOLM, Nov. 3, 2021 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced upcoming presentations concerning Nefecon, its lead product candidate, at the American Society of Nephrology (ASN) annual Kidney Week 2021 conference, which will be held virtually November 2-7, 2021.
  • In addition, Laura Prez-Als will present a poster titled "Treatment with Targeted Release Formulation Budesonide (Nefecon) Modulates the Complement System in Patients with IgA Nephropathy".
  • "We are delighted that these posters have been selected for the upcoming ASN Digital Kidney Week 2021 and congratulate the authors.
  • Calliditas is also planning to start clinical trials with NOX inhibitors in primary biliary cholangitis and head and neck cancer.

Calliditas announces poster presentations at ASN Digital Kidney Week 2021

Retrieved on: 
Mittwoch, November 3, 2021
Head, Chemokine, University, Nasdaq Stockholm, Goal, Inflammation, PDT, Person, CET, U.S. Securities and Exchange Commission, Private Securities Litigation Reform Act, Security (finance), FDA, Peyer's patch, ASN, Congenital nephrotic syndrome, CALT, Immunoglobulin A, American Society of Nephrology, Nasdaq, Risk, Complement system, TMA, NOX, TEL, Budesonide, Therapy, Society, Kidney, University of Leicester, CMO, Primary biliary cholangitis, Animal, Online gambling, Pharmaceutical industry

STOCKHOLM, Nov. 3, 2021 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced upcoming presentations concerning Nefecon, its lead product candidate, at the American Society of Nephrology (ASN) annual Kidney Week 2021 conference, which will be held virtually November 2-7, 2021.

Key Points: 
  • STOCKHOLM, Nov. 3, 2021 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced upcoming presentations concerning Nefecon, its lead product candidate, at the American Society of Nephrology (ASN) annual Kidney Week 2021 conference, which will be held virtually November 2-7, 2021.
  • In addition, Laura Prez-Als will present a poster titled "Treatment with Targeted Release Formulation Budesonide (Nefecon) Modulates the Complement System in Patients with IgA Nephropathy".
  • "We are delighted that these posters have been selected for the upcoming ASN Digital Kidney Week 2021 and congratulate the authors.
  • Calliditas is also planning to start clinical trials with NOX inhibitors in primary biliary cholangitis and head and neck cancer.

Annexon Broadens Autoimmune Franchise with Advancement of Third Anti-C1q Product Candidate and Strategic Expansion into New Autoantibody-driven Diseases

Retrieved on: 
Donnerstag, Juli 22, 2021
Medical specialties, Medicine, Clinical medicine, Complement system, Autoimmune diseases, Immunology, Antibodies, Syndromes, Classical complement pathway, Autoimmunity, Autoimmune disease, Autoantibody

SOUTH SAN FRANCISCO, Calif., July 22, 2021 (GLOBE NEWSWIRE) -- Annexon, Inc. (“Annexon”) (Nasdaq: ANNX), a clinical stage biopharmaceutical company developing a class of next generation complement medicines for patients with classical complement-mediated autoimmune and neurodegenerative disorders of the body, brain and eye, today announced the broadening of its autoimmune franchise through advancement of its third clinical-stage product candidate, ANX009, into Lupus Nephritis and expansion of the ANX005 clinical program into a second neuromuscular condition, Multifocal Motor Neuropathy (MMN).

Key Points: 
  • Our upstream complement approach has broad potential to deliver multiple, differentiated, first-in-class therapies for patients across a spectrum of autoantibody-driven autoimmune diseases.
  • Annexon is pioneering a targeted approach to tackling an array of antibody-mediated autoimmune diseases by inhibiting C1q and the early classical complement pathway.
  • Annexons third clinical-stage product candidate, ANX009, is a subcutaneous C1q inhibitor developed for antibody-mediated autoimmune diseases of blood and vascular tissues.
  • Annexon will host a virtual autoimmune R&D event on Wednesday, July 28, 2021, at 10:30 a.m.

Catalyst Biosciences to Host Research and Development Day on CB 4332 and Complement Programs

Retrieved on: 
Donnerstag, Juli 1, 2021
Organ systems, Complement system, Life sciences, Medical specialties, Biogen, Spinal muscular atrophy, Macular degeneration, Complement factor I, Protease

Catalysts complement portfolio is led by the development candidates CB 4332 and CB 2782-PEG, originating from the companys internal discovery platform, which has generated a rich pipeline of leads.

Key Points: 
  • Catalysts complement portfolio is led by the development candidates CB 4332 and CB 2782-PEG, originating from the companys internal discovery platform, which has generated a rich pipeline of leads.
  • CB 4332 is an engineered CFI protease with the potential to become a therapy addressing multiple complement related disorders.
  • CB 2782-PEG is a potential best-in-class C3 degrader product candidate in preclinical development for the treatment of dry AMD that Catalyst has licensed to Biogen.
  • Catalyst is a research and clinical development biopharmaceutical company focused on addressing unmet medical needs in rare disorders of the complement and coagulation systems.

European Hematology Association - Pegcetacoplan Maintains a Durable Response in Patients with Paroxysmal Nocturnal Hemoglobinuria Through Week 48

Retrieved on: 
Samstag, Juni 12, 2021
Medical specialties, Clinical medicine, Acquired hemolytic anemia, Complement system, Orphan drugs, Organ systems, Paroxysmal nocturnal hemoglobinuria, Eculizumab, Pegcetacoplan, Hemoglobinuria

After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.

Key Points: 
  • After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.
  • Here, we show the efficacy and safety of pegcetacoplan in 77 patients through 48 weeks of treatment.
  • Improvement of hemoglobin (Hb) level was maintained through Week 48 in all patients treated with pegcetacoplan monotherapy.
  • In conclusion, patients with suboptimal response to eculizumab experienced durable treatment effect when switched to pegcetacoplan, suggesting that pegcetacoplan is an effective therapeutic option for patients with paroxysmal nocturnal hemoglobinuria.

European Hematology Association - Pegcetacoplan Maintains a Durable Response in Patients with Paroxysmal Nocturnal Hemoglobinuria Through Week 48

Retrieved on: 
Samstag, Juni 12, 2021
Medical specialties, Clinical medicine, Acquired hemolytic anemia, Complement system, Orphan drugs, Organ systems, Paroxysmal nocturnal hemoglobinuria, Eculizumab, Pegcetacoplan, Hemoglobinuria

After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.

Key Points: 
  • After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.
  • Here, we show the efficacy and safety of pegcetacoplan in 77 patients through 48 weeks of treatment.
  • Improvement of hemoglobin (Hb) level was maintained through Week 48 in all patients treated with pegcetacoplan monotherapy.
  • In conclusion, patients with suboptimal response to eculizumab experienced durable treatment effect when switched to pegcetacoplan, suggesting that pegcetacoplan is an effective therapeutic option for patients with paroxysmal nocturnal hemoglobinuria.

Apellis and Sobi Report Positive Top-line Results from the Phase 3 PRINCE Study of EMPAVELI™ (pegcetacoplan) in Treatment-naïve Patients with PNH

Retrieved on: 
Dienstag, Mai 25, 2021
Complement system, Organ systems, Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, Pegcetacoplan, PNH, Medical specialties, Clinical medicine, Paroxysmal attack, Hemoglobinuria, Ham test

Combined with previous studies, these results emphasize the potential of EMPAVELI to provide disease control for all adults with PNH regardless of prior treatment.

Key Points: 
  • Combined with previous studies, these results emphasize the potential of EMPAVELI to provide disease control for all adults with PNH regardless of prior treatment.
  • At Week 26, 9% of patients in the EMPAVELI group experienced a serious adverse event (SAE) compared to 17% on standard of care.
  • The PRINCE study results reinforce the efficacy and safety profile of EMPAVELI in PNH, said Ravi Rao, head of research and development and chief medical officer at Sobi.
  • The PRINCE study (NCT04085601) is a 2:1 (EMPAVELI: standard of care) randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-nave adults with paroxysmal nocturnal hemoglobinuria (PNH).

Tony Gibney Joins InflaRx Board of Directors

Retrieved on: 
Freitag, Mai 21, 2021
Complement system, C5a, XE-class submarine, Complement component 5a, Mark Tuan, Complement component 5

Mr. Mark K\xc3\xbcbler and Ms. Lina Ma were re-appointed as Non-Executive Directors to the Board of Directors.\n\xe2\x80\x9cWe are excited that Tony is joining the InflaRx board.

Key Points: 
  • Mr. Mark K\xc3\xbcbler and Ms. Lina Ma were re-appointed as Non-Executive Directors to the Board of Directors.\n\xe2\x80\x9cWe are excited that Tony is joining the InflaRx board.
  • \xe2\x80\x9cOur entire team feels fortunate about his commitment and willingness to help InflaRx with its endeavors.
  • We are also very happy to announce the re-election of Lina Ma and Mark K\xc3\xbcbler as board members.
  • Both have contributed to InflaRx for many years and were instrumental in our successful IPO in 2017.\xe2\x80\x9d\nTony Gibney, Board member of InflaRx, said: "I am gratified to have been offered the opportunity to return to the Board of InflaRx, especially at such an important time in the Company\'s evolution as the leader in complement C5a inhibition.

Omeros Announces Extension of FDA Review Period for Narsoplimab in HSCT-TMA

Retrieved on: 
Donnerstag, Mai 20, 2021
Research, Managed care, Pharmaceutical, Oncology, Genetics, Clinical trials, Science, Biotechnology, Stem cells, Practice Management, FDA, Health, Medical specialties, Clinical medicine, Medicine, Food and Drug Administration, Acquired hemolytic anemia, Blood disorders, Complement system, Lectin pathway, Hematology, Thrombotic microangiopathy, Prescription Drug User Fee Act, Atypical hemolytic uremic syndrome

The new Prescription Drug User Fee Act (PDUFA) target action date is October 17, 2021.\nAs part of the ongoing BLA Priority Review, Omeros recently submitted a response to an FDA information request.

Key Points: 
  • The new Prescription Drug User Fee Act (PDUFA) target action date is October 17, 2021.\nAs part of the ongoing BLA Priority Review, Omeros recently submitted a response to an FDA information request.
  • FDA has classified the response as a major amendment, which requires additional time to review.\n\xe2\x80\x9cWe\xe2\x80\x99re pleased with our ongoing interactions with FDA on the narsoplimab BLA,\xe2\x80\x9d said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros.
  • Omeros\xe2\x80\x99 lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application under priority review by FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy.
  • Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, atypical hemolytic uremic syndrome and COVID-19.