Complement component 1q

Annexon to Host Virtual R&D Day on Guillain-Barré Syndrome, Focusing on its Serious Unmet Need and Annexon’s Novel Therapeutic Approach

Retrieved on: 
Wednesday, February 21, 2024
Doctor of Philosophy, MD, Neurology, Complement component 1q, University, GBS, MBBS, Medicine, Sintering

BRISBANE, Calif., Feb. 21, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for people living with devastating inflammatory-related diseases, today announced it will host a virtual R&D Day on Friday, March 1, 2024 at 10:00 AM ET. To register, click here.

Key Points: 
  • Event will feature GBS experts Lisa Butler of the GBS|CIDP Foundation International, Hugh Willison, MBBS, PhD, Professor Emeritus of Neurology of University of Glasgow, and David Cornblath, MD, Professor Emeritus of Johns Hopkins University School of Medicine
    BRISBANE, Calif., Feb. 21, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for people living with devastating inflammatory-related diseases, today announced it will host a virtual R&D Day on Friday, March 1, 2024 at 10:00 AM ET.
  • To register, click here .
  • The event will feature GBS experts who will provide an overview of Guillain-Barré syndrome (GBS), the most common cause of acute neuromuscular paralysis:
    Presentations from GBS experts and Annexon management will highlight:
    Targeting C1q in GBS with ANX005, Annexon's investigational monoclonal antibody designed to inhibit C1q and stop classical complement pathway activation before it starts
    A live question and answer will follow formal presentations.

Orphan designation: Humanised IgG4 monoclonal antibody against C1q Treatment of Guillain-Barré syndrome, 13/10/2024 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Complement component 1q, Immunoglobulin G, Committee, Patient, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Humanised IgG4 monoclonal antibody against C1q
    - Intended use
    - Treatment of Guillain-Barré syndrome
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2836
    - Date of designation
    - Sponsor
    Kinesys Consulting NL B.V.
  • 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:

Annexon Outlines 2024 Priorities with Late-Stage Clinical Milestones Across Upstream Complement Portfolio for Autoimmune, Ophthalmic and Neurodegenerative Diseases

Retrieved on: 
Monday, January 8, 2024
Patient, Geographic atrophy, Initiate, Conference, Outline, ARCHER, Immunoglobulin therapy, GBS, PRIME, EMA, Research, Paratriathlon, Cold agglutinin disease, AMD, CAD, Complement component 1s, Brain, Facial muscles, Eye, Clinical trial, Inflammation, Pharmacokinetics, Nerve, Death, Nervous system, Guillain, SAD, Complement component 1q, Webcast, Safety, Meta-analysis, Pharmaceutical industry

BRISBANE, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for people living with devastating inflammatory-related diseases, today outlined its strategic priorities for 2024 with late-stage clinical milestones, including ANX005 for Guillain-Barré syndrome (GBS), ANX007 for geographic atrophy (GA) and its first-in-kind oral small molecule complement inhibitor, ANX1502, for a range of autoimmune indications.

Key Points: 
  • The study completed enrollment in 2023 and the company is on-track to report Phase 3 data in the first half of 2024.
  • Annexon also plans to initiate the ARROW clinical trial, an injection-controlled head-to-head study against SYFOVRE® (pegcetacoplan injection) in late 2024.
  • A live webcast of the event can be accessed under the ‘Events & Presentations’ section on the Investors page at www.annexonbio.com.
  • A replay of the webcast will be archived on the Annexon website for 30 days following the presentation.

Annexon Outlines Global Registrational Program for ANX007 in Geographic Atrophy with FDA Alignment on Vision Preservation as Primary Endpoint

Retrieved on: 
Wednesday, December 20, 2023
Quality of life, Geographic atrophy, Food, PRIME, FDA, EMA, Paratriathlon, AMD, FAF, Inflammation, Visual acuity, Ophthalmology, Complement component 1q, Patient, Safety, Pharmaceutical industry

BRISBANE, Calif., Dec. 20, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for people living with devastating inflammation-related diseases, today outlined its global registrational program for ANX007, a first-in-class C1q and classical complement inhibitor, for the treatment of patients with geographic atrophy (GA).

Key Points: 
  • Notably, the FDA has not required Annexon to study the slowing of lesion growth as measured by fundus autofluorescence (FAF), an anatomical endpoint used for the approval of other GA programs.
  • “We are thrilled to have aligned with FDA on vision preservation as the primary endpoint in our Phase 3 GA program, based on the statistically significant and dose-dependent visual protection ANX007 demonstrated in the Phase 2 ARCHER trial,” said Douglas Love, chief executive officer of Annexon.
  • “Blocking C1q with ANX007 is designed to stop classical complement inflammation that drives photoreceptor damage and vision loss.
  • BCVA ≥15-letter loss is a well-established functional endpoint that has served as the basis for numerous ophthalmology drug approvals by the FDA and EMA.

Annexon Reports Phase 1 Results for ANX1502, its Oral Small Molecule Inhibitor of the Classical Complement Pathway

Retrieved on: 
Wednesday, December 20, 2023
Research, Cold agglutinin disease, Diarrhea, CAD, Complement component 1s, Nausea, Trial of the century, Serum, PD, Vomiting, SAD, Complement component 1q, Patient, Safety, Dietary supplement

BRISBANE, Calif., Dec. 20, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for people living with devastating inflammation-related diseases, today reported results from the Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) healthy volunteer study of ANX1502, a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. ANX1502 achieved target serum levels and demonstrated pharmacokinetic (PK) measures that support advancement into a proof-of-concept clinical study to assess pharmacodynamics (PD) and efficacy in patients with cold agglutinin disease (CAD) in 2024.

Key Points: 
  • “After more than a decade of groundbreaking research targeting the early classical complement pathway, we are excited to have reached an important step in the clinical development of ANX1502, our first-in-kind small molecule complement inhibitor that we believe can have meaningful impact on a range of autoimmune conditions,” said Ted Yednock, Ph.D., chief innovation officer of Annexon.
  • “We’re very encouraged by the results from our Phase 1 SAD/MAD trial showing that ANX1502 was well tolerated and achieved target drug levels with supportive impact on a key biomarker in healthy volunteers.
  • Results of the study were as follows:
    Across all doses evaluated, ANX1502 was generally well tolerated with mild to moderate treatment-emergent adverse events (TEAEs).
  • No serious adverse events were reported, and there were no significant clinical or lab findings.

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

Retrieved on: 
Monday, 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.

Annexon Announces Clinical and Regulatory Progress for ANX005 Pivotal Program in Guillain-Barré Syndrome (GBS)

Retrieved on: 
Tuesday, October 10, 2023
Food, Death, Inflammation, Standard of care, European Medicines Agency, Guillain, Immunoglobulin therapy, Disease, EMA, FDA, Health status of Asian Americans, Weakness, GBS, Facial muscles, Nerve, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Complement component 1q, Advantage, Patient, Pharmaceutical industry, EU

BRISBANE, Calif., Oct. 10, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX) a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced that the European Medicines Agency (EMA) granted orphan drug designation to ANX005 for the treatment of Guillain-Barré Syndrome (GBS). The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to ANX005 for the treatment of GBS.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to ANX005 for the treatment of GBS.
  • ANX005, a humanized monoclonal antibody, inhibits C1q, the initiator molecule of the classical complement pathway, and is designed to stop complement mediated inflammation and neuronal damage early in GBS.
  • In granting the designation, EMA stated that preliminary clinical data with ANX005 constitutes “a clinically relevant advantage” over IVIg for patients affected by GBS.
  • Importantly, Annexon has also achieved target enrollment of 225 patients in the randomized, double-blind, placebo-controlled Phase 3 trial of ANX005 in patients with GBS.

BioVie Presents Data Highlighting Baseline Characteristics of Study Population in Phase 3 Trial of NE3107 in Mild to Moderate Alzheimer’s Disease

Retrieved on: 
Monday, September 11, 2023
Cognition, Insulin resistance, WHR, DNA, Hyperglycemia, Dyslipidemia, Methylation, Hypertriglyceridemia, American Neurological Association, Calendar, Deficit spending, Risk, Inflammation, Biomarker, MCI, Hypertension, Hypercholesterolemia, CDR, RANTES, AAIC, Dementia, HIV disease progression rates, ANA, CRP, Complement component 1q, Patient, Probability, Abdominal obesity, Poster, CSF, ADAS, DNA methylation, AD, Gene, APOE, MMSE, Conference, Pharmaceutical industry, Nursing

CARSON CITY, Nev., Sept. 11, 2023 (GLOBE NEWSWIRE) -- BioVie Inc., (NASDAQ: BIVI) (“BioVie” or the “Company”) a clinical-stage company developing innovative drug therapies for the treatment of neurological and neurodegenerative disorders and advanced liver disease, today announced that preliminary baseline data from its multicenter, randomized, placebo-controlled Phase 3 study (NCT04669028) of NE3107 in patients with mild to moderate Alzheimer’s Disease (AD) was presented as a poster at the American Neurological Association (ANA) annual meeting, being held September 11-13, 2023 in Philadelphia, PA.

Key Points: 
  • The poster, Metabolic Dysregulation in Probable Alzheimer’s Disease (Christopher Reading, et al), is being presented by Joseph Palumbo, Chief Medical Officer of BioVie, and highlights the preliminary baseline metabolic and inflammation characteristics from the Phase 3 study population (see Table 1).
  • “Instead, it provides an understanding of the patient population at the start of the trial, as understood to date.
  • At baseline, the majority of the study population are coded with abdominal obesity (85%), hypertension (61%), and impaired glucose metabolism (IFG/T2D; 52%).
  • Both Aβ+ and Aβ− patients with dementia were enrolled in the study and had, at baseline, comparable CDR-SB scores indicative of mild dementia.

Annexon Highlights Recent Pipeline and Business Progress and Reports Second Quarter 2023 Financial Results

Retrieved on: 
Monday, August 7, 2023
Molecule, HD, Drug development, Geographic atrophy, ARCHER, G&A, Regulatory Agencies, Inflammation, Pivotal trial, LN, Differentiation, Complement component 1q, HIV disease progression rates, Investment, Appointment, Annual general meeting, GBS, R, Research, Visual acuity, ALS, Society, ASRS, Fine chemical, Pharmaceutical industry, EU, Patient

BRISBANE, Calif., Aug. 07, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today highlighted recent progress across its business and portfolio of complement therapies and reported second quarter 2023 financial results.

Key Points: 
  • Together, these data demonstrate the differentiated mechanism of inhibiting C1q to provide functional benefits for patients living with complement-mediated diseases.
  • Annexon plans to provide pipeline updates and outline key milestones across its business and portfolio of complement-targeted therapies during an R&D Day in the second half of 2023.
  • Following encouraging preliminary results which showed slowing of disease progression, additional data are expected in the second half of 2023.
  • General and administrative (G&A) expenses: G&A expenses were $7.4 million for the quarter ended June 30, 2023, compared to $8.3 million for the quarter ended June 30, 2022.

BioVie Presents Data Highlighting Role of Insulin Resistance and Neuroinflammation in the Development of Mild to Moderate in Alzheimer’s Disease

Retrieved on: 
Monday, June 26, 2023
Insulin resistance, WHR, Hyperglycemia, American Diabetes Association, Dyslipidemia, Hypertriglyceridemia, Deficit spending, Inflammation, Hypertension, Hypercholesterolemia, RANTES, HIV disease progression rates, CRP, Complement component 1q, Patient, Risk, Probability, Poster, ADAS, AD, APOE, MMSE, Safety, Pharmaceutical industry, Medical device

NE3107 is an oral small molecule, blood-brain permeable anti-inflammatory insulin sensitizer that binds extracellular signal-regulated kinase.

Key Points: 
  • NE3107 is an oral small molecule, blood-brain permeable anti-inflammatory insulin sensitizer that binds extracellular signal-regulated kinase.
  • BioVie’s Phase 3 trial is the largest study to date to evaluate the safety and efficacy of NE3107 in patients with AD.
  • NE3107 is the only anti-inflammatory agent currently in phase 3 development for AD.
  • Additional subgroup analysis revealed higher degrees of impaired glucose metabolism and insulin resistance among the APOE ε4− patients compared to their APOE ε4+ counterparts and comparable baseline MMSE scores, indicating that both groups had mild to moderate cognitive impairment.