Complement Inhibitors - Pipeline Insight, 2025

This “Complement Inhibitors - Pipeline Insight, 2025” report provides comprehensive insights about 50+ companies and 60+ pipeline drugs in Complement Inhibitors pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Complement Inhibitors: Understanding

Complement Inhibitors: Overview

Complement inhibitors are a class of drugs that target the complement system, an essential part of the immune system involved in inflammation, pathogen clearance, and tissue homeostasis. The complement system, composed of over 30 proteins, can be activated via three pathways: classical, alternative, and lectin. Activation results in a cascade that leads to the formation of the membrane attack complex (MAC), which can destroy pathogens and cells. However, excessive or uncontrolled complement activation is implicated in various diseases, including autoimmune disorders, kidney diseases, and conditions such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Complement inhibitors work by blocking key proteins in this cascade, thereby preventing tissue damage caused by overactivation.

The complement system plays a key role in the innate immune response, protecting the body against pathogens. However, its overactivation can result in tissue damage and inflammation. To maintain balance, the body utilizes natural complement inhibitors, and in clinical settings, biotechnologically developed agents are deployed to regulate complement activity effectively.

The complement system consists of a series of proteins that, when activated, work together to defend the body against pathogens, facilitate inflammation, and promote the clearance of debris or foreign agents. However, if not tightly regulated, the activation of this system can lead to tissue damage and contribute to the pathogenesis of various diseases, including autoimmune disorders and chronic inflammatory conditions. Complement inhibitors have been developed to modulate this system’s activity by targeting key proteins involved in its activation cascade. These inhibitors work through several mechanisms. Some inhibitors, such as monoclonal antibodies or small molecules, block the binding of complement proteins to their receptors on target cells or pathogens, thereby preventing their activation. Other inhibitors target the proteolytic enzymes in the complement pathway, such as C3 or C5 convertases, to prevent the cleavage of these proteins and halt further progression of the complement cascade.

Complement inhibitors represent a growing field of therapeutics with applications beyond rare diseases. In addition to PNH and aHUS, research is exploring their use in common diseases such as age-related macular degeneration (AMD) and lupus nephritis, where complement overactivity plays a critical role. The development of novel complement inhibitors is driven by advances in understanding complement biology, and with ongoing clinical trials, the landscape of complement therapeutics is expected to expand significantly. This growing market reflects the potential of complement inhibitors to address both unmet medical needs and new indications in diverse therapeutic areas.

“Complement Inhibitors - Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the Complement Inhibitors pipeline landscape is provided which includes the disease overview and Complement Inhibitors treatment guidelines. The assessment part of the report embraces, in depth Complement Inhibitors commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Complement Inhibitors collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Complement Inhibitors R&D. The therapies under development are focused on novel approaches to treat/improve Complement Inhibitors.

Complement Inhibitors Emerging Drugs Chapters

This segment of the Complement Inhibitors report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Complement Inhibitors Emerging Drugs

MY008: Wuhan Createrna Science and Technology

MY008 is an investigational therapeutic designed to target complement-mediated diseases, including Paroxysmal Nocturnal Hemoglobinuria (PNH) and IgA Nephropathy (IgAN). In PNH, MY008 works by inhibiting the complement system to prevent red blood cell destruction (hemolysis), reducing symptoms like anemia and fatigue. For IgA Nephropathy, MY008 aims to suppress complement activation in the kidneys, reducing inflammation and proteinuria, thereby slowing disease progression. With its targeted approach, MY008 holds promise as a novel treatment option for these rare and chronic conditions, addressing unmet clinical needs. Currently the drug is in Phase III stage of its development for the treatment of IgA nephropathy.

IONIS-FB-LRx: Ionis Pharmaceuticals

IONIS-FB-LRx, also known as RG6299, is an investigational RNA-targeted medicine designed to reduce the production of complement factor B (FB), and the alternative complement pathway. IONIS-FB-LRx, an antisense drug using Ionis' advanced LIgand Conjugated Antisense (LICA) technology, reduces the production of FB, a key protein in the complement innate immune system. FB is predominately produced in the liver and circulates throughout the vascular system, including vessels in the eye and kidney. This complement protein plays a pivotal role in an innate immunogenic cascade that, when overactivated, has been associated with the development of several complement-mediated diseases. Currently the drug is in Phase III stage of its development for the treatment of IgA nephropathy.

Empasiprubart: Argenx

Empasiprubart (formerly ARGX-117) is designed to be a humanized sweeping antibody that binds specifically to C2 in a pH- and Ca2+- dependent manner. C2 is a protein in the complement cascade which, when activated, leads to cell destruction. Binding of Empasiprubart is intended to inhibit the function of C2 and downstream complement activation.By blocking complement activity, Empasiprubart has the potential to reduce tissue inflammation and the adaptive immune response. Through this proposed mechanism, Empasiprubart could represent a broad pipeline opportunity across severe autoimmune indications. In October 2024, Argenx SE announced its third quarter 2024 financial results and provided a business update. Vision 2030 is the next phase in argenx’s long-term commitment to transform the treatment of autoimmune diseases by strengthening its leadership in FcRn biology, investing in its continuous pipeline of differentiated antibody candidates. The company also highlighted about the registrational study of empasiprubart in MMN to start by end of 2024.

Currently the drug is in Phase II stage of its development for the treatment of Multifocal Motor Neuropathy (MMN).

STSA 1002: Staidson (Beijing) Biopharmaceuticals Co., Ltd

STSA-1002 is an investigational fully humanized IgG1 monoclonal antibody targeting complement component C5a, developed by Staidson Biopharmaceuticals. By inhibiting C5a, it aims to reduce inflammation associated with conditions like acute respiratory distress syndrome (ARDS) and anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis. Additionally, a Phase I study is assessing its safety and tolerability in healthy subjects. These studies aim to establish STSA-1002's potential as a therapeutic option for complement-mediated inflammatory diseases. Currently the drug is in Phase I/II stage of its development for the treatment of Acute Respiratory Distress Syndrome.

ALS 205: Alsonex Pharmaceuticals

ALS-205 is a potent antagonist of the human C5a1 receptor with no activity at the C5a2 receptor. It binds to the human C5a1 receptor on white blood cells and is a functional insurmountable antagonist. ALS-205 has shown similar effects on other cell types including lymphocytes, monocytes and monocyte-derived macrophages. It is also highly receptor selective, failing even at 100 μM concentrations to antagonize myeloperoxidase release from leukocytes stimulated with fMet-Leu-Phe, leukotriene B4, platelet-activating factor, C3a, or IL-8. ALS-205 is chemically stable to peptidase degradation in blood and gastric fluids and is both small enough and sufficiently lipophilic to be orally active at ≤1 mg/kg/day in vivo and readily accesses the brain. Currently the drug is in Phase I stage of its development for the treatment of motor neuron disease.

Complement Inhibitors: Therapeutic Assessment

This segment of the report provides insights about the different Complement Inhibitors drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Complement Inhibitors

There are approx. 50+ key companies which are developing the therapies for Complement Inhibitors. The companies which have their Complement Inhibitors drug candidates in the most advanced stage, i.e. Phase III include, Wuhan Createrna Science and Technology and Ionis Pharmaceuticals.

Phases

The report covers around 60+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Complement Inhibitors pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Complement Inhibitors: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Complement Inhibitors therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Complement Inhibitors drugs.

Complement Inhibitors Report Insights

• Complement Inhibitors Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Complement Inhibitors Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Complement Inhibitors drugs?
• How many Complement Inhibitors drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Complement Inhibitors?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Complement Inhibitors therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Complement Inhibitors and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Wuhan Createrna Science and Technology
• Ionis Pharmaceuticals
• Argenx
• Staidson (Beijing) Biopharmaceuticals Co., Ltd
• Alsonex Pharmaceuticals
• AstraZeneca
• Aptarion Biotech
• NovelMed Therapeutics
• Arrowhead Pharmaceuticals
• BioCryst Pharmaceuticals
• I-MAB Biopharma
• 4D Molecular Therapeutics
• Akari Therapeutics Plc.

Nimble Therapeutics

• EyePoint

Key Products

• MY008
• IONIS-FB-LRx
• Empasiprubart
• STSA 1002
• ALS 205
• Gefurulimab
• AON-D21
• NM8074
• ARO-CFB
• BCX10013
• TJ 210
• 4D-175
• Nomacopan
• Research Program: Oral C5 Inhibitor
• Research Program

This product will be delivered within 1-3 business days.