Complement Inhibitors - Pipeline Insight, 2024

This “Complement Inhibitors - Pipeline Insight, 2024” report provides comprehensive insights about 40+ companies and 50+ 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.

Geography Covered

• Global coverage

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.

One of the most prominent complement inhibitors is eculizumab, which targets C5, a protein involved in the terminal complement pathway. By inhibiting C5, eculizumab prevents the formation of the MAC, reducing the risk of complement-mediated cell lysis. This drug has demonstrated efficacy in treating PNH, a rare hematologic disorder, and aHUS, a condition that leads to kidney damage. Newer complement inhibitors target different parts of the cascade, such as C3 or Factor D, offering more selective approaches to complement inhibition. These drugs are particularly significant for patients with diseases that are resistant to C5 inhibitors or for those where upstream inhibition might offer broader protection.

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, 2024" 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

Gefurulimab: AstraZeneca

Gefurulimab, also known as ALXN1720, is a bispecific antibody developed by AstraZeneca, specifically under its Alexion division. This innovative therapeutic agent is primarily designed to target the complement component C5 and human serum albumin, making it a promising candidate for treating various chronic disorders, particularly generalized myasthenia gravis (gMG).

Gefurulimab functions by inhibiting the cleavage of complement component C5 into its active forms, C5a and C5b, thereby blocking the activation of the terminal complement pathway. This mechanism is crucial in conditions where the complement system contributes to disease pathology. The antibody has been engineered from llama-derived variable heavy-chain domains (VHHs), which are known for their stability and ability to bind tightly to antigens. Specifically, gefurulimab binds to both C5 and albumin simultaneously, enhancing its therapeutic potential. The drug is in the Phase III stage of development for the treatment of generalized myasthenia gravis (gMG).

ANX005: Annexon, Inc.

ANX005 is a clinical-stage investigational monoclonal antibody developed by Annexon, Inc. for the treatment of Guillain-Barré Syndrome (GBS). ANX005 functions by selectively inhibiting C1q, thereby blocking its binding to peripheral nerves. This action is intended to halt the autoimmune attack that characterizes GBS, allowing for quicker recovery of muscle strength and overall function12. The therapy is administered intravenously and currently is in the Phase III stage of development for the treatment of Guillain-Barre Syndrome.

DNTH103: Dianthus Therapeutics

DNTH103 is an investigational monoclonal antibody developed by Dianthus Therapeutics, specifically designed to selectively inhibit the active form of the C1s protein in the classical complement pathway. This pathway plays a significant role in various autoimmune disorders, making DNTH103 a promising candidate for treating conditions such as generalized Myasthenia Gravis (gMG), Multifocal Motor Neuropathy (MMN), and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). The drug is in the Phase II stage of development for the treatment of generalized myasthenia gravis (gMG).

ALS 205: Alsonex Pharmaceuticals

ALS-205, also known as PMX205, is an investigational drug developed at the University of Queensland, targeting the C5a receptor (C5aR). This receptor is implicated in neuroinflammation, which plays a significant role in the progression of motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS). ALS-205 works by blocking C5aR, a molecule that has been found to be elevated in patients with MND. By inhibiting this receptor, the drug aims to reduce neuroinflammation associated with ALS and potentially slow disease progression. The drug is in the Phase I stage of 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. 40+ 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, AstraZeneca and Annexon, Inc.

Phases

The report covers around 50+ 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

• AstraZeneca
• Annexon, Inc.
• Dianthus Therapeutics
• Alsonex Pharmaceuticals
• Mallinckrodt
• CANbridge Pharmaceuticals
• Beijing Defengrei Biotechnology
• NovelMed Therapeutics
• DynamiCure Biotechnology
• CSL

Key Products

• Gefurulimab
• ANX005
• DNTH103
• ALS-205
• SLN-MNK-3
• CAN106
• BDB-1
• NM8074
• DCSZ11
• CSL040

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