Complement C3 inhibitors - Pipeline Insight, 2024

This “Complement C3 inhibitors - Pipeline Insight, 2024” report provides comprehensive insights about 6+ companies and 8+ pipeline drugs in Complement C3 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 C3 inhibitors Understanding

Complement C3 inhibitors: Overview

Complement component 3, often simply called C3, is a protein of the immune system. It plays a central role in the complement system and contributes to innate immunity. In humans it is encoded on chromosome 19 by a gene called C3. C3 plays a central role in the activation of the complement system. Its activation is required for both classical and alternative complement activation pathways. People with C3 deficiency are susceptible to bacterial infection. Several crystallographic structures of C3 have been determined and reveal that this protein contains 13 domains. The C3 precursor protein is first processed by the removal of 4 Arginine residues, forming two chains, beta and alpha, linked by a disulfide bond. The C3 convertase activates C3 by cleaving the alpha chain, releasing C3a anaphylatoxin and generating C3b (beta chain + alpha' (alpha prime) chain).

Complement C3 inhibitors - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Complement C3 inhibitors pipeline landscape is provided which includes the disease overview and Complement C3 inhibitors treatment guidelines. The assessment part of the report embraces, in depth Complement C3 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 C3 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 C3 inhibitors R&D. The therapies under development are focused on novel approaches to treat/improve Complement C3 inhibitors.

Complement C3 inhibitors Emerging Drugs Chapters

This segment of the Complement C3 inhibitors report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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 C3 inhibitors Emerging Drugs

Pegcetacoplan: Apellis Pharmaceuticals Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. The company reported top-line results from the Phase 3 DERBY and OAKS studies evaluating intravitreal pegcetacoplan, an investigational targeted C3 therapy, in 1,258 adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Based on results from the studies, the company plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in the first half of 2022.

AMY-101: Amyndas Pharmaceuticals AMY-101 is a novel complement C3-targeted therapeutic based on the 3rd-generation compstatin analog Cp40. Compstatins are synthetic cyclic peptides with strong affinity and selectivity for human and primate C3, discovered at the University of Pennsylvania by Professor John Lambris and his team. Compstatins inhibit complement centrally, at the level of C3, and interrupt all downstream pathways of the complement activation cascade. By inhibiting complement centrally, at the level of C3, AMY-101 and emerging 4th-generation compstatins, being developed by Amyndas, may prove more effective in treating a wide range of complement-mediated diseases than is possible with partial inhibitors of complement (such as anti-C5 agents) or other C3 inhibitors. It is currently being evaluated in Phase II stage of development for the treatment of Acute Respiratory Distress Syndrome Due to SARS-CoV-2 Infection and Gingivitis.

Complement C3 inhibitors: Therapeutic Assessment

This segment of the report provides insights about the different Complement C3 inhibitors drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Complement C3 inhibitors

There are approx. 6+ key companies which are developing the therapies for Complement C3 inhibitors. The companies which have their Complement C3 inhibitors drug candidates in the most advanced stage, i.e. phase III include Apellis Pharmaceuticals.

Phases

This report covers around 8+ 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 C3 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

• Subcutaneous
• Intravenous
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Peptides
• Polymer
• Small molecule
• Product Type

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

Complement C3 inhibitors: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Complement C3 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 C3 inhibitors drugs.

Complement C3 inhibitors Report Insights

• Complement C3 inhibitors Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Complement C3 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 C3 inhibitors drugs?
• How many Complement C3 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 C3 inhibitors?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Complement C3 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 C3 inhibitors and their status?
• What are the key designations that have been granted to the emerging drugs?

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