C3 Complement Inhibitor - Target Population, Competitive Landscape, and Market Forecast - 2034

Key Highlights:

• C3 is a key complement protein, located at the nexus of all complement activation pathways. Extracellular, tissue, cell-derived, and intracellular C3 plays critical roles in the immune response that is dysregulated in many diseases, making it an attractive therapeutic target.
• Currently, Apellis' pegcetacoplan is the only C3 inhibitor approved in paroxysmal nocturnal hemoglobinuria (PNH) and geographic atrophy, with brand name EMPAVELI and SYFOVRE, respectively.
• A few of the potential emerging C3 complement inhibitors are Arrowhead's ARO-C3 (RNAi based C3 inhibitor), and Amyndas' AMY-101.
• Both ARO-C3 and AMY-101 are currently being investigated in a Phase I study for C3 Glomerulopathy (C3G) and IgA Nephropathy (IgAN).
• The market size of C3 complement inhibitor is expected to increase at a steady growth rate, owing to anticipated commercial success of emerging and marketed therapies in multiple complement dysregulated disorders during our forecast period (2024-2034).

DelveInsight's "C3 Complement inhibitor - Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the C3 complement inhibitors, historical and forecasted epidemiology, competitive landscape as well as the C3 complement inhibitors market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The C3 complement inhibitors market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM C3 inhibitors market size from 2020 to 2034. The report also covers current C3 inhibitors treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

C3 Complement Inhibitors Disease Understanding and Treatment Algorithm

C3 Complement Inhibitors Overview

Three major pathways of complement which are the classical, alternative and lectin pathways converge on complement C3. As the central component among the intricate protein network of the complement system, C3 is associated with diverse immunomodulatory system and biological process that affect human physiology and pathophysiology, thereby representing a 'hot-spot' for current complement-targeted therapeutic intervention. It is the most abundant complement protein which mediates numerous protective effects or pathological effects of complement. C3 plays roles in complement cascade by active fragments or complexes, which are achieved through a panel of enzyme processing procedure. C4b2a is C3 convertase (C3-cleaving enzyme) that triggers the activation in classical (antibody) or lectin (sugar) pathways, whereas in the alternative pathway that is C3bBb.The alternative pathway, triggered by spontaneous hydrolysis of C3 to C3-H2O, also generates C3 convertase (C3(H2O)Bb), leading to C3 cleavage and amplification. The resulting C3b facilitates further complement activation, including the formation of the MAC complex, essential for cell membrane permeation and lysis.

C3 Complement Inhibitors Treatment

Apellis' pegcetacoplan is currently the only approved C3 inhibitor in the complement dysregulation disorder market. On February 17, 2023, FDA approved SYFOVRE (pegcetacoplan) for the treatment of Geographic atrophy secondary to age-related macular degeneration. The FDA previously approved pegcetacoplan (EMPAVELI) as a therapy for adult patients with PNH who are treatment naive and those switching from treatment with SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab). Pegcetacoplan works by inhibiting C3 to regulate downstream immune response in the complement system. Dysregulated activation of this system can cause both inflammation and damage to motor neurons.

C3 Complement Inhibitors Drug Chapters

The drug chapter segment of the complement inhibitors reports encloses a detailed analysis of complement inhibitors' marketed drugs and late-stage (Phase I and Phase II) pipeline drugs. It also helps understand the complement inhibitors' clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Marketed Drugs

Pegcetacoplan - Apellis

In 2021, EMPAVELI (pegcetacoplan) became the first C3 compliment inhibitor, administered subcutaneously, to be approved by the FDA for the treatment of PNH. Later, in 2023, Apellis launched it under a different brand name, SYFOVRE (pegcetacoplan injection), for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

EMPAVELI is available only through a restricted program under a risk evaluation and mitigation strategy. Meningococcal (a type of bacteria) infections can occur in patients taking Empaveli and can become life-threatening or fatal if not treated early.

Pegcetacoplan binds to C3 and its activation fragment C3b, thereby inhibiting the cleavage of C3 into C3a and C3b, and the downstream effectors of complement activation, leading to regulation of MAC-mediated intravascular haemolysis and C3-mediated extravascular haemolysis.

Emerging Drugs

ARO-C3: Arrowhead

ARO-C3 is designed to reduce production of complement component 3 as a potential treatment for various complement mediated diseases. It uses a technology called RNA interference (RNAi). When a gene is read to produce a protein, the code providing instructions for making the protein is copied into a temporary molecule called messenger RNA. This RNA molecule is then used by other cell machinery components as a template for making the protein.The basic idea behind RNAi therapies such as ARO-C3 is to inactivate the messenger RNA of a specific gene, in order to reduce the amount of protein that is produced.

The ARO-C3 is currently being investigated in a Phase I study involving adult patients with complement-mediated renal disease (C3 Glomerulopathy and IgA Nephropathy).

C3 Complement Inhibitors Market Outlook

The approval of Apellis' pegcetacoplan in Geographic atrophy and PNH has set a positive example for other companies who are investigating their drugs in multiple complement mediated diseases.

Furthermore, since C5 inhibitors are associated with heterogeneous responses among patients, C3 offers a potent alternative to the current C5 inhibitors such as SOLIRIS and ULTOMIRIS.

Also, since PNH and aHUS has already been crowded with a lot of marketed/emerging drugs, and anticipated generic entrants, the companies have now shifted their focus to other complement dysregulation disorders, including but not limited to C3G, IgA-N, and Geographic Atrophy.

Besides major players, small pharma companies are currently exploring RNAi based C3 inhibitors, with an aim to offer a more robust product with better clinical efficacy.

C3 Complement Inhibitors Drugs Uptake

This section focuses on the uptake rate of potential approved and emerging C3 Complement inhibitors expected to be launched in the market during 2020-2034.

C3 Complement Inhibitors Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III phase II, and phase I. It also analyzes key players involved in developing targeted therapeutics.

The presence of numerous drugs under different stages is expected to generate immense opportunity for C3 Complement Inhibitors market growth over the forecasted period.

Recent Events

• The report covers information on collaborations, acquisitions and mergers, licensing, patent details, and recently published results for complement inhibitors emerging therapies.
• May 2024 - Apellis Pharmaceuticals has announced positive one-year data from the Phase II NOBLE trial of pegcetacoplan in treating post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Below are the highlights of the results:
• The primary endpoint was met, with a significant proportion of patients showing a reduction in C3c staining after 12 weeks.
• Furthermore, 55% of patients, including those with IC-MPGN, showed zero C3c staining intensity, suggesting clearance of C3c deposits
• October 2023 - Apellis announced that the FDA has approved the EMPAVELI Injector for PNH patients. The EMPAVELI Injector is the first high-volume (20mL), subcutaneous on-body delivery system, which is developed in collaboration with Enable Injections, based on the enFuse(R) Syringe Transfer System.

KOL Views

To keep up with current and future market trends, we take Industry Experts' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on C3 complement inhibitors' evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.

DelveInsight's analysts connected with 15+ KOLs to gather insights; however, interviews were conducted with 7+ KOLs in the 7MM. We contacted institutes such as Duke University School of Medicine and others.

Their opinion helps understand and validate current and emerging therapy treatment patterns or C3 complement inhibitors market trends. This will support the clients in understanding potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of C3 complement inhibitors, explaining their mechanism, and therapies (current and emerging).
• Comprehensive insight into the competitive landscape, forecasts, the future growth potential of treatment rate, drug uptake, and drug information have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies that will impact the current landscape.
• A detailed review of the C3 complement Inhibitors market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM Complement inhibitors market.

C3 complement inhibitors report insights

• C3 complement Inhibitors Targeted Patient Pool
• Therapeutic Approaches
• C3 complement Inhibitors Pipeline Analysis
• C3 complement Inhibitors Market Size and Trends
• Existing and Future Market Opportunity

C3 complement inhibitors Report Key Strengths

• Eleven years Forecast
• The 7MM Coverage
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

C3 complement inhibitors Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT)

Key Questions:

• What was the C3 complement inhibitors total market size, the market size by therapies, market share (%) distribution, and what would it look like in 2034? What are the contributing factors for this growth?
• Which drug is going to be the largest contributor in 2034?
• Which is the most lucrative market for C3 complement inhibitors?
• Which drug accounted for maximum C3 complement inhibitors sales?
• What are the pricing variations among different geographies for approved therapies?
• How has the reimbursement landscape for C3 complement inhibitors evolved since the first one was approved? Do patients have any access issues that are driven by reimbursement decisions?
• What are the risks, burdens, and unmet needs of treatment with C3 complement inhibitors? What will be the growth opportunities across the 7MM for the patient population of C3 complement inhibitors?
• What are the key factors hampering the growth of the C3 complement inhibitors market?
• What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
• What key designations have been granted to the therapies for C3 complement inhibitors?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the C5 complement inhibitors Market.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of indication-wise current and emerging therapies under the conjoint analysis section to provide visibility around leading indications.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.