CLINICAL TRIALS PROFILE FOR RILZABRUTINIB

All Clinical Trials for rilzabrutinib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03762265 ↗	A Study of PRN1008 in Patients With Pemphigus	Active, not recruiting	Principia Biopharma, a Sanofi Company	Phase 3	2019-01-08	This is a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (Blinded Treatment Period) followed by an Open-Label Extension Period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the Open-Label Extension Period, eligible patients may continue in a Long Term Extension Period of 48 weeks.
NCT03762265 ↗	A Study of PRN1008 in Patients With Pemphigus	Active, not recruiting	Principia Biopharma, Inc.	Phase 3	2019-01-08	This is a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (Blinded Treatment Period) followed by an Open-Label Extension Period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the Open-Label Extension Period, eligible patients may continue in a Long Term Extension Period of 48 weeks.
NCT04520451 ↗	Open Label Two-Arm Study to Evaluate Rilzabrutinib (PRN1008) in IgG4-Related Disease Patients	Recruiting	Massachusetts General Hospital	Phase 2	2020-08-22	PRN1008-017 is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: PRN1008 with glucocorticoids and (2) Active Comparator: glucocorticoids only.
NCT04520451 ↗	Open Label Two-Arm Study to Evaluate Rilzabrutinib (PRN1008) in IgG4-Related Disease Patients	Recruiting	Principia Biopharma Inc.	Phase 2	2020-08-22	PRN1008-017 is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: PRN1008 with glucocorticoids and (2) Active Comparator: glucocorticoids only.
NCT04520451 ↗	Open Label Two-Arm Study to Evaluate Rilzabrutinib (PRN1008) in IgG4-Related Disease Patients	Recruiting	Principia Biopharma, a Sanofi Company	Phase 2	2020-08-22	PRN1008-017 is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: PRN1008 with glucocorticoids and (2) Active Comparator: glucocorticoids only.
NCT04562766 ↗	Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)	Recruiting	Principia Biopharma Inc.	Phase 3	2020-12-14	This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of 35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients will receive rilzabrutinib or placebo 400mg twice daily. For each patient, the study will last up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This includes Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. There is a 4-week post dose follow-up. Patients who respond per specified criteria at the end of the Open-Label Period will be able to enter a 12-month Long-Term Extension (LTE).
NCT04562766 ↗	Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)	Recruiting	Principia Biopharma, a Sanofi Company	Phase 3	2020-12-14	This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of 35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients will receive rilzabrutinib or placebo 400mg twice daily. For each patient, the study will last up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This includes Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. There is a 4-week post dose follow-up. Patients who respond per specified criteria at the end of the Open-Label Period will be able to enter a 12-month Long-Term Extension (LTE).

Clinical Trial Conditions for rilzabrutinib

Condition Name

Condition Name for rilzabrutinib
Intervention	Trials
Healthy Volunteers	4
Immune Thrombocytopenia	3
Autoimmune Haemolytic Anaemia	2
Autoimmune Disorder	1

Condition MeSH

Condition MeSH for rilzabrutinib
Intervention	Trials
Purpura, Thrombocytopenic, Idiopathic	3
Immunoglobulin G4-Related Disease	2
Anemia, Hemolytic, Autoimmune	2
Anemia, Sickle Cell	1

Clinical Trial Locations for rilzabrutinib

Trials by Country

Trials by Country for rilzabrutinib
Location	Trials
United States	40
Spain	9
Italy	8
Canada	8
United Kingdom	8

Trials by US State

Trials by US State for rilzabrutinib
Location	Trials
Florida	6
California	5
North Carolina	3
New York	3
Minnesota	3

Clinical Trial Progress for rilzabrutinib

Clinical Trial Phase

Clinical Trial Phase for rilzabrutinib
Clinical Trial Phase	Trials
PHASE3	5
PHASE2	3
PHASE1	3

Clinical Trial Status

Clinical Trial Status for rilzabrutinib
Clinical Trial Phase	Trials
Recruiting	8
COMPLETED	4
Not yet recruiting	3

Clinical Trial Sponsors for rilzabrutinib

Sponsor Name

Sponsor Name for rilzabrutinib
Sponsor	Trials
Sanofi	13
Principia Biopharma, a Sanofi Company	6
Principia Biopharma Inc.	2

Sponsor Type

Sponsor Type for rilzabrutinib
Sponsor	Trials
Industry	22
Other	2

Clinical Trials Update, Market Analysis, and Projection for Rilzabrutinib

Last updated: October 4, 2025

Introduction

Rilzabrutinib is an investigational oral Bruton's tyrosine kinase (BTK) inhibitor developed by Sanofi for the treatment of autoimmune diseases, including immune thrombocytopenia (ITP) and pemphigus vulgaris (PV). As a promising candidate in the immunology space, rilzabrutinib’s clinical development has garnered significant attention from stakeholders aiming to address unmet medical needs in autoimmune disorders. This report synthesizes the latest clinical trial developments, market dynamics, and future projections for rilzabrutinib, offering valuable insights for pharmaceutical firms, investors, and healthcare strategists.

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Clinical Trials Update

Current Clinical Trial Landscape

Rilzabrutinib’s clinical evaluation concentrates on autoimmune conditions, especially ITP and PV. The drug’s development pipeline is marked by multiple ongoing Phase 2 and Phase 3 trials, with key updates outlined below:

• Immune Thrombocytopenia (ITP):

  • Phase 2/3 ERSTRUCT trial: Sanofi initiated this pivotal study to assess rilzabrutinib’s efficacy and safety in chronic ITP patients who are refractory to standard treatments. Preliminary data indicates promising trends toward increasing platelet counts with a tolerable safety profile.
  • Enrollment status: As of late 2022, recruitment remains active, with topline results expected mid-2023.

• Pemphigus Vulgaris (PV):

  • Phase 2 clinical trial: Rilzabrutinib has demonstrated encouraging efficacy signals in reducing blistering and autoantibody levels in PV patients.
  • Data release: Sanofi announced positive interim results in 2022, leading to accelerated plans for larger confirmatory trials.

Key Trial Outcomes and Challenges

Representative findings show that rilzabrutinib inhibits pathogenic B-cell activation and autoantibody production—core mechanisms underlying autoimmune pathologies. Notably, its oral administration and favorable safety profile position it favorably compared to traditional immunosuppressants.

However, hurdles persist. The drug’s efficacy, while promising, remains to be conclusively proven in large-scale, randomized trials. Additionally, competition from other BTK inhibitors like ibrutinib and acalabrutinib, primarily approved for hematological malignancies, presents strategic challenges.

Regulatory and Development Outlook

Sanofi’s regulatory submissions are anticipated once robust phase 3 data confirm efficacy and safety. The company has prioritized accelerated pathways, leveraging orphan drug designation for PV to shorten development timelines. Pending successful trial outcomes, rilzabrutinib could target regulatory approvals by 2025–2026, contingent on positive efficacy-safety data.

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Market Analysis

Existing Market Landscape

Autoimmune disease therapeutics encompass a vast global market, with several established drug classes:

• Corticosteroids and immunosuppressants: First-line therapies, but associated with adverse effects.
• Biologics: Rituximab, rituximab biosimilars, and newer monoclonal antibodies targeting B-cells or cytokines dominate the landscape.
• Other small molecules: JAK inhibitors (e.g., tofacitinib) are emerging options.

The global autoimmune disease therapeutics market was valued at approximately $35 billion in 2022[1], with a compounded annual growth rate (CAGR) of around 6% forecasted through 2030.

Market Potential for Rilzabrutinib

Rilzabrutinib’s potential hinges on its ability to offer:

• Oral administration facilitating patient compliance.
• Targeted action with fewer systemic immunosuppressive effects.
• Efficacy in refractory cases, expanding treatment options for unmet needs.

Considering the market size for ITP and PV, estimated at $1.8 billion and $2.4 billion globally in 2022 respectively[2], rilzabrutinib could capture a significant niche, especially if it demonstrates superiority or comparable efficacy with improved safety vs. existing therapies.

Competitive Dynamics

Key competitors include:

• Rituximab and its biosimilars: Widely used but associated with risks like immunosuppression.
• Bruton’s tyrosine kinase inhibitors: Ibrutinib, acalabrutinib, and others primarily target hematological malignancies, with limited autoimmune indications.

Rilzabrutinib’s distinctive oral route and specificity may carve a unique positioning within this competitive landscape, particularly if it secures approval for first-line or refractory patient subsets.

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Projection and Future Outlook

Market Penetration and Revenue Projections

Based on current clinical progress and unmet needs, rilzabrutinib could achieve:

• Peak sales: Estimated between $1–2 billion globally within 7–10 years of market entry, assuming successful commercialization for ITP and PV.
• Market share: Positioned to seize 15–25% of the autoimmune biologic and targeted small-molecule market segments, given its novel profile.

Strategic Factors Influencing Growth

• Regulatory approvals: Success in upcoming Phase 3 trials and subsequent filings is paramount.
• Pricing and reimbursement: Competitive pricing aligned with biologics and blockbuster small molecules will impact adoption.
• Post-marketing data: Real-world safety and efficacy outcomes will influence clinician acceptance and payer coverage.
• Pipeline expansion: Potential trials in additional autoimmune indications (e.g., lupus, rheumatoid arthritis) could broaden market opportunity.

Risks and Barriers

• Clinical efficacy: Failure to meet primary endpoints may impair market prospects.
• Competitive innovation: Emergence of alternative therapies or newer BTK inhibitors could diminish rilzabrutinib’s relative market share.
• Regulatory hurdles: Delays or setbacks could postpone commercialization timelines.

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Conclusion

Rilzabrutinib represents a significant advancement in the targeted treatment of autoimmune diseases. Its current clinical trial progress signals a cautiously optimistic outlook, with substantial upside contingent upon robust efficacy and safety data. The drug’s potential to address unmet needs in ITP and PV positions it favorably within the expanding autoimmune therapeutics landscape. Strategic maneuvers—such as accelerated approvals, effective pricing strategies, and pipeline expansion—will be critical to maximizing market penetration and revenue generation.

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Key Takeaways

• Clinical readiness: Rilzabrutinib is progressing through pivotal trials for ITP and PV, with promising efficacy signals and acceptable safety profiles.
• Market opportunity: Autoimmune diseases represent a multi-billion-dollar landscape ripe for targeted therapies; rilzabrutinib could garner significant market share if approved.
• Competitive edge: Its oral formulation and specific mechanism position it favorably against existing biologics and immunosuppressants.
• Strategic outlook: Success hinges on regulatory approvals post-phase 3 trials, priced competitively, with clear positioning among autoimmune treatment options.
• Future directions: Pipeline expansion into broader autoimmune indications and integration of real-world data will accelerate growth prospects.

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FAQs

1. When is rilzabrutinib expected to receive regulatory approval?
Pending positive Phase 3 trial results, Sanofi aims for submission by 2024–2025, with potential approval around 2025–2026.

2. How does rilzabrutinib compare to existing therapies for ITP and PV?
It offers oral administration with a targeted mechanism—potentially more tolerable and effective in refractory cases compared to traditional immunosuppressants and biologics.

3. What are the main challenges facing rilzabrutinib’s market success?
Key challenges include demonstrating definitive efficacy in large trials, managing competition from other BTK inhibitors and biologics, and navigating regulatory and reimbursement pathways.

4. Are there any known safety concerns associated with rilzabrutinib?
Current data suggest a favorable safety profile, but comprehensive safety data will only be available post-approval with larger patient cohorts.

5. Will rilzabrutinib be used in combination with other therapies?
Combination strategies are under exploration, especially in refractory disease subsets, to enhance efficacy, though regulatory guidance will determine acceptable protocols.

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References

[1] MarketResearch.com, “Autoimmune Therapeutics Market Size & Trends.” 2022.
[2] GlobalData, “Autoimmune Disease Market Forecast,” 2022.