A Parallel-group Treatment, Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Umbrella Study to Evaluate the Efficacy and Safety of Frexalimab, SAR442970, and Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis (FSGS) or Minimal Change Disease (MCD)

This is a parallel, Phase 2a, double-blind, 6-arm study for the treatment of primary focal segmental glomerulosclerosis (FSGS) or primary minimal change disease (MCD).
The purpose of this study is to measure the change in proteinuria and its impact on the rates of remission of nephrotic syndrome with frexalimab, SAR442970, or rilzabrutinib compared with placebo in participants with primary FSGS or primary MCD aged 16 to 75 years.
Study details for each participant include:
The study duration will be up to 76 weeks. The treatment duration will be 24 weeks. There will be up to 18 visits.

Start Date19 Dec 2024

Sponsor / Collaborator

Sanofi

NCT06342700

/ CompletedPhase 1

A Randomized, Open-label, Single-dose, 4-period, 4--sequence, Crossover Phase I Relative Bioavailability Study Comparing Crystalline Formulation Tablet Versus Reference Amorphous Formulation Tablet of Rilzabrutinib (SAR444671) in Fasted and Fed Conditions in Healthy Male and Female Participants

This is a cross-over, Phase 1, 4-arm study. The purpose of this study is to measure the relative bioavailability and food effect of crystalline formulation rilzabrutinib and amorphous formulation rilzabrutinib in healthy male and female participants aged 18 to 55 years of age.
The total study duration per participant is expected to be up to 36 days, including:
Screening: up to 4 weeks
Treatment periods: once successfully screened, enrolled participants will be randomized to 1 of 4 treatment sequences with 4 single dose treatment periods.
Washout: One day washout is planned after each treatment period hence providing 2 days between doses.
Safety follow-up: participants will be asked to participate in an end-of-study safety assessment upon discharge from the clinical study unit, ie, on Day 8 of the study.

Start Date25 Mar 2024

Sponsor / Collaborator

Sanofi

ChiCTR2300069707

/ CompletedPhase 1IIT

A Phase 1, Single-center, Open-label Study to Evaluate the Pharmacokinetics, safety and Tolerability of Rilzabrutinib (PRN1008/ SAR444671) in Chinese Healthy Male and Female Subjects

Start Date23 Mar 2023

Sponsor / Collaborator-

100 Clinical Results associated with Rilzabrutinib

100 Translational Medicine associated with Rilzabrutinib

100 Patents (Medical) associated with Rilzabrutinib

Literatures (Medical) associated with Rilzabrutinib

01 Apr 2025·DRUGS

Update on the Treatment of Chronic Spontaneous Urticaria

Review

Author: Marcelino, Joao ; Kolkhir, Pavel ; Metz, Martin ; Kocatürk, Emek ; Okas, Tiia-Linda ; Li, Philip H ; Fok, Jie Shen

Chronic spontaneous urticaria (CSU) is a mast cell-mediated skin disease that presents with wheals, angioedema, or both for more than 6 weeks. Less than 10% of patients have complete control of their CSU (the main goal of CSU treatment) with second generation H1-antihistamines, the first-line treatment. About 70% of patients with antihistamine-refractory CSU do not reach complete control with omalizumab, the second-line treatment. Novel therapies are especially needed for patients with mast cell-activating immunoglobulin (Ig)G autoantibodies (autoimmune CSU) associated with nonresponse or late response to omalizumab. Furthermore, there is a lack of disease-modifying treatments that induce long-term CSU remission after drug withdrawal. Several emerging treatments can address these unmet needs including Bruton tyrosine kinase inhibitors, e.g., remibrutinib and rilzabrutinib; anti-KIT monoclonal antibodies, e.g., barzolvolimab; and anti-cytokine therapies, e.g., dupilumab. In clinical trials, 30-31%, 28-32%, and 38-51% of patients with CSU showed complete response to treatment with dupilumab (phase 3, week 24), remibrutinib (phase 3, week 24), and barzolvolimab (phase 2, week 12), respectively. The most common adverse events were injection site reactions for dupilumab (12%), respiratory tract infections (11%), headache (6%), and petechiae (4%) for remibrutinib and changes in hair color (14%), neutropenia / decreased neutrophil count (9%) and skin hypopigmentation (1%) for barzolvolimab. This review provides an update on the current state of development of treatments for CSU.

01 Mar 2025·AMERICAN JOURNAL OF HEMATOLOGY

Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study

Article

Author: Garg, Mamta ; Sholzberg, Michelle ; Kuter, David J. ; Tarantino, Michael D. ; Goncalves, Isaac ; McDonald, Vickie ; Daak, Ahmed ; Ypma, Paula F. ; Percy, Charles ; Diab, Remco ; Cooper, Nichola ; Gernsheimer, Terry B. ; Jansen, A. J. Gerard ; Bird, Robert ; Bogdanov, Lachezar H. ; Yao, Mengjie ; Mayer, Jiří ; Košťál, Milan

ABSTRACT:

Current treatments for persistent or chronic immune thrombocytopenia (ITP) are limited by inadequate response, toxicity, and impaired quality of life. The Bruton tyrosine kinase inhibitor rilzabrutinib was evaluated to further characterize safety and durability of platelet response. LUNA2 Part B is a multicenter, phase 1/2 study in adults with ITP (≥ 3 months duration, platelet count < 30 × 109/L) who failed ≥ 1 ITP therapy (NCT03395210, EudraCT 2017–004012‐19). Oral rilzabrutinib 400 mg bid was given over 24 weeks, with optional long‐term extension (LTE). Primary endpoints were safety and platelet counts ≥ 50 × 109/L on ≥ 8 of the last 12 weeks of main treatment without rescue medication. From 22 March2018 to 31 January2023, 26 patients were enrolled. Patients had baseline median platelet count 13 × 109/L, ITP duration 10.3 years, and six prior ITP therapies (46% splenectomized). Nine (35%) patients achieved the primary endpoint. Platelet counts ≥ 50 × 109/L or ≥ 30 × 109/L and doubling from baseline without rescue therapy were sustained for a mean 9.3 weeks. 11 (42%) LTE‐eligible patients were ongoing with median LTE platelet > 80 × 109/L. Three (12%) patients received rescue medication during main treatment, none in LTE. Clinically meaningful improvements were observed in fatigue and women's health. With a median treatment duration of 167 days (main treatment), 16 (62%) patients had ≥ 1 treatment‐related adverse event (AE), mainly grade 1, including diarrhea (35%), headache (23%), and nausea (15%). There was no treatment‐related grade ≥ 2 bleeding/thrombotic events/infections, serious AE, or death. Rilzabrutinib continues to demonstrate durable platelet responses with favorable safety profile in previously treated ITP patients.Trial Registration: NCT03395210, EudraCT 2017‐004012‐19

12 Nov 2024·Blood Advances

Selective Btk inhibition by PRN1008/PRN473 blocks human CLEC-2, and PRN473 reduces venous thrombosis formation in mice

Article

Author: Ivanova, Vanesa-Sindi ; Brown, Helena C. ; Storek, Michael ; Nunn, Philip A. ; LaStant, Jacob ; Chauhan, Abhi ; Watson, Steve P. ; Foulke, Matthew C. ; Theodore, Lindsay N. ; Cunningham, Adam F. ; Hicks, Alexandra ; Mukai, Shin ; Owens, Tim ; Langrish, Claire ; Garcia-Quintanilla, Lourdes ; Perez-Toledo, Marisol ; Rookes, Kieran ; Smith, Christopher W. ; Thomas, Mark R. ; Brill, Alexander ; Jossi, Sian ; Campos, Joana ; Francesco, Michelle ; Harbi, Maan ; Jooss, Natalie J. ; Nicolson, Phillip L. R.

Abstract:

Platelet C-type lectin-like receptor 2 (CLEC-2) is a hem-immunoreceptor tyrosine–based activation motif-containing receptor that has a critical role in venous thrombosis but minimal involvement in hemostasis. CLEC-2 can be blocked by Btk inhibitors. Treatment with ibrutinib is associated with increased bleeding due to off-target inhibition of Src family kinases (SFKs). Patients with X-linked agammaglobulinemia (XLA) who lack Btk, however, do not bleed, suggesting selective Btk inhibition as a viable antithrombotic strategy. We assessed the effects of selective Btk inhibitors PRN1008 (rilzabrutinib) and PRN473 on platelet signaling and function mediated by CLEC-2 and glycoprotein-VI. We used healthy donors and XLA platelets to determine off-target inhibitor effects. Inferior vena cava (IVC) stenosis and Salmonella infection mouse models were used to assess antithrombotic effects of PRN473 in vivo. PRN1008 and PRN473 potently inhibited CLEC-2–mediated platelet activation to rhodocytin. No off-target inhibition of SFKs was seen. PRN1008 treatment of Btk-deficient platelets resulted in minor additional inhibition of aggregation and tyrosine phosphorylation, likely reflecting inhibition of Tec. No effect on G protein-coupled receptor-mediated platelet function was observed. PRN473 significantly reduced the number of thrombi in podoplanin-positive vessels after Salmonella infection and the presence of IVC thrombosis after vein stenosis. The potent inhibition of human platelet CLEC-2 and reduced thrombosis in in vivo models, together with the lack of off-target SFK inhibition and absence of bleeding reported in rilzabrutinib-treated patients with immune thrombocytopenia, suggest Btk inhibition as a promising antithrombotic strategy.

News (Medical) associated with Rilzabrutinib

19 Mar 2025

·www.prnewswire.com

CABLIVI Market Reports Positive Growth - Strong Momentum Expected to Continue | DelveInsight

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultLicense out/inPhase 3Drug Approval

30 Jan 2025

·www.globenewswire.com

Press Release: Q4 sales growth of 10.3%, 2024 business EPS guidance exceeded, and strong business EPS rebound expected in 2025

Sanofi: Q4 sales growth of 10.3%, 2024 business EPS guidance exceeded, and strong business EPS rebound expected in 2025 Paris, January 30, 2025 Q4: sales growth of 10.3% at CER1 and business EPS2 of €1.31 Pharma launches up 56.5%, reaching sales of €0.8 billion, 8% of total sales, led by ALTUVIIIODupixent sales up 16.0% to €3.5 billionVaccines sales up 10.8% to €2.2 billion, driven by Beyfortus sales in EuropeBusiness EPS of €1.31, -11.0% at CER and -14.9% reported; IFRS EPS of €0.54 FY: double-digit sales growth and business EPS guidance exceeded Sales totaled €41.1 billion, an increase of 11.3% at CERSales targets exceeded: Dupixent >€13 billion and Beyfortus blockbuster status (€1.7 billion) in its first full yearResearch and Development expenses reached €7.4 billion, up 14.6%, in line with commitmentsBusiness EPS of €7.12, +4.1% at CER, above guidance, and -1.8% reported; IFRS EPS of €4.59The Board of Directors met on January 29, 2025; proposes a dividend of €3.92 for 2024, 30th year of consecutive increases Pipeline: increased investment and progress Q4: four regulatory approvals: Dupixent EoE (children) (EU), Kevzara PMR (EU), Cerdelga GD1 (children) (EU), Efluelda flu (JP)3FY: 14 regulatory approvals of medicines and vaccines, 21 regulatory submission acceptances, and eight positive phase 3 readouts emphasize a positive and improving pipeline momentum Further progress towards a focused biopharma business Intention to sell a controlling stake in Opella consumer health at an attractive valuation; closing in Q2 2025 at the earliest4 Guidance In 2025, sales are anticipated to grow by a mid-to-high single-digit percentage at CER5. Sanofi confirms the expectation of a strong rebound in business EPS with growth at a low double-digit percentage at CER (before share buyback).6Sanofi intends to execute a share buyback program in 2025 of €5 billion. Shares will be purchased preferably through block trades and in the open market with the purpose of cancellation. Paul Hudson, Chief Executive Officer: “We achieved double-digit sales growth in 2024 while pursuing the transformation of the company. Innovation was a key driver of our growth as launches already contributed 11 percent of sales, with Beyfortus becoming a blockbuster in its first full year of sales. We exceeded our business EPS guidance. In 2024, we announced an intention to sell a controlling stake in Opella consumer health, which will make Sanofi a focused, science-driven biopharma company. We increased R&D investments and achieved significant progress with our pipeline in 2024, including positive phase 3 study results for new medicines such as rilzabrutinib in rare diseases and tolebrutinib in multiple sclerosis. As we enter 2025, we expect continued, solid growth in sales and a strong rebound in earnings. We are also confident in the mid to long-term growth prospects of Sanofi, supported by ongoing launches, Dupixent (currently expected to reach sales of around €22 billion in 20307, in line with the current ambition), and expected future launches from our pipeline.” Q4 2024 Change Change at CER FY 2024 Change Change at CER IFRS net sales reported €10,564m +9.1% +10.3% €41,081m +8.6% +11.3% IFRS net income reported €683m — — €5,744m +6.4% — IFRS EPS reported €0.54 — — €4.59 +6.5% — Free cash flow8 €2,340m -25.5% — €5,955m -19.6% — Business operating income €2,078m -11.8% -7.7 % €11,343m +1.5% +7.6% Business net income €1,642m -15.1% -11.2 % €8,912m -1.8% +4.1% Business EPS €1.31 -14.9% -11.0% €7.12 -1.8% +4.1% 1 Changes in net sales are at constant exchange rates (CER) unless stated otherwise (definition in Appendix 9).2 To facilitate an understanding of operational performance, Sanofi comments on the business net income statement which is a non-IFRS financial measure (definition in Appendix 9). The income statement is in Appendix 3 and a reconciliation of reported IFRS to business net income is in Appendix 4.3 For the definition of medical and scientific terms, please see the first use of the word in the Pipeline update section.4 Subject to finalization of definitive agreements and subject to obtaining regulatory approvals from the competent authorities.5 In 2025, sales growth will exclude any impact from hyperinflation. In 2024, it is estimated that sales growth benefited by 1.8 percentage points.6 Applying average January 2025 exchange rates, the currency impacts are estimated between +2% and +3% on sales and +2% and +3% on business EPS.7 At CER.8 Free cash flow is a non-IFRS financial measure (definition in Appendix 9). Attachment Press_Release

Phase 3Clinical ResultFinancial StatementBiosimilarDrug Approval

31 Dec 2024

·www.echemi.com

Sanofi Submits Market Authorization Application for BTK Inhibitor Rilzabrutinib

According to information published on the CDE (Center for Drug Evaluation) website on December 30, Sanofi’s market authorization application for its BTK inhibitor Rilzabrutinib has been officially accepted. Sanofi has conducted several clinical trials in China for Rilzabrutinib to treat autoimmune diseases, including a Phase III study (CTR20220447) for immune thrombocytopenia (ITP) and a Phase IIb study (CTR20212864) for warm autoimmune hemolytic anemia. In April 2024, Sanofi announced that its Phase III LUNA 3 study achieved positive results in treating adult patients with persistent or chronic immune thrombocytopenia (ITP), meeting its primary endpoint. Based on these results, Sanofi planned to submit the market authorization application for Rilzabrutinib. If approved, Rilzabrutinib will become the first BTK inhibitor approved for the ITP indication. The LUNA 3 study was a randomized, multicenter, double-blind, placebo-controlled clinical trial. It evaluated the efficacy and safety of Rilzabrutinib (400 mg twice daily) versus placebo in treating adult and adolescent patients with persistent or chronic ITP who had previously received treatment. The double-blind treatment phase lasted 12 to 24 weeks, followed by a 28-week open-label treatment period with Rilzabrutinib, and concluded with a 4-week safety follow-up or a long-term extension study. The primary endpoint of the study was the proportion of patients achieving a platelet count of ≥50,000/μL for at least 8 weeks during the final 12 weeks of the 24-week double-blind treatment phase without requiring rescue therapy. The baseline platelet count of patients in the study was 15,000/μL, while the normal range is 150,000–450,000/μL. The study results showed that the proportion of patients achieving sustained platelet response was significantly higher in the Rilzabrutinib group compared to the placebo group, with both clinical and statistical significance. Furthermore, the study met its key secondary endpoints. Regarding safety, Rilzabrutinib’s safety profile was consistent with previous studies. Rilzabrutinib, developed by Principia Biopharma, is an oral, reversible, covalent BTK inhibitor. In August 2020, Sanofi acquired Principia Biopharma for $3.68 billion, gaining access to multiple products, including Rilzabrutinib. BTK is expressed in B cells, mast cells, and other cells involved in innate immune responses and plays a role in the development of various autoimmune diseases. Rilzabrutinib helps treat ITP complications by reducing the production of pathogenic autoantibodies and macrophage-mediated platelet destruction. Immune thrombocytopenia (ITP) is a serious acquired autoimmune hematologic disorder characterized by autoantibody-mediated platelet destruction and impaired platelet production, resulting in reduced platelet counts (<100,000/μL) and an increased risk of life-threatening bleeding, such as intracranial hemorrhage. ITP also significantly impacts patients’ quality of life, including fatigue and cognitive dysfunction. In addition to the ITP indication, Sanofi is exploring Rilzabrutinib’s therapeutic potential for other autoimmune diseases, such as IgG4-related disease, warm autoimmune hemolytic anemia, chronic spontaneous urticaria, and prurigo nodularis.

Clinical ResultPhase 3ImmunotherapyDrug ApprovalAcquisition

100 Deals associated with Rilzabrutinib

External Link

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Purpura, Thrombocytopenic, Idiopathic	NDA/BLA	China	Sanofi Srl	30 Dec 2024
Purpura, Thrombocytopenic, Idiopathic	NDA/BLA	China	Genzyme Corp.	30 Dec 2024
Pemphigus	Phase 3	United States	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	Argentina	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	Australia	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	Brazil	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	Bulgaria	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	Canada	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	Croatia	Principia Biopharma, Inc.	08 Jan 2019
Pemphigus	Phase 3	France	Principia Biopharma, Inc.	08 Jan 2019

Clinical Result

Indication

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05002777 (ASH2024)	Phase 2	Warm autoimmune hemolytic anemia	-	Rilzabrutinib 400 mg bid	kcicuhzdnj(epwbxjboyp) = 23% vogyprmiyd (aojhhvngfi ) View more	-	09 Dec 2024
				Concomitant CS
NCT04562766 (LUNA 3, ASH2024) Manual	Phase 3	Purpura, Thrombocytopenic, Idiopathic	202	rilzabrutinib	afkcyruhej(emtseiggpc) = kokuxvrukp xuonuainkq (azezqxbfww ) Met View more	Positive	08 Dec 2024
				placebo	afkcyruhej(emtseiggpc) = cwttwcjtrx xuonuainkq (azezqxbfww ) Met View more
ASH2024	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	-	Rilzabrutinib 400 mg bid	qdfpubtcst(kbkwmodqdw) = atghsgrxgr vjsxhgatqx (fwiofkzfxv ) View more	-	08 Dec 2024
				Placebo	mqfhababbk(eclfwgxpts) = tetsosvdgo jnybbupdcl (qvwcbbthtw, 2.9) View more
NCT05104892 (ATS2024) Manual	Phase 2	Asthma	64	Rilzabrutinib 400 mg BID	rehzlqjlcj(azvxhklowk) = uvwktngacx eexcjutsqy (cxhytklrsd ) View more	Positive	22 May 2024
NCT03395210 (LUNA 2, EHA2024) Manual	Phase 2	Purpura, Thrombocytopenic, Idiopathic Second line	71	Rilzabrutinib 400 mg bid	azfrdpivif(roqunvusex) = lohlqzydvb hsbhsialrt (slxkmxxkqm ) View more	Positive	14 May 2024
				Rilzabrutinibhrombopoietin-receptor agonists (TPO-RA) or corticosteroids (CS)	cstowcnpsk(tvqkaaykyx) = oaofxlqozz xeervtajwr (evobzxppbp ) View more
NCT04562766 (LUNA 3, EHA2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	202	Rilzabrutinib 400 mg bid	zxmbdzivhz(sunkspclwf) = fhygckfbss etclmsrnik (kewzxttajs ) View more	Positive	14 May 2024
NCT03395210 (EHA2024)	Phase 2	Purpura, Thrombocytopenic, Idiopathic	-	Rilzabrutinib 400 mg bid	egywimprkp(bkoayufxrw) = In the main and LTE periods, 11 (41%) patients had treatment-related adverse events (TRAEs). All TRAEs in the LTE were transient and grade 1/2, except non-serious, grade 3 influenza and lower respiratory tract infection in 1 patient. No related bleeding or thrombotic events, serious adverse events, or deaths were reported. jclywbecuq (xegpftcyzp )	-	14 May 2024
				Concomitant ITP therapy
NCT03395210 (Pubmed) Manual	Phase 1/2	Purpura, Thrombocytopenic, Idiopathic	16	Rilzabrutinib 400 mg	oqdnlhrdsp(bxrfdmqokn) = Adverse events related to treatment were grade 1 or 2 and transient, with no bleeding, thrombotic, or serious adverse events. twjpghpwvx (eoxcklxzon )	Positive	09 Apr 2024
NCT05107115 (RILECSU, AAAAI2024) Manual	Phase 2	Chronic Urticaria	160	rilzabrutinib 400mg once-every-evening	pyozdilduw(syecbhbubv) = Rilzabrutinib was well tolerated; adverse events occurring at a higher frequency with rilzabrutinib vs placebo included headache, nausea, and diarrhea. wshsyqbjfj (gsrcqgvwcc )	Positive	24 Feb 2024
				rilzabrutinib 400mg twice-a-day
NCT03395210 (Part B, ASH2023)	Phase 1/2	Purpura, Thrombocytopenic, Idiopathic	9	Rilzabrutinib 400 mg bid	mxdomgljpn(jrwpllnwal) = oqnvsdjeyl qxcoxmfnfa (olzzcvqbtp, 17% - 56)	-	11 Dec 2023

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