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 Date22 Aug 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

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.

01 Sep 2024·Expert Review of Hematology

Novel treatments for immune thrombocytopenia: targeting platelet autoantibodies

Review

Author: Arnold, Donald M. ; Harnett, Brian ; Al‐Samkari, Hanny ; Dalmia, Shreyash ; Al-Samkari, Hanny ; Arnold, Donald

INTRODUCTION:

Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by low platelets and an increased risk of bleeding. Platelet autoantibodies target major platelet glycoproteins and cause Fc-mediated platelet destruction in the spleen and reticuloendothelial systems. As mechanisms of disease, platelet autoantibodies are important therapeutic targets. Neonatal Fc receptor (FcRn) antagonists are a new class of therapeutics that reduce the half-life of immunoglobulin G including pathogenic platelet autoantibodies. Spleen tyrosine kinase (Syk) inhibitors interfere with Fc-mediated platelet clearance. Bruton's tyrosine kinase (BTK) inhibitors and B-cell activating factor (BAFF) inhibitors reduce antibody production. The efficacy of these targeted therapies provides new support for the role of platelet autoantibodies in pathogenesis of ITP even these antibodies can be difficult to detect.

AREAS COVERED:

This review includes an in-depth exploration of the pathophysiologic mechanisms of ITP, focusing on autoantibodies. Treatments outlined in this review include a) FcRn antagonists, b) complement inhibitors, c) B-cell directed therapies such as BTK inhibitors, and anti-BAFF agents, d) Syk inhibitors, e) plasma-cell directed therapies, and f) novel cellular therapeutic products.

EXPERT OPINION:

Platelet autoantibodies are often elusive in ITP, yet novel treatments targeting this pathway reinforce their role in the pathogenesis of this autoimmune platelet disorder.

01 Aug 2024·The Journal of investigative dermatology

Efficacy and Safety of Rilzabrutinib in Pemphigus: PEGASUS Phase 3 Randomized Study

Article

Author: Caux, Frédéric ; Hagino, Owen ; Rudnicka, Lidia ; Werth, Victoria P ; Gourlay, Steven G ; Arora, Puneet ; Joly, Pascal ; Patsatsi, Aikaterini ; Vassileva, Snejina ; Uzun, Soner ; Yen, Karl ; Ye, Jenny ; Murrell, Dedee F

TRIAL DESIGN:

Pemphigus is a rare but life-threatening autoimmune disease requiring long-term treatment that minimizes corticosteroid (CS) exposure while providing consistent disease control. The phase 2 pemphigus study of oral, reversible, covalent Bruton tyrosine kinase inhibitor rilzabrutinib demonstrated rapid and sustained efficacy with well-tolerated safety.

METHODS:

Adults (aged 18-80 years) were randomized 1:1 to 400 mg rilzabrutinib (n = 65) or placebo (n = 66) twice daily (with CS ≤ 0.5 mg/kg/d) for 37 weeks in the phase 3 PEGASUS study in moderate-to-severe pemphigus vulgaris/pemphigus foliaceus.

RESULTS:

The primary endpoint of complete remission from week 29 to week 37 with the amended endpoint CS dose ≤10 mg/d was not significant for 13 of 54 (24%) rilzabrutinib versus 10 of 55 (18%) placebo patients with PV (P = .45). Secondary endpoints showed numerical but nonsignificant improvements with rilzabrutinib (vs placebo) in reduced CS use, prolonged complete remission duration, and faster time to first complete remission.

CONCLUSIONS:

Overall, rilzabrutinib was well-tolerated, with similar adverse events reported in both groups. Using minimal CS dose ≤10 mg/d and excluding remote observations, the primary efficacy endpoint was not met. However, results from a prespecified sensitivity analysis using CS dose ≤5 mg/d, considering all observations, and including all patients support Bruton tyrosine kinase inhibition as a viable therapeutic approach for pemphigus.

News (Medical) associated with Rilzabrutinib

06 Nov 2024

·www.prnewswire.com

Severe Asthma Clinical Trial Pipeline Appears Robust With 40+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

The severe asthma market is fueled by the rising prevalence of the condition, particularly in urban areas, alongside the emergence of innovative therapies like biologics targeting IgE and cytokine modulators. A growing demand for personalized medicine enhances treatment efficacy and patient compliance. Government initiatives and increased awareness improve access to care, while advancements in inhaler technology boost medication adherence. Collaborative efforts in R&D are also vital for driving innovation in asthma treatments. LAS VEGAS, Nov. 6, 2024 /PRNewswire/ -- DelveInsight's ' Severe Asthma Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline severe asthma therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the severe asthma pipeline domain. Key Takeaways from the Severe Asthma Pipeline Report DelveInsight's severe asthma pipeline report depicts a robust space with 40+ active players working to develop 50+ pipeline therapies for severe asthma treatment. Key severe asthma companies such as Kinaset Therapeutics, Bio-Thera Solutions, CSPC ZhongQi Pharmaceutical Technology, AB Science, GlaxoSmithKline, Oneness Biotech, Biosion, Lanier Biotherapeutics, Kymera Therapeutics, Suzhou Connect Biopharmaceuticals, Upstream Bio, Teva Branded Pharmaceutical Industries, Sanofi, Jiangsu Hengrui Medicine, AstraZeneca, Advagene Biopharma, Genrix (Shanghai) Biopharmaceuticals, and others are evaluating new severe asthma drugs to improve the treatment landscape. Promising severe asthma pipeline therapies such as KN-002, BAT2606, CM326, Masitinib, GSK3511294, FB 704A, BSI-045B, LNR 125.38, KT 621, CBP-201, Verekitug, TEV'248, Rilzabrutinib, SHR-1905, Atuliflapon, AD17002, GR1802, and others are under different phases of severe asthma clinical trials. In September 2024, Kinaset Therapeutics announced two oral presentations and a late-breaking poster presentation at the 2024 European Respiratory Society (ERS) Congress. The presentations demonstrate the potential of Kinaset's lead clinical candidate, frevecitinib ( KN-002), to treat all patients with moderate to severe asthma and patients with COPD. In May 2024, GSK announced positive headline results from the phase III clinical trials SWIFT-1 and SWIFT-2, which assessed the efficacy and safety of depemokimab versus placebo in adults and adolescents with severe asthma with type 2 inflammation characterized by blood eosinophil count. In April 2024, Twenty-five abstracts across approved and investigational medicines will be presented at this year's American Thoracic Society (ATS) International Conference. Notable data presentations for Sanofi's immunology pipeline include the first presentation of phase IIb moderate to severe asthma data for rilzabrutinib. In October 2023, Upstream Bio announced positive interim results in its Phase Ib clinical study of UPB-101, a thymic stromal lymphopoietin receptor (TSLPR) inhibitor. The Phase Ib study is a randomized, double-blinded, placebo-controlled multiple ascending dose (MAD) study with subcutaneous (SC) administration conducted in asthma patients. In October 2023, Aiolos Bio with an asthma drug in clinical trials, announced that it raised $245 million in Series A funding. Atlas Venture, Bain Capital Life Sciences, Forbion Capital Partners and Sofinnova Investments led the round, with additional investment from RA Capital Management Request a sample and discover the recent advances in severe asthma treatment drugs @ Severe Asthma Pipeline Report The severe asthma pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage severe asthma drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the severe asthma clinical trial landscape. Severe Asthma Overview Severe asthma is a chronic respiratory condition characterized by persistent symptoms that are difficult to control, even with high-dose medications. Unlike milder forms of asthma, severe asthma can significantly impair the quality of life and may result in frequent exacerbations requiring hospitalization. The exact cause of severe asthma is not entirely clear, but it often arises from a combination of genetic and environmental factors. These may include respiratory infections, allergens (pollen, mold, dust mites), pollutants (tobacco smoke, air pollution), occupational irritants, and lifestyle factors such as obesity and stress. Severe asthma can also develop in people whose milder asthma is not well-controlled over time. Diagnosis of severe asthma begins with a comprehensive medical history, physical examination, and pulmonary function tests like spirometry to assess lung function. Doctors may also order additional tests, such as chest X-rays, blood tests, and allergy tests to identify triggers. A key factor in diagnosing severe asthma is the persistence of symptoms despite the optimal use of standard asthma treatments. Managing severe asthma typically requires a multifaceted approach. Inhaled corticosteroids and long-acting bronchodilators are the mainstays of treatment. For those who do not respond to these medications, biologic therapies (such as omalizumab, mepolizumab, or dupilumab) may be prescribed to target specific pathways involved in the inflammatory response. In addition, patients with severe asthma may benefit from lifestyle modifications, including weight management, avoiding triggers, and pulmonary rehabilitation. In extreme cases, systemic corticosteroids may be necessary, although long-term use can have serious side effects. Find out more about severe asthma treatment drugs @ Drugs for Severe Asthma Treatment A snapshot of the severe asthma Pipeline Drugs mentioned in the report: Learn more about the emerging severe asthma pipeline therapies @ Severe Asthma Clinical Trials Severe Asthma Therapeutics Assessment The severe asthma pipeline report proffers an integral view of the severe asthma emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Severe Asthma Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Interleukin 5 receptor antagonists, Thymic stromal lymphopoietin inhibitors, Thymic stromal lymphopoietin inhibitors, Thymic stromal lymphopoietin modulators, Interleukin 6 inhibitors, STAT6 transcription factor degraders, Interleukin 4 receptor alpha subunit antagonists Key Severe Asthma Companies: Kinaset Therapeutics, Bio-Thera Solutions, CSPC ZhongQi Pharmaceutical Technology, AB Science, GlaxoSmithKline, Oneness Biotech, Biosion, Lanier Biotherapeutics, Kymera Therapeutics, Suzhou Connect Biopharmaceuticals, Upstream Bio, Teva Branded Pharmaceutical Industries, Sanofi, Jiangsu Hengrui Medicine, AstraZeneca, Advagene Biopharma, Genrix (Shanghai) Biopharmaceuticals, and others. Key Severe Asthma Pipeline Therapies: KN-002, BAT2606, CM326, Masitinib, GSK3511294, FB 704A, BSI-045B, LNR 125.38, KT 621, CBP-201, Verekitug, TEV'248, Rilzabrutinib, SHR-1905, Atuliflapon, AD17002, GR1802, and others. Dive deep into rich insights for new drugs for severe asthma treatment, visit @ Severe Asthma Drugs Table of Contents For further information on the severe asthma pipeline therapeutics, reach out @ Severe Asthma Treatment Drugs Related Reports Severe Asthma Market Severe Asthma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key severe asthma companies including AstraZeneca plc, GlaxoSmithKline plc, Sanofi S.A., Novartis AG, Roche Holding AG, Teva Pharmaceutical Industries Ltd., Merck & Co., Inc., Boehringer Ingelheim International GmbH, Regeneron Pharmaceuticals, Inc., Johnson & Johnson, Pfizer Inc., Bristol Myers Squibb, Eli Lilly and Company, Vertex Pharmaceuticals Incorporated, Amgen Inc., AbbVie Inc., Genentech, Inc., Gilead Sciences, Inc., Biogen Inc., CSL Limited, among others. Asthma Market Asthma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key asthma companies including GlaxoSmithKline, 4D Pharma plc, AstraZeneca, Suzhou Connect Biopharmaceuticals, Avillion, Pearl Therapeutics, ARS Pharmaceuticals, Sinomab, Avalo Therapeutics, Kymab, Sanofi, Cumberland Pharmaceuticals, Genentech, Inc. , among others. Asthma Pipeline Asthma Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key asthma companies, including Jiangsu Hengrui Medicine, GlaxoSmithKline, Immunotek SL, AB Science, EMS, Avalo Therapeutics, Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd., ARS Pharmaceuticals, Sanofi, Zura Bio Ltd., AstraZeneca, EURRUS Biotech GmbH, T-Balance Therapeutics GmbH, Gossamer Bio, Aldeyra Therapeutics, Inc., Trio Medicines, Janssen Research & Development, Arrowhead Pharmaceuticals, Hoffman-La-Roche, GlaxoSmithKline, Biohaven Pharmaceutical, TAK-Circulator Co., Shanghai Hengrui Pharmaceutical, among others. Acute Respiratory Distress Syndrome Market Acute Respiratory Distress Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key ARDS companies, including MediciNova, Edesa Biotech, Light Chain Biosciences, Boehringer Ingelheim, Genentech, Windtree Therapeutics, Biomarck Pharmaceuticals, Athersys, Healios, Direct Biologics, Biohaven Pharmaceutical, Arch Biopartners, APEPTICO Forschung und Entwicklung GmbH, Staidson (Beijing) Biopharmaceuticals, Veru, Mesoblast Limited, Avalo Therapeutics, Pluristem Therapeutics, ILTOO Pharma, 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 ResultPhase 2Phase 3Phase 1

05 Nov 2024

·endpts.com

Sanofi reveals positive data for BTK inhibitor from $3.7B Principia takeover

Sanofi’s BTK inhibitor rilzabrutinib helped 23% of adults with a rare autoimmune blood disorder achieve target platelet counts in a Phase 3 trial, compared to zero patients in the placebo arm, the company said Tuesday. The data, unveiled ahead of the American Society of Hematology meeting in December, bolster rilzabrutinib’s case as Sanofi prepares a regulatory filing in the US for immune thrombocytopenia, or ITP. The drug was part of Sanofi’s $3.7 billion Principia Biopharma buyout in 2020, and the company now touts it as a potential blockbuster that could generate €2 billion ($2.2 billion) to €5 billion ($5.5 billion) in peak sales, according to a December R&D presentation . Of 133 adults randomized to receive rilzabrutinib in the LUNA 3 trial, 31 achieved the primary endpoint of durable platelet response, meeting platelet counts at or above 50,000/μL for at least eight of the last 12 weeks of the 24-week blinded treatment period, with no rescue therapy. Zero of 69 placebo patients achieved the primary endpoint. ITP is a rare autoimmune disorder that leads to low platelet counts and an increased risk of bleeding and bruising. Patients in the trial had failed on or were intolerant to prior therapies. Sanofi said 46% of participating adults have received five or more treatments, and some had been dealing with ITP for years. While the trial enrolled both adults and adolescents, Sanofi said this is the first report of efficacy and safety in the adult population. The company also reported “significant improvements” in key secondary endpoints, including a reduction in the need for rescue therapy by 53% compared to placebo, and improvements in physical fatigue. While Sanofi said a similar percentage of patients in the rilzabrutinib and placebo arms experienced adverse events, it noted that one patient “with multiple risk-factors” had a treatment-related Grade 3 peripheral embolism, also known as a blockage in an artery. Sanofi said on its second-quarter earnings call in July that it wants to file a regulatory submission this year, according to an AlphaSense transcript. Rilzabrutinib’s Phase 3 win is welcome news following the drug’s Phase 2 fail in atopic dermatitis last year and a disappointing Phase 3 readout in pemphigus in 2021. But Sanofi continues to pursue a handful of other mid-stage programs, including asthma, chronic spontaneous urticaria, IgG4-related disease and warm autoimmune hemolytic anemia, according to the company’s pipeline . Stay tuned for more coverage of ASH from Endpoints , and sign up for our virtual ASH event here .

Clinical ResultPhase 3ASHPhase 2Acquisition

05 Nov 2024

·firstwordpharma.com

ASH24: Sanofi reveals ITP response data supporting bid for US, EU approval of rilzabrutinib

Sanofi shared detailed results on Tuesday from a pivotal trial of rilzabrutinib to treat immune thrombocytopenia (ITP), after first revealing in April that the BTK inhibitor met the primary endpoint of the Phase III LUNA 3 study. At the time, the French pharma had announced it was targeting regulatory submissions in the US and Europe by year-end.A win for rilzabrutinib could put two feathers in Sanofi's cap. First, it would justify the company's $3.7-billion purchase of Principia Biopharma to gain the asset in 2020, and second, it would bring a key growth driver to market from its immunology and inflammation (I&I) pipeline, an area the drugmaker is increasingly relying on for revenue as it slims down its oncology portfolio. For related analysis, see Vital Signs: Sanofi builds momentum for its I&I bets.Sanofi has cause to be cautious, however, as the BTK inhibitor has previously fallen short in clinical studies for pemphigus and atopic dermatitis.Boosting platelet countThe LUNA 3 data were unveiled in an abstract ahead of the American Society of Hematology (ASH) meeting in December. The study randomised 202 adults with persistent or chronic ITP to receive rilzabrutinib twice daily or placebo. As promised, the BTK inhibitor met the trial's primary endpoint of durable platelet response, with 23% patients who received the therapy achieving platelet counts at or above 50,000/μL for at least eight out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, compared with zero patients in the placebo group. When Sanofi combined data from the double-blind and open-label periods, the durable response rate jumped to 29%. Additionally, rilzabrutinib significantly reduced the need for rescue therapy by 52% versus placebo, and led to significant and clinically meaningful improvements in physical fatigue and bleeding.The experimental therapy also appeared safe and tolerable, as patients in the investigational arm had a similar percentage of adverse events (AEs) as those who received placebo. The most common Grade 1/2 treatment-related AEs for rilzabrutinib were diarrhoea (23%), nausea (17%), headache (8%) and abdominal pain (6%).Sanofi also disclosed that one patient with multiple risk factors who received the BTK inhibitor experienced a treatment-related Grade 3 peripheral embolism. Another participant in the study died due to pneumonia unrelated to treatment.

Clinical ResultPhase 3AcquisitionASHASCO

100 Deals associated with Rilzabrutinib

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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	Phase 3	US	Sanofi (China) Investment Co. Ltd.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	US	Principia Biopharma, Inc.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	CN	Principia Biopharma, Inc.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	JP	Sanofi (China) Investment Co. Ltd.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	JP	Principia Biopharma, Inc.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	AR	Sanofi (China) Investment Co. Ltd.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	AR	Principia Biopharma, Inc.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	AU	Principia Biopharma, Inc.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	AU	Sanofi (China) Investment Co. Ltd.	14 Dec 2020
Purpura, Thrombocytopenic, Idiopathic	Phase 3	AT	Sanofi (China) Investment Co. Ltd.	14 Dec 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Warm autoimmune hemolytic anemia	-	Rilzabrutinib 400 mg bid	qcvbazazzx(ldserolsjs) = 23% grlyujjmjb (jpsfzgzvdz ) View more	-	09 Dec 2024
				Concomitant CS
NCT04562766 (LUNA 3, ASH2024) Manual	Phase 3	Purpura, Thrombocytopenic, Idiopathic	202	rilzabrutinib	anivemfbkb(szkndbeuwl) = hvirtfubqj evromcgfrd (luokyconoc ) Met View more	Positive	08 Dec 2024
				placebo	anivemfbkb(szkndbeuwl) = nvjxidjyyf evromcgfrd (luokyconoc ) Met View more
ASH2024	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	-	Rilzabrutinib 400 mg bid	neohblzqmd(vjonwlwnrf) = zcgwfnztgv jsvotdwwtl (klgqufivod ) View more	-	08 Dec 2024
				Placebo	uqulkxrkir(bxxeolektm) = ihddjiijpu jkyoztrbav (irbltgwons, 2.9) View more
NCT05104892 (ATS2024) Manual	Phase 2	Asthma	64	Rilzabrutinib 400 mg BID	vpelasfkxg(pgzrebiagt) = yczhgtuoas wkakkfwbml (reuhxrnycy ) View more	Positive	22 May 2024
NCT03395210 (LUNA 2, EHA2024) Manual	Phase 2	Purpura, Thrombocytopenic, Idiopathic Second line	71	Rilzabrutinib 400 mg bid + thrombopoietin-receptor agonist	luhzgrytss(usykhcsgup) = nmmvabsnvj lyseufmddl (vbxhzbbcly ) View more	Positive	14 May 2024
				Rilzabrutinib 400 mg bid + thrombopoietin-receptor agonist (responders)	zkeoppohdu(jfbwwwahnb) = pvqfmxntoo wqejgnlkxb (iylttigpvh ) View more
NCT03395210 (LUNA 2, EHA2024)	Phase 2	Purpura, Thrombocytopenic, Idiopathic	71	Rilzabrutinib 400 mg bid alone	djrmkupxcq(mbrpoarluc) = qiguoewsan kaiykqslrl (eisvoibxkx, 6 - 20)	Positive	14 May 2024
				Rilzabrutinib 400 mg bid with concomitant ITP therapy	djrmkupxcq(mbrpoarluc) = zyngqbcirt kaiykqslrl (eisvoibxkx, 6 - 20)
NCT04562766 (LUNA 3, EHA2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	202	Rilzabrutinib 400 mg bid	yyqdrlhchf(aqkfehsuap) = fsufuikknw gnoppojtiv (rvoxtjxpmt ) View more	Positive	14 May 2024
EHA2024	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	-	Rilzabrutinib 400 mg bid	fsumuyjzxu(flvlteheop) = 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. sbnrqxtjyo (plmshbmxko )	-	14 May 2024
				Concomitant ITP therapy
NCT03395210 (Pubmed) Manual	Phase 1/2	Purpura, Thrombocytopenic, Idiopathic	16	Rilzabrutinib 400 mg	vvwokvnnlo(lywwpzxzdc) = Adverse events related to treatment were grade 1 or 2 and transient, with no bleeding, thrombotic, or serious adverse events. xjpmszbmjd (tfiqqganvh )	Positive	09 Apr 2024
NCT05107115 (RILECSU, AAAAI2024) Manual	Phase 2	Chronic Urticaria	160	rilzabrutinib 400mg once-every-evening	fvsitfrnsg(wpbgjjmtbn) = Rilzabrutinib was well tolerated; adverse events occurring at a higher frequency with rilzabrutinib vs placebo included headache, nausea, and diarrhea. hoocqeoabt (yfsmmixprd )	Positive	24 Feb 2024
				rilzabrutinib 400mg twice-a-day

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