A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 3 Study of Masitinib as add-on Therapy in Patients With Mild to Moderate Alzheimer's Disease

Masitinib is an orally administered tyrosine kinase inhibitor that targets activated cells of the neuroimmune system (mast cells and microglia). Study AB21004 will evaluate masitinib as an adjunct to cholinesterase inhibitor and/or memantine in patients with mild-to-moderate Alzheimer's disease.

开始日期2024-01-01

申办/合作机构

AB Science SA

NCT05449444 / Recruiting临床2期

A 24-week, Multicenter, Randomized, Double Blind, Placebo-controlled, Dose-range Finding Phase II Study to Compare Efficacy and Safety of Oral Masitinib to Placebo in Treatment of Patients With Severe Mast Cell Activation Syndrome (MCAS) With Handicap Unresponsive to Optimal Symptomatic Treatment

To evaluate the efficacy and safety of two dosing schemes of oral masitinib versus matching placebo in the treatment of patients suffering from severe MCAS with handicap unresponsive to optimal symptomatic treatment.

开始日期2022-07-01

申办/合作机构

AB Science SA

NCT05441488 / Recruiting临床3期

A 96-Week, Prospective, Multicenter, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Study to Compare Efficacy and Safety of Masitinib Dose Titration to 4.5 mg/kg/Day Versus Placebo in the Treatment of Patients With Primary Progressive or Secondary Progressive Multiple Sclerosis Without Relapse

To evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients with primary progressive or secondary progressive multiple sclerosis without relapse.

开始日期2022-06-28

申办/合作机构

AB Science SA

100 项与甲磺酸马赛替尼相关的临床结果

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100 项与甲磺酸马赛替尼相关的转化医学

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100 项与甲磺酸马赛替尼相关的专利（医药）

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184

项与甲磺酸马赛替尼相关的文献（医药）

2024-12-01·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

In vitro assessment of inhibitory effects of kinase inhibitors on CYP2C9, 3A and 1A2: Prediction of drug-drug interaction risk with warfarin and direct oral anticoagulants

Article

作者: Cai, Weimin ; Kurkela, Mika ; Backman, Janne T ; Jin, Shasha ; Backman, Janne T. ; Neuvonen, Mikko ; Xiang, Xiaoqiang ; Kahma, Helinä ; Paludetto, Marie-Noëlle

OBJECTIVE:

This study aimed to evaluate the cytochrome P450 (CYP)-mediated drug-drug interaction (DDI) potential of kinase inhibitors with warfarin and direct oral anticoagulants (DOACs).

METHODS:

An in vitro CYP probe substrate cocktail assay was used to study the inhibitory effects of fifteen kinase inhibitors on CYP2C9, 3A, and 1A2. Then, DDI predictions were performed using both mechanistic static and physiologically-based pharmacokinetic (PBPK) models.

RESULTS:

Linsitinib, masitinib, regorafenib, tozasertib, trametinib, and vatalanib were identified as competitive CYP2C9 inhibitors (K_i = 1.4, 1.0, 1.1, 3.8, 0.5, and 0.1 μM, respectively). Masitinib and vatalanib were competitive CYP3A inhibitors (K_i = 1.3 and 0.2 μM), and vatalanib noncompetitively inhibited CYP1A2 (K_i = 2.0 μM). Moreover, linsitinib and tozasertib were CYP3A time-dependent inhibitors (K_I = 26.5 and 400.3 μM, k_inact = 0.060 and 0.026 min^-1, respectively). Only linsitinib showed time-dependent inhibition of CYP1A2 (K_I = 13.9 μM, k_inact = 0.018 min^-1). Mechanistic static models identified possible DDI risks for linsitinib and vatalanib with (S)-/(R)-warfarin, and for masitinib with (S)-warfarin. PBPK simulations further confirmed that vatalanib may increase (S)- and (R)-warfarin exposure by 4.37- and 1.80-fold, respectively, and that linsitinib may increase (R)-warfarin exposure by 3.10-fold. Mechanistic static models predicted a smaller risk of DDIs between kinase inhibitors and apixaban or rivaroxaban. The greatest AUC increases (1.50-1.74) were predicted for erlotinib in combination with apixaban and rivaroxaban. Linsitinib, masitinib, and vatalanib were predicted to have a smaller effect on apixaban and rivaroxaban AUCs (AUCR 1.22-1.53). No kinase inhibitor was predicted to increase edoxaban exposure.

CONCLUSIONS:

Our results suggest that several kinase inhibitors, including vatalanib and linsitinib, can cause CYP-mediated drug-drug interactions with warfarin and, to a lesser extent, with apixaban and rivaroxaban. The work provides mechanistic insights into the risk of DDIs between kinase inhibitors and anticoagulants, which can be used to avoid preventable DDIs in the clinic.

2024-10-01·Pharmacology Research & Perspectives

Cromolyn sodium and masitinib combination inhibits fibroblast‐myofibroblast transition and exerts additive cell‐protective and antioxidant effects on a bleomycin‐induced in vitro fibrosis model

Article

作者: Göksu, Azize Yasemin ; Kocanci, Fatma Gonca ; Dirol, Hulya

Abstract:

Idiopathic pulmonary fibrosis (IPF) is a progressive and fatal fibrotic lung disease. While recent studies have suggested the potential efficacy of tyrosine kinase inhibitors in managing IPF, masitinib, a clinically used tyrosine kinase inhibitor, has not yet been investigated for its efficacy in fibrotic lung diseases. In a previous study on an in vitro neurodegenerative model, we demonstrated the synergistic antitoxic and antioxidant effects of masitinib combined with cromolyn sodium, an FDA‐approved mast cell stabilizer. This study aims to investigate the anti‐fibrotic and antioxidant effects of the masitinib‐cromolyn sodium combination in an in vitro model of pulmonary fibrosis. Fibroblast cell cultures treated with bleomycin and/or hydrogen peroxide (H2O2) were subjected to masitinib and/or cromolyn sodium, followed by assessments of cell viability, morphological and apoptotic nuclear changes, triple‐immunofluorescence labeling, and total oxidant/antioxidant capacities, besides ratio of Bax and Bcl‐2 mRNA expressions as an indication of apoptosis. The combined treatment of masitinib and cromolyn sodium effectively prevented the fibroblast myofibroblast transition, a hallmark of fibrosis, and significantly reduced bleomycin / H2O2‐induced apoptosis and oxidative stress. This study is the first to demonstrate the additive anti‐fibrotic, cell‐protective, and antioxidant effects of the masitinib‐cromolyn sodium combination in an in vitro fibrosis model, suggesting its potential as an innovative therapeutic approach for pulmonary fibrosis. Combination therapy may be more advantageous in that both drugs could be administered in lower doses, exerting less side effects, and at the same time providing diverse mechanisms of action simultaneously.

2024-07-01·Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology

Safety of masitinib in patients with neurodegenerative diseases: a meta-analysis of randomized controlled trials

Article

作者: Hamad, Abdullah Ashraf ; Amer, Basma Ehab

Abstract:

Objectives:

This meta-analysis aimed to examine the safety of masitinib in patients with neurodegenerative diseases.

Methods:

We considered randomized controlled trials (RCTs) comparing different doses of masitinib versus placebo. We performed our analysis using the R (v.4.3.0) programming language and the incidence of adverse events was pooled using risk ratio (RR) and 95% confidence interval (CI).

Results:

We included five RCTs, focusing on multiple sclerosis (MS), Alzheimer's disease (AD), and amyotrophic lateral sclerosis. The meta-analysis revealed a significantly higher incidence of adverse events in the masitinib group compared to the control group, regardless of adverse event grade and masitinib dose (RR = 1.12, 95% CI [1.07 to 1.17], P < 0.01). Adverse events categorized as severe, non-fatal serious, leading to dose reduction, and leading to permanent discontinuation also showed a higher incidence in the masitinib group (P ≤ 0.01). Subgroup analysis for AD and MS supported these findings. The pooled incidence of adverse events, regardless of their grade, was higher in the masitinib group for both the 3 mg/kg/d dose (RR = 1.13, P = 0.01) and the 4.5 mg/kg/d dose (RR = 1.11, P < 0.01). However, there was no significant difference between masitinib 3 mg/kg/d dose and placebo regarding severe and non-fatal serious adverse events for the.

Conclusion:

Masitinib use in neurodegenerative diseases presents safety concerns that may impact patients' quality of life and require management. Further research is recommended to determine the optimal dose with minimal safety concerns in this patient population.

110

项与甲磺酸马赛替尼相关的新闻（医药）

2024-11-06

·PRNewswire

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

临床结果临床2期临床3期临床1期

2024-10-28

·GlobeNewswire-Health Care

AB Science receives notice of allowance for European patent covering masitinib until 2040 in the treatment of sickle cell disease

PRESS RELEASE AB SCIENCE RECEIVES NOTICE OF ALLOWANCE FOR EUROPEAN PATENT COVERING MASITINIB UNTIL 2040 IN THE TREATMENT OF SICKLE CELL DISEASE THIS DECISION FURTHER STRENGTHENS MASITINIB’S INTELLECTUAL PROPERTY ADDING ONE MORE INDICATION WITH LONG TERM PROTECTION MASITINIB IS IN PHASE 2 IN SICKLE CELL DISEASE Paris, October 28, 2024, 5.45pm CET AB Science SA (Euronext - FR0010557264 - AB) today announced that the European Patent Office has issued a Notice of Allowance for a patent relating to methods of treating sickle cell disease (i.e. a medical use patent) with its lead compound masitinib, based on preclinical data. This new European patent provides intellectual property protection for masitinib in this indication until November 2040. Professor Olivier Hermine, President of the AB Science Scientific Committee and member of the French Academy of Sciences and Head of Hematology Department at Necker Hospital commented: “Masitinib represents a promising novel strategy for treating sickle cell disease and its serious complication of acute chest syndrome, which can lead to the development of chronic lung disease and is a common cause of hospitalization or even death. There is an increasing need in sickle cell disease with several new drugs being retrieved from market for lack of efficacy or toxicity; however, unlike masitinib, none of these drugs target mast cells”. Sickle cell disease (SCD) is a group of inherited red blood cell disorders, with masitinib being developed to treat the severest forms of the disease, accounting for about 65% of SCD. Severe SCD represents a major public health challenge and leads to early death. Treatment for SCD can be curative based on gene therapy (targets the HbS mutation), however, such an option remains extremely limited due to scarcity of donors, unresolved safety challenges, and very high costs. Standard treatment for SCD includes red blood cell transfusions and treatment with hydroxyurea to manage complications, but significant unmet need remains. Mast cells, a major target for masitinib, appear to play a critical role for the severe forms of SCD and its complications, such as vaso-occlusive crises (VOC), acute chest syndrome (ACS), and pain [1-2]. Masitinib has demonstrated survival benefit in an SCD mouse model: all control SCD mice experienced VOC and 83% died in the first 3 hours, whereas SCD mice pretreated with masitinib for 4 days, experienced no VOCs and no death. Furthermore, lung histology and immune immunohistochemistry showed that masitinib protects from acute lung injuries and mast cell infiltration in an SCD mouse model. Masitinib clinical development in SCD is being conducted as part of the SICKMAST collaborative program, funded with 9.2 million euros [3], which aims to demonstrate in a phase 2 clinical trial the efficacy of masitinib in the treatment of acute and chronic complications of SCD in patients identified based on biomarkers. The Assistance Publique-Hôpitaux de Paris (AP-HP) will be the promoter of these phase 2 studies. AB Science will mainly be involved in supplying masitinib and monitoring masitinib pharmacovigilance data. AB Science remains free to carry out, as it sees fit, any potential phase 3 development following the success of phase 2. Sickle cell disease Sickle cell disease (SCD) is an autosomal recessive disorder affecting millions of people worldwide. Although life expectancy has increased over the last 20 years, acute and chronic complications still result in comorbidities, high social burden and premature death at around 40 years. Approximately 1.1% of couples worldwide are at risk of having a child with a hemoglobin disorder (sickle cell disease or thalassemia), and 2.3 conceptions per 1,000 are affected by sickle cell disease. Estimates suggest that each year, around 300,000 children are born with sickle cell disease, and this number could reach 400,000 by 2050 [4]. Sickle cell disease affects over 100,000 children and adults in the United States. In France, approximately 26,000 patients are affected (50% children, 50% adults). Rationale for the use of masitinib in this indication Inflammation mediated by innate immune cells and promoting vaso-occlusion has recently been shown to play a major role in sickle cell disease. In particular, our clinical observations and experimental work in mice, have revealed the involvement of mast cells and basophils in complications associated with sickle cell disease: The degree of mast cell activation in patients with sickle cell disease may contribute to the heterogeneity of inflammation and chronic and acute complications.The potential role of basophils in sickle cell disease has not been studied, however, given their role in various diseases and their ability to release substance P and histamine, they could also play important roles in the pathophysiology of sickle cell disease. Masitinib is an inhibitor of KIT, LYN, and FYN, three major kinases involved in the activation of mast cells and basophils. Medical need The classic view of sickle cell disease pathophysiology involves polymerization of mutated hemoglobin (HbS) leading to red blood cell (RBC) sickling with subsequent hemolytic anemia, painful vaso-occlusive crisis (VOC) and acute chest syndrome (ACS). Current treatment options such as hydroxycarbamide, chronic transfusion or anti-P-selectin antibodies, do not fully prevent life-threatening acute and chronic complications of sickle cell disease. Allogeneic stem cell transplantation and gene therapy are available only for a minority of patients, are associated with toxicity and are very expensive, which limits their use. There is a significant medical need to prevent the acute and chronic complications of sickle cell disease. References [1] Allali S, Lionnet F, Mattioni S, et al. Br J Haematol. 2019;186(1):125-129. [2] Allali S, Maciel TT, Hermine O, de Montalembert M. Haematologica. 2020;105(2):273-283. [3] AB Science Press Release, 27th November 2023. https://www.ab-science.com/the-clinical-development-of-masitinib-in-sickle-cell-disease-is-among-the-19-winning-projects-under-the-sixth-call-for-hospital-inuversity-research-in-health-rhu/ [4] Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017;376(16):1561-1573. About AB Science Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science’s website: www.ab-science.com. Forward-looking Statements - AB Science This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations. For additional information, please contact: AB Science Financial Communication & Media Relations investors@ab-science.com Attachment Sickle Cell Patent EPO vENG VF

临床2期临床结果基因疗法免疫疗法

2024-10-17

·BioSpace

AB Science provides an update on the application for conditional marketing authorisation of masitinib in ALS

PRESS RELEASE AB SCIENCE PROVIDES AN UPDATE ON THE APPLICATION FOR CONDITIONAL MARKETING AUTHORISATION OF MASITINIB IN THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS Paris, 17 October, 2024, 7.30pm CET AB Science SA (Euronext - FR0010557264 - AB) today announces that the European Medicines Agency (EMA) confirmed a negative opinion for the conditional marketing authorization of masitinib in the treatment of amyotrophic lateral sclerosis (ALS), following a vote adopted during the Committee for Medicinal Products for Human Use (CHMP) meeting on 14-17 October 2024. The Conditional Marketing Authorization of masitinib had been under review by the CHMP in response to the company’s request for a re-examination of the negative opinion issued in June 2024. Despite the difficulty of a conditional marketing authorization in ALS, AB Science had asked for a re-examination first and foremost considering the urgent need for patients to have early access to a promising treatment and the clinical data obtained from the first study AB10015, showing an increase in median overall survival of + 6 months (p= 0.0761) in the primary analysis population of Normal progressors, and a significant + 12 months survival benefit (p=0.0192) in a subgroup defined as patients prior to any complete loss of function (i.e. excluding patient with an ALSFRS-R score of 0 in any of the 12 items of the scale). A Scientific Advisory Group – Neurology (SAG-N) was conveyed for the first time, as part of the re-examination procedure. Importantly, the SAG-N experts agreed with the categorization approach to distinguish between Normal and Fast Progressors (and with the 1.1 points per month ALSFRS decline from onset as cut-off), if properly pre-specified. This opinion supports the design of the confirmatory study of masitinib in ALS, as the same dichotomization and the same cut-off have been pre-specified for the confirmatory study. The phase 2B/3 study AB10015 is considered hypothesis generating but not sufficient as a single pivotal study to support a marketing authorization. Separately, Health Canada recently informed AB Science that key analyses presented for the reconsideration submitted in May 2024 [1], have been considered as new data, rather than re-analyses of existing data. Considering that Health Canada guideline prevent the use of new data as part of the re-examination procedure, AB Science has decided to notify Health Canada it will not pursue the reconsideration. Health Canada has offered the possibility to submit a new application to resolve this issue. Alain Moussy, CEO and co-founder of AB Science said “ We are thankful to the patients and physicians that supported the re-examination process as part of the EMA review. We did all these efforts for the patients. We are convinced that masitinib is a promising drug when we see patients from the study surviving with the drugs for more than 10 years in our compassionate use program. Now AB Science objective is to focus on the confirmatory phase 3 program of masitinib in ALS to reach full approval ”. References [1] AB Science press release dated 3 April 2024 About AB Science Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science’s website: . Forward-looking Statements - AB Science This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words “expect”, “anticipate”, “believe”, “intend”, “estimate” or “plan” as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations. For additional information, please contact: AB Science Financial Communication & Media Relations investors@ab-science.com Attachment CP ALS REGUL EMA VENG VF

临床3期上市批准

100 项与甲磺酸马赛替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	甲磺酸马赛替尼	L01XE22	DB11526

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肌萎缩侧索硬化	申请上市	加拿大	AB Science SA	2022-05-26
肥大细胞增多症	申请上市	欧盟	AB Science SA	2017-09-14
胃肠道间质瘤	申请上市	欧盟	AB Science SA	-
多发性硬化症	临床3期	美国	AB Science SA	2022-12-29
哮喘	临床3期	阿根廷	AB Science SA	2016-12-01
哮喘	临床3期	马来西亚	AB Science SA	2016-12-01
哮喘	临床3期	秘鲁	AB Science SA	2016-12-01
哮喘	临床3期	菲律宾	AB Science SA	2016-12-01
哮喘	临床3期	乌克兰	AB Science SA	2016-12-01
黑色素瘤	临床3期	中国	AB Science SA	2016-08-11

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05441488 (MAXIMS, GlobeNewswire) 人工标引	临床3期	原发性进行性多发性硬化 \| 继发进展型多发性硬化 Expanded Disability Status Scale (EDSS)	-	Masitinib	糧遞醖願積築襯積構觸(夢築鬱築築網範醖鑰醖) = 醖鹹糧鹽簾鬱鬱壓衊積遞鹹網簾艱艱衊蓋鹹夢 (鏇範築膚餘網繭積簾膚 ) 更多	积极	2024-09-23
				Placebo	-
NCT04622865 (Biospace) 人工标引	临床2期	新型冠状病毒感染	95	masitinib + Isoquercetin + Best Supportive Care	顧壓築憲鹽窪觸鑰醖鹽(簾襯觸製鏇顧繭膚衊蓋) = odds ratio of 2.4 in favor of the treatment arm after 15 days of treatment, superior to the odds ratio of 2.2 initially hypothesized, with p=0.038 simulated with 200 patients and p=0.072 detected with 95 patients recruited. 選衊餘網築範窪艱蓋獵 (蓋築窪築範鹽糧壓網築 ) 更多	积极	2024-07-08
				Best Supportive Care
NCT02588677 (AB10015, AAN2023)	临床2/3期	肌萎缩侧索硬化追加	-	Masitinib 4.5 mg/kg/d	鏇願顧鑰艱範鹹獵範膚(顧願鑰窪選淵餘壓構顧) = 艱窪襯遞顧憲選夢願蓋選鹹積齋艱壓醖網餘壓 (觸製膚鹽築構遞構淵鹹 )	-	2023-04-25
				Riluzole alone	鏇願顧鑰艱範鹹獵範膚(顧願鑰窪選淵餘壓構顧) = 願簾蓋築襯蓋願鹽鹹選選鹹積齋艱壓醖網餘壓 (觸製膚鹽築構遞構淵鹹 )
NCT01433497 (Pubmed \| Neurol Neuroimmunol Neuroinflamm) 人工标引	临床3期	多发性硬化症	611	masitinib	獵顧範獵構膚繭選淵鑰(獵壓構遞遞鏇鑰鏇鏇願) = 範鑰構襯顧顧壓膚製顧壓選糧壓範壓憲網鹽衊 (艱鏇鬱繭觸蓋膚積夢淵 )	积极	2022-02-21
				Placebo	獵顧範獵構膚繭選淵鑰(獵壓構遞遞鏇鑰鏇鏇願) = 廠齋齋衊獵膚鏇鏇餘顧壓選糧壓範壓憲網鹽衊 (艱鏇鬱繭觸蓋膚積夢淵 )
AB12003 (AUA2021)	临床3期	转移性去势抵抗性前列腺癌一线	714	Masitinib 6.0 mg/kg/d plus Docetaxel	窪憲蓋餘構鏇餘簾齋鬱(膚構製積夢壓遞壓夢鹹) = 獵衊糧膚簾顧淵獵艱夢醖糧膚齋顧觸醖憲醖積 (簾選選構艱鹹遞鑰淵遞 ) 更多	积极	2021-09-01
				Placebo plus Docetaxel	窪憲蓋餘構鏇餘簾齋鬱(膚構製積夢壓遞壓夢鹹) = 獵鏇壓鏇糧範製鹽艱壓醖糧膚齋顧觸醖憲醖積 (簾選選構艱鹹遞鑰淵遞 ) 更多
NCT01872598 (AB09004, GlobeNewswire) 人工标引	临床3期	阿尔茨海默症	358	Masitinib mesylate	鑰憲積鹽廠衊淵淵構夢(鹽夢壓醖蓋蓋鏇窪餘網) = 鬱憲願製鏇夢築醖鹽製製製獵範鬱鏇鏇網淵齋 (淵範廠衊鬱餘顧鏇簾選 ) 更多	积极	2021-07-29
				Placebo	鑰憲積鹽廠衊淵淵構夢(鹽夢壓醖蓋蓋鏇窪餘網) = 廠範窪鏇積鹽壓齋網構製製獵範鬱鏇鏇網淵齋 (淵範廠衊鬱餘顧鏇簾選 ) 更多
NCT03766295 (AB12005, ASCO2021)	临床3期	胰腺癌一线	384	Masitinib plus gemcitabine	簾淵築醖構製遞獵築憲(淵憲繭觸夢壓齋獵鹹襯) = 襯壓鏇築網鏇顧觸範獵壓遞顧膚窪襯蓋鑰顧壓 (壓願簾積鬱醖構選憲糧 )	积极	2021-05-28
				Placebo plus gemcitabine	簾淵築醖構製遞獵築憲(淵憲繭觸夢壓齋獵鹹襯) = 廠衊憲簾夢遞積夢壓獵壓遞顧膚窪襯蓋鑰顧壓 (壓願簾積鬱醖構選憲糧 )
NCT03761225 (AB12003, Corporate Publications) 人工标引	临床3期	去势抵抗性前列腺癌一线	714	Docetaxel+Masitinib	鬱簾鑰製觸窪範鹽蓋築(築壓鬱鹽鏇艱膚膚鑰艱) = 憲願壓衊淵築淵鏇鑰構夢築構網餘獵築製遞憲 (糧膚衊憲窪顧糧壓窪衊, 4.9 ~ 6.3) 更多	不佳	2021-05-25
				Docetaxel+Placebo	鬱簾鑰製觸窪範鹽蓋築(築壓鬱鹽鏇艱膚膚鑰艱) = 艱鏇廠鬱範夢醖壓鹽製夢築構網餘獵築製遞憲 (糧膚衊憲窪顧糧壓窪衊, 4.9 ~ 5.9) 更多
NCT01433497 (AB07002, ECTRIMS2020)	临床3期	慢性进行性多发性硬化	-	Masitinib 4.5 mg/kg/d	淵網觸艱淵糧鏇範鑰構(鑰夢憲衊獵壓壓鑰夢築): ΔLSM = -0.097 (95% CI, -0.192 ~ -0.002), P-Value = 0.0256 更多	积极	2020-12-07
				Placebo
NCT03771040 (AB14001, GlobeNewswire) 人工标引	临床3期	重度哮喘	-	masitinib	艱鹹廠壓艱壓齋繭繭淵(壓範遞繭遞夢築積遞鹽) = 襯構醖窪構網鬱膚顧積網鬱鹽膚顧齋鹽廠淵鏇 (鹽衊夢壓遞憲膚範獵選 ) 更多	积极	2020-10-20
				Placebo	艱鹹廠壓艱壓齋繭繭淵(壓範遞繭遞夢築積遞鹽) = 蓋艱鹹觸網鹽淵觸選蓋網鬱鹽膚顧齋鹽廠淵鏇 (鹽衊夢壓遞憲膚範獵選 ) 更多