A Prospective, Multicenter, Randomised, Double-blind, Placebo-controlled, Parallel Groups, Phase 3 Trial to Compare the Efficacy and Safety of Masitinib in Combination With Standard of Care Versus Placebo in Combination With Standard of Care in the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)

The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).

开始日期2026-01-01

申办/合作机构

AB Science SA

NCT05564169

/ Not yet recruiting临床3期

A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 3 Clinical Trial to Evaluate the Safety and Efficacy of Masitinib as add-on Therapy in Patients With Mild Alzheimer's Disease, Treated With Standard of Care

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2026-06-01

申办/合作机构

AB Science SA

NCT05449444

/ Unknown status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

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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334

项与甲磺酸马赛替尼相关的文献（医药）

2025-11-01·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Structure-activity relationship studies of thiazole-based derivatives leading to the identification of novel and potent SARS-CoV-2 main protease inhibitors

Article

作者: Kong, Wai-Po ; Wong, Kwok-Yin ; Leung, Siu-Lun ; Yin, Weile ; Hung, Cheung-Hin

The COVID-19 pandemic has highlighted the need for effective antiviral agents targeting SARS-CoV-2. This study presents the development of thiazole-based inhibitors against SARS-CoV-2 Main Protease, a key enzyme for viral replication. Using Masitinib and MAC-5576 as leads, we designed 29 compounds featuring a pyridinyl ester for covalent binding to Cys145 and a thiazole core for S2 subsite interaction. Structure-activity relationship (SAR) analysis identified the pyridinyl ester as a critical pharmacophore, with the thiazole core providing superior inhibition compared to oxazole. Compound MC12 (IC₅₀ = 77.7 ± 14.1 nM) demonstrated inhibitory activities comparable to Nirmatrelvir (IC₅₀ = 58.4 ± 8.6 nM). Mass spectrometry and X-ray crystallography confirmed reversible covalent binding of MC compounds to SARS-CoV-2 Main Protease. These compounds also showed low cytotoxicity and dual inhibition of SARS-CoV and SARS-CoV-2 M^pro. Thiazole-based compounds thus emerge as promising leads for developing potent and safe SARS-CoV-2 M^pro inhibitors.

2025-10-01·JOURNAL OF PHARMACEUTICAL SCIENCES

Understanding the degradation behavior of masitinib by Stress testing, UHPLC, HRMS, NMR, APCI-MS, DFT and prediction of toxicity using in silico tools

Article

作者: Dhiman, Vivek ; Khemchandani, Rahul ; Samanthula, Gananadhamu ; Velip, Laximan ; Mishra, Deepak

The rising global incidence of cancer has led to widespread use of anti-cancer agents worldwide, raising concerns about their stability, degradation and potential toxicological risks. This study aimed to understand the degradation behavior of masitinib and the toxicological profiles of masitinib and its degradation products (DPs) under both stress and the International Council for Harmonization (ICH) stability conditions. Masitinib was subjected to hydrolytic, oxidative and photolytic conditions in accordance with the ICH guidelines (ICH Q1A (R2) and ICH Q1B). The Ultra High Performance liquid chromatography (UHPLC) with photo diode array detection resulted in the idetification of eight DPs. Photolytic exposure led discoloration of solid masitinib, warranting HRMS analysis that revealed 12 DPs, eight of which were distinct from those observed under other stress conditions. Regioisomeric DPs (DP7 and DP8) were identified, with their N-oxide formation was substantiated by a thermally induced Meisenheimer rearrangement in the atmospheric pressure chemical ionization (APCI) positive mode of HRMS. The proposed N-oxide positions were confirmed using nuclear magnetic resonance spectroscopy (1D and 2D NMR) on DP8. Additionally, the chemical reactivity and stability of masitinib and its N-oxidized DPs were predicted using Density Functional Theory (DFT). A plausible mechanistic pathway for the degradation of masitinib under different stress conditions was established. In silico toxicity predictions identified the three DPs exhibit teratogenic and mutagenic potential. DP6, a mutagenic DP, was further evaluated for genotoxicity using molecular docking. Considering masitinib's clinical relevance, this study highlights the importance of correlating degradation products with known human metabolites and assessing their potential toxicological impact in long-term therapeutic use and environmental exposure.

2025-08-01·Current Opinion in Allergy and Clinical Immunology

New treatments for systemic mastocytosis in 2025

Review

作者: Paoletti, Giovanni ; Marzio, Valentina ; Heffler, Enrico ; Costanzo, Giovanni ; Cavaglià, Edoardo

Purpose of review:

To provide an accessible, comprehensive overview of past, present, imminent, and future therapies for systemic mastocytosis.

Recent findings:

Based on recent trials, the Food and Drug Administration (FDA) and European Medicines Agency (EMA) have approved two drugs for treating advanced systemic mastocytosis: avapritinib and midostaurin. The FDA also approved imatinib for selected cases of aggressive systemic mastocytosis without the D816V c-Kit mutation. Moreover, for the first time, a cytoreductive molecule, avapritinib, has been approved for patients with indolent systemic mastocytosis.

Summary:

Despite the considerable therapeutic progress in recent years, systemic mastocytosis is an incurable disease. In the last 20 years, the management of systemic mastocytosis has transformed from a one-size-fits-all approach, characterized by nonspecific cytoreductive drugs, to a tailored strategy focused on increasingly precise molecular targets, with the most notable example being the KIT inhibitors. Recently, the FDA and EMA have approved two drugs for treating systemic mastocytosis: avapritinib and midostaurin. Moreover, numerous trials are currently assessing the efficacy of new molecules: most are testing new-generation KIT inhibitors (ripretinib, bezuclastinib, elenestinib, masitinib, nintedanib), others focusing on Bruton's kinase (TL-895), interleukin-6 (sarilumab), sialic acid-binding immunoglobulin-like lectin-8 (lirentelimab), mTOR and CD33, among others. Real-life data are needed to confirm preliminary preclinical results.

132

项与甲磺酸马赛替尼相关的新闻（医药）

2025-08-04

·GlobeNewswire-Health Care

AB Science announces the successful completion of a 2.55 million euros private placement

PRESS RELEASE AB SCIENCE ANNOUNCES THE SUCCESSFUL COMPLETION OF A EUR 2.55 MILLION PRIVATE PLACEMENT Paris, August 4, 2025, 8am CET AB Science S.A. (the “Company” or “AB Science”, Euronext – FR0010557264 – AB) announces today the successful completion of a capital increase of a total gross amount of EUR 2.55 million subscribed by a limited number of investors (the “Private Placement”). The Private Placement is not subject to a prospectus requiring an approval from the French Financial Market Authority (Autorité des Marchés Financiers – the “AMF”). In accordance with Article 1.5.(ba) of the Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, as amended (the “Prospectus Regulation”), the Company file with the AMF a document containing the information set out in Appendix IX of the Prospectus Regulation (the “Information document”), copies of which will be available free of charge on the Company’s website at www.ab-science.com and on the AMF’s website at www.amf-france.org. Use of proceeds The Company intends to use the net proceeds of the Private Placement to finance its ongoing activities, with a focus on the clinical development of the AB8939 program. This transaction strengthens the Company's cash position and enables it to cover its financing needs in 2025 and beyond the next 12 months, taking into account the explanations set out in section 5.2.1.5 (note 2) of the 2024 financial report. Terms and conditions of the Private Placement The Private Placement, for a total amount of EUR 2.55 million (including share issue premium), was carried out through the issuance, without preferential subscription rights and without a priority subscription period, of 2,276,787 new ordinary shares of the Company (the “New Shares”), each with one share warrant attached (a “BSA” and, together with the New Share to which it is attached, an “ABSA”), as part of a share capital increase with cancellation of shareholders’ preferential subscription rights for the benefit of investors within the category of persons defined by the 16th resolution of the Combined General Meeting of the Company’s shareholders of June 30, 2025 (the “General Meeting”), in accordance with Article L. 225-138 of the French commercial code (the “Private Placement”). The issue of the ABSAs, representing approximately 3.34% of the Company’s share capital, on a non-diluted basis, before completion of the Private Placement, and 3.23% of the Company’s share capital, on a non-diluted basis, after completion of the Private Placement, was decided on August 1st, 2025 by the Chief Executive Officer, pursuant to the delegation of competence granted to him by the board of directors dated July 24, 2025, pursuant to the delegation of competence granted to it under the 16th resolution of the General Meeting . The issue price of one ABSA is EUR 1.12 (including share issue premium), representing a facial discount of 24.68% (i.e. EUR 0.3669) to the volume-weighted average price of the AB Science shares on the regulated market of Euronext Paris (“Euronext Paris”) over the three trading days preceding the setting of such issue price, i.e. August 1st and July 31 and 30, 2025 (the “3-day VWAP”). The issue price of an ABSA, including the theoretical value of the BSA attached to it (as described below, together with the exercise price of such BSA) represents a total 17.87% discount per AB Science share to the 3-day VWAP, consistent with the maximum discount authorized by the General Meeting pursuant to its 16th resolution. Terms and conditions of the BSA One BSA is attached to each New Share. One BSA entitles their holder to subscribe to one new ordinary share of the Company, at a price of EUR 1.71 per ordinary share. The BSAs may be exercised at any time within 60 months of their issuance. In the event all BSAs are exercised, a total number of 2,276,787 additional ordinary shares of the Company will be issued, representing additional total proceeds of approximately EUR 3.89 million. The theoretical value of each BSA, assuming a volatility of 34.355%1 and based on closing price as of August 1st, 2025, is equal to EUR 0.3877 using Black & Scholes model. The BSAs will be immediately detached (détachés) from the New Shares upon issuance and are expected to be listed on Euronext Growth Paris (“Euronext Growth Paris”) on or prior to August 11, 2025. Impact of the Private Placement on the Company’s shareholding Following the issuance of the ABSAs, the Company’s total share capital will be EUR 704,695.95 (or EUR 727,463.82 in the event of exercise of all BSAs). It will be comprised of 63,706,916 ordinary shares (or of 65,983,703 ordinary shares in the event of exercise of all BSAs) with a par value of EUR 0.01. There will be no change on the number of preferred shares. To the Company’s knowledge, immediately prior to completion of the Private Placement and after completion of the Private Placement, the breakdown of the Company's share capital is as follows: ShareholdersBefore the capital increaseAfter the capital increase (before exercising the warrants)After the capital increase and exercise of the warrantNumber of shares ( 1) %Diluted base ( 2)Number of shares ( 1) %Diluted base ( 2)Number of shares ( 1) %Diluted base ( 2)A. Moussy7 345 39610,77%17,57%7 345 39610,42%17,12%7 345 39610,10%16,69%AMY SAS (3)12 273 00018,00%14,20%12 273 00017,42%13,84%12 273 00016,87%13,49%Subtotal concert A. Moussy19 618 39628,77%31,77%19 618 39627,84%30,96%19 618 39626,97%30,18%Other investors members of the concert2 625 3273,85%5,97%2 625 3273,73%5,82%2 625 3273,61%5,67%Actions in the pact1 123 9021,65%4,24%1 123 9021,59%4,13%1 123 9021,54%4,02%Actions outside the pact1 501 4252,20%1,74%1 501 4252,13%1,69%1 501 4252,06%1,65%Total concert22 243 72332,62%37,74%22 243 72331,56%36,78%22 243 72330,58%35,86%Other investors above 5%6 888 61010,10%9,33%6 888 6109,78%9,09%6 888 6109,47%8,86%Other investors39 060 47557,28%52,93%41 337 26258,66%54,14%43 614 04959,95%55,28%Total68 192 808100,00%100,00%70 469 595100,00%100,00%72 746 382100,00%100,00% (1) All classes of shares are affected. The number of ordinary shares amounts to 61,430,129 before the Private Placement, 63,706,916 after the Private Placement (but before exercise of the BSAs), and 65,983,703 after the Private Placement and exercise of the BSAs.(2) The diluted basis takes into account the exercise of all instruments giving access to the capital, the definitive allocation of all free shares and the conversion of all preferred shares into ordinary shares (aiming for the highest theoretical dilution).(3) AMY SAS is a company controlled by A. Moussy. On the basis of the share capital of the Company immediately after completion of the Private Placement, the interest of a shareholder who held 1.00% of the Company’s share capital prior to the above-mentioned capital increase and who did not subscribe to it now stands at 0.97% on a non-diluted basis and 0.77% on a diluted basis. Admission to trading of the New Shares The New Shares are expected to be admitted to trading on the regulated market of Euronext Paris on August 7, 2025. The New Shares will be subject to the provisions of the Company’s by-laws and will be assimilated to existing shares upon final completion of the Private Placement. They will bear current dividend rights and will be admitted to trading on the same listing line as the Company’s existing shares under the same ISIN code FR0010557264 – AB. Lock-up commitments The Company has signed a lock-up commitment (to the benefit of the investors) pursuant to which it has agreed to a lock-up period of 45 calendar days from the date of the settlement and delivery of the Private Placement, subject to certain customary exceptions. The directors and officers of the Company have signed a lock-up commitment pursuant to which they have agreed to a lock-up period of 90 calendar days from the date of the settlement and delivery of the Private Placement, subject to certain customary exceptions. Indicative timetable July 24, 2025Decisions of the Board of Directors deciding the principle of the Private Placement.August 1st, 2025Decisions of the Chief Executive Officer setting the terms and conditions of the Private Placement (including the subscription price of the ABSAs and the gross amount of the Private Placement).August 4, 2025Publication of this press release.Publication of the Information Document.August 7, 2025Publication of the Euronext notice of admission of the New Shares to trading on Euronext Paris. August 7, 2025Settlement-delivery of the ABSAs - Detachment of the BSA - Start of trading of the New Shares on Euronext Paris.August 11, 2025Admission of the BSAs on Euronext Growth Paris. Risk factors AB Science draws the attention of the public to the risk factors relating to the Company and its business described in its annual management reports and press releases, which are available free of charge on the Company's website (www.ab-science.com). In addition, the main risks specific to securities are as follows: The existing shareholders who do not participate in the Private Placement will see their shareholding in the share capital of AB Science diluted, and this shareholding may also be diluted in the event of exercise of the BSA, as well as in the event of new securities transactions. The volatility and liquidity of AB Science shares could fluctuate significantly. The market price of the Company's shares may fluctuate and fall below the subscription price of the shares issued in the context of the Private Placement. The sale of Company shares may occur on the secondary market, after the Private Placement, and have a negative impact on the Company share price. About masitinib Masitinib is a novel oral tyrosine kinase inhibitor that is being developed to target mast cells and macrophages, key immune cells, through inhibition of a limited number of kinases. Due to its unique mode of action, the Company believed that masitinib can be developed in a wide range of diseases, including oncology, inflammatory diseases, and certain central nervous system diseases. In oncology, through its immunotherapy activity, masitinib may have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglial cells and therefore its inhibitory effect on the activation of the inflammatory process, masitinib may have an effect on the symptoms associated with certain inflammatory and central nervous system diseases. About AB8939 AB8939 is a new synthetic microtubule-destabilizing drug candidate. Preclinical data suggests that AB8939 has broad anticancer activity, with a notable advantage over standard chemotherapies that target microtubules of being able to overcome P-glycoprotein (Pgp) and myeloperoxidase (MPO) mediated drug resistance. Development of drug resistance often restricts the clinical efficacy of microtubule-targeting chemotherapy drugs (for example, taxanes and vinca alkaloids); thus, AB8939 has the potential to be developed in numerous oncology indications. 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 being 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. Disclaimer This press release and the information contained herein do not constitute an offer to subscribe or purchase, or the solicitation of an order to purchase or subscribe, for the New Shares in the United States of America or in any other jurisdiction. Securities may not be offered or sold in the United States of America absent registration under the U.S. Securities Act or an exemption from registration under the U.S. Securities Act. AB Science does not intend to make a public offering of the New Shares in the United States of America or in any other jurisdiction. The distribution of this press release may be subject to legal or regulatory restrictions in certain countries. Persons in possession of this press release should inform themselves of and observe any local restrictions. The information contained herein is subject to change without notice. This information contains forward-looking statements, which are not guarantees of future performance. These statements are based on the current expectations and beliefs of AB Science’s management and are subject to several factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. AB Science and its affiliates, directors, officers, employees, consultants or agents do not undertake, and are not under any obligation, to release any updates to any forward-looking statement or to revise any forward-looking statement. For additional information, please contact: AB Science Financial communication and public relationsinvestors@ab-science.com 1Based on the volatility overt the last 12 months of the Euronext Next Biotech index. Attachment AB Science - August 2025 Press Release US VF

2025-07-30

·GlobeNewswire-Health Care

AB Science receives regulatory approval from European countries to initiate third stage of Phase I/II study combining its molecule AB8939 with venetoclax for the treatment of AML

PRESS RELEASE AB SCIENCE RECEIVES REGULATORY APPROVAL FROM EUROPEAN COUNTRIES TO INITIATE THIRD STAGE OF PHASE I/II STUDY COMBINING ITS MOLECULE AB8939 WITH VENETOCLAX FOR THE TREATMENT OF ACUTE MYELOID LEUKEMIA THE COMBINATION OF AB8939 TARGETING MICROTUBULE AND STEM CELLS AND VENETOCLAX TARGETING BCL-2 SHOWS POTENTIAL SYNERGISTIC EFFICACY AND GOOD HEMATOLOGICAL TOLERANCE COMPARED WITH STANDARD OF CARE CHEMOTHERAPIES, IN PARTICULAR IN THE POOR RESPONDERS TO STANDARD OF CARE CHEMOTHERAPIES TREATMENT OF THE FIRST PATIENTS IS UNDERWAY Paris, July 30, 2025, 8am CET AB Science SA (Euronext - FR0010557264 - AB) today announced the approval of the third of four stages of the Phase I/II study (AB18001) with the compound AB8939 in adult patients with relapsed/refractory acute myeloid leukemia (AML). The third stage has been approved in France, Germany, Spain and Greece. The objective of the Phase 1 study is to determine the maximum tolerated dose (MTD) for different treatment stages of AB8939. Stage 1: Determination of the MTD after 3 consecutive days of treatment with AB8939 alone. Stage 2: Determination of the MTD after 14 consecutive days of treatment with AB8939 alone. Step 3: Determination of the MTD after 14 consecutive days of treatment with AB8939 in combination with venetoclax. Stage 4: Determination of MTD after 14 consecutive days of treatment with AB8939 in combination with venetoclax and azacitidine. The first two stages of Phase 1 were completed with 28 and 13 patients enrolled, respectively, and determined the MTD of AB8939 after 3 consecutive days of treatment (21.3 mg/m2 ) and after 14 consecutive days of treatment (21.3 mg/m2 ). The third stage now consists of evaluating the MTD after 14 consecutive days of treatment with AB8939 in combination with venetoclax, a standard treatment for AML. Professor Olivier Hermine, MD, President of the Scientific Committee of AB Science and member of the Académie des Sciences in France said, “The approval of this third stage is a key milestone for the Phase I/II study with AB8939 in AML. The combination of AB8939 and venetoclax has the potential to change the standard of care in the treatment of relapsed/refractory AML”. Rationale for the AB8939 + venetoclax combination The combination of AB8939 + venetoclax has several potential benefits: 1 - Both molecules have low hematologic toxicity. This combination could therefore be less toxic than azacitidine + venetoclax as first-line treatment for AML. 2 - These two molecules act on different and complementary targets in cancer cells, which could have an additive or even synergistic effect in terms of efficacy. AB8939 has two modes of action: Microtubule destabilization: Microtubules are cellular structures essential for cell division (mitosis). By destabilizing them, AB8939 prevents cancer cells from dividing properly, leading to their death. The advantage is that this type of chemotherapy is independent of the TP53 mutation that creates the most resistance to standard chemotherapies.Inhibition of ALDH (aldehyde dehydrogenase): ALDH is an enzyme found in cancer stem cells, a subtype of cells in AML that are particularly resistant to treatment and responsible for relapses, as they can survive conventional chemotherapy. By inhibiting ALDH, AB8939 specifically targets these stem cells, reducing resistance to treatment and limiting the risk of relapse. Mechanism of action of venetoclax: Inhibition of BCL2 BCL2 is a protein that prevents apoptosis (programmed cell death) in cancer cells.BCL2 is another factor in AML resistance, as it allows cancer cells to survive despite treatment.By blocking BCL2, venetoclax promotes apoptosis, making cancer cells more vulnerable. There is an additive, even synergistic, potential for the combination By simultaneously targeting these mechanisms, the combination could reduce the chances of cancer cells escaping treatment (resistance). This potential synergistic effect arises from the fact that inhibiting ALDH and BCL2 could weaken the resistance mechanisms of cancer stem cells, while destabilizing microtubules prevents cell proliferation. Together, these actions are more powerful than if each molecule were used alone. 3 - Finally, AML is difficult to treat due to its heterogeneity and resistance mechanisms. AB8939 is effective in vitro and in vivo in animals and generates responses in human patients with the poorest prognostic factors when treated with standard of care chemotherapies, namely TP53 mutation, MECOM and complex karyotype. Non-clinical pharmacology data Animal studies have demonstrated the following properties of AB8939 that are relevant for the treatment of AML: AB8939 is active ex vivo against AML cancer cells from chemotherapy-naive or chemotherapy-refractory/relapsed patients, particularly those with TP53 mutations or complex karyotypes.AB8939 eradicates blasts in the blood and bone marrow in PDX models resistant to 5-AraC (cytarabine), particularly those with MECOM rearrangements.AB8939 increases survival and has an additive effect in combination with the standard treatments azacitidine and venetoclax.ALDH expression is a characteristic of cancer stem cells (CSCs), and AB8939 is an ALDH1/2 inhibitor. Therefore, AB8939 is a targeted therapy for leukemic cancer stem cells.AB8939 eradicates leukemia cancer stem cells in a human PDX model of AML. Potential market for AB8939 in AML Treatments for AML represent an estimated market potential of more than €2 billion per year. RegionIncidence(1)% of adressable market (poor responders to SoC chemotherapiesFirst line or relapse) (2)% Insured patients (2)Drug price (€)Market size(k€)United States/Canada23,70050%90100,000 (3)1,000,000Europe27,6009060,000770,000Asia-Pacific27,8003060,000250,000India11,0003060100,000Latin America7,2003060,00065,000MENA3,9003060,00035,000TOTAL90,200 2,200,000 Europe = EU27 + Norway + United Kingdom + Switzerland; Asia-Pacific = Australia, People's Republic of China, Japan, New Zealand, Singapore, Taiwan; Latin America = Argentina, Brazil, Chile, Colombia, Costa Rica, Mexico; MENA = Algeria, Bahrain, Egypt, Israel, Kuwait, Morocco, Oman, Qatar, Saudi Arabia, Tunisia, United Arab Emirates (1) Zhou, Y et al. Global, regional, and national burden of acute myeloid leukemia, 1990–2021: a systematic analysis for the global burden of disease study 2021. Biomark Res 12, 101 (2024). (2) Estimated(3) Choi M. et al. Costs per patient achieving remission with venetoclax-based combinations in newly diagnosed patients with acute myeloid leukemia ineligible for intensive induction chemotherapy. Journal of Managed Care & Specialty Pharmacy Volume 28, Number 9. https://doi.org/10.18553/jmcp.2022.22021 Intellectual property protection until 2036 (with potential 5 years extension) and 2044 for certain chromosomal abnormalities and orphan drug designation by EMA and FDA AB8939 was entirely discovered by AB Science, which retains full ownership of the intellectual property rights, reflecting AB Science's priority to develop innovative drugs aimed at improving patients' lives. The composition of AB8939, including its use in the treatment of AML, is covered until 2026 by a patent granted in all geographical areas where AB8939 could be marketed, including Europe (patent EP 3253749), the United States (US 10,570,122), Canada (CA 2975644), China (CN 107531685), South Korea (KR 10-2544132), Japan (JP 6713000), Hong Kong (HK 1243700), Israel (IL 253779), Australia (AU 2016214283), Russia (RU 2758259), Brazil (BR 112017016883-9), Mexico (MX 377742), India (IN 480996) and South Africa (ZA 2017/05537). An extension of this protection for 5 years is possible in certain countries. A second patent application for medical use has been filed to protect the use of AB8939 in the treatment of AML with certain chromosomal abnormalities. If this application is accepted, protection for AB8939 will be extended until 2044 for these subpopulations of AML patients. In addition to patent protection, AB8939 is also eligible for regulatory data protection in numerous countries, preventing generic competition for a period of up to 8 years from product registration. AB8939 has also received orphan drug designation for AML from both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). This orphan drug designation confers 10 and 7 years of marketing exclusivity in Europe and the US, respectively, from the date of product registration. About AB ScienceFounded 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 is crucial in cell signaling. Our programs target only diseases with high medical need, often fatal with low survival rates, rare, or resistant to first-line treatment.AB Science has developed its own portfolio of molecules, and AB Science's lead molecule, masitinib, has already been registered for veterinary use and is being developed in humans for oncology, neurodegenerative diseases, inflammatory diseases, and viral diseases. The Company is headquartered in Paris and is listed on Euronext Paris (Ticker: AB). For more information about the Company, please visit the website: www.ab-science.com Forward-looking statements – AB ScienceThis 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 regarding projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and potential or future performance. These forward-looking statements can often be identified by words such as "expect," "anticipate," "believe," "intend," "estimate," or "plan," as well as by other similar words. Although AB Science believes that these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties, many of which are difficult to predict and generally beyond the control of AB Science, which could cause actual results and events to differ materially from those expressed, implied or predicted in the forward-looking information and statements. These risks and uncertainties include, in particular, the uncertainties inherent in the development of the Company's products, which may not be successful, or in the granting by the competent authorities of marketing authorizations or, more generally, any factors that may affect the marketability of the products developed by AB Science as well as those developed or identified in the public documents published by AB Science. AB Science undertakes no obligation to update forward-looking information and statements, subject to applicable regulations, in particular Articles 223-1 et seq. of the AMF's General Regulations. For further information, please contact: AB Science Financial Communicationsinvestors@ab-science.com Attachment Authorisation Step 3 PhI AB8939 VENG VF

临床1期孤儿药

2025-07-25

·GlobeNewswire-Health Care

AB Science has received approval from several European countries to initiate the confirmatory phase 3 study of masitinib in ALS

AB SCIENCE HAS RECEIVED APPROVAL FROM SEVERAL EUROPEAN COUNTRIES TO INITIATE THE CONFIRMATORY PHASE 3 STUDY OF MASITINIB IN AMYOTROPHIC LATERAL SCLEROSIS THIS APPROVAL FOLLOWS PROTOCOL VALIDATION BY THE EMA AND FDA AUTHORIZATION July 24, 2025, 8am CET AB Science SA (Euronext - FR0010557264 - AB) today announced that the confirmatory Phase 3 study with masitinib in amyotrophic lateral sclerosis (ALS), study AB23005, has been authorized by the first set of European countries (Spain, Greece, Slovenia) in Step 2 of the Clinical Trials Information System (CTIS) procedure. This authorization follows EMA's validation of the harmonized protocol, approved at the end of Step 1 of the CTIS procedure, and followed the authorization from the FDA. Consequently, AB Science can now initiate this registration study in Europe and the United States. Professor Albert Ludolph, MD, PhD (University of Ulm, Germany), principal investigator of the study, said: "Masitinib ALS study AB23005 has great potential because it’s design is based on strong clinical and preclinical data. That is to say, results from the first 48-week Phase 2B/3 study, AB10015, which generated a strong hypothesis with significant survival of several months in the patient population being targeted in this confirmatory study, and also numerous preclinical studies that provide compelling insight into masitinib’s mechanism of action in ALS and its effect on biomarkers such as neurofilaments (NfL) by targeting mast cells and microglia." Alignment with the FDA and EMA on the Phase 3 design and, in particular, the study population focused on patients who respond best to masitinib. Study AB23005 is a prospective, multicenter, randomized, double-blind, placebo-controlled, two-arm study in patients with amyotrophic lateral sclerosis (ALS), to confirm the efficacy and safety of masitinib (at a dose of 4.5 mg/kg/day in combination with riluzole) as compared against riluzole in combination with placebo after 48 weeks of treatment. The study will include 408 patients (randomized 1:1) with ALS, with normal disease progression (i.e., functional decline of less than 1.1 points per month) and no total loss of function (i.e., a score of at least 1 on each of the 12 items of the ALSFRS-R score). US patients receiving edaravone will also be eligible to participate in the study, with the use of this drug being a stratification factor. This design has been validated in discussions with European health authorities, particularly with regard to the criteria for the optimal population selected for the confirmatory study: Patients without rapid progression: Experts of the EMA's Scientific Advisory Group on Neurology (SAG-N) considered the categorization of the study population with normal progressors, defined as an average rate of change in the ALSFRS-R of less than 1.1 points per month, as clinically relevant and consistent with the expected progression of the disease, and therefore acceptable when predefined, which is the case for this study. Patients without complete loss of function: The SAG-N experts considered that the ALSFRS-R scale is widely used in clinical practice and that administration criteria are available for healthcare professionals. Therefore, the subgroup of patients with very severe ALS (who have a score of zero on at least one of the 12 individual items of the ALSFRS-R) can be easily identified in clinical practice. In this subgroup, defined as patients before complete loss of function and with normal disease progression, which corresponds to the optimal population of best responders to masitinib that is targeted in study AB23005, the AB10015 study generated extremely robust results, with a median survival increase of +12 months. This optimal population represents approximately 75% of the total patient population. The optimal population included approximately 90 patients per treatment group in the AB10015 study. The effect of masitinib was statistically significant (p=0.0290) on the CAFS endpoint, which is the endpoint recognized by the FDA. The AB23005 study will enroll approximately 200 patients per treatment group, more than double the number in the AB10015 study, in order to achieve high statistical power for this test and maximize the chances of statistical success. Masitinib, thanks to its well-demonstrated mechanism of action, particularly its immunomodulatory properties targeting mast cells and microglia, preserves neuromuscular function. The immunomodulatory properties of masitinib in ALS have been well demonstrated in a preclinical setting using a relevant model that replicates the complexity of a multicomponent immune response with concomitant evidence of neurodegeneration [1-4]. The mechanism of action of masitinib in ALS has been confirmed by independent research. Harrison and colleagues showed that treatment of SOD1G93A mice with masitinib significantly reduced macrophage infiltration, prevented the loss of terminal Schwann cells and , and improved reinnervation of partially denervated plantar muscles [5]. Recent pharmacological data have shown that mast cells infiltrate the degenerating spinal cord in both mouse models of ALS and in patients with ALS [6]. The results indicate a protective effect of masitinib. Masitinib has demonstrated its ability to reduce blood levels of neurofilament light (NfL) in a model of neurodegenerative disease (EAE model) [7]. Masitinib has demonstrated its ability to restore motor performance in zebrafish expressing the TDP-43 mutant. Overall, these preclinical results establish the biological plausibility of masitinib for use in ALS and support the hypothesis that masitinib may offer clinical benefit if administered early in the disease course (i.e., before the point of permanent loss of function). Based on the results of the AB10015 study, AB Science has filed a patent application for methods of treating ALS (i.e., a secondary medical use patent) with its lead molecule, masitinib, and this patent has been granted in all jurisdictions where it has been filed. This patent provides strong protection for masitinib in the treatment of ALS until 2037 and in all geographical areas where masitinib could be marketed, including Europe (patent EP 3240538), the United States (US 10092564), Canada (CA 3018635), China (ZL201780019760. 9), South Korea (KR 10-2293847), Japan (JP 7250312B2), Singapore (SG 11201808106Y), Hong Kong (HK 1261581), Israel (IL 261856), Australia (AU M53001274), Eurasia (EA 201800499), Mexico (MX 390495), New Zealand (NZ 745778) and South Africa (ZA 2018/05810). In addition, masitinib has been designated as an orphan drug for ALS by both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). This orphan drug designation confers 10 and 7 years of marketing exclusivity in Europe and the United States, respectively, from the date of product registration. Such status may also be sought in Japan. If granted, the period of commercial exclusivity is extended to 10 years (compared with 4 to 6 years for conventional drugs). References: [1] Trias E, Kovacs M, King PH, et al. Glia. 2020;68(6):1165-1181. [2] Trias E, King PH, Si Y, et al. JCI Insight. 2018;3(19):e123249. [3] Trias E, Ibarburu S, Barreto-Núñez R, et al. JCI Insight. 2017;2(20):e95934. [4] Trias E, Ibarburu S, Barreto-Núñez R, et al. J Neuroinflammation. 2016;13(1):177. [5] Harrison JM, Rafuse VF. Neurobiol Dis. 2020;145:105052. [6] Kovacs M, Alamón C, Maciel C, et al. Acta Neuropathol Commun. 2021;9(1):136. [7] Hermine O, Gros L, Tran T-A, et al. (2025) PLoS ONE 20(4):e0322199. 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). The content above comes from the network. if any infringement, please contact us to modify.

临床结果孤儿药

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

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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	願憲選顧襯顧選糧窪獵(蓋艱鏇選積夢鹹鹽鑰鹽) = 醖顧範鬱鑰糧構積製遞淵觸網壓衊鏇壓糧網醖 (糧構鬱獵糧醖艱鹽網觸 ) 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	鹹襯壓製艱鏇窪積獵餘(積夢遞選糧壓鹹範網廠) = 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. 獵製壓範襯齋壓簾醖蓋 (構鑰廠艱蓋壓齋範餘憲 ) 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	蓋選獵膚範構衊壓糧餘(製醖醖襯醖遞齋鏇製淵) = 顧蓋艱觸鏇積觸鹹餘鑰蓋獵窪鏇願餘醖鏇獵顧 (蓋繭簾遞壓蓋齋醖鏇艱 )	-	2023-04-25
				Riluzole alone	蓋選獵膚範構衊壓糧餘(製醖醖襯醖遞齋鏇製淵) = 淵蓋範顧鑰夢簾鬱鏇範蓋獵窪鏇願餘醖鏇獵顧 (蓋繭簾遞壓蓋齋醖鏇艱 )
NCT01433497 (Pubmed \| Neurol Neuroimmunol Neuroinflamm) 人工标引	临床3期	多发性硬化症	611	masitinib	蓋鏇範簾窪鹹願鏇糧範(憲範鏇網構鹹網壓築觸) = 獵窪糧壓選構積積襯壓夢繭鹹鑰鬱顧鹹繭廠壓 (餘艱鹽鏇網顧襯餘鹽範 )	积极	2022-02-21
				Placebo	蓋鏇範簾窪鹹願鏇糧範(憲範鏇網構鹹網壓築觸) = 齋獵簾遞蓋鑰簾衊繭獵夢繭鹹鑰鬱顧鹹繭廠壓 (餘艱鹽鏇網顧襯餘鹽範 )
AB12003 (AUA2021)	临床3期	转移性去势抵抗性前列腺癌一线 alkaline phosphatase levels (ALP)	714	Masitinib 6.0 mg/kg/d plus Docetaxel	積壓獵蓋築鹽製夢鑰艱(膚築鹹積積積積蓋獵網) = 鑰憲廠廠願醖壓網製衊夢觸製觸齋積選餘願憲 (願遞遞築蓋糧築蓋壓觸 ) 更多	积极	2021-09-01
				Placebo plus Docetaxel	積壓獵蓋築鹽製夢鑰艱(膚築鹹積積積積蓋獵網) = 鑰憲築糧餘築艱築鹽餘夢觸製觸齋積選餘願憲 (願遞遞築蓋糧築蓋壓觸 ) 更多
NCT01872598 (AB09004, GlobeNewswire) 人工标引	临床3期	阿尔茨海默症	358	Masitinib mesylate	鬱網構糧製窪餘範選夢(衊築鏇鏇壓觸遞壓獵窪) = 鹽築鏇繭遞壓積廠襯選夢鏇襯醖窪糧廠窪構艱 (觸衊選積製顧蓋鹹獵鑰 ) 更多	积极	2021-07-29
				Placebo	鬱網構糧製窪餘範選夢(衊築鏇鏇壓觸遞壓獵窪) = 觸襯範繭製獵鬱積網艱夢鏇襯醖窪糧廠窪構艱 (觸衊選積製顧蓋鹹獵鑰 ) 更多
NCT03766295 (AB12005, ASCO2021)	临床3期	胰腺癌一线	384	Masitinib plus gemcitabine	鹽觸夢蓋鹽遞網鑰衊衊(窪簾遞遞鬱獵獵衊淵鬱) = 鑰淵範構顧鬱衊醖構積淵繭網鹽選積顧壓艱簾 (憲構醖衊壓衊衊蓋鬱鹽 )	积极	2021-05-28
				Placebo plus gemcitabine	鹽觸夢蓋鹽遞網鑰衊衊(窪簾遞遞鬱獵獵衊淵鬱) = 獵繭壓簾築遞鏇築積衊淵繭網鹽選積顧壓艱簾 (憲構醖衊壓衊衊蓋鬱鹽 )
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	廠壓積夢淵積膚構憲淵(網鹽範衊顧積鹽範壓膚) = 繭齋憲艱壓壓醖鹹構繭鑰憲獵廠鹹鏇齋襯齋範 (築築醖蓋遞鏇願膚築鏇, 4.9 ~ 6.3) 更多	不佳	2021-05-25
				Docetaxel+Placebo	廠壓積夢淵積膚構憲淵(網鹽範衊顧積鹽範壓膚) = 窪膚構淵憲鏇願範鏇遞鑰憲獵廠鹹鏇齋襯齋範 (築築醖蓋遞鏇願膚築鏇, 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	壓壓糧製蓋夢憲衊醖艱(憲獵網壓選壓壓壓蓋鑰): 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	衊衊鏇窪醖淵窪醖醖壓(壓製糧積壓鹹艱廠願範) = 網鏇獵壓艱顧夢蓋鑰衊窪夢壓壓廠鏇選製網淵 (鏇淵壓淵簾憲窪窪壓顧 ) 更多	积极	2020-10-20
				Placebo	衊衊鏇窪醖淵窪醖醖壓(壓製糧積壓鹹艱廠願範) = 壓蓋獵製製積窪廠鹽憲窪夢壓壓廠鏇選製網淵 (鏇淵壓淵簾憲窪窪壓顧 ) 更多