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

/ 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

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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370

项与甲磺酸马赛替尼相关的文献（医药）

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.

2025-07-01·NEUROSCIENCE LETTERS

Masitinib attenuates neuropathological changes in acrolein-induced sAD mouse model via NF-κB/NLRP3/Caspase-1 signaling pathway

Article

作者: Shen, Qianhui ; Jia, Kang ; Liang, Zhi ; Ren, Yu ; Wei, Cailv ; Zhang, Zhixian ; Pi, Rongbiao ; Chen, Yanping ; Ding, Chao ; Zhou, Sigui ; Liu, Yuan ; Wang, Shisong

Alzheimer's Disease (AD) is a global health crisis, with sporadic AD (sAD) accounting for more than 95 % of all cases. The lack of effective disease-modifying therapies for sAD, driven by its complex pathogenesis involving genetic, environmental, and lifestyle factors, underscores the urgent need for novel treatments. Masitinib, an oral tyrosine kinase inhibitor originally developed for cancer treatment, has shown potential to regulate mast cells and neuroinflammation, making it a promising candidate against AD. In this study, we investigated the therapeutic effects of masitinib (60 mg/kg/day) in acrolein-induced sAD mouse model. A comprehensive series of behavioral tests, including the buried food pellet tests, Morris water maze, Y-maze, open field, and elevated plus maze, along with Western blot, immunofluorescence and Golgi-Cox staining were used to evaluate pathological changes. The results showed that masitinib significantly improved acrolein-induced olfactory deficits, cognitive dysfunction, particularly in learning and memory, and anxiety-like behaviors. Additionally, masitinib not only reduced p-Tau levels, increased PSD95 expression and restored dendritic spine density, but also suppressed neuroinflammation by inhibiting the NF-κB/NLRP3/caspase-1 inflammatory pathway and microglial activation. These findings demonstrate that masitinib, for the first time, attenuates sAD pathology through dual mechanisms of cognitive enhancement and neuroprotection. Our study provides strong preclinical evidence to support further clinical development of masitinib as a disease-modifying therapy for sAD.

2025-07-01·ECOTOXICOLOGY AND ENVIRONMENTAL SAFETY

Dibutyl phthalate promotes central precocious puberty through primordial follicle activation by downregulating BMP15

Article

作者: Liu, Wenjuan ; Wang, Zhaoyi ; Liu, Fenghua ; Yao, Yangcheng ; Tang, Maoxing ; Zhang, Xiqian ; Li, Yufeng ; Xiao, Xiaomin

Dibutyl phthalate (DBP) has been reported to induce central precocious puberty (CPP). However, the mechanism by which DBP accelerates primordial follicle activation and contributes to CPP has not been studied. This study aims to explore the effects of DBP on puberty onset and the role of primordial follicle activation in DBP-related CPP. Female ICR pups (developed by the Institute of Cancer Research, USA) were exposed to DBP from PND5 to PND20. Vaginal opening, a marker of puberty onset, was monitored daily staring from PND20. The involvement of the hypothalamus-pituitary-ovary axis (HPOA) was evaluated, and the activation of primordial follicles was analysed. To investigate the role of primordial follicle activation in CPP development, masitinib was used to establish a model of primordial follicle silencing. Additionally, a neonatal ovary in vitro culture model was developed to explore the mechanism by which DBP accelerates primordial follicle activation. Compared to the control group, the DBP-exposed group exhibited significantly earlier vaginal opening. The hypothalamic gene expression of KISS1, GPR54, as well as serum levels of LH and E2, were elevated. Moreover, the activation of primordial follicle was accelerated in the DBP group. However, these effects were blocked by masitinib treatment. In vitro culture of neonatal ovaries revealed that DBP downregulates AMH expression via the BMP15/p38-MAPK pathway. In conclusion, our study firstly demonstrates that the activation of primordial follicles is essential for CPP development, and DBP contributes to CPP by accelerating primordial follicle activation through the BMP15/p38-MAPK/AMH pathway.

129

项与甲磺酸马赛替尼相关的新闻（医药）

2025-07-08

·GlobeNewswire-Health Care

AB Science announces the successful completion of a EUR 1.925 million private placement

PRESS RELEASE AB SCIENCE ANNOUNCES THE SUCCESSFUL COMPLETION OF A EUR 1.925 MILLION PRIVATE PLACEMENT Paris, July 8, 2025, 8am 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 1.925 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 1.925 million (including share issue premium), was carried out through the issuance, without preferential subscription rights and without a priority subscription period, of 1,645,302 new ordinary shares in 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 2.47% of the Company’s share capital, on a non-diluted basis, before completion of the Private Placement, and 2,41% of the Company’s share capital, on a non-diluted basis, after completion of the Private Placement, was decided on July 7, 2025 by the Chief Executive Officer, pursuant to the delegation of competence granted to him by the board of directors dated July 7, 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 1.17 (including share issue premium), representing a facial discount of 24.36% (i.e. EUR 0.38) 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. July 7, 4 and 3, 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 18.84% 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 1.78 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 1,645,302 additional ordinary shares of the Company will be issued, representing additional total proceeds of approximately EUR 2.93 million. The theoretical value of each BSA, assuming a volatility of 34.355%1 and based on closing price as of July 7, 2025, is equal to EUR 0.4392 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 July 14, 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 681,937.55 (or EUR 698,390.57 in the event of exercise of all BSAs). It will be comprised of 61,431,076 ordinary shares (or of 63,076,378 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. 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 0.98% on a non-diluted basis and 0.79% 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 July 10, 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 7, 2025Decisions of the Board of Directors deciding the principle of the Private Placement.July 7, 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).July 8, 2025Publication of this press release.Publication of the Information Document.July 10, 2025Publication of the Euronext notice of admission of the New Shares to trading on Euronext Paris. July 10, 2025Settlement-delivery of the ABSAs - Detachment of the BSA - Start of trading of the New Shares on Euronext Paris.July 14, 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 - AK July 2025 Press Release VEng VF

2025-07-04

·GlobeNewswire-Health Care

AB Science: Masitinib receives FDA and EMA authorization for confirmatory phase 3 trial in metastatic castrate-resistant prostate cancer

PRESS RELEASE MASITINIB RECEIVES FDA AND EMA AUTHORIZATION FOR CONFIRMATORY PHASE 3 TRIAL IN METASTATIC CASTRATE-RESISTANT PROSTATE CANCER, WITH BIOMARKER-DRIVEN PATIENT SELECTION TARGETING POPULATION MOST LIKELY TO BENEFIT Paris, July 4, 2025, 8am CET AB Science SA (Euronext - FR0010557264 - AB) today announced that a confirmatory phase 3 trial of masitinib in metastatic castrate resistant prostate cancer (study AB22007) has been authorized by FDA and EMA (harmonized protocol approved through step 1 of Clinical Trials Information System), with a biomarker that targets patients with less advanced metastatic disease. Professor Olivier Hermine, MD, President of the Scientific Committee of AB Science and member of the Académie des Sciences in France said, “The authorization of our confirmatory Phase 3 study by both the FDA and EMA represents a critical milestone for masitinib in metastatic castrate-resistant prostate cancer. With a validated biomarker guiding patient selection, this trial has the potential to establish the first targeted combination with docetaxel in nearly two decades for mCRPC.” Design of phase 3 study Study AB22007 is a prospective, multicenter, randomized, double blind, placebo-controlled, 2-parallel groups, phase 3 study to confirm the efficacy and safety of docetaxel (IV 75 mg/m² plus prednisone for up to 10 cycles) plus masitinib 6.0 mg/kg/d, versus docetaxel plus placebo in metastatic castrate resistant prostate cancer (mCRPC). The study will enroll 600 patients (randomization 1:1) with confirmed mCRPC eligible to docetaxel and with a biomarker (as measured by baseline alkaline phosphatase level) indicative of less advanced metastatic disease. The study’s primary endpoint will be radiographic progression free survival (rPFS), supported by overall survival as the first secondary endpoint. Masitinib is positioned in metastatic castrate resistant prostate cancer eligible to docetaxel, a high unmet medical need Masitinib is positioned in combination with docetaxel as a treatment of mCRPC patients who are eligible to docetaxel; that is to say, it is administered directly following resistance or relapse after the metastatic hormone-sensitive prostate cancer (mHSPC) treatments. While there are numerous treatments in the mHSPC treatment space, there is currently no drug registered for use in combination with standard of care treatment docetaxel in patients who have relapsed on hormone treatments, i.e., patients with mCRPC, despite docetaxel having been approved almost 20 years ago. Although localized disease is associated with high survival rates, metastatic prostate cancer still represents an unmet medical need with a 5-years survival rate of about 30% [1]. Up to 20% of men who undergo state-of-the art treatment for prostate cancer will develop CRPC within 5 years, and at least 84% of these will have metastases at the time of CRPC diagnosis [2]. Practically all patients with metastatic disease become resistant to androgen-deprivation therapy. Prostate cancer is the most common cause of cancer in men, with 137.9 new cases per 100,000 men per year [2]. The estimated prevalence of people living with prostate cancer is 113 per 100,000 [3], with approximately 15% of the patients having mCRPC eligible to chemotherapy [4]. As such, the population with mCRPC eligible to chemotherapy is around 75,000 in the EU and 50,000 in the USA. Results from study AB12003 demonstrated that the biomarker Alkaline Phosphatase predicts response of masitinib in mCRPC. The combination of masitinib plus docetaxel may provide a new first-line treatment option for mCRPC patients with low metastatic involvement Primary analysis: AB12003 was a prospective, placebo controlled, double blind, randomized, phase 3 trial, evaluating masitinib (6.0 mg/kg/d) in combination with docetaxel (IV 75 mg/m² plus prednisone for up to 10 cycles) as a first-line treatment of mCRPC. Eligible patients were chemo-naïve with confirmed mCRPC, who had progressed on previous abiraterone treatment or were indicated for docetaxel treatment, and had a ECOG ≤1. Primary analysis was performed on a pre-specified targeted subgroup, defined as patients with baseline alkaline phosphatase levels (ALP) ≤250 IU/L, and on the overall population. Primary endpoint was progression free survival (PFS) (PCWG2 definition). The study was successful if improvement in median PFS relative to control reached a 3.9% level of significance for the target subgroup (alpha split with fallback procedure to conserve overall type-I error at 5% for the overall study cohort). Primary analysis was based on 450 patients in the targeted subgroup (ALP ≤ 250 IU/L). There was a total of 712 patients in the overall study cohort. Masitinib (6.0 mg/kg/day) plus docetaxel confers a significant PFS benefit in mCRPC patients with ALP ≤ 250 IU/L. Hazard ratio of 0.79 [0.64;0.97] (p=0.0087), corresponding to a 21% reduction in risk of progression relative to control. Assessment of PFS rates was convergent with this primary outcome; 12, 18, and 24-month PFS rates showed significant improvement in favor of masitinib plus docetaxel relative to control: 1.6-fold (p=0.0035), 1.9-fold (p=0.0001) and 1.9-fold (p=0.0028), respectively. ALP as a biomarker: Importantly, a progressively greater masitinib treatment effect was observed for lower baseline ALP levels (less advanced metastatic disease), with a significant 47% reduced risk of progression in patients with ALP≤100 IU/L (hazard ratio=0.53, p=0.002). The efficacy and response of masitinib was in fact correlated to the level of ALP. The use of biomarker ALP for the confirmatory phase 3 study has been validated by FDA and EMA. The establishment of a biomarker predictive of the response to masitinib is a potentially important discovery. ALP measures the involvement in the bones and in the liver of metastasis. When used sufficiently early, masitinib in combination with docetaxel was able to slow down the progression of the metastatic cancer even resistant to hormone treatments. The masitinib plus docetaxel safety profile was acceptable with respect to control; consistent with the known masitinib profile and no new safety signals observed. Historically, there has been a high failure rate of trials studying combinations of docetaxel and new targeted agents, with study AB12003 being a rare example of a phase 3 clinical trial that showed improvement in progression-free survival (PFS) for masitinib in combination with docetaxel. Patent protection until 2042 Based on the results from AB12003 study, AB Science filed a patent application relating to methods of treating mCRPC (i.e. a secondary medical use patent) with its lead compound masitinib. The European Patent Office has granted this patent (EP4175639). It provides protection until 2042 for masitinib and related compounds for treatment of mCRPC in the patient subpopulation with low metastatic involvement (as measured by baseline alkaline phosphatase levels), which is the patient population in the approved phase 3 study of masitinib in mCRPC. Counterpart patent applications have also been filed in other major international markets, including the United States. References : [1] Cancer stat facts: prostate cancer. National Cancer Institute/ Surveillance, Epidemiology, and End Results Program. Accessed September 10, 2021. https://seer.cancer.gov/statfacts/html/prost.html[2] Crawford ED, Petrylak D, Sartor O. Navigating the evolving therapeutic landscape in advanced prostate cancer. Urol Oncol. 2017 May;35S:S1-S13. doi: 10.1016/j.urolonc.2017.01.020. [3] Bray F, Ferlay J, Soerjomataram I, Siegel RL, Torre LA, Jemal A. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68(6):394–424. [4] Scher 2015 – PLoSONE - Symptomatic mCRPC that has not been treated with or not progressed on chemotherapy 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 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 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 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 ScienceFinancial Communication & Media Relations investors@ab-science.com Attachment Authorisation Ph3 mRCPC VENG VF

临床3期临床结果

2025-07-02

·EINPresswire

Amyotrophic Lateral Sclerosis (ALS): Competitive Intelligence Insight into Next-Gen Therapies | DataM Intelligence

ALS treatment is evolving from symptom management to disease-modifying strategies gene therapies, antisense drugs, & neuroprotective agents promise real impact ALS, once incurable, is seeing remarkable progress. With gene interventions and neuroprotective molecules entering late-stage trials, the vision of slowing or halting progression is closer than ever.” — DataM Intelligence AUSTIN, TX, UNITED STATES, July 2, 2025 / EINPresswire.com / -- Amyotrophic Lateral Sclerosis (ALS) , often known as Lou Gehrig’s disease, is a fatal neurodegenerative disorder that progressively destroys motor neurons, leading to paralysis and death. It remains one of the most complex and devastating neurological diseases, with a prognosis of just 2 to 5 years post-diagnosis for most patients. However, the landscape in 2025 reflects significant progress. With deeper genetic insights and a diversified therapeutic pipeline, ALS is transitioning from symptom management toward disease modification. Download CI Sample Report: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🧬 Disease Overview ALS causes the degeneration of both upper and lower motor neurons, disrupting voluntary muscle activity. This leads to severe muscle wasting, difficulties in swallowing and breathing, and ultimately, respiratory failure. While about 90% of ALS cases are sporadic, the remaining 10% are familial, frequently linked to mutations in genes such as SOD1, C9orf72, TARDBP, and FUS. Diagnosis is typically clinical, supported by electromyography (EMG), nerve conduction studies, and exclusion of mimicking conditions. Biomarker development is ongoing, aiming to enable earlier diagnosis and patient stratification for clinical trials. 🌍 Epidemiology & Market Landscape Globally, ALS affects an estimated 2–5 people per 100,000, with an annual incidence of 1–2 per 100,000. The disease is more common in individuals aged 55–75 and is slightly more prevalent in men. Due to its high mortality and lack of curative therapies, ALS qualifies for orphan drug status in major markets. This regulatory incentive, combined with strong advocacy and rising R&D investment, is accelerating the therapeutic pipeline. The ALS treatment market, valued at around USD 2 billion in 2024, is projected to grow steadily with the entry of novel disease-modifying therapies over the next five years. 💊 Current Treatment Paradigm The current standard of care is limited to supportive and symptomatic therapies. These include: - Riluzole: Approved in 1995, it modestly extends survival by a few months by reducing glutamate-mediated excitotoxicity. - Edaravone: Approved in 2017, it acts as a free radical scavenger, slightly delaying functional decline in early-stage ALS. - Nuedexta: Although not approved specifically for ALS, it is commonly used to manage pseudobulbar affect (PBA), improving emotional stability. While helpful, these therapies do not halt or reverse the disease, highlighting a clear unmet need for disease-modifying approaches. Book Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🔬 Pipeline Innovation and Competitive Momentum The ALS pipeline in 2025 is driven by a multi-pronged approach, including gene silencing, neuroprotection, immunomodulation, and cellular therapies. Key areas of innovation include: Gene-Targeted Therapies - Biogen’s Tofersen, an antisense oligonucleotide (ASO) targeting SOD1 mutations, has shown promise in reducing SOD1 protein levels and slowing disease progression in familial ALS. It represents the first real attempt at targeting a genetic driver in ALS with precision. - Another gene-targeted agent, BIIB100, is in early-phase trials to treat C9orf72-associated ALS, a common cause of familial and sporadic cases. These therapies are designed to address root-cause mutations and could revolutionize ALS management in genetically stratified patients. Neuroprotective and Combination Therapies - AMX0035, a fixed-dose combination of sodium phenylbutyrate and taurursodiol, showed significant survival benefit and slower functional decline in late-phase trials. With a favorable safety profile and oral administration, it’s emerging as a strong candidate for broad use across ALS populations. - Other agents in this class aim to support mitochondrial function, reduce ER stress, or modulate excitotoxicity-core mechanisms implicated in ALS pathogenesis. Immuno-inflammatory Modulators - Masitinib, a tyrosine kinase inhibitor, targets neuroinflammation and microglial activation. Now in Phase III, it could become the first oral immune-modulating agent with broad applicability in ALS if efficacy is confirmed. - Avonex (interferon-beta) and other repurposed immune therapies are being evaluated to address inflammatory aspects of ALS, particularly in patients showing elevated biomarkers of immune activation. Stem Cell and Cellular Approaches Stem cell infusions, particularly mesenchymal stem cells (MSCs), are under evaluation for their potential to protect or repair damaged motor neurons. These approaches remain experimental but are gaining attention for their regenerative promise. 🔍 Competitive Intelligence – Descriptive Landscape Tofersen is leading the field in genetically targeted therapy for SOD1-mutated ALS. Its ability to slow functional decline and reduce neurofilament biomarkers puts it ahead in terms of precision and biological targeting. In the broader ALS population, AMX0035 offers a disease-modifying option with strong survival data and ease of use. It stands out as the first combination therapy likely to gain global traction for sporadic ALS. Masitinib, still under evaluation, represents a promising oral option for patients with inflammatory phenotypes. Its oral route and broad immunologic targeting could position it as a foundational therapy if efficacy and tolerability are confirmed. Meanwhile, stem cell therapies and early-stage gene-editing solutions such as BIIB100 are advancing with hopes of establishing novel, durable interventions. These will likely serve as adjuncts or niche solutions depending on patient genotype and disease stage. 🎯 Target Opportunity Profile (TOP): What Success Looks Like For any new therapy to thrive in the ALS market, it must deliver across a range of clinical and strategic attributes: - Clinical Efficacy: Demonstrated improvement in survival and/or functional endpoints such as ALSFRS-R or time to tracheostomy. - Safety and Tolerability: Minimal systemic side effects, especially critical in a fragile patient population with reduced physiological reserve. - Mechanism Differentiation: Gene-targeted therapies should address known mutations; other candidates must tackle neuroinflammation, oxidative stress, or neuronal energy failure. - Ease of Administration: Oral or infrequent dosing regimens are preferred. Intrathecal administration may be acceptable for precision therapies like ASOs. - Payer & Access Considerations: Orphan status, cost-effectiveness, and robust real-world data will be vital for widespread adoption and reimbursement. - Patient Stratification: Success depends on biomarker-driven selection, especially for genetic or inflammatory subtype-specific therapies. Ask for Customized CI Service as per Your Business Requirement: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🔮 Market Outlook: 2025 and Beyond The years ahead will witness critical inflection points: - Tofersen is expected to reshape treatment in genetically confirmed SOD1-ALS, catalyzing greater adoption of genetic testing. - AMX0035 will likely establish itself as the first broadly used disease-modifying therapy. - Masitinib may expand treatment choices for sporadic ALS if it confirms efficacy in ongoing trials. - Gene therapies and cell-based innovations will push the envelope toward true disease reversal or stabilization. ✅ Conclusion After decades of frustration, the ALS field is entering a breakthrough era. New therapies are moving beyond symptomatic support to targeting the underlying biology of the disease. From gene silencing to neuroprotection and immunomodulation, the coming years hold unprecedented potential to extend and improve lives. For patients, caregivers, and industry stakeholders, ALS is no longer a closed chapter-it’s a rapidly unfolding story of innovation. Read Related CI Reports: 1. Focal Segmental Glomerulosclerosis | Competitive Intelligence 2. Vitiligo | Competitive Intelligence Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 sai.k@datamintelligence.com Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

临床3期上市批准孤儿药寡核苷酸免疫疗法

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	襯艱襯膚夢蓋鏇鹹鹽簾(鏇壓範鹽網淵選齋範艱) = 觸願顧艱壓積構顧鏇夢餘觸壓構鏇簾積廠淵膚 (夢遞齋淵衊製鹹餘製夢 ) 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	鹽選構構蓋鬱鬱襯艱壓(衊選繭鹹鬱顧觸夢憲獵) = 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. 窪憲製築遞壓積鑰壓鬱 (觸鬱醖淵製鹽壓鏇夢糧 ) 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	簾築鏇廠襯繭願衊簾顧(選淵築範觸鬱積繭製壓) = 齋夢鏇簾夢網範襯顧構淵範鹹鹹鹹範構憲廠範 (鏇窪醖願壓願襯簾鬱夢 )	-	2023-04-25
				Riluzole alone	簾築鏇廠襯繭願衊簾顧(選淵築範觸鬱積繭製壓) = 鹽艱積壓膚範願壓願憲淵範鹹鹹鹹範構憲廠範 (鏇窪醖願壓願襯簾鬱夢 )
NCT01433497 (Pubmed \| Neurol Neuroimmunol Neuroinflamm) 人工标引	临床3期	多发性硬化症	611	masitinib	膚醖廠願醖廠鏇積積壓(構觸膚簾齋膚糧鏇淵鹹) = 蓋餘積廠鏇選築構餘窪顧鹽壓網構醖網夢鹽鏇 (襯構廠製醖願淵醖築築 )	积极	2022-02-21
				Placebo	膚醖廠願醖廠鏇積積壓(構觸膚簾齋膚糧鏇淵鹹) = 繭糧蓋製糧壓選製膚範顧鹽壓網構醖網夢鹽鏇 (襯構廠製醖願淵醖築築 )
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	廠範憲餘願淵窪積選積(窪鏇糧鑰餘範淵窪鏇窪) = 範衊築蓋夢網獵夢築鹽鏇築選遞齋觸鬱簾壓遞 (顧築憲繭構齋糧鏇簾觸 ) 更多	积极	2021-09-01
				Placebo plus Docetaxel	廠範憲餘願淵窪積選積(窪鏇糧鑰餘範淵窪鏇窪) = 膚鬱積鑰繭築遞顧蓋範鏇築選遞齋觸鬱簾壓遞 (顧築憲繭構齋糧鏇簾觸 ) 更多
NCT01872598 (AB09004, GlobeNewswire) 人工标引	临床3期	阿尔茨海默症	358	Masitinib mesylate	廠獵齋艱構蓋選遞選積(願襯築鏇鑰選醖構獵鏇) = 蓋襯壓觸顧獵衊艱選壓窪鑰衊鹽膚網獵鬱鏇齋 (鹹築製獵範網觸餘糧鹽 ) 更多	积极	2021-07-29
				Placebo	廠獵齋艱構蓋選遞選積(願襯築鏇鑰選醖構獵鏇) = 選願窪繭淵選鹽齋糧艱窪鑰衊鹽膚網獵鬱鏇齋 (鹹築製獵範網觸餘糧鹽 ) 更多
NCT03766295 (AB12005, ASCO2021)	临床3期	胰腺癌一线	384	Masitinib plus gemcitabine	壓淵鏇醖襯廠衊壓鏇齋(積壓窪遞衊餘憲憲齋築) = 網簾選築網鹽鏇願鹽網網選鏇積壓窪顧窪鬱顧 (遞築積醖壓廠廠夢鬱蓋 )	积极	2021-05-28
				Placebo plus gemcitabine	壓淵鏇醖襯廠衊壓鏇齋(積壓窪遞衊餘憲憲齋築) = 繭遞範窪鹽顧範鑰選餘網選鏇積壓窪顧窪鬱顧 (遞築積醖壓廠廠夢鬱蓋 )
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	鹹衊鬱壓積鏇蓋構觸製(壓簾鏇鏇壓艱遞衊艱簾) = 夢淵艱蓋觸衊選網淵製夢壓襯餘鹽鹽糧壓顧糧 (衊壓繭顧膚獵製壓構簾, 4.9 ~ 6.3) 更多	不佳	2021-05-25
				Docetaxel+Placebo	鹹衊鬱壓積鏇蓋構觸製(壓簾鏇鏇壓艱遞衊艱簾) = 範糧餘衊淵淵衊醖築築夢壓襯餘鹽鹽糧壓顧糧 (衊壓繭顧膚獵製壓構簾, 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	範遞構顧築憲艱獵窪築(衊襯簾壓簾簾醖鑰築淵): 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	鹹廠廠願蓋窪艱糧艱夢(夢繭簾觸壓鏇構積築窪) = 襯淵憲淵襯構壓繭遞膚艱糧願觸窪選齋淵鹹齋 (築鏇襯淵繭網鏇廠構廠 ) 更多	积极	2020-10-20
				Placebo	鹹廠廠願蓋窪艱糧艱夢(夢繭簾觸壓鏇構積築窪) = 鏇餘網鏇蓋蓋鹹廠簾構艱糧願觸窪選齋淵鹹齋 (築鏇襯淵繭網鏇廠構廠 ) 更多