PRESS RELEASE
AB SCIENCE PROVIDES UPDATE ON APPLICATION FOR CONDITIONAL MARKETING AUTHORIZATION OF MASITINIB IN THE TREATMENT OF AMIOTROPHIC LATERAL SCLEROSIS
Paris, June 28, 2024, 12:30 pm CET
AB Science SA (Euronext – FR0010557264 – AB) announces today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted, in line with the trend vote, a negative opinion on the application for conditional authorization for introduction into the masitinib market in the treatment of amyotrophic lateral sclerosis (ALS).
AB Science intends to request a reexamination based on:
1) Firstly, the urgent need for patients to have early access to a promising treatment.
2) The opportunity for the dossier to be re-examined by new rapporteurs and by a Scientific Advisory Board.
AB Science highlights the difficulty of a conditional marketing authorization in ALS and cannot guarantee a positive outcome following this review.
The grounds for requesting a review may be based on the following elements:
- Acceptable safety of masitinib:First, the CHMP confirmed that the safety of masitinib is considered acceptable, which is a key consideration in the context of a conditional marketing authorisation where confirmatory evidence of efficacy is required.
- Objection regarding deviations from Good Clinical Practices: As per EMA guidance (EMA/868942/2011), impact analyzes of all protocol deviations that could not be corrected were carried out and showed no impact, resolving Good Clinical Practice issues as per the guideline.
- Objection regarding the exclusion of fast pacers: The amendment that carried over from phase 2 to phase 3 excluding rapid progressors from the primary analysis population was necessary and well justified in order to have a more homogeneous population with a greater chance of reaching the week 48 time point and to minimize missing data. Furthermore, the amendment was implemented early enough and while the study was blinded to remove any methodological issues.
- Objection regarding the treatment of missing data in the primary analysis: Multiple sensitivity analysis of the primary analysis; using non-LOCF (Last observation made) methods for imputing missing data are positive and consistent, including two analyses previously recommended by the CHMP, demonstrating the robustness of the primary analysis, thus resolving the objection regarding the treatment of missing data.
- Objection about subgroup data: There was a significant imbalance in a subgroup of patients who experienced complete loss of function (i.e., ALSFRS-R score of zero) in one or more item scores (20% in the masitinib arm versus 8% in the placebo arm), because the ALSFRS-R score was minimized but not stratified by severity category. The subgroup defined as patients before any complete loss of function (i.e., excluding the biased subgroup mentioned above) accounted for 86% of the population and showed extremely convincing results, including a significant 12-month survival benefit. The subgroup analysis is a strict application of the EMA guidance (EMA/CHMP/539146/2013), which is applicable to post hoc analysis and single pivotal study registration, thus resolving the objection regarding subgroup data.
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 is essential 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, is already 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).
More information is available on the AB Science 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, and statements based on projects, objectives, intentions and expectations with respect to 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. 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 that are difficult to predict and generally beyond AB Science’s control and that could cause actual results and events to differ materially from those expressed, implied or forecasted in the forward-looking information and statements. These risks and uncertainties include uncertainties related to the Company’s product development that may not be successful or to the marketing authorizations granted by the competent authorities or, more generally, any factors that may affect the commercialization ability 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 applicable regulations, in particular Articles 223-1 et seq. of the AMF General Regulations.
For additional information, please contact:
AB Science
Financial Communication and Media Relations
investors@ab-science.com
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