ALS Phase 3 Study of Masitinib Reaches Target Patient Enrollment
AB Science recently announced it has completed its targeted enrollment of 381 patients for an ongoing Phase 3 study evaluating masitinib in the treatment of amyotrophic lateral sclerosis (ALS).
Masitinib is an orally administered tyrosine kinase inhibitor designed to target important immune system cells known as mast cells and macrophages. Its mechanism of action centers on an inhibitory process, and the company expects the drug to have a positive effect on symptoms associated with some inflammatory and central nervous system diseases, such as ALS, and their degenerative processes. The U.S. Food and Drug Administration has granted masitinib orphan drug designation for ALS.
Patients are being randomized into two treatment groups: masitinib plus riluzole, and placebo plus riluzole (riluzole is an approved oral drug to slow ALS progression). The primary study endpoints will measure the change at week 48 in ALSFRS-R, a validated rating instrument for monitoring the progression of disability in ALS patients, which correlates significantly with quality of life and survival. The study will also compare the efficacy and safety of masitinib.
In a company press release, Alain Moussy, chief executive officer of AB Science, said, “We have completed active recruitment of patients in our phase 3 study in amyotrophic lateral sclerosis as per schedule. Cumulative evidence on masitinib’s efficacy in ALS animal models and the underlying mechanism of action of masitinib based on its interaction with microglia and mast cells have contributed to the high level of recruitment and support from the medical community. The interim analysis will be a crucial milestone.”
This trial was initiated after masitinib showed positive results in a rat model of ALS that included:
- oral administration of masitinib started 7 days after disease onset prolonged post-paralysis survival by 40%, an unprecedented protective effect in ALS rat models;
- reduction in motor neuron pathology in the spinal cord.
An interim study analysis is planned for the first quarter of 2016.
AB Science, founded in 2001, is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins with key action in the signaling pathways within cells.