French biotech company AB Science has announced some positive news in its Alzheimer’s programme as its phase III trial has been successful at the first hurdle.
Given that the development path for Alzheimer’s drugs is littered with failures, AB Science is pleased that its trial of masitinib has passed a futility test and will now continue to its conclusion.
The company is planning a conference call later this week to discuss progress with masitinib, an orally-active tyrosine kinase inhibitor that dampens down the immune response by inhibiting the activity of two types of white blood cell, namely mast cells and macrophages. It is already sold as a veterinary medicine to treat mast cell tumours in dogs.
The drug is thought to work in Alzheimer’s by reducing the inflammation that is seen in the brains of Alzheimer’s patients, specifically by blocking the infiltration of mast cells across the blood brain barrier and into the central nervous system.
That means it works in a different way to many of the failed Alzheimer’s candidates, which overall have tried to inhibit the amyloid plaques that are seen in the brains of patients, with a host of failed projects including most recently Roche’s gantenerumab.
The phase III trial is comparing masitinib to placebo, both given on top of background therapy with cholinesterase inhibitors or the NMDA receptor antagonist memantine, in patients with mild-to-moderate Alzheimer’s disease.
The primary outcome measure – cognition and memory assessment after 24 weeks – is due to read out in December 2015, based on a target enrolment of around 400 patients.
The futility assessment was carried out after a third of the target population had been enrolled and concluded that the study “has the potential to achieve its stated efficacy objective,” said AB Science in a statement.
A smaller, phase II trial completed was published in 2011 and found that adding masitinib to standard therapy led to a small improvement in cognition and memory scores over 24 weeks, while the placebo group continued to decline.
AB Science seems to be taking a scattergun approach to masitinib’s development. The drug is in trials for a host of cancer indications, including phase III studies in gastrointestinal stromal tumours (GIST), colorectal cancer, multiple myeloma and melanoma, as well as phase II trials in stomach and breast cancer, amongst others.
However, it has also suffered some significant setbacks, having been turned down by the Committee for Medicinal Products for Human Use (CHMP) last year as a treatment for GIST in 2013 and pancreatic cancer last year.
Outside the cancer area it is being trialled in multiple sclerosis, rheumatoid arthritis, asthma and motor neuron disease.