AB Science announced today interim results from their current Phase 2/3 clinical trial of Masitinib in people with ALS. Before starting the clinical trial, the investigators predetermined that they would conduct this analysis when 50% (N=191) of the people with ALS enrolled completed the trial. It takes 48 weeks for each person with ALS to complete the trial. According to the press release, the report shows that the study has met its primary endpoint, which was defined as change in ALSFRS-R score.
The other 50% (N=190) of people with ALS in the program haven’t completed their 48 week time in the study. While this interim report is encouraging, it is important that all the data from all those in the trial be considered together, which can only occur when the study is completed. Only at that time will AB Science be able to determine whether or not their trial met its endpoints.
This interim report can be considered a “peek” at the trial’s progress toward determining whether or not the treatment met its endpoints. According to the press release, statistical significance was achieved in primary and secondary measures and the side effect profile wasn’t an issue. However, at least one conflicting report published in 2015 suggested the potential for acute and severe autoimmune issues with the treatment of masitinib in people with ALS. A total of 381 people with ALS in Spain were enrolled in the Masitinib clinical trial between 2012 and 2015, when the company reported achieving full enrollment. Therefore, the final report ought to be available sometime toward the end of 2016 or early 2017.
In the press release from the company, AB Science CEO & co-founder, Alain Moussy, suggested that the company will be sharing the data with the EMA and FDA and discussing “marketing authorization for masitinib in ALS”. Those discussions will undoubtedly remain confidential between the company and the regulators.
The company stated in their press release that they will conduct a webinar soon regarding the announcement, and it would be expected in that webinar that additional information will become known.
What is Masitinib?
Masitinib aims to slow progression of ALS by reducing inflammation. A kinase inhibitor, masitinib blocks mast cell-mediated degranulation, the release of cytotoxic substances that might further damage the motor nerves. Marketed by AB Science under the name Kinavet, masitinib is currently being used to treat certain cancers in dogs. It is important to note that the company is also exploring the use of its kinase inhibitor in many other diseases, such as multiple sclerosis, Alzheimer’s and different types of cancer, among others.
What preclinical evidence is there for Masitinib?
There have been preclinical experiments on masitinib reported, however the ALS Therapy Development Institute has not sought to independently reproduce those findings. There were some challenges with the preclinical reports, including that at least one of the studies was done solely with female mice. The full data sets on these preclinical experiments haven’t been published, so it is challenging to comment fully on them.
This is an exciting press release from AB Science regarding their clinical trial in ALS. The trial is 50% done now, and we look forward to seeing the rest of the trial completed and the final data reported. It is also encouraging that AB Science has stated that they will share the data from their predetermined interim analysis not only with regulatory agencies, but also with the general public via a webinar. A date for that webinar has not been set, but the company said it will be occur within the coming days.
We also encourage AB Science to take advantage of several upcoming opportunities to share their data in addition to the webinar they have planned. These opportunities exist within the ENCALS meeting in May and throughout the year. Doing so will enable scientists, researchers, and importantly people with ALS and their families to see and analyze data.
The ALS Therapy Development Institute would like to recognize the nearly 400 people with ALS and their families who are participating in this study of masitinib. Today, there are dozens of clinical trials occurring worldwide in ALS, including several on potential treatments. ALS.net maintains a comprehensive, unbiased resource of all ALS clinical trials occurring across the globe today, which you can access by clicking here. A lively discussion of this proposed treatment is occurring on the ALS Forum.