Call with Mesoblast re CHF interim

Hi all

As promised, I said I would get back to all you folks after I got in contact with Mesoblast re the CHF interim.

I had a call with them today and had three questions, and received these responses:

Q 1. In May 2016, we were advised that interim data would not be provided to Mesoblast, so what type of data can we expect from the upcoming interim?

Mesoblast's Answer:  The trial will remain double blinded, that is Mesoblast, patients, and investigators will not see any patient level data. The only party who has access to the patient level data is the DMC and independent statisticians. This upcoming interim is a "futility" test, similar to the aGVHD one done late 2016 i.e. no patient level data will be provided.

The process is that the DMC will review the patient level data for safety, and then the data is provided to the independent statisticians and they will provide a recommendation to either proceed or not proceed based on the results assessed against the primary endpoint.

Q2. What was the difference between the Apr16 and Oct16 DMC read-outs vs the upcoming interim read-out?

Mesoblast's Answer: The two read-outs in 2016 were for safety only, where the DMC had access to patient level data. No other party had access to patient level data, or else the entire trial would be compromised. The DMC only provided its recommendation on safety, and NOT futility (effectiveness).

This upcoming interim is a 'futility test', and the first to be taken for his phase 3 trial. To be clear, this is the first recommendation from the DMC & statisticians based on effectiveness of the treatment measured against the primary endpoint.

Q 3. What will this interim assess?
Mesoblast's Answer: Primary endpoint, which has prominence over all secondary end-points - just look at Novartis, their trial failed as it did not meet its primary endpoint. Secondary endpoints are not the be all end all, the primary end point is!


Summary
There will be no detailed data provided for this interim, it will be similar to the aGVHD announcement in terms of the level of detail/guidance provided on the interim. This will not impact the integrity of the trial as some fear.

And I did try my luck on when the announcement will be made, but got the standard 'hopefully soon' response.

Good luck all