Phase 2b CHF (LVAD) clinical trial

Hi all

Sorry, had to re-post this due to broken link. From earlier this morning (what a nice day it has been since!)

The market is still trying to come to terms with what meeting the primary endpoints of the phase 3 aGVHD trial means ... personally the real value is in the validation of MSB's MSC platform which has the same mechanism of action across all programs (aGVHD, RA, Chronic lower back paid, Chronic Heart Failure).

But I personally believe the watershed moment for MSB is yet to come,  and it is on its way later this year... I am confident of a partnership for the CHF program with a positive NIH sponsored CHF LVAD trial being the catalyst (study start date July 2015, read-out due Mar/Apr18).

Not to cast shade over the recent aGVHD phase 3 trial success, but the commercial implications for MSB will be dwarfed by a successful phsae 2b CHF (LVAD) clinical trial (which has been flagged as a potential catalyst to accelerated FDA approval through RMAT designation received in Dec17).

I am very confident that the phase 2b trial will be positive, not because the fortune cookie told me so... but a little research and reaffirmation from the aGVHD trial outcome (remarkably consistent with the EAP program) gives me confidence that a similar outcome will be achieved in the 2b trial as was achieved in the 2a trial back in 2013.

Here is a summary of the 2a trial:
- 30 patient, 2:1 randomised trial using 25 million MPCs.
- No safety events in the 90 day trial period, or the 12 month follow up (as we are now accustomed to with MSB's trials, but shouldn't be taken for granted);
- At 90 days, 50% of treated patients were able to be weened off their LVAD support for 30 minutes compared to 20% in the control group;
- At 90 days, 0% mortality in the treated group. vs 30% mortality rate (n=3)
- At 12 months, 85% of treated patients were able to be temporarily weened off their LVAD once or more compared to 40% in the control group.

Link to publication of trial results of 2a clinical trial:
2014 Phase 2a LVAD trial publication

On the back of the above phase 2 clinical trial (safety was the objective), the NIH sponsored a phase 2b trial with:
- 159 patients to test for safety and efficacy;
- 150 million MPCs (6 times more than the 2a trial)
- primary outcomes are:
i) successfully ween patient off their LVAD for 30 minutes (6 months); and
ii) number of adverse effects (12 months).

Phase 2b clinical trial
Phase 2b Clinical trial program