Ann: P3 Trial of MSB's Cell Therapy in CHF Completes Recruitment, page-17

Lets try and put this in some perspective. Looking back at previous Revascor clinical trial results we can see that no hospitalisations was a phenomenal, almost unbelievable achievement, compared to the control group who experienced HF MACE events of 27% over 15 months and 33% over 24 months. If people wanted to be cynical you could observe that it was a 60 patient trial split into 3 dosage levels and a control..but it is still a  statistically relevant result in my view. I should also point out that Dr Perin was disclosed as receiving consultancy fees for his work from Mesoblast.

https://www.tctmd.com/news/small-heart-failure-studies-show-positive-trends-cell-therapy

Now lets remember that we have now had an interim  futility analysis of 270 patients performed by the Revascor phase 3 clinical trials Independent Monitoring Committee which reported the trial was thus far successful in moving towards its primary endpoints...then we learned that :

https://globenewswire.com/news-release/2018/04/18/1480696/0/en/Mesoblast-Clinical-Program-Update-for-MPC-150-IM-in-Patients-With-Chronic-Heart-Failure.html

• In April 2018, the DMC for the Phase 3 trial undertook a scheduled review of available data from 465 randomized patients, including the primary and secondary endpoints of HF-MACE and TCEs, and all safety data. The DMC recommended continuation of the trial without modification. Enrollment of this Phase 3 trial is expected to be completed by the end of 2018.

I would ask the statisticians amongst you to try and calculate the probability of this trial not now being successful . 

If you have had the phase 3 clinical results successfully monitored over such a long period which include 465 out of a total trial size of 600 patients and you also know the published results of the Phase 2 results over 36 months which show no hospitalisations ..... surely the prospects of failing to reach the primary endpoint has to be very small now? Where am I going wrong in my analysis ?

 

Mesoblast reported in their slide presentation to JP Morgan in January 2018- Slide 22

 250K – 300K patients/yr suffer from advanced systolic HF (NYHA Class IV)1 50k patients/yr have end-stage heart failure Despite optimal medical therapy, 1-year mortality exceeds 50% in end-stage

heart failure patients1

We are talking here about a multi billion dollar revenue opportunity for Mesoblast. At what point will the FDA weigh  the statistical probability of this trial successfully reaching its clinical endpoints, against the interest of 25,000 Americans dying each year of stage 4 heart disease ! 

 I agree with a previous poster who  commented that the information released today is not in itself a major surprise....but from my perspective, the only surprise is that the market has yet to react to the likelihood that this trial will be meet its primary endpoints.  All biotech stocks must be classed as high risk...but what an opportunity !!! GLA DYOR