In another thread,
@aatisket suggested that case studies of aGVHD would be of interest to MNK. This got me thinking... what really would be of interest to MNK and what does MSB look like from their perspective?
Firstly I don't think MSB have a shortage of 'case studies' if they wanted to get these, as their expanded access program treated 241 patients with aGVHD (with a 65% response rate).
82% of those responders survived 100 days, which means overall 53% survival rate or 128 positive case studies.
Having said that, I don't think putting together individual case studies would necessarily be what would get MNK over the line. What I believe matters to MNK in their due diligence process is that the MSCs are safe (tick) and their method of action is demonstrated to be effective (see below).
So in light of this, I believe that on top of the trial data from the aGVHDand lower back pain programs - the on-going results of the CHF and RA programs would also be of interest to MNK - as they all rely on the same method of action (inflammation and tissue repair).
With this train of thought ...here is what MNK would have known when they announced their intention to partner, and what they know now just over 4 months after that announcement:
At time of announcement (Dec16):
- Jan15 Lower back pain phase 2 trial positive (24 month), Phase 3 360 patient trial initiated
- Feb16 Temcell launches in Japan
- Feb16 MSC 100-IV expanded access program for 241 patients has a 65% response rate
- Nov16 MSC100-IV passes interim futility analysis
- Dec16 21st Century Cures Act enacted
- Dec16 NIH fund trial on cancer patients
- Dec16 Lower back pain phase 3 trial expected to complete enrolment in 2017 (12 and 24 month read-outs). Top-line results expected in 2018.
After announcement:
- Feb17; RA phase 2 with 9 month durable results
- Mar17; MSC 100-IV is granted FDA fast track designation
- Apr 17; FDA clears heart trial funded by Boston Children's hospital
- Apr17; CHF passes interim futility
IMO the significance of the announcements in 2017 cannot be underestimated in the discussions with MNK. I believe MNK's due diligence process would be significantly influenced by these very positive announcements supporting the MSC's underlying mechanism of action.
Specific to the aGVHD program, the fast track designation would have been a BIG tick, however the RA program showing 9 month durable results and the CHF program passing an interim futility analysis, as well as MSCs being endorsed by the Boston Children's hospital - all build on the underlying method of action that MSB are trying to bring to market.
In an attempt to step into the shoe's of MNK, I also found it insightful to consider MNK's capital outlay if they chose to partner for aGVHD and chronic lower back pain .. because IMO
it would be relatively low.
I say this because typically programs are partnered PRIOR to phase 3 trials (as this is the most expensive stage).
However both the aGVHD program and the chronic lower back pain program are
nearing the end of their phase 3 enrolment. So they won't need to incur significant up-front costs to fund the set-up and enrolment of the phase 3 trials ... MSB already funded most of it!
So if MNK partner, they would be looking at minimal upfront capital outlay to continue these phase 3 programs... and they would not be expected to incur significant cash outlays until the marketing, sales and distribution phase.
This would be a key consideration and something that would significantly act in favour of MSB during discussions.
Would be good to get people's views on my above thoughts (positive and negative views are welcome).
(20min delay)