MSB Trading 2021 - paradigm shift, page-8579

Hey Team,

No deep analysis to do because it's all been done, just some timelining as it is excitingly coming up to crunch time and I like where the company is sitting.

In the short term, we have CHF and CLBP feedback and the CHF presentation to look forward to. We were first told that MSB had filed for a Type C meeting with the FDA on the 1st of July.



https://www.fda.gov/media/109951/download

As per the above guidance from the FDA regarding meeting times (which I'm sure we're all familiar with by now), meeting minutes should be available within 105 days of receipt of the meeting request. This means we should have received feedback from the FDA by the 14th October. I was surprised to see that they were still awaiting feedback in the quarterly. Does this mean there is perhaps a dialogue ongoing with the FDA? If so, that's incredibly encouraging. Otherwise, MSB is just keeping it confidential until they can announce the next steps alongside the FDA response.

Similarly, we were first told that MSB had filed for a type C meeting for CHF on the 31st of July. 105 days from this time is the 13th of November, so the minutes from that meeting are also due within the next two weeks.

The heart trial data to be presented at AHA is going to be great - I would love to understand the details of the biomarkers etc. more for the treatment. Regardless, it is a guaranteed positive catalyst, MSB wouldn't promote their data by applying to present it (and paying the application fee) if it wasn't going to create shareholder value.

I am also very confident about our investigator-led trial for Crohn's disease and ulcerative colitis. Alofisel has been commercial for many years now and its success is well known. The PI Amy Lightner and MSB will be well aware of this and the opportunity surrounding MSC therapies in this indication, and now have a third party leading a clinical trial for them at their own cost. This will almost certainly be another imminent positive catalyst.

So - short-term, things are looking great.

A lot of the medium-term catalysts hinge on the OTAT meeting. I am pretty confident with this, as the FDA had the bold (but ultimately I believe the appropriate considering the potential batch-batch consistency concerns) response to a 9-1 ODAC vote in the form of a CRL. Within this CRL, they raised three issues with the regulatory development of Remestemcel-L in pSR-GvHD to date.

1. They had concerns over the single-arm trial design
2. They had concerns over the proposed theoretical MoA
3. They had concerns over the corresponding potency assays

And on the 30th of April we were told this gem:

In a conference call, it was stated that the FDA had initially agreed to the trial design, and it seems that this concern has since been resolved. So MSB worked within FDA guidance to develop their potency assays and have since had feedback that the assays appear to be sufficient.

This gives us a lot of confidence for OTAT, which will unlock the reapplication for the BLA for pSR-GvHD, the initiation of the 2nd trial for ARDS and the potential closing of the Novartis deal. If OTAT is green, I doubt the FDA will provide guidance that suggests reapplying and then turn down a program that received a 9-1 in favour from their advisory committee twice. This positive OTAT should lead to two commercial products by EoY 22 (pSR-GvHD and ARDS).

On Novartis, I suspect part of their analysis is looking at the biomarkers for efficacy in covid-19 ARDS and comparing them with the biomarkers of non-Covid-19 ARDS to see if the biomarkers (and thus likely efficacy) are comparable.

Long-term: Stem cell therapies are too impactful to not get there, and MSB holds all the IP cards and are currently a mile out in front. A matter of when not if.

I reckon we'll end this month richer than we started, but I'm happy to hold off getting my Tesla until CY22 instead.

Gang Gang