Alright team, what an awesome day.
This was a huge derisk in a single announcement. This had one of the biggest question marks around it. The results of the ARDS trial weighed heavy on a lot.
Let's acknowledge how big a deal this is, a huge (75% reduction in mortality of Rem-l+Dex relative to Dex alone) (43% reduction in mortality in their own right) deal. That is a 75% improvement on the standard of care for a disease that is killing >800 people in the US per day, reducing mortality to just 12% from 43%. This clearly shows that Rem-L is an effective treatment against Covid-19 ARDS, which is clearly still a massive unmet medical need.
Not only does it show this, but the FDA now has:
- RCT data for safety
- RCT data for biomarkers (ie. mechanism of action)
- RCT data for a strong signal of efficacy (26% vs 42%, p=0.048 alone; 14% vs 45% in combination with Dex, p=0.006) for a cytokine-syndrome-like disease
Why do I point this out? Rem-L is also under review for regulatory pathways to market in GvHD. This gives gold standard (RCT) data on all of the above for Remestemcel-L, this data - as SI alluded to in the CC - will aid in FDA discussions regarding the best path forward for GvHD. The FDA could have even agreed to hold out to review this data prior to making a binding decision on GvHD - that would explain the delays. It's not the same disease, but you know, we have some pretty clear efficacy (shown by a met primary endpoint) to show we treat GvHD.
For people who aren't too familiar with stats and p values, that means that there was a 48/1000 and 6/1000 chance that this data could have happened by chance, respectively. I'm happy with those odds, as will most people - including the FDA as p<0.05 is the standard for significance. I saw one dude be like "Yea but they only just got under .05, this doesn't give me confidence" - I die a little reading this. You are saying that less than a 50/1000 chance of this happening by chance is insufficient evidence for you to back this result? What about the 6/1000 as a combination therapy? How many times do you need to run into a concrete wall before you finally believe it's a dumb idea? smh Also, addressing EUA, this is definitely on the cards - but thank god MSB seem to be becoming more conservative in their estimates and saying 'another trial' right off the bat.
The 4th point is demonstrated by the massive improvement on top of the current standard of care - Dexamethasone. Who knows, but we certainly have a worthy application.
So looking forward, things we know:
- Surg Centre, no doubt under an NDA to allow for a complete review of the internal functioning of the company, put down US$110 million at 2.30. (Means there are probably no shady dealings considering they appointed a SurgCentre official to the board.)
- Novartis held out to review data for ARDS before deciding on the next steps
- Each of the pursued indications by the company reflects massive market opportunities.
Things I consider almost certain:
- Gruenthal European partnership money and trial initiation for CLBP
- Novartis partnership money and trial for ARDS (After being told that the trial failed, what results could you possibly hope for other than what we got?A statistically significant lasting reduction in mortality up to 60 days that synergistically works with the SoC and a reduction in discharge time. Considering they stayed on following the trial, I don't believe in the face of this kind of data that Novartis would walk - that would be insane. The CEO of Novartis even gave us a shout out shortly after 1:24: https://www.linkedin.com/posts/vasnarasimhan_leadership-reimaginingmedicine-results-activity-6792709292706295808-T5Ah/?fbclid=IwAR2-ziNWC_0--gMvWtEgmrveilJmemoS25V-lWEdVtcYdMEenIGwvVm4Khk)
- Further SP gain on analysis of secondary markers from ARDS trial (similar to in January with the CHF results)
- Progression of Tasly partnership in China
- Eventual CHF partnership for US/EU commercialisation of Rexlemestrocel
Things I consider moderately risky:
- GvHD commercialisation has been massively derisked for the reasons highlighted above - but everything the FDA did with this one was weird so what's to stop them now. I think more likely to get conditional approval than not but that's just imo based on the new evidence presented by the ARDS RCT.
- CHF requiring a single trial. Coin flip tbh. We certainly have the data to validate it, but FDA wild card makes it too hard to call. Regardless, I strongly believe we will get a lucrative partnership prior to commercialisation.
- CLBP requiring a single trial. Ditto, coin flip but I think CHF is slightly more likely to land the right way up.
- EUA for Covid ARDS. We tick all the boxes, and the pandemic calls for it, but they're not given lightly. Is this the path to approval for the first approved commercial stem cell therapy in the US?
All just my thoughts and not investment advice, I did top up though - following my own thought process.
Gang Gang
(20min delay)