Fasten your seatbelts!

 

Fasten your seatbelts!

You’d be hard pressed to find a biotech company anywhere in the world with a more exciting calendar than Mesoblast in the next two months. 

Front and center is the lead product candidate Remestemcel-L, or Ryoncil. 

Not later than September 30th, the FDA will decide whether to approve Ryoncil for the treatment of pediatric patients suffering Steroid Refectory Graft Versus Host Disease (GVHD).  

Approval will see Mesoblast move from being a speculative biotech, to becoming an investment grade company able to generate sustainable cashflows to support its broad product pipeline. I don’t even want to think about it not being approved… 

That decision will be massive news for Mesoblast, but there’s a possibility that there could be even bigger news before that FDA decision. 

The same MSB product, Remestemcel-L, is approaching the latter stages of a 300 patient, phase 3 trial into Covid-19 ARDS (Acute Respiratory Distress Syndrome) and an interim look at the data for the first 90 patients is due early September.   

Many think the trial will be stopped at this juncture due to overwhelming efficacy, if this turns out to be the case there’s a good possibility that the event could make global headlines as there are currently no good treatment options for this desperately ill cohort. Though I’m happy to be wrong, I don’t share the view that the trial will stop this early in the trial.   

The standard of care for intubated patients has been refined since the results came in for an earlier study (EAP) in April, the result of that is that significantly more intubated patients are surviving today than they were at the beginning of the C19 crisis. How many more? It’s difficult to find reliable data to know for sure. 

Let’s not forget that the interim look is at the first 90 patients enrolled in the trial, 45 of those will be in the placebo group, 45 in the Remestemcel group.  Are 45 patients enough to provide a statistically significant efficacy outcome? I’m not sure. 

A continuation of the trial beyond the first 90 patients would still be a significant win for Mesoblast as a larger sample size will shore up the data for the many “secondary outcome measures” for this trial.  As long as MSB gain approval, does it matter if it takes a little longer? 

To give you an idea of the staggering number of secondary measures I’ve attached a link to the clinical trial site where you can see for yourself: 

 

https://clinicaltrials.gov/ct2/show/study/NCT04371393?term=mesoblast&recrs=ab&draw=2&rank=2 

 

This important extra data could prove vital to the design of future trials or even “label extensions” into other causes of ARDS down the track. A continuation of the trial to the second or third interim look is the outcome I’m expecting. 

If we assume the trial is told to continue, the last of the 300 patients should have been dosed by the last week in September.  30-days after the final patient is dosed, attention will turn to crunching the data from the trial. Ordinarily that task can take months, but given the gravity of the global pandemic, I believe significantly more resources will be thrown at the task meaning it could be done in weeks, not months. 

 

Moving on. 

Paraphrasing the old “Demtel” TV advertisements from years ago – “But wait! There’s more! 

As if the FDA decision and Covid ARDS trial were not enough, there’s the been little talk recently of MSB’s other potential blockbuster product candidates to treat lower back pain and heart failure. 

The two recently completed phase 3 trials are due to have data read outs in the next month or two. Originally these were due mid-year but it appears to have been delays due to Covid 19 and the company’s near-term focus on Remestemcel-L. 

Disregarding any potential approvals for Remestemcel, approval of Revascor for heart failure and MPC-06-ID for lower back pain would be transformational for Mesoblast. These products are targeting areas of huge unmet need. 

As with any clinical trial, there’s no way of knowing if the trial will be successful. Depending on where you look for your data, generally around 50% of phase 3 candidates are approved by the FDA, making it difficult to make an accurate assessment of MSB’s products.  

There are clues though. 

Demiurge Technologies is a Swiss based company that uses Artificial Intelligence to crunch the numbers of various clinical trails to predict the outcome of those trails. A link is provided below. 

 

https://www.demiurge.technology/ 

 

To validate their claims, the company publishes its predictions and the outcomes or the trials, usually on Twitter. The company claims to have achieved 84% accuracy in its predictions in 2020. 

MSB holders will no doubt be interested to know what Demiurge Technologies have to say about Mesoblast’s Revascor and MPC-06-ID. You will see their predictions below. 

 Success is the prediction for both products ( I can’t find if they have predicted the outcome for Remestemcel-L). Both indications are for multi-billion-dollar market opportunities. I’ll leave it up to you to decide if these predictions are believable or not, but they are interesting none the less. 

In conclusion, there’s never been a more exciting time to be involved in Mesoblast. Whether you are a large or small Mesoblast shareholder, you’d better fasten your seatbelts because these next few weeks are going to be a wild ride, one way or the other 🙂 

 

Author – Ratfink.