Hi Techinvestor – long time no see. Play devils advocate for me...

Hi Techinvestor – long time no see. Play devils advocate for me for the GVHD resubmission.

According to MSB:

“Long-term survival results were received for remestemcel-L from Mesoblast’s pivotal Phase 3 clinical trial (GVHD-001) in children with SR-aGVHD. The results showed durable survival through 4 years of follow-up. These new long-term survival data are a key component of the Company’s BLA resubmission to the FDA.”

Meanwhile at the Ad Com meeting the FDA stated in black and white that they were not interested in the survival outcomes from study 001:

“In addition, as described in the 2018 FDA guidance document for clinical trial endpoints, time-to- event measures such as overall survival are difficult to interpret in single-1 arm trials, and Study 001 was not designed to detect differences in survival. And therefore, survival endpoints will not be discussed further by FDA.” PM transcript – page 65.

So the one thing that the FDA have said they are not interested in discussing further is what MSB are submitting as the key component to their resubmission.

The CRL suggests “at least” one further RCT is required. “At least” meaning possibly two. But how could two more trials be required if the FDA has accepted that the single arm trial was “adequate and well-controlled”. Well it wouldn’t; because it hasn’t.

So my reading is MSB are starting from scratch. No amount of post-hoc survival analyses of trials that either failed (the early Osiris trials), the EAP protocols that are confounded by patients receiving other treatments or trials that are not deemed to be adequate and well controlled (the single arm trial) can address this basic problem caused from the single arm study having the unacceptable null hypothesis.

But there must be more to it surely. So what exactly is the argument that this its not doomed like all the other ASX microcaps that think they can battle things out with the FDA.