Defense Funding, page-9

Reference has already been made to the DoD 2016 Appropriations Bill which will allow funding to be used for clinical trials for Rett Syndrome. In order to gain some perspective on the question of why-is-it-so when the Army is already funding the trial of Trofinetide in TBI, its important to look at the respective size of pivotal clinical trials.

We have some useful comparisons.

Firstly, we know that Neuren, following discussions with the FDA, proposes to design a single Phase 3 clinical trial for Rett syndrome. It would appear likely, based upon the CEO Address in May 2013, that the Phase 3 trial would be the same size as the Phase 2 trial which could be completed within 1 year. The Phase 2 trial enrolled 56 patients.

What would be the likely size of a Phase 3 trial in TBI? The highly publicised clinical trial of progesterone in TBI provides highly probable answers. There were 2 trials of progesterone in patients with TBI. The PROTECT 111 trial involved 1140 patients and the SYNAPSE trial involved 1195 patients.

It remains to be seen whether military research funds will be granted towards funding a pivotal trial in Rett Syndrome. Nonetheless, the numbers appear compelling if the objective is to expedite the approval of the drug for CNS conditions including TBI.