Ann: FDA IND Approval for Phase 3 Study of DMX-200 in FSGS, page-48

Hi BTB,

This is awesome news! How many patients do we need for Interim Analysis again to take to the FDA? It’s n=72 & if you take into account we now have 44/75 actively recruiting, even if not yet recruiting PIs will be identifying suitable patients, until site initiation is completed & the green light given to commence - this isn’t even two patients per trial site in an Orphan Status (Rare disease) currently.

If all come onboard soon it’s roughly <1 patient per site. 3 patients per site is totally do-able to run smoothly if needed, event with multiple drug trials ongoing.

Something else also important is the “Intent to treat” population & that FSGS appears to be more common in black people & also those of Hispanic heritage. So take a look at trial sites, there is a lot of disparity in who gets recruited & benefits of clinical trials, white people suffer as well, but we want a representation of the FSGS sufferers in our trial & that may be interesting to you, as the FDA & World-Wide conferences are very much talking about inclusive clinical trials.

It’s a little different to REMAP-CAP & CLARITY 2.0 in that this Ph3 is Sponsor Lead (Dimerix) & an excellent Clinical Research Contract Organisation that deals with the day to day running of the trial, data collection portals & trial equipment. They are used often by Big Pharma trials also.

There has been a big shift in accommodating clinical trials with CRO’s & COVID-19, not only to support trial sites should they be affected by COVID-19, but also patients as well, to not miss visits (contingency put in place) with Decentralisation, that will not affect our data. A lot has changed in the last 2.5 years! We have been faring well. No reason we won’t on DXB also. It’s a vulnerable population with COVID-19, but so are other clinical areas.

This is great news!