Ann: OLE Study update reveals impressive Survival Data, page-121

Let me see if i can help a little.

HISTORY LESSON (sorry for ya'll who know this stuff... I have been accused of being repetitive so just to repeat - soz)

We started with 12 patients, all with MND for more than 1 year before joining and they were at various levels of progression and various speeds of disease progress. All patients were successful in completing Phase 1/2 in the 2 cohorts.

Cohort 1 had 7 patients > 1 moved to cohort 2, with 1 additional patient brought on to Cohort 1 to fill the "vacated" seat on the trial. ....... Cohort 1 had dosing of 2mg then 6mg regimen

Cohort 2 had 6 patients > note the 1 from cohort 1 ....... Cohort 2 had dosing of 4mg then 10mg regimen

NOTE, while all patients had various other tests including ALSFRS-R , only 3 of the 12 patients agreed to have NFL tests undertaken as they are extremely invasive. (wow, that's brave)

Compassionate use program was 6mg regimen for all comers and was always going to happen however they (the trial team) then decided it would be a great idea to also go to an Open Label Extension Trial to enable the company to get more data as compassionate use precludes any data being collected or tests being undertaken for use by the company.
ALL patients went on the compassionate use program and ALL patients agreed to enter the OLE trail if they qualified.

During the Compassionate use program, sadly 1 patient died. It is not known by the company what caused the death. Nor is it known if they stayed on MPL dosing to the last days. But, whatever the reason, we can just assume they did take the right dosing of tablets to the last day as that is basically the worst case (but may not have been the actual case) for the company.

THen, on entry assessments to the OLE trial, unfortunately 1 NSW based patient has increased liver enzymes so could not be included. NOTE: The increased liver enzymes had no relationship to MPL.

The way I read the graph below:
The single survival step down in purple line (Monepantel) at the top of the graph shows 1 patient passing away just after their 11 months on MPL and all other patients (11) being alive, hence the flat line with only 1 step down.

It is important to note, the graph is showing all 11 other patients being alive at the time of the data being collected. We don't know if any have subsequently passed away as they are now on a 2 monthly check in. We should reasonably expect that sadly more patients will pass away but hopefully not for a long time and when it does happen, we hope they have a better quality and longer life than would otherwise be the case without MPL.

From memory the blue, green,baby poo, orange represents the PRO-ACT "like" patients (30 per patient) were matched to each of our 12 patient (360 in total) - "Like" meaning matched for disease features and geno type (like age, gender etc).

The trajectory in blue, green,baby poo, orange is as compared to the PRO-ACT database (not the actual patients) and the dotted lines (top to bottom) show the period to which the individual patients have been on MPL.

The key observation is that, without MPL and at the time of the graph being compiled, basically every patient (exception of the one that passed away) has around 50% - 25% chance of being alive...

You might ask why the PRO-ACT is in 4 lines - it simply shows different data streams from the pro-act database.
Of the 30 matched PRO-ACT patients to each of our patients, the data was collected at different points / stages so hence the 4 channels.
That said, no matter how you slide and dice it, the survival of the MPL patients is remarkable when compared to similar patients within the PRO-ACT database.



If i am incorrect with anything, please yell and i hope that helped someone.