The Rome Cohort

There is much to understand and learn from the recent announcement in regards to the comparison with a matched natural history control. The first thing I needed to do was appreciate the point of difference with the initial reporting of the PUL 2.0 results from the ATL1102 Phase II study.

Of course, the results from the Phase II looked at the change from baseline to week 24 to for each of the nine boys in the study. We knew the results were encouraging because historical studies reported a progressive and continuous deterioration in physical function in non-ambulant patients with DMD over time.

The new data analysis provides the means for quantification. We now have the opportunity to compare the mean change for the nine boys against the Rome Cohort. In the words of the announcement, the matched comparison elevates both the quality and clinical importance of the efficacy outcomes.

The article Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data, published online in June 2018 provides a table setting out PUL 2.0 scores in ambulant and non ambulant patients at baseline, 12 and 24 months. The table provides even more contextual clarity.

Now, even more interesting. If ANP shareholders have the opportunity to appreciate the clinical significance of the Phase II study, how much more will biotech/pharma be able to prepare their own own analytical observations. Will they come up with the observation that the level of improvement in mean PUL 2.0 is the best yet seen in non ambulant DMD boys. Further, what are the implications for the therapeutic use of ATL1102 over a longer term in non ambulant boys and for use in ambulant boys in regards to lower limb function.

When we review the matched results in the PUL 2.0 data, bearing in mind the MRI and other data, its hard not to conclude that the therapeutic benefit of ATL1102 in controlling inflammation is as significant as restoring minute levels of dystrophin.