From the patients and parents perspective it doesn't matter whether the therapy is exon-skipping or gene therapy, whether its steroids or other anti inflammatory treatments. What actually matters is whether the medication is effective in improving quality of life and increasing life span without nasty side effects. Outside of a cure, its the ability to be self sufficient in the little things of life and gain enjoyment from daily activities that we all take for granted. So, PUL 2.0 is a proxy measurement for all those things.
Just as ANP can compare the mean change for the nine boys in their Phase II trial against the Rome Cohort, so too can Pharma/Biotech compare their drugs performance against ATL1102. There is a presentation Performance of Upper Limb Measure: PUL 2.0 made to the PPMD Conference in June 2016. Just for some context, before PUL 2.0 there was PUL 1.2; simply put PUL 2.0 is a refinement of PUL 1.2 to improve PUL's measurement properties and ease and clarity of testing. I wanted to highlight one slide titled Studies Using PUL. Its a list of Pharma/Biotech including Sarepta who use either PUL 1.2 or PUL 2.0 in studies. Remember this is from a presentation in 2016 so it would be interesting to see an update.
I mention the slide because it highlights the importance of PUL as an endpoint. I suspect that the announcement by ANP of comparison with a matched natural history control is of far greater importance than at first glance: the audience includes shareholders but will speak more loudly to advocacy groups, clinicians and pharmaceutical and biotech companies operating in the space.
It also begs the following question. What would be the PUL 2.0 results once an optimal dose for ATL1102 is established and what would be the PUL 2.0 results in combination therapy using ATL1102 and exon skipping or gene therapy drug?
ANP Price at posting:
10.5¢ Sentiment: Buy Disclosure: Held
