The upcoming trial need to show much better data for ATL1102 in...

The upcoming trial need to show much better data for ATL1102 in DMD or it will flop.

The data from the poster presentation doesn't look that great at all.

• Safety data - Acceptable so good start
• Clinical Data/Clinical Improvement - Minimal. Small percentage change in function/symptom improvement, no indication any significant PFS
• Radiographic data - Looks okay but doesn't mean much without significant clinical improvement
• Lung function - Again, looks okay but doesn't mean much without significant clinical improvement
• Patient-Reported Outcomes/QoL measurement - PROs are probably the best thing on the poster in my opinion but these parameters are subjective and almost meaningless without clinical data

A drug's commercial success hinges on it's clinical data. Even if there is some symptom improvement, it would have to be significant to be attracting. The dose is 25mg weekly for 24 weeks. So that's 6 months treatment. There is no way a drug with minimal symptom improvement will get anywhere near $1000 a dose. A few hundred dollars is a stretch. It will end up being a private market for parents who are truly desperate for something for their child. For ATL1102 to be a success, it will have to be $100s to $1000s per DOSE and most people won't pay for minimal improvement. At this price range, there have been so many cancer and neurological drugs with way better data that haven't taken off in the US and not subsidised in Australia.

However, assuming that there is clinical improvement, patent is until 2029 in US, unknown elsewhere in the world. Not sure if there is much time to commercialise ATL1102 even if it was clinical signficant.

So yeah, as someone who does clinical trials and health economics for a living, ATL1102 doesn't seem to be commercially viable at the moment.