I wanted to go back to the link provided by George in relation to cTAP, the Collaborative Trajectory Analysis Project re: study looking at natural history data compared to placebo arm data. The cTAP study was released in early September 2020 and results indicated that real-world and natural history data could augment or replace placebo controls in DMD clinical trials. Coincidentally, ANP released an announcement on 2 Oct 2020 that PUL2.0 data from its clinical trial showed a statistically significant mean improvement compared to matched natural history.
What to make of it? Its just another dot point that makes ATL1102 such a compelling drug candidate in DMD. In the final analysis, drug approval will come off the back of meeting endpoints. The Duchenne community have suffered a disheartening setback with the recent news that edasalonexent met neither its primary or secondary endpoints. Eyes are on ATL1102. Its not another corticosteroid replacement but has a unique mechanism of action validated by a seminal scientific paper, clinical data and now matched natural history. For advocacy groups, its appeared on American radar at the same time that the edasalonexent signal has been lost. Australian investors may not have picked up on the story but in the rest-of-the-world its a compelling hope that has come into view at the most opportune time.
ANP Price at posting:
11.0¢ Sentiment: Buy Disclosure: Held
