Ann: ATL1102 Revised Plans accelerate reporting of unblinded data, page-50

Part of a post i was putting together Charmaine has just been going over some of this but looks like we are stepping back into a PH2b trial


If you read below you will see the huge difference in trials, they have only reduced costs because they have cut the study down by one third from the original plan




ial The Phase IIb/III clinical trial is a multicentre, randomised, double-blind, placebo-controlled study to determine the efficacy, safety, and pharmacokinetic profile of ATL1102 (25 mg and 50 mg) administered once weekly by subcutaneous injection for 52 weeks in non-ambulatory participants with DMD, to be conducted as a potentially pivotal (approvable) trial with a follow-on open label extension trial. Participants will be randomised to either 25 mg ATL1102, 50 mg ATL1102 or placebo in a 1:1:1 ratio with stratification by corticosteroid use.


Up to 114 participants are to be enrolled (38 per treatment arm) with 108 participants expected to complete the trial. Additional trial details and timelines are outlined in the investor presentation lodged today with the ASX. As previously announced,






The Phase IIb study aims to enrol and randomize 45 non-ambulant boys with DMD. Following the initial six-month regimen of either placebo, 25 mg or 50 mg once weekly,

participants will be invited into a further six-month open label follow-up treatment period in which all boys will be on active treatment (25 or 50mg). This additional time period will be used to demonstrate longevity of response as well as collect additional safety data and facilitate streamlining and de-risking of a Phase III study, the most expensive phase of the drug’s development (i.e., may reduce the number of dosing arms). It is the Company’s view that if results from the Phase IIb proved to be highly successful, it would then engage with Regulatory Agencies in relation to obtaining an accelerated approval for the unmet medical need of non-ambulant DMD patients.

The Phase IIb/III clinical trial application submitted in Germany (BfArM) is undergoing evaluation.

( note undergoing evaluation ) ????


With the regulatory focus now directed to submission of the Phase IIb trial applications,

( PH2b applicatrions ) ?????

no additional Phase IIb/III trial submissions are planned at this time