Ann: US FDA Feedback on Type C Meeting for ATL1102 in the US, page-131

The best part of this report should be the following:

Regulators’ expectations appear to be aligned. While final feedback from the EMA is still pending, the feedback that ANP has received to date indicates that the FDA and EMA both have similar expectations regarding the key features that should be included in the design of a pivotal trial of ATL1102 in non-ambulant DMD patients, including the patient population, treatment duration and primary endpoint. The key difference appears to be the FDA’s requirement for a 9-month animal toxicology study (est. cost $1-2m).

The fact that the FDA would allow dosing of patients to commence before the 9-month monkey study is complete (providing a draft study report is submitted before the duration of dosing exceeds 6 months) may allow the initiation of the monkey study to also be deferred until after the topline data readout; given that by then safety data would be available from 12-month dosing of human study subjects it is possible the monkey study may no longer be required (subject to FDA feedback). If the results of the European Phase IIb trial are positive, ANP might consider applying for US approval based on the European trial data without conducting a separate US pivotal trial, subject to the feasibility of such a route.