Extract from Morgans Report: Event: Collaboration, reg feedback,...

Extract from Morgans Report:

Event: Collaboration, reg feedback, data supporting additional indications ▪ In recent weeks, a number of major catalysts have come to pass including information on new indications and preliminary feedback from the two major regulatory authorities (FDA Type-C / EMA PIP), and an FDA fast-track submission. ▪ Feedback from the authorities was broadly supportive with concessions on FDA’s requirement for usual 12 month tox data (ANP has 6 month data) to 9 month and included the ability to run these studies concurrently in the Ph2b/3 trial provided a draft tox report is submitted before patient dosing exceeds 6 months. ▪ The EMA’s advisory committee feedback suggests the regulator’s interest in seeing the trial design prior to design submission. Our view is that this is most likely a desire to see ambulant (ability to walk) boys being included in protocols which does add risk (to date untested), but also adds a further 50% in addressable market. Analysis: Driven by data ▪ Interest in the story will be driven by regulatory progression and partnering potential, which in turn is driven by data. The depth and breadth of ANP’s clinical/non-clinical data has increased substantially over the past 24 months and with positive data expected from additional animal and human plasma studies to drive a better understanding into the mechanism of action (MOA) and new indications, it’s beginning to look like a compelling package. ▪ A hint in the data suggesting potential use in combination with dystrophin restoration drugs was particularly interesting. Questions around efficacy for these treatments remains, while a number of sources suggest inflammation may play a functional role in the newly produced dystrophin not being able to penetrate the muscles effectively and lowering functional efficacy rates being observed in these patients.

So AMBULANT BOYS are going to be included....

Interesting the report saying " a number of sources suggest " ..

Sources??? I would love to know which source...Is it Prof Voit ??

Such important information" Newly produced Dystrophin not being able to penetrate the muscle effectively and lowering functional efficacy rates..

Love this stock..so much potential..

Good luck all