Thanks for the reminder, Wayne. The scientific literature points...

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Uboy

4,680 Posts.

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25/05/23

12:21

Post #: 67957921
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Originally posted by waynesworld ↑

Now it gets really interesting. The news of more time required by the FDA for Sarepta's DMD gene therapy and the likely accelerated approval of SRP-9001 initially for 4-5 years old, sets the hounds running on why the gene therapy might work on the youngest cohort but there are question marks all over older patients - in fact its likely ineffective for older patients. Long time readers of the ANP threads could probably provide an extremely plausible explanation.

Discussions go back to October 2021, Post #: 56951088 and a blog account brought to our notice by @Uboy;
Reference is made in the blog to the accumulation of extracellular matrix around the muscle fibres such that after a period of time the loss of muscle function is linked to fibrosis rather than the original mutation in the dystrophin gene.

Why does ATL1102 look ever more likely to be an effective therapeutic either as a monotherapy or in combination therapy? Drawing on my comments from that post, the more we discover about ATL1102 the more it looks like an effective treatment for DMD not simply for its role in modulating lymphocytes and managing the inflammatory process but because of the downstream modulation of several proteins critical to fibrosis.

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Thanks for the reminder, Wayne.

The scientific literature points to a lot of evidence that fibrosis plays a big role the decline of DMD boys after the age of 5.

The additional dystrophin is not material when the "accumulation of extracellular matrix" sets in...causing muscles to waste and accumulate fatty tissue.

If the fibrosis can be controlled i.e. like the MRI showed with ATL Phase 2 trial results...then the additional dystrophin may have a tangible impact?

Shame ANP did not move faster on Monkey TOX study as soon as they knew this was non-negotiable with FDA.

I suspect TOX study is the critical milestone for any partnership opportunities.

At EV of ~$23m...ANP is a bargain, BUT we have been here before

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Last edited by Uboy: 25/05/23

ANP Price at posting: 6.3¢ Sentiment: Buy Disclosure: Held
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