The "story" is now easy to understand - after 20 years of toil and shareholder frustration
- ATL1102 is addressing a key pathway in inflammation
- Not just for DMD - but also other disease indications like LGMD, Asthma, MS etc.
- CD49d as a target has been validated
- PUL 2.0 and MRI results from DMD Phase 2A provide exceptional functional data
- The PUL2.0 data, if replicated in EMA Phase 2B would be end point for EMA/FDA early approval
- Lymphocyte and T-cell modulation data presented by ANP show T-cells benefit from ATL1102
- After the 6 month ATL1102 trial - the DMD boys condition reverted back to baseline i.e. T-cell overaction
- Controlling inflammation in DMD is a key player to the boys functional outcomes
- Evidence shows changes at cellular level
- Treats 100% of boys ...compared to Sarepta's Exondys that only treats just 13% Exon skipping boys
- PRV voucher if ANP receives approval is worth ~ $100m
- ...
-...
Sarepta's failed gene therapy results highlight that extra Dystrophin is not the panacea for functional outcomes in DMD - especially after 5 years of age when fibrosis and inflammation sets in.
ATL1102 seems to help with 'matrix deposition' in the muscles - something additional dystrophin cannot help with.
FA wise the "story" could not get better.
I would be amazed if Sarepta didn't get involved with ATL1102 - and take over the US P2B.
Let's see - assuming as Sammy says... the Board don't get trigger happy and sell the farm cheap.
Exciting times.
