In addition to this -I believe that Sarepta’s current FDA approved DMD drug (Exondys-51) and their new pipeline drug (SRP-5051) are only appropriate with patients who are amenable to Exon-51 skipping (using an antisense oligonucleotide to take up that missing Exon space between Exon-49 and Exon-51).
- Link for Exon-51 skipping suitability percentage:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5675502/#!po=0.694444
I haven’t seen anything to date that suggests that LTBP4 is only implicated in muscle function in a certain percentage of individuals - if anyone has please reply with the link.
Also an important consideration to Mason’s point is that although Exondys-51 and SRP-5051 are ‘closer to the source’ of DMD in that it’s gene therapy, this doesn’t diminish ATL1102’s value in modulating growth factor proteins such at LTBP4 that regulate muscle function and elasticity.
In many ways, you could consider the ‘lower down the biological chain’ function that ATL1102 performs as beneficial commercially and therapeutically - Sarepta’s approach may be effective but will only work with those who are suited to Exon-51 gene therapy, whereas ATL1102’s function may allow it to be a more general therapy not constrained by specific DMD Exon activity.
I’d like to note that I also cannot find any efficacy data for Exondys-51 for grip strength or other functional outcomes that AT1102 trials have measured against, simply data showing increases in dystrophin levels:
https://www.exondys51hcp.com/about-exondys-51/clinical-data
Again, if anyone can find data showing that Exon-51 skipping has resulted in improvement in functional outcomes like the PUL 2.0 test, then please let me know.
ANP have maintained that they intend to be supplementary in the DMD therapeutics market rather than as direct competitors to companies like Sarepta, Santhera etc (see page 9 of the following ANP presentation:
http://www.antisense.com.au/wp-cont...SX-19_23-Jan_Updated-Company-Presentation.pdf)
I haven’t done much research into other DMD’s therapies in Santhera, Capricor and Pfizer’s domains, but I’d welcome anyone to also assist in looking into these and their efficacies. I believe Santhera is targetting respiratory issues specifically, whereas Pfizer and Capricor focus on muscle regenerative therapies.
My reasonably shallow understanding of LTBP4 suggests that ATL1102 has the capacity to target the muscle regenerative aspect of DMD therapeutics as well as their initial inflammation focus.
As always, I’d love to hear more on this from anyone who’s done their own research/has expertise in this field.
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