ASOs, DMD and CPPs, page-8

I’m pretty sure Sarepta would be aware of what PYC is doing. Firstly, as Sarepta is the leading company in DMD therapeutics and has multiple DMD drug candidates in its pipeline, it is its business to know what else is happening in the space. Also, there is the fact that PYC’s FPP-conjugated PMO experiments in mouse models of DMD and spinal muscular atrophy (SMA) were performed in collaboration with Murdoch University. In a separate collaboration with Sarepta, Murdoch University is establishing the Sarepta Translational Laboratory. The laboratory at Murdoch is said to be exploring the applicability of Sarepta’s PMO technology for disease targets beyond DMD, such as in SMA.

As stated in my post at the head of this thread, Sarepta’s FDA approved drug eteplirsen (Exondys 51) for patients with DMD gene mutation amenable to exon 51 skipping, is based on Sarepta’s first generation PMO chemistry. Eteplirsen was approved despite demonstrating only low impact on muscle dystrophin levels, especially in heart muscle. Sarepta is now in Phase 1/2 testing of a second-generation PPMO version of eteplirsen. In preclinical mouse and monkey models, the PPMO version resulted in substantially higher levels of exon skipping and expression of dystrophin protein in muscle. More detailed results can be found in the following company presentation which has almost 300 slides. The relevant section, PPMO for Duchenne Muscular Dystrophy, is right at the end of the presentation. In my view, these are the delivery results which PYC will need to better, rather than those of eteplirsen.

To date, Sarepta hasn’t announced any clinical programs for development of a PPMO therapy for SMA which might rival Biogen’s Spinraza, but it does state in a presentation released this month that PPMO has the potential to address multiple neuromuscular diseases beyond DMD and then, there is that collaborative work with Murdoch University.