A beautiful partnership

We know the cargo type. We don't know the disease indication. We know development is at an advanced stage. We don't know whose cargo is being evaluated for delivery.

We also know that Sarepta are the leaders in the PMO space which is the subset of ASOs which are highly amenable to intracellular delivery by CPPs. Sarepta are also on the cusp of taking CPPs into the clinic. Clearly, in the hypothetical case of Sarepta, Phylogica would need to demonstrate that it can deliver more of the PMO cargo with specificity, efficiency and safety than they can using their PPMO technology.

First thoughts would indicate DMD is the disease indication. Phylogica have "done this before with successful outcomes". in the case of Sarepta, however, they are already committed to driving forward with PPMO technology in a Phase 1 trial in DMD.

It is interesting that the AGM presentation for Phylogica highlights a comment by Doug Ingram that if the PPMO tech works, Sarepta would move beyond DMD into other therapeutic areas. Other therapeutic areas would include other rare neuromuscular diseases. Based upon an article Rare Diseases and the power of precision medicine published December 2017, Professor Wilton and his team at Murdoch are applying ASO/PMO drugs to treat neuromuscular diseases including cystic fibrosis, multiple sclerosis and spinal muscular atrophy. Could the disease indication be other neuromuscular diseases rather than DMD?

The time horizons for Sarepta and Phylogica seem to fit. For starters, Ingram makes it clear that DMD is the immediate priority so future clinical programs in other indications are somewhere in the future. Looking at slide 5 from the AGM presentation, Phylogica expect to see 'a molecule' emerging from pre-clinical to IND-enabling during 2019-20. The critical point in time appears to be the delivery of therapeutic in vivo outcomes which should happen in 2019. As slide 6 indicates, this is the primary reference point for Pharma partners to assess whether the molecule will translate into the clinic.

Sarepta is a biotech success story which has rapidly progressed to a US$8B market cap based upon one drug. Imagine what they could do with a pipeline of drugs in neuromuscular diseases including cystic fibrosis, multiple sclerosis and spinal muscular atrophy. They also understand the advantages conferred by CPP delivery of ASOs. It almost looks a perfect fit.