The treatment of inherited retinal diseases is the priority setting for the clinical development of our platform….
Multiple Pharmaceutical/Biotechnology companies are advancing drugs that are delivered inside cells by Cell Penetrating Peptides (CPPs) through clinical trials today….
Animal studies demonstrate that Phylogica’s CPPs deliver substantially more drug cargo inside their target cell than the current CPP benchmark for clinical development…
The animal studies... demonstrate the efficacy of Phylogica’s drug molecule at a dose that is substantially lower than competitive approaches for ASO administration in the vitreous without the benefit of PYC’s CPP delivery technology….
OK, PYC says it will focus on applying its CPP technology to inherited retinal disease. It says that lots of biotech and pharma are using CPPs in clinical trials. It says that its latest mouse studies show that its CPP can deliver more ASO drug cargo inside the target cell (Retinal Pigment Epithelium) than the “current CPP benchmark for clinical development”, which is apparently PepK. And finally, it says that its CPP enables the ASO drug cargo to work more effectively at a much lower dose than “naked” ASOs currently being developed in inherited retinal disease (by Wave Life Sciences and ProQR).
So far, so good.
From a bit of research I can see that inherited retinal disease is a very sexy space atm.
Janssen signed a $440 million headline collaboration and license deal with MeiraGTx in January to develop, manufacture and commercialize a portfolio of gene therapies for inherited retinal diseases.
Roche moved on Spark Therapeutics in February with a $4.8bn merger offer which it’s still trying to wrap up. The main prize is Luxturna (which I mentioned on the other thread), Spark’s once-only genetic treatment for individuals with inherited retinal disease caused by mutations in both copies of the RPE65 gene, which was approved by the FDA early last year. However, Spark’s pipeline also includes therapies for treating choroideremia and Stargardt diseases, both inherited retinal diseases.
Then, earlier this month,
Biogen announced it was acquiring Nightstar Therapeutics for $800 million. Nightstar is a clinical-stage gene therapy company focused on (you guessed it) inherited retinal diseases. Biogen has picked up two mid- to late- stage assets, as well as preclinical programs in opthamology.
But what do MeiraGTx, Spark Therapeutics and Nightstar have in common, other than inherited retinal disease targets?
Rather than using ASOs or CPPs in their retinal disease therapies, they are using AAVs.
Now, having a more effective CPP is great, as is having a CPP + ASO that outperforms a naked ASO. But what I’d really like to know is how does PYC’s CPP + ASO perform compared with AAV gene therapy. ProQR
compares its naked ASO approach to the AAV + gene approach (Slides 18-25).
My question for Phylogica is
What is/are the compelling advantage(s) of the CPP + ASO approach to the treatment of inherited retinal eye disease compared with gene therapy using an AAV?
(20min delay)