Rocket Pharma reports updated PhI data for Danon disease

 New Jersey biotech Rocket Pharmaceuticals has been working on an investigational gene therapy for gene disorder Danon disease — and the pharma announced updated data from the Phase I trial today.

The trial was looking at a single IV infusion of gene therapy RP-A501 at two different dosages in five patients — “low-dose” with three patients (6.7e13 vg/kg) and “high-dose” with the other two (1.1e14 vg/kg). While the gene therapy was well-tolerated at the low dose and conferred sustained clinical benefit, the same can’t be said for the high dose.

As previously made public, dose-dependent toxicity was seen in one of the two patients on the high dose regimen — and developed thrombotic microangiopathy that fully resolved with treatment, which included transient hemodialysis.

As such, the biotech will be cutting off the higher dosage and sticking with the lower amount moving forward.

“We are excited to announce positive data from our RP-A501 trial for Danon Disease showing clinical, functional and biomarker improvements at one year or beyond and potential early separation from the natural course of disease in adult and adolescent patients,” said Rocket CEO Gaurav Shah. “We have initiated treatment in the pediatric cohort at the low-dose and anticipate moving as rapidly as possible toward Phase II.”

https://endpts.com/cassava-being-investigated-by-unnamed-government-agencies-rocket-pharma-gives-updated-data-on-danon-disease/