Aug. 26 at 2:42 PM
$ARCT 810 for OTC deficiency was mentioned as a dark horse by Joe Payne. Yesterday, the latest presentation by Arcturus at Helsinki showed very positive phase 2 results for 810, proving safety and tolerability, and very importantly, it also demonstrated efficacy as shown in the normalisations of not just ammonia levels, but also glutamine levels and decreases in glycine being observed. So, there is high chance of obtaining FDA approval to proceed to phase 3. 810 has secured FDA Fast Track, Orphan Drug and Rare Paediatric Diseases designations.
For 810, this dark horse is finally coming of age. In US and Europe, there are about 10,000 patients, and cost per patient is USD1.2million. A 10% market share would translate to potential revenue of USD1.2billion. Outside of US and Europe, there could be market for 810 therapy too. So, all very positive and promising for Arcturus. Like what Elon Musk
$TSLA said, mRNA technology has tremendous promise for curing diseases.
$MRNA $BNTX
