Seattle-based biotech company PhaseRx develops drug candidates to treat rare, often life-threatening liver diseases, and one of their candidates just got a boost from the FDA.
The company’s leading drug, called PRX-OTC, was awarded the FDA’s orphan drug designation, which encourages companies to develop treatments for rare diseases. PRX-OTC treats ornithine transcarbamylase deficiency (OTCD), a rare and often life-threatening liver disorder that primarily affects children under the age of 12.
PhaseRx’s orphan drug status means it will get financial breaks as it moves the drug through the rigorous and lengthy FDA approval process, and will also be guaranteed seven years of market exclusivity if the drug is approved.
In response to the news, PhaseRx’s stock jumped almost 150 percent by market close on Monday, although it had dipped to $2.10 by Tuesday morning. PhaseRx went public in May, pricing shares on the Nasdaq under the ticker PZRX at $5 per share. It now has a market value of about $61 million.
The new interest in the company is promising as it prepares for clinical trials of the treatment, which are planned for 2018.
Early trials of the drug have proved promising, as PhaseRx CEO Robert Overell told GeekWire in a recent interview.
Overell said the drug works by leveraging a cell’s mRNA, molecules that communicate with the DNA and help enact the function of certain genes. PhaseRx has developed an mRNA delivery system that allows physicians to alter how DNA is expressed, which they hope to use to treat the genetic cause of OTCD.
“Using this system, we have shown complete reversal of urea cycle diseases in preclinical mouse models that are missing exactly the same gene as the children suffering from the disease,” said Overell, a former venture capitalist at Frazier Healthcare ventures who also previously worked for Immunex Corp. and Targeted Genetics Corp.