All eyes are on shares of PhaseRx Inc. (NASDAQ:PZRX) this morning, after the FDA has given Orphan Drug Designation for one of the company’s most talked about drugs. PRX-OTC is an intracellular enzyme replacement therapy aimed at treating children with a rare liver disorder referred to as ornithine transcarbamylase deficiency (OTCD).
OTCD causes hyperammonemia, which is an elevated level of ammonia in a person’s blood. It often has huge neurological effects on a patient and can result in coma and/or death.
The drug, PRX-OTC, has been tested on mice and has been shown to efficiently lower levels of blood ammonia, so much so that the survival rate in these mice was 100%. There is still a long way to go to bring this treatment to humans, and surely many obstacles to overcome, but investors see this as a potential blockbuster should eventual FDA approval of a human variation come to fruition.
Shares of PhaseRx Inc. (NASDAQ:PZRX) have jumped 115% to $2.44 this morning after the news was released. This is still well shy of the $5.77 52-week high we saw earlier in the year. The stock has traded with a volume of 2.85 million shares, compared the average daily volume of just 58,250 shares. It will be interesting to follow the price of this stock as the day unfolds.
“The FDA’s decision to grant PRX-OTC orphan drug designation for OTCD is another important milestone in the development of our lead product candidate, as we prepare to file the IND by the end of 2017 and initiate our clinical trial in 2018,” explained Robert W. Overell, Ph.D., president and chief executive officer. “PRX-OTC is the first of three drugs in development using our Hybrid mRNA Technology™, and we believe it has the potential to correct the disease in children, a population that could particularly benefit from treatment for this rare disease. Our team at PhaseRx is driving hard to advance these drugs to help the lives of families affected by this devastating liver disease that causes irreversible brain damage and potentially fatal ammonia toxicity.”