BioTech

PhaseRx Inc. (NASDAQ:PZRX) Spikes 66% On Orphan Drug Designation

PhaseRx Inc. (NASDAQ:PZRX)

PhaseRx Inc. (NASDAQ:PZRX)

PhaseRx Inc. (NASDAQ:PZRX) shares jumped 66.45% after the U.S. Food and Drug Administration (FDA) granted an Orphan Drug Designation for its treatment of argininosuccinate lyase deficiency (ASLD), PRX-ASL. According to Chief Executive Officer, Robert W. Overell, the designation represents an important milestone in the development of the company’s second therapeutic candidate.

Stock Performance

The Orphan Drug Designation helped reverse a strong downtrend that had plagued the stock since May. The stock has since risen above the $1.20 mark, a key resistance level. It awaits to be seen if the close above the $1.20 mark will help fuel a run to $1.90, which acts as another resistance level.

One month PZRX stock price chart

PhaseRx Inc. (NASDAQ:PZRX) Surges 66.45% is a biopharmaceutical company specializing in the development of mRNA products targeting life-threatening inherited liver disease in children. PRX-ASL is the company’s candidate drug for ASLD, a rare liver disorder caused by a single-gene deficiency that most of the time results in elevated ammonia in the blood.

PhaseRx’s PRX-ASL is a replacement therapy designed to replace missing or defective enzyme in patients with ASLD. Clinical trials have shown that the candidate drug can reduce compounds that form the hallmark of ASLD including Plasma and blood ammonia.

“PRX-ASL is our second drug to show preclinical proof of concept using our Hybrid mRNA Technology. Like our lead candidate PRX-OTC, we believe PRX-ASL also has the potential to correct the disease in children, a population that could particularly benefit from treatment for this rare disease,” said Mr. Overell.

Orphan Drug Designation Synergies

The Orphan Drug Designation is an important milestone because PhaseRx Inc. (NASDAQ:PZRX) will now be able to get assistance in clinical studies as well as design and drug development from the FDA. The company could also receive tax credits for clinical trial costs as well as exemptions from certain FDA applications.

Seven years of market exclusivity upon regulatory approval is another benefit that the company stands to enjoy. The European Medicine Agency has also granted Orphan Drug Designation for the novel treatment.

Separately, PhaseRx Inc. (NASDAQ:PZRX) posted a net loss of (-$3.5) million for Q2 2017 – less than half the net loss of (-$11.1) million the company reported a year ago. Operating expenses in the quarter totaled $3.3 million, down from $9.8 million reported in Q2 2016. The biopharmaceutical company exited the quarter with cash and cash equivalent of $8.4 million.

I have no positions in any of the stocks mentioned, and have no plans to initiate any positions within the next 72 hours. All information, including any data, is provided without any guarantees of accuracy.

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About the author: Monica has an undergraduate degree in Accounting and an MBA she earned – with Honors. She has six years of experience in the financial markets and has been an analyst for the past two years.

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Monica has an undergraduate degree in Accounting and an MBA she earned - with Honors. She has six years of experience in the financial markets and has been an analyst for the past two years.

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