Opko Health Inc. (OPK) Announces Orphan Drug Designation for CUR-1916 in Dravet Syndrome

Opko Health Inc. (NASDAQ:OPK) announces that the Company has received orphan drug designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for OPKO’s oligonucleotide-based AntagoNAT (CUR-1916) for the treatment of Dravet Syndrome.  Currently, there is no approved treatment for Dravet Syndrome in the U.S.  On March 7, 2017, OPKO Health received orphan drug designation for CUR-1916 for the treatment of Dravet Syndrome from the European Commission.

Orphan drug designation provides certain marketing exclusivity, tax credits for research and a waiver of the New Drug Application user fee.

AntagoNAT, anti-Natural Antisense Transcripts, is an OPKO platform technology in which single strand oligonucleotide molecules are designed to interfere with regulatory gene expression in order to enhance production of endogenous functional proteins.  The AntagoNAT technology, part of CURNA Pharmaceuticals, acquired by OPKO in 2011, was further developed in OPKO’s Miami research laboratories under the direction of Jane Hsiao, Ph.D., OPKO’s Vice Chairman and Chief Technical Officer.

OPKO has studied over 250 genes and confirmed involvement of natural antisense transcripts (NAT) in their regulatory pathways. Of those, 89 genes were demonstrated to be subject to significant upregulation of mRNA in in vitro screening, and seven AntagoNAT oligonucleotides have been validated in vivo to date. OPKO plans to initiate a clinical trial of CUR-1916 for treatment of Dravet Syndrome this year.

Oligonucleotides are synthetic chemical compounds consisting of mixtures of modified DNAs and RNAs. Only five oligonucleotide compounds are approved by FDA for various indications and others have been reported to be in late phase clinical development. The majority of the compounds work by down regulating, or depressing transcription (anti-sense) or by correcting gene defects. CUR-1916 works by upregulating a defective gene to increase the production of functional protein.

Shares of Opko Health closed yesterday at $7.86, down $0.14 or -1.75%. OPK has a 1-year high of $12.15 and a 1-year low of $7.13. The stock’s 50-day moving average is $8.24 and its 200-day moving average is $9.61.

On the ratings front, Opko Health has been the subject of a number of recent research reports. In a report issued on March 14, Guggenheim analyst Louise Chen initiated coverage with a Buy rating on OPK and a price target of $25, which represents a potential upside of 218% from where the stock is currently trading. Separately, on March 3, Standpoint Research’s Ronnie Moas reiterated a Buy rating on the stock and has a price target of $14.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Louise Chen and Ronnie Moas have a yearly average loss of 15.3% and a return of 5.7% respectively. Chen has a success rate of 33% and is ranked #4449 out of 4549 analysts, while Moas has a success rate of 70% and is ranked #73.

Sentiment on the street is mostly bullish on OPK stock. Out of 5 analysts who cover the stock, 4 suggest a Buy rating and one recommends to Hold the stock. The 12-month average price target assigned to the stock is $13.38, which represents a potential upside of 70% from where the stock is currently trading.

OPKO Health, Inc. is a diversified healthcare company. It operates through Diagnostics, and Pharmaceutical segments. The Diagnostics segment is consists of the pharmaceutical operates that the company acquired in Chile, Mexico, Ireland, Israel, and Spain and its pharmaceutical research and development operations. The Diagnostics segment is primarily comprised of the clinical and laboratory operations tit acquired through the acquisition of Bio-Reference and OPKO Lab and its point-of-care operations. 


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