BioWorld Today Contributing Writer

Opko Health Inc. plunked down $10 million cash this week to acquire Curna Inc., a privately held drug discovery company based in Jupiter, Fla., that has largely flown under the radar since it was launched in 2008 with technology from the Scripps Research Institute.

Unlike competitors in the field of oligonucleotide therapeutics, Curna's technology is based on the up-regulation of protein production through interference with noncoding RNAs (natural antisense). The strategy contrasts with established approaches, which down-regulate protein production.

Curna has designed a therapeutic modality termed AntagoNAT and has validated the approach for up-regulation of several therapeutically relevant proteins. Claes Wahlestedt developed the technology as a professor at Scripps' Jupiter campus.

He then partnered with Florida entrepreneur Joseph Collard to co-found the company, which exclusively licensed the technology from Scripps.

Curna has developed and patented molecules that increase the production of more than 90 key proteins involved in a number of diseases. Thus, the company's platform holds the potential to treat a wide variety of illnesses, including cancer, heart disease, metabolic disorders and genetic anomalies.

Miami-based Opko is a specialty health care company that has its hands in a variety of business lines, including the discovery, development and commercialization of pharmaceutical products, medical devices, vaccines, diagnostic technologies and imaging systems. Initially focused on the treatment and management of ophthalmic diseases, Opko received a rude wake-up call in 2009, when it yanked its lead compound, the siRNA bevasiranib, for wet age-related macular degeneration after a data monitoring committee determined its Phase III study was unlikely to meet its primary endpoint. (See BioWorld Today, March 9, 2009.)

Despite the bevasiranib setback, Opko continues to pursue RNAi technology, so Curna's platform technology was attractive, according to Steven D. Rubin, Opko's executive vice president of administration.

"We started out with and we still have RNAi technology, and this is an RNA-based technology, with the difference that it's not interfering," Rubin told BioWorld Today. "It actually up-regulates, so it stimulates the production of a protein."

Going forward, Curna will operate as a subsidiary of Opko.

"They have a great platform," Rubin said. "The business model has been to use their platform to validate the potential for a variety of diseases but not really go into the clinic. We hope to pretty quickly choose a couple of nice targets, focus on those and try to get something in the clinic as quickly as possible.

"They've done a nice job validating the utility of the technology, and we hope to take it to the next step," Rubin added.

Although Opko has diversified away from ophthalmics, the company continues to develop a glaucoma drainage device called aquashunt and the investigational compound doxovir for the treatment of viral conjunctivitis. In 2008, Opko acquired worldwide rights from Redox Pharmaceuticals to commercialize the cobalt-containing compound, which scavenges superoxide radicals and has displayed antiviral and anti-inflammatory properties in preclinical models.

In addition, the company has expanded into other activities, ranging from the development of vaccines for flu, dengue fever and West Nile virus to the rapid diagnostics of small molecules.

Opko hasn't shied away from other deals. Last month, the company inked an agreement with Bristol-Myers-Squibb Co. to investigate the use of its blood test technology to diagnose Alzheimer's disease and to identify individuals with early stage cognitive impairment that is likely to progress to Alzheimer's.

The technology, also licensed from Scripps, is designed to identify biomarkers for a variety of illnesses. (See BioWorld Today, Jan. 7, 2011.)

And in December, Opko flipped the rights to the nausea drug rolapitant, which it had earlier acquired from Schering-Plough Corp., to Tesaro Inc. for $121 million. (See BioWorld Today, Dec. 15, 2010.)

The clinical and financial diversity make it difficult to pin Opko to a single target.

"In the near term, our focus is going to be on the molecular diagnostics side," Rubin said.

"We're still looking to do something with RNAi, but like everyone else in the RNAi field, we're looking for a better way of delivering better penetration to the cell." Opko has several projects under way in that area – likely along with every other company with an RNAi portfolio, he acknowledged.

"We would not go back into the clinic until we are happy with that," Rubin said.

The Curna RNA technology platform "does not seem to have the same cell penetration issues, so the target could be in ophthalmology – in fact, that's a good possibility – but that's as much coincidental as anything else," he added.