Arrowhead Pharmaceuticals Inc. might be on the brink of a biotech drug breakaway.

It looks as though its shares have a head start in any case – up by about 300 percent so far this year, pushing its market capitalization to $1.4 billion.

The Pasadena-based biopharmaceutical company just won European and U.S. approvals for its orphan drug to treat liver disease tied to alpha-1 antitrypsin deficiency, a rare genetic disorder.

Early results of a clinical trial of the RNA-interference (RNAi) drug, meanwhile, suggest it can stymie a harmful protein linked to the disease. The process is known as “gene silencing,” which aims to turn off certain genes in order to inhibit the body’s production of disease-causing proteins.

“Near complete suppression,” Chris Anzalone, chief executive of the 14-year-old Arrowhead. “It’s exciting. We still have a long way to go until commercialization – but we have made a huge step forward.”

Arrowhead has a research laboratory in Wisconsin – picked up in a deal with Roche Holding AG – that works at the molecular level to develop the gene-silencing medicines to treat various intractable illnesses.

The company has a pipeline of eight drugs under development to treat liver and cardiovascular disease and hepatitis B. The research and development lineup on RNAi drugs give it a place in an emerging field of gene therapy that aims to wipe out proteins that cause disease.

Arrowhead’s standing – along with its shares – have also gotten recent boosts from licensing deals with Roche, Novartis AG and Amgen Inc., as well some progress by a competitor in the segment.

The recent activity has some viewing Arrowhead as a rising star in the growing Los Angeles biotech industry as well as in the field of RNAi drugs.

“Arrowhead has done a good job of moving their pipeline forward,” said Ahmed Enany, chief executive of the Southern California Biomedical Council, a Los Angeles-based trade group. “They’ve done a good job of hanging around, hanging tough, riding the market and raising money from the public to move their products.”

Reverse merger

Arrowhead was founded as a holding company for a variety of nanotechnology companies, Anzalone said. It went public in 2006, the same year that that Andrew Fire of Stanford University and Craig Mello of the University of Massachusetts Medical School were credited with the discovery of RNAi and awarded a Nobel Prize for medicine.

Arrowhead soon acquired two spinoff firms from California Institute of Technology in Pasadena. One of the acquisitions was Calando Pharmaceuticals Inc., the first company to develop an RNAi therapy.


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