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Snapshot: Despite Advances in RNAi Science, the Field Sees Departures of Would-Be Rx Developers


Last week, Targeted Genetics announced that it may shutter its doors as soon as the end of the month if it is unable to find new sources of financing (see related story, this issue).

While most companies working in the RNAi drugs field have been able to secure the money needed to maintain operations, despite a difficult economy, Targeted Genetics wouldn’t be the first to exit the space under unfavorable circumstances.

Below is a breakdown of key RNAi drug developers that have either shutdown, ceased their RNAi activities, or experienced significant restructuring other than a merger or acquisition since they were founded.

Nucleonics — Founded in 2001 to develop expressed RNAi therapeutics, Nucleonics was originally a high-flyer in RNAi, raising nearly $50 million in a Series B financing round in 2004. But the company began experiencing difficulties that very year when Australian rival Benitec sued for patent infringements. After a protracted legal battle, Benitec eventually dropped its suit, but Nucleonic again ran into trouble in 2008 after it failed to close a Series C round and was forced to shut down its operations. Legal filings later revealed that the financing was scuttled by allegations of scientific misconduct, including the falsification of data related to the company’s phase I hepatitis B therapy NucB 1000, between the company’s co-founders, its president and CEO, and its board of directors.

Galenea — The company was established in 2004 to develop an siRNA-based influenza treatment originating at the Massachusetts Institute of Technology and small-molecule drugs for cognitive disorders. From the start, Galenea’s RNAi activities were troubled: a $50 million deal with Otsuka Pharmaceutical only applied to the company’s non-RNAi programs, and the company struggled to compete with Alnylam Pharmaceuticals, which was at the time developing its own flu drug, for funding and partnerships. By 2006, Galenea had decided to bow out of the RNAi arena, and sold off its RNAi technology and intellectual property to Nastech Pharmaceutical (now MDRNA).

Benitec — Boasting one of the RNAi field’s first US patents, Australia’s Benitec came on the scene in 2003 with its chief executive telling RNAi News that his company planned to file lawsuits against a number of companies if licensing deals for the patent could not be struck. Of the three companies ultimately sued, only one — Nucleonics — declined to take a license, setting off years of costly legal wrangling that depleted Benitec’s coffers and tarnished its image. After replacing its CEO, Benitec successfully petitioned to have the Nucleonics suit dismissed, but Nucleonics’ efforts to invalidate the patent continued. Benitec eventually shut down its US operations and reorganized as a significantly smaller firm in Australia, and continues to struggle to maintain its US patent, the claims of which have been rejected multiple times on a non-final basis by the US Patent and Trademark Office.

Intradigm — An early player in therapeutic RNAi, Intradigm had long been committed to its vascular endothelial growth factor-targeting cancer therapy ICS-283. After repeatedly missing goals for moving the drug into the clinic, the company underwent a sweeping reorganization in 2006, which included a complete change in management and relocation from Maryland to California. By early this year, the company again replaced its chief executive and had dropped ICS-283, deciding to pursue new targets deemed undruggable by other technologies.

Nastech Pharmaceutical — Through it was founded as an intranasal drug-delivery company, Nastech dipped its toes in the RNAi waters when it acquired the RNAi assets of Galenea. However, its failures with its drug-delivery technologies, including the loss of a key collaborator, led the company to reorganize as a pure-play RNAi drugs shop, a move that included a change in its management and its name. The company now operates as MDRNA.

CytRx — CytRx became a key face in RNAi in 2003 when it licensed from the University of Massachusetts the so-called Tuschl-I intellectual property estate for certain indications including obesity, type II diabetes, and amyotrophic lateral sclerosis. Although the company provided ambitious guidance on its RNAi pipeline, at one time stating that it could file an investigational new drug application on an siRNA-based ALS drug in 2004, the company never gained any traction in the field and shifting its focus onto small-molecule drugs instead. In 2007, CytRx spun out its RNAi operations into pure-play subsidiary RXi Pharmaceuticals, which has since distanced itself from CytRx and now operates independently, although its one-time parent still holds about 39 percent of its outstanding stock.