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RNAi Therapeutic Pipeline Update


As the RNAi-based therapeutics space becomes increasingly crowded, keeping track of who's doing what and when can prove a challenge. As such, RNAi News has compiled a list of the major players in the space and where their publicly stated clinical-development efforts stand.

For the most part, the timelines provided are unchanged from those provided by the companies in recent months. There are a few exceptions, however.

The biggest shift occurred at Sirna Therapeutics, which just streamlined its pipeline in a bid to control costs. While Sirna had given estimates on six drug programs in January (see RNAi News, 1/14/2005), the company recently said it was focusing on its three core programs • in hepatitis C, age-related macular degeneration, and permanent hair removal • and other projects would only be "advanced with a combination of internal and external support" (see RNAi News, 5/13/2005).

A surprise came from Acuity Pharmaceuticals, which, after being the first company to get an RNAi drug into humans (see RNAi News, 11/12/2004), is already looking to the next stage of development. Acuity President and CEO Dale Pfost told RNAi News in an e-mail this week that his company is aiming to begin phase II development of its AMD drug before the end of the year.

Meanwhile, Intradigm readjusted its timeline for a cancer therapy, pushing it back to the first quarter of 2006 from previous projections of year-end 2005. It also dropped plans to work on diseases associated with ocular angiogenesis on its own. Chairman and CEO John Spears told RNAi News in an e-mail this week that the company would, however, consider working with a collaborator in the ocular field.

Benitec also changed up a development timeline, with CEO Sara Cunningham telling RNAi News that a phase I trial for an HIV treatment would likely start in the first half of next year, as opposed to sometime this year as stated on the company's website. Benitec also recently announced that a hepatitis C drug is expected to reach phase I testing in the second half of 2006.

Finally, newcomer Galenea provided for the first time guidance on its RNAi-based influenza drug, targeting an IND for the second half of 2007.

• Doug Macron ([email protected])

RNAi Therapeutic Pipeline Update
Acuity Age-related macular degeneration Phase II in 2005
Alnylam Pharmaceuticals Age-related macular degeneration Phase I in the second half of 2005
Respiratory syncytial virus Phase I in the first half of 2006
Atugen Cancer IND in 2006
Benitec Hepatitis C Phase I in the second half of 2006
HIV Phase I in first half of 2006
Calando No programs announced No timeline
CombiMatrix Conditions associated with unconventional warfare No timeline
Hepatitis C No timeline
HIV No timeline
CytRx Amyotrophic lateral sclerosis No timeline
Obesity No timeline
Type II diabetes No timeline
Cytomegalovirus No timeline
Galenea Influenza IND in the second half of 2007
GeneCare Cancer Phase I in 2007
Intradigm Cancer Phase I in first quarter 2006
Nastech Rheumatoid arthritis No timeline
Nucleonics Hepatitis B IND before end of 2005
Hepatitis C IND in third quarter 2006
Sirna Therapeutics Age-related macular degeneration Phase II in mid-2006
Permanent hair removal Phase I in second quarter of 2006; phase II in 2007; phase III in 2009
Hepatitis C Phase I by the end of 2006
Quark Biotech Age-related macular degeneration IND by the end of 2005
Diabetic retinopathy IND by the end of 2005

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