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Alnylam Files IND for RSV Treatment; Breakdown of RNAi Drugs Pipelines

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Alnylam Pharmaceuticals announced this week that it has submitted to US regulators an investigational new drug application to begin phase I testing of an intranasally delivered RNAi-based treatment for respiratory syncytial virus.

The news comes a little more than a month after Alnylam said it would drop development of its age-related macular degeneration treatment ALN-VEG01 amid competition in the field (see RNAi News, 9/23/2005).

The company said that, pending US Food and Drug Administration acceptance of the IND, it expects to begin testing the drug, called ALN-RSV01, in healthy adults before the end of the year. Alnylam plans to ultimately submit the drug for approval for use in pediatric patients.

Alnylam also said that it is planning to initiate a phase I trial of the drug in Europe. Company officials were not available to comment on when the European study might begin, or the rationale behind it.

According to Alnylam, ALN-RSV01 selectively silences the RSV nucleocapsid "N" gene, which is essential for RSV replication. "Data presented to date show both in vitro and in vivo efficacy of ALN-RSV01 in pre-clinical animal studies," the company said. "Intranasally delivered ALN-RSV01 specifically inhibits RSV replication in animals and is active in the prevention and treatment of RSV infection. Additionally, Alnylam scientists have reported completion of GLP toxicology studies for ALN-RSV01 without any significant toxicities being observed."

Should Alnylam begin phase I testing of ALN-RSV01 before the end of 2005, the company will have beaten its previously disclosed timeline of starting clinical testing in the first half of 2006. Below is a list of the major players in the RNAi drugs space and where their publicly stated clinical-development efforts currently are:

Company Indications Timeline
Acuity Pharmaceuticals Age-related macular degeneration Recently started phase II testing; phase III trials planned for late 2006/ early 2007
Alnylam Pharmaceuticals RSV Phase I planned for end of 2005
Pandemic influenza No timeline
Cystic fibrosis (with the Cystic Fibrosis Foundation) No timeline
Spinal cord injury (with Merck) No timeline
Parkinson's disease (with Medtronic) No timeline
Benitec AIDS lymphoma Phase I in the first half of 2006
Hepatitis C Phase I planned for the second half of 2006
Calando Pharmaceuticals 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 planned for the second half of 2007
GeneCare Cancer Phase I planned for 2007
Genesis Research
and Development
Asthma No timeline
Atopic dermatitis No timeline
IC-Vec Hepatitis B No timeline
Cancer No timeline
Intradigm Cancer Phase I in first quarter of 2006
Nastech Rheumatoid arthritis No timeline
Nucleonics Hepatitis B IND in first quarter of 2006
Sirna Therapeutics Age-related macular degeneration (recently licensed to Allergan) Currently in phase I
Hepatitis C Phase I expected in the second half of 2006
Permanent hair removal IND planned for late 2006
TransDerm Pachyonychia congenita IND in 2006

— Doug Macron ([email protected])

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