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Alnylam to Disclose Two Additional Drug Programs by Year-End


By Bernadette Toner

Alnylam plans to unveil two new drug programs in the third and fourth quarter, CEO John Maraganore disclosed this week.

Speaking at the 2011 Bank of America Merrill Lynch Health Care Conference this week, Maraganore said that the company is in the final stages of selecting the fourth and fifth candidates for its so-called 5x15 strategy, announced in January (GSN 1/13/2011), in which it aims to have five products in advanced clinical development by 2015.

"We have about four or five different discovery programs right now that are in an intense bake-off," Maraganore said. These potential programs "cover diseases ranging from thalassemia, hemophilia, to [hereditary angioedema] and other disorders," he added.

Maraganore added that the new programs will be in line with the company's "strategic decision to focus on genetically defined diseases" for its internal discovery programs.

Alnylam has three other programs in its 5x15 initiative: a treatment for transthyretin-mediated amyloidosis treatment called ALN-TTR01, along with a follow-on therapy called ALN-TTR02; a treatment for severe hypercholesterolemia called ALN-PCS; and a treatment for refractory anemia called HPN.

During his presentation, which was webcast, Maraganore reiterated previously disclosed timelines for the first three programs in Alnylam's pipeline.

ALN-TTR01 entered phase 1 testing last summer and the company expects to report human proof-of-concept data in the third quarter.

Alnylam plans to file an investigational new drug application for ALN-PCS in the first half of this year, with a phase 1 trial slated to start before the end of the year, while an IND for ALN-HPN is planned for 2012.

Alnylam also has two partnered programs — ALN-RSV01 for respiratory syncytial virus, which it is developing with partner Cubist Pharmaceuticals, and ALN-HTT for Huntington's disease, which it is developing with Medtronic.

The company is seeking a development partner for its liver cancer treatment ALN-VSP, Maraganore said.

Have topics you'd like to see covered in Gene Silencing News? Contact the editor at btoner [at] genomeweb [.] com.

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