Phase 1 clinical trial of SIRT1 inhibitor for the treatment of Huntington’s Disease commenced

Elixir Pharmaceuticals, Inc. announced today that its partner, Siena Biotech S.p.A., has commenced Phase 1 clinical testing of Elixir’s potent, first-in-class SIRT1 (sirtuin-1) inhibitor for the treatment of Huntington’s Disease. EX-527, also known as SEN0014196, is currently in a Phase 1a combined single and multiple ascending dose study in the European Union to assess safety, tolerability and pharmacokinetics in healthy volunteers.

“This is a major accomplishment for Elixir and its partner, Siena Biotech”

In addition to initiation of the Phase 1 study, Elixir announced EX-527/SEN0014196 was granted orphan designation by the European Medicines Agency (EMEA) on October 28, 2009, and by the Office of Orphan Products Development of the U.S. FDA on December 7, 2009. Orphan designation allows for various terms of market exclusivity, as well as possible regulatory fee exemptions or reductions.

“This is a major accomplishment for Elixir and its partner, Siena Biotech,” said Peter DiStefano, CSO of Elixir. “This represents the first sirtuin inhibitor to enter clinical trials and validates the work of our drug discovery and design program focused on the sirtuin family of enzymes, specifically, our selective SIRT1 inhibitor program. Based on in vivo preclinical and mechanistic studies, EX-527/SEN0014196 offers the potential for novel and effective therapy for Huntington’s Disease, a debilitating and ultimately fatal neurodegenerative disease.”

In preclinical studies, EX-527/SEN0014196 reduced neuronal death caused by mutant huntingtin protein in cell-based assays. In addition, the molecule was efficacious in a widely employed transgenic model of Huntington’s Disease while demonstrating highly favorable safety profiles and pharmaceutical properties.

Elixir entered into a research collaboration agreement with Siena in 2007, and Elixir licensed EX-527 to Siena in 2009. Under the terms of the license Siena has exclusive worldwide rights to EX-527/SEN0014196 in certain diseases.

Source: Elixir Pharmaceuticals, Inc.