Published on January 8, 2010 at 12:58 AM
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.
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