Feb 25 2010
Regulus Therapeutics Inc. today announced the establishment of a new
collaboration with GlaxoSmithKline (GSK) to develop and commercialize
microRNA therapeutics targeting microRNA-122 in all fields with
Hepatitis C Viral infection (HCV) as the lead indication. Under the
terms of the new collaboration, Regulus will receive additional upfront
and early-stage milestone payments with the potential to earn more than
$150 million in miR-122-related combined payments, and tiered royalties
up to double digits on worldwide sales of products.
“This new collaboration with GSK demonstrates the clear scientific
leadership that Regulus has established in advancing a whole new
frontier of pharmaceutical research. microRNA therapeutics target the
pathways of human diseases, not just single disease targets, and hold
considerable promise as novel therapies across a broad range of unmet
medical needs,” said Kleanthis G. Xanthopoulos, Ph.D., President and
Chief Executive Officer of Regulus. “It also further validates Regulus’
microRNA product platform built on fundamental biology of human diseases
and intellectual property, and also extends the therapeutic scope of our
existing collaboration formed with GSK in 2008. Furthermore, the funding
from this alliance supports Regulus’ efforts in advancing high impact,
novel medicines based on microRNA biology to patients.”
The collaboration provides GSK with access to Regulus’ comprehensive and
robust intellectual property estate. Regulus exclusively controls patent
rights covering miR-122 antagonists and their use as HCV therapeutics in
the United States, Europe, and Japan, including but not limited to the
patent families which encompass: the ‘Sarnow’ patent pertaining to the
method of use of anti-miR-122 to inhibit HCV replication, the ‘Esau’
patent application claiming the use of anti-miRs targeting miR-122 as
inhibitory agents, the ‘Tuschl III’ patent claiming composition of
matter for miR-122 and complementary oligonucleotides, and the
‘Manoharan’ patent claiming antagomirs, including antagomirs targeting
miR-122.
miR-122 is a liver-expressed microRNA that has been shown to be a
critical endogenous “host factor” for the replication of HCV, and
anti-miRs targeting miR-122 have been shown to block HCV infection
(Jopling et al. (2005) Science 309, 1577-81). In earlier
work, scientists at Alnylam and Isis demonstrated the ability to
antagonize miR-122 in vivo using chemically modified
single-stranded anti-miR oligonucleotides. Further, work by Regulus
scientists and collaborators showed that inhibiting miR-122 results in
significant inhibition of HCV replication in human liver cells,
suggesting that antagonism of miR-122 may comprise a novel “host factor”
therapeutic strategy. Regulus scientists have shown in multiple
preclinical studies a robust HCV antiviral effect following inhibition
of miR-122. Regulus plans to identify a clinical development candidate
in the second half of 2010 and file an investigational new drug (IND)
application in 2011.
SOURCE Regulus Therapeutics Inc.