Tens of millions of dollars milestone payment Lilly co-developed RNAi gene therapy new drug application authorized

On November 16, RNA Interference (RNAi) gene therapy drug developer Dicerna Pharmaceuticals, Inc., announced that the FDA has now authorized a new drug application (IND) code-named LY3561774, the first clinical phase drug candidate for Dierna's partnership with Lilly.
the authorization triggered Lilly's $10 million milestone payment to Diperna, while later Lilly will conduct a Phase I clinical trial of LY3561774.
, the pre-planned trial is expected to be conducted by the end of 2020, primarily for the treatment of an unknown cause of cardiovascular metabolic disease.
In addition, Dicerna is eligible for up to $350 million in future development and commercial milestone payments for liver metabolic disease applications on the GalXC™ platform and $355 million milestone payments for non-liver metabolic disease applications, as well as tiered licensing fees for potential product sales from the median to the lower double digits.
Agreement between Dierna and Lilly was reached in 2018 to identify, develop and commercialize potential new therapies for metabolic diseases, neurodegenerative diseases and pain.
partnership, the two sides have launched more than a dozen development projects.
and LY3561774's IND application is the first milestone achievement of Thena and Lilly's 2018 global licensing and research collaboration, which develops research cardiovascular metabolic therapies and future therapies primarily using Dicerna's RNAi technology and platform.
for Lilly, this provides strong support for the marketing of RNAi product candidates in 2021 and beyond, and helps improve research and development pipelines.
, RNAi-based drugs have the potential to become an effective and accessible treatment for more serious diseases that are not met by the drug market.
' proprietary RNAi technology platform, called GalXC™, aims to advance the next generation of RNAi-based therapies that will be able to silence the genes that drive liver disease.
addition, GalXC-based compounds enable off-the-skin delivery of RNAi therapy, which binds to the specific binding of the subjects on liver cells, causing the cell to internalize and enter the cell to perform RNAi's role.
the GalXC approach is that it optimizes the activity of the RNAi approach to operate in the most specific and efficient way.
Diperna is currently exploring new applications of its RNAi technology beyond the liver, targeting other tissues and implementing new therapeutic applications.
RNAi is a biological process in which some double-stranded RNA molecules inhibit the expression of disease-caused genes by destroying the messenger RNA (mRNA) of those genes.
it reflects new ways to develop specific and effective therapies.
RNAi does not target and bind to proteins to inhibit their activity, but rather plays an earlier role in gene silencing by targeting mRNA, the set of instructions that guide protein construction.
to this instruction set, RNAi is thought to have the ability to attack any target, including disease-caused genes that are not possible in conventional antibodies and small molecule forms.
, the RNAi platform has the potential to address diseases that are difficult to treat in other ways through silent disease-caused genes.