Dicerna Announces FDA Acceptance of Lilly’s Investigational New Drug (IND) Application for First GalXC™ RNAi Candidate Under Companies’ Global Research Collaboration and Licensing Agreement
Nov 16, 2020
— Milestone Achievement Reflects First Investigational Candidate to Emerge Under Multi-Target Collaboration and Triggers $10
– Lilly Targeting Initiation of Phase 1 Clinical Trial of LY3561774 by Year-End 2020 –
“The successful IND filing for LY3561774 marks an important milestone in what is expected to be the first of several resulting from our close, efficient collaboration with Lilly that could generate potentially life-saving RNAi-enabled therapies across a broad range of diseases,” said
The IND filing for LY3561774 is the first milestone achievement under a 2018 global licensing and research collaboration between Dicerna and Lilly focused on the discovery, development and commercialization of potential new therapies for cardiometabolic disease, neurodegeneration and pain. This investigational cardiometabolic therapy and future therapies to emerge from the two companies’ collaboration leverage Dicerna's proprietary RNAi technologies.
“We believe that RNAi-based medicines have the potential to be convenient and effective treatments for diseases with significant unmet need,” said
This first IND filing under the collaboration agreement with Lilly triggers a
RNA interference (RNAi) is a biologic process in which certain double-stranded RNA molecules inhibit the expression of disease-causing genes by destroying the messenger RNAs (“mRNAs”) of those genes. It reflects a new approach in the development of specific and powerful therapies. Rather than targeting and binding to proteins to inhibit their activity, RNAi exerts its effects one step earlier in the gene silencing process by targeting the mRNA, the instruction set that directs the building of the protein. By attaching to this instruction set, RNAi is believed to have the ability to attack any target, including disease-causing genes that are beyond the reach of conventional antibody and small-molecule modalities. Additionally, RNAi-based therapeutic approaches hold the potential to offer more convenience for patients via infrequent dosing and a long duration of effect.
About Dicerna's RNAi Technology Platform
Dicerna’s proprietary RNA interference (RNAi) technology platform, called GalXC™, aims to advance the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. GalXC-based compounds enable subcutaneous delivery of RNAi therapies that are designed to bind specifically to receptors on liver cells, leading to internalization and access to the RNAi machinery within the cells. The GalXC approach seeks to optimize the activity of the RNAi pathway so that it operates in the most specific and potent fashion. Dicerna has continued to innovate and is exploring new applications of its RNAi technology beyond the liver, targeting additional tissues and enabling new therapeutic applications.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements pertaining to the Company’s collaboration with Lilly, Lilly’s future clinical development plans for LY3561774 and future candidates, future milestone and other payments to Dicerna, the potential of applying RNAi technology to other diseases, as well as our business and operations, including the discovery, development and commercialization of our product candidates and technology platform, and the therapeutic potential thereof, the success of our collaboration with partners and any potential future collaborations and our strategy, business plans and focus. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those risks identified under the heading “Risk Factors” included in our most recent Form 10-Q filing and in other future filings with the
GalXC™ is a trademark of