On April 18, 2017 Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported a collaboration and license agreement with Suzhou Ribo Life Science Co., Ltd. (Ribo) to develop and commercialize RNA-targeted therapeutics in China (Press release, Ionis Pharmaceuticals, APR 18, 2017, View Source [SID1234518609]). Ionis granted Ribo a license for the right to commercialize in China two Ionis Generation 2+ antisense drugs in metabolic disease and cancer and an option to license a third pre-specified Generation 2+ antisense drug. In addition, Ribo will be responsible for conducting a multi-year research and drug discovery program to identify drugs that utilize Ionis’ ssRNAi technology. Ionis will receive an undisclosed up-front payment and equity in Ribo. Ionis retains the rights to develop and commercialize ssRNAi technology and all drugs under the collaboration outside of China. Schedule your 30 min Free 1stOncology Demo! Ionis Pharmaceuticals
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"The need for new medicines in China is growing rapidly, especially for metabolic diseases and certain genetically-defined cancers," said Brett Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals. "Ribo is the ideal partner for us in China. Ribo has made excellent progress in its RNAi therapeutic programs and we believe we will benefit from their drug development and regulatory expertise in China. We believe that our partnership with Ribo maximizes the value of our drugs in this collaboration by leveraging the clinical data generated in China to support the overall global development plan for each drug."
"We are committed to advancing new RNA-targeted therapeutics, like RNAi and antisense, to patients with high unmet medical need in China," said Liang Zicai, founder and Chairman of Ribo. "Ionis has made significant breakthroughs showing that chemically modified single-stranded oligonucleotides can activate the RNAi pathway. We believe that this work is the foundation for a robust drug discovery platform that takes advantage of using single-stranded RNA-like antisense drugs that harness the power of the RNAi pathway. We are excited by the opportunity to contribute our expertise and resources to advancing this promising technology."
Following the identification of a development candidate, Ribo may exercise its option to license each drug by paying Ionis a license fee. For each drug that Ribo licenses, Ribo will be responsible for all development and commercialization activities and costs in China. Ionis is eligible to receive development, regulatory and commercial milestone payments as each drug advances. In addition, Ionis is eligible to receive royalties on net sales of each drug.
Ribo will provide Ionis a royalty-free license to the data and intellectual property created under the collaboration.
ABOUT RNAi AND ssRNAi
As it is known to occur in nature, the RNAi pathway is mediated by short double-stranded RNA oligonucleotides called "small interfering RNAs" or "siRNAs." To date, efforts aimed at harnessing this pathway to silence disease-causing proteins have used double-stranded siRNAs. Double-stranded siRNA drugs require complex formulations or special conjugates to achieve sufficient delivery for systemic activity. This requirement has severely limited the development of safe and effective drugs that work through the RNAi pathway. In contrast, single-stranded RNA-like antisense drugs can be administered subcutaneously and distribute to tissues without the need for complex formulations or special conjugates. Using its expertise in oligonucleotide chemistry and design, Ionis has discovered strategies for designing single-stranded oligonucleotides that act through the RNAi mechanism. With further development, these chemically modified, single-stranded, RNA-like oligonucleotides have the potential for improved properties for systemic administration while harnessing the advantages of the RNAi mechanism.