Ionis Pharmaceuticals and Suzhou Ribo Life Science agreed to a collaboration and license agreement to develop and commercialize RNA-targeted therapeutics in China. The deal gives Ribo a license to commercialize two Ionis Generation 2+ antisense drugs for metabolic and cancer indications and an option to a third, prespecified Generation 2+ antisense candidate. Financial details were not disclosed, except to confirm that Ionis will receive an up-front payment and equity stake in Ribo. Ionis retains all rights to its single-stranded RNA interference (ssRNAi) technology and drugs developed through the collaboration outside China.
Ribo will in addition carry out an R&D program to identify drugs based on Ionis’ ssRNAi technology. It will have an option to license each drug for the Chinese market in return for an up-front license fee, plus development, regulatory, and commercialization milestones. Ionis will also have a royalty-free license to data and IP created through the collaboration.
“Ribo is the ideal partner for us in China,” said Brett Monia, Ph.D., svp of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals. “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,” added Liang Zicai, Ph.D., 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 ssRNA-like antisense drugs that harness the power of the RNAi pathway.”
Ionis is developing an in-house and partnered pipeline of RNA-targeted drugs for severe and rare diseases. The company's first approved product, Spinraza™ (nusinersen), was cleared by the FDA in December 2016 for treating spinal muscular atrophy. The drug is being developed globally by Biogen, which exercised its option to take over the nusinersen program in August 2016.
Ionis’ late-stage development pipeline includes volanesorsen, which is being developed through its wholly owned subsidiary Akcea Therapeutics for treating familial chylomicronemia syndrome and familial partial lipodystrophy. Akcea filed for its $100 million IPO last month. Ionis' Phase III-stage clinical candidate IONIS-TTRRx is partnered with GlaxoSmithKline (GSK) for the treatment of transthyretin (TTR) amyloidosis.
Since mid-December, Ionis has earned over $100 million in milestone payments through antisense drugs collaborations with Bayer and with AstraZeneca. In January, Ionis and Akcea Therapeutics inked a potentially $1 billion equity and cash deal with Novartis for cardiovascular disease antisense drugs.
Kunshan City-based Ribo is focused on developing RNAi drugs and is developing a clinical and preclinical pipeline of siRNA candidates against diseases that include hepatitis B, breast and liver cancers, hyperlipidemia, liver fibrosis, and HIV.
In 2012, Ribo and Quark Pharmaceutical established a joint venture—Kunshan RiboQuark Pharmaceutical Science and Technology, to progress R&D and clinical development of RNAi therapeutics. The joint venture's pipeline includes QPI-1007, an anti-NAION (nonarteritic anterior ischemic optic neuropathy) siRNA drug, and a candidate for APACG (acute primary angle-closure glaucoma), which are both in clinical development in China.