HONG KONG – A deal inked between Carlsbad, Calif.-based Ionis Pharmaceuticals Inc. and Kunshan, China-based Suzhou Ribo Life Science Co. Ltd. will ease the U.S. company's entry into the Chinese innovative drug market, while Ribo picks up a license for two antisense drugs – and an option for a third – aimed at treating metabolic diseases and cancer.

"There is a huge unmet need in China for the treatment of diseases that can be satisfied through nucleic acid therapy, and antisense therapy is in the field of nucleic acid therapy," Zicai Liang, CEO of Suzhou Ribo Life Science, told BioWorld Asia.

As part of the deal, Ribo will be responsible for the R&D work to identify drugs that can use Ionis' technology. Following the identification of a candidate, Ribo can exercise its option to license the drug. Ionis is eligible to receive an undisclosed up-front payment along with Ribo equity. The U.S. firm would also be in line for regulatory and commercial milestone payments as each drug advances and is eligible to receive royalties on net sales of each drug.

"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," he added, citing the company's "excellent progress in its RNAi therapeutic programs." The partnership "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."

Ionis will hang onto the rights to develop and sell the drugs outside of China.

"Ionis as a company has single-handedly developed a method for the antisense therapy to work and cure human diseases," said Liang. "It has founders who dedicated their lives to this new therapeutic field. On one hand, they developed the world's largest nucleic acid therapeutics drug pipeline, and on the other hand, they are visionaries, looking far into the long run."

pace of new drugs accelerating

China has been supportive in paving regulatory pathways for foreign innovative drugs, according to Liang.

"The regulatory environment in China is improving with the market pace, and it is paving the way for innovative drugs. Introduction of new drugs is accelerating rather than slowing down," said Liang.

And Ribo's right to sell the Ionis-licensed drugs out of China could pay off handsomely. Globally, the market for antisense gene therapy along with the RNAi therapeutics market is anticipated to reach $4.58 billion by 2022, according to U.S.-based research company Grand View Research Inc.

Part of the reason is that antisense gene therapy is emerging as one of the most beneficial therapeutics for various diseases such as hemorrhagic fever, cancer, cystic fibrosis, renal diseases, HIV and AIDS, spinal muscular atrophy and cardiovascular diseases.

In cancer, for example, antisense therapy works by a synthesized strand of nucleic acid binding to the specific messenger RNA (mRNA) and shutting off the genes that are causing the expression of tumor cells and other cancer-related genes.

Big pharma firms also are taking further interest in developing RNAi-based therapeutics, looking at RNAi therapeutics as one of the options to restock exhausted pipelines.

In January, Ionis signed a deal with Novartis AG to develop and commercialize two early stage next-generation therapies to reduce cardiovascular risk in patients with underlying lipid disorders. That good news came after spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) received FDA approval in December. (See BioWorld Today, Dec. 28, 2016.)

Spinraza was the first drug approved to treat children and adults with SMA, a rare and fatal genetic disease affecting muscle strength and movement. Partner Biogen Inc. reported in its first-quarter earnings this week that sales of Spinraza totaled $47 million for the three months ending March 31, blasting past consensus estimates of $16 million. (See BioWorld Today, April 26, 2017.)

Ionis has also reported positive data from two phase III trials for its triglyceride-lowering drug, volanesorsen.

Meanwhile, Ribo, which closed a $20 million series A in 2015, raised $39 million in a series B funding round, disclosed last month, led by China's State Development and Investment Corp.