OliX Pharmaceutical’s RNAi (RNA interference) technology is getting the notice of the global pharmaceutical industry as it prepares various clinical trials using its patented technology to treat disease with high unmet medical needs.
|Olix Pharmaceutical’s CEO Lee Dong-ki explains the company’s drug candidates and its future goals, during a recent interview with Korea Biomedical Review at the company’s headquarters in Suwon, Gyeonggi Province.|
RNAi medicines, which target genes that cause the diseases rather than inhibiting the protein of the disease, have recently received high expectations to open a new era of treating diseases.
As proof, the U.S. Food and Drug Administration approved Alnylam Pharmaceutical’s Onpattro, a hereditary transthyretin amyloidosis treatment, last year. While several other companies are conducting clinical trials for the commercialization of subsequent RNAi drugs, OliX Pharmaceutical is the fastest developer in Korea.
The company, founded in February 2010 by Lee Dong-ki, had been developing RNAi technology since 2004 and acquired a patent related to RNA interference technology in 2009.
“OliX is a company that develops drugs for various intractable diseases based on RNAi,” Lee said in a recent interview with Korea Biomedical Review.
Asked about the difference between RNAi-based therapy and other conventional ones, Lee said that RNA is the latest third-generation technology.
“The medicine we talk about is divided into three generations,” Lee said. “The first generation revolves around small molecules, the second generation on antibodies, and the third generation includes genomic drugs based on DNA or RNA nucleic acids, including oligonucleotide RNA, which is the company’s platform.”
The advantage of the third generation compared to the first and second generation is that while the previous drugs only targeted proteins that have already been produced, the company’s platform acts on the mRNA (messenger RNA), which conveys genetic information from DNA to the ribosome, thereby preventing the production of disease-causing proteins, he noted.
“It is known that there are 25,000 human genes, while first and second generations can only target 15 percent of these genes, the third generation treatment can suppress all the proteins produced in the human body,” Lee said.
OliX expects that the company’s technology will develop treatments for some genes that the researchers have found to have caused the disease but had no treatment due to the limitations in targeting the specific gene.
As of now, the company is focusing on local organs and skin diseases such as ophthalmology and lung disease.
According to Lee, a majority of the company’s pipeline is on treatments related to an ophthalmologic disease.
OLX301A, which is a first-in-class therapy that targets dry and wet age-related macular degeneration (AMD), has demonstrated excellence in both pre-clinical trials, and the company plans to apply for phase 1 clinical trials to the FDA in the second half of this year.
“Our company has many networks regarding ophthalmologic diseases both at home and abroad,” Lee said. “We plan to apply for additional trials for various other indications regarding the drug candidate by next year as the company plans to focus a lot on the ophthalmology in the short term.”
OliX’s most advanced treatment candidate is OLX101, a skin-scar therapeutic. Hugel has an exclusive license in the Asian region and is now conducting phase 2 clinical trials in Korea, while the company is conducting phase 1 clinical trials in the U.K. to accelerate its launch in the global market.
“OliX also has drug candidates in treating lung fibrosis and is in the process of discovering candidates for immunoncology,” Lee said. “Also as most oligonucleotide companies are developing drugs targeting the liver, OliX is also developing a substance in the area.”
To expand its candidates on a global level, the company has established a branch in the U.S.
“Our company's largest market is expected to be in the U.S., and one of the reasons for establishing a branch office is our commitment to recruiting talented American researchers,” Lee said. "OliX has recruited talented people such as Wei Li as our chief development officer to run the U.S. unit.”
As almost all the big pharmaceutical companies are gathered in Cambridge, Mass., we plan to use our branch office in meeting various multinational pharmaceutical companies and maintaining a close relationship, he said.
OliX’s ultimate goal is to get a deal for its platform technology with the multinational pharmaceutical companies through the U.S. branch.
Another reason the company is focusing on the international market is that there are not many RNA-specialized companies in Korea.
“Although the Korean government seems to give much consideration to gene or cell therapy companies, there are no specific moves related to RNA companies,” Lee said. “I guess one of the reasons is there are not many companies, and there is a limit to our efforts to move the Ministry of Food and Drug Safety to pay more attention to our field.”
It does not mean that there are no regulations in Korea, but the FDA and the U.K.’s National Health Service have regulations on the nucleic acid that differ significantly from the regulatory list of Korea, he said.
Therefore, the company’s strategy is to study and pass regulations in the U.S. and U.K. and come back to Korea with an approved treatment.
Lee stressed that the company plans to accelerate its global presence this year.
“I think OliX has a strong position in Asia. Therefore, we will focus on enhancing global presence this year,” Lee said. “To this end, we will create a unique presence by targeting diseases with high unmet medical needs.”
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