Rznomics is set for its first test to prove the efficacy of its RNA editing technology. After booking losses of around 100 billion won, attention is on whether the company can secure additional technology transfer momentum with global big pharma through this clinical data.
Rznomics plans to deliver an oral presentation of interim results from the phase 1b/2a trial of its liver cancer drug candidate RZ-001 at the American Association for Cancer Research Annual Meeting (AACR 2026), to be held in San Diego from the 17th to the 22nd.
The announcement is significant as the first clinical data showing whether the company's core technology, an RNA ribozyme-based editing platform, works inside actual patients. RNA editing technology remains in the early stages of development in the global market, leaving human clinical data scarce.
The industry views this announcement as a key indicator for judging the commercialization potential of Rznomics' platform technology, beyond the progress of an individual candidate.
Kim Min-jung, an analyst at DS Investment & Securities, said, "Having already confirmed safety in a phase 1 trial in glioblastoma patients, if efficacy is verified this time, it could lead to a reassessment of the platform's overall value."
Based on the closing price on the 2nd (199,000 won), Rznomics' market capitalization is about 2.7728 trillion won. Singapore's Wave Life Sciences saw its share price jump about 147% in a single day right after it released positive phase 1 results for its obesity drug candidate WVE-007 in Dec. last year.
◇ Standard-of-care ORR at 30% is the benchmark… Gauging the standalone platform's efficacy
The approach mainly used by global RNA editing corporations leverages existing intracellular editing mechanisms. However, critics have noted that because this method must rely on the cell's inherent editing capacity, therapeutic efficiency may be constrained.
In response, Rznomics built a platform that directly edits target RNA for therapy. In theory, it is expected to offer higher efficiency and the potential to expand into various diseases.
At this meeting, clinical data will be disclosed for RZ-001 in combination with the first-line standard-of-care for advanced liver cancer, Tecentriq and Avastin. According to the clinical design disclosed so far, the study proceeds through a three-step dose-escalation process to identify the optimal dose.
Each cohort consists of 15 patients, and the study employs a stepwise 3+3 design that first verifies safety in a small initial cohort before moving to the next stage. Given this, the announcement is expected to include basic safety readouts from the low-dose range.
The key metric the market is watching is the objective response rate (ORR), which can gauge efficacy. For ORR, which reflects the degree of tumor shrinkage, the roughly 30% figure recorded by the current standard-of-care (Tecentriq and Avastin) is expected to serve as a main benchmark.
◇ Expanding the technology base with circular RNA… "Potential for CAR-T development"
RNA-based therapeutics are cited as one of the next-generation modalities because they can directly control disease drivers that small molecules and antibodies have struggled to reach. The commercialization of the spinal muscular atrophy treatment Spinraza and the hyperlipidemia treatment Leqvio is cited as evidence that RNA technologies can expand beyond rare diseases into chronic conditions.
However, in indications where existing therapies have already secured sufficient clinical utility, some view the economics or necessity of RNA therapeutics as relatively lower.
Considering this market environment, Rznomics is broadening its technology base with a circular RNA platform that can complement the limitations of conventional linear RNA.
On the 27th of last month, the company published research results on circular RNA delivery technology in the international journal ACS Biomaterials Science & Engineering. The study verified whether circular RNA can be selectively delivered to the spleen via intravenous administration.
According to the results, circular RNA complexed with a polymer-based carrier reached the spleen about 10 times more than the liver after intravenous administration. Compared with conventional linear RNA, the duration of protein expression was about twice as long, and delivery efficiency to T cells, an immune cell type, was about three times higher.
The industry is focusing on the potential of this research to alleviate the chronic "liver tropism" issue of conventional lipid nanoparticle (LNP) approaches. If selective delivery to the spleen becomes possible, it could lead to the development of CAR-T Therapy that manipulates immune cells directly in the body.
Lee Sung-uk, CEO of Rznomics, said, "We plan to continuously expand the scope of application, including CAR-T, through additional research going forward."
◇ First revenue from the "Lilly effect"… Aiming for additional technology transfer in 2029
Rznomics posted its first revenue of about 7.9 billion won through a joint research and technology transfer agreement signed last year with Eli Lilly worth a total of $1.334 billion (about 1.9 trillion won).
However, with one-off factors such as expanded research and development (R&D) investment and listing-related expense reflected, the operating loss widened from about 12.9 billion won the previous year to around 14.6 billion won.
In particular, loss before income taxes expanded to about 102.7 billion won. Of that, 88.6 billion won is analyzed to be non-cash items with no actual cash outflow, such as derivative valuation losses.
This stems from accounting characteristics that arise when redeemable convertible preferred shares (RCPS) and the like convert into common stock. Under current accounting standards, the value of the conversion right is linked to the share price, resulting in an "accounting illusion" where book losses grow as corporate value rises.
On Dec. 18 last year, at listing, Rznomics opened at 90,000 won, the upper limit of the daily price band relative to its offer price of 22,500 won, and on Jan. 9 this year, it crossed 190,000 won on a closing basis. After temporary volatility from profit-taking, the stock has risen since last month and is now hovering around the 200,000-won level.
The industry expects that securing stable revenue sources, such as additional technology transfers, will be a key task to ease future earnings volatility.
The company plans to push for technology transfer of RZ-001 by 2029 as soon as it secures clinical data. It has signed confidentiality disclosure agreements (CDA) with two domestic and overseas pharmaceutical and biotech corporations and is in related discussions.
The company also plans to expand the scope of collaboration with Lilly to gradually broaden its revenue base. A company official noted, "Beyond retinal diseases, we are keeping open the possibility of joint research with Lilly in our key pipelines, including oncology and Alzheimer's."