[Edaily Reporter KIM SAE-MI ] “We expect ‘OASIS-DUO,’ which we are unveiling for the first time today, to become a next-generation treatment strategy that transcends the limitations of existing single-agent long-term therapies.”
Lee Dong-ki, CEO of Olix Pharmaceuticals, Inc.(226950), made this statement while unveiling OASIS-DUO for the first time at the “2026 R&D Day” event held at the Korea Exchange in Yeouido, Seoul, at 2:00 p.m. on the 14th.
OASIS-DUO is an RNA interference (RNAi) platform designed to deliver a single drug to two different organs to suppress target genes in each organ separately. A lipid ligand that migrates to adipose tissue is attached to one short interfering RNA (siRNA) unit, while GalNAc, which is delivered to the liver, is attached to the other; the two units are then connected by a cleavable linker. When administered into the body, the linker cleaves, allowing each siRNA to travel to the liver and adipose tissue, respectively, to silence different genes.
Olix Pharmaceuticals, Inc. explained that subcutaneous injection of OASIS-DUO into mice confirmed simultaneous suppression of target genes in both the liver and adipose tissue. The company’s vision is to treat diseases where pathological conditions in the liver and adipose tissue are intertwined—such as obesity and metabolic-associated steatohepatitis (MASH)—with a single drug. The company is currently working to expand this liver-adipose tissue combination to other organ pairs, such as the kidneys and muscles, and is also researching a platform that links three siRNAs. However, the results released this time represent an early-stage proof-of-concept (PoC) in mice, confirming the suppression of model gene expression.
CEO Lee stated, “I believe this will become a key drug development approach for complex diseases such as metabolic disorders,” adding, “Since OASIS-DUO is virtually the first of its kind in the world, it will serve as a springboard for developing first-in-class innovative drugs without falling behind the frontrunners.”
The new data that drew investors’ attention were the results of obesity monkey trials for the obesity treatment candidate “OLX501A.” OLX501A is an siRNA candidate that promotes lipolysis by inhibiting the ALK7 gene, which is expressed in fat cells.
Olix Pharmaceuticals, Inc. administered OLX501A and an unnamed clinical-stage competitor substance to obese monkeys at a dose of 3 mg/kg each, via subcutaneous injection once a month for a total of two doses. On day 70 of treatment, the ALK7 gene inhibition rate measured in subcutaneous fat was 89% for OLX501A and 88% for the competitor substance, showing similar results. In contrast, visceral fat assessed via magnetic resonance imaging (MRI) on day 49 of treatment decreased by 29.2% from baseline in the OLX501A group. The reduction rate in the competitor substance group was 10.0%, while the control group showed an 11.1% increase.
The expression of “ADRB3,” a pharmacodynamic biomarker indicating lipolysis following ALK7 inhibition, also remained at a higher level in the OLX501A group compared to the competitive compound group. The company highlighted the potential for OLX501A to become a best-in-class new drug, citing that the reduction in visceral fat was approximately three times greater than that of the competitive compound, despite similar ALK7 inhibition rates in subcutaneous fat.
CEO Lee explained, “We confirmed that the efficacy in reducing visceral fat in obese monkeys was significantly superior to that of a competitor’s compound currently in the clinical trial phase,” adding, “In summary, we have confirmed strong potential for the drug to be developed as a best-in-class treatment.”
However, these results are from an interim analysis. The number of animals per group, the margin of error, and the statistical significance of the differences between OLX501A and the competitor compound have not been disclosed. Additionally, the ALK7 inhibition rate was measured in subcutaneous fat, not visceral fat.
Olix Pharmaceuticals, Inc. plans to evaluate final body composition—including body fat and muscle mass—using dual-energy X-ray absorptiometry (DEXA) between days 112 and 119 of treatment. The company did not specify when the results will be released. The key question in the final analysis will be whether the “qualitative weight loss that reduces fat while preserving muscle”—a point the company has emphasized—was replicated in monkeys.
OLX501A is currently undergoing preclinical studies and production of candidate compounds for clinical trials, with the goal of entering clinical trials in the first half of 2027. The company expects that technology transfer will be possible even during the nonclinical or early clinical stages.
The dual-target program for obesity and metabolic diseases has also taken shape. Olix Pharmaceuticals, Inc. has finalized the target combination and completed drug design, and is currently conducting monkey trials. While specific targets and data were not disclosed due to the agreement with Lilly, the company plans to secure a large-animal data package—including pharmacokinetics (PK) and pharmacodynamics (PD)—by the next quarter.
“OLX702A,” a MASH treatment licensed to Lilly, completed the final patient visit for its Phase 1 clinical trial in Australia last May, and the company expects to receive the Clinical Study Report (CSR) in the second half of this year. CEO Lee stated, “We confirmed industry-leading results in terms of efficacy in reducing fatty liver and the duration of that effect,” adding, “In accordance with the agreement, we expect our partner to conduct subsequent clinical trials.”
The hair loss treatment “OLX104C” is currently undergoing Phase 1b and 2a clinical trials in Australia. Olix Pharmaceuticals, Inc. has completed a single-dose Phase 1a trial and is currently conducting a Phase 1b trial involving a total of three doses administered at 28-day intervals. The company plans to conclude the Phase 1b trial in the second half of this year and complete the Phase 2a trial by 2027. CEO Lee said, “To date, no side effects related to systemic exposure or changes in male hormone levels have been observed,” adding, “The efficacy of hair regrowth will be evaluated in the Phase 2a trial.”
Separately, Olix Pharmaceuticals, Inc. is developing “OLX104CC,” a cosmeceutical product utilizing the same technology, as a hair tonic under the “Yuberna” brand, with plans to officially launch it in the second half of this year. CEO Lee revealed, “We developed the cosmeceutical product OLX104CC under the Yuberna brand and conducted pilot production and field tests last year,” adding, “We received positive feedback from a large number of hair loss patients.”
Regarding the central nervous system (CNS) platform, the company presented the possibility of delivering siRNA to the brain via subcutaneous or intravenous injection, rather than through invasive intrathecal administration. Olix Pharmaceuticals, Inc. administered an antibody-RNA conjugate—combining siRNA with a blood-brain barrier (BBB) shuttle from VECT Co., Ltd.—to mice and confirmed the suppression of target genes in the cerebral cortex, striatum, hippocampus, and midbrain. In particular, the company explained that subcutaneous injections showed suppression patterns similar to those of intravenous injections. Olix Pharmaceuticals, Inc. plans to verify efficacy in primates following candidate optimization and the establishment of a manufacturing process.
Among the currently disclosed programs for expansion into extrahepatic tissues, OLX501A has reached the primate stage, while CNS and OASIS-DUO are still in the rodent phase. Attention is focused on whether the platform’s scalability, as presented by Olix Pharmaceuticals, Inc., will extend beyond simple target gene suppression to demonstrate therapeutic efficacy and success in human clinical trials.
CEO Lee emphasized, “Although our market capitalization, which stood at approximately 960 billion won during last year’s R&D Day, rose to about 4 trillion won based on the all-time high, it has since corrected to the 2.5 trillion to 2.6 trillion won range.” He added, “I am deeply sorry for the recent stock price situation, and we will continue to share our achievements to create an opportunity to surpass the all-time high.”
