OliX Pharmaceuticals, Inc. engages in the research and development of treatments and the manufacture and sale of new drugs. It develops technology acid-based therapeutics based on ribonucleic acid interference (RNAi) technology. The company was founded by Dong-ki Lee on February 26, 2010 and is headquartered in Suwon-si, South Korea.

Tags

Other Diseases

Endocrinology and Metabolic Disease

Digestive System Disorders

siRNA

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Endocrinology and Metabolic Disease	5
Nervous System Diseases	3
Neoplasms	1
Infectious Diseases	1

Top 5 Drug Type	Count

siRNA	18

Top 5 Target	Count

CTGF(Connective tissue growth factor)	3
AR(Androgen Receptor)	2
MTARC1(mitochondrial amidoxime reducing component 1)	1
ASGPR(Asialoglycoprotein receptor)	1

Drugs associated with OliX Pharmaceuticals, Inc.

OLX-10010

Target

CTGF

Mechanism

CTGF inhibitors [+1]

Active Org.

OliX Pharmaceuticals, Inc.

Originator Org.

OliX Pharmaceuticals, Inc.

Active Indication

Cicatrix [+1]

Inactive Indication-

Drug Highest PhasePhase 2

First Approval Ctry. / Loc.-

First Approval Date-

OLX-72021

Target

Mechanism

AR antagonists

Active Org.

OliX Pharmaceuticals, Inc.

Originator Org.

OliX Pharmaceuticals, Inc.

Active Indication

Androgenetic Alopecia

Inactive Indication-

Drug Highest PhasePhase 1

First Approval Ctry. / Loc.-

First Approval Date-

OLX-301A

Target-

Mechanism

RNA interference

Active Org.

OliX Pharmaceuticals, Inc.

Originator Org.

OliX Pharmaceuticals, Inc.

Active Indication

Inactive Indication-

Drug Highest PhasePhase 1

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with OliX Pharmaceuticals, Inc.

NCT05643118

/ RecruitingPhase 1

Evaluation of the Safety and Tolerability of OLX10212 in Patients With Neovascular Age-Related Macular Degeneration

This is a Phase 1, multicenter, open-label, single- and multi-dose, dose-escalating study of OLX10212 in patients with neovascular age-related macular degeneration (AMD). This study is composed of 2 parts: Part A and Part B. Part A is a single ascending dose study, and Part B is a multiple ascending dose study. The primary objective is to evaluate the safety and tolerability of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD. The exploratory objectives are to evaluate the preliminary efficacy of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD, and to evaluate the pharmacokinetics (PK) of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD.

Start Date04 Jan 2023

Sponsor / Collaborator

OliX Pharmaceuticals, Inc.

[+1]

NCT05038371

/ TerminatedNot ApplicableIIT

Connective Tissue Growth Factor in Patients With Neovascular Age Related Macular Degeneration

Age-related macular degeneration (AMD) remains a leading cause of blindness in United States and can be broadly divided into two forms: non-neovascular AMD (NNVAMD) and neovascular AMD (NVAMD) AMD. Among the several mechanisms underlying AMD, hypoxia and oxidative stress have been implicated and cause upregulation of several signaling proteins. About 20% of patients with NNVAMD develop choroidal neovascularization and hence convert to NVAMD. Upregulation of vascular endothelial growth factor (VEGF) plays a critical role in conversion from NNVAMD to NVAMD.
Connective tissue growth factor (CTGF) is a polypeptide that has been shown to be overexpressed in various fibrotic disorders, suggesting its involvement in scarring. After the development of choroidal neovascularization, subretinal fibrosis may occur and result in permanent reduction of vision. An important question is, does CTGF contribute to subretinal fibrosis. An important first step in addressing this question is to determine if CTGF levels are increased in the eyes of patients with NVAMD and this is the objective of this study.
The investigators plan to measure levels of connective tissue growth factor (CTGF) in the aqueous humor of patients with neovascular age-related macular degeneration and compare to controls. Levels of VEGF will be measured as a positive control.

Start Date01 Sep 2021

Sponsor / Collaborator

The Johns Hopkins University

[+1]

NCT04877756

/ CompletedPhase 2

Phase 2a Prospective, Randomized, Double-blind, Intra-subject, Placebo-controlled, Proof of Concept Study to Evaluate the Efficacy of OLX10010 as an Adjunct Therapy to Reduce the Recurrence of Hypertrophic Scars After Scar Revision Surgery

Phase 2a, prospective, randomized, double-blind, intra-subject, placebo-controlled, proof of concept study. Approximately twenty subjects will be randomized 1:1 to one of two treatment arms:
Arm A: 2.0 mg/cm OLX10010 biweekly (every two weeks) Arm B: 5.0 mg/cm OLX10010 biweekly (every two weeks) Each treatment arm will have approximately 10 subjects. Each subject will receive both active (OLX10010) and control (placebo) treatment post-hypertrophic scar surgery biweekly (every two weeks) for a total of six doses. Dosing will occur post-surgery on Weeks 2, 4, 6, 8, 10, and 12. Post-treatment follow-up visits will occur at Weeks 18 and 24, and a long-term follow-up visit will occur at Month 12. The Patient and Observer Scar Assessment Scale (both physician and patient scales), Stony Brook Scar Evaluation Scale, Vancouver Scar Scale, and a photograph-based visual analog scale by blinded experts will be completed prior to scar revision surgery, at Weeks 2, 8, 12, 18, and 24, and at Month 12. The overall opinion responses on the physician scales of the POSAS at Week 24 will be used for primary endpoint analysis.
The total length of the linear hypertrophic scar line will be divided equally for treatment with OLX10010 and placebo, injected intradermally per centimeter (cm). The OLX10010 end and placebo end of the scar line will be separated by a 2 cm or greater distance depending on the scar length.
After the Week 24 visit, all data collected will be cleaned and all data management activities will be completed. After the database is frozen/locked, the primary endpoint efficacy analysis will be completed. If at least one of the treatment arms are shown to be appropriate to reduce recurrence of hypertrophic scars, all analyses will be performed. If the efficacy analysis of Arms A and B indicate the study doses are not as effective as expected, the sponsor may decide to add a third arm (Arm C) to explore the weekly dosing regimen. See detailed summary.
Subjects in all study arms will continue in the study until their Month 12 visit. After all subjects complete that visit, data are collected, and data management activities are completed, Month 12 data will be analyzed.

Start Date19 Aug 2021

Sponsor / Collaborator

OliX Pharmaceuticals, Inc.

[+1]

100 Clinical Results associated with OliX Pharmaceuticals, Inc.

0 Patents (Medical) associated with OliX Pharmaceuticals, Inc.

Literatures (Medical) associated with OliX Pharmaceuticals, Inc.

01 Nov 2024·DRUG METABOLISM AND DISPOSITION

Nonclinical Pharmacokinetics Study of OLX702A-075-16, N-Acetylgalactosamine Conjugated Asymmetric Small Interfering RNA (GalNAc-asiRNA)

Article

Author: Park, Shin-Young ; Ban, Jihye ; Seo, Bong Kyo ; Kim, So Hee ; Yu, Yunmi ; Lee, Dong-Ki ; Choe, Jeongyong ; Park, June Hyun ; Kim, Minkyeong

In this study, the nonclinical pharmacokinetics of OLX702A-075-16, an RNA interference (RNAi) therapeutic currently in development, were investigated. OLX702A-075-16 is a novel N-acetylgalactosamine conjugated asymmetric small-interfering RNA (GalNAc-asiRNA) used for the treatment of an undisclosed liver disease. Its unique 16/21-mer asymmetric structure reduces nonspecific off-target effects without compromising efficacy. We investigated the plasma concentration, tissue distribution, metabolism, and renal excretion of OLX702A-075-16 following a subcutaneous administration in mice and rats. For bioanalysis, high-performance liquid chromatography with fluorescence detection (HPLC-FD) was used. The results showed rapid clearance from plasma (0.5 to 1.5 h of half-life) and predominant distribution to the liver, and/or kidney. Less than 1% of the liver concentration of OLX702A-075-16 was detected in the other tissues. Metabolite profiling using liquid chromatography coupled with high-resolution mass spectrometry (LC-HRMS) revealed that the intact duplex OLX702A-075-16 was the major compound in plasma. The GalNAc moiety was predominantly metabolized from the sense strand in the liver, with the unconjugated sense strand of OLX702A-075-16 accounting for more than 95% of the total exposure in the rat liver. Meanwhile, the antisense strand was metabolized by the sequential loss of nucleotides from the 3'-terminus by exonuclease, with the rat liver samples yielding the most diverse truncated forms of metabolites. Urinary excretion over 96 h was less than 1% of the administered dose in rats. High plasma protein binding of OLX702A-075-16 likely inhibited its clearance through renal filtration. Significance Statement This study presents the first comprehensive characterization of the in vivo pharmacokinetics of GalNAc-asiRNA. The pharmacokinetic insights gained from this research will aid in understanding toxicology and efficacy, optimizing delivery platforms, and improving the predictive power of preclinical species data for human applications.

01 Oct 2023·Nucleic acid therapeutics

Development of Long Asymmetric siRNA Structure for Target Gene Silencing and Immune Stimulation in Mammalian Cells.

Article

Author: Lee, Dong-Ki ; Kim, Jaejin ; Kim, Soonkap ; Dua, Pooja ; Kang, Young Gyu

Post-transcriptional regulation of transcript abundances by RNA interference (RNAi) is a widely conserved regulatory mechanism to control cellular processes. We previously introduced an alternative siRNA structure called asymmetric siRNA (asiRNA), and showed that asiRNA exhibits comparable gene-silencing efficiency with reduced off-target effects compared with conventional siRNAs. However, to what extent the length of the guide strand affects the gene-silencing efficiency of asiRNAs is still elusive. In this study, we analyzed in detail the gene-silencing ability of asiRNAs along the guide strand length and immunostimulatory capacity of asiRNAs. We generated asiRNAs containing various guide strand lengths ranging from 25 to 29 nt, called long asiRNA (lasiRNA). We found that the gene-silencing activity of lasiRNAs decreased as the length of the guide strand increased. Nonetheless, the 3'-end overhangs that are complementary to the target gene have higher efficiency for gene silencing compared with mismatched overhangs. In addition, we found that the silencing efficiency of lasiRNAs correlates with their Ago2-binding affinity. Finally, replacing the mismatched overhang with a TLR7- or TLR9-associated immune response motif induced a toll-like receptor (TLR)-specific immune response and retained gene-silencing activity. Our findings demonstrate that lasiRNA structures can be tailored to function as bifunctional siRNA, which trigger a specific immune response combined with target gene silencing. Taken together, we anticipate that our findings provide a road map for the subsequent development of immune-stimulating lasiRNA, which bear the potential to be applied for therapeutic benefits.

01 Aug 2022·Nucleic acid therapeutics

RIG-I-Mediated Innate Immune Stimulation by Chemically Synthesized Long Double-Stranded RNAs Is Structure and Sequence Dependent

Article

Author: Kim, Jaejin ; Lee, Dong Ki ; Kang, Young Gyu ; Choe, Jeong Yong ; Lee, Kyeongmin ; Dua, Pooja

Double-stranded RNAs (dsRNAs) longer than 30 bp have not been considered desirable RNA interference (RNAi) triggering structures in mammalian cells as they nonspecifically activate innate immune response. However, in earlier studies, not only dsRNA length but also 5'-triphosphate moiety produced by in vitro transcription might have affected the stimulation of innate immune system. Herein, using chemically synthesized long dsRNAs without 5'-triphosphate, we elucidated direct relationship between length of dsRNAs and innate immune stimulation. First, we found that blunt-ended, chemically synthesized 38/40-60 bp-long dsRNAs induced retinoic acid-inducible gene I (RIG-I)-mediated innate immune response, which was suppressed by the introduction of the 2-nt 3' overhang structure. Surprisingly, we discovered that RIG-I activation by these long dsRNAs is also sequence dependent, and the sequence composition at dsRNA termini is important for RIG-I activation. In addition, we identified that long dsRNAs over 38 bp could elicit single- or dual-target gene silencing in a Dicer-independent manner. Taken together, our findings may serve as guidelines to develop an immunostimulatory RNAi trigger to exploit host's innate immune system, as well as a specific dual-gene targeting RNAi therapeutics platform without nonspecific innate immune stimulation by RIG-I.

News (Medical) associated with OliX Pharmaceuticals, Inc.

13 Feb 2025

·pmlive.com

Eli Lilly and OliX partner to advance early-stage metabolic disease candidate

Eli Lilly and RNAi therapeutics specialist OliX Pharmaceuticals have entered into a global licensing agreement to advance a phase 1 candidate for metabolic-associated steatohepatitis (MASH) and other cardiometabolic indications. MASH, also known as nonalcoholic steatohepatitis, occurs when too much fat builds up in the liver. The condition is associated with an increased risk of advanced liver disease, including liver cancer, and is expected to affect more than 19 million adults in the US by 2039. The new alliance focuses on the development and commercialisation OliX’s OLX75016, which has already demonstrated efficacy in preclinical studies in addressing MASH, liver fibrosis and other cardiometabolic diseases. Though financial details of the deal have not been disclosed, OliX said it will receive an initial payment from Lilly to complete the phase 1 clinical trial of the candidate. OliX’s chief executive officer, Dong Ki Lee, said the collaboration “marks a significant step forward for OLX75016’s development and its potential to improve treatment outcomes for a range of metabolic conditions”. “Partnering with an industry leader like Lilly enables us to advance innovative solutions that address medical needs with a strong focus on both efficacy and safety. Together, we aim to develop therapies that offer meaningful benefits for patients and drive progress in metabolic health,” he added. Lilly’s once-weekly GIP/GLP-1 receptor agonist tirzepatide, which holds approvals for weight management and type 2 diabetes, has already shown promise in patients with biopsy-proven MASH and stage 2 or 3 fibrosis. The announcement comes just one month after Lilly said it would be expanding its oncology pipeline by acquiring Scorpion Therapeutics’ PI3Kα inhibitor programme in a deal worth up to $2.5bn. STX-478 is currently being evaluated in a phase 1/2 trial as a monotherapy and as part of a combination treatment for breast cancer and other advanced solid tumours. Lilly has also already partnered with Alchemab Therapeutics this year to develop new therapies for amyotrophic lateral sclerosis. The collaboration agreement centres around Alchemab’s antibody discovery platform, which uses patient samples from those with unusually slow rates of disease progression to identify antibodies associated with resilience.

Phase 1License out/in

11 Feb 2025

·www.pharmaceutical-technology.com

Eli Lilly pledges $630m on OliX’s Phase I RNA candidate for MASH

MASH is a form of non-alcoholic liver disease. Credit: SEBASTIAN KAULITZKI / SCIENCE PHOTO LIBRARY via Getty Images. Eli Lilly is paying up to $630m for a Phase I metabolic-associated steatohepatitis (MASH) candidate from South Korea-based biotech OliX Pharmaceuticals, diversifying its pipeline for the disease with an RNA-based asset. Known as OLX75016, the candidate is an antisense RNAi oligonucleotide developed using OliX’s therapeutic RNAi platform. OliX focuses on developing therapies by inhibiting the expression of genes that cause disease through its technology. Lilly will make an initial payment to complete a Phase I trial that kicked off in Australia in February 2024. The study, set to enrol 58 healthy subjects, will evaluate the safety and tolerability of single and multiple ascending doses of OLX75016. While the ongoing Phase I trial is investigating OLX75016 for the treatment of MASH and liver fibrosis, the candidate is also in preclinical development for the treatment of obesity in combination with glucagon-like peptide-1 receptor agonist (GLP-1RA) semaglutide. The $630m agreement includes an upfront payment, as well as development and commercial milestones, according to a securities filing on Korea’s stock exchange. The deal establishes Lilly with a diverse pipeline to target the large market opportunity within MASH. The drugmaker’s blockbuster GLP-1RA drug tirzepatide – the active ingredient in Mounjaro and Zepbound – is being investigated as a treatment for MASH. In a Phase II trial (NCT04166773) readout in June 2024, Lilly reported that 51% to 54.9% of patients achieved an improvement in fibrosis without worsening of their disease at 52 weeks, compared to 21.3% to 25.2% on placebo. Lilly also invested in its nucleic acid infrastructure, unveiling a $700m R&D centre in Boston, US, in August 2024 – a facility focused on developing novel RNA-based and DNA-based medicine. MASH is a form of non-alcoholic liver disease, which describes a spectrum of conditions like steatosis, inflammation, and fibrosis marked by fat buildup in the liver. The disease, also associated with comorbidities such as obesity and diabetes, has been notoriously difficult to treat. The MASH treatment landscape has seen multiple trial failures and setbacks, with companies such as AstraZeneca terminating the development of its MASH therapies. In June 2023, the US Food and Drug Administration (FDA) rejected Intercept Pharmaceuticals’ new drug application (NDA) for its MASH therapy, obeticholic acid. The FDA cited concerns over the therapy’s safety profile, especially the potential risks of causing liver injury and diabetes. In March 2024, Madrigal Pharmaceuticals’ Rezdiffra (resmetirom), a type of thyroid hormone receptor beta (THR-beta) agonist, became the first and only approved therapy that does not require a liver biopsy for the treatment of patients with MASH and Stage 2–3 fibrosis.

Phase 1Phase 2Oligonucleotide

10 Feb 2025

·pipelinereview.com

OliX and Lilly Enter Global Licensing Agreement for MASH and Other Cardiometabolic Indications

– OliX to Develop OLX75016 for MASH and Other Cardiometabolic Indications – OliX to Receive Initial Payment and Complete Phase 1 Clinical Trial in Australia – OLX75016: A GWAS-derived therapeutic targeting MASH, liver fibrosis, and other cardiometabolic indications SEONGNAM, South Korea I February 10, 2025 I OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced a global licensing agreement with Eli Lilly and Company (“Lilly”). This collaboration focuses on the development and commercialization of OliX’s OLX75016, a Phase 1 candidate primarily targeting metabolic-associated steatohepatitis (MASH) and other cardiometabolic indications. OliX will receive an initial payment from Lilly to complete the Phase 1 clinical trial, currently underway in Australia. OLX75016 derives from a robust target discovered through a genome-wide association study (GWAS), a research approach for identifying genomic variants in large populations. In preclinical studies, OliX has demonstrated the efficacy of OLX75016 in addressing both MASH and liver fibrosis, as well as other cardiometabolic indications. OliX is currently developing OLX75016 as a novel therapeutic in treating MASH accompanied by liver fibrosis, and other cardiometabolic indications. Dong Ki Lee, Ph.D., CEO of OliX Pharmaceuticals, commented, “This collaboration with Lilly marks a significant step forward for OLX75016’s development and its potential to improve treatment outcomes for a range of metabolic conditions – MASH and other cardiometabolic indications. Partnering with an industry leader like Lilly enables us to advance innovative solutions that address medical needs with a strong focus on both efficacy and safety. Together, we aim to develop therapies that offer meaningful benefits for patients and drive progress in metabolic health.” About OliX Pharmaceuticals OliX Pharmaceuticals is a clinical-stage pharmaceutical company developing therapeutics against a variety of disorders by down-regulating the expression of disease-causing genes based on its own proprietary RNAi technology. The Company’s core RNAi platform, asymmetric siRNA (asiRNA), is a unique gene silencing technology based on RNA interference (RNAi), which is considered the most efficient gene silencing technology. Utilizing this proprietary asiRNA technology, OliX has developed cell penetrating asiRNA (cp-asiRNA), a therapeutic RNAi platform to effectively target diseases including hypertrophic scarring, dry and wet age-related macular degeneration (AMD), and subretinal fibrosis. OliX has also developed another therapeutic RNAi platform, GalNAc-asiRNA, to target a variety of liver diseases. Learn more: https://www.oliXpharma.com/eng SOURCE: OliX Pharmaceuticals

License out/inPhase 1

100 Deals associated with OliX Pharmaceuticals, Inc.

100 Translational Medicine associated with OliX Pharmaceuticals, Inc.

Corporation Tree

Boost your research with our corporation tree data.

view full example data

Pipeline

Pipeline Snapshot as of 25 Mar 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Discovery

Preclinical

IND Approval

Phase 1

Phase 2

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

OLX-10010 ( CTGF )	Cicatrix, Hypertrophic More	Phase 2
OLX-702A ( MTARC1 )	Nonalcoholic Steatohepatitis More	Phase 1
OLX-301A	Macular Degeneration, Age-Related, 1 More	Phase 1
OLX-72021 ( AR )	Androgenetic Alopecia More	Phase 1
OLX-104C ( AR )	Alopecia More	IND Approval

Deal

Boost your decision using our deal data.

view full example data

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Profit

Explore the financial positions of over 360K organizations with Synapse.

view full example data

Grant & Funding(NIH)

Access more than 2 million grant and funding information to elevate your research journey.

view full example data

Investment

Gain insights on the latest company investments from start-ups to established corporations.

view full example data

Financing

Unearth financing trends to validate and advance investment opportunities.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis