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

Endocrinology and Metabolic Disease

Skin and Musculoskeletal Diseases

Other Diseases

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
Infectious Diseases	1
Neoplasms	1

Top 5 Drug Type	Count

siRNA	18

Top 5 Target	Count

AR(Androgen Receptor)	2
CTGF(Connective tissue growth factor)	2

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-702A

Target-

Mechanism

RNA interference

Active Org.

OliX Pharmaceuticals, Inc.

Originator Org.

OliX Pharmaceuticals, Inc.

Active Indication

Metabolic dysfunction-associated steatotic liver disease [+2]

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 Oct 2023·Nucleic acid therapeutics

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

Article

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

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, Kyeongmin ; Lee, Dong Ki ; Dua, Pooja ; Choe, Jeong Yong ; Kang, Young Gyu

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.

01 Dec 2019·Molecular Therapy - Methods & Clinical DevelopmentQ2 · MEDICINE

ALPPL2 Is a Potential Diagnostic Biomarker for Pancreatic Cancer-Derived Extracellular Vesicles

Q2 · MEDICINE

ArticleOA

Author: Lee, Dong Ki ; Shin, Hye-Su ; Park, Sungho ; Jung, Sang Baek ; Dua, Pooja

Pancreatic cancer is an aggressive malignancy that often goes undiagnosed in the early stages. Non-invasive, early, and accurate diagnosis is therefore undoubtedly the "holy grail" of pancreatic cancer research. However, despite extensive research efforts, there is no definitive biomarker for this cancer. Previously, we identified alkaline phosphatase placental-like 2 (ALPPL2) as a diagnostic biomarker for pancreatic ductal adenocarcinoma and developed a 2'-fluoro modified RNA aptamer toward it. In this study, we show that ALPPL2 is present in pancreatic cancer extracellular vesicles (EVs) and therefore has potential application in liquid biopsy-based diagnostic strategies. We also developed ALPPL2 direct and sandwich aptamer-linked immobilized sorbent assay (ALISA) for EVs, which could sensitively and specifically detect the protein. We believe that our ALISA format may have a potential diagnostic utility in screening pancreatic-cancer-derived EVs.

News (Medical) associated with OliX Pharmaceuticals, Inc.

30 Aug 2024

·www.businesswire.com

RNAi Technologies and Global Markets Report 2024, Featuring Revenue Data for 2021- 2023, Estimated Figures for 2024, Forecasts for 2029 and Projected CAGRs through 2029 - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "RNAi Technologies and Global Markets" report has been added to ResearchAndMarkets.com's offering. The global market for RNAi technology is estimated to increase from $2.1 billion in 2023 to reach $4.8 billion by 2029, at a compound annual growth rate (CAGR) of 14.8% from 2024 through 2029. The scope of this study encompasses an investigation of the ribonucleic acid interference (RNAi) technology market. The report analyzes RNAi technology based on technology type: tools and reagents, diagnostics, therapeutics, and agriculture. The publisher determines the current market status in each segment, examines its impact on future needs, and presents growth forecasts over the next six years. The report provides a detailed analysis of the market's drivers, restraints, challenges, and opportunities. It includes company profiles of key players, with detailed information about their business segments, financials, product portfolios, and recent developments. The report also provides detailed information on emerging technologies and new developments in the market; examines the regulatory landscape; provides a patent and pipeline analysis; and discusses ESG developments, the investment outlook, and deals within the market. Report Scope: 26 data tables and 53 additional tables An overview of the global market for RNAi technologies Analysis of global market trends, featuring revenue data for 2021- 2023, estimated figures for 2024, forecasts for 2029, and projected CAGRs through 2029 Evaluation of the current market's size and revenue growth prospects, along with a market share analysis by type, therapeutic area, delivery method, and region A look at innovations, technological advances, and recent product launches in the RNAi market Analysis of the industry's regulatory framework and policies A discussion of ESG challenges and ESG practices in the RNAi technologies industry An analysis of the key companies' market shares, proprietary technologies, and strategic alliances Company profiles of major players within the industry, including Alnylam Pharmaceuticals Inc., Novartis AG, Thermo Fisher Scientific Inc., and Revvity Inc. Key Topics Covered: Chapter 1 Executive Summary Chapter 2 Market Overview Background: Genetic Material RNA Interference Overview RNA Interference: Mechanism of Action Initiation Phase Effector Phase Small Interfering RNAs (siRNAs) MicroRNAs (miRNAs) Small Hairpin RNAs (shRNAs) Chapter 3 Market Dynamics Market Drivers Increasing Prevalence of Chronic Disease Increasing Approval of RNAi Products Advancements in RNAi Delivery Systems Increasing Strategic Initiatives Convenient Dosing Market Restraints High Treatment Costs Lack of Awareness Market Opportunities Expansion of Disease Targets Combination Therapies Untapped Genetic Targets Chapter 4 Emerging Technologies and Developments Therapeutics Diagnostics Circulating miRNA Diagnostics for Non-invasive Health Monitoring Agriculture Chapter 5 Pipeline Analysis Chapter 6 Patent Analysis Chapter 7 Market Segmentation Analysis Market Analysis by Technology RNAi Therapeutics Market, by Region RNAi Therapeutics Market, by Therapeutic Area RNAi Therapeutics Market, by Delivery Method Market Analysis by Region Chapter 8 Competitive Intelligence Strategic Initiatives Competitive Analysis Chapter 9 Sustainability in RNAi Technology Market: An ESG Perspective Introduction to ESG Sustainability in RNAi Technology Industry: An ESG Perspective Environmental Initiatives Social Initiatives Governance Initiatives Case Study Concluding Remarks Company Profiles Alnylam Pharmaceuticals Inc. Arrowhead Pharmaceuticals Inc. Arbutus Biopharma Corp. Bayer Merck Novartis Novo Nordisk Olix Pharmaceuticals Inc. Qiagen Revvity Inc. Silence Therapeutics Sirnaomics Sylentis S.A. Switch Therapeutics Thermo Fisher Scientific Inc. For more information about this report visit https://www.researchandmarkets.com/r/xokiai About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

siRNA

13 Feb 2024

·www.biospace.com

OliX Pharmaceuticals Commences Patient Dosing in Phase 1 Clinical Trial of OLX75016 for MASH

- Australia phase 1 clinical trials on 70 healthy + NAFLD participants - OLX75016’s safety and tolerability to be evaluated - Demonstrated enhanced weight loss effects when administered with GLP-1 obesity drug - Additional nonclinical study is ongoing to identify weight-managing efficacy of OLX75016-dual GIP/GLP-1 receptor agonists combi treatment SUWON, South Korea--(BUSINESS WIRE)-- OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced that the first participant was dosed today with OLX75016. OLX75016 is being developed to provide a novel treatment approach for metabolic dysfunctions associated with non-alcoholic fatty liver disease linked to obesity. The phase 1 trial is ongoing in Brisbane, Australia. The safety and tolerability of OLX75016 is being evaluated in a randomized, double-blind, placebo-controlled, single and multiple ascending dose study in healthy volunteers and early-stage patients with non-alcoholic fatty liver disease. OLX75016 acts as a mechanism to reduce liver fat content as well as inflammation and liver fibrosis which is frequently found in obese individuals. This development program is based on emerging Genome-Wide Association Study (GWAS) data that suggest certain genes to play a key role in these processes. This human genetic evidence was corroborated in a series of nonclinical studies. The Company showed data supporting the utility of OLX75016 either as mono-therapy or in combination with a GLP-1 receptor agonist or a dual GIP/GLP-1 receptor agonist for MASH and weight management. Dong Ki Lee, Ph.D., Founder and Chief Executive Officer of OliX Pharmaceuticals, said, "With the obesity market expected to reach $100 bn by 2030, many companies opt to develop GLP-1/GIP agonists for their weight management therapies. Given a differentiated mechanism of action, OLX75016 demonstrates its robust multimodality potential not just for additive weight loss effects but for NASH and liver fibrosis alleviation. This is how we believe OLX75016 is set apart, and we anticipate this novel therapeutic to bring hope to patients with multiple metabolic diseases, starting in phase 1 clinical trial." 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. Learn more:

Phase 1

22 Dec 2023

·www.biospace.com

OliX Pharmaceuticals Receives HREC Approval to Initiate Phase 1 Clinical Trial of NASH and Obesity Drug

SUWON, South Korea--(BUSINESS WIRE)-- OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced that the Company has received approval from the Alfred Human Research Ethics Committee (HREC) to conduct a Phase 1 clinical trial of drug candidate OLX75016 (the candidate of OLX702A) for the treatment of non-alcohol steatohepatitis (NASH). This randomized, double-blind, placebo-controlled, single and multiple ascending dose study will assess the safety and tolerability of OLX75016 in healthy volunteers. OliX Pharmaceuticals is developing OLX75016 as a novel therapeutic in treating NASH accompanied by liver fibrosis and managing weight. The Company has secured positive results from a preclinical non-human primates (NHP) study on the efficacy of OLX75016. OLX75016 administered together with a glucagon-like peptide-1 (GLP-1) receptor agonist (semaglutide) demonstrated additional weight-loss effects, mitigated the weight-rebound effect shown upon termination of semaglutide, lowered total body fat mass, and reduced abdominal girth. According to the Company, these favorable effects were not mediated by changes in food intake. This suggests that not dietary changes or changes in appetite regulation, but beneficial effects on energy expenditure mediate the weight-loss effects of this combination treatment. 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:

Phase 1siRNAIND

100 Deals associated with OliX Pharmaceuticals, Inc.

100 Translational Medicine associated with OliX Pharmaceuticals, Inc.

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Pipeline

Pipeline Snapshot as of 15 Nov 2024

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 Clinical

Phase 2 Clinical

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

OLX-10010 ( CTGF )	Cicatrix, Hypertrophic More	Phase 2
OLX-702A	Metabolic dysfunction-associated steatotic liver disease More	Phase 1
OLX-72021 ( AR )	Androgenetic Alopecia More	Phase 1
OLX-301A	Macular Degeneration, Age-Related, 1 More	Phase 1
OLX-104C ( AR )	Alopecia More	IND Approval

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