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KDDF-201706-03 Non-clinical GMP Toxicology Study & Clinical IND approval for the development of idopathic pulmonary fibrosis using a novel RNAi-based nanoparticle technology (SAMiRNA)(Respiratory Diseases, Genetics) [2020-06-24]

Ongoing Project
Section Lead
Preclinical Phase I Phase II Phase III

Development and Market Objectives

To develop effective and novel therapeutic drugs for idiopathic pulmonary fibrosis (IPF), specific target molecules have been identified using animal models of pulmonary fibrosis.  After synthesis of highly specific and effective siRNA against these target genes, the siRNAs have been integrated into our patented SAMiRNA platform technology. The effectiveness of the drug will be determined through a variety of Non-clinical toxicity studies such as general toxicity, Toxicokinetics, and PK/PD properties. Based on these comprehensive evaluations, the best candidates with significant therapeutic potential for IPF will be developed for IND filing

Unmet Medical Need & Target Patients

Unmet Medical Needs

① The mortality rate for lung fibrosis is extremely high, with a 5-year survival rate lower than 30%.

② Pirfenidone, which is approved as an orphan drug for the treatment in Japan, Europe and India, is expensive and exhibits limited efficacy. Novel drug development for the disease is urgently needed as no approved treatments exist in many countries including the US and the Republic of Korea.

③ By developing potent antifibrotic agents, applications can be extended to diseases that are frequently accompanied by fibrosis (such as hepatocirrhosis, kidney fibrosis, etc).


Target Patients

Patients aged 50 and older, with lung fibrosis confirmed by high resolution computed Tomography (HRCT) or by surgical biopsy, with mild to moderate symptoms between 50% to 80% on a post-bronchodilator forced vital capacity pulmonary function test.


After the discovery of a first-in-class amphiregulin gene through advanced research and analysis of a highly specific siRNA-medicated inhibition sequence, down regulation of fibrosis-associated biomarkers has been confirmed after in-vitro administration of SAMiRNA drug treatment, which exhibits a safe and persistent effect.

Research of SAMiRNA-Amphiregulin in IPF to develop a First-in-class therapeutic drug will be performed through Non-clinical toxicology studies and the approval of IND application.

Intellectual Property

Patents for the SAMiRNA RNAi drug platform (siRNA conjugate and preparing method) have been filed and pended for domestic and major international markets such as PCT. Patents for specific fibrosis-related genes are also pending for submission as well. Through further research, follow-up patent applications will be pending, and exclusive rights will be obtained in major markets by 2035.

Competitive Advantages

① Robust SAMiRNA technology fundamental patents for lung fibrosis-related siRNA sequences  

② Favorable comparisons for therapeutic efficacy compared to approved and on-going clinical trial drugs in pulmonary fibrosis animal models

③ Market assessment will be faster due to a shorter clinical trial period, as idiopathic lung fibrosis medicines are classified as orphan drug.

④ Potential extended applications for the diseases in other organs which are frequently accompanied with fibrosis symptoms (hepatocirrhosis, kidney fibrosis, etc).

Contact & Company Overview

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