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KDDF-201312-11 Selection of optimized lead compounds for preclinical studies for idiopathic pulmonary fibrosis using a novel RNAi-based nanoparticle technology SAMiRNA™, with a goal of IND application(Respiratory Diseases, Genetics) [03.31.2014]

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Development and Market Objectives

Our objective is to develop the effective therapeutic drugs for the treatment of Idiopathic Pulmonary Fibrosis (IPF), a respiratory fibrotic diseases. It will be the first siRNA-based therapeutic drug against IPF, fatal respiratory diseases with no available efficient drugs yet. We are developing multiple preclinical candidate substances for idiopathic lung fibrosis with overall evaluations of efficacy and PK/PD analysis, applying the SAMiRNA™ RNAi nanoparticle technology covered by patent protection. 

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 Esbriet® (Pirfenidone) and Ofev® (Nintedanib), which are approved by FDA in 2014, is expensive and exhibits limited efficacy. Urgent need for the development of new effective drug which can actively reverse the process of fibrosis. 
③ By developing potent antifibrotic agents, applications can be extended to diseases that are frequently accompanied by fibrosis (such as NASH, systemic scleroderma, 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. 

Status

In-vitro screening: Several hundreds of siRNA molecules were in-vitro screened to find highly specific and potent siRNAs against these target genes. Hundreds of SAMiRNA™ have been synthesized and screened for the highly potent therapeutic candidates.

PK/PD: PK/PD analysis of SAMiRNA was performed by in vivo imaging of biodistribution and real-time qPCR-based quantification of gene knock-down in various organs. SAMiRNA-IPF is delivered to lung only in the IPF induced model animals both of Bleomycin-induced and TGF-b transgenic model, consequently knock down.

in vivo efficacy test: The efficacy of the SAMiRNA-IPF drug has been evaluated with TGF-ß transgenic & Bleomycin induced mouse models of pulmonary fibrosis. Based on these comprehensive evaluations, the candidates show the more significant therapeutic potential for IPF treatment than Pirfenidone of InterMune, Inc and FG-3019 (CTGF mAb) of Fibrogen, Inc.

Intellectual Property

 

Country

Application

Date

Application

Number

Registration

Date

Registration

Number

Name of

Invention

KR

2009-05-14

10-2009-042297

2013-01-16

1224828

siRNA conjugate and preparing method thereof

KR

2009-05-14

10-2012-0069988

2013-03-05

1241852

KR

2009-05-14

10-2012-0114011

2014-04-30

1392973

US

2010-05-13

13/319885

2014-07-15

8779114

US

2010-05-13

13/613071

2014-07-08

8772472

US

2014-05-30

14/291,540

2015-12-15

9211343

EP

2010-05-13

10775118

2015-08-26

2463371

JP

2010-05-13

2012-510752

2015-06-12

5758381

JP

2013-05-08

2013-098798

2015-08-28

5797688

CN

2010-05-13

201080021324.3

2015-07-01

ZL201080021324.3

CN

2010-05-13

201210301551.2

2015-06-17

ZL20120301551.2

CN

2013-04-25

201310147988.X

2015-09-16

ZL01310147988.X

CA

2010-05-13

2761794

   

CA

2014-11-20

2,761,749

   

AU

2010-05-13

2010248239

2015-05-07

2010248239

AU

2015-01-13

2015200143

   

BR

2010-05-13

1012141-2

   

IN

2010-05-13

2336/MUMNP/2011

   

RU

2010-05-13

2011150787

2015-07-01

2558258

RU

2013-11-21

2013151977

   

KR

2015-04-06

10-2015-7008814

   

Composition for the prevention or treatment of respiratory diseases including gene-specific double strand oligo-RNA

US

2015-04-05

14/433627

   

EP

2015-04-05

13843342

   

CN

2015-05-06

201380058057

   

JP

2015-04-03

2015-535571

   

AU

2015-04-05

2013325384

   

CA

2015-04-05

2887069

   

BR

2015-04-05

11-2015-007637-8

   

IN

2015-04-05

1964/CHENP/2015

   

KR

2016-01-26

10-2016-7002234

   

Respiratory disease related genes-specific siRNA, double-stranded oligo RNA molecules comprising the siRNA, and composition for the prevention or treatment of respiratory diseases comprising the same

US

2016-01-02

14/902,566

   

JP

       

CN

       

EP

2016-02-03

14820458.9

   

CA

2016-01-04

2917320

   

AU

2016-01-22

2014284836

   

RU

       

BR

2016-01-05

BR 11 2016 000163 0

   

IN

       

MX

2016-01-07

MX/a/2016/000019

   

KR

2015-04-06

10-2015-0048443

   

Novel double strand oligo RNA and pharmaceutical compositions for preventing or treating fibrosis or respiratory diseases containing the same

PCT

2015-04-06

PCT/KR2015/003400

 

 

Patents for the SAMiRNA™ RNAi drug platform (siRNA conjugate and preparing method thereof) have been filed for domestic and major international markets, including patents for specific fibrosis-related genes. The period of monopoly by 2035 will be obtained in the major markets by follow-up patent applications with further studies. 
 

Competitive Advantages

① Robust SAMiRNA™ technology fundamental patents for lung fibrosis-related siRNA sequences   
② Synergy for the lung fibrosis treatment effect will be verified by administration of an siRNA cocktail simultaneously targeting two critical genes.
③ Favorable comparisons for therapeutic effect in various animal models (TGF-β transgenic mouse model, smoking model, bleomycin-induced lung fibrosis mouse model)
④ Market assessment will be faster due to a shorter clinical trial period, as idiopathic lung fibrosis medicines are classified as orphan drug.
⑤ Potential extended applications for the diseases in other organs which are frequently accompanied with fibrosis symptoms (hepatocirrhosis, kidney fibrosis, etc). 

Indication

idopathic pulmonary fibrosis

Research Period

Feb, 2014 ~ Oct, 2015

Company

Bioneer Corp.

Developmental Stage

Lead Optimization

Additional Information

Contact Information

Contact
Address Company Name: Bioneer Corporation
WebSite Homepage: http://www.bioneer.com Contact Person: Pyoung Oh Yoon
E-mail: poyoon@bioneer.co.kr Contact: 82-42-930-8753

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