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Research programme: siRNA therapeutics - OliX Pharmaceuticals

Drug Profile

Research programme: siRNA therapeutics - OliX Pharmaceuticals

Alternative Names: asiRNA therapeutics - OliX Pharmaceuticals; BMT 101; cp-asiRNA therapeutics - OliX Pharmaceuticals; OLX 10020; OLX 103; OLX 201; OLX 301; OLX 301A; OLX 401; OLX 701

Latest Information Update: 03 Apr 2020

At a glance

  • Originator BioMolecular Therapeutics
  • Developer OliX Pharmaceuticals; University of Virginia School of Medicine
  • Class Antifibrotics; Antivirals; Eye disorder therapies; Skin disorder therapies; Small interfering RNA
  • Mechanism of Action Connective tissue growth factor inhibitors; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Age-related macular degeneration; Atopic dermatitis; Hepatic fibrosis; Idiopathic pulmonary fibrosis; Neuropathic pain
  • Research COVID 2019 infections
  • Discontinued Cancer; Herpes simplex virus infections

Most Recent Events

  • 30 Mar 2020 OliX Pharmaceuticals signs an exclusive licensing and supply agreement with AM Chemicals for GalNAc-conjugation technology for liver targeting
  • 20 Mar 2020 Early research in COVID-2019 infections in South Korea (unspecified route)
  • 20 Mar 2020 Olix Pharmaceuticals plans to initiate preclinical study for COVID-2019 infections within three months

Development Overview

Introduction

siRNA therapeutics are being developed by OliX Pharmaceuticals (formerly BioMolecular Therapeutics) for the treatment of dermatological, ophthalmic, neurological, pulmonary disorders and COVID-2019. Company's proprietary cell penetrating asymmetric siRNA (cp-asiRNA) compounds are different from traditional siRNA. These are based on asymmetric shorter-duplex siRNA (asiRNA), which is a 15-16 bp sense strand paired to a 19-21 bp antisense strand. Asymmetric siRNA is designed to specifically target implicated disease gene(s) and silence them. The siRNA therapeutic potentially avoids triggering the innate immune system, an unwanted off-target effect, which is often a problem associated with conventional siRNA therapies. cp-asiRNA compounds have shown to efficiently induce gene silencing in animal models and can deliver into hard to transfect cells including primary and suspension cells, without requiring special formulation or instrument. Lead molecules from connective tissue growth factor (CTGF) targeting programmes include, OLX 201 which is being developed as an aerosol formulation in collaboration with Korea Institute of Toxicology for idiopathic pulmonary fibrosis. Another candidate, OLX 301 (OLX 10020) that targets an undruggable gene involved in both wet and dry age-related macular degeneration is being developed in collaboration with University of Virginia. OLX 301 is a first-in-class therapeutic with potential to treat both wet and dry age-related macular degeneration. OLX 701 is being developed for liver fibrosis. These molecules target CTGF, whose increased expression in injuries leads to scarring and fibrosis. OliX Pharmaceuticals is also developing OLX 103 for atopic dermatitis and OLX 401 for neuropathic pain. Preclinical development in underway in South Korea and the US. Early research for COVID-19 is underway in South Korea.

OliX Pharmaceuticals was developing siRNA therapeutics for herpes simplex virus infections, and cancer however as at January 2018, these indications are not listed on company's pipeline and hence the development has been discontinued.

The company is also developing siRNA technology for cosmetic use including skin whitening, hair loss and anti-wrinkle treatments.

In September 2014, Biomolecular Therapeutics changed its name to OliX Pharmaceuticals (OliX Pharmaceuticals Website, January 2018).

OLX 10010 [see Adis Insight Drug profile 800052077] and OLX 304A [see Adis drug profile 800054779] have emerged from this research programme.

Company Agreements

In March 2020, OliX Pharmaceuticals signed an exclusive worldwide licensing agreement with AM Chemicals (AMC) for the rights to patents and know-how encompassing the technology to conjugate N-acetylgalactosamine (GalNAc) to OliX' asymmetric small interfering RNA (asiRNA) molecules for liver targeting. Under the agreement, AMC will supply a selection of GalNAc-modified phosphoramidites and solid supports to OliX Pharmaceuticals. AMC will also provide support for RNA synthesis, for which AMC is entitled to receive upfront milestone and royalty payments. [1]

In March 2019, OliX Pharmaceuticals entered into a license and collaboration agreement with Thea Open Innovation, to develop and commercialize OLX 301A. Under the agreement, OliX receives a non-refundable and non-creditable upfront payment of 2 million euro ($US2.26 million) and is eligible to receive milestone payments upon achievement of clinical milestones, plus royalty for the development and commercialisation. Thea has been granted licensing rights for OLX 301A program in EU countries, Middle East and Africa. OliX continues to hold the rights of OLX 301A program for US and Asia. [2]

In January 2012, BioMolecular Therapeutics announced it had entered a research collaboration with Samyang, a Korean-based company, for the development of RNAi therapeutics. As part of the agreement BioMolecular Therapeutics will conduct validation studies regarding the use of the RNAi technology against targets identified by Samyang.

Key Development Milestones

In January 2019, OliX Pharmaceuticals reported its intention to submit an IND application to the US FDA for OLX 301 for initiation of a phase I trial in dry age-related macular degeneration (geographic atrophy) in 2019. OliX Pharmaceuticals completed preliminary safety studies of OLX 301 and initiated GLP toxicology studies [3] .

In preclinical trials, OLX 301 demonstrated efficacy in animal models of both wet and dry-age related macular degeneration [3] .

In October 2010, BioMolecular Therapeutics reported research on the immune stimulation effect of asymmetric shorter-duplex siRNA [4] .

Financing information

In July 2018, Olix Pharmaceuticals raised a capital of KRW43 billion ($US38 million) in the Korea Securities Dealer Automated Quotation (KOSDAQ) listing, which would be used for research and development of therapeutic programs including idiopathic pulmonary fibrosis (OLX 201), and age-related macular degeneration (OLX 301A and OLX 301D) [5] .

In April 2015, Olix Pharmaceuticals secured a grant from The Small and Medium Business Administration, Korea for the development of OLX 103, for atopic dermatitis (Olix Pharmaceuticals website, January 2018).

In October 2014, Olix Pharmaceuticals received a three years funding of $US1.8 million from A*STAR, Singapore and Korea Health Industry Development Institute, for the development of OLX 201 for the treatment of idiopathic pulmonary fibrosis (Olix Pharmaceuticals website, January 2018).

Patent Information

In February 2020, Olix Pharmaceuticals filed a provisional patent application covering composition of matter claims to more than 30 small interfering RNAs (siRNAs) designs that target available genomes of the COVID virus [6] .

Olix Pharmaceuticals also holds patents for cp-asiRNA in South Korea (PCT/KR2013/004463). Other patent applications are under review in the US, Europe, Japan and China (Olix Pharmaceuticals website, January 2018).

A Korean patent, entitled 'Novel siRNA Structure for Minimizing Off-target Effects and Relaxing Saturation of RNAi Machinery and the Use thereof', has been issued to BioMolecular Therapeutics covering its asiRNA technology, and a PCT (international) patent is pending (PCT/KR2008/007530).

Drug Properties & Chemical Synopsis

  • Route of administration Inhalation, Topical
  • Formulation Aerosol, unspecified
  • Class Antifibrotics, Antivirals, Eye disorder therapies, Skin disorder therapies, Small interfering RNA
  • Target Connective tissue growth factor
  • Mechanism of Action Connective tissue growth factor inhibitors; RNA interference
  • WHO ATC code

    A05B-A (Liver therapy)

    D03A (Cicatrizants)

    D11 (Other Dermatological Preparations)

    J05 (Antivirals for Systemic Use)

    L01 (Antineoplastic Agents)

    N02 (Analgesics)

    R07 (Other Respiratory System Products)

    S01 (Ophthalmologicals)

  • EPhMRA code

    A5B (Hepatic Protectors, Lipotropics)

    D11 (Other Dermatological Preparations)

    D3 (Wound Healing Agents)

    J5 (Antivirals for Systemic Use)

    L1 (Antineoplastics)

    N2 (Analgesics)

    R7 (Other Respiratory System Products)

    S1 (Ophthalmologicals)

Development Status

Summary Table

Indication Qualifier Patient Segment Phase Countries Route / Formulation Developers Event Date
Age-related macular degeneration - - Preclinical South Korea unspecified / unspecified OliX Pharmaceuticals, University of Virginia School of Medicine 22 Jan 2018
Atopic dermatitis - - Preclinical South Korea Topical / unspecified OliX Pharmaceuticals 22 Jan 2018
COVID 2019 infections - - Research South Korea unspecified / unspecified OliX Pharmaceuticals 20 Mar 2020
Cancer - - Discontinued (Preclinical) South Korea unspecified / unspecified OliX Pharmaceuticals 22 Jan 2018
Hepatic fibrosis - - Preclinical South Korea unspecified / unspecified OliX Pharmaceuticals 22 Jan 2018
Herpes simplex virus infections - - Discontinued (Preclinical) South Korea unspecified / unspecified OliX Pharmaceuticals 22 Jan 2018
Idiopathic pulmonary fibrosis - - Preclinical South Korea Inhalation / Aerosol OliX Pharmaceuticals 22 Jan 2018
Neuropathic pain - - Preclinical South Korea unspecified / unspecified OliX Pharmaceuticals 22 Jan 2018

Commercial Information

Involved Organisations

Organisation Involvement Countries
BioMolecular Therapeutics Originator South-Korea
OliX Pharmaceuticals Owner South-Korea
Hugel Licensee Asia
Thea Open Innovation Licensee Africa, European Union, Middle East
AM Chemicals Technology Provider USA
Small and Medium Business Administration Funder South-Korea
Korea Health Industry Development Institute Funder South-Korea
A STAR Funder Singapore
Korea Drug Development Fund Funder South-Korea
Samyang Biopharmaceuticals Corporation Collaborator South-Korea
Korea Institute of Toxicology Collaborator South-Korea
University of Virginia School of Medicine Collaborator USA

Future Events

Expected Date Event Type Description Updated
30 Jun 2020 Trial Update Olix Pharmaceuticals plans to initiate preclinical study for COVID-2019 infections within three months [6] 30 Mar 2020
31 Dec 2019 Regulatory Status OliX Pharmaceuticals intends to file an IND application for OLX 301 with the US FDA for Dry age-related macular degeneration in 2019 [3] 22 Jan 2019
31 Dec 2019 Trial Update OliX Pharmaceuticals plans a phase I trial for OLX 301 for Dry age-related macular degeneration in USA in 2019 [3] 22 Jan 2019
01 Aug 2019 Trial Update Hugel plans a phase II trial for Hypertrophic scars in South Korea in August 2019 (NCT04012099) 10 Jul 2019

Development History

Event Date Update Type Comment
30 Mar 2020 Licensing Status OliX Pharmaceuticals signs an exclusive licensing and supply agreement with AM Chemicals for GalNAc-conjugation technology for liver targeting [1] Updated 03 Apr 2020
20 Mar 2020 Phase Change Early research in COVID-2019 infections in South Korea (unspecified route) [6] Updated 30 Mar 2020
20 Mar 2020 Trial Update Olix Pharmaceuticals plans to initiate preclinical study for COVID-2019 infections within three months [6] Updated 30 Mar 2020
25 Feb 2020 Patent Information Olix Pharmaceuticals has patents pending for composition of matter patent for siRNAs for COVID-2019 [6] Updated 30 Mar 2020
09 Jul 2019 Trial Update Hugel plans a phase II trial for Hypertrophic scars in South Korea in August 2019 (NCT04012099) Updated 10 Jul 2019
18 Mar 2019 Licensing Status OLX 301A licensed to Thea Open Innovation in European Union, Middle East and Africa [2] Updated 25 Mar 2019
17 Jan 2019 Regulatory Status OliX Pharmaceuticals intends to file an IND application for OLX 301 with the US FDA for Dry age-related macular degeneration in 2019 [3] Updated 22 Jan 2019
17 Jan 2019 Trial Update OliX Pharmaceuticals plans a phase I trial for OLX 301 for Dry age-related macular degeneration in USA in 2019 [3] Updated 22 Jan 2019
22 Jan 2018 Patent Information Olix Pharmaceuticals has patent pending for cp-asiRNA technology in USA, Europe, Japan and China (Olix Pharmaceuticals website, January 2018) Updated 22 Jan 2018
22 Jan 2018 Patent Information Olix Pharmaceuticals has patent protection for cp-asiRNA technology in South Korea (Olix Pharmaceuticals website, January 2018) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Discontinued(Preclinical) Discontinued - Preclinical for Cancer in South Korea (unspecified route) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Discontinued(Preclinical) Discontinued - Preclinical for Herpes simplex virus infections in South Korea (unspecified route) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Preclinical Preclinical trials in Age-related macular degeneration in South Korea (unspecified route) (Olix Pharmaceuticals pipeline, January 2018) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Preclinical Preclinical trials in Atopic dermatitis in South Korea (Topical) (Olix Pharmaceuticals pipeline, January 2018) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Preclinical Preclinical trials in Hepatic fibrosis in South Korea (unspecified route) (Olix Pharmaceuticals pipeline, January 2018) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Preclinical Preclinical trials in Idiopathic pulmonary fibrosis in South Korea (Inhalation) (Olix Pharmaceuticals pipeline, January 2018) Updated 22 Jan 2018
22 Jan 2018 Phase Change - Preclinical Preclinical trials in Neuropathic pain in South Korea (unspecified route) (Olix Pharmaceuticals pipeline, January 2018) Updated 22 Jan 2018
16 Jul 2016 Phase Change - No development reported No recent reports of development identified for preclinical development in Age-related-macular-degeneration in South Korea Updated 16 Jul 2016
01 Sep 2014 Company Involvement BioMolecular Therapeutics is now called OliX Pharmaceuticals Updated 16 Jan 2018
24 Apr 2012 Company Involvement Boston Biomedical has been acquired by Dainippon Sumitomo Pharma Updated 27 Apr 2012
03 Jan 2012 Phase Change - No development reported(Preclinical) No development reported - Preclinical for Cancer in South Korea (unspecified route) Updated 03 May 2012
03 Jan 2012 Phase Change - No development reported(Preclinical) No development reported - Preclinical for Herpes simplex virus infections in South Korea (unspecified route) Updated 03 May 2012
20 Jan 2009 Phase Change - Preclinical Preclinical trials in Cancer in South Korea (unspecified route) Updated 28 Oct 2010
20 Jan 2009 Phase Change - Preclinical Preclinical trials in Herpes simplex virus infections in South Korea (unspecified route) Updated 28 Oct 2010
20 Jan 2009 Phase Change - Preclinical Preclinical trials in Age-related macular degeneration in South Korea (unspecified route) Updated 26 Oct 2010

References

  1. OliX Pharmaceuticals and AM Chemicals Announce Exclusive Licensing and Supply Agreement for GalNAc-Conjugation Technology for Liver Targeting.

    Media Release
  2. Tha and OliX Announce Collaboration to Develop RNAi Therapies for the Treatment of AMD.

    Media Release
  3. OliX Announces Clinical Candidate Nomination for the Treatment of Geographic Atrophy.

    Media Release
  4. BioMolecular Therapeutics, a New Global Player in RNAi Therapeutics, Launches Company and Adds Key Advisors.

    Media Release
  5. OliX, a RNAi therapeutics developer, gets approval for KOSDAQ listing.

    Media Release
  6. OliX Pharmaceuticals Advances RNAi Approaches to Target Highly Conserved Regions of Coronavirus RNAs.

    Media Release
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