Completed Projects

HOME > R&D Pipeline > Current status > Completed Projects

KDDF-201210-09 Development of an innovative drug for Alzheimer’s disease modulating microRNA(CNS Diseases, Genetics) [01.17.2013]

PRINT

Development and Market Objectives

•      To develop an innovative drug for Alzheimer’s disease.
•      To perform further confirmation and optimization based on Antagomir-206.
•      To analyze in vivo efficacy and preliminary toxicity of optimized substances.
•      To derive drug candidates for Alzheimer’s disease-modulating microRNA and enter preclinical study.

Unmet Medical Need & Target Patients

•      The prevalence of patients with Alzheimer’s disease has increased yearly to 35.6 million as of 2010, a doubling rate of every 20 years.
•      Commercially available drugs (Donepezil, Rivastigmine, Galantamine, Memantine) exhibit weak efficacy. Currently available therapeutics modulate symptoms, but cannot suppress disease progression.
•      As of 2012 the market for commercial drugs for Alzheimer’s is $5.3Bn.
•      Synaptic degeneration is observed from the initial disease stages, which causes degradation of cognitive function.
•      Our drug candidate induces synapse regeneration through modulation of BDNF and could cure the disease.

Status

•      It was determined that an increase of miR-206, which regulates BDNF, aggravates dementia in the brains of patients with Alzheimer’s disease.
•      We discovered a lead compound modulating miR-206.
•      It was demonstrated that the substance was effectively delivered to the brain through intranasal delivery.
•      It was confirmed that cognitive function was restored through synapse regeneration in mice with Alzheimer’s disease.
•      Lead optimization and toxicity experiments are in progress.

Intellectual Property

•      Domestic patent registration and international PCT patent application; PCT/KR2011/006718.
•      “Therapy on neurodegerative disease using Antagomir-206”

Competitive Advantages

•      This is an innovative drug that operates via microRNA modulation, a core component of Alzheimer’s disease.
•      The drug clearly demonstrates disease-modifying properties such as synapse regeneration, unlike existing temporary symptom-improving agents.
•      Overcoming the blood-brain barrier through intranasal delivery is an area of present effort.
•      As it is expected to be sufficient with single weekly intranasal delivery, application of the drug is convenient.

Indication

Alzheimer Disease

Research Period

Jan 01, 2013 ~Dec. 31, 2014

Company

Seoul National University Hospital

Developmental Stage

Discovery

Additional Information

Contact Information

Contact
Address Company Name: Seoul National University Hospital, Neurology
WebSite Homepage: http://cnupharm.cnu.ac.kr Contact Person: Geon Joo, Professor
E-mail: stemcell.snu@gmail.com Contact: 02-2072-1878

Related Projects

Related Project
list
Update to 2013 © KOREA DRUG DEVELOPMENT FUND. ALL RIGHTS RESERVED. QR Code