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Copernicus Therapeutics, Inc.
Copernicus:Delivering the Promise
of Nucleic Acid Therapeutics
Robert C. Moen, M.D., Ph.D.President & [email protected]
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Copernicus Therapeutics, Inc.
A leader in the emerging field of nucleic acid delivery and therapeutics (12 issued patents, more pending)
Enable first in class therapeutic molecules employing DNA, RNA, siRNA
Address unmet medical needs Cystic fibrosis Viral lung diseases (influenza, avian flu, SARS) Eye disorders (macular degeneration, retinitis
pigmentosa) Established proof-of-concept in humans (CF trial)
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The Copernicus Platform Technology:
Nucleic Acid Nanoparticles
DNA (or RNA/siRNA) Compaction Single molecules of NA condensed with amino acids
Simple, reproducible manufacturing process cGMP grade raw materials available (final
assembly) Stable (>3 years at 4°C, > 9 months room temp) Stable at clinically relevant concentrations IP covers composition of matter, methods and
uses
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Advantages of Copernicus Technology
Non-toxic Non-immunogenic Repetitive dosing possible Drug does NOT alter human genomic
DNA, but introduces nucleic acids into the cell
Highly efficient delivery to cells Both dividing AND non-dividing cells
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Uptake and Trafficking of Nanoparticles
NON-DEGRADATIVETRAFFICKING PATHWAY
Nanoparticle
nucleolus
In collaboration with D. Kube and P. Davis, CWRUnucleolin
BINDING TO CELLSURFACE NUCLEOLINCOMPLEX
nuclearpore
Released nucleic acid
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Why a Cystic Fibrosis Focus?
Copernicus treatment successful in CF animal modelModels
Support
Treatments
Cystic Fibrosis Foundation and State of Ohio TAF support.
No existing corrective treatments, only symptomatic therapy with limited effectiveness
Provides proof of concept yet large market size
FDA Accelerated approval process & orphan drug status
Mutations in a single gene (CFTR) cause CF, only ~5% of lung cells need to be corrected, a wide range of expression is therapeutic and nontoxic
Single gene
Market
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Cystic Fibrosis Clinical Trial has Provided Proof of Principle
Formulation well-tolerated and safe Strong gene transfer endpoints
Efficient DNA uptake into target cells Correction of the CFTR chloride channel
defect Konstan MW et al, “Single Dose Escalation Study to Evaluate Safety of Nasal Administration of CFTR001 Gene Transfer Vector to Subjects with Cystic Fibrosis”, Human Gene Therapy, 15:1255-69, 2004.
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Platform Technology Addresses Multiple Indications With
Significant Markets DNA and/or siRNA Aerosol delivery
Cystic Fibrosis Influenza RSV, SARs, Bird flu, BioWarfare
Eye (retina) Macular degeneration (> 5 million pts in US) Retinitis pigmentosa (~ 70,000 pts in US) Diabetic retinopathy (~ 900,000 pts in US with
severe disease)
Others (neural, neovasculature, tumors)
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Influenza A Mouse ModelCompacted siRNA Therapy
Fisher’s exact testp2 = 0.0280 1 2 3 4
0102030405060708090
100
control (anti-luciferase)
experimental (anti-NP, anti-AP)
Time (days)
Per
cen
t su
rviv
al
Dosing of compacted siRNA followedby influenza A virus
NP and AP conserved across all strains of
Influenza A, not flu season specific !
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Subretinal Injection of DNA Nanoparticles Encoding EGFP
OUTER NUCLEAR LAYER(nuclei of rods and cones)
INNER NUCLEAR LAYER(nuclei of second order neurons)
IHC DETECTION
nearly 100% gene transfer ofrods and cones in the retina
Results 10-20 times better than seen with any other system
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Direct Brain Gene Transfer
Comparable to best results for viral vectors (lentivirus, AAV)
Viral vectors have toxicities related to immunity No inflammation or immune response to DNA
nanoparticles Partnering opportunities for large markets with
unmet medical needs (e.g. Parkinson’s disease) Efficient transfer into neural stem cells in vitro
Product Summary
Parkinson’s Disease
Macular Degeneration
Retinitis Pigmentosa
Influenza, etc.
Cystic Fibrosis
Pre-ClinicalDevelopmentClinical
I II III
Management Robert C. Moen, M.D., Ph.D. (CEO and President)
VP of Clinical and Regulatory Affairs at Baxter Healthcare VP of Clinical Development at Geneic Sciences Co-founder and Director of Clinical and Regulatory Affairs and
Director of Cell Engineering at Genetic Therapy, Inc. Mark J. Cooper M.D. (Sr. VP Science and Medical Affairs)
Co-founder of Copernicus Therapeutics, Inc. Associate Professor, Oncology at CWRU Oncology Fellowship, NCI , NIH
Joseph Ashley (Chairman of the Board) Chief Financial/Business Development Officer (To be
hired)
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Key Achievements To Date $19 M equity investment $4 M support from CFF, state of Ohio, CDC,
NIH Human clinical trial data (Phase I/II) Strong IP position Unique, powerful mechanism of action Preclinical data to support multiple other
applications of core technology Initiating ~$15M Series B financing round
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Use of Series B Funds
Milestones 2006 Complete lung aerosol CF trial Initiate at least one additional product
development Corporate partnerships Obtain additional non-diluting support Augment management team
Milestones 2007 Initiate multi-dose CF trial, complete in 2008 Initiate and complete at least one additional
product clinical trial
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ConclusionCopernicus Therapeutics, Inc
Lead product for CF with excellent toxicology profile and demonstrated biological effect in patients
Technology solves the problems with past attempts to develop nucleic acid therapeutics
Platform technology can generate products for multiple indications
IP estate for compacting nucleic acids protects lead product and will be attractive to large pharma/biotech
CF product demonstrates proof-of-concept for technology platform with a large market application
