TOPIGEN Begins IND-Enabling Studies for TPI-1100 the First RNA-Targeting Compound to Treat COPD - RNA

TOPIGEN Begins IND-Enabling Studies for TPI-1100 the First RNA-Targeting Compound to Treat COPD
9/18/2006
GEN
TOPIGEN Pharmaceuticals Inc., an emerging biopharmaceutical company specializing in developing treatments for respiratory diseases, today announced that it has selected its next clinical candidate for Chronic Obstructive Pulmonary Disease (COPD). The compound, TPI-1100, is a novel, potent RNA-targeting inhibitor of multiple phosphodiesterases (PDEs), a group of enzymes that are validated targets of COPD. The compound will begin full scale development and undergo formal scale-up, manufacturing and toxicology studies to support clinical development activities, which are scheduled to begin in 2007.
"We are excited about the profile for this new inhaled PDE inhibiting compound," commented Paul K. Wotton, Ph.D., President and Chief Executive Officer of TOPIGEN. "This is the second compound originating from our internal research to be nominated for clinical development demonstrating TOPIGEN's discovery capabilities in RNA-targeting therapeutics. Similar to our lead Phase II clinical drug candidates (TPI-ASM8 and TPI-1020), this drug utilizes a multi-targeted approach designed to inhibit multiple inflammatory pathways that are known to be linked to disease symptoms. The Company is now focused on completing supporting trials needed for regulatory submission and to move TPI-1100 into Phase I testing in 2007."
Results of preclinical studies on TPI-1100 performed to date demonstrated significant and selective PDE gene-knockdown while reducing neutrophil influx and down regulating inflammatory mediators in lung tissue. "PDEs are some of the most important biological targets known to be involved in the regulation of lung inflammation associated with COPD," stated Paolo Renzi, M.D., TOPIGEN's Founder and Chief Scientific Officer. "TPI-1100 efficiently blocks key forms of PDEs in animal models of COPD and exhibits promising in vivo characteristics. While the development of other medications targeting PDEs has been hindered due to dose limiting systemic toxicity, TPI-1100 is the first inhaled, gene-targeting drug of its kind, designed to minimize systemic drug exposure while significantly inhibiting multiple PDEs locally in the lungs."
About TPI-1100
TPI-1100 is a selective and dual-acting RNA-targeting oligonucleotide inhibitor of phosphodiesterase isoforms PDE4 and PDE7. The drug is designed to inhibit multiple gene expression pathways known to be linked to progressive airway inflammation and remodeling in COPD. Delivered topically via aerosol to the lungs, TPI-1100 represents a novel RNA-targeting therapy with a highly selective mechanism for reducing lung inflammation without the dose-limiting systemic side effects widely associated with known small molecule inhibitors. The drug utilizes TOPIGEN's proprietary FANA(TM) Technology, a new generation of oligonucleotide chemistry with potential for broad gene-targeting drug applications. Clinical studies are expected to begin in 2007.
About COPD
Chronic obstructive pulmonary disease (COPD) is a disease characterized by persistent airflow reduction that makes it difficult to move air into and out of the lungs. As the disease progresses, the airways of the lungs become inflamed and obstructed. Approximately 80 to 90 percent of COPD cases are caused by cigarette smoking. Today, COPD is the fourth leading cause of death and claims the lives of hundred of thousands of people worldwide annually. Chronic airflow limitation is believed to be a consequence of an abnormal recruitment of inflammatory cells such as neutrophils (cells releasing enzymes that destroy lung tissue) and response to exposure to cigarette smoke. Acute exacerbations are the most common complication and a primary contributor to the morbidity and mortality. The disease is often associated with other significant disorders, notably asthma and heart disease. Although existing treatments may improve quality of life and provide symptomatic relief in some patients, there are currently no drugs available that can slow the progressive decline in lung function that is a hallmark of the disease.
About FANA(TM) Technology Platform
FANA oligonucleotides are chemically-modified molecules (nucleotides) that are designed to bind to a specific sequence of a messenger RNA (mRNA) target through base-pairing interactions, thereby interfering with expression of the protein encoded by the mRNA. Scientists are focused on using antisense and RNA-interference (RNAi) oligonucleotides to design new, more effective drugs to inhibit gene expression and production of abnormal levels of cell proteins. FANA(TM) (Fluoro-Arabino-Nucleic Acid) is unique among oligonucleotide chemistries and significantly enhances efficacy of oligonucleotides indicated for gene targeting and silencing and offers significant performance benefits, compared to the traditional antisense and RNAi approaches.
About TOPIGEN
TOPIGEN is a privately held biopharmaceutical company focused on developing new classes of inhaled drugs for respiratory disorders. TOPIGEN's pipeline includes small molecule and RNA-based therapeutics. The Company has two products currently undergoing Phase II trials and research programs uniquely focused on inhibiting multiple inflammatory pathways at the cellular site of lung inflammation. By topically targeting multiple affected airway receptors with its drugs, the Company expects to improve outcomes for many patients with respiratory diseases. This approach has shown promising preclinical and clinical results in inhibiting the underlying inflammation associated with many pulmonary conditions such as asthma, COPD and allergic rhinitis. For more detailed information on TOPIGEN visit www.topigen.com.