COPD And Asthma - Proprietary FANA™ Chemistry Platform For RNA-Silencing Therapeutics Described

Main Category: Respiratory / Asthma
Article Date: 05 May 2008 - 3:00 PDT

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TOPIGEN Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company specialized in developing products for respiratory disorders, described progress of its proprietary FANA™ oligonucleotide chemistry platform at the RNAi World Congress on April 30, 2008, Boston, MA.

Nicolay Ferrari, Ph.D., TOPIGEN's Director of Pharmacology, presented preclinical data for TPI 1100, a novel, inhaled, FANA™- based RNA-silencing therapeutic for the treatment of chronic obstructive pulmonary disease (COPD) and asthma. TPI 1100 is TOPIGEN's first drug candidate created with its FANA™ chemistry.

About FANA™ Chemistry Platform and TPI 1100

FANA™ (2'-deoxy-2'fluoroarabinonucleic) is a third-generation antisense oligonucleotide technology. FANA™'s key chemistry modification substitutes the naturally-occurring D-ribose sugar moiety in oligonucleotides with a D-arabinose sugar containing fluorine. This change enhances RNA-targeting properties and allows the creation of RNA-targeting agents that have demonstrated increased resistance to nuclease breakdown, increased activity and duration of action, and a favorable safety profile in preclinical studies with activity at lower doses compared with other chemistries.

Dr. Ferrari, commented on his presentation, "The superior efficacy of TPI 1100, which is TOPIGEN's first FANA™-modified RNA-silencing therapeutic, in reducing lung inflammation in a COPD model provides validation for advancement of our product candidate into human clinical studies. TPI 1100 acts by inhibiting several forms of the enzyme phosphodiesterase (PDE), known to promote inflammation in the lung in COPD. Traditional PDE inhibitors inhibit only the PDE 4 form of the enzyme. Topigen's multi-pathway targeted approach inhibits several enzyme isoforms and provides more potent anti-inflammatory activity compared with roflumilast (an investigational small molecule phosphodiesterase 4 inhibitor) at very low doses of TPI 1100.

TPI 1100 is delivered via inhalation directly to the lungs. Systemic exposure is minimal and markedly lower compared to other PDE inhibitors which should result in a significantly better safety and tolerability profile.

About TOPIGEN

TOPIGEN is a privately held, clinical-stage company focused on developing new classes of inhaled drugs for respiratory diseases such as chronic obstructive pulmonary disease (COPD) and asthma. TOPIGEN is actively progressing two drug candidates in Phase 2 trials for COPD and asthma, including TPI 1020, a novel anti-inflammatory, licensed from NicOx S.A. and TPI ASM8, which utilizes its proprietary mRNA-silencing technology. These drug candidates target multiple pathways and pro-inflammatory mediators involved in chronic pulmonary diseases.

Current venture investors include NovaQuest, MMV Financial, Inc., BDC Venture Capital, Fonds de solidarité FTQ, Desjardins Venture Capital, Caisse de Dépot et Placement du Québec (Caisse), T2C2/BIO 2000 and Lothian Partners 27 (sarl) SICAR. For more detailed information on TOPIGEN visit www.topigen.com.