IDRAIdera Pharmaceuticals, Inc. (NASDAQ:IDRA) announced the presentations of new pre-clinical data on the mechanism of action supporting selective targeting of single point mutations.  Also, data was presented on 3GA targeting the NLRP3 gene for the treatment of inflammatory disorders.

“The understanding we have gained of the mechanism of action of 3GA is providing insights into the increased potency and specificity of its gene silencing.  This data has illustrated to us a way in which 3GA could be used to target point mutations,” stated Sudhir Agrawal, D. Phil., President of Research at Idera Pharmaceuticals.  “We are continuing to employ 3GA technology to target multiple genes, including NLPR3, with a goal of prioritizing a candidate for clinical development.”

In the oral presentation, entitled “Selective Targeting of Point Mutations by Third Generation Antisense Oligonucleotides,” Reina Improgo, Ph.D., Research Scientist of Idera’s Discovery Team presented an overview of the novel mechanism of action of 3GA technology.  These preclinical studies were conducted both in cell-based assays as well as in vivo models.  The novel mechanism of action of 3GA leads to excision of the targeted RNA in the central region.  Insertion of a single mismatch led to significant loss of gene-silencing activity.  Based on this data, 3GAs were designed to target single point mutations.  These proof-of-concept studies were conducted using two targets, the BRAF V600E and MYD88 L265P mutations.  The 3GA targeted to BRAF V600E showed mutation-specific inhibition, whereas a 3GA targeted to wild type BRAF showed minimal activity.   Similarly, 3GA targeted to MYD88 L265P showed mutation-specific inhibition, and had insignificant impact on wild type MYD88 expression. Based on the specificity in targeting RNA the data indicates that 3GA could be used to successfully treat diseases that require allele-specificity.

Additionally, Fugang Zhu, Ph.D. and Wayne Jiang, M.D., Ph.D., scientists from Idera’s Discovery Team, presented a poster entitled, “Third generation antisense (3GA) targeting NLRP3 for the treatment of inflammatory disorders.”  In the presentation, they showed that 3GA targeting NLRP3 led to the suppression of the NLRP3 mRNA and protein and inhibition of the downstream cascade, including IL-1β and IL-18.   The data also demonstrated that 3GA targeting of NLRP3 resulted in marked improvement in disease-associated parameters in both interstitial cystitis and uveitis preclinical models. (Original Source)

Shares of Idera Pharmaceuticals closed yesterday at $2.66, down $0.10 or -3.62%. IDRA has a 1-year high of $4.42 and a 1-year low of $1.19. The stock’s 50-day moving average is $2.07 and its 200-day moving average is $1.78.

On the ratings front, Wedbush analyst Heather Behanna reiterated a Buy rating on IDRA, with a price target of $6, in a report issued on August 25. The current price target implies an upside of 125.6% from current levels. According to, Behanna has a yearly average return of 15.7%, a 47.5% success rate, and is ranked #506 out of 4183 analysts.

Idera Pharmaceuticals, Inc. is a clinical stage biotechnology company, which is engaged in discovery, development and commercialization of novel nucleic acid therapeutics to treat patients with serious and life-threatening diseases. Idera’s immunotherapy approach is based on the modulation of toll-like receptors (TLRs) while its third-generation antisense technology is designed to inhibit the production of disease-associated proteins by targeting RNA.