Idera Pharmaceuticals Presents New Third Generation Antisense (3GA) Data at the 12th Annual Meeting of the Oligonucleotide Th...
September 27 2016 - 7:30AM
Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), a clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel nucleic acid-based therapeutics for
oncology and rare diseases, today 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.
These presentations are currently available on
Idera’s website at
http://www.iderapharma.com/our-approach/key-publications/.
Previously the company has announced the
identification of NLRP3 (NOD-like receptor family, pyrin domain
containing protein 3) and DUX4 (Double Homeobox 4) as initial gene
targets to advance into IND-enabling activities, which will occur
throughout 2016. Potential disease indications related to
these targets include, but are not limited to, interstitial
cystitis, lupus nephritis, uveitis and facioscapulohumeral muscular
dystrophy (FSHD). The Company is currently conducting
clinical, regulatory and commercial analysis activities and
conducting IND-enabling studies with the plan to enter the clinic
in 2017 for the first clinical development program. In
addition to these activities, over the first half of 2016, Idera
generated 3GA compounds for a series of additional gene
targets. These will enable the Company to continue to expand
both its future pipeline opportunities for internal development as
well as its opportunities for partnerships in areas outside of
Idera’s focus. Additionally, Idera is party to a
collaboration and license agreement with GSK to research, develop
and commercialize compounds from its 3GA technology for the
treatment of undisclosed, selected renal targets.
About Idera’s Third Generation Antisense
Platform (3GA)Idera’s proprietary third-generation
antisense (3GA) platform technology is focused on silencing the
mRNA associated with disease causing genes. Idera has
designed 3GA oligonucleotides to overcome specific challenges
associated with earlier generation antisense technologies and RNAi
technologies.
About Idera Pharmaceuticals,
Inc.Idera Pharmaceuticals is a clinical-stage
biopharmaceutical company developing novel nucleic acid-based
therapies for the treatment of certain cancers and rare diseases.
Idera’s proprietary technology involves using a TLR-targeting
technology, to design synthetic oligonucleotide-based drug
candidates to act by modulating the activity of specific TLRs. In
addition to its TLR programs, Idera has created a third generation
antisense technology platform using its proprietary technology to
inhibit the production of disease-associated proteins by targeting
RNA. To learn more about Idera, visit www.iderapharma.com.
Forward Looking Statements This
press release contains forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933, as amended,
and Section 21E of the Securities Exchange Act of 1934, as amended,
including statements about potential treatments for cancer or other
diseases employing combinations of drug therapies including Idera’s
third generation gene silencing technology. Such statements
may be identified by words such as "believe," "expect," "may,"
"plan," "potential," "will" and similar expressions, and are based
on the company’s current beliefs and expectations. Development of
drug therapies involves a high degree of risk, and only a small
percentage of research and development programs undertaken may
result in the commercialization of a product. Positive
preclinical data does not ensure that later stage clinical trials
will be successful. For more detailed information on the
risks and uncertainties associated with Idera’s development
activities, please review the Risk Factors section of Idera's most
recent annual or quarterly report filed with the Securities
and Exchange Commission. Any forward-looking statements speak only
as of the date of this press release and the company assumes no
obligation to update any forward-looking statements, whether as a
result of new information, future events or otherwise.
Investor Contact:
Robert Doody
VP, IR & Corporate Communications.
617-679-5515 (office)
484-639-7235 (mobile)
rdoody@iderapharma.com
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