Idera Pharmaceuticals and Parent Project Muscular Dystrophy Announce Collaboration to Advance New Treatment Approach for Duchenne Muscular Dystrophy

  • Toll-like receptor antagonism designed to treat muscle inflammation central to disease progression
  • New approach offers potential to treat broad population of patients regardless of their genetic mutation

CAMBRIDGE, Mass. and HACKENSACK, N.J., Nov. 7, 2014 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals (Nasdaq:IDRA) and Parent Project Muscular Dystrophy (PPMD) today announced a collaboration to advance Idera's proprietary Toll-like receptor (TLR) technology for the treatment of Duchenne muscular dystrophy. As part of this collaboration, Idera and PPMD intend to work together to conduct preclinical studies and develop a clinical development strategy for an investigational TLR antagonist candidate. In previous preclinical studies in models of Duchenne, treatment with a TLR antagonist candidate led to a reduction in disease-associated markers of inflammation and improved muscle function. Idera is a clinical-stage biopharmaceutical company focused on developing nucleic acid therapeutics for patients with rare and serious diseases. PPMD is the largest and most comprehensive nonprofit organization in the U.S. focused on finding a cure for Duchenne.

"We look forward to partnering with Idera Pharmaceuticals, a leader in the development of nucleic acid therapeutics for rare diseases, to advance a novel approach to controlling muscle inflammation with the potential to treat all patients regardless of their genetic mutation," said Pat Furlong, Founding President and CEO of PPMD. "One of our organization's primary objectives is to advance potential treatments so that all people with Duchenne have an opportunity to live longer and fuller lives. For 20 years, we have done whatever we can to support new and promising technologies. PPMD looks forward to working with Idera on this new area of biology to advance treatments applicable to all patients."

"We are very pleased to work with PPMD on advancing the potential application of TLR antagonism to address the underlying inflammation that propagates disease progression in Duchenne. PPMD brings decades of experience working with families affected by Duchenne, as well as close ties with scientists and clinicians working in the field worldwide," said Sudhir Agrawal, D.Phil., Chief Executive Officer of Idera Pharmaceuticals. "We look forward to collaborating with PPMD and researchers from Children's National Health System, to conduct preclinical studies to inform a clinical development strategy."

Duchenne is a rare, fatal neuromuscular disorder characterized by progressive muscle weakness, increasing disability limiting activities of daily living, pulmonary and cardiac dysfunction, and death typically before age 30. It affects approximately 15,000 to 20,000 patients in the United States and is caused by the lack of dystrophin, an essential protein that stabilizes the membranes of muscle cells.

Due to compromised cellular membranes, dying and damaged muscle cells release self RNA and other molecules which are recognized by the body's innate immune system as Damage Associated Molecular Patterns (DAMPs). In preclinical studies, researchers have shown that DAMPS stimulate TLR-mediated signaling pathways that trigger an inflammatory response. This TLR-mediated inflammatory response is believed to cause additional muscle cell damage, propagating a cycle of tissue damage that contributes to disease progression. TLRs represent novel targets in Duchenne upstream of other traditional inflammatory targets such as NF-KB and TNF-α.

Summary of Preclinical Data Supporting TLR Antagonism in Duchenne

"Over the last five years, we have demonstrated in preclinical studies that TLRs play a critical role in triggering inflammation in neuromuscular diseases such as Duchenne. Based on this research, we believe there is a clear scientific rationale for the evaluation of TLR antagonism as a potential treatment approach," said Kanneboyina Nagaraju, Ph.D., DVM, Professor of Integrative Systems Biology and Pediatrics at the George Washington University School of Medicine and Health Sciences and Children's National Health System.

Independent research published by investigators from Children's National Health System in Washington, DC, have demonstrated the role of TLRs in the pathogenesis of DMD. Results from this study showed a statistically significant 6.6-fold over expression of TLR 7 in the muscle fibers of patients with Duchenne compared with healthy controls aged 5 to 12 years. In addition, investigators reported that TLR 7 was also over expressed in pre-symptomatic infants, suggesting TLR activation is an early trigger of muscle inflammation in Duchenne.1

Additional preclinical research published by investigators from Children's National Health System and Idera showed that inhibition of TLR activity improved disease-associated measures in Duchenne models. To assess the potential utility of inhibiting TLR activity, investigators knocked out the gene for MYD88, an adaptor protein in the TLR signaling pathway, and evaluated muscle function in the mdx mouse model of Duchenne. Results showed statistically significant improvements in skeletal and cardiac muscle function. In a separate experiment, mdx mice treated with an antagonist of TLR 7 and TLR 9 showed significant reductions in muscle inflammation and creatine kinase, a marker of muscle damage, and increased muscle force.2

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest, most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy – PPMD's mission is to end Duchenne.

PPMD invests deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. PPMD advocates in Washington, DC, and has secured hundreds of millions of dollars in funding. PPMD demands optimal care, and strengthens, unites, and educates the global Duchenne community.

Everything PPMD does -- and everything it has done since its founding in 1994 -- helps boys with Duchenne live longer, stronger lives. PPMD will not rest until every young man has a treatment to end Duchenne. Go to for more information or to learn how you can support our efforts and help families affected by Duchenne.

About Children's National Health System

Children's National Health System, based in Washington, DC, has been serving the nation's children since 1870. Children's National is Magnet® designated, and is consistently ranked among the top pediatric hospitals by U.S. News & World Report. Home to the Children's Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children's National is one of the nation's top NIH-funded pediatric institutions. With a community-based pediatric network, seven regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children's National is recognized for its expertise and innovation in pediatric care and as an advocate for all children. For more information, visit, or follow us on Facebook and Twitter.

About Idera Pharmaceuticals

Idera Pharmaceuticals is a clinical-stage biopharmaceutical company developing a novel therapeutic approach for the treatment of genetically defined forms of B-cell lymphoma and rare diseases. Idera's proprietary technology involves creating novel nucleic acid therapeutics designed to inhibit over-activation of Toll-like Receptors (TLRs). In addition to its TLR programs, Idera is developing gene silencing oligonucleotides (GSOs) that it has created using its proprietary technology to inhibit the production of disease-associated proteins by targeting RNA. To learn more about Idera, visit


1 Chen Y.-W., et al. Early onset of inflammation and later involvement of TGFβ in Duchenne muscular dystrophy. Neurology. 2005;65;826-834.

2 Henriques A, et al. Role of Toll-like receptors in the pathogenesis of dystrophin-deficient skeletal and heart muscle. Hum Mol Genet. 2014 May 15;23(10):2604-17.

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. All statements, other than statements of historical fact, included or incorporated in this press release, including statements regarding the Company's strategy, future operations, collaborations, intellectual property, cash resources, financial position, future revenues, projected costs, prospects, plans, and objectives of management, are forward-looking statements. The words "believes," "anticipates," "estimates," "plans," "expects," "intends," "may," "could," "should," "potential," "likely," "projects," "continue," "will," and "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Idera cannot guarantee that it will actually achieve the plans, intentions or expectations disclosed in its forward-looking statements and you should not place undue reliance on the Company's forward-looking statements. There are a number of important factors that could cause Idera's actual results to differ materially from those indicated or implied by its forward-looking statements. Factors that may cause such a difference include: whether results obtained in preclinical studies and clinical trials such as the results described in this release will be indicative of the results that will be generated in future clinical trials; whether products based on Idera's technology will advance into or through the clinical trial process on a timely basis or at all and receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether, if the Company's products receive approval, they will be successfully distributed and marketed; whether the Company's collaborations will be successful; and such other important factors as are set forth under the caption "Risk Factors" in the Company's Quarterly Report on Form 10-Q for the three months ended September 30, 2014. Although Idera may elect to do so at some point in the future, the Company does not assume any obligation to update any forward-looking statements and it disclaims any intention or obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

CONTACT: Parent Project Muscular Dystrophy Media Contact: Will Nolan Vice President, Communications and Administration (201) 250-8440 Idera Pharmaceuticals Investor and Media Contact: Jim Baker Executive Director, Corporate Affairs (617) 679-5516

Source:Idera Pharmaceuticals