Release Details

Phase 2 clinical programs progressing in two genetically targeted lymphoma indications

New partnerships with myositis and GvHD communities further upcoming clinical development programs

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 12, 2014-- Idera Pharmaceuticals, Inc. (NASDAQ: IDRA), a clinical stage biopharmaceutical company developing nucleic acid therapeutics for rare diseases, today reported financial and operational results for the quarter ended June 30, 2014. The Company highlighted progress in advancing drug candidates for the treatment of mutation-specific forms of B-cell lymphoma and autoimmune diseases, as well as its next-generation antisense technology platform.

“During the past quarter, we made significant progress in advancing our clinical programs. Our trials in Waldenström’s macroglobulinemia (WM) and diffuse large B-cell lymphoma (DLBCL) are advancing, and we are making progress towards initiating clinical development in myositis and graft versus host disease (GvHD) by year-end,” said Sudhir Agrawal, D. Phil., Chief Executive Officer of Idera Pharmaceuticals. “We are also pleased to have put in place invaluable relationships with global key opinion leaders and patient foundations to help us advance these important programs."

Recent Developments

Idera has significantly progressed both its clinical development programs for rare disease indications and its gene silencing oligonucleotide (GSO) technology, a novel third-generation antisense platform.

Programs for Genetically Defined Forms of B-cell Lymphoma

• Activated additional clinical sites at leading U.S. institutions and continued enrollment in a Phase 1/2 trial of IMO-8400 in patients with WM who have relapsed or were refractory to prior therapies.
• Activated the first two sites and initiated patient screening in a Phase 1/2 clinical trial of IM0-8400 in patients with DLBCL who harbor the MYD88 L265P mutation and have relapsed or were refractory to prior therapies.
• Significantly advanced the development of a companion diagnostic for the MYD88 L265P mutation under the Company's collaboration with Abbott Molecular. The Company now expects to augment ongoing patient screening activities with Abbott’s PCR-based screening assay in the third quarter.
• Presented new preclinical data at the ASH Meeting on Lymphoma Biology 2014 in Colorado Springs, CO, showing that IMO-8400 treatment of MYD88 L265P mutation-positive DLBCL cell lines inhibited tumor cell growth and key mediators of signaling, including IRAK-1, IRAK-4, BTK, STAT3, NF-κB, IκB, and IL-10.
• Demonstrated that IMO-8400 treatment of primary bone marrow cells from WM patients positive for the MYD88 L265P mutation led to dose-dependent inhibition of cell growth, apoptosis and down-regulation of genes implicated in cell survival. In addition, IMO-8400 treatment in a WM-derived tumor model led to inhibition of tumor growth and suppression of systemic IgM levels. The Company plans to present these data at the 8^thInternational Workshop on Waldenström’s Macroglobulinemia on August 14 in London.
• Long-term observational data published by independent investigators in the second quarter revealed that DLBCL patients harboring the MYD88 L265P mutation experienced poor outcomes and demonstrated decreased response to current therapies, as compared to patients without the mutation. These data further highlight the serious unmet medical need in this population and the potential utility of a mutation-specific treatment approach.¹

Programs for Rare Autoimmune Diseases

• Completed a strategic analysis of rare autoimmune diseases in which TLRs play a significant role in the disease pathogenesis and there is significant unmet medical need. This evaluation led to the prioritization of myositis and GvHD as the Company's lead autoimmune indications for its product candidate IMO-8400. Myositis is a rare inflammatory muscle disease with limited treatment options. GvHD is a significant complication of allogeneic stem cell transplantation.
• Formed a Myositis Expert Advisory Board, made up of world-class medical experts in myositis (including Anthony Amato, M.D., Lisa Christopher-Stine, M.D., M.P.H., David Fiorentino, M.D., Ph.D., David Isenberg, M.D., Ingrid Lundbery, M.D., Frederick W. Miller, M.D., Ph.D., Tahseen Mozaffar, M.D., Chester Oddis, M.D., and Lisa Rider, M.D.), and moved forward with plans to initiate clinical development in polymyositis and dermatomyositis by year-end.
• Announced a collaboration with The Myositis Association (TMA) to educate patient and clinical communities about TLR antagonism as a potential therapeutic approach, including highlighting opportunities to participate in the Company’s upcoming clinical trial.
• Entered into collaborations with leading GvHD investigators at Ohio State University, Washington University in St. Louis, and the University of Minnesota to carry out preclinical evaluations of TLR antagonist candidates in both acute and chronic GvHD animal models, in concert with Idera's plan to initiate clinical development of IMO-8400 for GvHD by year-end.

Additional pipeline programs

• Completed the clinical portion of a placebo-controlled Phase 2 trial of IMO-8400 in psoriasis at four escalating dose levels. Top-line data showed that IMO-8400 was well tolerated at all dose levels and demonstrated clinical activity as evidenced by improvements in the Psoriasis Area Severity Index (PASI) score, an established rating system for psoriasis. Detailed data from the trial will be submitted for presentation at a future medical meeting.
• Completed non-clinical safety studies to support initial clinical evaluation of IMO-9200, a second novel antagonist of TLR7, TLR8 and TLR9. The Company expects to initiate a Phase 1 clinical study of IMO-9200 in healthy volunteers in the second half of 2014.

Gene Silencing Oligonucleotide (GSO) Platform

• Conducted preclinical studies of compounds from its GSO platform, a novel third-generation antisense technology designed to mitigate immunotoxicity and provide an increased therapeutic index. Data from these research experiments showed treatment-related improvements in disease-associated biomarkers and improved potency against multiple clinically relevant gene targets, as compared to other current antisense technologies. Based on these experiments, Idera has initiated preclinical studies on proprietary targets with a goal of initiating proof-of-concept clinical studies for two indications during the second half of 2015.
• Announced the publication of new preclinical data showing that GSOs inhibited specific microRNAs involved in the regulation of neovascularization. In the current online edition of Circulation Research, studies conducted in collaboration with academic investigators enabled validation of the GSO-targeted inhibition of microRNAs and also demonstrated activity in animal models.²
• Presented data at the Nucleic Acid Summit in June in San Diego, entitled “Gene Silencing Oligos (GSOs): Third Generation Antisense,” that support the Company's belief that GSO's may offer superior potency and an improved therapeutic index compared to other current antisense platforms.

"Collectively these achievements in the second quarter testify both to the scientific and clinical potential of Idera's programs and to the quality and dedication of our team, which has continued to add experienced talent," said Dr. Agrawal. "We look forward to updating investors on continued progress in the coming quarters."

Second Quarter 2014 Financial Results

As of June 30, 2014, Idera's cash, cash equivalents and investments totaled $64.7 million compared to $35.6 million as of December 31, 2013.

Net loss applicable to common stockholders for the three months ended June 30, 2014 was $8.4 million, or $0.10 per diluted share, compared to a net loss applicable to common stockholders of $5.6 million, or $0.15 per diluted share, for the same period in 2013. For the six month period, the Company’s net loss applicable to common stockholders was $17.6 million, or $0.22 per diluted share, compared to a net loss applicable to common stockholders of $9.7 million, or $0.30 per diluted share, for the same period in 2013. There was nominal revenue recognized in the second quarter and six month periods of 2014 and 2013.

Research and development expenses for the three months ended June 30, 2014 totaled $5.6 million compared to $2.0 million for the same period in 2013. For the sixth month period, R&D expenses totaled $12.6 million compared to $4.3 million for the same period in 2013.

General and administrative expenses for the three months ended June 30, 2014 totaled $2.7 million compared to $1.6 million for the same period in 2013. For the six-month period, G&A expenses totaled $4.8 million compared to $3.1 million for the same period in 2013.

“To date we’ve made significant progress in building out the operational infrastructure to support our ongoing and planned clinical and preclinical initiatives,” said Lou Arcudi, Chief Financial Officer of Idera Pharmaceuticals. “We believe we are well capitalized and have the resources in place to be able to execute on our current business objectives and carry us through the second quarter of 2016.”

Webcast and Conference Call

Idera will host a conference call today at 8:30 a.m. EDT to discuss second quarter 2014 financial results and corporate highlights.

In order to participate in the conference call, please dial 1-800-901-5241 (domestic) or 1-617-786-2963 (international) and provide the access code 28510971. The live webcast also can be accessed under “Investor Events” in the Investors section of the Company’s website at www.iderapharma.com.

A replay of the call will be available at 1:30 p.m. EDT on August 12, 2014. To access the replay, please dial 1-888-286-8010 (domestic) or 1-617-801-6888 (international) and reference the access code 67213242. The archived webcast will be available for 90 days in the Investors section of Idera’s website at www.iderapharma.com.

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 autoimmune 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 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. 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, including in clinical trials in different disease indications; whether products based on Idera’s technology will advance into or through the clinical trial process when anticipated, 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 six months ended June 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.

References

¹ Fernandez-Rodriguez C, et al. MYD88 (L265P) mutation is an independent prognostic factor for outcome in patients with diffuse large B-cell lymphoma. Leukemia. 2014 Jun 6. Epub ahead of print.

² Welten SM, et al. Inhibition of 14q32 MicroRNAs miR-329, miR-487b, miR-494 and miR-495 Increases Neovascularization and Blood Flow Recovery after Ischemia. Circ Res. 2014 Aug 1. Epub ahead of print.

Source: Idera Pharmaceuticals, Inc.

Investor and Media Contact:
Idera Pharmaceuticals, Inc.
Jim Baker, 617-679-5516
jbaker@iderapharma.com