Idera Pharmaceuticals Reports Positive Data From Ongoing Phase 1/2 Clinical Trial of IMO-8400 in Patients With Waldenstrom's Macroglobulinemia
ORLANDO, Fla., Dec. 05, 2015 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (NASDAQ: IDRA), a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare diseases, today presented initial clinical data from its ongoing Phase 1/2 clinical trial for IMO-8400, a Toll-like receptor 7, 8 and 9 antagonist, being evaluated for the treatment of patients with relapsed or refractory Waldenström's Macroglobulinemia (WM). These results provide evidence that IMO-8400 has clinical activity and is well tolerated. Today's results were presented during a poster session (Abstract #1540) at the 57th Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL.
"Our clinical trial in Waldenström's Macroglobulinemia represents the first step in our understanding of the potential role that TLR antagonism could play in B-cell malignancies, specifically in those harboring the MYD88-L265P oncogenic mutation which is highly prevalent in Waldenström's Macroglobulinemia," stated Vincent Milano, Idera's Chief Executive Officer. "We are pleased that the initial results from this ongoing trial met our objectives in determining safety and tolerability, as well as clinical activity of IMO-8400 in this patient population. We are further encouraged that the safety profile seen to date will enable us to expand this study to evaluate higher dosing levels of IMO-8400.
The results being reported are from 15 evaluable patients with Waldenström's Macroglobulinemia who had a history of relapse or failure to one or more prior therapies and who completed at least one cycle of therapy with IMO-8400. Patients enrolled in the multi-center, open-label, dose ranging clinical trial which evaluated 3 dose levels of IMO-8400 (06. mg/kg weekly, 1.2 mg/kg weekly, 1.2mg/kg twice a week) administration for a period of up to 24 weeks. The primary objectives of the study were to assess safety and tolerability. Secondary objectives were to assess clinical activity, PK and define the optimal dose for further clinical evaluation. In addition to clinical treatment parameters, cytokine levels were analyzed as an exploratory endpoint in the trial.
Top Line Results
- IMO-8400 was generally well tolerated at all dose levels studied.
- The Maximum Tolerated Dose of IMO-8400 has not yet been identified.
- Across all dose cohorts, 6 of 15 patients (40%) with relapsed or refractory WM had an objective response.
- Three responders were refractory to their last treatment, including 1 patient who was refractory to ibrutinib.
- In the highest dose cohort (1.2 mg/kg twice a week):
- 3 of 6 patients (50%) had an objective response and two had stable disease.
- The median time to first response was ~10.5 weeks.
- There was improvement in bone marrow findings, hemoglobin and disease symptoms.
- An exploratory analysis showed a significant correlation between change in M-protein and a change in IL-10, with decreases in IL-10 being seen in responding patients.
- These data in patients with WM provide the first clinical evidence supporting inhibition of the TLR pathway as a potential therapeutic approach for B-cell malignancies characterized by the MYD88 L265P oncogenic mutation.
- Evaluation of higher IMO-8400 dose levels is planned.
The full poster presentation is currently available on the Investors Page of the Idera corporate website which can be found at www.iderapharma.com.
Investor Event and Webcast
Idera will host a conference call and live webcast on Monday, December 7 at 9:00 A.M. EST to review the data being presented at ASH along with discussion of next steps for the IMO-8400 development program in B-cell Lymphomas. To participate in the conference call, please dial (866) 379-0841 (domestic) and (440) 996-5667 (international). The webcast can be accessed live or in archived form in the "Investors" section of the company's website at www.iderapharma.com. The company has also posted a slide presentation to the Idera corporate website which will be referenced during the conference call.
About Waldenström's macroglobulinemia (WM)
Waldenström's macroglobulinemia is a rare and slow-growing form of B-cell lymphoma with approximately 1,000 to 1,500 new cases diagnosed in the United States each year.1 The median age at diagnosis is between 60 and 70 years of age. Symptoms include fatigue, night sweats, headaches, visual problems, pain and abnormal bleeding due to complications such as anemia, retinopathy and peripheral neuropathy.2 About 90 percent of WM patients harbor the MYD88 L265P oncogenic mutation.3
About Toll-Like Receptors (TLRs) and Idera's Proprietary TLR Antagonism Technology Platform
TLRs are receptor proteins that play a central role in the innate immune system. In healthy people, TLRs recognize invading pathogens and endogenous molecules released from damaged or dysfunctional cells, and initiate signaling cascades that trigger an inflammatory response. Through these signaling cascades, TLRs are also involved in activating the adaptive immune system, in which B-cells play a critical role. Based on the company's proprietary chemistry-based discovery platform, Idera discovered and is developing the synthetic oligonucleotide-based TLR antagonist, IMO-8400. This clinical-stage candidate has demonstrated activity in multiple preclinical models of cancer and autoimmune disease.
About Idera Pharmaceuticals
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 is developing a third generation antisense technology platform 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.
1 American Cancer Society. What are the key statistics about Waldenstrom macroglobulinemia? Available at: http://www.cancer.org/cancer/waldenstrommacroglobulinemia/detailedguide/waldenstrom-macroglobulinemia-key-statistics-w-m. Accessed December 2014.
2 American Cancer Society. Signs and Symptoms of Waldenstrom macroglobulinemia. Available at: http://www.cancer.org/cancer/waldenstrommacroglobulinemia/detailedguide/waldenstrom-macroglobulinemia-signs-symptoms. Accessed December 2014.
3 Treon SP, et al. MYD88 L265P somatic mutation in Waldenström's macroglobulinemia. N Engl J Med. 2012 Aug 30;367(9):826-33.
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 preclinical data 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 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; and such other important factors as are set forth under the caption "Risk Factors" in the Company's Annual Report and on Form 10-Q for the period ended September 30, 2015. 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.