Idera Announces FDA Orphan Drug Designation for IMO-8400 for the Treatment of Diffuse Large B-Cell Lymphoma

Idera Pharmaceuticals, Inc.

, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for oncology and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for IMO-8400, an antagonist of the endosomal Toll-like receptors (TLRs) 7, 8 and 9, for the treatment of diffuse large B-cell lymphoma (DLBCL). Idera is currently conducting a clinical trial of IMO-8400 in patients with relapsed or refractory DLBCL harboring MYD88 L265P oncogenic mutation (ClinicalTrials.gov identifier NCT02252146). Preclinical studies have shown that in B‐cell lymphomas characterized by the MYD88 L265P oncogenic mutation, including DLBCL, TLR signaling is over-activated, thereby enabling tumor cell survival and proliferation. As a TLR antagonist, IMO-8400 inhibits TLR signaling. The objectives of the trial are to evaluate the compound's safety, tolerability and clinical activity. The protocol includes three dose-escalation cohorts of IMO-8400 administered subcutaneously. "The Orphan Drug designation granted today represents another positive milestone for our B-Cell Lymphoma clinical development program," stated James J. O'Leary, MD, Idera's interim Chief Medical Officer. "We continue to advance our efforts in DLBCL, as well as our ongoing clinical trial in Waldenstrom's macroglobulinemia (WM), which we expect to complete and have full data available in the fourth quarter of this year." Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs intended for the treatment of a rare disease or condition that affects fewer than 200,000 people in the United States. This designation provides certain incentives, including eligibility for federal grants, research and development tax credits, waiver of PDUFA filing fees and a seven-year marketing exclusivity period, once the product is approved and as long as orphan drug designation is maintained. The approval of an orphan drug designation request does not alter the standard regulatory requirements and processes for obtaining marketing approval of an investigational drug. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.