Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), 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-8400administered 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.
About Diffuse Large B-cell Lymphoma (DLBCL)
DLBCL is the most common form of non-Hodgkin lymphoma (NHL) with approximately 20,000 new cases diagnosed in the United States each year.1DLBCL is a fast-growing and potentially lethal lymphoma. Initial symptoms may include rapid swelling in the neck, armpit and groin due to enlarged lymph nodes. Other symptoms include night sweats, unexplained fevers and weight loss.2 The company believes that about 10 percent of DLBCL patients harbor the MYD88 L265P oncogenic mutation, and data from an independent trial of DLBCL patients have shown that prognosis in this population is poor, with overall survival markedly impaired compared to patients without the MYD88 L265P mutation.3,4,5
Idera's Toll-like receptor (TLR) antagonist drug candidates have been created using a proprietary chemistry-based drug discovery platform. IMO-8400 is a first-in-class synthetic oligonucleotide-based antagonist of endosomal TLRs 7, 8, and 9. In April 2014, Idera presented preclinical data at the American Association for Cancer Research Annual Meeting from preclinical studies in which IMO-8400inhibited the survival and proliferation of human B-cell lymphoma cells, including Waldenstrom's macroglobulinemia (WM) cells, harboring the oncogenic MYD88 L265P genetic mutation. IMO-8400 has been well-tolerated in a Phase 1 trial in 42 healthy subjects at single and multiple escalating doses up to 0.6 mg/kg for four weeks, and has shown inhibition of immune responses mediated by TLRs 7, 8, and 9. Idera is pursuing clinical development of IMO-8400 in genetically defined forms of B-cell lymphoma, including WM and diffuse large B-cell lymphoma in patients harboring the MYD88 L265P mutation, and in rare autoimmune diseases, including dermatomyositis.
Shares of Idera closed yesterday at $3.71 . IDRA has a 1-year high of $5.48 and a 1-year low of $1.94. The stock's 50-day moving average is $4.36 and it's 200-day moving average is $3.59.
On the ratings front, Idera has been the subject of a number of recent research reports. In a report issued on December 17, Piper Jaffray analyst Edward Tenthoff reiterated a Buy rating on IDRA, with a price target of $7, which implies an upside of 88.7% from current levels. Separately, on December 9, Cowen's Boris Peaker maintained a Buy rating on the stock .
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Edward Tenthoff and Boris Peaker have a total average return of 29.4% and 36.4% respectively. Tenthoff has a success rate of 68.3% and is ranked #164 out of 3562 analysts, while Peaker has a success rate of 64.8% and is ranked #26.
Idera Pharmaceuticals Inc is a clinical stage biotechnology company.The Company is engaged in the discovery, development and commercialization of novel therapeutics for oncology and rare diseases.