Orphan Autoimmune diseases

Idera’s business strategy is focused on developing targeted therapies to treat rare and severe autoimmune diseases with high unmet medical need. In autoimmune diseases, overactivation of Toll-like receptors (TLRs) by tissue and organ damage is implicated in the exacerbation of the disease. This overactivation of TLRs leads to the production of multiple cytokines, including Tumor Necrosis Factor Alpha, and other interleukins. Current therapeutic approaches involve antibodies that block specific cytokines.

By contrast, Idera’s therapeutic approach is to block the overactivation of TLRs, a biologic target that is upstream of traditional cytokine targets, thereby inhibiting multiple cytokines without affecting their normal function. Our goal is to develop novel therapies to treat autoimmune diseases, with improved long-term safety profiles and impact on disease remission.

Clinical Proof-of-concept of TLR Antagonism

To date, Idera has established clinical proof-of-concept of TLR antagonism in patients with an autoimmune disease. In a recently completed randomized, double-blind, placebo-controlled Phase 2 trial of patients with moderate-to-severe plaque psoriasis, treatment with IMO-8400 was well tolerated over a 12-week treatment period. In addition, IMO-8400 treatment led to improvement in Psoriasis Area and Severity Index (PASI) scores. (Learn more) In an earlier Phase 2 trial, PASI score improvements were correlated with downregulation of activated genes in the IL-17 pathway. Full data from the Phase 2 trial of IMO-8400 in psoriasis will be submitted for presentation at an upcoming medical meeting.

Prioritization of Orphan Autoimmune Indications for Development

With this encouraging proof-of-concept data on the role of TLR antagonism in an autoimmune disease the Company undertook an extensive evaluation of rare autoimmune diseases to prioritize indications for development and commercialization. The three indications chosen for near-term development are polymyositis, dermatomyositis, and graft-versus-host disease (GvHD).

Polymyositis and dermatomyositis are devastating, rare inflammatory myopathies that cause inflammation and progressive weakness in muscles. Polymyositis and dermatomyositis patients can develop serious disabilities, including loss in mobility, difficulty breathing and swallowing, and have an increased risk of certain cancers. Dermatomyositis is also accompanied by a purple or red skin rash. There are an estimated 10, 000 polymyositis patients and 67, 000 dermatomyositis patients in the U.S. alone. Both polymyositis and dermatomyositis have been designated as orphan diseases by the U.S. Food and Drug Administration (FDA).

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