The company is working on a new approach to treating bone erosion based on a common pathway in the cell that infiltrates bone and joints. Bone erosion disables millions of people each year. It occurs in children and adults with cancer, after trauma and infections, but most cases are idiopathic. A prime idiopathic example is the millions of adults who live with chronic RA. Patients with multiple myeloma suffer from painful bone lesions caused by cancer-induced overproduction of the cell that normally controls bone development.

Bone lesions often require treatments with serious side effects. Later in arthritis, bone erosion is a major problem that causes severe pain and debilitation. There is no small-molecule drug available to treat arthritis bone erosion specifically. Bisphosphonates used for multiple myeloma patients to control bone lesions have severe side effects. We show that osteoclast maturation is suppressed by blocking ion channels. Our leading drug candidate, ion-channel antagonist ELP-004, suppresses osteoclast maturation and strongly suppresses bone erosion in two mouse models of arthritis, even after symptoms of arthritis were measurable, and prevents bone lesions from developing with multiple myeloma in mice.

We hypothesize that signals mediated by specific ion channels modulate the final differentiation of osteoclasts resulting in bone and joint degradation. We propose that pharmacologically suppressing specific ion channels with ELP-004 will prevent bone erosion due to idiopathic and pathologic stimuli without major adverse effects. Preclinical toxicity assays show that ELP-004 has minimal toxicity to the immune and cardiovascular systems and has no measurable genotoxicity. Continued comprehensive pharmacokinetic and toxicokinetic testing, in addition to mechanistic experiments with ion-channel signaling, immunology, and bone biology expertise, is underway.