Infinity Expands Clinical Development Program for IPI-926, an Oral Smoothened Antagonist Targeting the Hedgehog Pathway

Infinity Pharmaceuticals, Inc. INFI today announced that it is expanding its clinical development program for IPI-926, a novel, oral small molecule that inhibits Smoothened, a key component of the Hedgehog pathway. As part of this expansion, Infinity plans to initiate an exploratory Phase 2 clinical trial in patients with myelofibrosis, an incurable malignancy of the bone marrow which is characterized by the replacement of normal bone marrow by fibrotic tissue and the production of blood cells in other organs, such as the spleen and liver. The single-arm, Phase 2 trial is designed to evaluate the safety and efficacy of IPI-926 administered orally once daily in up to 45 patients with myelofibrosis. The primary endpoint of the trial is hematologic response rate. The trial is expected to begin in the third quarter of 2011. "The important role of the Hedgehog pathway in pathogenic fibrosis leads us to believe that IPI-926 may have application across a range of malignancies, including pancreatic cancer and myelofibrosis, in which malignant cells create a dense, fibrotic, protective microenvironment via the Hedgehog pathway signaling to the stroma," stated Pedro Santabárbara, M.D., Ph.D., chief medical officer at Infinity. "While other agents in development for the treatment of myelofibrosis reduce spleen size, there is still a significant need for novel treatment options that can directly target the malignant fibrosis underlying this disease."