— Company Granted U.S. Orphan Drug Designation for IPI-926 for the Treatment of Chondrosarcoma —
— Rationale for Developing IPI-926 in Chondrosarcoma to be Highlighted During a Hedgehog Pathway Symposium at AACR —
Infinity Pharmaceuticals, Inc. (Nasdaq:INFI) today announced the initiation of a randomized, double-blind Phase 2 clinical trial to evaluate the safety and efficacy of IPI-926 compared to placebo in patients with metastatic or locally advanced, inoperable chondrosarcoma. The trial is expected to enroll over 100 patients worldwide. Chondrosarcoma is a rare, life-threatening bone cancer. There are no approved systemic treatments for this disease and, when surgery is no longer possible, chondrosarcoma is uniformly fatal.
IPI-926 is a novel, oral molecule that inhibits Smoothened, a key component of the Hedgehog pathway. In chondrosarcoma, activation of the Hedgehog pathway is believed to be involved in tumor initiation, growth, survival and metastases. Inhibiting Smoothened with IPI-926 may represent a fundamentally new approach to treating chondrosarcoma by disrupting the Hedgehog pathway and inhibiting tumor growth.
"There is a strong preclinical rationale for treating chondrosarcoma by inhibiting the Hedgehog pathway with IPI-926, and we look forward to further exploring its potential in this rigorous Phase 2 trial," stated Julian Adams, Ph.D., president of research and development at Infinity. "Chondrosarcoma has been a challenging disease to treat because of its resistance to chemotherapy and radiation. With surgery as the primary treatment option, there is a significant unmet need for a therapy for patients with metastatic or inoperable chondrosarcomas."
Infinity also announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to IPI-926 for the treatment of chondrosarcoma. Orphan Drug Designation is a status granted by the FDA under the Orphan Drug Act for a product that treats a rare disease or condition and is designed to encourage companies to develop therapies to treat rare diseases or conditions. Companies that receive Orphan Drug Designation for their products may receive funding for certain clinical trials, clinical trial design assistance, tax credits, a waiver of Prescription Drug User Fee Act (PDUFA) fees and marketing exclusivity for up to seven years following regulatory approval.