— 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 —
CAMBRIDGE, Mass., March 3, 2011 (GLOBE NEWSWIRE) -- 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.
Phase 2 Chondrosarcoma Trial Design
The Phase 2 trial is designed to compare the safety and efficacy of IPI-926, administered orally once daily, to matching placebo in patients with metastatic or locally advanced, inoperable chondrosarcoma. The primary endpoint of the trial is progression free survival. Secondary endpoints include time to progression, overall survival, overall response rate and response duration. Patients in the placebo treatment arm who experience disease progression will have the option to crossover and receive IPI-926 in an open-label arm of the trial.
Rationale for IPI-926 in Chondrosarcoma to Be Highlighted at AACR
Through a collaboration with Drs. Jay Wunder and Ben Allman at University of Toronto, preclinical data have been generated to suggest that the Hedgehog pathway is activated in chondrosarcoma tumor cells and that inhibition of the pathway may delay tumor progression. The rationale for development of IPI-926 in chondrosarcoma will be discussed by Margaret Read, Ph.D., senior director, product development at Infinity, during a symposium session at the upcoming American Association for Cancer Research (AACR) 102nd Annual Meeting 2011. Dr. Read's presentation, entitled "Direct targeting of tumor cells with Smoothened inhibitor IPI-926," will take place during "The Hedgehog Signaling Pathway: Biology and Therapeutics" symposium on Sunday, April 3, 2011, from 1:00 p.m. – 3:00 p.m. ET.
About Chondrosarcoma
Chondrosarcoma is a rare, life-threatening bone cancer. In the U.S., chondrosarcoma accounts for approximately one-third of the 2,000 cases of primary bone cancer diagnosed each year.i The most common locations for chondrosarcoma tumors are the bones of the extremities and the pelvis.i Chondrosarcoma predominantly affects middle-aged and older adults, usually occurring in patients over 40 years old, with the incidence gradually increasing up to age 75.ii,iii Symptoms associated with chondrosarcoma depend upon the size and location of the tumor and often include pain that increases in severity over time, localized swelling and decreased range of motion in joints near the affected bone.iii,iv As chondrosarcomas are largely resistant to chemotherapy and radiotherapy, the standard therapeutic strategy is surgery. For patients with metastatic disease or with locally advanced tumors who are not candidates for surgery, no treatment has been shown to be effective and there is no established standard of care.
About the Hedgehog Pathway and IPI-926
Malignant activation of the Hedgehog pathway occurs in a broad range of cancers through three distinct mechanisms: signaling to the tumor microenvironment, signaling to tumor progenitor cells, and signaling directly to tumor cells. IPI-926 is a small molecule that inhibits Smoothened (Smo), a key component of the Hedgehog pathway. Smo inhibition represents a significant anti-cancer opportunity for addressing a number of difficult-to-treat cancers by disrupting malignant activation of the pathway.
IPI-926 is currently being evaluated in the Phase 2 portion of an ongoing trial in combination with gemcitabine (also known as Gemzar®) in previously untreated patients with metastatic pancreatic cancer and in a Phase 2 trial as a single agent in patients with chondrosarcoma. In a Phase 1 trial of IPI-926 in advanced solid tumors, including a cohort of patients with basal cell carcinoma, IPI-926 has been generally well-tolerated and demonstrated evidence of clinical activity. These clinical trials build upon a robust set of supporting data that provide a strong rationale for evaluating the potential of IPI-926 for treatment across a broad range of cancers.
About Infinity Pharmaceuticals, Inc.
Infinity is an innovative drug discovery and development company seeking to discover, develop, and deliver to patients best-in-class medicines for difficult-to-treat diseases. Infinity combines proven scientific expertise with a passion for developing novel small molecule drugs that target emerging disease pathways. Infinity's programs in the inhibition of the Hedgehog pathway, the Hsp90 chaperone system, fatty acid amide hydrolase, and phosphoinositide-3-kinase are evidence of its innovative approach to drug discovery and development. For more information on Infinity, please refer to the company's website at www.infi.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include those regarding the potential utility of Hedgehog pathway inhibition and IPI-926 in addressing chondrosarcoma and other cancers, the presenting of rationale for development of IPI-926 in chondrosarcoma, and clinical trial enrollment expectations. Such statements are subject to numerous factors, risks and uncertainties that may cause actual events or results to differ materially from the company's current expectations. For example, there can be no guarantee that IPI-926 will successfully complete necessary preclinical and clinical development phases or that Infinity's strategic alliance with Mundipharma International Corporation Ltd. will continue for its expected term or that it will fund Infinity's programs as agreed. Management's expectations could also be affected by risks and uncertainties relating to: results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites; Infinity's ability to enroll patients in its clinical trials; unplanned cash requirements and expenditures, including in connection with business development activities; the development of agents by Infinity's competitors for diseases in which Infinity is currently developing its product candidates; and Infinity's ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing. These and other risks which may impact management's expectations are described in greater detail under the caption "Risk Factors" included in Infinity's quarterly report on Form 10-Q filed with the Securities and Exchange Commission on November 9, 2010. Any forward-looking statements contained in this press release speak only as of the date hereof, and Infinity expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Gemzar® is a registered trademark of Eli Lilly and Company.
Sources:
i National Comprehensive Cancer Network Practice Guidelines in Oncology – V.3.2010. Bone Cancer.
ii Dorfman HD, Czerniak B, Kotz R, Vanel D, Park YK, Unni KK. WHO classification of tumours of bone: Introduction. In: Fletcher CDM. Unni KK, Mertens F.eds. World Health Organization Classification of Tumours. Pathology and Genetics of Tumours of Soft Tissue and Bone. Lyon, France: IARC Press; 2002.
iii Yasko Aw, Chow W, Fressica D. Bone Sarcomas. In: Pazdur R, Wagman LD, Camphausen, Hoskins WJ, eds. Cancer Management: A Multidisciplinary Approach (ed 12): CMPMedicas LLC; 2008.
iv Springfield D, Rosen G. Bone Tumors. In: Kufe DW, Bast RC, Hait WN, Hong WK, Pollock RE, Weichselbaum RR, Holland JF, Frei E, eds. Cancer Medicine 7 (ed 5). Hamilton, Ontario: BC Decker; 2006; 1686-1687.