Positive data from Gloucester Pharmaceuticals' ISTODAX study presented at the ASH meeting

Dec 7 2009

Gloucester Pharmaceuticals announced today the presentation of positive data from an analysis of ISTODAX^® (romidepsin) in a subset of patients from Gloucester’s registration study in cutaneous T-cell lymphoma (CTCL) at the 51^st American Society of Hematology (ASH) Annual Meeting being held in New Orleans, LA. This analysis is an assessment of patients with CTCL who—in addition to skin disease and potential lymph node and visceral involvement—also had blood involvement. Blood involvement is characterized as having greater than five percent circulating Sézary (malignant) cells and is typically associated with advanced stage, aggressive disease. ISTODAX is a member of a new class of cancer drugs known as histone deacetylase (HDAC) inhibitors and was recently approved by the U.S. Food and Drug Administration for the treatment of cutaneous T-cell lymphoma in patients who have received at least one prior systemic therapy.

“CTCL that includes blood involvement represents a major therapeutic challenge and the results of this analysis suggest that ISTODAX may help address a treatment void for these patients,” said Dr. Youn Kim, an investigator in studies of ISTODAX and Professor, Department of Dermatology and Director, Multidisciplinary Cutaneous Lymphoma Program, Stanford Cancer Center, Stanford, CA. “In this subset of 37 patients with blood involvement, the response rate is consistent with the response rate from the overall population of 96 patients in the registration trial. Additionally, the rapid and sustained reduction in Sézary counts observed in many patients with a higher blood tumor burden, which is typically associated with advanced stage, more aggressive disease, is noteworthy."

The results were described in a poster presentation entitled, “Clinically Significant Responses Achieved with Romidepsin in 37 Patients with Cutaneous T-Cell Lymphoma (CTCL) with Blood Involvement.” Of the 96 patients enrolled in the registration study, 37 patients had blood involvement. In this subset of patients with blood involvement> The composite assessment included measurement of skin, lymph node and visceral involvement in addition to measurement of circulating malignant T-cells (Sézary cells). The median duration of response in the 37 patients with blood involvement has not been reached; however, the current maximum duration of response is 20 months. Pruritus, intense itching, was measured in these patients using a 100mm visual analog scale (VAS). 14 of 24 patients (58%) with moderate to severe pruritus at baseline (≥30 mm VAS) had a clinically meaningful decrease (≥30 mm) in VAS. The safety profile in this subset of 37 patients was similar to the overall safety profile of ISTODAX in the registration study which was characterized principally by mild (grade 1 and 2) gastrointestinal disturbances, hematologic toxicities, clinical chemistry abnormalities and asthenic conditions. The only study-drug related serious adverse event occurring in more than one patient was tumor lysis syndrome (2 patients).

“We’re pleased to see that the results in this subset of patients with more challenging disease are similar to those observed overall in the registration study,” commented Jean Nichols, President and Chief Operating Officer of Gloucester Pharmaceuticals. “These data demonstrate that ISTODAX has the potential to provide sustained clinical benefit to patients with CTCL with blood involvement who have received at least one prior systemic therapy. In addition, the observed improvements in pruritus are encouraging because pruritus can be a particularly troubling symptom for this patient population."