While taking a break from pouring over Pharma market cap data, I checked out how the FDA ODAC meetings were going today.
This morning, Gloucester Pharma were slated first and many VC's and investors must have been a tad nervous after the two negative votes for Genzyme and Vion in AML yesterday. Gloucester recently announced $29M in series D financing to further the development of their HDAC inhibitor, romidepsin. Everyone needn't have worried, since the committee voted with 10 in favour and 1 abstained.
The discussion this morning focused on refractory CTCL, a rare form of non-Hodgkins Lymphoma (NHL). CTCL is caused by a mutation of T cells, unlike most non-Hodgkin's lymphomas which are generally of B-cell origin. The malignant T cells involve the skin, causing plaques, patches, erythroderma and/or tumors and can involve other organs, including the blood lymph nodes and viscera.
In the Phase 2B, international, multicenter registration study of romidepsin in patients with CTCL (n=96, intent-to-treat), patients refractory to prior therapy who received romidepsin had an overall response rate of 34% i.e. 33 out of 96 patients. This exceeded the primary endpoint of the study, which was a 15% improvement.
Overall, complete and partial responses were observed at all stages of disease. The median duration of response was 14.9 months. Despite the exclusion of steroid and antihistamine use, 63% of patients with moderate to severe pruritus at baseline experienced significant pruritus relief, a key indicator of clinical benefit. The most common adverse effects in clinical trials of romidepsin include fatigue, gastrointestinal disturbances and generally mild to moderate hematologic toxicity.
Currently, treatments for CTCL include Merck's Zolinza (vorinostat), the first HDAC (approved in 2006) for this indication, as well as Ontak (denileukin diftitox), approved in 1999, and Targretin (bexarotene), all of which were approved on the basis of single arm studies.
A Prescription Drug User Fee Act (PDUFA) date of November 12, 2009 has
been set, so it looks likely that the FDA may well approve romidepsin for
treatment of cutaneous T-cell lymphoma (CTCL), including relief of pruritus, in patients who have received at least one prior systemic therapy before 2009 is out. Patients will then have a new therapy option once initial treatment has failed.