XERECEPT®
Treatment of Peritumoral edema
XERECEPT® is being developed for the treatment of brain edema (swelling) associated with cerebral tumors. The symptoms of brain edema include headaches, hallucinations, psychotic behavior, memory loss and coma. If left untreated, it can lead to death. The effective management of brain edema is crucial to keeping the patient alive in order to treat the underlying ailment, so it impacts directly on the survival prognosis for these patients.
XERECEPT® is being evaluated as both mono-therapy or in conjunction with conventional steroid therapy e.g. dexamethasone. It is a synthetic version of the natural peptide hormone corticotropin-releasing factor, which reduces the permeability of blood vessel walls. XERECEPT® is expected to permit reduction or in some cases potentially eliminate dexamethasone usage, thus avoiding the severe and sometimes life-threatening side effects associated with high-dose steroid treatment.
XERECEPT® is currently in three Phase III trials:
NTI 0302, an ongoing study, is a randomized, double-blind study comparing Human Corticotropin-Releasing Factor (hCRF) to dexamethasone for the control of symptoms associated with peritumoral brain edema in patients with primary malignant glioma. NTI 0302 is planned to enroll approximately 120 patients with acute symptoms. The endpoint will be a greater than 25% reduction in dexamethasone use, with stabilization or improvement of neurological function as evidenced by stable or improved scores on both the 10-item neurological exam and the Karnofsky scale, which are widely accepted measures of neurological function.
NTI 0303 is a multi-center, placebo-controlled, randomized clinical study being conducted at more than 25 sites in the US and Canada and is planned to include approximately 200 patients with peritumoral brain edema who require treatment with dexamethasone. The primary endpoint of the study will be a 50% reduction in dexamethasone use by the end of Week Two and maintained to Week Five, with stabilization or improvement of neurological function as evidenced by stable or improved scores on both the 10-point neurological exam and the Karnofsky scale.
NTI 0501 is an open-label, extended use study of XERECEPT® for patients who enrolled in either of the 0302 or 0303 studies and elect to receive the drug on a long-term basis through this extension protocol.
Celtic Pharma anticipates a first NDA filing with the FDA in 2008. XERECEPT® has been assigned orphan drug status in the US and a Special Protocol Assessment has been granted by the FDA.
Market
Approximately 35,000 patients are diagnosed every year with primary brain tumors and another 160,000-180,000 patients are diagnosed with metastatic (secondary) brain tumors, in the United States alone. There is a clear wish to reduce the amount of steroids used in managing this patient group. The side effects of high-dose dexamethasone can be distressing for the patient and difficult and expensive to treat successfully.
Clinical Trials
For trial information: http://www.braintumortrials.com/