Molecular Insight Pharmaceuticals Inc announced that the European Medicines Agency (EMEA) has approved its phase-3 protocol for Onalta (Yttrium-90 edotreotide). Onalta is the company's lead radiotherapeutic product candidate under development for the treatment of metastatic carcinoid and pancreatic neuroendocrine tumours in patients whose symptoms are not controlled by conventional therapy. The compound has shown the potential to selectively deliver lethal radiation to cancer cells.
The proposed phase-3 trial will confirm that administration of Onalta results in stabilization, regression or complete remission of the carcinoid tumour, and improves carcinoid-related symptoms when compared to a high-dose regimen of the current standard therapy for this disease, Sandostatin. With EMEA's approval of its proposed phase-3 protocol in hand, Molecular Insight can proceed with the final clinical trial that will position Onalta for marketing authorization in the EU.
EMEA notified Molecular Insight that its Onalta phase-3 protocol is acceptable, including the plan to assess and manage renal tolerance, that the endpoints and measures are appropriate, and that the number of patients proposed to be presented in support of safety for the future Marketing Authorization Application (MAA) is acceptable.
John W Babich, executive vice president, chief scientific officer and president of Research and Development of Molecular Insight, said that, "This is a significant Company milestone that allows us to proceed with a pivotal trial for Onalta in Europe. A successful trial would mean an innovative and well-understood therapy could be made available to thousands of patients whose lives are being cut short and who suffer from the debilitating symptoms that accompany this disease."
"The notification by EMEA comes on the heels of our Special Protocol Assessment (SPA) agreement with FDA allowing us to proceed to a pivotal phase-2 trial for Azedra Ultratrace (Iobenguane I-131) in another neuroendocrine cancer, pheochromocytoma. Azedra is also currently being evaluated for treatment of neuroblastoma. These drug candidates represent significant opportunities to provide neuroendocrine cancer patients with innovative therapies. Molecular Insight has selected Progression Free Survival (PFS) as the primary endpoint for the proposed phase-3 study and overall survival will be assessed as a key secondary endpoint. The regulatory progress in Europe will hopefully be followed by regulatory progress in the USA."
According to Dr Val Lewington, consultant physician, Royal Mardsen NHS Foundation Trust, "EMEA approval of this Phase 3 protocol represents a landmark in radionuclide therapy. The potential of this approach is well-recognized in Europe but this will be the first opportunity to evaluate this peptide-based radiotherapy in a major, multi-center, randomized clinical trial. It is difficult to overstate the importance of this step in the development of innovative therapies for inoperable neuroendocrine tumors which so often prove refractory to other treatment options. News of the EMEA decision will be welcomed enthusiastically both by clinicians and by patients with neuroendocrine disease."
The proposed phase-3 protocol will evaluate 194 patients with metastatic, progressive, somatostatin receptor-positive, carcinoid cancer, receiving either Onalta or the standard of care. Carcinoid cancer is a rare, serious and life-threatening condition that affects a group of patients with few treatment options. Once the disease has metastasized, the patients' prognosis is poor: the best five-year survival rate is reported to be 20 to 30 per cent.
Onalta is intended to complement Azedra, Molecular Insight's other clinical stage radiotherapeutic candidate for the treatment of neuroendocrine tumours. In 2007, Molecular Insight acquired Onalta from Novartis Pharma AG, which had conducted three phase-1 and three phase-2 clinical trials involving more than 300 patients.
Molecular Insight has been developing Onalta as a treatment for metastatic pancreatic neuroendocrine and carcinoid tumours in patients whose symptoms are not controlled by current somatostatin analogue therapy.
Molecular Insight Pharmaceuticals is a biopharmaceutical company specializing in the emerging field of molecular medicine, applying innovations in the identification and targeting of disease at the molecular level to improve healthcare for patients with life-threatening diseases.