May 15 2015
Dicerna Pharmaceuticals, Inc., a leader in the development of RNAi therapeutics, today announced it is expanding its ongoing Phase 1 study of DCR-MYC in solid tumors, multiple myeloma, or lymphoma to include a cohort of patients with pancreatic neuroendocrine tumors (PNETs) following early signs of clinical and metabolic response and tumor shrinkage in PNET patients.
DCR-MYC is an investigational Dicer substrate short-interfering RNA (DsiRNA) therapeutic targeting the MYC oncogene and the first MYC-targeting short-interfering RNA (siRNA) to enter clinical trials.
“Based on the clinical activity seen in two out of three patients with PNET, evidence of a complete metabolic response (based on FDG-PET) and a partial response (based on RECIST criteria), as well as published evidence on the role of MYC in growth and maintenance of PNET tumors, we are adding a PNET expansion cohort in the ongoing Phase 1 study,” said Pankaj Bhargava, M.D., chief medical officer of Dicerna. “Most patients with PNET have unresectable or metastatic disease at the time of diagnosis. Despite recent improvements in treatment, advanced PNET remains an incurable disease. There are only two approved targeted therapies (Sutent® and Afinitor®) for advanced PNET, both with modest response rates, and there are no approved treatments for patients who progress after standard therapies. Therefore, there remains a clear unmet medical need for patients with advanced PNET.”
The ongoing Phase 1 study, initiated in April 2014, is a multi-center, dose-escalation trial designed to assess the safety and tolerability of DCR-MYC in patients with solid tumors, multiple myeloma, or lymphoma who are refractory or unresponsive to standard therapies. The study is designed to identify the maximum tolerated dose (MTD), the pharmacokinetic profile, potential pharmacodynamic effects, and antitumor activity of DCR-MYC. Once the MTD is established, an expansion cohort will be opened to enroll patients with PNET. This multi-center expansion cohort will enroll up to 20 patients with low- to intermediate-grade PNET who have demonstrated disease progression after treatment with standard therapies.
Preliminary safety, tolerability, clinical, and metabolic response data from the ongoing Phase 1 trial of DCR-MYC will be presented at the 2015 ASCO Annual Meeting during the Tumor Biology Oral Abstract Session on Monday, June 1, 2015.
Source: http://dicerna.com/