November 21, 2016
Ionis Pharmaceuticals announced positive data from a phase II study evaluating IONIS-FXIRx in patients with end-stage renal disease (ESRD) on hemodialysis. The study was a double-blinded, randomized, placebo-controlled study in 43 patients. The primary purpose of the study was to understand the drug’s behavior and activity in patients with severe kidney disease on dialysis. Patients received 200mg or 300mg of IONIS-FXIRx or placebo for 12 weeks. In patients treated with 200mg and 300mg of IONIS-FXIRx, a mean percent reduction in FXI activity of 56% (p=<0.001) and 71% (p=<0.001), respectively, was achieved at week 13, compared to a mean percent reduction of 4% for placebo-treated patients. Furthermore, a decrease in severe clotting events in the dialysis circuit after six weeks compared to baseline was observed. IONIS-FXIRx displayed a favorable safety and tolerability profile. In this small three-month study, there were no clinically meaningful reductions in platelets and no treatment-related major or clinically relevant non-major bleeding events. There were no treatment-related serious adverse events. An increase in minor bleeding events was observed in patients receiving the 300mg dose. In addition, there were no clinically meaningful changes in laboratory values, including those related to liver function. IONIS-FXIRx was well-tolerated in the study with no flu-like or injection site reactions.
February 25, 2013
Argos Therapeutics reported results from a phase II trial of AGS-003 for metastatic renal cell carcinoma (mRCC). This open-label study enrolled 21 patients with unfavorable risk mRCC and with an expected survival of approximately 15 months. Subjects received standard six-week cycles of sunitinib plus AGS-003, administered every three weeks for five doses, and then every 12 weeks until progression. Results showed median overall survival was 30.2 months. When analyzed by baseline Heng risk status, the median overall survival for intermediate risk patients (n=11) has not been reached, but is estimated to be longer than 39.5 months with continued follow-up. Median overall survival for poor risk patients (n=10) was 9.1 months. Of the 21 patients who participated in the study, seven (33%) patients are still alive after nearly four years or longer following study registration. Argos Therapeutics is currently enrolling patients in a global phase III study of AGS-003.
February 18, 2013
AVEO Oncology and Astellas Pharma reported results from a phase III trial of tivozanib compared to sorafenib for the treatment of advanced renal cell carcinoma (RCC). This global, randomized study, TIVO-1, enrolled 517 patients with RCC. Subjects received either tivozanib or sorafenib. The final overall survival (OS) analysis showed a median OS of 28.8 months (95% confidence interval [CI]: 22.5–NA) for tivozanib versus a median OS of 29.3 months (95% CI: 29.3–NA) for the comparator arm, sorafenib. No statistical difference between the two arms (HR=1.245, p=0.105) was observed. Patients randomized to the sorafenib arm were eligible to cross over to tivozanib therapy under a separate protocol after radiographic confirmation of disease progression. No crossover protocol was available for patients randomized to the tivozanib arm. Tivozanib was well tolerated. Based on these data, AVEO and Astellas have filed a New Drug Application (NDA) for tivozanib for the treatment of RCC.
November 28, 2011
Mologen released results from a phase I/II trial of MGN1601, a therapeutic vaccine for the treatment of renal cancer. This open, non-randomized study enrolled 19 subjects in whom the standard treatment proved to be unsuccessful. The subjects were treated with MGN1601 several times over a period of 12 weeks. Nine subjects completed the full three month treatment cycle. In one subject the size of the metastases was reduced by more than 50%, and in two cases the progress of renal cancer was stopped. Two subjects are still on treatment and the therapeutic success with these two subjects has lasted for more than six months. One subject is in the first phase of the trial. MGN1601 was well tolerated and no serious adverse effects were reported. Due to these results, enrollment was stopped early and the treatment of the remaining three subjects will continue as planned.
May 6, 2002
Phase II trial results suggest a strong correlation between clinical response and pharmacological action for GD0039, GlycoDesign's lead anti-cancer drug. The trial, which was conducted at five Canadian centers, included 18 subjects with metastatic renal cancer and 22 subjects with metastatic colorectal cancer resistant to 5-fluorouracil. In 14 trial subjects, the cell-surface carbohydrate structures targeted by GD0039 were highly inhibited. Within this population, four of six renal cancer subjects and three of eight colorectal cancer subjects experienced tumor shrinkage or stable disease.