Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of July 18, 2016

CytRx issued results of a phase III trial of aldoxorubicin compared to investigator’s choice of therapy in patients with relapsed or refractory soft tissue sarcomas (STS). The randomized, controlled trial enrolled 433 patients at 79 sites in 15 countries. Patients with metastatic, locally advanced or unresectable soft tissue sarcomas who had either not responded to, or who had progressed following treatment with one or more systemic regimens of non-adjuvant chemotherapy, were randomized 1:1 to be treated with aldoxorubicin or the investigator’s choice of an approved chemotherapeutic regimen, including doxorubicin, ifosfamide, dacarbazine, pazopanib (Votrient) or gemcitabine plus docetaxel. The primary endpoint of the study was PFS. Secondary endpoints include overall survival, response rates and safety. The study did not show a significant difference between aldoxorubicin and investigator’s choice of therapy for PFS, with a median of 4.17 months and 4.04 months, respectively, for the study’s primary endpoint (hazard ratio: 0.91). However, the most immediate indications of therapeutic activity, objective response rate (ORR) and disease control rate (ORR + stable disease four months), showed a near doubling in the aldoxorubicin arm compared to investigator’s choice, including in patients who previously received treatment with doxorubicin. Disease control rate for aldoxorubicin was significantly greater than investigator’s choice therapy in the intent-to-treat population (p=0.048) as well as in patients who received prior doxorubicin (p=0.0415). Patients continue to be followed for overall survival (OS), a secondary endpoint of the trial.

Sage Therapeutics released results of a phase II trial of SAGE-547 for the treatment of severe postpartum depression (PPD). The multicenter, placebo-controlled, double-blind, 1:1 randomization trial was designed to enroll up to 32 women. The population studied were women with severe PPD (HAM-D ≥26) who developed severe depression either in the third trimester or within four weeks of childbirth. At baseline, the mean HAM-D scores for both groups was greater than 28. The primary objective of the trial was to evaluate the effect of SAGE-547 on depression as measured by the HAM-D score, compared to placebo, at 60 hours. In addition, patients were monitored during a 30-day follow-up period to assess both safety and efficacy. SAGE-547 achieved the primary endpoint of a significant reduction in the HAM-D score compared to placebo at 60 hours (p=0.008). This represented a greater than 20-point mean reduction in the depression scores of the SAGE-547 group at the primary endpoint of 60 hours through trial completion with a greater than 12-point difference from placebo. The statistically significant difference in treatment effect began at 24 hours, (p=0.006) with an effect that was maintained at similar magnitude through to the 30-day follow-up (p=0.01). Remission from depression, as determined by a HAM-D ≤7, measured at 60 hours, was seen in seven of 10 in the SAGE-547 group compared with one of 11 in the placebo group (p=0.008). Similarly, at 30 days, seven of 10 of the SAGE-547 group and two of 11 in the placebo group were in remission (p=0.03). SAGE-547 was found to be generally well-tolerated with no serious adverse events reported during the treatment and follow-up periods. The company has initiated an expansion program to determine optimal dosing of SAGE-547 in PPD.

Valeant Pharmaceuticals International and Nicox announced results of a phase III study for latanoprostene bunod (LBN) ophthalmic solution 0.024% for open angle glaucoma (OAG) or ocular hypertension (OHT). The prospective, double-masked, parallel group trial compared LBN 0.024% with timolol maleate 0.5%. The results of this study showed that mean IOP was significantly lower in the LBN 0.024% group than in the timolol 0.05% group at all time points (range 17.7 - 18.7 mm Hg for LBN 0.024%; 18.8-19.6 mm Hg for timolol 0.5%; p≤0.025) except for week two at 8 a.m. (19.2 mm Hg for LBN 0.024% v. 19.6 mm Hg for timolol 0.5%; p=0.216). These differences corresponded to a reduction from baseline ranging from 29.1% to 32.1% in the LBN 0.024% group and 25.2% to 28.7% in the timolol group. Adverse events, though uncommon, were slightly higher in the LBN group. They included conjunctival hyperemia, eye irritation and eye pain, and were mostly mild-to-moderate in severity.