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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 May 22, 2017

Ionis Pharmaceuticals reported results of the phase III NEURO-TTR study of inotersen (IONIS-TTRRx) in patients with familial amyloid polyneuropathy (FAP). Inotersen was evaluated in a randomized (2:1), double-blind, placebo-controlled, international study in 172 patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The study was designed to support an application for marketing approval of inotersen in patients with hATTR-PN. The 15-month study measured the effects of inotersen on neurological dysfunction and on quality of life by measuring the change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Over the 15-month period of the study, inotersen-treated patients achieved statistically significant benefit compared to placebo in mNIS+7 and Norfolk QoL-DN (p<0.0001 and p=0.0006, respectively). Statistically significant differences were also observed for both endpoints at eight months. Treatment-emergent adverse events considered related to treatment were seen more commonly with inotersen than placebo. Two key safety findings were observed during the study that required changes to the monitoring schedule. Three serious adverse events of thrombocytopenia were observed in inotersen-treated patients; two patients recovered and one patient died due to intracranial hemorrhage. The preparation of regulatory marketing applications for inotersen is underway. GSK has the option to license inotersen following review of additional data and prior to the submission of regulatory applications.

Photocure released phase III study results of Blue Light Flexible Cystoscopy (BLFC) with Hexvix/Cysview for bladder cancer. The study was a prospective, open, comparative, within-patient controlled study, and included 304 patients with non-muscle invasive bladder cancer (NMIBC) enrolled at 17 academic institutions in the US. In the study, BLFC with Cysview was used with the Karl Storz D-Light C PDD Flexible Videoscope System. The study showed that BLFC with Cysview detected bladder cancer recurrence in 21.5% of the patients undergoing surveillance cystoscopy that otherwise would have been missed with white light (WL) alone, which is highly significant (p<0.0001). Of note, the study showed that nine out of 26 patients (34.6%) with flat, more aggressive high grade lesions (carcinoma in situ; CIS) were diagnosed using confirmatory Blue Light Cystoscopy with Cysview alone and not WL (p<0.0001). The study also showed that there was no increase in the rate of related adverse events after repeated administration of Cysview in bladder cancer patients undergoing cystoscopy examination. The company intends to submit the data to the FDA to seek approval of BLFC with Cysview in the surveillance setting in the U.S., and an expansion of the indication to include CIS.

Synergy Pharmaceuticals issued data from an analysis of patients with moderate to very severe bloating at baseline who participated in two phase III studies evaluating Trulance (plecanatide) for the treatment of adults with chronic idiopathic constipation (CIC). Over 12 weeks, patients with CIC and moderate, severe or very severe bloating symptoms at baseline and were treated with Trulance 3mg or 6mg doses achieved a significantly greater efficacy responder rate—the primary endpoint defined by the FDA for regulatory approval in CIC—in this analysis compared to placebo (18.8% for 3mg and 16.3% for 6mg compared to 9.5% for placebo). Efficacy responders were defined as patients who had at least three complete spontaneous bowel movements (CSBMs) in a given week and an increase of at least one CSBM over baseline in the same week for at least nine weeks out of the 12-week treatment period, including at least three of the last four weeks. The symptom of bloating among these patients also showed statistically significant improvements for Trulance 3mg and 6mg compared to placebo. Improvements in abdominal bloating scores were statistically significant after one week and continued throughout the 12-week treatment period. In both studies, the most common adverse event was diarrhea (4.1% at 3mg and 4.5% at 6mg compared to 0.7% for placebo). Trulance is a once-daily tablet approved by the FDA for the treatment of adults with CIC and is currently being evaluated for the treatment of adults with irritable bowel syndrome with constipation (IBS-C).

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