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 6, 2015

AstraZeneca reported results of a phase III, double-blind, multicenter, placebo-controlled trial of lesinurad used in combination with febuxostat for gout. The study evaluated lesinurad (200mg or 400mg) in combination with febuxostat 80mg in patients who had at least one measurable tophus (deposits of uric acid crystals in joints and skin). Patients were administered febuxostat 80mg orally once daily for three weeks before randomization to the combination treatments. Results showed lesinurad 200mg in combination with febuxostat demonstrated greater (nominal p<0.05) sUA lowering to the target for tophaceous gout of <5mg/dL compared to febuxostat alone at all months except at the time of the primary endpoint, month 6 (56.6% v. 46.8%, non-significant). In the important subgroup of subjects with baseline sUA=5mg/dL, lesinurad 200mg in combination with febuxostat did result in more subjects reaching target sUA of <5mg/dL compared to febuxostat alone at month six (44.1% v. 23.5% respectively; nominal p=0.0243). Lesinurad 400mg in combination with febuxostat met the primary endpoint, with a significantly (p<0.0001) higher proportion of patients reaching the target sUA goal of <5mg/dL at month six compared to febuxostat alone (76.1% v. 46.8%). The MAA and NDA for lesinurad 200mg tablets in combination with an XOI (febuxostat or allopurinol) currently are under review by the CHMP/EMA and the FDA.

Avanir Pharmaceuticals issued results of a phase IIIb trial comparing AVP-825 22mg to sumatriptan 100mg tablets for the treatment of acute migraine. The study is a multicenter, randomized, double-blind, double-dummy crossover study in which migraine sufferers were treated with either 22mg of AVP-825 and placebo or Breath Powered device-delivered placebo and 100mg sumatriptan tablet. Study participants were instructed to treat up to five migraine attacks in each treatment period of up to 12 weeks. Migraine pain and presence of migraine associated symptoms of phonophobia, photophobia and nausea/vomiting were assessed immediately before dosing and at 10, 15, 30, 45, 60, 90, 120 minutes and at 24 and 48 hours after administration. 275 participants and 1,531 migraines were assessed during the study. Results showed that patients treated with AVP-825 achieved pain relief and pain freedom in significantly more migraine attacks at all time points from 15-90 minutes post-dose compared to sumatriptan tablets. AVP-825 and sumatriptan tablets achieved sustained pain relief and pain freedom at 24 and 48 hours in a similar number of attacks. In addition, AVP-825 treated patients experienced early resolution of all migraine-associated symptoms of nausea, sensitivity to sound and to light in more attacks compared with sumatriptan tablets. An NDA for AVP-825 has been accepted and currently is under review by the FDA with a PDUFA goal date of Nov. 6.

BioMarin Pharmaceutical released results of a phase II study of BMN 111 (vosoritide) in children with achondroplasia. The trial was an open-label, sequential cohort dose-escalation study. Patients were treated with either 2.5μg/kg/daily, 7.5μg/kg/ daily or 15μg/kg/ daily. A total of 26 children with achondroplasia with an average age of 7.8 years were enrolled. The 10 children in cohort three treated with 15 micrograms per kilogram per day had a mean increase of 50% (p=0.01) in their annualized growth velocity compared to their annualized prior six month natural history baseline growth velocity. Changes from baseline in proportionality as measured by upper to lower body ratio were not observed. Based on the safety profile observed to date across the three dose cohorts, all subjects have been switched to the highest dose of 15μg/kg/ daily for the duration of the 18 month extension study. Vosoritide has Orphan designation in both the U.S. and Europe.

Radius Health reported results of a phase III trial of abaloparatide for osteoporotic fractures. Radius previously reported positive results for the ACTIVE Trial, meeting the primary endpoint of new vertebral fracture reduction (-86%, p<0.0001) and secondary endpoints of non-vertebral fracture reduction (-43%, p=0.0489) and BMD increases at spine (18M, 9.20% p<0.0001), femoral neck (18M 2.90% p<0.0001) and total hip (18M 3.44% p<0.0001). ACTIVExtend results show that the group previously treated with abaloparatide had no new vertebral fractures during the first six months on alendronate. From the start of the ACTIVE study, this group showed an 87% reduction in new vertebral fractures (p<0.0001), 52% reduction in non-vertebral fractures (p=0.0168), 48% reduction in clinical fractures (p=0.0139) and a 58% reduction in major osteoporotic fractures (p=0.0122) over the 25-month period. A recent exploratory analysis showed that, for major osteoporotic fractures, there was a 67% reduction in major osteoporotic fractures (p=0.0014) for the abaloparatide treatment group v. placebo, and a 53% reduction in major osteoporotic fractures (p=0.0437) for the abaloparatide treatment group as compared to teriparatide. Over the 25-month period, patients in the abaloparatide-alendronate treatment group on average achieved a 12.8% increase in BMD at the lumbar spine, a 5.5% increase in BMD at total hip and a 4.5% increase in BMD at the femoral neck. In this treatment group, 20.4% of patients achieved a 6% increase or greater in BMD at all three sites (i.e., lumbar spine, total hip and femoral neck). The company is preparing regulatory submissions.