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New Medical Therapies™

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 September 22, 2014

Avanir Pharmaceuticals reported results of a phase II trial of AVP-923 for agitation related to Alzheimer’s disease. The trial was a 10- week, multicenter, randomized, double-blind, placebo-controlled study utilizing a SPCD design intended to reduce placebo response rates, enrolling 220 Alzheimer’s patients in the U.S. Patients were initially randomized 3:4 to receive either AVP-923 (dose escalated from DM 20mg/ Q 10mg to DM 30mg/ Q 10mg) or placebo. At the end of week five, patients who initially received placebo were stratified according to their response to treatment and subsequently re-randomized 1:1 to receive either AVP-923 or placebo for the remainder of the study. Patients who initially received AVP-923 continued to receive AVP-923 DM 30mg/Q 10mg for the remainder of the study. Treatment with AVP-923 was associated with significantly reduced agitation as measured by the primary endpoint, the agitation/aggression domain score of the neuropsychiatric inventory (NPI) compared to placebo (p=0.00008). The reduction in agitation was observed in both stage one (p=0.0002) and stage two (p=0.021) of the sequential parallel comparison study design. In addition, improvements also were seen in a majority of the secondary endpoints including the NPI total score (p=0.014), clinical global impression of change-agitation (p=0.0003), patient global impression of change (p=0.001) and measures of caregiver burden (p≤0.05).

QLT issued results of a phase Ib trial of QLT091001 in subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) due to inherited genetic mutations in retinal pigment epithelium (RPE65) or lecithin: retinol acyltransferase (LRAT). This multicenter, open-label retreatment trial was an extension study in which 27 LCA or RP subjects with RPE65 or LRAT mutations received up to three seven-day courses of QLT091001 to assess visual outcomes and safety following retreatment. Subjects received treatment with QLT091001 at 10mg/m2, 40mg/m2 or 60mg/m2, with the majority of subjects receiving 40mg/m2. Results showed 19 of 27 subjects (70%) had a visual field response (increase in retinal area of at least 20% from the study baseline at two consecutive visits starting within six months of any study treatment), and 19 of 27 subjects (70%) having a visual acuity response (increase of at least five letters at two consecutive visits starting within six months of any study treatment). These responses were durable, with the visual field response lasting an average of 235 days (Min-Max = 7 – 742 days), and the visual acuity response lasting an average of 232 days (Min-Max = 7 – 616 days). Overall, 10 of 13 LCA subjects (77%), and 12 of 14 RP subjects (86%) were classified as responders for either visual field retinal area or visual acuity.

Sangamo BioSciences released results of a phase I trial of SB-728-T for HIV/AIDS. The study was an open-label trial to evaluate single infusions of an escalating dose of an autologous (a patient’s own) CD4+ T-cell product genetically modified at the CCR5 gene by CCR5-specific ZFNs (SB-728-T). The trial enrolled nine HIV-infected subjects (three cohorts of three subjects each) who had sub-optimal T-cell levels and no detectable viral load on long-term ART, so-called immunologic non-responders (INRs). Three CCR5 delta 32 heterozygote subjects have controlled VL to undetectable or <1000 copies during a treatment interruption (TI) from ART, one for more than 59 weeks (to last measurement taken). Two subjects experienced a two-log decrease in viral load from peak (with Cytoxan conditioning of 1gm/m and 1.5gm/m), which has been sustained in one subject for more than 39 weeks. Five subjects currently remain on extended TI (longer than the 16 week period defined in the protocol) with VLs <10,000 copies and CD4 counts of >500. Analyses showed a large increase in CCR5-modified cells in the long-lived T(SCM) compartment, which may explain why CCR5- modified cells from a single infusion can be detected in all subjects over a prolonged period (more than 42 months). A median 0.9 log decrease in the size of the HIV reservoir at 36 months was observed in nine of nine subjects treated, as demonstrated by measurement of HIV total DNA in PBMCs. The decrease in reservoir showed a statistically significant correlation with an improvement in CD4 count. Sangamo is conducting an ongoing phase II clinical trial, SB-728-mR-1401 (1401).

Ultragenyx Pharmaceutical issued results of a phase I/II extension study of KRN23 (UX023) in adult patients with X-linked hypophosphatemia (XLH). The extension study (INT-002) was designed to follow an initial four-month dose escalation study (INT-001) that was conducted in the U.S. and Canada. 22 adult patients with XLH were evaluated over an additional 12 months. Patients received monthly subcutaneous injections of KRN23 administered at a dose range of 0.1mg/kg to 1mg/kg. Data demonstrated the increases in serum phosphorus levels, urinary phosphorus reabsorption and 1,25 dihydroxy vitamin D levels observed in the initial INT-001 study were generally sustained during the 12-month extension. All patients continued to demonstrate increases in serum phosphorus levels. Approximately 52.6%-85.7% of subjects in the extension study had serum phosphorus levels that reached the normal range (2.5mg/dL to 4.5mg/dL) at peak time on day seven or day 14 after each dose over this 12-month period. The company plans to continue development in adult XLH patients and is enrolling pediatric XLH patients in an ongoing phase II study.

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