Cytori Therapeutics Inc., of San Diego, said the SCLERADEC-I trial of Cytori cell therapy (ECCS-50) showed sustained benefit at two-year follow-up in patients with hand dysfunction associated with scleroderma. Two-year data from the investigator-initiated, open-label trial, which enrolled 12 patients, showed sustained improvement in the key efficacy endpoints of Cochin Hand Function Score, Scleroderma health assessment questionnaire, Raynaud's condition score and hand pain, as assessed by a standard visual analogue scale. Average values for the four endpoints at two years showed statistically significant improvement over baseline and similar improvement to one-year scores. No safety issues related to Cytori cell therapy were reported. (See BioWorld Today, Aug. 7, 2015.)
Eisai Ltd., of Hatfield, UK, said it will present data in a poster session at the San Antonio Breast Cancer Symposium exploring the mode of action of Halaven (eribulin) and showing its impact in reversing the epithelial-to-mesenchymal transition. The company also will report data from a study of stage I/II hormone receptor-positive/Her2 negative breast cancer suggesting that women with the luminal B form of the disease might benefit most from eribulin, which induces a luminal A phenotype. Data highlighting the potential of eribulin to work in combination with other therapies will include the design of a phase Ib/II study to evaluate the efficacy and safety of eribulin in combination with pembrolizumab (Keytruda, Merck & Co. Inc.) in patients with metastatic triple-negative breast cancer. A second study will explore whether PEGPH20 (pegylated recombinant human hyaluronidase, Halozyme Therapeutics Inc.) enhances the efficacy of eribulin in triple negative breast cancer xenografts. Eribulin is approved in Europe to treat adults with locally advanced or metastatic breast cancer who progressed after at least one chemotherapeutic regimen for advanced disease.
Flexion Therapeutics Inc., of Burlington, Mass., said it enrolled the first patient in a pharmacokinetic study of Zilretta (FX006) in osteoarthritis of the knee. The U.S. multicenter, open-label trial will enroll approximately 50 patients, who will receive 40 mg of Zilretta administered as a single 5 mL intra-articular injection. The study is designed to provide additional characterization of local concentrations of triamcinolone acetonide, the active ingredient in Zilretta, in the knee over 20 weeks to support the company's new drug application to the FDA, expected in the second half of next year. (See BioWorld Today, Sept. 5, 2014.)
Immunovaccine Inc., of Halifax, Nova Scotia, said initial results from a phase II study suggested that DPX-Survivac, its lead cancer immune therapy, can induce a strong immune response in hematologic cancers, including diffuse large B cell lymphoma (DLBCL). Researchers observed changes in tumor-infiltrating T cells following administration of the DPX-Survivac therapy, correlating with an immune response produced by DPX-Survivac and detected in the blood, according to Immunovaccine. Analysis of tumors collected from vaccinated patients also provided a rationale for combining the DPX-Survivac therapy with checkpoint inhibitors, including anti-PD-1 agents, to enhance the potential benefit of DPX-Survivac in DLBCL patients. The results came from the first two evaluable patients who completed the scheduled therapy.
Kamada Ltd., of Ness Ziona, Israel, said it will unblind its current clinical trial testing of its human alpha-1 antitrypsin (AAT) in newly diagnosed pediatric and young adult patients with type 1 diabetes (T1D) at the planned interim analysis, which will accelerate the timeline for future commercialization of the product, should the analysis be positive. The double-blind, placebo-controlled trial is evaluating the safety and efficacy of intravenous AAT to halt disease progression and maintain the ability of the pancreas to produce insulin. It is evaluating beta cell functioning as measured by C-peptide parameters, glycemic control expressed in HbA1C levels, hypoglycemic events and insulin daily dose, among other measurements, all of which will be part of the final report. Kamada previously reported preliminary data from the extension portion of its phase I/II trial of AAT to treat pediatric patients newly diagnosed with T1D, showing that at about 26 months from diagnosis and about 10 months following the last AAT infusion, 60 percent of study subjects who participated in the extension portion of the trial had peak C-peptide levels greater than 0.2 pmol/ml, which indicates a functioning beta cell capacity and is considered to be a higher percentage than would be expected without intervention.
Newron Pharmaceuticals SpA, of Milan, said it started a U.S. phase II study with its sodium channel blocker, NW-3509, in patients with schizophrenia. The, four-week, double-blind, placebo-controlled randomized trial will test NW-3509, administered as add-on treatment, in schizophrenic patients experiencing breakthrough psychotic symptoms while on stable and adequate doses of their current atypical antipsychotics. About 60 patients will be enrolled. Results are expected in the fourth quarter of 2016.
Oncogenex Pharmaceuticals Inc., of Bothell, Wash., said its phase III AFFINITY trial is continuing based on the pre-planned interim futility analysis of the intent-to-treat population, following an independent data monitoring committee (DMC) meeting. In the final safety review, no new safety issues were identified. Both the DMC and Oncogenex remain blinded to all analyses and final results are expected in the second half of 2016, depending on timing of the event-driven final analysis. AFFINITY is designed to evaluate whether clusterin inhibitor custirsen, when combined with Jevtana (cabazitaxel, Sanofi SA),improves survival in men with metastatic castrate-resistant prostate cancer whose disease has progressed following treatment with docetaxel. Oncogenex also reported results from an analysis of a prospectively defined subpopulation of men in the AFFINITY trial who had multiple poor prognostic risk factors, showing that the combination of custirsen and cabazitaxel did not meet the rigorous criteria required to demonstrate an improvement in overall survival (hypothesized hazard ratio ≤0.69, one-sided p ≤0.015). That subpopulation was identified and evaluated based on a retrospective analysis of a previous phase III trial of men with similar clinical features who experienced a reduced risk of death when custirsen was added to chemotherapy. Shares of Oncogenex (NASDAQ:OGXI) fell 99 cents, or 37.8 percent, to close Wednesday at $1.63. (See BioWorld Today, April 29, 2014.)
Ra Pharmaceuticals Inc., of Cambridge, Mass., said it launched a phase I study of its synthetic peptide C5 inhibitor, RA101495. The randomized, double-blind study is designed to test the safety of single, escalating doses of RA101495 and to establish the pharmacokinetics and pharmacodynamics after subcutaneous administration in healthy volunteers.
Scynexis Inc., of Jersey City, N.J., said it dosed the first group of patients in its phase II study of antifungal candidate SCY-078, an oral glucan synthase inhibitor, as a treatment for vulvovaginal candidiasis (VVC). The randomized, evaluator-blinded study will compare one of two doses of SCY-078 to oral fluconazole in at least 90 adult female patients with moderate to severe VVC. The study will assess efficacy, safety, tolerability and pharmacokinetics.
Shionogi Inc., of Florham Park, N.J., started a phase III trial to evaluate the safety and efficacy of Osphena (ospemifene) for the treatment of moderate to severe vaginal dryness, a symptom of vulvovaginal atrophy (VVA) due to menopause. The randomized, double-blind, placebo-controlled study will assess the safety and efficacy of Osphena 60 mg once-daily oral therapy in postmenopausal women with vaginal dryness as the most bothersome symptom of VVA. About 600 postmenopausal women between the ages of 40 and 80 will be randomized in a 1:1 ratio to treatment with Osphena or placebo. Safety and efficacy will be assessed at 12 weeks, and study participants will be followed for two years. Osphena is already FDA-approved for the treatment of moderate to severe painful sex, known as dyspareunia.
Ultragenyx Pharmaceutical Inc., of Novato, Calif., reported interim data from the first 12 patients in the ongoing pediatric phase II study of its recombinant human monoclonal antibody KRN23 against fibroblast growth factor 23 (FGF23) for the treatment of X-linked hypophosphatemia (XLH). An improvement in mean rickets score was observed after 40 weeks of treatment with investigational KRN23. Ultragenyx is conducting the study under a collaboration and license agreement with Kyowa Hakko Kirin Co. Ltd. to develop and commercialize KRN23. No serious adverse events have been reported in the study to date and there have been no discontinuations from the study so far. For the 12 patients who had reached 40 weeks at the time of the interim analysis, the most common adverse events considered to be treatment-related were injection site reactions. All patients in the study of the FDA-fast tracked drug continue to receive KRN23. An additional 40-week analysis for 36 patients is planned for the fourth quarter of 2015.