Acelrx Pharmaceuticals Inc., of Redwood City, Calif., said ARX-04 (sufentanil sublingual tablet, 30 mcg) met the primary and secondary endpoints in the SAP301 trial, a double-blind, placebo-controlled phase III study of the short-term treatment of patients with moderate-to-severe acute pain following ambulatory abdominal surgery. In the trial, patients who received ARX-04, which was given in a disposable, prefilled single-dose applicator, showed greater pain reduction compared to placebo. Most common adverse events were nausea, headache and vomiting. Data from the trial will be presented at the American Society of Anesthesiologist Annual Meeting in San Diego, Oct. 24-28.
Aduro Biotech Inc., of Berkeley, Calif., said it entered a clinical trial agreement with Wilmington, Del.-based Incyte Corp. to evaluate the safety, tolerability and preliminary efficacy of Aduro's lead LADD immunotherapy, CRS-207, in combination with Incyte's oral indoleamine dioxygenase 1 inhibitor, epacadostat (INCB24360), in patients with ovarian cancer. The phase I/II trial, which is being funded equally between the two companies, is designed to test combinations of CRS-207 with two dose levels of epacadostat in dose escalation and then will expand to a phase II study evaluating the combination at the optimal dose level compared to CRS-207 alone based on safety and tumor biomarkers. The study plans to enroll up to 40 patients in phase I and up to 86 patients in phase II with platinum-resistant ovarian, fallopian or peritoneal cancers. The trial is expected to begin enrolling patients in early 2016.
Akebia Therapeutics Inc., of Cambridge, Mass., reported top-line results from its phase II study of vadadustat (formerly AKB-6548) in dialysis patients with anemia related to chronic kidney disease, showing the study achieved its primary objective, maintaining stable hemoglobin levels throughout the 16-week treatment period following conversion from recombinant erythropoiesis-stimulating agent therapy. Vadadustat, which is designed to stabilize hypoxia inducible factor, also demonstrated a favorable safety profile with no drug-related serious adverse events and no deaths. The 94-patient trial tested the drug in one of three dose cohorts, with patients in the first two cohorts receiving once-daily doses, while patients in the third cohort received vadadustat three times per week in conjunction with their hemodialysis schedule. The positive data sent shares of Akebia (NASDAQ:AKBA) up $3.55, or 45.5 percent, to close Wednesday at $11.36.
Astrazeneca plc, of London, presented updated data on AZD9291 (osimertinib) in first-line patients with epidermal growth factor receptor mutation (EGFRm)-positive advanced non-small-cell lung cancer (NSCLC) and previously treated patients with EGFR T790M mutation-positive NSCLC at the World Conference on Lung Cancer in Denver. Data in the first-line setting demonstrated that in 60 patients who received AZD9291 once daily, 72 percent were progression-free at 12 months. Confirmed overall response rate (ORR) was 75 percent and the longest duration of response (DoR) was ongoing at 18 months. Preliminary data from the phase II AURA studies in previously treated patients with EGFR T790M mutation showed an efficacy and tolerability profile for AZD9291 consistent with previously reported data. In the AURA extension trial, ORR was 61 percent and median DoR and median progression-free survival were not calculable.
Bind Therapeutics Inc., of Cambridge, Mass., said it started patient dosing in the iNSITE 2 trial, a phase II, two-stage trial of BIND-014 in patients with four tumor histologies. The trial is designed to determine the activity, safety and tolerability of BIND-014 and will enroll up to 160 patients. An imaging and biomarker program that can potentially inform the future clinical utility of BIND-014 is also planned. Data readout is expected in the first half of 2016. BIND-014 is a prostate-specific membrane antigen-targeted Accurin containing docetaxel.
Bristol-Myers Squibb Co., of New York, and Pfizer Inc., also of New York, said the first patient has been enrolled in the phase IV trial, Augustus, a two-by-two factorial, randomized controlled trial that will evaluate the safety of the companies' Eliquis (apixaban) vs. warfarin or other vitamin K antagonists (VKA) in patients with non-valvular atrial fibrillation (NVAF) and a recent acute coronary syndrome or undergoing percutaneous coronary intervention, also known as a stent. In addition, patients will be randomized to aspirin or placebo. All patients will receive a P2Y12 inhibitor (such as clopidogrel) in combination with either Eliquis or a VKA. Eliquis is approved to reduce the risk of stroke and systemic embolism in patients with NVAF.
Cara Therapeutics Inc., of Shelton, Conn., said it started a phase III program of intravenous (IV) CR845, a peripherally acting kappa opioid receptor agonist, in postoperative pain, with the dosing of the first subjects in an adaptive pivotal trial in patients undergoing a range of abdominal surgeries. The randomized, double-blind, placebo-controlled, parallel-group adaptive design CLIN3001 trial will test repeated doses of IV CR845 or placebo administered both prior to and following abdominal surgery in male and female patients. The trial will enroll up to 600 patients undergoing hysterectomy, prostatectomy, hemi-colectomy or ventral hernia and will test three dose levels. The primary efficacy measure is the change in pain intensity over the 24-hour postoperative period using the patient-reported Numeric Rating Scale score collected at pre-specified time points through 24 hours. Postoperative nausea and vomiting will be evaluated as a secondary efficacy measure, and the study will explore the impact of treatment of inflammatory biomarkers. Top-line data are expected in the first half of 2016.
Chimerix Inc., of Durham, N.C., provided an update on brincidofovir, for the prevention of cytomegalovirus (CMV) and the treatment of adenovirus infection. The SUPPRESS trial that will evaluate brincidofovir to prevent clinically significant CMV infection in those who have recently undergone hematopoietic cell transplant, or bone marrow transplant, has enrolled 450 patients. The AdVise open-label trial of brincidofovir for disseminated or localized adenovirus infection in patients at increased risk of rapid progression to disseminated disease is fully enrolled with 200 participants. The FDA has recommended that the initial new drug application for brincidofovir be reviewed for efficacy based only on data from SUPPRESS.
Data at the International Epilepsy Congress in Istanbul showed that Tokyo-based Eisai Co. Ltd.'s epilepsy drug Fycompa (perampanel) may significantly reduce seizure frequency, both in patients with primary generalized tonic-clonic (PGTC) seizures (seizure frequency was reduced 62.3 percent versus 31.7 percent placebo shown in a subset analysis of a recently published phase III study  in patients having PGTC seizures with idiopathic generalized epilepsy), and partial epilepsy (8.6 percent of patients were seizure free at six months shown in a retrospective analysis of Spanish data of 315 patients with partial-onset seizures). A subgroup analysis of another phase III trial, showed that Inovelon (rufinamide) demonstrated favorable efficacy as adjunctive treatment for adults with Lennox-Gastaut syndrome. Median change from baseline in seizure frequency was -31.5 percent for rufinamide versus +22.1 percent for placebo.
Endo International plc, of Dublin, said its subsidiary Endo Pharmaceuticals Inc. and Biodelivery Sciences International Inc., of Raleigh, N.C., presented data from a phase II study for the investigational drug buprenorphine buccal film using Biodelivery's Bioerodible Mucoadhesive drug delivery technology. The findings, presented in a poster session at Painweek 2015 in Las Vegas, showed that study participants receiving around-the-clock therapy with an opioid full agonist (morphine or oxycodone) could be switched to buprenorphine buccal film, at about half the full agonist dose, without increasing the risk of experiencing opioid withdrawal or a loss of pain relief. These findings also will be presented later this month at the 26th Annual Meeting of the American Academy of Pain Management in National Harbor, Md. The product is being developed under the name Belbuca and is under review by the FDA for use in patients with pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. The PDUFA action date is next month.
Essentialis Inc., of Carlsbad, Calif., dosed the first Prader-Willi syndrome patients in a six-month open-label extension to study PC025. Patients who complete the first two phases of study PC025 are being enrolled in the extension study, which will document the long-term therapeutic benefits of lead drug, DCCR, on hyperphagia; body fat; lean body mass; aggressive, threatening and destructive behaviors; and stamina. The company expects to report top-line data from the extension study in the first quarter of 2016.
Five Prime Therapeutics Inc., of South San Francisco, started patient dosing in the phase Ia/Ib trial evaluating the immunotherapy combination of FPA008, Five Prime's monoclonal antibody that inhibits colony stimulating factor-1 receptor, with Opdivo (nivolumab), New York-based Bristol-Myers Squibb Co.'s PD-1 immune checkpoint inhibitor, in six tumor types.
Flexion Therapeutics Inc., of Burlington, Mass., said top-line results from the first of two pivotal trials of lead candidate FX006 in patients with moderate to severe osteoarthritis (OA) knee pain showed that treatment with 40 mg of FX006, compared to placebo (saline), demonstrated statistical significance in average pain relief over weeks one through 12 and over weeks one through 24. At weekly time points, 40 mg of FX006 also demonstrated superiority to placebo in pain relief beginning at week one, continuing to week 11 and also at week 13 at each time point. However, the trial missed its primary endpoint, defined as superiority in pain relief at 12 weeks, which did not reach statistical significance. A pre-specified, commonly applied sensitivity analysis (Baseline Observation Carried Forward/Last Observation Carried Forward) that addresses patient dropouts, however, did demonstrate statistical significance for the primary endpoint at 12-weeks. Overall, the 40-mg dose of FX006 performed better than the 20-mg dose, and the frequency of treatment-related adverse events across the three groups (FX006 40 mg, FX006 20 mg and placebo) was comparable, with no drug-related serious adverse events observed. Shares of Flexion (NASDAQ:FLXN) fell $6.98, down 24.1 percent, to close Wednesday at $22.02.
Genkyotex SA, of Geneva, disclosed top-line data from its phase II program with GKT137831, its lead NOX1&4 inhibitor. In patients with diabetic kidney disease, GKT137831 demonstrated an excellent safety profile and statistically significant reduction in both liver enzyme and inflammatory marker levels, the company said. Treatment with GKT137831 for 12 weeks resulted in fewer adverse events than placebo, confirming its safety profile. However, a reduction in albuminuria, the primary efficacy endpoint of the study, was not achieved within this timeframe. The company said it will continue to explore the potential of GKT137831 in various fibrotic indications.
Genspera Inc., of San Antonio, reported interim data from its phase II study of mipsagargin (G-202) in adult patients with recurrent or progressive glioblastoma which demonstrate that mipsagargin confers clinical benefit to a subset of patients and that activity may correlate with the level of prostate-specific membrane antigen (PSMA) expression in the primary tumor. Mipsagargin is activated by the enzyme PSMA, which is highly expressed in some glioblastoma tumor vasculature.
Helsinn Group SA, of Lugano, Switzerland, said additional study results from the ROMANA phase III program presented at the World Conference on Lung Cancer in Denver showed that a significantly higher proportion of non-small-cell lung cancer (NSCLC) patients treated with anamorelin maintained or gained lean body mass in both studies compared to placebo (ROMANA 1: 58.1 percent vs. 36.9 percent; p<0.001; ROMANA 2: 51.5 percent vs. 26.5 percent; p<0.001). In an exploratory analysis of overall survival combining both studies, patients who were able to stabilize or increase lean body mass had a significant improvement in overall survival vs. patients who lost lean body mass, regardless of treatment, showing that maintaining or improving lean body mass enables advanced NSCLC patients with cachexia to survive longer. Anamorelin HCl is a selective, orally active ghrelin receptor agonist in development for the treatment of anorexia, cachexia and unintended weight loss in NSCLC.
Marinus Pharmaceuticals Inc., of Radnor, Pa., disclosed the successful completion of an end-of-phase II meeting with the FDA on ganaxolone for the adjunctive treatment of focal onset seizures in adults. Based on guidance, the company believes its plan will support registration. Marinus expects to finish its ongoing phase III trial and start another next year.
Mirati Therapeutics Inc., of San Diego, said it would present data at the International Association of Lung Cancer 16th World Conference on Lung Cancer in Denver on the first non-small-cell lung cancer (NSCLC) patient with AXL gene amplification enrolled in the MGCD265 phase Ib expansion cohort. Data will be presented showing the patient had a confirmed partial response (PR) based on RECIST criteria. The firm also made known a confirmed PR in an NSCLC patient with MET gene amplification who was enrolled in the MGCD265 expansion cohort.
Neovacs SA, of Paris, presented extended follow-up data from the phase I/IIa trial of interferon alpha (IFNa)-kinoid during Lupus 2015, the 11th International Congress on Systemic Lupus Erythematosus in Vienna. The results showed a lasting, polyclonal, neutralizing anti-IFNa response in lupus patients. A phase IIb trial is starting soon.
Oncomed Pharmaceuticals Inc., of Redwood City, Calif., reported new biomarker and updated clinical data for the company's phase II anti-Notch2/3 therapeutic candidate, tarextumab (OMP-59R5). These data show the potential of Notch3 overexpression as a prognostic factor in small-cell lung cancer and update OncoMed's phase Ib results for tarextumab in combination with standard-of-care chemotherapy for the first-line treatment of patients with extensive-stage disease. The data were presented in a mini-oral presentation at the 16th World Lung Conference on Lung Cancer in Denver.
Oramed Pharmaceuticals Inc., of Jerusalem, reported that 98 patients have already been enrolled in the company's phase IIb study of its oral insulin capsule, ORMD-0801, which represents more than half of the 180 patients expected to be recruited. The double-blind, randomized phase IIb study for type 2 diabetes is designed to generate ample data for both efficacy and safety endpoints for the oral delivery of insulin.
OSE Pharma SA, of Paris, reported results of survival and T-specific immune response in the patients with brain metastases treated with the company's T-specific immunotherapy presented during the World Conference on Lung Cancer, in Denver. Six patients with brain metastases were identified, out of the 64 patients treated with Tedopi (OSE2101). The heavily pretreated patients had a median survival of 13.75 months, with extremes ranging from seven months in a patient whose cancer was still progressing, to a survival exceeding 41 months.
Otonomy Inc., of San Diego, reported a successful end-of-phase II meeting with the FDA for OTO-104 in the treatment of Ménière's disease. The company expects to initiate two parallel phase III trials for OTO-104 in Ménière's patients, with the first to begin by the end of 2015 and the second to begin during the first quarter of 2016.
Pharmacyte Biotech Inc., of Silver Spring, Md., started a phase IIb trial designed to test the effectiveness of its pancreatic cancer treatment (the combination of microcapsules produced using the Cell-in-a-Box technology and low doses of the anticancer drug ifosfamide) in patients with this disease.
Pherecydes Pharma SA, of Romainville, France, said it launched the Phagoburn trial, a randomized and monitored phase I/II, single-blind trial, to evaluate the tolerance and effectiveness of two anti-infection bacteriophage treatments in serious burn patients. The effect of the bacteriophages is compared to reference treatment silver sulfadiazine. The international study is involving 220 patients across two arms – 110 patients for each of the bacteriophage cocktails. One targets bacterial infections caused by Escherichia coli, and the other targets infections caused by Pseudomonas aeruginosa. Phagoburn is part of a European Framework Programme 7 project. The study, launched in June 2013 for a period of three years, has received €3.85 million (US$4.3 million) in EU funding.
Protalix Biotherapeutics Inc., of Carmiel, Israel, reported positive phase I/II clinical trial data for the 1 mg/kg dose of PRX-102 for the treatment of Fabry disease. PRX-102 is a recombinant plant cell chemically modified version of the human alpha-Galactosidase-A enzyme. The efficacy results for the 1 mg/kg cohort appear more robust than those previously announced for the 0.2 mg cohort. The trial is treating up to 18 naïve male and female adult patients. Based on an analysis of kidney biopsies with randomized blinded scoring, PRX-102 demonstrated a reduction in renal peritubular capillary Gb3 of 86 percent using a quantitative Barisoni Lipid Inclusion Scoring System. PRX-102 was well tolerated, with the majority of adverse events being mild and moderate. Protalix expects to report full top-line results from all dosing cohorts in the fourth quarter of 2015.
Revance Therapeutics Inc., of Newark, Calif., said it started dosing patients with axillary hyperhidrosis (excessive underarm sweating) in a phase II trial of its RT001 (now referred to as RTT150 [botulinum toxin type A] topical gel) candidate. The randomized, double-blind, dose-ranging, placebo-controlled study is designed to evaluate the safety and efficacy of a single, bilateral application of RT001 topical gel for the treatment of primary axillary hyperhidrosis. About 60 adults will be enrolled. Revance expects to release interim data by year-end.
Seattle Genetics Inc., of Bothell, Wash., and partner, Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, said they completed patient enrollment in the phase III Alcanza clinical trial, which is evaluating Adcetris (brentuximab vedotin) vs. investigator's choice of methotrexate or bexarotene in 132 patients with CD30-expressing cutaneous T-cell lymphoma (CTCL) who received prior systemic therapy. Adcetris is an antibody-drug conjugate directed to CD30, which is expressed on skin lesions in at least 50 percent of patients with CTCL. The primary endpoint of the study is overall response rate lasting at least four months in patients with CD30-expressing MF or pcALCL. Key secondary endpoints are complete response, progression-free survival and burden of symptoms during treatment.
Spark Therapeutics Inc., of Philadelphia, said the database is locked for its phase III trial of lead program SPK-RPE65 for the treatment of RPE65-mediated inherited retinal dystrophies. The datasets have been transferred to the company's independent external statistical consultants, who have initiated the analysis of the data, and Spark anticipates providing top-line results in October.
TC Biopharm Ltd., of Edinburgh, Scotland, was granted Clinical Trial Authorisation by UK regulators to treat cancer patients with Immunicell, which uses patients' own cells grown in culture to target a wide variety of different cancers. The trial design allows patients with melanoma, kidney and lung cancer to be treated in a single study.
TG Therapeutics Inc., of New York, started a phase I/II study to investigate the use of TGR-1202, an oral PI3K delta inhibitor and TG-1101 (ublituximab), the company's glycoengineered anti-CD20 monoclonal antibody in combination with pembrolizumab the anti-PD-1 immune checkpoint inhibitor, in patients with relapsed or refractory chronic lymphocytic leukemia. This will be the first clinical trial evaluating the safety, tolerability and effectiveness of the triple combination of a PI3K delta inhibitor with an anti-CD20 mAb and an anti-PD-1 checkpoint inhibitor.
Vitae Pharmaceuticals Inc., of Fort Washington, Pa., reported positive top-line results from its phase I single ascending dose clinical study of VTP-43742 in autoimmune disorders. VTP-43742 is Vitae's first-in-class, wholly owned RORgammat inhibitor being developed for the treatment of a range of autoimmune disorders, potentially including psoriasis, psoriatic arthritis, rheumatoid arthritis, multiple sclerosis and irritable bowel disease, as well as numerous orphan diseases. In this study that evaluated single oral doses of VTP-43742 in 53 healthy human volunteers, VTP-43742 was safe and generally well tolerated at all dose levels across a 60-fold dose range.
Zafgen Inc., of Boston, completed enrollment of ZAF-203, a phase IIb clinical trial of beloranib in the treatment of patients with both severe obesity and type 2 diabetes. The trial enrolled 152 patients across 16 sites in Australia. The trial is designed to determine the long-term weight loss benefits of MetAP2 inhibitor treatment with beloranib in patients with severe obesity complicated by type 2 diabetes. The company remains on track to release six-month interim data in a subset of 95 patients in late 2015 or very early 2016.
Zealand Pharma A/S, of Copenhagen, reported results from a phase Ib trial with its glucagon analogue, ZP4207 for type I diabetes, which show good safety and tolerability after multiple dosing. In addition, it was shown that ZP4207 leads to clinically relevant increases in blood glucose levels. The trial objectives were to evaluate safety and tolerability and, secondarily, to assess pharmacokinetics and pharmacodynamics of ZP4207 after multiple dosing.