Atyr Pharma Inc., of San Diego, reported the first administration of Resokine IV to healthy human volunteers in Europe. The company is developing Resokine IV as a potential therapeutic for rare disorders where a patient’s immune system is imbalanced. Resokine belongs to a new class of proteins called physiocrines, which were discovered by Atyr scientists.

Biothera Inc., of Eagan, Minn., reported positive data from its phase IIb trial of Imprime PGG, used as first-line treatment in late-stage non-small-cell lung cancer (NSCLC) in combination with cetuximab (Erbitux, Eli Lilly and Co.), carboplatin and paclitaxel. The combination therapy met its primary endpoint with a statistically significant improvement in objective response rate (ORR) – more than double for the Imprime PGG group compared to the control group Erbitux and the chemotherapies alone (48 percent vs. 23 percent, p = 0.0468). A subgroup of Imprime PGG biomarker-positive patients showed 67 percent improvement in ORR (p = 0.0088) and a 23-week improvement in median overall survival. The findings were reported at the International Association for the Study of Lung Cancer meeting on targeted therapies in Santa Monica, Calif.

Calithera Biosciences Inc., of South San Francisco, dosed the first patients in its first phase I study of CB-839 in advanced solid tumors. The potent, selective, orally bioavailable inhibitor of glutaminase interferes with tumor metabolism and blocks cancer cell growth and survival. Two additional phase I studies, one in patients with advanced multiple myeloma and non-Hodgkin’s lymphoma and another in patients with acute leukemias, are being conducted in parallel. The three trials are single-arm, open-label, dose escalation studies that allow for expansion in specific tumor types once maximum tolerated dose is reached. The studies primarily are seeking to determine the safety and tolerability of CB-839 and to establish a dose for phase II studies. Secondary endpoints include pharmacokinetics, pharmacodynamics and evidence of anti-tumor response. Predictive biomarkers are also being evaluated. The trials are being conducted at multiple U.S. sites. (See BioWorld Today, Oct. 30, 2013.)

Cellular Biomedicine Group Inc., of Palo Alto, Calif., reported an updated analysis of its phase I/IIa trial for human adipose-derived mesenchymal precursor cell (haMPC) therapy for knee osteoarthritis (KOA), which tests the safety and efficacy of intra-articular injections of autologous haMPCs in order to reduce inflammation and repair damaged joint cartilage. Completed in Q4 2013, the data show no serious adverse events, suggesting Rejoin cell therapy for KOA patients is safe. Three-month follow-up data reveal statistically significant improvement in KOA from the baseline in clinical scores for WOMAC, NRS-11, SF-36 and KSCRS knee osteoarthritis indices, showing significantly reduced knee pain, improved knee mobility and prolonged walking distance. Reduction of bone marrow lesions was observed in some patients. MRI examination revealed an increase in cartilage thickness as early as three months after the therapy. The trial is conducted by Shanghai’s Renji Hospital, a tertiary hospital affiliated with Shanghai Jiao Tong University School of Medicine.

Celsion Corp., of Lawrenceville, N.J., said the FDA reviewed and cleared the protocol for the company’s planned pivotal, double-blind, placebo-controlled phase III trial of Thermodox, its heat-activated liposomal encapsulation of doxorubicin in combination with radio frequency ablation (RFA), in hepatocellular carcinoma (HCC). The trial design is based on a comprehensive analysis of data from the company’s phase III HEAT study, which missed its primary endpoint but showed that treatment with Thermodox resulted in a 55 percent improvement in overall survival in HCC patients who received an optimized RFA treatment. Celsion expects to launch OPTIMA in the first half of 2014. The study is expected to enroll 550 patients across up to 100 sites in the U.S., Europe, China and Asia Pacific, studying Thermodox in conjunction with RFA, standardized to a minimum of 45 minutes across all investigators and sites to treat lesions measuring 3 cm to 7 cm, vs. standardized RFA alone. The primary endpoint is overall survival. The statistical plan calls for two interim efficacy analyses by an independent data monitoring committee. (See BioWorld Today, Feb. 1, 2013, and Sept. 17, 2013.)

Mast Therapeutics Inc., of San Diego, said 40 U.S. sites were opened by year-end 2013 for EPIC, its pivotal phase III study of MST-188 in sickle cell disease. The company expects additional clinical sites to open in at least three more countries during the first quarter, with approximately 30 ex-U.S. sites open by the end of the year. The company affirmed prior guidance that the trial is expected to be fully enrolled by the end of 2015. Mast also is amending the study’s entry criteria to expand the genotype and age range, based on physician response to the study and identification of otherwise eligible adult candidates. In parallel, Mast plans to add a limited number of adult centers to the 40 pediatric-focused sites that already are open to generate additional safety and efficacy data on MST-188 in adults. The decision was made following a determination by disease experts and the EPIC steering committee that expansion will not compromise the ability to enroll a sufficiently homogenous patient population and conduct a successful study. (See BioWorld Today, June 17, 2013.)

Palatin Technologies Inc., of Cranbury, N.J., presented new analyses from its phase IIb trial of bremelanotide, which demonstrated dose-dependent improvements in sexual desire and treatment satisfaction in premenopausal women with hypoactive sexual desire disorder (HSDD) and combined HSDD/female sexual arousal disorder, both which are forms of female sexual dysfunction. The data were presented at the International Society for the Study of Women’s Sexual Health Conference in San Diego. Palatin will likely enroll patients in phase III trials in the second half of 2014.

Synchroneuron Inc., of Waltham, Mass., said the first patient has been dosed in a phase II, multicenter, randomized, double-blind, placebo-controlled trial evaluating the efficacy, safety and pharmacokinetic behavior of orally administered SNC-102, a new formulation of acamprosate calcium for tardive dyskinesia (TD). Synchroneuron expects to enroll 90 patients across 12 clinical sites. The study will evaluate the clinical efficacy of SNC-102 tablets as measured by the Abnormal Involuntary Movement Scale, the standard rating scale for measuring severity of involuntary movements in TD. SNC-102 is a unique formulation of acamprosate calcium, an FDA-approved drug for treating alcohol dependence.