Amgen Inc., of Thousand Oaks, Calif., said the phase III TESLA (Trial Evaluating PCSK9 Antibody in Subjects with LDL Receptor Abnormalities) trial evaluating evolocumab met its primary endpoint of the percent reduction from baseline at week 12 in low-density lipoprotein cholesterol (LDL-C). The percent reduction in LDL-C, or “bad” cholesterol, was clinically meaningful and statistically significant. Evolocumab is a fully human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9, a protein that reduces the liver’s ability to remove LDL-C from the blood. Separately, the company disclosed findings from a pre-specified retrospective analysis of patients with metastatic melanoma that showed talimogene laherparepvec reduced the size of injected tumors and also non-injected tumors that had metastasized to other parts of the body. The analysis recorded tumor-level responses from a pivotal phase III study evaluating talimogene laherparepvec in patients with injectable unresected stage IIIB, IIIC or IV melanoma compared to granulocyte-macrophage colony-stimulating factor. Full results were presented during an oral session at the Society of Surgical Oncology Annual Cancer Symposium in Phoenix. Talimogene laherparepvec is an oncolytic immunotherapy designed to selectively replicate in tumor tissue and to initiate a systemic anti-tumor immune response. Of the 295 patients treated with talimogene laherparepvec, almost 4,000 tumor lesions were tracked for the analysis.

Bellicum Pharmaceuticals Inc., of Houston, said the first clinical study is under way of a Chimeric Antigen Receptor T Cell therapy that incorporates a safety switch to enable rapid elimination of the administered T cells if they threaten the life or health of the patient. Researchers from the National Cancer Institute have begun treating pediatric patients with osteosarcoma and other non-neuroblastoma. GD2-expressing solid tumors are being treated with a third generation CAR T-cell therapy that incorporates Bellicum’s Caspacide “safety switch” technology.

Can-Fite Biopharma Ltd., of Petach Tikva, Israel, said a phase II study protocol for the treatment of advanced liver cancer with its drug CF102 has been approved by an institutional review board in Israel. The company plans to conduct the study in Israel, Europe and the U.S., and it will include 78 subjects that will be dosed with the drug as a second-line treatment of advanced hepatocellular carcinoma with Child-Pugh class B cirrhosis. CF102 is a described as an orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor.

Edison Pharmaceuticals Inc., of Mountain View, Calif., said it started a phase II trial, titled “A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone) in Children with Pearson Syndrome,” designed as a subject-controlled study lasting 12 months. The primary endpoint is the incidence of episodes of sepsis, metabolic crisis and hepatic failure, while secondary endpoints include transfusion avoidance and other disease-relevant endocrine and neurological outcome assessments. Pearson syndrome, which is characterized by transfusion-dependent anemia, neutropenia and pancreatic dysfunction, is estimated to affect less than one out of 1 million and is typically diagnosed during infancy. EPI-743 is a member of the para-benzoquinone class and is believed to work by augmenting endogenous glutathione biosynthesis, which is essential for the control of oxidative stress.

GW Pharmaceuticals plc, of London, said it completed a phase I trial of GWP42006, which features nonpsychoactive cannabinoid cannabidivarin, in epilepsy, with data from the 66 healthy subject trial showing that the drug demonstrated no safety or toxicity signals and was well tolerated at even the highest dose. The company also reported that it had started a 12-week phase IIb study testing GWP42004, an oral product featuring plant-derived tetrahydrocannabivarin, with a primary objective of comparing change in glycemic control in patients with type II diabetes when treated with one of three doses of drug or placebo at add-on therapy to metformin. That trial is expected to enroll about 200 patients, with an estimated completion date in the second half of 2015. GW also has started a phase IIa schizophrenia trial testing GWP42003, which features purified CBD, with a primary objective of comparing the change in symptom severity in patients with schizophrenia or related psychotic disorder when given drug or placebo in addition to first-line antipsychotic therapy over a period of six weeks.

Medivir AB, of Stockholm, Sweden, reported new phase III data showing that once-daily protease inhibitor simeprevir achieved its primary endpoint in the ATTAIN study in treatment-experienced adults with chronic hepatitis C virus (HCV) and compensated liver disease, demonstrating noninferiority to Incivek (telaprevir, Vertex Pharmaceuticals Inc.) when both are given in combination with pegylated interferon plus ribavirin (PegIFN/RBV). Simeprevir demonstrated a superior safety profile, including fewer adverse events, fewer serious adverse events and less anemia vs. telaprevir. Those data were presented at the Conference of the Asian Pacific Association for the Study of the Liver in Brisbane, Australia. The company also presented pooled analysis of data from the phase III QUST-1 and QUEST-2 studies, confirming efficacy in treatment-naïve genotype 1b HCV patients, with 85 percent of patients achieved sustained viral response at week 12 when treated with simeprevir in combination with PegIFN/RBV compared to placebo in combination with PegIFN/RBV.

Neostem Inc., of New York, said the data safety monitoring board recommended continuing the PreSERVE AMI phase II trial of AMR-001, an autologous bone marrow-derived CXCR4-expressing cell, following a fourth interim data and safety review. The study is designed to enroll 160 patients to test AMR-001 vs. placebo for the preservation of heart function after a severe heart attack. Full enrollment has been completed, and data are expected in the second half of this year.

Neuralstem Inc., of Rockville, Md., reported final results from the phase I trial testing NSI-566 spinal cord stem cells in the treatment of amyotrophic lateral sclerosis, published in Annals of Neurology, which included data from the last six patients in the trial and the first to receive cervical stem cell transplants. Results showed that the stem cells can be safety transplanted in both the lumbar and cervical spinal cord segments, did not accelerate disease progression and warrant further study on dosing and therapeutic efficacy. Researchers also were able to identify potential therapeutic windows, suggesting that more injections, as well as multiple injections, are better and may increase both the length and the magnitude of the potential benefits.

Ocular Therapeutix Inc., of Bedford, Mass., said it started a phase III program to test its sustained-release dexamethasone, an ophthalmic corticosteroid, for the treatment of postoperative inflammation and pain following cataract surgery. The product is administered as a one-time bioabsorbable intracanalicular plug for release of the steroid to the ocular surface for up to 30 days. In the first of two studies, 240 patients undergoing clear corneal cataract surgery will be enrolled and randomized, after surgery, to either the dexamethasone treatment group or placebo vehicle plug. Primary endpoints are absence of anterior chamber cells at day 14 and reduction of pain at day eight. The second phase III trial is expected to launch later this year.

Repros Therapeutics Inc., of The Woodlands, Texas, said it received guidance from the FDA indicating that it may proceed to phase I and phase II studies of oral Proellex under two separate investigational new drug applications, for endometriosis and uterine fibroids, while remaining on partial clinical hold. The FDA guidance provides that the highest allowed dose will be 12 mg daily. Repros currently is conducting a phase II study of Proellex in severe endometriosis and plans to submit a phase II protocol for the treatment of symptomatic fibroids.

Resolve Therapeutics LLC, of Seattle, said the first doses to humans of its lead compound RSLV-132 have been administered. The targeted nuclease therapy is designed to decrease the burden of circulating RNA-containing immune complexes in systemic lupus erythematosus (SLE) patients. Ongoing studies are designed to evaluate the safety, tolerability, pharmacokinetics and biological activity of RSLV-132. A single escalating dose study in healthy volunteers began earlier in March, with a multidose study in SLE patients planned to begin in the second quarter.

Theracos Inc., of Marlborough, Mass., reported positive results of a multinational 96-week phase II study evaluating the efficacy and safety of the SGLT-2 inhibitor THR1442. The double-blinded, placebo-controlled study enrolled a total of 288 subjects with type II diabetes from sites in the US, Mexico and Colombia and evaluated the efficacy of THR1442 compared to placebo as a monotherapy for the treatment of diabetes. The primary efficacy endpoint was change in HbA1c at week 24. Treatment with THR1442 resulted in a statistically significant lowering of HbA1c, relative to placebo, of 0.79 percent, a difference that increased to 1.02 percent following 96 weeks of treatment.