Asterias Biotherapeutics Inc., of Fremont, Calif., said that including the sixth and final patient in the AIS-A 10 million cell cohort in the company's ongoing SCiStar phase I/IIa trial has further confirmed previously disclosed motor function improvements at six months following administration of AST-OPC1. The product is comprised of oligodendrocyte progenitor cells manufactured from our pluripotent embryonic stem cell platform. The AST-OPC1 stem cell lines were derived from an embryo that was originally meant for an in vitro fertilization procedure in the late 1990's, the company said.
Astrazeneca plc, of London, disclosed results of the first large real-world evidence study of its kind evaluating the risk of hospitalization for heart failure and death from any cause in patients with type 2 diabetes (T2D) receiving SGLT-2 inhibitors (SGLT-2i). The CVD-REAL study assessed data from more than 300,000 patients across six countries, 87 percent of whom did not have a history of cardiovascular disease. The data showed that across this broad population of patients with T2D compared to other T2D medicines, treatment with SGLT-2 inhibitor medicines Farxiga (dapagliflozin), canagliflozin (Invokana, Janssen Pharmaceuticals Inc.), and empagliflozin (Jardiance, Boehringer Ingelheim GmbH / Eli Lilly and Co.) reduced the rate of hospitalization for heart failure by 39 percent (HR 0.61; 95 percent CI 0.51-0.73; p<0.001) and death from any cause by 51 percent (HR 0.49; 95 percent CI 0.41-0.57; p<0.001). For the composite endpoint of hospitalization for heart failure and death from any cause, the reduction was 46 percent (HR 0.54; 95 percent CI 0.48-0.60; p<0.001).
Aveo Oncology Inc., of Cambridge, Mass., said the first patient has been dosed in the phase I/II Aveo-sponsored TiNivo trial evaluating tivozanib in combination with New York-based Bristol-Myers Squibb Co.'s anti-PD-1 therapy, Opdivo (nivolumab), in advanced renal cell carcinoma. Tivozanib is an oral, once-daily, vascular endothelial growth factor tyrosine kinase inhibitor.
Beyondspring Inc., of New York, said an abstract on the molecular action describing the immunological involvement in the anticancer effect of Beyondspring's lead asset, plinabulin, was accepted for poster presentation at this year's Keystone Symposia's "Cancer Immunology and Immunotherapy: Taking a Place in Mainstream Oncology" meeting in Whistler, British Columbia. Plinabulin is a small molecule that alters the tumor micro-environment and stimulates dendritic cell maturation and T-cell activation. The company is advancing it into a global phase III trial for non-small-cell lung cancer and a global phase II/III trial for neutropenia prevention. Specifically, the compound inhibits alpha and beta tubulin from polymerization, shown in a recently solved crystal structure, with a differentiated mechanism of action compared to other tubulin-targeting agents. The poster showed that guanine nucleotide exchange factor-H1 signaling is critical for induction of antitumor immunity induced by microtubule-depolymerizing drugs.
Galapagos NV, of Mechelen, Belgium, started a phase I study with potentiator GLPG3067 for cystic fibrosis. The company will receive a $7.5 million milestone payment from its collaboration partner Abbvie Inc., of North Chicago. The aim of the study is to evaluate the safety, tolerability and pharmacokinetics of oral single and multiple ascending doses of GLPG0367. (See BioWorld Today, Sept. 25, 2013.)
Hepatera LLC, of Moscow, completed the patient recruitment for phase IIb trials of Myrcludex B, described as a drug designed to inhibit the essential hepatitis B virus (HBV) receptor on the liver cell surface, preventing the infection of healthy cells and viral spreading in the liver. Specifically, the mechanism of antiviral action is the binding and inactivation of HBV receptors NTCP, which misdirects HBV to an unproductive pathway and thereby prevents an infection of the cell.
Intec Pharma Ltd., of Jerusalem, started a phase I trial of AP-CBD/THC, its Accordion Pill platform with the two primary cannabinoids contained in Cannabis sativa, cannabidiol and tetrahydrocannabinol, being developed for indications that include including low back pain and fibromyalgia.
Novartis AG, of Basel, Switzerland, disclosed results from the global phase III RELAX-AHF-2 study investigating the efficacy, safety and tolerability of RLX030 (serelaxin) in patients with acute heart failure. RELAX-AHF-2 did not meet its primary endpoints of reduction in cardiovascular death through day 180 or reduced worsening heart failure through day five when added to standard therapy, the company said. The drug is a recombinant form of human relaxin-2, a naturally occurring peptide.
Oncoceutics Inc., of Philadelphia, announced the publication of an article in Clinical Cancer Research that describes the phase I trial of the company's lead compound at the Rutgers Cancer Institute of New Jersey. The trial demonstrated that ONC201 is well-tolerated at plasma concentrations that meet or exceed the targeted therapeutic threshold and is biologically active in advanced cancer patients when orally administered at the recommended phase II dose (RP2D) of 625 mg every three weeks. ONC201 selectively binds to and antagonizes DRD2, a member of the dopamine receptor family and of the superfamily of G protein-coupled receptors (GPCRs) that is dysregulated in many cancers. GPCRs represent therapeutic targets that have largely been untapped in oncology despite evidence showing that selective inhibition of DRD2 has the potential to be a promising novel therapeutic concept in oncology. The completion of the first-in-human clinical trial enables phase II studies that are now underway for ONC201.
Origin Inc., of Princeton, N.J., said treatment has begun on the first patients in its U.S. dose-ranging GENESIS trial, a phase IIb-equivalent test of its technology to produce and deliver therapeutic quantities of plasma-generated nitric oxide for a wide range of potential benefits. The GENESIS trial is designed to demonstrate healing and optimize the treatment regimen for chronic diabetic foot ulcers.