Industry Wire

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(Compiled and edited by Sadik H. Kassim)

Neurologix, Inc. (Fort Lee, NJ), a biotechnology company engaged in the development of gene therapies for disorders of the brain and central nervous system, announced its financial results for the 6 months ended June 30, 2011. The company reported a net loss of approximately $5.0 million, as compared with a net loss of approximately $8.0 million for the 6 months ended June 30, 2010. The company had cash and cash equivalents of approximately $3.5 million as of June 30, 2011. Neurologix recently presented promising results to the NIH's Recombinant DNA Advisory Committee (RAC) for their novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD). The company plans to submit a Phase 3 protocol to the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment later in 2011.

GenVec, Inc. (Gaithersburg, MD), announced its financial results for the 6 months ended June 30, 2011. The company's net loss for this period was $3.5 million ($0.27 per share) compared with a net loss of $8.9 million ($0.72 per share) for the 6 months ended June 30, 2010. Revenue for the 6-month period ending June 30, 2011 was $10.0 million compared with the revenue of $6.1 million in the comparable prior year period. GenVec ended the second quarter of 2011 with $30.6 million in cash, cash equivalents, and short-term investments. GenVec recently announced that it has entered into a collaborative agreement with Merial, the animal health division of Sanofi-Aventis to develop and commercialize GenVec's proprietary vaccine technology for use against foot-and-mouth disease and other areas of animal health.

Genzyme (Cambridge, MA) and Isis Pharmaceuticals, Inc. (Carlsbad, CA), announced that Genzyme has submitted a marketing authorization application (MAA) to the European Medicines Agency seeking approval for the 200-mg weekly dose of mipomersen for the treatment of homozygous and severe heterozygous familial hypercholesterolemia. Mipomersen is a first-in-class antisense therapeutic that inhibits apolipoprotein-B (apoB) synthesis and is currently in late-stage development for the reduction of low-density lipoprotein (LDL) cholesterol.

VIRxSYS Corporation (Gaithersburg, MD) presented data indicating that a simian version of its lentiviral vector-based HIV vaccine, VRX1273, achieved a functional cure in a subset of rhesus macaques that had been challenged with a high dose of simian immunodeficiency virus (SIV). Eighteen months after the SIVmac251 infection, VIRxSYS researchers report that some of the vaccinated animals displayed viral loads below levels of detection in blood plasma, blood cells, and cells residing in reservoir tissues. The data were presented at the 6th International AIDS Society Conference on HIV Pathogenesis, Treatment, and Prevention.

Jennerex, Inc. (San Francisco, CA), announced positive results from a proof-of-concept clinical trial using JX-594 to treat research subjects with metastatic melanoma. JX-594 is an engineered oncolytic poxvirus that is designed to selectively target and destroy cancer cells. The mechanistic proof-of-concept study enrolled 10 treatment-refractory subjects with stage IV melanoma, 8 of whom completed the study. All subjects received up to nine weekly intratumoral injections of JX-594 at a low dose. JX-594 activity was assessed in blood samples and tumor biopsies. Blood samples collected over the course of treatment demonstrated JX-594 replication after repeated dosing as well as the expression and functionality of JX-594-encoded transgenes. Tumor biopsies showed tumor cell necrosis (death) and immune cell recruitment into tumors. JX-594 was safe and well tolerated, with flulike symptoms as the most common side effect for research subjects.

Transgene, Inc. (Strasbourg, France), announced detailed results of its phase I open-label, dose-escalating study performed in France with its therapeutic vaccine TG4040 (MVA-HCV) in research subjects infected with chronic hepatitis C (HCV). TG4040 is a recombinant vector-based on the modified vaccinia Ankara (MVA) virus carrying and expressing three of the major nonstructural proteins (NS3, NS4, and NS5B) of HCV. Phase I clinical results in 39 treatment-naive genotype 1 HCV subjects showed that the product is safe and well tolerated at all dose levels tested. Immunological analyses of 15 treatment-naive subjects support the expected mechanism of action of TG4040, which aims at inducing an effective HCV-specific T cell-based immune response, able to control viral replication.

Alnylam Pharmaceuticals, Inc. (Cambridge, MA), announced that it has completed its phase I study with ALN-VSP, a systemically delivered RNA interference (RNAi) therapeutic for the treatment of advanced solid tumors with liver involvement. ALN-VSP is a systemically delivered RNAi therapeutic comprising two small interfering RNAs (siRNAs) designed to target vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP). In the phase I study with ALN-VSP, 41 research subjects were enrolled and treated at doses ranging from 0.1 to 1.5 mg/kg. A total of 209 doses were administered, with a range of 1 to 28 doses per patient. Disease control (stable disease or better) was observed in 13 of 31 evaluable patients (42%) treated at doses of 0.4 to 1.5 mg/kg. The average duration of disease control is approximately 5 months, with a range of 2–14 months. ALN-VSP is Alnylam's first systemic RNAi program and represents the company's first clinical program in oncology. The company expects to partner its ALN-VSP program before initiating a phase II clinical study.

Alnylam also reported its consolidated financial results for the second quarter of 2011. As of June 30, 2011, Alnylam had cash, cash equivalents, and total marketable securities of $316.0 million, as compared with $349.9 million at December 31, 2010. The company's net loss for the first half of this year was $13.8 million as compared with a net loss of $14.6 million for the same period in the previous year. Revenues were $20.6 million for the second quarter of 2011, as compared with $26.6 million for the same period last year.

AMT (Amsterdam, The Netherlands) reported its financial results for the first half of 2011. Total net loss for the period ended June 30, 2011 amounted to € 8.7 million, a reduction of 7% compared with the net loss for the period ended June 30, 2010, which amounted to € 9.4 million. Cash and cash equivalents amounted to € 9.1 million on June 30, 2010, a decrease of € 8.8 million compared with € 17.9 million on December 31, 2010. The decrease in cash and cash equivalents stems mainly from the operational cash outflow, which amounted to € 8.8 million for the period ended June 30, 2011 (compared with an operating cash outflow of € 8.9 million for the period ended June 30, 2010). In June 2011, the European Medical Agency's Committee for Medicinal Products for Human Use (CHMP) rejected AMT's Marketing Authorisation Application (MAA) for Glybera, an AAV based gene therapy for the treatment of Lipoprotein Lipase Deficiency (LPLD). AMT has subsequently generated additional data, including results showing that Glybera significantly reduces the risk of pancreatitis in LPLD patients. AMT has filed for re-examination of the Glybera MAA. This process will be completed by the end of 2011.