• Ariad Pharmaceuticals Inc., of Cambridge, Mass., said it submitted a marketing authorization application for ponatinib, its BCR-ABL inhibitor, to the European Medicines Agency to treat adults with resistant or intolerant chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ALL). The Committee for Medicinal Products for Human Use granted accelerated assessment of the application. A new drug application was submitted to the FDA last month for ponatinib, which has orphan drug status in both the U.S. and Europe in CML and Ph+ALL. (See BioWorld Today, June 5, 2012.)

• Armgo Pharma Inc., of Tarrytown, N.Y., said a study published in Cell revealed the underlying role of calcium leak through the ryanodine receptor calcium-release channel on stress-induced cognitive dysfunction – the focus of the company's small-molecule drugs known as Rycals. The preclinical mouse study suggested that orally available and brain-penetrant Rycals drugs may have therapeutic utility in stress-induced cognitive dysfunction and a potentially broad variety of learning and memory disorders. Mice treated with the company's Rycal, S107, showed improvement in all tested measures, including learning and memory.

• Arrowhead Research Corp., of Pasadena, Calif., said its Calando Pharmaceuticals subsidiary completed enrollment of a Phase Ib trial of CALAA-01 , an RNAi therapeutic candidate targeting solid tumors. All patients participating in the trial have completed dosing, with the exception of one patient who is still receiving treatment and will continue to be monitored. An analysis of the final data is being prepared.

• Bayer HealthCare, of Wayne, N.J., and Onyx Pharmaceuticals Inc., of South San Francisco, disclosed that Bayer submitted a new drug application to the FDA for the oral multikinase inhibitor regorafenib in metastatic and/or unresectable gastrointestinal stromal tumors in patients whose disease has progressed despite prior treatment. (See BioWorld Today, Oct. 13, 2011.)

• CLC bio, of Aarhus, Denmark, reported that it will develop a user-friendly and computationally efficient solution for pathogen identification, host biomarker identification, pathogen variant characterization and molecular epidemiology in a clinical setting using part of an EU-funded clinical research grant. The Pathseek study has a total project budget of $10 million of which the company will receive $2 million. The objective of Pathseek is to demonstrate the potential of next-generation sequencing technologies in clinical microbiology labs, to enable the detection of pathogens directly from clinical samples and the early detection of drug-resistant mutations.

• Exelixis Inc., of South San Francisco, reported that its new drug application (NDA) for cabozantinib as a treatment for patients with progressive, unresectable, locally advanced, or metastatic medullary thyroid cancer has been removed from the FDA's Oncologic Drugs Advisory Committee (ODAC) meeting agenda scheduled for the Nov. 8 and Nov. 9. As a result, the company said it does not anticipate a discussion of the NDA by ODAC. The PDUFA date for cabozantinib is Nov. 29. Piper Jaffray analyst Edward A. Tenthoff said in a research note that the news is "a likely positive" and he continues to expect Cabo approval by the PDUFA date.

• Isis Pharmaceuticals Inc., of Carlsbad, Calif., said two papers published in Cell demonstrated that single-stranded short interfering RNA (ss-siRNA) molecules, distributed broadly, activated the RNA interference (RNAi) pathway and reduced expression of targeted genes in animal models. In one study, Isis scientists evaluated the absorption, distribution, metabolism and activity of the ss-siRNAs designed to utilize the RNAi pathway, showing that single-stranded RNA-like antisense oligonucleotides are broadly distributed to and active in multiple organs and effectively engage and trigger the RNAi pathway without the need for complex formulations or delivery vehicles. In the second study, researchers designed ss-siRNA molecules to mimic microRNA activity and selectively inhibit the production of a specific mutant, disease-causing HTT protein associated with the cause and progression of Huntington's disease. The data suggested ss-siRNAs engaged the RNAi pathway to produce selective silencing of the huntingtin gene in an animal model of Huntington's disease.

• Pluristem Therapeutics Inc., of Haifa, Israel, said that it is requesting orphan drug status from the FDA for PLacental eXpanded (PLX) cells to treat aplastic anemia. It is the company's second application with the FDA granting orphan status to its PLX cells for the treatment of Buerger's disease. (See BioWorld Today, Aug.31, 2011.)

• Soligenix Inc., of Princeton, N.J., reported results from a Phase Ib trial showing that an aluminum hydroxide adjuvanted formulation of RiVax was safe and well tolerated and induced greater ricin-neutralizing antibodies in humans than adjuvant-free RiVax. The study in healthy volunteers was designed to evaluate the long-term safety and immunogenicity of escalating doses of the vaccine up to one year after a primary vaccination. Results were published in Clinical and Vaccine Immunology.

• Sucampo Pharmaceuticals Inc., of Bethesda, Md., said that majority shareholder S&R Technology Holdings LLC (S&R) has converted all of its shares of Sucampo's class B common stock into shares of its class A common stock. S&R held all 26,191,050 issued and outstanding shares of class B common stock. Class B common stock holders were entitled to 10 votes per share while class A common stock holders were entitled to one vote per share.

• Teva Pharmaceutical Industries Ltd., of Jerusalem, said the FDA approved tbo-filgrastim (XM02 filgrastim), a short-acting recombinant form of granulocyte colony-stimulating factor (G-CSF), to reduce the duration of severe neutropenia in patients with non-myeloid malignancies receiving chemotherapy that affects the bone marrow. Teva currently markets filgrastim in Europe under the trade name Tevagrastim, a biosimilar to Neupogen. Tbo-filgrastim was filed in the U.S. as a biologics license application since a biosimilar approval pathway had not been established at the time of submission. Teva will market tbo-filgrastim as early as November 2013, in accordance with a settlement reached with Amgen Inc., of Thousand Oaks, Calif. Separately, Teva UK Ltd. launched Irbesartan, a generic version of Aprovel (Irbesartan, Sanofi, Bristol-Myers Squibb), in the treatments of essential hypertension and renal disease in patients with hypertension and Type II diabetes.