Pharmaceuticals Inc. (Carlsbad, Calif.) ISIS-STAT3Rx An antisense drug Advanced metastatic hepatocellular carcinoma Started a Phase Ib/IIa study 5/7/13 Jennerex Biotherapeutics Inc. (San Francisco) Pexa-Vec JX-594, pexastimogene devacirepvec Solid tumors Phase I data demonstrated the ability of it to induce functional anticancer immunity 5/16/13 Mirna Therapeutics Inc. (Austin, Texas) MRX34 miRNA mimic of tumor suppressor miR-34 delivered using a liposomal delivery formulation Cancer Started a

Company (Location) Product Description Indication Status Date AUTOIMMUNE Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.) and Sanofi SA (Paris) Sarilumab Fully human monoclonal antibody directed against the alpha subunit of the IL-6 receptor complex Moderate to severe rheumatoid arthritis Enrolled their first patients in Phase III trials 5/16/13 Xenoport Inc. (Santa Clara, Calif.) Arbaclofen placarbil A prodrug of the R-isomer of baclofen Spasticity due to multiple sclerosis Terminated the

Company (Location) Product Description Indication Status Date AUTOIMMUNE GW Pharmaceuticals plc (Porton Down, UK) Sativex Cannabinoid product Moderate to severe spasticity in multiple sclerosis Received commercial approval for Sativex in Italy 5/8/13 CANCER Aveo Pharmaceuticals Inc. (Cambridge, Mass.) and Astellas Pharma Inc. (Tokyo) Tivozanib A once-daily, VEGF receptor tyrosine kinase inhibitor Advanced renal cell carcinoma Astellas no longer intends to submit an MAA and will not fund any

an oncolytic immunotherapy Hepatocellular carcinoma FDA granted orphan drug status 5/10/13 Seattle Genetics Inc. (Bothell, Wash.) Adcetris Brentuximab vedotin Relapsed Hodgkin lymphoma and systemic anaplastic large-cell lymphoma FDA accepted for filing its sBLA 5/15/13 CARDIOVASCULAR Biogen Idec Inc. (Weston, Mass.) Eloctate Recombinant Factor VIII Fc fusion protein Hemophilia A FDA accepted the firm's BLA 5/14/13 CENTRAL NERVOUS SYSTEM Titan Pharmaceuticals Inc. (South San Francisco) Probuphine

This was a difficult conclusion for us because it will come as a tremendous disappointment to patients and their families, as well as for the larger RCC community, including our investigators and nurses." – Tuan Ha-Ngoc, president and CEO, Aveo Pharmaceuticals Inc., on discontinuing further development of lead product tivozanib in renal cell carcinoma

sites 5/23/13 Oncogenex Pharmaceuticals Inc. (Bothell, Wash.) OGX-427 A heat-shock protein 27 Advanced or metastatic bladder cancer Started the Phase II Borealis-2 trial 5/1/13 Oncogenex Pharmaceuticals Inc. (Bothell, Wash.) OGX-427 Heat-shock protein 27 Advanced squamous cell lung cancer Began a Phase II trial 5/24/13 Oncolytics Biotech Inc. (Calgary, Alberta) Reolysin Reovirus variant for Ras-mutant cancers Metastatic melanoma Met the primary endpoint in the first stage of a Phase II trial

Sunesis Pharmaceuticals Inc., of South San Francisco, said the independent data and safety monitoring board for the pivotal VALOR trial completed its latest period safety review and recommended that the study continue as planned without changes

It's much more important to show me how you can compete and how much of the market you can penetrate," Neil Berkley, director of business development at San Diego-based Cadence Pharmaceuticals Inc., told the audience

Ohr Pharmaceutical Inc., of New York, said it received confirmation that its application to list on Nasdaq was approved

Biota Pharmaceuticals Inc., of Alpharetta, Ga., began dosing in a Phase II trial of laninamir octanoate, for influenza A or B infection...Ritter Pharmaceuticals Inc., of Los Angeles, said Phase II data with RP-G28 in patients with lactose intolerance demonstrated the agent's ability to alter the composition of microbes in patients' intestines

Anacor Pharmaceuticals Inc., of Palo Alto, Calif., said it entered a loan agreement with Hercules Technology Growth Capital Inc

A handful of development-only biotech firms have popped up in recent years, such as Furiex Pharmaceuticals Inc., which in-licenses early compounds, sees them through proof-of-concept development before licensing them back out for late-stage development and commercialization...Discovered in an Emory lab, the nucleoside reverse transcriptase inhibitor was licensed in 1996 by Durham, N.C.-based Triangle Pharmaceuticals Inc. – later acquired by Gilead Sciences Inc. – and has since gone on to be a

In 2011 the company out-licensed PEP02 to Merrimack Pharmaceuticals Inc., of Cambridge, Mass., for an up-front fee of $10 million for commercialization rights of the drug renamed MM-398 in Europe and Asia, excluding Taiwan, with another $210 million due on reaching development, regulatory and sales milestones, and for royalties...OBI Pharma was formed in Taiwan in 2002 initially as a subsidiary of U.S.-based Optimer Pharmaceuticals Inc., but operating since 2012 as an independent entity

Meanwhile, Sound Pharmaceuticals Inc., of Seattle, has SPI-1005, which is designed to protect the inner ear from sound-associated damage, in clinical development

The potential of UPS as a source of drug targets is underlined by two marketed proteasome inhibitors, the blockbuster Velcade (bortezomib, Millennium: The Takeda Oncology Co.) and Kyprolis (carfilzomib, Onyx Pharmaceuticals Inc.), both treatments for multiple myeloma

Questcor Pharmaceuticals Inc., of Anaheim, Calif., acquired rights to Synacthen and Synacthen Depot for the U.S...Vertical Pharmaceuticals Inc., of Sayreville, N.J., signed a co-promotion agreement with Nautilus Neurosciences Inc., of Bedminster, N.J., for Lorzone (chlorzoxazone), a muscle relaxant approved for musculoskeletal pain

Agios Pharmaceuticals Inc., of Cambridge, Mass., filed for an initial public offering (IPO) to raise up to $86 million to fund development of its pipeline in cancer and inborn errors of metabolism (IEMs), a subset of orphan genetic metabolic diseases

last year. (See BioWorld Today, July 20, 2011, and Sept. 5, 2012.) Antibiotic developer Paratek Pharmaceuticals Inc., a potential competitor with tetracycline-derived compound PTK-AR01 under investigation in acne and rosacea, also filed and priced but has not yet closed an initial public offering. (See BioWorld Today, Oct. 1, 2012, and March 21, 2013.) And new entrants pursuing acne continue to come

But biosimilars offer no improvement, so the only incentive for trial participation might be getting the drug for a little less, Craig Wheeler, president and CEO of Momenta Pharmaceuticals Inc., told BioWorld Today