o Avigen Inc., of Alameda, Calif., and the Children's Hospital of Philadelphia filed an investigational new drug application with the FDA for a gene therapy approach to the treatment of hemophilia B, using Avigen's adeno-associated viral vector delivery system. Preparations for a Phase I trial are under way.
o Immunex Corp., of Seattle, and the Pediatric Rheumatology Collaborative Study Group, headquartered at Cincinnati Children's Hospital Medical Center, reported on a study that showed Enbrel is effective in treating children suffering from juvenile rheumatoid arthritis. Enbrel is a protein-based drug that inhibits the binding of tumor necrosis factor (TNF), a protein secreted by different types of cells that that regulate the immune response. It was recently approved for use in adults. (See BioWorld Today, Nov. 8, 1998, p. 1.)
o Ligand Pharmaceuticals Inc., of San Diego, completed the final closing of its strategic alliance with Elan Corp. plc, of Dublin, which was announced Sept. 29. In that agreement, Elan agreed to purchase $20 million of Ligand common stock and to purchase from Ligand up to $100 million in issue price of zero coupon, convertible senior notes due 2008 with an 8 percent per annum yield to maturity. Elan also licensed to Ligand in the U.S. and Canada its proprietary product, Morphelan, a once-daily sustained-release, solid oral dosage form of morphine for pain in oncology and HIV patients. (See BioWorld Today, Sept. 30, 1998, p. 1.)
o Shaman Pharmaceuticals Inc., of South San Francisco, proposed restructuring a convertible note financing originally completed in August 1997. The current balance on the notes is $4.7 million. The company said reclassification of this debt to equity is an important part of its efforts to maintain compliance with Nasdaq listing requirements.
o Sugen Inc., of South San Francisco, reported that Phase I interim data showed the angiogenesis inhibitor SU5416 appeared to be well-tolerated with minimal side effects. The data also indicated that SU5416 might have clinical activity in cancer, including colorectal, lung, renal cell and AIDS-related Kaposi's sarcoma. SU5416 is a small molecule inhibitor of the Flk-1/KDR signaling pathway and prevents the formation of new blood vessels required for tumor growth by blocking the Flk-1/KDR receptor, found in the endothelial cells of these blood vessels.
o Texas Biotechnology Corp., of Houston reported that TBC11251, the company's endothelin A receptor antagonist, reduced pulmonary vascular resistance and pulmonary artery pressure in patients with congestive heart failure. The Phase IIA data were presented at the American Heart Association's scientific session in Dallas. (See BioWorld Today, Feb. 9, 1998, p. 1.)
o Trega Biosciences Inc., of San Diego, reported that Novartis Pharma AG, of Basel, Switzerland, purchased $7 million of common stock as part of an option announced last May. Novartis received 1.8 million shares of Trega stock priced at $3.75 per share. The stock purchase is part of a transaction that included a research, development and license agreement to develop an orally active small molecule for the treatment of certain diseases mediated by the melanocortin-4 receptor pathway, including obesity, diabetes and Syndrome X. (See BioWorld Today, May 27, 1998, p. 1.)
o Vertex Pharmaceuticals Inc., of Cambridge, Mass., reported that double protease inhibitor therapy that includes Agenerase (amprenavir) results in a significant reduction in viral replication and is well tolerated through 48 weeks in HIV patients. The results of this small pilot study were presented at the 4th International Congress on Drug Therapy in HIV Infection, in Glasgow, Scotland. Agenerase is currently in Phase III development and is being co-developed and co-licensed with Glaxo Wellcome plc, of London.