• AVI BioPharma Inc., of Portland, Ore., said the FDA has granted orphan drug designation to AVI-4658 for the treatment of Duchenne muscular dystrophy (DMD). AVI-4658 uses AVI's ESPRIT technology (Exon Skipping Pre-RNA Interference Technology). By "skipping" exon 51, the proper RNA reading frame can be restored in suitable patients, resulting in the production of functional dystrophin. AVI is planning clinical development of AVI-4658 including a dose-ranging trial using systemic administration of the drug. The trial will be conducted in conjunction with the company's DMD cross-licensing and development partner, Ercole Biotech Inc., of Research Triangle Park, N.C.

• Cytori Therapeutics Inc., of San Diego, said it has received European approval for the second-generation Celution stem- and regenerative-cell processing system. The Celution system is the only device approved in Europe to extract and concentrate a patient's own stem and regenerative cells from within fat tissue, or adipose, in order to be delivered back to the same patient in the same procedure, the firm said. Adipose is a rich, accessible and non-embryonic source of stem and regenerative cells. The Celution system was evaluated in an 11-patient investigator-initiated safety and feasibility study conducted in Japan. The study investigated adipose-derived stem and regenerative cells extracted and processed with the Celution system to treat the complications of surgery and radiation damage following partial mastectomy for the treatment of breast cancer. Based on preliminary results, Cytori is planning two clinical studies to further evaluate the use of adipose-derived stem and regenerative cells in breast reconstruction following partial mastectomy for the purpose of offering optimal reimbursement in Europe.

• Discovery Laboratories Inc., of Warrington, Pa., submitted its formal response to the FDA's April 2006 approvable letter for Surfaxin for the prevention of respiratory distress syndrome in premature infants. Within 14 days of the submission, the agency will report whether it accepts the response and will determine a review classification. Discovery Labs said it anticipates a Class 2 classification, which would allow for a six-month review period, with a target PDUFA date in the second quarter of 2008. The approvable letter did not require any additional clinical trials, though it requested additional information involving drug product specifications and stability, analytical methods and related controls. Surfaxin is an engineered version of natural human lung surfactant containing Discovery Labs' KL-4 peptide. (See BioWorld Today, April 6, 2006.)

• Enanta Pharmaceuticals Inc., of Watertown, Mass., reported that its hepatitis C protease inhibitors demonstrated antiviral activity in the cell-based replicon assay and had favorable preclinical pharmacokinetic properties with the potential for once-daily dosing in humans. Data were presented at the hepatitis C resistance workshop in Boston. Enanta partnered with Abbott Park, Ill.-based Abbott in 2006 to develop and commercialize HCV protease inhibitors, with the HCV NS 3/4A serine protease at the focus of the collaboration.

• Generex Biotechnology Corp., of Worcester, Mass., received approval for its Oral-lyn oral insulin spray, a non-injectable buccal insulin, in India. In connection with that approval, the company signed a product licensing and distribution agreement with Shreya Life Sciences Pvt. Ltd., of Mumbai, India, and Shreya is preparing for commercial launch. Specific financial terms of the deal were not disclosed.

• ImmunoGen Inc., of Cambridge, Mass., signed Princeton, N.J.-based Laureate Pharma Inc. as its contract manufacturer for the huC242 antibody used in the production of the company's huC242-DMA tumor-activated prodrug compound, which is in Phase II testing. Terms of the agreement were not disclosed.

• Indevus Pharmaceuticals Inc., of Lexington, Mass., said that the FDA has accepted for review the firm's new drug application (NDA) for Nebido, a long-acting injectable testosterone preparation for the treatment of male hypogonadism, a condition characterized by a reduced or absent secretion of testosterone from the testes. The Prescription Drug User Fee Act target action date for Nebido is June 27, 2008. The NDA, which was submitted to the FDA on Aug. 28, contains data from six clinical studies in which more than 400 patients received at least one dose of Nebido, with more than 300 of those patients treated for more than one year. More than 3,000 injections of Nebido have been given to hypogonadal males during the clinical development program, the company said. The NDA also includes postmarketing data from safety reporting through June 2007.

• King Pharmaceuticals Inc., of Bristol, Tenn., said it filed a Citizen Petition with the FDA that raises specific concerns related to a biologics license application (BLA) filed by ZymoGenetics Inc., of Seattle, for a recombinant topical thrombin product currently under review by the FDA. The petition asserts that ZymoGenetics is pursuing an abbreviated pathway to approval of its recombinant thrombin in violation of the law regarding BLA approvals. King contends that ZymoGenetics' clinical program has resulted in only a limited number of patients exposed to its product and believes that the Seattle biotech is relying on data from nonrecombinant thrombin products, including Kings bovine thrombin product Thrombin-Jmi (thrombin, topical, bovine), to demonstrate safety, purity and potency, contrary to law. King is asking the FDA to adhere to its long-held scientific and legal requirements that, with limited exceptions, BLA approvals be based on at least two adequate and well-controlled clinical studies and that those studies each feature clinically meaningful efficacy endpoints.

• Pozen Inc., of Chapel Hill, N.C., said the FDA accepted for review its amended response to the Aug. 1 approvable letter for Trexima, a combination of sumatriptan and naproxen sodium. The agency also notified Pozen that response warrants a six-month review, which means a decision on the migraine drug could come around April 15, 2008. The approvable letter raised concerns regarding a preclinical genotoxicity study that the company said it could address with a small, single-dose trial. (See BioWorld Today, Aug. 3, 2007.)

• Progenics Pharmaceuticals Inc., of Tarrytown, N.Y., said it has been awarded grants totaling $1.9 million from the National Institutes of Health. The proceeds, to be disbursed over two years, were awarded to support the development and initiation of clinical testing of a novel antibody-drug conjugate and vaccine immunotherapies that target prostate-specific membrane antigen (PSMA), a molecular marker found on prostate cancer cells. The firm was awarded $940,000 to support preclinical activities and clinical testing of a PSMA antibody-drug conjugate, a novel immunotherapeutic agent that combines the molecular targeting of a fully human monoclonal antibody that is specific for PSMA with the cell-eradicating properties of a highly potent cytotoxic drug. The company received a $970,000 grant for translational research and initial human testing of PSMA-VRP, a vaccine designed to enable a patient's own immune system to recognize and eliminate prostate cancer cells. PSMA-VRP is a viral vector vaccine that utilizes alphavirus technology under license from AlphaVax Inc., of Research Triangle Park, N.C. PSMA-VRP is designed to infect key antigen-presenting cells in the body.

• ProMetic Life Sciences Inc., of Montreal, said new preclinical results indicated that a once-a-day oral administration of PBI-1402, the firm's lead compound for anemia, increases circulating red-blood cells and hemoglobin level comparable with normal range values. PBI-1402 was tested in the 5/6 nephrectomized rat model, which simulates chronic renal failure in humans resulting in loss of kidney functions and anemia subsequent to a reduced level of erythropoietin normally produced by the kidneys. ProMetic recently expanded its clinical program for PBI-1402 into the treatment of anemia in patients with myelodysplastic syndrome. The firm said clinical trials in patients with anemia associated with chronic kidney diseases also are scheduled to commence shortly.

• Science Applications International Corp., of San Diego, was awarded a contract extension by the National Institute of Allergy and Infectious Diseases' Division of Microbiology and Infectious Diseases to provide malaria vaccine production and support services. The seven-year, cost-plus-fixed-fee deal has an estimated value of $32.7 million, and the contract extension represents and follow-on effort for previous work SAIC has been performing for NIAID since 2000.