Amgen Inc., of Thousand Oaks, Calif., said the FDA granted fast-track designation for two of the company's experimental therapies, AMG 531 and AMG 706. AMG 531, which received orphan drug designation in 2003, is Amgen's first peptibody and represents a new approach to treat immune thrombocytopenic purpura, an autoimmune bleeding disorder. Amgen said AMG 706, its first oral cancer therapy, might hold promise for certain tumor types and is in Phase II trials for the treatment of imatinib-resistant gastrointestinal stromal tumors.
ArQule Inc., of Woburn, Mass., enrolled the first patient in a Phase Ib combination trial of ARQ 501 and Taxotere. It is an open-label, dose-escalation study that is being conducted at a single site in the U.S., enrolling patients with advanced cancer, some of whom previously received Taxotere. ARQ is ArQule's most-advanced compound developed using its Activated Checkpoint Therapy.
BrainStorm Cell Therapeutics Ltd., of Tel Aviv, Israel, leased space in Kiryat Ha'Mada Ve Ha'Technologia in Petach Tikva. The space will be used as an R&D facility, and for corporate offices. Brainstorm is focused on developing NurOwn cell-therapy products derived from adult stem cells that might treat neurodegenerative diseases.
Cellerant Therapeutics Inc., of Palo Alto, Calif., said data demonstrate how myeloid progenitor (MP) cells improve survival after opportunistic infections in a murine model of chemotherapy-induced neutropenia. The data were published online in Blood. The data add to previously published work showing the protective effects of those cells in a murine model of neutropenia following lethal irradiation. In the experiments, mice developed neutropenia after a dose of 5-fluorouracil and were then exposed to Aspergillus fumigatus. Only 33 percent of control animals survived, compared with 54 percent of animals treated with MP cells.
Cryptome Pharmaceuticals Ltd., of Melbourne, Australia, signed a memorandum of understanding with the University of Queensland's Institute for Molecular Biosciences to collaborate on the identification of potential therapeutics for cardiovascular diseases. The IMB will provide Cryptome with compound libraries, which Cryptome will screen for activity using its Cryptomics platform technology. That platform is based on finding small protein fragments with previously undiscovered therapeutic activities.
CV Therapeutics Inc., of Palo Alto, Calif., and Solvay Pharmaceuticals Inc., of Marietta, Ga., entered a co-promotion agreement for Aceon (perindopril erbumine) tablets, an angiotensin-converting enzyme inhibitor with tissue activity approved in the U.S. in hypertension. The agreement covers the U.S. and extends through at least 2010. CV will be responsible for marketing activities and establishing a cardiology-focused sales force to promote the product. Solvay will continue to handle manufacturing and distribution, and its primary-care sales force also will continue to promote the product. Solvay will book all sales and CV will receive a royalty on all sales above a pre-specified baseline. There are no up-front payments by either party.
Cytos Biotechnology AG, of Zurich, Switzerland, initiated a combined Phase I/II trial with the immunodrug candidate CYT006-AngQb, a therapeutic vaccine for hypertension. The study will include 16 normotensive and 72 hypertensive participants with mild to moderate hypertension. Efficacy will be determined by measuring systolic and diastolic blood pressure. First results are expected in the second half of 2006.
Discovery Laboratories Inc., of Warrington, Pa., said it saw encouraging preliminary data in its Surfaxin Phase II trial for acute respiratory distress syndrome in adults. As of Nov. 15, a total of 89 patients were enrolled and completed the 28-day study. The 78 evaluable patients received either one of two dosing regimens or standard care. Both Surfaxin dose groups showed a 31 percent relative improvement in overall mortality assessed at day 28 vs. standard care. With respect to the primary endpoint of the incidence rate of being alive and off mechanical ventilation at day 28, 69 percent of dose group A patients, 82.8 percent of dose group B patients and 75 percent of the standard-care patients were alive and off mechanical ventilation. Based on those data and in consultation with clinical advisers, the Phase II protocol has been modified to establish the endpoint signal in clinical outcomes in order to design a potential Phase III trial. The modified protocol allows for increased enrollment of up to 160 patients. The remainder of the trial will be comprised of Surfaxin dose group B (lavage with bolus) and standard care. The trial is expected to be completed by the fourth quarter. Also, the company said its deal with Barcelona, Spain-based Laboratorios del Dr. Esteve SA has been restructured. Discovery regained full commercialization rights in certain European markets, Central America and South America for its Surfactant replacement therapies, including Surfaxin for respiratory distress syndrome in premature infants and acute respiratory distress syndrome in adults. Discovery's stock (NASDAQ:DSCO) rose $1.40 Tuesday, or 20.5 percent, to close at $8.22.
DOR BioPharma Inc., of Miami, received notice from the American Stock Exchange that after review of the company's Form 10-Q for the quarter ended Sept. 30, the company was not in compliance with an AMEX continued listing standard. The notice requires DOR to submit a plan by Dec. 30, advising AMEX of the action it has taken, or will take, to bring the company into compliance with the continued listing standards by May 30. DOR is focused on the development of biodefense vaccines and therapeutics.
Endovasc Inc., of Montgomery, Texas, said that with the recent completion of the Phase II trial of Liprostin, a liposome-encapsulated form of prostaglandin E1, it is working toward initiating a Phase III trial in 2005. The company is finalizing the documents to be submitted to the FDA, as well as designing a Phase III protocol. Also, Endovasc has retained Synergos Inc., a Houston-based contract research organization, to assist in the preparation of those documents.
ID Biomedical Corp., of Vancouver, British Columbia, said it will ship an additional 1.2 million doses of its influenza vaccine, Fluviral, to the Canadian market due to pressure for supply within the Canadian private market system. Those are the remaining doses of vaccine available from ID Biomedical for the 2004-05 flu season, it said.
Illumina Inc., of San Diego, said it was successful in reducing the amount of punitive damages awarded in a wrongful termination lawsuit. In a Dec. 3 opinion, the Fourth Appellate District Court of Appeal in San Diego found the original punitive damage award to be "grossly excessive" and reduced it to $2.2 million. Illumina expects to record a one-time gain of about $3.3 million and make a $5.9 million cash payment. Illumina develops tools for large-scale analysis of genetic variation and function.
Imcor Pharmaceutical Co., of San Diego, began a Phase II trial designed to investigate alternative methods of administration for Imagent (perflexane lipid microsphere), including repeat dosing and infusion. The study is part of a series of clinical trials to establish the safety and efficacy of Imagent when administered at higher doses. It will provide data for Imagent's use in new indications, such as myocardial perfusion imaging and radiology applications.
Invitrogen Corp., of Carlsbad, Calif., and the Mayo Clinic in Rochester, Minn., entered a collaboration to discover and develop high-content biomarkers in cancer and general laboratory medicine. Under the collaboration and associated licensing agreement, Invitrogen will bring its portfolio of advanced research technologies to the effort and the clinic will contribute clinical resources and expertise. Invitrogen will provide financial and research support for multiple biomarker discovery programs at the clinic, and will have an option to license and develop resulting technology on an exclusive and nonexclusive basis. Other terms were not disclosed.
MediGene AG, of Martinsried, Germany, completed its pivotal Phase III trial of Polyphenon E ointment to treat anogenital and perianal warts. The company plans to submit a new drug application in the middle of 2005. The randomized and double-blinded trial enrolled more than 500 patients and demonstrated significant efficacy of the ointment compared to placebo. It also showed good tolerability. The trial resulted in the clearance of baseline warts and new warts in about 57 percent of patients treated.
Nabi Biopharmaceuticals, of Boca Raton, Fla., filed a shelf registration with the SEC that will allow the company to offer and sell up to $175 million of equity or debt securities. Nabi completed an equity offering in December 2003 that provided it with enough money to complete the development and launch of its lead candidate StaphVax. The company intends to use net proceeds from an offering conducted under the shelf registration for general corporate purposes, including clinical trials, research, development and marketing expenses, as well as new acquisition and licensing costs.
Nanobac Life Sciences Inc., of Tampa, Fla., said Histopathology published a study on the presence of nanobacteria in psammoma bodies of ovarian cancer. Nanobac's nanobacteria-specific 8D10 antibody and nanobacterial culture technique were used in the experiments. The authors reported that 100 percent of calcified ovarian cancers had nanobacteria present and 100 percent of uncalcified ovarian cancers lacked nanobacteria.
Neoprobe Corp., of Dublin, Ohio, said researchers reported data on Lymphoseek at the Fourth Biennial World Sentinel Node Congress meeting in Los Angeles showing the product identified lymphatic tissue in all of the patients treated to date in breast and melanoma cancer trials. Lymphoseek is intended to be used in sentinel node biopsy procedures for the detection of potentially cancerous lymph nodes.
Neurochem Inc., of Montreal, completed the treatment phase of the double-blind, placebo-controlled, randomized, international Phase II/III trial to measure the efficacy and safety of 1,3- propanedisulfonate (Fibrillex) to treat amyloid A amyloidosis. Neurochem expects to issue data in the second quarter. All patients who completed the trial have been invited to join the two-year, open-label Phase II/III extension study. The two-year, multicenter, randomized, double-blind and placebo-controlled trial enrolled 183 patients at 27 sites across North America, Europe, North Africa and Israel.
NovaDel Pharma Inc., of Flemington, N.J., initiated a pilot pharmacokinetic feasibility study in humans of a lingual spray formulation of zolpidem, which is marketed in the U.S. as the sleep-inducing agent Ambien by Sanofi-Aventis Group, of Paris. NovaDel's lingual spray drug delivery technology is designed to achieve more rapid onset of therapeutic activity by allowing drugs to enter the bloodstream directly through the mucosa lining the mouth. The pilot study will test the drug in a range of doses in humans.
Proneuron Biotechnologies Inc., of Los Angeles, said the Mount Sinai Medical Center and University Hospital in collaboration with Kessler Institute for Rehabilitation are enrolling patients in the Phase II study of ProCord for complete spinal cord injury. Progenitor Cell Therapy LLC, of Hackensack, N.J., will provide the required cell processing facility for the manufacturing of ProCord for both sites. ProCord consists of macrophages isolated from the patient's own blood, activated through Proneuron's process, and then injected into the patient's injured spinal cord.
Rho Ventures in New York announced the final closing of Rho Ventures V LP with commitments of $425 million to finance companies seeking early- and expansion-stage funding in the communications, health care and information technology sectors. Rho has invested in several biotech companies, including San Diego-based Diversa Corp.; Gaithersburg, Md.-based MedImmune Inc.; and Rockville, Md.-based Human Genome Sciences Inc.
RxGen Inc., of New Haven, Conn., said the National Institutes of Health funded Phase I of the company's $1.1 million fast-track Small Business Innovation Research grant to uncover genomic markers of Parkinson's disease. RxGen is using genomics and bioinformatics technologies and its experience with primate models of Parkinson's disease to profile in vivo brain RNA expression patterns following exposure to compounds known to cause the neuronal injury and dopamine depletion associated with Parkinson's disease.
Savient Pharmaceuticals Inc., of East Brunswick, N.J., received approval from the FDA for its premarket approval application for Nuflexxa, which is indicated to treat pain in osteoarthritis of the knee in patients who have failed to respond adequately to conservative non-pharmacologic therapy and simple analgesics. Nuflexxa is approved in Europe and will be marketed there under the brand name Euflexxa. Savient plans to file a supplement with the FDA in the first quarter to support room-temperature storage vs. the refrigerated expiration dating approved by the FDA. Savient also is in negotiations with a global marketing partner, it said.
Serologicals Corp., of Atlanta, intends to offer 4.2 million shares of its common stock, plus up to 630,000 shares to cover overallotments, for sale under an existing shelf registration statement. It expects to use the proceeds to repay existing term debt and for general corporate purposes. The company also said certain stockholders are offering about 1.4 million shares for sale in an offering that will be conducted concurrently with the company's offering. Serologicals will not receive any of the proceeds of that offering. Serologicals' stock (NASDAQ:SERO) fell $1.32 Tuesday to close at $22.51.
Tapestry Pharmaceuticals Inc., of Boulder, Colo., said it is on track for filing an investigational new drug application for its taxane, TPI 287, prior to the end of the year. The company is completing all toxicology and pharmacology testing required for beginning clinical trials, and it is manufacturing GMP material for Phase I testing. Tapestry expects to file an IND for its second compound, TPI 273, later than originally planned, during the first half of 2005.
Transmolecular Inc., of Birmingham, Ala., said that based on the favorable initial safety and tolerability profile of its cancer therapeutic, 131I-TM-601 for recurrent glioma or metastatic brain cancer, it is initiating a Phase II multicenter North American trial to include 66 patients with adult recurrent glioma. 131I-TM-601 incorporates a tumor-targeting, biologically active and chemically synthesized 36-amino-acid peptide derived from a naturally occurring protein found in scorpion venom that is conjugated with a medicinal radioisotope referred to as iodine 131.
Vaxon-Biotech, of Evry-Genopole, France, completed the Phase I trial of its vaccine, VX-001, which was conducted at Heraklion University in Greece. VX-001 was well tolerated, stimulated T-cell immunity, and showed clinical benefits in patients. It contains hTERT572Y, a single optimized cryptic peptide that targets tumors expressing the telomerase antigen. The trial involved 19 patients with advanced-stage metastatic cancer of different types that progressed despite chemotherapy.