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Biotechnol Healthc. 2010 Spring; 7(1): 10–11.
PMCID: PMC2873725

Research, Conferences, and FDA Actions

Amy Rossi, Assistant Editor

Late-stage successes

A phase 3 trial that compared the efficacy and safety of ustekinumab (Stelara) and etanercept (Enbrel) in the treatment of moderate to severe plaque psoriasis showed that over 12 weeks, 67.5 percent of ustekinumab patients experienced at least a 75 percent improvement in the psoriasis area and severity index, compared with 56.8 percent of etanercept patients. Ustekinu-mab also demonstrated efficacy in patients who were unresponsive to etanercept.

In a late-stage study, Amgen’s denosumab (Prolia) minimized the risk of serious bone complications in advanced breast cancer patients by 23 percent. ... Denosumab proved more effective than Novartis’ zoledronic acid (Zometa) in a phase 3 trial, delaying by 18 percent the time advanced prostate cancer patients suffered a fracture or needed surgery to ease bone pain.

The primary endpoint was met in a late-stage study of Roche’s ocrelizumab for rheumatoid arthritis (RA). Combined with methotrexate, ocrelizumab significantly improved the signs and symptoms of RA in hard-to-treat patients at 24 and 28 weeks. … Roche’s tocilizumab (Actemra) met a late-stage trial primary endpoint of significant improvement of disease signs and symptoms after 12 weeks for systemic onset juvenile idiopathic arthritis.

Shire’s velaglucerase alfa demonstrated long-term clinical efficacy and safety in a phase 3 study as an investigational enzyme replacement therapy for type 1 Gaucher’s disease. ... A phase 3 trial of Protalix BioTherapeutics’ taliglucerase alfa (Uplyso) for patients with Gaucher’s disease led to significantly reduced mean spleen volume after 9 months in two dosing arms compared with baseline; the primary endpoint of a 20 percent mean reduction in spleen volume after 9 months was achieved in both treatment groups after 6 months of therapy.

Human Genome Sciences and GlaxoSmith Kline say a second late-stage trial of belimumab (Benlysta), an experimental lupus therapy, was statistically superior to placebo. A low-dose formulation did not show a statistically superior response.

Trial disappointments

Pfizer ended a late-stage study of experimental lung cancer drug figitumumab after finding that the addition of the drug to paclitaxel and carboplatin was unlikely to meet the main goal of improving overall survival compared with paclitaxel and carboplatin alone. ... Genentech’s bevacizumab (Avastin) did not meet a late-stage trial’s primary endpoint of extended overall survival. Patients with stomach cancer were given bevacizumab in combination with chemotherapy agents fluorouracil or Roche’s capecitabine (Xeloda) and cisplatin.

Mixed results were reported by Ception for reslizumab (Cinquil) for the treatment of pediatric eosinophilic esophagitis. Patients met the primary endpoint of a decrease in esophageal eosinophil levels; however, when compared with the control arm, patients did not achieve a statistically significant improvement in symptoms. With an acquisition opportunity for Ception in the works, Cephalon has decided to wait for results from a future reslizumab trial before finalizing the acquisition.

Enrollment was halted on two ecallantide (Kalbitor) studies after a safety monitoring board determined there was a higher death rate among high-dose patients who were took the drug to reduce bleeding during on-pump cardiac surgery.

Spectrum Pharmaceuticals has ended development of ozarelix for enlarged prostate treatment after a mid-stage trial failed to show statistically significant improvement compared with a dummy drug.

Did you hear?

Shire’s velaglucerase alfa, for the treatment of Gaucher disease, has been granted priority review by the U.S. Food and Drug Administration and will be reviewed in 6 months rather than the standard 10.

Fabry disease treatment Replagal, also from Shire, has received a fast-track designation by the FDA. Patients with Fabry disease experienced substantial and sustained benefits in all areas studied, including heart and kidney function, pain, and quality of life after 5 years of enzyme replacement therapy.

Nanosensors have been used to detect cancers quickly through blood tests, and nanomaterials also have enabled the release of medicine at targeted organs. Now, investigators at Yale University have discovered tiny amounts of cancer biomarkers in a small volume of whole blood in 20 minutes.

Scientists have identified five genetic variants that influence lung function, potentially leading to better lung disease treatments. … The two genes involved in inflammatory processes that result in ankylosing spondylitis have been identified as ERAP1 and IL23R, allowing for further treatment development for the condition. … The entire genetic codes of skin and lung cancer have been revealed through the cataloguing of their genetic maps, pinpointing the specific mutations within DNA that can lead to dangerous tumors. ... Scientists are developing a fast, low-priced test in which a drop of saliva can determine a person’s chances of developing certain genetic diseases, potentially replacing conventional DNA testing.

Action dateManufacturerDrug (trade name)IndicationAdministration
Biologics license approval
Jan. 8Genentechtocilizumab (Actemra)Rheumatoid arthritis; first to block interleukin-6 activityIntravenous infusion
New indication
Feb. 18Genentechrituximab (Rituxan)Chronic lymphocytic leukemiaIntravenous infusion
Genetic test marketing clearance
Jan. 21AbaxisC-reactive protein assayTest for infections, inflammatory diseases, tissue injuries, and abnormal cell growth
ManufacturerDrug (trade name)Type of drugIndication soughtNotes
Biologics license application
Dendreonsipuleucel-T (Provenge)Cell-based vaccineAdvanced prostate cancerWould be first active cellular immunotherapy in the United States
Protalix Biotherapeuticstaliglucerase alfa (Uplyso)Plant cell-expressed form of GCDGaucher’s disease
Shireagalsidase alfa (Replagal)Enzyme replacement therapyFabry diseaseCurrently available to U.S. Fabry patients under FDA-approved regimen
TevaXM02 (Neutroval)Granulocyte colony-stimulating factorBoost white blood cell count in chemotherapy patientsBiosimilar of Amgen’s filgrastim (Neupogen)
Supplemental biologics license application
Humane Genome Sciencesalbinterferon alfa-2b (Zalbin)Genetic fusion of human albumin and interferon alfaHepatitis C
Orphan drug designation
GenVecTNFeradeAdenovector, or DNA carrierAdvanced pancreatic cancer
Denials or delays
Human Genome Sciencesraxibacumab (ABthrax)Human monoclonal antibodyAnthraxFDA asked for more information on how drug compares with current anthrax treatments


Sources: Manufacturers’ news releases, FierceBiotech, BIO SmartBrief, wire reports, and weblogs

Debiopharm is giving tremelimumab another chance; previously, a safety monitoring board recommended halting a late-stage study of the anticancer immunotherapy when the drug proved no more effective than chemotherapy. If Debiopharm succeeds, Pfizer will be responsible for its commercialization.

Following its merger with Wyeth, Pfizer has discontinued about 100 drug development programs because of portfolio overlap or scientific evidence that discouraged continued testing of a drug. The 500 remaining programs will pertain to six key areas: oncology, pain, inflammation, Alzheimer’s disease, psychoses, and diabetes.

All clinical trials described in Drug Track are phase 3, randomized controlled studies unless otherwise specified.

Articles from Biotechnology Healthcare are provided here courtesy of MediMedia, USA