In the Pipeline
In the Pipeline
Transkaryotic Therapies Inc., reports positive pivotal clinical results for its investigational enzyme replacement therapy, agalsidase alfa (Replagal), for the treatment of Fabray disease. The data suggest that treatment with agalsidase alfa has broad clinical effects in patients with Fabray disease. The phase II study assessed the safety and efficacy of agalsidase alfa over six months. Results showed that the compound is well-tolerated with an excellent safety profile. Patients showed clinically and statistically significant improvements in multiple manifestations of the disease. At six months, patients receiving 0.2 mg/kg every other week in 20- to 40-minute infusions had a reduction in severe pain, compared to no change for the placebo group. In addition, patient kidney function stabilized or improved in the treatment group, whereas kidney function declined in the placebo group.
CEL-SCI Corp. has received an approval for funding of a phase II study from the National Institute of Allergy and Infectious Diseases (NIAID) to further develop a herpes simplex vaccine. The grant will support the development of a herpes simplex virus vaccine based on CEL-SCI’s patented LEAPS (Ligand Epitope Antigen Presentation System) technology. LEAPS is a novel T-cell modulation technology that allows for the vaccine to selectively target particular immune cells and cause cellular antibody responses.
MacroMed has announced that the FDA has given a favorable review of the investigational new drug application (IND) for OncoGel, a novel formulation of paclitaxel that uses ReGel, MacroMed’s proprietary drug delivery system. IND approval allows initiation of clinical trials in the United States with OncoGel to direct high levels of paclitaxel to the tumor site while protecting the body from systemic side effects normally associated with paclitaxel when administered in its traditional form.
ViroPharma Inc. has initiated clinical trials with a new antiviral compound for the treatment of respiratory syncytial virus (RSV). Initial trials are designed to evaluate the safety and pharmacokinetic profile of the compound in healthy volunteers. The compound, known as VP14637, has shown high potency and selectivity in laboratory studies in which it inhibited RSV by affecting functions of the viral fusion protein, a protein essential for virus reproduction.
D-Pharm Ltd. has completed phase I safety assessment for its compound, DP-b99, a neuroprotective drug candidate. DP-b99 is being developed as a first-line therapy to improve clinical outcomes in stroke and traumatic brain injury patients, and as a prophylactic agent to protect against neurological complications often associated with open-heart surgery. The pharmacology of the compound involves a novel mechanism of action targeting apoptosis, which underlies the neurological damage that occurs after an ischemic insult to the brain.
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