Pharmos Corporation (OTC-PINK: PARS) announced that it has successfully completed a proof-of-concept clinical trial using its compound levotofisopam (S-tofisopam) to treat patients with hyperuricemia and gout. This phase 2a trial was conducted at the Duke Clinical Research Unit of Duke University and the principal investigator was John Sundy, MD, PhD, an expert and key opinion leader in the treatment of gout. The trial was designed to assess the safety and efficacy of levotofisopam as a uric acid-lowering agent in patients with gout.
The trial enrolled 13 patients in an open label study with patients confined in the Duke facility. The study enrolled patients with screening serum urate between 8 and 12 mg/dL. Subjects received a single dose of 50 mg on days 1 and 7 and 50 mg TID on days 2 through 6. Levotofisopam was well tolerated. The mean reduction in serum urate was over 45%. All 13 patients were responders, and demonstrated a serum urate level of less than 6 mg/dL on day 7. Seven subjects achieved a serum urate level less than 4 mg/dL on day 7. Additionally there was an increase in the fractional excretion of urate, confirming the compound's mechanism of action as a uricosuric agent that enhances urate excretion by the kidneys.
Commenting on the results of this Phase 2a trial, the principal investigator Dr. John Sundy said, "monotherapy with levotofisopam was well tolerated and induced clinically important reductions in serum urate levels in all patients studied. These results support further development of levotofisopam for treating hyperuricemia in patients with gout."
This trial follows two prior phase 1 clinical studies conducted by Vela Pharmaceuticals (merged with Pharmos in October 2006). In these studies, conducted in healthy volunteers in the United Kingdom and The Netherlands, levotofisopam treatment was generally well tolerated and was associated with a large and rapid reduction in serum uric acid values.
Commenting on the results, S. Colin Neill, President, stated, "given that the compound is safe, and has been exposed to 90 subjects in total, levotofisopam should be an attractive compound for a pharmaceutical company to partner or license. Achieving a partnership is now our primary objective."
Levotofisopam is the S-enantiomer of the racemic mixture RS-tofisopam, a well tolerated agent used for the treatment of a variety of disorders associated with stress or autonomic instability. Racemic tofisopam is not marketed in the U. S. but has been approved since 1974 and marketed in more than 20 other countries around the world. Dextofisopam, the R-enantiomer, is being developed for the treatment of irritable bowel syndrome (IBS) and has completed testing through phase 2b in the U. S.
Pharmos believes levotofisopam has the potential to address a poorly served population of gout patients who respond inadequately to first-line treatment, and that this population represents a substantial global market. Pharmos owns the rights to both R- and S-tofisopam, the two enantiomers of racemic tofisopam, through two US issued composition-of-matter patents. The Company is actively seeking a partnership to continue the clinical development of levotofisopam in gout.
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