Idiopathic Inflammatory Myopathies

June 15, 2009

Novartis released positive results from a phase III trial of Ilaris (canakinumab) for the treatment of cryopyrin-associated periodic syndrome (CAPS). This 48-week, multinational, randomized, double-blind and placebo-controlled study enrolled 35 subjects, aged 9 to74 years old. The trial was divided into three parts: in the first part, lasting 8 weeks, 35 subjects received a single dose of Ilaris 150 mg by subcutaneous injection. All but one showed a rapid and complete clinical response. Thirty-one subjects proceeded to part two, a randomized 24-week, double-blind placebo-controlled phase. Subjects were treated every 8 weeks with either Ilaris or placebo. Part two of the study included the primary endpoint: a comparison Ilaris and placebo in the proportion of subjects who experienced disease relapse or flares at the end of 8 weeks. The primary endpoint was reached; none of the subjects in the Ilaris group experienced a disease flare compared to 13 out of 16 subjects in the placebo group (0% versus. 81%, p<0.001). The subjects who relapsed during part two of the study discontinued and entered part three. Thirty-one subjects entered the last phase of the study, 29 of whom were evaluable for response. This open label period evaluated two further doses of Ilaris for a minimum of 16 weeks. Clinical and biochemical remission of CAPS was sustained in 97 % of this population.

August 28, 2006

Kemia reported positive results from two phase I trials of KC706 for the treatment of chronic inflammation. A total of 79 healthy volunteers received oral administration of KC706 in three studies designed to evaluate the safety and pharmacokinetics of the drug. The initial escalating, single-dose study revealed that the compound was well tolerated. The second escalating, multiple-dose study demonstrated the safety of various dosages and determined the range of doses to be used in upcoming trials. The third phase I trial established the bioavailability of a capsule formulation of KC706 for use in future trials. Based on these results, Kemia planned to initiate a phase IIa study in Europe by the end of September 2006.

June 20, 2005

Regeneron Pharmaceuticals has issued preliminary results of a pilot study of IL-1 Trap, for the treatment of the autoimmune disorder CIAS1-associated periodic syndrome (CAPS). Results from the ongoing study indicated that the drug was efficacious in reducing levels of several inflammatory biomarkers, including C-reactive protein, erythrocyte sedimentation rate, and serum amyloid A protein. Furthermore, the drug produced significant improvements in daily patient diary symptom severity scores. Weekly dosing was seen to maintain favorable responses. This open-label pilots study has enrolled 4 subjects to date, who received a loading dose regimen of IL-1 Trap (100 mg per day for three days), followed by a drug free washout to investigate symptom recurrence, then followed resumption of a once weekly regimen of the drug. The company hopes that full results from this study will identify optimal dosing regimens for future investigations.

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