Scleroderma

Daval International announced yesterday, positive results from its Double-Blind Placebo-Controlled Phase II Clinical Study evaluating the safety and tolerability of AIMSPRO given as a monotherapy to patients with Late Stage Established Diffuse Cutaneous Systemic Sclerosis (diffuse scleroderma). The study evaluated the effects of 4.5mg/ml doses of AIMSPRO, administered subcutaneously, twice weekly for 26 weeks compared with placebo.

The primary endpoint of the study was to evaluate the safety and tolerability of AIMSPRO in the treatment of twenty patients with Systemic Sclerosis (diffuse scleroderma) through a period of 26 weeks of study participation. The secondary outcome measures of the study were to assess the efficacy of AIMSPRO as a therapeutic agent for scleroderma using amongst others, the Scleroderma Health Assessment Questionnaire, the Modified Rodnan Skin Score, the Scleroderma UK Functional Score, the Patient and Physician Global Assessment (VAS), the SF-36 (Short form 36), and the MRC Sum Score.

At the end of the 26 week period AIMSPRO proved to be a safe and well-tolerated medication when administered to these especially sick patients at such a late-stage of the disease. There was no deterioration in haematological, biochemical, immunologic, cardiologic or in pulmonary parameters that were measured. In addition to the positive safety result, there were encouraging signals of clinical benefit in the secondary outcome measures. There were also trends towards benefit for lung function measures.

"Although requiring further confirmatory studies, these results are quite exciting, especially when compared to what has been seen in other pilot trials in scleroderma as there is a clear unmet medical need for patients suffering with this life-threatening disease" said Professor Christopher Denton, Professor of Experimental Rheumatology at the Royal Free Hospital in London, United Kingdom, the principal investigator for the trial. "The important value of the safety data from such a well-conducted trial in such a serious disease is clear, apart from the signals of therapeutic benefit."

Daval's Clinical and Scientific Director, Professor Syed Haq commented, "This study, conducted at one of the world's leading scleroderma centres, was an important first step towards understanding whether AIMSPRO could significantly help patients with diffuse scleroderma and potential overlapping connective tissue disorders, for which there is no cure, by arresting the disease even in its latter stages. AIMSPRO has been shown to be well-tolerated and safe and with the several positive therapeutic signals demonstrated, some of which were significant, it certainly warrants further investigation on a larger scale to determine the full efficacy of AIMSPRO in Late Stage Established Diffuse Cutaneous Systemic Sclerosis."

At the completion of the 26 week period of the study, AIMSPRO was offered to the patients on a compassionate use basis and the results from the 52 week visit will be presented as an addendum to the initial study report.

Daval is now assessing the biomarker data collected in this clinical trial and correlating this with the changes observed, so as to shed more light into the mechanism of action of AIMSPRO in the context of Late Stage Established Diffuse Cutaneous Systemic Sclerosis.

Source: PRNewswire