Sjogren's Syndrome

August 15, 2016

Aldeyra Therapeutics released results of a randomized, parallel-group, double-blind, vehicle-controlled, multicenter trial of topical dermatologic NS2 for the treatment of the dermal manifestations of Sjögren-Larsson Syndrome (SLS). Twelve subjects with SLS and moderate to severe ichthyosis were randomized equally to receive NS2 1.0% dermatologic formulation or vehicle formulation administered once daily (QD) on a 4x10 inch area of skin for two months. Ichthyosis was graded by a central review of digital photographs, as well as by clinical exam, using the Ichthyosis Severity Score, which is comprised of assessments of global impression, scaling, erythema (redness), lichenification (thickness) and excoriation (abrasion). In addition, dermal tissue from subjects was analyzed for biomarkers relevant to fatty aldehyde dehydrogenase deficiency. NS2 consistently produced clinically meaningful effects in reducing the severity of ichthyosis. As assessed by central review, five of six subjects (83%) treated with NS2 achieved a rating of “almost clear” or “mild” on global assessment. Six of six (100%) subjects treated with NS2 improved over the course of therapy as assessed by central review, and the improvement was greater than that observed with vehicle-treated patients (p<0.05). For NS2-treated subjects, mean reductions in ichthyosis severity were greater after eight weeks of therapy than after four weeks of therapy, suggesting a disease modifying effect of NS2. Consistent with the clinical activity of NS2, significant dermal biomarker changes were noted in NS2-treated patients but not vehicle-treated patients (p<0.004). Cholesterol, which is significantly elevated in the skin of SLS patients, was reduced in NS2-treated patients more than in vehicle-treated patients (p<0.001). NS2 was observed to be generally well-tolerated and there were no significant adverse events, serious adverse events or discontinuations in the trial.

April 14, 2008

Parion issued mixed results from a phase I trial of P-552-02 for the treatment of dry mouth associated with primary Sjogren's syndrome. This twenty-eight day, randomized, double-blind, placebo- controlled, crossover study enrolled thirty subjects who received a six-time daily oral rinse formulation of P-552-02 or placebo. The primary efficacy endpoint, a global improvement in the "feeling of dry mouth" as determined by a single retrospective "recall" report at day twenty eight, was not met. However, an assessment of "global change in dry mouth" as determined by the change in visual analog scale (VAS) measurements twelve hours after dosing at day seven and day twenty eight revealed significant improvement in the global dry mouth scores over placebo. Based on the results Parion plans to proceed with a phase I/IIa study designed to test the efficacy of higher concentrations of P-552-02.

TaiGen released positive results from a phase II trial of nemonoxacin for the treatment of community-acquired pneumonia (CAP). This randomized, double-blind, comparative trial enrolled two hundred and sixty seven subjects. The primary endpoint was the non-inferiority of nemonoxacin in the clinical cure rate of CAP compared with levofloxacin, the current standard of care. Nemonoxacin 750 mg administered orally once daily over a period of seven days achieved non-inferiority in clinical cure rate of CAP compared with levofloxacin 500 mg once daily. Overall, 82.6% of the intent-to-treat population and 90% of the evaluable population were cured with nemonoxacin compared with 80.0% and 91% cure rate in the levofloxacin arm. Treatment was safe and well tolerated, with an adverse event profile similar between the two treatment arms. Based on the positive results, a phase II trial evaluating once-a-day intravenous dosing is scheduled to enter clinical trial the second quarter of 2008.

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