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Clinical Data Detailed Study Results:At ICNMD, Dr. Howard reported that 18 of the study’s 22 pre-specified analyses achieved p-values <0.05. As previously reported, these included the first three secondary efficacy endpoints of change from baseline to week 26 in Quantitative Myasthenia Gravis (QMG) total score, a physician-administered assessment of MG clinical severity (p=0.0129), as measured by a worst-rank analysis; the proportion of patients with at least a 3-point reduction in MG-ADL total score and no rescue therapy from baseline to week 26 (p=0.0229); and the proportion of patients with at least a 5-point reduction in QMG total score and no rescue therapy from baseline to week 26 (p=0.0018). The primary efficacy endpoint of change from baseline in MG-ADL total score at week 26 did not reach statistical significance (p=0.0698) as measured by a worst-rank analysis.Several prospectively defined sensitivity analyses were conducted to validate results for the primary and secondary endpoints, including sensitivity analyses for change from baseline in MG-ADL, QMG, MGC, and MG-QOL15 using repeated measures, all of which achieved p-values <0.05. As previously reported, the pre-specified sensitivity analyses of the primary and first secondary endpoints of MG-ADL and QMG using repeated measures had p-values of 0.0058 and 0.0006, respectively. The pre-specified sensitivity analyses of MGC and MG-QOL15 using repeated measures at week 26 showed a mean change from baseline in MGC with eculizumab treatment at week 26 of -8.1 versus a mean change with placebo of -4.8 (p=0.0134), and a mean change from baseline in MG-QOL15 with eculizumab treatment at week 26 of -12.6 versus a mean change with placebo of -5.4 (p=0.0010).Additional results from the study, including detailed outcomes from the MG-ADL and QMG responder analyses, were presented at ICNMD.

The safety of eculizumab in this study was consistent with the Soliris labels. The most common adverse events in patients receiving eculizumab and placebo, respectively, were: headache (16.1%, 19.0%), upper respiratory tract infection (16.1%, 19.0%), nasopharyngitis (14.5%, 15.9%), nausea (12.9%, 14.3%), and myasthenia gravis (9.7%, 17.5%). Serious adverse events were reported in 14.5% of eculizumab patients and 28.6% of placebo patients. Four patients receiving eculizumab (6.5%) discontinued treatment due to an adverse event. There were no discontinuations due to adverse events in the placebo arm. Half as many patients treated with eculizumab received rescue therapy compared with placebo (9.7%, 19.0%).Ninety-four percent of patients (117 of 125) from the REGAIN study continued into a Phase 3 open-label extension study evaluating the safety and efficacy of eculizumab in the treatment of patients with refractory gMG. Eculizumab has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S., EU, and Japan. Eculizumab is not approved in any country for the treatment of patients with gMG.

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