Recently, Alexion Pharmaceuticals (ALXN) presented encouraging data from a small mid-stage study (n=14) which evaluated its drug Soliris in patients suffering from severe and refractory generalized myasthenia gravis (gMG).
Data from the randomized, double-blind, placebo-controlled, cross-over study were presented at the annual Scientific Session of the Myasthenia Gravis Foundation of America, Inc. in San Francisco. The patients evaluated in the mid-stage study were suffering from moderate to severe muscle weakness despite being treated with immunosuppressants. The patients were divided equally into two groups – one receiving Soliris, the other placebo.
Data from the study revealed strong signals regarding improvement of the disease for patients in the Soliris arm. Improvement was measured through the Quantitative Myasthenia Gravis disease severity score (QMG score).
Data further revealed that after 16 weeks of treatment 6 of the 7 patients in the Soliris arm achieved a three-point reduction in their QMG score. Only 4 of the 7 patients in the placebo group achieved similar results.
We remind investors that Soliris is already marketed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare genetic blood disorder. Alexion is studying Soliris for other indications as well, including myasthenia gravis.
Alexion has made giant strides towards getting Soliris approved for treating patients suffering from atypical hemolytic uremic syndrome (aHUS). The disorder often leads to heart attack, stroke or kidney failure which can even prove to be fatal. Soliris is under priority review in the US for the aHUS indication. Alexion expects to launch the drug in the US by year-end, if approved for the additional indication. Soliris is also under review in the European Union (EU) for aHUS.
Approvals for marketing Soliris in the EU and the US for aHUS were sought on the basis of encouraging data from two mid-stage studies. The 26-week studies evaluated Soliris for treating adults and adolescents suffering from aHUS.
We note that the aHUS market provides a lucrative opportunity for the company as patients suffering from the disorder have limited treatment options. Therefore, if Soliris is approved for treating the disease then the market potential for this product will further expand.
Our Recommendation
Currently, we have a Neutral stance on Alexion. The stock carries a Zacks #3 Rank (short-term Hold recommendation).
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