So this is kind of a bummer: Clinical trials looking into using Alexion Pharmaceutical’s drug Soliris for treatment-resistant generalized myasthenia gravis have just come up short of expectations.
Soliris, which is already approved for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), works to inhibit a certain part of the immune system called “terminal complement” thought to play a role in disease activity. Researchers had hoped that patients in the trial would show significant improvement in activities of daily living; while there were some improvements, they were not statistically significant.
What’s next for this line of research? It’s hard to say. Alexion will make the argument that even though the results shown for the primary endpoint were more modest than what they had hoped for, the unmet need for this segment of the MG population justifies moving forward with applying for FDA approval. And they can at least point to much better results in the trial’s secondary endpoint: Improvements in patients’ responses to the Quantitative Myasthenia Gravis (QMG) Test.
Whether that will be enough to push Soliris over the finish line remains to be seen—Alexion shares dropped 9% after the trial results were announced. But progress isn’t always a straight line forward; sometimes you have to take a step or two back before you can make the next giant leap into the future.
Don’t give up the fight! Go here to learn about other MG clinical trials in progress.
