Alexion Pharmaceuticals is a company focused on ultra-rare diseases, although it is working on a shift into rare diseases. This may sound like something of a minor shift, but it represents developing drugs for more diseases with larger patient populations. The company’s success has been built primarily on its Soliris (eculizumab), a complement inhibitor. The drug has been approved for paroxysmal nocturnal hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS) and for generalized Myasthenia Gravis (gMG).
Soliris, as well as the company’s next-generation complement inhibitor, Ultomiris, underline the company’s expertise in complement-related diseases. The complement system is part of the immune system made up of about 30 different proteins. As such it plays a role in numerous diseases at some level, and alone in several diseases, including PNH, aHUS and others.
John Orloff, Alexion’s executive vice president and Head of Research and Development, and Brian Goff, executive vice president and Chief Commercial Officer, spoke with BioSpace about Alexion and new trends and developments the company is reporting.
One piece of news was Alexion presented an abstract regarding a Phase III clinical trial of Soliris in gMG at the recent American Academy of Neurology Annual Meeting held in Philadelphia. Myasthenia gravis is a chronic autoimmune neuromuscular disease resulting in weakness in the skeletal muscles. The muscles most affected control eye and eyelid movement, facial expression, chewing, talking and swallowing. Muscles that control breathing, and neck and limb movements may also be affected.
Goff told BioSpace, “The storyline here is that Soliris was approved for gMG in October 2017 in the U.S. That was based on the pivotal Phase III trial, which was the first FDA approval in more than 60 years in gMG. The concept is a totally different way of treating these patients with complement inhibition. Historically, and to this day, many patients are treated with continuous cycles of immunosuppressant therapy (IST). That’s a different way of treating the disease. Soliris goes to the core mechanisms of complement, the key core of the disease.”
The current trial evaluated the drug in 117 adults with gMG who were on IST who had completed the REGAIN clinical trial, a 6-month, randomized trial of Soliris. During REGAIN, changes in concomitant myasthenia gravis therapies weren’t permitted. But during the open-label expansion (OLE) study, patients received Soliris every two weeks after a 4-week blinded induction phase. Changes to the MG therapies, including ISTs, were at the investigator’s discretion, but weren’t required. Goff notes that they were looking to answer two questions. First, can patients sustain the durability of clinical effect. “More and more we’re seeing that the answer is yes.”
The second question is, in patients receiving IST, if they take Soliris, can they reduce or eliminate IST. And the answer, Goff says, is “yes, there is a cohort of patients who we do believe can reduce the level of IST, which creates a treatment sustainability option for those patients as well.”
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