Alexion Pharmaceutical shares were down to 9.7 percent last Monday, June 6, after the patients participating in the clinical trial reported no significant changes in their daily activities.
The company is working on a neuromuscular drug to treat a rare disease called Myasthenia gravis. In the final stage of the trial, the results failed to meet the primary end-point or goal of the study. Their candidate drug is Soliris or eculizumab is trying to expand their treatment application by targeting this muscle weakening disease.
In the phase 3 of the clinical study, the patients did not have any statistically significant changes after 26 weeks of treatment as compared with the placebo.
After the stock market closed in New York, the company share was down to 11 percent with $137.85 per share.
David Hallal, Chief Executive Officer of Alexion, said in a press release statement, that there were positive signs in the trial. He noted that some patients had improvements during the secondary measure of the clinical study. Hallal also pointed out during the investors meeting that there are a number of therapies for rare condition which are unable to meet the requirements of FDA particularly the primary end-point, but has been granted an approval.
The Lead Drug
Soliris is approved by FDA to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Last year, Soliris generated $2.59 billion sales for Alexion. In fact, it is the company’s main pipeline which generated most of its revenue.
In the secondary measurements of the clinical study were doctors assessed the severity of their disease, the patients reported some improvement.
The full result of the study will be presented by Alexion at the International Congress on Neuromuscular Diseases in Toronto, Canada on July 7.
Understanding Myasthenia Gravis
Acquired myasthenia gravis is a rare neuromuscular disease that affects 20 out of 100,000 people in the US based from the report of Myasthenia Gravis Foundation of America. It is an autoimmune disease that attacks the connection between the nerves and muscles or the neuromuscular junction. This disease is characterized by muscle weakness that fluctuates, worsen with exertion, and improve with rests.
About two-thirds of the patients, presents with an involvement of the extrinsic ocular muscles as the initial symptoms. The disease progressed to involve other muscles including the limb muscles that results to a generalized form of myasthenia gravis. There is no known cause of the disease but scientist were able to figure out the role of circulating antibodies that targets the nicotinic receptor as the main culprit of the disease condition.
Soliris is used to treat refractory generalized form of myasthenia gravis that affects 10 to 15 percent of the patients.
Myasthenia is Highly Treatable
Myasthenia can be treated as long as the disease is recognized on its early stages. There are a lot of new treatment modalities that leads to the significant reduction of the disease. Alexion’s lead drug, Soliris is aiming to be one of the alternative drugs for the treatment of Myasthenia gravis.
Although, the results were not in favor of the company, maybe in the coming days, the company will release combination treatment that would increase the potency of the drug. At the end, it will be the patients who will benefit more in this study considering that they will have another treatment option.
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Trouth, A. J. (2012). Myasthenia Gravis: A Review. Autoimmune Dis .