Rare Diseases, Results

Roche, Alexion ripe for showdown in a race to treat rare CNS disorder

Months after Alexion’s $ALXN flagship Soliris enthralled investors with impressive pivotal data for patients with neuromyelitis optica spectrum disorder (NMSOD), researchers at Roche’s Chugai on Wednesday reported positive Phase III data testing its drug, satralizumab, as a monotherapy in the same patient population.

There are no treatments approved specifically for the rare disorder, which is often confused with multiple sclerosis as the immune system attacks its host, damaging healthy tissue in the spine and eyes. Patients frequently experience a relapsing disease course with repeated attacks leading to accumulated neurological damage and disability and, sometimes, even death.

The study, which tested satralizumab against a placebo, adds to previous results from another trial conducted by the Tokyo-based drugmaker that showed satralizumab conferred improvements in patients who were on baseline treatment.

A statistically significant reduction in the risk of relapse was observed in patients who received satralizumab, compared to those who received placebo, Chugai said, adding that the company is now preparing a regulatory filing. Detailed data from the 90-patient study will be provided at a future medical meeting. The FDA, meanwhile, has rewarded the Roche subsidiary with a breakthrough therapy designation for the therapy, which bodes well for a swift review.

Credit Suisse analysts covering Alexion were unperturbed, however, saying although the Chugai data underscore satralizumab’s efficacy in the indication and suggest the drug has potential to capture market share in earlier lines of therapy, there are no changes to their projections for Soliris for the patient population. They reiterated their estimate that the expensive drug will likely reap between $400 million and $500 million in global peak sales, based on a 10-15% penetration in the market for patients with relapsing NMSOD.


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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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Catalytic Data Science Charleston, SC
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Flatiron Health New York City or San Francisco

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