Alexion Pharmaceuticals yesterday announced promising trial results from its Phase 3 study of eculizumab (trade name Soliris) in patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD).
NMOSD is a rare and debilitating disorder of the central nervous system characterized by relapses. Each relapse results in accelerated incidence of disability, including blindness and paralysis, and sometimes premature death. Patients with anti-AQP4 auto-antibodies represent about three-quarters of all NMOSD patients.
There are presently no approved therapies for this disease.
“These results far exceeded our expectations. The remarkable reduction in relapse risk demonstrates the unique ability of Soliris to inhibit complement, and suggests a promising new treatment for NMOSD,” said John Orloff, MD, Alexion’s executive vice president and head of R&D. “Given that patients currently have no approved therapies, we are moving quickly to discuss these results with regulators and file for approval in the U.S., EU, and Japan.”
Detailed results from this Phase 3 study will be presented at a future medical congress.
What you don’t know can kill you
A genomic screening approach of more than 50,000 people shows that more than 80 percent of those who carry an identifiable genetic risk for breast, ovarian, prostate and pancreatic cancer don’t know it, even despite regular interaction with the health-care system.
In the absence of routine screening, most discover they carry cancer-associated variants in the BRCA1 or BRCA2 genes only when that person or family members receive a cancer diagnosis.
“It usually takes a tragedy for people to get tested,” said Michael Murray, professor of genetics at the Yale School of Medicine and senior author of the study, conducted at Geisinger Medical Center in Danville, Pa. “Our reliance on a documented personal or family history as a trigger to offer testing is not working. Hopefully, one day we can change that with effective DNA-based screening for everyone.”
The findings were published Sept. 21 in the journal JAMA Network Open.
Precipio expands lung-cancer detection kits
Physicians will soon have the ability to use a liquid biopsy to identify lung cancer patients eligible for Tyrosine Kinase Inhibitor (TKI) therapies through analysis of tumor DNA for critical EGFR mutations.
The liquid-biopsy kit, trade name ICEme, was this month launched by New Haven-based Precipio Inc. It uses proprietary technology to complements Precipio previously-launched TKI-resistance detection kit, providing doctors with a full arsenal of sequential lung-cancer diagnostic tools critical to determining patient treatment.
