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On September 25, 2020, Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved ULTOMIRIS® (ravulizumab) for adults and children living with atypical hemolytic uremic syndrome (aHUS). ULTOMIRIS is the first and only long-acting C5 inhibitor for aHUS and is administered every other month for adults and children (20 kg or more) and monthly for children (<20 kg). Atypical HUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.

“The goal of aHUS treatment is to prevent the body from attacking its own immune system through the inhibition of uncontrolled C5 complement activation,” said Prof. Shoichi Maruyama, Director, Department of Nephrology, Nagoya University Hospital. “Importantly, ULTOMIRIS demonstrated good control, while also offering more time between infusions, which provides a relevant difference to patients and providers.”

Atypical HUS affects both adults and children and many patients present in critical condition in the hospital setting, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, with 56 percent of adults and 29 percent of children developing end-stage renal disease or dying within a year of diagnosis with supportive care alone, so a timely and accurate diagnosis in addition to treatment, is critical to improving patient outcomes.

“Today’s approval marks another important step in our efforts to continue innovating for patients and improving their treatment experience,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “ULTOMIRIS’ extended dosing interval offers patients more flexibility and time to focus on living their lives beyond their disease while also reducing the burden on healthcare systems, hospitals and providers, which are all under a tremendous amount of stress in the current environment.”

The approval is based on data from two ongoing, global, single-arm open-label studies of ULTOMIRIS – one in adults and one in children, referred to as pediatrics in the study. A total of 18 out of 21 complement inhibitor treatment-naïve children and 56 out of 58 complement inhibitor treatment-naïve adults were enrolled and included in the interim analysis. Efficacy evaluation of Complete TMA Response was defined by normalization of hematologic parameters (platelet count and LDH) and improved kidney function (as measured by ≥25 percent improvement in serum creatinine from baseline). In the initial 26-week treatment periods, 54 percent of adults and 77.8 percent (interim data) of children demonstrated Complete TMA Response. Treatment with ULTOMIRIS resulted in normalization of platelet count in 84 percent of adults and 94 percent of children, normalization of LDH (marker of hemolysis) in 77 percent of adults and 90 percent of children, and improved kidney function in 59 percent of adults and 83 percent (interim data) of children (for patients on dialysis at enrollment, baseline was established after they had come off dialysis). In the 52-week follow-up period, 4 additional adult patients and 3 pediatric patients had a Complete TMA Response that was confirmed after the 26-week Initial Evaluation Period resulting in an overall Complete TMA Response of 61 percent in adults and 94 percent in children (interim data). A second cohort of 10 pediatric patients who were SOLIRIS-experienced were included in the pediatric study, demonstrating that switching to ULTOMIRIS maintained disease control as evidenced by stable hematologic and renal parameters, with no apparent impact on safety.

The most frequently observed adverse reactions reported in these studies were upper respiratory tract infection, diarrhea, nausea, fatigue, headache, nasopharyngitis, and pyrexia. Serious meningococcal infections have occurred in patients treated with ULTOMIRIS, however in aHUS studies, no meningococcal infections occurred in the 89 patients receiving treatment with ULTOMIRIS. To minimize the risk for patients, specific risk-mitigation plans, including a Risk Management Plan, have been established for ULTOMIRIS.

ULTOMIRIS will be available at approval for the treatment of aHUS.

aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. aHUS occurs when the complement system—a part of the body’s immune system—over-responds, leading the body to attack its own healthy cells. aHUS can cause sudden organ failure or a slow loss of function over time—potentially resulting in the need for a transplant, and in some cases, death. aHUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosis—in addition to treatment—is critical to improving patient outcomes. Available tests can help distinguish aHUS from other hemolytic diseases with similar symptoms.

On September 21, 2020, Alexion announced that the Committee for Medicinal Products for Human Use, or CHMP, has recommended marketing authorization in the European Union for a new 100 mg/mL intravenous, or IV, advanced formulation of Ultomiris. Ultomiris is a long-acting C5 inhibitor administered every eight weeks for the treatment of two ultra-rare diseases-paroxysmal nocturnal hemoglobinuria, or PNH, and atypical hemolytic uremic syndrome, or aHUS. Ultomiris 100 mg/mL would constitute an advancement in the treatment experience for patients with aHUS and PNH by reducing average annual infusion times by approximately 60% compared to Ultomiris 10 mg/mL while delivering comparable safety and efficacy. With Ultomiris 100 mg/mL, most patients will spend six hours or less a year receiving treatment.

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