Alexion Pharmaceuticals, Inc. announced the planned initiation of a pivotal Phase 3 study of ULTOMIRIS (ravulizumab) in amyotrophic lateral sclerosis (ALS). The 50-week global study, called CHAMPION-ALS, will evaluate approximately 350 adults across a broad patient population, and the primary endpoint will be change in ALS functional rating scale-revised (ALSFRS-R) score. The company submitted an investigational new drug application (IND) for ULTOMIRIS in ALS to the FDA in the fourth quarter of 2019 and plans to initiate the Phase 3 study this quarter. The Phase 3 CHAMPION-ALS trial is a randomized, double-blind, placebo-controlled multicenter global study designed to evaluate the efficacy and safety of ULTOMIRIS across a broad ALS population. The study will enroll approximately 350 adults with sporadic or familial ALS who have had disease onset (in the form of first motor symptoms) within the prior 36 months, demonstrate a slow vital capacity (SVC) of at least 65% predicted, and are not dependent on respiratory support. Study participants will be randomized on a 2:1 basis to receive ULTOMIRIS or placebo every 8 weeks following an initial loading dose and may continue to receive their existing standard of care treatment for ALS. After 50 weeks, all patients will receive ULTOMIRIS in a 2-year open-label extension phase of the study. The study will be conducted at approximately 90 clinical trial sites across North America, Europe and Asia-Pacific. The primary study endpoint will be change from baseline in ALS functional rating scale-revised (ALSFRS-R) score. Secondary endpoints will include ventilation assistance-free survival (VAFS), respiratory capacity, muscle strength, neurofilament light chain (NfL) serum concentrations and safety. ULTOMIRIS (ravulizumab-cwvz) is the first and only approved long-acting C5 complement inhibitor. It is administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose. ULTOMIRIS works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders. ULTOMIRIS is approved in the U.S., Japan, and the EU as a treatment for adults with PNH and in the U.S. for aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. ULTOMIRIS is a prescription medicine called a monoclonal antibody. ULTOMIRIS is used to treat adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). ULTOMIRIS is used to treat adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). It is not known if ULTOMIRIS is safe and effective in children with PNH. It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age in aHUS. ULTOMIRIS is a medicine that affects the immune system. ULTOMIRIS can lower the ability of the immune system to fight infections. ULTOMIRIS increases the chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early. Meningococcal vaccines must be received at least 2 weeks before the first dose of ULTOMIRIS if one has not already had this vaccine. If one’s doctor decides that urgent treatment with ULTOMIRIS is needed, meningococcal vaccination should be administered as soon as possible. If one has not been vaccinated and ULTOMIRIS therapy must be initiated immediately, 2 weeks of antibiotics should also be administered with the vaccinations. If one had a meningococcal vaccine in the past, additional vaccination might be needed before starting ULTOMIRIS. One’s doctor will decide if additional meningococcal vaccination is needed. Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call one’s doctor or get emergency medical care right away if any of these signs and symptoms of a meningococcal infection occur: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms, and eyes sensitive to light. ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. ULTOMIRIS may also increase the risk of other types of serious infections. People who take ULTOMIRIS may have an increased risk of getting infections caused by Streptococcus pneumoniae and Haemophilus influenzae. Certain people may also have an increased risk of gonorrhea infection. To find out if one is at risk for gonorrhea infection, about gonorrhea prevention, and regular testing, talk to the doctor. Call the doctor right away if one has any new signs or symptoms of infection. ULTOMIRIS can cause serious side effects including infusion reactions. Infusion reactions may happen during one’s ULTOMIRIS infusion. Symptoms of an infusion reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in the arms or legs. Tell the doctor or nurse right away if these symptoms develop, or any other symptoms during the ULTOMIRIS infusion that may mean one is having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of the face, tongue, or throat, and feel faint or pass out. One’s doctor will treat the symptoms as needed. The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory infection and headache. The most common side effects of ULTOMIRIS in people with aHUS are upper respiratory infections, diarrhea, nausea, vomiting, headache, high blood pressure, and fever.