- ULTOMIRIS is the first approved, long-acting complement inhibitor for PNH, administered every other month, reducing the treatment burden for patients -
- ULTOMIRIS has the potential to become the new standard of care for both complement inhibitor-naïve patients and patients switching from SOLIRIS® (eculizumab) -
PNH is an ultra-rare and severe disease that, when left untreated, may cause a wide range of debilitating symptoms and complications, including thrombosis. Thrombosis occurs when a blood clot presents inside a blood vessel and slows or blocks the flow of blood through the circulatory system. Serious cases of thrombosis can occur throughout the body and result in organ damage, stroke, heart attack, and potentially premature death.2-9
“More than a decade after SOLIRIS was approved, ULTOMIRIS provides a major step forward in the treatment of PNH,” said Dr.
PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.3,10 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than five years.11
“At Alexion, our goal is to continue to improve the lives of people and families affected by PNH and other serious rare diseases,” said
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating and life-threatening ultra-rare blood disorder characterized by hemolysis (destruction of red blood cells) that is mediated by an uncontrolled activation of the complement system, a component of the body’s immune system.4,5,16 PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.4,10 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than five years.11 Patients with PNH may experience a wide range of signs and symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia.6-9,12,13 The most devastating consequence of chronic hemolysis is thrombosis, which can occur in blood vessels throughout the body, damage vital organs and cause premature death.17 The first thrombotic event can be fatal.4,17,18 Despite historical supportive care, including transfusion and anticoagulation management, 20 to 35 percent of patients with PNH die within five to 10 years of diagnosis.19,20 Patients with certain types of hemolytic anemia, bone marrow disorders and unexplained venous or arterial thrombosis are at increased risk of PNH.11,21-25
About ULTOMIRIS®
ULTOMIRIS® (ravulizumab) is the first and only long-acting C5 inhibitor administered every eight weeks. It is approved in the EU for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with hemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with SOLIRIS® (eculizumab) for at least the past six months. ULTOMIRIS (ravulizumab-cwvz) is approved in the U.S. and in
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 like PNH, including atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG) and anti-aquaporin-4 (AQP4) auto-antibody-positive neuromyelitis optica spectrum disorder (NMOSD). In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH14 and patients with PNH who had been stable on SOLIRIS® (eculizumab),15 intravenous treatment with ULTOMIRIS every eight weeks demonstrated non-inferiority to intravenous treatment with SOLIRIS every two weeks on all 11 endpoints.
The Phase 3 study of ULTOMIRIS, administered intravenously every eight weeks in adult patients with aHUS, met its primary objective.
ULTOMIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S. and
U.S. Important Safety Information for ULTOMIRIS® (ravulizumab-cwvz)*
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 decided 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. 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 with a stiff neck or stiff back, fever and a rash, muscle aches with flu-like symptoms, headache and fever, fever, confusion, and eyes sensitive to light.
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 healthcare provider. Call the healthcare provider right away if one has any new signs or symptoms of infection.
Before one receives ULTOMIRIS, tell the doctor about all of the medical conditions, including if one: has an infection or fever, is pregnant or plans to become pregnant, and is breastfeeding or plans to breastfeed. It is not known if ULTOMIRIS will harm an unborn baby. It is not known if ULTOMIRIS passes into the breast milk. One should not breast feed during treatment and for 8 months after one’s final dose of ULTOMIRIS.
Tell the doctor about all the medicines one takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ULTOMIRIS and other medicines can affect each other causing side effects. Know the medications one takes and the vaccines one receives. Keep a list of them to show the doctor and pharmacist when one gets a new medicine.
If one stops receiving ULTOMIRIS, the doctor will need to monitor closely for at least 16 weeks after one stops ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of the red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of the red blood cell count, tiredness, blood in the urine, stomach-area (abdomen) pain, blood clots, shortness of breath, trouble swallowing, and erectile dysfunction (ED) in males.
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, or feeling faint. 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 are upper respiratory infection and headache.
For more information, please see the full U.S. Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis, also available at: www.ultomiris.com.
About SOLIRIS® (eculizumab)
SOLIRIS® (eculizumab) is a first-in-class complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade, a part of the immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe rare and ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG), and anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). SOLIRIS is approved in the U.S., EU,
SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU,
U.S. Important Safety Information for SOLIRIS® (eculizumab)*
SOLIRIS is a medicine that affects the immune system. SOLIRIS can lower the ability of the immune system to fight infections. SOLIRIS 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 SOLIRIS if one has not already had this vaccine. If one’s doctor decided that urgent treatment with SOLIRIS is needed, meningococcal vaccination should be administered as soon as possible. If one has not been vaccinated and SOLIRIS 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 SOLIRIS. 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.
SOLIRIS may also increase the risk of other types of serious infections. If one’s child is treated with SOLIRIS, make sure that the child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Certain people may be at risk of serious infections with gonorrhea. Talk to the doctor about whether one is at risk for gonorrhea infection, about gonorrhea prevention, and regular testing. Certain fungal infections (Aspergillus) may also happen if one takes SOLIRIS and has a weak immune system or a low white blood cell count.
Before one receives SOLIRIS, tell the doctor about all of the medical conditions, including if one: has an infection or fever, is pregnant or plans to become pregnant, and is breastfeeding or plans to breastfeed. It is not known if SOLIRIS will harm an unborn baby. It is not known if SOLIRIS passes into the breast milk.
Tell the doctor about all the medicines one takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. SOLIRIS and other medicines can affect each other causing side effects.
It is important that one: has all recommended vaccinations before starting SOLIRIS, receives 2 weeks of antibiotics if one immediately starts SOLIRIS, and stays up-to-date with all recommended vaccinations during treatment with SOLIRIS. Know the medications one takes and the vaccines one receives. Keep a list of them to show the doctor and pharmacist when one gets a new medicine.
If one has PNH, the doctor will need to monitor closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS may cause breakdown of the red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of the red blood cell count, drop in the platelet counts, confusion, kidney problems, blood clots, difficulty breathing, and chest pain.
SOLIRIS can cause serious side effects including serious allergic reactions. Serious allergic reactions can happen during one’s SOLIRIS infusion. Tell the doctor or nurse right away if one gets any of these symptoms during the SOLIRIS infusion: chest pain, trouble breathing or shortness of breath, swelling of the face, tongue, or throat, and feeling faint or pass out. If one has an allergic reaction to SOLIRIS, the doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS. The most common side effects in people with PNH treated with SOLIRIS include: headache, pain or swelling of the nose or throat (nasopharyngitis), back pain, and nausea.
For more information, please see the accompanying full U.S. Prescribing Information and Medication Guide for SOLIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections, also available at: www.soliris.net.
About Alexion
Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases as well as several early-stage therapies, including one for light chain (AL) amyloidosis and a second anti-FcRn therapy. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, and metabolic disorders. Alexion has been named to the Forbes’ list of the World’s Most Innovative Companies seven years in a row and is headquartered in
[ALXN-G]
Forward-Looking Statement
This presentation contains forward-looking statements, including statements related to: the belief that ULTOMIRIS has the potential to become the new standard of care for both complement inhibitor-naïve PNH patients and PNH patients switching from SOLIRIS® (eculizumab); the expectation that PNH patients using ULTOMIRIS can experience greater freedom in their lives; the Company’s goal is to continue to improve the lives of people and families affected by PNH and other serious rare diseases; the Company believes ULTOMIRIS will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained for eight weeks, and reducing the number of infusions per year from 26 with SOLIRIS to only six or seven with ULTOMIRIS; the Company’s plans to make future regulatory filings for approval of certain products and product candidates and the timing of such filings; ULTOMIRIS intravenously administered every eight weeks is a potential treatment for patients with gMG; the Company’s plans for future clinical trials and studies (including plans to initiate a Phase 3 trial for ULTOMIRIS in patients with NMOSD) and the timing for the commencement of future clinical trials; ULTOMIRIS subcutaneously administered once per week is a potential treatment for patients with PNH and aHUS; and the potential benefits of current products and products under development and in clinical trials (including ULTOMIRIS as a treatment for patients with PNH). Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those forward-looking statements, including for example: any potential post-approval restrictions that the
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*U.S. prescribing information included due to issuing country of the release
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Alexion:
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Megan Goulart, 857-338-8634
Senior Director, Corporate Communications
Investors
Susan Altschuller, Ph.D., 857-338-8788
Vice President, Investor Relations