Market Overview

IBD

According to the Crohn’s & Colitis Foundation of America, there were an estimated 1.6 million IBD patients in the United States in 2013, an increase of approximately 200,000 patients since 2011. As many as 70,000 new cases of IBD are diagnosed in the United States each year. As of 2008, annual direct treatment costs for patients with IBD in the United States were estimated to exceed $6.3 billion, while indirect costs such as missed work days were estimated to cost an additional $5.5 billion.

In 2015, GlobalData estimated that the UC market reached approximately $4.8 billion across seven major markets: United States, France, Germany, Italy, Spain, United Kingdom and Japan and is expected to increase at a compound annual growth rate of approximately 1.3% to $5.5 billion by 2025. In 2016, GlobalData estimated that the CD market reached approximately $9.2 billion, across those same seven major markets and is expected to grow approximately 3.8% per year to $13.4 billion by 2026.

The current tumor necrosis factor-alpha (TNF-α) antibody drugs approved for moderate-to-severe IBD, Humira® and Remicade®, are both injectable. According to GlobalData, the 2013 sales for Humira and Remicade for IBD were $3.4 billion in the United States. Takeda Pharmaceuticals reported 2016 fiscal year sales for Entyvio® for IBD of ¥143.2 billion JPY (approximately US $1.3 billion). Like Entyvio, which is an injectable antibody drug, the oral peptide PTG-100 inhibits alpha-4-beta-7 integrin.

Approximately one third of IBD patients are nonresponders to TNF-α antibody drugs and approximately another 30% to 40% become refractory within the first year of treatment. Additionally, TNF-α antibody drugs may predispose patients to an increased risk of serious infection and the development of anti-drug antibodies (ADAs), which over time can cause loss of drug response. Thus, while available treatments exist for moderate-to-severe IBD, there continues to be a significant medical need for efficacious, safer, and convenient treatments.

β-Thalassemia

Beta-Thalassemia is a rare genetic blood disorder that is characterized by impaired red blood cell production that can result in life-threatening chronic anemia. Patients usually require regular and life-long blood transfusions for survival which can lead to iron overload in target organs such as the heart and liver.

β-Thalassemia results from mutations in the HBB gene, which holds instructions for making beta-globin, an essential part of hemoglobin. Depending on the mutation, affected individuals have either a partial or complete reduction in beta-globin, causing red blood cell damage in turn leading to ineffective erythropoiesis and chronic anemia. β-Thalassemia is classified into two subtypes; transfusion-dependent thalassemia and non-transfusion dependent thalassemia.

β-Thalassemia is most prevalent in people of Mediterranean descent, such as Italians, Greeks, Turkish and is also found in the Arabian Peninsula, Iran, Africa, Southeast Asia and southern China. The prevalence of β-Thalassemia was estimated to be approximately 300,000 patients worldwide in 2008, with at least 60,000 patients born each year with the disease, according to the CDC. In 2018, Decision Resource Group reported that while β-Thalassemia has a worldwide carrier rate of 1.5%, the disease is rare in the US, Italy, Germany, UK, Spain, and France with a total diagnosed prevalence of 16,307. The prevalence in the US is low, with an estimated 3,000 patients and approximately 300 patients born each year with the disease.

The greatest unmet need for β-Thalassemia is for more effective treatment for chronic anemia to decrease the burden of frequent blood transfusions and thus eliminate complications associated with the disease and its management and costs associated with RBC transfusions.

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