Bayer's Phase III Clinical Trial Of Nifurtimox In Pediatric Patients With Chagas Disease Meets Primary Endpoint

Largest clinical treatment study of 330 pediatric patients with Chagas disease

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Mar 14, 2019, 14:00 ET

WHIPPANY, N.J., March 14, 2019 /PRNewswire/ -- Bayer presented results today from the CHICO part (CHagas disease In Children treated with NifurtimOx) of the phase III clinical study of investigational nifurtimox in pediatric patients with Chagas disease at the "XV Taller sobre la enfermedad de Chagas" conference in Barcelona, Spain. The study met its primary endpoint, which was the serological response at one year after end of treatment, by demonstrating superiority of the 60-day nifurtimox treatment compared with historical placebo control. Safety and efficacy were evaluated throughout the clinical trial.

CHICO was the first part of a prospective, randomized (to dosing regimen), double-blind, historically placebo controlled phase III trial to evaluate the efficacy, safety, and pharmacokinetics of nifurtimox in 330 pediatric patients with acute or chronic Chagas disease. The study was conducted at 25 investigational sites in Argentina, Bolivia and Colombia between 2016 and 2018.

Chagas is an infectious disease caused by a parasite (Trypanosoma cruzi) and transmitted to humans via the feces of triatomines, also known as kissing bugs. Efforts to control Chagas disease include eliminating transmission, controlling the vector, treating girls and women of childbearing age (to avoid transplacental transmission), and testing blood prior to transfusion. In addition, the aim is to make healthcare access available for the infected population at all stages of the disease.

According to the World Health Organization, 8 million people are infected with Chagas disease worldwide, mainly in Latin America where the condition remains one of the biggest public health problems, causing incapacity in infected individuals and more than 10,000 deaths per year.^ⁱ Chagas disease is also a growing health concern in the U.S. with an estimated 300,000 people who are infected with T. cruzi, according to the Centers for Disease Control and Prevention (CDC).

"Children are the most vulnerable patient group suffering from Chagas disease. With the successful completion of the CHICO study, an important milestone in our long-standing commitment to improve the treatment of Chagas has been achieved," said Dr. Joerg Moeller, Member of the Executive Committee of Bayer AG´s Pharmaceutical Division and Head of Research and Development.

The treatment of Chagas disease is based on only two nitroheterocyclic compounds, nifurtimox and benznidazole. Nifurtimox is not approved by the U.S. Food and Drug Administration (FDA), but is currently available in the U.S. as a 120 mg tablet exclusively through the CDC under an investigational protocol. For the CHICO trial, Bayer developed a new formulation of both the 30 mg and 120 mg tablets, which can be dissolved in water to form a slurry when administered to pediatric patients. The new formulations were developed to allow for dosing accuracy and administration to those who have difficulty swallowing tablets, especially young children.

"An adequate dispersable formulation of nifurtimox is a big step forward toward achieving the goal of treating all infected children," said Dr. Jaime Altcheh, Head of the Department of Parasitology and Chagas disease at the Ricardo Gutierrez Children's Hospital in Buenos Aires, Argentina, and Coordinating Investigator of the phase III CHICO trial. "Early treatment after infection is very important to prevent manifestation of the disease in adulthood."

Today less than 1% of people infected with Chagas disease worldwide are treated due to low disease awareness and limited access to treatment. Bayer is committed to improve access to nifurtimox by expanding the number of countries where it is currently approved and making the new formulations available in countries with high disease burden.

Nifurtimox is registered in several Latin American countries (Argentina, Uruguay, Chile, El Salvador, Guatemala, and Honduras) for treatment of acute and chronic Chagas disease in adults and children.

About Chagas Disease

Chagas disease is named after Carlos Ribeiro Justiniano Chagas, a Brazilian physician and researcher who discovered the disease in 1909. Chagas is an infectious disease caused by single-cell parasites (Trypanosoma cruzi) which are transmitted to humans via the feces/urine of kissing bugs or triatomines. Chagas disease can also be transmitted by infected blood transfusions, organ transplantation, or during pregnancy or birth from the infected mother to her unborn child.

Infected patients are mainly present in endemic countries of Latin America. However, due to local and transnational migration, the disease has extended with exceptional or no vectorial transmission to other countries.

During the acute phase of the disease – immediately after the infection occurs – only a third of victims suffer non-specific symptoms, which include fever, fatigue, swollen lymph glands for several weeks; the majority of infected patients are unaware.

Without successful antiparasitic treatment, the disease evolves into a chronic lifelong phase, which is asymptomatic for the majority of patients. About 30% of patients develop serious irreversible complications decades after the infection. The chronic complications include enlarged heart (cardiomyopathy) and other heart conditions, abnormal enlargement of the digestive tract or even neurological or mixed conditions. Many die as a result of sudden cardiac arrest or heart failure.

Bayer and Chagas disease

Although one quarter of the Latin American population is at risk of contracting the disease, Chagas is still called a "forgotten disease", but even more it is a "silent disease" as it remains "hidden" for years or even decades due to lack of specific symptoms. For a long time there were no means to treat Chagas disease effectively. Nifurtimox was discovered by Bayer in Wuppertal, Germany. The product was first introduced as Lampit® (nifurtimox) in Argentina in the 1970s, and shortly afterwards also in other Latin American countries.

In 2002, the World Health Organization (WHO) and Bayer collaborated in the fight against Chagas disease. Since then, Bayer has been providing the WHO nifurtimox free of charge, as well as financial resources for logistics and distribution, recently also for awareness, education and training as well as surveillance activities. Nifurtimox is on the WHO's lists of "Essential Medicines" for both adults and children.

As the originator of nifurtimox and as a signatory of the 'London Declaration on NTDs of 2012, Bayer is committed to making the drug available for patients until Chagas disease is eliminated. The fight against Chagas disease, a neglected tropical disease (NTD), poses a special challenge, as it has turned from an endemic disease in Latin America to a global public health threat. The control of the disease by screening programs, early diagnosis, access to treatment and vector control are important measures on the path of eliminating the disease by 2030.

A prerequisite to achieve this goal is that all stakeholders in an aligned approach join efforts and contributions. This was concluded by all partners at the first Chagas Stakeholder Meetings with PAHO and WHO in 2018. Bayer is a dedicated partner in this network. The decision to joining the Global Chagas Coalition in 2018 was a clear endorsement of the company's special emphasis to fight Chagas disease.

Bayer: Science For A Better Life

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros. For more information, go to www.bayer.us.

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Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports, which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

^ⁱ World Health Organization; chagas facts; https://www.who.int/chagas/epidemiology/en/

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