Bayer Announces Phase III Study to Evaluate New Pediatric Formulation of Nifurtimox for Potential Treatment of Chagas Disease

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Nov 15, 2016, 05:00 ET

ATLANTA, Nov. 15, 2016 /PRNewswire/ -- Bayer announced today a Phase III clinical study evaluating the efficacy and safety of a newly developed formulation of the investigational medication Nifurtimox in children of all age groups who have been diagnosed with Chagas disease. The announcement was made at the 65^th annual meeting of the American Society of Tropical Medicine & Hygiene (ASTMH) in Atlanta. Nifurtimox is not approved by the U.S. Food and Drug Administration (FDA) and there are no FDA-approved treatments for children with Chagas disease.

Chagas disease is a potentially life-threatening illness caused by the protozoan parasite Trypanosoma cruzi (T. cruzi). It's estimated more than 300,000 people in the United States are infected with Chagas diseaseⁱ. The pathogen is often transmitted by an infected mother to her newborn during pregnancy or childbirth. Children, therefore account for the majority of new infections. In addition to systematic screening and diagnosis, treatment of infected women of child-bearing age and children is an effective tool in combating the spread of disease.

"As the manufacturer of Nifurtimox, we have a long-term commitment to the fight against this disease," said Dieter Weinand, Member of the Board of Management of Bayer AG and President of Bayer's Pharmaceuticals Division. "Children are the most vulnerable patient group. Every child who can be treated is a step toward getting the disease under control."

Nifurtimox is on the World Health Organization's (WHO) list of "Essential Medicines" for both adults and children. The WHO is distributing Nifurtimox in its efforts to control Chagas disease by 2020. Since 2004, as part of Bayer's commitment to neglected tropical diseases, the company has given the WHO a permanent supply guarantee for Nifurtimox and provided financial assistance to support logistics and drug distribution.

"While children are particularly at risk from the disease, they also have the best chances of being completely cured," said Dr. Pedro Albajar Vinas, Medical Officer for Neglected Tropical Diseases at the World Health Organization. "Therefore, investigating a treatment today is an utmost priority, together with broad-based health education and early diagnosis."

"We regard Chagas as a children's disease," said Dr. Jaime Altcheh, Head of the Department of Parasitology and Chagas disease at the Ricardo Gutierrez Children's Hospital in Buenos Aires. "An infected adult is usually a child that went untreated. Most infections are caused in childhood."

About the Clinical Trial
The CHICO study (Chagas disease In Children treated with NifurtimOx) is a prospective, historically controlled study to evaluate the efficacy, safety and pharmacokinetics of Nifurtimox in children (up to the age of 17) with Chagas disease. The study involves 390 pediatric patients including newborn babies.

Researchers aim to develop an appropriate, weight-adjusted dose of Nifurtimox for newborns and children using fast-disintegrating 30mg and 120mg tablets that can be divided for weight-adjusted dosing in all age groups. The study design is a two-arm study with 30- and 60-day treatment cycles vs. historical untreated control; this is because a placebo-controlled study is not feasible. The study will be conducted at 25 locations in Argentina, Colombia and Bolivia. Patient enrollment began in January 2016.

Chagas disease is spreading beyond Latin America as a result of increased migration and travel. After successful completion of the study, Bayer plans to register Nifurtimox both in disease-endemic and in non-endemic countries with disease burden.

About Nifurtimox
Bayer is the manufacturer of the investigational medicine, Nifurtimox. The product was first introduced in Argentina in 1970, and shortly afterwards in other South American countries.

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) 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.

During the acute phase – immediately after the victim is bitten by the triatomine bug – only a third of victims suffer symptoms, which include fever, fatigue, swollen lymph glands and heart pain for several weeks. The majority of infected people have no symptoms.

Without successful antiparasitic treatment, the subsequent chronic phase becomes a life-long phase during which the infected person can be completely free of any symptoms. By the time Chagas disease symptoms are again noticed, years or even decades later, some organs will have already been irreversibly damaged by the parasite.

One third of people affected develop chronic heart diseases. Others suffer from an abnormal enlargement of the digestive organs or even neurological or mixed alterations. Many die as a result of sudden cardiac arrest or heart failure.

The disease has already been recognized as a global problem: the world's population movements (i.e. mainly migration) have carried the parasite abroad, mainly to North America and Europe. There it can be transmitted either congenitally, to the offspring of infected mothers, or horizontally via blood or organ donations.

Bayer's commitment to Neglected Tropical Diseases (NTD)
Inspired by the World Health Organization's 2020 Roadmap on Neglected Tropical Diseases, the leaders of several prominent global health and development organizations, together with industry partners, met in London in 2012 where they pledged to unite in their efforts on NTDs. Controlling Chagas disease is now one of the goals of this London Declaration on NTDs. Bayer and its engagement to fight Chagas disease, African Sleeping Sickness and River Blindness is part of the London Declaration.

Bayer: Science For A Better Life
Bayer is a global enterprise with core competencies in the Life Science fields of health care and agriculture. Its products and services are designed to benefit people and improve their quality of life. At the same time, the Group aims to create value through innovation, growth and high earning power. Bayer is committed to the principles of sustainable development and to its social and ethical responsibilities as a corporate citizen. In fiscal 2015, the Group employed around 117,000 people and had sales of EUR 46.3 billion. Capital expenditures amounted to EUR 2.6 billion, R&D expenses to EUR 4.3 billion. These figures include those for the high-tech polymers business, which was floated on the stock market as an independent company named Covestro on October 6, 2015.

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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 Centers for Disease Control, Neglected Parasitic Infections in the U.S.; accessed 11.13.16; https://www.cdc.gov/parasites/resources/pdf/npi_chagas.pdf

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