FDA grants orphan drug designation to Boehringer Ingelheim's investigational anti-CD33 monoclonal antibody BI 836858 for treatment of myelodysplastic syndromes

- Myelodysplastic syndromes are caused by changes in bone marrow and, in some cases, may lead to acute myeloid leukemia (AML)

- BI 836858 was previously granted orphan drug designation for the treatment of patients with AML

RIDGEFIELD, Conn., March 9, 2017 /PRNewswire/ -- Boehringer Ingelheim today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its anti-CD33 monoclonal antibody BI 836858 for the treatment of myelodysplastic syndromes (MDS). Orphan drug designation is granted by the FDA to investigational compounds intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people.

MDS are a group of bone marrow disorders. The bone marrow produces blood cells. In MDS, the bone marrow is characterized by cell abnormalities and ineffective blood cell production. Various subtypes of the disease exist with variable prognoses and treatment options. In some cases, MDS can progress to acute myeloid leukemia (AML), an aggressive and devastating blood cancer. According to data from the National Cancer Institute, an estimated 15,000 new cases of MDS are identified each year.

"Preclinical studies with our anti-CD33 monoclonal antibody have shown promising therapeutic potential in myelodysplastic syndromes, a rare group of bone marrow disorders," said Martina Flammer, M.D., Vice President, Clinical Development & Medical Affairs Specialty Care, Boehringer Ingelheim. "Orphan drug designation for our investigational candidate in MDS is an important recognition of our ongoing research efforts and underscores Boehringer Ingelheim's commitment to studying treatment options for rare cancers."

A Phase I/II multi-center, open-label, dose escalation and randomized trial (NCT02240706) evaluating BI 836858 in patients with MDS is ongoing. BI 836858 previously received orphan drug designation for the treatment of patients with AML and is currently being evaluated as part of the Leukemia & Lymphoma Society's (LLS) groundbreaking, first-of-its-kind Beat AML Master trial program to advance treatment for patients with AML. Using the latest genomic technology, the trial finds and matches specific AML mutations in newly-diagnosed patients over the age of 60 with an investigational drug or drugs best suited to attack the specific genetic mutations found within the cancer.

About BI 836858
BI 836858 is a monoclonal antibody that targets a protein called CD33, which is expressed on the surface of certain cancerous cells. BI 836858 has been engineered for improved antibody-dependent cell-mediated cytotoxicity (ADCC), a type of immune reaction in which a target cell or microbe is coated with antibodies and killed by certain types of white blood cells. Preclinical studies with the compound have shown promising ADCC against malignant cells.

About Boehringer Ingelheim in Oncology
Boehringer Ingelheim's oncology research is driven by a passion to advance clinical practice and a determination to improve the lives of patients who are battling cancer. Through our own scientific innovation and partnerships, we are focused on discovering and providing novel best-in-class, breakthrough cancer medications that fit the needs of patients, caregivers and healthcare professionals. We have a clear strategy to become a leader in the field of lung cancer. Boehringer Ingelheim has successfully launched two products globally for NSCLC that have been widely adopted and established as valuable additions to current clinical practice. Continuous insights and learnings from research and development are key parts of innovation and our way forward to advance clinical practice in lung cancer and other cancer types.

About Boehringer Ingelheim Pharmaceuticals, Inc.
Boehringer Ingelheim Pharmaceuticals, Inc., based in Ridgefield, CT, is the largest U.S. subsidiary of Boehringer Ingelheim Corporation.

Boehringer Ingelheim is one of the world's 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, the company operates globally with 145 affiliates and about 50,000 employees. Since its founding in 1885, the family-owned company has been committed to researching, developing, manufacturing and marketing novel treatments for human and veterinary medicine.

Boehringer Ingelheim is committed to improving lives and providing valuable services and support to patients and their families. Our employees create and engage in programs that strengthen our communities. To learn more about how we make more health for more people, visit our Corporate Social Responsibility Report.

In 2015, Boehringer Ingelheim achieved net sales of about $15.8 billion (14.8 billion euros). R&D expenditure corresponds to 20.3 percent of its net sales.

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SOURCE Boehringer Ingelheim Pharmaceuticals, Inc.

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