Value through Innovation26 October 2014
17 September 2013

Volasertib* receives FDA Breakthrough Therapy designation for treatment of patients with acute myeloid leukaemia

Administration (FDA) to potentially provide a new treatment option to improve survival related outcomes for patients with acute myeloid leukaemia (AML)

Ingelheim, Germany, 17 September, 2013 – Boehringer Ingelheim is pleased to announce that the FDA has granted a Breakthrough Therapy designation to volasertib*, a selective and potent polo-like kinase (Plk) inhibitor, for the treatment of patients with AML, a type of blood cancer.

Professor Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim

Professor Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim

"Volasertib"s* innovative mode of action offers a new approach and may potentially provide a new therapy option for AML patients who have a high unmet medical need," said Professor Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim. "We are looking forward to exploring opportunities with the FDA to expedite the final stage of the development of volasertib* with the aim of making this treatment available for patients."

The Breakthrough Therapy designation pathway was launched by the US Food and Drug Administration (FDA) in 2012, and is intended for any drug that "treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies."1

A Phase II study in patients with previously untreated AML ineligible for intensive therapy compared volasertib* in combination with the established therapy of low-dose cytarabine (LDAC) versus LDAC alone.2 The primary endpoint for the Phase II study was objective response. Objective responses were observed in 31 percent of patients (13 of 42 patients) treated with the combination of volasertib* plus LDAC compared to 13.3 percent of the patients (6 of 45 patients) treated with LDAC alone (p = 0.0523).

Secondary endpoints included event-free survival (EFS), overall survival (OS) and safety. A trend for OS benefit (8.0 months for the volasertib* combination compared to 5.2 months for LDAC alone, p = 0.0996) was observed. EFS was measured from the date of randomisation to the date of disease progression (treatment failure), relapse or death from any cause, whichever occurred first. In patients treated with the combination of volasertib* plus LDAC, the median EFS was 5.6 months compared to 2.3 months in patients treated with LDAC alone (hazard ratio: 0.56; 95% CI: 0.34, .93; p=0.0237).

These encouraging results led to the initiation of the Phase III study, POLO-AML-2, investigating volasertib* in combination with LDAC, in patients aged 65 years and above with previously untreated AML who are ineligible for intensive remission induction therapy.

"The most common treatment approach for AML is intensive remission induction therapy; however, many older patients are ineligible for this approach which involves high doses of chemotherapy which these patients are often unable to tolerate," said Professor Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim, "These patients are in particular need of new treatments and we hope that volasertib* may be able to fill this gap by providing a tolerable option that improves survival related outcomes."

Notes to Editors

About the POLO-AML-2 Study
For additional information on the trial, please visit http://clinicaltrials.gov./ct2/show/NCT01721876?term=Volasertib&rank=4

About volasertib*
Volasertib* is a selective and potent Plk inhibitor, is currently being evaluated in clinical trials for AML and is one of several late-stage compounds that Boehringer Ingelheim is currently evaluating in clinical trials for cancer.

Of the company's ongoing research in cell cycle inhibition, the volasertib* clinical development programme is the furthest advanced. Boehringer Ingelheim is one of the first companies to advance Plk inhibitors into clinical development.

Volasertib* is designed to inhibit the activity of Plk. Plk1 – the
best characterised of the five known human Plks – is an enzyme that regulates cell division (mitosis). This inhibition results in cell cycle arrest and ultimately cell death (apoptosis).

About Acute Myeloid Leukaemia (AML)
Leukaemia is a rare cancer of the bone marrow and blood. AML is one of the most common types of leukaemia in adults, accounting for approximately one third of all adult leukaemias in the Western world.4 AML has one of the lowest survival rates of all leukaemias.5

AML predominantly occurs in older adults; the average age of newly diagnosed patients is 65 year.5 In AML patients the prognosis worsens with increasing age, with a median survival of six months or less following diagnosis in older patients who are often ineligible for intensive remission induction therapy (the most common treatment approach for younger patients). This involves high doses of chemotherapy which older patients often are unable to tolerate.5

About Boehringer Ingelheim in Oncology
Building on scientific expertise and excellence in the fields of pulmonary and cardiovascular medicine, metabolic disease, neurology, virology and immunology, Boehringer Ingelheim has embarked on a major research programme to develop innovative cancer drugs. Working in close collaboration with the international scientific community and a number of the world’s leading cancer centres, Boehringer Ingelheim’s commitment to oncology is underpinned by using advances in science to develop a range of targeted therapies for various solid tumours and haematological cancers.

The current focus of research includes compounds in three areas: angiogenesis inhibition, signal transduction inhibition and cell-cycle kinase inhibition. Volasertib* is the most advanced pipeline compound for haematological cancers. The pipeline for haematological cancers also includes two new biological entities (NBEs) using an immunotherapeutic approach: BI 836858 is an anti-CD33 antibody targeting AML and BI 836826 is an anti-CD37 antibody targeting chronic lymphocytic leukemia (CLL) and B-cell non-Hodgkin's lymphoma (B-NHL).

Boehringer Ingelheim's oncology pipeline is evolving and demonstrates the company's continued commitment to advance the disease area.

Boehringer Ingelheim
The Boehringer Ingelheim group is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, it operates globally with 140 affiliates and more than 46,000 employees. Since it was founded in 1885, the family-owned company has been committed to researching, developing, manufacturing and marketing novel medications of high therapeutic value for human and veterinary medicine.

Social responsibility is a central element of Boehringer Ingelheim's culture. Involvement in social projects, caring for employees and their families, and providing equal opportunities for all employees form the foundation of the global operations. Mutual cooperation and respect, as well as environmental protection and sustainability are intrinsic factors in all of Boehringer Ingelheim’s endeavours.

In 2012, Boehringer Ingelheim achieved net sales of about 14.7 billion euro. R&D expenditure in the business area Prescription Medicines corresponds to 22.5% of its net sales.

For more information please visit:
www.boehringer-ingelheim.com and www.newshome.com

References
1FDA Frequently Asked Questions: Breakthrough Therapies. http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/ucm341027.htm [Last accessed: June 2013]
2Maertens J, et al. Phase I/II study of volasertib (BI 6727), an intravenous Polo-like kinase (Plk) inhibitor, in patients with acute myeloid leukemia (AML): results from the randomized phase II part for volasertib in combination with low-dose cytarabine (LDAC) versus LDAC monotherapy in patients with previously untreated AML ineligible for intensive treatment. Oral Presentation at ASH Annual Meeting and Exposition 2012
3Schöffski P. Polo-Like Kinase (PLK) Inhibitors in Preclinical and Early Clinical Development in Oncology. Oncologist. 2009;14(6):559-570
4Jemal A, et al. Cancer Statistics, 2009; CA Cancer J Clin 2009;59:225-249
5Deschler B et al. Acute Myeloid Leukemia: Epidemiology and Etiology. Cancer. 2006;107(9):2099-107

*Volasertib is an investigational compound and is not yet approved. Its safety and efficacy have not yet been fully established.

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