With the enrollment of the first patient, Boehringer Ingelheim is
pleased to announce on international Rare Disease Day, the initiation of
a Phase III study (POLO-AML-2) investigating volasertib*, a selective
and potent polo-like kinase (Plk) inhibitor, in combination with
chemotherapy, in patients with acute myeloid leukaemia (AML) ineligible
for intensive therapy.
Acute leukaemias are rare diseases, with AML being the most deadly acute leukaemia in adults.1 28 February, 2013 marked the sixth international Rare Disease Day, and more than 60
countries around the world have joined to raise awareness for those
affected by rare diseases. In Europe, rare disease is defined as a
life-threatening or chronically debilitating disease which affects fewer
than five people per 10, 000.2
"Rare diseases are often incorrectly diagnosed
and even once they are correctly diagnosed, there is often a lack of
viable treatment options" said Professor Klaus Dugi, Corporate Senior
Vice President Medicine, Boehringer Ingelheim. "The initiation of the
POLO-AML-2 trial is a significant milestone as therapeutic options are
limited in AML patients ineligible for intensive therapy."
AML is characterised by the rapid proliferation
of abnormal blood precursor cells that accumulate in the bone marrow and
interfere with the production of normal blood cells. The primary
endpoint of POLO-AML-2 is objective response to the combination
treatment compared to the chemotherapy alone. The main secondary
endpoint of POLO-AML-2 is overall survival.
The study was initiated following positive
results from a Phase II study which demonstrated higher rates of
objective response and an improvement in event free survival in patients
receiving volasertib* in combination with chemotherapy versus
"Boehringer Ingelheim is committed
to developing innovative medications that improve patients’ lives and
has put considerable effort into research and development of treatments
for orphan† diseases." said Professor Klaus Dugi, Corporate Senior Vice
President Medicine, Boehringer Ingelheim.
In addition to AML, Boehringer Ingelheim is
investigating therapeutic approaches for other rare diseases including
idiopathic pulmonary fibrosis (IPF). IPF is a severely debilitating and
fatal respiratory disease. It is characterised by progressive loss of
lung function ultimately leading to the death of half of the patient
population two to three years after diagnosis.
Nintedanib*, a small molecule tyrosine kinase
inhibitor (TKI), targets growth factor receptors which have been shown
to be potentially involved in the pathomechanism of pulmonary fibrosis.
The pivotal INPULSISTM-1 and INPULSISTM-2 Phase
III trials have completed recruitment and are ongoing in study centres
worldwide to assess the clinical outcomes in IPF patients treated with
nintedanib*. The Phase III INPULSIS trials aim to build upon the
promising results of the Phase II TOMORROW trial, which demonstrated a
positive trend in reducing lung function decline in IPF patients treated
with 150 mg of nintedanib* twice daily when compared to placebo.4
Additionally, nintedanib* has received orphan-drug designation from the
U.S. Food and Drug Administration in June 2011 and by the Ministry of
Health, Labour and Welfare of Japan in September 2011.
- Deschler B, et al. Acute Myeloid Leukemia: Epidemiology and Etiology. Cancer. 2006. 2009-2107
- European Commission. Available at: http://ec.europa.eu/health/ph_information/documents/ev20040705_rd05_en.pdf. [Last accessed: February 2013]
- Döhner H, 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
- Richeldi L, et al. Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis. N Engl J Med. 2011;365(12):1079-87
Source: Boehringer Ingelheim