Basel, Switzerland, May 4, 2015
- Basilea Pharmaceutica Ltd. (SIX: BSLN) reported today the start of a
clinical phase 1 study with the orally administered investigational drug
BAL3833/CCT3833 in patients with advanced solid tumors. BAL3833/CCT3833
is the lead compound of a series of small molecule panRAF inhibitors
recently in-licensed by Basilea.
The open-label
dose-escalation phase 1 study is designed to investigate the safety and
tolerability of once-daily oral doses of BAL3833/CCT3833 in adult
patients with advanced solid tumors. The study aims to establish a safe
maximum tolerated dose for future clinical studies.
"A
significant range of cancers are associated with mutations in key
factors involved in the transmission of growth signals, such as the RAF
kinases, leading to uncontrolled tumor growth," said Dr. Laurenz
Kellenberger, Basilea's Chief Scientific Officer. "The BRAF kinase, for
example, is mutated in a range of cancers including 50% of melanomas.
Preclinical data show that our novel panRAF kinase inhibitors are able
to block growth of BRAF mutant melanoma cells in vitro and in vivo.
This includes activity in tumor models resistant to currently marketed
anti-cancer drugs targeting the BRAF pathway. Moreover, as these panRAF
kinase inhibitors also target other growth modulators relied upon by
drug-resistant tumor cells and non-melanoma cancers, it is anticipated
that their spectrum of activity will extend beyond currently available
BRAF inhibitors. This supports the potential for use of these panRAF
inhibitors in the treatment of expanded melanoma patient populations as
well as other cancer types."
Prof. Achim Kaufhold,
Basilea's Chief Medical Officer, said: "The initiation of the first
clinical study with this promising compound is a significant development
milestone and we are very pleased to add BAL3833/CCT3833 as the second
oncology drug in clinical stage to our expanding oncology development
portfolio."
The study is sponsored by The Institute
of Cancer Research, London, and The Royal Marsden NHS Foundation Trust
and is being carried out at The Royal Marsden, and The Christie NHS
Foundation Trust in Manchester, with biomarker work by the Cancer
Research UK Manchester Institute at The University of Manchester and
Basilea. The study will be funded by the Wellcome Trust, the NIHR
Biomedical Research Centre at The Royal Marsden and The Institute of
Cancer Research, The Christie charity and the Cancer Research UK
Manchester Institute. Basilea will assume full operational
responsibility after phase 1.
About BAL3833
BAL3833
(also known as CCT3833) is an orally available small-molecule panRAF
kinase inhibitor targeting certain cell proliferation signaling pathways
that are associated with tumor growth. It is the lead compound of a
series of panRAF inhibitors recently in-licensed by Basilea under an
agreement with The Institute of Cancer Research, London, Cancer Research
Technology, the Wellcome Trust, and The University of Manchester. The
compound originates from research at The Institute of Cancer Research
and the Cancer Research UK Manchester Institute, by scientists funded by
Cancer Research UK and the Wellcome Trust. These panRAF kinase
inhibitors target both BRAF and the growth pathways that tumor cells
rely on when they become resistant to existing drugs targeting BRAF.
BRAF is mutated in a range of cancers including melanomas, colorectal
and serous ovarian cancer. Data from preclinical studies suggest that
compounds from this class are active in tumors derived from patients
which have developed resistance to currently available RAF pathway
inhibitors.[1]
About Wellcome Trust
The
Wellcome Trust is a global charitable foundation dedicated to improving
health. It provides more than GBP 700 million a year to support bright
minds in science, the humanities and the social sciences, as well as
education, public engagement and the application of research to
medicine. Its GBP 18 billion investment portfolio gives it the
independence to support such transformative work as the sequencing and
understanding of the human genome, research that established front-line
drugs for malaria, and Wellcome Collection, its free venue for the
incurably curious that explores medicine, life and art.
About The Institute of Cancer Research, London
The
Institute of Cancer Research, London, is one of the world's most
influential cancer research institutes. Scientists and clinicians at The
Institute of Cancer Research (ICR) are working every day to make a real
impact on cancer patients' lives. Through its unique partnership with
The Royal Marsden NHS Foundation Trust and "bench-to-bedside" approach,
the ICR is able to create and deliver results in a way that other
institutions cannot. Together the two organisations are rated in the top
four cancer centres globally. The ICR has an outstanding record of
achievement dating back more than 100 years. It provided the first
convincing evidence that DNA damage is the basic cause of cancer, laying
the foundation for the now universally accepted idea that cancer is a
genetic disease. Today it leads the world at isolating cancer-related
genes and discovering new targeted drugs for personalized cancer
treatment. As a college of the University of London, the ICR provides
postgraduate higher education of international distinction. It has
charitable status and relies on support from partner organisations,
charities and the general public. The ICR's mission is to make the
discoveries that defeat cancer. For more information visit www.icr.ac.uk
About The Royal Marsden NHS Foundation Trust
The
Royal Marsden opened its doors in 1851 as the world's first hospital
dedicated to cancer diagnosis, treatment, research and education. Today,
together with its academic partner, The Institute of Cancer Research
(ICR), it is the largest and most comprehensive cancer centre in Europe
treating over 50,000 NHS and private patients every year. It is a centre
of excellence with an international reputation for groundbreaking
research and pioneering the very latest in cancer treatments and
technologies. The Royal Marsden, with the ICR, is the only National
Institute for Health Research Biomedical Research Centre for Cancer.
First awarded the status in 2006, it was re-awarded in 2011. A total of
GBP 62 million is being provided over five years, to support pioneering
research work, and is being shared out over eight different cancer
themes. Since 2004, the hospital's charity, The Royal Marsden Cancer
Charity, has helped raise over GBP 100 million to build theatres,
diagnostic centres, and drug development units. For more information
visit www.royalmarsden.nhs.uk
About The University of Manchester
The
University of Manchester, a member of the prestigious Russell Group of
British universities, is the largest and most popular university in the
UK. It has 20 academic schools and hundreds of specialist research
groups undertaking pioneering multi-disciplinary teaching and research
of worldwide significance. The University of Manchester is one of the
country's major research institutions, rated fifth in the UK in terms of
'research power', and has had no fewer than 25 Nobel laureates either
work or study there. The University had an annual income of GBP 886
million in 2013/14. Cancer is one of The University of Manchester's
research beacons - examples of pioneering discoveries, interdisciplinary
collaboration and cross-sector partnerships that are tackling some of
the biggest questions facing the planet. For more information visit www.manchester.ac.uk/research/beacons/cancer/
About Cancer Research UK Manchester Institute
Cancer
Research UK Manchester Institute is a world leading cancer research
institute within The University of Manchester and is core-funded by
Cancer Research UK, the largest independent cancer research organisation
in the world. It is at the heart of the Manchester Cancer Research
Centre, a dynamic partnership between CRUK, The University of Manchester
and The Christie Hospital NHS Foundation Trust that integrates cancer
research across Manchester. Research at CRUK MI spans the whole spectrum
of cancer research, from programmes investigating the molecular and
cellular basis of cancer, to those focused on translational research and
the development of novel therapeutics. Strong links with The Christie
Hospital NHS Foundation Trust, Western Europe's largest specialist
cancer hospital, provide exceptional opportunities for interactions
between basic and clinical research teams, and facilitating rapid
translation of fundamental research findings into patient benefit.
About Cancer Research UK
Cancer
Research UK is the world's leading cancer charity dedicated to saving
lives through research. Cancer Research UK's pioneering work into the
prevention, diagnosis and treatment of cancer has helped save millions
of lives. Cancer Research UK receives no government funding for its
life-saving research. Every step it makes towards beating cancer relies
on every pound donated. Cancer Research UK has been at the heart of the
progress that has already seen survival rates in the UK double in the
last forty years. Today, 2 in 4 people survive cancer. Cancer Research
UK's ambition is to accelerate progress so that 3 in 4 people will
survive cancer within the next 20 years. Cancer Research UK supports
research into all aspects of cancer through the work of over 4,000
scientists, doctors and nurses. Together with its partners and
supporters, Cancer Research UK's vision is to bring forward the day when
all cancers are cured. For further information about Cancer Research
UK's work or to find out how to support the charity, please call +44
(0)300 123 1022 or visit www.cancerresearchuk.org. Follow Cancer Research UK on Twitter and Facebook.
About Cancer Research Technology
Cancer
Research Technology (CRT) is a specialist commercialisation and
development company, which aims to develop new discoveries in cancer
research for the benefit of cancer patients. CRT works closely with
leading international cancer scientists and their institutes to protect
intellectual property arising from their research and to establish links
with commercial partners. CRT facilitates the discovery, development
and marketing of new cancer therapeutics, vaccines, diagnostics and
enabling technologies. CRT is a wholly owned subsidiary of Cancer
Research UK, the world's leading cancer charity dedicated to saving
lives through research. Further information about CRT can be found at www.cancertechnology.com.
About The Christie NHS Foundation Trust
The
Christie specialise in cancer treatment, research and education. It is
the first UK centre to be accredited as a comprehensive cancer centre.
It is the largest single site cancer centre in Europe treating more than
40,000 patients a year. It serves an immediate population of 3.2
million in the Greater Manchester and Cheshire area and also delivers a
number of regional and national services from the main Christie site.
Its patients are at the heart of everything that The Christie does. As a
centre of excellence The Christie focuses solely on improving outcomes
for oncology patients. It is able to provide services based on expert
staff and a specialised infrastructure dedicated to the delivery of
cancer treatment, care, research and education. Its focus and size
enables it to uniquely deliver effective and efficient specialist care
offering patients the best possible outcomes from its research
programme. Its charity, which is one of the largest in the UK, provides
enhanced services over and above what the NHS funds. It has over 30,000
supporters, who helped raise a record breaking GBP 14.8m last year, with
83p in every pound going directly to patients.
About Basilea
Basilea
Pharmaceutica Ltd. is a biopharmaceutical company developing products
that address increasing resistance and non-response to current treatment
options in the therapeutic areas of bacterial infections, fungal
infections and cancer. The company uses the integrated research,
development and commercial operations of its subsidiary Basilea
Pharmaceutica International Ltd. to develop and commercialize innovative
pharmaceutical products to meet the medical needs of patients with
serious and potentially life-threatening conditions. Basilea
Pharmaceutica Ltd. is headquartered in Basel, Switzerland and listed on
the SIX Swiss Exchange (SIX: BSLN). Additional information can be found
at Basilea's website www.basilea.com.
Disclaimer
This
communication expressly or implicitly contains certain forward-looking
statements concerning Basilea Pharmaceutica Ltd. and its business. Such
statements involve certain known and unknown risks, uncertainties and
other factors, which could cause the actual results, financial
condition, performance or achievements of Basilea Pharmaceutica Ltd. to
be materially different from any future results, performance or
achievements expressed or implied by such forward-looking statements.
Basilea Pharmaceutica Ltd. is providing this communication as of this
date and does not undertake to update any forward-looking statements
contained herein as a result of new information, future events or
otherwise.
For further information, please contact:
This press release can be downloaded from www.basilea.com.
References
[1]
M. R. Girotti et al., Paradox-breaking RAF inhibitors that also target
SRC are effective in drug-resistant BRAF mutant melanoma, Cancer Cell
2015 (27), 85-96
