Draft NICE Guidance for Aggressive non-Hodgkin Lymphoma Treatment, PIXUVRI®
SEATTLE, April 10, 2013
SEATTLE, April 10, 2013 /PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (NASDAQ
and MTA: CTIC) today announced that the National Institute for Health and Care
Excellence (NICE), a non-departmental public body of the Department of Health
in the United Kingdom, issued draft guidance on PIXUVRI^® (pixantrone) as a
monotherapy for the treatment of adult patients with multiply relapsed or
refractory aggressiveB-cell non-Hodgkin lymphoma (aggressive B-cell NHL).
The draft guidance does not recommend funding of pixantrone (PIXUVRI) by the
UK's National Health Service (NHS) and requests that consultees, including
CTI, healthcare professionals and members of the public, comment on the
preliminary recommendations, which are available to view on the NICE website.
The consultation will be open until May 1, 2013, and any comments received
will be fully considered by the independent committee of experts during the
next stage of guidance development.
In May 2012, the European Commission (EC) granted conditional marketing
authorization in the European Union (E.U.) for PIXUVRI as a monotherapy for
adult patients with multiply relapsed or refractory aggressive B-cell NHL
based on the results of the EXTEND, or PIX301, pivotal randomized Phase 3
clinical trial. PIXUVRI was made available to patients in eight countries in
the European Union in the fourth quarter of 2012, and some patients in other
countries have already started to receive the treatment. Prior to the
approval of PIXUVRI in the E.U., there were no approved agents or standard of
care in this disease. The PIX301 trial was designed utilizing agents in the
comparator arm that have anti-tumor activity in relapsed disease and are
typically employed as palliative therapy for these patients.
"We look forward to working with NICE to bring PIXUVRI to what we believe is
an unserved patient population for whom PIXUVRI represents the first
pharmaceutical product approved for patients with aggressive B-cell NHL in the
E.U. in over 25 years," said James A. Bianco, M.D., President and CEO of CTI.
"The overall data from the Phase 3 PIX301 clinical trial demonstrated a
favorable benefit-to-risk ratio for a disease where there is a clear unmet
medical need and we believe this was confirmed by the European Commission in
May 2012 with its decision to grant conditional marketing authorization in the
E.U. for PIXUVRI. We believe strong support among UK lymphoma experts and the
data in the labeled population of patients with aggressive B-cell NHL who
failed 2 or 3 prior lines of therapy demonstrates PIXUVRI is a cost-effective
therapy for this end-of-life disease population."
About PIXUVRI (pixantrone)
PIXUVRI is a novel aza-anthracenedione with unique structural and
physiochemical properties. Unlike related compounds,PIXUVRI forms stable DNA
adducts and in preclinical models has superior anti-lymphoma activity compared
to related compounds. PIXUVRI was structurally designed so that it cannot
bind iron and perpetuate oxygen radical production or form a long-lived
hydroxyl metabolite -- both of which are the putative mechanisms for
anthracycline induced acute and chronic cardiotoxicity. These novel
pharmacologic properties allow PIXUVRI to be administered to patients with
near maximal lifetime exposure to anthracyclines without unacceptable rates of
InMay 2012, the European Commission (EC) granted conditional marketing
authorization for PIXUVRI as a monotherapy for the treatment of adult patients
with multiply relapsed or refractory aggressiveNHL. The benefit of PIXUVRI
treatment has not been established in patients when used as fifth line or
greater chemotherapy in patients who are refractory to last therapy. The
Summary of Product Characteristics (SmPC) has the full prescribing
information, including the safety and efficacy profile of PIXUVRI in the
approved indication. TheSmPCis available atwww.pixuvri.eu.
CTI is currently accruing patients into a Phase 3 trial comparing PIXUVRI and
rituximab with gemcitabine and rituximab in the setting of aggressive B-cell
NHL. PIXUVRI does not have marketing approval inthe United States.
About Non-Hodgkin Lymphoma
Non-Hodgkin lymphoma is the sixth most common cancer in the UK; in 2010,
12,180 people were diagnosed with the disease.^1 NHL is caused by the abnormal
proliferation of lymphocytes, cells that are key to the functioning of the
immune system. It usually originates in lymph nodes and spreads through the
lymphatic system. NHL can be broadly classified into two main
forms—aggressive and indolent NHL. Aggressive NHL is a rapidly growing form
of the disease that moves into advanced stages much faster than indolent NHL,
which progresses more slowly.
There are many subtypes of NHL, but aggressive B-cell NHL is the most common
and accounts for about 50 percent of NHL cases.^2 After initial therapy for
aggressive NHL with anthracycline-based combination therapy, one-third of
patients typically develop progressive disease.^3 Approximately half of these
patients are likely to be eligible for intensive second-line treatment and
stem cell transplantation, although 50 percent are expected not to respond.^3
For those patients who fail to respond or relapse following second-line
treatment, treatment options are limited, and usually palliative only.^3
About Conditional Marketing Authorization
Similar to accelerated approval regulations intheUnited States, conditional
marketing authorizations are granted in the E.U. to medicinal products with a
positive benefit/risk assessmentthat address unmet medical needs and whose
availability would result in a significant public health benefit. A
conditional marketing authorization is renewable annually. Under the
provisions of the conditional marketing authorization for PIXUVRI, CTI will be
required to complete a post-marketing study aimed at confirming the clinical
benefit previously observed.
The European Medicines Agency's (the EMA)Committee for Medicinal Products for
Human Usehas accepted PIX306, CTI's ongoing randomized controlled Phase 3
clinical trial, which compares PIXUVRI-rituximab to gemcitabine-rituximab in
patients who have relapsed after one to three prior regimens for aggressive
B‑cellNHLand who are not eligible for autologous stem cell transplant. As a
condition of approval, CTI has agreed to have available the PIX306 clinical
trial results byJune 2015.
About Cell Therapeutics, Inc.
CTI (NASDAQ and MTA: CTIC) is a biopharmaceutical company committed to the
development and commercialization of an integrated portfolio of oncology
products aimed at making cancer more treatable. CTI is headquartered in
Seattle, WA. For additional information and to sign up for email alerts and
get RSS feeds, please visit www.CellTherapeutics.com.
This press release includes forward-looking statements that involve a number
of risks and uncertainties, the outcome of which could materially and/or
adversely affect actual future results and the market price of CTI's
securities. Specifically, the risks and uncertainties that could affect the
development of PIXUVRI include risks associated with preclinical and clinical
developments in the biopharmaceutical industry in general and with PIXUVRI in
particular including, without limitation, that NICE may not determine that
PIXUVRI provides an additional benefit; that NICE's draft guidance may not
change even if comments are received during the comment period; that the NICE
decision regarding PIXUVRI may not be made in the second quarter of 2013; that
the NICE decision may have an impact on the reimbursement of PIXUVRI in the
United Kingdom and other countries in the E.U.; that CTI may not obtain
reimbursement in certain markets in the European Union as planned; that CTI
may not be able to complete the PIX306 clinical trial of PIXUVRI-rituximab
compared to gemcitabine-rituximab in patients who have relapsed after 1 to 3
prior regimens for aggressive B-cellNHL and who are not eligible for
autologous stem cell transplant byJune 2015or at all as required by the EMA
or have the results of such clinical trial available byJune 2015or at all;
that CTI may not be able complete a post-marketing study aimed at confirming
the clinical benefit observed in the PIX301 trial; that results in future
studies of PIXUVRI may differ from the results of past studies; that the
conditional marketing authorization for PIXUVRI may not be renewed; that CTI
cannot predict or guarantee the pace or geography of enrollment of its
clinical trials or the total number of patients enrolled; that CTI's average
net operating burn rate may increase; CTI's may not be able to continue to
raise capital as needed to fund its operations in general, and other risks,
including, without limitation, competitive factors, technological
developments, costs of developing, producing, and selling PIXUVRI, and the
risk factors listed or described from time to time in CTI's filings with
theSecurities and Exchange Commissionincluding, without limitation, CTI's
most recent filings on Forms 10-K, 10-Q and 8-K. Except as may be required by
law, CTI does not intend to update or alter its forward-looking statements
whether as a result of new information, future events, or otherwise.
PIXUVRI is a registered trademark of Cell Therapeutics, Inc.
1. Cancer Research UK
Accessed April 2013.
2. Harris NL, et al. Ann Oncol. 1999;10(12):1419-32
3. Friedberg ASH Education Book 2011;1:498-505
CTI Life Sciences Limited, Milan Branch
Laura Villa or Elena Bellacicca
T: +39 02 89659700
SOURCE Cell Therapeutics, Inc.
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