Second Draft NICE Guidance for Aggressive non-Hodgkin Lymphoma Treatment, PIXUVRI® (pixantrone)

  Second Draft NICE Guidance for Aggressive non-Hodgkin Lymphoma Treatment,
                            PIXUVRI® (pixantrone)

PR Newswire

SEATTLE, July 1, 2013

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SEATTLE, July 1, 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in the United Kingdom, issued a second draft guidance on the use of PIXUVRI^®
(pixantrone) as a monotherapy for the treatment of adult patients with
multiply relapsed or refractory aggressiveB-cell non-Hodgkin lymphoma
(patients with aggressive B-cell NHL who failed 2 or 3 prior lines of
therapy). The draft final appraisal document (FAD) does not recommend funding
of pixantrone (PIXUVRI) by the UK's National Health Service (NHS). The FAD is
open to appeal until July 11, 2013, and any appeal received will be considered
by the independent committee of experts during the next stage of guidance
development, in accordance with the process set out by NICE. In addition, CTI
in coordination with NICE's Patient Access Scheme Liaison Unit (PASLU) has
reached agreement with the UK's Department of Health on a patient access
scheme and is applying for rapid reappraisal by NICE. These schemes involve
innovative pricing agreements designed to improve cost effectiveness and
facilitate patient access to specific drugs or other technologies.

"Our intention is to continue to work with NICE to provide support for PIXUVRI
as an important therapy for what we believe is an unserved patient population
and for whom PIXUVRI represents the first pharmaceutical product approved
specifically for patients with aggressive B-cell NHL who have failed 2 or 3
prior lines of therapy," said James A. Bianco, M.D., President and CEO of CTI.
"The overall data from the pivotal Phase 3 PIX301 clinical trial demonstrated
a favorable benefit-to-risk ratio. We believe continued support among UK
lymphoma experts, the data in the labeled population of patients with
aggressive B-cell NHL who failed 2 or 3 prior lines of therapy, and the recent
approval of a patient access scheme by the Department of Health in England and
Wales demonstrates our commitment to bringing this new approved therapy to
patients with aggressive NHL in the 3^rd line salvage setting." 

"There are no other licensed products for use in the 3^rd or 4th line," said
Professor Finbarr E. Cotter, Professor of Haematology and Chair of
Experimental Haematology, Centre for Haemato-Oncology, Barts Cancer Institute.
"As a representative for the British Society for Haematology, I feel it is
important for NICE to approve pixantrone to make it available with clear
guidance for its licensed indication only by haemato-oncologists in England
for the small number of unfortunate patients when the opportunity for
potentially curative stem cell transplantation is still a possibility if a
complete response is achieved".

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.

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
cardiotoxicity.

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.

Forward-Looking Statements

This press release includes forward-looking statements that are made pursuant
to the Safe Harbor provisions of the Private Securities Litigation Reform Act
of 1995, including, but not limited to, statements regarding the Company's
intentions and expectations regarding NICE's processes, pricing arrangements
and the development of PIXUVRI and the Company in general. Such statements
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 these matters 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; 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; that CTI 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.

References:

1. Cancer Research UK
http://www.cancerresearchuk.org/cancer-info/cancerstats/incidence/commoncancers/
Accessed April 2013.
2. Harris NL, et al. Ann Oncol. 1999;10(12):1419-32
3. Friedberg ASH Education Book 2011;1:498-505

Contacts:

Monique Greer
+1 206-272-4343
mgreer@ctiseattle.com

Ed Bell
+1 206.282.7100
ebell@ctiseattle.com

In Europe

CTI Life Sciences Limited, Milan Branch
Laura Villa
E: lvilla@cti-lifesciences.com
T: +39 02 89659706
http://www.celltherapeutics.com/italiano

SOURCE Cell Therapeutics, Inc.

Website: http://www.celltherapeutics.com
 
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