German Federal Joint Committee (G-BA) Issues Final Assessment Report for PIXUVRI® (pixantrone)

   German Federal Joint Committee (G-BA) Issues Final Assessment Report for
                            PIXUVRI® (pixantrone)

- Pricing Negotiations to Begin in June -

PR Newswire

SEATTLE, May 20, 2013

SEATTLE, May 20, 2013 /PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (NASDAQ
and MTA: CTIC) today reported that Germany's Federal Joint Committee (G-BA)
has issued its final benefit assessment report for PIXUVRI® (pixantrone),
which is indicated as a monotherapy for the treatment of adult patients with
aggressive B-cell non-Hodgkin lymphoma (NHL) who have failed two or three
prior lines of therapy. The G-BA reported that additional benefit could not be
determined for PIXVURI versus the comparator therapies assigned by G-BA, the
ultimate authority in determining reimbursement for drugs in Germany. PIXUVRI
is the first medicinal product approved in the European Union (E.U.) for
patients with aggressive B-cell NHL who have failed two or three prior lines
of therapy; as such, there was no approved standard of care. The Federal Joint
Committee also decided that the prescribability for pixantrone should be
limited to hematologists and oncologists and that this limitation might be of
relevance for the GKV-SV, the Federal Association of Statutory Health
Insurance Funds, with regard to the therapy cost, since the cost for PIXUVRI
was only marginally higher than the cost of the appropriate comparative
therapy. CTI also reported that the Company was notified by the GKV-SV that
pricing discussions will begin in June.

The G-BA's decision was based on the assessment that the submitted Phase 3
EXTEND study of PIXUVRI was found not to be sufficient to allow a conclusion
of a 'proven additional benefit' because:

  othe chosen comparator arm differed from the G-BA selected appropriate
    comparative therapy , and
  othe pre-treatment of patients in the pivotal Phase 3 study of PIXUVRI
    (PIX301) did not reflect the current treatment used in German health care
    practice with regard to previous therapies patients received.

"We recognize the challenge of quantifying the additional benefit for a
therapy for which under AMNOG law there is no comparator because there was no
approved standard of care for this stage of the disease, prior to the approval
of PIXUVRI in the E.U.," said James A. Bianco, M.D., President and CEO of CTI.
"PIXUVRI is the first medicinal product approved in the E.U. for the treatment
of patients with aggressive non-Hodgkin B-cell lymphomas who have failed two
or three prior lines of therapy, which was based on results from the only
Phase 3 study conducted in this patient population. We appreciate the Joint
Committee recognizing this conundrum and providing CTI the opportunity to work
with payors to arrive at an equitable price for PIXUVRI, based on the cost
associated with unlicensed agents or regimens currently being utilized by
physicians in Germany."

CTI will begin pricing discussions in June with the GKV-SV, the Federal
Association of Statutory Health Insurance Funds. Under AMNOG, the annual
treatment costs must not exceed those of the appropriate comparator.

PIXUVRI is already available in Germany for healthcare professionals to
prescribe in accordance with local guidance. InMay 2012, the European
Commission granted conditional marketing authorization for PIXUVRI as a
monotherapy for the treatment of adult patients with multiply relapsed or
refractory aggressiveNHL based on the results of the EXTEND, or PIX301,
pivotal randomized Phase 3 clinical trial. 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 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 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.

About Non-Hodgkin Lymphoma

In the E.U., there are approximately 37,000 new cases of aggressive B-cell NHL
every year.^1,2 ^NHL is caused by the abnormal proliferation of lymphocytes,
cells 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

Safe Harbor Statement

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 CTI may not obtain
reimbursement in certain markets in the E.U. as planned; that results in
future studies of PIXUVRI may differ from the results of past studies; that
the G-BA's decision regarding PIXUVRI and limitation of prescribability to
hematologists and oncologists may have an impact on the reimbursement or
adoption of PIXUVRI in Germany or elsewhere; that the GKV-SV pricing
discussions may not begin in June and may not result in favorable pricing
determinations; 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 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 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.


1.European Cancer Observatory, Cancer Fact Sheets, 2008
2.Harris NL, et al. Ann Oncol. 1999;10(12):1419-32
3.Friedberg ASH Education Book 2011;1:498-505

PIXUVRI is a registered trademark of Cell Therapeutics, Inc.

CTI Media and Investor Contacts:

Monique Greer
+1 206-272-4343

Ed Bell
+1 206-282-7100

In Europe:

Laura Villa
Elena Bellacicca

T: +39 02 89659700
F: +39 02 89659719

SOURCE Cell Therapeutics, Inc.

Press spacebar to pause and continue. Press esc to stop.