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

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Excellence (NICE), a non-departmental public body of the Department of Health in the United Kingdom, issued a second draft guidance on the use of PIXUVRI® (pixantrone) as a monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive B-cell non-Hodgkin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 3rd line salvage setting."  

"There are no other licensed products for use in the 3rd 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.

In May 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 aggressive NHL. 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. The SmPC is available at www.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 in the 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 in the United States, conditional marketing authorizations are granted in the E.U. to medicinal products with a positive benefit/risk assessment that 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 Use has 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‑cell NHL and 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 by June 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-cell NHL and who are not eligible for autologous stem cell transplant by June 2015 or at all as required by the EMA or have the results of such clinical trial available by June 2015 or 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 the Securities and Exchange Commission including, 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.



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