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

- Pricing Negotiations to Begin in June -

News provided by

May 20, 2013, 01:30 ET

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:

the chosen comparator arm differed from the G-BA selected appropriate comparative therapy , and
the 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. In May 2012, the European Commission granted conditional marketing authorization for PIXUVRI as a monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive NHL 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. 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 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 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.

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-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 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 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 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.

References: