Assistant Managing Editor

Cell Therapeutics Inc.'s pixantrone met its primary endpoint in the pivotal EXTEND trial in patients with advanced, relapsed aggressive non-Hodgkin's lymphoma, an event company CEO James Bianco called "a big win."

"I think the company has several opportunities to see a pretty significant turnaround," he told investors during a webcast presentation at the Rodman & Renshaw Healthcare Conference in New York.

Preliminary analysis of the pivotal data showed that pixantrone, a DNA intercalating cancer drug designed with a safer profile than existing anthracycline therapies, demonstrated a 3.5-fold higher complete remission/unconfirmed complete remission rate compared to standard chemotherapy. Twenty percent (14/70) of patients on pixantrone had a complete response vs. 5.7 percent (4/70) of patients receiving chemotherapy.

"All confirmed complete responses were in the pixantrone group," Bianco added.

Pixantrone treatment also showed an improvement in the secondary endpoint of overall response rate (ORR), demonstrating 37 percent (26/70) ORR vs. 14 percent (10/70) in the control group. Data have not yet been compiled for the other secondary endpoint of progression-free survival, but Bianco said the data gleaned so far indicate that an improvement from pixantrone likely will be seen there as well.

During the trial, about 74 percent of patients discontinued treatment due to either disease progression or death, but the "super-majority of those were in the control arm," Bianco said.

Seattle-based Cell Therapeutics plans to request a pre-new drug application meeting with the FDA and expects to begin submission of a rolling NDA in the first quarter of next year. Pixantrone has fast-track status, so the company anticipates a priority six-month review process.

With the positive Phase III data in hand, Cell Therapeutics also expects to hear from partner Novartis AG on whether the Basel, Switzerland-based pharma giant will seek to option rights to pixantrone under the companies' 2006 collaboration. That deal involved Novartis gaining rights to lung cancer drug Opaxio (poliglumex paclitaxel, formerly Xyotax) in exchange for up to $285 million in up-front equity and potential milestone payments, with an option to license pixantrone for up to $11.5 million. (See BioWorld Today, Sept. 19, 2006.)

If Novartis exercises that option, Cell Therapeutics can expect a $7.5 million option fee, with another $15 million coming at the time of FDA approval, Bianco said. Specific terms for the sales and marketing end of the arrangement are subject to further negotiation should Novartis opt for pixantrone rights, he added.

Pixantrone is in development as an improved treatment compared to existing anthracycline therapy. While anthracyclines have been active against a number of cancer types, including lymphomas, the "major limitation" to their use has been the heart damage caused by increasing exposure, Bianco said.

Cell Therapeutics' drug is designed specifically to reduce the free radical production that leads to cardiac toxicity. And clinical data to date have shown that pixantrone is "much better tolerated than traditional anthracyclines," he added.

In addition to the EXTEND study, pixantrone is being tested in a supporting trial in chemotherapy-naïve patients. It also is in a Phase III trial in indolent NHL patients who have relapsed after first-line therapy.

In the indolent setting, Frazer, Pa.-based Cephalon Inc.'s Treanda (bendamustine) recently gained approval, and anti-CD20 antibody Rituxan (rituximab, Genentech Inc. and Biogen Idec Inc.) is approved for both the aggressive and indolent forms of the disease.

Cell Therapeutics also markets Zevalin ([90Y]-ibritumomab tiuxetan), a radioimmunotherapy for NHL, and recently submitted a supplemental biologics license application to expand the drug's use after a pivotal trial showed that the drug significantly improved progression-free survival in patients with advanced follicular NHL who had a partial or complete response to first-line chemotherapy regimens. (See BioWorld Today, Oct. 17, 2008.)

The company ended the third quarter with about $11.7 million in cash, though it added an additional $7.5 million to its bank account last month.

Shares of Cell Therapeutics (NASDAQ:CTIC) closed at 35 cents Tuesday, up 2 cents.

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