BioWorld Today Contributing Writer

Kite Pharma Inc. is a new company created to pursue what its leadership described as "the next therapeutic modality" in treating cancer: recruiting the body's own immune system to fight malignancies. Its pipeline of immunotherapeutic candidates combines tumor-specific antigens and targeted immune-activating components.

Kite, of Los Angeles, is developing two antigens, carbonic anhydrase IX (CAIX) and alpha fetoprotein, as initial therapeutic targets. In March, Kite Pharma raised $15 million in its first financing round through a private placement with a syndicate of venture investors.

"We're aiming at harnessing the patient immune response to fight tumor cells," Kite CEO Aya Jakobovits told BioWorld Today. "Our product and strategy are aimed to increase and produce a robust immune response against tumor cells."

According to Kite, the immune response in patients with advanced cancer is almost always insufficient to attack and clear the diseased cells. Those cells grow out of control and spread. The company's strategy is to enhance the immune system's ability to attack those tumor cells.

Kite's CAIX entity marries granulocyte-macrophage colony-stimulating factor (GM-CSF), a potent immune activating molecule, to the CAIX gene. In preclinical studies, GM-CAIX immunization has resulted in inhibition of kidney cancer in mice.

Cells expressing GM-CAIX also caused the death of tumor cells from renal cancer patients. CAIX antigen is expressed in many tumor types including kidney, bladder, cervical, ovarian, head and neck, colon and breast cancers, showing the utility of the approach across a range of cancers. Kite is developing GM-CAIX for renal cell carcinoma.

Kite's product targeting alpha fetoprotein (AFP) has potential as a treatment for hepatocellular carcinoma. AFP is expressed in large amounts in that cancer, but not in normal adult tissues. In preclinical studies, immunization against AFP inhibited growth of tumors in mice.

Immune system therapies for cancer have attracted increasing interest since last year's annual meeting of the American Society of Clinical Oncology (ASCO), where clinical studies showing efficacy of such treatments made a big splash. (See BioWorld Today, June 8, 2010.)

At the conference, Bristol-Myers Squibb Co. reported that ipilimumab met its endpoint and achieved statistical significance in the difficult indication of melanoma in a large Phase III trial.

Ipilimumab is a human antibody targeted to cytotoxic T lymphocyte-associated antigen 4 (CTLA-4), which is a regulator of immune response. Blocking the activity of CTLA-4 is thought to enable a sustained immune response against cancer cells. The FDA approved ipilimumab in March 2011, and BMS is marketing it under the brand name Yervoy. (BioWorld Today, March 28, 2011.)

Dendreon's Provenge (sipuleucel-T) made history a year ago as the first therapeutic cancer vaccine approved by the FDA. Provenge sales have been strong, at $28.1 million in the first quarter, and $15 million in April alone. Dendreon's guidance for 2011 sales of Provenge comes in at $350 million to $400 million, weighted heavily toward the end of the year, as it expects the product to build. (See BioWorld Today, April, 30, 2010, and May 3, 2010.)

Drugs like Provenge provide a potential template model for the success of similar therapies, like Kite's CAIX and AFP products. "With the approval of Provenge . . . there is some maturation of understanding of why the immune system is failing in patients with cancer," Jakobovits pointed out.

Kite Pharma was founded in late 2010, but received its first major financing March 9. The private placement, agented by Riverbank Capital Inc., raised $15 million.

Jakobovits credited the successful financing and launch of the company to the experience of its management team, which includes Arie Belldegrun, one of the founders of Agensys Inc. (purchased by Astellas Pharma Inc. in December 2007 for $537 million), and Gloria Lee, who led the clinical team through the global registration of Boniva (ibandronate) at Hoffman La-Roche.

Jakobovits also hails from Agensys, where she served as its executive vice president and head of research and development.

According to Jakobovits, the Kite management team has a "proven track record of managing and growing companies from product pipeline all the way to approval."