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Halozyme Reports Fourth Quarter And Full Year 2016 Financial Results

On February 28, 2017 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results and recent highlights for the fourth quarter and full year ended December 31, 2016 (Press release, Halozyme, FEB 28, 2017, View Source [SID1234517886]).

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"We exited 2016 in a strong position through the benefit of our differentiated business model and having made substantial progress across both of our value-creating pillars," said Dr. Helen Torley, president and chief executive officer. "The positive Phase 2 data we reported last month affirms our conviction in PEGPH20, our lead investigational oncology drug, and supports our ongoing Phase 3 study in pancreas cancer patients. We have made strong progress initiating global sites in the Phase 3 study and see momentum building in the number of patients we are screening.

"Our ENHANZE platform remains an important value driver as royalty revenue demonstrated yet another quarter of robust growth, and exceeded $50 million for the year. With positive data presented by Genentech and Janssen at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting for their ENHANZE-partnered programs and a recent BLA filing in the U.S. by Genentech, we remain very encouraged by the royalty growth potential from current and new products over the long term."

Fourth Quarter 2016 and Recent Highlights include:

Reporting a statistically significant improvement in the primary endpoint of progression-free survival (PFS) of all evaluable patients, and in the secondary endpoint of PFS in patients with high levels of hyaluronan (HA), as of a December 2016 data cut in the Phase 2 randomized HALO-202 study of PEGPH20 in combination with ABRAXANE (nab-paclitaxel) and gemcitabine in advanced pancreas cancer patients. The primary safety endpoint to evaluate and demonstrate a reduction in the rate of thromboembolic events in the PEGPH20 arm was also achieved.

In the population that closely mirrors the HALO-301 Phase 3 study, a 91 percent improvement was achieved in median PFS for HA-High patients in the PEGPH20 arm, 8.6 months compared to 4.5 months in the control arm. A 50 percent improvement was also reported in median overall survival (OS) for HA-High patients in the PEGPH20 arm, 11.7 months compared to 7.8 months in the control arm.

Screening patients at nearly 200 global sites and receiving approval in all 22 participating countries for HALO-301, the company’s Phase 3 study of pancreas cancer patients.

Entering the dose expansion phase of the ongoing Phase 1b clinical study evaluating PEGPH20 in combination with KEYTRUDA (pembrolizumab) in relapsed non-small cell lung and gastric cancer patients. The company expects to enroll approximately 50 patients with high levels of HA at 30 U.S. sites.

Announcing a broad clinical collaboration agreement with Genentech to evaluate PEGPH20 and TECENTRIQ (atezolizumab) in up to 8 tumor types. Halozyme plans to initiate a Phase 1b trial to evaluate the combination in gallbladder cancer and cholangiocarcinoma, and Roche plans to initiate multiple Phase 1b/2 trials within their novel immunotherapy MORPHEUS clinical trial platform to evaluate the combination in pancreas and gastric cancers in the second half of 2017.

Presenting supportive clinical data by ENHANZE partners at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in November. Genentech presented data from the SABRINA Phase 3 study indicating comparable response rates and time-to-event data for subcutaneous rituximab compared to IV administration. The data presentation followed acceptance by the Food and Drug Administration (FDA) of a Biologics License Application for a subcutaneous formulation of rituximab using Halozyme’s ENHANZE technology. Roche has indicated it is seeking approval in chronic lymphocytic leukemia and non-Hodgkin’s lymphoma, with an action date in June.

In addition, Janssen presented data indicating its subcutaneous formulation of daratumumab on the ENHANZE platform was well tolerated and had an efficacy and pharmacokinetic profile consistent with the IV formulation, demonstrating the feasibility of a 30-minute, 90 mL dose and supporting further study in a Phase 3 clinical trial.
Fourth Quarter and Full Year 2016 Financial Highlights

Revenue for the fourth quarter was $39 million compared to $52.2 million for the fourth quarter of 2015. The year-over-year decrease was driven by $25 million received upon signing the Lilly collaboration agreement in 2015, offset by increases in royalties from partner sales of Herceptin SC, MabThera SC and HYQVIA, API sales to partners, and manufacturing and clinical supply reimbursements from ENHANZE partners. Revenue for the fourth quarter included $14.3 million in royalties, an increase of 50 percent from the prior-year period, $9 million in sales of bulk rHuPH20 primarily for use in manufacturing collaboration products and $4.4 million in HYLENEX recombinant (hyaluronidase human injection) product sales.

Revenue for 2016 totaled $146.7 million, an increase of 9 percent from 2015, including royalty revenue of $51 million, an increase of 65 percent from 2015.

Research and development expenses for the fourth quarter were $41.3 million, compared to $27.7 million for the fourth quarter of 2015. The planned increases were primarily due to a ramp in spending associated with the HALO-301 study, personnel expenses, and manufacturing and clinical supply expenses that are reimbursed by ENHANZE partners.

Selling, general and administrative expenses for the fourth quarter were $12.2 million, compared to $10.6 million for the fourth quarter of 2015. The increase was primarily due to personnel expenses, including stock compensation, for the period.

Operating expenses for 2016 totaled $229.9 million, an increase of 41 percent from 2015.

Net loss for the fourth quarter was $27.4 million, or $0.21 per share, compared to net income in the fourth quarter of 2015 of $4.3 million, or $0.03 per share. Net loss for 2016 totaled $103 million, or $0.81 per share.

Cash, cash equivalents and marketable securities were $205 million at December 31, 2016, compared to $221.1 million at September 30, 2016.
Financial Outlook for 2017

For 2017, the company reiterated and updated the cash portions of its financial guidance, now expecting:

Net revenue of $115 million to $130 million, excluding any new ENHANZE collaboration agreements;
Operating expenses of $240 million to $250 million;
Operating cash burn of $75 million to $85 million; and
Year-end cash balance of $110 million to $125 million, an increase from its prior range of $100 million to $110 million.
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Actinium Announces Expansion of Intellectual Property Portfolio with Notice of Allowance for U.S. Patent Related to Actimab-A, Actimab-M and the Company’s Technology Platform

On February 28, 2017 Actinium Pharmaceuticals, Inc. (NYSE MKT:ATNM) ("Actinium" or "the Company"), a biopharmaceutical Company developing innovative targeted payload immunotherapeutics for the treatment of advanced cancers, reported that the Company received a notice of allowance from the United States Patent and Trademark Office (USPTO) for a patent claiming the methods for generating a radioimmunoconjugate comprised of actinium-225, an alpha emitting radioisotope, conjugated to monoclonal antibodies (Press release, Actinium Pharmaceuticals, FEB 28, 2017, View Source [SID1234517883]).

Actinium-225 is the radioisotope used in Actinium’s Actimab-A, a drug candidate in a Phase 2 clinical trial for patients newly diagnosed with acute myeloid leukemia (AML) who are over the age of 60; Actimab-M, a drug candidate in a Phase 1 trial for patients with relapsed or refractory multiple myeloma (MM); and the Company’s alpha particle immunotherapy technology platform. Actinium currently has 61 issued and pending patents that are owned or licensed relating to isotope production methods, drug preparation methods and the Company’s platform technology. This patent is expected to expire in July of 2030.

Sandesh Seth, Actinium’s Executive Chairman said, "Actinium-225 is an excellent isotope for medical applications given its half-life, potency, safety profile and relatively ease of handling. As a result, it is an important aspect of Actinium’s Actimab-A and Actimab-M programs and alpha particle immunotherapy technology (APIT) platform. We welcome this addition to our intellectual property portfolio, which now consists of more than 60 patents. We will continue to invest in our intellectual property portfolio to further enhance our position in the use of alpha particles to enhance outcomes for patients."

The claims of this patent are for a method for producing actinium-225 radioconjugates, the method comprising the steps of: a) conjugating a chelating agents to a biological molecule in a conjugations reaction mixture to generate a conjugated biological molecule, b) purifying the reaction mixture so as to remove unconjugated chelating agents, and c) chelating one or more actinium-225 radionuclides with the conjugated biological molecule in a chelation reaction mixture to generate an actinium-225 radioconjugate.

Kite Reports Fourth Quarter and Full Year 2016 Financial Results

On February 28, 2017 Kite Pharma, Inc. (Nasdaq:KITE) reported a corporate update and fourth quarter and full-year 2016 financial results for the period ended December 31, 2016 (Press release, Kite Pharma, FEB 28, 2017, View Source [SID1234517879]).

In a separate announcement today, Kite issued positive topline results from the primary analysis of the ZUMA-1 study of axicabtagene ciloleucel in patients with aggressive non-Hodgkin lymphoma (NHL). Kite continues to expect completion of its rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for axicabtagene ciloleucel (KTE-C19) for the treatment of aggressive NHL by the end of the first quarter 2017, with potential approval and commercial launch in 2017.

"The accomplishments, leadership additions to the company, and performance of Kite during 2016 put us in a position of strength as we prepare to potentially deliver the first CAR-T therapy for aggressive NHL to patients later this year," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "Beyond axicabtagene ciloleucel, we have taken important steps to expand Kite’s global footprint and pipeline of cell therapy candidates. Through our recently announced strategic collaborations with Fosun Pharma and Daiichi Sankyo, our plans for submission and potential launch in Europe, and ongoing clinical studies of axicabtagene ciloleucel, we look ahead to an increasingly strong future for Kite."

Fourth Quarter and Full Year 2016 Financial Results

Revenues were $4.9 million for the fourth quarter of 2016 and $22.2 million for the full year of 2016.
Research and development expenses were $58.9 million for the fourth quarter of 2016, which includes $8.9 million of non-cash stock-based compensation expense. For the full year of 2016, research and development expenses were $197.9 million, which includes $34.7 million of non-cash stock-based compensation expense.
General and administrative expenses were $31.8 million for the fourth quarter of 2016, which includes $10.8 million of non-cash stock-based compensation expense. For the full year of 2016, general and administrative expenses were $97.4 million, which includes $38.8 million of non-cash stock-based compensation expense.
Net loss was $84.9 million, or $1.70 per share, for the fourth quarter of 2016. For the full year of 2016, net loss was $267.1 million, or $5.46 per share.
Non-GAAP net loss for the fourth quarter of 2016 was $65.2 million, or $1.31 per share, excluding non-cash stock-based compensation expense of $19.7 million. For the full year of 2016, non-GAAP net loss was $193.5 million, or $3.95 per share, excluding non-cash stock-based compensation expense of $73.6 million.
As of December 31, 2016, Kite had $414.4 million in cash, cash equivalents, and marketable securities. In January 2017, Kite received a $50 million upfront payment from Daiichi Sankyo related to the recently announced strategic collaboration in Japan.
2017 Financial Guidance

Kite expects full year 2017 net cash burn to be between $325 million and $340 million, which includes approximately $30 million in capital expenditures but excludes cash inflows or cash outflows from business development activities, if any, and excludes planned upfront payments totaling $90 million from recently announced strategic collaborations in Asia. Estimated full year 2017 cash burn is driven primarily by a projected GAAP net loss of between $450 million and $465 million. The 2017 projected net loss includes non-cash stock-based compensation expenses of approximately $135 million.
Kite expects full year 2017 revenue to be between $40 million and $50 million, which assumes no product revenue, and full year 2017 GAAP operating expenses to be between $490 million and $515 million.
As previously announced, Kite expects to have sufficient cash resources to fund its current operations, including planned clinical development programs, through the first half of 2018. This projection excludes cash inflows or cash outflows from future business development activity, if any.
2016 Highlights

Axicabtagene Ciloleucel/KTE-C19 Progress

Presented positive interim results from the ZUMA-1 Phase 2 study of axicabtagene ciloleucel in aggressive NHL in a late-breaker oral presentation at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.
Reported a Phase 1 update from the ZUMA-3 and ZUMA-4 trials of KTE-C19 in adult and pediatric relapsed/refractory acute lymphoblastic leukemia at the 2016 ASH (Free ASH Whitepaper) annual meeting.
Initiated ZUMA-6, a Phase 1b/2 study of axicabtagene ciloleucel in combination with the checkpoint inhibitor atezolizumab in patients with chemorefractory diffuse large B-cell lymphoma (DLBCL) in collaboration with Genentech.
Presented results from SCHOLAR-1, the first patient-level pooled analysis of outcomes in chemorefractory DLBCL, providing an important historical benchmark for studies of the disease.
Regulatory Milestones

Initiated BLA rolling submission to the FDA for axicabtagene ciloleucel in December 2016.
Received Orphan Drug Designation from the FDA for KTE-C19 in the treatment of five additional B-cell malignancies, granting Kite the designation for the major indications in hematologic malignancies in the US. KTE-C19 also has Orphan Drug Designation in the EU for these indications.
Received Priority Medicines (PRIME) status from the European Medicines Agency for KTE-C19 in the treatment of chemorefractory DLBCL.
Axicabtagene Ciloleucel Commercial & Manufacturing Readiness

Achieved 99 percent success rate in the manufacturing of clinical product patient dose from a single apheresis for the multi-center ZUMA-1 clinical trial.
Marked the official opening of Kite’s commercial manufacturing plant, a state-of-the-art facility in El Segundo, California estimated to have the capacity to produce more than 4,000 patient therapies per year.
Initiated development of Kite Konnect, a cloud-based solution for commercial-scale ordering, logistics, monitoring and delivery of T-cell therapies, designed to enable a positive prescriber and patient experience.
Pipeline Expansion and Developments

Submitted an investigational new drug (IND) application for KITE-718, a TCR product candidate that targets MAGE-A3/A6 antigens expressed on solid tumors.
Initiated plans to file an IND application in 2017 for KITE-585, a CAR product candidate targeting B cell maturation antigen (BCMA) for multiple myeloma.
Initiated plans to file IND applications in 2018 for KITE-796, a CAR product candidate targeting CLL-1 in acute myeloid leukemia (AML), and for KITE-439, a TCR product candidate targeting human papillomavirus (HPV)-16 E7 for cervical cancer, head and neck cancer, and other solid tumors.
Strategic Collaborations

Entered into a new Cooperative Research and Development Agreement (CRADA) for the development of a fully human anti-CD19 CAR product candidate to treat B-cell malignancies with the National Cancer Institute (NCI).
Licensed intellectual property related to a fully human anti-CD19 CAR to treat B-cell malignancies from the National Institutes of Health (NIH).
Expanded the TCR portfolio by licensing intellectual property from the NIH related to multiple TCR product candidates for the treatment of solid tumors expressing mutated KRAS antigens.
Entered into a new CRADA for the development of TCR product candidates directed against HPV-16 E6 and E7 for the treatment of HPV-associated cancers with the NCI.
Licensed a technology platform from The Regents of the University of California, on behalf of the University of California, Los Angeles, for the scalable production of T cells using pluripotent stem cell lines capable of indefinite self-renewal and designed to overcome limitations of current allogeneic approaches.
Partnered with Leiden University Medical Center to develop TCR product candidates targeting solid tumors associated with HPV-16 infection.

FDA Accepts the Biologics License Application for Avelumab for the Treatment of Metastatic Urothelial Carcinoma for Priority Review

On February 28, 2017 EMD Serono, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany, in the US and Canada, and Pfizer Inc. reported that the US Food and Drug Administration (FDA) has accepted for Priority Review EMD Serono’s Biologics License Application (BLA) for avelumab* as a treatment for patients with locally advanced or metastatic urothelial carcinoma (mUC) with disease progression on or after platinum-based therapy (Press release, Pfizer, FEB 28, 2017, View Source [SID1234517878]). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of August 27, 2017, for avelumab in this indication.

"Taken together with last year’s filing for metastatic Merkel cell carcinoma, this BLA acceptance confirms our rapid and continued progress in the clinical development of avelumab," said Luciano Rossetti, M.D., Executive Vice President, Global Head of Research & Development at the biopharma business of Merck KGaA, Darmstadt, Germany. "We continue to evaluate avelumab in cancers that have limited or suboptimal treatment choices, such as metastatic or locally advanced urothelial carcinoma, to hopefully be able to provide patients with new treatment options for fighting their disease."

Despite advances in the treatment of UC, the prognosis for patients remains poor, particularly when the disease has metastasized. Bladder cancer makes up approximately 90% of urothelial cancers and is the sixth most common cancer in the US.[1],[2] "

Advanced urothelial carcinoma remains a difficult-to-treat tumor, which is why we are developing a comprehensive clinical development program that involves Phase I and III trials designed to address this challenge," said Chris Boshoff, M.D., Ph.D., Senior Vice President and Head of Immuno-oncology, Early Development and Translational Oncology, Pfizer Global Product Development. "We’re continuing to accelerate our urothelial carcinoma development program and look forward to continuing our dialogue with the FDA."

Avelumab is an investigational, fully human anti-PD-L1 antibody. The FDA’s Priority Review status reduces the review time from 10 months to a goal of six months from the day of filing acceptance and is given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists. In November 2016, the FDA accepted, and granted Priority Review status to, the BLA for avelumab for the treatment of patients with metastatic Merkel cell carcinoma.

The international clinical development program for avelumab, known as JAVELIN, involves at least 30 clinical programs, including nine Phase III trials, and more than 4,000 patients evaluated across more than 15 tumor types. In December 2015, Merck KGaA, Darmstadt, Germany, and Pfizer announced the initiation of a Phase III study (JAVELIN Bladder 100) of avelumab in the first-line setting as a maintenance treatment in patients with locally advanced or metastatic UC. This trial is currently enrolling patients.

*Avelumab is not approved for any indication in any market. This marks the second acceptance of an application by the FDA to review the investigational product, avelumab.

References

1.National Comprehensive Cancer Network. NCCN Guidelines Version 1.2017 Updates. Bladder Cancer. Available from: View Source (link is external). Last Accessed: February 2017.

2.Siegel RL, et al. Cancer Statistics, 2017. CA Cancer J Clin 2017;67:7-30. Available from: View Source (link is external). Last Accessed: February 2017.

3.American Cancer Society. Key Statistics for Bladder Cancer. Available from: View Source (link is external). Last Accessed: February 2017.

About Metastatic Urothelial Carcinoma

Urothelial Carcinoma includes several tumors originating from the cells lining the bladder, renal pelvis and urethra. While cancers outside of the bladder are relatively uncommon, accounting for an estimated 10% of cases, bladder cancer represents 90% of urothelial cancers and is the ninth most common cancer globally.[1],[3] Worldwide, approximately 400,000 new cases of bladder cancer are diagnosed and 150,000 deaths are attributed to this disease each year.[3] The incidence and mortality of bladder cancer have remained unchanged over the past 25 years.[3]

About Avelumab

Avelumab is a fully human antibody specific for a protein found on tumor cells called PD-L1, or programmed death ligand-1. By inhibiting PD-L1 interactions, avelumab is thought to enable the activation of T-cells and the adaptive immune system. By retaining a native Fc-region, avelumab is thought to potentially engage the innate immune system and induce antibody-dependent cell-mediated cytotoxicity (ADCC). In November 2014, Merck KGaA, Darmstadt, Germany, and Pfizer announced a strategic alliance to co-develop and co-commercialize avelumab. Common adverse reactions include fatigue, musculoskeletal pain, diarrhea, nausea peripheral edema, decreased appetite, and rash. Immune-mediated adverse reactions have also been reported.

Alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc., New York, US

Immuno-oncology is a top priority for Merck KGaA, Darmstadt, Germany, and Pfizer Inc. The global strategic alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc., New York, US, enables the companies to benefit from each other’s strengths and capabilities and further explore the therapeutic potential of avelumab, an investigational anti-PD-L1 antibody initially discovered and developed by Merck KGaA, Darmstadt, Germany. The immuno-oncology alliance will jointly develop and commercialize avelumab and advance Pfizer’s PD-1 antibody. The alliance is focused on developing high-priority international clinical programs to investigate avelumab as a monotherapy, as well as in combination regimens, and is striving to find new ways to treat cancer.

Kite Announces Positive Topline Primary Results of Axicabtagene Ciloleucel from First Pivotal CAR-T Trial in Patients with Aggressive Non-Hodgkin Lymphoma

On February 28, 2017 Kite Pharma, Inc., (Nasdaq:KITE) reported positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel (previously referred to as KTE-C19), in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL) (Press release, Kite Pharma, FEB 28, 2017, View Source [SID1234517877]). The study met the primary endpoint of objective response rate (ORR), or rates of tumor response (complete response + partial response) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p < 0.0001).

These results demonstrate the treatment effect of axicabtagene ciloleucel in a patient population with multiple types of aggressive NHL, including diffuse large B-cell lymphoma (DLBCL) enrolled in Cohort 1, as well as primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) enrolled in Cohort 2.

One hundred one patients were treated in ZUMA-1. The following table shows the ORR and rate of complete response (CR) as well as the month 6 ORR and CR:

DLBCL (n=77)
TFL/PMBCL (n=24)
Combined (n=101)
ORR (%) CR (%) ORR (%) CR (%) ORR (%) CR (%)
ORR 82 49 83 71 82 54
Month 6 36 31 54 50 41 36

Four of the 101 patients in ongoing CR did not have a month 6 tumor assessment prior to the data cut-off and are therefore categorized as non-responders for month 6 in the table above. These patients have an opportunity to be counted as a month 6 CR in a follow-up analysis, which may increase the month 6 response and month 6 CR rate.

At month 6, 41 percent of treated patients achieved a response, including 36 percent in CR. Five of the 101 patients (5 percent) continue to experience highly significant and durable partial responses (PR) with minimal abnormalities in PET scans. One of these PRs converted to a CR at month 9.

With a median follow-up of 8.7 months for this primary analysis, the median overall survival (OS) has not yet been reached. In a similar patient population, the median OS was estimated to be 6.6 months (SCHOLAR-1 study, ASCO (Free ASCO Whitepaper) 2016).

The most common grade 3 or higher adverse events included anemia (43 percent), neutropenia (39 percent), decreased neutrophil count (32 percent), febrile neutropenia (31 percent), decreased white blood cell count (29 percent), thrombocytopenia (24 percent), encephalopathy (21 percent) and decreased lymphocyte count (20 percent). As compared to the interim analysis, grade 3 or higher cytokine release syndrome (CRS) decreased from 18 percent to 13 percent and neurologic events decreased from 34 percent to 28 percent. There were no cases of cerebral edema.

As previously reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in 2016, there were three deaths not due to disease progression in the study. Two events, one hemophagocytic lymphohistiocytosis and one cardiac arrest in the setting of CRS, were deemed related to axicabtagene ciloleucel. The third case, a pulmonary embolism, was deemed unrelated. Between the interim analysis that included 62 patients, and this primary analysis which now includes all 101 patients, there were no additional deaths due to adverse events.

"These results with axicabtagene ciloleucel are exceptional and suggest that more than a third of patients with refractory aggressive NHL could potentially be cured after a single infusion of axicabtagene ciloleucel," said Jeff Wiezorek, M.D., Senior Vice President of Clinical Development. "The ZUMA-1 study was built on a foundation of support and commitment from Dr. Steven Rosenberg and the National Cancer Institute and our ZUMA-1 clinical trial investigators who believed in the potential for CAR-T therapy to change the paradigm of cancer treatment."

Kite intends to seek regulatory approval of axicabtagene ciloleucel in aggressive NHL based upon the combined data from all 101 patients and plans to complete its rolling submission of the Biologics License Application (BLA) by the end of the first quarter of 2017. In addition, Kite plans to submit a marketing authorization application (MAA) for axicabtagene ciloleucel for the treatment of relapsed or refractory DLBCL, PMBCL and TFL with the European Medicines Agency (EMA) in 2017.

"We know as clinicians that patients with aggressive lymphoma who do not respond to their previous treatments have a very poor prognosis. In fact, we know from the SCHOLAR-1 study, these patients have only an eight percent chance of achieving a complete response with current therapies," said Frederick L. Locke, M.D., ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida. "Several patients we treated at Moffitt Cancer Center experienced a rapid and durable complete response with this first-of-its kind therapy. The ZUMA-1 study results suggest that axicabtagene ciloleucel could become a new standard of care for patients with refractory aggressive lymphoma."

Sattva S. Neelapu, M.D., Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, served as a co-lead investigator in the ZUMA-1 trial.

Full data from the primary analysis will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April 2017 in Washington, D.C.

ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program.

About axicabtagene ciloleucel
Kite Pharma’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.