On February 25, 2016 Heat Biologics, Inc. ("Heat") (Nasdaq: HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported that the company will no longer enroll new patients in its Phase 2 monotherapy trial arm evaluating HS-410 alone for the treatment of non-muscle invasive bladder cancer (NMIBC) (Filing, 8-K, Heat Biologics, FEB 25, 2016, View Source [SID:1234509202]). Heat added the monotherapy trial arm in response to the intermittent global shortage of standard of care Bacillus Calmette-Guérin (BCG) in early 2015. The shortage has since then been resolved and as such, Heat will no longer enroll new patients in this trial arm based on discussions with the U.S. Food and Drug Administration (FDA). The decision does not relate to concerns regarding the safety profile of HS-410. The 16 patients currently enrolled, out of the anticipated 25 patients, can continue receiving HS-410 monotherapy per the study protocol. Heat anticipates reporting topline 6-month data from these 16 patients in the fourth quarter of 2016, contemporaneous with reporting data from the company’s BCG combination cohorts.

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This decision does not impact the three additional randomized Phase 2 trial arms evaluating HS-410 in combination with BCG for the treatment of NMIBC. As previously announced, Heat completed enrollment of the 75 patients for these three combination trial arms in October 2015 and continues to expect to report one-year topline efficacy, immune-response and safety data in the fourth quarter of 2016.

About HS-410 (vesigenurtacel-L)

HS-410 is an investigational product candidate for NMIBC based on Heat’s proprietary ImPACT immunotherapy platform, designed to generate CD8+ "killer" T cells that attack cancer cells. HS-410 is currently being evaluated in a Phase 2, placebo-controlled NMIBC trial at multiple centers and has been granted U.S. FDA Fast Track Designation for the treatment of NMIBC.

On February 25, 2016 BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported financial results for both the fourth quarter and year ended December 31, 2015 (Press release, BioMarin, FEB 25, 2016, View Source [SID:1234509260]). For the quarter ended December 31, 2015, Non-GAAP net loss was $70.0 million, compared to non-GAAP net loss of $10.7 million for the fourth quarter of 2014. For the year ended December 31, 2015, non-GAAP net loss was $142.6 million, compared to non-GAAP net loss of $25.7 million for the year ended December 31, 2014. The increased non-GAAP net loss for the year ended December 31, 2015, compared to the prior year, is primarily due to increased research and development and selling, general and administrative expenses, partially offset by increased revenues from the global launch of Vimizim and strong Kuvan sales.

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GAAP net income was $68.6 million, or $0.43 per basic and $0.39 diluted share, respectively, for the fourth quarter of 2015, compared to GAAP net loss of $69.8 million, or $0.47 per basic and diluted share, for the fourth quarter of 2014. GAAP net income for the quarter included a $369.5 million gain on the sale of intangible assets due to the sale of talazoparib to Medivation, Inc. and a $47.9 million credit to contingent consideration expense primarily due to the U.S. FDA complete response letter for Kyndrisa. The complete response letter triggered a reversal of previously accrued Contingent Value Rights (CVR) related to the potential $80 million payment to former Prosensa shareholders for U.S. FDA approval prior to May 15, 2016. These gains and credits were partially offset by a $198.7 million impairment of intangible asset charge related to the decline in value of the U.S. rights to Kyndrisa due to the U.S. FDA complete response letter for Kyndrisa and increased research and development and selling, general and administrative expenses. GAAP net loss for the year ended December 31, 2015 was $171.8 million, or $1.07 per basic and diluted share, compared to GAAP net loss of $134.0 million, or $0.92 per basic and diluted share for the year ended December 31, 2014.

Total BioMarin Revenue was $889.9 million for the year ended December 31, 2015 an increase of 18.8% compared to the same period in 2014. Fourth quarter 2015 Total BioMarin Revenue of $227.9 million decreased 1.0% due to the timing of government orders impacting Naglazyme revenue. The increase in Total BioMarin Revenue for full-year 2015 was driven by the continued global launch of Vimizim and growth in the number of Kuvan patients on therapy. The number of patients being treated with Vimizim increased 10% quarter to quarter in the fourth quarter compared to the third quarter of 2015. Sales of Vimizim in 2015 were recorded in 13 new countries to a total of 33 countries through year-end and totaled $228.1 million for the full year. Kuvan Net Product Revenue increased 17.9% to $239.3 million driven primarily by patient number increases and high rates of compliance. At year-end 2015, commercial patients on Kuvan increased 15.8% year over year.

As of December 31, 2015, BioMarin had cash, cash equivalents and investments totaling $1,018.3 million, as compared to $1,043.1 million on December 31, 2014.

"BioMarin is entering 2016 from a position of strength as supported by four factors. First, we expect that our established and growing commercial business may generate over one billion dollars in revenues this year and believe it can grow to $1.5 billion by 2020. Second, data readouts for cerliponase alfa and pegvaliase may potentially lead to two new product filings later this year and two potential new product launches in 2017. Third, we have two potential $1 billion opportunities in development with vosoritide and BMN 270 for hemophilia A. And fourth, we expect to manage this growing business with the goal of achieving non-GAAP break-even or better in 2017 regardless of the regulatory outcome of Kyndrisa in Europe," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We look forward to hosting our annual R&D Day on April 20th in New York where we will share one year data with vosoritide for achondroplasia as well as an update on our gene therapy program with BMN 270 for the treatment of hemophilia A. In addition, in the second quarter we expect to receive an opinion from the Committee for Medicinal Products (CHMP) on a potential approval of Kyndrisa in Europe. Kyndrisa is under regulatory review in the E.U., where there is currently no approved treatment option for children with Duchenne muscular dystrophy amenable to exon 51 skipping."

Net Product Revenue (in millions, unaudited)

Total Revenue

Three Months Ended December 31, Twelve Months Ended December 31,
2015 2014 $ Change % Change 2015 2014 $ Change % Change

Vimizim (1) $ 58.5 $ 36.9 $ 21.6 58.5 % $ 228.1 $ 77.3 $ 150.8 195.1 %
Naglazyme (1) 59.7 88.5 (28.8 ) (32.5 )% 303.1 334.4 (31.3 ) (9.4 )%
Kuvan 64.8 57.4 7.4 12.9 % 239.3 203.0 36.3 17.9 %
Aldurazyme 39.0 40.9 (1.9 ) (4.6 )% 98.0 105.6 (7.6 ) (7.2 )%
Firdapse 4.4 4.1 0.3 7.3 % 16.0 18.1 (2.1 ) (11.6 )%
Net product revenues 226.4 227.8 (1.4 ) (0.6 )% 884.5 738.4 146.1 19.8 %

Collaborative agreement revenues 0.2 0.3 (0.1 ) 1.0 1.6 (0.6 )
Royalty, license and other revenues 1.3 2.0 (0.7 ) 4.4 9.3 (4.9 )
Total BioMarin revenues $ 227.9 $ 230.1 $ (2.2 ) (1.0 )% $ 889.9 $ 749.3 $ 140.6 18.8 %

(1) Vimizim and Naglazyme revenues experience quarterly fluctuations due to the timing of government ordering patterns in certain countries. The Company does not believe these fluctuations reflect a change in underlying demand.

Reconciliation of Aldurazyme Revenues

Three Months Ended December 31, Twelve Months Ended December 31,
2015 2014 $ Change % Change 2015 2014 $ Change % Change
Aldurazyme revenue reported by Genzyme $ 54.1 $ 56.3 $ (2.2 ) (3.9 )% $ 217.8 $ 228.8 $ (11.0 ) (4.8 )%

Three Months Ended December 31, Twelve Months Ended December 31,
2015 2014 $ Change 2015 2014 $ Change
Royalties earned from Genzyme $ 26.7 $ 27.9 $ (1.2 ) $ 95.8 $ 97.0 $ (1.2 )
Net product transfer revenues (2) 12.3 13.0 (0.7 ) 2.2 8.6 (6.4 )
Total Aldurazyme net product revenues $ 39.0 $ 40.9 $ (1.9 ) $ 98.0 $ 105.6 $ (7.6 )

(2) To the extent units shipped to third party customers by Genzyme exceed BioMarin inventory transfers to Genzyme, BioMarin will record a decrease in net product revenue from the royalty payable to BioMarin for the amount of previously recognized product transfer revenue. If BioMarin inventory transfers exceed units shipped to third party customers by Genzyme, BioMarin will record incremental net product transfer revenue for the period. Positive net product transfer revenues result in the period if BioMarin transferred more units to Genzyme than Genzyme sold to third-party customers.

2016 Financial Guidance

Revenue Guidance ($ in millions)

Item
2016 Guidance
Total BioMarin Revenues $1,050 to $1,100
Vimizim Net Product Revenue $300 to $330
Naglazyme Net Product Revenue $290 to $320
Kuvan Net Product Revenue $320 to $350

Select Income Statement Guidance ($ in millions, except percentages)

Item
2016 Guidance
Cost of Sales (% of Total Revenue) 18.0% to 19.0%
Selling, General and Admin. Expense $470 to $490
Research and Development Expense $680 to $720
Non – GAAP Net Loss $(75) to $(100)
GAAP Net Loss $(400) to $(430)

Anticipated Milestones in 1H16

Cerliponase alfa for CLN2, late-infantile form of Batten disease: Complete results from the Phase 1/2 study of cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1), for the treatment of patients with late-infantile neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease will be announced at the WORLD LSD Symposium on March 2, 2016. If data are supportive, the Company plans to submit in the U.S. and E.U. for regulatory approval mid-year 2016.

Pegvaliase for phenylketonuria (PKU): The Company expects to share top-line results from this study in the first quarter of 2016 and, if the data are supportive, submit a Biologics License Application (BLA) to U.S. FDA in the second half of 2016.

BMN 270 gene therapy product for hemophilia A: In the fourth quarter of 2015, the first patient was dosed in a Phase 1/2 trial with BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. Subjects in that study are now being dosed with the third highest dose in this dose ranging safety study. BioMarin will provide a program update at the R&D Day in April 2016.

Vosoritide for achondroplasia: In June 2015, the Company published results from the Phase 2 study showing a 50 percent in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (P-value= 0.01). In addition, to support further exploration of a dose that may enable "catch-up" growth in the event of delayed treatment, a fourth cohort with 30 micrograms per kilogram daily completed enrollment in the fourth arm of the Phase 2 study. BioMarin will provide 12-month results with vosoritide at the 15 µg/kg dose, preliminary safety update on the 30 µg/kg dose and an update on Phase 3 plans at the R&D Day in April 2016.

Kyndrisa (drisapersen) for Duchenne muscular dystrophy: The Committee for Medicinal Products (CHMP), the arm of the European Medicines Agency that is currently reviewing the Marketing Authorization Application for Kyndrisa, is expected to provide an opinion on the application in the second quarter of 2016. If the CHMP provides a positive opinion, Kyndrisa could potentially be approved in the E.U. in the second half of 2016.

Reveglucosidase alfa for Pompe disease: In January 2016, the Company shared interim results from the single-arm Phase 2/3 trial with patients previously treated with alglucosidase alfa who were then switched to treatment with reveglucosidase alfa. The primary endpoint of the study showed an improvement from baseline in the respiratory parameter Maximal Inspiratory Pressure (MIP) as well as the secondary endpoint 6 minute walk test. The Company is currently determining next steps for the program.

Other Corporate Achievements in 2015

October 1, 2015, BioMarin to Acquire Global Rights to PKU Franchise from Merck Serono
BioMarin and Merck Serono announced that BioMarin will acquire all global rights to Kuvan (sapropterin dihydrochloride), excluding Japan, and pegvaliase from Merck Serono (Merck). Under the terms of the agreement, BioMarin provided Merck with an upfront payment of $371.8 million. An additional €60 million in milestones will be paid to Merck if combined sales of Kuvan and pegvaliase reach undisclosed cumulative sales thresholds. In addition, €125 million will be paid to Merck conditional on the achievement of undisclosed regulatory milestones related to pegvaliase. Previously, BioMarin had exclusive rights to Kuvan in the United States and Canada and to pegvaliase in the United States and Japan. Under the terms of the transaction, BioMarin will now have exclusive worldwide rights to Kuvan and pegvaliase with the exception of Kuvan in Japan. Approved in 2007 in the U.S., Kuvan is a commercialized product for the treatment of patients with phenylketonuria (PKU). Pegvaliase is currently in registration-enabling pivotal studies as a potential therapeutic option for adult patients with phenylketonuria. With the potential approval of pegvaliase, the two products combined would expand and globalize BioMarin’s leadership position by offering a wider range of treatment options to patients worldwide with PKU.

August 24, 2015, Talazoparib acquired by Medivation, Inc.:
Medivation, Inc. and BioMarin Pharmaceutical Inc. announced that BioMarin entered into a definitive agreement to sell Medivation all worldwide rights to talazoparib (formerly referred to as BMN 673), a highly-potent, orally available poly ADP ribose polymerase (PARP) inhibitor currently in a Phase 3 study for the treatment of patients with deleterious germline BRCA 1 or BRCA 2 mutations and locally advanced and/or metastatic breast cancer. The transaction closed on October 6, 2015 and as a result, Medivation is now responsible for all research, development, regulatory and commercialization activities for all indications on a global basis. Under the terms of the agreement, Medivation paid BioMarin $410 million upfront, and will pay up to an additional $160 million upon the achievement of regulatory and sales-based milestones and mid-single digit royalties for talazoparib.

On February 24, 2016 The Clinical Center at the National Institutes of Health reported that they are investigating the potential use of a new generation of a computerized tomography (CT) scanner, called a photon-counting detector CT scanner, in a clinical setting (Press release, NIH, FEB 24, 2016, View Source [SID:1234509173]). The prototype technology is expected to replicate the image quality of conventional CT scanning, but may also provide health care specialists with an enhanced look inside the body through multi-energy imaging. Patients could receive a minimum amount of radiation, while the maximal amount of information needed would be delivered to health care providers.

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Over the next five years, David Bluemke, M.D., Ph.D., chief of the Department of Radiology and Imaging Sciences, and his team will continue to develop scan protocols and image processing algorithms, which could improve screening, imaging, and treatment planning for health conditions like cancer and cardiovascular disease.

"The NIH Clinical Center has helped shape and share research advances and health care for decades. Now is an exciting time for us and for our study participants here in the Clinical Center as we help test and develop this CT technology so that it may one day help patients around the world and impact the health care they receive," said Dr. Bluemke.

As the world’s largest hospital solely dedicated to research, the NIH Clinical Center sees thousands of patients every year, many of whom have rare and complicated illnesses. In the treatment and study of disease, surgery is often viewed as the last option. CT scanning is one way that doctors can examine the body’s internal features in a non-surgical way. In collaboration and through a partnership known as a cooperative research and development agreement with the manufacturer, Siemens Healthcare, and researchers in the CT technology field, the Clinical Center is testing this technology to help the health care field optimize the scanner for clinical use across the U.S. and around the globe.

Image of photon-counting CT scan
Photon CT scan image of a research subject at the NIH. Greater amounts of iodine contrast are shown in brighter, yellow colors.
The Clinical Center is one of three sites in the world to use this technology and is the first hospital-based research setting of the device. More than 45 volunteers enrolled in a research protocol have benefited from this cutting edge equipment. Initial findings have been reported in Radiology (link is external).

By advancing this technology, researchers aim to improve the diagnosis that doctors can offer by increasing the resolution and contrasts available for analysis. Areas of research investigation with the new technology include:

Doctors can identify materials in the body with anatomic precision. A dye, or contrast, is often given to a patient so that researchers can see a selected area in more detail. Different materials in the body can be displayed in different colors for faster diagnosis and precision.
The new technology may be used to help identify and characterize tumors, plaques or vessels that are smaller than half a millimeter. For many patients, finding a tumor that size may make a difference in identifying if it is benign or could be cancerous.
The technology may help to more accurately identify soft tissues such as proteins, tendons or collagen which are hard to differentiate with current equipment.

On February 24, 2016 bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2015 (Press release, bluebird bio, FEB 24, 2016, View Source;p=RssLanding&cat=news&id=2142878 [SID:1234509175]).

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"In 2015 bluebird bio defined an accelerated regulatory path for LentiGlobin in ß-thalassemia and established a powerful reason to believe in LentiGlobin in sickle cell disease, though there is still more to learn as we treat additional patients. We also fully enrolled our Starbeam study of Lenti-D in cerebral adrenoleukodystrophy and made significant advances in building a competitive T cell oncology franchise," said Nick Leschly, chief bluebird. "In 2016 we are excited to learn even more across all of our programs and continue to innovate and improve. We are particularly looking forward to sharing data from the Starbeam study for the first time and presenting more data with longer follow-up from all three of our LentiGlobin clinical studies."

Recent Highlights

ADVANCED FIRST ONCOLOGY PROGRAM INTO THE CLINIC – Earlier in February, the first patient was infused in the CRB-401 study of bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene has exercised its option to exclusively license bb2121, under the terms of the collaboration agreement between the two companies. bluebird bio will receive a $10.0 million option exercise payment from Celgene and is also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.

PRESENTED UPDATED CLINICAL DATA IN ß-THALASSEMIA FROM HGB-204 AND HGB-205 CLINICAL STUDIES OF LENTIGLOBIN AT ASH (Free ASH Whitepaper) – In December 2015, investigators presented data from bluebird bio’s ongoing clinical studies in transfusion-dependent ß-thalassemia (TDT) and severe sickle cell disease (SCD). Data in patients with TDT from the HGB-204 and HGB-205 studies showed that 100% of patients with non-ß0/ß0 genotypes achieved sustained transfusion independence as of the data cut-off, ranging from 7.1 months to 23.4 months. Patients with the ß0/ß0 genotype all saw reductions in their transfusion needs, ranging from a 33% to 100% reduction.

PRESENTED CLINICAL DATA IN SICKLE CELL DISEASE FROM HGB-205 AND HGB-206 CLINICAL STUDIES OF LENTIGLOBIN AT ASH (Free ASH Whitepaper) –Marina Cavazzana, M.D., Ph.D., of Hospital Necker, University Paris Descartes, presented updated data on one patient with SCD from the HGB-205 study, who remained free of transfusions, hospitalizations and acute SCD-related events for more than nine months as of the data cut-off. At the 12-month post-drug infusion follow-up, the proportion of anti-sickling hemoglobin in this patient accounted for 49% (47% HbAT87Q + 2% HbF) of all hemoglobin production – well above the 30% threshold anticipated to achieve a disease-modifying effect. John Tisdale, M.D., of the National Institutes of Health presented early data from the HGB-206 study, in which two patients had at least three months of post-infusion follow-up. At the three-month post-infusion follow-up for Subject 1301, anti-sickling hemoglobin accounted for 17% of all hemoglobin production (4% HbAT87Q + 13% HbF). At the six-month post-infusion follow-up for subject 1303, anti-sickling hemoglobin accounted for 16 percent of all hemoglobin production (12% HbAT87Q + 4% HbF).

PRESENTED PRE-CLINICAL AND MANUFACTURING DATA FROM CAR T ONCOLOGY PROGRAMS AT ASH (Free ASH Whitepaper) – bluebird bio scientists presented three posters at ASH (Free ASH Whitepaper), covering critical basic research, translational and manufacturing aspects of the Company’s T cell oncology programs. One poster discussed an important observation made by bluebird bio scientists: culturing anti-BCMA CAR T cells with a PI3K inhibitor generated a product with many of the properties of younger, less differentiated T cells. Consistent with a younger T cell phenotype, this product showed improved in vivo efficacy and persistence in multiple model systems.
SHARED DATA ON PLATFORM IMPROVEMENTS – In an investor event at ASH (Free ASH Whitepaper), bluebird bio chief scientific officer Philip Gregory, D.Phil., presented data from ongoing research to improve the cell transduction process for LentiGlobin. The presentation showed that in preclinical experiments, adding selected compounds to the transduction process resulted in substantially increased vector copy number and transduction efficiency (i.e. percentage of corrected cells). Importantly, the new process was shown to be robust with similar improvements seen across multiple donors and vector lots.

ENTERED INTO CAR T LICENSE WITH VIROMED – Signed exclusive license agreement with Viromed Co., Ltd., to research, develop and commercialize CAR T therapies using Viromed’s proprietary humanized antibody to an undisclosed cancer target in solid tumors.

Upcoming Anticipated Milestones

Presentation of interim data from the Starbeam study of Lenti-D in patients with cerebral adrenoleukodystrophy (CALD) at the American Academy of Neurology annual meeting in April 2016

Update on LentiGlobin process improvements in the second half of 2016

Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016

Presentation of updated data from the HGB-204, HGB-205 and HGB-206 studies at the ASH (Free ASH Whitepaper) annual meeting in December 2016
Fourth Quarter and Full Year 2015 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2015 were $865.8 million, compared to $492.0 million as of December 31, 2014, an increase of $373.8 million, which was primarily driven by the June 2015 equity financing partially offset by cash used to fund operations.

Revenues: Collaboration revenue was $1.5 million for the fourth quarter of 2015 and $14.1 million for the year ended December 31, 2015, compared to $6.3 million and $25.0 million in the comparable periods in 2014. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.

R&D Expenses: Research and development expenses were $35.7 million for the fourth quarter of 2015 and $134.0 million for the year ended December 31, 2015, compared to $20.5 million and $62.6 million for the comparable periods in 2014. The increase in research and development expenses was primarily attributable to increased employee compensation expense due to increased headcount, in-licensing milestones and fees, and manufacturing and clinical trial-related costs to support our advancing pipeline.

G&A Expenses: General and administrative expenses were $14.4 million for the fourth quarter of 2015 and $46.2 million for the year ended December 31, 2015, compared to $5.3 million and $23.2 million for the comparable periods in 2014. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting and facilities-related costs to support our overall growth.

Net Loss: Net loss was $47.3 million for the fourth quarter of 2015 and $166.8 million for the year ended December 31, 2015, compared to net loss of $19.5 million and $48.7 million for the comparable periods in 2014.

Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $865.8 million as of December 31, 2015 will be sufficient to fund its current operations through 2018.

On February 24, 2016 Kite Pharma, Inc. (Nasdaq:KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous cell therapy (eACT) products for the treatment of cancer, reported the formation of the company’s integrated commercial leadership team with the appointment of three senior-level industry executives (Press release, Kite Pharma, FEB 24, 2016, View Source [SID:1234509179]). The team will be responsible for all aspects of commercial and medical affairs strategy, planning, and analysis for the potential launch of KTE-C19, an investigational engineered T cell therapy for patients with relapsed and/or refractory B cell malignancies.

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The new commercial and medical affairs leadership team includes Diane L. Parks, Senior Vice President, Marketing, Sales & Market Research; Elizabeth A. Faust, Ph.D., Vice President, Medical Affairs; and Kimberly A. Metcalf, Vice President, Customer Engagement, Training & Development. The group will report to Shawn Tomasello, Kite’s Chief Commercial Officer. Dr. Faust will also report to Jeff Wiezorek, M.D., M.S., Senior Vice President of Clinical Development.

"Commercial readiness is a top priority for Kite as we prepare for pivotal data from the ZUMA-1 study and potential U.S. regulatory filing for KTE-C19 this year," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "Such a potentially transformative therapy as KTE-C19 will require a creative and strategic commercialization model to optimize access and impact for patients. We believe this proven and accomplished team, under the direction of Shawn Tomasello, can deliver on this promise."

"It is a privilege to welcome Diane, Elizabeth, and Kimberly to Kite," said Shawn Tomasello, Chief Commercial Officer. "As a group, they bring the range of critical skills and proven capabilities needed to prepare for commercialization. They have been closely involved in the launch of several major oncology therapies, and they understand how markets are reshaped by therapeutic innovations that improve patient outcomes. Our team is already fully engaged in market analysis and strategy development activities."

Diane L. Parks, Senior Vice President, Marketing, Sales & Market Research

Ms. Parks joins Kite from Pharmacyclics, where she was Vice President of Marketing. During her tenure at Pharmacyclics, Ms. Parks led the development and execution of all global marketing strategies for IMBRUVICA. Previously, she held senior leadership roles as Vice President of Sales for Amgen, representing oncology and nephrology products, and Senior Vice President of Specialty Biotherapeutics and Managed Care at Genentech, where she led the launches of multiple products as well as commercial development of LUCENTIS and RITUXAN. Ms. Parks began her career at Marion Merrell Dow. She holds a BS from Kansas State University and an MBA from Georgia State University.

Elizabeth A. Faust, Ph.D., Vice President, Medical Affairs

Prior to joining Kite, Dr. Faust was Vice President of Medical Affairs at Pharmacyclics, during which time she planned and successfully executed four IMBRUVICA launches for various indications. Before Pharmacyclics, Dr. Faust was Vice President of Clinical Sciences Research at Celgene Corporation, serving as the national lead for oncology Regional Medical Liaisons in the US, and as Senior Director of Medical Affairs at Gloucester Pharmaceuticals, which was acquired by Celgene in 2010. From 1995 to 2007, Dr. Faust held roles of increasing responsibility in research, development, and medical affairs at Amgen, including Executive Director, Oncology Regional Medical Liaisons. Dr. Faust has co-authored numerous publications and was the former President-Elect for the Pacific Southwest Chapter of the American Medical Writers Association. She holds a BS in microbiology from Auburn University, a MA in biology from University of California Riverside, and a PhD in microbiology and molecular genetics from University of California Los Angeles.

Kimberly A. Metcalf, Vice President, Customer Engagement, Training & Development

Ms. Metcalf most recently served as Vice President of Commercial Training and Administration at Pharmacyclics, where she was responsible for developing and executing commercial and medical affairs training including launch plans for IMBRUVICA. Previously, she spent eight years at Celgene Corporation where she contributed significantly to building the commercial training department and assumed roles of increasing responsibility in commercial training, market access, and sales and marketing operations, including Executive Director, US National and Regional Accounts. During her tenure at Celgene, she launched POMALYST, ISTODAX and OTEZLA. Ms. Metcalf began her career in biopharma at Pfizer. She holds a BS from Northwestern University, an MBA from West Chester University, and a Master of Health Systems from The University of Medicine & Dentistry of New Jersey.

About KTE-C19

KTE-C19 is an investigational therapy in which a patient’s T cells are genetically modified to express a CAR designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias. Kite is currently enrolling four pivotal studies (also known as ZUMA studies) for KTE-C19 in patients with various B cell malignancies. The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation status to KTE-C19, for the treatment of patients with refractory diffuse large B cell lymphoma, primary mediastinal B cell lymphoma, and transformed follicular lymphoma. KTE-C19 has also secured Orphan Drug Designation in the U.S. for DLBCL and in the EU for various hematological indications.