On FEBRUARY 29, 2016 BioMotiv, a drug development accelerator associated with The Harrington Project, and Rutgers, the State University of New Jersey, reported the formation of a new biotechnology startup, Z53 Therapeutics. Z53 Therapeutics aims to develop novel anti-cancer drugs that target tumors with p53 mutations (Press release, Z53 Therapeutics, FEB 29, 2016, View Source [SID:1234509511]).

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Z53 Therapeutics is based on intellectual property exclusively licensed from Rutgers University and SUNY Upstate Medical University in Syracuse, N.Y., which was developed by the laboratory of scientific founder Darren Carpizo, M.D., Ph.D., Surgical Oncologist at The Rutgers Cancer Institute of New Jersey. The work was done in collaboration with the synthetic chemistry laboratories of Rutgers Translational Sciences, headed by S. David Kimball, Ph.D., Associate Vice President Research Translation and Commercialization, and the biochemistry and molecular biology laboratory of Stewart Loh, Ph.D., at SUNY Upstate.

"I am very excited to collaborate with BioMotiv to advance our research from our initial discoveries and mechanistic work to the identification of compounds that can be used in the clinic to target a wide spectrum of tumors with specific p53 mutations", said Dr. Carpizo.

The p53 protein has been called the "the guardian of the human genome" for its central role in suppressing tumor formation. The gene encoding p53 is the most commonly mutated gene in human cancer with a large number of mutations resulting in a defect in the protein’s structure due to an impairment in the ability of the protein to bind zinc (so-called zinc deficient mutant p53). Dr. Carpizo and colleagues discovered that small molecules could restore the normal structure and function to zinc deficient mutant p53. Dr. Carpizo, along with Dr. Loh, went on to elucidate the mechanism of action of these compounds to be a restoration of zinc binding in mutant p53 which they have named "zinc metallochaperones".

Zinc metallochaperones represent a novel class of anti-cancer drugs. "The pharmacologic restoration of structure and function of a mutant protein by the delivery of a metal ion is unprecedented in drug development," said Dr. Carpizo. Together with Drs. Loh and Kimball, the research team has identified novel zinc metallochaperones which will be developed by Z53 Therapeutics. Dr. Carpizo goes on to say, "Targeting mutant p53 with small molecule compounds has been one of the holy grails of drug development, so it is very exciting to be a forefront of this field of research."

Christopher Molloy, Ph.D., Senior Vice President for Research and Economic Development at Rutgers, said, "The rapid progress in identifying a biologically active lead chemical series provides validation for Rutgers’ investment in pre-clinical translational research, which is being led by Dr. Kimball. We are pleased that this agreement provides additional resources to allow Dr. Carpizo and our translational sciences team to advance the research on these promising discoveries."

"The discoveries made by Dr. Carpizo have great promise," said Baiju R. Shah, Chief Executive Officer of BioMotiv. "We are looking forward to partnering with him in developing those discoveries in Z53 Therapeutics."

Dr. Carpizo is a Harrington Scholar-Innovator whose work has been supported by the Harrington Discovery Institute of University Hospitals in Cleveland. His laboratory is also funded by the National Cancer Institute, Sidney Kimmel Cancer Foundation, and the Breast Cancer Research Foundation.

# # #

About BioMotiv
BioMotiv is the mission-driven accelerator associated with The Harrington Project for Discovery & Development, a $250 million national initiative for advancing medicine centered at University Hospitals in Cleveland. The focus is to accelerate breakthrough discoveries from research institutions into therapeutics for patients through an innovative model that efficiently aligns capital and collaborations. The company leverages an experienced team and advisory board to select, fund, and actively manage and advance a portfolio of drug development programs.
Learn more at www.biomotiv.com.

About Rutgers University
Rutgers, The State University of New Jersey, is a leading national research university. Established in 1766 and celebrating a milestone 250th anniversary in 2016, the university is the eighth oldest higher education institution in the United States. More than 67,000 students and 22,000 faculty and staff learn, work, and serve the public at Rutgers locations across New Jersey and around the world. Rutgers University–New Brunswick is the only public institution in New Jersey represented in the prestigious Association of American Universities. Rutgers is a member of the Big Ten Conference and its academic counterpart, the Committee on Institutional Cooperation, a consortium of 15 world-class research universities. Rutgers is among the top 30 universities nationally for total R&D funding and last year achieved an 18.3 percent increase in overall funding for research and sponsored programs over the previous year, from $517.6 million in fiscal year 2014 up to $612.5 million in fiscal year 2015. The Office of Research and Economic Development is a central point for industry to access Rutgers and offers a website designed for the business community, www.businessportal.rutgers.edu.

About SUNY Upstate
SUNY Upstate Medical University in Syracuse, NY, is the only academic medical center in Central New York and the region’s largest employer with 9,460 employees. Affiliated with the State University of New York and anchored by its four colleges—Medicine, Nursing, Health Professions and Graduate Studies (biomedical sciences), Upstate’s mission is to improve the health of the community through education, biomedical research and health care. As a biomedical research enterprise, Upstate focuses on the most prevalent human diseases, including cancer, diabetes, heart disease, nervous system disorders, vision, and infectious diseases. The quest for treatments and cures is built upon expertise in structural, molecular and systems biology. The Upstate University Health System serves 1.8 million people, often the most seriously ill and injured, and includes Upstate University Hospital; Upstate University Hospital at Community Campus; Upstate Golisano Children’s Hospital, and numerous satellite sites across Upstate New York. Research Administration at Upstate, which includes the Offices of Technology Transfer, Sponsored Programs and Clinical Trials, assists companies interested in licensing new technologies or in working with Upstate researchers or clinicians (View Source).

On February 29, 2016 PeptiMimesis reported the inception and launch of its activities to develop a novel class of therapeutic peptides using a breakthrough technology from the University of Strasbourg and INSERM (French Institute of Health and Medical Research) (Press release, PeptiMimesis, FEB 29, 2016, View Source [SID:1234509510]). PeptiMimesis will now apply this cutting edge approach to a first set of 60 targets including the most promising in the chosen therapeutic areas.

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Over the past 2 years, the techtransfer office SATT Conectus supported the team to develop the technology up to the proof of concept. PeptiMimesis was founded by three academic researchers, each having more than 15 years of experience in the field of transmembrane peptides, and Domain Therapeutics, a drug discovery company with a long and successful track record in targeting membrane receptors.

"This new venture highlights the quality of the incubation phase conducted by SATT Conectus and the high standard of research carried out by the University of Strasbourg and INSERM," said Pascal Neuville, CEO of Domain Therapeutics.

"The creation of PeptiMimesis is the culmination of many years of work developing our technology," said Dr Dominique Bagnard, founder. "With my two long – standing collaborators and co – founders, Dr Gérard Crémel and Dr Pierre Hubert, we are pleased to partner with Domain Therapeutics to develop our research, and launch PeptiMimesis on the road to success."

"Domain Therapeutics is ideally positioned to help bridge the gap between a breakthrough technology developed by a nascent company and the expectations of pharma partners. We strongly believe that our expertise will allow PeptiMimesis’ platform to deliver promising and valuable peptides to answer patient needs," said Pascal Neuville.

About therapeutic peptides
There are more than 60 US Food and Drug Administration (FDA) approved peptide medicines currently on the market. This is expected to grow significantly, with approximately 140 peptide drugs in clinical trials and more than 500 therapeutic peptides in preclinical development. In terms of value, the global peptide drug market is predicted to increase from $14.1Bn ( € 12.1Bn) in 2011 to an estimated $25.4Bn ( € 22.7Bn) in 2018, with an underlying increase in novel innovative peptide drugs from $8.6Bn ( € 7.7Bn) in 2011 (60%) to $17.0Bn ( € 15.2Bn) in 2018 (66%).

About transmembrane therapeutic peptides
Peptides possess key competitive advantages over antibodies such as a faster drug discovery process and a reduced manufacturing cost. In addition, the novel class of peptides developed by PeptiMimesis presents lower immunogenicity as they rapidly set within cellular membranes. The peptides also demonstrate an amplified therapeutic efficacy through indirect inhibition of multiple co – receptors and their associated signalling pathways. This innovative approach relies on the disruption of dimerization of membrane receptors using peptides that interfere with the transmembrane sites of oligomerization.

About PeptiMimesis
Created in October 2015, PeptiMimesis is a biopharmaceutical company based in Strasbourg, France dedicated to the development of transmembrane therapeutic peptides. The company’s proprietary platform delivers a set of drug candidates that act on strategic targets in the field of immuno-oncology, oncology and immune diseases. PeptiMimesis’ business model aims at moving forward internal assets up to a significant value inflexion point before out – licensing them to pharma partners. PeptiMimesis is also looking to establish partnerships on its unique technology with Pharma and Biotechs. www.pep timimesis.com

About Domain Therapeutics
Domain Therapeutics is a biopharmaceutical company based in Strasbourg, France and Montreal Canada. Domain is dedicated to the discovery and development of drugs targeting transmembrane receptors, including G Protein – Coupled Receptors (GPCRs), one of the most important classes of drug targets. Domain identifies and develops allosteric modulators and biased ligands through its innovative approach and its distinctive technologies. The company’s business model is to g enerate revenues through drug discovery partnerships while developing its own pipeline of drug candidates for central nervous system disorders and cancer. View Source

On February 29, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported that it has entered into a collaboration agreement with Merck, known as MSD outside the US and Canada, through a subsidiary, to develop and commercialize a novel diagnostic assay to predict response to KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy (Press release, NanoString Technologies, FEB 29, 2016, View Source [SID:1234509311]). Under the terms of the collaboration agreement, NanoString will be responsible for seeking regulatory approval for and commercialization of the diagnostic test. NanoString will be eligible to receive up to $24 million for technology access and near-term milestones, in addition to development funding and other potential regulatory milestone payments.

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Previously, the companies had engaged in a research collaboration to develop an assay to evaluate the potential to predict benefit from KEYTRUDA. The expanded collaboration is for the development and commercialization of the selected gene expression signature on NanoString’s nCounter Dx Analysis System as a diagnostic assay to predict response to KEYTRUDA in multiple tumor types.

"We look forward to working with NanoString on the development of their diagnostic assay to help identify patients who are most likely to benefit from KEYTRUDA in multiple additional tumor types," said Dr. Eric Rubin, vice president, oncology early-stage development, Merck Research Laboratories.

"We are excited to expand our collaboration with Merck to develop this novel assay for predicting response to anti-PD-1 therapies such as KEYTRUDA. We believe this gene signature has the potential to become the basis for a universally available assay that serves as the ‘gold standard’ for informing treatment with immuno-oncology therapies," said Brad Gray, President and Chief Executive Officer of NanoString Technologies. "This collaboration solidifies NanoString’s position as the leader in immuno-oncology biomarker signatures, and builds on our ongoing research collaborations with MD Anderson and the Cancer Immunotherapy Trials Network."

Under the terms of the expanded collaboration agreement, NanoString retains the flexibility to independently develop and commercialize additional indications for the resulting diagnostic assay. In particular, this signature could form the basis for a comprehensive immuno-oncology test with the ability to direct use of multiple therapeutic classes, alone or in combination.

On February 29, 2016 MacroGenics, Inc. (NASDAQ:MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported a corporate progress update and reported financial results for the year ended December 31, 2015 (Press release, MacroGenics, FEB 29, 2016, View Source [SID:1234509310]).

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"I am thrilled with MacroGenics’ progress towards advancing breakthrough biologics and life-changing medicines, and particularly our industry leadership in creating bi-specific molecules," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "There are now six Dual-Affinity Re-Targeting, or DART, molecules based on our proprietary platform, in or entering clinical development — four led by MacroGenics and two led by our collaborators, Janssen Biotech and Pfizer. In addition, we are actively enrolling patients with metastatic breast cancer in SOPHIA, our Phase 3 study of our Fc-optimized HER2 antibody, margetuximab. During 2015, in addition to updating our Phase 1 margetuximab data at the ASCO (Free ASCO Whitepaper) annual meeting, we also reported encouraging initial results from an ongoing Phase 1 study of enoblituzumab, our Fc-optimized B7-H3 antibody, at the SITC (Free SITC Whitepaper) annual meeting. Finally, given the strength of our cash and investments balance, we continue to be well positioned to advance our proprietary pipeline of immunotherapeutic product candidates."

"For 2016, we have already initiated a Phase 1b/2 study combining margetuximab and pembrolizumab in advanced HER2-positive gastric cancer," added Dr. Koenig. "Later this year, we plan to share additional enoblituzumab clinical data and provide clinical updates on multiple DART molecules being evaluated in Phase 1 studies. We also expect to submit an Investigational New Drug (IND) application for MGA012. Over the next few years, we intend to continue to advance at least one additional IND per year."

Pipeline Update

Margetuximab is an Fc-optimized monoclonal antibody that targets the human epidermal growth factor receptor 2, or HER2. Recent highlights include:

Phase 1b/2 Gastric Cancer Study: MacroGenics recently dosed the first patient in a Phase 1b/2 clinical trial of margetuximab in combination with pembrolizumab, an anti-PD-1 therapy, in patients with advanced HER2-positive gastric cancer. Treatment options for these patients are limited and our proposed combination regimen would avoid chemotherapy while exploiting the expected enhanced immune-mediated killing properties of both margetuximab and pembrolizumab. We recently elected to expand the scope of this trial to include centers in both Asia and the United States. This study is being conducted in collaboration with Merck.
SOPHIA Study: The Company’s Phase 3 pivotal study in patients with HER2-positive metastatic breast cancer is ongoing, as the Company continues to initiate sites and enroll patients. This study is evaluating the efficacy of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in approximately 530 patients following progression after at least two lines of previous therapy. The Company is targeting completion of this study in 2018.

B7-H3 Franchise. MacroGenics is developing a portfolio of therapeutics that target B7-H3, a member of the B7 family of molecules involved in immune regulation. The Company is advancing multiple programs that target B7-H3 through complementary mechanisms of action and take advantage of this antigen’s broad expression across multiple solid tumor types. Recent highlights of ongoing clinical programs include:

Enoblituzumab (MGA271): Data from the ongoing monotherapy study of enoblituzumab, an Fc-optimized monoclonal antibody that targets B7-H3, was presented in a late-breaking abstract session at the 2015 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November. Enoblituzumab has been generally well tolerated in patients and has shown encouraging initial single-agent, anti-tumor activity in heavily pre-treated patients, including those with prostate and bladder cancer as well as melanoma. In addition, evidence of T-cell immunomodulatory function has been observed in patients treated with enoblituzumab. The Company has expanded its development program to include two combination studies with either ipilimumab or pembrolizumab.
MGD009: This DART molecule targeting B7-H3 and CD3 is being evaluated in a Phase 1 study across multiple solid tumor types.

DART Product Candidates. There are currently six DART molecules in or entering Phase 1 clinical development, including MGD006 (CD123 x CD3, also known as S80880), MGD007 (gpA33 x CD3), MGD011 (CD19 x CD3, also known as JNJ-64052781), MGD010 (CD32B x CD79B), MGD009 (B7-H3 x CD3) and PF-06671008 (P-cadherin x CD3). The Company expects to submit IND applications for two additional DART molecules in 2017. These two product candidates are:

MGD013: MacroGenics is developing an Fc-bearing DART molecule, MGD013, to simultaneously block two immune checkpoint molecules, PD-1 and LAG-3. The Company has presented promising pre-clinical data demonstrating the activity of a DART molecule with these specificities and expects that this bi-specific combination may be useful for treatment of a wide range of solid tumors and hematological malignancies.
MGD014: In 2015, MacroGenics presented pre-clinical data on MGD014, a DART molecule that is being developed to eliminate latent HIV infection. MGD014 is being developed under a contract awarded to MacroGenics by the National Institute of Allergy and Infectious Diseases for up to $24.5 million. This is the first infectious disease DART program planned for clinical testing.

Beyond MGD013 and MGD014, MacroGenics is generating and evaluating multiple other candidates that target a range of immune regulatory and other molecules using both its DART and Trident platforms, the latter for generating tri-specific molecules.

Corporate Update

Commercial Preparation: Tom Farrell recently joined MacroGenics as Vice President, Market Development and Strategy and will lead the Company’s effort in preparing for commercialization of its lead product candidates. Tom was most recently at Genentech, a Member of the Roche Group, where he was Global Pricing & Market Access Head (Oncology/Hemophilia), and responsible for leading the development and implementation of global pricing and payer strategies for all oncology (including Perjeta and Kadcyla) and hemophilia molecules from early- through late-stage development.
Pfizer’s DART Molecule Advances: MacroGenics’ collaboration partner, Pfizer, recently advanced PF-06671008, a DART molecule that targets P-cadherin and CD3, by submitting an IND application that has been cleared by the FDA. Increased levels of the protein P-cadherin have been reported in various tumors, including breast, gastric, endometrial, colorectal and pancreatic cancers, and is correlated with poor survival of patients.

2015 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and investments as of December 31, 2015 were $339.0 million, compared to $157.6 million as of December 31, 2014. In the first quarter of 2015, MacroGenics closed a global collaboration and license agreement for MGD011 with Janssen Biotech, Inc. and received a $50 million upfront license fee. Johnson & Johnson Innovation – JJDC, Inc. also purchased $75 million of newly issued shares of MacroGenics common stock. In July 2015, MacroGenics completed an equity offering that raised net proceeds of $141 million.

Revenue: Total revenues, consisting primarily of revenue from collaborative agreements, were $100.9 million for the year ended December 31, 2015, compared to $47.8 million for the year ended December 31, 2014. Revenue from collaborative agreements includes the recognition of deferred revenue from payments received in previous periods as well as payments received during the year.

R&D Expenses: Research and development expenses were $98.3 million for the year ended December 31, 2015, compared to $70.2 million for the year ended December 31, 2014. This increase was due primarily to the initiation of SOPHIA, a margetuximab Phase 3 study, and a Phase 1b/2 study of margetuximab in combination with pembrolizumab, increased activity in our pre-clinical immune checkpoint programs, including MGD013, and the initiation of a Phase 1a study of MGD010.

G&A Expenses: General and administrative expenses were $22.8 million for the year ended December 31, 2015, compared to $15.9 million for the year ended December 31, 2014. This increase was primarily due to higher labor-related costs, including stock-based compensation expense and information technology-related expenses.

Net Loss: Net loss was $20.1 million for the year ended December 31, 2015, compared to a net loss of $38.3 million for the year ended December 31, 2014.

Shares Outstanding: Shares outstanding as of December 31, 2015 were 34,345,754.

Financial Guidance: MacroGenics expects that its current cash, cash equivalents and investments, combined with anticipated funding under its strategic collaborations, should fund the Company’s operations into 2018.

On February 29, 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 a corporate update and reported full-year and fourth quarter 2015 financial results for the period ended December 31, 2015 (Press release, Kite Pharma, FEB 29, 2016, View Source [SID:1234509308]).

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"2015 was a year of solid execution and transformation for Kite," noted Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "The development of KTE-C19, our breakthrough immunotherapy candidate for patients with refractory hematological malignancies who have limited or no treatment options, is currently advancing in four Kite-sponsored multi-center clinical studies. We expect interim pivotal data from ZUMA-1, our lead study of KTE-C19, in the second half of this year, positioning us to submit our registrational filing with the FDA by year end. Construction of our commercial manufacturing facility is complete, and commercial planning is currently progressing under the leadership of our Chief Commercial Officer, Shawn Tomasello, and her capable team."

Dr. Belldegrun continued, "We are advancing what we believe is the most robust clinical pipeline of chimeric antigen receptors and T cell receptors for both solid and hematological oncology. This effort is supported by our ongoing collaborations with distinguished academic and industry leaders in immuno-oncology. We expect that our pipeline progress in 2016 will include additional human proof of concept data supporting the potential of our TCRs for the treatment of solid tumors."

Full Year 2015 and Recent Highlights

KTE-C19 Program

Initiated Kite’s lead multi-center study (ZUMA-1) for KTE-C19 for the treatment of aggressive, refractory diffuse large B cell lymphoma (DLBCL), primary mediastinal B cell lymphoma (PMBCL), and transformed follicular lymphoma (TFL). DLBCL is the most common form of non-Hodgkin Lymphoma (NHL).

Announced positive data from the Phase 1 portion of ZUMA-1 and advanced the study to the pivotal phase. The overall safety, efficacy, and biomarker data were generally consistent with previously published data from the National Cancer Institute (NCI).

Initiated three additional pivotal multi-center studies of KTE-C19 for the treatment of relapsed/refractory mantle cell lymphoma (MCL), adult relapsed/refractory acute lymphoblastic leukemia (ALL), and pediatric relapsed/refractory ALL.

Received Breakthrough Therapy Designation for KTE-C19 for the treatment of patients with refractory DLBCL, PMBCL, and TFL.

Secured Orphan Drug Designations in the EU for KTE-C19 for all of Kite’s hematological indications.

Completed Kite’s clinical manufacturing facility that is manufacturing KTE-C19 and that will manufacture future product candidates.

Completed construction of Kite’s commercial manufacturing facility, which is on track for qualification and validation later this year.

Acquisition and Strategic Collaborations

Established European operations with the acquisition of T-Cell Factory, now known as Kite Pharma EU. Through this acquisition, Kite also gained the proprietary TCR-GENErator discovery platform developed by Ton Schumacher, Ph.D., Chief Scientific Officer of Kite Pharma EU.

Expanded clinical and research partnership with the NCI including:
An enhanced Cooperative Research and Development Agreement (CRADA) with the NCI’s Surgery Branch to advance multiple CAR and TCR programs led by Steven Rosenberg, M.D., Ph.D.
A separate CRADA with the NCI’s Experimental Transplantation and Immunology Branch to advance the development of a fully human anti-CD19 CAR product candidate. James Kochenderfer, M.D. is leading the Phase 1 study.

Expanded agreement with the Netherlands Cancer Institute for the exclusive option to license multiple TCR gene sequences for the development and commercialization of immunotherapy candidates targeting solid tumors.

Entered into a research and license agreement with Leiden University Medical Center in the Netherlands to identify and develop TCR product candidates targeting solid tumors that are associated with the human papillomavirus (HPV) type 16 infection.

Partnered with Amgen to develop and commercialize the next generation of novel CAR T cell immunotherapies.

Entered into a collaboration with bluebird bio to advance second-generation TCR cell therapy products to treat HPV-associated cancers.

Secured an exclusive license to Alpine Immune Sciences’ transmembrane immunomodulatory protein (TIPTM) technology for eACT-based products.

Corporate Milestones

Strengthened the organization with the addition of more than 100 new hires across all functions.

Formed Kite’s integrated commercial leadership team, led by Shawn Tomasello, Kite’s Chief Commercial Officer.

Appointed Dr. Franz B. Humer, former Chairman and Chief Executive of Roche Holding Ltd., to Kite’s Board of Directors.

Strengthened Kite’s Scientific Advisory Board with the addition of James Allison, Ph.D. and Padmanee Sharma, M.D., Ph.D., recognized immunotherapy leaders from MD Anderson Cancer Center.

Raised $272.6 million in net proceeds from a public offering in December 2015.

2016 Goals

Announce interim Phase 2 pivotal data from the ZUMA-1 study for the first 50 patients with 90-day follow-up in the second half of 2016.

Submit the KTE-C19 registration filing to the U.S. Food and Drug Administration (FDA) based on interim data from ZUMA-1 study by the end of 2016.

Expand KTE-C19 clinical program to Europe in preparation for a European registration filing in 2017.

Complete the qualification and validation testing of the commercial manufacturing facility in anticipation of pre-approval inspection by the FDA.

Initiate a second series of KTE-C19 studies for additional indications and earlier lines of therapy in DLBCL patients.

Present longer term follow-up KTE-C19 data in patients with refractory aggressive DLBCL from the Phase 1 portion of ZUMA-1.

File investigational new drug (IND) application with the FDA for a TCR product candidate that targets a MAGE antigen expressed on solid tumors.

Advance the Kite-Amgen collaboration resulting in the filing of an IND application with the FDA for a next generation CAR T cell immunotherapy candidate, the first of multiple INDs expected to originate from the collaboration.

Fourth Quarter and Full Year 2015 Financial Results

Revenue was $4.9 million for the fourth quarter of 2015 compared to $0 for the fourth quarter of 2014, and $17.3 million for the full year of 2015, compared to $0 for the full year of 2014. The increase was primarily due to revenue recognized under the Kite-Amgen collaboration.

Research and development expenses were $28.8 million for the fourth quarter of 2015, compared to $7.9 million for the fourth quarter of 2014, and $76.4 million for the full year of 2015 compared to $23.1 million in 2014. The full-year increase of $53.3 million was primarily attributable to a $31.5 million increase in research and development and manufacturing expenses supporting the advancement of KTE-C19 and other programs, $11.8 million in expenses related to increased personnel and consulting costs, and $10.0 million of non-cash stock-based compensation expense.

General and administrative expenses were $14.1 million for the fourth quarter of 2015, compared to $5.4 million for the fourth quarter of 2014, and $44.2 million for the full year of 2015, compared to $13.6 million in 2014. The full-year increase of $30.6 million was primarily attributable to an $8.2 million increase in personnel related expenses, $7.8 million for public company expenses and license obligations, and $14.6 million of non-cash stock-based compensation.

Net loss attributable to common stockholders was $38.2 million, or $0.85 per share, for the fourth quarter of 2015, compared to $13.0 million, or $0.33 per share, for the fourth quarter of 2014. For the full year of 2015, the net loss was $101.7 million, or $2.33 per share, compared to $43.7 million, or $1.91 per share, in 2014.

Non-GAAP net loss attributable to common stockholders for the fourth quarter of 2015 was $24.5 million, or $0.54 per share, which excludes non-cash stock-based compensation expense of $13.8 million for the fourth quarter of 2015. Non-GAAP net loss attributable to common stockholders for the full year of 2015 was $61.0 million, or $1.40 per share, which excludes non-cash stock-based compensation expense of $40.7 million for the full year of 2015.

As of December 31, 2015, Kite had $614.7 million in cash, cash equivalents, and marketable securities, compared to $367.0 million as of December 31, 2014.

Kite expects the full year 2016 net cash burn to be $235 to $250 million dollars, which includes approximately $20 million in capital expenditures, but excludes any inflows or outflows from business development activities. The estimated full year 2016 net cash burn is primarily driven by an estimated net loss of $295 to $310 million, which includes an estimated $80 million of non-cash stock-based compensation expense.