On February 16, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new immunotherapies, reported financial results for the fourth quarter ended December 31, 2016, and provided an update on the Company’s recent activities (Press release, Ziopharm, FEB 16, 2017, View Source [SID1234517763]).

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"ZIOPHARM made significant progress over the course of 2016, putting us on track to enter a pivotal study and prosecute multiple high-value studies across our oncology programs in 2017, focusing on gene therapy, CAR and TCR T-cell therapies, as well as off-the-shelf NK cells," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. "This strategy moves us not just into a registration pathway with Ad-RTS-hIL-12 + veledimex, but clinically validates the broad potential of our technologies, including our switch systems and the Sleeping Beauty non-viral platform. These advances will be applied across our program areas, enabling us to increase control, reduce complexity, and manage costs, addressing some of the most significant needs in gene and cell therapy. Ultimately, the ability to deliver controlled, point-of-care therapy makes individualized treatment targeting each patient’s tumor neoantigens possible, a goal with profound clinical implications."

Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM, added: "In a recently updated presentation of results from our Phase 1, multi-center study of Ad-RTS-hIL-12 + veledimex in patients with recurrent high-grade gliomas, we observed a median overall survival of 12.7 months in the 20mg veledimex dose group. This remains a very encouraging outcome compared to historic controls. We look forward to providing an update on our registration pathway in this indication in the first quarter, and to initiating a pivotal study by the end of 2017."

Program Updates

Ad-RTS-IL-12 + veledimex

Ad-RTS-hIL-12 + veledimex is a gene therapy candidate for the controlled expression of interleukin 12 (IL-12), a critical protein for stimulating an anti-cancer immune response, using the RheoSwitch Therapeutic System (RTS) gene switch. ZIOPHARM is currently conducting a multi-center Phase 1 study of Ad-RTS-hIL-12 + orally-administered veledimex in patients with recurrent or progressive glioblastoma multiforme (GBM), an aggressive form of brain cancer.

Announced End-of-Phase 2 Meeting with the FDA. In its presentation at the 35th Annual J.P. Morgan Healthcare Conference, the Company announced that it was granted an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) to determine a pivotal trial design for Ad-RTS-hIL-12 + veledimex for recurrent GBM. The Company expects to announce the outcome of this meeting in the first quarter of 2017, with the goal of initiating a pivotal clinical trial of Ad-RTS-hIL-12 + veledimex in recurrent GBM in 2017.

Presented Data Demonstrating Survival Benefits in Patients with Recurrent Brain Cancer Treated with Ad-RTS-hIL-12 + Veledimex. In November 2016, ZIOPHARM presented clinical data for Ad-RTS-hIL-12 + veledimex for recurrent brain cancer at the 21st Annual Scientific Meeting of the Society for Neuro-Oncology (SNO) in Scottsdale, Arizona. In the study, recombinant IL‑12, leading to the body’s production of interferon-gamma (IFN-gamma), were measured in the bloodstream and found to be proportional to the three doses of veledimex (20mg, n=7; 30mg, n=4; and 40mg, n=6), demonstrating that this orally-delivered activator crossed the blood-brain barrier to engage the RTS gene switch and express IL-12 and IFN-gamma in a dose-dependent manner. Toxicities were consistent with those previously reported and all related side effects were reversed upon cessation of veledimex. Based on tolerability and survival benefit (median OS=12.7 months, n=15, as of January 6, 2017), 20 mg was selected for an expansion cohort, with patients being followed for overall survival. Updated results from the study have been submitted for presentation at an upcoming scientific meeting.

Presented Results of Pre-Clinical Study Supporting Initiation of New Clinical Trial of Ad-RTS-hIL-12 + Veledimex in Pediatric Brain Tumors. In November 2016, ZIOPHARM presented results from a preclinical study of Ad-RTS-mIL-12 + veledimex as an investigational therapy for pediatric glioma in a poster titled "Local regulated IL-12 expression as an immunotherapy for the treatment of pontine glioma" at the 21st Annual Scientific Meeting of the SNO in Scottsdale, Arizona. In an orthotopic pons model, veledimex was shown to cross the blood-brain barrier to control mouse IL-12 production from the tumor, which stimulated the immune system and resulted in increased overall survival. Based on these results, the Company plans to initiate a Phase 1 clinical trial in pediatric brain tumors, including diffuse intrinsic pontine glioma (DIPG) in 2017.

Presented Data Demonstrating Activation of Anti-Tumor Immune Response Using Ad-RTS-hIL-12 + Veledimex in Patients with Advanced Breast Cancer. In October 2016, ZIOPHARM announced the presentation of preliminary data from the Company’s Phase 1b/2 study of Ad-RTS-hIL-12 + veledimex following standard chemotherapy for the treatment of patients with locally advanced or metastatic breast cancer at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress in Copenhagen, Denmark. Results showed that Ad-RTS-hIL-12 + seven days of veledimex consistently elicited production of IL-12 which in turn produced IFN-gamma. It was notable that the influx of CD8+ T cells making IFN-gamma within the tumor were present five weeks after completion of veledimex, consistent with the ability of Ad-RTS-hIL-12 to favorably impact the tumor environment over the long term. In two patients, Ad-RTS-hIL-12 + veledimex provided a meaningful chemotherapy holiday, with durable responses for 18 and 35 weeks.
Adoptive Cell Therapies

ZIOPHARM is developing various immuno-oncology programs, including chimeric antigen receptor (CAR) T cell (CAR-T), T-cell receptor (TCR) T cell (TCR-T), and natural killer (NK) adoptive cell-based therapies. These programs are being advanced in collaboration with Intrexon Corporation (NYSE:XON), MD Anderson Cancer Center, and Merck Serono, the biopharmaceutical business of Merck KGaA (CAR-T only).

Announced Advances in Point-of-Care Approach for Rapidly Producing CAR-Expressing T Cells Utilizing the Sleeping Beauty System. In January 2017, ZIOPHARM announced improved production times utilizing its non-viral platform to engineer T cells in an ongoing Phase 1 study of second-generation Sleeping Beauty (SB) CD19-specific CAR+ T cells. Plans to progress the Company’s point-of-care (POC) approach with administration of CAR-T cell therapy in less than 2 days are also underway, helping expand access to innovative T-cell therapies.

Cooperative Research and Development Agreement with the National Cancer Institute Utilizing Sleeping Beauty System to Generate T cells Targeting Neoantigens. In January 2017, ZIOPHARM and partner Intrexon announced the signing of a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) for the development of adoptive cell transfer-based immunotherapies genetically modified using the SB system to express TCRs for the treatment of solid tumors. Research conducted under the CRADA will be at the direction of Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI’s Center for Cancer Research.

Results from Four Studies of Adoptive Cell-based Therapeutic Programs Presented at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. In December 2016, ZIOPHARM presented data from the Company’s adoptive cell-based therapeutic programs at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition held December 3-6, 2016 in San Diego. The research, conducted at the MD Anderson Cancer Center and Intrexon, demonstrates, among other results, that T cells can be very rapidly produced with the SB system and that a non-viral approach to gene therapy can be harnessed to generate CAR- and TCR-expressing effector cells.

Announced Publication Demonstrating Membrane-bound IL-15 (mbIL15) Enhanced Persistence of CD19-Specific T Cells. In November 2016, ZIOPHARM announced the publication of data demonstrating enhanced persistence of genetically modified T cells targeting leukemia through utilization of its non-viral SB system to co-express mbIL15 and a CD19-specific CAR. The article, titled "Tethered IL-15 augments antitumor activity and promotes a stem-cell memory subset in tumor-specific T cells," was published in the Proceedings of the National Academy of Sciences and is available online here.

Using the SB system, researchers generated genetically modified T cells that preserved stem-cell memory (TSCM) cells by co-expressing the CAR with mbIL15. Engineered T cells were effective in treating established CD19+ leukemia in mice by facilitating the long-term persistence of TSCM cells sustained by signaling through recombinant IL-15. These findings provide for a translational pipeline of immunotherapies with improved potential by combining mbIL15 and T cells with diverse specificities.

The Company previously announced the publication of data highlighting the benefits of using the non-viral SB system to genetically modify T cells to express a CAR for use against CD19-expressing leukemias and lymphomas. The article, titled "Phase I trials using Sleeping Beauty to generate CD19-specific CAR T cells," was published in the Journal of Clinical Investigation (doi:10.1172/JCI86721), and is available online here.
Anticipated 2017 Milestones

Intra-tumoral IL-12 RheoSwitch programs:
– Clinical data from Phase 1 of Ad-RTS-hIL-12 + veledimex for GBM to be presented at a scientific meeting in 2017
– Initiate pivotal clinical trial for GBM in 2017
– Initiate combination study of Ad-RTS-hIL-12 + veledimex with immune checkpoint inhibitor (PD-1) during the first half of 2017
– Initiate Phase 1 study in the treatment of brain tumors in children during the first half of 2017
CAR-T programs:
– Continue CD19-specific CAR+ T clinical study in 2017 enrolling patients under shortened manufacturing process towards point of care
– Initiate a CD33-specific CAR+ T clinical study for relapsed or refractory AML in 2017
– Advance CAR+ T-cell preclinical studies for at least one hematological malignancy under a shortened manufacturing process towards point of care
TCR-T programs:
– Execute CRADA with NCI utilizing SB to generate T cells targeting neoantigens
NK cell programs:
– Initiate a Phase 1 study of off the shelf NK cells for AML in 2017
GvHD programs:
– Advance preclinical studies in 2017
2017 RBC Global Healthcare Conference

The Company also announced that Dr. Cooper will present at the 2017 RBC Global Healthcare Conference on Thursday, February 23rd, 2017 at 1:35 p.m. ET. The conference will be held at the Lotte New York Palace in New York City.

To access a live audio webcast of the presentation, please visit the Investor Relations section at www.ziopharm.com.

Fourth-Quarter 2016 Financial Results

Net loss applicable to common shareholders for the fourth quarter of 2016 was $14.8 million, or $(0.11) per share, compared to a net loss of $9.5 million, or $(0.07) per share, for the fourth quarter of 2015. The increase in net loss for the three months ended December 31, 2016 is primarily due to a $1.2 million increase in expenses related to the gene therapy and cell therapy programs, decreased collaboration revenue of $0.3 million, an increase in the charge for the change in derivative liabilities of $0.1 million and income attributable to preferred stockholders of $3.5 million.

Research and development expenses were $9.4 million for the fourth quarter of 2016 compared to $8.1 million for the fourth quarter of 2015. The increase in research and development expenses for the three months ended December 31, 2016 is primarily due to a $1.2 million increase in expenses related to the gene therapy and cell therapy programs.

General and administrative expenses were $3.3 million for the fourth quarter of 2016 and 2015.
The Company ended the quarter with cash and cash equivalents of approximately $81.1 million, which the Company believes will be sufficient to fund its currently planned activities into the fourth quarter of 2017. The cash burn is expected to increase substantially during 2017 due to the anticipated initiation of a pivotal trial in GBM.

On February 16, 2017 Cytokinetics, Inc. (Nasdaq:CYTK) reported total revenues for the fourth quarter of 2016 were $33.1 million, compared to $9.8 million, during the same period in 2015. Net income for the fourth quarter was $7.2 million, or $0.18 and $0.16 per basic and diluted share, respectively (Press release, Cytokinetics, FEB 16, 2017, View Source;p=RssLanding&cat=news&id=2246868 [SID1234517762]). This is compared to a net loss for the same period in 2015 of $9.2 million, or $0.24 per basic and diluted share. As of December 31, 2016, cash, cash equivalents and investments totaled $163.9 million. The 2016 year-end cash and cash equivalents does not include $100 million received from a royalty monetization deal with Royalty Pharma that closed in February 2017.

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"2016 culminated with another productive quarter marked by progress and momentum across our pipeline of late-stage muscle biology-directed drug candidates," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "With two drug candidates in Phase 3 clinical trials and a third advancing in multiple Phase 2 clinical trials, we are executing well against our Vision 2020 strategic initiative, alone and in collaboration with our partners. We expect 2017 to be a pivotal year highlighted by the expected results from VITALITY-ALS, our Phase 3 trial of tirasemtiv, as well as key data from our Phase 2 trial of CK-2127107 in patients with spinal muscular atrophy."

Recent Highlights

Cardiac Muscle Program

omecamtiv mecarbil

Announced the start of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil which is being conducted by Amgen, in collaboration with Cytokinetics.

Announced additional results from COSMIC-HF (Chronic Oral Study of Myosin Activation to Increase Contractility in Heart Failure), a Phase 2 trial evaluating omecamtiv mecarbil in patients with chronic heart failure, showing that omecamtiv mecarbil improved left atrial (LA) structure and function in patients with chronic heart failure with reduced systolic function. The results were presented in a Clinical Poster Session at the American Heart Association’s Scientific Sessions 2016 in New Orleans.

Completed enrollment of a Phase 2 clinical trial of omecamtiv mecarbil in Japanese patients with chronic heart failure.
Skeletal Muscle Program

tirasemtiv

Announced the first patient has been enrolled in VIGOR-ALS (Ventilatory Investigations in Global Open-Label Research in ALS), an open-label extension clinical trial designed to assess the long-term safety and tolerability of tirasemtiv, in patients with ALS who have completed their participation in VITALITY-ALS.

Announced new data at the 27th International Symposium on ALS/MND in Dublin, Ireland including:

– Baseline characteristics from VITALITY-ALS showed patients enrolled in VITALITY-ALS are similar to those from BENEFIT-ALS and other recently conducted clinical trials in patients with ALS.

– Results from the first part of a research collaboration to refine and prospectively validate a computer model developed by Origent Data Sciences to predict the course of ALS disease progression analyzing data from the placebo groups of earlier clinical trials in patients with ALS (including BENEFIT-ALS, Cytokinetics’ Phase 2b clinical trial of tirasemtiv) and the open-access Pro-Act database. The analyses showed that the Gradient Boosting Machine (GBM) algorithm was the optimal model to predict slow vital capacity (SVC) at times subsequent to baseline and that forced vital capacity (FVC) records could be used to predict slow vital capacity (SVC) scores of ALS patients using this machine learning technique.

– Results of an international physician survey on the use of non-invasive ventilation (NIV) in the treatment of ALS presented by Terry Heiman-Patterson, M.D., Director of the Center for Neurodegenerative Disorders, and Professor of the Department of Neurology at the Lewis Katz School of Medicine at Temple University, revealed similarities in best practices for initiating NIV in North America and Europe, and differences in the time to initiation.
CK-2127107

Continued enrollment of Cohort 1 in the Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy (SMA), conducted by Cytokinetics in collaboration with Astellas.

Continued enrollment in a Phase 2 clinical trial of CK-2127107 in patients with COPD, conducted by Astellas in collaboration with Cytokinetics.
Pre-Clinical Research

Continued research activities under our joint research program with Amgen directed to the discovery of next-generation cardiac muscle activators and under our joint research program with Astellas directed to the discovery of next-generation skeletal muscle activators. In addition, company scientists continued independent research activities directed to our other muscle biology programs.

Announced the publication of preclinical data characterizing a smooth muscle myosin inhibitor that induces smooth muscle relaxation. The manuscript titled, "Highly Selective Inhibition of Myosin Motors Provides the Basis of Potential Therapeutic Application," published in PNAS, Proceedings of the National Academy of Sciences, illustrates a mechanism of action with potential relevance for diseases of smooth muscle hypercontractility such as asthma and chronic obstructive pulmonary disease.
Corporate

Received a $26.7 million milestone payment from Amgen coincident to the start of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil which is being conducted by Amgen in collaboration with Cytokinetics.

Announced Cytokinetics has been selected for addition to the Nasdaq Biotechnology Index (NBI) as part of the annual re-ranking of the NBI.

Announced that Cytokinetics has agreed to sell to Royalty Pharma a 4.5% royalty on potential worldwide sales of omecamtiv mecarbil for $90 million and $10 million of Cytokinetics common stock.

Announced that Cytokinetics agreed to exercise its option under its collaboration agreement with Amgen to co-invest $40 million in the Phase 3 development program of omecamtiv mecarbil. As a result, Cytokinetics is eligible to receive an incremental royalty of up to 4% on increasing worldwide sales of omecamtiv mecarbil outside of Japan. Exercising its option and co-funding will afford Cytokinetics the right to co-promote omecamtiv mecarbil in institutional care settings in North America, with reimbursement by Amgen for certain sales force activities.
Financials

Revenues for the fourth quarter of 2016 were $33.1 million, compared to $9.8 million during the same period in 2015. Revenues for the fourth quarter of 2016 included $26.0 million from our collaboration with Amgen, and $3.6 million of research and development revenues and $3.2 million of license revenues from our collaboration with Astellas, and $0.3 million in research and development revenues from our collaboration with The ALS Association (ALSA). Revenues from our collaboration with Amgen included $26.7 million in a milestone payment, and $0.6 million in research and development revenue, partially offset by a payment of $1.3 million related to the option to co-fund a Phase 3 clinical trial for an increased royalty percentage. Revenues for the same period in 2015 were comprised of $5.1 million of license revenues and $4.0 million of research and development revenues from our collaboration with Astellas, and $0.6 million of research and development revenues from our collaboration with Amgen.

Total research and development (R&D) expenses for the fourth quarter of 2016 were $18.8 million, compared to $13.2 million for the same period in 2015. The $5.6 million increase in R&D expenses for the fourth quarter of 2016, compared with the same period in 2015, was primarily due to an increase of $4.0 million in outsourced clinical costs mainly associated with VITALITY-ALS, our ongoing Phase 3 trial of tirasemtiv, an increase of $1.2 million in personnel related expenses due to increased headcount costs and increased non-cash stock compensation expense, and an increase of $0.3 million in laboratory expenses, partially offset by a decrease of $0.3 million in outsourced pre-clinical costs.

Total general and administrative (G&A) expenses for the fourth quarter of 2016 were $6.7 million compared to $5.5 million for the same period in 2015. The $1.2 million increase in G&A expenses for the fourth quarter of 2016, compared to the same period in 2015, was primarily due to an increase of $0.8 million in personnel related expenses due to increased headcount and non-cash stock compensation expense and an increase of $0.2 million in outsourced costs related to commercial development, recruitment, consulting, and patent legal fees.

Revenues for the twelve months ended December 31, 2016 were $106.4 million, compared to $28.7 million for the same period in 2015. Revenues for the twelve months of 2016 included $62.2 million of license revenues, $13.1 million of research and development revenue, and $2.0 million of milestone payments from our collaboration with Astellas, and $27.9 million from our collaboration Amgen, and $1.1 million in research and development revenues from our collaboration with ALSA. Revenues from our collaboration with Amgen included $26.7 million in a milestone payment, and $2.5 million in research and development revenue, partially offset by a payment of $1.3 million related to the option to co-fund a Phase 3 clinical trial for an increased royalty percentage. Revenues for the same period in 2015 included $13.9 million of license revenues and $12.2 million of research and development revenues from our collaboration with Astellas, and $2.5 million of research and development revenues from our collaboration with Amgen.

Total R&D expenses for the twelve months ended December 31, 2016 were $59.9 million, compared to $46.4 million for the same period in 2015. The $13.5 million increase in R&D expenses for the twelve months of 2016, over the same period in 2015, was primarily due to an increase of $12.1 million in outsourced clinical costs, $4.5 million in personnel related expenses and non-cash stock compensation expense, and $0.8 million in outsourced research costs, partially offset by a decrease of $4.2 million in outsourced preclinical costs mainly associated with clinical manufacturing activities. The increase in outsourced clinical costs was comprised of an increase of $16.6 million in outsourced clinical costs mainly associated with VITALITY-ALS offset by a $4.5 million litigation settlement in June 2016 from a contract research organization for BENEFIT-ALS, our Phase 2 clinical trial of tirasemtiv which was concluded in 2014.

Total G&A expenses for the twelve months ended December 31, 2016 were $27.8 million, compared to $19.7 million for the same period in 2015. The $8.1 million increase in G&A spending in the twelve months of 2016 compared to the same period in 2015, was primarily due to $4.2 million in personnel related expenses due to increased headcount and non-cash stock compensation expense, an increase of $1.7 million in corporate and patent legal fees, and an increase of $1.7 million in outsourced costs related to commercial development, grants and sponsorships, and accounting and finance and recruitment related costs.

Net income for the twelve months ended December 31, 2016, was $16.5 million, or $0.41 and $0.39 per basic and diluted share, respectively, compared to a net loss of $37.5 million, or $0.97 per basic and diluted share, for the same period in 2015.

2017 Financial Guidance

Cytokinetics also announced financial guidance for 2017. The company anticipates cash revenue will be in the range of $21 to $23 million, cash R&D expenses will be in the range of $108 to $112 million, and cash G&A expenses will be in the range of $30 to $32 million. This guidance excludes approximately $7.0 million in unearned revenue from the 2014 amendment of our collaboration with Astellas, which will be recognized in 2017 under generally accepted accounting principles, as well as any potential future milestones that may be achieved in accordance with our collaboration agreements with our partner Astellas. This guidance also excludes an estimated $8.9 million in non-cash related operating expenses primarily related to stock compensation expense.

2017 Corporate Milestones

Skeletal Muscle Program
Tirasemtiv

Expect results from VITALITY-ALS in Q4 2017.

Expect to continue to enroll patients who complete VITALITY-ALS into VIGOR-ALS, an open-label extension trial throughout 2017.
CK-2127107

Expect data from a Phase 2 clinical trial of CK-2127107 in patients with SMA in 2H 2017.

Expect Astellas to continue enrollment in a Phase 2 clinical trial of CK-2127107 in patients with COPD in 2017.

Expect Astellas to begin a Phase 1b clinical trial of CK-2127107 in elderly patients with limited mobility in 1H 2017.

Expect to begin a Phase 2 clinical trial of CK-2127107 in patients with ALS in mid-2017.
Cardiac Muscle Program

omecamtiv mecarbil

Expect to continue to enroll patients with chronic heart failure in GALACTIC-HF, our Phase 3 clinical trial of omecamtiv mecarbil, throughout 2017.

Expect data from a Phase 2 clinical trial of omecamtiv mecarbil in Japanese patients with chronic heart failure in Q3 2017.
Pre-Clinical Research

Expect to continue research activities under our joint research program with Amgen directed to the discovery of next-generation cardiac muscle activators and under our joint research program with Astellas directed to the discovery of next-generation skeletal muscle activators.

On February 16, 2017 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration (AMD) and fibrotic diseases, reported that it has initiated patient dosing in its Phase 3 TAPPAS (TRC105 And Pazopanib versus Pazopanib alone in patients with advanced AngioSarcoma) trial of TRC105 (Press release, Tracon Pharmaceuticals, FEB 16, 2017, View Source;p=irol-newsArticle&ID=2246642 [SID1234517740]).

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"Angiosarcoma is an aggressive cancer with limited therapeutic options, and we are excited to begin this randomized Phase 3 clinical trial of TRC105 as a potential therapy for patients with this disease," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "TRC105 has orphan drug designation for soft tissue sarcoma in the U.S. and the EU, and the first patient dosed represents the culmination of TRACON’s extensive work with regulators in both regions to design a robust Phase 3 trial. We are encouraged by the activity seen to date in the ongoing Phase 2 trial of TRC105 in combination with Votrient, and look forward to providing further updates as the TAPPAS trial progresses."

About the Phase 3 TAPPAS Study

TRACON is conducting the Phase 3 TAPPAS trial (a randomized Phase 3 trial of TRC105 And Pazopanib versus Pazopanib alone in patients with advanced AngioSarcoma) under Special Protocol Assessment (SPA) with the FDA at sites in the U.S. and Europe. This one-to-one randomized trial of TRC105 in combination with Votrient (pazopanib) versus single agent Votrient features an adaptive enrichment design that allows for greater flexibility and efficiency to identify potential signs of clinical benefit. The trial has an initial enrollment target of 124 patients and, based on an interim analysis, allows for sample size re-estimation up to a maximum of 200 patients, as well as enrichment of potentially more responsive patients with cutaneous angiosarcoma. The primary endpoint is progression-free survival, with overall survival as a secondary endpoint.

Further details of the study are available on www.clinicaltrials.gov under NCT02979899.

About TRC105

TRC105 is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis, the process of new blood vessel formation. TRC105 is currently being studied in multiple Phase 2 clinical trials sponsored by TRACON or the National Cancer Institute (NCI) for the treatment of solid tumors in combination with VEGF inhibitors. TRC105 has received orphan designation for the treatment of soft tissue sarcoma in both the U.S. and EU. The ophthalmic formulation of TRC105, DE-122, is currently in a Phase 1/2 trial for patients with wet AMD. TRC205, a second generation antibody to endoglin, is undergoing preclinical testing in models of fibrosis.