On November 6, 2015 CTI BioPharma Corp. (CTI) (NASDAQ and MTA: CTIC) reported that data highlighting two compounds (pacritinib and tosedostat) from the company’s investigational pipeline will be presented at the upcoming 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held December 5-8 in Orlando, FL – including three oral presentations (Press release, CTI BioPharma, NOV 5, 2015, View Source;p=RssLanding&cat=news&id=2107857 [SID:1234508044]).

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The presentations will include data analyses from the Phase 3 PERSIST-1 trial of pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R that is currently under investigation for the treatment of patients with myelofibrosis and acute myeloid leukemia (AML) as well as findings from a Phase 2 study of tosedostat, an investigational oral selective inhibitor of aminopeptidases that has demonstrated anti-tumor responses in blood-related cancers and solid tumors in Phase 1-2 clinical trials.

"We look forward to the presentation of data for both pacritinib and tosedostat at this year’s ASH (Free ASH Whitepaper) annual meeting," said James A. Bianco, M.D., President and CEO of CTI BioPharma. "With six abstracts accepted, the data involving two of our investigational compounds is a further example of our focused commitment on advancing the development of our pipeline candidates."

A summary of the oral and poster presentations are below, and full abstracts can be accessed on the ASH (Free ASH Whitepaper) website at www.hematology.org.

Oral Presentations

Analysis of Outcomes by Patient Subgroups in Patients with Myelofibrosis Treated with Pacritinib vs Best Available Therapy (BAT) in the Phase III PERSIST-1 Trial
First Author: Alessandro M Vannucchi, M.D., Associate Professor of Hematology, University of Florence, Italy
Date/Time: Saturday, December 5 at 10:15 a.m. ET
Location: W224, Level 2
Oral Session: 634. Myeloproliferative Syndromes: Clinical: Early and Late Stage Trials in MPN
Abstract #58

Pacritinib, a Dual FLT3/JAK2 Inhibitor, Reduces IRAK-1 Signaling in Acute Myeloid Leukemia
First Author: Megan M Cleary, B.S., B.A, Knight Cancer Institute, Division of Hematology and Medical Oncology, Oregon Health & Science University, Portland, OR
Date/Time: Monday, December 7 at 11:45 a.m. ET
Location: Room W109
Oral Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Novel Targeting Approaches
Abstract #570

Tosedostat Plus Low Dose Cytarabine Induces a High Rate of Responses That Can be Predicted By Genetic Profiling in Elderly AML
First Author: Giuseppe Visani, M.D., Director of Hematology and Stem Cell Transplant Center at AORMN, Pesaro, Italy
Date/Time: Sunday, December 6 at 5:30 p.m. ET
Location: Room W109
Oral Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: New Drugs
Abstract #329

Poster Presentations

Relationship Between Patient-Reported Outcomes (PROs) and Health-Related Quality of Life (HRQoL) and Efficacy in Patients with Myelofibrosis in the Phase III PERSIST-1 Trial of Pacritinib Vs. Best Available Therapy (BAT)
First Author: Ruben Mesa, M.D., Chair, Hematology and Medical Oncology Division Mayo Clinic, Scottsdale, AZ
Date/Time: Saturday, December 5 at 5:30-7:30 p.m. ET
Location: Hall A, Level 2
Poster Session: 634. Myeloproliferative Syndromes: Clinical: Poster I
Abstract #1609

Targeting JAK2 by Gene Knockout or Pacritinib Treatment Reduces GVHD and Xenograft Rejection by Promoting Induced Treg Differentiation
First Author: Brian C. Betts, M.D., Medical Oncologist and Assistant Member of the Department of Blood and Marrow Transplant, Moffitt Cancer Center, Tampa, FL
Date/Time: Saturday, December 5 at 5:30-7:30 p.m. ET
Location: Hall A, Level 2
Poster Session: 702. Experimental Transplantation: Immune Function, GVHD and Graft-versus-Tumor Effects
Abstract #1874

Pacritinib (PAC) Synergistically Potentiates Azacitidine (5AZA) Cytotoxicity in Chronic Myelomonocytic Leukemia (CMML)
First Author: Yan Ma, M.D., H. Lee Moffitt Cancer Center, Tampa, FL
Date/Time: Saturday, December 5 at 5:30-7:30 p.m. ET
Location: Hall A, Level 2
Poster Session: 636. Myelodysplastic Syndromes – Basic and Translational Studies: Poster I
Abstract #1658

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis, including but not limited to patients with disease-related thrombocytopenia, patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy, or patients who are intolerant of, or whose symptoms are sub-optimally managed on other JAK2 inhibitor therapy. The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Pacritinib does not have regulatory approval and is not commercially available.

CTI BioPharma and Baxalta (NYSE:BXLT) are parties to a worldwide license agreement to develop and commercialize pacritinib. CTI BioPharma and Baxalta will jointly commercialize pacritinib in the U.S. while Baxalta has exclusive commercialization rights for all indications outside the U.S.

About Tosedostat

Tosedostat is an investigational oral aminopeptidase inhibitor that has demonstrated anti-tumor responses in blood-related cancers and solid tumors in Phase 1-2 clinical trials. Tosedostat is currently being evaluated in multiple Phase 2 clinical trials for the treatment of patients with AML or high-risk MDS. Tosedostat is not approved or commercially available.

(Filing, 10-Q, Provectus Pharmaceuticals, NOV 5, 2015, View Source [SID:1234507974])

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Sunesis has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Sunesis, NOV 5, 2015, View Source [SID1234508060]).

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On November 05, 2015 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported an interim data analysis showing that its INO-3112 DNA-based immunotherapy generated specific T-cell responses and was well tolerated in all evaluable patients with head and neck cancer associated with human papillomavirus (HPV) types 16 and 18 (Press release, Inovio, NOV 5, 2015, View Source;in-Patients-With-Head–Neck-Cancer/default.aspx [SID:1234507968]).

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The immunology results show that INO-3112 generated robust HPV16/18 specific CD8+ T cell responses and antibodies against HPV16/18 in all 10 tested patients who received all treatments. These results will be presented today and tomorrow at the 30th Anniversary Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in National Harbor, MD and on Nov 20-22 at the European Society for Medical Oncology Symposium on Immuno-Oncology in Lausanne, Switzerland.

INO-3112, an active immunotherapy targeting HPV 16/18 combined with a DNA plasmid for IL-12 as an immune activator, is designed to activate patient’s immune responses to specifically kill HPV associated tumors. In this phase I/IIa study, patients with HPV positive head and neck cancer received INO-3112 once every three weeks for a total of four injections.

The characteristics of these immune response data mirror those previously observed in a phase II clinical study of VGX-3100 for HPV-associated cervical dysplasia. In that study, strong CD8+ T cell immune responses were positively correlated with achievement of primary and secondary efficacy endpoints. Data from that trial was recently published in a peer-reviewed article in The Lancet. This publication details that VGX-3100 is the first therapy to demonstrate that activated killer T cells induced in the body have the power to clear neoplastic lesions as well as the virus which caused the disease.

Dr. Charu Aggarwal, MD, MPH, Assistant Professor of Medicine, Medical Oncologist at Abramson Cancer Center, University of Pennsylvania, Philadelphia and the principal investigator of this study said, "These results are in line with our hypothesis that DNA immunotherapy would lead to activation of the immune system. We are excited to follow these patients and learn about long-term results with this immunotherapy."

Dr. J. Joseph Kim, Inovio’s President and CEO, said, "These results demonstrate we’re on the right path using our DNA immunotherapies to fight cancer. In immuno-oncology, it’s all about the T cells. Here we show in cancer patients that we can generate antigen-specific CD8+ killer T cell responses, which are essential to an effective immunotherapy."

This open label study is intended to assess the safety, tolerability, and immunogenicity of INO-3112 in up to twenty five adults with HPV-positive head and neck squamous cell carcinoma. The study (NCT02163057) includes patients who are being treated with INO-3112 before and after resection of their tumor as well as patients being treated with INO-3112 after completion of chemotherapy and radiation therapy. This study currently continues patient enrollment at Abramson Cancer Center of University of Pennsylvania in Philadelphia. In August 2015, Inovio licensed INO-3112 to MedImmune, the global biologics research and development arm of AstraZeneca, for an upfront payment of $27.5 million, $700 million in potential development and commercial milestone payments, and royalties on INO-3112 product sales.

About HPV-Caused Head & Neck Cancer

Human papillomavirus (HPV) is the most common sexually transmitted disease in the United States, infecting 79 million Americans. HPV is known to play a major role in the development of head and neck cancers, which include cancers of the oral cavity, oropharynx, nose/nasal passages and larynx. Head and neck cancers associated with HPV account for nearly 3 percent of all cancers in the United States and are twice as prevalent in men as in women. Incidence rates of HPV-caused head and neck cancers have been on the rise, especially HPV-associated oropharyngeal cancer in men, and are expected to continue growing. By 2025, researchers believe that HPV will be the causative factor of 90% of all head and neck cancers.

On November 5, 2015 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported business highlights and financial results for the third quarter ended September 30, 2015 (Press release, Agios Pharmaceuticals, NOV 5, 2015, View Source;p=RssLanding&cat=news&id=2107178 [SID:1234507987]).

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"We have made significant progress this year toward realizing our goal of what’s possible for patients with our IDH inhibitors, by deploying a comprehensive development strategy of speed and breadth with AG-221 and AG-120 in AML and other cancers," said David Schenkein, M.D., chief executive officer at Agios. "In addition, we are pleased to have selected our fifth molecule, AG-519, for clinical development in PK deficiency. This coupled with DRIVE PK, our ongoing Phase 2 study of AG-348, may optimize our potential to help people with this rare genetic disorder."

KEY UPCOMING MILESTONES IN CANCER METABOLISM

Agios anticipates the following milestones from its IDH clinical development programs in collaboration with Celgene:

AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein

Present new data from the ongoing Phase 1 dose-escalation and expansion studies of AG-221 in advanced IDH2-mutant positive hematologic malignancies at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place December 5-8, 2015 in Orlando.

AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein

Present first data from the ongoing Phase 1 dose-escalation trial of AG-120 in advanced IDH1-mutant positive solid tumors in an oral presentation at AACR (Free AACR Whitepaper)-EORTC-NCI AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) on November 8, 2015 in Boston.

Present new data from the ongoing Phase 1 dose-escalation and expansion studies of AG-120 in advanced IDH1-mutant positive hematologic malignancies at the ASH (Free ASH Whitepaper) Annual Meeting.

Initiate a global registration-enabling Phase 3 study in patients with acute myeloid leukemia (AML) harboring an IDH1 mutation in the first half of 2016.

AG-221 and AG-120 front-line AML combination trials

Initiate a Phase 1b combination study of either AG-221 or AG-120 with standard induction (7+3, Ara-C and idarubicin/daunorubicin) and consolidation (Ara-C, or mitoxantrone with etoposide) chemotherapy in newly diagnosed AML patients eligible for intensive chemotherapy by the end of 2015.

Initiate a Phase 1/2 combination study of either AG-221 or AG-120 with VIDAZA (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in the first quarter of 2016.

KEY UPCOMING MILESTONES IN RARE GENETIC METABOLIC DISORDERS

AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency

Present data from the Phase 1 healthy volunteers study of AG-348 and new findings from the Natural History Study of PK deficiency (being conducted with Boston Children’s Hospital) at the ASH (Free ASH Whitepaper) Annual Meeting.
AG-519: a novel, oral activator of PKR for the treatment of PK deficiency

Initiate an integrated single ascending dose (SAD) and multiple ascending dose (MAD) placebo-controlled Phase 1 study in healthy volunteers in the first quarter of 2016.
RECENT DEVELOPMENT UPDATES IN CANCER METABOLISM

Agios has provided the following updates on its clinical development programs in collaboration with Celgene:

AG-221

IDHENTIFY, the Phase 3 study of AG-221, was initiated in October. This is an international, multi-center, open-label, randomized clinical trial designed to compare the efficacy and safety of AG-221 versus conventional care regimens in patients 60 years or older with IDH2 mutant-positive AML that is refractory to or relapsed after second- or third-line therapy. This study is being conducted by Celgene.

The expansion phase of the Phase 1 trial of AG-221 is on track and continues to enroll. It includes four cohorts with 25 patients each and a fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or reinduction treatment, or have relapsed after allogeneic transplantation.
The ongoing Phase 1 trial of AG-221 in IDH2-mutated advanced solid tumors and angioimmunoblastic T-cell lymphoma continues to enroll patients.

AG-120

The expansion phase of the Phase 1 trial of AG-120 is on track and continues to enroll. It includes three expansion cohorts of a total of 175 patients with IDH1-mutated advanced hematologic malignancies, including one cohort with 125 patients with relapsed and/or refractory AML.

The ongoing Phase 1 trial of AG-120 in IDH1-mutated advanced solid tumors continues to enroll.
AG-881: a brain-penetrant, first-in-class, oral, potent pan-inhibitor of the mutated IDH1 and IDH2 proteins

Two Phase 1, open-label, dose-escalation and expansion studies are on track and continue to enroll – the first in advanced IDH mutant-positive solid tumors and the second in patients with advanced IDH mutant-positive hematologic malignancies whose cancer has progressed on a prior IDHm inhibitor therapy.

RECENT DEVELOPMENT UPDATES IN RARE GENETIC DISORDERS OF METABOLISM

AG-348

DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency, is on track and enrolling.
A natural history study of PK deficiency is also ongoing and patient enrollment is on track.

AG-519

Agios selected a fifth molecule for clinical development, AG-519, a novel, oral activator of PKR for the treatment of PK deficiency.

THIRD QUARTER 2015 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of September 30, 2015 were $408.0 million, compared to $467.4 million as of December 31, 2014. The decrease was driven by cash used to fund operating activities of approximately $101.2 million, which was offset by funding of approximately $54.8 million made by Celgene during the nine months ended September 30, 2015 related to our collaboration agreements.

Collaboration revenue was $5.5 million for the third quarter of 2015, compared to $33.9 million for the comparable period in 2014. In July 2014, the company amended its collaboration agreement with Celgene. As a result, for the third quarter of 2014 the company recognized a total of $25.9 million under the previous accounting guidance and upon the modification in addition to $8.0 million in revenue subsequent to the modification through September 30, 2014.

Research and development (R&D) expense was $36.0 million, including $4.9 million of stock- based compensation expense in the third quarter of 2015, compared to $25.5 million, including $1.4 million in stock-based compensation expense for the comparable period in 2014. The increase in R&D expense was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development.

General and administrative (G&A) expense was $9.9 million, including $4.5 million of stock-based compensation expense, in the third quarter of 2015, compared to $5.2 million, including $1.4 million of stock-based compensation expense, for the comparable period in 2014. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the third quarter of 2015 was $40.3 million, compared to net income of $3.7 million for the comparable period in 2014. The third quarter of 2014 includes additional revenue recognition related to the amendment of the company’s collaboration agreement with Celgene.

FINANCIAL GUIDANCE FOR THE FULL YEAR 2015

Agios is reiterating that it expects to end 2015 with more than $350.0 million of cash, cash equivalents and marketable securities. The anticipated year end 2015 cash position does not include any additional program-specific milestone payments. Agios expects that its cash, cash equivalents and marketable securities would be sufficient to fund its operating expenses and capital expenditure requirements until late 2017.