Agios Reports Third Quarter 2017 Financial Results

On November 1, 2017 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the third quarter ended September 30, 2017. In addition, Agios highlighted select corporate milestones and preclinical and clinical data from its development programs (Press release, Agios Pharmaceuticals, NOV 1, 2017, View Source [SID1234521379]).

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"We achieved two key 2017 goals in the third quarter with the approval and launch of IDHIFA with our partner Celgene for patients with IDH2m R/R AML and the design completion of the AG-348 pivotal program in PK deficiency," said David Schenkein, M.D., chief executive officer at Agios. "We are now focused on completing the NDA for our first wholly owned product, ivosidenib for IDH1m R/R AML and will present the core data from the submission next month at ASH (Free ASH Whitepaper). In addition, the year-end submission of our IND for AG-270 targeting MTAP-deleted tumors remains on track, highlighting our commitment to remain a research-focused organization pursuing novel science with the potential to change patients’ lives."
THIRD QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS

• The U.S. Food and Drug Administration (FDA) granted Celgene full approval of IDHIFA (enasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved diagnostic test. IDHIFA, an oral targeted inhibitor of the IDH2 enzyme, is the first and only FDA-approved therapy for patients with R/R AML and an IDH2 mutation.

• Finalized two global, pivotal trial designs evaluating AG-348 in adults with pyruvate kinase (PK) deficiency:

• A randomized, placebo-controlled trial with a 1:1 randomization expected to enroll approximately 80-100 non-transfusion dependent patients. The primary endpoint of the study is the proportion of patients who achieve at least a 1.5 gram per deciliter (g/dL) increase in hemoglobin.

• A single arm trial of approximately 20 regularly transfused patients with a primary endpoint of reduction in transfusion burden over six months.
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• Presented the first preclinical data for AG-881 in IDHm solid and hematologic malignancies at the AACR (Free AACR Whitepaper)-NCI-EORTC 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) in October. The data support that AG-881 potently suppresses 2-hydroxyglutarate (2-HG) production by both IDH1 (isocitrate dehydrogenase-1) and IDH2 mutant proteins in biochemical, cell-based and in vivo systems.
FOURTH QUARTER 2017 DATA PRESENTATIONS
IDH Mutant Inhibitors:

• Updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at the 2017 Society for NeuroOncology Annual Meeting on November 17 in San Francisco.

• First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper) on December 9-12 in Atlanta.

• First data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy ("7 +3" and consolidation) in patients with newly diagnosed AML with an IDH2 or IDH1 mutation at ASH (Free ASH Whitepaper).

• First data from the ongoing Phase 1/2 combination trial of enasidenib or ivosidenib with VIDAZA in patients with newly diagnosed AML with an IDH2 or IDH1 mutation ineligible for intensive chemotherapy at ASH (Free ASH Whitepaper).
Rare Genetic Diseases:

• Updated data from the AG-348 Phase 2 DRIVE PK study in PK deficiency at ASH (Free ASH Whitepaper).

• Updated data from the PK Deficiency Natural History Study being conducted with Boston Children’s Hospital at ASH (Free ASH Whitepaper).
KEY UPCOMING MILESTONES
The company expects to achieve the following milestones:

• Submit an NDA (New Drug Application) to the U.S. FDA for ivosidenib for IDH1m positive R/R AML by the end of 2017.

• Submit an Investigational New Drug (IND) application for AG-270, the development candidate targeting MTAP-deleted tumors, by the end of 2017.

• Initiate two global, pivotal trials of AG-348 in PK deficiency in the first half of 2018.

• Initiate a global registry for adult and pediatric patients with PK deficiency in the first half of 2018. The registry will include approximately 60 sites in 20 countries and will follow patients for at least two years.

• Initiate a perioperative ‘window’ study with ivosidenib and AG-881 in low grade glioma to further investigate their effects on brain tumor tissue in the first half of 2018.
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THIRD QUARTER 2017 FINANCIAL RESULTS
Revenue for the quarter ended September, 30, 2017 was $11.4 million, which includes $10.7 million of collaboration revenue and $0.7 million of royalty revenue from net sales of IDHIFA. Revenue for the comparable period in 2016, was $9.0 million. Revenue increased compared to the prior year period primarily due to reimbursement by Celgene of our share of the commercialization effort for IDHIFA and the IDHIFA royalty revenue.
Research and development (R&D) expense was $72.9 million, including $7.6 million of stock-based compensation expense, for the quarter ended September 30, 2017, compared to $60.6 million, including $7.9 million in stock-based compensation expense, for the quarter ended September 30, 2016. The increase in R&D expense was primarily attributable to the ivosidenib program, including activities needed to prepare for a potential NDA submission in 2017, start-up costs for the Phase 3 AGILE clinical trial, and on-going site activation and patient enrollment of the Phase 3 ClarIDHy clinical trial. R&D expense also increased compared to the quarter ended September 30, 2016 due to IND enabling activities for AG-270 as well as ongoing research efforts across our discovery platform programs.
General and administrative (G&A) expense was $17.5 million, including $4.6 million stock-based compensation expense, for the quarter ended September 30, 2017, compared to $11.9 million, including $4.2 million of stock-based compensation expense, for the quarter ended September 30, 2016. The increase in G&A expense was attributed to an increase of $5.7 million related to support our growing commercial organization for the launch of IDHIFA and the potential launch of ivosidenib in 2018.
Net loss for the quarter ended September 30, 2017 was $77.1 million, compared to a net loss of $62.8 million for the quarter ended September 30, 2016.
Cash, cash equivalents and marketable securities as of September 30, 2017 were $641.7 million, compared to $573.6 million as of December 31, 2016. The increase in cash was driven by net proceeds of $270.2 million from the April financing, $12.3 million of cost reimbursements related to our collaboration agreements with Celgene and $12.4 million received from employee stock transactions. This was offset by expenditures to fund operations of $226.4 million during the nine months ended September 30, 2017.
The company expects that its cash, cash equivalents and marketable securities as of September 30, 2017, together with anticipated interest income, anticipated expense reimbursements, and royalty payments under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2019.
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CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2017 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 99771605. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

Unum Therapeutics Announces Presentation of Initial Clinical Data from the ATTCK-20-2 Trial and Pre-Clinical Data on its Antibody-Coupled T Cell Receptor (ACTR) Platform at the Upcoming 59th American Society of Hematology (ASH) Annual Meeting

On November 1, 2017 Unum Therapeutics, a clinical-stage biopharmaceutical company focused on the development of novel immunotherapy products designed to harness the power of a patient’s immune system to cure cancer, reported that initial clinical data from the ATTCK-20-2 Phase I study and pre-clinical data on its Antibody-Coupled T cell Receptor (ACTR) platform will be presented in an oral presentation and a poster presentation, respectively, at the 59th ASH (Free ASH Whitepaper) Annual Meeting, which is being held in Atlanta, GA December 9-12, 2017 View Source The oral presentation will highlight early data from an ongoing Phase I dose escalation study, ATTCK-20-2 (ClinicalTrials.gov No. NCT02776813), of ACTR087 used in combination with rituximab in subjects with relapsed/refractory CD20+ B cell Non-Hodgkin lymphoma. In the first dose level studied (Cohort 1), ACTR087 used in combination with rituximab induced two complete responses (CR) and one partial response (PR) in patients evaluable for response (n=6), with no ACTR087-related serious adverse events (SAEs), no adverse events (AEs) leading to treatment discontinuation, no cytokine-release syndrome (CRS), and no neurotoxicity. The Company is currently enrolling and treating patients in Cohort 2. The poster presentation will provide data from non-clinical studies demonstrating the adaptability of ACTR T Cells to target T cell targets such as CD38 via combination with daratumumab, and bypass the challenges of scFv-based CAR-T cell production to effectively target CD38-expressing tumor cells.

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Presentation Details:

Oral Presentation

Presentation Title: ACTR087, Autologous T Lymphocytes Expressing Antibody Coupled T-cell Receptors (ACTR), Induces Complete Responses in Patients with Relapsed or Refractory CD20-Positive B-cell Lymphoma, in Combination with Rituximab
Authors (affiliation): L. Akard (IBMT), S. Jaglowski (OSU), S. Devine (OSU), M. McKinney (Duke), M. Vasconcelles, H. Huet, S. Ettenberg, A. Ranger, J. Abramson (MGH)
Presenter: L. Akard (IBMT)
Presentation Date: Monday, December 11, 2017
Presentation Time: 7:45 AM
Location: Georgia World Congress Center, Bldg A, Lvl 4, A411-A412
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Immune-Based Therapeutic Approaches
Abstract Number: 580

Poster Presentation

Presentation Title: Adaptability of Antibody-Coupled T Cell Receptor (ACTR) Engineered Autologous T Cells in Combination with Daratumumab Over CAR-based Approaches
Authors: Taylor Hickman, Adrianna Graziano, Katie O’Callaghan, Ryan Boomer, Eugene Choi, Allison Nelson, Greg Motz, Jessica Sachs, Birgit Schultes, Seth Ettenberg, and Tooba Cheema
Presenter: Taylor Hickman, Senior Associate Scientist, Unum Therapeutics
Presentation Date: Sunday, December 10, 2017
Presentation Time: 6:00 PM – 8:00 PM
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Session: 703. Adoptive Immunotherapy: Poster II
Abstract Number: 3189

The poster will be posted on Unum’s website following the presentations.

About Antibody-Coupled T cell Receptor (ACTR) Technology

Unum’s proprietary ACTR is a chimeric protein that combines components from proteins normally found on two different human immune cell types – natural killer (NK) cells and T cells – to create a novel approach to cancer cell killing. T cells bearing the ACTR receptor protein can be directed to attack a tumor by combining with a monoclonal antibody that binds antigens on the cancer cell surface.

In contrast to other T cell therapy approaches for cancer that are limited to a single cancer cell surface target and, therefore, treat a narrow set of tumors, Unum’s approach is not restricted by a specific tumor cell antigen and, thus, we believe may have applications for treating many different types of cancers when combined with the right antibodies.

Unum is developing ACTR in combination with a range of tumor-targeting antibodies for use in both hematologic and solid tumor indications. ACTR087 used in combination with rituximab, an anti-CD20 antibody, is Unum’s most advanced product candidate, currently in Phase I clinical testing for the treatment of adult patients with relapsed/refractory CD20-positive B cell non-Hodgkin lymphoma. The Company has two additional product candidates on track for imminent clinical testing under Investigational New Drug Applications (INDs) in effect with the FDA: ACTR707 used in combination with rituximab for the treatment of adult patients with relapsed/refractory CD20-positive B cell non-Hodgkin lymphoma, and ACTR087 in combination with a novel anti-BCMA antibody, SEA-BCMA, for the treatment of adult patients with relapsed/refractory multiple myeloma.

Gamida Cell to Present New Data from Key Programs at 2017 ASH Annual Meeting

On November 1, 2017 Gamida Cell, a leading cellular and immune therapeutics company, reported that two oral presentations on new data from the company’s pipeline programs will be presented during the 59th Annual Meeting of the American Society for Hematology (ASH) (Free ASH Whitepaper) (Press release, Gamida Cell, NOV 1, 2017, View Source [SID1234521461]).

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The oral presentations will highlight safety and efficacy data for a completed phase 2 study of the company’s lead clinical asset, NiCord, as a single cord blood graft, and proof-of-concept data for the advancement of natural killer cells (NK cells) as an immunotherapeutic modality for patients with cancer. These abstracts are now available on the ASH (Free ASH Whitepaper) conference website.

Oral Presentations

NiCord Single Unit Expanded Umbilical Cord Blood Transplantation (UCBT): Final Results of a Multicenter Phase I/ II Trial

Date & Time: Monday, December 11, 2017 at 4:30 p.m. ET

Session Name: Clinical Allogeneic Transplantation: Results: Donor Selection in the Haplo Transplant Era

Abstract Number: 847

Location: Georgia World Congress Center, Building C, Level 2, Room C202-204

Enhanced In Vivo Persistence and Proliferation of NK Cells Expanded in Culture with the Small Molecule Nicotinamide: Development of a Clinical-Applicable Method for NK Expansion

Date & Time: Monday, December 11, 2017 at 11:00 a.m. ET

Session Name: Cell Collection and Processing: Optimization of Hematopoietic Graft Using Small Molecules in Culture

Abstract Number: 657

Location: Georgia World Congress Center, Building B, Level 2, Room B216-217

Vedanta Biosciences to Advance Cancer Immunotherapy Candidates Based on Microbiome-Derived Live Biotherapeutics that Induce CD8+ T Cells

On November 1, 2017 Vedanta Biosciences, an affiliate of PureTech Health (LSE: PRTC) developing a new category of therapies for immune and infectious diseases based on rationally designed consortia of human microbiome-derived bacteria, reported that it has exclusively sub-licensed key intellectual property from JSR Corporation to develop and commercialize microbiome-derived cancer immunotherapies based on live biotherapeutics (Press release, Vedanta Biosciences, NOV 1, 2017, View Source [SID1234521457]). These live biotherapeutics have been shown to activate CD8+ T cells, a type of white blood cell that is the predominant effector in cancer immunotherapy. The sub-licensed intellectual property is based on the pioneering research of Dr. Kenya Honda, Professor, of Keio University School of Medicine and his collaborators in the University of Tokyo in Japan. An IND filing for the lead product candidate is planned in 2018.

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“Dr. Honda’s research suggests an entirely new approach to cancer immunotherapy harnessing the human microbiome, which has the potential to significantly enhance the effectiveness of cancer therapies,” said Bernat Olle, PhD, Chief Executive Officer of Vedanta Biosciences. “We are actively developing product candidates containing defined bacterial consortia that activate CD8+ T cells and stimulate the immune system to fight cancer alone and in combination with checkpoint inhibitors. This license to additional intellectual property from Dr. Honda’s institution further strengthens Vedanta’s leading IP position in the microbiome field.”

In validated pre-clinical models of cancer, Vedanta’s orally administered product candidates containing defined bacterial consortia induced CD8+ T cells, potentiating the immune system’s attack of tumor cells. The tumor fighting effects of a variety of checkpoint inhibitors were significantly amplified when combined with the bacterial consortia, suggesting a potentially novel approach for combination cancer immunotherapy.

About Key Intellectual Properties
Keio University and The University of Tokyo joined the Leading Advanced Projects for Medical Innovation (“LEAP”), part of Advanced Research and Development Programs for Medical Innovation at The Japan Agency for Medical Research and Development (“AMED”), in 2016. Their research and development is focused on the development of therapeutic cocktails of bacteria isolated from the gut microbiota. The licensed intellectual properties were developed as a part of the AMED-LEAP Research Program and are exclusively licensed to JSR Corporation (TSE: JSR) from Keio University and The University of Tokyo.

New Data of Emicizumab Presented at The American Society of Hematology 2017

On November 1, 2017 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that oral and poster presentations will be given with regard to emicizumab at The American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 in Atlanta, Georgia, United States. Emicizumab is a bispecific antibody under development for hemophilia A (Press release, Chugai, NOV 1, 2017, View Source [SID1234521451]).

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Data from additional six-month follow-up of global Phase lll studies in hemophilia A with inhibitors to factor VIII, HAVEN 1 study (NCT02622321) and HAVEN 2 study (NCT02795767), will be shown at the conference. The HAVEN 2 will be presented at 7:30 EST on December 9 as part of the official Press Program at ASH (Free ASH Whitepaper). Both studies have been conducted in collaboration with Roche and Genentech, while HAVEN 1 is for adults and adolescents and HAVEN 2 is for children.

In addition, preliminary data from HAVEN 4 study (NCT03020160), a Phase lll study with hemophilia A patients with or without inhibitors which examines emicizumab prophylaxis administered subcutaneously once every four weeks, and real-world data from a non-interventional trial in children under 12 years of ages with hemophilia A with inhibitors will be presented.