10-Q – Quarterly report [Sections 13 or 15(d)]

bluebird bio has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, bluebird bio, 2017, NOV 1, 2017, View Source [SID1234521378]).

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Kitov Pharmaceuticals Announces Receipt of FDA’s Favorable Response to
NT219’s pre-IND Meeting Package

On November 1, 2017 Kitov Pharmaceuticals (NASDAQ: KTOV; TASE: KTOV), an innovative biopharmaceutical company, reported that it has received the U.S. Food and Drug Administration’s (FDA) response to the NT219’s pre-IND meeting package (Press release, Kitov Pharmaceuticals , NOV 1, 2017, View Source [SID1234521437]). FDA has agreed to the proposed Chemistry Manufacturing and Controls (CMC), preclinical, and clinical development plans for NT219. For the clinical development plan, the FDA agreed with TyrNovo’s proposed development plan to test NT219 in combination with gemcitabine for the treatment of advanced pancreatic cancer. The FDA further agreed that the initial clinical trial with NT219 will be a Phase I/II clinical trial, and that "the overall design of proposed first-in-human trial appears reasonable". The FDA further agreed that one-month animal toxicology studies for NT219 would be sufficient to support the IND and that no toxicology studies of NT219 together with gemcitabine would be necessary.

"We are very pleased that FDA’s Division of Oncology Products has accepted our chemistry, non-clinical, and clinical development plans for TyrNovo’s cancer drug, NT219, and we are moving forward with these development plans. We appreciate FDA’s helpful guidance and look forward to continuing to work with the FDA toward an IND submission, which based on our current development plans, we now expect to submit during the first half of 2019," said Dr. J. Paul Waymack, Chairman of Kitov’s Board and Chief Medical Officer.

Dr. Hadas Reuveni, TyrNovo’s Founder and CTO added: "According to the National Cancer Institute, pancreatic cancer is the fourth leading cause of cancer death in the US and is known to be one of the most aggressive and difficult-to-treat cancer types. NT219 works by overcoming drug resistance and can be combined with various oncology drugs. Our drug’s efficacy was demonstrated in preclinical patient-derived xenograft (PDX) models with various oncology therapies such as chemotherapy agents, EGFR Antibodies, MEK and mTOR inhibitors, and also in combination with immuno-oncology agents such as Keytruda. We will initially be clinically testing NT219 in combination with gemcitabine on advanced pancreatic cancer patients, based on our consistent encouraging results in preclinical PDX models. Our long-term strategy is to develop NT219 in combination with other oncology drugs and for additional oncology indications in collaboration with potential strategic partners, who have expressed solid preliminary interest in NT219."

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CytomX to Present at the Jefferies 2017 London Healthcare Conference

On November 1, 2017 CytomX Therapeutics, Inc. (Nasdaq:CTMX), a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, reported that it will present at the Jefferies 2017 London Healthcare Conference. Sean McCarthy, D.Phil., president and chief executive officer will deliver a corporate overview on November 15, 2017, at 4:40 p.m. BT (Press release, CytomX Therapeutics, NOV 1, 2017, View Source;p=RssLanding&cat=news&id=2313476 [SID1234521423]).

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A live audio webcast of the presentation will be available through the Investors and News section of CytomX’s website. An archived replay will be available for 90 days following the event.

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.