On November 5, 2015 Cellectis (Alternext: ALCLS – Nasdaq: CLLS) reported that Great Ormond Street Hospital (GOSH) and University College London (UCL) will present encouraging data from a first in man clinical use of UCART19, at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando during the poster session (Press release, Cellectis, NOV 5, 2015, View Source [SID:1234507990]).

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GOSH has treated in June 2015 a young leukemia patient under a special license from the Medicines & Healthcare products Regulatory Agency(MHRA) with Cellectis’ TALEN gene edited allogeneic UCART19 product candidate because no other therapies were available for refractory relapsed Acute Lymphoblastic Leukemia (ALL) following mismatched allogeneic stem cell transplantation.

In response to an unsolicited request from Professor Waseem Qasim, Consultant Immunologist at GOSH and Professor of Cell and Gene Therapy at University College London (UCL) Institute of Child Health, Cellectis gave its approval for the use of its UCART19 product candidate and technologies under GOSH’s "Specials" license and responsibility, for the particular clinical needs of that individual patient.

Professor Qasim says: "The successful treatment of a patient with UCART19 cells represents a landmark in the use of new gene engineering technology. If replicated in other patients, it could represent a huge step forward in treating leukaemia and other cancers."

"We are very glad for this young patient to have benefited from our highly innovative TALEN gene edited allogeneic CAR T therapy UCART19. We expect to accelerate our clinical development of TALEN gene-edited allogeneic CAR-T therapies to further confirm this encouraging clinical proof of concept," said Doctor Mathieu Simon, MD, Executive Vice President, Chief Operating Officer at Cellectis.

"Our team aims to provide to patients, with unmet medical needs, access to the first allogeneic CAR-T therapy, UCART19 made with Cellectis’ TALEN gene-editing technologies," said Doctor André Choulika, Founder, Chairman and Chief Executive Officer of Cellectis. "Cellectis had, is and will invest significant amounts of energy and creativity to provide cancer patients with an accessible, cost-effective, off-the-shelf allogeneic CAR-T therapies across all geographies. UCART19 has been provided for to a patientwho could not undergo an autologousCAR-T therapy. Our goal is to make our product candidates accessible to anyone."

About UCART19
UCART19 is a potential best-in-class allogeneic engineered T-cell product for treatment of CD19 expressing hematologic malignancies, initially developed in Chronic lymphocytic leukemia (CLL) and Acute lymphoblastic leukemia (ALL). Servier has an option under the collaboration agreement to acquire the exclusive rights to further develop and commercialize UCART19. Engineered allogeneic CD19 T-cells currently stand out as a real therapeutic innovation for treating various types of leukemia and lymphoma. Cellectis’ approach with UCART19 is based on the preliminary positive results from clinical trials using products based on the CAR technology and has the potential to overcome the limitation of the autologous current approach by providing an allogeneic frozen, "off the shelf" T-cell based medicinal product.

About Great Ormond Street Hospital (GOSH)
Great Ormond Street Hospital for Children NHS Trust is the country’s leading centre for treating sick children, with the widest range of specialists under one roof. With the UCL Institute of Child Health, they are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.

About the UCL department of hematology
The UCL department of hematology is the major tertiary referral center in the UK for all types of hematological malignancies. They have assumed a global leadership position in stem cell transplantation and adoptive cell therapy for leukemia patients.

On November 5, 2015 BioLineRx Ltd. (NASDAQ, TASE: BLRX), a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, reported positive results from the dose escalation part of BL-8040’s Phase 2 clinical trial in relapsed or refractory acute myeloid leukemia (r/r AML) (Press release, BioLineRx, NOV 5, 2015, View Source;p=RssLanding&cat=news&id=2107352 [SID:1234507989]). The results will be presented at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, to be held December 5-8, 2015 in Orlando, Florida.

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Results showed that BL-8040, as a single agent and in combination with Cytarabine (Ara-C), was safe and well tolerated at all doses tested up to and including the highest dose level of 1.5 mg/kg, with no major adverse events. The composite complete remission rate, including both complete remission (CR) and complete remission with incomplete blood count recovery (CRi), was 38% in subjects receiving only one cycle of BL-8040 treatment at doses of 1 mg/kg and higher (n=16). Patients included in this part of the study were patients that had undergone a significant number of prior treatment cycles or that were refractory to induction treatment.

Treatment with BL-8040 had a triple effect on the leukemic cells. First, following only two days of monotherapy, BL-8040 triggered an average 40-fold mobilization of immature AML progenitor cells from the bone marrow to the peripheral blood, thereby sensitizing these cells to the Ara-C chemotherapy and improving its efficacy. Second, BL-8040 showed a direct and significant apoptotic effect on the immature leukemia progenitor cells in the bone marrow following the two days of monotherapy. Last, BL-8040 induced leukemia progenitor cells towards differentiation, as evidenced by a 58% median decrease in the number of bone marrow leukemia progenitor cells, along with a three-fold increase in differentiated granulocytes, in the bone marrow biopsy conducted on Day 3 of the treatment cycle prior to the Ara-C treatment, as compared to the biopsy performed at baseline.

Dr. Jorge Cortes, Chief of the AML and CML Sections at the MD Anderson Cancer Center in Houston, stated, "The clinical results from the dose escalation stage of the Phase 2 study for BL-8040 in r/r AML are very encouraging and fully support continued development of the compound in the AML space."

Dr. Kinneret Savitsky, CEO of BioLineRx, commented, "We are very pleased and encouraged by the positive results from the first part of this Phase 2 trial with BL-8040 for the treatment of r/r AML, especially in light of the severity of the patient population recruited and the short treatment regimen of one cycle. The results continue to demonstrate that BL-8040 not only significantly induces mobilization of leukemic cells from the protective microenvironment of the bone marrow into the peripheral blood, but also directly leads to apoptosis of leukemic progenitor cells and triggers terminal differentiation of the cells into granulocytes. Combined with the reported 38% remission rate from subjects receiving BL-8040 doses of at least 1 mg/kg, the results strongly suggest that BL-8040 has potent anti-leukemic activity and, in combination with Ara-C, may improve the response typically achieved in this advanced AML population." Dr. Savitsky continued, "In light of the encouraging results, we look forward to discussions with the regulatory authorities regarding the future development plan for AML. We currently anticipate reporting topline results from the full study by early next year."

"In order to further expand and enhance the potential of this unique oncology platform, we are continuing to perform and plan multiple additional clinical studies for BL-8040. These include a Phase 2b study which we recently initiated for BL-8040 as an AML consolidation treatment; a Phase 2 study in the planning stages as a novel stem cell mobilization treatment, based on input that we received from the FDA last month; and two additional studies in certain bone marrow failure indications and for the treatment of AML patients with the FLT3-ITD mutation," concluded Dr. Savitsky.

About the r/r AML Phase 2 study

The Phase 2 trial is a multicenter, open-label study under an IND, conducted at ten clinical sites in the U.S. and Israel, and is designed to assess the safety, efficacy pharmacodynamics and pharmacokinetic parameters of BL-8040 in combination with Cytarabine (Ara-C) for the treatment of adult relapsed or refractory AML patients. Twenty-two patients with r/r AML were enrolled in the dose escalation stage of the study (16 of which received a dose of 1 mg/kg and higher), which includes a dose escalation stage followed by an expansion stage. In the dose escalation stage, each patient received a once daily dose of BL-8040 monotherapy (from 0.5 to 1.5 mg/kg) on days 1-2 followed by the same dose of BL-8040 plus Ara-C on days 3-7. Extensive pharmacodynamic parameters, such as mobilization of leukemic cells and induction of apoptosis, were assessed after monotherapy with BL-8040 using peripheral blood sampling and bone marrow aspirates at baseline and on Day 3 prior to Ara-C administration. Clinical response to treatment was evaluated by bone marrow biopsy on Day 30.

About BL-8040

BL-8040 is a clinical-stage drug candidate for the treatment of acute myeloid leukemia, as well as other hematological indications. It is a short peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis (growth of new blood vessels in the tumor), metastasis (spread of the disease to other organs or organ parts) and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a Phase 1/2, open-label, dose escalation, safety and efficacy clinical trial in 18 multiple myeloma patients, BL-8040, when combined with G-CSF, demonstrated an excellent safety profile at all doses tested and was highly effective in the mobilization of hematopoietic stem cells and white blood cells from the bone marrow to the peripheral blood. Additionally, in a Phase 1 stem-cell mobilization study in healthy volunteers, BL-8040 as a single agent was safe and well tolerated at all doses tested and resulted in efficient stem-cell mobilization and collection in all study participants. Importantly, the results of this study support the use of BL-8040 as one-day, single-dose collection regimen, which is a significant improvement upon the current standard of care.

BL-8040 also mobilizes cancer cells from the bone marrow and may therefore sensitize these cells to chemo- and bio-based anti-cancer therapy. Importantly, BL-8040 has also demonstrated a direct anti-cancer effect by inducing apoptosis. Pre-clinical studies show that BL-8040 inhibits the growth of various tumor types including multiple myeloma, non-Hodgkin’s lymphoma, leukemia, non-small cell lung carcinoma, neuroblastoma and melanoma. BL-8040 also significantly and preferentially stimulated apoptotic cell death of malignant cells (multiple myeloma, non-Hodgkin’s lymphoma and leukemia). Significant synergistic and/or additive tumor cell killing activity has been observed in-vitro and in-vivo when tumor cells were treated with BL-8040 together with Rituximab, Bortezomib, Imatinib, Cytarabine and the FLT-3 inhibitor AC-220 (in NHL, MM, CML, AML, and AML-FLT3-ITD models, respectively). In addition, the current Phase 2 clinical trial in AML patients has demonstrated robust mobilization and apoptosis of cancer cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and is the most common type of acute leukemia in adults. According to the American Cancer Society, approximately 19,000 new cases of AML were diagnosed in the United States in 2014, and the median age of AML patients was 67 years old. The first treatment line for patients with AML includes a combination of chemotherapy drugs and is called induction treatment. The median survival for AML patients receiving induction chemotherapy is less than two years, with shorter survival for patients over the age of 60 or for those with certain gene or chromosome aberrations. Due to relapsed or refractory disease (where the disease is not responsive to standard treatments), the overall five-year survival rate for AML is between 10 and 40 percent.

On November 5, 2015 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, reported that data in six abstracts on the investigational protein therapeutics sotatercept, luspatercept and ACE-1332 will be presented at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held in Orlando, Florida on December 5-8, 2015 (Press release, Acceleron Pharma, NOV 5, 2015, View Source [SID:1234507988]).

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Oral presentations

Title: Luspatercept Treatment Leads to Long Term Increases in Hemoglobin and Reductions in Transfusion Burden in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS): Preliminary Results from the Phase 2 PACE-MDS Extension Study (Abstract #92)
Session: 637. Myelodysplastic Syndromes – Clinical Studies: New Therapeutic Approaches
Date: Saturday, December 5th
Time: 12:15 pm EST (Orange County Convention Center, W331)

Title: TGFβ1 Antagonist Inhibits Fibrosis in a Murine Model of Myelofibrosis (Abstract #605)
Session: 635. Myeloproliferative Syndromes: Basic Science II
Date: Monday, December 7th
Time: 11:30 am EST (Orange County Convention Center, W331)

Title: RAP-536 (Murine ACE-536/Luspatercept) Inhibits Smad2/3 Signaling and Promotes Erythroid Differentiation by Restoring GATA-1 Function in Murine β-Thalassemia (Abstract #751)
Session: 112. Thalassemia and Globin Gene Regulation: Therapeutic Approaches to Thalassemia and Their Mechanisms
Date: Monday, December 7th
Time: 4:35 pm EST (Orange County Convention Center, Valencia A (W415A))

Title: Luspatercept (ACE-536) Reduces Disease Burden, Including Anemia, Iron Overload, and Leg Ulcers, in Adults with Beta-Thalassemia: Results from a Phase 2 Study (Abstract #752)
Session: 112. Thalassemia and Globin Gene Regulation: Therapeutic Approaches to Thalassemia and Their Mechanisms
Date: Monday, December 7th
Time: 4:45 pm EST (Orange County Convention Center, Valencia A (W415A))

Poster presentations

Title: Biomarkers of Ineffective Erythropoiesis Predict Response to Luspatercept in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS): Final Results from the Phase 2 PACE-MDS Study (Abstract #2862)
Session: 637. Myelodysplastic Syndromes – Clinical Studies: Poster II
Date: Sunday, December 6th
Time: 6:00 – 8:00 pm EST (Orange County Convention Center, Hall A)

Title: Phase 1 Dose-Escalation Study of Sotatercept (ACE-011) in Combination with Lenalidomide and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma (Abstract #4241)
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster III
Date: Monday, December 7th
Time: 6:00 – 8:00 pm (EST) (Orange County Convention Center, Hall A)

The posters and presentation slides will be available in the "Publications" section on Acceleron’s website (www.acceleronpharma.com).

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Acceleron and Celgene are initiating phase 3 clinical trials that are designed to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the "MEDALIST" study) and in patients with beta-thalassemia (the "BELIEVE" study). For more information, please visit www.clinicaltrials.gov.

About Sotatercept

Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β) superfamily involved in the late stages of erythropoiesis (red blood cell production). Sotatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing sotatercept as part of a global collaboration. Sotatercept is currently in multiple phase 2 clinical trials. For more information, please visit www.clinicaltrials.gov.

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.

On November 5, 2015 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) reported that new data from the ongoing dose-escalation and expansion studies of AG-221 and AG-120 in advanced hematologic malignancies will be presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, December 5-8, 2015 (Press release, Agios Pharmaceuticals, NOV 5, 2015, View Source;p=RssLanding&cat=news&id=2107405 [SID:1234507986]).

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In total, five abstracts led by Agios describing new clinical data from the company’s cancer metabolism and rare genetic metabolic disorders programs have been accepted for presentation at ASH (Free ASH Whitepaper), as well as two abstracts detailing new findings from the Natural History Study of pyruvate kinase (PK) deficiency being conducted with Boston Children’s Hospital. Agios’ IDH inhibitors AG-221 and AG-120 are being developed in collaboration with Celgene.

The accepted abstracts are listed below and are now available online on the ASH (Free ASH Whitepaper) conference website: View Source

Oral Presentation

Date & Time: Sunday, December 6, 2015 at 5:30 p.m. ET
Session Title: 613. Acute Myeloid Leukemia: Clinical Studies: Advances in Targeted Therapy
Abstract Number: 323
Presentation Title: Safety and Efficacy of AG-221, a Potent Inhibitor of Mutant IDH2 That Promotes Differentiation of Myeloid Cells in Patients with Advanced Hematologic Malignancies: Results of a Phase 1/2 Trial
Location: Orange County Convention Center, W110

Poster Presentations

Date & Time: Saturday, December 5, 2015 from 5:30 p.m. to 7:30 p.m. ET
Session Title: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I
Abstract Number: 1306
Presentation Title: Molecular Profiling and Relationship with Clinical Response in Patients with IDH1 Mutation-Positive Hematologic Malignancies Receiving AG-120, a First-in-Class Potent Inhibitor of Mutant IDH1, in Addition to Data from the Completed Dose Escalation Portion of the Phase 1 Study
Location: Orange County Convention Center, Hall A

Date & Time: Saturday, December 5, 2015 from 5:30 p.m. to 7:30 p.m. ET
Session Title: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I
Abstract Number: 1310
Presentation Title: Longitudinal Pharmacokinetic/Pharmacodynamic Profile of AG-120, a Potent Inhibitor of the IDH1 Mutant Protein, in a Phase 1 Study of IDH1-Mutant Advanced Hematologic Malignancies
Location: Orange County Convention Center, Hall A

Date & Time: Sunday, December 6, 2015 from 6:00 p.m. to 8:00 p.m. ET
Session Title: 613. Acute Myeloid Leukemia: Clinical Studies: Poster II
Abstract Number: 2509
Presentation Title: Evaluation of the Pharmacokinetics of AG-221, a Potent Mutant IDH2 Inhibitor, in Patients with IDH2-Mutation Positive Advanced Hematologic Malignancies in a Phase 1/2 Trial
Location: Orange County Convention Center, Hall A

Date & Time: Sunday, December 6, 2015 from 6:00 p.m. to 8:00 p.m. ET
Session Title: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster II
Abstract number: 2136
Presentation Title: The Phenotypic Spectrum of Pyruvate Kinase Deficiency (PKD) from the PKD Natural History Study (NHS): Description of Four Severity Groups By Anemia Status
Location: Orange County Convention Center, Hall A

Date & Time: Monday, December 7, 2015 from 6:00 p.m. to 8:00 p.m. ET
Session Title: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster III
Abstract Number: 3337
Presentation Title: Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New Variants
Location: Orange County Convention Center, Hall A

Date & Time: Monday, December 7, 2015 from 6:00 p.m. to 8:00 p.m. ET
Session Title: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster III
Abstract Number: 3336
Presentation Title: Population Pharmacokinetics and Pharmacodynamics of AG-348 in Healthy Human Volunteers Guide Dose Selection for the Treatment of Pyruvate Kinase Deficiency
Location: Orange County Convention Center, Hall A