On November 1, 2018 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that nine abstracts from its complement research and development program have been accepted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, December 1 to 4, 2018 (Press release, Alexion, NOV 1, 2018, View Source [SID1234530490]). Key data will include both new analyses and previously announced results from the two Phase 3 studies of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH), in addition to further data on Soliris (eculizumab) for the treatment of PNH and atypical hemolytic uremic syndrome (aHUS). Collectively, the breadth of the data to be presented at ASH (Free ASH Whitepaper) demonstrates continued progress extending the company’s leadership in understanding and treating rare complement-mediated diseases.

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The accepted abstracts are listed below and are now available on the ASH (Free ASH Whitepaper) website.

ALXN1210

A Phase 3 Study of Ravulizumab (ALXN1210) versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria Naive to Complement Inhibitors: Results of a Subgroup Analysis with Patients Stratified by Baseline Hemolysis Level, Transfusion History, and Demographics. Abstract ID#: 110623 – Oral Presentation, December 3, 2018, 11:00-11:15 a.m. PST, Grand Hall C.

Results from a Phase 3, Multicenter, Non-Inferiority Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Eculizumab. Abstract ID#: 119147 – Oral Presentation, December 3, 10:30-10:45 a.m. PST, Grand Hall C.

Ravulizumab (ALXN1210) versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter Studies. Abstract ID#: 110858 – Oral Presentation, December 3, 10:45-11:00 a.m. PST, Grand Hall C.

A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria. Abstract ID#: 110874 – Poster Presentation, December 2, 6:00-8:00 p.m. PST, Hall GH.

Soliris (eculizumab)

Efficacy of Eculizumab in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria in the International PNH Registry. Abstract ID#: 111306 – Poster Presentation, December 3, 6:00-8:00 p.m. PST, Hall GH.

Economic Benefit of Early In-hospital Diagnosis and Treatment Initiation of Eculizumab in aHUS. Abstract ID#: 112893 – Poster Presentation, December 2, 6:00-8:00 p.m. PST, Hall GH.

PNH

Prognostic Value of Clone Size in Paroxysmal Nocturnal Hemoglobinuria (PNH) for Thrombotic Events in Untreated Patients in the International PNH Registry. Abstract ID#: 111324 – Poster Presentation, December 1, 6:15-8:15 p.m. PST, Hall GH.

Baseline Characteristics of Patients with Paroxysmal Nocturnal Hemoglobinuria Identified in the Department of Defense Database. Abstract ID#: 113478 – online.

The Value of Population Based Data to Study Rare Diseases: An Example Using the Department of Defense Healthcare System. Abstract ID#: 113497 – online.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder that can strike men and women of all races, backgrounds, and ages without warning, with an average age of onset in the early 30s.1,2,3 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.2 In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, results in hemolysis (the destruction of red blood cells)4, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath.5,6,7 The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.8 Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.2 PNH is more common among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).9,10,11 In certain patients with thrombosis of unknown origin, PNH may be an underlying cause.4

About ALXN1210

ALXN1210 is an innovative, investigational, long-acting C5 inhibitor discovered and developed by Alexion that works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH, and patients with PNH who had been stable on Soliris, intravenous treatment with ALXN1210 every eight weeks demonstrated non-inferiority to intravenous treatment with Soliris every two weeks, with numeric results for all primary and key secondary endpoints favoring ALXN1210. ALXN1210 is also currently being evaluated in a Phase 3 clinical study in complement inhibitor-naïve patients with aHUS, administered intravenously every eight weeks. In addition, Alexion plans to initiate a Phase 3 clinical study of ALXN1210 delivered subcutaneously once per week as a potential treatment for patients with PNH and aHUS.

ALXN1210 has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU, and Japan, and for the subcutaneous treatment of patients with aHUS in the U.S.

About Soliris (eculizumab)

Soliris is a first-in-class complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade, a part of the immune system that, when activated in an uncontrolled manner, plays a role in severe rare and ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). Soliris is approved in the U.S., EU, Japan, and other countries as the first and only treatment for patients with PNH and aHUS, in the EU as the first and only treatment of refractory generalized MG (gMG) in adults who are anti-AchR antibody-positive, in the U.S. for the treatment of adult patients with gMG who are anti-AchR antibody-positive, and in Japan for the treatment of patients with gMG who are AChR antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin (IVIG) therapy or plasmapheresis (PLEX). Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).

Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU, Japan, and many other countries, for the treatment of patients with aHUS in the U.S., EU, and many other countries, for the treatment of patients with MG in the U.S. and EU, for the treatment of patients with refractory gMG in Japan, and for the treatment of patients with neuromyelitis optica spectrum disorder (NMOSD) in the U.S., EU, and Japan. Alexion and Soliris have received some of the pharmaceutical industry’s highest honors for the medical innovation in complement inhibition: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).

For more information on Soliris, please see full prescribing information for Soliris, including BOXED WARNING regarding risk of serious meningococcal infection, available at www.soliris.net.

Important Soliris Safety Information

The U.S. prescribing information for Soliris includes the following warnings and precautions: Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Comply with the most current Centers for Disease Control (CDC)’s Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with meningococcal vaccines at least two weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-SOLIRIS (1-888-765-4747) or at www.solirisrems.com.

Patients may have increased susceptibility to infections, especially with encapsulated bacteria. Aspergillus infections have occurred in immunocompromised and neutropenic patients. Children treated with Soliris may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenza type b (Hib). Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. Administration of Soliris may result in infusion reactions, including anaphylaxis or other hypersensitivity reactions.

In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis, back pain, and nausea. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, and pyrexia. In patients with gMG who are anti-AchR antibody-positive, the most frequently reported adverse reaction observed with Soliris treatment in the placebo-controlled clinical study (≥10%) was musculoskeletal pain.

On November 1, 2018 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, 2018 (Press release, Agios Pharmaceuticals, NOV 1, 2018, View Source [SID1234530489]).

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"On the heels of the third quarter U.S. approval of TIBSOVO, our first wholly owned precision medicine, we remain focused on executing on our remaining 2018 milestones and continuing to create value from our portfolio," said David Schenkein, M.D., chief executive officer at Agios. "Based on regulatory discussions, we are accelerating our frontline strategy in IDH1m AML with the planned submission of an sNDA expanding TIBSOVO’s label to newly diagnosed AML patients not eligible for standard treatment and a shorter enrollment timeline for the Phase 3 AGILE trial. In addition, we continue to activate new sites globally for our pivotal PK deficiency program and accrue patients in the dose-escalation portion of the Phase 1 study in MTAP-deleted tumors. We believe these programs coupled with our robust preclinical pipeline, support our next phase of growth toward becoming a fully integrated, sustainable biopharmaceutical company."

THIRD QUARTER & RECENT 2018 HIGHLIGHTS

Launched TIBSOVO (ivosidenib) for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by a test approved by the U.S. Food and Drug Administration (FDA).
Awarded the U.S. Prix Galien Award for Best Pharmaceutical Product of 2018 for IDHIFA (enasidenib), an isocitrate dehydrogenase-2 (IDH2) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation as detected by a test approved by the FDA. Each year, the Prix Galien USA Committee recognizes outstanding achievements in improving the human condition through the development of innovative therapies.
Reached agreement with the FDA to submit a supplemental new drug application (sNDA) for single agent TIBSOVO in newly diagnosed AML patients with an IDH1 mutation who are not eligible for standard treatment.
Reached agreement with the FDA that event free survival (EFS) is an acceptable primary endpoint for the Phase 3 AGILE trial of ivosidenib combination with azacitidine in newly diagnosed AML patients with an IDH1 mutation who are ineligible for intensive chemotherapy. Full enrollment for AGILE is now expected to complete in 2020 vs. previous guidance of 2021.
Received global rights to AG-881, a brain-penetrant, pan-IDH inhibitor that was previously part of a joint worldwide collaboration with Celgene.
Submitted an investigational new drug (IND) application for AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies.
Announced that effective February 1, 2019, CEO David Schenkein, M.D., will transition to the role of executive chairman of the board of directors and Jacqualyn ("Jackie") Fouse, Ph.D., will succeed Dr. Schenkein as Agios’ next chief executive officer.
KEY UPCOMING MILESTONES

The company expects to achieve the following near-term milestones:

Cancer:

Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO for the treatment of adult patients with R/R AML with an IDH1 mutation by year end 2018.
Support, in conjunction with Celgene, the initiation of HOVON 150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy followed by a maintenance therapy period in frontline AML patients with an IDH1 or IDH2 mutation, respectively, by year end 2018.
Submit an sNDA to the FDA for TIBSOVO for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy by the end of January 2019.
Rare Genetic Diseases:

Initiate a Phase 2 proof-of-concept trial of mitapivat in thalassemia by year end 2018.
FOURTH QUARTER CLINICAL DATA PRESENTATIONS

Updated data from Phase 1 trial of AG-881 in solid tumors, including glioma, has been accepted as an oral presentation at the 2018 Society for Neuro-Oncology (SNO) Annual Meeting on November 15-18 in New Orleans.
Updated data from the ongoing Phase 1 trial of single agent ivosidenib in IDH1m hematologic malignancies in a subset of patients with newly diagnosed AML not eligible for standard therapy has been accepted as an oral presentation at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition on December 1-4 in San Diego.
Updated data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation also been accepted as an oral presentation at ASH (Free ASH Whitepaper).
Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of single agent ivosidenib in IDH1m hematologic malignancies has been accepted as a poster presentation at ASH (Free ASH Whitepaper).
THIRD QUARTER 2018 FINANCIAL RESULTS

Revenue for the quarter ended September 30, 2018 was $15.2 million, which includes $4.5 million of net product revenue from U.S. sales of TIBSOVO, $8.7 million of collaboration revenue and $2.0 million of royalty revenue from net global sales of IDHIFA under our collaboration agreements with Celgene. Revenue for the quarter ended September 30, 2017 was $11.4 million and consisted of $10.6 million of collaboration revenue and $0.7 million of royalty revenue under our agreements with Celgene. The year-over-year increase in total revenue for the third quarter was primarily driven by U.S. sales of TIBSOVO and royalty revenue from U.S. sales of IDHIFA, offset by a decrease in collaboration revenue recognized during the quarter.

Cost of sales for the quarter ended September 30, 2018 were $0.7 million and relate to manufacturing costs associated with TIBSOVO sales.

Research and development (R&D) expenses were $82.6 million, including $13.4 million of stock-based compensation expense, for the quarter ended September 30, 2018, compared to $72.9 million, including $7.6 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the mitapivat (AG-348) pivotal program in PK deficiency and IND enabling activities for AG-636, our DHODH inhibitor. R&D expense also increased as a result of ongoing research efforts across our discovery platform programs.

Sales, general and administrative (SG&A) expenses were $31.1 million, including $10.8 million of stock-based compensation expense, for the quarter ended September 30, 2018, compared to $17.5 million, including $4.6 million of stock-based compensation expense, for the quarter ended September 30, 2017. The increase in SG&A expense was primarily attributable to the growth in our U.S. commercial organization to support the launch of TIBSOVO and personnel costs related to increased headcount.

Net loss for the quarter ended September 30, 2018 was $94.7 million, compared to a net loss of $77.1 million for the quarter ended September 30, 2017.

Cash, cash equivalents and marketable securities as of September 30, 2018 were $878.4 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow-on offering, $14.8 million of cost reimbursements and royalty payments under our collaboration agreements with Celgene, $12.0 million under our collaboration agreement with CStone and $29.2 million received from employee stock transactions. This was offset by expenditures to fund operations of $263.0 million during the nine months ended September 30, 2018.

The company expects that its cash, cash equivalents and marketable securities as of September 30, 2018, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license 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 2020.

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 2018 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 referring to conference ID 5285068. 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.

On November 1, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that updated data from its isocitrate dehydrogenase (IDH) programs and pyruvate kinase (PK) deficiency program will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 1 – 4, 2018 in San Diego (Press release, Agios Pharmaceuticals, NOV 1, 2018, View Source [SID1234530488]).

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In total, five abstracts led by Agios describing updated data from the company’s IDH programs and PKD program have been accepted for presentation at ASH (Free ASH Whitepaper).

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

Oral presentations by Agios:

Title: Ivosidenib (AG-120) Induced Durable Remissions and Transfusion Independence in Patients with IDH1-Mutant Untreated AML: Results from a Phase 1 Dose Escalation and Expansion Study
Date & Time: Monday December 3, 2018 at 7:30 a.m. PST
Oral Abstract Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Targeted Therapy
Abstract: 561
Location: Manchester Grand Hyatt San Diego, Seaport Ballroom F
Presenter: Gail J. Roboz, M.D., Weill Cornell Medical College

Title: Ivosidenib or Enasidenib Combined with Induction and Consolidation Chemotherapy in Patients with Newly Diagnosed AML with an IDH1 or IDH2 Mutation is Safe, Effective, and Leads to MRD-Negative Complete Remissions
Date & Time: Monday December 3, 2018 at 7:15 a.m. PST
Oral Abstract Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Targeted Therapy
Abstract: 560
Location: Manchester Grand Hyatt San Diego, Seaport Ballroom F
Presenter: Eytan Stein, M.D., Memorial Sloan Kettering Cancer Center

Poster presentations by Agios:

Title: Ivosidenib (AG-120) Induced Durable Remissions and Transfusion Independence in Patients with IDH1-Mutant Relapsed or Refractory Myelodysplastic Syndrome: Results from a Phase 1 Dose Escalation and Expansion Study
Poster Session Date & Time: Saturday December 1, 2018 from 6:15-8:15 p.m. PST
Poster Session: 637. Myelodysplastic Syndromes—Clinical Studies: Poster I
Abstract: 1812
Poster Location: San Diego Convention Center, Hall GH
Author: Courtney Denton DiNardo, M.D., University of Texas MD Anderson Cancer Center

Title: Population Pharmacokinetics of Ivosidenib (AG-120) in Patients with IDH1-Mutant Advanced Hematologic Malignancies
Poster Session Date & Time: Saturday December 1, 2018 from 6:15-8:15 p.m. PST
Poster Session: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I
Abstract: 1394
Poster Location: San Diego Convention Center, Hall GH
Author: Kha Le, Ph.D., Agios Pharmaceuticals

Title: Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency
Poster Session Date & Time: Monday December 3, 2018 from 6:00-8:00 p.m. PST
Poster Session: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster III
Abstract: 3621
Poster Location: San Diego Convention Center, Hall GH
Author: Charles Kung, Ph.D., Agios Pharmaceuticals

On November 1, 2018 Actinium Pharmaceuticals, Inc. (NYSE American: ATNM) reported that multiple abstracts highlighting its Antibody Radiation Conjugates (ARCs) have been accepted for presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, including an oral presentation of preliminary feasibility and safety results from its pivotal Phase 3 SIERRA trial of Iomab-B (Press release, Actinium Pharmaceuticals, NOV 1, 2018, View Source [SID1234530487]). The ASH (Free ASH Whitepaper) Annual Meeting is being held December 1 – 4, 2018 in San Diego, California. Data presented will highlight Actinium’s lead product candidate, Iomab-B, that is intended to be a targeted conditioning agent prior to a bone marrow transplant for patients with active relapsed or refractory Acute Myeloid Leukemia (AML) who are over the age of 55. Patients with active relapsed or refractory AML do not routinely undergo allogeneic bone marrow transplant due to a lack of efficacy using standard approaches and typically the survival of such patients without a transplant is less than six months.

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Iomab-B Oral Presentation Details
Abstract # 1017
Title: Targeted Conditioning of Iomab-B (131I-anti-CD45) Prior to Allogeneic Hematopoietic Cell Transplantation Versus Conventional Care in Relapsed or Refractory Acute Myeloid Leukemia (AML): Preliminary Feasibility and Safety Results from the Prospective, Randomized Phase 3 Sierra Trial
Session Name: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Conditioning Intensity and Novel Approaches with Targeted therapy
Session Date: Monday, December 3, 2018
Presentation Time: 6:45 PM
Room: Manchester Grand Hyatt San Diego, Seaport Ballroom A
Presenter: Dr. Agura, Baylor University Medical Center
Results as of July 5, 2018

Dr. Mark Berger, Actinium’s Chief Medical Officer said, "We are honored that results from our ongoing Phase 3 trial have been accepted for an oral presentation at this year’s ASH (Free ASH Whitepaper) annual meeting as approximately just ten percent of accepted abstracts receive this designation. Most importantly, patients with active, relapsed or refractory AML have severely restricted access to bone marrow transplant, the only potentially curative treatment option, so we are we are elated that the initial feasibility and safety data from SIERRA has demonstrated the ability to enable transplant and engraftment for not only all patients initially randomized to Iomab-B but also all those that crossed-over from the control arm when salvage chemotherapy failed to produce a complete response. Importantly, this occurred in patients with high blast counts as the median blast count was 30% and 47% in the Iomab-B arm and cross-over patients, respectively. We look forward to having our data presented at ASH (Free ASH Whitepaper), providing additional updates on this important trial as it progresses and completing the SIERRA trial with the goal of bringing this important targeted conditioning agent to patients with a significant unmet need."

Sandesh Seth, Actinium’s Chairman and Chief Executive Officer said, "We are delighted that data representing an important cross-section of our Antibody Radiation Conjugate pipeline will be featured at this year’s ASH (Free ASH Whitepaper), particularly the presentation highlighting preliminary results from the SIERRA trial for our lead targeted conditioning asset, Iomab-B. Recognizing that a bone marrow transplant is a potentially curative treatment option for many hematologic diseases, Actinium is focused on improving bone marrow transplant access and outcomes through improved targeted conditioning, which is currently underserved by chemotherapy. We are excited that the data presented in the various forums at ASH (Free ASH Whitepaper) will demonstrate the capabilities of our highly differentiated ARC approach for targeted conditioning that we believe is unmatched by other technologies or approaches. We are committed to continuing to expand our targeted conditioning pipeline as we have done with Actimab-MDS with the goal of building an independent fully integrated company."

Data from the Company’s CD33 program ARC, Ac-225 – Lintuzumab, and the recently completed Actimab-A Phase 2 trial from for patients newly diagnosed with AML who are unfit for intensive chemotherapy has been accepted for poster presentation. Actinium recently announced in a CD33 program update that, based on the results of the Phase 2 Actimab-A trial, Actinium is continuing to develop Ac-225 – Lintuzumab in two combination trials for patients with relapsed or refractory AML, one being with Venetoclax and the other being with Venetoclax and Hypomethylating agents. Ac-225 – Lintuzumab is also being studied in patients with multiple myeloma and as a targeted conditioning agent to enable a bone marrow transplant for patients with high-risk Myelodysplastic Syndrome.

Actimab-A Abstract Details
Abstract # 1457
TITLE: A Phase 2 Study of Actinium-225 (225Ac)-Lintuzumab in Older Patients with Untreated Acute Myeloid Leukemia (AML) – Interim Analysis of 1.5 µci/Kg/Dose
Session Name: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster I
Date: Saturday, December 1, 2018
Presentation Time: 6:15 PM – 8:15 PM
Location: San Diego Convention Center, Hall GH

Actinium also submitted preliminary data from its Iomab-ACT program for next generation targeted lymphodepletion prior to CAR-T therapy. This data will be published online coinciding with the start of the 2018 ASH (Free ASH Whitepaper) Annual Meeting.

On November 1, 2018 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that new data for its approved and investigational medicines across a range of blood diseases, and including several first-in-class medicines, will be presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting from 1-4 December, 2018 (Press release, Hoffmann-La Roche, NOV 1, 2018, View Source [SID1234530483]). Ten Roche medicines will be featured in more than 70 abstracts, including 25 oral presentations, across 15 blood diseases.

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"We look forward to sharing progress from our broad development programme in haematology at ASH (Free ASH Whitepaper) this year, reflecting our approach to understand mechanisms of blood diseases at the molecular level," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We are excited to be presenting data across multiple blood diseases, including studies of several first-in-class medicines with the potential to transform standards of care and improve patients’ lives."

Hemlibra (emicizumab), which represents the first new class of medicine in nearly 20 years for people with haemophilia A, will be featured in 12 abstracts at the congress. New data in children younger than 12 with haemophilia A with and without factor VIII inhibitors will be presented, including the full results from the pivotal HAVEN 2 study evaluating three different Hemlibra dosing options (once weekly, every two weeks or every four weeks) in children with haemophilia A with factor VIII inhibitors. Additionally, treatment preference data from the pivotal HAVEN 3 study in people with haemophilia A without factor VIII inhibitors and the pivotal HAVEN 4 study in people with haemophilia A with and without factor VIII inhibitors will be presented. Hemlibra was recently approved by the US Food and Drug Administration (FDA) for the treatment of haemophilia A without factor VIII inhibitors and is the only haemophilia treatment that can be administered subcutaneously and at multiple dosing options for all people with haemophilia A, with and without factor VIII inhibitors.

Roche will also share data for medicines for a range of blood cancers, across multiple lines of treatment. Highlights include updated results from the phase III MURANO study evaluating Venclexta/Venclyxto (venetoclax) in chronic lymphocytic leukaemia (CLL). In addition, data evaluating Venclexta/Venclyxto in acute myeloid leukaemia (AML) will be featured, including two phase Ib/II combination studies (M14-358 study and M14-387 study). Venclexta/Venclyxto was recently approved in Europe and the United States as a treatment for relapsed or refractory CLL, and is currently under review by the FDA for the treatment of previously untreated AML in combination with a hypomethylating agent or in combination with low dose cytarabine, with a decision expected by end of year. Venclexta/Venclyxto is being developed by AbbVie and Roche.

Updated efficacy data from the phase II GO29365 study evaluating polatuzumab vedotin, an investigational anti-CD79b antibody drug conjugate, in combination with MabThera/Rituxan (rituximab) plus bendamustine, in relapsed or refractory diffuse large B-cell lymphoma (DLBCL), will also be presented. The results of the DLBCL portion of the GO29365 study will be submitted to health authorities around the world for approval consideration. Data from the phase III GALLIUM study of Gazyva/Gazyvaro (obinutuzumab) in previously untreated follicular lymphoma which support the prognostic value of minimal residual disease status at the end of induction treatment will also be presented.

Finally, Roche will present early data for two novel T-cell engaging bispecific antibodies in non-Hodgkin lymphoma (NHL), which includes initial efficacy and safety results from the first clinical trials for the investigational medicines mosunetuzumab and CD20-TCB. These bispecific antibodies redirect T-cells to engage and eliminate malignant B-cells. This builds on Roche’s extensive history and expertise in the development of anti-CD20 antibodies for the treatment of numerous B-cell malignancies.

Key abstracts featuring Roche medicines that will be presented at ASH (Free ASH Whitepaper) can be found in the table below.

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About Roche in haematology
For more than 20 years, Roche has been developing medicines that redefine treatment in haematology. Today, we are investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera//Rituxan (rituximab), Gazyva/Gazyvaro (obinutuzumab), and Venclexta/Venclyxto (venetoclax) in collaboration with AbbVie, Roche’s pipeline of investigational haematology medicines includes Tecentriq (atezolizumab), an anti-CD79b antibody drug conjugate (polatuzumab vedotin/RG7596) and a small molecule which inhibits the interaction of MDM2 with p53 (idasanutlin/RG7388). Roche’s dedication to developing novel molecules in haematology expands beyond malignancy, with the development of Hemlibra (emicizumab), a bispecific monoclonal antibody for the treatment of haemophilia A.