On November 1, 2018 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the third quarter ended September 30, 2018 (Press release, bluebird bio, NOV 1, 2018, View Source [SID1234530577]).

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"2018 has been a year of tremendous growth and transition at bluebird, notably exemplified by the filing of our first regulatory application for LentiGlobin in transfusion dependent β-thalassemia (TDT) with the European Medicines Agency (EMA)," said Nick Leschly, chief bluebird. "As we prepare to serve patients with LentiGlobin first in Europe, and later in the U.S., we are advancing the development of our programs in SCD and multiple myeloma, the latter in collaboration with Celgene. We have outlined an accelerated development plan utilizing a surrogate endpoint that we believe allows us to continue to evolve our understanding of the potential of LentiGlobin in patients with SCD. In cerebral adrenoleukodystrophy (CALD), we have regulatory alignment on the path forward and are on track. In multiple myeloma, we and Celgene have begun to lay the plans to bring bb2121 into earlier lines of therapy, while building for the future with bb21217. We look forward to sharing clinical and pre-clinical data across our portfolio – a breadth of progress that is a testament to the tireless work of the bluebird flock."

"The emerging data from our LentiGlobin program in SCD demonstrate the potential to rapidly produce high levels of anti-sickling HbAT87Q that we hope will fundamentally reduce sickling and hemolysis, thereby providing a meaningful increase in total hemoglobin and leading to a reduction in clinical events," said David Davidson, M.D., chief medical officer, bluebird bio. "We continue to have ongoing dialogue with the U.S. Food and Drug Administration (FDA), in the context of our Regenerative Medicine Advanced Therapy (RMAT) designation, and now plan to pursue an accelerated development path that may allow us to initially validate, and then leverage these clinically meaningful biomarkers to form the primary endpoint of a registration-enabling study for patients with a history of vaso-occlusive events (VOEs). We also plan to conduct future clinical investigation of LentiGlobin in patients with other SCD phenotypes, including those at risk of stroke."

Recent Highlights

TDT

LENTIGLOBIN MAA ACCEPTANCE – In October 2018, the EMA accepted for review the company’s marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with TDT and a non-β0/β0 genotype. LentiGlobin was previously granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the EMA in July 2018, potentially reducing the EMA’s active review time of the MAA from 210 days to 150 days.
SCD

LENTIGLOBIN DEVELOPMENT STRATEGY – Based on ongoing discussions with the FDA, bluebird bio is modifying and expanding its clinical development plans to explore efficacy endpoints that may allow the company to pursue a more accelerated development path in the United States for the treatment of patients with SCD who have a history of VOEs. Enrollment of the HGB-206 study has been expanded to enroll up to 50 adult and adolescent patients. The expanded HGB-206 study has a new primary efficacy endpoint based on HbAT87Q and total hemoglobin, and a key secondary endpoint of frequency of VOEs. As modified, the HGB-206 study will increase the overall set of clinical data regarding the relationship between anti-sickling hemoglobin and clinical outcomes and has the potential to validate the new primary efficacy endpoint as a surrogate endpoint for other SCD clinical outcomes such as VOEs. In 2019, bluebird bio intends to initiate a multi-site, international Phase 3 study of LentiGlobin for the treatment of patients with SCD and a history of VOEs with the same primary and secondary endpoints, and comparable design, as the HGB-206 study. bluebird bio is also engaged in ongoing discussions with the EMA regarding proposed development plans for LentiGlobin in SCD in Europe.
CALD

UPDATED DATA AT THE SOCIETY FOR THE STUDY OF INBORN ERRORS OF METABOLISM SYMPOSIUM – In September 2018, bluebird bio announced updated results from the Phase 2/3 Starbeam study (ALD-102) of its investigational Lenti-D gene therapy in boys 17 years of age and under with CALD, and initial data from ALD-103, the ongoing observational study of outcomes from allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD. bluebird bio has also reached general agreement with the FDA and the EMA to use data from ALD-102 and ALD-103 to support future marketing applications for Lenti-D in CALD.
ALD-104 STUDY – In early 2019, bluebird bio intends to initiate a multi-site Phase 3 study of the Lenti-D product candidate for the treatment of patients with CALD to enable access following completion of enrollment in the Starbeam study, and to evaluate the suitability of additional conditioning regimens for use with the Lenti-D product candidate.
COMPANY

REGENERON COLLABORATION – In August 2018, bluebird bio and Regeneron Pharmaceuticals, Inc. (Regeneron) announced a collaboration to apply their respective technology platforms to the discovery, development and commercialization of novel immune cell therapies for cancer. The collaborators will specifically leverage Regeneron’s VelociSuite technology platforms for the discovery and characterization of fully human antibodies, as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides, and bluebird bio will contribute its field-leading expertise in gene transfer and cell therapy.
GRITSTONE COLLABORATION – In August 2018, bluebird bio and Gritstone Oncology, Inc. (Gritstone) announced a collaboration to utilize Gritstone’s proprietary technology platform to identify and validate tumor-specific targets and provide TCRs directed to selected targets for use in bluebird’s gene therapy products. bluebird bio will conduct all development, manufacturing and commercial activities.
STRENGTHENED BALANCE SHEET – In July 2018, bluebird bio raised approximately $600.6 million in net proceeds through a public equity offering. bluebird bio anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations into 2022 based on the company’s current business plan.
Upcoming Anticipated Milestones

TDT
Presentation of LentiGlobin clinical data from the Northstar (HGB-204) clinical study in patients with TDT at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting
Presentation of LentiGlobin clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-β0/β0 genotypes at the ASH (Free ASH Whitepaper) Annual Meeting
Presentation of LentiGlobin clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype at the ASH (Free ASH Whitepaper) Annual Meeting
SCD
Presentation of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD at the ASH (Free ASH Whitepaper) Annual Meeting
Presentation early data from the investigator-initiated Phase 1 study of shRNAmiR lentiviral vector targeting BCL11A for autologous gene therapy in SCD at the ASH (Free ASH Whitepaper) Annual Meeting
Multiple Myeloma
Presentation of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma at the ASH (Free ASH Whitepaper) Annual Meeting
Initiation by Celgene of a Phase 3 clinical study of bb2121 in third line multiple myeloma
Third Quarter 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2018 and December 31, 2017 were $2.0 billion and $1.6 billion, respectively. The increase in cash, cash equivalents and marketable securities is primarily related to the completion of a public offering of common stock in July 2018, which raised net proceeds of approximately $600.6 million, and the receipt of $100.0 million from Regeneron made in connection with the company’s collaboration with Regeneron which was entered into in August 2018. The overall increase in cash, cash equivalents and marketable securities was offset by $40.0 million paid to Gritstone in connection with the company’s collaboration with Gritstone, which was also entered into in August 2018, and cash used in operating activities.
Revenues: Total revenues were $11.5 million for the three months ended September 30, 2018 compared to $7.7 million for the three months ended September 30, 2017. Total revenues were $35.3 million for the nine months ended September 30, 2018 compared to $31.3 million for the nine months ended September 30, 2017. The increase in both periods was primarily attributable to increased manufacturing services under the company’s collaboration agreement with Celgene, offset by decreased license and royalty revenue.
R&D Expenses: Research and development expenses were $116.7 million for the three months ended September 30, 2018 compared to $61.5 million for the three months ended September 30, 2017. Research and development expenses were $328.9 million for the nine months ended September 30, 2018 compared to $180.5 million for the nine months ended September 30, 2017. The increase in both periods was driven by costs incurred to advance and expand the company’s pipeline and is attributable to increased clinical trial-related costs and manufacturing costs for development programs, increased laboratory expenses, increased employee-related costs due to headcount growth, and increased license milestones and fees under the company’s strategic collaboration and license agreements.
G&A Expenses: General and administrative expenses were $44.5 million for the three months ended September 30, 2018 compared to $23.0 million for the three months ended September 30, 2017. General and administrative expenses were $120.6 million for the nine months ended September 30, 2018 compared to $64.5 million for the nine months ended September 30, 2017. The increase in both periods was attributable to increases in employee-related costs due to increased headcount to support overall growth, commercial-readiness activities, and professional and consulting fees.
Net Loss: Net loss was $145.5 million for the three months ended September 30, 2018 compared to $78.8 million for the three months ended September 30, 2017. Net loss was $406.6 million for the nine months ended September 30, 2018 compared to $218.4 million for the nine months ended September 30, 2017.
Conference Call & Webcast Information
bluebird bio will host a conference call and live webcast at 8:00 a.m. ET on Friday, November 2, 2018. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or (315) 625-3227 from outside the United States. Please refer to conference ID number 5595967.

On November 1, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported consolidated financial results for the third quarter 2018 and reviewed recent highlights and upcoming milestones (Press release, NewLink Genetics, NOV 1, 2018, View Source [SID1234530642]).

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"NewLink Genetics continues to produce encouraging data supporting indoximod in targeted cancer indications. We remain confident in the advancement of our clinical programs as we strive to develop novel therapies addressing areas of great unmet need," said Charles J. Link, Jr, MD, Chairman and Chief Executive Officer. "We look forward to presenting data at SITC (Free SITC Whitepaper) and ASH (Free ASH Whitepaper) this fall."
Data to be Presented at Upcoming Medical Meetings

Abstract accepted for oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting, December 1-4, 2018
◦
Abstract 332: Indoximod combined with standard induction chemotherapy is well tolerated and induces a high rate of complete remission with MRD-negativity in patients with newly diagnosed AML: results from a Phase 1 trial, Emadi, A., et al. – to be presented during the oral session, 616 entitled "Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Combination Therapy" Sunday, Dec 2, 2018, 9:30-11:00 AM PT. Data indicate that a high percentage of newly diagnosed AML patients treated with indoximod plus standard-of-care (SOC) chemotherapy achieved complete response (CR) and showed no evidence of minimal residual disease (were MRD-negative). Indoximod was well tolerated.
•
Abstracts accepted for poster presentation at the SITC (Free SITC Whitepaper) Annual Meeting, November 7-11, 2018
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Abstract 11213: A phase 1a clinical trial of NLG802, a prodrug of indoximod with enhanced pharmacokinetic properties, Rixe, O., et al. (Poster #P331)
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Abstract 10294: The immunogenomic impact of indoximod on the tumor microenvironment of melanoma patients, Yu, J., et al. (Poster #P142)
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Abstract 10304: Effects of indoximod plus gemcitabine/nab-paclitaxel on tumor microenvironment of patients with metastatic pancreatic cancer, Yu, J., et al. (Poster #P706)
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Posters are being presented on Friday, November 9th, and Saturday, November 10th, from 8 AM to 8 PM, in Exhibition Hall E of the Walter E. Washington Convention Center.
Outlook for 2019

Updated results from Phase 1 trial of indoximod plus radiotherapy for pediatric patients with recurrent malignant brain tumors including initial survival data expected to be presented 1H 2019

Updated data from Phase 1 trial of indoximod plus radiotherapy in DIPG anticipated in 2019

Data from Phase 2 trial of NLG207 (CRLX101), a nanoparticle formulation of the topoisomerase 1 inhibitor, camptothecin, plus paclitaxel in recurrent ovarian cancer anticipated in 2019

Exhibit 99.1

Clinical Update
NewLink Genetics continues its clinical trials of indoximod in combination therapies for adult patients with newly diagnosed AML, pediatric patients with recurrent brain tumors, and pediatric patients with newly diagnosed DIPG. These targeted indications are those with unmet need where indoximod has produced encouraging early data and where standard-of-care therapy has not changed significantly for decades.
A Phase 2 study evaluating NLG207, a nanoparticle formulation of the topoisomerase 1 inhibitor camptothecin, in combination with paclitaxel for patients with recurrent ovarian cancer is complete, and data analysis is underway. NLG207 is an asset acquired from Cerulean Pharma Inc. in 2017. This trial is being conducted in conjunction with the Gynecological Oncology Group.
Board Changes
Paolo Pucci has resigned his position as a Director on NewLink Genetics’ Board effective October 31, 2018 due to the increasing responsibilities associated with his current position as CEO of ArQule, Inc. and current guidelines of proxy advisors regarding the number of directorships to be held by a CEO. With Mr. Pucci’s departure, NewLink’s Board will consist of seven directors.
Financial Results for the Three-Month Period Ended September 30, 2018
Cash Position: NewLink Genetics ended the quarter on September 30, 2018, with cash and cash equivalents totaling $122.1 million compared to $158.7 million for the year ending December 31, 2017.
R&D Expenses: Research and development expenses for the third quarter of 2018 were $7.6 million, a decrease of $10.9 million from $18.5 million for the same period in 2017. The decrease was due to reductions of $7.3 million in contract research and manufacturing spend, $2.5 million in clinical trial expense, $570,000 in personnel-related and stock compensation expense, $560,000 in supplies and licensing, and $100,000 in restructuring costs. These reductions were offset by an increase of $70,000 in legal and consulting expense.
G&A Expenses: General and administrative expenses for the third quarter of 2018 were $7.6 million, a decrease of $320,000 from $7.9 million for the same period in 2017. The decrease was due to reductions of $300,000 in restructuring costs, $240,000 in legal and consulting expenses, and $10,000 in personnel-related and stock compensation expense. These reductions were offset by an increase of $230,000 in supplies and other expense.
Net Loss: NewLink Genetics reported a net loss of $7.4 million or ($0.20) per diluted share for the third quarter of 2018 compared to a net loss of $20.6 million or ($0.69) per diluted share for the third quarter of 2017.
NewLink Genetics ended the quarter with 37,216,892 shares outstanding.
Conference Call and Webcast Details
The Company has scheduled a conference call and webcast for 4:30 p.m. ET today to discuss the results and to give an update on clinical and business development activities. NewLink Genetics’ senior management team will host the call, which will be open to all listeners. There will also be a question and answer session following the prepared remarks.
Access to the live conference call is available by dialing (855) 235-8286 (U.S.) or (267) 753-2161 (international) five minutes prior to the start of the call. The conference call will be webcast live and a link to the webcast can be accessed through the NewLink Genetics website at www.NewLinkGenetics.com in the "Investors & Media" section under "Events and Presentations" or by clicking here. To ensure a timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast. A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and using the passcode 1753698. The replay will be available for two weeks from the date of the call.
About Indoximod
Indoximod is an investigational, orally available small molecule targeting the IDO pathway. The IDO pathway is a key immuno-oncology target, suppressing immune response and allowing for immune escape by degrading tryptophan

Exhibit 99.1

with the resultant production of kynurenine. Indoximod reverses the immunosuppressive effects of low tryptophan and high kynurenine through mechanisms that include modulation of the AhR-driven transcription of genes that control immune function. This results in increased proliferation of effector T cells, increased differentiation into helper T cells rather than regulatory T cells, and downregulation of IDO expression in dendritic cells. Indoximod is being evaluated in combination with treatment regimens including chemotherapy, radiation, checkpoint blockade and cancer vaccines across multiple indications including recurrent pediatric brain tumors, DIPG, and AML

On November 1, 2018 Seattle Genetics, Inc. (Nasdaq:SGEN) reported that 31 abstracts featuring data from the broad ADCETRIS (brentuximab vedotin) development program have been accepted for presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place from December 1-4, 2018 in San Diego, Calif (Press release, Seattle Genetics, NOV 1, 2018, View Source [SID1234530659]). ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, which is expressed on the surface of Hodgkin lymphoma (HL) cells and several types of non-Hodgkin lymphoma. ADCETRIS is being evaluated globally as the foundation of care for CD30-expressing lymphomas in more than 70 corporate- and investigator-sponsored clinical trials.

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Data accepted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting include the following:

Data from the phase 3 ECHELON-2 clinical trial evaluating ADCETRIS in combination with chemotherapy in previously untreated patients with CD30-expressing peripheral T-cell lymphoma (PTCL) patients will be presented in an oral presentation on Monday, December 3, 2018 at 6:15 p.m. PT. Seattle Genetics and its partner Takeda Pharmaceutical Company Limited (Takeda) reported positive top-line results from the ECHELON-2 trial in October 2018. The trial demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS) of ADCETRIS in combination with CHP (cyclophosphamide, doxorubicin, prednisone) versus the control arm, CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone). The ADCETRIS plus CHP arm also demonstrated superior overall survival (OS), a key secondary endpoint, compared to CHOP. The ECHELON-2 trial is the first trial to demonstrate an OS advantage in this difficult to treat type of non-Hodgkin lymphoma. Seattle Genetics expects to submit in November 2018 a supplemental Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of ADCETRIS plus CHP in frontline CD30-expressing PTCL.
Several analyses from the phase 3 ECHELON-1 clinical trial evaluating ADCETRIS in combination with chemotherapy in frontline Stage III or IV classical HL adult patients, which formed the basis of FDA approval in this indication in March 2018, will be presented. Data presentations include additional analyses from the ECHELON-1 study, including PFS per investigator and outcomes in younger patients (18-30 years of age). These analyses are consistent with the previously reported modified PFS data and demonstrate improved outcomes in the ADCETRIS plus AVD (doxorubicin, vinblastine, dacarbazine) arm versus ABVD (doxorubicin, bleomycin, vinblastine, dacarbazine).
Preliminary results from a phase 2 study of ADCETRIS in combination with Opdivo (nivolumab) among patients with relapsed or refractory primary mediastinal large B-cell lymphoma (CHECKMATE 436 trial), as well as updated results from an ongoing phase 1/2 study evaluating the combination therapy in relapsed or refractory HL.
"There will be more than 30 data presentations from both corporate- and investigator-sponsored studies presented at the 2018 ASH (Free ASH Whitepaper) Annual Meeting evaluating ADCETRIS in a variety of CD30-expressing lymphoma settings. These presentations are reflective of a robust ADCETRIS clinical development program that we, in partnership with the oncology community, are conducting to improve the treatment outcomes for patients," said Roger Dansey, M.D., Chief Medical Officer of Seattle Genetics. "Importantly, and in collaboration with our partner Takeda, the results of the phase 3 ECHELON-2 clinical trial evaluating ADCETRIS in combination with CHP chemotherapy in frontline CD30-expressing peripheral T-cell lymphoma patients will be presented in an oral presentation on Monday, December 3rd. These data are the basis for our planned supplemental Biologics License Application to the FDA requesting approval of ADCETRIS in this setting, which we intend to submit in November 2018."

ADCETRIS is not currently approved for use in frontline PTCL or in combination with Opdivo.

Multiple corporate and investigator presentations will be featured at ASH (Free ASH Whitepaper). Abstracts can be found at www.hematology.org and include the following:

Saturday, December 1, 2018

Brentuximab Vedotin with Chemotherapy in Adolescents and Young Adults (AYA) with Stage III or IV Hodgkin Lymphoma: A Subgroup Analysis from the Phase 3 ECHELON-1 Study (Abstract #1647, poster presentation)
Older Patients (pts) with Previously Untreated Classical Hodgkin Lymphoma (cHL): A Detailed Analysis from the Phase 3 ECHELON-1 Study (Abstract #1618, poster presentation)
Phase 1/2 Study of Brentuximab Vedotin in Combination with Nivolumab in Patients with Relapsed or Refractory Classic Hodgkin lymphoma: Part 3 (Concurrent Dosing) Results and Updated Progression-Free Survival Results from Parts 1 and 2 (Staggered Dosing) (Abstract #1635, poster presentation)
Phase 1 Study of MDR1 Inhibitor Plus Brentuximab Vedotin in Relapsed/Refractory Hodgkin Lymphoma (Abstract #1636, poster presentation)
Real World Prevalence of Diagnostic Revision Among Patients with Peripheral T-cell Lymphomas (PTCL) in the US: Results of an Administrative Claims and Electronic Medical Record Analyses (Abstract #1633, poster presentation)
Superior Clinical Benefit of Brentuximab Vedotin in Mycosis Fungoides Versus Physician’s Choice Irrespective of CD30 Level of Large Cell Transformation Status in the Phase 3 ALCANZA Study (Abstract #1646, poster presentation)
A Phase II Study of Brentuximab Vedotin plus Adriamycin and Dacarbazine without Radiation in Non-Bulky Limited Stage Classical Hodgkin Lymphoma (Abstract #1654, poster presentation)
Treatment Patterns and Outcomes of Relapsed/Refractory Peripheral T-Cell Lymphoma (RR-PTCL) Patients Treated in the Community Oncology Setting (Abstract #1656, poster presentation)
Nivolumab Combined with Brentuximab Vedotin for Relapsed/Refractory Primary Mediastinal Large B-Cell Lymphoma: Preliminary Results From the Phase 2 CheckMate 436 Trial (Abstract #1691, poster presentation)
The Development and Validation of an Electronic Health Record (EHR)-Based Algorithm for Identifying Treatment Failure in Newly Diagnosed Hodgkin Lymphoma (HL) Treated in a US Community Oncology Setting (Abstract #2261, poster presentation)
Real World Evidence in Relapsed/Refractory Classical Hodgkin Lymphoma Patients Who Are Ineligible for Stem Cell Transplant in the United States (US) (Abstract #2268, poster presentation)
Toxicity Profile of Brentuximab Vedotin in Combination with Chemotherapy for Newly Diagnosed Patients with ALK+ ALCL: a Children’s Oncology Group Study ANHL12P1 (Abstract #1625, poster presentation)
Phase I Study of the Antibody-Drug Conjugate Brentuximab Vedotin Combined with Re-Induction Chemotherapy in Patients with CD30-Expressing Relapsed/Refractory Acute Myeloid Leukemia (Abstract #1431, poster presentation)
Phase 1 Results from a Phase 1/2 Study to Assess the Safety, Tolerability and Recommended Phase 2 Dose (RP2D) of Brentuximab Vedotin Plus Doxorubicin, Vinblastine and Dacarbazine (A+AVD) in Pediatric Patients (Pts) with Advanced Stage Newly Diagnosed Classical Hodgkin Lymphoma (cHL) (Abstract #1644, poster presentation)
Sunday, December 2, 2018

Brentuximab Vedotin Plus Chemotherapy in Patients with Advanced-Stage Classical Hodgkin Lymphoma (cHL): Evaluation of Modified Progression-Free Survival (mPFS) and Traditional PFS in the Phase 3 ECHELON-1 Study (Abstract #2904, poster presentation)
Resolution of Peripheral Neuropathy (PN) in Patients Who Received A+AVD or ABVD in the Phase 3 ECHELON-1 Trial (Abstract #2921, poster presentation)
Interim Analysis Results from an International, Multi-Centre, Non-Interventional Retrospective Study to Describe Treatment Pathways, Outcomes, and Resource Use in Patients with Classical Hodgkin Lymphoma: B-CD30+ Hodgkin Lymphoma International Multi-Centre Retrospective Study of Treatment Practices and Outcomes (B-HOLISTIC) (Abstract #2917, poster presentation)
Combining Brentuximab Vedotin with DHAP as Salvage Treatment in Relapsed/Refractory Hodgkin Lymphoma: the Phase II HOVON/LLPC Transplant BRaVE Study (Abstract #2923, poster presentation)
Peripheral T-Cell Lymphomas in Spain: Profiling Clinical, Phenotypic and Genetic Characteristics in Spanish Population (Abstract #2938, poster presentation)
Primary Mediastinal B-Cell Lymphoma: Evaluation of Clinicopathologic Diagnosis Compared to Gene Expression Based Diagnosis in a Clinical Trial with CD30+ B-Cell Lymphomas (Abstract #2959, poster presentation)
Utilization of a Novel Method of Detection of CD30 Expression in Diffuse Large B-cell Lymphoma (Abstract #2978, poster presentation)
Health-Related Quality of Life (HRQL) Trajectories during Treatment for Advanced Stage Pediatric Hodgkin Lymphoma (HL) (Abstract #3587, poster presentation)
Monday, December 3, 2018

The ECHELON-2 Trial: Results of a Randomized, Double-Blind, Active-Controlled Phase 3 Study of Brentuximab Vedotin and CHP (A+CHP) Versus CHOP in the Frontline Treatment of Patients with CD30+ Peripheral T-Cell Lymphomas (Abstract #997, oral presentation at 6:15 p.m. PT)
Longitudinal Adverse Event Assessment of the Combination of Ipilimumab, Nivolumab And Brentuximab Vedotin in Relapsed/Refractory Hodgkin Lymphoma: A Trial of the ECOG-ACRIN Cancer Research Group (E4412: Arms A-F) (Abstract #623, oral presentation at 8:00 a.m. PT)
A Phase I Study with an Expansion Cohort of the Combinations of Ipilimumab, Nivolumab and Brentuximab Vedotin in Patients with Relapsed/Refractory Hodgkin Lymphoma: A trial of the ECOG-ACRIN Research Group (E4412: Arms G-I) (Abstract #679, oral presentation at 10:30 a.m. PT)
B-CAP (brentuximab vedotin, cyclophosphamide, doxorubicin and predniso(lo)Ne) in Older Patients with Advanced-Stage Hodgkin Lymphoma: Results of a Phase II Intergroup Trial By the German Hodgkin Study Group (GHSG) and the Nordic Lymphoma Group (NLG) (Abstract #926, oral presentation at 4:45 p.m. PT)
Response-Adapted Therapy with Nivolumab and Brentuximab Vedotin (BV), Followed by BV and Bendamustine for Suboptimal Response, in Children, Adolescents, and Young Adults with Standard-Risk Relapsed/Refractory Classical Hodgkin Lymphoma (Abstract #927, oral presentation at 5:00 p.m. PT)
Productivity Loss Among Parent Caregivers is Associated with Poor Health-Related Quality of Life (HRQL) at the Initial Diagnosis Of Pediatric Advanced Stage Hodgkin Lymphoma (HL) (Abstract #975, oral presentation at 5:00 p.m. PT)
Baseline Tumor Transcriptome Characteristics Associated with the Response of Relapsed/Refractory Hodgkin Lymphoma Patients to Brentuximab Vedotin in Combination with Nivolumab (Abstract #2837, poster presentation)
Patient and Physician Preferences for First-Line Treatment of Classical Hodgkin Lymphoma in the United States (Abstract #4786, poster presentation)
Prolonged Overall Survival (OS) in a Subset of Responders to the Combination of Brentuximab Vedotin (Bv) and Bendamustine (B) in Heavily Treated Patients with Relapsed or Refractory Hodgkin Lymphoma (HL): Results of an International Multi-Center Phase I/II Experience (Abstract #2907, poster presentation)
About ADCETRIS (brentuximab vedotin)

ADCETRIS is being evaluated broadly in more than 70 clinical trials in CD30-expressing lymphomas. These include the recently completed phase 3 ECHELON-2 trial in frontline peripheral T-cell lymphomas (also known as mature T-cell lymphoma), the completed phase 3 ECHELON-1 trial in previously untreated Hodgkin lymphoma, the completed phase 3 ALCANZA trial in cutaneous T-cell lymphoma, and the ongoing CHECKMATE 812 trial of ADCETRIS in combination with Opdivo (nivolumab) for relapsed/refractory Hodgkin lymphoma.

ADCETRIS is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics’ proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS injection for intravenous infusion has received FDA approval for five indications in adult patients with: (1) previously untreated Stage III or IV classical Hodgkin lymphoma (cHL), in combination with chemotherapy, (2) cHL at high risk of relapse or progression as post-autologous hematopoietic stem cell transplantation (auto-HSCT) consolidation, (3) cHL after failure of auto-HSCT or failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (4) sALCL after failure of at least one prior multi-agent chemotherapy regimen, and (5) primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy.

Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL in 2013, and non-conditional approval for post-autologous stem cell transplantation (ASCT) consolidation treatment of Hodgkin lymphoma patients at increased risk of relapse or progression.

ADCETRIS received conditional marketing authorization from the European Commission in October 2012. The approved indications in Europe are: (1) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following ASCT, or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, (2) the treatment of adult patients with relapsed or refractory sALCL, (3) for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT, and (4) for the treatment of adult patients with CD30-positive cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

ADCETRIS has received marketing authorization by regulatory authorities in 72 countries for relapsed or refractory Hodgkin lymphoma and sALCL. See select important safety information, including Boxed Warning, below.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs

On November 1, 2018 Nordic Nanovector ASA (OSE: NANO) reported that an abstract reporting promising results from a research collaboration to develop a novel CD37-targeting alpha therapy for B-cell malignancies has been published (Press release, Nordic Nanovector, NOV 1, 2018, View Source [SID1234553489]). The complete results will be presented in a poster at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (1-4 December 2018 in San Diego, CA, USA).

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The research collaboration was established in June 2015 with Orano Med (formerly known as AREVA Med) to develop and investigate a next-generation targeted alpha therapy, comprising Nordic Nanovector’s chimeric anti-CD37 antibody (NNV003) and lead-212 (212Pb), for the treatment of B-cell malignancies.

The results published in the abstract relate to promising findings from preclinical studies investigating the dose-dependent efficacy and tolerability of 212Pb-NNV003 in human cell and mouse models of chronic lymphocytic leukaemia (CLL) and Burkitt’s lymphoma (a type of non-Hodgkin’s lymphoma, NHL).

In the studies, 212Pb-NNV003 was found to be well tolerated and led to a 90-100% survival rate in mouse models of CLL and NHL.

Jostein Dahle, Chief Scientific Officer of Nordic Nanovector, said: "There is a strong scientific rationale for combining our CD37-targeting approach with other cytotoxic payloads, including radionuclides and toxins. CD37 is an important target for B-cell malignancies as it is selectively expressed on the surface of B-cell malignancies. Alpha-particles have demonstrated good potential for targeted cancer therapies because their high energy is limited to a very short distance of just a few cell widths resulting in localised cytotoxicity while sparing surrounding healthy tissues. The development of 212Pb-conjugated CD37-targeted alpha therapy therefore offers the potential to treat leukaemias and lymphomas where there is no substantial tumour mass and tumour cells are near healthy tissues. We look forward to further results from this research programme."

Abstract 4422

Abstract title: Targeted Alpha Therapy with 212Pb-NNV-003 for the Treatment of CD37 Positive B-Cell Chronic Lymphocytic Leukemia (CLL) and Non-Hodgkin Lymphoma (NHL)

Authors: A. Saidi et al.

Session Name: 642. CLL: Therapy, excluding Transplantation: Poster III

Date: Monday, 3 December 2018

Presentation Time: 6:00 PM – 8:00 PM Pacific time

Location: San Diego Convention Center, Hall GH

The abstract is available at View Source and the poster will be published on the Nordic Nanovector website to coincide with the session.

About ASH (Free ASH Whitepaper)

The ASH (Free ASH Whitepaper) annual meeting is the premier event for scientific exchange in the field of haematology, attracting more than 20,000 attendees from all over the world. Typically, more than 5,000 scientific abstracts are submitted each year, and more than 3,000 abstracts are accepted for oral and poster presentations through an extensive peer review process.

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.

Follow Roche on Twitter via @Roche and keep up to date with ASH (Free ASH Whitepaper) Annual Meeting news and updates by using the hashtag #ASH18.

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.