On November 6, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the third quarter 2019 and provided an operational update (Press release, Bellicum Pharmaceuticals, NOV 6, 2019, View Source [SID1234550444]).

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"Bellicum made significant strides in the third quarter repositioning the company to focus exclusively on our GoCAR programs, as we seek to clinically demonstrate how our technology may extend the impact of CAR-T therapies to more patients," said Rick Fair, President and Chief Executive Officer of Bellicum Pharmaceuticals. "By streamlining the organization and other cost-saving initiatives and executing a financing in August, we believe we have positioned the company to generate meaningful clinical data on our ongoing GoCAR programs and to advance a third GoCAR program toward the clinic."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-601 GoCAR-T

Bellicum anticipates presenting new translational data from cohort 5B in the BPX-601 Phase 1/2 trial in early 2020. These data are expected to provide additional evidence on the proposed mechanisms of action of iMC activation in BPX-601, including cell expansion and persistence, cytokine production, tumor infiltration, and CAR-T activity in the tumor microenvironment. Bellicum is currently enrolling cohort 5C. Data from this cohort will be used to evaluate the safety of repeat rimiducid dosing to re-activate iMC over time, which is intended to deepen and extend the treatment effect. Patient enrollment has proceeded more slowly than anticipated primarily as the result of the amended protocol limiting enrollment to second line patients. Initial results from Cohort 5C are expected to be presented in the second half of 2020.
BPX-603 GoCAR-T

In response to Bellicum’s IND application for BPX-603, the FDA has responded with a request for additional preclinical data to better characterize the potential risk of off-tumor / on-target toxicity before IND clearance. Bellicum is engaged in discussions with the FDA to align on a plan to address the request.
Research Programs

An abstract for a preclinical investigation from Bellicum’s natural killer cell GoCAR program has been accepted for poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting on November 8, 2019.
Rivo-cel

Bellicum is actively pursuing a strategic partner for rivo-cel to lead future development and commercialization for this product candidate. Bellicum has reduced and expects to continue to reduce its rivo-cel related activities.
Corporate Highlights

In August, Bellicum announced receipt of aggregate gross proceeds of $69.6 million, including gross proceeds of $57.5 million from an underwritten public offering and a $12.1 million private placement option fee related to its private placement of up to $70 million in additional potential gross proceeds.
Bellicum is actively pursuing a partner to purchase its manufacturing facility and establish a preferred supply agreement with the goal of reducing operating costs while maintaining viral vector and cell therapy development capabilities and dedicated manufacturing capacity for its programs.
Third Quarter 2019 Financial Results and Outlook

R&D Expenses: Research and development (R&D) expenses were $14.3 million for the third quarter of 2019, compared to $16.4 million for the third quarter of 2018. The reduction in expenses in the third quarter of 2019 resulted primarily from reduced expenses related to rivo-cel and reduced general R&D expenses, partially offset by higher expenditures related to the GoCAR-T platform. R&D expenses for the nine months ended September 30, 2019 were $51.0 million compared to $51.4 million for the comparable period in the prior year.

G&A Expenses: General and administrative (G&A) expenses were $9.2 million for the third quarter of 2019 compared to $7.0 million during the comparable period in 2018. The higher expenses in the third quarter 2019 relative to the comparable period in 2018 were primarily due to an accrual of severance costs arising from reductions in rivo-cel related activities. G&A expenses for the nine months ended September 30, 2019 were $24.3 million compared to $18.0 million for the first nine months of 2018.

Net Loss: Bellicum reported a net loss of $32.0 million for the third quarter of 2019 compared to a net loss of $23.8 million for the third quarter of 2018. The results included non-cash, share-based compensation charges of $1.6 million and $3.7 million for the third quarter of 2019 and 2018, respectively. Net loss for the nine months ended September 30, 2019 was $83.5 million compared to a loss of $70.8 million for the nine months ended September 30, 2018.

Shares Outstanding: At October 31, 2019, Bellicum had 49,616,316 shares of common stock outstanding and 541,500 shares of preferred stock outstanding. Each preferred share can be converted into 100 shares of common stock.

Cash Position and Guidance: Bellicum reported cash, restricted cash and investments totaling $106.9 million as of September 30, 2019, compared to $98.0 million at December 31, 2018. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into 2021.

Conference Call and Webcast

Bellicum’s management will host a webcast and conference call today at 5 p.m. ET / 2 p.m. PT, November 6, 2019, to discuss the financial results for the third quarter 2019 and provide a corporate update. The live call may be accessed by dialing (877) 407-3103 for domestic callers and (201) 493-6791 for international callers. A live webcast of the call will be available from the Investors and Media section of the company’s website at www.bellicum.com and a replay will be available shortly after the live event.

About BPX-601

BPX-601, the company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, gastric, and prostate cancers.

On November 6, 2019 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH / TSX: AUP) (the "Company") reported that it will release its third quarter 2019 financial results on Thursday, November 14, 2019, after the market closes (Press release, Aurinia Pharmaceuticals, NOV 6, 2019, View Source [SID1234550443]). Aurinia’s management team will host a conference call to discuss the Company’s financial results and to provide a general business update.

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The conference call and webcast is scheduled for November 14, 2019 at 4:30pm ET. In order to participate in the conference call, please dial +1-877-407-9170 (Toll-free U.S. & Canada). An audio webcast can be accessed under "News/Events" through the "Investors" section of the Aurinia corporate website at www.auriniapharma.com. A replay of the webcast will be available on Aurinia’s website.

On November 6, 2019 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended September 30, 2019 (Press release, Athersys, NOV 6, 2019, View Source [SID1234550442]).

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Highlights of the third quarter of 2019 and recent events include:

Steady advancement of both MultiStem ischemic stroke clinical trials, including the HEALIOS K.K. ("Healios") TREASURE study in Japan and in our Phase 3 registrational study MASTERS-2;

Continued support for the Healios’ acute respiratory distress syndrome ("ARDS") trial, the ONE-BRIDGE study in Japan, which continues to have steady enrollment;

Completion of one-year follow up assessments for our ARDS trial, the MUST-ARDS study, and initiation of planning for subsequent ARDS trial;

Advanced preparations for planned Phase 2 trauma clinical study;

Progressed planning for commercial readiness with our manufacturing process, technical transfer operations and future manufacturing plans;

Engaged in partnering discussions with companies interested in MultiStem commercialization rights in Europe and other regions;

MultiStem recognized in the first ESOT Leonardo Da Vinci Research Innovation Award received by Emily R. Thompson from the University of Newcastle for her work using MultiStem in human kidney transplant research;

Entered in new equity facility that will replace the current facility, providing us with access of up to $100 million to support operational and other initiatives over the next several years;

Recognized revenues of negative $0.4 million and net loss of $12.0 million, or $0.08 net loss per share, for the quarter ended September 30, 2019; and

Ended the 2019 third quarter with $40.4 million of cash and cash equivalents.

"We continue to make steady progress in our ongoing clinical programs, maintaining our focus on the critical care indications where our prior clinical results and extensive preclinical data suggest there is an excellent opportunity for MultiStem to address substantial areas of unmet medical need. Each of the indications we are focused on represent significant market opportunities, where current standard of care is limited, cost of care is high, and the disability burden and the quality of life impact on the patient and family is substantial," commented Dr. Gil Van Bokkelen, Chairman & CEO of Athersys. "We believe that MultiStem can help address substantial gaps and limitations in current standard of care, meaningfully improving clinical outcomes for patients, and delivering substantial value to the healthcare system.

"We are also heavily focused on strategic partnering activities in Europe and other geographies of interest, while we also pursue other strategic opportunities that have the potential to add value and strengthen our balance sheet. We are focused on preparing for commercialization in the future and maintaining a strong financial position while we pursue these initiatives," concluded Dr. Van Bokkelen.

Third Quarter Results
Our revenues are generally derived from license fees, manufacturing-related activities for Healios, other contract revenue from our collaborations and grant revenue. Revenues were negative $0.4 million for the three months ended September 30, 2019 compared to $2.3 million for the three months ended September 30, 2018, which were primarily related to our collaboration with Healios. In the third quarter of 2019, we determined that the estimated variable transaction price of product supply decreased due to a reduction in the underlying cost per dose that occurred during the quarter. This reduction exceeded the amount of revenue generated during the quarter. Royalty revenue ceased late in 2018 upon a licensee’s decision to discontinue distribution of the licensed product.
Research and development expenses decreased to $8.9 million for the three months ended September 30, 2019 from $9.5 million for the comparable period in 2018. The $0.6 million net decrease is associated with decreases in clinical trial and manufacturing process development costs of $1.1 million and license fees of $0.2 million, with such decreases partially offset by increases in personnel costs of $0.2 million, outside services of $0.2 million, stock compensation costs of $0.2 million, and consulting costs of $0.1 million. Included in our clinical expenses are costs associated with providing manufacturing services to Healios, which are invoiced to Healios in accordance with our collaboration agreements.
General and administrative expenses increased to $3.0 million for the three months ended September 30, 2019 from $2.6 million in the comparable period in 2018. The $0.4 million increase was due primarily to increased legal and professional fees, outside services and stock compensation costs compared to the same period last year.
Net loss for the third quarter of 2019 was $12.0 million compared to a net loss of $9.7 million in the third quarter of 2018. The difference reflects the above variances, as well as an increase in other income.
During the nine months ended September 30, 2019, net cash used in operating activities was $25.2 million compared to $8.8 million in the nine months ended September 30, 2018, with 2018 being impacted by proceeds from the Healios collaboration expansion. At September 30, 2019, we had $40.4 million in cash and cash equivalents, compared to $51.1 million at December 31, 2018.

Conference Call
Gil Van Bokkelen, Chairman and Chief Executive Officer, Laura Campbell, Senior Vice President of Finance, and Karen Hunady, Director of Corporate Communications and Investor Relations will host a conference call today to review the results as follows:
Date

November 6, 2019
Time

4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada

(877) 396-3286
Telephone access: International

(647) 689-5528
Access code

4183148
Live webcast

www.athersys.com, under the Investors/Events section

We encourage shareholders to listen using the webcast link and to use the phone line if you intend to ask a question. A replay will be available on the webcast at www.athersys.com under the Investors section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on November 13, 2019 by dialing (800) 585-8367 or (416) 621-4642 and entering Encore passcode 4183148.

On November 6, 2019 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported that it will present long-term tab-cel (tabelecleucel) clinical outcomes from a multicenter Expanded Access Protocol (EAP) study for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) (Press release, Atara Biotherapeutics, NOV 6, 2019, View Source [SID1234550441]). These results, along with findings described in three additional abstracts, will be presented at the 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, to be held December 7-10, 2019, in Orlando, Florida.

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"I am pleased with the tab-cel long-term clinical outcomes for patients with EBV+ PTLD we continue to observe in our EAP study," said Pascal Touchon, President and Chief Executive Officer of Atara Biotherapeutics. "The well-tolerated safety profile, high response rate and durable 2-year survival data are consistent with our previous tab‑cel clinical experience and reinforces our confidence in tab-cel as a potentially transformative off-the-shelf, allogeneic T-cell immunotherapy for this often-deadly ultra-rare cancer."

Twenty-six EBV+ PTLD patients who failed prior rituximab treatment regimens were enrolled in a tab‑cel EAP study (EAP-201) as of June 2018, after which EAP-201 was amended to focus on expanded access for patients with EBV+ PTLD and other EBV+ diseases who are not eligible for Atara’s ongoing tab‑cel Phase 3 studies (EAP-901, NCT02822495). The findings presented here are as of June 3, 2019. Consistent with prior studies, no tab-cel related adverse events leading to discontinuation or death occurred.

A subgroup of 22 EAP-201 EBV+ PTLD patients with adequate ECOG performance status, no CNS disease and no PTLD-related ventilatory support, would have likely met the eligibility criteria for Atara’s ongoing tab‑cel Phase 3 studies.

The overall response rate (ORR) for patients in this EAP-201 subgroup with EBV+ PTLD following HCT (n=11) and SOT (n=11) was 55 and 82 percent with an estimated two-year overall survival of 79 and 81 percent, respectively.

For all EBV+ PTLD patients enrolled in EAP-201, the ORR was 50 and 83 percent for HCT (n=14) and SOT (n=12), respectively.

Atara will also present additional findings describing the hospitalization burden of patients with EBV+ PTLD following SOT who failed first-line rituximab or rituximab plus chemotherapy.

In addition, Atara’s Moffitt Cancer Center collaborators will present two abstracts detailing next-generation CAR T technologies, licensed exclusively to Atara, and designed to enhance persistence while reducing susceptibility to exhaustion and suppressive immune microenvironments.

Details of the ASH (Free ASH Whitepaper) presentations and abstracts are as follows:

Abstract 4071: Long-Term Outcomes of Subjects with Epstein-Barr Virus-Driven Post-Transplant Lymphoproliferative Disorder (EBV+PTLD) Following Solid Organ (SOT) or Allogeneic Hematopoietic Cell Transplants (HCT) Treated with Tabelecleucel on an Expanded Access Program
Poster Presentation Date and Time: Monday, December 9, 6:00 – 8:00 p.m. EST
Session Title: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphoma) – Results from Prospective Clinical Trials: Poster III
Location: Orange County Convention Center, Hall B
Authors: Susan Prockop, M.D.1, Ran Reshef, M.D.2, Donald E. Tsai, M.D., Ph.D.3, Nancy Bunin, M.D.4, Rolla Abu-Arja, M.D.5, Kris Michael Mahadeo, M.D.6, Wen-Kai Weng, M.D., Ph.D.7, Koen Van Besien, M.D., Ph.D.8, David Loeb, M.D., Ph.D.9, Sunita Dwivedy Nasta, M.D.10, Eneida R. Nemecek, M.D., M.B.A., M.S.11, Minoti Hiremath, MBBS, Ph.D.12, Susan Yue, M.D.13, Yan Sun, Ph.D.13, Willis H Navarro, M.D.12 and Sarah Nikiforow, M.D., Ph.D.14
Affiliations: 1Memorial Sloan Kettering Cancer Center, New York, NY; 2Columbia University Irving Medical Center, New York, NY; 3Loxo Oncology, Stamford, CT; 4Children’s Hospital of Philadelphia, Philadelphia, PA; 5Nationwide Children’s Hospital, Columbus, OH; 6MD Anderson Cancer Center, Houston, TX; 7Division of Blood and Marrow Transplantation, Department of Medicine, Stanford Univ. School of Med., Stanford, CA; 8Division of Hematology and Oncology, Weill Cornell Medical College, New York, NY; 9Montefiore, Bronx, NY; 10University of Pennsylvania, Philadelphia; 11Pediatric Hematology/Oncology & Bone Marrow Transplantation, OHSU Knight Cancer Institute Doernbecher Children’s Hospital, Portland, OR; 12Atara Biotherapeutics, South San Francisco, CA; 13Atara Biotherapeutics, Thousand Oaks, CA; 14Dana-Farber Cancer Institute, Boston, MA

Abstract 65: Burden of Hospitalizations Due to Epstein-Barr Virus-Driven Post-Transplant Lymphoproliferative Disorder (EBV+ PTLD) in Patients Who Failed First Line Rituximab or Rituximab Plus Chemotherapy Following Solid Organ Transplant (Post-SOT): A Retrospective Chart Review Study of German PTLD Registry
Oral Presentation Date and Time: Saturday, December 7, 8:30 a.m. EST
Session Title: 902. Health Services Research – Malignant Conditions (Lymphoid Disease): Health Care Utilization and Quality of Life
Location: Orange County Convention Center, W308
Authors: Heiner Zimmermann, M.D.1, Hairong Xu, M.D., Ph.D.2, Arie Barlev, Pharm.D.3, Yang Zhang, Ph.D.2, Dhanalakshmi Thirumalai2, Crystal Watson, M.S.3 and Ralf Ulrich Trappe, M.D.1
Affiliations: 1Internal Medicine II: Hematology and Oncology, Diako Hospital, Bremen, Germany; 2Atara Biotherapeutics, Inc, Thousand Oaks, CA; 3Atara Biotherapeutics, Inc, South San Francisco, CA
Summary: Approximately one-half of PTLD patients treated with rituximab, currently first-line therapy for PTLD, are relapsed or refractory to treatment. This review of the German PTLD registry database showed a substantial hospitalization burden for patients failing rituximab treatment, accounting for approximately 20% of patients’ hospitalization time after initial PTLD diagnosis with approximately 10% of the time spent in the ICU.

Abstract 5826: The Economic Burden of Short-Term Adverse Events Associated with the CHOP Chemotherapy Regimen in Patients with Lymphoproliferative Disorders in the United States; A Comprehensive Literature Review
Presentation Date and Time: N/A; will appear in the November supplemental online-only issue of Blood
Authors: Crystal Watson, M.S. 1, Arie Barlev, Pharm.D. 1, Jodie Worrall1, Steve Duff, M.S.3, Rachel Beckerman, Ph.D.2
Affiliations: 1Atara Biotherapeutics, Inc, South San Francisco, CA; 2Maple Health Group, New York, NY; 3Veritas Health Economic Consulting, Carlsbad, CA

Abstract 867: Mutation of the CD28 Costimulatory Domain Confers Enhanced CAR T Cell Function
Oral Presentation Date and Time: Monday, December 9, 5:00 p.m. EST
Session Title: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: New Approaches
Location: Orange County Convention Center, W224ABEF
Authors: Justin C Boucher1, Gongbo Li1, Hiroshi Kotani1, Maria L Cabral2, Dylan Morrissey3, Sae Bom Lee1,4, Kristen Spitler1, Nolan Beatty1,4, Bishwas Shrestha1, Bin Yu1, Aslamuzzaman Kazi5, Xuefeng Wang6, Said M Sebti5, Marco L Davila1,3
Affiliations: 1Department of Blood & Marrow Transplant and Cellular Immunotherapy, Division of Clinical Science, H. Lee Moffitt Cancer Center, Tampa, FL; 2Department of Cell Biology, Microbiology, and Molecular Biology, College of Arts and Sciences, University of South Florida, Tampa, FL; 3Morsani College of Medicine, University of South Florida Health, Tampa, FL; 4Cancer Biology PhD Program, University of South Florida, Tampa, FL; 5Drug Discovery Program, H. Lee Moffitt Cancer Center, Tampa, FL; 6Department of Biostatistics and Bioinformatics, H. Lee Moffitt Cancer Center, Tampa, FL
Summary: Current generation Chimeric antigen receptors (CARs) utilizing CD28 co-stimulatory domains have been reported to drive high levels of T cell activation that also lead to exhaustion and shortened persistence. Three signaling subdomains present on CD28 differentially regulate this drive towards exhaustion. Using a combination of in vitro and in vivo genomic studies, this study demonstrates CAR T cells using a modified version of CD28, with tailored signaling driven through the PYAP (mut06) subdomain, optimizes CAR T cell signaling by lowering transcription factors that drive exhaustion.

Abstract 4438: MDSC Suppression of CAR T cell can be Reduced by Targeted Signaling Disruption
Poster Presentation Date and Time: Monday, December 9, 6:00 – 8:00 p.m. EST
Session Title: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: Poster III
Location: Orange County Convention Center, Hall B
Authors: Estelle V Cervantes1, Justin C Boucher2, Sae Bom Lee2,3, Kristen Spitler2, Kayla Reid2, Marco L Davila1,2
Affiliations: 1Morsani College of Medicine, University of South Florida, Tampa, FL, 33612; 2Department of Blood & Marrow Transplant and Cellular Immunotherapy, Division of Clinical Science, H. Lee Moffitt Cancer Center, Tampa, FL, 33612; 3Cancer Biology PhD Program, University of South Tampa, Tampa, FL, 33612
Summary: CAR T persistence is one of the challenges faced by CAR T cell therapy. Myeloid derived suppressor cells (MDSCs) function as key contributors to preventing persistence of CAR T cells. Data from this study show MDSCs can suppress CAR T cell function when present in vitro, including during CAR T production, as demonstrated by reductions in CAR T cell activation and cytotoxicity. CAR T cells expressing an optimized CD28 co-stimulatory domain (mut06) were less susceptible to the suppressive effects of MDSCs in vitro and in vivo. These data support that mut06 CARs may improve activity and persistence in the presence of MDSCs and may also improve CAR T production in vitro by overcoming the effects of MDSCs.

On November 6, 2019 AstraZeneca reported that it will present the first data from the Phase III ELEVATE-TN trial assessing Calquence (acalabrutinib), a next-generation selective Bruton’s tyrosine kinase (BTK) inhibitor, in patients with previously untreated chronic lymphocytic leukaemia (CLL), as well as data from novel-combination trials across multiple blood cancers at the 2019 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, USA, December 7-10 (Press release, AstraZeneca, NOV 6, 2019, View Source [SID1234550440]).1

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The Company will present over 30 abstracts, including seven oral presentations, in CLL, mantle cell lymphoma (MCL), acute myeloid leukaemia (AML), diffuse large B-cell lymphoma (DLBCL) and multiple myeloma (MM). Key data include:

The first presentation of data from the pivotal Phase III ELEVATE-TN trial evaluating Calquence in combination with obinutuzumab and Calquence monotherapy versus obinutuzumab combined with chlorambucil chemotherapy in previously untreated CLL
Long-term efficacy, safety and tolerability data on Calquence in relapsed or refractory CLL from the Phase I/II ACE-CL-001 trial
First-time data on roxadustat as a potential new treatment for anaemia in patients with primary myelodysplastic syndrome (MDS)
Dave Fredrickson, Executive Vice President, Oncology Business Unit said: "AstraZeneca continues to demonstrate its strength in haematology, presenting new research at ASH (Free ASH Whitepaper) that spans targeted therapies across eight blood cancers. This year we are especially excited to present the ELEVATE-TN data demonstrating the impressive efficacy and tolerability of Calquence in 1st-line chronic lymphocytic leukaemia."

Key headline data from the Calquence Phase III ELEVATE-TN trial

Efficacy measure

Calquence plus obinutuzumab

N = 179

Calquence monotherapy

N = 179

Obinutuzumab plus chlorambucil

N = 177

Stratified analysis, median follow-up 28 months

Hazard ratio for PFS endpoint (vs. obinutuzumab + chlorambucil), stratified analysis

HR 0.10
(primary endpoint)

95% CI 0.06–0.17, P<0.0001

median not reached

HR 0.20
(secondary endpoint)

95% CI 0.13–0.30,
p<0.0001

median not reached

n/a

median 22.6 months

Select adverse events (AEs) include infusion reactions, which were less frequent with Calquence plus obinutuzumab (13%) than with obinutuzumab plus chlorambucil (40%). Additionally, AEs led to treatment discontinuation in 11% of patients on Calquence plus obinutuzumab, 9% of patients on Calquence, and 14% of patients on obinutuzumab plus chlorambucil. With >2 y of follow-up, 79% of patients in both the Calquence-containing arms remain on Calquence as a monotherapy. Other select AEs (Calquence plus obinutuzumab or Calquence vs chlorambucil plus obinutuzumab) included atrial fibrillation (any grade: 3% or 4% vs. 1%), bleeding (any grade/Grade ≥3: 43%/2% or 39%/2% vs. 12%/0%), and hypertension (Grade ≥3: 3% or 2% vs. 3%).

Full data from the ELEVATE-TN trial will be presented at ASH (Free ASH Whitepaper) by the primary investigators. AstraZeneca has submitted Calquence for US regulatory review in 1st-line and relapsed/refractory CLL.

Raising the bar for CLL treatment outcomes with Calquence

In addition to the oral presentation of the ELEVATE-TN results, key presentations include:

An oral presentation on preliminary data from a Phase II investigator-initiated trial evaluating Calquence combined with obinutuzumab and venetoclax in patients with previously untreated CLL, including high-risk disease status and a trial-in-progress poster detailing an ongoing Phase III trial to evaluate this novel combination in patients with previously untreated CLL without del(17p) or TP53 mutation.
Long-term (42-month) follow-up results from the Phase I/II ACE-CL-001 trial confirming Calquence initial efficacy from this trial for the treatment of relapsed or refractory CLL and providing additional data on duration of response and long-term tolerability.
Exploring a potential treatment option for a challenging comorbidity in blood cancer

An oral presentation on first-time data from a global Phase III trial evaluating roxadustat to treat anaemia in patients with primary MDS. Considered a type of cancer, MDS is a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells. Approximately one in three MDS patients can progress to AML.2
Exploring potential new medicines from the pipeline and new treatment strategies for aggressive or treatment-resistant blood cancers

In AML, an oral presentation and four poster presentations, including results from an Imfinzi (durvalumab) and azacitidine combination for the 1st-line treatment of older, chemotherapy-ineligible patients and data from a Phase I/II clinical trial of AZD2811(nanoparticles) as a monotherapy or in combination with azacitidine in previously untreated or relapsed/refractory patients who are not eligible for intensive induction therapy.
In DLBCL, five abstracts, including a poster presentation detailing the ongoing Phase I PRISM trial of Calquence in four different combinations with potential new medicines targeting STAT3, ATR, CD47 and BRD4.
In MM, three poster presentations, including results of a Phase I trial of MEDI2228, a BCMA antibody-PBD conjugate and potential new medicine, as a monotherapy and in combinations with bortezomib and DNA-damage response medicines and results from an in vitro trial of AZD4785 alone or with proteasome inhibitors targeting mutant KRAS.
Key AstraZeneca presentations at ASH (Free ASH Whitepaper) 2019

Lead author

Abstract title

Presentation details

Chronic lymphocytic leukaemia

Sharman, J.

ELEVATE TN: Phase 3 Study of Acalabrutinib Combined with Obinutuzumab (O) or Alone vs O Plus Chlorambucil (Clb) in Patients (Pts) With Treatment-Naive Chronic Lymphocytic Leukemia (CLL)

Oral Presentation

Saturday 7 December

07:30 ET

Orange County Convention Center, Hall D

Lampson, BL.

Preliminary Safety and Efficacy Results from a Phase 2 Study of Acalabrutinib, Venetoclax and Obinutuzumab in Patients with Previously Untreated Chronic Lymphocytic Leukemia (CLL)

Oral Presentation

Saturday 7 December

07:45 ET

Orange County Convention Center, Hall D

Frei, CR.

Treatment Patterns and Outcomes of 1205 Patients on Novel Agents in the US Veterans Health Administration (VHA) System: Results from Retrospective EMR and Chart Review Study in the Real-World Setting

Oral Presentation

Monday 9 December

15:15 ET

Orange County Convention Center, Valencia A (W415A)

Goyal, RK.

Overall Survival, Adverse Events, and Economic Burden in Medicare Patients with Chronic Lymphocytic Leukemia Receiving Cancer-Directed Therapy

Oral Presentation

Monday 9 December

15:15 ET

Orange County Convention Center, Valencia A (W415A)

Furman, RR.

Acalabrutinib Monotherapy in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia: 42-Month Follow-Up of a Phase 2 Study

Poster Presentation

Sunday 8 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Brown, JR.

A Phase 3 Trial Comparing the Efficacy and Safety of Acalabrutinib in Combination with Venetoclax with or without Obinutuzumab, Compared with Investigator’s Choice of Chemoimmunotherapy in Patients with Previously Untreated Chronic Lymphocytic Leukemia (CLL) without del(17p) or TP53 Mutation

Poster Presentation

Monday 9 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Mantle cell lymphoma

Kabadi, S.

Overall Survival, Adverse Events, and Economic Burden in Medicare-Insured Patients with Mantle Cell Lymphoma Receiving Cancer-Directed Therapy

Oral Presentation

Saturday 7 December

08:00 ET

Orange County Convention Center, W308

Ryan, K.

Characteristics of Mantle Cell Lymphoma (MCL) and Chronic Lymphocytic Leukemia (CLL) Patients Treated with Acalabrutinib in a Real World Setting in the United States

Poster Presentation

Sunday 8 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Acute myeloid leukaemia

Zeidan, A.

Efficacy and Safety of Azacitidine (AZA) in Combination with the Anti-PD-L1 Durvalumab (durva) for the Front-line Treatment of Older Patients (pts) with Acute Myeloid Leukemia (AML) Who Are Unfit for Intensive Chemotherapy (IC) and Pts with Higher-Risk Myelodysplastic Syndromes (HR-MDS): Results from a Large, International, Randomized Phase 2 Study

Oral Presentation

Monday 9 December

16:30 ET

Orange County Convention Center, Chapin Theater (W320)

Donnellan, W.

A Phase I/II Study of AZD2811NP as Monotherapy or in Combination in Treatment-Naïve or R/R AML/MDS Patients Not Eligible for Intensive Induction Therapy

Poster Presentation

Monday 9 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Diffuse large B-cell lymphoma

Roschewski, M.

PRISM: A Platform Protocol for the Treatment of Relapsed/Refractory Aggressive Non-Hodgkin Lymphoma

Poster Presentation

Sunday 8 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Moskowitz, CH.

Safety and Antitumor Activity Study of Loncastuximab Tesirine and Durvalumab in Diffuse Large B-Cell, Mantle Cell, or Follicular Lymphoma

Multiple myeloma

Xing, L.

Anti-BCMA PBD MEDI2228 combats drug resistance and synergizes with bortezomib and inhibitors to DNA damage response in multiple myeloma

Poster Presentation

Saturday 7 December

17:30 – 19:30 ET

Orange County Convention Center, Hall B

Sacco, A.

Specific targeting of KRAS using a novel high-affinity KRAS antisense oligonucleotide in myeloma.

Poster Presentation

Sunday 8 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Xing, L.

MEDI2228, a novel BCMA antibody-PBD conjugate, sensitizes human multiple myeloma cells to NK cell-mediated cytotoxicity and upregulates CD38 expression in MM cells: clinical implication

Poster Presentation

Sunday 8 December

18:00 – 20:00 ET

Orange County Convention Center, Hall B

Primary MDS-induced anaemia

Henry, D.

Roxadustat (FG4592; ASP1517; AZD9941) in the Treatment of Anemia in Patients with Lower Risk Myelodysplastic Syndrome (LR-MDS) and Low Red Blood Cell (RBC) Transfusion Burden (LTB)

Oral Presentation

Monday 9 December

16:30 – 18:00 ET

Orange County Convention Center, W311ABCD

About ELEVATE-TN

ELEVATE-TN (ACE-CL-007) is a randomised, multicentre, open-label Phase III trial evaluating the safety and efficacy of Calquence in combination with obinutuzumab, a CD20 monoclonal antibody, or Calquence alone vs. chlorambucil, a chemotherapy, in combination with obinutuzumab in previously untreated patients with CLL. In the trial, 535 patients were randomised (1:1:1) into three arms. Patients in the first arm received chlorambucil in combination with obinutuzumab. Patients in the second arm received Calquence (100mg twice daily until disease progression or unacceptable toxicity) in combination with obinutuzumab. Patients in the third arm received Calquence monotherapy (100mg twice daily until disease progression or unacceptable toxicity).3

The primary endpoint is progression-free survival (PFS) in the Calquence and obinutuzumab arm compared to the chlorambucil and obinutuzumab arm, assessed by an independent review committee (IRC), and a key secondary endpoint is IRC-assessed PFS in the Calquence monotherapy arm compared to the chlorambucil and obinutuzumab arm. Other secondary endpoints include objective response rate, time to next treatment and overall survival.3

About AstraZeneca in Haematology

Leveraging its strength in oncology, AstraZeneca has established haematology as one of four key oncology disease areas of focus. The Company’s haematology franchise includes two US FDA-approved medicines and a robust global development programme for a broad portfolio of potential blood cancer treatments. Acerta Pharma serves as AstraZeneca’s haematology research and development arm. AstraZeneca partners with like-minded science-led companies to advance the discovery and development of therapies to address unmet need.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of

new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline

of small molecules and biologics in development, we are committed to advance Oncology as one of AstraZeneca’s four Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.