GlycoMimetics Presents New AML Data with Uproleselan at 60th ASH Annual Meeting

On December 3, 2018 GlycoMimetics, Inc. (NASDAQ:GLYC) reported that new data on uproleselan-treated high risk patients with both relapsed/refractory and newly diagnosed AML were presented at an oral session during the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, GlycoMimetics, DEC 3, 2018, View Source [SID1234531812]). An analysis of clinical outcomes from the Phase 1/2 clinical study showed that uproleselan (GMI-1271) resulted in the majority of evaluable patients achieving a stringent level of measurable residual disease (MRD) negativity, an effect which translated into extended overall survival relative to matched, historical controls.1-4

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Additionally, an analysis of E-selectin ligand expression on leukemic cells demonstrated that detectable levels are present in every patient tested, providing strong clinical evidence of biological relevance in this disease setting. In bone marrow blasts, leukemic stem cell expression of E-selectin ligand correlated with leukemic blast E-selectin ligand expression (p<0.0001), consistent with the hypothesis that E-selectin-mediated interactions are a mechanism of chemoresistance. Additionally, investigators assessed the association between baseline E-selectin ligand expression and clinical outcomes using a log-rank test. In the R/R cohort of patients (n=22), this analysis demonstrated that ≥10% E-selectin ligand expression at baseline is correlated with prolonged survival (p<0.01) for patients treated with uproleselan. This observation is important since separately Chien et al. (Abstract 1513) report that high levels of E-selectin ligand in patients not treated with uproleselan correlate with worse clinical prognosis.

"The new MRD and correlative efficacy data in difficult-to-treat patients, when combined with the already encouraging response rate and survival results from this trial, further demonstrate the potential of uproleselan to be an important new treatment option in AML," said Daniel J. DeAngelo, M.D., Ph.D., Chief of the Division of Leukemia at the Dana-Farber Cancer Institute and Brigham and Women’s Hospital, and the trial’s lead investigator. "The fact that more than half of the evaluable patients achieved a stringent level of MRD negativity is particularly noteworthy as uproleselan’s mechanism of action is to selectively disrupt the relationship between leukemic cells and the bone marrow microenvironment."

"It is now clearly established that patients with AML who express E-selectin ligands on their leukemic cells have more infiltrative, aggressive disease and significantly worse clinical outcomes when not treated with uproleselan," said Helen Thackray, M.D., FAAP, GlycoMimetics Senior Vice President, Clinical Development and Chief Medical Officer. "While we would expect patients with R/R AML and >10% E-selectin ligand expression on their leukemic blasts to do very poorly, it is extremely exciting to see that the addition of uproleselan is resulting in statistically significant improvements in clinical outcomes in these high-risk patients. This is completely counterintuitive to what you would expect and provides robust scientific evidence suggesting that uproleselan is exerting biologic activity."

The ASH (Free ASH Whitepaper) presentations referenced above include:

Publication Number: 331
TITLE: Uproleselan (GMI-1271), an E-Selectin Antagonist, Improves the Efficacy and Safety of Chemotherapy in Relapsed/Refractory (R/R) and Newly Diagnosed Older Patients with Acute Myeloid Leukemia: Final, Correlative and Subgroup Analyses

Publication Number: 1513
TITLE: E-Selectin Ligand Expression by Leukemic Blasts Is Associated with Prognosis in Patients with AML

References
1 Feldman et al, J Clin Oncol. 2005 Jun 20;23(18):4110-6.
2 Greenberg et al, J Clin Oncol. 2004 Mar 15;22(6):1078-86.
3 Lowenberg et al, N Engl J Med. 2009 Sep 24;361(13).
4 Lancet et al, Blood. 2014 May 22;123(21):3239-46.

About Uproleselan (GMI-1271)

Uproleselan (yoo’ pro le’sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed/refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better than expected remission rates and overall survival compared to historical controls, which have been derived from results from third party clinical trials evaluating standard chemotherapy, as well as lower than expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects. The U.S. Food and Drug Administration (FDA) has granted uproleselan Breakthrough Therapy Designation for the treatment of adult AML patients with relapsed/refractory (R/R) disease. GlycoMimetics is currently implementing a comprehensive development program across the clinical spectrum of AML. This includes a company sponsored Phase 3 trial in R/R AML and two consortia-sponsored trials in newly diagnosed patients. One consortium trial is being sponsored by the NCI and will enroll newly diagnosed patients fit for intensive chemotherapy. The other trial is sponsored by the HOVON group in Europe and will enroll newly diagnosed patients unfit for intensive chemotherapy.

Aduro Biotech and Dana-Farber Cancer Institute Present Preclinical Data Supporting Anti-APRIL Antibody BION-1301 for the Treatment of Multiple Myeloma at the 60th American Society of Hematology Annual Meeting and Exposition

On December 3, 2018 Aduro Biotech, Inc. (NASDAQ: ADRO) reported preclinical data in two abstracts for its first-in-class anti-APRIL antibody BION-1301 supporting its potential use as a treatment for multiple myeloma (MM) at the 60TH American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, CA (Press release, Aduro Biotech, DEC 3, 2018, View Source;p=RssLanding&cat=news&id=2379030 [SID1234531808]). Data from the studies demonstrated that therapies blocking a proliferation inducing ligand (APRIL) from binding to B cell maturation antigen (BCMA) and transmembrane activator and cyclophilin ligand interactor (TACI) may simultaneously target MM cells and APRIL-induced immunosuppression.

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"Our newest findings from the data presented at ASH (Free ASH Whitepaper) indicate that combining an anti-APRIL antibody such as BION-1301, which inhibits APRIL binding to TACI and BCMA, with a BCMA-targeted therapy may have the potential to augment anti-myeloma activity," commented Dr. Kenneth C. Anderson, director of the Lebow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center at Dana-Farber Cancer Institute. "These studies support the rationale for use of the investigational agent, BION-1301, in the treatment of multiple myeloma."

Moreover, studies showed patient serum levels of APRIL were elevated at all stages of MM investigated, suggesting a role for APRIL in early development and pathogenesis. In addition, APRIL was found to induce production of key chemokines with osteolytic capacity. Blocking APRIL could modulate the tumor microenvironment more broadly, illustrating the potential of BION-1301 as a therapeutic agent for MM, particularly in combination therapies.

"We are encouraged by the data we presented at ASH (Free ASH Whitepaper), which support our ongoing clinical evaluation of BION-1301," said Andrea van Elsas, Ph.D., chief scientific officer of Aduro. "We believe BION-1301 could represent a first-in-class therapy for the treatment of multiple myeloma and that its differentiated approach to blocking APRIL may have further potential in combination strategies."

Aduro is currently conducting a Phase 1/2 multi-center, open-label study (see www.clinicaltrials.gov, identifier NCT03340883) designed to evaluate the safety and activity of BION-1301 in patients with relapsed or refractory MM whose disease has progressed after at least 3 prior systemic therapies, including immunomodulatory drugs, proteasome inhibitors, chemotherapies, or monoclonal antibodies.

The abstract, "APRIL Is Significantly Elevated at All Stages of Multiple Myeloma (MM) and Interferes with Anti-BCMA Monoclonal Antibody-Mediated Cytolysis, Supporting the Clinical Evaluation of BION-1301 As a Novel Therapeutic Approach in MM," was presented in a poster session titled "652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster II" and is available online at the ASH (Free ASH Whitepaper) Annual Meeting Website.

The abstract, "BION-1301 Blocks APRIL-Induced Anti-Apoptotic Signaling, Immune Suppressive Phenotype, and Chemokine Production Associated with Multiple Myeloma," was presented in a poster session titled "651. Myeloma: Biology and Pathophysiology, excluding Therapy: Poster I" and is available online at the ASH (Free ASH Whitepaper) Annual Meeting Website.

About APRIL
APRIL is a member of the tumor necrosis factor superfamily and is primarily secreted by bone marrow and/or myeloid cells. APRIL is overproduced in patients with MM and binds to BCMA and TACI to stimulate a wide variety of responses that promote MM growth and survival and suppress the immune system so that the tumor cells are protected and sustained in the bone marrow.

About BION-1301
BION-1301 is currently being evaluated in a Phase 1/2 clinical study in patients with relapsed or refractory MM. Aduro also plans to explore BION-1301 as a potential treatment for IgA nephropathy (IgAN). Despite new treatments recently approved in MM, this disease remains incurable as patients relapse, or become resistant to, currently-available therapies. In prior preclinical studies, Aduro established APRIL plays a crucial part in the protective bone marrow tumor microenvironment. In these studies, APRIL, through BCMA, was shown to be critically involved in the survival, proliferation and chemoresistance of MM, and upregulates mechanisms of immunoresistance, including PD-L1 upregulation. BION-1301, a humanized antibody that blocks APRIL from binding to its receptors, has been shown in preclinical studies to halt tumor growth and overcome drug resistance. In addition, BION-1301 also demonstrated the ability to inhibit immune suppressive effects of regulatory T cells via TACI but not BCMA in MM blood and bone marrow

BerGenBio reports 43% response rate with bemcentinib monotherapy in AXL positive R/R AML/MDS patients at ASH

On December 3, 2018 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company focused on developing a pipeline of first-in-class AXL kinase inhibitors to treat multiple cancer indications, reported that it presented a comprehensive analysis of monotherapy data from its BGBC003 clinical trial (NCT02488408) with selective AXL inhibitor bemcentinib in patients with relapsed/refractory Acute Myeloid Leukaemia (AML) or high-risk Myelodysplastic Syndrome (MDS) (Press release, BerGenBio, DEC 3, 2018, View Source [SID1234531807]).

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In a poster presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) in San Diego entitled: "Comprehensive Analysis of the Dose Escalation, Expansion and Correlates in the Ph I/II Trial BGBC003 with the Selective Oral AXL Inhibitor Bemcentinib (BGB324) in Relapsed/Refractory AML and MDS", Professor Sonja Loges, attending physician at the University Hospital in Hamburg-Eppendorf and lead investigator of the BGBC003 trial, detailed the following:

14 of 26 (54%) of patients found to be AXL positive (denoted by low serum AXL, sAXL, levels at start of treatment).
Response rate of 43% CR/Cri/CRp to bemcentinib monotherapy in AXL positive patients and 22% overall.
Mild and manageable side effect profile with a low incidence of Grade 3/4 events and low incidence of haematological toxicity.
Furthermore, the Company announces that enrolment is complete into the phase II combination cohort of bemcentinib and decitabine in first line AML. Analysis of the activity of the combination will be submitted for presentation at a future medical congress.

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "I am very encouraged by the data presented today, we are consistently seeing AXL positive patients report a superior response to bemcentinib therapy alone or in combination with standard of care therapy. The value of our biomarkers to identify patients most likely to benefit from bemcentinib is tremendous. Based on our clinical data, more than 50% of AML/MDS patients are AXL positive, of which 43% reported complete response within the first few weeks of starting bemcentinib monotherapy treatment. Bemcentinib is very well tolerated by patients as a single agent and in combination with other drugs, which is an important feature supporting bemcentinib’s broad utility. We are making excellent progress recruiting the chemotherapy combination arms of this study and look forward to reporting the data from these patients in the coming months".

END

About AML
AML is the most common form of acute leukaemia in adults where malignant AML blasts interfere with the normal functioning of the bone marrow leading to a multitude of complications like anaemia, infections and bleeding. AML is diagnosed in over 20,000 patients in the US annually and is rapidly lethal if left untreated. Successful treatment typically requires intensive therapy or bone marrow transplantation, and relapse and resistance are common. Consequently, there is an urgent need for effective novel therapies in relapsed/refractory patients, particularly those that are ineligible for intensive therapy or bone marrow transplant.

About the BGBC003 trial
The BGBC003 trial is a phase Ib/II multi-centre open label study of bemcentinib as a single agent in patients with AML or high risk MDS or in a combination with cytarabine and decitabine in AML patients. Up to 75 patients will be enrolled at centres in the US, Norway, Germany and Italy.

For more information please access trial NCT02488408 at www.clinicaltrials.gov.

About the 60th ASH (Free ASH Whitepaper) Annual Meeting in San Diego, California
The 60th American Society of Haematologist Annual Meeting and Exposition is the most comprehensive haematology conference worldwide and attracts more than 25,000 haematology professionals from every subspecialty.

Aprea Therapeutics Closes EUR 50 Million Financing

On December 2, 2018 Aprea Therapeutics, a clinical-stage biotechnology company developing novel anticancer therapies targeting the p53 tumor suppressor protein, reported the closing of a EUR 50 million Series C financing round led by the Redmile Group, with participation by new investor Rock Springs Capital and existing investors 5AM Ventures, Versant Ventures, HealthCap, Sectoral Asset Management and Karolinska Development AB (Nasdaq Stockholm: KDEV) (Press release, Aprea, DEC 3, 2018, View Source [SID1234531806]). A representative from Redmile Group will also be appointed as Director to the Aprea Therapeutics Board of Directors.

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"We are thrilled to close this financing, initiate a Phase III clinical study in myelodysplastic syndromes (MDS) with our lead compound, APR-246, and drive the company toward its next value inflection point," said Christian S. Schade, President and Chief Executive Officer of Aprea. "The addition of Redmile and Rock Springs to our investor group broadens the US investor base and we believe positions the company for future strategic opportunities. We are committed to bringing this important new therapy to cancer patients with limited therapeutic options."

Proceeds from the financing will be used to advance the clinical development of APR-246, a first-in-class anticancer agent that reactivates mutated p53 tumor suppressor protein. Aprea is planning to begin a Phase 3 clinical study in myelodysplastic syndromes (MDS) and is nearing completion of a Phase Ib/II clinical trial in p53 mutated high-risk myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML) with APR-246 and azacitidine. The Phase II part of the study is ongoing, with updated data to be presented at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis.

About p53 and APR-246

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 has been shown to reactivate mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – and thereby induce programmed cell death in human cancer cells. APR-246 has demonstrated pre-clinical anti-tumor activity in a wide variety of solid and hematological (blood) tumors, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase I/II clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

NantHealth® to Present New Research Findings at the San Antonio Breast Cancer Symposium

On December 3, 2018 Significant developments in breast cancer research reported that it will be presented by NantHealth (NASDAQ: NH) at the San Antonio Breast Cancer Symposium this week (Press release, NantHealth, DEC 3, 2018, View Source;p=RssLanding&cat=news&id=2378988 [SID1234531804]). NantHealth, a leader in breakthrough cancer research and solutions to improve patient care and lower healthcare costs, will discuss the findings of three investigations, which examine the theme of providing oncologists with insights that enable cancer treatment tailored to the individual characteristics of each patient.

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The Symposium, which will be held at the Henry B. Gonzalez Convention Center in San Antonio from Dec. 4-8, is designed to provide state-of-the-art information on the experimental biology, etiology, prevention, diagnosis and therapy of breast cancer and premalignant breast disease, to an international audience of academic and private physicians and researchers.

"About 1 in 8 U.S. women or about 12.4% will develop invasive breast cancer over the course of her lifetime. Such staggering statistics make breast cancer research and progress critical," said Sandeep "Bobby" Reddy MD, Chief Medical Officer, NantHealth. "We are honored to have an integral role at the San Antonio Breast Cancer Symposium, which provides a dynamic forum for interaction, communication, and education for a broad spectrum of researchers, health professionals, and those with a special interest in breast cancer."

Title: "Germline potentially pathogenic variants in breast cancer intrinsic molecular subtypes are not associated with somatic TMB"
Presenting Author: Elias Obeid, MD, MPH
Senior Author: Christopher Szeto, PhD
Contributors: Sandeep "Bobby" Reddy MD; Lori J. Goldstein, MD; Mary B. Daly, MD, PhD; Stephen C. Benz, PhD; and Michael J. Hall, MD, MS
Description: Comprehensive DNA and RNA profiling of 270 patients revealed intrinsic molecular subtypes of breast cancer have significantly distinct profiles of pathogenic germline variants. However, despite some breast cancer subtypes having higher frequency of germline pathogenic variants within DNA-damage repair genes, there is little evidence that these patients have subsequently higher mutational burden within their somatic genomes.
Key Takeaway: Pathogenic germline variants in key DNA damage repair genes such as BRCA1/2 are likely not adequate biomarkers for immune checkpoint therapy response, but are potentially biomarkers of differential tumorigenesis pathways.

Title: "Time-course DNA and RNA profiling of tumors from intra-patient cross-over trial of sequential use of aromatase inhibitors"
Presenting Author: Charles Vaske
Senior Authors: Vessela Kristensen and Jürgen Geisler
Contributors: Rahul Parulkar, Nazli Bahrami, Torill Sauer, Marie Loeng, Berit Gravdehaug, Belal Aljabri, Vahid Bemanian, Jonas Lindstrøm and Torben Lüders
Description: Early analysis from the ongoing NEOLETEXE trial examines a time-series of biopsies taken while transitioning patients from steroidal to non-steroidal aromatase inhibitors (AI) and vice versa. Acquired markers of AI-therapy resistance, and potential markers of sequential therapy sensitization, were explored. Two months after initial therapy, mutational burden decreased and clonality increased, yet by 4mo post-initialization mutations particularly in PIK3CA had repopulated.
Key Takeaway: Switching AI therapies sequentially in a clinical study is a model system to study differences in anti-tumor-effects of AIs. This ongoing trial may lead to a novel strategy to resensitize tumors to hormonal treatment and to elucidate the differences between steroidal and non-steroidal AIs.

Title: "Identification of a neoantigen targeted by tumor-infiltrating lymphocytes in a patient with Her2+ breast cancer"
Presenting Author: Hannah Kranich
Senior Authors: Peter A. Fasching and Anita N. Kremer
Contributors: Andrew Nguyen, Hanna Hübner, Erber Ramona, Judith Bausenwein, Edith D. van der Meijden, Michael P. Lux, Sebastian Jud, Claudia Rauh, John Zachary Sanborn, Stephen C. Benz, Shahrooz Rabizadeh, Matthias W. Beckmann, Andreas Mackensen and Matthias Rübner
Description: In our study, we identify tumor infiltrating T-cells (TILS) that recognize tumor specific mutations (Neoepitopes) found by next-generation sequencing of breast cancer tumors. These identified TILS are further immortalized and characterized to bind the patient’s specific HLA alleles.
Key Takeaway: Identification of TILS recognizing patient specific neoepitopes allow development of personalized medicine in a pre-clinical setting.

Since 1977, the San Antonio Breast Cancer Symposium mission has been to provide state-of-the-art information on breast cancer research. From a one-day regional conference, the Symposium has grown to a five-day program attended by a broad international audience of academic and private researchers and physicians from over 90 countries. For more information visit: View Source