On April 10, 2021 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that an abstract presenting the results of a recent preclinical study on the therapeutic effects of GMI-1757, a new glycomimetic with dual antagonism to E-selectin and galectin-3, has been accepted for a poster presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Annual Meeting, to be held virtually on April 10-15 and May 17-21 (Press release, GlycoMimetics, APR 10, 2021, View Source [SID1234577817]).

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The study demonstrates that GMI-1757 significantly improved anti-PD-L1 therapeutic activity in a pancreatic adenocarcinoma model. Results showed 50% partial regressions and an approximate 99% reduction of median tumor volume. Microscopic evaluations also showed that in groups treated with GMI-1757 both the incidence and area of intratumoral fibrosis were markedly reduced. Additionally, GMI-1757 strongly increased the incidence of mononuclear cell tumor infiltration. These results suggest that the decreased intratumoral fibrotic development and increased mononuclear cell infiltration obtained with GMI-1757 created a favorable immune environment so that when combined with anti-PD-L1 it produced a more robust anti-tumor effect compared to anti-PD-L1 treatment alone. Investigations will continue on GMI-1757’s impact when combined with immune modulators where fibrosis and restricted host cell infiltration negatively impact tumor response.

GlycoMimetics Senior Vice President, Research and Chief Scientific Officer John Magnani commented, "We look forward to presenting the latest research on our novel glycomimetic compound at AACR (Free AACR Whitepaper)’s annual meeting. Our hope is that as this research progresses, we will move ever closer to achieving promising therapies for individuals living with forms of cancer where medical needs remain unmet."

Details on GlycoMimetics e-presentation at the AACR (Free AACR Whitepaper) Meeting are as follows:

Title: A novel glycomimetic compound (GMI-1757) with dual functional antagonism to E-selectin and galectin-3 attenuates fibrosis, facilitates mononuclear cell infiltration and optimizes anti-PD-L1 therapeutic activity in a pancreatic adenocarcinoma model
Presenter: William E. Fogler, Ph.D., GlycoMimetics
Session: e-Presentation
Date and Time: Saturday, April 10, 2021 (available online through Monday, June 21)

About GMI-1757

An innovative dual antagonist of E-selectin and galectin-3, GMI-1757 has shown anti-thrombotic and anti-fibrotic activity in preclinical models presented at major scientific meetings. Data suggest the compound may be able to play a role in the treatment of a variety of cancers and fibrotic conditions.

On April 10, 2021 OncXerna Therapeutics, Inc., a precision medicine company using an innovative RNA-expression based biomarker platform to predict patient responses to its first-in-class targeted oncology therapies, reported new results from its Xerna TME Panel during Week 1 of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, OncXerna Therapeutics, APR 10, 2021, View Source [SID1234577834]). In this study, OncXerna demonstrated that the first panel (TME Panel) from its Xerna platform revealed prognostic subtypes in colorectal cancer (CRC) by analyzing tumor samples from over 600 CRC patients. The XernaTM TME Panel uses proprietary RNA-based gene expression data and a machine learning-based algorithm to classify patients based on their dominant biologies of the tumor microenvironment (TME), and has been developed as a clinical assay.

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"Our goal is to expand the applicability of precision medicine in cancer through a novel approach that matches patients to the appropriate therapies by using RNA expression to identify patients with common biological drivers," said Laura Benjamin, Ph.D., President and Chief Executive Officer at OncXerna Therapeutics. "In our AACR (Free AACR Whitepaper) presentation, we presented results that verify the capabilities of our TME Panel and enable its expanded use for patients with colorectal cancer. These exciting findings pave the way for future prospectively-driven trials with the Xerna TME Panel and our clinical-stage programs, navicixizumab and bavituximab."

The results from the study presented at this year’s AACR (Free AACR Whitepaper) Annual Meeting revealed:

An expansion of the TME Panel’s capabilities to include CRC, as the RNA-based gene signature identified unique subtypes of patients with angiogenic and immune biologies that dominated the stroma. These biologies were prognostic for recurrence-free and overall survival, supporting the potential use of the TME Panel as a novel, pan-tumor biomarker.
A comparison of the TME Panel’s subtypes to the Consensus Molecular Subtypes (CMS) model. The CMS model represents gene expression data from both colorectal cancer cells and their microenvironment. In contrast, the Xerna TME Panel integrates the interplay of angiogenic and immunogenic properties of the tumor microenviroment, and because of this focus, could be more predictive for treatments that target angiogenic and immunogenic properties of the TME.
The following two datasets were used to explore the application of the TME Panel in colorectal cancer: 1) A public dataset from the Cartes d’Identite des Tumeurs (CIT) containing 566 primary tumor samples collected from patients with stage 1-4 CRC between 1988 and 2007 in France, and 2) A proprietary collection of 92 samples from the Wood Hudson Cancer Research Laboratory (WH) of patients with metastatic CRC who were treated with various regimens including targeted therapies following surgery.

OncXerna’s electronic poster, #348 entitled: "RNA-based Diagnostic Panel Matches TME Phenotype to Therapeutic Mechanism of Action in Colorectal Cancer," is included in the "Biomarkers Predictive of Therapeutic Benefit" poster session at the AACR (Free AACR Whitepaper) annual meeting. A link to the poster can be found here.

On April 10, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC) reported data from multiple poster presentations highlighting the breadth of the company’s precision therapy pipeline at the virtual American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (Press release, Blueprint Medicines, APR 10, 2021, View Source [SID1234577850]). Collectively, the presentations, including foundational preclinical data for multiple programs, demonstrate the productivity of the company’s scientific platform. Additional presentations of clinical data for AYVAKIT (avapritinib) and BLU-263 will be reported on Sunday, April 11, 2021.

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"Our data presentations at the AACR (Free AACR Whitepaper) annual meeting showcase Blueprint Medicines’ next wave of precision therapies, which have the potential to impact large global patient populations," said Fouad Namouni, M.D., President, Research & Development at Blueprint Medicines. "Building on the foundation of our approved medicines AYVAKIT and GAVRETO, these presentations highlight our efforts to address significant patient needs in areas such as EGFR-driven lung cancer, cyclin E-aberrant cancers and cancer immunotherapy. As we celebrate our 10th anniversary as a company, and our rapidly expanding pipeline now includes our ninth development candidate, we look forward to continuing to leverage our productive research platform to advance potent and selective inhibitors that have the potential to enable transformative benefit for patients."

BLU-701 and BLU-945: Double- and triple-mutant inhibitors in EGFR-driven NSCLC

Lung cancer is the leading cause of cancer death globally, and approximately 17 percent of patients with lung adenocarcinoma, the most common form of non-small cell lung cancer (NSCLC), have EGFR-driven disease. While first- and third-generation EGFR inhibitors have improved treatment outcomes for patients with EGFR-driven NSCLC, resistance inevitably emerges, with the T790M and C797S mutations being the most common on-target resistance mechanisms. Together, BLU-701 and BLU-945 are designed to provide comprehensive coverage of the most common activating and on-target resistance mutations, spare wild-type EGFR to limit toxicities driven by wild-type EGFR inhibition and treat or prevent central nervous system (CNS) metastases. Ultimately, with these characteristics, BLU-701 and BLU-945 have the potential to be used either as monotherapy or in combination, together or with other agents, to potentially overcome or prevent on-target resistance across multiple lines of treatment.

BLU-701 is a potential best-in-class, selective, potent, fourth-generation double-mutant EGFR inhibitor with activity against EGFR activating mutations and the C797S osimertinib-resistant mutation. Preclinical data presented at the conference showed strong and durable inhibition of tumor growth at doses that are EGFR wild-type sparing. BLU-701 also indicated significant CNS penetration in preclinical models, with comparable exposure in the plasma and brain, which illustrates its potential to treat or prevent CNS metastases in patients with EGFR-driven tumors. With activity also shown against the activating EGFR mutants, BLU-701 has potential to be used in both first- and second-line settings.

BLU-945 is a potential first- and best-in-class, selective, potent, fourth-generation triple-mutant EGFR inhibitor with activity against the T790M and C797S resistance mutations. BLU-945 is highly selective over wild-type EGFR and off-target kinases, highlighting its potential to enable tolerable combinations with BLU-701 or other therapies. Data presented at the conference demonstrated potent anti-tumor activity in triple-mutant osimertinib-resistant tumor models, as well as activity in a triple-mutant intracranial patient-derived xenograft model. In addition, the combination of BLU-945 with either gefitinib or osimertinib showed enhanced anti-tumor activity when compared with either gefitinib or osimertinib alone.

The preclinical data presented for BLU-701 and BLU-945 support the continued development of both candidates in patients with EGFR-driven NSCLC. An investigational new drug (IND) application for BLU-945 has been cleared by the U.S. Food and Drug Administration (FDA) and an international Phase 1 dose escalation trial is expected to begin this quarter. Future clinical development of BLU-945 in combination with other agents across multiple treatment settings is planned. BLU-701 is expected to enter clinical development later this year.

BLU-222: CDK2 inhibitor in cyclin E-aberrant cancers

Cyclin dependent kinases (CDKs) and their cyclin partners regulate the cell cycle. In subsets of patients across multiple cancer types, aberrant cyclin E (CCNE) hyperactivates CDK2, resulting in cell cycle dysregulation and tumor proliferation. Aberrant CCNE has been observed as a primary driver of disease as well as a mechanism of resistance to CDK4/6 inhibitors and other therapies. In addition, data have shown that ovarian and hormone-receptor-positive breast cancer patients with aberrant CCNE have poor outcomes. Prior drug discovery efforts targeting CDK2 have been hindered by challenges in achieving selectivity over other CDK family members associated with toxicity.

At AACR (Free AACR Whitepaper), preclinical data highlighted a set of potent and selective CDK2 inhibitors designed by Blueprint Medicines. The data showed that selective CDK2 inhibition arrested the cell cycle and blocked tumor proliferation in CCNE-amplified cell lines and demonstrated robust and sustained anti-tumor activity in vivo in models of CCNE-amplified ovarian, breast and gastric cancer. A selective CDK2 inhibitor also showed improved tolerability compared to a pan-CDK inhibitor and chemotherapy, as measured by animal body weight.

Based on this work and further optimization, Blueprint Medicines reported the nomination of a potentially best-in-class selective and potent CDK2 inhibitor development candidate, BLU-222, which is expected to enter clinical development in the first half of 2022.

BLU-852: MAP4K1 inhibitor

MAP4K1 is a well-characterized immunokinase target involved in the regulation of immune cells; however, prior drug discovery efforts have been hindered by challenges in achieving selectivity over other MAP4K family members associated with toxicity. In January 2021, Blueprint Medicines announced the nomination of a highly selective and potent MAP4K1 inhibitor development candidate, BLU-852, with best-in-class potential.

Data presented at AACR (Free AACR Whitepaper) highlighted a set of potent and highly selective MAP4K1 inhibitors designed by Blueprint Medicines, including BLU-852. The inhibitors were shown to enhance intratumoral immune cell activation, overcome T cell suppression, and reduce tumor burden both as a monotherapy and in combination with checkpoint inhibition. The data support the continued development of BLU-852 under the company’s cancer immunotherapy collaboration with Roche, with Phase 1 trial initiation anticipated in 2022.

Copies of Blueprint Medicines data presentations from the AACR (Free AACR Whitepaper) annual meeting are available in the "Science—Publications and Presentations" section of the company’s website at www.BlueprintMedicines.com.

Conference Call Information

Blueprint Medicines will host a live webcast on Monday, April 12, 2021 beginning at 8:00 a.m. ET to review data for multiple research- and clinical-stage programs presented at the AACR (Free AACR Whitepaper) annual meeting. To access the live call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international) and refer to conference ID 5548976. A webcast of the conference call will be available under "Events and Presentations" in the Investors & Media section of Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On April 10, 2021 Arcus Biosciences, Inc. (NYSE:RCUS), an oncology-focused biopharmaceutical company working to create best-in-class cancer therapies, today presented progression-free survival (PFS) and overall survival (OS) data in patients with advanced metastatic colorectal cancer (mCRC) from the ARC-3 study at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Arcus Biosciences, APR 10, 2021, View Source [SID1234577866]). ARC-3 was a Phase 1/1b, multicenter, open-label, dose-escalation and dose-expansion study that evaluated the safety, tolerability, PK and early clinical activity of etrumadenant, the first dual adenosine A2a/A2b receptor antagonist in the clinic, in subjects with mCRC.

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Additionally, Arcus presented details of its Hypoxia-Inducible Factor 2α (HIF-2α) research program, including the design of a novel series of HIF-2α inhibitors, which has resulted in the identification of molecules such as AB521, with excellent potency, selectivity, biological activity and pharmacokinetic properties suitable for further development.

ARC-3: Updated Results of Etrumadenant (AB928) + mFOLFOX-6 in Patients with Metastatic Colorectal Cancer

Initial results from ARC-3 demonstrated that etrumadenant + modified FOLFOX-6 (mFOLFOX-6) in patients with mCRC was well tolerated and associated with a substantial disease control rate (DCR) across all lines of therapy, including in patients with microsatellite stable disease and RAS/BRAF-mutated mCRC1,2.

Updated data from the 3L+ cohort (median of 3 and a range of 2 to 7 prior lines), in which 87% of the patients had received prior FOLFOX, demonstrated the following:

Safety Results (n=23 safety-evaluable 3L+ patients as of the DCO of Feb. 26, 2021)
Etrumadenant + mFOLFOX-6 was well tolerated, and etrumadenant, at the evaluated doses of 75mg and 150mg once daily (QD), did not appear to add significant toxicity to that expected for mFOLFOX-6.
No grade 3 or above neuropathy events were observed in this heavily pretreated population.
The most common treatment emergent adverse events (TEAEs) for the etrumadement- mFOLFOX-3 combination were fatigue (70%), thrombocytopenia (57%), diarrhea (52%) and nausea (52%).
Efficacy Results (n=22 efficacy-evaluable 3L+ patients as of the DCO of Feb. 26, 2021)
Median progression-free survival (PFS) of 4.2 months. Reported data for current standard-of-care (SOC) therapies have shown a median PFS of 2.0 and 1.9 months for trifluridine-tipiracil and regorafenib, respectively3,4.
Median overall survival (OS) of 13.6 months. Reported data for trifluridine-tipiracil and regorafenib have shown a median OS of 7.1 and 6.4 months, respectively3,4.
Objective response rate (ORR) of 9.1% and an encouraging 8-week DCR of 86%. Reported data for trifluridine-tipiracil and regorafenib have shown ORRs of 1.6% and 1%, respectively 3,4.
Patients with higher tumor mutation burden and intra-tumoral expression of CD73 demonstrated improved outcomes compared to patients with lower levels of these biomarkers, consistent with previous findings1, which may be reflective of an etrumadenant-mediated effect.
"Based on these very encouraging early results, we have advanced etrumadenant into ARC-9, a randomized Phase 2 platform study to evaluate this first-in-class molecule in combination with zimberelimab, our anti-PD-1 antibody, and FOLFOX +/- bevacizumab in second- and third-line mCRC," said Bill Grossman, M.D., Ph.D., Chief Medical Officer of Arcus. "The results presented today, combined with our recent promising early data evaluating AB680, our small-molecule CD73 inhibitor, in pancreatic cancer, support an expanding rationale for targeting the ATP-adenosine axis to meet critical unmet needs in gastrointestinal cancers."

"Few options exist today to treat third-line colorectal cancer, and these therapies are associated with significant toxicity and limited efficacy. While this was an early-stage study, etrumadenant’s efficacy in this difficult-to-treat patient population was impressive, particularly the doubling of progression-free and overall survival that was observed relative to what has been reported for current standard-of-care therapies," said Michael Cecchini, MD, Assistant Professor of Medicine (Medical Oncology), Yale Cancer Center and Smilow Cancer Hospital. "Importantly, etrumadenant also added very little toxicity, enabling patients to remain on treatment for extended periods of time. I look forward to working with Arcus to advance etrumadenant, and Arcus’s other ATP-adenosine axis-targeting agents, into later-stage studies for gastrointestinal cancers in order to broaden access to these innovative potential therapies."

Preliminary data from ARC-9, a global follow-on study to ARC-3, are expected to be presented in the first half of 2022. For additional information on this trial, please visit NCT04660812, at www.clinicaltrials.gov.

Discovery and Characterization of AB521, a Novel, Potent, and Selective HIF-2 α Inhibitor

Preclinical and clinical evidence indicate that HIF-2α inhibition is a validated approach for the treatment of clear cell renal cell carcinoma (ccRCC) and tumors associated with mutant/deficient Von Hippel-Lindau (VHL). Arcus has developed a broad research program to identify drug candidates against this target. Despite the inherent difficulties in identifying drug-like inhibitors of HIF-2α, Arcus scientists have generated highly optimized inhibitors, including AB521, which exhibit high potency, selectivity and biological activity, as well as a favorable pharmacokinetic profile in preclinical species, that we expect will facilitate achieving optimal plasma levels of drug in the clinic.

We expect to initiate clinical development for our lead HIF-2α inhibitor in the second half of 2021 and plan to combine this molecule with other product candidates from our pipeline, including our first-in-class adenosine axis-targeting agents.

Additional information about these presentations may be found on the Arcus website at Arcus Publications.

About Etrumadenant

Etrumadenant (AB928), the first dual A2a/A2b adenosine receptor antagonist in the clinic, is designed to maximally inhibit the adenosine-driven impairment of tumor-infiltrating lymphocytes (mainly CD8+ T cells and NK cells) and myeloid cells (dendritic cells, macrophages), mediated by A2aR and A2bR, respectively. A2bR is also upregulated by certain cancer cells, such as in prostate cancer and KRAS- mutated cancers. As a result, etrumadenant may uniquely block adenosine’s immunosuppressive and cancer cell-intrinsic effects. Developed specifically for the oncology setting, etrumadenant achieves high penetration of tumor tissue, robust potency in the presence of high adenosine concentrations, and minimal shift in potency from non-specific protein binding. Etrumadenant has demonstrated a favorable safety profile with a variety of combination regimens and exhibits pharmacokinetics / pharmacodynamics consistent with once-daily dosing. AB928 is currently being evaluated in several Phase 1b/2 studies across multiple indications.

On April 10, 2021 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that a Phase 1b trial of GMI-1359, being conducted at Duke University Cancer Center, showed evidence of on-target effects, immune-activation and cell mobilization in the initial two patients treated with the Company’s dual antagonist of E-selectin and CXCR4 (Press release, GlycoMimetics, APR 10, 2021, View Source [SID1234577818]). Dorothy Sipkins, MD, PhD, Associate Research Professor in Pharmacology and Cancer Biology at Duke University School of Medicine, will present results from the proof-of-concept clinical study as well as a separate preclinical study supporting the positive biologic findings of the Phase 1b study. The presentation will be made at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Annual Meeting, which is being held virtually on April 10-15 and May 17-21. GMI-1359 is GlycoMimetics’ novel small molecule drug candidate, a dual antagonist of E-selectin and CXCR4, designed to target tumor-microenvironment resistance to chemotherapy in cancers with bone metastases.

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The initial data from the study confirmed the dual CXCR4 and E-selectin antagonist’s on-target effects. In the two patients who completed treatment, evaluations of peripheral blood showed a consistent mobilization of CD34+ hematopoietic stem and progenitor cells at doses beginning at 5 mg/kg and a reduction of plasma levels of soluble E-selectin. Furthermore, in one individual, following the administration of 7.0 mg/kg of GMI-1359, an immunophenotyping assessment of peripheral blood showed a redistribution of myeloid derived suppressor cells (MDSCs) as evidenced by increased percentages of both the monocytic and granulocytic MDSCs. In this same individual following administration of 7.0 mg/kg GMI-1359, the incidence of M1 proinflammatory macrophages increased while the M2 anti-inflammatory macrophages, often associated with tumor progression, decreased. The clinical poster concludes that GMI-1359 demonstrated an acceptable safety and tolerability profile in the patients treated to date. No dose limiting toxicities were observed following multiple dose administration up to 7 mg/kg.

Dr. Sipkins noted, "Despite the fact that our patient numbers are very small due to COVID’s impact on recruitment, we are seeing the on-target effects of antagonizing both CXCR4 and E-selectin with use of GMI-1359, and that the drug is well-tolerated at all dose levels. Our pilot immune profile analysis also suggests that the drug could have favorable effects on the tumor immune microenvironment, echoing results seen in our preclinical work."

Dr. Sipkins will disclose preclinical evidence that it may be possible for GMI-1359 to augment immune recognition of the tumor. The data in the poster from a mouse metastatic breast carcinoma model demonstrated a reduction in the immune suppressive monocytic MDSCs at the primary tumor site and a significant increase in the CD8/Treg ratio in both the primary tumor and at the bone metastatic sites. These findings on immune cell redistributions strongly suggest the induction of a more favorable anti-tumor environment following GMI-1359 administration.

According to Dr. Eric J. Feldman, GlycoMimetics Senior Vice President and Chief Medical Officer, "The information shared in this AACR (Free AACR Whitepaper) poster provides us with important understandings upon which we expect to identify a potential indication for advancing GMI-1359 in the clinic. It suggests that this small molecule drug candidate could improve responses to therapies and potentially reduce the burden of metastatic breast cancer disease."

In prior preclinical research supported by GlycoMimetics, Dr. Sipkins’ laboratory demonstrated that E-selectin and CXCR4/SDF-1 interactions were critical for breast carcinoma cells (BCCs) invasion and retention, respectively, into bone. Moreover, they found that dormant and proliferating BCCs occupy distinct regions of the bone microenvironment, with dormant BCCs predominantly found in SDF-1 and E-selectin rich regions. These dormant BCCs are expected to be highly susceptible to GMI-1359 mobilization, suggesting a new intervention to break the foothold of dormant BC micrometastases in bone.

Details on the GMI-1359 e-presentation at the AACR (Free AACR Whitepaper) Meeting are as follows:

Title: Development of GMI-1359, a novel agent targeting tumor-microenvironment cross-talk in bone metastatic cancer
Presenter: Dorothy Sipkins, MD, PhD, Associate Research Professor in Pharmacology and Cancer Biology at Duke University School of Medicine
Session: e-Presentation
Date and Time: Saturday, April 10, 2021 (available online through Monday, June 21)

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4, which are adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow, such as AML and multiple myeloma, or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor affecting about 900 adolescents a year in the United States. GMI-1359 completed a Phase 1 clinical trial in healthy volunteers, and a Phase 1b clinical study is underway in breast cancer patients and is designed to enable investigators to identify study dose ranging and to generate initial biomarker data around the drug’s activity. In the first two patients evaluated, the study showed evidence of on-target effects, immune-activation and cell mobilization. GMI-1359 has received Orphan Drug designation and Rare Pediatric Disease designation from the FDA for the treatment of osteosarcoma.