On November 19, 2018 GlycoMimetics, Inc. (NASDAQ: GLYC) reported dosing of the first patient in the company’s Phase 3 clinical trial of uproleselan (GMI-1271) in relapsed/refractory AML (Press release, GlycoMimetics, NOV 19, 2018, View Source [SID1234531445]). The trial’s protocol provides for administration of the investigational drug in combination with MEC (mitoxantrone, etoposide and ara-C) or in combination with FAI (fludarabine, ara-C and idarubicin), both of which are standard of care for this indication in the United States. In 2017, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to GlycoMimetics for uproleselan in this patient population. The company anticipates the initiation of two complementary Phase 2/3 trials from two leading clinical consortia in early 2019.

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"The dosing of the first patient in our pivotal Phase 3 trial for uproleselan is an important milestone for GlycoMimetics," said Helen Thackray, M.D., FAAP, Senior Vice President, Clinical Development, and Chief Medical Officer of GlycoMimetics. "This is a rigorously designed Phase 3 trial that has the potential to bring us one step closer to meeting the significant unmet needs of individuals living with relapsed/refractory AML. The trial is an important component of our comprehensive late-stage development program for uproleselan that positions us to evaluate the use of our product candidate across the spectrum of AML. It is the first of three randomized, controlled trials for uproleselan in AML, which we believe should provide clear efficacy and safety outcome measures in each of the settings being assessed."

"Our clinical development pipeline sets us up for multiple, value-creating clinical data readouts," added Rachel K. King, GlycoMimetics Chief Executive Officer. "During 2019, we anticipate topline data from the Phase 3 study of rivipansel being conducted by Pfizer in patients with sickle cell disease. Then, beginning at the end of 2020, we expect to generate topline data from the several trials that we will have underway in AML."

GlycoMimetics plans to enroll approximately 380 adult patients worldwide in the single pivotal randomized, double-blind, placebo-controlled Phase 3 AML trial, which is designed to align with guidance received from regulatory agencies. The primary endpoint is overall survival, and censoring for transplant in the primary efficacy analysis will not be required. Key secondary endpoints include incidence of severe mucositis and remission rate, which will be assessed in a hierarchical fashion for potential inclusion in the product labeling. The majority of the study sites will be in the United States, with meaningful participation from leading clinical centers in other strategic countries to support regulatory filings in major markets.

More information on the clinical trial can be found at www.clinicaltrials.gov.

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, as well as lower than expected induction-related mortality rates, as compared to historical controls which have been derived from results from third party clinical trials evaluating standard chemotherapy 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.

On November 19, 2018 Guardant Health, Inc. (Nasdaq:GH), a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary blood tests, vast data sets and advanced analytics, reported financial results for the third quarter ended September 30, 2018 (Press release, Guardant Health, NOV 19, 2018, View Source [SID1234531532]).

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Recent Highlights

Revenues of $21.7 million for the third quarter of 2018, representing a 95% increase over the third quarter of 2017

Reported 7,027 tests to clinical customers and 2,505 tests to biopharmaceutical customers in the third quarter, representing increases of 14% and 67% respectively, over the third quarter 2017

Lung cancer study published in JAMA Oncology demonstrated Guardant360 outperformed tissue biopsy alone in identification of targetable mutations

Awarded Medicare coverage for the Guardant360 assay in non-small cell lung cancer through a local coverage determination from Palmetto GBA, a Medicare Administrative Contractor; successfully received first payment

Completed initial public offering, raising approximately $249.5 million of net proceeds, after underwriting fees and other expenses
"Our third quarter accomplishments reflect the ongoing strength of our business," said Helmy Eltoukhy, PhD, Chief Executive Officer. "We are continuing to build proof points for a blood-first paradigm ahead of traditional tissue genotyping, as demonstrated by the study from the University of Pennsylvania which was recently published in JAMA Oncology."
"In addition, we are pleased with the successful completion of our IPO," continued Dr. Eltoukhy. "We are grateful for the support of our investors who participated in the offering, and we are focused on the creation of long-term shareholder value through unprecedented access to cancer’s molecular information throughout all stages of the disease."
Third Quarter 2018 Financial Results
Revenue was $21.7 million in the three months ended September 30, 2018, a 95% increase from $11.1 million in the three months ended September 30, 2017. Precision oncology revenue increased 78% driven by higher testing volume and increases in revenue per test. Tests for clinical customers increased 14% (after excluding tests in the third quarter of 2017 from a customer that began processing tests in-house in March 2018 based on a joint development agreement) and tests for biopharmaceutical customers increased 67%.
Gross profit, or total revenue less cost of precision oncology testing and cost of development services, was $11.6 million for the third quarter of 2018, an increase of $9.1 million from $2.5 million in the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 53.7%, as compared to 22.2% in the corresponding prior year period.
Operating expenses were $35.8 million for the third quarter of 2018, as compared to $31.1 million in the corresponding prior year period, an increase of 15%.
Net loss attributable to Guardant Health, Inc. common stockholders was $24.5 million in the third quarter of 2018, as compared to $33.3 million in the corresponding period of the prior year. Net loss per share attributable to Guardant Health, Inc. common stockholders was $1.94 in the third quarter of 2018, as compared to $2.76 in the corresponding period of the prior year.
Cash, cash equivalents and marketable securities were $274.3 million as of September 30, 2018. In October 2018, Guardant completed an initial public offering, raising approximately $249.5 million net of underwriting fees and other expenses.
2018 Financial Guidance
Guardant Health expects full year 2018 revenue to be in the range of $82.0 million to $84.0 million.
Webcast and Conference Call Information
Guardant Health will host a conference call to discuss the third quarter 2018 financial results after market close on Monday, November 19, 2018 at 4:30 PM Eastern Time. The conference call can be accessed live over the phone (866) 417-5537 for U.S. callers or (409) 217-8233 for international callers (Conference ID: 9156645). The webcast can be accessed at View Source

On November 16, 2018 ArQule, Inc. (Nasdaq: ARQL) reported the presentation of clinical and preclinical data on ARQ 751 in three poster presentations at the 30th EORTC/AACR/NCI Symposium held from November 13 to 16, 2018 in Dublin, Ireland (Press release, ArQule, NOV 16, 2018, View Source [SID1234531401]). The data presented highlight clinical data from ARQ 751-101, a Phase 1 study in adult patients with refractory and/or metastatic tumors that harbor AKT, PI3K or PTEN genetic alterations, and preclinical data on ARQ 751 in combination with other agents.

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Clinical data highlights and key conclusions include:

1. A Phase 1 Dose Escalation Study of ARQ 751 in Adult Patients with Advanced Solid Tumors with AKT1, 2, 3 Genetic Alterations, Activating PI3K Mutations, PTEN-null, or Other Known Actionable PTEN Mutations

ARQ 751 demonstrated manageable toxicity at doses from 5 mg QD to 75 mg QD, and the recommended Phase 2 dose was determined to be 75 mg QD
Evidence of clinical activity was observed with two partial responses in ER+/PR+/HER2- stage IV breast cancer patients, one with PTEN C296fs*2 mutation, one with PIK3CA H1047R mutation, 11 patients had stable disease
The data support continued development of ARQ 751 as a monotherapy or in combination with other anti-cancer agents due to its manageable safety profile and preliminary evidence of biological activity
"ARQ 751, as a highly specific allosteric AKT inhibitor, holds great potential in treating patients with solid tumors harboring mutations in the AKT/PI3K/PTEN pathways," said Brian Schwartz, M.D., Chief Medical Officer of ArQule. "The presented data are very encouraging and demonstrate both preliminary signs of clinical activity and a favorable safety profile while also determining the recommended Phase 2 dose. At ArQule, we are committed to developing genetically targeted cancer treatments to provide effective new treatment options for patients, particularly those with advanced or relapsed disease, and look forward to advancing the ARQ 751 clinical program."

Preclinical data highlights include:

2. Combination of the AKT inhibitor ARQ 751 with Immune Checkpoint Inhibitor and Other Therapeutic Agents

In preclinical cellular models, ARQ 751 exerted greater anti-proliferative and biochemical effects when in combination with multiple therapeutic agents including an ER antagonist, aromatase inhibitor, androgen receptor antagonist and a BTK inhibitor
In an in vivo colon cancer animal model, ARQ 751 in combination with an anti-PD-1 antibody exhibited superior anti-tumor activity compared to single agents
3. Miransertib and ARQ 751 exhibit superior cell-death-inducing properties compared to other AKT inhibitors and can overcome resistance to other allosteric AKT inhibitors

ArQule’s AKT inhibitors, miransertib and ARQ 751 showed superior activity in comparison to other allosteric and ATP-competitive AKT inhibitors currently in clinical development
Miransertib and ARQ 751 have the potential to overcome some mechanisms of resistance to AKT inhibitors
Miransertib and ARQ 751 in combination with ATM inhibition demonstrated synergistic effects
Dr. Shubham Pant, MD, Associate Professor in the Department of Investigational Cancer Therapeutics at MD Anderson Cancer Center, said "AKT inhibitors have significant potential to treat a broad range of solid tumors in molecularly defined patient populations. The presented data show that ARQ 751 exhibits unique properties that differentiate it from other AKT inhibitors. It is our hope that by combining ARQ 751 with a broad spectrum of therapeutic agents, including hormonal agents, we could provide new opportunities for combinatorial interventions in oncology."

All posters presented by ArQule at the EORTC/AACR/NCI Symposium are available on the company’s website at View Source

About ARQ 751
ARQ 751 is an orally bioavailable, selective small molecule inhibitor of the AKT serine/threonine kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications. ARQ 751 is currently in a Phase 1 study in adult patients with refractory and/or metastatic tumors that harbor genetic alterations along the AKT pathway.

About Miransertib
Miransertib (ARQ 092) is an orally bioavailable, selective, pan-AKT (protein kinase B) inhibitor that potently inhibits AKT1, 2 and 3 isoforms. Dysregulation of AKT has been implicated in a variety of rare overgrowth diseases and cancers; however, there are currently no approved inhibitors of AKT. AKT inhibitors, either as single agent or combination therapy, show significant promise in molecularly defined patient populations. Miransertib is currently in a Phase 1/2 company-sponsored study for PIK3CA-Related Overgrowth Spectrum (PROS), a Phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health (NIH/NHGRI), and a Phase 1b study in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer with AKT and PI3K mutations. Miransertib has been granted Rare Pediatric Disease Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA), as well as Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome.

On November 16, 2018 apceth Biopharma GmbH, an established contract manufacturing organization in the field of gene and cell therapy, reported it has started supplying clinical batches of DCprime’s cell-based cancer vaccine DCP-001 to a phase II clinical study for the treatment of Acute Myeloid Leukemia (AML) (Press release, apceth, NOV 16, 2018, View Source [SID1234531454]). AML is a haematological cancer characterized by high risk of relapse, even after initial response to chemotherapy. Cancer vaccination with dendritic cells could be a successful strategy to boost the patient’s immune system and result in lasting disease control. DCprime announced yesterday that the first patient has been treated with the product DCP-001 at the Amsterdam University Medical Center (UMC).

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"We are proud to be the manufacturing partner of DCprime for DCP-001, the first off-the shelf dendritic cell vaccine in a cancer indication with high unmet medical need", explained Dr Christine Günther, CEO of apceth Biopharma. "Our long-term collaboration with DCprime has always been constructive and cooperative, from technology transfer to the large-scale manufacturing process."

Dr Erik Manting, CEO of DCprime, commented: "Based on the recently published encouraging results of the phase I trial with our lead product DCP-001, we aim to confirm these results in the phase II ADVANCE-II trial and we are happy with the enrollment of the first patient. We would like to thank the apceth Biopharma team for their important contribution to the manufacturing of our product."

On November 16, 2018 AIVITA Biomedical reported that it has dosed the first two patients in its Phase 2 clinical trial in patients with newly diagnosed glioblastoma (Press release, AIVITA Biomedical, NOV 16, 2018, View Source [SID1234531407]). The trial is designed to investigate AIVITA Biomedical’s next-generation patient-specific cancer treatment, uniquely targeting the patient’s tumor-initiating cells.

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The first two patients were treated at the University of California Irvine (UCI) Comprehensive Brain Tumor Program under the direction of UCI Health neuro-oncologist and Principal Investigator Daniela Bota, MD, PhD.

AIVITA will enroll approximately 55 patients in its ROOT OF CANCER Glioblastoma trial to receive the Company’s patient-specific cancer treatment, which is administered in a series of subcutaneous injections alongside standard care. AIVITA is also expanding access to the trial, having recently adding the University of California San Diego and John Wayne Cancer Institute as clinical sites.

"I am very proud of the AIVITA team for so effectively running three clinical programs in two countries," said Dr. Bob Dillman, Chief Medical Officer at AIVITA. "This is enabled by a quick, reliable and inexpensive manufacturing process and a treatment protocol that truly complements the regime of cancer care."

AIVITA’s ROOT OF CANCER technology is also the subject of an ongoing multi-center Phase 2 clinical trial treating ovarian cancer in the USA, and an application to commercialize the treatment of melanoma patients in Japan. Previously, this treatment was tested in two Phase 2 trials in patients with advanced melanoma and approved for Phase 3 testing. These clinical studies demonstrated a 72% 2-year survival rate and a 54% 5-year survival rate, supporting AIVITA’s conditional commercial approval application in Japan. The Company is considering Japanese strategic partners for this program.

About the ROOT OF CANCER Glioblastoma trial

AIVITA’s treatment is a platform technology applicable to any solid tumor type and consists of autologous dendritic cells loaded with autologous tumor antigens from autologous self-renewing tumor-initiating cells.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established by AIVITA, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC).

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917