Medicare Coverage of Prolaris® Test Expands with the Addition of Men Diagnosed with Favorable Intermediate Risk Prostate Cancer

On May 25, 2017 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported that Palmetto GBA, a Medicare Administrative Contractor (MAC) that assesses molecular diagnostic technologies, has issued a positive final Local Coverage Determination (LCD) to expand Medicare coverage of the Prolaris test (Press release, Myriad Genetics, MAY 25, 2017, View Source [SID1234519305]).

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Today’s decision extends coverage to Medicare beneficiaries with favorable intermediate risk prostate cancer and builds on a prior decision that provided coverage for men with low- and very low-risk prostate cancer. The new LCD is posted to the Medicare Coverage Database on the Centers for Medicare & Medicaid Services (CMS) website with an effective date of July 10, 2017. Prolaris is the first and only genetic test to receive Medicare coverage for favorable intermediate and low- or very low-risk prostate cancer in the United States.

"We are excited that the MolDX program has expanded Prolaris coverage to the thousands of Medicare beneficiaries with favorable intermediate risk prostate cancer," said Mark C. Capone, president and CEO, Myriad Genetics. "The coverage decision is another important step to make sure the Prolaris test is broadly accessible to the patients who need it."

Of the newly diagnosed patients with prostate cancer, approximately 20 percent will be favorable intermediate risk prostate cancer, which is defined by National Comprehensive Cancer Network (NCCN) as a Gleason score of 3+4 or less, a percentage of positive biopsy cores less than 50 percent and, at most, one NCCN determinant of intermediate-risk prostate cancer. When combined with the previous Medicare coverage decision, more than 70 percent of Medicare patients with prostate cancer will have access to the Prolaris test.

"It is clinically challenging to determine how best to treat men with favorable intermediate risk prostate cancer. Our goal is to provide physicians with genetic information and help them tailor treatments based on patients’ individual risk profiles." said Michael Brawer, M.D., senior vice president of Urology, Myriad Genetic Laboratories. "The Prolaris test accurately measures the aggressiveness of prostate cancer and give’s both the patient and physician the confidence to make appropriate medical management decisions."

About Prolaris
Prolaris is a novel 46-gene RNA-expression test that directly measures tumor cell growth characteristics to quantify the aggressiveness of prostate cancer and help guide patient care. Prolaris is the only prognostic signature for prostate cancer which has been validated to predict 10-year prostate cancer specific mortality in an untreated patient cohort allowing men to make treatment decisions prior to surgical intervention. Additionally, Prolaris has been extensively validated on its ability to also predict patients that are at higher risk for biochemical recurrence and metastases. Studies have shown that following the Prolaris test almost two-thirds of men are good candidates for more conservative patient management, leading to lower treatment associated side effects and lower overall healthcare costs. For more information on how the Prolaris test can provide information to help decision-making in managing an individual’s localized prostate cancer visit www.Prolaris.com.

Venn Therapeutics acquires a novel β-catenin/BCL9 Inhibitor to turn “Cold” tumors “Hot”

On May 25, 2017 The University of Utah Research Foundation reported to have partnered with Venn Therapeutics, a biotechnology company focused on developing immune priming oncology therapies, to advance its novel small molecule β-catenin/BCL9 inhibitor, invented by Dr. Haitao (Mark) Ji (Press release, Venn Therapeutics, MAY 25, 2017, View Source [SID1234519738]).

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Current immuno-oncology therapies generate objective responses in 10% to 30% of patients and Venn aims to develop therapies that will expand the number of patients benefiting from these new treatment modalities.

The Wnt/β-catenin signaling pathway plays a critical role in regulating oncogenesis and metastasis of many cancers. The hyper-activation of β-catenin contributes to many malignant features of cancer cells. Recent clinical and basic science studies have repeatedly pointed out that the β-catenin signaling drives cancer cells to escape immune surveillance and suppresses anti-tumor immunity. A β-catenin inhibitor can short circuit this process generating a more robust immune response to tumors.

Dr. Haitao (Mark) Ji, currently an Associate Member at Moffitt Cancer Center, developed a novel small-molecule technology while at the University of Utah Research Center, to disrupt alpha-helix-mediated protein–protein interactions. Using this technology, he and his co-workers produced potent and selective small-molecule inhibitors for specific β-catenin interactions.

Dr. Ji was quoted as saying: "β-catenin is a highly relevant target for cancer treatment. I have been fortunate to work with a group of talented individuals while at the University of Utah, to tackle this important problem by developing new drug design technology. Our goal is to bring a small-molecule β-catenin/BCL9 inhibitor to the clinic and contribute to the fight against cancer."

Dr. Darrell Irvine, Professor at MIT, Howard Hughes Medical Institute and Koch Institute for Integrative Cancer Research Investigator remarked, "Recent studies suggest an important role for β-catenin in determining whether tumors are immunologically "hot" or "cold": Beta catenin expression in melanoma has been shown to lead to a blockade in recruitment of dendritic cells to tumors, and subsequently, lack of T cell infiltration. Thus this pathway may represent an important key to enhancing the efficacy of immunotherapies in general."

Dr. Paul Rennert, a highly respected oncology consultant and a member of Venn Therapeutics scientific advisory board further added, "Inhibition of β-catenin will give us an important tool to directly attack tumor cells while overcoming resistance to immune checkpoint therapy."

"We are excited to advance this novel small molecule towards the clinic. This is an important acquisition for us, as the asset has the safety and selectivity profile that we are looking for in a drug. It deepens our pipeline of early stage, innovative cancer therapies. This is a perfect complement to our STING agonist, which targets another critical pathway that promotes elimination of cancer in animal studies." said Sam Shrivastava, Venn Therapeutics’ chairman and chief executive officer.

Venn Therapeutics recently joined the University of South Florida’s Tampa Bay Technology Incubator (TBTI), where the company will utilize TBTI laboratories and facilities to perform essential R&D functions. USF’s TBTI supports both USF spinout companies and community startups through their early stage commercialization by offering programs, services and high-tech facilities to enrich the skills and ability of entrepreneurs to grow and develop their companies.

TESARO Announces Presentations of Abstracts at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting

On May 25, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported the presentation of three ZEJULA (niraparib) abstracts at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, June 2 to June 6, 2017, in Chicago (Press release, TESARO, MAY 25, 2017, View Source [SID1234519300]). In addition, TESARO will host an investor and analyst briefing in Chicago on Saturday, June 3 at 6:00 PM local time in conjunction with the ASCO (Free ASCO Whitepaper) Annual Meeting.

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"We’re excited to see presentations of additional data from the landmark ENGOT-OV16/NOVA trial presented at this year’s ASCO (Free ASCO Whitepaper) Annual Meeting," said Mary Lynne Hedley, Ph.D., President and COO, TESARO. "We also look forward to sharing initial data from the TOPACIO trial of niraparib plus pembrolizumab, as well as data from the Phase 1 trial of TSR-042, our anti-PD-1 antibody, at our ASCO (Free ASCO Whitepaper) investor briefing."

Please plan to visit TESARO at Booth #18097 to learn more about ZEJULA (niraparib), VARUBI (rolapitant) and our immuno-oncology pipeline.

Presentation Details:

Saturday, June 3, 2017, 1:15 PM to 4:45PM
Efficacy of Niraparib on Progression-free Survival (PFS) in Patients (Pts) with Recurrent Ovarian Cancer (OC) with Partial Response to the Last Platinum-based Chemotherapy
Abstract #5517, Poster Board #339, Location: Hall A
Poster Discussion: Saturday, June 3, 2017, 4:45 PM to 6:00 PM, Location: Aerie Crown Theater

Saturday, June 3, 2017, 1:15 PM to 4:45PM
Long-Term Benefit of Niraparib Treatment of Recurrent Ovarian Cancer (OC)
Abstract #5534, Poster Board #356, Location: Hall A

Saturday, June 3, 2017, 1:15 PM to 4:45PM
The Successful Phase 3 Niraparib ENGOT-OV16/NOVA Trial Included a Substantial Number of Patients with Platinum Resistant Ovarian Cancer
Abstract #5560, Poster Board #382, Location: Hall A

GlycoMimetics’ GMI-1271 Receives EU Orphan Drug Designation for Acute Myeloid Leukemia

On May 25, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported that the European Commission, based on a favorable recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products, has granted orphan designation for the company’s drug candidate GMI-1271 for the treatment of acute myeloid leukemia (AML) (Press release, GlycoMimetics, MAY 25, 2017, View Source [SID1234519296]). The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation for GMI-1271 for the treatment of AML in May of 2015.

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GMI-1271, a specific E-selectin inhibitor is being evaluated in the company’s ongoing Phase 1/2 clinical trial, in which clinicians are evaluating the use of GMI-1271 along with chemotherapy in patients with relapsed or refractory AML as well as those with newly diagnosed AML. Earlier this month, the company announced that GMI-1271 had been granted Breakthrough Therapy designation by the FDA. The company also announced that abstracts had been published by both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the European Hematology Association (EHA) (Free EHA Whitepaper) highlighting new data from the Phase 2 portion of the company’s ongoing Phase 1/2 trial that will be presented at their upcoming annual meetings in June.

"The European orphan designation will provide incentives for the commercialization and development of GMI-1271 in AML, where there are limited therapies available to patients," said Helen Thackray, M.D., FAAP, Senior Vice President, Clinical Development and Chief Medical Officer of GlycoMimetics. "We believe that GMI-1271, when combined with standard chemotherapy, has the potential to address an unmet therapeutic need for individuals living with AML, and we are encouraged by both our clinical results to date and achieving this designation from the European Commission."

The European Commission grants orphan designation to drugs intended to treat, prevent or diagnose life-threatening or chronically debilitating rare disorders, defined as diseases with prevalence of no more than 5 in 10,000 in the EU, for which no satisfactory method of diagnosis, prevention or treatment yet exists. Orphan designation provides benefits including commercialization incentives, protection of intellectual property, including 10 years of market exclusivity and protocol assistance through the EMA’s Scientific Advice program.

About AML

AML is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults. The National Cancer Institute estimates that there will be over 21,000 new cases of AML diagnosed in 2017 in the United States, and over 10,000 people will die from all forms of the disease in 2017. AML is more commonly present in elderly patients. Unlike other cancers that start in an organ and spread to the bone marrow, AML is known for rapid growth of abnormal white blood cells that gather in the bone marrow, getting in the way of normal blood cell production. The lack of normal blood cells can cause some of the symptoms of AML, including anemia (shortage of red blood cells resulting in tiredness and weakness), neutropenia (shortage of white blood cells that may lead to increased infections), and thrombocytopenia (shortage of platelets in the blood that may lead to excessive bleeding). Current treatment options for AML consist of reducing and eliminating cancer cells mainly through chemotherapy, radiation therapy, and stem cell transplantation.

X4 Pharmaceuticals Announces Initiation of the Phase 2 Expansion of its Phase 1/2 Study of X4P-001 in Patients with Advanced Clear Cell Renal Cell Carcinoma

On May 25, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare diseases, reported dosing of the first patient in the Phase 2 portion of the ongoing Phase 1/2 study evaluating X4P-001, the company’s lead CXCR4 inhibitor, in combination with Inlyta (axitinib) in patients with advanced clear cell renal cell carcinoma (ccRCC) (Press release, X4 Pharmaceuticals, MAY 25, 2017, View Source [SID1234519294]). Full results from the Phase 1 portion of the study including the selection of the once daily oral dose used in the expansion cohort will be presented at an upcoming medical meeting.

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In addition to safety and tolerability, the Phase 2 portion of the study will evaluate clinical efficacy as measured by objective response rate (ORR), duration of response (DOR), and progression free survival (PFS) and will explore the correlation of biomarkers with efficacy. Multiple cancer centers with leading renal cell carcinoma researchers in the U.S. and South Korea are participating in the study.

"Efficiently progressing this study into Phase 2 is an important milestone for the development of X4P-001 and the CXCR4-targeted therapeutic approach," said Sudha Parasuraman, MD, Chief Medical Officer of X4. "Having established the Phase 2 combination dose of X4P-001, we are now focused on augmenting proof of concept data for this critically important biological axis known to play a key role in immune cell trafficking."

In addition to this Phase 1/2 study of X4P-001 in combination with Inlyta, a VEGF kinase inhibitor, X4 has additional oncology clinical studies ongoing, including a Phase 1/2 study in patients with advanced ccRCC to evaluate X4P-001 in combination with Opdivo (nivolumab), and a Phase 1b biomarker study in patients with advanced melanoma to evaluate X4P-001 in combination with Keytruda (pembrolizumab).

About X4P-001 in Cancer

X4P-001, the company’s lead drug candidate, is currently in Phase 1/2 testing in refractory clear cell renal cell carcinoma (ccRCC) and other solid tumor indications. Based on promising preclinical studies, X4P-001 is being evaluated in clinical studies in combination with approved cancer therapies, including tyrosine kinase inhibitors and checkpoint inhibitors. X4P-001 is an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, the receptor for the chemokine CXCL12. Recent studies demonstrate that CXCR4/CXCL12 is a primary receptor-ligand pair that cancer cells and surrounding stromal cells use to block normal immune function and promote angiogenesis through the trafficking of T-effector and T-regulatory cells, as well as myeloid derived suppressor cells (MDSCs), in the tumor microenvironment.1,2

About Renal Cell Carcinoma

Kidney cancer is among the ten most common cancers in both men and women with more than 60,000 new diagnoses each year in the United States.3 Clear cell renal cell carcinoma (ccRCC) is the most common form of kidney cancer, and advanced ccRCC accounts for approximately 20% of the patient population. Therapies for advanced ccRCC include immunotherapies, mammalian target of rapamycin (mTOR) kinase inhibitors, and angiogenesis inhibitors, such as vascular endothelial growth factor (VEGF) inhibitors.4 There continue to be unmet medical needs with advanced ccRCC because durable responses remain a serious clinical challenge for patients with advanced disease.