On September 5, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX) reported that target enrollment for the UNITY-CLL Phase 3 trial has been achieved (Press release, TG Therapeutics, SEP 5, 2017, View Source [SID1234520372]). The UNITY-CLL Phase 3 trial is a randomized study of TG-1101 (ublituximab), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, in combination with TGR-1202 (umbralisib), the Company’s PI3K delta inhibitor (together referred to as the U2 regimen), compared to an active control arm of obinutuzumab plus chlorambucil, in patients with both treatment naïve and relapsed or refractory Chronic Lymphocytic Leukemia (CLL) . Per the protocol, the target enrollment was 175 patients in each the U2 arm and the active control arm. While target enrollment has been reached, in order to provide an opportunity for all patients already identified to participate, enrollment is expected to continue until mid-October. The UNITY-CLL Phase 3 trial is being conducted pursuant to a Special Protocol Assessment (SPA) agreement with the Food and Drug Administration (FDA).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "We are extremely pleased to announce the completion of target enrollment in the UNITY-CLL clinical trial, nearly 9 months earlier than our original projections. From the beginning, enrollment in this study has exceeded our expectations, putting us in position to deliver top-line data on the ORR endpoint earlier than anticipated, now expected in the second quarter of 2018. If positive, pursuant to our SPA, the ORR data may be used to support a filing for accelerated approval." Mr. Weiss continued, "We want to thank the UNITY-CLL Investigators, research staff, and their patients, for their participation in this important clinical research. Without their commitment and trust, advancing novel medicines would not be possible."

John Gribben, MD, DSc, of the Barts Cancer Institute in London, UK, and Global Study Chair for the UNITY-CLL study stated, "The design of the UNITY-CLL trial is a paradigm shift in the way we approach oncology research, allowing two novel drugs to be evaluated together in a single four-arm study for potential approval. The speed in which target enrollment has completed is even more impressive, considering the size and scope of this innovative study, which also underscores the need for new treatment options despite recent advances. I am eagerly looking forward to presenting results from this study next year."

Ian Flinn, MD, PhD, Director of the Blood Cancer Research Program, Sarah Cannon Research Institute, and a lead US enroller in the UNITY-CLL study stated, "We were pleased at Tennessee Oncology to not only lead enrollment in the first-in-human study of umbralisib, but to now be one of the leading enrollers to the Phase 3 UNITY-CLL trial. The rapid rate of enrollment for both the first-in-human study of umbralisib and the Phase 3 study UNITY study is a testament to the continued need for better, patient-friendly, easy to use, treatment options for both treatment naïve CLL patients and those relapsed or refractory to prior therapy. BTK intolerance and difficulties in delivering anti-bcl2 therapy are challenges for delivering effective chemo-free treatment options for many patients. Approval of the ublituximab + umbralisib regimen would provide new hope to many of those patients.

ABOUT UNITY-CLL PHASE 3 TRIAL

UNITY-CLL is a global Phase 3 randomized controlled clinical trial in patients with Chronic Lymphocytic Leukemia (CLL) that includes two key objectives: first, was to demonstrate contribution of each agent in the TG-1101 (ublituximab) + TGR-1202 (umbralisib) or U2 regimen, and second, to demonstrate superiority in Progression Free Survival (PFS) over the standard of care to support the submission for full approval of the combination. In addition, upon completion of enrollment, this trial will evaluate Overall Response Rate (ORR) for accelerated approval. The study initially randomized patients into four treatment arms: TG-1101 plus TGR-1202, TG-1101 single agent, TGR-1202 agent, and an active control arm of obinutuzumab plus chlorambucil. Pursuant to the Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA), an early interim analysis was conducted to assess contribution of each single agent which allowed for early termination of both single agent arms.

On September 5, 2017 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported that Anthem has issued a positive coverage decision for the Prosigna Breast Cancer Gene Signature Assay (Press release, NanoString Technologies, SEP 5, 2017, View Source [SID1234520370]). This positive coverage decision is in line with updated ASCO (Free ASCO Whitepaper) guidelines released in February 2016. The ASCO (Free ASCO Whitepaper) guidelines recommend the use of the Prosigna Breast Cancer assay to guide decisions on adjuvant systemic therapy for women with early-stage invasive breast cancer with known hormone receptor and HER2 status.

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"With this positive coverage decision from Anthem, Prosigna is now covered by every national commercial plan in the United States and the vast majority of regional plans," said Brad Gray, president and chief executive officer of NanoString Technologies. "We now estimate that over 95% of US patients that are indicated for Prosigna are covered, and our team continues to work with the remaining regional plans to ensure that their patients have access to this state-of-the-art test."

About the Prosigna Breast Cancer Prognostic Gene Signature Assay and nCounter Dx Analysis System
The Prosigna Assay provides a risk category and numerical score for assessment of the risk of distant recurrence of disease at 10 years in postmenopausal women with node-negative (Stage I or II) or node-positive (Stage II), hormone receptor-positive (HR+) breast cancer. Based on the PAM50 gene signature initially discovered by Charles Perou, Ph.D. and colleagues, the Prosigna Assay is an in vitro diagnostic tool that utilizes gene expression data weighted together with clinical variables to generate a risk category and numerical score to assess a patient’s risk of distant recurrence of disease. The Prosigna Assay measures gene expression levels of RNA extracted from formalin-fixed paraffin embedded (FFPE) breast tumor tissue previously diagnosed as invasive breast carcinoma.

The Prosigna Assay requires minimal hands-on time and runs on NanoString’s proprietary nCounter Dx Analysis System, which offers a reproducible and cost-effective way to profile many genes simultaneously with high sensitivity and precision.

The nCounter Dx Analysis System is a highly automated and easy-to-use platform that utilizes a novel digital barcoding chemistry to deliver high precision multiplexed assays. The system is available in the multi-mode FLEX configuration, which is designed to meet the needs of high-complexity clinical laboratories seeking a single platform with the flexibility to run the Prosigna Breast Cancer Assay and, when operated in the "Life Sciences" mode, process translational research experiments and multiplexed assays developed by the laboratory.

In the United States, the Prosigna Assay is 510(k) cleared for use on the nCounter Dx Analysis System, and is available for diagnostic use when ordered by a physician. The Prosigna Assay has been CE-marked and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as Canada, Israel, Australia, New Zealand and Hong Kong. In the U.S., the Prosigna Assay is indicated in female breast cancer patients who have undergone surgery in conjunction with locoregional treatment consistent with standard of care, either as:

(1) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors or (2) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-positive (1-3 nodes), Stage II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. The device is not intended for patients with four or more positive nodes.

For more information, please visit www.prosigna.com.

On September 5, 2017 Acceleron Pharma Inc. (NASDAQ: XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, reported that The Lancet Oncology has published results from the Phase 2 study of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) (Press release, Acceleron Pharma, SEP 5, 2017, View Source [SID1234520369]).

“The efficacy and safety results in this Phase 2 study support continued research into luspatercept for the treatment of refractory anemia which often requires red blood cell transfusions in lower-risk MDS patients,” said Uwe Platzbecker, M.D., Professor of Hematology and Head of the MDS program at the University Hospital in Dresden, Germany. “This novel investigational therapy has the potential to address a significant need in MDS patients who currently have very limited options for managing chronic anemia. We have already begun exploring luspatercept’s activity across a range of MDS patient populations.”

“We are pleased that The Lancet Oncology chose to publish these results,” said Matthew Sherman, M.D., Chief Medical Officer of Acceleron. “The positive clinical activity demonstrated in this study informed our earlier decision to initiate the pivotal MEDALIST Phase 3 trial in lower-risk MDS, and we expect to report top-line results from this trial in mid-2018.”

The article, entitled “Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: a phase 2 dose-finding study with long-term extension study” is now available online and will be published in a future print issue of The Lancet Oncology. The journal also published online a companion Comment article, “Defeating anaemia in myelodysplastic syndromes: another step forward,” by Valeria Santini, M.D., Associate Professor of Hematology at the University of Florence Medical School in Florence, Italy.

Phase 2 presentations of luspatercept in MDS presented at recent medical conferences include updated longer-term follow-up and new expansion cohort preliminary results beyond that incorporated in this publication. Presentations outlining these results are available online under the science page on the Company’s website at www.acceleronpharma.com.

The MEDALIST trial, a global Phase 3 study of luspatercept in lower-risk MDS patients, is fully enrolled and top-line results are expected in mid-2018. The MEDALIST trial enrolled patients who are ring sideroblast-positive, red blood cell transfusion dependent, and are erythropoiesis-stimulating agent (ESA)-refractory or ESA-treatment ineligible, based on erythropoietin levels greater than 200 units per liter at baseline. In early 2018, Acceleron and Celgene plan to initiate the COMMANDS Phase 3 trial in first-line, lower-risk MDS patients.

Luspatercept is an investigational product that is not approved for use in any country.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the transforming growth factor-beta superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoiesis stimulating agents (ESAs), which stimulate the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Phase 3 clinical trials are underway to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the MEDALIST study) and in patients with beta-thalassemia (the BELIEVE study). For more information, please visit www.clinicaltrials.gov.