On September 11, 2019 Veracyte, Inc. (Nasdaq: VCYT) reported the publication of new data demonstrating the clinical and analytical validity of its Afirma Xpression Atlas (XA) genomic test, which is used to help guide surgery and treatment decisions for patients with likely or confirmed thyroid cancer (Press release, Veracyte, SEP 11, 2019, View Source [SID1234539437]). The new findings appear online in the journal Frontiers in Endocrinology.

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The Afirma XA uses RNA whole-transcriptome sequencing to detect expressed DNA variants and RNA fusion partners in over 500 genes that are associated with thyroid cancer. The test is performed on fine needle aspiration (FNA) samples of thyroid nodules deemed suspicious for cancer by Veracyte’s Afirma Genomic Sequencing Classifier (GSC), as well as those that are suspicious for or have been diagnosed as cancer based on cytopathology. Importantly, the Afirma XA is performed on the same FNA sample as the Afirma GSC, obviating the need for patients to undergo an additional FNA procedure to obtain this genomic information about their thyroid.

To evaluate the Afirma XA’s clinical validity, researchers compared the test’s ability to identify genomic variants in an FNA sample’s transcriptome to currently accepted methods of targeted DNA and RNA sequencing. Using 943 blinded FNA samples, they found the Afirma XA had high positive predictive agreement (PPA) with targeted DNA sequencing (88 percent) and targeted RNA sequencing (89 percent). Similarly, using 695 blinded FNA samples to look for RNA fusions, the Afirma XA had an 82 percent PPA with targeted RNA sequencing. Conversely, 95 percent or more of variants and fusions identified by Veracyte’s RNA whole-transcriptome sequencing test were also identified by the reference method.

"Our findings suggest that the Afirma XA is sensitive and accurate in identifying gene alterations that are associated with thyroid cancer and confirm that the test can do this using one patient sample for all molecular testing," said Trevor E. Angell, assistant professor of clinical medicine at the Keck School of Medicine of USC and lead author of the new paper. "The extensive genomic-alteration information provided by the Afirma test can help physicians tailor initial treatment for patients with likely or confirmed cancer and also provides information about the potential benefits of targeted therapies for those cancers that don’t respond to standard treatment."

The researchers also investigated the reproducibility of the Afirma XA across laboratories and reagent lots. Using 69 variant-positive FNA samples, they found that the Afirma XA showed high accuracy between two different labs with different personnel for detecting variants (90 percent) and fusions (94 percent).

"The use of pre-operative molecular testing with minimally invasive FNA samples is expanding beyond cytologically indeterminate thyroid nodules to include those nodules with clear malignancy. Our findings demonstrate that physicians can be confident in the Afirma XA’s results at the time of diagnosis, which help guide surgery and treatment decisions," said Richard T. Kloos, M.D., medical director of endocrinology for Veracyte and an author of the new study.

On September 11, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has accepted for review its supplemental New Drug Application (sNDA) for neratinib in combination with capecitabine for the treatment of patients with HER2-positive metastatic breast cancer who have failed two or more prior lines of HER2-directed therapy (third-line disease) (Press release, Puma Biotechnology, SEP 11, 2019, View Source [SID1234539435]). The FDA has informed the Company that it is not currently planning to hold an advisory committee meeting to discuss this application. The FDA confirmed that the review will have an action date of late April, 2020.

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"The FDA’s acceptance of our sNDA marks another important regulatory milestone for my team," said Alan H. Auerbach, Chief Executive Officer and President of Puma. "We look forward to working with the FDA during its review of this submission, which targets patients with HER2-positive metastatic breast cancer who have progressed on two or more prior treatments and who need additional treatment options."

Neratinib was originally approved by the U.S. Food and Drug Administration in July 2017 for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer following adjuvant trastuzumab-based therapy and is marketed in the United States as NERLYNX tablets. In September 2018 NERLYNX was granted marketing authorization by the European Commission for the extended adjuvant treatment of adult patients with early stage hormone receptor-positive HER2-overexpressed/amplified breast cancer and who are less than one year from completion of prior adjuvant trastuzumab-based therapy.

The sNDA is supported by the results of the Phase III NALA trial, a randomized controlled trial of neratinib plus capecitabine versus Tykerb (lapatinib) plus capecitabine in patients with third-line HER2-positive metastatic breast cancer.

About HER2-Positive Breast Cancer

Approximately 20 to 25 percent of breast cancer tumors over-express the HER2 protein. HER2-positive breast cancer is often more aggressive than other types of breast cancer, increasing the risk of disease progression and death. Although research has shown that trastuzumab can reduce the risk of early stage HER2-positive breast cancer returning after surgery, up to 25% of patients treated with trastuzumab experience recurrence.

About NALA

The NALA trial is a randomized controlled Phase III trial of neratinib plus capecitabine versus Tykerb (lapatinib) plus capecitabine in patients with third-line HER2-positive metastatic breast cancer. The trial enrolled 621 patients who were randomized (1:1) to receive either neratinib plus capecitabine or lapatinib plus capecitabine. The trial was conducted globally at sites in North America, Europe, Asia-Pacific and South America. The co-primary endpoints of the trial are centrally confirmed progression free survival (PFS) and overall survival (OS). An alpha level of 1% was allocated to the PFS and 4% allocated to OS. The study was to be considered positive if either of the co-primary endpoints was positive. Puma reached agreement with the FDA under a Special Protocol Assessment (SPA) for the design of the Phase III clinical trial and the European Medicines Agency (EMA) also provided follow-on scientific advice (SA) consistent with that of the FDA regarding the Company’s Phase III trial design and endpoints used in the trial.

On September 11, 2019 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, reported the pricing of the sale of 4,000,000 shares of common stock of OncoCyte Corporation at a price to buyers of $1.66 per share, the closing price as of September 10, 2019 (Press release, Lineage Cell Therapeutics, SEP 11, 2019, View Source [SID1234539433]). Gross proceeds from the sale were $6.6 million, before payment of $100,000 in sales commissions and offering expenses. The sale is expected to close on September 13, 2019, subject to customary closing conditions. Following the completion of the sale, Lineage will own approximately 16% or 8.4 million shares of OncoCyte’s outstanding common stock. Based on the closing price of OncoCyte’s common stock on September 10, 2019, the value of Lineage’s remaining OncoCyte shares following the closing is approximately $14.0 million. Lineage has agreed not to sell additional shares of OncoCyte common stock until January 1, 2020 or unless the OncoCyte common stock price is above $3.00.

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"Our objective is to build Lineage into a premier cell therapy company. This latest transaction involving the sale of OncoCyte shares is part of a larger strategy to raise operating capital from time to time to support our three clinical-stage programs through sources other than Lineage common stock," stated Brian M. Culley, Chief Executive Officer. "We have no plans to conduct additional sales of OncoCyte in the near-term. We continue to have other funding mechanisms at our disposal via our remaining investments in AgeX Therapeutics, Inc. and Hadasit Bio-Holdings Ltd., as well as a $21.6 million promissory note due to us in August 2020 from Juvenescence Ltd. In parallel, we continue to assess other funding and strategic alliance opportunities which may be available through our existing or future potential partners. While it is imperative to support our internal programs, we remain one of OncoCyte’s largest shareholders and are highly supportive of their short and long-term growth plans. We look forward to their continued progress toward becoming a comprehensive diagnostic content company serving the needs of lung cancer patients across disease stages."

The Special Equities Group, LLC a division of Bradley Woods & Co. Ltd. acted as exclusive placement agent with respect to part of this transaction.

On September 11, 2019 ExCellThera Inc., an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, reported that Guy Sauvageau, President and CEO, will present a company update at the annual Cell & Gene Meeting on the Mesa to be held October 2-4 in Carlsbad, California (Press release, ExCellThera, SEP 11, 2019, View Source [SID1234539432]).

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ExCellThera’s lead technology, ECT-001, is a combination of a small molecule and an optimized culture system. The technology, capable of expanding stem and immune cells exponentially in as little as seven days, is used in novel curative cell therapies for patients with blood cancers and other hematologic malignancies, allowing more rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients. ECT-001 has received FDA orphan drug designation (ODD) for the prevention of graft-versus-host disease and regenerative medicine advanced therapy (RMAT) designation in the treatment of hematologic malignancies.

The following are specific details regarding ExCellThera’s presentation at the conference:

Event: 2019 Cell & Gene Meeting on the Mesa

Date: October 2, 2019

Time: 1:45pm PT

Location: Cognate Bioservices Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers. Complimentary attendance at this event is available for credentialed investors and members of the media only. www.meetingonthemesa.com

A live webcast of this presentation will be available at: View Source View Source/and will also be published on the conference website shortly after the event.

On September 11, 2019 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, reported that it has submitted an Orphan Drug Designation (ODD) request with the U.S. Food and Drug Administration (FDA) for 64Cu-SARTATE, a diagnostic for the clinical management of neuroendocrine tumours (NETs) (Press release, Clarity Pharmaceuticals, SEP 11, 2019, View Source [SID1234539431]).

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NETs are a group of cancers that originates in the diffuse neuroendocrine system. They most commonly occur in the gastrointestinal tract (48%), lung (25%), and pancreas (9%), but may also occur in other areas including the breast, prostate, thymus and skin. NET patients commonly present at an advanced (metastatic) disease stage. Due to the rarity of NETs and nonspecific presentation of symptoms, a delay in diagnosis or misdiagnosis is common. Mean patient-reported time from first symptom onset to diagnosis was 52 months; 29% of patients required ≥ 5 years for a NET diagnosis, and 58% of patients had metastases at the time of diagnosis. As a result, the median survival for patients can be as short as 10 months.

FDA grants ODDs to facilitate the development of investigational therapies intended to treat, diagnose or prevent rare diseases affecting fewer than 200,000 people in the United States. An ODD provides a number of benefits to pharmaceutical development companies, including potential tax credits for clinical costs, exemptions from certain administrative FDA fees, eligibility for grants to fund future clinical work and seven years of marketing exclusivity if a marketing application is approved.

Dr Alan Taylor, Clarity’s Executive Chairman, said "At Clarity, we are continuing to progress our products to market and this is an important regulatory step for the development of 64Cu-SARTATE in NETs.

"Despite the recent advances in diagnostic methods with gallium-68 (68Ga) labelled somatostatin analogs becoming a preferred diagnostic imaging agent for well-differentiated NETs, most NETs are still diagnosed at an advanced stage and can rarely be cured in these cases.

The issues relating to 68Ga mean that many patients are not getting access to the latest diagnostic tools. 68Ga is normally eluted from expensive and inefficient generators on site which require highly trained personnel to prepare the products and maintain the generators at each treatment centre. The short half life and limited supply of 68Ga also present challenges in scheduling and scanning of patients leading to significant patient backlogs.

"64Cu-SARTATE utilises copper-64, which has a half life that allows finished product to be centrally manufactured in large volumes to meet fluctuating end-user demands without the logistical and financial challenges associated with 68Ga. 64Cu-SARTATE therefore has the potential to improve early-stage diagnosis to prolong survival. Given the superior affinity of our products to copper, 64Cu-SARTATE also has the benefit of measuring later time points compared to 68Ga-based products, which may have the added benefit of better diagnostic and therapeutic outcomes.

"The receipt of the ODD status from the US FDA would enable us to advance the development of this product more quickly and efficiently, getting Clarity closer to our ultimate goal of better treatment of children and adults with cancer."

References
Cheung, Vincent T F, and Mohid S Khan. 2015. "A Guide to Midgut Neuroendocrine Tumours (NETs) and Carcinoid Syndrome." Frontline Gastroenterology 6 (4): 264–69. View Source

Hallet, Julie, Calvin How Lim Law, Moises Cukier, Refik Saskin, Ning Liu, and Simron Singh. 2015. "Exploring the Rising Incidence of Neuroendocrine Tumors: A Population-Based Analysis of Epidemiology, Metastatic Presentation, and Outcomes." Cancer 121 (4): 589–97. View Source

2018 Raphael, Michael J., David L. Chan, Calvin Law, and Simron Singh. 2017. "Principles of Diagnosis and Management of Neuroendocrine Tumours." CMAJ : Canadian Medical Association Journal 189 (10): E398–404. View Source