On September 11, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of novel cancer therapies, reported that CEO Saiid Zarrabian will deliver a presentation on the Company and meet with investors at the Fall Investor Summit on September 16, 2019 at 1:30 PM ET in Track 1 at Essex House, 160 Central Park South in New York City (Press release, DelMar Pharmaceuticals, SEP 11, 2019, View Source [SID1234539440]). The conference is being held September 16-17, 2019.

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Interested investors may register for the conference at https://fall-investor-summit.events.issuerdirect.com/signup, or may request a meeting time by contacting John Marco at Core IR, either by calling 516-222-2560 or via [email protected].

On September 11, 2019 Merck KGaA, Darmstadt, Germany, a leading science and technology company, which operates its biopharmaceutical business as EMD Serono in the US and Canada, reported that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for the investigational targeted therapy tepotinib* in patients with metastatic non-small cell lung cancer (NSCLC) harboring MET exon 14 skipping alterations who progressed following platinum-based cancer therapy (Press release, Merck KGaA, SEP 11, 2019, View Source [SID1234539439]).

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"Tepotinib was associated with robust objective responses with durability that has not previously been seen in patients with metastatic NSCLC harboring MET exon 14 skipping alterations, selected by either tissue or liquid biopsy approaches," said Luciano Rossetti, Global Head of Research & Development for the Biopharma business of Merck KGaA, Darmstadt, Germany. "This breakthrough therapy designation further underscores the potential of tepotinib, and we aim to advance this program and deliver this medicine as quickly as possible to NSCLC patients who may benefit."

Lung cancer is the most common type of cancer worldwide, with 2 million cases diagnosed annually.1 Alterations of the MET signaling pathway are found in various cancer types, including 3-5% of NSCLC cases, and correlate with aggressive tumor behavior and poor clinical prognosis.2-4

Discovered in-house at Merck KGaA, Darmstadt, Germany, tepotinib is an investigational oral MET kinase inhibitor that is designed to be highly potent and selective5 and to inhibit the oncogenic signaling caused by MET (gene) alterations, including both MET exon 14 skipping alterations and MET amplifications, or MET protein overexpression.

In March 2018, tepotinib’s potential was recognized by the Japanese Ministry of Health, Labour and Welfare (MHLW), which granted SAKIGAKE ‘fast-track’ designation for tepotinib in advanced NSCLC harboring MET exon 14 skipping alterations. SAKIGAKE designation promotes research and development in Japan, aiming at early practical application for innovative pharmaceutical products, medical devices and regenerative medicines.

Tepotinib is also being investigated in the INSIGHT 2 study (NCT03940703) in combination with the tyrosine kinase inhibitor (TKI) osimertinib in epidermal growth factor receptor (EGFR) mutated, MET amplified, locally advanced or metastatic NSCLC having acquired resistance to prior EGFR TKI.

The Breakthrough Therapy Designation is based on data from the ongoing VISION study (NCT02864992), showing preliminary clinical evidence that tepotinib may offer an improvement over available therapy in patients with metastatic NSCLC harboring MET exon 14 skipping alterations detected by liquid biopsy (LBx) or tissue biopsy (TBx) across different lines of treatment.

Results from an interim analysis of the ongoing VISION study in 73 efficacy-evaluable patients with NSCLC with MET exon 14 skipping alterations identified by LBx or TBx testing demonstrate overall objective response rate (ORR) of 50.0% for LBx-identified patients as assessed by Independent Review Committee (IRC), and 55.3% as assessed by investigators. The ORR for TBx-identified patients was 45.1% and 54.9%, respectively. The overall median duration of response (DOR) was 12.4 months and 17.1 months among LBx-identified patients, as assessed by IRC and investigators, respectively, while among TBx-identified patients, 15.7 and 14.3 months were observed, respectively.

Most treatment-related adverse events (TRAEs) were Grade 1 and 2. No Grade 4 or 5 TRAEs were observed. Any grade TRAEs reported by ≥10% of 87 patients evaluable for safety were peripheral edema (48.3%), nausea (23.0%) diarrhea (20.7%) and increased blood creatinine (12.6%). Other relevant TRAEs of any grade include increased lipase (4.6%), fatigue (3.4%) and vomiting (3.4%). TRAEs led to permanent discontinuation in four patients (two patients due to peripheral edema, one due to interstitial lung disease, one due to diarrhea and nausea).

Results from this study were presented in an oral presentation at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.6 The use of both LBx and TBx to identify patients for the VISION study is intended to support improved patient selection and is consistent with the company’s focus on patient-centric drug development.

*Tepotinib is the recommended International Nonproprietary Name (INN) for the MET kinase inhibitor (MSC2156119J). Tepotinib is currently under clinical investigation and not approved for any use anywhere in the world.

About Breakthrough Therapy Designation

Breakthrough Therapy Designation is designed to expedite the development and review of drugs which are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). The FDA’s granting of the Breakthrough Therapy Designation for advanced NSCLC does not alter the standard regulatory requirement to establish the safety and effectiveness of a drug through adequate and well-controlled studies to support approval.

About Non-Small Cell Lung Cancer

With 2 million cases diagnosed annually, lung cancer (including trachea, bronchus and lung) is the most common type of cancer worldwide, and the leading cause of cancer-related death, with 1.7 million mortality cases worldwide.1 Alterations of the MET signaling pathway, including MET exon 14 skipping alterations and MET amplifications, occur in 3-5% of NSCLC cases.2-4

About Tepotinib

Tepotinib, discovered in-house at Merck KGaA, Darmstadt, Germany, is an investigational oral MET inhibitor that is designed to inhibit the oncogenic MET receptor signaling caused by MET (gene) alterations, including both MET exon 14 skipping alterations and MET amplifications, or MET protein overexpression. It has been designed to have a highly selective mechanism of action, with the potential to improve outcomes in aggressive tumors that have a poor prognosis and harbor these specific alterations.

Tepotinib is currently being investigated in NSCLC and Merck KGaA, Darmstadt, Germany is actively assessing the potential of investigating tepotinib in combination with novel therapies and in other tumor indications.ray F, et al. CA Cancer J Clin. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. 2018;68(6):394–424. View Source View Source.
Reungwetwattana T, et al. Lung Cancer 2017;103:27-37.
Mo HN, et al. Chronic Dis Transl Med 2017; 3(3):148-153.
Lutterbach B, et al. Cancer Res 2007;67:2081–8.
Bladt, F, et al. Clin Cancer Res 2013;19:2941-2951.
Paik P, et al. J Clin Oncol 2019;37: (suppl; abstr 9005).
All Merck KGaA, Darmstadt, Germany, press releases are distributed by e-mail at the same time they become available on the EMD Group Website. In case you are a resident of the USA or Canada please go to www.emdgroup.com/subscribe to register for your online subscription of this service as our geo-targeting requires new links in the email. You may later change your selection or discontinue this service.

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.