On April 19, 2018 Argos Therapeutics, Inc. (Nasdaq:ARGS), an immuno-oncology company focused on the development and commercialization of individualized immunotherapies based on the Arcelis precision immunotherapy technology platform, reported interim results from its randomized, active controlled, open-label, multi-center Phase 3 ADAPT trial of Rocapuldencel-T in combination with sunitinib/standard-of-care for the treatment of newly diagnosed metastatic renal cell carcinoma. Based on these results, the Company has decided to discontinue the trial (Press release, Argos Therapeutics, APR 19, 2018, View Source [SID1234525543]).

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As previously reported, a total of 462 patients with previously untreated advanced or metastatic renal cell carcinoma were enrolled in the ADAPT trial and randomized 2:1 between combination treatment with Rocapuldencel-T and sunitinib (combination arm) vs. sunitinib monotherapy (control arm) after undergoing cytoreductive nephrectomy. The Company recently submitted a protocol amendment to the U.S. Food and Drug Administration providing for four co-primary endpoints focused on various measures of survival. Based upon review of the interim data, the Company does not believe that it would achieve these endpoints if the trial were to be continued. After consulting with the principal investigators of the trial, the Company has therefore decided to discontinue the trial and has informed the FDA of its decision.

The most recent interim analysis was conducted after 51 new events (deaths) had occurred since the time of the February 2017 interim analysis. Median overall survival for the intent-to-treat patient population, one of the four co-primary endpoints, was estimated using the Kaplan-Meier method. The estimated median overall survival for the combination arm was 28.2 months (95% Confidence Interval (CI): 23.4, 35.2) compared to 31.2 months (95% CI: 23.0, 44.5) for the control arm. The hazard ratio was 1.10 (95% CI: 0.85, 1.42). The two other co-primary endpoints that were evaluated at this time, including overall survival for the patients who remained alive at the time of the February 2017 interim analysis and overall survival for all patients for whom at least 12 months of follow-up was available, also did not demonstrate a favorable result. A fourth endpoint, five-year survival, was not evaluated because there was insufficient data at this time to perform this analysis.

Based on a review of the status of its internal programs, resources and capabilities, Argos plans to explore a wide range of strategic alternatives that may include a potential merger or sale of the Company, among other potential alternatives that could maximize both near and long-term value for our shareholders. The Company has retained Stifel, Nicolaus & Company, Incorporated to serve as its financial advisor in the process.

Argos does not have a defined timeline for the exploration of strategic alternatives and is not confirming that the process will result in any strategic alternative being announced or consummated. Argos does not intend to discuss or disclose further developments during this process unless and until its Board of Directors has approved a specific action or otherwise determined that further disclosure is appropriate.

Argos also today reported that it does not expect to regain compliance with The Nasdaq Capital Market continued listing requirements by the April 24, 2018 deadline. As a result, Argos expects that its common stock will be delisted from The Nasdaq Capital Market and that trading in the Company’s common stock on The Nasdaq Capital Market will be suspended effective at the open of business on April 23, 2018. The Company has filed an application to transfer trading and quotation of its common stock to the OTCQB Venture Market, operated by OTC Markets Group Inc., under its current trading symbol "ARGS," effective as of April 23, 2018. Quotation and trading information for the common stock will be available on www.otcmarkets.com.

On April 19, 2018 Epigenomics AG (FSE: ECX, OTCQX: EPGNY) reported promising results from two clinical studies published in EBioMedicine supported by Cell Press and The Lancet, demonstrating high accuracy of Epigenomics’ proprietary epigenetic circulating biomarker mSEPT9 in detecting liver cancer among patients with cirrhosis (Press release, Epigenomics, APR 19, 2018, View Source [SID1234525523]).

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The two independent clinical studies (observational/case-control) included 289 cirrhosis patients with or without liver cancer from France (initial study) and Germany (replication study). Overall, the mSEPT9 test demonstrated high sensitivity of 90.6 percent at a specificity of 87.2 percent (using the "2 out of 3" algorithm). Importantly, a triple-negative mSEPT9 test had the highest negative predictive value for excluding liver cancer (97.2 percent), whereas a triple-positive mSEPT9 test had the highest positive predictive value for retaining a diagnosis of liver cancer (91.5 percent).

The results from the replication study were consistent with those of the initial study with regard to all diagnostic accuracy measures. Furthermore, the mSEPT9 blood test exhibited higher diagnostic accuracy compared to alpha-fetoprotein (AFP), which has been widely used as a diagnostic marker for liver cancer.

"The detection of liver cancer, one of the deadliest cancer types worldwide, still represents a high medical need," said Abderrahim Oussalah MD, PhD, Department of Molecular Medicine at the University Hospital of Nancy (France). "Findings from two independent clinical studies reveal that the mSEPT9 test constitutes a promising opportunity in this respect. As more clinical evidence is needed, we have initiated a further, prospective clinical study with 440 patients in order to confirm the diagnostic accuracy of mSEPT9 in the diagnosis of liver cancer (SEPT9-CROSS study, ClinicalTrials ID: NCT03311152). Future prospective studies should assess the mSEPT9 test in a screening algorithm for patients with cirrhosis to improve risk prediction and the personalized therapeutic management of liver cancer."

According to the World Health Organization, liver cancer is the fifth most common cancer in men and the seventh in women, and ranks second in annual cancer mortality rates worldwide, with liver cancer diagnosed in more than 700,000 people annually. Major risk factors for liver cancer include cirrhosis, infection with hepatitis B or C virus, alcoholic liver disease, and non-alcoholic fatty liver disease.

"We are very excited about the promising clinical results of our mSEPT9 blood test in the diagnosis of liver cancer", said Jorge Garces, President & Chief Scientific Officer of Epigenomics AG. "In the future, an accurate blood test could offer the opportunity to closely monitor patients at high risk for developing liver cancer."

The full-length paper is available here: View Source(18)30116-6/pdf

On April 19, 2018 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that data from pre-clinical studies supporting the potential of prexigebersen (BP1001, liposomal Grb2 antisense), in the treatment of solid tumors in gynecologic malignancies were presented in a poster at the annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), which took place in Chicago, IL (Press release, Bio-Path Holdings, APR 19, 2018, View Source [SID1234525544]).

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The poster titled, "Grabbing GRB2: The use of Liposome-incorporated Grb2 antisense oligonucleotides as a novel therapy in gynecologic malignancies," was presented by Olivia D. Lara, M.D., University of Texas MD Anderson Cancer Center, Department of Gynecologic Oncology during the Experimental and Molecular Therapeutics Poster Session. The data summarize results from studies investigating the expression of GRB2 in a series of in vitro experiments in high-grade serous (HGSC) and uterine (UC) carcinoma models. The study also examined the biological effects of prexigebersen in HGSC mice models (OVCAR 5), first in a dose-defining experiment then in combination with standard dose paclitaxel.

The data showed there was an eighty-six percent (86%) decrease in tumor burden (p<0.05), and multinodular burden (p<0.01) in the combination prexigebersen/paclitaxel group compared to control. In addition, there was no apparent toxicity with mice on combination therapy losing less weight than the paclitaxel-only group (P = 0.005).

"We are delighted to present these very encouraging findings at this year’s AACR (Free AACR Whitepaper) before an audience of the world’s leading cancer researchers and clinicians," stated Peter H. Nielsen, chief executive officer of Bio-Path Holdings. "Our research continues to suggest that prexigebersen-based combination therapy may offer an attractive method for targeting solid tumors and these findings establish the GRB2/GAB2 complex as an important target for prexigebersen. We look forward to advancing this promising therapy in gynecologic and other solid tumor cancers and plan to initiate first-in-human studies as early as the end of this year."

On April 19,2018 Novartis reported the appointment of John Tsai, M.D. as Head of Global Drug Development (GDD) and Chief Medical Officer (Press release, Novartis, APR 19, 2018, View Source [SID1234525524]). Dr. Tsai will join Novartis on May 1, 2018, and will be based in Basel, Switzerland. He will report to Vas Narasimhan, M.D., CEO of Novartis and will become a member of the Executive Committee of Novartis (ECN). He succeeds Dr. Narasimhan who became CEO of Novartis on February 1, 2018. Dr. Rob Kowalski, who led GDD ad interim since February 1, 2018, will resume his responsibilities as Head of Global Regulatory Affairs for GDD.

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Dr. Tsai has been Chief Medical Officer and Senior Vice President of Global Medical at Amgen Inc., since May 2017 and oversees all clinical and medical functions across multiple sites worldwide. At Novartis, he will be responsible for advancing the company’s industry-leading pipeline of innovative medicines and biosimilars. Dr. Tsai will also lead the GDD organization’s ongoing transformation embracing the power of advanced data sciences and digital technologies to create a more agile model for drug development.

"I am delighted to welcome John to Novartis," said Dr. Narasimhan. "As we continue to reimagine drug development, his expertise across multiple therapeutic areas, including cardiovascular, oncology and neuroscience combined with his background in electrical engineering will be a source of great strength for Novartis. John has a great track record in nurturing talent across clinical development, medical affairs and development operations and shares our commitment to build an empowered and inspired organization. I also want to express my sincere gratitude to Rob Kowalski for his excellent ad interim leadership of the GDD organization."

Dr. Tsai said: "I feel honored to have the opportunity to lead the Novartis Global Drug Development organization and do my part in bringing forward the company’s strong pipeline of medicines that address some of humanity’s biggest health challenges. I am also excited to work with my colleagues at Novartis to pioneer novel paradigms for drug development with data and digital technologies at the core."

Prior to joining Amgen, Dr. Tsai spent eleven years with Bristol-Myers Squibb (BMS), where he served as Global Head of Clinical Development for marketed products and global clinical operations. He also played a leadership role in advancing the company’s late-stage pipeline across multiple therapeutic areas including cardiovascular, oncology and neuroscience. As the company’s Chief Medical Officer, Europe and prior to that, as Head of U.S. Medical and Vice President of Cardiovascular Medical, Dr.Tsai played a critical role in driving multiple transformation initiatives within the development and medical organizations. Before joining BMS, he was a cardiovascular group leader at Pfizer where he led the strategy development and execution of over 26 pivotal trials for a major cardiovascular medicine. He started his career at GE as an electrical engineer before returning to school to study medicine.

Dr. Tsai holds a medical degree from the University of Louisville School of Medicine and a Bachelor of Science in Electrical Engineering from Washington University in St. Louis. He completed his residency at Kaiser Permanente in San Francisco, California, where he then served as a physician in internal medicine, Chief Resident and as a member of the hospital’s faculty.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "will," "pipeline," "ongoing," "to create," "continue," "commitment," "opportunity," "excited," "to pioneer," or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, or regarding potential future revenues from any such products express or regarding potential future sales or earnings of the Novartis Group. You should not place undue reliance on these statements. Such forward looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward looking statements. There can be no guarantee that any new products will be approved for sale in any market, or that any new indications will be approved for any existing products in any market, or that any approvals which are obtained will be obtained at any particular time, or that any such products will achieve any particular revenue levels. Nor can there be any guarantee Novartis will be commercially successful in the future, or achieve any particular financial results. In particular, our expectations could be affected by, among other things: regulatory actions or delays or government regulation generally; the potential that the strategic benefits, synergies or opportunities expected from the significant reorganizations of recent years may not be realized or may take longer to realize than expected; the uncertainties inherent in the research and development of new healthcare products; our ability to obtain or maintain proprietary intellectual property protection on key products; safety, quality or manufacturing issues; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; uncertainties regarding actual or potential legal proceedings; and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

On April 19, 2018 The U.S. Food and Drug Administration (FDA) approved AstraZeneca’s Tagrisso (osimertinib) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) in tumors with epidermal growth factor receptor (EGFR) mutations (Press release, BioSpace, APR 19, 2018, View Source [SID1234525545]).

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The mutations, exon 19 deletions or exon 21 L858R mutations, will be detected by an FDA-approved test. The decision was based on data form the Phase III FLAURA clinical trial. Results were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress and published in the New England Journal of Medicine.

The FLAURA trial compared Tagrisso to current first-line EGFR tyrosine kinase inhibitors, erlotinib or gefitinib, in patients who had not received previous treatment for locally-advanced or metastatic EGFR-mutation NSCLC. It met the primary endpoint of progression-free survival, and were consistent across all pre-specified patient subgroups.

Patients receiving Tagrisso had about 18.9 months on average before the disease got worse compared to 10.2 months for patients receiving traditional treatments.

Tagrisso is a third-generation, irreversible EGFR-TKI designed to inhibit both EGFR-sensitizing and EGFR T790M-resistance mutations, with clinical activity against central nervous system cancers. It has been approved in the U.S. and Brazil for first-line EGFRm advanced NSCLC, and in more than 75 countries, including the U.S., Europe, Japan and China, for patients with EGFR T790M mutation-positive advanced NSCLC. It is also being evaluated as an adjuvant and in combination with other treatments.

It has also been approved in the U.S. for second-line treatment of patients with metastatic EGFRm NSCLC, whose disease has on or after a first-line EGFR-TKI round of therapy and who have developed the secondary T790 mutation. The drug received both Breakthrough Therapy and Priority Review designations from the FDA in 2017 for the first-line setting.

"Today’s FDA approval of Tagrisso in the first-line setting is an exciting milestone for patients and our company," said Dave Fredrickson, executive vice president, Head of the Oncology Business Unit at AstraZeneca, in a statement. "Tagrisso delivered unprecedented median progression-free survival data across all pre-specified patient subgroups, including patients with or without CNS metastases, and could prolong the lives of more patients without their tumors growing or spreading."

The drug is being evaluated in Europe and Japan as a first-line treatment, with regulatory decisions expected in the second half of this year. AstraZeneca has projected that Tagrisso could, at its peak, bring in $4 billion per year.

Lung cancer accounts for about one-fifth of all cancer deaths, more than breast, prostate and colorectal cancers combined. It is the leading cause of cancer death for both men and women. About 10 to 15 percent of patients in the U.S. and Europe and about 30 to 40 percent in Asia, have EGFRm NSCLC. They are especially sensitive to EGFR-TKI treatments, but those cancers typically develop resistance to that treatment, leading to disease progression.

"The approval of osimertinib in the first-line setting represents a major advance in the treatment of patients with EGFR mutations and a significant change in the treatment paradigm," stated Suresh Ramalingam, principal investigator of the FLRUA trial, a researcher with the Winship Cancer Institute of Emory University in Atlanta. "Osimertinib provides robust improvements in progression-free survival with no unexpected safety signals compared to the previous generation of EGFR inhibitors."