On May 20, 2019 Early research using blood-based methylated DNA markers, identified through the longstanding collaboration between Exact Sciences and Mayo Clinic, reported a potential to achieve 92% sensitivity and 92% specificity for detecting the most common type of pancreatic cancer (Press release, Exact Sciences, MAY 20, 2019, View Source [SID1234536475]). Researchers presented the findings at Digestive Disease Week (DDW) 2019, the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery.

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"These pancreatic cancer data showcase the strength of our marker discovery collaboration with Mayo Clinic, a partnership that is fueling our product pipeline," said Kevin Conroy, chairman and CEO of Exact Sciences. "We aim to identify a minimally-invasive marker for a disease that often goes undetected until it progresses to an advanced stage, when fewer treatment options exist."

Pancreatic cancer is the third-leading cause of cancer-related deaths in the United States. According to the American Cancer Society, half of all pancreatic cancer patients are diagnosed in later stages when the five-year median survival rate is only 3%. No major guidelines recommend routine screening for average risk patients because the current detection options have not shown to lower a patient’s risk of death.

Lead author and Mayo Clinic gastroenterologist Shounak Majumder, M.D. presented results from the 340-sample, case-control study. A panel of methylated DNA markers in plasma in combination with CA 19-9, achieved a cross-validated sensitivity of 79% in Stage 1, 82% in Stage 2, 94% in Stage 3 and 99% in Stage 4 pancreatic ductal adenocarcinoma (PDAC) with 92% specificity (81-100%). This combination was significantly better than CA 19-9 alone. As Majumder writes in the abstract, "CA 19-9 is unreliable for early detection and may be normal in advanced disease."

Statistical modeling was used to identify the best algorithm to predict disease status in this sample set. Subsequently, the panel was cross validated by randomly splitting the entire data set into training and testing sets. The fitted model from the training set was used to predict disease status in the test set over multiple iterations. This study used a small number of archival patient blood samples, which could lead to over-fitting to this particular sample set and cause subsequent sensitivity and specificity to decline in a larger, prospective population. A prospective validation study is currently underway at Mayo Clinic.

"Powered by more than a decade of work with Exact Sciences, Mayo Clinic researchers continue producing promising data, in this case on blood-based biomarkers for pancreatic cancer detection," said Paul Limburg, MD, MPH, AGAF, Exact Sciences chief medical officer and Mayo Clinic gastroenterologist.i "While further research must be completed, this serves as an early indicator of encouraging news in the fight against this deadly disease."

The Exact Sciences and Mayo Clinic collaboration focuses on identifying biomarkers for 15 of the deadliest cancers. A poster showing Mayo Clinic researchers work on esophageal cancer will also be presented at DDW.

On May 20, 2019 Emergent BioSolutions Inc. (NYSE: EBS) reported that a member of the company’s senior management team will participate in the following investor conferences during the second quarter of 2019 (Press release, Emergent BioSolutions, MAY 20, 2019, View Source;p=RssLanding&cat=news&id=2399088 [SID1234536474]):

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RBC Capital Markets – 2019 Global Healthcare Conference
New York, NY
May 21-22, 2019

Goldman Sachs – 40th Annual Global Healthcare Conference (1×1 Meetings Only)
Rancho Palos Verdes, CA
June 11-13, 2019

Three Part Advisors – East Coast IDEAS Conference
Boston, MA
June 12-13, 2019

BMO – Prescriptions for Success Healthcare Conference 2019
New York, NY
June 25, 2019

Presentation dates and times will be updated on the Emergent website www.emergentbiosolutions.com under "Investors" as the information becomes available.

For these conferences, the company will be webcasting its presentation, which may include a discussion of the company’s recent business developments as well as its most recently reported financial results and guidance. The webcasts will be available both live and by replay, accessible from the Emergent website.

On May 20, 2019 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST) reported that Dr. Patricia Keller, Senior Scientist at Constellation Pharmaceuticals, presented a poster titled Targeting epigenetic dysregulation in bladder cancer through inhibition of EZH2 at the Bladder Cancer: Transforming the Field meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in Denver on May 19 (Press release, Constellation Pharmaceuticals, MAY 20, 2019, View Source [SID1234536473]).

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The poster discusses results of Constellation’s work with its second-generation EZH2 inhibitor CPI-0209 in bladder cancer models, exploring whether ARID1A mutation status affected responsiveness to CPI-0209 treatment. ARID1A, a protein that is a key component of the SWI/SNF chromatin remodeling complex, is mutated in about 25% of muscle-invasive bladder cancers.

Constellation used CPI-0209 in long-term phenotypic growth assays in a panel of 21 bladder cancer cell lines, demonstrating preferential treatment sensitivity that correlated with increased cell death in cell lines harboring ARID1A mutations. Transcriptional profiling after CPI-0209 treatment showed widespread activation of EZH2 target gene expression. In vivo, once-daily treatment with CPI-0209 in bladder cell-line derived xenografts harboring ARID1A mutations produced dose-dependent tumor growth inhibition and regression. Treatment with CPI-0209 and the chemotherapeutic agent cisplatin demonstrated combinatorial effects on cell viability in vitro and on tumor growth in vivo. The poster can be viewed in the Investors & Media/Presentations section of our website.

On May 20, 2019 Compugen Ltd. (NASDAQ: CGEN), a leader in predictive discovery and development of first-in-class therapeutics for cancer immunotherapy, reported financial results for the first quarter ended March 31, 2019 (Press release, Compugen, MAY 20, 2019, View Source [SID1234536472]).

"2019 continues to be marked by strong execution and we anticipate reaching additional important milestones in the second half of the year," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "Patient recruitment and site engagement in our Phase 1 study for COM701 are progressing as planned, and we now have 10 leading sites participating in our study. Enrollment in the monotherapy dose escalation arm is expected to be completed by the end of the third quarter, clearing the way for us to begin enrolling patients in the monotherapy expansion cohorts later this year. In addition, based on the progress made in the COM701 monotherapy dose escalation arm, we are confident in moving forward with the combination dose escalation arm of the study for which we expect to complete enrollment this year."

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"In parallel, we are also advancing our earlier stage pipeline focused on first-in-class molecules modulating immuno-suppressive cells in the tumor microenvironment, mainly cells from the myeloid lineage, as well as pursuing discovery efforts targeted at identifying proteins and pathways which are involved in immune resistance mechanisms to PD-1 therapies. Our goal is to generate a sustainable pipeline to ensure we have high potential first-in-class assets in addition to COM701, both for internal development as well as for additional partnering opportunities."

Recent Corporate Highlights

Dosed first patient in the combination arm of Phase 1 study, combining escalating doses of COM701 with a fixed dose of Opdivo (nivolumab) in patients with advanced solid tumors. Combination arms of the study are conducted under the clinical collaboration agreement signed with Bristol-Myers Squibb in October 2018.

Presented trial-in-progress posters at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium in January and the 2019 AACR (Free AACR Whitepaper) annual meeting in April. At AACR (Free AACR Whitepaper), the Company reported that the fifth dose level patient cohort of COM701 monotherapy has been completed and that no dose-limiting toxicities were found. Clinical and laboratory assessment for safety and tolerability are ongoing for this and earlier dose level patient cohorts.

Awarded by the U.S. Patent and Trademark Office U.S. Patent No. 10,213,505, which covers the composition comprising the COM701 and the backup antibodies and U.S. Patent No. 10,227,408, which covers composition comprising an anti-PVRIG antibody having CDRs of COM701 and backup antibodies.

Published two peer-reviewed papers in Cancer Immunology Research demonstrating the role of PVRIG as a novel immune checkpoint target for cancer immunotherapy.

Financial Results
Revenues for the first quarter of 2019 were $0, compared with $10 million in the comparable period of 2018. The revenues for the first quarter of 2018 reflect the upfront payment of $10 million from the license agreement with MedImmune/AstraZeneca.

R&D expenses for the first quarter ended March 31, 2019 were $6.3 million, compared with $7.1 million for the comparable period in 2018. The decrease in R&D expenses is attributed to the cost reduction measures announced by the Company during the first quarter of 2019. Further reduction in expenses will be reflected over the course of 2019.

Net loss for the first quarter of 2019 was $8.4 million, or $0.14 per basic and diluted share, compared with a net income of $0.1 million, or $0.0 per basic and diluted share, in the comparable period of 2018.

As of March 31, 2019, cash, cash related accounts, short-term and long-term bank deposits totaled $38.2 million, compared with $45.7 million at December 31, 2018. During the three months ended March 31, 2019, the Company sold approximately 961,000 ordinary shares under its "at-the-market" (ATM) facility pursuant to a sales agreement entered into with Cantor Fitzgerald & Co. in May 2018 for aggregate proceeds of $3.4 million, net of commissions to Cantor and expenses related to the offering. The Company has no debt.

Conference Call and Webcast Information
Compugen will hold a conference call to discuss its first quarter 2019 results today, May 20, 2019, at 8:30 a.m. ET. To access the live conference call by telephone, please dial 1-888-668-9141 from the U.S., or +972-3-918-0687 internationally. The conference call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

On May 20, 2019 Ayala Pharmaceuticals, Inc., a clinical-stage company developing medicines for cancers that are genetically defined, reported updates on AL101, a pan-Notch inhibitor, that will be presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, Il (Press release, Ayala Pharmaceuticals, MAY 20, 2019, View Source [SID1234536471]).

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The details of the poster presentations are as follows:

Title: ACCURACY: phase (P) 2 trial of AL101, a pan-Notch inhibitor, in patients (pts) with recurrent/metastatic (R/M) adenoid cystic carcinoma (ACC) with Notch activating mutations (Notchact mut)

Date and Time: Saturday, June 1 at 1:15 – 4:15 p.m. CT
Location: McCormick Place, Exhibit Hall A, Poster Section: Head and Neck Cancer
Poster Board Number: 82B
Abstract Number: TPS6098

Title: AL101 mediated tumor inhibition in notch-altered TNBC PDX models

Date and Time: Sunday, June 2 at 8:00 – 10:00 a.m. CT
Location: McCormick Place, Exhibit Hall A, Poster Section: Breast Cancer – Metastatic
Poster Board Number: 145
Abstract Number: 1064

Ayala Pharmaceuticals is developing its investigational product AL101 for treatment of tumors with Notch activated mutations. Ayala has prioritized its research to focus on indications where the role of Notch activation is well characterized, such as ACC and TNBC. AL101 is a small-molecule that inhibits gamma secretase, an enzyme which plays a key role in the activation of the Notch signaling pathway by releasing the Notch intracellular domain (NICD) which migrates to the nucleus initiating a complex transcription program.

The FDA recently granted Orphan Drug Designation for AL101 for the treatment of ACC. The ACCURACY Phase 2 trial is a multicenter, open-label trial in ACC patients with Notch activated mutations. The primary endpoint of the trial is to assess overall response rate upon weekly administration of AL101 4 mg IV. Secondary endpoints include duration of response, progression free survival and overall survival. Twelve patients are currently being treated in stage one of the phase 2 ACCURACY trial.

"I’m very excited about this trial given the early signs of activity previously observed in Phase 1b study and in ACC Patient Derived Xenograft animal models with AL101 in tumors that harbor Notch activated mutations," said Dr. Renata Ferrarotto, M.D., Associate Professor, Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center. "This trial is an important step toward understanding the role AL101 may have in treating ACC patients with Notch activating mutations."

Additionally, Ayala Pharmaceuticals recently completed a preclinical study for TNBC, a sub-type of breast cancer associated with poor prognosis and limited availability of targeted therapies. The Notch pathway is activated during mammary gland development and has been implicated as a key driver in a subset of TNBC.

The preclinical data to be presented at ASCO (Free ASCO Whitepaper) demonstrate that the response to AL101 monotherapy correlates with Notch activating mutations and fusions. Specifically, nine patients derived xenograft (PDX) models of TNBC were selected, whereby two of the PDX models had wild type (WT) Notch, four PDX models had Notch activating mutations and/or fusions and three models had Notch mutations which were not predicted to result in activation. Mice were randomized to Vehicle or AL101 treatment arms and significant inhibition of tumor growth was observed only in models with Notch activating mutations and fusions. These data support the clinical development of AL101 as a targeted therapy for TNBC tumors bearing Notch activating mutations/fusions.

"We are passionate about bringing innovative treatment options to people living with ACC and TNBC. I’m pleased with the meaningful advances Ayala is making to offer new hope to those living with underserved cancers," said Roni Mamluk, Ph.D., Chief Executive Officer at Ayala Pharmaceuticals. "Our clinical programs are advancing swiftly, in fact, by the end of this year, we expect to have initial response rates from 12 patients of the first stage of our ongoing ACCURACY study in ACC."

About AL101

AL101 is a gamma secretase inhibitor developed as a Notch inhibitor for oncology indications. Notch signaling pathway plays an important role in tumorigenesis in several solid and hematological malignancies. Upon ligand binding of the Notch receptor, an important step in the activation of Notch receptors is cleavage by gamma secretase, which frees the Notch intracellular signaling domain.

AL101 is currently in Phase 2 for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY). For additional information about AL101 ACCURACY clinical trial, please go to www.clinicaltrials.gov. Interested patients and physicians can contact Medical Affairs at Ayala Pharmaceuticals for more information at: [email protected] or call +1-857-444-0553.

About Adenoid Cystic Carcinoma (ACC)

ACC is a rare form of cancer. In the U.S., there are approximately 566,000 people diagnosed with cancer each year, and only about 1,224 of them are diagnosed with ACC. According to the Adenoid Cystic Carcinoma Organization International, there are approximately 14,873 Americans alive today living with this disease. Current treatment options include surgery, chemotherapy and/or radiation therapy; however, there are no approved drugs for the treatment of ACC.

About Triple Negative Breast Cancer (TNBC)

TNBC is a heterogeneous disease entity constituting about 15 percent of breast cancer cases worldwide. TNBC is associated with poor prognosis and lack of sustained response to conventional chemotherapeutic agents. Patients with recurrent and/or metastatic TNBC has a median survival of approximately 13 months; therefore, there is an urgent need to identify new therapeutic strategies for these patients.