On December 11, 2017 The OHSU Knight Cancer Institute and Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported the presentation of preclinical data demonstrating that CG’806, a pan-FLT3/pan-BTK inhibitor, has broad and potent drug activity against acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL) and other hematologic disease subtypes (Press release, Aptose Biosciences, DEC 11, 2017, View Source [SID1234522544]). The data were highlighted in a poster presentation on Monday, December 11, 2017 at the American Society of Hematology (ASH) (Free ASH Whitepaper) 59th Annual Meeting & Exposition, being held December 9-12 in Atlanta, GA.

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The poster CG’806, a First-in-Class Pan-FLT3/BTK Inhibitor, Exhibits Potent Growth Inhibition as a Single Agent and in Combination with a BET Bromodomain Inhibitor and a Bcl2 Inhibitor Against AML and CLL Patient Samples, evaluated the activity of CG’806 on various hematologic malignancy cell lines and patient primary bone marrow specimens through the Beat AML Initiative. CG’806 exhibited broad and potent activity against primary patient samples over a diverse range of hematologic malignancy subtypes, including AML, CLL, myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN), and acute lymphoblastic leukemia (ALL).

The poster presentation can be accessed on the Events & Presentations section of the Aptose website at the following link.

"In patient samples and cultured cell lines, CG’806 demonstrated potent and broad inhibitory activity against hematologic malignancies alone and in combination," said Stephen E. Kurtz, Ph.D., lead author and Research Assistant Professor at the OHSU Knight Cancer Institute. "Both AML and CLL are in urgent need of effective therapies that provide more durable clinical responses. CG’806 represents a potential new treatment approach that warrants further development."

OHSU researchers used an ex vivo drug sensitivity assay to determine the activity of CG’806 as a single agent and in combination with the BET bromodomain inhibitor OTX-015 or the Bcl2 inhibitor venetoclax on freshly isolated primary patient samples. Across the four general subtypes of hematologic malignancies in the dataset with patient samples, there was broad sensitivity to CG’806, with 55% (90/164) AML, 48% (46/96) CLL, 22% (6/27) ALL, and 53% (14/26) MDS/MPN cases exhibiting an IC50 < 100nM. CG’806 demonstrated median IC50 values of 70nM and 140nM against primary AML and CLL cells, respectively. CG’806 also exerted potent picomolar to low-nanomolar IC50 anti-proliferative activity against human AML, B-ALL, mantle-cell lymphoma, Burkitt’s lymphoma, and diffuse large B-cell lymphoma cell lines. CG’806 in combination with OTX-015 demonstrated median IC50 values of 20nM and 40nM against primary AML and CLL cells, respectively. CG’806 in combination with venetoclax demonstrated median IC50 values of 20nM and 10nM against primary AML and CLL cells, respectively.

"Collaborating with OHSU and the Beat AML initiative has provided us an exceptional opportunity to explore the activity of CG‘806 against a large and diverse set of freshly isolated patient bone marrow samples from patients with AML, CLL and other hematologic malignancies," commented William G. Rice, Ph.D., Chairman and Chief Executive Officer of Aptose. "CG’806 continues to reveal compelling preclinical results that are superior to other FLT3 or BTK inhibitors, and we are eagerly preparing CG’806 for clinical studies and look forward to an IND submission in 2018."

Separately, Aptose and The University of Texas MD Anderson Cancer Center researchers also presented new data on CG’806 at ASH (Free ASH Whitepaper) (see press release here).

In addition to the abstracts that were presented at ASH (Free ASH Whitepaper), two additional abstracts on CG’806 and two abstracts on APTO-253, Aptose’s small molecule c-Myc Inhibitor, have been published on the ASH (Free ASH Whitepaper) abstracts site. All abstracts will become part of the permanent ASH (Free ASH Whitepaper) and Blood abstracts archive.

For more information on Beat AML refer to View Source

About CG’806

CG‘806 is an oral, first-in-class pan-FLT3/pan-BTK inhibitor. This small molecule demonstrates potent inhibition of all wild type and mutant forms of FLT3 tested (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates AML tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with FLT3-driven AML. Likewise, CG’806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinases operative in B cell malignancies, suggesting CG’806 may be developed for CLL and MCL patients that are resistant/refractory/intolerant to covalent BTK inhibitors.

On December 11, 2017 Kura Oncology, Inc. (Nasdaq:KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, today reported new findings supporting the development of lead candidate tipifarnib, a potent and selective inhibitor of farnesyl transferase, in the treatment of certain bone marrow cancers (Press release, Fate Therapeutics, DEC 11, 2017, View Source;p=RssLanding&cat=news&id=2322103 [SID1234522555]). These results were featured in presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Atlanta. Copies of the posters are now available on the company’s website at www.kuraoncology.com.

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Among the findings presented at ASH (Free ASH Whitepaper) were the identification of CXCR4/CXCR2 expression ratio and bone marrow homing of myeloid cells as potential biomarkers of tipifarnib activity across the bone marrow cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML), further showing that the CXCL12/CXCR4 pathway is a potential therapeutic target of farnesyl transferase inhibitors.

"Although tipifarnib has previously demonstrated clinical responses in certain patients with AML and MDS, no molecular mechanism of action was identified that could explain the activity of the drug candidate in those patient populations," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "These new findings are exciting because they may define potential biomarkers for tipifarnib in bone marrow tumors and characterize a subgroup of patients that are most likely to derive clinical benefit from a targeted therapy such as tipifarnib."

Previously, Kura Oncology reported preliminary results from an ongoing Phase 2 clinical trial of tipifarnib in patients with peripheral T-cell lymphoma (PTCL) identifying the CXCL12/CXCR4 pathway as a potential target of tipifarnib. Specifically, high levels of CXCL12 gene expression and absence of single nucleotide gene variations in the 3′-untranslated region of the CXCL12 gene were associated with observed clinical activity of tipifarnib in these PTCL patients.

In the ASH (Free ASH Whitepaper) presentation entitled, "The CXCL12/CXCR4 Pathway As a Potential Target of Tipifarnib: Preliminary Results from an Open-Label, Phase II Study in Relapsed or Refractory Peripheral T-Cell Lymphoma," Kura Oncology extends these observations and provides data supporting the observed tipifarnib-derived clinical benefit for the CXCL12-positive population.

CXCL12 is a chemokine that is secreted in large amounts by lymph nodes, bone marrow stroma, liver, and lung, and plays key roles in tumor invasion, bone marrow homing and site of metastasis. Among its multiple functions, CXCL12 is essential for homing of myeloid cells to the bone marrow and lymphoid cells to lymph nodes and other organs.

Based on its initial observations in PTCL, the company investigated a role for the CXCL12/CXCR4 pathway and bone marrow homing of myeloid cells as biomarkers of tipifarnib activity in AML and MDS studies.

In the ASH (Free ASH Whitepaper) presentation entitled, "The CXCL12/CXCR4 Pathway As a Potential Target of Tipifarnib in Acute Myeloid Leukemia and Myelodysplastic Syndromes," Kura Oncology presented results that identify the ratio of CXCR4/CXCR2 gene expression and bone marrow homing of myeloid cells as potential biomarkers of the activity of tipifarnib in certain bone marrow tumors. The results were obtained by analyzing data from previous studies of tipifarnib in AML and MDS, as well as data from the ongoing Phase 2 clinical trial of tipifarnib in CMML.

"We were very encouraged to identify CXCR4/CXCR2 expression ratio and bone marrow homing as markers of tipifarnib’s activity in MDS, AML and CMML," said Antonio Gualberto, M.D., Ph.D., Chief Medical Officer at Kura Oncology. "Although our analysis is retrospective, the fact that we observe a consistent clinical benefit across different endpoints, treatment settings and indications gives us increased confidence in the potential for these biomarkers. Based on our preliminary data, we believe CXCL12/CXCR4 may have the potential to unlock the therapeutic value of farnesyl transferase inhibition across multiple bone marrow neoplasias."

About Tipifarnib

Kura Oncology’s lead candidate, tipifarnib, is a potent and selective inhibitor of the enzyme farnesyl transferase, a key cell signaling process implicated in cancer initiation and development. Tipifarnib has previously been studied in more than 5,000 patients in more than 70 clinical trials has a well-established safety profile and has demonstrated compelling and durable anti-cancer activity in certain patient subsets.

Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, Kura Oncology is seeking to identify patients most likely to benefit from tipifarnib. The company is conducting clinical and preclinical studies in multiple disease indications where tipifarnib has previously shown signs of activity with the goal of identifying and validating biomarkers associated with the observed clinical activity of tipifarnib.

In September 2017, Kura Oncology reported that its Phase 2 trial of tipifarnib in patients with HRAS mutant head and neck squamous cell carcinomas (HNSCC) achieved its primary efficacy endpoint prior to the completion of patient enrollment. The company is now planning to initiate a registration-enabling study of tipifarnib in HRAS mutant HNSCC in 2018.

On December 11, 2017 Integra LifeSciences Holdings Corporation (NASDAQ: IART), a leading global medical technology company, reported that it will host an Investor Day meeting with analysts and institutional investors in New York City, beginning at 8:00 a.m. EST (Press release, Integra LifeSciences, DEC 11, 2017, View Source [SID1234522528]). Members of Integra’s executive leadership team will discuss the company’s financial performance and outlook.

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Highlights of today’s conference include:

•
Reaffirming 2017 financial guidance as provided on October 26, 2017, including:
o
Full-year 2017 revenue in the range of $1.165 billion to $1.175 billion, which includes about 4% organic growth
o
Full-year 2017 adjusted earnings per share in the range of $1.83 to $1.87
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Providing 2018 preliminary revenue estimate in the range of $1.46 billion to $1.48 billion, which includes about 5% organic growth
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Reaffirming 2018 adjusted earnings per share in the range of $2.25 to $2.35, consistent with preliminary estimates provided on October 26, 2017
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Establishing five-year financial targets of approximately $2 billion in revenue and an adjusted EBITDA margin range of 28% to 30%

The company will host a livestream of the presentation and conference materials are available through the Investors section of Integra’s website at www.integralife.com. A replay of the conference will be archived on the company website.

On December 11, 2017 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") reported the data of the investigational agent gilteritinib from the ongoing, open-label, dose escalation/expansion Phase 1 study (NCT02236013) in newly diagnosed patients with acute myeloid leukemia (AML) (Press release, Astellas, DEC 11, 2017, View Source [SID1234522548]). The data are being presented today in an oral presentation at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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"These initial data shed encouraging light on the safety and tolerability of gilteritinib when combined with intensive chemotherapy for newly diagnosed AML patients," said Keith W. Pratz, M.D., of John Hopkins Sidney Kimmel Comprehensive Cancer Center, who is the principal investigator for the study. "In addition, while evaluating antitumor effects is an exploratory goal, the response rates in FLT3mut+ patients are promising and warrant expanded investigation of gilteritinib in this upfront treatment setting. Continuing research to evaluate the potential role for a FLT3 inhibitor in newly diagnosed patients and other stages of AML should continue to be a priority in our collective efforts to improve outcomes for patients."

The primary objective of this Phase 1 study is to assess the safety/tolerability profile, including dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD), of gilteritinib when combined with 7+3 induction (cytarabine and idarubicin) and high-dose cytarabine (HiDAC) consolidation chemotherapy, followed by single agent maintenance therapy in patients 18 years of age and older who have been newly diagnosed with AML. Assessment of antitumor effects of this combination therapy is an exploratory objective.

The two-part trial first enrolled patients to successive cohorts to determine the MTD. Successive cohorts received gilteritinib doses of 40, 80 or 120 mg/day. Dose escalation decisions were made based on DLTs that occurred following the first dose of gilteritinib during induction. Patients in the dose expansion cohort received gilteritinib at the recommended expansion dose established during dose escalation. Patients also received gilteritinib during consolidation, and then received maintenance therapy with once-daily gilteritinib over a 28-day cycle for up to 26 cycles.

"We are very encouraged by this initial data from our ongoing study of gilteritinib in combination with intensive chemotherapy in newly diagnosed AML patients, and pleased that it earned selection for oral presentation at ASH (Free ASH Whitepaper)," said Steven Benner, M.D., senior vice president and global therapeutic area head, Oncology Development, Astellas. "Mutations of FLT3 in AML are associated with a poor prognosis across the course of disease treatment and, through our comprehensive clinical development program, Astellas is committed to understanding how selective inhibition by gilteritinib might be beneficial to as many patients as possible."

As of July 9, 2017, 50 patients (n=17, dose escalation cohort; n=33, dose expansion cohort) had been enrolled in this ongoing study and 49 had received at least one dose of gilteritinib. Of the 48 patients with documented FLT3 mutation status, 23 (47.9%) were FLT3mut+, of whom 13 (56.5%) had internal tandem duplications (ITD).

Additional key findings include:

During dose escalation, two subjects in the 40 mg/day cohort who had received gilteritinib on days 1-14 experienced DLTs (neutropenia, thrombocytopenia and decreased ejection fraction). After gilteritinib induction schedule modification, no additional DLTs were observed.
The maximum tolerated dose was not reached; gilteritinib 120 mg/day was chosen as the recommended expansion dose.
Grade ≥ 3 treatment-emergent adverse events (TEAEs) occurring in ≥ 10% of subjects were febrile neutropenia (36.7%), thrombocytopenia (18.4%), neutropenia (16.3%) and decreased platelet count (12.2%).
Serious drug-related TEAEs occurring in >1 subject were febrile neutropenia (n=8), sepsis (n=2), small intestinal obstruction (n=2), lung infection (n=2), and decreased ejection fraction (n=2).
In FLT3mut+ and FLT3 wild type subjects, end-of-treatment CRc rates were 100% and 60.9%, respectively.
About Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is a cancer that impacts the blood and bone marrow, and its incidence increases with age. The American Cancer Society estimates that in 2017, approximately 21,000 new patients will be diagnosed with AML in the United States and about 10,000 cases will result in death.

About Gilteritinib

Gilteritinib is an investigational compound that has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) as well as FLT3 tyrosine kinase domain (TKD), two common types of FLT3 mutations that are seen in approximately one-third of patients with AML. Further, gilteritinib has also demonstrated inhibition of the AXL receptor in AML cell lines, which has been reported to be associated with therapeutic resistance. Astellas is currently investigating gilteritinib in various AML patient populations through several additional Phase 3 trials. Visit d to learn more about ongoing gilteritinib clinical trials.

Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and potentially commercialize gilteritinib. Gilteritinib has been granted Orphan Drug designation and Fast Track designation by the U.S. FDA, and SAKIGAKE designation by the Japan Ministry of Health, Labor and Welfare.

The safety and efficacy of the agent discussed herein are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval and become commercially available for the uses being investigated. Information about pharmaceutical products (including products currently in development), which is included in this press release are not intended to constitute an advertisement or medical advice.

On December 11, 2017 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that preliminary safety and efficacy data from a phase 1 study of DS-3201, an investigational and potential first-in-class EZH1/2 dual inhibitor, in patients with relapsed or refractory non-Hodgkin lymphomas (NHLs) were presented during a poster session at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Atlanta, Georgia (Press release, Daiichi Sankyo, DEC 11, 2017, View Source [SID1234522553]).

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Preliminary exploratory efficacy results from an ongoing phase 1 dose escalation study showed that an overall response rate of 58.8 percent (10 of 17 patients) was observed with single agent DS-3201 in 17 evaluable patients with NHLs, including B-cell and T-cell lymphomas, who were relapsed from or refractory to standard treatment or for whom no standard treatment was available. Among the 10 patients with response, there were one complete remission and nine partial remissions. Additionally, four patients experienced stable disease and three patients experienced progressive disease.

An overall response rate of 45.5 percent (5 of 11 patients) was observed with DS-3201 in 11 evaluable patients with B-cell lymphomas, including follicular lymphoma (5 patients), diffuse large B-cell lymphoma (3 patients), extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (2 patients) and lymphoplasmacytic lymphoma (1 patient). An overall response rate of 83.3 percent (5 of 6 patients) was observed with DS-3201 in six evaluable patients with T-cell lymphomas, including peripheral T-cell lymphoma not otherwise specified (2 patients), angioimmunoblastic T-cell lymphoma (2 patients) and adult T-cell leukemia-lymphoma (2 patients).

"Based on these preliminary safety and efficacy data on DS-3201 in a clinical setting, further evaluation of DS-3201 is warranted," said Dai Maruyama, MD, PhD, Department of Hematology, National Cancer Center Hospital, Tokyo, Japan. "As the first dual inhibitor of EZH1 and EZH2 in clinical development, DS-3201 may represent a new epigenetic approach to treating blood cancers. We look forward to reviewing additional data as it becomes available to evaluate the potential of this approach."

Following observation of dose-limiting toxicities (DLTs) in three of 18 evaluable patients, dose expansion is ongoing to determine a conclusive recommended phase 2 dose. Four DLTs were observed in three patients who received either the 200 mg or 300 mg dose: there were three cases of temporary grade 4 platelet count decreases (one patient in the 200 mg cohort and two patients in the 300 mg cohort) and one case of grade 3 anemia requiring transfusion in a patient in the 300 mg cohort. Preliminary safety data from 18 evaluable patients in the study also were reported. The most common treatment emergent hematologic adverse events of any grade seen in all patients included decreased platelet count (77.8 percent), anemia (55.6 percent), decreased lymphocyte count (50.0 percent) and decreased neutrophil count (44.4 percent). The most common treatment emergent non-hematologic adverse events were dysgeusia (50.0 percent), alopecia (33.3 percent), diarrhea (22.2 percent), decreased appetite (22.2 percent), nasopharyngitis (22.2 percent), alanine aminotransferase increased (22.2 percent), rash (16.7 percent), aspartate aminotransferase increased (16.7 percent) and dry skin (16.7 percent). One serious adverse event of grade 3 pneumocystis jirovecii pneumonia (PJP) led to discontinuation from the study.There was one additional non-serious case of PJP observed, leading to the institution of prophylactic treatment for all subsequent patients enrolled into the study.

DS-3201 targets epigenetic regulation by inhibiting both the EZH1 (enhancer of zeste homolog 1) and EZH2 (enhancer of zeste homolog 2) enzymes, which may reactivate various genes that have been silenced by the protein H3K27me3.1 Reactivation of the silenced genes has been shown to result in decreased proliferation of EZH2-expressing cancer cells. Preclinical research has shown that DS-3201 suppressed trimethylation of H3K27 in cells (IC50: 0.55 nM) more potently than EZH2 selective inhibitors.1

"Targeting epigenetic regulation is an approach to treating cancer that aims to reverse aberrant epigenetic changes that contribute to cancer cell growth and to maintain normal gene expression. The dual inhibition of EZH1/2 is theoretically able to provide a different spectrum of activity compared to EZH2-specific inhibitors already in the clinic. Our phase 1 program is designed to address the question of the potential benefit for this dual mode of action," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "In addition to the phase 1 study in non-Hodgkin lymphomas, we also are evaluating targeting epigenetic regulation with DS-3201 in patients with acute myeloid leukemia and acute lymphocytic leukemia."

About Non-Hodgkin Lymphoma
Non-Hodgkin lymphoma (NHL) is a form of cancer that originates in lymphocytes, a type of white blood cell.2 The two main types of NHL are B-cell lymphomas and T-cell lymphomas, which are classified into subtypes based on the origin and stage of the cancer.2 There were an estimated 386,000 new cases and about 200,000 deaths globally from NHL in 2012.3 In Japan, there were nearly 21,000 new cases of NHL in 2012, accounting for around five percent of cases worldwide.3 While recent treatment advances have led to improved outcomes for patients with certain types of NHL, patients with aggressive NHL subtypes or relapsed or refractory disease still face a poor prognosis.2,4

About the DS-3201 Phase 1 Study
A multicenter, non-randomized, open-label phase 1 dose escalation trial in Japan is enrolling adult patients with non-Hodgkin lymphomas (NHL) who have relapsed from or are refractory to standard treatment or for whom no standard treatment is available. The primary objectives are to evaluate the safety and pharmacokinetics of multiple-dose monotherapy of DS-3201 and to determine the recommended phase 2 dose. Secondary objectives are to determine the maximum tolerated dose of DS-3201 and to conduct exploratory evaluations of DS-3201-related biomarkers and the efficacy of DS-3201. For more information about the clinical trial, visit ClinicalTrials.gov.

About DS-3201
Part of the AML Franchise of the Daiichi Sankyo Cancer Enterprise, DS-3201 is an investigational and potential first-in-class EZH1/2 dual inhibitor in phase 1 clinical development for hematologic cancers including acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and non-Hodgkin lymphoma (NHL). DS-3201 is an investigational agent that has not been approved by the FDA or any other regulatory agency worldwide as a treatment for any indication. Safety and efficacy have not been established.

About Daiichi Sankyo Cancer Enterprise
The vision of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking in order to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by our Antibody Drug Conjugate (ADC) and Acute Myeloid Leukemia (AML) Franchises, our cancer pipeline includes more than 20 small molecules, monoclonal antibodies and ADCs stemming from our powerful research engines: our two laboratories for biologic/immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in development include: quizartinib, an oral FLT3 inhibitor, for newly-diagnosed and relapsed or refractory AML with FLT3-ITD mutations; DS-8201, an ADC for HER2-expressing breast and gastric cancer, and other HER2-expressing solid tumors; and pexidartinib, an oral CSF-1R inhibitor, for tenosynovial giant cell tumor (TGCT), which is also being explored in a range of solid tumors in combination with the anti-PD1 immunotherapy pembrolizumab.