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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the development of medicines to control the expression of disease-driving genes, today announced that the Company will present new data on three of its clinical and preclinical programs at the American Association for Cancer Research (AACR) Annual Meeting taking place April 1-5 in Washington, D.C. The new data will be highlighted in five presentations: “The presentations at AACR showcase both the productivity of Syros’ gene control platform and the potential of our first-in-class programs to provide a meaningful benefit for patients with a range of aggressive cancers both as single agents and in combination with other targeted therapies,” said Nancy Simonian, M.D., Chief Executive Officer of Syros. “Our platform is the first focused solely on the regulatory genome to systematically identify and target disease-causing alterations in gene expression with the aim of treating diseases that have eluded other genomic-based approaches. In just three years since our inception, this pioneering approach has led to a robust and growing pipeline, with our lead program in a Phase 2 clinical trial, our second program poised to start clinical development in the first half of this year and multiple other programs in preclinical development. We are excited to be presenting data from multiple programs across all stages of our pipeline.” Details on the presentations are as follow: Date & Time: Monday, April 3, from 8 a.m. - 12 p.m. ET Presentation Title: AML patient clustering by super-enhancers reveals an RARA associated transcription factor signaling partner Session Category: Molecular and Cellular Biology / Genetics Session Title: Targeting Aberrant Transcription in Cancer Presenter: Michael R. McKeown, Ph.D., Senior Scientist, Translational Biology, Syros Abstract Number: 1511 Location: Walter E. Washington Convention Center, Halls A-C, Poster Section 20 Date & Time: Monday, April 3, from 8 a.m. - 12 p.m. ET Presentation Title: SY-1365, a potent and selective CDK7 inhibitor, exhibits promising anti-tumor activity in multiple preclinical models of aggressive solid tumors Session Category: Experimental and Molecular Therapeutics Session Title: New Targets 1 Presenter: Christian Fritz, Ph.D., Vice President, Biology, Syros Abstract Number: 1151 Location: Walter E. Washington Convention Center, Halls A-C, Poster Section 4 Date & Time: Monday, April 3, from 8 a.m. - 12 p.m. ET Presentation Title: Targeting the transcriptional kinases CDK12 and CDK13 in breast and ovarian cancer Session Category: Experimental and Molecular Therapeutics Session Title: New Targets 1 Presenter: Michael Bradley, Ph.D., Principal Scientist, Biochemistry & Biophysics, Syros Abstract Number: 1143 Location: Walter E. Washington Convention Center, Halls A-C, Poster Section 4 Date & Time: Monday, April 3, from 1 - 5 p.m. ET Presentation Title: SY-1425, a selective RARα agonist, induces high levels of CD38 expression in RARA-high AML tumors creating a susceptibility to anti-CD38 therapeutic antibody treatment Session Category: Immunology Session Title: Immune Response to Hematopoietic Tumors: New Development in Tumor Immunology Presenter: Kathryn Austgen, Ph.D., Senior Scientist, Immuno-Oncology, Syros Abstract Number: 2644 Location: Walter E. Washington Convention Center, Halls A-C, Poster Section 26 Date & Time: Tuesday, April 4, from 8 a.m. – 12 p.m. ET Presentation Title: SY-1425 (tamibarotene), a selective RARα agonist, shows synergistic anti-tumor activity with hypomethylating agents in a biomarker selected subset of AML Session Category: Experimental and Molecular Therapeutics Session Title: Differentiation Therapy Presenter: Michael R. McKeown, Ph.D., Senior Scientist, Translational Biology, Syros Abstract Number: 3085 Location: Walter E. Washington Convention Center, Halls A-C, Poster Section 3 About Syros Pharmaceuticals Syros Pharmaceuticals is pioneering the understanding of the non-coding region of the genome to advance a new wave of medicines that control expression of disease-driving genes. Syros has built a proprietary platform that is designed to systematically and efficiently analyze this unexploited region of DNA in human disease tissue to identify and drug novel targets linked to genomically defined patient populations. Because gene expression is fundamental to the function of all cells, Syros’ gene control platform has broad potential to create medicines that achieve profound and durable benefit across a range of diseases. Syros is currently focused on cancer and immune-mediated diseases and is advancing a growing pipeline of gene control medicines. Syros’ lead drug candidates are SY-1425, a selective RARα agonist in a Phase 2 clinical trial for genomically defined subsets of patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-1365, a selective CDK7 inhibitor with potential in a range of solid tumors and blood cancers. Led by a team with deep experience in drug discovery, development and commercialization, Syros is located in Cambridge, Mass. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including without limitation statements regarding the clinical progress of and potential benefits from treatment with SY-1425, the initiation of clinical development of SY-1365, the ability to advance preclinical programs, and the benefits of Syros’ gene control platform. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: Syros’ ability to: advance the development of its programs, including SY-1425 and SY-1365, under the timelines it projects ; obtain and maintain patent protection for its drug candidates and the freedom to operate under third party intellectual property; demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; replicate scientific and non-clinical data in clinical trials; successfully develop a companion diagnostic test to identify patients with biomarkers associated with the RARA super-enhancer; obtain and maintain necessary regulatory approvals; identify, enter into and maintain collaboration agreements with third parties; manage competition; manage expenses; raise the substantial additional capital needed to achieve its business objectives; attract and retain qualified personnel; and successfully execute on its business strategies; risks described under the caption “Risk Factors” in the company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which is on file with the Securities and Exchange Commission; and risks described in other filings that the company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Syros expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.


Late-Breaking Presentation Highlighting Interim Phase 2b Selinexor Data in Patients with Relapsed or Refractory DLBCL (SADAL Study) Overview of Key Selinexor Myeloma Data Also Featured at the 16th International Myeloma Workshop NEWTON, Mass., March 01, 2017 (GLOBE NEWSWIRE) -- Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today announced that 12 abstracts describing the Company's product candidates in development for hematological and solid tumor malignancies have been selected for presentation at the 2017 Annual Meeting of the American Association for Cancer Research (AACR) taking place April 1-5, 2017 in Washington, DC. The abstracts, which represent both company- and investigator-sponsored studies, describe data related to Karyopharm’s lead product candidate, selinexor (KPT-330), an oral Selective Inhibitor of Nuclear Export / SINE™ compound, as well as two of its promising Phase 1 oncology programs, KPT-8602, a second-generation oral SINE compound, and KPT-9274, a first-in-class oral dual inhibitor of PAK4 and NAMPT. “The ongoing randomized Phase 2b SADAL study, which was initiated based on encouraging Phase 1 data in patients with diffuse large B-cell lymphoma (DLBCL), was designed to evaluate the overall response rate of single-agent oral selinexor in patients with relapsed or refractory DLBCL,” said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm.  “We look forward to presenting interim results from this important trial at AACR this year.” Karyopharm is also presenting an overview of selinexor myeloma data at the 16th International Myeloma Workshop (IMW) held March 1-4, 2017 in New Delhi, India.  In an oral presentation, titled “Oral Selinexor Shows Single Agent Activity Enhanced with PI or IMiD Combinations in Refractory Multiple Myeloma,” (Abstract #234) Sagar Lonial, MD, FACP, Professor and Chair, Hematology and Medical Oncology, Emory University, provided an overview of clinical data demonstrating selinexor’s activity in combination with proteasome inhibitors (PIs) and immunomodulatory drugs (IMiDs) for the treatment of relapsed or refractory multiple myeloma.  The 2017 IMW is a prestigious biannual event where myeloma experts from around the world gather to discuss basic, preclinical and clinical aspects in the biology and treatment of multiple myeloma. Title: A Phase 2b randomized study of selinexor in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) demonstrates durable responses in both GCB and non-GCB subtypes Presenter: Marie Maerevoet, Institute Jules Bordet Poster Board #: 13 Session: Phase I-III Clinical Trials and Pediatric Clinical Trials Location: Convention Center, Halls A-C, Poster Section 33 Date and Time: Tuesday, April 4, 2017 from 1:00 PM - 5:00 PM Title: KPT-9274 inhibits cellular NAD and synergizes with doxorubicin to treat dogs with lymphoma Presenter: Cheryl London, Tufts University Poster Board #: 16 Session: Late-Breaking Research: Experimental and Molecular Therapeutics 2 Location: Convention Center, Halls A-C, Poster Section 34 Date and Time: Wednesday, April 5, 2017 8:00 AM - 12:00 PM Title: Selinexor or KPT-8602 mediated XPO1 inhibition synergizes with dexamethasone to repress convergent pathways in the mTORC1 signaling network and drive cell death in multiple myeloma Presenter: Christian Argueta, Karyopharm Therapeutics Inc. Poster Board #: 15 Session: Molecular and Cellular Biology/Genetics – Cell Growth Signaling Pathways 1 Location: Convention Center, Halls A-C, Poster Section 14 Date and Time: Sunday, April 2, 2017 1:00 PM - 5:00 PM Title: Novel role of XPO1 in regulating microRNAs related to pancreatic ductal adenocarcinoma invasion and metastasis Presenter: Asfar Azmi, Wayne State University Poster Board #: 5 Session: Molecular and Cellular Biology/Genetics – MicroRNA Regulation of Cancer Biology 1 Location: Convention Center, Halls A-C, Poster Section 19 Date and Time: Sunday, April 2, 2017 1:00 PM - 5:00 PM Title: Synergistic effects of the XPO1 inhibitor selinexor with proteasome inhibitors in pediatric high-grade glioma and diffuse intrinsic pontine glioma Presenter: John DeSisto, University of Colorado Denver Poster Board #: 18 Session: Tumor Biology:  Pediatric Cancer 1: Biomarkers, Preclinical Models, and New Targets Location: Convention Center, Halls A-C, Poster Section 42 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Anti-tumor activity of selinexor is enhanced by palbociclib in preclinical models of HER2+ breast cancer Presenter: Hua Chang, Karyopharm Therapeutics Inc. Poster Board #: 12 Session: Experimental and Molecular Therapeutics – Combination Therapy 1 Location: Convention Center, Halls A-C, Poster Section 2 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Disruption of nuclear export with selinexor or KPT-8602 reduces androgen receptor expression and leads to potent anti-tumor activity in preclinical models of androgen-independent prostate cancer Presenter: Christian Argueta, Karyopharm Therapeutics Inc. Poster Board #: 13 Session: Endocrinology – Prostate Cancer Biology and Therapy Location: Convention Center, Halls A-C, Poster Section 25 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: p21 activated kinase 4 (PAK4) as a novel therapeutic target for non-Hodgkin's lymphoma Presenter: Asfar Azmi, Wayne State University Poster Board #: 9 Session: Molecular and Cellular Biology/Genetics – Cell Growth Signaling Pathways 4 Location: Convention Center, Halls A-C, Poster Section 14 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Nuclear export of E2F7 in squamous cell carcinoma is an actionable event that reverses resistance to anthracyclines Presenter: Alba Natalia Saenz Ponce, University of Queensland, Brisbane, Australia Poster Board #: 28 Session: Experimental and Molecular Therapeutics: Reversal of Drug Resistance Location: Convention Center, Halls A-C, Poster Section 6 Date and Time: Monday, April 3, 2017 8:00 AM -­ 12:00 PM Title: Exportin-1 (XPO1) is a novel therapeutic biomarker for patients with neuroblastoma Presenter: Basia Galinski, Albert Einstein College of Medicine Poster Board #: 10 Session: Pediatric Cancer 1: Biomarkers, Preclinical Models, and New Targets Location: Convention Center, Halls A­C, Poster Section 42 Date and Time: Monday, April 3, 2017 8:00 AM -­ 12:00 PM Title: Combined targeting of estrogen receptor alpha and nuclear transport pathways remodel metabolic pathways to induce apoptosis and overcome tamoxifen resistance Presenter: Eylem Kulkoyluoglu-Cotul, University of Illinois Urbana-Champaign Poster Board #: 14 Session: Endocrinology: Nuclear Receptors and Endocrine Oncology Therapies Location: Convention Center, Halls A-C, Poster Section 25 Date and Time: Tuesday, April 4, 2017 8:00 AM - 12:00 PM Title: Selinexor synergizes with DNA damaging agents through down-regulation of key DNA damage response genes Presenter: Trinayan Kashyap, Karyopharm Therapeutics Inc. Poster Board #: 26 Session: Experimental and Molecular Therapeutics – New Targets and New Drugs Location: Convention Center, Halls A-C, Poster Section 5 Date and Time: Tuesday, April 4, 2017 1:00 PM - 5:00 PM Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 1,900 patients have been treated with selinexor and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Karyopharm plans to initiate a pivotal randomized Phase 3 study of selinexor in combination with bortezomib (Velcade®) and low-dose dexamethasone (BOSTON) in patients with multiple myeloma in early 2017. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform the Company's clinical development priorities for selinexor. The latest clinical trial information for selinexor is available at www.clinicaltrials.gov. Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a clinical-stage pharmaceutical company focused on the discovery and development of novel first-in-class drugs directed against nuclear transport and related targets for the treatment of cancer and other major diseases. Karyopharm's SINE™ compounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). In addition to single-agent and combination activity against a variety of human cancers, SINE™ compounds have also shown biological activity in models of neurodegeneration, inflammation, autoimmune disease, certain viruses and wound-healing. Karyopharm, which was founded by Dr. Sharon Shacham, currently has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com. This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, including the timing of initiation of certain trials and of the reporting of data from such trials. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company's current expectations. For example, there can be no guarantee that any of Karyopharm's SINE™ compounds, including selinexor (KPT-330), KPT-8602 and KPT-9274, will successfully complete necessary preclinical and clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical studies; Karyopharm's ability to obtain and maintain requisite regulatory approvals and to enroll patients in its clinical trials; unplanned cash requirements and expenditures; development of drug candidates by Karyopharm's competitors for diseases in which Karyopharm is currently developing its drug candidates; and Karyopharm's ability to obtain, maintain and enforce patent and other intellectual property protection for any drug candidates it is developing. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which was filed with the Securities and Exchange Commission (SEC) on November 7, 2016, and in other filings that Karyopharm may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


Late-Breaking Presentation Highlighting Interim Phase 2b Selinexor Data in Patients with Relapsed or Refractory DLBCL (SADAL Study) Overview of Key Selinexor Myeloma Data Also Featured at the 16th International Myeloma Workshop NEWTON, Mass., March 01, 2017 (GLOBE NEWSWIRE) -- Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today announced that 12 abstracts describing the Company's product candidates in development for hematological and solid tumor malignancies have been selected for presentation at the 2017 Annual Meeting of the American Association for Cancer Research (AACR) taking place April 1-5, 2017 in Washington, DC. The abstracts, which represent both company- and investigator-sponsored studies, describe data related to Karyopharm’s lead product candidate, selinexor (KPT-330), an oral Selective Inhibitor of Nuclear Export / SINE™ compound, as well as two of its promising Phase 1 oncology programs, KPT-8602, a second-generation oral SINE compound, and KPT-9274, a first-in-class oral dual inhibitor of PAK4 and NAMPT. “The ongoing randomized Phase 2b SADAL study, which was initiated based on encouraging Phase 1 data in patients with diffuse large B-cell lymphoma (DLBCL), was designed to evaluate the overall response rate of single-agent oral selinexor in patients with relapsed or refractory DLBCL,” said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm.  “We look forward to presenting interim results from this important trial at AACR this year.” Karyopharm is also presenting an overview of selinexor myeloma data at the 16th International Myeloma Workshop (IMW) held March 1-4, 2017 in New Delhi, India.  In an oral presentation, titled “Oral Selinexor Shows Single Agent Activity Enhanced with PI or IMiD Combinations in Refractory Multiple Myeloma,” (Abstract #234) Sagar Lonial, MD, FACP, Professor and Chair, Hematology and Medical Oncology, Emory University, provided an overview of clinical data demonstrating selinexor’s activity in combination with proteasome inhibitors (PIs) and immunomodulatory drugs (IMiDs) for the treatment of relapsed or refractory multiple myeloma.  The 2017 IMW is a prestigious biannual event where myeloma experts from around the world gather to discuss basic, preclinical and clinical aspects in the biology and treatment of multiple myeloma. Title: A Phase 2b randomized study of selinexor in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) demonstrates durable responses in both GCB and non-GCB subtypes Presenter: Marie Maerevoet, Institute Jules Bordet Poster Board #: 13 Session: Phase I-III Clinical Trials and Pediatric Clinical Trials Location: Convention Center, Halls A-C, Poster Section 33 Date and Time: Tuesday, April 4, 2017 from 1:00 PM - 5:00 PM Title: KPT-9274 inhibits cellular NAD and synergizes with doxorubicin to treat dogs with lymphoma Presenter: Cheryl London, Tufts University Poster Board #: 16 Session: Late-Breaking Research: Experimental and Molecular Therapeutics 2 Location: Convention Center, Halls A-C, Poster Section 34 Date and Time: Wednesday, April 5, 2017 8:00 AM - 12:00 PM Title: Selinexor or KPT-8602 mediated XPO1 inhibition synergizes with dexamethasone to repress convergent pathways in the mTORC1 signaling network and drive cell death in multiple myeloma Presenter: Christian Argueta, Karyopharm Therapeutics Inc. Poster Board #: 15 Session: Molecular and Cellular Biology/Genetics – Cell Growth Signaling Pathways 1 Location: Convention Center, Halls A-C, Poster Section 14 Date and Time: Sunday, April 2, 2017 1:00 PM - 5:00 PM Title: Novel role of XPO1 in regulating microRNAs related to pancreatic ductal adenocarcinoma invasion and metastasis Presenter: Asfar Azmi, Wayne State University Poster Board #: 5 Session: Molecular and Cellular Biology/Genetics – MicroRNA Regulation of Cancer Biology 1 Location: Convention Center, Halls A-C, Poster Section 19 Date and Time: Sunday, April 2, 2017 1:00 PM - 5:00 PM Title: Synergistic effects of the XPO1 inhibitor selinexor with proteasome inhibitors in pediatric high-grade glioma and diffuse intrinsic pontine glioma Presenter: John DeSisto, University of Colorado Denver Poster Board #: 18 Session: Tumor Biology:  Pediatric Cancer 1: Biomarkers, Preclinical Models, and New Targets Location: Convention Center, Halls A-C, Poster Section 42 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Anti-tumor activity of selinexor is enhanced by palbociclib in preclinical models of HER2+ breast cancer Presenter: Hua Chang, Karyopharm Therapeutics Inc. Poster Board #: 12 Session: Experimental and Molecular Therapeutics – Combination Therapy 1 Location: Convention Center, Halls A-C, Poster Section 2 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Disruption of nuclear export with selinexor or KPT-8602 reduces androgen receptor expression and leads to potent anti-tumor activity in preclinical models of androgen-independent prostate cancer Presenter: Christian Argueta, Karyopharm Therapeutics Inc. Poster Board #: 13 Session: Endocrinology – Prostate Cancer Biology and Therapy Location: Convention Center, Halls A-C, Poster Section 25 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: p21 activated kinase 4 (PAK4) as a novel therapeutic target for non-Hodgkin's lymphoma Presenter: Asfar Azmi, Wayne State University Poster Board #: 9 Session: Molecular and Cellular Biology/Genetics – Cell Growth Signaling Pathways 4 Location: Convention Center, Halls A-C, Poster Section 14 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Nuclear export of E2F7 in squamous cell carcinoma is an actionable event that reverses resistance to anthracyclines Presenter: Alba Natalia Saenz Ponce, University of Queensland, Brisbane, Australia Poster Board #: 28 Session: Experimental and Molecular Therapeutics: Reversal of Drug Resistance Location: Convention Center, Halls A-C, Poster Section 6 Date and Time: Monday, April 3, 2017 8:00 AM -­ 12:00 PM Title: Exportin-1 (XPO1) is a novel therapeutic biomarker for patients with neuroblastoma Presenter: Basia Galinski, Albert Einstein College of Medicine Poster Board #: 10 Session: Pediatric Cancer 1: Biomarkers, Preclinical Models, and New Targets Location: Convention Center, Halls A­C, Poster Section 42 Date and Time: Monday, April 3, 2017 8:00 AM -­ 12:00 PM Title: Combined targeting of estrogen receptor alpha and nuclear transport pathways remodel metabolic pathways to induce apoptosis and overcome tamoxifen resistance Presenter: Eylem Kulkoyluoglu-Cotul, University of Illinois Urbana-Champaign Poster Board #: 14 Session: Endocrinology: Nuclear Receptors and Endocrine Oncology Therapies Location: Convention Center, Halls A-C, Poster Section 25 Date and Time: Tuesday, April 4, 2017 8:00 AM - 12:00 PM Title: Selinexor synergizes with DNA damaging agents through down-regulation of key DNA damage response genes Presenter: Trinayan Kashyap, Karyopharm Therapeutics Inc. Poster Board #: 26 Session: Experimental and Molecular Therapeutics – New Targets and New Drugs Location: Convention Center, Halls A-C, Poster Section 5 Date and Time: Tuesday, April 4, 2017 1:00 PM - 5:00 PM Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 1,900 patients have been treated with selinexor and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Karyopharm plans to initiate a pivotal randomized Phase 3 study of selinexor in combination with bortezomib (Velcade®) and low-dose dexamethasone (BOSTON) in patients with multiple myeloma in early 2017. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform the Company's clinical development priorities for selinexor. The latest clinical trial information for selinexor is available at www.clinicaltrials.gov. Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a clinical-stage pharmaceutical company focused on the discovery and development of novel first-in-class drugs directed against nuclear transport and related targets for the treatment of cancer and other major diseases. Karyopharm's SINE™ compounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). In addition to single-agent and combination activity against a variety of human cancers, SINE™ compounds have also shown biological activity in models of neurodegeneration, inflammation, autoimmune disease, certain viruses and wound-healing. Karyopharm, which was founded by Dr. Sharon Shacham, currently has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com. This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, including the timing of initiation of certain trials and of the reporting of data from such trials. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company's current expectations. For example, there can be no guarantee that any of Karyopharm's SINE™ compounds, including selinexor (KPT-330), KPT-8602 and KPT-9274, will successfully complete necessary preclinical and clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical studies; Karyopharm's ability to obtain and maintain requisite regulatory approvals and to enroll patients in its clinical trials; unplanned cash requirements and expenditures; development of drug candidates by Karyopharm's competitors for diseases in which Karyopharm is currently developing its drug candidates; and Karyopharm's ability to obtain, maintain and enforce patent and other intellectual property protection for any drug candidates it is developing. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which was filed with the Securities and Exchange Commission (SEC) on November 7, 2016, and in other filings that Karyopharm may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


Late-Breaking Presentation Highlighting Interim Phase 2b Selinexor Data in Patients with Relapsed or Refractory DLBCL (SADAL Study) Overview of Key Selinexor Myeloma Data Also Featured at the 16th International Myeloma Workshop NEWTON, Mass., March 01, 2017 (GLOBE NEWSWIRE) -- Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today announced that 12 abstracts describing the Company's product candidates in development for hematological and solid tumor malignancies have been selected for presentation at the 2017 Annual Meeting of the American Association for Cancer Research (AACR) taking place April 1-5, 2017 in Washington, DC. The abstracts, which represent both company- and investigator-sponsored studies, describe data related to Karyopharm’s lead product candidate, selinexor (KPT-330), an oral Selective Inhibitor of Nuclear Export / SINE™ compound, as well as two of its promising Phase 1 oncology programs, KPT-8602, a second-generation oral SINE compound, and KPT-9274, a first-in-class oral dual inhibitor of PAK4 and NAMPT. “The ongoing randomized Phase 2b SADAL study, which was initiated based on encouraging Phase 1 data in patients with diffuse large B-cell lymphoma (DLBCL), was designed to evaluate the overall response rate of single-agent oral selinexor in patients with relapsed or refractory DLBCL,” said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm.  “We look forward to presenting interim results from this important trial at AACR this year.” Karyopharm is also presenting an overview of selinexor myeloma data at the 16th International Myeloma Workshop (IMW) held March 1-4, 2017 in New Delhi, India.  In an oral presentation, titled “Oral Selinexor Shows Single Agent Activity Enhanced with PI or IMiD Combinations in Refractory Multiple Myeloma,” (Abstract #234) Sagar Lonial, MD, FACP, Professor and Chair, Hematology and Medical Oncology, Emory University, provided an overview of clinical data demonstrating selinexor’s activity in combination with proteasome inhibitors (PIs) and immunomodulatory drugs (IMiDs) for the treatment of relapsed or refractory multiple myeloma.  The 2017 IMW is a prestigious biannual event where myeloma experts from around the world gather to discuss basic, preclinical and clinical aspects in the biology and treatment of multiple myeloma. Title: A Phase 2b randomized study of selinexor in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) demonstrates durable responses in both GCB and non-GCB subtypes Presenter: Marie Maerevoet, Institute Jules Bordet Poster Board #: 13 Session: Phase I-III Clinical Trials and Pediatric Clinical Trials Location: Convention Center, Halls A-C, Poster Section 33 Date and Time: Tuesday, April 4, 2017 from 1:00 PM - 5:00 PM Title: KPT-9274 inhibits cellular NAD and synergizes with doxorubicin to treat dogs with lymphoma Presenter: Cheryl London, Tufts University Poster Board #: 16 Session: Late-Breaking Research: Experimental and Molecular Therapeutics 2 Location: Convention Center, Halls A-C, Poster Section 34 Date and Time: Wednesday, April 5, 2017 8:00 AM - 12:00 PM Title: Selinexor or KPT-8602 mediated XPO1 inhibition synergizes with dexamethasone to repress convergent pathways in the mTORC1 signaling network and drive cell death in multiple myeloma Presenter: Christian Argueta, Karyopharm Therapeutics Inc. Poster Board #: 15 Session: Molecular and Cellular Biology/Genetics – Cell Growth Signaling Pathways 1 Location: Convention Center, Halls A-C, Poster Section 14 Date and Time: Sunday, April 2, 2017 1:00 PM - 5:00 PM Title: Novel role of XPO1 in regulating microRNAs related to pancreatic ductal adenocarcinoma invasion and metastasis Presenter: Asfar Azmi, Wayne State University Poster Board #: 5 Session: Molecular and Cellular Biology/Genetics – MicroRNA Regulation of Cancer Biology 1 Location: Convention Center, Halls A-C, Poster Section 19 Date and Time: Sunday, April 2, 2017 1:00 PM - 5:00 PM Title: Synergistic effects of the XPO1 inhibitor selinexor with proteasome inhibitors in pediatric high-grade glioma and diffuse intrinsic pontine glioma Presenter: John DeSisto, University of Colorado Denver Poster Board #: 18 Session: Tumor Biology:  Pediatric Cancer 1: Biomarkers, Preclinical Models, and New Targets Location: Convention Center, Halls A-C, Poster Section 42 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Anti-tumor activity of selinexor is enhanced by palbociclib in preclinical models of HER2+ breast cancer Presenter: Hua Chang, Karyopharm Therapeutics Inc. Poster Board #: 12 Session: Experimental and Molecular Therapeutics – Combination Therapy 1 Location: Convention Center, Halls A-C, Poster Section 2 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Disruption of nuclear export with selinexor or KPT-8602 reduces androgen receptor expression and leads to potent anti-tumor activity in preclinical models of androgen-independent prostate cancer Presenter: Christian Argueta, Karyopharm Therapeutics Inc. Poster Board #: 13 Session: Endocrinology – Prostate Cancer Biology and Therapy Location: Convention Center, Halls A-C, Poster Section 25 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: p21 activated kinase 4 (PAK4) as a novel therapeutic target for non-Hodgkin's lymphoma Presenter: Asfar Azmi, Wayne State University Poster Board #: 9 Session: Molecular and Cellular Biology/Genetics – Cell Growth Signaling Pathways 4 Location: Convention Center, Halls A-C, Poster Section 14 Date and Time: Monday, April 3, 2017 8:00 AM - 12:00 PM Title: Nuclear export of E2F7 in squamous cell carcinoma is an actionable event that reverses resistance to anthracyclines Presenter: Alba Natalia Saenz Ponce, University of Queensland, Brisbane, Australia Poster Board #: 28 Session: Experimental and Molecular Therapeutics: Reversal of Drug Resistance Location: Convention Center, Halls A-C, Poster Section 6 Date and Time: Monday, April 3, 2017 8:00 AM -­ 12:00 PM Title: Exportin-1 (XPO1) is a novel therapeutic biomarker for patients with neuroblastoma Presenter: Basia Galinski, Albert Einstein College of Medicine Poster Board #: 10 Session: Pediatric Cancer 1: Biomarkers, Preclinical Models, and New Targets Location: Convention Center, Halls A­C, Poster Section 42 Date and Time: Monday, April 3, 2017 8:00 AM -­ 12:00 PM Title: Combined targeting of estrogen receptor alpha and nuclear transport pathways remodel metabolic pathways to induce apoptosis and overcome tamoxifen resistance Presenter: Eylem Kulkoyluoglu-Cotul, University of Illinois Urbana-Champaign Poster Board #: 14 Session: Endocrinology: Nuclear Receptors and Endocrine Oncology Therapies Location: Convention Center, Halls A-C, Poster Section 25 Date and Time: Tuesday, April 4, 2017 8:00 AM - 12:00 PM Title: Selinexor synergizes with DNA damaging agents through down-regulation of key DNA damage response genes Presenter: Trinayan Kashyap, Karyopharm Therapeutics Inc. Poster Board #: 26 Session: Experimental and Molecular Therapeutics – New Targets and New Drugs Location: Convention Center, Halls A-C, Poster Section 5 Date and Time: Tuesday, April 4, 2017 1:00 PM - 5:00 PM Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 1,900 patients have been treated with selinexor and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Karyopharm plans to initiate a pivotal randomized Phase 3 study of selinexor in combination with bortezomib (Velcade®) and low-dose dexamethasone (BOSTON) in patients with multiple myeloma in early 2017. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform the Company's clinical development priorities for selinexor. The latest clinical trial information for selinexor is available at www.clinicaltrials.gov. Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a clinical-stage pharmaceutical company focused on the discovery and development of novel first-in-class drugs directed against nuclear transport and related targets for the treatment of cancer and other major diseases. Karyopharm's SINE™ compounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). In addition to single-agent and combination activity against a variety of human cancers, SINE™ compounds have also shown biological activity in models of neurodegeneration, inflammation, autoimmune disease, certain viruses and wound-healing. Karyopharm, which was founded by Dr. Sharon Shacham, currently has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com. This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, including the timing of initiation of certain trials and of the reporting of data from such trials. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company's current expectations. For example, there can be no guarantee that any of Karyopharm's SINE™ compounds, including selinexor (KPT-330), KPT-8602 and KPT-9274, will successfully complete necessary preclinical and clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical studies; Karyopharm's ability to obtain and maintain requisite regulatory approvals and to enroll patients in its clinical trials; unplanned cash requirements and expenditures; development of drug candidates by Karyopharm's competitors for diseases in which Karyopharm is currently developing its drug candidates; and Karyopharm's ability to obtain, maintain and enforce patent and other intellectual property protection for any drug candidates it is developing. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which was filed with the Securities and Exchange Commission (SEC) on November 7, 2016, and in other filings that Karyopharm may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.


News Article | February 27, 2017
Site: globenewswire.com

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Achaogen, Inc. (NASDAQ:AKAO), a clinical-stage biopharmaceutical company discovering and developing novel antibacterials addressing multi-drug resistant (MDR) gram-negative infections, today announced the addition of Ms. Janet Dorling as Chief Commercial Officer effective today. Ms. Dorling will report to Blake Wise, Achaogen’s President and Chief Operating Officer. “We are very pleased to welcome Janet to the Achaogen team at this pivotal time, as we pursue regulatory approval of our lead product candidate, plazomicin, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae,” said Mr. Wise. “Janet is a highly effective commercial strategist with proven experience in driving global commercial success, including hands-on experience leading a large hospital-based products business in the U.S. With her proven ability to build and lead cross-functional teams, Janet’s appointment to this role fortifies our management team and underscores Achaogen’s commitment to commercialize new treatment options for serious MDR gram-negative infections.” Ms. Dorling brings to Achaogen more than 14 years of experience in commercial sales and marketing of pharmaceutical products. Most recently, Ms. Dorling was the Vice President of Global Product Strategy, Breast Cancer at Roche/Genentech. In this role, Ms. Dorling was responsible for developing and implementing global commercialization and lifecycle strategies for the Breast Cancer Franchise. Previously, as Vice President of the Lytics Franchise, Ms. Dorling led hospital-focused Sales and Marketing teams that delivered double-digit revenue growth to achieve over one billion dollars in revenue in the acute care setting. Ms. Dorling has also led the Market Analysis and Strategy group at Genentech with portfolio-wide responsibility. Ms. Dorling has a Bachelor of Arts in Molecular and Cellular Biology from the University of California at Berkeley, a Master’s in Science in Pharmacology and Cancer Biology from Duke University, and a Master’s in Business Administration from the Haas School of Business at the University of California at Berkeley. “The CDC has characterized carbapenem-resistant Enterobacteriaceae as ‘nightmare bacteria’ and an immediate public health threat and, unfortunately, patients with serious MDR gram-negative infections have limited therapeutic options,” said Ms. Dorling. “I look forward to joining the committed and passionate team at Achaogen to deliver on the Company’s commercial objectives for plazomicin and to contribute to advancing an exciting pipeline.” About Achaogen Achaogen is a clinical-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of novel antibacterial treatments for MDR gram-negative infections. Achaogen is developing plazomicin, Achaogen’s lead product candidate, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae. Achaogen’s plazomicin program is funded in part with a contract from the Biomedical Advanced Research and Development Authority. Plazomicin is the first clinical candidate from Achaogen’s gram-negative antibiotic discovery engine, and Achaogen has other programs in early and late preclinical stages focused on other MDR gram-negative infections. All product candidates are investigational only and have not been approved for commercialization.  For more information, please visit www.achaogen.com. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, Achaogen’s expectations regarding potential regulatory approval of plazomicin, Achaogen’s commercial objectives and Achaogen’s pipeline of product candidates. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause Achaogen's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the preclinical and clinical development process; the risks and uncertainties of the regulatory approval process; the risks and uncertainties of commercialization and gaining market acceptance; the risk when bacteria will evolve resistance to plazomicin; Achaogen's reliance on third-party contract manufacturing organizations to manufacture and supply its product candidates and certain raw materials used in the production thereof; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Achaogen's patents or proprietary rights; and the risk that Achaogen's proprietary rights may be insufficient to protect its technologies and product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward- looking statements, as well as risks relating to Achaogen's business in general, see Achaogen's current and future reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and its Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Achaogen does not plan to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.


News Article | February 27, 2017
Site: globenewswire.com

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Achaogen, Inc. (NASDAQ:AKAO), a clinical-stage biopharmaceutical company discovering and developing novel antibacterials addressing multi-drug resistant (MDR) gram-negative infections, today announced the addition of Ms. Janet Dorling as Chief Commercial Officer effective today. Ms. Dorling will report to Blake Wise, Achaogen’s President and Chief Operating Officer. “We are very pleased to welcome Janet to the Achaogen team at this pivotal time, as we pursue regulatory approval of our lead product candidate, plazomicin, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae,” said Mr. Wise. “Janet is a highly effective commercial strategist with proven experience in driving global commercial success, including hands-on experience leading a large hospital-based products business in the U.S. With her proven ability to build and lead cross-functional teams, Janet’s appointment to this role fortifies our management team and underscores Achaogen’s commitment to commercialize new treatment options for serious MDR gram-negative infections.” Ms. Dorling brings to Achaogen more than 14 years of experience in commercial sales and marketing of pharmaceutical products. Most recently, Ms. Dorling was the Vice President of Global Product Strategy, Breast Cancer at Roche/Genentech. In this role, Ms. Dorling was responsible for developing and implementing global commercialization and lifecycle strategies for the Breast Cancer Franchise. Previously, as Vice President of the Lytics Franchise, Ms. Dorling led hospital-focused Sales and Marketing teams that delivered double-digit revenue growth to achieve over one billion dollars in revenue in the acute care setting. Ms. Dorling has also led the Market Analysis and Strategy group at Genentech with portfolio-wide responsibility. Ms. Dorling has a Bachelor of Arts in Molecular and Cellular Biology from the University of California at Berkeley, a Master’s in Science in Pharmacology and Cancer Biology from Duke University, and a Master’s in Business Administration from the Haas School of Business at the University of California at Berkeley. “The CDC has characterized carbapenem-resistant Enterobacteriaceae as ‘nightmare bacteria’ and an immediate public health threat and, unfortunately, patients with serious MDR gram-negative infections have limited therapeutic options,” said Ms. Dorling. “I look forward to joining the committed and passionate team at Achaogen to deliver on the Company’s commercial objectives for plazomicin and to contribute to advancing an exciting pipeline.” About Achaogen Achaogen is a clinical-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of novel antibacterial treatments for MDR gram-negative infections. Achaogen is developing plazomicin, Achaogen’s lead product candidate, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae. Achaogen’s plazomicin program is funded in part with a contract from the Biomedical Advanced Research and Development Authority. Plazomicin is the first clinical candidate from Achaogen’s gram-negative antibiotic discovery engine, and Achaogen has other programs in early and late preclinical stages focused on other MDR gram-negative infections. All product candidates are investigational only and have not been approved for commercialization.  For more information, please visit www.achaogen.com. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, Achaogen’s expectations regarding potential regulatory approval of plazomicin, Achaogen’s commercial objectives and Achaogen’s pipeline of product candidates. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause Achaogen's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the preclinical and clinical development process; the risks and uncertainties of the regulatory approval process; the risks and uncertainties of commercialization and gaining market acceptance; the risk when bacteria will evolve resistance to plazomicin; Achaogen's reliance on third-party contract manufacturing organizations to manufacture and supply its product candidates and certain raw materials used in the production thereof; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Achaogen's patents or proprietary rights; and the risk that Achaogen's proprietary rights may be insufficient to protect its technologies and product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward- looking statements, as well as risks relating to Achaogen's business in general, see Achaogen's current and future reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and its Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Achaogen does not plan to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.


Genes common to both the human T-cell leukemia virus and high-risk human papillomaviruses activate survival mechanisms in cancer cells. An SMU lab, with National Cancer Institute funding, is hunting ways to inhibit those genes to halt the development of c SMU virologist and cancer researcher Robert L. Harrod has been awarded a $436,500 grant from the National Cancer Institute to further his lab's research into how certain viruses cause cancers in humans. Under two previous NCI grants, Harrod's lab discovered that the human T-cell leukemia virus type-1, HTLV-1, and high-risk subtype human papillomaviruses, HPVs, share a common mechanism that plays a key role in allowing cancers to develop. Now the lab will search for the biological mechanism -- a molecular target -- to intervene to block establishment and progression of virus-induced cancers. The hope is to ultimately develop a chemotherapy drug to block the growth of those tumor cells in patients. "The general theme of our lab is understanding the key molecular events involved in how the viruses allow cancer to develop," said Harrod, an associate professor in SMU's Department of Biological Sciences whose research focuses on understanding the molecular basis of viral initiation of cancer formation. While HTLV-1 and HPV are unrelated transforming viruses and lead to very different types of cancers, they've evolved a similar mechanism to cooperate with genes that cause cancer in different cell types. The lab discovered that the two viruses tap a common protein that cooperates with cellular genes to help the viruses hide from the immune system. That common protein, the p30 protein of HTLV-1, binds to a different protein in the cell, p53, which normally has the job of suppressing cancerous growth or tumor development. Instead, however, p30 manages to subvert p53's tumor suppressor functions, which in turn activates pro-survival pathways for the virus. From there, the virus can hide inside the infected cell for two to three decades while evading host immune-surveillance pathways. As the cell divides, the virus divides and replicates. Then ultimately the deregulation of gene expression by viral encoded products causes cancer to develop. "They are essentially using a similar mechanism, p30, to deregulate those pathways from their normal tumor-suppressing function," Harrod said. About 15 percent to 20 percent of all cancers are virus related. Worldwide, about 10 million people are infected with HTLV-1 and, as with other viral-induced cancers, about 3 percent to 5 percent of those infected go on to develop malignant disease. Cancer is often associated with the process of normal aging, because our tumor suppression and DNA damage-repair pathways begin to break down and fail, explained Harrod. Our pathways don't as easily repair genetic mutations, which makes us more susceptible to cancers like adult T-cell leukemia and HPV-associated cervical cancers or head-and-neck carcinomas, he said. The human T-cell leukemia virus is transmitted through blood and body fluid contact, usually infecting infants and children via breastfeeding from their mother. A tropical infectious disease, it's endemic to Southeast Asia, primarily Japan, Taiwan, China and Malaysia, as well as certain regions in the Middle East, Northern Africa and islands of the Caribbean. In the United States, Hawaii and Florida have the highest incidence of adult T-cell leukemia. HTLV-1 is highly resistant to most modern anticancer therapies, including radiotherapy and bone marrow or matching donor stem cell transplants. The life expectancy of patients with acute or lymphoma-stage disease is about six months to two years after diagnosis. In the case of HPV, certain high-risk sub-types aren't inhibited by today's available HPV vaccines. It's considered the high-risk HPVs are sexually transmitted through direct contact with the tissues of the virus-producing papillomas or warts. High-risk HPVs can also cause cervical cancers and head and neck carcinomas, many of which are associated with poor clinical outcomes and have high mortality rates. How do viruses cause cancer? For both HTLV-1 and HPV, the virus itself does not cause cancer to develop. "It's cooperating with oncogenes -- cellular genes that become deregulated and have the potential to cause cancer," Harrod said. "The role of these viruses, it seems, is to induce the proliferation of the cell affected with cancer. We're trying to understand some of the molecular events that are associated with these cancers. " The lab's three-year NCI grant runs through 2019. Harrod's two previous grants awarded by the National Institutes of Health were also three-year-grants, for $435,000 and $162,000. Each one has targeted HTLV-1 and the p30 protein. The lab's first NCI grant came after the researchers provided the first demonstration that p30 could cooperate with cellular oncogenes, which have the potential to cause cancer, to cause deregulated cell growth leading to normal cells transforming into cancer cells. That original discovery was reported in 2005 in the article "A human T-cell lymphotropic virus type 1 enhancer of Myc transforming potential stabilizes Myc-TIP60 transcriptional interactions," in the high-profile journal Molecular and Cellular Biology. "We find that the p30 protein is involved in maintaining the latency of these viruses. These viruses have to persist in the body for 20 to 40 years before a person develops disease. To do that they have to hide from the immune response," Harrod explained. "So p30 plays a role in silencing the viral genome so that the affected cells can hide, but at the same time it induces replication of the affected cells. So when the cell divides, the virus divides. We call that pro-viral replication." The term "latency maintenance factor" in reference to p30 originated with Harrod's lab and has gained traction in the HTLV-1 field. Under the lab's second NCI grant, the researchers figured out how to block pro-survival pathways to kill tumor cells. In the current grant proposal, Harrod's lab demonstrated that by inhibiting specific downstream targets of p53 -- essentially blocking pathways regulated by the p53 protein -- they could cause infected tumor cells to collapse on themselves and undergo cell death. "We do that independent of chemotherapy," Harrod said. "So that was a big find for us." Goal is to eliminate cancer cells by inhibiting pathway Each grant project builds upon the one before it, and the third grant extends the work, to now include high-risk HPVs. "Now that we've shown we can block one or two of these factors to cause cell death, we're starting to get an eye really on how we can inhibit these cancer cells and what potentially down the road may lead to a therapeutic," Harrod said. "That's the ultimate goal." One of the biggest challenges will be to inhibit the pathways in the tumor cells without targeting normal cells, he said. The lab's recent findings indicate the researchers may soon be within reach of identifying a new strategy to eliminate cancer cells by inhibiting pathways key to their survival. Harrod's lab collaborates on the research with: Lawrence Banks, Tumor Virology Group Leader, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy; Brenda Hernandez, Associate Director, Hawaii Tumor Registry, University of Hawaii Cancer Center, Honolulu; and Patrick Green, Director, Center for Retrovirus Research, The Ohio State University.


News Article | February 27, 2017
Site: globenewswire.com

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Achaogen, Inc. (NASDAQ:AKAO), a clinical-stage biopharmaceutical company discovering and developing novel antibacterials addressing multi-drug resistant (MDR) gram-negative infections, today announced the addition of Ms. Janet Dorling as Chief Commercial Officer effective today. Ms. Dorling will report to Blake Wise, Achaogen’s President and Chief Operating Officer. “We are very pleased to welcome Janet to the Achaogen team at this pivotal time, as we pursue regulatory approval of our lead product candidate, plazomicin, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae,” said Mr. Wise. “Janet is a highly effective commercial strategist with proven experience in driving global commercial success, including hands-on experience leading a large hospital-based products business in the U.S. With her proven ability to build and lead cross-functional teams, Janet’s appointment to this role fortifies our management team and underscores Achaogen’s commitment to commercialize new treatment options for serious MDR gram-negative infections.” Ms. Dorling brings to Achaogen more than 14 years of experience in commercial sales and marketing of pharmaceutical products. Most recently, Ms. Dorling was the Vice President of Global Product Strategy, Breast Cancer at Roche/Genentech. In this role, Ms. Dorling was responsible for developing and implementing global commercialization and lifecycle strategies for the Breast Cancer Franchise. Previously, as Vice President of the Lytics Franchise, Ms. Dorling led hospital-focused Sales and Marketing teams that delivered double-digit revenue growth to achieve over one billion dollars in revenue in the acute care setting. Ms. Dorling has also led the Market Analysis and Strategy group at Genentech with portfolio-wide responsibility. Ms. Dorling has a Bachelor of Arts in Molecular and Cellular Biology from the University of California at Berkeley, a Master’s in Science in Pharmacology and Cancer Biology from Duke University, and a Master’s in Business Administration from the Haas School of Business at the University of California at Berkeley. “The CDC has characterized carbapenem-resistant Enterobacteriaceae as ‘nightmare bacteria’ and an immediate public health threat and, unfortunately, patients with serious MDR gram-negative infections have limited therapeutic options,” said Ms. Dorling. “I look forward to joining the committed and passionate team at Achaogen to deliver on the Company’s commercial objectives for plazomicin and to contribute to advancing an exciting pipeline.” About Achaogen Achaogen is a clinical-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of novel antibacterial treatments for MDR gram-negative infections. Achaogen is developing plazomicin, Achaogen’s lead product candidate, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae. Achaogen’s plazomicin program is funded in part with a contract from the Biomedical Advanced Research and Development Authority. Plazomicin is the first clinical candidate from Achaogen’s gram-negative antibiotic discovery engine, and Achaogen has other programs in early and late preclinical stages focused on other MDR gram-negative infections. All product candidates are investigational only and have not been approved for commercialization.  For more information, please visit www.achaogen.com. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, Achaogen’s expectations regarding potential regulatory approval of plazomicin, Achaogen’s commercial objectives and Achaogen’s pipeline of product candidates. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause Achaogen's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the preclinical and clinical development process; the risks and uncertainties of the regulatory approval process; the risks and uncertainties of commercialization and gaining market acceptance; the risk when bacteria will evolve resistance to plazomicin; Achaogen's reliance on third-party contract manufacturing organizations to manufacture and supply its product candidates and certain raw materials used in the production thereof; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Achaogen's patents or proprietary rights; and the risk that Achaogen's proprietary rights may be insufficient to protect its technologies and product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward- looking statements, as well as risks relating to Achaogen's business in general, see Achaogen's current and future reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and its Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Achaogen does not plan to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.


News Article | February 27, 2017
Site: globenewswire.com

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Achaogen, Inc. (NASDAQ:AKAO), a clinical-stage biopharmaceutical company discovering and developing novel antibacterials addressing multi-drug resistant (MDR) gram-negative infections, today announced the addition of Ms. Janet Dorling as Chief Commercial Officer effective today. Ms. Dorling will report to Blake Wise, Achaogen’s President and Chief Operating Officer. “We are very pleased to welcome Janet to the Achaogen team at this pivotal time, as we pursue regulatory approval of our lead product candidate, plazomicin, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae,” said Mr. Wise. “Janet is a highly effective commercial strategist with proven experience in driving global commercial success, including hands-on experience leading a large hospital-based products business in the U.S. With her proven ability to build and lead cross-functional teams, Janet’s appointment to this role fortifies our management team and underscores Achaogen’s commitment to commercialize new treatment options for serious MDR gram-negative infections.” Ms. Dorling brings to Achaogen more than 14 years of experience in commercial sales and marketing of pharmaceutical products. Most recently, Ms. Dorling was the Vice President of Global Product Strategy, Breast Cancer at Roche/Genentech. In this role, Ms. Dorling was responsible for developing and implementing global commercialization and lifecycle strategies for the Breast Cancer Franchise. Previously, as Vice President of the Lytics Franchise, Ms. Dorling led hospital-focused Sales and Marketing teams that delivered double-digit revenue growth to achieve over one billion dollars in revenue in the acute care setting. Ms. Dorling has also led the Market Analysis and Strategy group at Genentech with portfolio-wide responsibility. Ms. Dorling has a Bachelor of Arts in Molecular and Cellular Biology from the University of California at Berkeley, a Master’s in Science in Pharmacology and Cancer Biology from Duke University, and a Master’s in Business Administration from the Haas School of Business at the University of California at Berkeley. “The CDC has characterized carbapenem-resistant Enterobacteriaceae as ‘nightmare bacteria’ and an immediate public health threat and, unfortunately, patients with serious MDR gram-negative infections have limited therapeutic options,” said Ms. Dorling. “I look forward to joining the committed and passionate team at Achaogen to deliver on the Company’s commercial objectives for plazomicin and to contribute to advancing an exciting pipeline.” About Achaogen Achaogen is a clinical-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of novel antibacterial treatments for MDR gram-negative infections. Achaogen is developing plazomicin, Achaogen’s lead product candidate, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae. Achaogen’s plazomicin program is funded in part with a contract from the Biomedical Advanced Research and Development Authority. Plazomicin is the first clinical candidate from Achaogen’s gram-negative antibiotic discovery engine, and Achaogen has other programs in early and late preclinical stages focused on other MDR gram-negative infections. All product candidates are investigational only and have not been approved for commercialization.  For more information, please visit www.achaogen.com. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, Achaogen’s expectations regarding potential regulatory approval of plazomicin, Achaogen’s commercial objectives and Achaogen’s pipeline of product candidates. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause Achaogen's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the preclinical and clinical development process; the risks and uncertainties of the regulatory approval process; the risks and uncertainties of commercialization and gaining market acceptance; the risk when bacteria will evolve resistance to plazomicin; Achaogen's reliance on third-party contract manufacturing organizations to manufacture and supply its product candidates and certain raw materials used in the production thereof; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Achaogen's patents or proprietary rights; and the risk that Achaogen's proprietary rights may be insufficient to protect its technologies and product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward- looking statements, as well as risks relating to Achaogen's business in general, see Achaogen's current and future reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and its Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Achaogen does not plan to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.


News Article | January 22, 2016
Site: www.scientificcomputing.com

Harvard’s John A. Paulson School of Engineering and Applied Sciences (SEAS), Center for Brain Science (CBS), and the Department of Molecular and Cellular Biology have been awarded over $28 million to develop advanced machine learning algorithms by pushing the frontiers of neuroscience. The Intelligence Advanced Research Projects Activity (IARPA) funds large-scale research programs that address the most difficult challenges facing the intelligence community. Today, intelligence agencies are inundated with data — more than they are able to analyze in a reasonable amount of time. Humans, naturally good at recognizing patterns, can’t keep pace. The pattern-recognition and learning abilities of machines, meanwhile, still pale in comparison to even the simplest mammalian brains. IARPA’s challenge: figure out why brains are so good at learning, and use that information to design computer systems that can interpret, analyze and learn information as successfully as humans. To tackle this challenge, Harvard researchers will record activity in the brain's visual cortex in unprecedented detail, map its connections at a scale never before attempted, and reverse engineer the data to inspire better computer algorithms for learning. “This is a moonshot challenge, akin to the Human Genome Project in scope,” said project leader David Cox, assistant professor of molecular and cellular biology and computer science. “The scientific value of recording the activity of so many neurons and mapping their connections alone is enormous, but that is only the first half of the project. As we figure out the fundamental principles governing how the brain learns, it's not hard to imagine that we’ll eventually be able to design computer systems that can match, or even outperform, humans.” These systems could be designed to do everything from detecting network invasions, to reading MRI images, to driving cars. The research team tackling this challenge includes Jeff Lichtman, the Jeremy R. Knowles Professor of Molecular and Cellular Biology; Hanspeter Pfister, the An Wang Professor of Computer Science; Haim Sompolinsky, the William N. Skirball Professor of Neuroscience; and Ryan Adams, assistant professor of computer science; as well as collaborators from MIT, Notre Dame, New York University, University of Chicago, and Rockefeller University. The multi-stage effort begins in Cox’s lab, where rats will be trained to recognize various visual objects on a computer screen. As the animals are learning, Cox’s team will record the activity of visual neurons using next-generation laser microscopes built for this project with collaborators at Rockefeller University, to see how brain activity changes as the animals learn. Then, a substantial portion of the rat's brain — one-cubic millimeter in size — will be sent down the hall to Lichtman’s lab, where it will be diced into ultra-thin slices and imaged under the world’s first multi-beam scanning electron microscope, housed in the Center for Brain Science. “This is an amazing opportunity to see all the intricate details of a full piece of cerebral cortex,” says Lichtman. “We are very excited to get started but have no illusions that this will be easy.” This difficult process will generate over a petabyte of data — equivalent to about 1.6 million CDs worth of information. This vast trove of data will then be sent to Pfister, whose algorithms will reconstruct cell boundaries, synapses and connections, and visualize them in three dimensions. “This project is not only pushing the boundaries of brain science, it is also pushing the boundaries of what is possible in computer science,” said Pfister. “We will reconstruct neural circuits at an unprecedented scale from petabytes of structural and functional data. This requires us to make new advances in data management, high performance computing, computer vision and network analysis.” If the work stopped here, its scientific impact would already be enormous — but it doesn’t. Once researchers know how visual cortex neurons are connected to each other in three dimensions, the next question is to figure out how the brain uses those connections to quickly process information and infer patterns from new stimuli. Today, one of the biggest challenges in computer science is the amount of training data that deep learning systems require. For example, in order to learn to recognize a car, a computer system needs to see hundreds of thousands of cars. But humans and other mammals don’t need to see an object thousands of times to recognize it — they only need to see it a few times. In subsequent phases of the project, researchers at Harvard and their collaborators will build computer algorithms for learning and pattern recognition that are inspired and constrained by the connectomics data. These biologically-inspired computer algorithms will outperform current computer systems in their ability to recognize patterns and make inferences from limited data inputs. For example, this research could improve the performance of computer vision systems that can help robots see and navigate through new environments. "We have a huge task ahead of us in this project, but at the end of the day, this research will help us understand what is special about our brains," Cox said. "One of the most exciting things about this project is that we are working on one of the great remaining achievements for human knowledge — understanding how the brain works at a fundamental level."

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