On November 1, 2018 ImmunSYS, Inc. reported that Chairman & CEO Eamonn Hobbs presented on October 31st at the 2018 NYC Oncology Investor Conference, held at Rockefeller Center in Manhattan (Press release, ImmunSYS, NOV 1,2018, View Source [SID1234577208]). ImmunSYS was one of 31 companies accepted to present to investors attending the conference.

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The NYC Oncology Investor Conference 2018 hosted by OneMed Forum, and sponsored by the National Foundation for Cancer Research, the International Cancer Impact Fund, Klosters Innovation Partners, Venable LLP, Torreya Partners, and Marcum is the leading conference for early-stage private and public cancer investing.

Meeting attendees included leading life science and oncology venture capitalists, family offices, lawyers, pharma executives, startup public and private cancer companies, and cancer foundations. The conference provides a forum for discussion of trends, opportunities, and risks in oncology investing, corporate presentations by a select group of public and private oncology companies, and updates on cutting edge science.

On November 1, 2018 Geron Corporation (Nasdaq: GERN) reported that clinical data related to imetelstat, the Company’s first-in-class telomerase inhibitor, will be the subject of two oral presentations at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held in San Diego, California from December 1-4, 2018 (Press release, Geron, NOV 1, 2018, View Source [SID1234532271]). The abstracts, summarizing clinical data from Part 1 of IMerge in myelodysplastic syndromes and the IMbark primary analysis in myelofibrosis, were published today on the ASH (Free ASH Whitepaper) website at www.hematology.org.

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"We are pleased the abstracts for Part 1 of IMerge and IMbark were accepted for presentation at ASH (Free ASH Whitepaper)," said John A. Scarlett, M.D., Geron’s President and Chief Executive Officer. "We are looking forward to the oral presentations as they underscore imetelstat’s potential to address the unmet medical need in lower risk MDS and relapsed/refractory myelofibrosis."

Oral Presentations

Title: Imetelstat Treatment Leads to Durable Transfusion Independence (TI) in RBC Transfusion-Dependent (TD), Non-Del(5q) Lower Risk MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Who Are Lenalidomide and HMA Naïve (Abstract #463)

Session Name: 637. Myelodysplastic Syndromes—Clinical Studies: Novel Therapeutics II
Session Date: Sunday, December 2, 2018
Session Time: 4:30 p.m. PT – 6:00 p.m. PT
Presentation Time: 4:30 p.m. PT

The oral presentation is expected to provide more mature efficacy and safety data from the combined initial and expansion cohorts in Part 1 of IMerge, a Phase 2 clinical trial of imetelstat in transfusion dependent, lower risk myelodysplastic syndromes (MDS) patients who are relapsed or refractory to an erythropoiesis stimulating agent (ESA), do not have a del(5q) chromosomal abnormality and are hypomethylating agent (HMA) and lenalidomide treatment naïve.

Title: Imetelstat Is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels (Abstract #685)

Session Name: 634. Myeloproliferative Syndromes: Clinical: Emerging Therapies and Prognostic Scoring in Myelofibrosis and Other MPNs
Session Date: Monday, December 3, 2018
Session Time: 10:30 a.m. PT – 12:00 p.m. PT
Presentation Time: 10:30 a.m. PT

The oral presentation will highlight efficacy and safety data from a primary analysis of IMbark, a Phase 2 clinical trial that evaluated two starting dose levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered by intravenous infusion every three weeks) in more than 100 patients with Intermediate-2 or High-risk myelofibrosis (MF) who have relapsed after or are refractory to prior treatment with a JAK inhibitor. More mature data from the extension phase of IMbark, including median overall survival, is expected to be presented.

Analyst and Investor Event

On December 10, 2018, Geron plans to host a webcasted event for analysts and investors. At the event, an investigator from each of the IMbark and Part 1 of IMerge trials will reprise the oral presentations from the ASH (Free ASH Whitepaper) Annual Meeting. A press release with event details, including how to access a webcast link, will be available at the end of November.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat include a Phase 2/3 trial called IMerge in lower risk myelodysplastic syndromes (MDS) and a Phase 2 trial called IMbark in Intermediate-2 to High-risk myelofibrosis. Imetelstat received Fast Track designation from the United States Food and Drug Administration for the treatment of patients with transfusion-dependent anemia due to lower risk MDS who are non-del(5q) and refractory or resistant to an erythroid stimulating agent.

On November 1, 2018 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the European Commission has approved Venclyxto (venetoclax) in combination with MabThera (rituximab) for the treatment of adult patients with chronic lymphocytic leukaemia (CLL) who have received at least one prior therapy (Press release, Hoffmann-La Roche, NOV 1, 2018, View Source [SID1234530480]).

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"There are approximately 30,000 people living with chronic lymphocytic leukaemia in Europe, an incurable blood cancer that becomes harder to treat with each relapse," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We are pleased that, thanks to this approval, Venclyxto plus MabThera will provide a new chemotherapy-free option for people with previously treated chronic lymphocytic leukaemia, helping them to live longer without their disease progressing compared to a standard-of-care therapy."

This approval is based on results from the randomised phase III MURANO study which showed that a fixed duration of treatment with Venclyxto plus MabThera significantly reduced the risk of disease progression or death (progression-free survival [PFS] as assessed by investigators [INV], primary endpoint of the study) by 83% compared with bendamustine plus MabThera (BR), a current standard of care (HR=0.17; 95% CI 0.11-0.25; p<0.0001). PFS assessed by independent review committee was consistent. In addition, minimal residual disease (MRD)-negativity in peripheral blood at the end of combination treatment was 62.4% with Venclyxto plus MabThera compared to 13.3% with BR. Being MRD-negative means no cancer can be detected in the blood and or bone marrow using a sensitive test. In Europe, MRD is used as an indicator of a patient achieving longer endpoints such as PFS and overall survival. The most commonly observed side effects of Venclyxto plus MabThera included low white blood cell count (neutropenia), diarrhoea and upper respiratory tract infection.

Venclyxto was previously granted conditional marketing authorisation in the EU in December 2016 as a single agent for the treatment of CLL in the presence of 17p deletion or TP53 mutation in people who are unsuitable for or have failed a B-cell receptor pathway inhibitor. Today’s EU approval follows the US Food and Drug Administration approval in June 2018 of Venclexta in combination with Rituxan for the treatment of people with CLL or small lymphocytic lymphoma, with or without 17p deletion, who have received at least one prior therapy. Additional submissions of the MURANO data to health authorities around the world are ongoing.

Venclyxto is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the United States, under the brand name Venclexta, and commercialised by AbbVie outside of the United States.

About the MURANO Study
MURANO (NCT02005471) is a phase III open-label, international, multicentre, randomised study evaluating the efficacy and safety of fixed duration Venclexta/Venclyxto (venetoclax) in combination with MabThera/Rituxan (rituximab) compared to standard of care bendamustine in combination with MabThera/Rituxan (BR) in patients with relapsed or refractory chronic lymphocytic leukaemia (CLL). Patients on the Venclexta/Venclyxto plus MabThera/Rituxan arm received six cycles of Venclexta/Venclyxto plus MabThera/Rituxan followed by Venclexta/Venclyxto monotherapy for up to two years total. Patients on the BR arm received six cycles of BR. The study included 389 patients with CLL who had been previously treated with at least one line of therapy. Patients were randomly assigned in a 1:1 ratio to receive either Venclexta/Venclyxto plus MabThera/Rituxan or BR. The primary endpoint of the study was progression-free survival (PFS) as assessed by investigator (INV). Secondary endpoints included overall survival (OS), overall response rate (ORR), complete response rate (with or without complete blood count recovery, CR/CRi), minimal residual disease (MRD) and safety.

*Data at median follow-up of 24 months

The most common adverse reactions (≥20%) of any grade in patients receiving Venclyxto in the combination study with MabThera were neutropenia, diarrhoea, and upper respiratory tract infection. Death occurred in 11% of people who received Venclexta/Venclyxto plus MabThera/Rituxan compared to 16% for BR.

About Venclexta/Venclyxto (venetoclax)
Venclexta/Venclyxto is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumours, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta/Venclyxto blocks the BCL-2 protein and works to restore the process of apoptosis.

Venclexta/Venclyxto is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the United States and by AbbVie outside of the United States. Together, the companies are committed to research with Venclexta/Venclyxto, which is currently being studied in clinical trials across several types of blood and other cancers.

In the United States, Venclexta has been granted four Breakthrough Therapy Designations by the FDA: in combination with Rituxan for people with relapsed or refractory chronic lymphocytic leukaemia (CLL); as a monotherapy for people with relapsed or refractory CLL with 17p deletion; in combination with hypomethylating agents (azacitidine or decitabine) for people with untreated acute myeloid leukaemia (AML) ineligible for intensive chemotherapy; and in combination with low-dose cytarabine for people with untreated AML ineligible for intensive chemotherapy.

Venclexta/Venclyxto is approved in more than 50 countries. Roche and AbbVie are currently working with regulatory agencies around the world to bring this medicine to additional eligible patients in need.

About Chronic Lymphocytic Leukaemia
Chronic lymphocytic leukaemia (CLL) is the most common type of leukaemia in the Western world.[1]
CLL mainly affects men and the median age at diagnosis is about 70 years.[2] In Europe, the incidence of all leukaemias is estimated to be almost 95,000[3] and CLL is estimated to affect around one-third of all people newly diagnosed with leukaemia.[1]

On November 1, 2018 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported data presentations at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting that highlight the Company’s robust off-the-shelf, allogeneic T-cell immunotherapy pipeline and next-generation CAR T technologies (Press release, Atara Biotherapeutics, NOV 1, 2018, View Source [SID1234530500]). The event will be held December 1-4, 2018, at the San Diego Convention Center in San Diego, CA.

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"We continue to rapidly advance our T-cell and CAR T immunotherapy pipeline across multiple therapeutic areas leveraging cutting edge discoveries by our external collaborators," said Dietmar Berger, M.D., Ph.D., Global Head of Research and Development of Atara Biotherapeutics. "Eight highlighted ASH (Free ASH Whitepaper) abstracts include an oral presentation on December 3 that describes a next-generation CAR T technology that may have wide applications including as a component of our pipeline programs in acute myeloid leukemia (AML) and B-cell malignancies. In addition, promising clinical results from our Phase 2 studies of tab-cel for patients with EBV+ PTLD involving the CNS as well as health outcome and treatment pattern assessments for transplant and PTLD patients will be featured. We are pleased with these strong results and continued demonstration of the broad potential of our off-the-shelf, allogeneic T cell platform."

Atara is also scheduled to present tab-cel efficacy and safety results in patients with EBV-associated leiomyosarcoma (EBV+ LMS) solid tumors at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology Congress held December 13-16, 2018, in Geneva, Switzerland.

60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting

Abstract 966: Mutation of the CD28 Costimulatory Domain Confers Increased CAR T Cell Persistence and Decreased Exhaustion
Session: 703. Adoptive Immunotherapy: Preclinical Studies to Improve Safety and Efficacy of CAR-T Cells
Oral Presentation Date and Time: Monday, December 3, 2018 at 5:45 pm PST
Location: Marriott Marquis San Diego Marina, San Diego Ballroom B
Authors: Justin C Boucher, Gongbo Li, Bishwas Shrestha, Maria L Cabral, Dylan Morrissey, Lawrence Guan, Marco L Davila
Affiliations:Moffitt Cancer Center
Summary: The investigators created mutated CAR T cells with mutations in intracellular co-stimulatory domains (PYAP-only CAR T-cell) resulting in reduced cytokine secretion, but maintained cytotoxic activity compared to non-mutated (CD28 CAR T cells). PYAP-only CAR T treated immunocompetent mice showed a significant survival advantage compared to treatment with non-mutated CAR T cells. PYAP-only CAR T cells exhibited increased persistence in spleen and bone marrow. The work enables the development of next-generation CAR T cell therapies with potential improved persistence and reduced toxicities.

Abstract 4590: Adoptive Therapy with EBV-Specific T Cells for Treatment of CNS EBV Post-Transplant Lymphoproliferative Disease Arising after Hematopoietic Stem Cell Transplant or Solid Organ Transplant
Session: 723. Clinical Allogeneic and Autologous Transplantation: Late Complications and Approaches to Disease Recurrence: Poster III
Poster Presentation Date & Time: Monday, December 3, 2018 from 6:00 pm – 8:00 pm PST
Location: Hall GH
Authors: Susan Prockop, MD, Stephanie Suser, Ekaterina Doubrovina, MD, PhD, Hugo R. Castro-Malaspina, MD, Esperanza B. Papadopoulos, MD, James W. Young, MD, Victoria Szenes, PNP, Alison Slocum, MA, Karim Baroudy, MS and Richard J. O’Reilly, MD
Affiliations: Memorial Sloan Kettering Cancer Center
Summary: Off-the-shelf, allogeneic EBV-specific T cells (tab-cel) have demonstrated efficacy in the treatment of EBV+ PTLD. However, PTLD involving the central nervous system is particularly challenging due to poor CNS bioavailability of the current available treatment (rituximab). This study evaluated the activity of tab-cel in treating CNS EBV+ PTLD in patients who have failed prior rituximab therapy. The overall response rate was 63% of the 19 patients treated in the study. The one-year overall survival (OS) was 60% with responding and non-responding patients demonstrating a one-year OS of 92% and 14%, respectively. The authors concluded that tab-cel can treat an otherwise often lethal CNS complication of transplantation and that the availability of tab-cel enables early treatment of the disease.

Abstract 4777: Treatment Patterns for Patients with Post-Transplant Lymphoproliferative Disorder Who Fail Rituximab after Allogeneic Hematopoietic Stem Cell Transplantation: Findings from a Systematic Literature Review
Session: 902. Health Services Research—Malignant Diseases: Poster III
Poster Presentation Date & Time: Monday, December 3, 2018 from 6:00 pm – 8:00 pm PST
Location: Hall GH
Authors: Hairong Xu, MD, PhD, Crystal Watson, MS, Shan Ashton Garib, MA, Anna Forsythe, PharmD, MSc, MBA and Arie Barlev, PharmD
Affiliations:Atara Biotherapeutics, Purple Squirrel Economics

Abstract 3556: Estimating Long-Term Survival in a Cohort of Allogeneic Hematopoietic Stem Cell Transplant Patients
Session: 902. Health Services Research—Malignant Diseases: Poster II
Poster Presentation Date & Time: Sunday, December 2, 2018 from 6:00 pm – 8:00 pm PST
Location: Hall GH
Authors: Stephen Palmer, MSc, Casey Quinn, PhD, MPhil, Crystal Watson, MS and Arie Barlev, PharmD
Affiliations:Centre for Health Economics, University of York, PRMA Consulting Ltd., Atara Biotherapeutics

Abstract 4596: Dual-Sensitized T-Cells Responding to EBV Blcl and Either CMVpp65 or WT-1 Peptide Pools Have Distinct or Shared HLA Restrictions That May Depend on the Presenting HLA Alleles
Session: 723. Clinical Allogeneic and Autologous Transplantation
Poster Presentation Date & Time: Monday, December 3, 2018 from 6:00 pm – 8:00 pm PST
Location: Hall GH
Authors: Ekaterina Doubrovina, MD, PhD, Aisha N. Hasan, MD, Susan Prockop, MD, Karim Baroudy, MS, and Richard O’Reilly, MD
Affiliations: Memorial Sloan Kettering Cancer Center

Abstract 5839: A Systematic Literature Review of Real-World Evidence in Post-Transplant Lymphoproliferative Disorder
Authors: Hairong Xu, MD, PhD, Anna Forsythe, PharmD, MSc, MBA, Arie Barlev, PharmD, Nazia Rashid, PharmD and Crystal Watson, MS
Affiliations:Atara Biotherapeutics, Purple Squirrel Economics

Abstract 5841: Younger Patients Are Impacted By Post-Transplant Lymphoproliferative Disorder: Findings from a Systematic Literature Review of Real-World Evidence
Authors: Crystal Watson, MS, Hairong Xu, MD, PhD, Anna Forsythe, PharmD, MSc, MBA, Shan Ashton Garib, MA and Arie Barlev, PharmD
Affiliations:Atara Biotherapeutics, Purple Squirrel Economics

Abstract 5840: Risk of Patients Developing Post-Transplant Lymphoproliferative Disorder within the First Year after an Allogeneic Hemopoietic Stem Cell Transplant, 2011 to 2016: A US Claims Database Analysis
Authors: Arie Barlev, PharmD, Hairong Xu, MD, PhD, Nicole Fulcher, MA, Crystal Watson, MS, Ila Sruti, MPH and Akshay Sudhindra, MD
Affiliations:Atara Biotherapeutics, IBM Watson Health

European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology Congress 2018

Abstract 222 (Session 37): Efficacy and safety of tabelecleucel in patients with Epstein‑Barr Virus-associated leiomyosarcomas (EBV+ LMS)
Oral Presentation Date and Time: Saturday, December 15, 2018 at 8:05 a.m.
Location: Room A, Palexpo Exhibition Centre, Geneva, Switzerland
Authors:L.S. Kurlander, A. Srinivasan, A. Ghobadi, S. Suser, E. Doubrovina, F. Boulad, L. Mascarenhas, M. Laquaglia, A. Price, G. Behr, B. Shulkin, A. Sudhindra, Y. Wei, M. Hiremath, W. Navarro, R. O’Reilly, S. Prockop
Affiliations: Weill Cornell New York Presbyterian Hospital, St. Jude Children’s Research Hospital, Washington University School of Medicine, Children’s Hospital of Los Angeles, Atara Biotherapeutics, Memorial Sloan Kettering Cancer Center

About EBV+ PTLD
Since its discovery as the first human oncovirus, Epstein-Barr virus (EBV) has been implicated in the development of a wide range of lymphoproliferative disorders, including lymphomas, and other cancers. EBV is widespread in all human populations and persists as a lifelong, asymptomatic infection. In immunocompromised patients, such as those undergoing allogeneic hematopoietic cell transplants (HCT) or solid organ transplants (SOT), EBV-associated post-transplant lymphoproliferative disorder (EBV+ PTLD) represents a life-threatening condition. Median overall survival in patients with EBV+ PTLD following HCT who have failed rituximab-based first line therapy is 16-56 days. In EBV+ PTLD following SOT, patients failing rituximab experience increased chemotherapy-induced treatment-related mortality compared to other lymphoma patients. One- and two-year survival in patients with high-risk EBV+ PTLD following SOT is 36% and 0%, respectively.

About tab-cel (tabelecleucel)
Atara’s most advanced T-cell immunotherapy in development, tab-cel, is a potential treatment for patients with Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (EBV+ PTLD) who have failed rituximab, as well as other EBV-associated hematologic and solid tumors, including nasopharyngeal carcinoma (NPC). In February 2015, FDA granted tab-cel Breakthrough Therapy Designation for EBV+ PTLD following allogeneic hematopoietic cell transplant (HCT), and in October 2016, tab-cel was accepted into the EMA Priority Medicines (PRIME) regulatory pathway for the same indication, providing enhanced regulatory support. Atara also received positive regulatory feedback from Health Canada in September 2017 supporting the submission of tab-cel for an expedited approval pathway. In addition, tab-cel has orphan status in the U.S. and EU. Tab-cel is in Phase 3 clinical development for the treatment of EBV+ PTLD following an allogeneic hematopoietic cell transplant (MATCH study) or solid organ transplant (ALLELE study), and Atara is planning a Phase 1/2 study in NPC.

On November 1, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that ten abstracts have been selected for presentation, including three oral presentations, at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 Annual Meeting being held December 1-4, 2018 in San Diego (Press release, Karyopharm, NOV 1, 2018, View Source [SID1234530516]). Four key abstracts to be presented at the meeting will feature clinical data for selinexor, the Company’s first in class, oral SINE compound, from Karyopharm-sponsored trials. The presentations will include: top-line results from the Phase 2b SADAL study in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), additional data from the pivotal Phase 2b STORM study in patients with penta-refractory multiple myeloma, and updated data from the Darzalex (daratumumab) and Pomalyst (pomalidomide) arms of the Phase 1b/2 STOMP study of selinexor in combination with backbone therapies for the treatment of patients with relapsed or refractory multiple myeloma.

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"With ten abstracts to be presented at this year’s ASH (Free ASH Whitepaper) meeting, we believe the depth and breadth of the selinexor clinical data is highly compelling," said Sharon Shacham, PhD, MBA, Founder, President and Chief Scientific Officer of Karyopharm. "We look forward to the presentation of top-line results from the fully enrolled SADAL study in DLBCL. These data, if positive, could support our second planned New Drug Application in the first half of 2019, with a request for accelerated approval for selinexor as a new treatment for patients with relapsed or refractory DLBCL. In the previously reported interim analysis for the Phase 2b SADAL study, single-agent oral selinexor demonstrated activity and independently-confirmed durable responses in patients with heavily pretreated DLBCL, and these responses correlated to improved overall survival. We are delighted that data from the selected research abstracts will be shared with the medical community at ASH (Free ASH Whitepaper) this year."

In addition to top-line data from the SADAL study, this year’s ASH (Free ASH Whitepaper) meeting will also feature updated data from the STORM study in an oral presentation. As part of this presentation, data from an independent database of patients with heavily pretreated myeloma will also be presented, further underscoring how poor the prognosis is for patients with penta-refractory multiple myeloma. In October 2018, the U.S. FDA accepted Karyopharm’s New Drug Application for selinexor seeking accelerated approval as a new treatment for patients with penta-refractory multiple myeloma. The FDA has assigned a Priority Review and given an action date of April 6, 2019 under the Prescription Drug User-Fee Act (PDUFA).

Other key abstracts at the meeting include data from two arms of the Phase 1b/2 STOMP study. There will be an oral presentation with updated data from the arm evaluating selinexor in combination with Darzalex and low-dose dexamethasone (SDd). In previously reported data, the once weekly SDd combination without a proteasome inhibitor or immunomodulatory drug demonstrated high response rates in the patient population as a doublet regimen. Finally, a poster presentation will highlight updated data from the arm evaluating selinexor in combination with Pomalyst and low-dose dexamethasone (SPd). In data reported previously from these arms, selinexor demonstrated evidence of synergistic anti-myeloma activity when combined with these standard approved therapies.

Details for the ASH (Free ASH Whitepaper) 2018 presentations are as follows:

Oral Presentations – Company-Sponsored Trials

Title:Results of the Pivotal STORM Study (Part 2) in Penta-Refractory Multiple Myeloma (MM): Deep and Durable Responses with Oral Selinexor Plus Low Dose Dexamethasone in Patients with Penta-Refractory MM
Presenter:Ajai Chari, Icahn School of Medicine at Mount Sinai, New York, New York
Abstract Number/Publication ID: 598
Session: 653. Myeloma: Therapy, excluding Transplantation: Antibodies and Targeted Therapies
Date and Time:Monday, December 3, 2018; 7:45 AM PT
Location:San Diego Convention Center, Room 6F

Title:Deep and Durable Responses with Selinexor, Daratumumab, and Dexamethasone (SDd) in Patients with Multiple Myeloma (MM) Previously Exposed to Proteasome Inhibitors and Immunomodulatory Drugs: Results of Phase 1b Study of SDd
Presenter:Cristina Gasparetto, Duke University Cancer Center, Durham, North Carolina
Abstract Number/Publication ID: 599
Session: 653. Myeloma: Therapy, excluding Transplantation: Antibodies and Targeted Therapies
Date and Time:Monday, December 3, 2018; 8:00 AM PT
Location:San Diego Convention Center, Room 6F

Oral Presentations – Investigator-Sponsored Trials

Title:Selinexor, a First-in-Class XPO1 Inhibitor, Is Efficacious and Tolerable in Patients with Myelodysplastic Syndromes Refractory to Hypomethylating Agents
Presenter:Virginia M. Klimek, Memorial Sloan Kettering Cancer Center, New York, New York
Abstract Number/Publication ID: 233
Session: 637. Myelodysplastic Syndromes—Clinical Studies: Novel Therapeutics I
Date and Time:Saturday, December 1, 2018; 5:00 PM PT
Location: Manchester Grand Hyatt San Diego, Grand Hall A

Poster Presentations – Company-Sponsored Trials

Title: Single Agent Oral Selinexor Demonstrates Deep and Durable Responses in Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL) in Both GCB and Non-GCB Subtypes: The Phase 2b SADAL Study
Presenter: Marie Maerevoet, Institute Jules Bordet, Brussels, Belgium
Abstract Number/Publication ID: 1677
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster I
Date and Time:Saturday, December 1, 2018; 6:15-8:15 PM PT
Location:San Diego Convention Center, Hall GH

Title:Selinexor Plus Pomalidomide and Low Dose Dexamethasone (SPd) in Patients with Relapsed or Refractory Multiple Myeloma
Presenter:Christine Chen, Princess Margaret Cancer Center, Toronto, Ontario
Abstract Number/Publication ID: 1993
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster I
Date and Time:Saturday, December 1, 2018; 6:15-8:15 PM PT
Location:San Diego Convention Center, Hall GH

Poster Presentations – Investigator-Sponsored Trials

Title:Phase I Study of the Selinexor in Relapsed/Refractory Childhood Acute Leukemia
Presenter:Andrew E. Place, Dana-Farber Cancer Institute and Boston Children’s Hospital, Boston, Massachusetts
Abstract Number/Publication ID: 1405
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I
Date and Time:Saturday, December 1, 2018; 6:15 PM PT
Location:San Diego Convention Center, Hall GH

Title:E2F1 Is a Biomarker of Selinexor Resistance in Relapsed/Refractory Multiple Myeloma Patients
Presenter:Alessandro Lagana, Icahn School of Medicine at Mount Sinai, New York, New York
Abstract Number/Publication ID: 3216
Session: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster II
Date and Time:Sunday, December 2, 2018; 6:00-8:00 PM PT
Location:San Diego Convention Center, Hall GH

Title:Final results from a phase I trial combining selinexor with high-dose cytarabine (HiDAC) and mitoxantrone (Mito) for remission induction in acute myeloid leukemia (AML)
Presenter:Hongtao Liu and Amy Wang, University of Chicago Medicine, Chicago, Illinois
Abstract Number/Publication ID: 4073
Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III
Date and Time:Monday, December 3, 2018; 6:00-8:00 PM PT
Location:San Diego Convention Center, Hall GH

Title:NAMPT inhibitor KPT-9274 selectively targets self-renewal capacity in acute myeloid leukemia
Presenter:Shaneice Mitchell, Ohio State University College of Medicine, Columbus, Ohio
Abstract Number/Publication ID: 3931
Session: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Poster III
Date and Time:Monday, December 3, 2018; 6:00-8:00 PM PT
Location:San Diego Convention Center, Hall GH

Title:Inhibition of Nicotinamide Phosphoribosyltransferase (NAMPT) Activity Selectively Targets Human Acute Myeloid Leukemia Stem Cells
Presenter:Amit Subedi, Princess Margaret Cancer Centre, University Health Network, Toronto, Ontario
Abstract Number/Publication ID: 3932
Session: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases
Date and Time: Monday, December 3, 2018; 6:00-8:00 PM PT
Location: San Diego Convention Center, Hall GH

About Selinexor

Selinexor 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 2,600 patients have been treated with selinexor. In April and September 2018, Karyopharm reported positive data from the Phase 2b STORM study evaluating selinexor in combination with low-dose dexamethasone in patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. Karyopharm’s New Drug Application (NDA) has been accepted for filing and granted Priority Review by the FDA, and oral selinexor is currently under review by the FDA as a possible new treatment for patients with penta-refractory multiple myeloma. The Company also plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in early 2019 with a request for conditional approval. Selinexor is also being evaluated in several other mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), as a potential backbone therapy in combination with approved therapies (STOMP), in diffuse large B-cell lymphoma (SADAL), liposarcoma (SEAL), and an investigator-sponsored study in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.