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Rocket Pharmaceuticals Announces Strategic Research Collaboration

On May 17, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, and the Stanford University School of Medicine reported a strategic collaboration to support the advancement of Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD) gene therapy research (Press release, Rocket Pharmaceuticals, MAY 17, 2018, View Source;p=RssLanding&cat=news&id=2349565 [SID1234526774]). Rocket’s lentiviral vector (LVV)-based gene therapy program for FA is currently in clinical trials with academic partners in the U.S. and Europe. The LVV-based gene therapy program for PKD is currently in preclinical development in Europe.

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Under the terms of the agreement, Stanford will serve as a lead clinical trial research center in the United States for a planned upcoming registrational trial for FA, and would also be the lead site for PKD clinical trials. Maria Grazia Roncarolo, M.D., director of the Stanford Center for Definitive and Curative Medicine and co-director of the school’s Institute for Stem Cell Biology and Regenerative Medicine, will lead the school’s effort. The center is a joint initiative of the School of Medicine, Stanford Health Care and Stanford Children’s Health and is focused on bench to bedside development of innovative cell- and gene-based therapies.

Gaurav Shah, M.D., Chief Executive Officer and President of Rocket, commented, "Rocket is delighted to expand the reach of our gene therapy program in the U.S. and prepare for our registrational trial. We are committed to developing FA and PKD programs in collaboration with outstanding gene therapy centers and pioneers in the field. This collaboration with the Stanford Center for Definitive and Curative Medicine is a critical step within our overall strategy of building relationships with gene therapy experts, with investigators who have dedicated their careers to improving the care of patients afflicted with these disorders, and within the broader FA and PKD communities."

"This project will also evaluate the introduction of conditioning regimens for both FA and PKD, where we hope to develop best-in-class gene therapy approaches for both clinical indications. The regulatory design and preparation for our registrational trial for FA is ongoing and we remain on track to advance this program to a registration study in 2019. Our PKD program continues to advance in preclinical studies with an Investigational Medicinal Product Dossier (IMPD) expected to be filed in early 2019," continued Dr. Shah.

For more information about this news on the Stanford website, please visit View Source

Novartis data at ASCO and EHA reinforce company’s commitment to reimagining cancer

On May 17, 2018 Novartis reported it will present data from across its oncology portfolio at the upcoming 54th Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) to be held June 1-5 in Chicago; and the 23rd Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), scheduled for June 14-17 in Stockholm (Press release, Novartis, MAY 17, 2018, View Source [SID1234526773]). With studies highlighting more than 25 compounds investigated in a range of disease areas including breast, renal cell and lung cancers, leukemias and other blood disorders, and myeloproliferative neoplasms (MPNs), the 84 abstracts that are part of the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) scientific programs illustrate the breadth and depth of Novartis’ work in oncology.

"Novartis is improving the lives of people with cancer through a relentless commitment to scientific innovation," said Liz Barrett, CEO, Novartis Oncology. "Whether examining investigational treatment combinations, investigating treatment options that may redefine treatment goals of diseases like CML, or demonstrating the value of our therapies through real-world studies, Novartis continues to push boundaries as we reimagine cancer."

Novartis data at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting will highlight the following:

New data evaluating Kisqali (ribociclib)* in broad range of patients with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced breast cancer and additional update on investigational treatment, BYL719 (alpelisib):

Ribociclib (RIB) + fulvestrant (FUL) in postmenopausal women with hormone receptor-positive (HR+), HER2-negative (HER2-) advanced breast cancer (ABC): Results from MONALEESA-3 [Abstract #1000; Sunday, June 3, 8:00 AM CDT]
Ribociclib (RIBO) + letrozole (LET) in patients (pts) with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC) with no prior endocrine therapy (ET) for ABC: Preliminary results from the phase 3b CompLEEment-1 trial [Abstract #1056; Saturday, June 2, 8:00 AM CDT]
Ribociclib (RIB) + tamoxifen (TAM) or a non-steroidal aromatase inhibitor (NSAI) in premenopausal women with hormone receptor-positive (HR+), HER2-negative (HER2-) advanced breast cancer (ABC) who received prior chemotherapy (CT): MONALEESA-7 subgroup analysis [Abstract #1047; Saturday, June 2, 8:00 AM CDT]
First-line ribociclib (RIB) + letrozole (LET) in hormone receptor-positive (HR+), HER2 -negative (HER2-) advanced breast cancer (ABC): MONALEESA-2 biomarker analyses [Abstract #1022; Saturday, June 2, 8:00 AM CDT]
BYLieve: A phase II study of alpelisib (ALP) with fulvestrant (FUL) or letrozole (LET) for treatment of PIK3CA mutant, hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (aBC) progressing on/after cyclin-dependent kinase 4/6 inhibitor (CDK4/6i) therapy [Abstract #TPS1107; Saturday, June 2, 8:00 AM CDT]
Data evaluating real-world effectiveness and safety outcomes of first-line Votrient (pazopanib) in patients with advanced renal cell carcinoma (RCC):

Prospective, multinational, observational study of real-world treatment outcomes with pazopanib in patients with advanced or metastatic renal cell carcinoma (PRINCIPAL Study) [Abstract #4574; Saturday, June 2, 8:00 AM CDT]
Comparison of clinical outcomes with first-line pazopanib in clinical trial eligible and non-clinical trial eligible patients with renal cell carcinoma [Abstract #4561; Saturday, June 2, 8:00 AM CDT]
New analyses of ENESTop and ENESTfreedom evaluating Treatment-free Remission (TFR) at 144-week follow-up after Tasigna (nilotinib) treatment discontinuation in eligible adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP):

Long-term treatment-free remission (TFR) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP) after stopping second-line (2L) nilotinib: ENESTop 144-week results [Abstract #7003; Saturday, June 2, 4:00 PM CDT]
Long-term treatment-free remission (TFR) following frontline (1L) nilotinib in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP): ENESTfreedom 144-wk results [Abstract #7063; Monday, June 4, 8:00 AM CDT]
Additional data presented at ASCO (Free ASCO Whitepaper) include:

Trametinib in pediatric patients with neurofibromatosis type 1 (NF-1)-associated plexiform neurofibroma: A phase I/IIa study [Abstract #10504; Saturday, June 2, 4:12 PM CDT]
Dabrafenib in pediatric patients with BRAF V600-positive high-grade glioma (HGG) [Abstract #10505; Saturday, June 2, 4:24 PM CDT]
Efficacy and safety results from a phase I/IIa study of dabrafenib in pediatric patients with BRAF V600-mutant relapsed refractory low-grade glioma [Abstract #10506; Saturday, June 2, 4:36 PM CDT]
Phase I/II study of spartalizumab (PDR001), an anti-PD1 mAb, in patients with anaplastic thyroid cancer [Abstract #6024; Saturday, June 2, 1:15 PM CDT]
Phase I/II study of LAG525 ± spartalizumab (PDR001) in patients (pts) with advanced malignancies [Abstract #3012; Monday, June 4, 8:00 AM CDT]
A phase I study of LXH254 in patients (pts) with advanced solid tumors harboring MAPK pathway alterations [Abstract #2586; Monday, June 4, 8:00 AM CDT]
Sandoz, a Novartis division and the pioneer and global leader in biosimilars will present data for the company’s filgrastim biosimilar:

Comparison of efficacy and safety of biosimilar filgrastim in a randomized clinical trial (PIONEER) and real-world practice (MONITOR-GCSF) [Abstract #111; Monday, June 4, 10:21 AM CDT]
Advanced Accelerator Applications, a Novartis company and leader in nuclear medicine theragnostics, will present an update on outcomes from the NETTER-1 study evaluating Lutathera (lutetium Lu 177 dotatate) in patients with progressive midgut neuroendocrine tumors:

First update on overall survival, progression-free survival, and health-related time-to-deterioration quality of life from the NETTER-1 study: 177Lu-Dotatate vs. high dose octreotide in progressive midgut neuroendocrine tumors [Abstract #4099; Sunday, June 3, 8:00 AM CDT]
Novartis data at the 2018 EHA (Free EHA Whitepaper) Annual Congress will highlight the following:

Updates on outcomes with Kymriah (tisagenlecleucel) in adult relapsed or refractory (r/r) patients with diffuse large b-cell lymphoma (DLBCL) and patient-reported quality of life in pediatric and young adult patients with r/r B-cell acute lymphoblastic leukemia (B-ALL)**:

An updated analysis of JULIET, a global pivotal phase 2 trial of tisagenlecleucel in adult patients with relapsed or refractory (r/r) diffuse large b-cell lymphoma (DLBCL) [Abstract #S799; Saturday, June 16, 11:30 AM CEST]
Outcomes of young adult patients (>= 18-25 years) with relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL) following treatment with chimeric antigen receptor (CAR) T-cell therapy [Abstract #S1565; Sunday, June 17, 8:00 AM CEST]
Improvement of patient-reported quality of life following tisagenlecleucel infusion in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia [Abstract #PF181; Friday, June 15, 5:30 PM CEST]
Initial experience in US commercial manufacturing of tisagenlecleucel, a chimeric antigen receptor (CAR)-T cell therapy for pediatric relapsed/refractory B-cell precursor acute lymphoblastic leukemia [Abstract #PS1156; Saturday, June 16, 5:30 PM CEST]
New data evaluating Jakavi (ruxolitinib)*** for patients with myelofibrosis, including those with early stage disease, and patients with polycythemia vera who are resistant to or intolerant of hydroxyurea:

Comparison of ruxolitinib and real-world best available therapy in terms of overall survival and thrombosis in patients with polycythemia vera who are resistant or intolerant to hydroxyurea [Abstract #PF628; Friday, June 15, 5:30 PM CEST]
Safety and efficacy of ruxolitinib (RUX) in patients (pts) with DIPSS low-risk myelofibrosis (MF) in the phase 3B expanded-access JUMP study [Abstract #PF623; Friday, June 15, 5:30 PM CEST]
Predictors of response to ruxolitinib (RUX) in patients (pts) with myelofibrosis (MF) in the phase 3B expanded-access JUMP study [Abstract #PF616; Friday, June 15, 5:30 PM CEST]
Results from 48-week follow-up of the EXPAND study: a phase 1b, open-label, dose-finding study of ruxolitinib in patients with myelofibrosis and low platelet counts (50-99 × 109/L) at baseline [Abstract #PF611; Friday, June 15, 5:30 PM CEST]
Further analyses of ENESTop and ENESTfreedom evaluating TFR at 144-week follow-up after Tasigna (nilotinib) treatment discontinuation in eligible adult patients with Ph+ CML-CP:

Long-term treatment-free remission (TFR) following second-line (2L) nilotinib (NIL) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP): ENESTop 144-wk results [Abstract #PF377; Friday, June 15, 5:30 PM CEST]
Long-term treatment-free remission (TFR) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP) following frontline (1L) nilotinib (NIL): Results from ENESTfreedom [Abstract #PF368; Friday, June 15, 5:30 PM CEST]
Safety analysis of the SUSTAIN study evaluating crizanlizumab in patients with sickle cell disease:

Crizanlizumab treatment is not associated with the development of proteinuria and hematuria in patients with sickle cell disease: A safety analysis from the SUSTAIN study [Abstract #PF712; Friday, June 15, 5:30 PM CEST]
New data evaluating Revolade (eltrombopag) in patients with either persistent or chronic immune thrombocytopenia (ITP):

Eltrombopag treatment improved platelet counts in patients with persistent or chronic immune thrombocytopenia: Efficacy and safety results from the Phase III EXTEND study and a Phase IV study [Abstract #PF671; Friday, June 15, 5:30 PM CEST]
Interim results from the ITP World Impact Survey (I-WISh) about the burden of disease and impact of ITP on patient quality of life and productivity:

The burden of disease and impact of Immune Thrombocytopenia (ITP) on patient quality of life and productivity: Results from the ITP World Impact Survey (I-WISh) [Abstract #PF654; Friday, June 15, 5:30 PM CEST]
Throughout the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting and EHA (Free EHA Whitepaper) Annual Congress, Novartis will host dedicated content on View Source that will feature unique insights and perspectives on emerging areas of cancer care and research.

Product Information
Approved indications for products vary by country and not all indications are available in every country. The product safety and efficacy profiles have not yet been established outside the approved indications. Because of the uncertainty of clinical trials, there is no guarantee that compounds will become commercially available with additional indications.

For full prescribing information, including approved indications and important safety information about marketed products, please visit View Source

For Lutathera full prescribing information, including approved indications and important safety, please visit View Source

Alpelisib (BYL719), LAG525, spartalizumab (PDR001), LXH254 and crizanlizumab (SEG101) are investigational compounds. Efficacy and safety have not been established. There is no guarantee these compounds will become commercially available.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

MorphoSys Announces Presentation of Clinical Data on Proprietary Blood Cancer Programs at Upcoming EHA 2018 Conference

On May 17, 2018 MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX; Nasdaq: MOR; OTC: MPSYY) reported the publication of two abstracts on its proprietary hemato-oncological drug candidates MOR208 and MOR202 submitted to the 23rd European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, to be held in Stockholm/Sweden from June 14-17, 2018 (Press release, MorphoSys, MAY 17, 2018, View Source [SID1234526772]).

In a poster presentation, first clinical data from the exploratory phase 2 COSMOS trial with the Fc-engineered CD19 antibody MOR208 in combination with idelalisib in chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), after discontinuation of an ibrutinib therapy, will be presented. In an oral presentation, updated maturing data from a phase 1/2a study with the CD38 antibody MOR202 alone and in combination with pomalidomide or lenalidomide in relapsed/refractory multiple myeloma will be presented.

"We are pleased that clinical data from trials investigating our proprietary antibody programs MOR208 and MOR202 in indications with high medical need were selected for presentation at the upcoming EHA (Free EHA Whitepaper) conference," commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

Details about the abstracts from MorphoSys’s proprietary programs MOR208 and MOR202 accepted for presentation at EHA (Free EHA Whitepaper) 2018:

Abstract Code: PF350

Two-cohort, phase II study in R/R CLL (COSMOS): First preliminary safety and efficacy results of MOR208 treatment in combination with idelalisib in patients who discontinued prior ibrutinib therapy

The poster presentation will include clinical results from the phase 2 study COSMOS with MOR208 in combination with idelalisib (cohort A) in adult patients with relapsed/refractory CLL who failed prior treatment with Bruton’s Tyrosine Kinase inhibitor (BTKi) ibrutinib.

The poster will be presented during the session "Chronic lymphocytic leukemia and related disorders – Clinical" on Friday, June 15, 2018 5:30-7:00 pm CEST (11:30am-1:00pm EDT), in the poster area at the Stockholmsmässan in Stockholm.

In addition, the abstract will be on display on the E-poster screens at the conference from Friday, June 15, 2018, 9:30 am CEST (3:30 am EDT) to Sunday, June 17, 2018 1:00 pm CEST (7:00 am EDT).

Abstract Code: S848

MOR202 with low-dose dexamethasone (DEX) or pomalidomide/DEX or lenalidomide/DEX in relapsed or refractory multiple myeloma (r/r MM): A phase I/IIa, multicenter, dose-escalation study

The oral presentation will include updated clinical data from the phase 1/2a study with MOR202 alone or in combination with the immunomodulatory drugs lenalidomide or pomalidomide, plus low-dose dexamethasone (DEX). The trial is being conducted in pre-treated patients with relapsed/refractory multiple myeloma.

The oral presentation will be given during the session "New therapeutic strategies to improve the outcome of relapse/refractory plasma cell disorders" on Saturday, June 16, 2018, from 4:15-4:30pm MEST (10:15-10:30am EDT), in Room A1 at the Stockholmsmässan in Stockholm.

Additional information can be found at www.ehaweb.org, including the abstracts.

MabVax Therapeutics Receives U.S. Patent for Fully Human Antibodies Targeting the Antigen GD2 Expressed on Sarcoma, Melanoma, and Neuroblastoma

On May 17, 2018 MabVax Therapeutics Holdings, Inc. (Nasdaq: MBVX), a clinical-stage biotechnology company with a fully human antibody discovery platform focused on the development of antibody-based products to address unmet medical needs in the treatment of cancer, reported that the Company has been issued Patent No. 9,856,324 by the United States Patent and Trademark Office (USPTO) for a patent titled, "HUMAN MONOCLONAL ANTIBODIES TO GANGLIOSIDE GD2 (Press release, MabVax, MAY 17, 2018, View Source [SID1234526770])."

This is the second patent issued in the antibody portfolio created through MabVax’s unique discovery platform, which uses blood samples from patients vaccinated with selected tumor associated carbohydrate antigens. The recovery of human antibodies directly from vaccinated cancer patients makes the antibodies discovered by MabVax highly unique and potentially useful while reducing the kinds of toxicities induced by non-human antibodies. Similar to the Company’s sialyl lewis A (sLea) and Thomsen-nouveau (Tn) antigen targeting antibodies, the ganglioside GD2 targeting antibodies have the potential to be developed as therapeutic and diagnostic products. Additional disease targets for anti-GD2 antibodies include sarcoma, melanoma, and neuroblastoma.

"The issuance of this patent represents another valuable intellectual property asset brought forward by MabVax and is an important component of our unique discovery strategy and expands our robust patent estate. Importantly, this filing is timely as we evaluate additional product candidates that have the potential to address significant unmet needs in the treatment of cancers to fuel our pipeline and expand our partnering discussions," stated David Hansen, MabVax’s President and Chief Executive Officer.

The Company has already made substantial progress required to translate the GD2 preclinical development program into clinical development. In collaboration with St. Jude Children’s Research Hospital, data from the preclinical development program were presented as a poster at the World Molecular Imaging Conference last year. The results of the study demonstrated the anti-GD2 antibodies had high specificity and antibody drug conjugates based on these antibodies were readily internalized and cytotoxic. Immuno-PET agents developed with these antibodies showed good accumulation on osteosarcoma tumors and demonstrated potential utility as a diagnostic imaging agent.

Karyopharm to Present Selinexor Phase 1b/2 STOMP Data at the European Hematology Association 2018 Annual Meeting

On May 17, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that three posters highlighting clinical data from the ongoing Phase 1b/2 STOMP study will be presented at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) 2018 Annual Meeting taking place June 14-17, 2018 in Stockholm, Sweden (Press release, Karyopharm, MAY 17, 2018, View Source [SID1234526769]). These three poster presentations will feature updated data from the STOMP arms evaluating selinexor, the Company’s lead, novel, oral SINE compound, and dexamethasone in combination with standard approved therapies, Velcade, Pomalyst or Darzalex in patients with heavily pretreated multiple myeloma.

"Following our positive results for selinexor in the STORM study in patients with penta-refractory multiple myeloma, we are pleased to be presenting updated results of selinexor combinations in earlier lines of myeloma treatment," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "In the previously reported Phase 1b/2 STOMP study results, selinexor has shown robust anti-myeloma activity and a manageable safety profile in patients with relapsed or refractory myeloma after at least one prior therapy. The STOMP results not only provide the underlying clinical rationale for the ongoing pivotal Phase 3 BOSTON study evaluating selinexor in combination with once weekly Velcade, but also provide supportive data for selinexor as a potential new backbone therapy in combination with approved myeloma therapies, often with synergistic activity. We look forward to sharing these updated STOMP results from the Velcade, Pomalyst and Darzalex arms with the medical community at EHA (Free EHA Whitepaper) this year."

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

Title: Selinexor combined with pomalidomide and low dose dexamethasone (SPd) in a relapsed/refractory multiple myeloma patient population
Presenter:Christine Chen, University of Toronto, Princess Margaret Cancer Center
Final Abstract Code: PF586
Topic/Session Title: Myeloma and other monoclonal gammopathies – Clinical
Date and Time:Friday, June 15, 2018; 17:30 – 19:00 CEST
Location: Poster area

Title: Selinexor combined with low dose bortezomib and dexamethasone (SVd) induces a high response rate in patients with relapsed or refractory multiple myeloma (MM)
Lead author: Nizar Bahlis, Southern Alberta Cancer Research Institute
Final Abstract Code: PS1322
Topic/Session Title: Myeloma and other monoclonal gammopathies – Clinical
Date and Time: Saturday, June 16, 2018; 17:30 – 19:00 CEST
Location: Poster area

Title: A Phase 1b study using the combination of selinexor, daratumumab, and dexamethasone in multiple myeloma patients previously exposed to proteasome inhibitors and immunomodulatory drugs
Lead author:Cristina Gasparetto, Duke University
Final Abstract Code: PS1329
Topic/Session Title: Myeloma and other monoclonal gammopathies – Clinical
Date and Time: Saturday, June 16, 2018; 17:30 – 19:00 CEST
Location: Poster area

About Selinexor

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 2,400 patients have been treated with selinexor. In April 2018, Karyopharm reported positive top-line 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 plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) during the second half of 2018, with a request for accelerated approval for oral selinexor as a 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) and as a potential backbone therapy in combination with approved therapies (STOMP), and 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 one or more 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.