1stOncology™: Cancer Intelligence Service – Page 13902 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Cytokinetics Reports First Quarter 2019 Financial Results

On May 9, 2019 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the first quarter of 2019 (Press release, Cytokinetics, MAY 9, 2019, View Source [SID1234536034]). Net loss for the first quarter was $29.4 million, or $0.54 per share, compared to net loss for the first quarter of 2018 of $30.3 million, or $0.56 per share. Cash, cash equivalents and investments totaled $176.6 million at March 31, 2019.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"We are pleased with the progress made across our pipeline of muscle-directed investigational medicines during the first quarter of 2019," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "Recently, we shared encouraging data from FORTITUDE-ALS demonstrating consistency of effect for doses, endpoints and timepoints in patients treated with reldesemtiv and we believe the results may support progression to further clinical trials toward potential registration. We also passed through the first planned interim analysis of GALACTIC-HF and opened enrollment in METEORIC-HF while we also independently continued the conduct of the Phase 1 study of CK-274 and prepared for potential progression to Phase 2. Our strategy to advance multiple drug candidates, under our collaborations and independently, continues to generate upside potential for patients and shareholders."

Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Continued conduct of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil, following first planned interim analysis for futility. We expect screening in this event-driven trial to complete in the second quarter of 2019.

Opened METEORIC-HF, (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), the second Phase 3 trial of omecamtiv mecarbil, to enrollment. METEORIC-HF is a randomized, placebo-controlled, double-blind, parallel group, multicenter clinical trial designed to evaluate the effect of treatment with omecamtiv mecarbil compared to placebo on exercise capacity as determined by cardiopulmonary exercise testing (CPET) following 20 weeks of treatment. We expect to continue enrollment of METEORIC-HF throughout 2019.

John Teerlink, M.D., Professor of Clinical Medicine, University of California San Francisco and Director of Heart Failure, San Francisco Veterans Affairs Medical Center, presented additional results from COSMIC-HF (Chronic Oral Study of Myosin Activation to Increase Contractility in Heart Failure) at the American College of Cardiology’s 68th Annual Scientific Session. The results of a post hoc subgroup analysis of the COSMIC-HF data showed that, between 32 patients with atrial fibrillation (AF) and 117 patients without AF, there were no statistically significant differences in the effects of treatment with omecamtiv mecarbil on cardiac function, including systolic ejection time and stroke volume, as well as ventricular volumes, heart rate, and NT-proBNP.
AMG 594 (cardiac troponin activator)

Continued conduct of the Phase 1 study of AMG 594 to assess its safety, tolerability, pharmacokinetics and potential to increase cardiac function in healthy volunteers. AMG 594 is a novel, selective, oral, small molecule cardiac troponin activator, discovered under a joint research program with Amgen. This Phase 1 study is being conducted by Amgen in collaboration with Cytokinetics. We expect the conduct of this study to continue throughout 2019.
CK-3773274 (CK-274, cardiac myosin inhibitor)

Continued conduct of the Phase 1 double-blind, randomized, placebo-controlled, multi-part, single and multiple ascending dose clinical study of CK-274 in healthy adult subjects. CK-274 is a wholly-owned, novel cardiac myosin inhibitor, discovered by company scientists, in development for the potential treatment of hypertrophic cardiomyopathy (HCM). We expect results from this study in the third quarter of 2019 and are preparing for potential progression of CK-274 to Phase 2 in the second half of 2019.
Skeletal Muscle Program

reldesemtiv (next-generation fast skeletal muscle troponin activator (FSTA))

Results from FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS), the Phase 2 clinical trial of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS), were presented during a platform presentation at the American Academy of Neurology 71st Annual Meeting in Philadelphia on Sunday, May 5, 2019.

FORTITUDE-ALS did not achieve statistical significance for a pre-specified dose-response relationship in its primary endpoint of change from baseline in slow vital capacity (SVC) after 12 weeks of dosing (p=0.11). Similar analyses of ALSFRS-R and slope of the Muscle Strength Mega-Score yielded p values of 0.09 and 0.31, respectively. While the dose-response analyses for the primary and secondary endpoints did not achieve statistical significance at the level of 0.05, in a post-hoc analysis pooling the doses together, patients who received reldesemtiv in FORTITUDE-ALS declined less than patients who received placebo. The trial showed effects favoring reldesemtiv across dose levels and timepoints with clinically meaningful magnitudes of effect observed at 12 weeks for the primary and secondary endpoints. The differences between reldesemtiv and placebo in SVC and ALSFRS-R total score observed after 12 weeks of treatment were still evident at follow-up, four weeks after the last dose of study drug.
Pre-Clinical Development and Ongoing Research

Continued pre-clinical development of CK-3762601 (CK-601), a next-generation fast skeletal muscle troponin activator (FSTA), under our collaboration with Astellas.

Continued research in collaboration with Astellas directed to the discovery of next-generation skeletal muscle activators; Astellas is sponsoring Cytokinetics’ activities through 2019.

Continued independent research activities directed to our other muscle biology research programs.
Corporate

Joined the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to recognize Rare Disease Day, an international campaign elevating the public understanding of rare diseases.
Financials

Revenues for the first quarter of 2019 increased to $8.5 million from $5.3 million for the first quarter of 2018, primarily due to increased research and development revenues from our collaborations with Astellas and Amgen. License revenues in the first quarter of 2018 were related to the Phase 2 study of reldesemtiv in spinal muscle atrophy completed in 2018.

Research and development expenses for the first quarter of 2019 increased to $23.5 million from $22.1 million for the first quarter of 2018, primarily due to increased spending related to the opening of METEORIC-HF and development of CK-274, offset in part by reduced spending for reldesemtiv as well as for tirasemtiv, following suspension of development of tirasemtiv in late 2017. General and administrative expenses increased slightly to $9.4 million for the first quarter of 2019 from $9.3 million for the first quarter of 2018.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s first quarter 2019 results via a webcast and conference call today at 4:30 PM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 4486595.

An archived replay of the webcast will be available via Cytokinetics’ website until May 16, 2019. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 4486595 from May 9, 2019 at 7:30 PM Eastern Time until May 16, 2019.

TG Therapeutics to Host Conference Call on First Quarter 2019 Financial Results and Business Update

On May 9, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held, Friday, May 10, 2019 at 8:00 AM ET to discuss results for the first quarter of 2019 and provide a business outlook for the remainder of the year (Press release, TG Therapeutics, MAY 9, 2019, http://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-first-quarter-2019 [SID1234536009]). Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer will host the call.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics First Quarter 2019 Business Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for replay on the Company’s website, for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.

Spectrum Announces Asset Purchase and License for a Novel Immuno-Oncology Platform including Two Early Stage Assets

On May 9, 2019 Spectrum Pharmaceuticals, Inc. (NASDAQ-GS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported the closure of an asset purchase and license agreement with ImmunGene, Inc., a privately held biotechnology company (Press release, Spectrum Pharmaceuticals, MAY 9, 2019, View Source [SID1234536008]). The deal includes an exclusive license for the intellectual property related to the FIT antibody-interferon fusion technology drug delivery platform originally developed by scientists at UCLA.

The license also includes two novel assets derived from this platform. The first asset is an antibody-interferon fusion molecule directed against CD20 (Anti–CD20-IFNα). This drug candidate is in Phase 1 development for treating relapsed or refractory non-Hodgkin lymphoma, including diffuse large b-cell lymphoma patients where a considerable unmet medical need exists. Research for this program received financial support through the Therapy Acceleration Program of The Leukemia & Lymphoma Society, Inc. (LLS), and an LLS research grant to UCLA. The second asset is an antibody-interferon fusion molecule directed against GRP94, a target for which currently there are no existing approved therapies. It has the potential for treating both solid and hematologic malignancies.

"The FIT platform that we’ve acquired today represents a new class of biotherapeutics which may have the potential to make the administration of an antibody-interferon fusion protein feasible and allows Spectrum to harness this powerful immune activating cytokine," said Francois Lebel, M.D., F.R.C.P.C., Chief Medical Officer of Spectrum Pharmaceuticals. "In pre-clinical models, anti–CD20-IFNα has been shown to have significant proapoptotic activity. Anti–CD20-IFNα is essentially a 2-pronged attack whereby both CD20 and IFNAR signaling pathways can be activated to induce tumor cell apoptosis. Although IFNα has potent biologic activities against B-cell malignancies, its clinical utility has been curtailed by systemic toxicities. Preclinical results suggest that anti–CD20-IFNα fusion proteins have an improved therapeutic index, while still exhibiting the ability to eradicate tumor cells."

Originally developed by scientists at UCLA and licensed to Spectrum by UCLA Technology Development Group, the FIT platform fuses interferon with various monoclonal antibodies targeting various tumor antigens. Interferons are highly potent and well-established anticancer cytokines but have been associated with significant dose-related side effects. The FIT technology may be able to maintain the potency and efficacy of interferon while reducing toxicity traditionally associated with interferon therapy. FIT therapies have potential application as single agents or in combination with other therapies, such as checkpoint inhibitors.

"Adding a promising platform and two early stage assets is consistent with our commitment to serving the needs of cancer patients by developing innovative drugs for unmet need," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "We are aggressively building a robust oncology pipeline anchored by our late-stage assets, poziotinib and ROLONTIS."

Under the terms of the agreement, Spectrum will pay an upfront cash payment of approximately $3 million, up to $156 million in development and sales milestones, and royalties on net sales in the high-single digits.

Mersana Therapeutics Announces First Quarter 2019 Financial Results and Provides Business Updates

On May 9, 2019 Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported financial results and a business update for the first quarter ended March 31, 2019 (Press release, Mersana Therapeutics, MAY 9, 2019, View Source [SID1234536007]).

"We have continued to make important progress in advancing XMT-1536, our first-in-class ADC clinical candidate targeting NaPi2b. We are very pleased with the safety, efficacy, and duration of treatment seen to date in unselected and heavily pretreated ovarian cancer and NSCLC adenocarcinoma patients. This profile provides us with the confidence to move forward with expansion studies in these two indications with high unmet medical need. We look forward to presenting interim data from the dose escalation phase of the study at the upcoming ASCO (Free ASCO Whitepaper) 2019 annual meeting," said Anna Protopapas, President and CEO of Mersana Therapeutics. "We also continue to advance our earlier-stage ADC programs and remain on track to disclose our next clinical candidate in the fourth quarter of 2019. With our successfully completed financing, we have the resources necessary to drive these promising programs forward."

Recent Highlights and Updates

Clinical Program

· The Phase 1 dose escalation study of XMT-1536 for the treatment of NaPi2b-expressing cancers remains ongoing. XMT-1536 is a first-in-class Dolaflexin ADC targeting NaPi2b, which is broadly expressed in epithelial ovarian cancer and non-small cell lung cancer (NSCLC) adenocarcinoma. The data to date from the ongoing XMT-1536 dose escalation study indicate that the trial has reached clinically relevant dose levels, starting at 20 mg/m2 but has not yet reached a maximum tolerated dose. The Company continues to evaluate patients in the 36 mg/m2 once-every-four-week dosing cohort. Mersana plans to present data from the once-every-three-week dosing schedule and from patients dosed up to and including, the 30 mg/m2 dose cohort in the once-every-four-week dosing schedule, at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting in June 2019.

· Site initiation for the dose expansion portion of the XMT-1536 Phase 1 study is underway. Mersana is in the process of initiating additional sites in anticipation of dosing patients in two expansion groups in the third quarter of 2019. In the first group, the Company plans to enroll platinum-resistant ovarian cancer patients who have failed standard therapy. The second patient group will enroll NSCLC adenocarcinoma patients who have failed front line platinum-based chemotherapy as well as anti-PD-1 or anti-PDL-1 therapy.

Discovery & Platform Progress

· On track to disclose its next ADC clinical candidate in the fourth quarter of 2019, further strengthening its scientific leadership in ADC development. The Company is targeting the filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in the first half of 2020.

·Presented data on its novel Dolasynthen platform and modular Synthemer scaffold at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2019. Key details of the data presented are summarized below:

· The presentation, titled "Dolasynthen – A Novel, Homogeneous Auristatin F Hydroxypropyl Amide Antibody-Drug Conjugate Platform" provided an overview of Dolasynthen, Mersana’s novel, fully synthetic, structurally homogeneous drug conjugation platform with a tunable drug-to-antibody ratio (DAR) from 2 to 24. Precisely defined ADCs created for multiple targets displayed excellent drug-like properties, as well as potent activity, excellent tolerability, and a broad therapeutic index in preclinical in vivo models, demonstrating the significant potential for clinical application and differentiation.

·The poster, titled "An Antibody-Drug Conjugate Carrying a Microtubule Inhibitor and a DNA Alkylator Exerts Both Mechanisms of Action on Tumor Cells" characterized Mersana’s use of its modular Synthemer platform to engineer a dual-payload ADC to deliver two mechanistically distinct payloads simultaneously to each target cell. This data further demonstrates the flexibility of the modular Synthemer approach and its potential to address a broad set of set of applications.

Corporate Updates

· Successful completion of equity financing further strengthens balance sheet. On March 5, 2019, the Company announced the closing of a public offering with aggregate gross proceeds from the offering of approximately $97.8 million.

· Completed non-dilutive debt financing for additional financial flexibility. On May 8, 2019, the Company completed a non-dilutive debt financing with Silicon Valley Bank (SVB) that provides Mersana with the ability to draw up to $20.0 million, the proceeds of which will be used for general corporate and working capital purposes. The first tranche of $5.0 million was drawn down by the Company upon execution of the relevant Loan and Security Agreement, and under the terms of the Agreement, the Company has the option to draw additional advances over time. Further information describing the Agreement with SVB will be included in the Form 10-Q to be filed by Mersana with the Securities and Exchange Commission.

Upcoming Events

·The Company will present interim Phase 1 dose escalation clinical data for XMT-1536 at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 medical meeting on June 1, 2019 in Chicago, IL. The presentation format will be a poster, followed by a poster discussion.

2019 Financial Results

Cash, cash equivalents and marketable securities as of March 31, 2019, were $137.3 million, compared to $70.1 million as of December 31, 2018. On March 5, 2019 the Company completed a public equity offering with gross proceeds of $97.8 million. The Company expects that its cash, cash equivalents and marketable securities will enable it to fund its operating plan into at least mid-2021.

First Quarter 2019

·Collaboration revenue for the first quarter 2019 was approximately $41.0 million, compared to $3.1 million for the same period in 2018. The increase in collaboration revenue was primarily due to the recognition of the remaining $40.0 million in deferred revenue associated with the discontinuation of the XMT-1522 program and Takeda collaboration announced in January 2019.

·Research and development expenses for the first quarter 2019 were approximately $15.1 million, compared to $12.3 million for the same period in 2018, driven primarily by an increase in external costs for the manufacturing activities for XMT-1536 and the Company’s next clinical candidate as well as modest increases in headcount and facilities costs. The increase was offset by a decrease in external clinical and regulatory expenses due to the discontinuation of the XMT-1522 clinical program.

·General and administrative expenses for the first quarter 2019 were approximately $4.4 million, compared to $3.6 million for the same period in 2018, driven primarily by increased employee-related expenses due to an increase in headcount and increased professional fees.

· Net income for the first quarter 2019 was $21.9 million, or $0.72 per share, compared to a net loss of $12.4 million, or $0.54 per share, for the same period in 2018. Net income for the first quarter 2019 was driven by recognition of the remaining $40.0 million in deferred revenue associated with the discontinuation of the XMT-1522 program and Takeda collaboration announced in January 2019. Weighted average common shares outstanding for the quarters ended March 31, 2019 and March 31, 2018, were 30,299,650 and 22,816,521 respectively.

Conference Call

Mersana Therapeutics will host a conference call and webcast today at 8:00 a.m. ET to report financial results for the first quarter of 2019 and provide certain business updates. To access the call, please dial 877-303-9226 (domestic) or 409-981-0870 (international) and provide the Conference ID 1378664. A live webcast of the presentation will be available on the Investors & Media section of the Mersana website at www.mersana.com.

About XMT-1536

XMT-1536 is a Dolaflexin ADC targeting the sodium-dependent phosphate transport protein (NaPi2b) and is comprised of an average of 10-15 DolaLock payload molecules conjugated to XMT-1535, a proprietary humanized anti-NaPi2b antibody. NaPi2b is an antigen highly expressed in the majority of non-small cell lung cancer (NSCLC) adenocarcinoma and ovarian cancer. XMT-1536 is in Phase 1 clinical trials in patients with tumors expressing NaPi2b, including ovarian cancer, NSCLC adenocarcinoma and other cancers. More information on the ongoing Phase 1 clinical trial can be found at clinicaltrials.gov.

Karyopharm Reports First Quarter 2019 Financial Results and Highlights Recent Company Progress

On May 9, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the first quarter 2019 and provided an overview of recent accomplishments and clinical development progress for its innovative drug pipeline, including for selinexor, Karyopharm’s first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound (Press release, Karyopharm, MAY 9, 2019, View Source [SID1234536006]).

"It’s been an active first quarter of 2019, which included a U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) meeting to review our selinexor New Drug Application (NDA) and the subsequent extension of the Prescription Drug User Fee Act (PDUFA) action date by three months to July 6, 2019," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "We are working closely with the FDA while they complete their Priority Review of the selinexor NDA. Looking ahead to the remainder of 2019, we are highly focused on the potential U.S. commercial launch for selinexor. We are also continuing to advance our additional selinexor programs, including in earlier line multiple myeloma, diffuse large B-cell lymphoma, liposarcoma and endometrial cancer."

First Quarter 2019 and Recent Events

Selinexor in Multiple Myeloma

FDA Extends Review Period for NDA to July 6, 2019. On Feb 26, 2019, the FDA held an ODAC meeting to review the selinexor NDA requesting accelerated approval. The committee was specifically asked to vote on whether the approval of selinexor should be delayed until the results from the ongoing, randomized Phase 3 BOSTON study are available. In a vote of 8 Yes and 5 No, the ODAC recommended that the approval decision for selinexor should be delayed until the results of the BOSTON study are available. Following the ODAC meeting, and at the FDA’s request, Karyopharm submitted additional, existing clinical information as an amendment to the NDA. On March 14, 2019, the FDA extended the PDUFA action date for the selinexor NDA by three months to July 6, 2019. The NDA is currently under Priority Review by the FDA and is seeking accelerated approval for selinexor in combination with dexamethasone as a new treatment for patients based on the results of the Phase 2b STORM study in patients with triple class refractory multiple myeloma who were previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. Although the FDA will consider the recommendation of the ODAC panel, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.

European Medicines Agency (EMA) Validates Marketing Authorization Application (MAA). On January 8, 2019, Karyopharm submitted a MAA to the EMA requesting conditional approval for selinexor, in combination with dexamethasone, as a new treatment for patients based on the results of the Phase 2b STORM study in patients with triple class refractory multiple myeloma who were previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. As a customary part of the marketing application review process, Karyopharm received the consolidated list of questions from EMA in early May 2019 and anticipates receiving additional feedback based on routine site audits and other activities. Karyopharm plans to promptly address the questions and feedback with EMA. To provide adequate time to evaluate the application and allow Karyopharm to respond to questions and feedback, the EMA has switched from an accelerated review to a traditional review. The Company expects to receive a decision on the application by the end of 2019.

Pivotal Phase 3 BOSTON Study in Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is progressing and, in January 2019, the Company announced the completion of patient enrollment in the study. Top-line data is expected by the end of 2019 or into 2020 contingent upon the occurrence of progression-free survival (PFS) events, the primary endpoint of the study. The BOSTON study is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (bortezomib) (once weekly) and low dose dexamethasone (SVd), compared to standard twice weekly Velcade and low dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. Data from the BOSTON study, if positive, would be used to support regulatory submissions to the FDA and EMA requesting the use of selinexor in patients with multiple myeloma who received at least one prior therapy. Regulatory approvals based on the results from the BOSTON study would also confirm accelerated and/or conditional approvals based on data from the Phase 2b STORM study.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

NDA and MAA Expected to be Submitted in the First Half of 2020. Following the positive top-line results from the Phase 2b SADAL study which were presented at ASH (Free ASH Whitepaper) 2018, Karyopharm expects to submit an NDA to the FDA and MAA to the EMA requesting accelerated and conditional approval, respectively, for patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for stem cell transplantation including CAR-T (chimeric antigen receptor modified T cell) therapies. In addition to Orphan Drug Designation, selinexor was granted Fast Track designation by the FDA in 2018.
Selinexor in Solid Tumors

Phase 3 SIENDO Study Evaluating Selinexor as Maintenance Therapy in Endometrial Cancer Will Now Be Conducted as a Company Sponsored Study. During the first quarter of 2019, an Investigational New Drug Application (IND) was submitted and accepted by FDA for a randomized, blinded Phase 2/3 study evaluating selinexor versus placebo as a maintenance therapy in patients with advanced or recurrent endometrial cancer following one prior platinum-based treatment. There are currently no approved therapies to treat patients with advanced or recurrent endometrial cancer in the maintenance setting. The primary endpoint of the SIENDO study is PFS. This study was previously an investigator sponsored study and has subsequently transitioned to a company sponsored study led by Professor Ignace Vergote, MD, PhD, Head of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, Belgium. Karyopharm is targeting enrollment completion for SIENDO in 2020.

Ongoing Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma. Karyopharm previously reported positive results from the Phase 2 portion of the randomized, blinded Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. Enrollment is currently ongoing in the Phase 3 portion of the SEAL study and, assuming a positive outcome on the primary endpoint of PFS, the Company intends to use the data from the SEAL study to support NDA and MAA submissions requesting approval for selinexor for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated in 2020.
Eltanexor

Phase 1/2 Eltanexor Data in Metastatic Castrate-Resistant Prostate Cancer (mCRPC) Presented at ASCO (Free ASCO Whitepaper)-GU 2019. At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Genitourinary Cancers Symposium in February, Jingsong Zhang, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute, presented preliminary results from an ongoing Phase 1/2 investigator-sponsored study investigating eltanexor (+/- abiraterone) in patients with advanced cancers. The presented results showed that amongst the 23 patients with mCRPC evaluable for efficacy, 9% achieved a partial response and 74% achieved stable disease (≥8 weeks). The median treatment duration was 145 days, with three patients remaining on treatment as of November 19, 2018. Amongst the 30 patients evaluable for safety, treatment-related adverse events (TRAEs) occurring in ≥30% of patients included fatigue (80%; 17% Grade (Gr)≥3), nausea (70%; 0% Gr≥3), decreased appetite (60%; 0% Gr≥3), diarrhea (47%; 3% Gr≥3), weight decreased (47%; 3% Gr≥3), vomiting (40%; 7% Gr≥3), anemia (40%; 13% Gr≥3), neutropenia (33%; 13% Gr≥3), dysgeusia (33%; 0% Gr≥3) and thrombocytopenia (30%; 7% Gr≥3). There were two Gr4 TRAEs (neutropenia and elevated AST). Enrollment in the mCRPC arm of the study is now complete.
Verdinexor

$1.25 Million Grant from the U.S. Department of Defense (DoD) Awarded to Explore Use of Verdinexor in Spinal Cord Injury (SCI). A $1.25 million grant from the DoD was secured by an academic collaborator, with the assistance of Karyopharm, to study verdinexor (KPT-335) in preclinical models of SCI. The goal of this research is to establish the extent to which oral administration of verdinexor promotes tissue preservation and recovery of function following spinal cord injury. Verdinexor is also being studied as a potential anti-viral agent in a variety of human viral indications and for the treatment of systemic lupus erythematosus (SLE). For SLE, a $2.0 million grant was awarded from the National Institute of Health in 2018 and research is ongoing. The purpose of this grant is to investigate oral verdinexor activity in models of B-cell generation, double-stranded DNA antibody levels, and persistence of self-reactive antibody secreting cells.
Medical Congress Activity and Presentation of Data

Two Selinexor Abstracts Selected for Presentation at ASCO (Free ASCO Whitepaper) 2019. Two selinexor abstracts have been selected for presentations at the upcoming ASCO (Free ASCO Whitepaper) 2019 Annual Meeting taking place May 31-June 4, 2019 in Chicago. The first abstract, titled "Efficacy and Safety of Selinexor in Recurrent Glioblastoma," (Lassman, et al; Abstract #2005) was selected for an oral presentation and describes results from the Phase 2 KING study evaluating single-agent selinexor in patients with recurrent glioblastoma. The second abstract, titled "Overall Survival (OS) with Oral Selinexor Plus Low Dose Dexamethasone (Sd) in Patients with Triple Class Refractory-Multiple Myeloma (TCR-MM)," (Richardson, et al; Abstract #8014; Poster Board #340) was selected for a poster presentation and highlights overall survival (OS) data from the Phase 2b STORM study evaluating selinexor and low-dose dexamethasone in patients with triple class refractory multiple myeloma who have been previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available.

Seven Preclinical Abstracts Presented at AACR (Free AACR Whitepaper) 2019. Seven posters highlighting preclinical data for selinexor were presented at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2019 Annual Meeting. These posters described research evaluating selinexor in various preclinical models of high unmet need cancers, including multiple myeloma, chronic lymphocytic leukemia, breast, pancreatic, lung and metastatic brain cancers. A complete list of these presentations can be accessed by visiting the AACR (Free AACR Whitepaper) 2019 website at View Source
Corporate Updates

Michael P. Mason Appointed Chief Financial Officer. Karyopharm appointed Michael P. Mason as Chief Financial Officer. Mr. Mason formerly served as Vice President of Finance and Treasurer at Alnylam Pharmaceuticals, Inc., a public biopharmaceutical company. He brings over 18 years of diversified financial experience to Karyopharm and has extensive expertise in global financial operations and controls, financing transactions, business planning and supporting pharmaceutical product launches.

Tina Clark Beamon, Esq., Appointed Chief Compliance Officer. Karyopharm appointed Tina Clark Beamon, Esq. as Chief Compliance Officer. Ms. Beamon formerly served as Executive Director of Compliance and Ethics at Alexion Pharmaceuticals. Prior to Alexion, she served as the head attorney for the Oncology Division and the Consumer Healthcare Division at Boehringer Ingelheim USA Corporation. She brings 21 years of healthcare industry experience to Karyopharm and will serve an integral role in building upon Karyopharm’s existing high operating standards and its ongoing commitment to corporate compliance and ethics.
First Quarter 2019 Financial Results

Cash, cash equivalents and investments as of March 31, 2019, including restricted cash, totaled $265.1 million, compared to $330.9 million as of December 31, 2018.

License and other revenue for the quarter ended March 31, 2019 was $0.2 million, compared to $10.0 million for the quarter ended March 31, 2018. The revenue in 2018 was from the $10.0 million upfront payment for the asset sale of KPT-350 to Biogen in the first quarter of 2018.

For the quarter ended March 31, 2019, research and development expense was $38.0 million compared to $41.3 million for the quarter ended March 31, 2018. Karyopharm expects research and development expense to decrease going forward in 2019 compared to the first quarter of 2019. For the quarter ended March 31, 2019, general and administrative expense was $27.1 million compared to $7.6 million for the quarter ended March 31, 2018. The increase in general and administrative expenses compared to the prior year period was due primarily to the hiring of the Karyopharm commercial team and related commercial launch preparation activities to support the potential U.S. commercial launch of selinexor.

Karyopharm reported a net loss of $66.2 million, or $1.09 per share, for the quarter ended March 31, 2019, compared to a net loss of $38.5 million, or $0.78 per share, for the quarter ended March 31, 2018. Net loss includes stock-based compensation expense of $3.9 million and $4.2 million for the quarters ended March 31, 2019 and March 31, 2018, respectively.

Financial Outlook

Based on its current operating plans, which assume a selinexor commercial launch by July 2019, Karyopharm expects its operating expenses, excluding stock-based compensation expense, for the full year 2019 to be in the range of $200 million to $215 million. The Company expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the second half of 2020, also assuming the commercial launch of selinexor by July 2019. If the FDA decides to delay its approval decision for selinexor until the BOSTON data are available, Karyopharm will re-evaluate its spending expectations for 2019. Additional key activities expected in 2019 include supporting the ongoing multiple myeloma regulatory filings for selinexor in the U.S. and Europe, progressing the pivotal Phase 3 BOSTON study in multiple myeloma and preparing for NDA and MAA submissions in the U.S. and Europe, respectively, in DLBCL.

Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma

The FDA instituted its Accelerated Approval Program to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint or an intermediate clinical endpoint thought to predict clinical benefit, like overall response rate. Accelerated approval is available only for drugs that provide a meaningful therapeutic benefit over existing treatments at the time of consideration of the application for accelerated approval, which the FDA has reiterated in its feedback to the Company. Particularly in disease areas with multiple available and potential new therapies, such as multiple myeloma, accelerated approval carries a high regulatory threshold. Consistent with its general guidance, the FDA has noted to the Company its preference for randomized studies geared toward full approval, which the Company has undertaken with the ongoing pivotal, Phase 3 BOSTON study, and has reminded the Company that accelerated approval requires patients to have exhausted all available approved therapies.

Conference Call Information

Karyopharm will host a conference call today, Thursday, May 9, 2019, at 8:30 a.m. Eastern Time, to discuss the first quarter 2019 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 9756776. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.