On March 14, 2016 Vericel Corporation (NASDAQ: VCEL), a leading developer of patient-specific expanded cellular therapies for the treatment of severe diseases and conditions, reported financial results and business highlights for the fourth quarter and year ended December 31, 2015 (Filing, Q4/Annual, Vericel, 2015, MAR 14, 2016, View Source [SID:1234510447]).

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!

Recent Business Highlights

During and since the fourth quarter of 2015, the company:
• Announced positive top-line results from the Phase 2b ixCELL-DCM clinical trial of ixmyelocel-T in patients with heart failure due to ischemic dilated cardiomyopathy;

• Received U.S. Food and Drug Administration (FDA) approval of the Epicel (cultured epidermal autografts) HDE supplement, which revised the Epicel product label to include pediatric patients and specify the probable survival benefit for adult and pediatric patients treated with Epicel, and allows the company to sell Epicel for profit on up to 360,400 grafts per year;

• Announced that the FDA has accepted for filing the BLA for MACI (matrix applied characterized autologous cultured chondrocytes), the company’s investigational third-generation autologous cultured chondrocyte implant intended for the treatment of symptomatic full-thickness cartilage defects of the knee in adult patients;

• Announced a long-term supply agreement with Matricel GmbH for the collagen membrane used in the production of MACI;

• Achieved 14% growth in total Carticel (autologous cultured chondrocytes) and Epicel net product revenues for 2015 over pro-forma Carticel and Epicel revenues for 2014, and 5% growth in total Carticel and Epicel net product revenues in the fourth quarter versus the fourth quarter of 2014;

• Achieved 60% and 24% growth in Epicel net product revenues for 2015 and the fourth quarter, respectively, versus the same periods in 2014; and

• Entered into a $10 million credit facility and $5 million term loan agreement with Silicon Valley Bank.

"2015 was an extremely productive year during which we completed our corporate transformation into a sustainable and growing commercial enterprise, substantially increased revenues and gross margins, and made significant progress on our clinical and regulatory objectives that we expect will drive current and long-term growth for the company," said Nick Colangelo, president and CEO of Vericel. "We believe that we have positioned the company as one of the leading cell therapy and regenerative medicine companies in the industry."

Financial Highlights

Total revenues for the fourth quarter and year ended 2015 were generated primarily from net sales of Carticel implants and surgical kits and Epicel, which were acquired on May 30, 2014 as part of the acquisition of Sanofi’s cell therapy and regenerative medicine business.

Total net revenues for the quarter ended December 31, 2015 were approximately $15.4 million and included approximately $11.3 million of net sales of Carticel implants and surgical kits and approximately $4.1 million of net sales of Epicel. Total Carticel and Epicel net product revenues in the fourth quarter increased approximately 5% over the same period in 2014.

Total net revenues for the year ended December 31, 2015 were approximately $51.2 million, including approximately $35.2 million of net sales of Carticel implants and surgical kits and approximately $15.2 million of net sales of Epicel. Total Carticel and Epicel net product revenues for 2015 increased approximately 14% over pro-forma Carticel and Epicel net product revenues for 2014. Total revenues for the quarter and year ended December 31, 2015 included approximately $0.1 million and $0.7 million of sales, respectively, from our Marrow Donation business which ceased operations in December, 2015.

Gross profit for the quarter and year ended December 31, 2015 was $8.2 million, or 53% of net product sales, and $24.7 million, or 48% of net product sales, respectively, compared to $8.0 million, or 54% of net product sales, and $11.5 million, or 40% of net product sales, for the quarter and year ended December 31, 2014, respectively.

Research and development expenses for the quarter and year ended December 31, 2015 were $7.4 million and $18.9 million, respectively, versus $5.8 million and $21.3 million for the same periods in 2014. The increase in research and development expenses in the fourth quarter is primarily due to additional research, development and regulatory costs incurred for the Biologics License Application (BLA) for MACI and Humanitarian Device Exemption (HDE) supplement submitted in December 2015 to revise the labeled indications for use of Epicel, which included $2.2 million in regulatory consulting expenses and a Prescription Drug User Fee Act (PDUFA) filing fee of $2.4 million paid in the fourth quarter of 2015.

The decrease in full-year research and development expenses is primarily due to a reduction in expenses associated with the ixCELL-DCM clinical trial, which completed enrollment in January 2015, and other clinical trial expenses, and a $3.2 million payment in 2014 to the former shareholders of Verigen pursuant to a settlement agreement that eliminated all future milestone payments related to the development and commercialization of MACI in the United States.

Selling, general and administrative expenses for the quarter and year ended December 31, 2015 were $5.7 million and $22.5 million, respectively, compared to $4.5 million and $13.8 million for the same periods in 2014. The increase in SG&A expenses is primarily due to Vericel being a commercial business for all of 2015 compared to only seven months in 2014, as well as an increase in sales and marketing expenses associated with Carticel and Epicel and strategic planning activities for MACI.

Loss from operations for the quarter and year ended December 31, 2015 was $5.0 million and $16.7 million, respectively, compared to $2.3 million and $23.5 million for the same periods a year ago. The operating loss for the quarter ended December 31, 2015 included $2.2 million for MACI BLA and Epicel HDE supplement regulatory consulting expenses and a $2.4 million PDUFA filing fee for MACI. Excluding these one-time expenses, the company would have had an adjusted operating loss of $0.4 million in the fourth quarter. Material non-cash items impacting the operating loss for the quarter and year included $0.6 million and $2.7 million, respectively, of stock-based compensation expense and $0.4 million and $1.6 million, respectively, in depreciation and amortization expense.

Other income (expense) for the quarter and year ended December 31, 2015 was less than $0.1 million and $0.3 million, respectively, compared to less than ($0.1) million and $3.6 million for the same periods in 2014. The change in other income for the quarter is primarily due to a decrease in the fair value of warrants in the fourth quarter of 2015 compared to the same period in 2014. The decrease in other income for the full year 2015 is primarily due to a bargain purchase gain of $3.5 million recognized in 2014 and a decrease in the fair value of warrants in 2015 compared to 2014.

Vericel reported a net loss for the quarter and year ended December 31, 2015 of $4.9 million, or $0.28 per share, and $16.3 million, or $0.97 per share, respectively, compared to a net loss of $2.4 million, or $0.17 per share, and $19.9 million, or $2.23 per share, for the same periods in 2014.

As of December 31, 2015, the company had $14.6 million in cash and cash equivalents compared to $30.3 million in cash and cash equivalents at December 31, 2014.

On March 14, 2016 Immunomedics, Inc., (Nasdaq:IMMU) reported that the Company is terminating the Phase 3 PANCRIT-1 trial with yttrium-90-labeled (90Y) clivatuzumab tetraxetan in patients with metastatic pancreatic cancer who had received at least two prior therapies, one of which must have been a gemcitabine-containing regimen (Filing, 8-K, Immunomedics, MAR 14, 2016, View Source [SID:1234509542]).

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!

The decision to terminate the trial early is based on the recommendation from the independent Data and Safety Monitoring Board (DSMB), following a planned interim analysis of data on overall survival (OS) after more than 50% of the required 371 deaths had occurred. The interim analysis showed that the treatment arm of 90Y-clivatuzumab tetraxetan combined with low-dose gemcitabine and best supportive care did not demonstrate a sufficient improvement in OS as compared to placebo plus low-dose gemcitabine and best supportive care.

"Given the significant unmet medical need in pancreatic cancer, we are disappointed that clivatuzumab tetraxetan did not produce the desired outcome and will review our future strategy for this antibody with key opinion leaders," remarked Cynthia L. Sullivan, President and Chief Executive Officer. "We would like to thank our clinical investigators and their patients and families for participating in this study. Pancreatic cancer is a challenging disease to treat but we remain fully committed to bringing innovative products, such as IMMU-132 (antibody-drug conjugate, sacituzumab govitecan) and (E1)-3s (T-cell redirecting immuno-oncology bispecific antibody), to address this unmet need," Ms. Sullivan added. "Both these other products have shown activity in pancreatic cancers, either clinically (IMMU-132) or preclinically [(E1)-3s]," Ms. Sullivan commented further.

On March 14, 2016 Oncothyreon Inc. (NASDAQ:ONTY), a clinical-stage biopharmaceutical company dedicated to the development of therapeutic products that can improve the lives and outcomes of patients with cancer, reported a corporate update and reported financial results for the year and quarter ended December 31, 2015 (Press release, Oncothyreon, MAR 14, 2016, View Source [SID:1234509544]).

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!

"2015 was a significant year for Oncothyreon, with the company reporting encouraging data from two combination trials of ONT-380 demonstrating that this product candidate may be impactful on HER2-positive breast cancer patients, including those with brain metastases – a patient population in desperate need of new treatment options," said Christopher S. Henney, Chairman and interim CEO of Oncothyreon. "We believe our clinical results to date provided a strong foundation for us to advance ONT-380 into our recently initiated randomized, double-blind, placebo-controlled Phase 2 trial in combination with Herceptin and Xeloda, which includes enrolling patients with progressing central nervous system disease."

Corporate Update & Recent Highlights

Clinical Development:

ONT-380 Phase 2 Combination Trial Underway in Patients with HER2-Positive Breast Cancer. The randomized, double-blind, placebo control trial is evaluating ONT-380 in combination with Herceptin (trastuzumab) and Xeloda (capecitabine). ONT-380 is an oral, HER2-selective, central nervous system (CNS)-active tyrosine kinase inhibitor. The trial is targeted to enroll approximately 180 heavily pretreated patients with advanced HER2-positive breast cancer who present with or without brain metastases. Building on encouraging Phase 1b results, the primary and secondary endpoint objectives are designed to measure ONT-380’s contribution on both systemic and CNS disease, an area of unmet need for patients.

Data from Ongoing ONT-380 Phase 1b Combination Trials Show Objective, Durable Responses and Favorable Tolerability Profile. During 2015, data from two ongoing trials of ONT-380 were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the San Antonio Breast Cancer Symposium (SABCS).

ONT-380 in combination with Xeloda and/or Herceptin:
This Phase 1b trial enrolled patients with metastatic HER2-positive breast cancer with progression following prior treatment with Herceptin and Kadcyla (ado-trastuzumab emtansine or T-DM1). In addition to patients without a prior history of CNS disease, patients with untreated, asymptomatic CNS disease and patients with progressing CNS disease after prior local therapy were allowed to enroll in the trial and were followed for responses both systemically and in the CNS. Overall, ONT-380 in combination with Xeloda, Herceptin or both Xeloda and Herceptin has been well tolerated. As reported at ASCO (Free ASCO Whitepaper) 2015, in a total of 32 patients treated with these combinations, the majority of adverse events were Grade 1 or 2 in severity, with no reported Grade 3 diarrhea. As reported at SABCS 2015, the objective response rate across treatment groups was 42 percent and the CNS response rate was 33 percent, providing encouraging data to support moving forward with a follow-on Phase 2 study of ONT-380 in combination with Xeloda and Herceptin.

ONT-380 in combination with Kadcyla:
This Phase 1b trial enrolled patients with metastatic HER2-positive breast cancer with progression following prior treatment with Herceptin and a taxane. Patients may have received prior treatment with Perjeta (pertuzumab) and Tykerb (lapatinib). Patients with or without brain metastases were eligible. This trial has completed enrollment, but is ongoing with patients continuing to receive treatment. Clinical data from this trial were presented at SABCS 2014 and 2015, and at ASCO (Free ASCO Whitepaper) 2015. Overall, the combination of ONT-380 and Kadcyla was clinically well tolerated in 50 patients treated at the maximum tolerated dose of ONT-380, with the majority of adverse events either Grade 1 or Grade 2 in severity. Grade 3 diarrhea was reported in 4 percent of patients. Durable ( > 6 months) systemic and CNS responses and disease stabilization were seen, with an objective response rate of 41 percent and a CNS response rate of 33 percent.

Internal Research & Discovery Collaborations:

Research Collaboration Moving Chk1 Inhibitors Forward. During 2015, Oncothyreon continued preclinical activities to develop a small molecule against the checkpoint kinase 1 (Chk1) target in collaboration with Sentinel Oncology. IND-enabling studies are expected to begin in 2017.

Protocell Research Program Progressing. During 2015, Oncothyreon continued research on protocells, a novel nanoparticle platform technology that may enable the targeted delivery of a variety of therapeutic agents.

Leadership:

Expanded Leadership Team, CEO Search Underway. In January 2016, Oncothyreon announced the appointment of Christopher S. Henney, the company’s chairman, as interim CEO. The company’s comprehensive search for a new CEO is actively ongoing. Additionally, Oncothyreon appointed three new members to its Board of Directors: Steven P. James in March 2015 and Mark Lampert and Gwen Fyfe, M.D. in January 2016.

Full Year and Fourth Quarter 2015 Financial Highlights

Cash, cash equivalents and investments totaled $56.4 million as of December 31, 2015, compared to $63.7 million at December 31, 2014, a decrease of $7.3 million, or 11.5%. The decrease was primarily attributable to $28.9 million of cash used in operations during the year ended December 31, 2015, partially offset by the net proceeds of $22.4 million from the closing of concurrent but separate underwritten offerings of common stock and Series B convertible preferred stock in February 2015.

Research and development expenses for the fourth quarter of 2015 decreased by $19.1 million to $6.9 million from $26.0 million in the fourth quarter of 2014. Full year research and development expenses decreased by $18.4 million to $23.5 million in 2015 from $41.9 million in 2014. The fourth quarter and full year decrease in expense was primarily the result of the one-time upfront payment made to Array Biopharma in December 2014.

General and administrative expenses for the fourth quarter of 2015 increased by $0.4 million to $2.3 million, from $1.9 million in the fourth quarter of 2014. Full year 2015 general and administrative expenses were $9.3 million, an increase of $0.3 million from $9.0 million in 2014. This increase was primarily due to patent expenses related to our product candidates.

Net loss for the year ended December 31, 2015 was $32.6 million, or $0.34 per basic and diluted share, compared with a net loss of $50.0 million, or $0.64 per basic and diluted share, for the comparable period in 2014. Net loss for the three months ended December 31, 2015 was $9.1 million, or $0.10 per basic and diluted share, compared with a net loss of $27.6 million, or $0.30 per basic and diluted share, for the comparable period in 2014. The decrease in net loss for the year and quarter was primarily attributable to a $20.0 million upfront payment Oncothyreon made to Array BioPharma upon entering into an exclusive license agreement in December 2014. The decrease in net loss was partly offset by slightly higher general and administrative expenses and lower non-cash income from the change in the fair value of our warrant liability.

Financial Guidance

Oncothyreon believes the following financial guidance to be correct as of the date provided. Oncothyreon is providing this guidance as a convenience to investors and assumes no obligation to update it.

Oncothyreon currently expects operating expenses in 2016 to be higher than in 2015. This increase will primarily be related to expenditures associated with the Phase 2 trial of ONT-380. Oncothyreon currently expects cash used in operations in 2016 to be approximately $38.0 million to $40.0 million. With cash, cash equivalents and investments of $56.4 million as of December 31, 2015, Oncothyreon estimates that its cash, cash-equivalents and investments will be sufficient to fund operations for at least the next 12 months.

On March 14, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended December 31, 2015 (Press release, Stemline Therapeutics, MAR 14, 2016, View Source [SID:1234509512]).

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!

Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "During the fourth quarter, we made significant progress across all three of our development programs. Key achievements included 1) generating and presenting data from our ongoing Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) which we have designed to support potential registration, 2) treating our first patient in the second stage of our Phase 2 program of SL-701 in advanced brain cancer, and 3) the opening of our IND for SL-801 on schedule. At the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December, we presented very strong data from our ongoing SL-401 trial in BPDCN, demonstrating a high overall response rate in BPDCN, with multiple complete responses. We are also very pleased with the pace of enrollment in BPDCN, and note that we are tracking in-line with our expectations. We look forward to sharing key clinical and regulatory updates on SL-401 throughout this year."

Dr. Bergstein continued, "We continue to evaluate SL-401 in several additional malignancies that overexpress IL-3R. Specifically, we have single agent trials enrolling AML patients with minimal residual disease and patients with advanced high-risk myeloproliferative neoplasms. We also expect our first clinical studies that combine SL-401 with other therapies will begin this year."

Dr. Bergstein concluded, "Our other pipeline candidates, SL-701 and SL-801, continue to advance. Our SL-701 trial is currently enrolling patients and we expect updates from this program in the second half of the year. The SL-801 IND is open, and we are on track to treat our first patient this quarter. With our strong cash position, we believe we have sufficient financial resources to achieve significant clinical and regulatory milestones this year and beyond."

Fourth Quarter 2015 Financial Results Review

Stemline ended the fourth quarter of 2015 with $97.5 million in cash, cash equivalents and investments, as compared to $104.0 million as of September 30, 2015, which reflects a cash burn of $6.5 million for the quarter. The company ended the fourth quarter of 2015 with 18.2 million shares outstanding.

For the fourth quarter of 2015, Stemline had a net loss of $10.2 million, or $0.58 per share, compared with a net loss of $6.9 million, or $0.53 per share, for the same period in 2014. The net loss for full year 2015 was $37.2 million, or $2.15 per share, as compared with a net loss of $28.8 million, or $2.23 per share, in the prior year.

Research and development expenses were $7.9 million for the fourth quarter of 2015, which reflects an increase of $2.8 million, or 56%, compared with $5.1 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to the ramp up in our clinical trial activities for SL-401 and SL-801.

General and administrative expenses were $2.6 million for the fourth quarter of 2015, which reflects an increase of $0.6 million, or 30%, compared with $2.0 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to an increase in compensation expense relating to administrative employees.

On March 14, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the fourth quarter and full year 2015 and commented on recent accomplishments and clinical development plans for its pipeline of several SINE-based therapeutics including selinexor, its lead product candidate (Press release, Karyopharm, MAR 14, 2016, View Source [SID:1234509521]).

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!

"During 2015, we continued to expand our leadership position in the development of oral SINE-based oncology and non-oncology therapies with clinical advancement of selinexor across a number of hematologic and solid tumor indications and the presentation of encouraging data on our earlier-stage pipeline programs," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "Our broad and aggressive oncology clinical development program continues in 2016 with both company- and investigator-sponsored trials underway or planned to evaluate selinexor both as a single-agent and in combination with other existing and emerging oncology therapies. In addition, we made important progress with our non-oncology focused SINE compounds in a number of disease areas in which XPO1 is critically involved. We look forward to several potentially value-creating data readouts over the next 18 months."

Clinical Development Plans:
Selinexor in hematologic malignancies. Karyopharm is actively enrolling patients in several later phase clinical studies evaluating single-agent selinexor in hematologic malignancies, including patients with relapsed/refractory multiple myeloma (MM) (STORM study), older patients with relapsed/refractory acute myeloid leukemia (AML) (SOPRA study) and patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) (SADAL study). Karyopharm expects data from the first 80 patients in the STORM study to be available in mid-2016, at which point, it will evaluate whether to expand the study. Karyopharm also expects to provide an interim analysis update from the SOPRA study in late-2016 with top-line data expected in mid-2017. Based on discussions with the U.S. Food and Drug Administration (FDA), Karyopharm amended the protocol for the ongoing SADAL study to remove dexamethasone from the regimen and to evaluate selinexor as a single-agent in this patient population. The study will continue to compare 60 mg and 100mg twice weekly doses of selinexor with 100 patients per arm and at least 50% of patients with GCB-type DLBCL. Based on these amendments, the company expects to report top-line data from this study in early 2017.

Single-agent selinexor in solid tumors. Karyopharm is currently conducting company-sponsored trials of single-agent selinexor in three solid tumor indications including advanced unresectable dedifferentiated liposarcoma (SEAL study), heavily pretreated patients with gynecologic malignancies (SIGN study) and recurrent glioblastoma multiforme (KING study). Karyopharm is planning to present updated data from the SIGN and KING studies at an appropriate medical meeting in mid-2016. The Phase 2/3 SEAL study, evaluating single-agent oral selinexor versus placebo, was initiated earlier this year based on promising clinical data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Annual Meeting in which durable stable disease and improvement in progression free survival compared to previous chemotherapies were observed. Top-line data from the Phase 2 portion of this study are expected in mid-2017.

Selinexor combinations. A number of investigator-sponsored and company-sponsored trials evaluating selinexor in combination with either chemotherapy or targeted agents in hematologic and solid tumor indications are currently ongoing or planned. In mid-2016, Karyopharm plans to initiate a Phase 2/3 study (SCORE study) evaluating the combination of selinexor, carfilzomib and dexamethasone in patients with refractory MM who were previously treated with a proteasome inhibitor and an immunomodulatory agent. In addition, Karyopharm expects to report top line data from the Phase 1 portion of a Phase 1b/2 study (STOMP study) evaluating selinexor in combination with commonly used treatments, including bortezomib, pomalidomide, or lenalidomide, for relapsed/refractory MM in late-2016.

KPT-8602, second generation SINE compound. In January 2016, Karyopharm initiated a Phase 1/2 study of oral KPT-8602, a novel, second generation, SINE compound, in patients with relapsed/refractory MM. Data from preclinical studies presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated single-agent anti-MM activity of KPT-8602 along with the potential for higher and/or more frequent dosing compared with selinexor. Top-line safety and tolerability data from this Phase 1/2 study are expected in late 2016.

KPT-9274, oral dual inhibitor of PAK4 and NAMPT. In mid-2016, Karyopharm plans to initiate clinical development in patients with heavily pretreated solid tumors or lymphoma for its oral dual inhibitor of PAK4 and NAMPT, KPT-9274.

Verdinexor (KPT-335). In May 2015, Karyopharm began clinical testing of verdinexor in a randomized, double-blind, placebo-controlled, dose-escalating Phase 1 clinical trial in healthy human volunteers in Australia. Verdinexor was found to be generally safe and well tolerated. Karyopharm is preparing to advance verdinexor for certain viral indications with an initial focus on influenza. Preclinical data provide strong support for other potential indications for verdinexor, including human immunodeficiency virus (HIV).
KPT-350. KPT-350 is an investigational new drug application-ready oral compound with a preclinical data package supporting potential efficacy in a number of neurological, autoimmune and inflammatory conditions. We plan to partner with a collaborator to undertake the clinical development and potential commercialization of KPT-350 in one or more mutually agreed indications.

Scientific Presentations and Publications:
Positive clinical data demonstrating the activity and tolerability of selinexor across multiple hematologic malignancies including MM and AML were presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting held in December 2015 in Orlando, Florida.

Updated data from an ongoing investigator-sponsored trial of selinexor in combination with carfilzomib and dexamethasone in refractory MM were presented by Andrzej Jakubowiak, MD, PhD, from The University of Chicago. The data demonstrated encouraging efficacy of selinexor in combination with carfilzomib and dexamethasone in heavily pretreated patients with highly refractory MM, including patients whose disease is refractory to previous carfilzomib-based combinations, suggesting the potential of this combination to overcome carfilzomib resistance. Based on these data, Karyopharm expects to initiate a Phase 2/3 clinical study (SCORE study) in mid-2016.

Karyopharm researchers and collaborators also presented preclinical data demonstrating the potential of selinexor to synergize with proteasome inhibitors to overcome drug resistance in MM.

Karyopharm presented data demonstrating the activity and tolerability of selinexor in combination with standard of care agents in the AML setting, including data from a Phase 2 trial investigating the efficacy and tolerability of selinexor in combination with arabinoside cytosine (Ara-C) and idarubicin in "fit" patients with relapsed and/or refractory AML. Karyopharm also presented data from a Phase 1 study determining the safety and efficacy of selinexor in combination with fludarabine and cytarabine in pediatric patients with relapsed and/or refractory acute leukemia.

Karyopharm researchers presented data establishing 60mg as the most appropriate selinexor dose for both efficacy and tolerability across many hematologic and solid cancers. These data support the selinexor first-in-human Phase 1 clinical trial solid tumor data published in Journal of Clinical Oncology in March 2016, which recommended a dose of 60mg given twice a week as the Phase 2 dose. This recommendation was based on superior patient tolerability, longer duration of therapy, and no demonstrable improvement in radiologic response or disease stabilization compared with higher doses.

Fourth Quarter and Year Ended December 31, 2015 Financial Results

Cash, cash equivalents and investments as of December 31, 2015, including restricted cash, totaled $210.0 million, compared to $214.8 million as of December 31, 2014.

For the year ended December 31, 2015, research and development expense was $97.7 million compared to $60.1 million for the year ended December 31, 2014. For the year ended December 31, 2015, general and administrative expense was $21.6 million compared to $15.9 million for the year ended December 31, 2014. The increase in research and development expense resulted primarily from the increase in expenses related to the continued clinical development of selinexor. The increase in general and administrative expense resulted primarily from an increase in personnel costs, including stock-based compensation expense.

Karyopharm reported a net loss of $118.2 million, or $3.32 per share, for the year ended December 31, 2015, compared to a net loss of $75.8 million, or $2.43 per share, for the year ended December 31, 2014. Net loss includes stock-based compensation expense of $17.1 million and $14.2 million for the years ended December 31, 2015 and 2014, respectively.

For the quarter ended December 31, 2015, research and development expense was $24.1 million compared to $20.0 million for the quarter ended December 31, 2014. For the quarter ended December 31, 2015, general and administrative expense was $5.3 million compared to $5.9 million for the quarter ended December 31, 2014. The increase in research and development expenses resulted primarily from the increase in expenses related to the continued clinical development of selinexor.

Karyopharm reported a net loss of $29.0 million, or $0.81 per share, for the quarter ended December 31, 2015, compared to a net loss of $25.9 million, or $0.79 per share, for the quarter ended December 31, 2014. Net loss includes stock-based compensation expense of $5.4 million and $4.6 million for the quarters ended December 31, 2015 and December 31, 2014, respectively.

Financial Outlook
Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will fund its research and development programs and operations into the middle of 2018, including advancing the four later-stage clinical studies to their next data inflection points. Karyopharm expects to end 2016 with at least $120 million in cash, cash equivalents and investments.