On March 13, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the fourth quarter and full year ended December 31, 2017 (Press release, Bellicum Pharmaceuticals, MAR 13, 2018, View Source [SID1234524726]).

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"In the past year, we made substantial progress toward our vision of delivering cures through controllable cell therapy," said Bellicum’s President & CEO Rick Fair. "We completed enrollment in our first registrational trial of BPX-501 and remain on track for our first filing for product approval in Europe in 2019. We moved three new projects featuring our industry-leading cellular control technology into clinical trials, including the first-ever GoCAR-T with our iMC activation switch. We also made substantial preclinical progress on our next generation ‘dual-switch’ platform containing both activation and safety switches in the same CAR-T cell, and initiated plans to move two dual-switch CAR-T projects into clinical trials in 2019."

2017 HIGHLIGHTS AND CURRENT UPDATES

Bellicum Submits Response to FDA Clinical Hold on BPX-501 Trials
Last week, the Company submitted a full response to the FDA clinical hold notification, including requested changes to study protocols to provide guidelines for comprehensive monitoring and management of neurologic adverse events associated with hematopoietic stem cell transplants. The Company expects the response to satisfy the conditions for removal of the clinical hold, which applies to BPX-501 clinical trials in the U.S.

Recruitment Complete in BPX-501 E.U. Registration Trial
The Company completed enrollment in the treatment arm of its BP-004 E.U. registration trial in pediatric patients undergoing haploidentical hematopoietic stem cell transplant (haplo-HSCT) and expects to report updated data from this trial at upcoming medical meetings. The Company remains on track to file European Marketing Authorization Applications for BPX-501 and rimiducid in 2019.

Positive BPX-501 Interim Results Reported in AML and Primary Immunodeficiencies
Earlier today, the Company announced interim survival results in pediatric patients with acute myeloid leukemia (AML) suggesting that the addition of BPX-501 T cells to a haplo-HSCT may improve the anti-leukemic effect of stem cell transplantation. The Company also reported interim data in pediatric patients with primary immunodeficiencies (PIDs) undergoing a curative haplo-HSCT with BPX-501 demonstrating favorable disease-free and overall survival rates at one year. These interim results have been submitted for presentation at an upcoming medical meeting.

Robust BPX-601 GoCAR-TCell Expansion Observed Following Rimiducid Administration
The Phase 1 study of BPX-601—the first product featuring the Company’s iMC activation switch—is enrolling patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA). The first patient dosed with rimiducid—to activate iMC following infusion of BPX-601 cells—showed a robust expansion of circulating BPX-601 cells following a single dose of rimiducid, providing the first clinical proof of concept of iMC. The patient continues to be evaluated for safety and efficacy, and the clinical site is enrolling additional patients. Bellicum expects to report findings from the initial cohorts of pancreatic cancer patients at an upcoming medical meeting and to expand the trial to other PSCA-expressing cancers later this year.

Collaborator CD19 CAR-T Trial Initiated
The first patients have been treated in a Phase 1 pediatric ALL clinical trial of a CD19 CAR-T incorporating the CaspaCIDe safety switch, which is being conducted in collaboration with Ospedale Pediatrico Bambino Gesù (OPBG), a leading European pediatric research center and hospital. The trial is designed to assess the impact of CaspaCIDe in managing the acute toxicities of CAR-T therapy.

Completed Buildout of In-House Manufacturing and Vector Production Facility
The Company recently completed the buildout and initial launch of a 30,400 square foot state-of-the-art cell manufacturing and vector production facility at its headquarters in Houston, Texas. This facility is designed and constructed to satisfy both U.S. and European regulatory standards, and the Company expects the facility will meet U.S. clinical trial and early commercialization requirements.

Bellicum Continues to Strengthen its Management Team and Board of Directors
Since August 2017, the Company has added Gregory Naeve, Ph.D. (Chief Business Officer), William Grossman, M.D., Ph.D. (Chief Medical Officer), and several key leadership appointments to strengthen its clinical and quality functions. Additionally, Edmund P. Harrigan, M.D. was recently appointed to Bellicum’s Board of Directors, bringing 28 years of cross-functional pharmaceutical industry experience, most recently as Senior Vice President, Worldwide Safety and Regulatory at Pfizer.

ANTICIPATED 2018 MILESTONES

Report updated data from the BP-004 study of BPX-501
Initiate pivotal clinical trials of BPX-501 in adult AML and in either pediatric AML or PIDs, pending regulatory clearances
Report initial results from the BPX-601 clinical trial, and expand the trial to include additional PSCA-expressing cancers
Present initial findings from the BPX-701 clinical trial at upcoming medical meetings

Fourth Quarter and Full Year 2017 Financial Results

Cash Position and Guidance: Bellicum ended the year on December 31, 2017 with cash, restricted cash and investments totaling $106.5 million, compared to $113.4 million at December 31, 2016. In the fourth quarter of 2017, the Company paid off its Hercules Capital debt facility with a $35.0 million loan from Oxford Finance. The new loan provided approximately $2.1 million in additional liquidity, interest-only payments until February 1, 2020 and a lower interest rate. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through the first quarter of 2019.

R&D Expenses: Research and development expenses were $14.3 million and $65.7 million for the fourth quarter and year ended December 31, 2017, respectively, compared to $15.1 million and $51.3 million during the comparable periods in 2016. The higher expenses in 2017 were primarily due to increased clinical trial costs, particularly for BPX-501, start-up costs related to Bellicum’s in-house manufacturing facility and contract manufacturers in Europe and increased personnel and consulting expenses. The higher R&D expenses in the fourth quarter of 2016 were attributable to costs associated with characterization studies of rimiducid.

G&A Expenses: General and administrative expenses were $5.1 million and $21.0 million for the fourth quarter and year ended December 31, 2017, respectively, compared to $4.2 million and $16.9 million during the comparable periods in 2016. The increased G&A expenses in 2017 were primarily due to Bellicum’s overall growth, including an increase in personnel-related costs, facility costs, and other administrative costs.

Net Loss: Bellicum reported a net loss of $21.9 million for the fourth quarter of 2017 and $91.8 million for the year ended December 31, 2017, compared to a net loss of $19.9 million and $69.2 million for the comparable periods in 2016. The results included non-cash, share-based compensation charges of $3.4 million and $13.6 million for the fourth quarter and year ended December 31, 2017, respectively, and $3.1 million and $12.3 million for the comparable periods in 2016.

Shares Outstanding:
At December 31, 2017, Bellicum had 33,285,177 shares of common stock outstanding.

Conference Call and Webcast
Bellicum management will host a webcast and conference call at 5:00 p.m. Eastern today to discuss the financial results. To access the call, participants should dial 877-407-3103 (domestic) and 201-493-6791 (international) at least 10 minutes prior to the start of the call. The event will be webcast live and can also be accessed in the Investors & Media section of bellicum.com. An archived version of the webcast will also be available for replay in the Investors & Media section of the Bellicum website for at least two weeks following the call.

About BPX-501
BPX-501 is an adjunct T cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD or other T-cell mediated complications occur. This enables physicians to more safely perform stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemic effect without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

About BPX-601
BPX-601 is a GoCAR-T product candidate containing Bellicum’s proprietary inducible MyD88/CD40, or iMC, activation switch, designed to treat solid tumors expressing prostate stem cell antigen, or PSCA. Preclinical data show enhanced T cell proliferation, persistence and in vivo anti-tumor activity compared to traditional CAR-T therapies. A Phase 1 clinical trial in patients with nonresectable pancreatic cancer is ongoing. In addition to pancreatic cancer, PSCA is expressed in several other solid tumor indications, including: gastric, esophageal, cholangiocarcinoma, glioblastoma, prostate and bladder cancers. The Company plans to expand the clinical development of BPX-601 to include additional PSCA expressing cancer types.

About BPX-701
BPX-701 is a high affinity T cell receptor product candidate designed with the CaspaCIDe safety switch. It is currently being tested in a Phase 1 study of patients with refractory or relapsed acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) who test positive for PRAME, or preferentially expressed antigen in melanoma. In preclinical studies, PRAME-specific clones showed high reactivity against a panel of PRAME positive tumor cell lines, metastatic melanoma, sarcomas and neuroblastoma tissues. In vitro study data showed that BPX-701 demonstrated strong affinity to panels of cancer cells presenting PRAME peptides and low affinity to non-tumor cells, as well as complete elimination of BPX-701 cells in response to rimiducid.

On March 14, 2018 Atreca, Inc., a biotechnology company focused on developing novel therapeutics based on a deep understanding of the human immune response, reported that the Company will present new research findings from its advancing pipeline programs at the 2018 American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, being held April 14-18, 2018, at the McCormick Place Convention Center, Chicago, IL (Press release, Atreca, MAR 13, 2018, View Source [SID1234524725]). The two presentations will highlight the antibody responses in patients who have achieved positive clinical outcomes following checkpoint inhibitor therapy.

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"We are excited to announce new results generated via our discovery engine, using our Immune Repertoire Capture (IRC) technology," said Tito A. Serafini, Ph.D., Atreca’s President, Chief Executive Officer, and Co­Founder. "In patients having responses to cancer immunotherapy, we demonstrate that an immune system attack on tumor tissue involving anti-tumor antibodies is indeed a hallmark of such responses across diverse cancer types. We have also discovered functional anti-tumor antibodies from these patients with potential application against a variety of tumors. We are pleased to announce these findings during AACR (Free AACR Whitepaper)."

The abstracts are available in the program section of the annual AACR (Free AACR Whitepaper) meeting website, and details for the poster presentations are as follows:

Abstract Title: Increased somatic hypermutation in the immunoglobulin sequences of melanoma patients who have durable response to checkpoint inhibitor therapy (Abstract 615 / Poster 9)

Poster Session Title: Immune Response to Therapies 1
Presentation Date & Time: Sunday, April 15, 2018, 1:00 PM – 5:00 PM CT
Location: Poster Section 27, Exhibit Hall A, McCormick Place South

Abstract Title: Mining the cancer immuno-responsome: The identification of functional antitumor antibodies from patients receiving checkpoint inhibitors (Abstract 3966 / Poster 19)

Poster Session Title: Targeting Oncogenes, Tumor Suppressors, or Gene Products
Presentation Date & Time: Tuesday, April 17, 2018; 8:00 AM – 12:00 PM CT
Location: Poster Section 40, Exhibit Hall A, McCormick Place South

On March 13, 2018 Athersys, Inc. (NASDAQ:ATHX) reported its fourth quarter 2017 and annual 2017 financial results and highlights (Press release, Athersys, MAR 13, 2018, View Source [SID1234524724]).

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"As we announced today in a separate press release, we have entered a letter of intent to expand our collaboration with Healios, and we are actively working with them with to complete the broader collaboration expansion by April 30, 2018, as we disclosed earlier today. In doing so, this would result in committed capital in the amount of $56.1 million, of which $31.1 million is already committed, in the form of the initial equity investment and license fee payments. Importantly, the broadened collaboration would lead to increased development of MultiStem treatment in Asia and provide us with capital to support our pivotal registration study for ischemic stroke, MASTERS-2, in the United States and Europe, as well as other important activities," commented Dr. Gil Van Bokkelen, CEO of Athersys.

Fourth Quarter 2017 and Recent Highlights:

Announced today plans to significantly expand our existing HEALIOS K.K. ("Healios") collaboration, including a $21.1 million equity investment and $10 million in guaranteed license fees, and, if the expansion is consummated, would also further provide an additional $25 million in committed payments over time. As part of the expansion, Healios would receive a license to MultiStem products for acute respiratory distress syndrome ("ARDS") and trauma in Japan, and Healios’ organ bud technology and certain ophthalmological indications globally. Also, Healios would receive an exclusive option to license MultiStem products for ischemic stroke, ARDS and trauma in China, and Athersys would be entitled to license fees, milestone payments and escalating royalties for the licensed indications;
Advanced our preparations for the MASTERS-2 Phase 3 registration study for ischemic stroke, to enable initiation of this important study;
Entered into a new equity facility in February 2018 as follow-on to current facility, with right to sell up to $100 million of common stock to Aspire Capital, LLC over three-year period, providing access to capital as needed to support our operations;
Recorded revenues of $1.2 million and a net loss of $13.1 million for the quarter ended December 31, 2017, noting that included in the net loss for the quarter was a $4.7 million non-recurring charge ($3.2 million of which was non-cash) related to a settlement and license agreement to resolve a long-standing intellectual property dispute; and
Ended 2017 with $29.3 million in cash and cash equivalents.

Other 2017 Highlights:

Received multiple special designations from regulators for our stroke program this year, including Regenerative Medicine Advanced Therapy designation and Fast Track designation from U.S. Food and Drug Administration, as well as a Final Scientific Advice positive opinion from European Medicines Device Agency;
Expanded manufacturing and process development collaborative relationships, including Nikon CeLL innovation Co., Ltd., and progressed key manufacturing campaigns and process development projects; and
Recorded revenues of $3.7 million and a net loss of $32.2 million, or $0.29 net loss per share, for the year ended December 31, 2017, again, factoring in the 2017 charge of $4.7 million for the intellectual property settlement and license.

"Over the course of 2017 and into 2018, we have undertaken and completed multiple initiatives that are intended to advance MultiStem therapy into registrational studies and ultimately to commercialization," stated Dr. Van Bokkelen. "We plan to launch our MASTERS-2 study in the second quarter, and continue to advance our manufacturing platform and capabilities. Importantly, through these activities and our collaboration expansion with Healios, we have further strengthened our financial position, while retaining North American and European rights for MultiStem therapy in ischemic stroke and other indications, while we continue to evaluate additional collaborative opportunities."

Fourth Quarter 2017 Financial Results

Total revenues for the fourth quarter of 2017 were $1.2 million compared to $1.0 million in the same period in the prior year, reflecting a combination of contract revenues and grant revenues.

Research and development expenses increased to $12.1 million in the 2017 fourth quarter from $7.1 million in the same period in the prior year. In 2017, approximately $4.7 million of license fees were expensed (of which $3.2 million was non-cash) related to a settlement and license agreement to resolve a long-standing intellectual property dispute. After factoring in this one-time charge, the remaining difference of $0.3 million from year-to-year was primarily due to increased clinical and preclinical development costs, which vary based on trials underway, clinical manufacturing and process development activities.

General and administrative expenses remained relatively consistent at $2.1 million and $2.0 million in the 2017 and 2016 fourth quarters, respectively.

Net loss was $13.1 million in the fourth quarter of 2017, compared to net loss of $7.1 million for the same period of 2016. The increase in net loss was primarily due to the variances outlined above (e.g., settlement and license fees) and a $1.1 million gain in the fourth quarter of 2016 related to the fair value of our warrant liabilities (non-cash), with no corresponding warrant activity in the 2017 fourth quarter, since all of our warrants were either exercised or expired early in 2017.

Full Year 2017 Financial Results

Revenues decreased to $3.7 million for the year ended December 31, 2017 from $17.3 million in 2016, related to a $15.0 million payment received and recognized as revenue for the Healios collaboration entered into in January 2016, partially offset by 2017 increases in other contract revenues, including a $1.0 million milestone payment from our collaboration with RTI Surgical, Inc. and manufacturing and service proceeds from Healios.

Research and development expenses increased to $27.8 million for the year ended December 31, 2017 from $24.8 million for the year ended December 31, 2016. After factoring in the non-recurring charge of $4.7 million for license fees referred to above, the decrease in research and development expenses year-over-year of $1.7 million related primarily to reduced spending on research supplies of $0.9 million and sponsored research of $0.5 million.

General and administrative expenses increased to $8.5 million in 2017 from $7.8 million in 2016. The $0.7 million increase was due primarily to increases in personnel costs and legal and professional services.

Net loss was $32.2 million in 2017, compared to $15.3 million in 2016. The difference of $16.9 million reflects the variances above, particularly the $15.0 million Healios revenue in 2016 and the $4.7 million one-time license fee expense in 2017, as well as an increase in 2017 of $1.3 million in the gain in the fair value of warrant liabilities, a decrease in 2017 of $0.7 million in the gain from insurance proceeds, and overall variances in operational activities.

Cash used in operating activities was $24.0 million and $10.9 million for full year 2017 and full year 2016, respectively, which takes into account the $15.0 million initial license fee revenue from Healios in 2016 and the other variances noted above.

As of December 31, 2017, we had $29.3 million in cash and cash equivalents, compared to $14.8 million at December 31, 2016.

Conference Call

Gil Van Bokkelen, Chairman and Chief Executive Officer, William (BJ) Lehmann, President and Chief Operating Officer, and Laura Campbell, Senior Vice President of Finance will host a conference call today to review the results as follows:
Date Tuesday, March 13, 2018
Time 4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada 800-273-1254
Telephone access: International 973-638-3440
Access code 8898346
Live webcast www.athersys.com, under the Investors section

A replay will be available for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on March 27, 2018 at the aforementioned URL, or by dialing (800) 585-8367 or (855) 859-2056 in the U.S. and Canada, or from abroad (404) 537-3406, and entering access code 8898346. The archived webcast will be available for one year at the aforementioned URL.

On March 13, 2018 Advaxis, Inc. (NASDAQ:ADXS), a late-stage biotechnology company ("Advaxis" or the "Company") focused on the discovery, development and commercialization of cancer immunotherapies, reported that Company management will present at the following upcoming March 2018 conferences (Press release, Advaxis, MAR 13, 2018, View Source [SID1234524711]):

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Barclays Global Healthcare Conference

Date and Time: Thursday, March 15, 2018 at 9:30am ET
Venue: Loews Miami Beach Hotel, Miami, FL
Presenter: Anthony Lombardo, Interim Chief Executive Officer
Presentation: Company overview
25 th Annual Future Leaders in the Biotech Industry Conference

Date and Time: Friday, March 23rd at 3:00pm ET
Venue: Millennium Broadway Hotel & Conference Center, New York, NY
Presenter: Robert Petit, Ph.D., EVP and Chief Scientific Officer
Presentation: Company overview

On March 13, 2018 Verastem, Inc. (NASDAQ:VSTM), focused on developing and commercializing drugs to improve the survival and quality of life of cancer patients, reported financial results for the year ended December 31, 2017 and provided an overview of certain corporate developments and plans (Press release, Verastem, MAR 13, 2018, View Source;p=RssLanding&cat=news&id=2337744 [SID1234524706]).

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"The last year has been marked by significant achievement for Verastem with the reporting of positive data from the pivotal Phase 3 DUO study and culminating in the recent submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking full approval for duvelisib for the treatment of relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and accelerated approval for the treatment of relapsed or refractory follicular lymphoma (FL)," said Robert Forrester, President and Chief Executive Officer of Verastem. "As we await the potential acceptance and approval of the duvelisib NDA, we are diligently working to build our commercial infrastructure and preparing for our first potential product launch. I am delighted that Joe Lobacki has joined the team. Joe’s formidable expertise in commercialising oncology drugs at Medivation, Micromet and Genzyme positions Verastem to successfully execute on our launch plan for duvelisib in the US."

Fourth Quarter 2017 and Recent Highlights:

Duvelisib

Duvelisib NDA submitted to FDA – In early February 2018, Verastem submitted an NDA to the FDA seeking full approval for its lead product candidate duvelisib, a first-in-class oral dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, for the treatment of relapsed or refractory CLL/SLL and accelerated approval for the treatment of relapsed or refractory FL. The NDA is supported by clinical data from the randomized Phase 3 DUO study, which met its primary endpoint by demonstrating statistically significant efficacy, along with a consistent and manageable safety profile, for duvelisib monotherapy in patients with relapsed or refractory CLL/SLL. The NDA is also supported by results from the Phase 2 DYNAMO study, which also met its primary endpoint by demonstrating a statistically significant improvement in overall response rate (ORR) compared to an historical control in patients with indolent non-Hodgkin’s lymphoma that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy.
Clinical Data from Pivotal Phase 3 DUO Study Highlighted in an Oral Presentation at ASH (Free ASH Whitepaper) 2017 – Verastem presented results from the Phase 3 DUO study at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 Annual Meeting (ASH 2017). The presentation, titled "Results from the Phase 3 DUO Trial: A Randomized Comparison of Duvelisib vs Ofatumumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma," was presented by principal investigator Ian Flinn, M.D., Ph.D., Director of the Blood Cancer Research Program at Sarah Cannon Research Institute. The DUO study met its primary endpoint with oral duvelisib monotherapy achieving a statistically significant improvement in progression free survival (PFS) compared to ofatumumab in patients with relapsed or refractory CLL/ SLL per a blinded independent review committee (IRC) using modified international workshop on CLL (iwCLL) and revised International Working Group (IWG) Response Criteria (median PFS=13.3 months versus 9.9 months, respectively; HR=0.52, p<0.0001), representing a 48% reduction in the risk of progression or death. Oral duvelisib monotherapy also achieved a statistically significant improvement in ORR compared to ofatumumab (74% vs 45%, respectively; p<0.0001), and reduced lymph node burden of less than 50% in most patients compared to ofatumumab (85% vs 16%, respectively). Duvelisib monotherapy demonstrated a manageable safety profile, with results from this study consistent with the well-characterized safety profile of duvelisib monotherapy in patients with advanced hematologic malignancies in previous studies. For duvelisib-treated patients, the median time on treatment was 50.3 weeks (range, 0.9 – 160.0) compared to 23.1 weeks (range, 0.1 – 26.1) for ofatumumab.
Additional Duvelisib Abstracts Presented at ASH (Free ASH Whitepaper) 2017 – Along with the Phase 3 DUO results, two additional duvelisib abstracts were presented at ASH (Free ASH Whitepaper) 2017. The abstract, titled "In Vitro, In Vivo, and Parallel Phase I Evidence Support the Safety and Activity of Duvelisib, a PI3K-δ,γ Inhibitor, in Combination with Romidepsin or Bortezomib in Relapsed/Refractory T-Cell Lymphoma," was given as an oral presentation by Alison Moskovitz, M.D., Memorial Sloan Kettering Cancer Center.
Preclinical Data Highlighting the Synergistic Effects in Combination with Immunotherapy Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Clinical Immuno-Oncology Symposium (ASCO-SITC) – In January 2018, Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem, presented preclinical data highlighting the potential synergistic effects of duvelisib in combination with immune checkpoint or co-stimulatory antibodies in B-cell lymphoma. This data, outlined in a poster titled "The Dual PI3K-δ,γ Inhibitor Duvelisib Stimulates Anti-Tumor Immunity and Enhances Efficacy of Immune Checkpoint and Co-Stimulatory Antibodies in a B-Cell Lymphoma Model," supports the further exploration of duvelisib in combination with anti-PD-1/PD-L1 or co-stimulatory antibodies in patients with B-cell malignancies.
Defactinib

Defactinib Preclinical Abstract Presented at ASH (Free ASH Whitepaper) 2017 – A poster describing preclinical data in combination with B-cell lymphoma 2 (BCL-2) was presented at ASH (Free ASH Whitepaper) 2017. The abstract, titled "Combinatorial Inhibition of Focal Adhesion Kinase and BCL-2 in AML," was presented by Xiangmeng Wang, Ph.D., MD Anderson Cancer Center.
Corporate and Financial

Joseph Lobacki Appointed Chief Commercial Officer – In January 2018, Verastem announced the appointment of Joseph Lobacki as Executive Vice President and Chief Commercial Officer. Mr. Lobacki, formerly Chief Commercial Officer and Executive Council Member at Medivation, is responsible for overseeing the commercial strategy and execution for Verastem’s lead product candidate, duvelisib. Mr. Lobacki is a skilled leader in commercializing oncology drugs and his strong experience in hematologic oncology commercialization and marketing make him an invaluable addition to the Verastem team.
Additional Financing Through Increasing Debt Facility to up to $50.0 Million and a $25.0 Million Public Offering – In January 2018, Verastem amended its loan and security agreement with Hercules Capital, Inc. (Hercules), increasing its existing borrowing limit under the loan facility from up to $25.0 million to up to $50.0 million in financing, subject to certain conditions of funding. In December 2017, the Company successfully completed an underwritten public offering of shares of common stock with gross proceeds totaling approximately $25.0 million.
NgocDiep Le, MD, PhD, Appointed Chief Medical Officer – In October 2017, Verastem announced the appointment of Dr. Le as its Chief Medical Officer. A trained medical oncologist, Dr. Le is board certified in internal medicine and has 15 years of drug development experience across all phases in both solid and liquid tumors, with specialized expertise in clinical development. Dr. Le joins Verastem from MedImmune (a wholly owned subsidiary of AstraZeneca) where she served as Vice President, Immuno-Oncology Innovative Medicines and led the product development teams for multiple high-priority immuno-oncology assets. Dr. Le oversees the development strategy and activities for Verastem’s core assets, duvelisib and defactinib.
Paid First Development Milestone to Infinity Pharmaceuticals – In October 2017, Verastem paid to Infinity Pharmaceuticals, Inc. (Infinity) a $6.0 million milestone payment, representing the first milestone under the duvelisib license agreement. This milestone is based on the achievement of positive top-line results from the Phase 3 DUO study evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory CLL/SLL. The milestone was paid using funds drawn from Verastem’s existing loan and security agreement with Hercules.
Full Year 2017 Financial Results

Net loss for the year ended December 31, 2017 (2017 Period) was $67.8 million, or $1.76 per share, as compared to a net loss of $36.4 million, or $0.99 per share, for the year ended December 31, 2016 (2016 Period). Net loss includes non-cash stock-based compensation expense of $5.0 million and $6.2 million for the 2017 Period and 2016 Period, respectively. Verastem used $57.3 million of cash for operating activities during the 2017 Period.

Research and development expense for the 2017 Period was $46.4 million compared to $19.8 million for the 2016 Period. The $26.6 million increase from the 2016 Period to the 2017 Period was primarily related to an increase of $13.4 million in external clinical research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, the achievement of a $6.0 million milestone pursuant to our license agreement with Infinity, an increase of $5.1 million in consulting fees, and an increase in personnel related costs of $1.9 million.

General and administrative expense for the 2017 Period was $21.4 million compared to $17.2 million for the 2016 Period. The increase of $4.2 million from the 2016 Period to the 2017 Period primarily resulted from increases in consulting and professional fees of $4.4 million, including $2.5 million related to commercial launch preparation, and an increase in personnel costs of $1.0 million. These increases were partially offset by a decrease in stock-based compensation expense of $1.5 million.

As of December 31, 2017, Verastem had cash, cash equivalents and investments of $86.7 million compared to $80.9 million as of December 31, 2016.

The number of outstanding common shares as of December 31, 2017, was 50,800,908.

Financial Guidance

Based on our current operating plans, we expect to have sufficient cash, cash equivalents and investments to fund our current operating plan and capital expenditure requirements into the second half of 2018.

About Duvelisib

Duvelisib is a first-in-class investigational, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints and Verastem has submitted a new drug application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL). Duvelisib is also being developed by Verastem for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Defactinib

Defactinib is an investigational inhibitor of focal adhesion kinase (FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling in response to cellular adhesion and growth factors.7 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment and enhancement of anti-tumor immunity.8,9 Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic cancer, ovarian cancer, non-small cell lung cancer (NSCLC), and mesothelioma. These studies are combination clinical trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.