On March 13, 2018 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative anti-infective therapies for difficult-to-treat and often life-threatening infections, reported financial results for the year ended December 31, 2017, and provided an update on recent operational and clinical developments (Press release, Scynexis, MAR 13, 2018, View Source [SID1234524738]).

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"In 2017, we made meaningful progress in advancing our lead product candidate, SCY-078, across a range of indications, most notably with the initiation and continued progression of our Phase 2b DOVE study evaluating oral SCY-078 for the treatment of vulvovaginal candidiasis (VVC)," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "Additionally, we now have a path forward with our IV SCY-078 program and plan to start Phase 1 testing of our new liposomal IV formulation in the third quarter of 2018. Overall, we continued to expand the body of evidence showing SCY-078’s potential as a potent therapy for the treatment of multiple fungal infections associated with significant unmet medical needs. Following the completion of our recent $30 million equity offering, we are well-positioned to advance the SCY-078 platform in 2018 and beyond."

Advancement of Oral SCY-078 Programs

Initiated Phase 2b dose-finding study in lead indication of VVC
DOVE Study. Dosing is ongoing in the Phase 2b, dose-finding trial designed to evaluate the safety and efficacy of oral SCY-078 compared to oral fluconazole (the standard of care) for the treatment of VVC, our most advanced indication. The study continues to enroll rapidly, and SCYNEXIS expects to report top-line results in mid-2018.
If successful, following completion of the DOVE study and following an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), SCYNEXIS anticipates initiating a Phase 3 VVC study in the fourth quarter of 2018, with the objective of filing the New Drug Application (NDA) for acute VVC in 2020.
Two studies for the treatment of refractory invasive fungal infections opened for enrollment.
FURI Study. Commenced patient dosing in a global, open-label study, designed to evaluate oral SCY-078 for the treatment of fungal infections that are refractory to or intolerant of standard therapy. A total of 24 locations throughout the U.S. and Europe are now active, and enrollment continues to progress.
CARES Study. The global, open-label study, designed to evaluate oral SCY-078 for the treatment of Candida auris infections, an often multidrug-resistant pathogen associated with high mortality infections, opened for enrollment in the fourth quarter of 2017. The CARES study is intended to provide rapid access to oral SCY-078 for patients suffering from this life-threatening infection.
Potential for streamlined development pathway for both studies. SCYNEXIS believes that compelling data from the FURI and/or CARES studies could allow oral SCY-078 to become eligible for the regulatory Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD), potentially resulting in an initial NDA based on streamlined development. SCYNEXIS plans to conduct a preliminary data review in the fourth quarter of 2018.
Promising pre-clinical data generated to support "Combination Study Design" for treatment of invasive aspergillosis. Based on promising data of SCY-078 in combination with an azole agent in pre-clinical models of invasive aspergillosis, SCYNEXIS plans to initiate a randomized, double-blind, Phase 2 study of oral SCY-078 in combination with azole therapy, the standard of care, for this indication. SCYNEXIS is finalizing the trial design and expects to initiate this study in the third quarter of 2018.

Advancement of IV SCY-078 Program with Liposomal IV Formulation

Pre-clinical work on the liposomal IV formulation of SCY-078 continues, and SCYNEXIS remains on track to initiate a Phase 1 trial evaluating the safety and tolerability of this formulation in healthy volunteers in the third quarter of 2018.
If successful, following completion of the Phase 1 study and pending FDA review, SCYNEXIS plans to initiate a Phase 2b IV-oral step-down study of SCY-078 in invasive candidiasis patients with the liposomal IV and oral formulations of SCY-078 in the fourth quarter of 2018.

Pre-Clinical Data Support Continued Development of SCY-078

In February 2018, at the 8th Advances Against Aspergillosis (AAA), SCYNEXIS presented new, pre-clinical data demonstrating synergistic activity and improved outcomes of SCY-078 in combination with isavuconazole for the treatment of invasive pulmonary aspergillosis.
In October 2017, at IDWeek 2017, SCYNEXIS presented results from three studies supporting the potent and broad antifungal activity of SCY-078 against Candida and Aspergillus species. These results showed SCY-078’s potent activity against wild-type (WT) and azole-resistant strains of A. fumigatus, as well as against WT, azole-resistant and echinocandin-resistant strains of C.parapsilosis. In addition, SCY-078 showed clinically-meaningful penetration into tissues relevant for the targeted indications, including lung, vaginal mucosa and kidney, following oral and IV administration in rats and mice.
In August 2017, at the IDSOG Annual Meeting, SCYNEXIS presented results showing SCY-078’s high penetration into vaginal tissue after oral administration and its potent anti-Candida activity in acidic pH conditions, characteristic of the vaginal setting, supporting the use of SCY-078 as a novel treatment of VVC.

Corporate Update

On March 6, 2018, SCYNEXIS raised $30.0 million in gross proceeds by issuing 17,751,500 shares of the Company’s common stock and two series of warrants to purchase up to an aggregate of 21,301,800 shares of the Company’s common stock. The offering resulted in approximately $27.8 million of net proceeds after deducting the underwriting discount and estimated offering expenses.
In November 2017, SCYNEXIS announced the appointment of Scott Sukenick, J.D., as General Counsel. With more than ten years of legal experience in life sciences, Mr. Sukenick joined SCYNEXIS most recently from Cooley LLP, where he focused on life sciences litigation and strategic intellectual property management.

Full Year 2017 Financial Results

Cash, cash equivalents and short-term investments totaled $43.9 million as of December 31, 2017.

Based upon its existing operating plan and the net proceeds from the March 6, 2018 offering, SCYNEXIS believes that its existing cash, cash equivalents and short-term investments will enable the Company to fund its operating requirements into 2020.

Research and development expenses decreased to $18.3 million in 2017, compared to $20.1 million in 2016. The decrease of $1.8 million, or 8.7%, was primarily driven by a decrease of $1.7 million in clinical development, a decrease of $1.3 million in chemistry, manufacturing, and controls (CMC), a decrease of $0.6 million in consulting fees; offset by an increase of $0.9 million in salary and personnel related costs and an increase of $0.9 million in other research and development expenses.

Selling, general and administrative expenses increased to $8.3 million in 2017, compared to $8.0 million in 2016. The increase of $0.3 million, or 3.2%, was primarily driven by an increase of $0.6 million in business development related activities, a $0.3 million increase in stock-based compensation, and a net increase of $0.2 million in other selling, general and administrative expenses; offset by a decrease of $0.4 million in both professional and consulting expenses.

Loss from operations in 2017 was $26.3 million, compared to a loss from operations of $27.8 million in 2016. The $1.5 million decrease in the loss from operations between the two periods was due to $1.8 million decrease in research and development expense, offset by an increase in selling, general and administrative expense of $0.3 million.

Total other income was $1.3 million in 2017, compared to other expense of $2.2 million in 2016 due to a $2.7 million non-cash gain recorded on the adjustment in the fair value of the warrant liability offset by an increase in interest expense of $1.1 million.

Net loss in 2017 was $25.1 million, or $0.94 per share. This compares to a net loss in 2016 of $30.0 million, or $1.58 per share.

On March 13, 2018 Seattle Genetics, Inc. (NASDAQ:SGEN) announced today that management will present at the Barclays Global Healthcare Conference 2018 on Wednesday, March 14, 2018 at 10:45 a.m. Eastern Time (Press release, Seattle Genetics, MAR 13, 2018, View Source;p=RssLanding&cat=news&id=2337768 [SID1234524739]). The presentation will be webcast live and available for replay from Seattle Genetics’ website at www.seattlegenetics.com in the Investors section.

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On March 13, 2018 Crown Bioscience, a wholly-owned subsidiary of Crown Bioscience International (TWSE: ticker 6554) and a global drug discovery and development services company providing translational platforms to advance oncology, inflammation, cardiovascular and metabolic disease research, reported it has been selected by Phanes Therapeutics as a strategic partner to advance their oncology drug discovery pipeline (Press release, Phanes Therapeutics, MAR 13, 2018, View Source [SID1234527491]).

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CrownBio provides the world’s largest collection of patient derived xenograft (PDX) models and is a leading provider of integrated oncology solutions that enable development of cutting-edge cancer treatments. Phanes will gain access to CrownBio’s clinically relevant CDX models, exclusive PDX models and robust in vitro assays to propel their innovative drug discovery pipeline.

Dr. Ming Wang, President and CEO of Phanes Therapeutics, expressed enthusiasm about this partnership, "We are an innovative drug discovery company with a robust pipeline and need access to top notch in vivo pharmacology efficacy models to quickly evaluate and advance our lead molecules. We are very glad to partner with CrownBio. They have very unique MuPrime models, as well as Humanized GEMMs (HuGEMM) and Patient-Derived Xenograft (PDX) models for our oncology programs. We look forward to a productive partnership."

"We are very pleased that Phanes Therapeutics has selected us as a trusted partner to advance their leading-edge oncology compounds," said Laurie Heilmann, CrownBio’s Chief Business Officer. "Our unique capabilities will help Phanes efficiently and thoroughly assess drugs in their pipeline to identify and advance candidates showing the most promise for improved treatment of cancer."

On March 13, 2018 MorphoSys Reported Updated Data from L-MIND Study of MOR208 plus Lenalidomide in Aggressive Lymphoma (r/r DLBCL) (Press release, MorphoSys, MAR 13, 2018, View Source [SID1234556340]).

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– New data from the ongoing L-MIND trial of MOR208 plus lenalidomide in relapsed/refractory DLBCL patients ineligible for high-dose chemotherapy and autologous stem cell transplantation confirm earlier data reported from this trial

– 81 patients enrolled, 68 available for efficacy assessment at cut-off date

– Preliminary median progression-free survival (PFS) not reached, preliminary PFS rate at 12 months of 50.4%

– Overall response rate (ORR) of 49% with 29 out of 33 responses ongoing, complete response (CR) rate of 31%

– Data show that MOR208 in combination with lenalidomide has been well tolerated in the study: no unexpected toxicities were observed for the treatment combination and no infusion-related reactions were reported for MOR208

– MorphoSys continues to have productive discussions with the FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study.

MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) today reported updated data from the ongoing single-arm phase 2 clinical trial known as L-MIND. L-MIND is designed to investigate the antibody MOR208 plus lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). MOR208 is an investigational Fc-engineered monoclonal antibody directed against CD19 and is currently in clinical development in blood cancer indications.

The L-MIND study enrolled patients with r/r DLBCL, who are ineligible for HDC and ASCT, after up to three prior lines of therapy, with at least one prior therapy including an anti-CD20 targeting therapy (such as rituximab). In November 2017, the trial completed patient enrollment with 81 patients. The updated interim data reported today (cut-off date December 12, 2017) included all 81 patients enrolled in the L-MIND trial, 68 of whom were available for efficacy assessment by the investigators at the time of data cut-off. Patients enrolled had a median age of 72 years and had received a median of two prior treatment lines.

Data reported today, with a median observation time of 8.3 months, showed a response in 33 out of 68 patients (overall response rate (ORR) 49%) and a complete response (CR) in 31% of the patients. The preliminary progression-free survival (PFS) rate at 12 months was 50.4% (95% confidence interval 40 – 67%) and the preliminary median PFS had not been reached (95% confidence interval: 4.3 months-not reached). 29 out of 33 responses (88%) were ongoing at the time of data-cut off. Median time to response was 1.8 months, median time to complete response was 3.6 months.

No unexpected toxicities were observed for the treatment combination and no infusion-related reactions (IRRs) were reported for MOR208. The most frequent adverse events with a toxicity grading of 3 or higher were neutropenia, thrombocytopenia, febrile neutropenia and pneumonia, observed in 36%, 12%, 7% and 7% of patients, respectively.

The results reported today confirm and corroborate earlier interim data reported from this trial (Salles et al, ASH (Free ASH Whitepaper) 2017), which had been based on 51 patients enrolled, 44 of whom had been eligible for investigators’ efficacy assessment at the June 13, 2017 cut-off date.

"We are truly excited about this data and our productive discussions with FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study. We look forward to continuing the analysis of maturing data from the L-MIND trial and to maintaining our interactions with the FDA," commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

"There is a very high unmet medical need for patients with r/r DLBCL who, after having failed initial therapies, are ineligible for high-dose chemotherapy and autologous stem cell transplantation," said Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG. "We are very encouraged by our most recent clinical data from the ongoing L-MIND trial, which support our plan to develop MOR208 in combination with lenalidomide as a chemo-free treatment option for this patient population."

About DLBCL
Diffuse large B-cell lymphoma (DLBCL) is the most frequent type of malignant lymphoma worldwide and accounts for approximately 30% of all non-Hodgkin lymphomas. Between 30% and 40% of all patients with DLBCL either fail to respond to or show a relapse to initial therapy. Patients who failed frontline therapy and are not eligible to high dose chemotherapy and autologous transplantation have a very poor outcome and require more therapeutic options.

About CD19 and MOR208
CD19 is broadly and homogeneously expressed across different B cell malignancies including DLBCL and CLL. CD19 has been reported to enhance B cell receptor (BCR) signaling, which is assumed important for B cell survival, making CD19 a potential target in B cell malignancies.
MOR208 (previously Xmab(R)5574) is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. Fc-modification of MOR208 is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. MOR208 has been observed in preclinical models to induce direct apoptosis by binding to CD19, which is assumed to be a crucial component for B cell receptor (BCR) signaling.
MorphoSys AG is clinically investigating MOR208 as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of MOR208 in combination with lenalidomide in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). Based on interim data from L-MIND, in October 2017 the FDA granted Breakthrough Therapy Designation for MOR208 plus lenalidomide in this patient population. The pivotal phase 2/3 B-MIND study is designed to investigate MOR208 in combination with the chemotherapeutic agent bendamustine in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) in comparison to the combination of the anti-CD20 antibody rituximab plus bendamustine. In addition, MOR208 is currently being investigated in patients with relapsed/refractory CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

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.