On November 7, 2017 Nektar Therapeutics (Nasdaq: NKTR) reported that new clinical data will be presented for NKTR-214, Nektar’s lead immuno-oncology candidate, a CD122-biased agonist, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting at the Gaylord National Hotel & Convention Center in National Harbor, Maryland (Press release, Nektar Therapeutics, NOV 7, 2017, View Source [SID1234521653]). Data from the ongoing PIVOT Phase 1/2 study, which is evaluating NKTR-214 in combination with the checkpoint inhibitor nivolumab, will be presented in an oral presentation on Saturday, November 11th. Six additional presentations for immuno-oncology (I-O) candidates in Nektar’s pipeline will also be presented at the meeting; specifically, one additional clinical and three preclinical presentations on NKTR-214; one preclinical presentation on NKTR-255, an IL-15 memory T-cell stimulating cytokine; and one preclinical presentation on NKTR-262, a novel toll-like receptor (TLR) agonist. The abstracts published in advance of the SITC (Free SITC Whitepaper) Annual Meeting were made available at 8 a.m. Eastern Time today on the Journal for ImmunoTherapy of Cancer’s website at View Source

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"The initial abstract data for these first set of 16 patients from the PIVOT study evaluating NKTR-214 in combination with nivolumab is very exciting," said Mary Tagliaferri, M.D., Senior Vice President of Clinical Development at Nektar Therapeutics. "We look forward to presenting additional new data from all of the 38 patients enrolled in the dose escalation cohort of the study in Dr. Diab’s oral presentation at SITC (Free SITC Whitepaper) on Saturday afternoon, including the first efficacy data for patients with PD-L1 negative non-small cell lung cancer who had progressed on prior chemotherapy. We believe there is tremendous need for more efficacious and better tolerated I-O combination treatment regimens, particularly treatments that can be used for the majority of cancer patients whose tumors don’t express PD-L1 as these patients can’t currently benefit from treatment with checkpoint inhibitors."

NKTR-214 is an investigational immuno-stimulatory therapy designed to expand specific cancer-fighting CD8+ effector T cells and natural killer (NK) cells directly in the tumor micro-environment and increase expression of PD-1 on these immune cells.

Details of the SITC (Free SITC Whitepaper) presentations for Nektar’s I-O portfolio are below.

Oral Presentation

Date: Saturday, November 11, 2017, Presentation Time: 5:00 p.m. Eastern Time
Session Title: Clinical Trials: Novel Combinations
Presentation Title: PIVOT-02: Preliminary safety, efficacy and biomarker results from the Phase 1/2 study of CD-122-biased agonist NKTR-214 plus nivolumab in patients with locally advanced/metastatic solid tumors
Presenter: Dr. Adi Diab, MD Anderson Cancer Center
Location: Gaylord National Hotel & Convention Center, Maryland Ballroom A

The online abstract for the oral clinical presentation includes preliminary data as of July 25, 2017 for 16 patients with Stage IV melanoma and renal cell carcinoma enrolled in the dose-escalation phase of the PIVOT study of NKTR-214 in combination with nivolumab (NCT02983045). New and updated interim data for a total of 38 patients with Stage IV melanoma, renal cell carcinoma and non-small cell lung cancer which were enrolled in the dose-escalation phase of the PIVOT study, will be presented at the SITC (Free SITC Whitepaper) Annual Meeting. A copy of the slides from Dr. Diab’s presentation will be made available on Nektar’s corporate website on Saturday, November 11th at 6:00 p.m. Eastern Time, in accordance with the embargo policies set forth by SITC (Free SITC Whitepaper).

Poster Presentations

Biomarkers and Immune Monitoring

Clinical Data Abstract #P77: The Novel IL-2 Cytokine Immune Agonist NKTR-214 Harnesses the Adaptive and Innate Immune System for the Treatment of Solid Cancers
Presenter: Salah Eddine Bentebibel, University of Texas MD Anderson Cancer Center
Date and Time: Friday, November 10, 2017, 12:30-2:00 p.m. Eastern Time

Cancer Vaccines

Preclinical Data Abstract #P140: NKTR-214 enhances anti-tumor T-cell immune responses induced by checkpoint blockade or vaccination
Presenter: Meenu Sharma, University of Texas MD Anderson Cancer Center
Date and Time: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Combination Therapy

Preclinical Data Abstract #P274: Combination of NKTR-214 and radiotherapy (RT) to reverse anergy and expand tumor-specific CD8 T Cells
Presenter: Joshua Walker, Oregon Health & Science University
Date and Time: Saturday, November 11, 2017,12:30-2:00 p.m. Eastern Time

Preclinical Data Abstract #P275: Harnessing the innate and adaptive immune system to eradicate treated and distant untreated solid tumors
Presenter: Saul Kivimae, Nektar Therapeutics
Date and Time: Friday, November 10, 2017, 12:30-2:00 p.m. Eastern Time

Immune Modulation, Cytokines, and Antibodies

Preclinical Data Abstract #P332: Pre-clinical efficacy and tolerability of NKTR-255, a polymer-conjugated IL-15 for immuno-oncology
Presenter: Peiwen Kuo, Nektar Therapeutics
Date and Time: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Personalized Vaccines and Technologies/Personalized Medicines

Preclinical Data Abstract #P434: Great Apes Adenoviral vaccine encoding neoantigens synergizes with immunomodulators to cure established tumors in mice
Presenter: Anna Morena D’Alise, Nouscom srl
Date and Time: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Analyst and Investor Event
Nektar Therapeutics will webcast an analyst and investor event with clinical investigators during the SITC (Free SITC Whitepaper) Annual Meeting beginning on Saturday, November 11, 2017 at 6:15 p.m. Eastern Time. Featured speakers include Dr. Patrick Hwu of MD Anderson Cancer Center, Dr. Adi Diab of MD Anderson Cancer Center, Dr. Michael E. Hurwitz of Yale Cancer Center, Dr. Antoni Ribas of UCLA Medical Center and Dr. Nizar M. Tannir of MD Anderson Cancer Center. The webcast is accessible from the investor relations page of Nektar’s website at www.nektar.com.

About NKTR-214
NKTR-214 is a clinical-stage, immuno-stimulatory therapy designed to expand specific cancer-fighting CD8+ effector T cells and natural killer (NK) cells directly in the tumor micro-environment and increase expression of PD-1 on these immune cells. NKTR-214 is a CD122-biased IL-2 pathway agonist that targets cancer-fighting immune cells to stimulate their proliferation and activation. In clinical and preclinical studies, treatment with NKTR-214 resulted in expansion of these cells and their mobilization into the tumor.1,2 NKTR-214 has an antibody-like dosing regimen similar to the existing checkpoint inhibitor class of approved medicines.

About NKTR-255
NKTR-255 is an immune-stimulating cytokine designed to engage all three IL-15 receptors and signal through all of these to activate the IL-15 pathway. NKTR-255 stimulates proliferation and survival of Natural Killer (NK) Cells, CD8+ T Cells and enhances long-term immunological memory which may lead to sustained anti-tumor immune response.

About NKTR-262
NKTR-262 is a first-in-class investigational small molecule intended to target toll-like receptors (TLRs) on innate immune cells in the body. NKTR-262 is designed to overcome the body’s dysfunction of antigen-presenting cells (APC), and is being developed as a single intra-tumoral injection to be administered at the start of therapy with NKTR-214 to induce an abscopal response and achieve the goal of complete tumor regression in cancer patients treated with both therapies.

On November 7, 2017 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration and fibrotic diseases, reported financial results for the third quarter ended September 30, 2017 (Press release, Tracon Pharmaceuticals, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315121 [SID1234521692]).

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Third Quarter 2017 and Recent Corporate Highlights

We continue to enroll patients in the randomized Phase 3 TAPPAS trial of TRC105 combined with Votrient (pazopanib) for the treatment of advanced angiosarcoma. Currently, more than 20 sites are open for accrual in the U.S., and sites in EU countries are expected to be open by year-end. As a reminder, the initial TAPPAS sample size of 124 patients is designed to detect an improvement in progression-free survival (PFS) from four to seven months. The trial uses an adaptive design that allows for expansion of patient numbers and selective enrollment of a responsive subtype of angiosarcoma while preserving type-1 error. TRACON expects that the interim analysis will be conducted in the second half of 2018.

In October, two posters highlighting TRC105 were presented at the 18th World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (IASLC) in Yokohama, Japan. Initial data from patients in the ongoing Phase 1b study of TRC105 in combination with Avastin (bevacizumab) and chemotherapy for the treatment of non-squamous cell lung cancer were presented. Three of eight (37%) evaluable patients had partial responses by RECIST 1.1, including one patient who achieved an 81% reduction in tumor volume. The complete enrollment of approximately 18 patients is expected by the end of 2018. In addition, investigators at the University of Alabama-Birmingham presented a "trials in progress" poster which detailed the study design of the Phase 1b dose-escalation trial of TRC105 and Opdivo (nivolumab) in patients with metastatic non-small cell lung cancer that is open for accrual.

In September, TRACON announced that enrollment was completed in the randomized Phase 2b TRAXAR study of TRC105 and Inlyta (axitinib) in patients with advanced or metastatic renal cell carcinoma (RCC). The Company expects to report top-line PFS data from this study in early 2018 based on the observed rate of events of disease progression or death that define the PFS endpoint. We expect the study to have at least 80 events confirmed by the independent central review committee at the time of data readout, which should provide at least 70% power to detect an improvement in PFS from 4.8 months with Inlyta to 7.2 months with the combination of TRC105 and Inlyta. PFS will also be assessed in patients with predefined levels of two soluble biomarkers, osteopontin and TGF-β receptor III, which correlated with response in the Phase 1 portion of the trial.

In July, following completion of the Phase 1/2 PAVE trial, TRACON and its partner, Santen Pharmaceutical Co. Ltd. (Santen), announced initiation of the randomized Phase 2a AVANTE study of DE-122, the ophthalmic formulation of TRC105, for the treatment of patients with wet age-related macular degeneration (AMD). The Phase 2a study is a randomized controlled trial designed to assess the safety and efficacy of repeated intravitreal injections of two different doses of DE-122 in combination with Lucentis (ranibizumab) compared to single agent Lucentis in patients with wet AMD. The Phase 2a study initiation resulted in a $7.0 million milestone payment to TRACON.
"The recently achieved progress across our pipeline positions us well for several important potentially value-creating milestones in the first half of 2018," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "In addition, we are highly encouraged by our partner Santen’s decision to advance DE-122 into the randomized Phase 2 AVANTE study in wet AMD."

Expected Upcoming Milestones Through 1H ‘18

Presentation of updated safety, pharmacokinetics and efficacy data from the completed Phase 1b/2 trial of TRC105 and Votrient in patients with angiosarcoma at the annual meeting of the Connective Tissue Oncology Society (CTOS) in Maui on November 9, 2017.

Presentation of early response data from the multicenter Phase 1/2 trial of TRC105 and Nexavar (sorafenib) in patients with hepatocellular carcinoma.

Completion of dose escalation in the Phase 1/2 clinical trial of TRC253 in patients with prostate cancer.

Announcement of top-line data from the randomized Phase 2 TRAXAR trial of TRC105 in combination with Inlyta.

Presentation of data from Santen’s completed Phase 1/2 PAVE trial of DE-122.
Third Quarter 2017 Financial Results

Cash, cash equivalents and short-term investments were $35.9 million at September 30, 2017, compared to $44.4 million at December 31, 2016.

Collaboration revenue for the third quarter of 2017 was $7.5 million compared to $0.8 million for the third quarter of 2016.

Research and development expenses for the third quarter of 2017 were $4.3 million compared to $4.5 million for the third quarter of 2016.

General and administrative expenses for the third quarter of 2017 were $1.8 million compared to $1.9 million for the third quarter of 2016.

Net income for the third quarter of 2017 was $1.2 million compared to a net loss of $5.9 million for the third quarter of 2016.
Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EST / 1:30 p.m. PST to provide an update on corporate activities and to discuss the financial results of its third quarter of 2017. The dial-in numbers are (855) 779-9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 7458759. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

On November 7, 2017 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results and recent highlights for the third quarter ended September 30 (Press release, Halozyme, NOV 7, 2017, View Source/investors/news-releases/news-release-details/2017/Halozyme-Reports-Third-Quarter-2017-Results/default.aspx" target="_blank" title="View Source/investors/news-releases/news-release-details/2017/Halozyme-Reports-Third-Quarter-2017-Results/default.aspx" rel="nofollow">View Source [SID1234521677]).

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"During the third quarter, we realized clear benefits from strategies we have been executing against in both the ENHANZE and PEGPH20 pillars of our business," said Dr. Helen Torley, president and chief executive officer. "On ENHANZE we significantly increased 2017 revenue and the potential future value of the technology with the signing of the landmark collaboration agreement with Bristol-Myers Squibb for development of up to 11 immuno-oncology targets, and with the expansion of our collaboration agreement with Roche. In addition, our partner Janssen took an important step towards the commercialization of a subcutaneous formulation of Darzalex with the initiation of a Phase 3 study.

"In our PEGPH20 pillar, investigator interest remains strong in our HALO-301 study, resulting in continued progress with enrollment. An interim analysis will be conducted for the first primary endpoint of progression-free survival when we achieve the target number of events, which we project will occur in late Q4 2018."

Third Quarter 2017 and Recent Highlights include:

Announcing a Global Collaboration and License Agreement with Bristol-Myers Squibb to develop subcutaneously administered Bristol-Myers Squibb immuno-oncology medicines using Halozyme’s ENHANZE drug-delivery technology. The agreement is the largest in company history including a $105 million upfront payment and $160 million in potential milestones for each of 11 immuno-oncology targets, including the initial target selection of programmed death 1 (PD-1).
Roche licensing a new ENHANZE target in exchange for a $30 million upfront payment and up to $160 million in potential development, regulatory and sales-based milestones. The agreement serves as an extension to the original collaboration between the companies, under which Roche has developed two subcutaneous formulations of cancer drugs for markets worldwide.
Janssen initiating the first of three planned Phase 3 studies of the subcutaneous formulation of DARZALEX (daratumumab). Halozyme’s ENHANZE technology has the potential to enable a 15 ml injection to be delivered in five minutes or less, with no requirement for an intravenous loading dose. Data informing this decision from the Phase 1 PAVO study in patients with relapsed or refractory multiple myeloma were accepted for presentation at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. Upon the dosing of the third patient in the recently initiated study, Halozyme will earn a $15 million milestone payment.
Genentech launching RITUXAN HYCELA (rituximab/hyaluronidase human) for subcutaneous injection, a combination of rituximab and Halozyme’s hyaluronidase human ENHANZE technology, for patients with follicular lymphoma, diffuse large B-cell lymphoma and chronic lymphocytic leukemia.
Continued progress screening and enrolling patients in the HALO-301 study of PEGPH20 in combination with ABRAXANE (nab-paclitaxel) and gemcitabine in first line metastatic pancreas cancer patients with high levels of tumor hyaluronan (HA-High). An interim analysis will be conducted for the first primary endpoint of progression-free survival when the target number of events has been reached, which the company projects will be in late Q4 2018. At that time, Halozyme projects approximately 500 patients will have been enrolled in the study.
Initiating multiple trials in collaboration with Genentech to evaluate PEGPH20 in combination with TECENTRIQ (atezolizumab) in four tumor types. Studies include a Halozyme-sponsored randomized clinical trial in patients with previously untreated, unresectable, locally advanced, or metastatic cholangiocarcinoma and gallbladder adenocarcinoma and two Genentech-funded and operated, Phase 1b/2 multi-arm clinical studies evaluating patients with previously treated metastatic pancreatic ductal adenocarcinoma and previously treated locally advanced unresectable or metastatic gastric cancer. The studies are part of a clinical collaboration agreement announced in 2016 to evaluate PEGPH20 and atezolizumab in up to eight tumor types.
Third Quarter 2017 Financial Highlights

Revenue for the third quarter was $63.7 million compared to $31.9 million for the third quarter of 2016. The year-over-year increase was driven by a $30 million upfront payment from Roche and growth in royalties from partner sales of Herceptin (trastuzumab) SC, MabThera (rituximab) SC and HYQVIA (Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase) offset by a decrease in research and development reimbursements and license payments from ENHANZE partners. Revenue for the third quarter included $17.1 million in royalties, an increase of 31 percent from the prior-year period, $9.8 million in sales of bulk rHuPH20 primarily for use in manufacturing collaboration products and $3.8 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Research and development expenses for the third quarter were $34.0 million, compared to $33.9 million for the third quarter of 2016. The increase was primarily due to a ramp in spending associated with the HALO-301 study.
Selling, general and administrative expenses for the third quarter were $13.3 million, compared to $11.6 million for the third quarter of 2016. The increase was primarily due to personnel expenses, including stock compensation, for the period.
Net income for the third quarter was $2.7 million, or $0.02 per share, compared to net loss in the third quarter of 2016 of $28.9 million, or $0.23 per share.
Cash, cash equivalents and marketable securities were $316.9 million at September 30, 2017, compared to $297.5 million at June 30, 2017.
Financial Outlook for 2017

Halozyme updated year-end guidance, now expecting:

Net revenue increasing from the prior range of $245 million to $260 million announced on Sept. 14 to $265 million to $280 million, driven by stronger product sales, royalties, and sponsored research;
Operating expenses decreasing from the prior range of $240 million to $250 million to $230 million to $240 million;
Positive operating cash flow increasing from the prior range of $50 million to $60 million to $70 million to $85 million;
Year-end cash balance increasing from the prior range of $380 million to $395 million to $400 million to $415 million.
Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the third quarter of 2017 today, Tuesday, November 7 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Helen Torley, president and chief executive officer, will lead the call. The call will be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit View Source approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed at (877) 410-5657 (domestic callers) (334) 323-7224 (international callers) using passcode 769890. A telephone replay will be available after the call by dialing (877) 919-4059 (domestic callers) or (334) 323-0140 (international callers) using replay ID number 19320711.

Alnylam has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Alnylam, 2017, NOV 7, 2017, View Source [SID1234521660]).

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On November 7, 2017 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to improve the survival and quality of life of cancer patients, reported financial results for the third quarter ended September 30, 2017 and provided an overview of certain corporate developments and plans (Press release, Verastem, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2314866 [SID1234521654]).

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"The third quarter was a pivotal time for Verastem with the announcement of positive top-line data from the pivotal Phase 3 DUO study evaluating oral duvelisib monotherapy in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL)," said Robert Forrester, President and Chief Executive Officer of Verastem. "The DUO study met its primary endpoint, with progression-free survival (PFS) significantly favoring duvelisib monotherapy over ofatumumab, an approved standard of care treatment for patients with CLL/SLL. We recently met with the U.S. Food and Drug Administration (FDA), and based on the meeting and written feedback, we intend to submit, during the first quarter of 2018, 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). The NDA will be based on a comprehensive clinical data package, including the Phase 3 DUO and Phase 2 DYNAMO studies evaluating duvelisib in patients with advanced hematologic malignancies."

Mr. Forrester concluded, "At the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 Annual Meeting (ASH 2017), we will be presenting data from multiple studies, including the detailed positive results from the Phase 3 DUO study, which were selected for an oral presentation."

Third Quarter 2017 and Recent Highlights:

Duvelisib

Announced Regulatory Strategy for Duvelisib NDA – Verastem recently announced that a meeting was held with the U.S. Food and Drug Administration (FDA) regarding the regulatory path for duvelisib. Based on the meeting with, and written feedback from the FDA, Verastem intends to submit 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 FL. Along with the clinical data from the DUO study, the duvelisib NDA submission will also contain the favorable results from the Phase 2 DYNAMO study in double-refractory indolent non-Hodgkin’s lymphoma (iNHL), which also achieved its primary endpoint with an ORR of 46% (p<0.0001). In the subset of patients enrolled in the DYNAMO study with double-refractory FL (n=83), duvelisib demonstrated an ORR of 41%. The Company expects to submit the duvelisib NDA during the first quarter of 2018.
Reported Positive Top-line Data from the Pivotal Phase 3 DUO Study in Relapsed or Refractory CLL/SLL – In September 2017, Verastem reported that the Phase 3 DUO study met its primary endpoint with oral duvelisib monotherapy demonstrating superiority over ofatumumab for PFS in patients with CLL/SLL. In this study, duvelisib achieved a statistically significant improvement in median PFS of 13.3 months, compared to 9.9 months for ofatumumab with a hazard ratio of 0.52 (p<0.0001), representing a 48% reduction in the risk of progression or death. Duvelisib monotherapy had 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.
Expanded Duvelisib Program to Include Peripheral T-Cell Lymphoma – In September 2017, Verastem announced the expansion of its duvelisib development program to include targeting the treatment of patients with Peripheral T-Cell Lymphoma (PTCL). Duvelisib was granted Fast Track designation by the FDA for the treatment of patients with PTCL who have received at least one prior therapy. Development of duvelisib in PTCL is supported by encouraging Phase 1 clinical data which demonstrated a 50% investigator-assessed overall response rate in 16 heavily pre-treated patients with relapsed or refractory PTCL, including 3 (19%) complete responses and 5 (31%) partial responses. Verastem intends to initiate an open-label, multicenter, Phase 2 clinical trial evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory PTCL by the end of 2017.
Clinical Data from Pivotal Phase 3 DUO Study Selected for Oral Presentation at ASH (Free ASH Whitepaper) 2017 – Verastem recently announced that an abstract highlighting clinical data from the Phase 3 DUO study was selected for oral presentation at ASH (Free ASH Whitepaper) 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," will be presented by principal investigator, Ian Flinn, M.D., Ph.D., Director of the Blood Cancer Research Program at Sarah Cannon Research Institute, on Sunday, December 10, 2017 at 4:30pm ET at the Georgia World Congress Center, in Building B, Level 5, Murphy BR 3-4.
Additional Duvelisib Abstracts Selected for Presentation at ASH (Free ASH Whitepaper) 2017 – Along with the Phase 3 DUO results, two additional duvelisib abstracts were selected for presentation 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," will be given as an oral presentation by Steven Horowitz, M.D., Memorial Sloan Kettering Cancer Center, on Monday, December 11, 2017 at 5:00pm ET at the Georgia World Congress Center, in Building A, Level 4, Marcus Auditorium. The abstract, 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," will be given as a poster presentation by Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem, on Saturday, December 9, 2017 from 5:30-7:30pm ET at the Georgia World Congress Center, in Building A, Level 1, Hall A2.
Verastem to Host Key Opinion Leader Event at ASH (Free ASH Whitepaper) 2017 – On Sunday, December 10, 2017, Verastem will host an investor and analyst reception, which will feature a moderated panel discussion/Q&A including Ian Flinn, MD, Director of the Blood Cancer Research Program at Sarah Cannon Cancer Center in Nashville, TN. The event will take place during the ASH (Free ASH Whitepaper) 2017 annual meeting and interested parties can access a live webcast of the event beginning December 10, 2017 at 8pm ET by going to the "News and Press" section of the Verastem website at www.verastem.com.
Defactinib

Published Scientific Data Highlighting Potential Role of Focal Adhesion Kinase (FAK) Inhibition in Pancreatic and Breast Cancer – In July 2017, Verastem announced the publication of two papers in the peer-reviewed journals, PLoS One and Oncotarget. The two published articles reported scientific findings from studies evaluating FAK inhibition in preclinical models of pancreatic and breast cancer and continue to validate the underlying thesis for ongoing clinical collaborations evaluating Verastem’s lead FAK inhibitor, defactinib, in combination with chemotherapeutic and leading immunotherapeutic agents in several difficult to treat types of cancer. The PLoS One paper in pancreatic cancer is available here and the Oncotarget paper in breast cancer is available here.
Defactinib Preclinical Abstract Selected for Presentation at ASH (Free ASH Whitepaper) 2017 – An abstract describing preclinical data in combination with BCL-2 was selected for presentation at ASH (Free ASH Whitepaper) 2017. The abstract, titled "Combinatorial Inhibition of Focal Adhesion Kinase and BCL-2 in AML," will be given as a poster presentation by Xiangmeng Wang, Ph.D., on Sunday, December 10, 2017 from 6:00-8:00pm ET at the Georgia World Congress Center, in Building A, Level 1, Hall A2.
Corporate and Financial

Brian Stuglik, R.Ph. Appointed to the Board of Directors – In September 2017, Verastem announced the appointment of Mr. Stuglik to its Board. Mr. Stuglik, former Chief Marketing Officer for Lilly Oncology, brings to Verastem 35 years of experience in pharmaceutical and oncology commercialization in both the U.S. and international markets. He has successfully launched several multi-billion dollar brands over his career, including Gemzar, Alimta and Erbitux.
NgocDiep Le, M.D., Ph.D., 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 hematologic malignancies as well as IND and NDA submissions. Dr. Le joins Verastem from MedImmune (a 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. Prior to joining MedImmune, Dr. Le held roles of increasing responsibilities at Novartis and at GlaxoSmithKline where she led the MEK inhibitor, trametinib (MekinistTM), from the first-in-human studies to FDA approval. Dr. Le received a Bachelor in Science degree from the California Institute of Technology, and earned both MD and PhD degrees from Stanford University School of Medicine. Dr. Le will oversee the development strategy and activities for Verastem’s duvelisib and defactinib.
Julie B. Feder Appointed Chief Financial Officer – In July 2017, Verastem announced the appointment of Ms. Feder as its Chief Financial Officer. Ms. Feder is an accomplished financial professional with invaluable leadership experience in the healthcare industry. She joins Verastem from the Clinton Health Access Initiative, Inc. (CHAI), where she served as Chief Financial Officer. Prior to joining CHAI, Ms. Feder held finance roles of increasing responsibility at Genzyme Corporation including leading the internal audit function. Ms. Feder began her career at Deloitte & Touche LLP and she holds a Bachelor of Science in Accounting from Yeshiva University’s Sy Syms School of Business.
Achieved First Development Milestone Related to the Duvelisib License Agreement – In September 2017, upon achievement of positive top-line results from the Phase 3 DUO study, Verastem determined that the pre-specified criteria for the first milestone under the license agreement with Infinity Pharmaceuticals, Inc. (Infinity) had been met and recorded $6.0 million as research and development expense. Subsequently, in October 2017, Verastem made the milestone payment of $6.0 million to Infinity. The milestone was paid using funds drawn from Verastem’s existing loan and security agreement with Hercules Capital, Inc.
Third Quarter 2017 Financial Results

Net loss for the three months ended September 30, 2017 (2017 Quarter) was $23.1 million, or $0.61 per share, as compared to a net loss of $7.9 million, or $0.21 per share, for the three months ended September 30, 2016 (2016 Quarter). Net loss includes non-cash stock-based compensation expense of $1.7 million and $1.3 million for the 2017 Quarter and 2016 Quarter, respectively. Verastem used $11.8 million for operating activities during the 2017 Quarter.

Research and development expense for the 2017 Quarter was $17.7 million compared to $4.2 million for the 2016 Quarter. The $13.5 million increase from the 2016 Quarter to the 2017 Quarter was primarily related to the achievement of a $6.0 million milestone pursuant to Verastem’s license agreement with Infinity, an increase of $4.8 million in contract research organization (CRO) expense for outsourced biology, development and clinical services, which includes Verastem’s clinical trial costs, an increase of approximately $2.0 million in consulting fees, an increase in stock-based compensation of approximately $423,000 and an increase in personnel related costs of approximately $153,000.

General and administrative expense for the 2017 Quarter was $5.4 million compared to $3.8 million for the 2016 Quarter. The increase of $1.6 million from the 2016 Quarter to the 2017 Quarter primarily resulted from increases in consulting and professional fees of $1.3 million and personnel costs of approximately $330,000.

As of September 30, 2017, Verastem had cash, cash equivalents and investments of $60.3 million compared to $80.9 million as of December 31, 2016.

The number of outstanding common shares as of September 30, 2017, was 39,945,028.

Financial Guidance

Based on our current operating plans, we expect to have sufficient cash, cash equivalents and investments to fund our research and development programs and operations into the second half of 2018.

About Duvelisib

Duvelisib is an 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-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib is currently being evaluated in late- and mid-stage clinical trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (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 is preparing to submit a New Drug Application to the U.S. Food and Drug Administration for the treatment of patients with relapsed or refractory CLL/SLL and patients with follicular lymphoma (FL) whose disease has progressed and are refractory to rituximab and to either chemotherapy or radioimmunotherapy. 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 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, enhancement of anti-tumor immunity, and reduction of cancer stem cells.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, ovarian, non-small cell lung cancer, 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.