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Galectin Therapeutics Reports 2017 Third Quarter Financial Results and Provides Business Update

On November 7, 2017 Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, reported financial results for the three months ended September 30, 2017 (Press release, Galectin Therapeutics, NOV 7, 2017, View Source [SID1234521648]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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"All of the patients in our NASH Cirrhosis, NASH-CX Phase 2b Clinical Trial have completed all 52 weeks of infusions and 100% of the doses have been administered," said Peter G. Traber, M.D., president, chief executive officer and chief medical officer of Galectin Therapeutics. "The dropout rate was well below expectations, with 151 subjects out of the 162 enrolled having completed the full trial regimen. The data will be compiled and analyzed in expectation that we will meet our original target of reporting top line data in early December 2017."

Expected Upcoming Milestones

Company remains on track to report top line data from the NASH-CX Phase 2b Clinical Trial in December 2017.
Summary of Key Development Programs and Updates

Company is funded through February 2018, which is sufficient to report top line data of the NASH-CX Phase 2b Clinical Trial.
Dr. Peter G. Traber, M.D., the Company’s president, chief executive officer and chief medical officer was Chair of the Conference for NASH Summit Europe 2017, an industry nonalcoholic steatohepatitis (NASH) drug development forum that was held in Frankfurt, Germany from October 10-12, 2017.
The Company received a Decision to Grant from the Chinese Patent Office for its patent application for "Composition of Novel Carbohydrate Drug for Treatment of Human Diseases," which, when issued, will extend composition of matter coverage of the Company’s lead compound, GR-MD-02, to China, where the prevalence of fatty liver disease has approximately doubled over the past two decades, with around 15% of the population experiencing NASH.
Financial Results

For the three months ended September 30, 2017, the Company reported a net loss applicable to common stockholders of $4.7 million, or $0.13 per share, compared with a net loss applicable to common stockholders of $4.5 million, or $0.16 per share, for the three months ended September 30, 2016. The decrease is largely due to lower general and administrative expenses and to lower stock compensation expenses.

Research and development expense for the three months ended September 30, 2017 was $3.5 million, compared with $3.3 million for the three months ended September 30, 2016. The increase primarily is primarily related to higher pre-clinical and drug manufacturing expenses.

General and administrative expense for quarter was approximately $900,000, compared with $1.2 million for the prior year, with the decrease being primarily related to lower investor relations and non-cash stock compensation expenses.

As of September 30, 2017, the Company had $7.0 million of non-restricted cash and cash equivalents. The Company believes it has sufficient cash to fund currently planned operations and research and development activities through December 31, 2017.

Infinity Provides Company Update and Reports Third Quarter 2017 Financial Results

On November 7, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its third quarter 2017 financial results and provided an update on the company, including its progress with IPI-549, an oral immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase-gamma (PI3K-gamma) (Press release, Infinity Pharmaceuticals, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2314924 [SID1234521651]). Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in a four-part Phase 1/1b study in patients with advanced solid tumors. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor, M2, to an anti-tumor, M1, phenotype and is able to overcome resistance to checkpoint inhibition as well as to enhance the activity of checkpoint inhibitors.1,2 IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

On November 10, 2017, a late-breaking abstract summarizing data from the recently completed monotherapy dose-escalation component of the study will be presented at the 2017 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting taking place in National Harbor, MD. The presentation, which will be made during an oral session on new agents, will include an analysis of the safety, activity, pharmacokinetics and pharmacodynamics of IPI-549 in 19 patients who received once daily (QD) doses of IPI-549 ranging from 10 mg to 60 mg. Initial translational data describing how inhibiting PI3K-gamma may elicit an immune response and identifying potential biomarkers of immune response will also be presented. In August, Infinity announced that the company designated the 60 mg QD dose for evaluation in the monotherapy expansion component of the study, which is now under way.

"We are looking forward to sharing the full data from the completed monotherapy dose escalation component of our study later this week at the SITC (Free SITC Whitepaper) Annual Meeting. In addition to reporting on the safety and activity of IPI-549 monotherapy, for the first time we will present initial translational data from patients treated with IPI-549 as a monotherapy that can help us better understand how IPI-549 may activate an immune response," stated Adelene Perkins, Infinity’s chair and chief executive officer. "Patient enrollment in the monotherapy expansion component of the study is progressing very well, and we expect to initiate the combination expansion portion of the study by the end of this year, positioning us for data readouts from the monotherapy expansion cohort as well as data from the combination dose escalation and disease-specific expansion cohorts, all in the first half of 2018."

The Phase 1/1b study of IPI-549 is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and activity of IPI-549 as a monotherapy and in combination with the approved dose of Opdivo in approximately 200 patients with advanced solid tumors. The study includes four components: monotherapy dose escalation, combination therapy dose escalation, monotherapy expansion and combination expansion. The combination expansion will include multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma, and head and neck squamous cell carcinoma (HNSCC) whose tumors show initial resistance or subsequently develop resistance to immune checkpoint blockade therapy. Additionally, the combination expansion component will also evaluate a cohort of patients with triple negative breast cancer (TNBC) who have not been previously exposed to anti-PD-1 or anti-PD-L1 therapy.

Based on progress made during 2017, Infinity expects to achieve the following IPI-549 data milestones in 2018:

Report data from the monotherapy expansion component of the study in the first half of 2018
Report data from the combination dose-escalation component of the study in the first half of 2018
Report initial data from the combination expansion component of the study in the first half of 2018, with more mature data from the combination expansion in the second half of 2018
"We are also focused on maintaining strong fiscal discipline and anticipate that we have cash runway into the first quarter of 2019, allowing us to generate safety and activity data on all four components of our Phase 1/1b study of IPI-549," commented Lawrence Bloch, M.D., J.D., president of Infinity. "We also have the potential to receive a future $22 million payment from Verastem upon the first regulatory approval of duvelisib, which we out-licensed in 2016, that could further strengthen our financial profile."

Recent Corporate Developments

$6.0 million payment received from Verastem: In October, Infinity received a $6.0 million cash payment from Verastem, Inc. The payment was made following the determination that the Phase 3 DUO clinical study evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma met certain pre-specified criteria at completion. Infinity out-licensed duvelisib to Verastem in November 2016.

Verastem recently provided guidance that it plans to submit a new drug application for duvelisib in the first quarter of 2018. Infinity is entitled to receive a $22 million payment from Verastem upon the first regulatory approval for duvelisib inside or outside the U.S.
Recent Clinical Developments

IPI-549 selected for an oral presentation at SITC (Free SITC Whitepaper) Annual Meeting: In October, Infinity announced that a late-breaking abstract describing new data for IPI-549 was selected for presentation during an oral session on new agents at the SITC (Free SITC Whitepaper) Annual Meeting. The abstract, "Monotherapy dose escalation clinical and translational data from first-in-human study in advanced solid tumors of IPI-549, an oral, selective, PI3K-gamma inhibitor targeting tumor macrophages" (Abstract O43), will be presented on Friday, November 10, 2017, from 2:15 p.m. – 2:30 p.m. ET. The lead author and presenter is David Hong, M.D., Deputy Chair, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX. An identically titled accompanying poster will also be presented on Friday, November 10, 2017, from 12:30 p.m. – 2:00 p.m. ET and 6:30 p.m. – 8:00 p.m. ET.

Additionally, a clinical trials in progress poster, "Phase 1/1b, first-in-human study of the PI3K-gamma inhibitor IPI-549 as monotherapy and combined with nivolumab in patients with advanced solid tumors" (Abstract P219), will be presented on Friday, November 10, 2017, from 12:30 p.m. – 2:00 p.m. ET and 6:30 p.m. – 8:00 p.m. ET. The lead author on the poster is Antoni Ribas, M.D., Ph.D., Parker Institute Center Director at the University of California, Los Angeles (UCLA).

Infinity will host a reception for investors and analysts on Friday, November 10, 2017, from 6:00 a.m. to 8:00 a.m. ET to discuss the clinical development of IPI-549, including a review of data from the ongoing clinical study. The presentation portion of the reception will be webcast live beginning at 6:30 a.m. ET. The webcast and accompanying slides will be available in the "investors/media" section of the company’s website, www.infi.com. A replay of the event will also be available.
Expanded clinical collaboration with Bristol-Myers Squibb: In September, Infinity announced that it expanded its existing clinical collaboration with Bristol-Myers Squibb evaluating IPI-549 in combination with Opdivo to include patients with TNBC who have not been previously exposed to anti-PD-1 or anti-PD-L1 therapy. The TNBC cohort will be part of the combination expansion component of the Phase 1/1b study.
Phase 1/1b study of IPI-549 advancing: The company is continuing to advance the Phase 1/1b study of IPI-549 both as a monotherapy and in combination with Opdivo in patients with solid tumors. Infinity has completed an evaluation of escalating monotherapy doses of IPI-549 ranging from 10 mg QD to 60 mg QD and selected the 60 mg dose for evaluation in the monotherapy expansion component of the study. Dose escalation evaluating IPI-549 40 mg QD in combination with Opdivo is ongoing, and Infinity expects to initiate the combination expansion component of the study by the end of 2017.
Third Quarter 2017 Financial Results

At September 30, 2017, Infinity had total cash, cash equivalents and available-for-sale securities of $55.6 million, compared to $66.2 million at June 30, 2017. The September 30, 2017 cash and investments balance excludes the $6.0 million payment from Verastem, which Infinity received in October 2017.
Revenue for the third quarter of 2017 was $6.0 million, all of which related to the amounts due from Verastem for the DUO study meeting the pre-specified criteria at completion. Infinity did not record any revenue during the third quarter of 2016.
Research and development (R&D) expense for the third quarter of 2017 was $9.3 million, compared to $12.8 million for the same period in 2016. The decrease in R&D expense was related to the company’s 2016 restructuring activities, partially offset by the $6.0 million convertible note issued to Takeda in exchange for eliminating Infinity’s royalty obligation to Takeda for selective inhibitors of PI3K gamma, including IPI-549.
General and administrative (G&A) expense was $4.5 million for the third quarter of 2017 compared to $7.1 million for the same period in 2016. The decrease in G&A expense was primarily due to the company’s 2016 restructuring activities.
Net loss for the third quarter of 2017 was $7.1 million, or a basic and diluted loss per common share of $0.14, compared to net loss of $19.5 million, or a basic and diluted loss per common share of $0.39, for the third quarter of 2016.
Cash and Investments Outlook

Infinity’s 2017 financial outlook remains as follows:

Net loss: Infinity expects net loss for 2017 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2017 with a cash, cash equivalents and available-for-sale securities balance ranging from $40 million to $50 million.
Based on its current operational plans, Infinity expects that its cash, cash equivalents and available-for-sale securities at September 30, 2017, will be adequate to satisfy the company’s capital needs into the first quarter of 2019. The company’s financial outlook excludes additional funding or business development activities.

Conference Call Information
Infinity will host a conference call today, November 7, 2017, at 8:30 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) or 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 97100725. An archived version of the webcast will be available on Infinity’s website for 60 days.

About the IPI-549 and the Ongoing Phase 1 Study
IPI-549 is an investigational, orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor, M2, to an anti-tumor, M1, phenotype and is able to overcome resistance to checkpoint inhibition as well as to enhance the activity of checkpoint inhibitors.1,2 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1/1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.3 The four-part study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. Patient enrollment is complete in monotherapy dose-escalation, and monotherapy expansion is ongoing. Combination dose-escalation is also ongoing, and combination expansion is expected to begin by the end of 2017.

The combination expansion component includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma, and head and neck squamous cell carcinoma (HNSCC) whose tumors show initial resistance or subsequently develop resistance to immune checkpoint blockade therapy. This combination expansion component will now also add a cohort of patients with triple negative breast cancer (TNBC) who have not been previously exposed to immune checkpoint blockade therapy. Although there has been great progress in the treatment of cancer, there remains a need for additional treatment options. NSCLC, melanoma, HNSCC and TNBC account for more than 22 percent of all new cancer cases in the U.S.4,5

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

Keryx Biopharmaceuticals Announces Third Quarter 2017 Financial Results

On November 7, 2017 Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX), a biopharmaceutical company focused on bringing innovative medicines to people with kidney disease, reported its financial results for the quarter ended September 30, 2017 (Press release, Keryx Biopharmaceuticals, NOV 7, 2017, View Source [SID1234521652]). The company also reviewed its progress with Auryxia, upcoming milestones, and withdrew its 2017 financial guidance.

"Auryxia prescriptions grew 18 percent in the third quarter compared to the second quarter of 2017, reflecting continued adoption of Auryxia; however, this growth was offset by a change in our payer mix," said Greg Madison, president and chief executive officer of Keryx Biopharmaceuticals. "Our confidence in Auryxia remains strong with brand awareness and clinical experience expanding. With the approval of Auryxia for the treatment of iron deficiency anemia in patients with chronic kidney disease, not on dialysis, we are thrilled to have the opportunity to now make Auryxia available to millions of people in the United States living with this condition. With a medicine that can treat two complications of chronic kidney disease, we believe we have a strong foundation from which to build a leading kidney care company."

Third Quarter 2017 Financial Results

Approximately 24,900 Auryxia prescriptions were written in the third quarter of 2017, representing 18 percent growth over the second quarter of 2017.
Keryx reported third quarter 2017 net U.S. Auryxia product sales of $13.6 million; sequential quarter prescription growth was offset by an increase in the gross-to-net adjustment due to a change in the mix of business, with greater prescription volume coming from patients with Medicare Part D insurance.
Given the potential of the additional Auryxia indication on future prescription demand and the dynamics surrounding Auryxia’s payer mix, prescription demand growth and the resulting net U.S. Auryxia product sales in the third quarter, Keryx is withdrawing its 2017 financial guidance.
Recent Business Highlights

The FDA approved Auryxia for an additional indication for the treatment of iron deficiency anemia in adults with chronic kidney disease, not on dialysis. Additional information on the approval is available in a separate press release issued today by Keryx Biopharmaceuticals.
Three abstracts related to Auryxia – one oral presentation and two poster presentations – were presented at the American Society of Nephrology’s (ASN) 2017 Kidney Week. The accepted abstracts are available online on the ASN conference website.
Third Quarter September 30, 2017 Financial Results
"Third quarter net U.S. product sales were lower than anticipated due to a change in the payer mix for Auryxia as well as lower-than-expected growth in prescriptions," said Scott Holmes, senior vice president and chief financial officer of Keryx Biopharmaceuticals. "The growth in Medicare Part D prescriptions, aided by the two large payer contracts added in March and June of this year, outpaced growth in the remainder of the business, leading to a faster acceleration in our gross-to-net adjustment than anticipated. The broad payer coverage across Medicare Part D and commercial plans we have today is critical to continued adoption of Auryxia for the treatment of hyperphosphatemia and positions us well in terms of access for those patients with iron deficiency anemia and chronic kidney disease, not on dialysis."

Total revenues for the quarter ended September 30, 2017 were $15.0 million, compared with $6.3 million during the same period in 2016. Total revenues for the third quarter of 2017 include $13.6 million in net U.S. Auryxia product sales, compared to $5.1 million in the third quarter of 2016. Total revenues also include $1.4 million in license revenue during the third quarter of 2017 and $1.3 million in the third quarter of 2016.

Cost of goods sold for the quarter ended September 30, 2017 were $5.9 million, compared with $18.2 million during the same period in 2016.

Selling, general and administrative expenses for the quarter ended September 30, 2017 were $22.7 million, as compared to $20.5 million during the same period in 2016. The increase was related to the continued commercialization of Auryxia, and preparations for Auryxia’s launch in iron deficiency anemia.

Research and development expenses for the quarter ended September 30, 2017 were $9.3 million, as compared to $8.7 million during the same period in 2016. The increase was primarily related to manufacturing and clinical activities to support the long-term growth of Auryxia.

Net loss for the quarter ended September 30, 2017 was $23.5 million, or $0.20 per share, compared to a net loss of $41.7 million, or $0.39 per share, for the comparable period in 2016.

Cash and cash equivalents as of September 30, 2017 totaled $114.0 million compared to $111.8 million as of December 31, 2016.

Conference Call Information
Keryx Biopharmaceuticals will host an investor conference call today at 8:00 a.m. ET to discuss financial results for the third quarter of 2017 and the FDA approval of Auryxia for the treatment of iron deficiency anemia in patients with chronic kidney disease, not on dialysis. To participate in the conference call, please call 1-(888) 396-2320 (U.S.), 1-(774) 264-7560 (outside the U.S.), call-in ID: 2193619. The call will also be webcast with slides, which will be accessible through the Investors section of the company’s website at www.keryx.com. The audio replay will be available at View Source for approximately 15 days after the call.

About Iron Deficiency Anemia in Adults with Chronic Kidney Disease, not on Dialysis
One out of every seven adults in the U.S. has chronic kidney disease. This disease carries a significant burden with complex issues requiring many different medications. A common complication of CKD is iron deficiency anemia. Iron is an essential mineral for the human body and is typically obtained from the diet. It is a critical component of human blood as it is necessary to make healthy red blood cells. People with chronic kidney disease often have anemia as a result of insufficient iron (called iron deficiency anemia) and do not produce enough hemoglobin, the component of the red blood cell that carries oxygen throughout the body. Iron deficiency anemia can negatively impact a patient’s quality of life and is associated with cardiovascular complications and increased mortality risk. Based on market research, Keryx estimates that nephrologists currently treat 650,000 people for iron deficiency anemia who have chronic kidney disease and are not on dialysis. There are estimated to be an additional 250,000 – 400,000 people under the care of a nephrologist who have chronic kidney disease and iron deficiency anemia but are not treated today. The prevalence and severity of iron deficiency anemia increases as kidney disease progresses.

About Auryxia (ferric citrate) tablets
Auryxia (ferric citrate) was approved by the U.S. Food and Drug Administration on September 5, 2014 for the control of serum phosphorus levels in adults with chronic kidney disease on dialysis and approved on November 6, 2017 for the treatment of iron deficiency anemia in adults with chronic kidney disease not on dialysis. Auryxia tablets were designed to contain 210 mg of ferric iron, equivalent to 1 gram of ferric citrate, and offers convenient mealtime dosing. The starting dose of Auryxia for the treatment of hyperphosphatemia for patients on dialysis is six tablets per day (two per meal) and for the treatment of iron deficiency anemia in patients not on dialysis is three tablets per day (one per meal). For more information about Auryxia and the U.S. full prescribing information, please visit www.Auryxia.com.

IMPORTANT U.S. SAFETY INFORMATION FOR AURYXIA (ferric citrate)

CONTRAINDICATION

AURYXIA (ferric citrate) is contraindicated in patients with iron overload syndromes, e.g., hemochromatosis.

WARNINGS AND PRECAUTIONS

Iron Overload: Increases in serum ferritin and transferrin saturation (TSAT) were observed in clinical trials with AURYXIA in patients with chronic kidney disease (CKD) on dialysis treated for hyperphosphatemia, which may lead to excessive elevations in iron stores. Assess iron parameters prior to initiating AURYXIA and monitor while on therapy. Patients receiving concomitant intravenous (IV) iron may require a reduction in dose or discontinuation of IV iron therapy.
Risk of Overdosage in Children Due to Accidental Ingestion: Accidental ingestion and resulting overdose of iron-containing products is a leading cause of fatal poisoning in children under 6 years of age. Advise patients of the risks to children and to keep AURYXIA out of the reach of children.
ADVERSE REACTIONS

Most common adverse reactions with AURYXIA were:

Hyperphosphatemia in CKD on Dialysis: Diarrhea (21%), discolored feces (19%), nausea (11%), constipation (8%), vomiting (7%) and cough (6%)
Iron Deficiency Anemia in CKD Not on Dialysis: Discolored feces (22%), diarrhea (21%), constipation (18%), nausea (10%), abdominal pain (5%) and hyperkalemia (5%)
SPECIFIC POPULATIONS

Pregnancy and Lactation: There are no available data on AURYXIA use in pregnant women to inform a drug-associated risk of major birth defects and miscarriage. However, an overdose of iron in pregnant women may carry a risk for spontaneous abortion, gestational diabetes and fetal malformation. Data from rat studies have shown the transfer of iron into milk, hence, there is a possibility of infant exposure when AURYXIA is administered to a nursing woman.
To report suspected adverse reactions, contact Keryx Biopharmaceuticals at 1-844-445-3799

Verastem Reports Third Quarter 2017 Financial Results

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]).

"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.

New Data Featuring Nektar Therapeutics’ Wholly-Owned Immuno-Oncology Pipeline to be Presented at the 2017 Society for Immunotherapy of Cancer 32nd Annual Meeting

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

"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.