On November 3, 2016 Juno Therapeutics, Inc. (NASDAQ: JUNO), a biopharmaceutical company focused on re-engaging the body’s immune system to revolutionize the treatment of cancer, reported that seven oral and four poster presentations detailing updated clinical and preclinical results generated in partnership with its collaborators will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Juno, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219226 [SID1234516282]). Senior executives will also review results and provide an update on Juno’s clinical development program at an analyst and investor event, which will also be available via webcast.

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"Presentations at ASH (Free ASH Whitepaper) will showcase new data that support our best-in-class CAR T strategy across a range of B-cell malignancies," said Mark J. Gilbert, M.D., Juno’s Chief Medical Officer. "The responses we are seeing with our product candidates, including updated JCAR017 results in adults with relapsed/refractory NHL, demonstrate the potential of our CAR T programs for patients in need of more treatment options. We are also learning more about cell characterization and toxicities, which are critical to improving the patient experience, our product candidates’ benefit-to-risk ratio, and potentially moving CAR T treatment into earlier lines of therapy."
New data with JCAR017 in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), which is a subtype of NHL, and final data from the PLAT-02 trial for pediatric patients with relapsed/refractory acute lymphoblastic leukemia (ALL) will be presented. JCAR017 uses a defined CD4:CD8 cell composition and 4-1BB as the costimulatory domain, which differentiates it from other CD19-directed CAR T product candidates advancing to commercialization. In addition, final data from a Phase I trial with JCAR014 in high-risk, ibrutinib-refractory patients with chronic lymphocytic leukemia (CLL) will also be presented in an oral presentation.
The following will be presented at ASH (Free ASH Whitepaper):
Oral Presentations
CD19 CAR T Cells Are Highly Effective in Ibrutinib-Refractory Chronic Lymphocytic Leukemia (Abstract #56)
Presenter: Cameron J. Turtle, M.B.B.S., Ph.D., Fred Hutchinson Cancer Research Center
Date: Saturday, December 3, 2016, 7:45 a.m. Pacific Time
Location: San Diego Convention Center, Room 6AB
Implications of Concurrent Ibrutinib Therapy on CAR T-Cell Manufacturing and Phenotype and on Clinical Outcomes Following CD19-Targeted CAR T-Cell Administration in Adults with Relapsed/Refractory CLL (Abstract #58)
Presenter: Mark B. Geyer, M.D., Memorial Sloan Kettering Cancer Center
Date: Saturday, December 3, 2016, 8:15 a.m. Pacific Time
Location: San Diego Convention Center, Room 6AB
CD19 CAR T Cell Products of Defined CD4:CD8 Composition and Transgene Expression Show Prolonged Persistence and Durable MRD-Negative Remission in Pediatric and Young Adult B-Cell ALL (Abstract #219)
Presenter: Rebecca Gardner, M.D., Seattle Children’s Research Institute
Date: Saturday, December 3, 2016, 4:30 p.m. Pacific Time
Location: Marriott Marquis San Diego Marina, Marriott Grand Ballroom Salons 11-13
Minimal Residual Disease Negative Complete Remissions Following Anti-CD22 Chimeric Antigen Receptor (CAR) in Children and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL) (Abstract #650)
Presenter: Nirali Shah, Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute
Date: Monday, December 5, 2016, 7:15 a.m. Pacific Time
Location: San Diego Convention Center, Room 6CF
Creation of The First Non-Human Primate (NHP) Model That Faithfully Recapitulates Chimeric Antigen Receptor (CAR) T cell-mediated Cytokine Release Syndrome (CRS) and Neurologic Toxicity Following B Cell-directed CAR T Cell Therapy (Abstract #651)
Presenter: Agne Taraseviciute, Seattle Children’s Research Institute
Date: Monday, December 5, 2016, 7:15 a.m. Pacific Time
Location: San Diego Convention Center, Room 6CF
Decreased Rates of Severe CRS Seen with Early Intervention Strategies for CD19 CAR T Cell Toxicity Management (Abstract #586)
Presenter: Rebecca Gardner, M.D., Seattle Children’s Research Institute
Date: Monday, December 5, 2016, 7:45 a.m. Pacific Time
Location: Marriott Marquis San Diego Marina, Marriott Grand Ballroom Salons 2-4
EBV-Specific Donor Cells Transduced to Express a High-Affinity WT1 TCR Can Prevent Recurrence in Post-HCT Patients with High-Risk AML (Abstract #1001)
Presenter: Aude G. Chapuis, M.D., Fred Hutchinson Cancer Research Center
Date: Monday, December 5, 2016, 3:45 p.m. Pacific Time
Location: San Diego Convention Center, Room 24
Poster Presentations
Biomarkers of Cytokine Release Syndrome and Neurotoxicity after CD19 CAR-T Cells and Mitigation of Toxicity by Cell Dose (Abstract #1852)
Presenter: Cameron J. Turtle, M.B.B.S., Ph.D., Fred Hutchinson Cancer Research Center
Date: Saturday, December 3, 2016, 5:30 p.m. – 7:30 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH
Preclinical Analyses Support Clinical Investigation of Combined Anti-CD19 CAR-T Cell, JCAR017 with Ibrutinib for the Treatment of Chronic Lymphocytic Leukemia (Abstract #3231)
Presenter: Jim Qin, Senior Research Associate, Juno Therapeutics
Date: Sunday, December 4, 2016, 6:00 p.m. – 8:00 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH
Transcend NHL 001: Immunotherapy with the CD19-Directed CAR T Cell Product JCAR017 Results in High Complete Response Rates in Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma (Abstract #4192)
Presenter: Jeremy S. Abramson, Massachusetts General Hospital Cancer Center
Date: Monday, December 5, 2016, 6:00 p.m. – 8:00 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH
Identification of Small Molecule Modulators to Enhance the Therapeutic Properties of Chimeric Antigen Receptor T Cells (Abstract #4712)
Presenter: Jonathan Rosen, Ph.D., Senior Director, Research & Technology Core, Fate Therapeutics
Date: Monday, December 5, 2016, 6:00 p.m. – 8:00 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH

On November 3, 2016 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported financial results for the quarter ended September 30, 2016 and provided a business update (Press release, Insmed, NOV 3, 2016, View Source [SID1234516280]).

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Business Update

Achieved enrollment objective in global phase 3 study of ARIKAYCE. Today the company announced that it has achieved its patient enrollment objective in the phase 3 study of ARIKAYCE (liposomal amikacin for inhalation). The study, which is known as CONVERT or INS-212, is evaluating ARIKAYCE in treatment refractory nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC). The primary efficacy endpoint is the proportion of subjects who achieve culture conversion at Month 6 in the ARIKAYCE plus multi-drug regimen arm compared to the multi-drug regimen without ARIKAYCE arm.
Published phase 2 study of ARIKAYCE. In October 2016, the American Journal of Respiratory and Critical Care Medicine published the company’s phase 2 study of ARIKAYCE in NTM lung disease. The manuscript, which is entitled "Randomized Trial of Liposomal Amikacin for Inhalation in Nontuberculous Mycobacterial Lung Disease" by Olivier et al. is accessible online at View Source
Presented new analyses of phase 2 study of ARIKAYCE at CHEST. A poster describing the stability and consistency of effect with ARIKAYCE treatment in the phase 2 study was recently presented at the CHEST annual meeting.
Presented new NTM disease burden data at ISPOR European Congress. Three posters describing the burden of NTM lung disease were recently presented at the International Society for Pharmacoeconomic and Outcomes Research (ISPOR) Annual European Congress.
Secured global exclusive rights to novel oral inhibitor of dipeptidyl peptidase I.
In October, the company acquired the global exclusive rights to INS1007 (formerly known as AZD7986) from AstraZeneca. INS1007 is a small molecule, reversible inhibitor of dipeptidyl peptidase I (DPP1), an enzyme responsible for activating neutrophil serine proteases (NSPs) in neutrophils when they are formed in the bone marrow. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. The company expects to begin a phase 2 dose-ranging study of INS1007 in non-cystic fibrosis (non-CF) bronchiectasis in 2017. Non-CF bronchiectasis is a rare, progressive, neutrophil-driven pulmonary disorder with no approved therapies.
Presented phase 1 study of INS1009 at ERS 2016. In September, results from a phase 1 study of INS1009 were presented at the European Respiratory Society International Congress. The study was a randomized, double-blind, placebo-controlled single ascending dose study of INS1009 to determine its safety, tolerability, and pharmacokinetics in healthy volunteers. The pharmacokinetic characteristics supported once- or twice-daily whereas existing inhaled therapies are dosed four to nine times per day. The adverse event profile was consistent with other inhaled prostanoids. INS1009 is the company’s inhaled treprostinil prodrug, which may offer therapeutic potential in rare pulmonary disorders.
Appointed chief commercial officer. The company enhanced its senior leadership team with the appointment of Roger Adsett as chief commercial officer. Prior to joining Insmed, Mr. Adsett was senior vice president, head of the gastrointestinal and internal medicine business unit at Shire plc. In that capacity he oversaw Shire’s global commercial P&L across six specialty and two rare disease brands. Before joining Shire, Mr. Adsett worked at AstraZeneca for 11 years. Mr. Adsett will be charged with overseeing the development and execution of Insmed’s global commercial strategy.
"Recent months have been marked by strong progress across all aspects of our business," said Will Lewis, president and chief executive officer of Insmed. "We are pleased to report that the enrollment phase of the CONVERT study is now complete, which positions us for top-line data next year. There is a significant need for new treatments for patients with refractory NTM lung disease and we look forward to advancing the development of ARIKAYCE. With respect to our earlier-stage pipeline, we are encouraged by the high-level of inbound physician interest in our clinical program for INS1007 in non-CF bronchiectasis. Physicians are eagerly awaiting new treatment options for this debilitating disorder and we believe INS1007 has the potential to achieve disease modification by impeding tissue destruction, inflammation, and mucus hypersecretion through the inhibition of DPP1."

Third Quarter Financial Results

For the third quarter of 2016, Insmed posted a net loss of $37.8 million, or $0.61 per share, compared with a net loss of $31.0 million, or $0.50 per share, for the third quarter of 2015.

Research and development expenses were $23.4 million for the third quarter of 2016, compared with $19.2 million for the third quarter of 2015. The increase was primarily due to the advancement of the company’s global phase 3 CONVERT study of ARIKAYCE in NTM lung disease, as well as an increase in headcount and related expenses. These increases were partially offset by a decrease in manufacturing expenses primarily due to the completion of the build-out of additional production capacity at a contract manufacturer in 2015.

General and administrative expenses for the third quarter of 2016 were $13.7 million, compared with $11.0 million for the third quarter of 2015. The increase was primarily related to pre-commercial activities, namely the buildout of the company’s infrastructure and NTM disease awareness activities.

Balance Sheet Highlights and Cash Guidance

As of September 30, 2016, Insmed had cash and cash equivalents of $201 million. Excluding depreciation and stock-based compensation expense, the company’s cash operating expenses for the nine months ended September 30, 2016 were $91 million. Insmed ended the third quarter of 2016 with $35 million in debt and $183 million of working capital.

On September 30, 2016, Insmed closed a $55 million debt agreement with Hercules Capital, Inc. The transaction refinanced the company’s existing debt of $25 million and added a total of $30 million of new debt, $20 million of which was funded in early October in connection with the upfront payment for the global exclusive rights to INS1007.

The company is investing in the following activities in 2016: (i) clinical development of ARIKAYCE, (ii) regulatory and pre-commercial initiatives for ARIKAYCE, and (iii) preclinical and clinical activities for its earlier-stage pipeline. Insmed continues to expect its cash-based operating expenses for the second half of 2016 to be in the range of $62 to $72 million.

On November 3, 2016 Incyte Corporation (Nasdaq: INCY) reported that more than 20 abstracts including data from its ongoing clinical development program for Jakafi (ruxolitinib) and its small molecule development programs targeting JAK1, LSD1 and PI3Kδ will be presented at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2016 in San Diego, California from December 3-6, 2016 (Press release, Incyte, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219274 [SID1234516279]).

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"We are pleased to have an array of data highlighting the potential of our portfolio accepted for presentation at this year’s ASH (Free ASH Whitepaper) meeting, including the pooled overall survival analysis of five-year data from our COMFORT-I and COMFORT-II trials, which reinforces the long-term clinical profile of Jakafi; data from INCB39110, our selective JAK1 inhibitor, in acute graft-versus-host disease; and emerging data from our LSD1 and PI3Kδ inhibitor programs in other hematologic diseases," said Steven Stein, M.D., Incyte’s Chief Medical Officer.

Key abstract presentations and publications, inclusive of studies sponsored by Incyte, Novartis and independent investigator studies, include:

Myelofibrosis
Effects of Long-Term Ruxolitinib (RUX) on Bone Marrow (BM) Morphology in Patients (pts) with Myelofibrosis (MF) Enrolled in the COMFORT-I Study (Abstract #1949)

• Saturday, December 3, 2016, 5:30-7:30 PM PST, Hall GH, Poster Session: 634

Ruxolitinib (RUX) plus Pomalidomide in Myelofibrosis (MF): Updated Results from the MPNSG-0212 Trial (NCT01644110) (Abstract #1939)
• Saturday, December 3, 2016, 5:30-7:30 PM PST, Hall GH, Poster Session: 634

Clinical Outcomes with Ruxolitinib (RUX) in Patients (pts) with Myelofibrosis (MF) Stratified by Transfusion Status: A Pooled Analysis of the COMFORT-I and -II Trials (Abstract #3118)
• Sunday, December 4, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

A Pooled Overall Survival Analysis of 5-Year Data from the COMFORT-I and COMFORT-II Trials of Ruxolitinib (RUX) for the Treatment of Myelofibrosis (MF) (Abstract #3110)
• Sunday, December 4, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

Safety and Efficacy of Ruxolitinib (RUX) for the Final Enrollment of JUMP: An Open-Label, Multicenter, Single-Arm, Expanded-Access Study in Patients (pts) with Myelofibrosis (MF) (N = 2233) (Abstract #3107)
• Sunday, December 4, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

Ruxolitinib (RUX) in Combination with 5-Azacytidine (AZA) as Therapy for Patients (pts) with Myelofibrosis (MF) (Abstract #1127)
• Monday, December 5, 2016, 4:30-6:00 PM PST, Ballroom Salons 15-17, Oral Presentation: 634

Exploring the potential of JAK1/2 inhibitor Ruxolitinib (RUX) with reduced intensity hematopoietic cell transplantation (HCT) for Myelofibrosis (MF): Stage I results of a prospective trial conducted through the Myeloproliferative Disorders – Research Consortium (MPD-RC) (Abstract #1126)
• Monday, December 5, 2016, 4:30-6:00 PM PST, Pacific Ballroom Salons 15-17, Oral Presentation: 634

Polycythemia Vera
Examining the Clinical Features and Underlying Cardiovascular Risk Among Patients with Polycythemia Vera in the REVEAL Study (Abstract #1934)
• Saturday, December 3, 2016, 5:30-7:30 PM PST, Hall GH, Poster Session: 634

Essential Thrombocythemia
Correlation between Treatment Outcomes, Baseline Characteristics and Molecular Responses in the MAJIC Study which compared Ruxolitinib (RUX) to Best Available Therapy in Essential Thrombocythemia (ET) (Abstract #1929)
• Saturday, December 3, 2016, 5:30-7:30 PM PST, Hall GH, Poster Session: 634

Myeloproliferative Neoplasms
Impact of Myeloproliferative Neoplasms on Patients’ Employment Status and Work Productivity in the United States: Results from the Living with MPN Patient Survey (Abstract #4265)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

The impact of Myeloproliferative Neoplasms (MPNs) on patients’ quality of life and productivity: Results from the international MPN LANDMARK survey (Abstract #4267)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

Other Hematologic Malignancies
A Phase I Study of Ruxolitinib (RUX) Plus Nilotinib in Chronic Phase CML Patients with Molecular Evidence of Disease (Abstract #1892)
• Saturday, December 3, 2016, 5:30-7:30 PM PST, Hall GH, Poster Session: 632

A Phase 1 Trial of Janus Kinase (JAK) Inhibition with INCB039110 in Acute Graft-Versus-Host Disease (aGVHD) (Abstract #390)
• Sunday, December 4, 2016, 12:00-1:30 PM PST, Grand Hall C, Oral Presentation: 722

LSD1 Inhibition Induces Fetal Hemoglobin Expression and Provides a Novel Therapeutic Approach to Sickle Cell Disease (Abstract #2472)
• Sunday, December 4, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 113

Effects of INCB052793, a Selective JAK1 Inhibitor, in Combination with Anti Myeloma Agents on Human Multiple Myeloma (MM) In Vitro and In Vivo (Abstract #3297)
• Sunday, December 4, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 652

Safety and efficacy of combined Ruxolitinib (RUX) and Decitabine in patients with blast-phase MPN and post-MPN AML: Results of a Phase I study (Myeloproliferative Diseases Research Consortium 109 trial) (Abstract #1124)
• Monday, December 5, 2016, 4:30-6:00 PM PST, Pacific Ballroom Salons 15-17, Oral Presentation: 634

An ongoing open-label phase 1/2 study of INCB050465, a selective PI3Kδ inhibitor, in patients with previously treated B-cell malignancies (Abstract #4195)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 626

Ruxolitinib (RUX) in Combination with Azacytidine (AZA) in Patients (pts) with Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPNs) (Abstract #4246)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

Phase I Study of Ruxolitinib (RUX) for Patients (pts) with Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) Who Failed at Least One Line of Therapy (Abstract #4318)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 637

Phase I/II study of Ruxolitinib (RUX) with decitabine (DAC) in patients with post-myelofibrosis neoplasm acute myeloid leukemia (post-MPN AML): Phase I results (Abstract #4262)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 634

A LYSA phase II study of oral JAK1/2 inhibitor Ruxolitinib in advanced relapsed/refractory (R/R) Hodgkin lymphoma (HL) (Abstract #4160)
• Monday, December 5, 2016, 6:00-8:00 PM PST, Hall GH, Poster Session: 624

Full sessions details and data presentation listing for ASH (Free ASH Whitepaper) 2016 can be found at: View Source
About Jakafi (ruxolitinib)
Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration, for treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF, and post–essential thrombocythemia MF.
Jakafi is also indicated for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.
Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.
Important Safety Information
Jakafi can cause serious side effects, including:
Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.
Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.
Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.
Increases in Cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.
The most common side effects of Jakafi include: low platelet count, low red blood cell counts, bruising, dizziness, headache.
These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.
Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider. Do not drink grapefruit juice while on Jakafi.
Women should not take Jakafi while pregnant or planning to become pregnant, or if breast-feeding.
Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi, is available at www.jakafi.com.

On November 3, 2016 Geron Corporation (Nasdaq:GERN) reported financial results for the three and nine months ended September 30, 2016 and recent events (Press release, Geron, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219487 [SID1234516274]).

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For the third quarter of 2016, the company reported a net loss of $3.6 million, or $(0.02) per share, compared to net income for the third quarter of 2015 of $27.2 million, or $0.17 per share. Net loss for the first nine months of 2016 was $21.1 million, or $(0.13) per share, compared to net income for the first nine months of 2015 of $8.5 million, or $0.05 per share. The company ended the third quarter of 2016 with $129.8 million in cash and investments and has not incurred any impairment charges on its marketable securities portfolio.

Revenues for the three and nine months ended September 30, 2016 were $5.1 million and $6.1 million, respectively, compared to $35.4 million and $36.2 million for the comparable 2015 periods. Revenues for the three and nine month periods ending September 30, 2016 included license fee revenue of $5.0 million in connection with an upfront payment due under a license agreement signed in September 2016 with Janssen Pharmaceuticals, Inc. for certain rights to specialized oligonucleotide backbone chemistry and novel amidates. Revenues for the three and nine month periods ending September 30, 2015 included the full recognition of the $35.0 million upfront payment from Janssen Biotech, Inc. (Janssen) as collaboration revenue upon the company’s transfer of the imetelstat license rights and completion of technology transfer-related activities outlined under the imetelstat collaboration agreement with Janssen. The upfront cash payment was received in December 2014 and recorded as deferred revenue at that time.

Total operating expenses for the three and nine months ended September 30, 2016 were $9.0 million and $27.9 million, respectively, compared to $8.3 million and $28.1 million for the comparable 2015 periods. Research and development expenses for the three and nine months ended September 30, 2016 were $4.3 million and $13.9 million, respectively, compared to $4.1 million and $13.8 million for the comparable 2015 periods. General and administrative expenses for the three and nine months ended September 30, 2016 were $4.7 million and $14.0 million, respectively, compared to $4.3 million and $12.9 million for the comparable 2015 periods. Year-to-date operating expenses for 2015 also included restructuring charges of $1.3 million in connection with the company’s organizational resizing announced in March 2015.

The increase in research and development expenses for the three and nine month periods ending September 30, 2016, compared to the same periods in 2015, primarily reflected the net result of higher costs for the company’s proportionate share of clinical development expenses under the imetelstat collaboration with Janssen, partially offset by reduced personnel-related costs resulting from the March 2015 organizational resizing and lower costs for the manufacturing of imetelstat drug product. The company expects research and development expenses to be higher in 2016 compared to 2015 as the clinical development of imetelstat continues in collaboration with Janssen. The increase in general and administrative expenses for the three and nine month periods ending September 30, 2016, compared to the same periods in 2015, primarily reflected higher non-cash stock-based compensation expense and an increased allocation of facilities and other overhead costs to general and administrative activities.

Interest and other income for the three and nine months ended September 30, 2016 was $322,000 and $871,000, respectively, compared to $187,000 and $481,000 for the comparable 2015 periods. The increase in interest and other income for the three and nine month periods ending September 30, 2016, compared to the same periods in 2015, primarily reflected higher yields on the company’s marketable securities portfolio.

Recent Company Events

In the third quarter of 2016, Janssen conducted planned internal reviews of initial data from IMbarkTM and IMergeTM, the ongoing clinical trials of imetelstat.

IMbarkTM was designed to evaluate two dose levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered every three weeks) in approximately 200 patients (approximately 100 patients per dosing arm) with Intermediate-2 or High risk myelofibrosis (MF) who have relapsed after or are refractory to prior treatment with a JAK inhibitor. The co-primary efficacy endpoints for the trial are spleen response rate and symptom response rate at 24 weeks.

Janssen’s review included data from 20 patients from each dosing arm who had been followed on the trial for at least 12 weeks. In this review, no new safety signals were identified and the safety profile was consistent with previous imetelstat clinical trials in hematologic myeloid malignancies. Activity in the 4.7 mg/kg dosing arm did not warrant further investigation of that dose, and this arm has been closed to new patient enrollment. In the 9.4 mg/kg dosing arm, even though at the week 12 data assessment an insufficient number of patients met the protocol defined interim criteria, this arm warranted further investigation because encouraging trends in the efficacy data were observed. New enrollment in the 9.4 mg/kg arm has been suspended while the trial continues in order to obtain additional and more mature data that includes a longer follow-up of these patients at 24 weeks.

Enrolled patients in both arms are permitted to continue to receive imetelstat. Janssen has submitted a protocol amendment to health authorities that includes allowing eligible patients in the 4.7 mg/kg dosing arm to increase their dose to 9.4 mg/kg per investigator discretion.

IMergeTM is a two-part clinical study in patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS). Part 1 is a Phase 2, open-label, single-arm design in approximately 30 patients, which has been fully enrolled, and Part 2 is a Phase 3, randomized, double-blind, placebo-controlled design in approximately 170 patients. Janssen’s review of data in a subset of patients in Part 1 indicated that emerging safety and efficacy in IMergeTM is consistent with data reported from the pilot study conducted at Mayo Clinic in MDS patients. The primary efficacy endpoint for the trial is 8-week transfusion independence. IMergeTM continues unmodified at this time.

Second internal data reviews of additional and more mature data from both trials are planned by the end of the second quarter of 2017.

Three abstracts describing non-clinical data on imetelstat were accepted for presentation at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held in San Diego, California from December 3-6, 2016. The abstracts were published on November 3, 2016 on the ASH (Free ASH Whitepaper) website at www.hematology.org.

On November 3, 2016 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported a corporate update and reported financial results for the third quarter ended September 30, 2016 (Press release, Five Prime Therapeutics, NOV 3, 2016, View Source [SID1234516271]).

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"We have continued to make progress on all of our clinical and preclinical programs during the quarter," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "We achieved an important milestone by advancing into the Phase 1b portion of our clinical trial evaluating the immunotherapy combination of cabiralizumab (FPA008) with nivolumab in multiple tumor types. We believe targeting both the CSF1R and PD-1 pathways has the potential to produce a synergistic therapeutic effect. Our Phase 1 trial evaluating FPA144 in patients with gastric cancer is also progressing well. We have added cohorts to enroll gastric cancer patients with tumors at varying levels of FGFR2b expression as well as a cohort to evaluate bladder tumors that overexpress FGFR2b. Additionally, we continue to advance our pre-clinical programs and have begun IND-enabling activities for three of our immuno-oncology therapeutic candidates."

Business Highlights and Recent Developments

Clinical Pipeline:

Cabiralizumab (FPA008): an investigational antibody that inhibits CSF1R and has been shown to block the activation and survival of monocytes and macrophages. In the setting of advanced cancer, tumor-associated macrophages can inhibit the immune system’s ability to eradicate the disease. In pigmented villonodular synovitis (PVNS), a CSF-1-driven tumor, the bulk of the tumor mass in joints is formed by the macrophages themselves. Five Prime and Bristol-Myers Squibb (BMS) have an exclusive worldwide collaboration agreement for the development and commercialization of cabiralizumab for these and potentially additional indications.

Initiated Phase 1b portion of cabiralizumab/OPDIVO trial.
In October 2016, Five Prime initiated the Phase 1b portion of the clinical trial evaluating the immunotherapy combination of cabiralizumab with the PD-1 immune checkpoint inhibitor OPDIVO (nivolumab) in multiple tumor types. Five Prime and BMS are evaluating the safety, tolerability and preliminary efficacy of the combination in advanced solid tumors, including non-small cell lung cancer, squamous cell carcinoma of the head and neck, pancreatic cancer, glioblastoma, renal cell carcinoma and ovarian cancer. The Phase 1a/1b trial is expected to enroll approximately 280 patients.

Advanced the Phase 2 trial of cabiralizumab in patients with PVNS.
Five Prime continued enrollment and dosing in the Phase 2 trial of cabiralizumab in PVNS. During Phase 2, Five Prime is evaluating clinical measures, including response rate, pain and range of motion, in approximately 30 PVNS patients.

FPA144: an isoform-selective antibody in development as a targeted immuno-therapy for tumors that overexpress FGFR2b, a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. FPA144 has been engineered for enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) to increase direct tumor cell killing by recruiting natural killer (NK) cells. Five Prime retains global development and commercialization rights to FPA144.

Opened new gastric cancer cohorts and added a bladder cancer cohort in Phase 1 monotherapy trial of FPA144. Enrollment continues in the expansion portion of the trial evaluating the safety, PK and efficacy of biweekly 15 mg/kg infusions of FPA144 in patients with gastric cancer whose tumors highly overexpress FGFR2b. During the quarter, Five Prime added cohorts to evaluate FPA144 in patients with bladder cancer whose tumors overexpress FGFR2b and in patients with gastric cancer whose tumors express moderate or low levels of FGFR2b. Five Prime reported encouraging initial single-agent efficacy and safety data at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

Announced FDA Orphan Drug Designation for FPA144 in July for the treatment of gastric cancer and cancer of the gastroesophageal junction in patients whose tumors overexpress FGFR2b.

FP-1039: a protein drug designed to intervene in FGF signaling. As a ligand trap, FP-1039 binds to and neutralizes FGF ligands (such as FGF2), preventing these signaling proteins from reaching FGFR1 on the surface of tumor cells.

Although Five Prime regained full rights to FP-1039 from GlaxoSmithKline (GSK) in September, GSK will complete the ongoing Phase 1b trial combining FP-1039 with first-line pemetrexed and cisplatin in untreated, unresectable mesothelioma. GSK concluded trial recruitment with 25 patients enrolled at the 15 mg/kg dose, and continues to dose and follow patients. Five Prime plans to make decisions on potential future development of FP-1039 in mesothelioma once objective response rate, disease control rate and progression-free survival data are sufficiently mature.
Preclinical Research and Development:

IND-enabling studies initiated for preclinical development candidates. Five Prime advanced multiple preclinical immuno-oncology programs and initiated IND-enabling activities for three therapeutic candidates. In vivo data from preclinical studies of Five Prime’s tetravalent GITR agonist antibody (FPA154) have been accepted for presentation in a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting on November 11, 2016. Five Prime intends to share further details on all three programs during its R&D Day in New York City on December 8. The company’s goal is to file at least one IND application for a new molecule each year for the foreseeable future, beginning in 2017.

Completed multiple immuno-oncology research screens to identify new targets and drug candidates. Five Prime’s research team completed functional screens on CD8 T cells and regulatory T cells, as well as a comprehensive screen of all extracellular binding interactions in the "immunome," a defined subset of extracellular proteins selectively expressed on immune cells. The screens were conducted in order to identify new immuno-oncology targets, which the company is prioritizing for further development as new drug candidates, either as monotherapies or as part of combination regimens.
Summary of Financial Results and Guidance:

Cash Position. Cash, cash equivalents and marketable securities totaled $440.7 million on September 30, 2016, compared to $517.5 million on December 31, 2015. The decrease in cash was primarily attributable to cash used in operations to advance the FPA144 clinical trial, preclinical programs and tax payments.
Revenue. Collaboration revenue for the third quarter of 2016 increased by $0.8 million to $6.7 million from $5.9 million in the third quarter of 2015. This increase was primarily due to revenue recognized under the 2015 cabiralizumab collaboration agreement with BMS, under which Five Prime is reimbursed for the expenses from the cabiralizumab immuno-oncology trial.
R&D Expenses. Research and development expenses for the third quarter of 2016 decreased by $0.8 million to $23.9 million from $24.7 million in the third quarter of 2015. In the third quarter of 2015, the company recorded an $8.0 million expense related to in-licensing. Adjusted for this expense, research and development expenses increased $7.2 million over the prior year’s quarter, primarily related to advancing the FPA144 clinical trial, preclinical development and immuno-oncology research programs.
G&A Expenses. General and administrative expenses for the third quarter of 2016 increased by $3.9 million to $9.1 million from $5.2 million in the third quarter of 2015. This increase was primarily due to increases in payroll and stock-based compensation expenses.
Net Loss. Net loss for the third quarter of 2016 was $19.4 million, or $0.72 per basic and diluted share, compared to a net loss of $24.0 million, or $0.93 per basic and diluted share, for the third quarter of 2015.
Shares Outstanding. Total shares outstanding were 28.4 million as of September 30, 2016.
Cash Guidance. Five Prime continues to expect full-year 2016 net cash used in operating activities to be less than $120 million, comprising less than $90 million used in operations and less than $30 million used for tax payments. The company estimates ending 2016 with more than $400 million in cash, cash equivalents and marketable securities.