On November 1, 2017 Fate Therapeutics, Inc. (NASDAQ:FATE), a biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that two oral and four poster presentations detailing clinical and preclinical results will be featured at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition(Press release, Fate Therapeutics, NOV 1, 2017, View Source [SID1234521387]). The meeting will be held December 9-12, 2017 in Atlanta, Georgia.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

An oral presentation will describe the generation of CD8αβ+ T cells from an induced pluripotent stem cell (iPSC) line engineered to express a chimeric antigen receptor (CAR). This breakthrough was led by Dr. Michel Sadelain, MD, PhD, Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center (MSK), under the Company’s multi-year sponsored research collaboration with MSK. As part of the collaboration, Fate Therapeutics has created clonal iPSC master cell lines engineered to express CARs and other functional elements and are also modified to attenuate alloreactivity and enhance persistence for off-the-shelf CAR T-cell immunotherapy. The Company’s first iPSC-derived CAR T-cell product candidate FT819, which is derived from a clonal iPSC master cell line engineered to express a CAR targeting CD19 and edited to remove T-cell receptor (TCR) expression, is undergoing preclinical development.

A second oral presentation will describe the production under current good manufacturing practice (cGMP) conditions of FT500, the Company’s first-of-kind natural killer (NK) cell product candidate derived from a clonal iPSC master cell line. The transformative manufacturing paradigm, which will be described by Jeffrey S. Miller, MD, Deputy Director of the Masonic Cancer Center, University of Minnesota, enables the efficient production of a large clonal population of NK cells in a single production run and is capable of yielding thousands of doses of homogeneous drug product for off-the-shelf delivery to patients. Fate Therapeutics plans to file a landmark Investigational New Drug (IND) application with the U.S. Food & Drug Administration (FDA) in the first quarter of 2018 to initiate first-in-human clinical investigation of FT500 in combination with FDA-approved checkpoint inhibitors for the treatment of advanced solid tumors.

In addition, Fate Therapeutics will present clinical data from the PROTECT study of ProTmune, a next-generation hematopoietic cell graft for patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). Key clinical outcomes, including incidence rates of acute graft-versus-host disease, cancer relapse and survival at 100 days following HCT, for the seven subjects administered ProTmune in the Phase 1 stage of PROTECT will be released. Three other poster presentations will highlight additional iPSC-derived immuno-oncology product candidates that the Company is developing.

2017 ASH (Free ASH Whitepaper) Oral Presentations

FT819 iPSC-derived CAR19 T-Cell Cancer Immunotherapy
Title: Generation of Clonal Antigen Specific CD8αβ+ Cytotoxic T Lymphocytes from Renewable Pluripotent Stem Cells for Off-the-Shelf T-Cell Therapeutics
Last Author: Michel Sadelain, MD, PhD, Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center
Publication Number: 163
Session: 703. Adoptive Immunotherapy: Immune Therapeutics for Hematologic Cancers
Date and Time: Saturday, December 9, 2017, 12:00 PM
Location: Building B, Level 2, B206

FT500 iPSC-derived NK Cell Cancer Immunotherapy
Title: Clinical Translation of Pluripotent Cell-Derived Off-the-Shelf Natural Killer Cell Cancer Immunotherapy
Last Author: Jeffrey S. Miller, MD, Deputy Director of the Masonic Cancer Center, University of Minnesota
Publication Number: 656
Session: 711. Cell Collection and Processing
Date and Time: Monday, December 11, 2017, 10:45 AM
Location: Building B, Level 2, B216-B217
2017 ASH (Free ASH Whitepaper) Poster Presentations

iPSC-derived CAR NK Cell Cancer Immunotherapy
Title: Engineering Human Induced Pluripotent Stem Cells with Novel Chimeric Antigen Receptors to Generate Natural Killer Cell Cancer Immunotherapies with Targeted Anti-Tumor Activity
Last Author: Dan S. Kaufman, MD, PhD, Director of Cell Therapy, UCSD
Publication Number: 1905
Session: 703. Adoptive Immunotherapy: Poster I
Date and Time: Saturday, December 9, 2017, 5:30 PM – 7:30 PM
Location: Building A, Level 1, Hall A2

iPSC-derived Cancer Immunotherapy Product Platform
Title: Multi-Functional Genetic Engineering of Pluripotent Cell Lines for Universal Off-the-Shelf Natural Killer Cell Cancer Immunotherapy
Last Author: Bahram Valamehr, PhD, VP Cancer Immunotherapy, Fate Therapeutics
Publication Number: 3187
Session: 703. Adoptive Immunotherapy: Poster II
Date and Time: Sunday, December 10, 2017, 6:00 PM – 8:00 PM
Location: Building A, Level 1, Hall A2

FT516 iPSC-derived hnCD16 NK Cell Cancer Immunotherapy
Title: Genetically Engineered Pluripotent Cell-Derived Natural Killer Cell Therapy Provides Enhanced Antibody Dependent Cellular Cytotoxicity Against Hematologic Malignancies and Solid Tumors in Combination with Monoclonal Antibody Therapy
Last Author: Dan S. Kaufman, MD, PhD, Director of Cell Therapy, UCSD
Session: 703. Adoptive Immunotherapy: Poster III
Publication Number: 4452
Date and Time: Monday, December 11, 2017, 6:00 PM – 8:00 PM
Location: Building A, Level 1, Hall A2

ProTmune
Title: ProTmune, the Next Generation Graft for Allogeneic Hematopoietic Cell Transplantation: Phase 1 Safety and Efficacy Data
First Author: Richard Maziarz, MD, Principal Investigator, Oregon Health Sciences University
Session: 722. Clinical Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution
Publication Number: 4498
Date and Time: Monday, December 11, 2017, 6:00 PM – 8:00 PM
Location: Building A, Level 1, Hall A2
About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables genetic engineering, high-throughput single-cell isolation and clonal selection of human iPSCs and supports long-term maintenance of human iPSCs as master pluripotent cell lines. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. Similar to master cell lines used for the manufacture of monoclonal antibodies, clonal iPSC master cell lines can serve as a renewable cell source for the consistent and repeated manufacture of homogeneous cell products with the potential to treat many different diseases and many thousands of patients in an off-the-shelf manner. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 90 issued patents and 100 pending patent applications.

On November 1, 2017 Incyte Corporation (Nasdaq:INCY) reported that more than 30 abstracts including data from its clinical development programs for Jakafi (ruxolitinib), JAK1, PI3Kδ, PIM and BRD will be presented at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 in Atlanta, Georgia from December 9-12, 2017 (Press release, Incyte, NOV 1, 2017, View Source [SID1234521410]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

“Data accepted for presentation include long-term, 4-year follow-up from our RESPONSE trial, updated data from our PI3Kδ inhibitor program in relapsed or refractory B-cell malignancies as well as first-in-man data from our BRD and PIM kinase inhibitor programs.”
“We look forward to presenting new data from across our clinical development portfolio,” said Steven Stein, M.D., Chief Medical Officer at Incyte. “Data accepted for presentation include long-term, 4-year follow-up from our RESPONSE trial, updated data from our PI3Kδ inhibitor program in relapsed or refractory B-cell malignancies as well as first-in-man data from our BRD and PIM kinase inhibitor programs.”

Select key abstract presentations include:

Jakafi (ruxolitinib)

Promising Results of a Phase 1/2 Clinical Trial of Ruxolitinib in Patients with Chronic Myelomonocytic Leukemia (Abstract #162)

Saturday, December 9, 2017, 12:00-1:30 PM, Building B, Level 3, B312-B314, Oral Session 637, Myelodysplastic Syndromes—Clinical Studies: Predicting Drug Response Using Novel Genomic Algorithms
Examining The Treatment Patterns And Blood Counts Among Patients With Polycythemia Vera Treated With Hydroxyurea In The United States: An Analysis From The REVEAL Study (Abstract #1633)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Role Of Symptom Burden In Disability Leave Among Patients With Myeloproliferative Neoplasms (MPNs): Findings From The Living With MPN Patient Survey (Abstract #1637)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Ruxolitinib Or Dasatinib In Combination With Chemotherapy For Patients With Relapsed/Refractory Philadelphia (Ph)-Like Acute Lymphoblastic Leukemia: A Phase I-II Trial (Abstract #1322)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
The Combination Of Ruxolitinib (RUX) With Decitabine (DAC) In Patients (Pts) With Post-Myeloproliferative Neoplasm Acute Myeloid Leukemia (Post-MPN AML): Interim Report Of A Phase I/II Trial (Abstract #1379)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
A Phase Ib Study To Assess The Safety And Tolerability Of Oral Ruxolitinib In Combination With Azacitidine In Patients With Advanced Phase Myeloproliferative Neoplasms (MPN), Including Myelodysplastic Syndromes (MDS) Or Acute Myeloid Leukaemia (AML) Arising From MPN (The Bloodwise / TAP PHAZAR Study On Behalf Of The UK MPN CSG) (Abstract #1649)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Results From The 208-Week (4-year) Follow-Up Of RESPONSE Trial, A Phase 3 Study Comparing Ruxolitinib (Rux) With Best Available Therapy (BAT) For The Treatment Of Polycythemia Vera (PV) (Abstract #322)

Sunday, December 10, 2017, 8:15 AM, Building C, Level 2, C208-C210,
Oral Session 634, Myeloproliferative Syndromes: Clinical: Phase III and Long-Term Outcome Studies in MPNs
Patient-Reported Symptom Burden And Peripheral Blood Counts Among Patients With Polycythemia Vera: And Analysis From The REVEAL Study (Abstract #2924)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
Safety And Efficacy Of Ruxolitinib (Rux) In An Open-Label, Multicenter, Expanded Treatment Protocol In Patients (Pts) With Polycythemia Vera (PV) Who Are Hydroxyurea (HU) Resistant Or Intolerant And For Whom No Alternative Treatments Are Available (Abstract #2918)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
Primary Analysis Of JUMP, A Phase 3b, Expanded-Access Study Evaluating The Safety And Efficacy Of Ruxolitinib In Patients With Myelofibrosis (N=2233) (Abstract #4204)

Monday, December 11, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Characteristics Of 809 Patients With Essential Thrombocythemia In Real-World Clinical Practice: A Chart Review Study In The United States (Abstract #1636)

Monday, December 11, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Real-World Patterns Of First-Line Hydroxyurea Treatment Among Patients With Essential Thrombocythemia In US Community Oncology Practices (Abstract #4203)

Monday, December 11, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Ponatinib

First Report of the Gimema LAL1811 Phase II Prospective Study of the Combination of Steroids with Ponatinib As Frontline Therapy of Elderly or Unfit Patients with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Abstract #99)

Saturday, December 9, 2017, 9:30-11:00 AM, Building C, Level 2, C211-C213,
Oral Session 612, Acute Lymphoblastic Leukemia: Clinical Studies: Advances in the Treatment of ALL
Efficacy And Safety Of Ponatinib In Chronic Phase-Chronic Myeloid Leukemia (CP-CML) According To The Extent Of Treatment With Prior Tyrosine Kinase Inhibitors (TKIs): Final (5-Year) Results Of The PACE Study (Abstract #1617)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2
Arterial Occlusive Events (AOEs) In The Phase 2 Ponatinib PACE Trial: 5-Year Update In Heavily Treated Patients (Pts) With Chronic-Phase Chronic Myeloid Leukemia (CP-CML) (Abstract #2896)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
Pipeline

Results From A Phase 1/2 Study Of INCB050465, A Highly Selective And Highly Potent PI3Kδ Inhibitor, In Patients With Relapsed Or Refractory B-Cell Malignancies (CITADEL-101) (Abstract #410)

Sunday, December 10, 2017, 12:15 PM, Building C, Level 1, Hall C1, Oral Session 623, Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Indolent Lymphomas, Novel Therapies and Diagnostics
Preliminary Results From An Ongoing Phase 1/2 Study Of INCB053914, A Pan-Proviral Integration Sites For Moloney Virus (PIM) Kinase Inhibitor, In Patients With Advanced Hematologic Malignancies (Abstract #2585)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
A Phase 1/2 Study Of The Oral Novel JAK1 Inhibitor INCB052793 As Monotherapy And In Combination With Standard Therapies In Patients With Advanced Hematologic Malignancies (Abstract #640)

Monday, December 11, 2017, 10:30 AM-12:00 PM, Building B, Level 5, Murphy BR1-2,
Oral Session 613, Acute Myeloid Leukemia: Clinical Studies: Novel Therapies for AML and APL
Preliminary Results From An Ongoing Phase 1/2 Study Of INCB057643, A Bromodomain And Extraterminal (BET) Protein Inhibitor, In Patients (pts) With Advanced Malignancies (Abstract #4048)

Monday, December 11, 2017, 6:00–8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Preclinical

The Pan-PIM Inhibitor INCB053914 Displays Potent Synergy At Low Doses In Combination With Ruxolitinib In Pre-Clinical Models Of MPNs (Abstract #1661)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Targeting Cell Non-Autonomous MAPK Activation As A Novel Therapeutic Strategy In Myeloproliferative Neoplasms (Abstract #381)

Sunday, December 10, 2017, 10:00 AM, Building C, Level 2, C208-C210, Oral Session 635, Myeloproliferative Syndromes: Basic Science: Identification of novel targets for the treatment of myeloproliferative neoplasms
Redundant JAK, SRC And PI3 Kinase Signaling Pathways Regulate Cell Survival In Human Ph-Like ALL Cell Lines And Primary Cells (Abstract #717)

Monday, December 11, 2017, 2:45-4:15 PM, Building B, Level 2, B216-B217
Full session details and data presentation listings for ASH (Free ASH Whitepaper) 2017 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 October 30, 2017 Acorda Therapeutics, Inc. (Nasdaq:ACOR) provided a financial and pipeline update for the third quarter ended September 30, 2017 (Press release, Acorda Therapeutics, OCT 31, 2017, View Source [SID1234521332]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

“We have had a constructive dialogue with the FDA since the issuance of its Refusal to File letter, and we plan to resubmit the INBRIJA NDA in the fourth quarter. We believe our resubmission reflects a strong package that incorporates feedback we received from FDA,” said Ron Cohen, M.D., Acorda’s President and CEO. “We are also on track to announce top-line data from our Phase 3 study of tozadenant in the first quarter of 2018.”

“INBRIJA and tozadenant are being developed as therapies for people with Parkinson’s, INBRIJA for on-demand use to treat symptoms of OFF periods and tozadenant as a daily oral treatment to increase overall ON time. If approved, they have the potential to position Acorda as a leader in the development of Parkinson’s therapy, creating substantial value for shareholders.”

Third Quarter 2017 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended September 30, 2017, the Company reported AMPYRA net revenue of $132.6 million compared to $128.8 million for the same quarter in 2016.

FAMPYRA (prolonged-release fampridine tablets) – For the quarter ended September 30, 2017, the Company reported FAMPYRA royalties from sales outside of the U.S. of $3.1 million compared to $2.6 million for the same quarter in 2016.

Research and development (R&D) expenses for the quarter ended September 30, 2017 were $33.3 million, including $2.0 million of share-based compensation and $.03 million of restructuring expenses compared to $54.8 million, including $2.9 million of share-based compensation, for the same quarter in 2016.

Sales, general and administrative (SG&A) expenses for the quarter ended September 30, 2017 were $40.7 million, including $4.6 million of share-based compensation and $0.01 million of restructuring expenses compared to $54.4 million, including $7.1 million of share-based compensation for the same quarter in 2016.

The Company recorded a non-cash intangible asset impairment charge of $39.4 million in the quarter ended September 30, 2017 for Selincro. Selincro is currently marketed in Europe by the licensor for the reduction of alcohol consumption in alcohol dependent adults. The Company re-assessed its valuation assumptions, including expected future growth related to the expansion into new markets, and determined that the intangible asset was impaired.

Provision for income taxes for the quarter ended September 30, 2017 was $18.9 million, including $3.7 million of cash taxes, compared to a provision for income taxes of $3.0 million, including $1.0 million of cash taxes, for the same quarter in 2016.

The Company reported a GAAP net loss attributable to Acorda of $(25.2) million for the quarter ended September 30, 2017, or $(0.55) per diluted share. GAAP net loss in the same quarter of 2016 was $(12.7) million, or $(0.28) per diluted share.

Non-GAAP net income for the quarter ended September 30, 2017 was $20.1 million, or $0.43 per diluted share. Non-GAAP net loss in the same quarter of 2016 was $(1.9) million, or $(0.04) per diluted share. This quarterly non-GAAP net income measure, more fully described below under “Non-GAAP Financial Measures,” excludes share-based compensation charges, non-cash interest charges on our debt, restructuring expenses, changes in the fair value of acquired contingent consideration, intangible asset impairment charges and acquisition-related expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At September 30, 2017, the Company had cash and cash equivalents of $192.5 million.

Guidance for 2017

The Company reiterates AMPYRA 2017 net revenue of $535-$545 million.
R&D expenses for the full year 2017 are expected to be $160-$170 million. This guidance is a non-GAAP projection that excludes share-based compensation and restructuring costs, as more fully described below under “Non-GAAP Financial Measures.”
The Company is reducing its SG&A expense guidance for the full year 2017 from $170-$180 million to $160-$170 million. This guidance is a non-GAAP projection that excludes share-based compensation and restructuring costs, as more fully described below under “Non-GAAP Financial Measures.”
The Company expects to be cash flow positive in 2017, with a projected year-end cash balance in excess of $200 million.
Third Quarter 2017 Highlights

INBRIJA (levodopa inhalation powder) in Parkinson’s disease
In August, the Company received a Refusal to File (RTF) letter regarding its NDA for INBRIJA. After constructive dialogue with the FDA, the Company expects to resubmit the NDA in Q4 2017.
As a result, the Company has revised the timing for its end-of-year submission of the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) to Q1 2018.
INBRIJA is an investigational treatment for symptoms of OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen.
Tozadenant in Parkinson’s disease
The Company expects to report topline Phase 3 in Q1 2018.
Tozadenant is an investigational treatment for the reduction of OFF time in people with Parkinson’s disease.
AMPYRA (dalfampridine)
The Company filed its opening brief for its appeal to the U.S. Court of Appeals for the Federal Circuit of the District Court’s decision in the AMPYRA patent litigation. The defendants have filed their opposition and cross-appeal opening brief. Reply briefs from both parties are expected to be filed in November 2017, followed by oral argument to be scheduled by the appellate court.
Both BIO and PhRMA filed amicus briefs in support of the Company’s appeal, raising important issues in conjunction with biopharmaceutical innovation.
The Company expects to maintain exclusivity of AMPYRA at least through July 2018.
Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate, please dial (844) 579-6824 (domestic) or (763) 488-9145 (international) and reference the access code 95686626. A replay of the call will be available from 11:30 a.m. ET on October 31, 2017 until 2:59 p.m. ET on November 30, 2017. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference the access code 95686626. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.On October 30, 2017

On October 31, 2017 AstraZeneca and MedImmune, its global biologics research and development arm, reported the expansion of their clinical collaboration with Incyte Corporation (Press release, AstraZeneca, OCT 31, 2017, View Source [SID1234521325]). As part of the agreement, the companies will evaluate the efficacy and safety of epacadostat, Incyte’s investigational selective IDO1 enzyme inhibitor, in combination with AstraZeneca’s Imfinzi (durvalumab), a human monoclonal antibody directed against PD-L1, compared to Imfinzi alone.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The exclusive collaboration for the study population allows for the two companies to conduct a Phase III trial in patients with locally-advanced (Stage III), unresectable non-small cell lung cancer (NSCLC) whose disease has not progressed following platinum-based chemotherapy concurrent with radiation therapy (CRT).

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "Imfinzi has shown exciting clinical potential in treating patients with locally-advanced lung cancer. We are pleased to build on recent data from the PACIFIC trial to further explore how Imfinzi, in combination with an IDO inhibitor could provide additional benefit to patients with locally-advanced lung cancer."

Steven Stein, MD, Chief Medical Officer, Incyte, said: "We are pleased to expand our ongoing clinical collaboration with AstraZeneca and to further explore the potential of epacadostat in patients with locally-advanced unresectable lung cancer. We look forward to beginning an additional pivotal trial for epacadostat, as we seek to position IDO1 enzyme inhibition as a key component of combination immunotherapy."

The Phase III trial, which will be co-funded by the two companies and will be conducted by AstraZeneca, is expected to begin enrolling patients in the first half of 2018. This agreement builds on an existing clinical collaboration for Imfinzi and epacadostat announced by both companies in May 2014.

NOTES TO EDITORS

About Locally-Advanced (Stage III) NSCLC

Stage III lung cancer is divided into two stages (IIIA and IIIB), which are defined by how much the cancer has spread locally and the possibility of surgery.

Stage III lung cancer represents approximately one-third of NSCLC incidence and was estimated to affect around 105,000 patients in seven leading markets[1] in 2016. More than half of these patients have tumours that are unresectable. The current standard of care is chemotherapy and radiation followed by active surveillance to monitor for progression. The prognosis remains poor and long-term survival rates are low.

About Epacadostat (INCB024360)

The immunosuppressive effects of indoleamine 2,3-dioxygenase 1 (IDO1) enzyme activity on the tumour microenvironment help cancer cells evade immunosurveillance. Epacadostat is an investigational, highly-potent and selective oral inhibitor of the IDO1 enzyme. In single-arm studies, the combination of epacadostat and immune checkpoint inhibitors has shown proof-of-concept in patients with unresectable or metastatic melanoma, non-small cell lung cancer, renal cell carcinoma, squamous cell carcinoma of the head and neck and bladder cancer. In these studies, epacadostat combined with the CTLA-4 inhibitor ipilimumab or the PD-1 inhibitors pembrolizumab or nivolumab improved response rates compared with studies of the immune checkpoint inhibitors alone.

On October 31, 2017 Incyte Corporation (Nasdaq: INCY) reported 2017 third-quarter financial results, highlighting 38 percent year-on-year growth in product-related revenue, driven by increased sales of Jakafi (ruxolitinib) in the U.S. and Iclusig (ponatinib) in Europe, and royalties from ex-U.S. sales of Jakavi (ruxolitinib) by Novartis and Olumiant (baricitinib) by Lilly (Press release, Incyte, OCT 31, 2017, View Source [SID1234521336]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We exit the third quarter of 2017 with excellent momentum across the whole business," stated Hervé Hoppenot, Incyte’s Chief Executive Officer. "Jakafi and Iclusig continue to outperform our expectations, and we are now evaluating ten different indications across our five late-stage development candidates. We are also on track to initiate the next wave of pivotal trials planned for the epacadostat development program. We are striving to build Incyte into a world-class biopharmaceutical company, and we are very pleased to report another quarter of significant progress towards that goal."

Know more, wherever you are:
Latest on Incyte’s Cancer Pipeline, book your free 1stOncology demo here.

Portfolio Update
Cancer – Targeted Therapies
The REACH1 pivotal trial studying ruxolitinib in patients with steroid-refractory acute graft-versus-host disease (GVHD) is on track to deliver results in the first half of 2018. If successful, Incyte anticipates submitting an sNDA seeking accelerated approval of ruxolitinib in this indication during 2018. Three additional pivotal trials are evaluating the role of JAK inhibition in GVHD (REACH2 and REACH3 with ruxolitinib, and GRAVITAS-301 with itacitinib).
The FIGHT and CITADEL programs evaluating INCB54828 (FGFR1/2/3) and INCB50465 (PI3Kδ), respectively, now include multiple different indications in potentially-pivotal trials.

Indication

Status Update
Ruxolitinib (JAK1/JAK2)
Steroid-refractory acute GVHD Pivotal Phase 2 (REACH1) and Phase 3 (REACH2)
Ruxolitinib (JAK1/JAK2)
Steroid-refractory chronic GVHD Phase 3 (REACH3)
Ruxolitinib (JAK1/JAK2)
Essential thrombocythemia Pivotal Phase 2 (RESET-272) open for enrollment
Itacitinib (JAK1)
Treatment-naïve acute GVHD Phase 3 (GRAVITAS-301)
Itacitinib (JAK1)
Non-small cell lung cancer Phase 1/2 in combination with osimertinib (EGFR)
INCB52793 (JAK1)
Advanced malignancies Phase 1/2 dose-escalation
INCB50465 (PI3Kδ)
Diffuse large B-cell lymphoma, follicular lymphoma, marginal zone lymphoma, mantle cell lymphoma Phase II (CITADEL-202 initiated; CITADEL-203, CITADEL-204, CITADEL-205 all open for enrollment)
INCB54828 (FGFR1/2/3)
Bladder cancer, cholangiocarcinoma; 8p11 MPNs
Phase 2 (FIGHT-201, FIGHT-202, FIGHT-203)
INCB57643 (BRD)
Advanced malignancies Phase 1/2 dose-escalation
INCB53914 (PIM)
Advanced malignancies Phase 1/2 dose-escalation
INCB59872 (LSD1)
Acute myeloid leukemia, small cell lung cancer Phase 1/2 dose-escalation
INCB62079 (FGFR4)
Hepatocellular carcinoma Phase 1/2 dose-escalation

Cancer – Immune Therapies
The pivotal Phase 3 ECHO-301 trial of epacadostat plus pembrolizumab in patients with unresectable or metastatic melanoma is now fully-recruited and data are expected in the first half of 2018.
In collaboration with Merck and Bristol-Myers Squibb, preparations for the next wave of eight pivotal Phase 3 trials of epacadostat plus PD-1 antagonists continue as planned. Initiation of these trials are expected before the end of 2017.

In October, Incyte and AstraZeneca announced an expanded clinical trial collaboration and the companies intend to initiate a Phase 3 trial of epacadostat in combination with AstraZeneca’s PD-L1 antagonist durvalumab in patients with Stage III non-small cell lung cancer.
In October, Incyte and MacroGenics announced an exclusive global collaboration and license agreement for MacroGenics’ MGA012, an investigational monoclonal antibody that inhibits PD-1. Under this agreement, Incyte will obtain exclusive worldwide rights for the development and commercialization of MGA012 in all indications.

Indication

Status Update
Epacadostat (IDO1)
Unresectable or metastatic melanoma Phase 3 (ECHO-301) in combination with pembrolizumab (PD-1)
Epacadostat (IDO1)
NSCLC, renal, bladder and head & neck cancer Phase 3 in combination with pembrolizumab (PD-1) expected to begin in 2017
Epacadostat (IDO1)
NSCLC, head & neck cancer Phase 3 in combination with nivolumab (PD-1) expected to begin in 2017
Epacadostat (IDO1)
NSCLC Phase 3 in combination with durvalumab (PD-L1) expected to begin in H1 2018
Epacadostat (IDO1)
Multiple tumor types Phase 2 (ECHO-202) expansion cohorts in combination with pembrolizumab (PD-1)
Epacadostat (IDO1)
Multiple tumor types Phase 2 (ECHO-204) expansion cohorts in combination with nivolumab (PD-1)
Epacadostat (IDO1)
Multiple tumor types Phase 2 (ECHO-203) expansion cohorts in combination with durvalumab (PD-L1)
INCB01158 (ARG)1
Solid tumors Phase 1/2
INCSHR1210 (PD-1)2
Solid tumors Phase 1/2; enrollment halted
INCAGN1876 (GITR)3
Solid tumors Phase 1/2
INCAGN1949 (OX40)3
Solid tumors Phase 1/2
PD-1 platform study
Solid tumors Phase 1/2, pembrolizumab (PD-1) in combination with itacitinib (JAK1) or INCB50465 (PI3Kδ)
JAK1 platform study
Solid tumors Phase 1/2, itacitinib (JAK1) in combination with epacadostat (IDO1) or INCB50465 (PI3Kδ)

Notes:
1) INCB01158 co-developed with Calithera
2) INCSHR1210 licensed from Hengrui
3) INCAGN1876 & INCAGN1949 from discovery alliance with Agenus

Non-oncology


Indication

Status Update
Topical ruxolitinib (JAK1/JAK2)
Atopic dermatitis, vitiligo Phase 2

Partnered
In August, Lilly and Incyte announced that Lilly plans to resubmit the New Drug Application (NDA) for baricitinib to the U.S. Food & Drug Administration (FDA) before the end of January 2018. The companies anticipate the FDA will classify the application as a Class II resubmission, which would start a new six-month review cycle.
In September, Lilly and Incyte announced that baricitinib met the primary endpoint in a Phase 2 study in patients with moderate-to-severe atopic dermatitis.
Novartis has stated that it anticipates submitting an NDA for capmatinib, a potent and selective c-MET inhibitor licensed from Incyte, in 2018.

Indication

Status Update
Baricitinib (JAK1/JAK2)1
Rheumatoid arthritis Approved in Europe and Japan; CRL issued by FDA
Baricitinib (JAK1/JAK2)1
Psoriatic arthritis Lilly expects the Phase 3 program to begin in 2018
Baricitinib (JAK1/JAK2)1
Atopic dermatitis Lilly expects the Phase 3 program to begin in late 2017
Baricitinib (JAK1/JAK2)1
Systemic lupus erythematosus Phase 2
Capmatinib (c-MET)2
Non-small cell lung cancer, liver cancer Phase 2 in EGFR wild-type ALK negative NSCLC patients with c-MET amplification and mutation

Notes:
1) Baricitinib licensed to Lilly
2) Capmatinib licensed to Novartis

2017 Third-Quarter Financial Results
Revenues For the quarter ended September 30, 2017, net product revenues of Jakafi were $304 million as compared to $224 million for the same period in 2016, representing 36 percent growth. For the nine months ended September 30, 2017, net product revenues of Jakafi were $831 million as compared to $615 million for the same period in 2016, representing 35 percent growth. For the quarter ended September 30, 2017, net product revenues of Iclusig were $18 million as compared to $13 million for the same period in 2016, representing 42 percent growth. For the nine months ended September 30, 2017, net product revenues of Iclusig were $47 million as compared to $17 million for the same period in 20161.

For the quarter and nine months ended September 30, 2017, product royalties from sales of Jakavi, which has been out-licensed to Novartis outside of the United States, were $41 million and $104 million, respectively, as compared to $30 million and $77 million for the same periods in 2016. For the quarter and nine months ended September 30, 2017, product royalties from sales of Olumiant outside of the United States received from Lilly were $3 million, and $4 million, respectively.

For the quarter and nine months ended September 30, 2017, contract revenues were $15 million and $105 million, respectively, as compared to $3 million and $70 million for the same periods in 2016. The contract revenues in 2017 relate to milestone payments earned.

For the quarter ended September 30, 2017, total revenues were $382 million as compared to $269 million for the same period in 2016. For the nine months ended September 30, 2017, total revenues were $1.1 billion as compared to $779 million for the same period in 2016.
1 In June 2016, Incyte obtained an exclusive license from ARIAD to develop and commercialize Iclusig in Europe and other select ex-U.S. countries.

Year Over Year Revenue Growth
(in thousands, unaudited)

Three Months Ended Nine Months Ended
September 30, % September 30, %
2017 2016 Change 2017 2016 Change
Revenues:
Jakafi net product revenues $ 303,929 $ 223,892
36%

$ 831,044 $ 615,285
35%

Iclusig net product revenues
18,100 12,731
42%

47,459 16,721 -
Product royalty revenues 44,487 29,626
50%

108,477 77,486
40%

Product-related revenues 366,516 266,249
38%

986,980 709,492
39%

Contract revenues 15,000 3,214 - 105,000 69,643 -
Other revenues
18 6 - 80 86 -
Total revenues $ 381,534 $ 269,469
42%

$ 1,092,060 $ 779,221
40%

Research and development expenses Research and development expenses for the quarter and nine months ended September 30, 2017 were $270 million and $879 million, respectively, as compared to $143 million and $420 million for the same periods in 2016. The increase in research and development expenses was primarily due to the expansion of the Company’s clinical portfolio as well as upfront and milestone expenses of $209 million related to our collaboration and license agreements with Agenus, Calithera and Merus. Included in research and development expenses for the quarter and nine months ended September 30, 2017 were non-cash expenses related to equity awards to our employees of $23 million and $68 million, respectively.

Selling, general and administrative expenses Selling, general and administrative expenses for the quarter and nine months ended September 30, 2017 were $91 million and $269 million, respectively, as compared to $76 million and $207 million for the same periods in 2016. Increased selling, general and administrative expenses were driven primarily by additional costs related to the commercialization of Jakafi and the geographic expansion in Europe. Included in selling, general and administrative expenses for the quarter and nine months ended September 30, 2017 were non-cash expenses related to equity awards to our employees of $12 million and $31 million, respectively.

Change in fair value of acquisition-related contingent consideration The change in fair value of acquisition-related contingent consideration for the quarter and nine months ended September 30, 2017 was a benefit of $16 million and $2 million, respectively, as compared to expense of $8 million and $10 million for the same periods in 2016. The change in fair value of acquisition-related contingent consideration represents the fair market value adjustments of the Company’s contingent liability related to the acquisition of the European business of ARIAD Pharmaceuticals, Inc.
Unrealized gain (loss) on long term investments Unrealized gain on long term investments for the quarter ended September 30, 2017 was $23 million as compared to $24 million for the same period in 2016. The unrealized loss on long term investments for the nine months ended September 30, 2017 was $2 million as compared to an unrealized gain of $20 million for the same period in 2016. The unrealized gain or loss on long term investments for the quarter and nine months ended September 30, 2017 represents the fair market value adjustments of the Company’s investments in Agenus and Merus.

Expense related to senior note conversions Expense related to senior note conversions for the nine months ended September 30, 2017 was $55 million related to the conversions of certain of our 2018 and 2020 convertible senior notes.

Net income (loss) Net income for the quarter ended September 30, 2017 was $36 million, or $0.17 per basic and diluted share, as compared to net income of $37 million, or $0.20 per basic and $0.19 per diluted share for the same period in 2016. Net loss for the nine months ended September 30, 2017 was $164 million, or $0.81 per basic and diluted share, as compared to net income of $95 million, or $0.51 per basic and $0.49 per diluted share for the same period in 2016.

Cash, cash equivalents and marketable securities position As of September 30, 2017, cash, cash equivalents and marketable securities totaled $1.3 billion as compared to $809 million as of December 31, 2016. The increase in cash, cash equivalents and marketable securities from December 31, 2016 to September 30, 2017 is primarily due to the recent public offering of 4,945,000 shares of our common stock resulting in net proceeds of $649 million.
2017 Financial Guidance

The Company has updated its full year 2017 financial guidance, as detailed below.

Current

Previous
Jakafi net product revenues $1,125-$1,135 million $1,090-$1,120 million
Iclusig net product revenues $60-$65 million Unchanged
Research and development expenses* $1,250-$1,300 million $1,050-$1,150 million
Selling, general and administrative expenses $340-$360 million Unchanged
Change in fair value of acquisition-related contingent consideration $5-$7 million $30-$35 million

* Includes upfront and milestone expenses of $359 million related to the amended Agenus collaboration, and the Merus, Calithera, and MacroGenics collaborations

Conference Call and Webcast Information

Incyte will hold its 2017 third-quarter financial results conference call and webcast this morning at 10:00 a.m. ET. To access the conference call, please dial 877-407-3042 for domestic callers or 201-389-0864 for international callers. When prompted, provide the conference identification number, 13672268.
If you are unable to participate, a replay of the conference call will be available for 30 days. The replay dial-in number for the United States is 877-660-6853 and the dial-in number for international callers is 201-612-7415. To access the replay you will need the conference identification number, 13672268.
The conference call will also be webcast live and can be accessed at www.incyte.com in the Investors section under "Events and Presentations".