On November 3, 2016 Seattle Genetics, Inc. (Nasdaq:SGEN) reported that 18 abstracts including eight oral presentations have been accepted for presentation at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place in San Diego, Calif. from December 3-6, 2016 (Press release, Seattle Genetics, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219399 [SID1234516296]). Collectively, the abstracts highlight advancement of the ADCETRIS (brentuximab vedotin) and vadastuximab talirine (SGN-CD33A) development programs and the company’s expanding global leadership in antibody-drug conjugates (ADCs), as well as its commitment to developing investigational compounds that have the potential to improve therapeutic options for patients with blood-related cancers.

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Data accepted for presentation at this year’s ASH (Free ASH Whitepaper) Annual Meeting include the following:

An oral presentation of full results from the phase 3 ALCANZA clinical trial evaluating ADCETRIS in patients with CD30-expressing cutaneous T-cell lymphoma (CTCL)
Numerous oral and poster presentations highlighting additional progress within the ADCETRIS development program including:
Preliminary results from a phase 1/2 study of ADCETRIS in combination with Opdivo (nivolumab) among patients with relapsed or refractory Hodgkin lymphoma (HL)
Long-term (e.g., four-year) survival and durability results in patients with CD30-expressing peripheral T-cell lymphomas who received ADCETRIS with cyclophosphamide, hydroxydaunorubicin, and prednisone (CHP) as frontline therapy
Final five-year survival and durability results in relapsed systemic anaplastic large cell lymphoma (sALCL) patients who received ADCETRIS monotherapy
Four oral presentations featuring data from clinical studies exploring vadastuximab talirine in acute myeloid leukemia (AML), including newly diagnosed patients
"At this year’s ASH (Free ASH Whitepaper) Annual Meeting, we will present data from 18 abstracts, highlighting ADCETRIS, vadastuximab talirine and multiple pipeline programs," said Jonathan Drachman, M.D., Chief Medical Officer and Executive Vice President, Research and Development at Seattle Genetics. "The oral presentation of the complete results of the ALCANZA trial represents the fourth consecutive registrational trial for ADCETRIS with a positive outcome. Data across our pipeline programs continue to support the potential for antibody-drug conjugates to improve outcomes for patients with cancer."

Seattle Genetics is the world leader in the development and commercialization of a new generation of ADCs. The company’s novel, proprietary technology is designed to harness the targeting ability of antibodies to deliver cell-killing agents directly to cancer cells. There are more than 20 ADCs in clinical development that utilize Seattle Genetics’ proprietary ADC technology. ADCETRIS is the first drug approved utilizing this technology. The company also has vadastuximab talirine in a global phase 3 clinical study (CASCADE) in newly diagnosed, older AML patients.

ADCETRIS is currently not approved for the treatment of CTCL frontline treatment of non-Hodgkin lymphoma or as a combination therapy for HL.

Multiple corporate and investigator presentations will be featured at ASH (Free ASH Whitepaper). Abstracts can be found at www.hematology.org and include the following:

Saturday, December 3, 2016

Results of an Ongoing Phase 2 Study of Brentuximab Vedotin with RCHP as Frontline Therapy in Patients with High-Intermediate/High-Risk Diffuse Large B-Cell Lymphoma (Abstract #104, oral presentation at 9:45 a.m. PT)
Brentuximab Vedotin Demonstrates Significantly Superior Clinical Outcomes in Patients with CD30-Expressing Cutaneous T-Cell Lymphoma Versus Physician’s Choice (Methotrexate or Bexarotene): the Phase 3 ALCANZA Study (Abstract #182, oral presentation at 2:15 p.m. PT)
A Phase 1b Study of Vadastuximab Talirine in Combination with 7+3 Induction Therapy for Patients with Newly Diagnosed Acute Myeloid Leukemia (Abstract #211, oral presentation at 4:00 p.m. PT)
Safety and Activity of Brentuximab Vedotin plus Ifosfamide, Carboplatin, and Etoposide (ICE) for Relapsed/Refractory Classical Hodgkin Lymphoma: Initial Results of a Phase I/II Trial (Abstract #1834, poster presentation)
Real World Clinical and Economic Burden of Hematopoietic Cell Transplantation Among a Large US Commercially Insured Population (Abstract #2368, poster presentation)
Sunday, December 4, 2016

The Use of a Novel CME Format to Elicit and Develop True Competence in Hematologist Ability to Risk Stratify Patients with Hodgkin Lymphoma (Abstract #3560, poster presentation)
A Phase 1b Study of Vadastuximab Talirine as Maintenance and in Combination with Standard Consolidation for Patients with Acute Myeloid Leukemia (Abstract #340, oral presentation at 10:15 a.m. PT)
Four-Year Survival and Durability Results of Brentuximab Vedotin in Combination with CHP in the Frontline Treatment of Patients with CD30-Expressing Peripheral T-cell Lymphomas (Abstract #2993, poster presentation)
Toxicity Burden of Bleomycin Treatment in Hodgkin Lymphoma: A Systematic Literature Review (Abstract #3566, poster presentation)
Monday, December 5, 2016

Vadastuximab Talirine Monotherapy in Older Patients with Treatment Naive CD33-Positive Acute Myeloid Leukemia (Abstract #590, oral presentation at 7:15 a.m. PT)
Vadastuximab Talirine Plus Hypomethylating Agents: A Well-Tolerated Regimen with High Remission Rate in Frontline Older Patients With Acute Myeloid Leukemia (Abstract #591, oral presentation at 7:30 a.m. PT)
Preliminary Results from a Phase 1/2 Study of Brentuximab Vedotin in Combination with Nivolumab in Patients with Relapsed or Refractory Hodgkin Lymphoma (Abstract #1105, oral presentation at 4:30 p.m. PT)
A Phase I Study with an Expansion Cohort of the Combination of Ipilimumab and Nivolumab and Brentuximab Vedotin in Patients with Relapsed/Refractory Hodgkin Lymphoma: A Trial of the ECOG-ACRIN Cancer Research Group (E4412 Arms D and E) (Abstract #1106 oral presentation at 4:45 p.m. PT)
Brentuximab Vedotin Plus ESHAP (BRESHAP) Is a Highly Effective Combination for Inducing Remission in Refractory and Relapsed Hodgkin Lymphoma Patients Prior to Autologous Stem Cell Transplant: A Trial of the Spanish Group of Lymphoma and Bone Marrow Transplantation (GELTAMO) (Abstract #1109, oral presentation at 5:30 p.m. PT)
Five-Year Survival Data from a Pivotal Phase 2 Study of Brentuximab Vedotin in Patients with Relapsed or Refractory Systemic Anaplastic Large Cell Lymphoma (Abstract #4144, poster presentation)
Denintuzumab Mafodotin Stimulates Immune Responses and Synergizes with CD20 Antibodies to Heighten Anti-tumor Activity in Preclinical Models of Non-Hodgkin Lymphoma (Abstract #4177, poster presentation)
SGN-CD48A: A Novel Humanized Anti-CD48 Antibody-Drug Conjugate for the Treatment of Multiple Myeloma (Abstract #4470, poster presentation)
A Phase 1/2 Clinical Trial of Brentuximab Vedotin and Bendamustine in Elderly Patients with Previously Untreated Advanced Hodgkin Lymphoma (Halo Study): Preliminary Report (Abstract #4154, poster presentation)
About ADCETRIS

ADCETRIS is being evaluated broadly in more than 70 ongoing clinical trials, including two phase 3 studies, ECHELON-1 in frontline classical Hodgkin lymphoma and ECHELON-2 in frontline mature T-cell lymphomas, as well as trials in many additional types of CD30-expressing malignancies, including B-cell lymphomas.

ADCETRIS is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics’ proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS for intravenous injection has received approval from the FDA for three indications: (1) regular approval for the treatment of patients with classical Hodgkin lymphoma after failure of autologous hematopoietic stem cell transplantation (auto-HSCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (2) regular approval for the treatment of classical Hodgkin lymphoma patients at high risk of relapse or progression as post-auto-HSCT consolidation, and (3) accelerated approval for the treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen. The sALCL indication is approved under accelerated approval based on overall response rate. Continued approval for the sALCL indication may be contingent upon verification and description of clinical benefit in confirmatory trials. Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL.

ADCETRIS was granted conditional marketing authorization by the European Commission in October 2012 for two indications: (1) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following autologous stem cell transplant (ASCT), or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, and (2) the treatment of adult patients with relapsed or refractory sALCL. ADCETRIS has received marketing authorization by regulatory authorities in 65 countries.

In June 2016, the European Commission extended the current conditional approval of ADCETRIS and approved ADCETRIS for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT. See important safety information below.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

About Vadastuximab Talirine (SGN-CD33A)

Vadastuximab talirine (SGN-CD33A) is a novel ADC targeted to CD33 utilizing Seattle Genetics’ newest ADC technology. CD33 is expressed on most AML cells. The CD33 antibody is attached to a highly potent DNA binding agent, a pyrrolobenzodiazepine (PBD) dimer, via a proprietary site-specific conjugation technology to a monoclonal antibody with engineered cysteines (EC-mAb). PBD dimers are significantly more potent than systemic chemotherapeutic drugs and the site-specific conjugation technology (EC-mAb) allows uniform drug-loading of the cell-killing PBD agent to the anti-CD33 antibody. The ADC is designed to be stable in the bloodstream and to release its potent DNA binding agent upon internalization into CD33-expressing cells. SGN-CD33A is being evaluated in ongoing phase 1 clinical trials for patients with AML. More information about SGN-CD33A and ongoing clinical trials can be found at www.ADC-CD33.com.

About Denintuzumab Mafodotin (SGN-CD19A)

Denintuzumab mafodotin (SGN-CD19A) is an ADC targeting CD19, a protein expressed broadly on B-cell malignancies. Denintuzumab mafodotin is comprised of an anti-CD19 monoclonal antibody linked to a synthetic cytotoxic cell-killing agent, monomethyl auristatin F (MMAF). The ADC is designed to be stable in the bloodstream, and to release its cytotoxic agent upon internalization into CD19-expressing tumor cells. This approach is intended to spare non-targeted cells and thus reduce many of the toxic effects of traditional chemotherapy while enhancing the antitumor activity. SGN-CD19A is being evaluated in two ongoing phase 1 clinical trials for patients with B-cell ALL and aggressive NHL as well as a phase 2 clinical trial in relapsed or refractory DLBCL.

On November 3, 2016 MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX, OTC: MPSYY) reported upcoming presentations of data on the Company’s proprietary hemato-oncological programs MOR208 and MOR202 at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 3-6, 2016 in San Diego, California/USA (Press release, MorphoSys, NOV 3, 2016, View Source [SID1234516292]).

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"We are pleased that updated clinical trial results with our antibodies MOR208 and MOR202 in patients with B-cell malignancies and multiple myeloma, respectively, will be shown at the upcoming ASH (Free ASH Whitepaper) conference as oral presentations," commented Dr. Arndt Schottelius, Chief Development Officer of MorphoSys AG. "We expect this will further demonstrate the potential of our proprietary development candidates for patients suffering from those hemato-oncological malignancies that have a particularly high unmet medical need."

Abstracts from MorphoSys’s proprietary programs accepted for presentation at ASH (Free ASH Whitepaper) 2016:

Single-Agent MOR208 in Relapsed or Refractory (R-R) Non-Hodgkin’s Lymphoma (NHL): Results from Diffuse Large B-Cell Lymphoma (DLBCL) and Indolent NHL Subgroups of a Phase IIa Study

The oral presentation will include updated clinical results, in particular with respect to duration of responses, from a phase 2a MOR208 monotherapy trial in adult patients with relapsed/refractory NHL including DLBCL and iNHL.
Abstract #623
Session Name: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas) – Results from Prospective Clinical Trials: Novel Agents
Session Date: Monday, December 5, 2016
Session Time: 7:00am-8:30am (PST) (4:00pm-5:30pm CET)
Presentation Time: 8:00am PST (5:00pm CET)
Room: San Diego Convention Center, Room 6B

A Phase I/IIa Study of the CD38 Antibody MOR202 Alone and in Combination with Pomalidomide or Lenalidomide in Patients with Relapsed or Refractory Multiple Myeloma

The oral presentation will include updated safety and efficacy results from the ongoing phase 1/2a MOR202 dose-escalation study in pre-treated multiple myeloma patients. Results will in particular include maturing data from the MOR202 8mg/kg patient cohorts in combination with lenalidomide and pomalidomide as well as first results from the highest dose cohorts of 16mg/kg MOR202 in combination with lenalidomide and pomalidomide.
Abstract #1152
Session Name: 653. Myeloma: Therapy, excluding Transplantation: Immunotherapy
Session Date: Monday, December 5, 2016
Session Time: 4:30pm-6:00pm (PST) (December 6, 2016, 1:30am-3:00am CET)
Presentation Time: 5:45pm (PST) (December 6, 2016, 2:45am CET)
Room: San Diego Convention Center, Hall AB

Updated Results from a Phase II Study of the Fc Engineered CD19 Antibody MOR208 in Combination with Lenalidomide for Patients with Chronic Lymphocytic Leukemia (CLL) and Richter’s Transformation or Ibrutinib for Patients with Ibrutinib-Resistant Clones
The poster presentation will include safety and efficacy results from the ongoing phase 2 investigator initiated trial (IIT) of MOR208 in combination with lenalidomide or ibrutinib in CLL.
Abstract #4386
Session Name: 642. CLL: Therapy, excluding Transplantation: Poster III
Date: Monday, December 5, 2016
Presentation Time: 6:00pm-8:00pm (PST) (December 6, 2016, 3:00am-5:00am CET)
Location: San Diego Convention Center, Hall GH

In addition to the presentations, all abstracts will be published online in the December 1, 2016 supplemental volume of Blood.
Additional information can be found at www.hematology.org, including the abstracts.

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.