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 Poseida Therapeutics Inc. ("Poseida"), a San Diego-based company translating best-in-class gene editing technologies into lifesaving therapeutics, reported that three abstracts detailing preclinical data on P-BCMA-101, the company’s BCMA-specific CAR-T drug candidate, have been selected for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Poseida Therapeutics, NOV 3, 2016, View Source [SID1234516624]). The studies explore utility and performance of Poseida’s proprietary CAR-T modifications using its piggyBac non-viral gene delivery system and Centyrin binding scaffold. Data will be presented at the 2016 ASH (Free ASH Whitepaper) Annual Meeting in San Diego on December 3rd and 5th. Abstracts are currently available online at View Source

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The following abstracts will be presented:

Presentation Title: A Novel BCMA-Specific, Centyrin-Based CAR-T Product for the Treatment of Multiple Myeloma
Abstract Number: 2127
Date and Time: 5:30 p.m. – 7:30 p.m., Saturday, December 3, 2016
Location: San Diego Convention Center, Hall GH

Presentation Title: piggyBac-Produced CAR-T Cells Exhibit Stem-Cell Memory Phenotype
Abstract Number: 2167
Date and Time: 5:30 p.m. – 7:30 p.m., Saturday, December 3, 2016
Location: San Diego Convention Center, Hall GH

Presentation Title: Development of Novel Non-Immunoglobulin Centyrin-Based CARs (CARTyrins) Targeting Human BCMA
Abstract Number: 4557
Date and Time: 6:00 p.m. – 8:00 p.m., Monday, December 5, 2016
Location: San Diego Convention Center, Hall GH

On November 3, 2016 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative and effective drug candidates for the treatment of human cancers, reported its financial results for the third quarter ended September 30, 2016 (Press release, Curis, NOV 3, 2016, View Source [SID1234516211]).

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"We are pleased with our progress this quarter, and remain focused on patient enrollment within our two clinical programs. CA-170’s Phase 1 trial is progressing rapidly through the dose escalation stage with no limiting adverse safety effects," said Ali Fattaey, Ph.D., Curis’s CEO. "Additionally, we continue to enroll at multiple centers in the Phase 2 trial of CUDC-907 in patients with relapsed/refractory DLBCL. Our goal is to assess CUDC-907’s efficacy in patients with MYC-altered DLBCL and we expect to use this information for discussion with the FDA in 2017."

Dr. Fattaey continued, "Our collaboration with Aurigene continues to progress well. In September, we completed a $24.5M financing with Aurigene. In October, we licensed a second immuno-oncology program, designating CA-327 as an oral small molecule development candidate targeting PDL1 and TIM3. We expect to file an IND for CA-327 in 2017."

Third Quarter 2016 Financial Results

Curis reported a net loss of $28.3 million, or $(0.21) per share on both a basic and diluted basis for the third quarter of 2016, as compared to a net loss of $5.5 million, or $(0.04) per share on both a basic and diluted basis for the same period in 2015. Curis reported a net loss of $49.1 million or $(0.38) per share on both basic and diluted basis for the nine months ended September 30, 2016, as compared to a net loss of $45.5 million, or $(0.37) per share on both basic and diluted basis for the same period in 2015. The net loss for the three and nine months ended September 30, 2016 includes a non-cash in-process research and development charge of $18.0 million related to the amendment of Curis’s license agreement with Aurigene. The net loss for the nine months ended September 30, 2015 includes a non-cash in-process research and development charge of $24.3 million related to Curis’s license agreement with Aurigene.

Revenues for the third quarter of 2016 were $1.8 million, as compared to $2.0 million for the same period in 2015. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge. Revenues for the nine months ended September 30, 2016 were $5.2 million, as compared to $5.8 million for the same period in 2015.

Operating expenses were $29.5 million for the third quarter of 2016, as compared to $6.9 million for the same period in 2015.

Operating expenses for the nine months ended September 30, 2016 were $52.4 million, as compared to $49.0 million for the same period in 2015, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the third quarter of 2016 and 2015. Cost of royalty revenues for the nine months ended September 30, 2016 and 2015 were $0.3 million for both periods.

In-Process Research and Development Expense. In-process research and development expense was $18.0 million for the third quarter of 2016, as compared to $24.3 million for the same period in 2015. These charges are associated with the stock issuances of 10,208,333 and 17,120,131 shares of Curis common stock to Aurigene, respectively. These shares were issued as consideration for the rights granted under the terms of the September 2016 amendment to the collaboration agreement and partial consideration for the rights granted under the terms of the January 2015 collaboration agreement, respectively.

Research and Development Expenses. Research and development expenses were $6.8 million for the third quarter of 2016, as compared to $4.0 million for the same period in 2015. The increase was primarily due to increased direct spending related to clinical activities of CUDC-907 and programs under the Aurigene collaboration over the prior year period. Employee-related expenses increased over the prior year period primarily due to additional headcount to support the multiple programs. Research and development expenses were $22.4 million for the nine months ended September 30, 2016 as compared to $14.7 million for the same period in 2015.

General and Administrative Expenses. General and administrative expenses were $4.7 million for the third quarter of 2016 as compared to $2.8 million for the same period in 2015. The increase in general and administrative expenses was driven primarily by higher personnel costs and stock-based compensation expense due to increased headcount and an increase in legal service costs. General and administrative expenses were $11.7 million for the nine months ended September 30, 2016, as compared to $9.7 million for the same period in prior 2015.

Other expense, net was $0.6 million for the third quarter of 2016, as compared to $0.7 million for the same period in 2015. Other expense, net primarily consisted of interest expense related to the loan made by BioPharma-II (an investment fund managed by Pharmakon Advisors) to Curis Royalty (a wholly owned subsidiary of Curis). Other expense, net was $1.8 million and $2.3 million for the nine months ended September 30, 2016 and 2015, respectively.

As of September 30, 2016, Curis’s cash, cash equivalents, marketable securities and investments totaled $53.4 million and there were approximately 140.0 million shares of common stock outstanding.

Recent Operational Highlights

Curis – Aurigene collaboration:

In October, 2016 Curis licensed the second oral small molecule immuno-oncology development candidate under the collaboration. This program is focused on the development of oral, small molecule antagonists that target two distinct checkpoint pathways: the programmed death-1 (PD1) and T-cell immunoglobulin and mucin domain containing protein-3 (TIM3) pathways. Curis has designated CA-327 that targets programmed death ligand-1 (PDL1) and TIM3 as the development candidate.

In September, 2016 Curis’s collaboration Aurigene Discovery Technologies Ltd. invested in Curis at a premium by acquiring 10.2 million shares of Curis’s common stock in lieu of receiving up to $24.5 million of milestone and other payments from Curis that may become due under the companies’ 2015 collaboration agreement.
Upcoming Activities

Curis expects that it will make presentations at the following conferences through December 2016:

Presentation of preclinical results from CA-170 and CA-327 at the SITC (Free SITC Whitepaper) conference
Presentation of preclinical results from combination of CUDC-907 with other treatments at the ASH (Free ASH Whitepaper) annual conference

On November 3, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that SL-401, a novel targeted therapeutic directed to CD123, will be featured in 7 presentations, including 3 oral presentations, at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held December 3-6, 2016 at the San Diego Convention Center in San Diego, CA (Press release, Stemline Therapeutics, NOV 3, 2016, View Source [SID1234516297]). The full abstracts are now available on the ASH (Free ASH Whitepaper) conference website.

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Investigators will deliver an oral presentation on updated clinical data from the SL-401 Phase 2 trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN). Additional presentations include early clinical data from ongoing SL-401 trials in patients with acute myeloid leukemia (AML) in remission with high relapse risk and minimal residual disease (MRD), high-risk myeloproliferative neoplasms (MPN), and relapsed/refractory multiple myeloma. Preclinical data of SL-401 against AML, myelodysplastic syndrome (MDS), and myeloma cancer stem cells, as well as SL-401 in combination with SL-801, a novel XPO1 inhibitor, against myeloma and other malignancies will be presented as well.

Ivan Bergstein, M.D., Stemline’s CEO, commented, "We are honored to be presenting a broad range of SL-401 studies, including three oral presentations, at this year’s ASH (Free ASH Whitepaper) conference. SL-401 is rapidly becoming recognized by the community as not only an active anticancer agent, but also one with the potential versatility, due to its unique mechanism of action and manageable safety profile, to be utilized as single agent or in combination in a broad range of indications." Dr. Bergstein concluded, "Importantly, our clinical data in BPDCN continue to strengthen with increasing patient numbers and exposure, and we look forward to providing a robust and detailed update on this potentially pivotal program at the upcoming conference."

Details on the presentations are as follows:

SL-401 – BPDCN (Clinical) – Oral Presentation
Title: Results from Phase 2 Trial Ongoing Expansion Stage of SL-401 in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
Abstract: 342
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Optimizing Current AML Therapy
Date/Time: Sunday, December 4, 2016 10:45 AM PT
Location: Marriott Marquis San Diego Marina, Pacific Ballroom

SL-401 – AML in CR with MRD (Clinical) – Oral Presentation
Title: Results from Ongoing Phase 2 Trial of SL-401 As Consolidation Therapy in Patients with Acute Myeloid Leukemia (AML) in Remission with High Relapse Risk Including Minimal Residual Disease (MRD)
Presenter: Andrew Lane, MD, PhD; Dana-Farber Cancer Institute
Abstract: 215
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Innovations in Induction Therapy
Date/Time: Saturday, December 3, 2016 5:00 PM PT
Location: Marriott Marquis San Diego Marina, San Diego Ballroom AB

SL-401 – Myeloproliferative neoplasms (Clinical)
Title: Results from Ongoing Phase 2 Trial of SL-401 in Patients with Advanced, High-Risk Myeloproliferative Neoplasms Including Chronic Myelomonocytic Leukemia
Presenter: Mrinal Patnaik, MBBS; Mayo Clinic
Abstract: 4245
Session: 634. Myeloproliferative Syndromes: Clinical: Poster III
Date/Time: Monday, December 5, 2016 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH

SL-401 — Multiple myeloma (Clinical)
Title: Results from Ongoing Phase 1/2 Trial of SL-401 in Combination with Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma
Presenter: Myo Htut, MD; City of Hope
Abstract: 5696
Date/Time: Thursday, December 1, 2016 publication release
Location: Published online on ASH (Free ASH Whitepaper) abstract website

SL-401 – AML and MDS cancer stem cells – Oral Presentation
Title: SL-401 Mediates Potent Cytotoxicity Against CD123+ AML and MDS with Excess Blasts and Demonstrates Therapeutic Benefit in PDX Model
Presenter: Rajeswaran Mani, PhD; Ohio State University
Abstract: 580
Session: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Targeting Leukemia-Initiating Cells
Date/Time: Monday, December 5, 2016?7:45 AM PT
Location: San Diego Convention Center, Room 24

SL-401 in combination with SL-801 — Multiple myeloma and other malignancies
Title: SL-401, a Targeted Therapy Directed to the Interleukin-3 Receptor (CD123), and SL-801, a Reversible Inhibitor of Exportin-1 (XPO1), Display Synergistic Anti-Tumor Activity Against Hematologic Malignancies in Vitro
Presenter: Janice Chen, PhD; Stemline
Abstract: 4724
Session: 802. Chemical Biology and Experimental Therapeutics: Poster III
Date/Time: Monday, December 5, 2016 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH

SL-401 – Multiple myeloma
Title: SL-401, a Novel IL-3Rα/CD123—Directed Agent Targets Stem-like Cells in Multiple Myeloma
Presenter: Arghya Ray, PhD; Dana-Farber Cancer Institute
Abstract: 4463
Session: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster III
Date/Time: Monday, December 5, 2016 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GHa

On November 2, 2016 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported business highlights and financial results for the third quarter ended September 30, 2016 (Press release, bluebird bio, NOV 2, 2016, View Source;p=RssLanding&cat=news&id=2218784 [SID1234516184]).

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"In recent months, we have made important advances in our gene therapy transduction and manufacturing processes, translational research and clinical development that will be critical for us to successfully bring LentiGlobin drug product to patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD). This includes implementing a new LentiGlobin manufacturing process that has been shown in vitro to consistently improve the percentage of cells transduced and vector copy number, and making a number of changes to the protocol for our ongoing severe SCD study that we believe have the potential to improve patient outcomes. We look forward to sharing initial clinical data from these improvements in 2017," said Nick Leschly, chief bluebird. "In September we announced a strategic alliance with Medigene around TCRs against four targets, a significant step forward in our efforts to build a broad, fully integrated immuno-oncology franchise. We anticipate sharing further progress in oncology in the first half of 2017, with presentation of initial data from our Phase 1 study of bb2121, our anti-BCMA CAR T program in multiple myeloma."

Recent Highlights

PHASE 3 HGB-207 STUDY OF LENTIGLOBIN OPENED – In September, bluebird bio opened the company’s first Phase 3 study of LentiGlobin drug product. HGB-207 is a global, multi-center study in patients with TDT with non-β0/β0 genotypes. This study will incorporate the addition of bluebird bio’s transduction enhancers to the drug product manufacturing process, and will be conducted under the same Investigational New Drug application (IND) as previous studies of LentiGlobin drug product in TDT. This study, which will include adult and adolescent patients (cohort #1) and pediatric patients (cohort #2), is intended to be pivotal in the U.S. and confirmatory in the E.U. bluebird bio plans to initiate the HGB-212 pivotal study of LentiGlobin drug product in patients with TDT with β0/β0 genotypes in 2017.

GENE THERAPY DAY – In October, bluebird bio held a Gene Therapy Day to provide updates on its LentiGlobin product candidate and its research, development and commercialization strategies. Highlights included:
A head-to-head in vitro comparison of manufacturing Process 1 and Process 2 consistently improved the percentage of cells transduced and vector copy number (VCN) in cells from TDT patients of all genotypes and severe SCD patients.

bluebird bio has implemented several amendments to the protocol of the ongoing HGB-206 study in severe SCD, with the goal of improving patient outcomes by increasing successful engraftment of transduced cells.

bluebird bio has reached general agreement with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) on the regulatory paths forward for LentiGlobin in TDT in the U.S. and E.U.

STRATEGIC TCR ALLIANCE WITH MEDIGENE – In September, bluebird bio and Medigene announced that they have established a strategic alliance in cancer immunotherapy focused on four TCR targets. Under the terms of the agreement, Medigene will be responsible for the generation and delivery of TCRs using its TCR isolation and characterization platform. Following collaborative preclinical development, bluebird bio will assume sole responsibility for the clinical development and commercialization of the TCR product candidates and will receive an exclusive license for intellectual property covering the resulting TCRs.
LENTIGLOBIN ACCEPTED INTO EUROPEAN MEDICINES AGENCY (EMA) PRIME PROGRAM – In September, the EMA granted access to its Priority Medicines (PRIME) scheme for LentiGlobin drug product in the treatment of patients with TDT. The PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly. To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical data or nonclinical data.
Upcoming Anticipated Milestones

Presentation of updated TDT and SCD clinical data from the Northstar (HGB-204), HGB-205 and HGB-206 clinical studies at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December
Presentation of early data from CRB-401, the Phase 1 trial of bb2121 in relapsed/refractory multiple myeloma in the first half of 2017
Third Quarter 2016 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2016 were $727.6 million, compared to $865.8 million as of December 31, 2015, a decrease of $138.2 million.
Revenues: Collaboration revenue was $1.6 million for the third quarter of 2016 compared to $1.3 million for third quarter of 2015.
R&D Expenses: Research and development expenses were $64.0 million for the third quarter of 2016 compared to $30.4 million for the third quarter of 2015. The increase in research and development expenses was primarily attributable to increased in-licensing milestones and fees, increased manufacturing costs and increased information technology and facilities costs to support the advancement of our clinical and pre-clinical programs, and increased employee payroll costs to support our overall growth.
G&A Expenses: General and administrative expenses were $14.6 million for the third quarter of 2016 compared to $13.7 million for the third quarter of 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, partially offset by decreased stock-based compensation expense.
Net Loss: Net loss was $77.0 million for the third quarter of 2016 compared to $42.9 million for the third quarter of 2015.
Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $727.6 million as of September 30, 2016 will be sufficient to fund its current operations through 2018.