Aptose to Present New APTO-253 Data at the 2016 ASH Annual Meeting

On November 3, 2016 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing new therapeutics and molecular diagnostics that target the underlying mechanisms of cancer, reported that new preclinical data for its lead investigational anticancer therapeutic APTO-253 will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 3-6, 2016, in San Diego, CA (Press release, Aptose Biosciences, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219559 [SID1234516219]).

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!

Poster Presentation Details:
Inhibition of c-Myc By Apto-253 As an Innovative Therapeutic Approach to Induce Cell Cycle Arrest and Apoptosis in Acute Myeloid Leukemia
Date & Time: Saturday, December 3, 2016, 5:30-7:30 p.m.
Session Title: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Poster I
Abstract Number: 1716
Location: San Diego Convention Center, Hall GH
The abstract can be viewed on the ASH (Free ASH Whitepaper) conference website at the following link:
View Source

Acceleron Pharma Reports Third Quarter 2016 Financial and Operational Results

On November 3, 2016 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, reported a corporate update and reported financial results for the third quarter ended September 30, 2016 (Press release, Acceleron Pharma, NOV 3, 2016, View Source [SID1234516218]).

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!

"In the third quarter, we continued to make strong progress across our pipeline of clinical and preclinical programs," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "Enrollment continues to go well in both of the ongoing luspatercept Phase 3 studies in patients with myelodysplastic syndromes and beta-thalassemia. We look forward to providing new and updated preliminary results from several Phase 2 studies at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December. Within our muscle program, we’re on track to initiate the ACE-083 Phase 2 study in patients with facioscapulohumeral muscular dystrophy before year-end."

Continued Dr. Knopf, "I would also like to extend a warm welcome to the Company’s newly appointed Chief Executive Officer, Habib Dable, who will assume this role on December 1st. Habib’s global pharmaceutical operational and commercial experience will serve Acceleron well as we advance toward becoming a commercial-stage company."

THIRD QUARTER 2016 HIGHLIGHTS

Development Programs

Hematology

Enrollment is ongoing in the pivotal luspatercept Phase 3 clinical trials for the treatment of myelodysplastic syndromes (MDS) patients (the MEDALIST study) and beta-thalassemia patients (the BELIEVE study).
Enrollment is ongoing in Phase 2 trial cohorts evaluating luspatercept in a first-line treatment setting in low- or intermediate-risk MDS patients.
Data from five clinical abstracts on luspatercept and sotatercept will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held in San Diego, California on December 3-6, 2016.
Neuromuscular Disease

Initiation of the ACE-083 Phase 2 study in facioscapulohumeral muscular dystrophy (FSHD) patients expected to start by the end of the year.
Oncology

Enrollment continues in randomized part 2 of the Phase 2 DART study of dalantercept in combination with axitinib in patients with advanced renal cell carcinoma (RCC). The primary endpoint of this trial, progression-free survival (PFS), is an event-driven assessment with preliminary data expected in the first half of 2017.
Preclinical Research and Development

Acceleron continues its research on several molecules targeting musculoskeletal diseases, fibrotic disorders and other serious diseases. The most advanced molecule from the IntelliTrap platform, ACE-2494, continues to advance through IND-enabling activities. The Company plans to initiate a Phase 1 clinical trial with ACE-2494 in mid-2017.
Other Corporate Updates

Habib Dable appointed as President and CEO effective December 1, 2016. Mr. Dable joins Acceleron from Bayer AG, where he most recently served as President of U.S. Pharmaceuticals.
John Knopf retiring as President and CEO. Dr. Knopf is expected to apply his scientific expertise as an advisor to the Company.
Upcoming Events in Q4 2016

Present data at the ASH (Free ASH Whitepaper) annual meeting from ongoing luspatercept Phase 2 studies and an ongoing sotatercept Phase 2 investigator initiated trial in patients with myelofibrosis.
Initiate ACE-083 Phase 2 study in patients with FSHD, one of the most prevalent forms of muscular dystrophy.
Provide sotatercept development plan update before year-end.
Present at the 25th Annual Credit Suisse Healthcare Conference on Monday, November 7, 2016 in Scottsdale, Arizona.
Participate at the Citi Global Healthcare Conference to be held on December 7-8, 2016 in New York.
Financial Results

Cash position – Cash, cash equivalents and investments as of September 30, 2016 were $251.0 million. As of December 31, 2015 the Company had cash, cash equivalents and investments of $136.0 million. The Company believes that existing cash, cash equivalents and investments will be sufficient to fund its projected operating requirements into the second half of 2019.
Revenue – Collaboration revenue for the third quarter was $3.0 million. Cost sharing reimbursement revenue from the Company’s Celgene partnership was $2.9 million and is related to expenses incurred by the Company in support of its partnered programs.
Costs and expenses – Total costs and expenses for the third quarter were $23.5 million. This includes R&D expenses of $17.1 million and G&A expenses of $6.4 million.
Other expense, net – Other expense for the third quarter was $0.3 million and includes a $0.8 million, non-cash, loss on marking the Company’s common stock warrant liability to market.
Net loss – The Company posted a net loss for the third quarter ended September 30, 2016 of $20.8 million.

Geron Announces Presentations at American Society of Hematology Annual Meeting

On November 3, 2016 Geron Corporation (Nasdaq:GERN) reported that four abstracts related to the imetelstat program have been accepted for presentation at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held in San Diego, California from December 3-6, 2016 (Press release, Geron, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219235 [SID1234516217]). The abstracts were published today on the ASH (Free ASH Whitepaper) website at www.hematology.org.

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!

Oral Presentation

One abstract containing non-clinical data on imetelstat was selected for oral presentation. These data extend previous preclinical work conducted in a patient-derived model of acute myeloid leukemia.

Title: The preclinical efficacy of a novel telomerase inhibitor, imetelstat, in AML – a randomized trial in patient-derived xenografts (Abstract #578)
Session Name: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Targeting Leukemia-Initiating Cells
Session Date: Monday, December 5, 2016
Presentation Time: 7:15 a.m. PT

Poster Presentations

Three abstracts related to imetelstat were selected for presentation as posters. One abstract investigates the potential impact of imetelstat on leukemia stem cells in a non-clinical model of chronic myeloid leukemia. The second abstract reports new data on telomere length dynamics from essential thrombocythemia patients providing further support that imetelstat treatment may suppress the neoplastic clones underlying the disease. The third abstract presents an analysis of treatment patterns and outcomes of patients with myelofibrosis from the United States medical claims databases that underscores the need for additional treatment options in that disease.

Title: Telomerase Inhibition with Imetelstat Eradicates β-catenin Activated Blast Crisis Chronic Myeloid Leukemia Stem Cells (Abstract #3065)
Session Name: 632. Chronic Myeloid Leukemia: Therapy: Poster II
Session Date: Sunday, December 4, 2016
Session Time: 6:00 p.m. – 8:00 p.m. PT

Title: Dynamics of Telomere Length Reflect the Clonal Suppression Seen with the Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia (Abstract #1938)
Session Name: 634. Myeloproliferative Syndromes: Clinical: Poster I
Session Date: Saturday, December 3, 2016
Session Time: 5:30 p.m. – 7:30 p.m. PT

Title: Characterization of Disease, Treatment Patterns, and Outcomes of Patients with Myelofibrosis: Analysis of 2 United States Commercial Claims Databases (Abstract #4769)
Session Name: 904. Outcomes Research – Malignant Conditions: Poster III
Session Date: Monday, December 5, 2016
Session Time: 6:00 p.m. – 8:00 p.m. PT

In accordance with ASH (Free ASH Whitepaper) policies, abstracts submitted to the ASH (Free ASH Whitepaper) Annual Meeting are embargoed from the time of submission. To be eligible for presentation at the ASH (Free ASH Whitepaper) Annual Meeting, information contained in the abstract, as well as additional data and information to be presented at the Annual Meeting, may not be made public before the abstract has been presented in connection with the ASH (Free ASH Whitepaper) Annual Meeting.

About Imetelstat

Imetelstat (GRN163L; JNJ-63935937) is a potent and specific inhibitor of telomerase that is administered by intravenous infusion. This first-in-class compound, discovered by Geron, is a specially designed and modified short oligonucleotide, which targets and binds directly with high affinity to the active site of telomerase. Preliminary clinical data suggest imetelstat has disease-modifying activity by inhibiting malignant progenitor cell clones associated with hematologic malignancies in a relatively select manner. Most commonly reported adverse events in imetelstat clinical studies include fatigue, gastrointestinal symptoms and cytopenias. Patients in these studies also experienced elevated liver enzymes, which resolved to normal or baseline in the majority of patients followed after imetelstat treatment was withdrawn. Imetelstat has not been approved for marketing by any regulatory authority.

About the Collaboration with Janssen

On November 13, 2014, Geron entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc., to develop and commercialize imetelstat for oncology, including hematologic myeloid malignancies, and all other human therapeutics uses. Under the terms of the agreement, Geron received an upfront payment of $35 million and is eligible to receive additional payments up to a potential total of $900 million for the achievement of development, regulatory and commercial milestones, as well as royalties on worldwide net sales. All regulatory, development, manufacturing and promotional activities related to imetelstat are being managed through a joint governance structure, with Janssen responsible for these activities.

Genocea Reports Third Quarter 2016 Financial Results

On November 3, 2016 Genocea Biosciences, Inc. (NASDAQ:GNCA), a company developing T cell-directed vaccines and immunotherapies, reported corporate highlights and financial results for the third quarter ended September 30, 2016 (Press release, Genocea Biosciences, NOV 3, 2016, View Source [SID1234516216]). Genocea’s lead clinical candidate, GEN-003, is a T cell-directed immunotherapy for the treatment of genital herpes infections, designed to elicit both a T cell and B cell (antibody) immune response that, if approved, the Company believes would be the first-ever therapeutic vaccine for an infectious disease.

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 achieved an important GEN-003 milestone in the third quarter with the selection of our Phase 3 dose, demonstrating a significant reduction in viral shedding for the third consecutive clinical trial, this time with an improved, Phase 3-ready formulation," said Chip Clark, president and chief executive officer of Genocea. "We also continue to advance our immuno-oncology program and are now focusing all of our early stage research and pre-clinical resources to these efforts. We believe ATLAS enables better cancer vaccine antigen selection than existing methods and that our demonstrated vaccine development expertise can be a further competitive advantage in this exciting space."

Mr. Clark continued: "We expect to maintain our strong momentum this quarter and throughout 2017. In December, we will be hosting our first R&D day as we set the stage for the expected start of the GEN-003 Phase 3 clinical trials in the second half of 2017, including the important Phase 2b six month placebo-controlled clinical efficacy data expected in January 2017. We will also set out in detail our maturing immuno-oncology strategy and neoantigen cancer vaccine development plans."

Recent Business Highlights

GEN-003 – Immunotherapy for treatment of genital herpes expected to enter Phase 3 development in 2H 2017.

September 2016 – data confirm optimal dose for Phase 3 trials; dose response consistent with T cell therapies and with previous GEN-003 clinical trials
October 2016 – IDWeek presentation: GEN-003 induced durable polyfunctional T cells, IgG and neutralizing antibody titers
In September 2016, Genocea announced positive viral shedding data from its ongoing Phase 2b study. The study achieved its primary endpoint, with GEN-003 demonstrating a statistically significant (versus placebo and baseline) 40 percent reduction in the viral shedding rate immediately after dosing in the 60 µg per protein / 50 µg of adjuvant dose group, using a new Phase 3-ready formulation. This result was consistent with a statistically significant (versus placebo and baseline) viral shedding rate reduction of 41 percent at this same dose and time point in the prior Phase 2 trial. Subsequent data from that prior Phase 2 trial demonstrated virologic and clinical efficacy durable through at least one year after dosing.

The 60 µg per protein / 75 µg of adjuvant dose group in the Phase 2b trial reduced the viral shedding rate by 27 percent, a smaller reduction than that observed in the prior trial, and also showed a less acceptable reactogenicity profile than the prior trial. Research has shown that overstimulation of the T cell immune system, as is suggested by this increase in reactogenicity, leads to a loss in efficacy for T cell therapies. We believe the likely driver of this effect is a more potent adjuvant formulation following customary manufacturing process changes to prepare for Phase 3 trials and commercialization.

In October 2016, the Company presented immunogenicity data from its previous Phase 2 trial at IDWeek 2016, the premier annual meeting for healthcare professionals focusing on infectious diseases. These data show that GEN-003 induced antigen-specific polyfunctional T cell responses in immunized subjects, a hallmark of potent T cell immunity. These data also demonstrated that GEN-003 elicited increases in IgG and neutralizing antibody levels above baseline that persisted for one year after the last dose, consistent with viral shedding and clinical symptom reduction seen at 12 months.

Anticipated Milestones and Events

GEN-003

Phase 2b 6-month placebo-controlled clinical efficacy data expected in January 2017
End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) expected in 1Q 2017
Antiviral combination study now planned as part of GEN-003 Phase 3 program
Immuno-oncology collaborations and cancer vaccine strategy

Data showing ATLAS’s differentiated neoantigen selection capabilities from the ongoing partnership with Memorial Sloan Kettering Cancer Center to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 31st Annual Meeting & Associated Programs in National Harbor, Maryland. The poster, #374, entitled Genome-scale neoantigen screening using ATLAS prioritizes candidates for immunotherapy in a non-small cell lung cancer patient will be presented on Saturday November 12, between 11:45 am and 1:00 pm and 6:45 pm and 8 pm ET
Immuno-oncology strategy and neoantigen cancer vaccine development plan update expected at R&D Day in December
Upcoming Events & Presentations

Neoantigen Summit 2016, Boston, November 15
Stifel 2016 Healthcare Conference, New York City, November 16
Piper Jaffray 28th Annual Health Care Conference, New York City, November 30
Virtual R&D Day, week of December 12
Updated Financial Guidance:

Genocea now expects that its existing cash, cash equivalents and marketable securities are sufficient to support its operating expenses and capital expenditure requirements into the first quarter of 2018, without assuming any receipt of proceeds from potential business development partnerships, equity financings or debt drawdowns. This guidance is made on the basis of Genocea’s current operating plans, which include focusing its research activities on immuno-oncology, conducting the antiviral combination study as part of the GEN-003 Phase 3 program and initiating Phase 3 trials for GEN-003 in the second half of 2017.

Third Quarter 2016 Financial Results

Cash Position: Cash, cash equivalents and investments as of September 30, 2016 were $75.5 million compared to $86.0 million as of June 30, 2016.

Research and Development (R&D) Expenses: R&D expenses for the quarter ended September 30, 2016 increased $2.8 million, to $8.8 million, from the same period in 2015. The increase was driven by increases in headcount and related expenses to support the GEN-003 program and higher clinical costs for the ongoing and anticipated GEN-003 trials. Higher personnel and lab-related costs to advance Genocea’s pre-clinical product candidates and develop the ATLAS platform for immuno-oncology also contributed to the increase. These higher R&D costs were partially offset by lower GEN-004 costs due to the Phase 2a trial which was ongoing in the third quarter of 2015 and has since been completed.

General and Administrative (G&A) Expenses: G&A expenses for the quarter ended September 30, 2016 were unchanged at approximately $3.6 million from the same three-month period in 2015.

Net Loss: Net loss was $12.8 million for the third quarter ended September 30, 2016, compared to a net loss of $9.8 million for the same period in 2015.

Genmab Announces Daratumumab and Ofatumumab Data to Be Included in 18 Presentations at American Society of Hematology Annual Meeting (ASH)

On November 3, 2016 Genmab A/S (Nasdaq Copenhagen: GEN) reported that 17 daratumumab abstracts have been accepted for presentation at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place December 3-6 in San Diego, California (Press release, Genmab, NOV 3, 2016, View Source [SID1234516215]). The daratumumab abstracts are sponsored by our collaboration partner Janssen Biotech, Inc., with the exception of one that was based on an investigator-sponsored study, and one that used commercial daratumumab. One abstract on a study with ofatumumab has also been scheduled for a poster presentation at the meeting. All abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org. A list of the key daratumumab abstracts is included below.

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!

"A number of presentations will highlight additional analyses and details from the daratumumab Phase III POLLUX and CASTOR studies that formed the basis of regulatory filings earlier this year. Additional presentations include the Phase I study of the subcutaneous formulation of daratumumab, data on the immuno-modulatory properties of daratumumab, and novel ‘real life’ clinical practice data on daratumumab plus pomalidomide retreatment of refractory multiple myeloma patients. We are excited that daratumumab will be featured in so many presentations at this key hematology conference," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.
Key Daratumumab Abstracts
Efficacy of Daratumumab, Bortezomib, and Dexamethasone Versus Bortezomib and Dexamethasone in Relapsed or Refractory Myeloma Based on Prior Lines of Therapy: Updated Analysis of CASTOR – Oral presentation
Efficacy of Daratumumab, Lenalidomide, and Dexamethasone Versus Lenalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma Patients with 1 to 3 Prior Lines of Therapy: Updated Analysis of POLLUX – Oral presentation
Efficacy of Daratumumab, Lenalidomide and Dexamethasone Versus Lenalidomide and Dexamethasone Alone for Relapsed or Refractory Multiple Myeloma Among Patients with 1 to 3 Prior Lines of Therapy Based on Previous Treatment Exposure: Updated Analysis of POLLUX – Oral presentation
Evaluation of Minimal Residual Disease (MRD) in Relapsed/Refractory Multiple Myeloma (RRMM) Patients Treated with Daratumumab in Combination with Lenalidomide Plus Dexamethasone or Bortezomib Plus Dexamethasone – Oral presentation
Open-Label, Multicenter, Dose Escalation Phase 1b Study to Assess the Subcutaneous Delivery of Daratumumab in Patients (pts) with Relapsed or Refractory Multiple Myeloma (PAVO) – Oral presentation
Clinical Efficacy of Daratumumab, Pomalidomide and Dexamethasone in Relapsed, Refractory Myeloma Patients: Utility of Retreatment with Daratumumab Among Refractory Patients — Oral presentation
This is an independent study, not sponsored by Janssen.
Daratumumab, Bortezomib and Dexamethasone Versus Bortezomib and Dexamethasone Alone for Relapsed or Refractory Multiple Myeloma Based on Prior Treatment Exposure: Updated Efficacy Analysis of CASTOR – Poster presentation
Daratumumab in Combination with Lenalidomide Plus Dexamethasone Induces Clonality Increase and T-Cell Expansion: Results from a Phase 3 Randomized Study (POLLUX) – Poster presentation
High-parameter Mass Cytometry (CyTOF) Evaluation of Relapsed/Refractory Multiple Myeloma (MM) Pts (Pts) Treated with Daratumumab Supports Immune Modulation as a Novel Mechanism of Action – Poster presentation