Idera Pharmaceuticals Reports Third Quarter 2015 Financial Results and Provides Corporate Update

On November 06, 2015 Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel nucleic acid-based therapeutics for oncology and rare diseases, reported its financial and operational results for the third quarter ended September 30, 2015 (Press release, Idera Pharmaceuticals, NOV 6, 2015, View Source;p=RssLanding&cat=news&id=2107923 [SID:1234508068]).

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"We’ve made a great deal of progress at Idera through the first three quarters of 2015, which sets up our company for a number of important catalysts in this last quarter of the year," stated Vincent Milano, Idera’s Chief Executive Officer. "As we announced yesterday, we will be presenting the clinical and safety data analysis from our Phase 1/2 clinical trial of IMO-8400 in Waldenström’s macroglobulinemia at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December. We also are initiating our clinical studies of our TLR9 agonist, IMO-2125 in combination with ipilmumab in patients with metastatic melanoma and IMO-8400 Phase 2 study in dermatomyositis, this quarter. Finally, as detailed in the following text, we are today announcing the first two gene targets for our third generation antisense technology platform."

Continued Milano, "I’m very pleased with the progress that our team at Idera has made over the past months and quarters as we’ve integrated the existing Idera team with many new contributors all the while maintaining the rigor and momentum to ensure our company is positioned for both near and long-term success."

Research and Development Program Updates

IMO-8400 and IMO-2125 are our lead clinical development drug candidates. IMO-8400 is an oligonucleotide-based antagonist of Toll-like receptors (TLRs) 7, 8, and 9. IMO-2125 is an oligonucleotide-based agonist of TLR9.

Toll-like Receptor (TLR) Agonism Program

Immuno-Oncology Program

In June 2015, the company announced that it had entered into a strategic clinical research alliance with MD Anderson Cancer Center to advance the clinical development of intra-tumoral TLR9 agonists in combination with checkpoint inhibitors. The company also announced that it expects to initiate the first trial from the alliance, a Phase 1/2 study to assess the safety and efficacy of intra-tumoral IMO-2125 in combination with ipilimumab in approximately 45 patients with metastatic melanoma. The company is on track to initiate this study in the fourth quarter of 2015. Planning of additional studies as part of the clinical research alliance with MD Anderson Cancer Center is in progress. Additionally, the company presented new preclinical data demonstrating the combination of IMO-2125 and PD1 in cancer models at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) on November 5th in Boston, MA.

Toll-like Receptor (TLR) Antagonism Programs

Genetically Defined Forms of B-cell Lymphoma

Idera’s program in genetically defined forms of B-cell lymphoma is based on pre-clinical studies that have demonstrated, in certain B-cell lymphomas that the presence of the MYD88 L265P oncogenic mutation led to over-activation of TLR7 and TLR9 signaling and that blocking these TLRs with our antagonists promoted tumor cell death.

In the company’s Phase 1/2 study in Waldenstrom’s macroglobulinemia, the targeted number of patients at each of the three dose levels completed assessment through the end of their first cycle of treatment; the dose escalation portion of the study has also been completed. The trial is designed to evaluate IMO-8400’s safety, tolerability and potential clinical activity in patients who have a history of relapse after or failure to respond to prior therapies. Idera announced yesterday that data from this Phase 1/2 clinical study will be presented as a poster at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on Saturday, December 5th from 5:30 PM ET – 7:30 PM ET.

During the third quarter the company also continued to enroll patients into the first of three dose cohorts of our Phase 1/2 clinical trial of IMO-8400 in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are harboring the MYD88 L265P oncogenic mutation. The company currently anticipates that data from this trial will become available in 2016.

Idera previously announced that the U.S. Food and Drug Administration (FDA) granted us orphan drug designation for IMO-8400 for the treatment of Waldenström’s macroglobulinemia and DLBCL.

Rare Disease Programs

The company is planning to initiate clinical development of IMO-8400 for the treatment of rare autoimmune and autoinflammatory diseases. The company has selected dermatomyositis and Duchenne muscular dystrophy (DMD) as the first rare diseases for which we plan to develop IMO-8400. The company selected these indications for development based on the reported increase in TLR expression in these disease states, expression of cytokines indicative of key TLR-mediated pathways, the identification of prospective biomarkers for evaluation in early clinical trials and significant unmet needs. The company plans to progress clinical development in these two indications by initiating a Phase 2 clinical trial in dermatomyositis in the fourth quarter of 2015 and a Phase 2 clinical trial in DMD in 2016.

Third Generation Antisense Platform

Throughout 2015, the company undertook an analysis and prioritization of oncology and rare disease indications for potential development of drug candidates derived from our third generation antisense technology platform. The key considerations in identifying disease indications from our third generation antisense program included: strong evidence that the disease is caused by a specific protein; clear criteria to identify a target patient population; biomarkers for early assessment of clinical proof-of-concept; a targeted therapeutic mechanism for action; and unmet medical need to allow for a well-defined development path to approval and commercial opportunity. As a result of this analysis, the company has selected NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4) as gene targets to advance into IND-enabling activities, which will occur throughout 2016. Potential disease indications include, but are not limited to interstitial cystitis, uveitis and facioscapulohumeral muscular dystrophy (FSHD), respectively. The company intends to describe these programs in more detail during the 2016 JP Morgan Healthcare Conference early next year.

Financial Results

Third Quarter 2015 Results
Net loss applicable to common stockholders for the three months ended September 30, 2015 was $11.4 million, or $ (0.10) per diluted share, compared to a net loss applicable to common stockholders of $9.6 million, or $ (0.11) per diluted share, for the same period in 2014. For the nine month period ended September 30, 2015, the Company’s net loss applicable to common stockholders was $36.6 million, or $ (0.32) per diluted share, compared to a net loss applicable to common stockholders of $27.1 million, or $ (0.33) per diluted share, for the same period in 2014. The company recognized nominal revenue in the third quarter and nine month periods of 2015 and 2014.

Research and development expenses for the three months ended September 30, 2015 totaled $7.5 million compared to $6.7 million for the same period in 2014. For the nine month period ended September 30, 2015, research and development expenses totaled $25.1 million compared to $19.2 million for the same period in 2014.

General and administrative expenses for the three months ended September 30, 2015 totaled $4.0 million compared to $2.9 million for the same period in 2014. For the nine month period ended September 30, 2015, general and administrative expenses totaled $11.7 million compared to $7.6 million for the same period in 2014.

As of September 30, 2015, Idera’s cash, cash equivalents and investments totaled $94.7 million compared to $48.6 million as of December 31, 2014.

Bellicum Announces Data Presentations on Lead Product Candidate BPX-501 and CAR-T and TCR Pipeline Programs at the 57th American Society of Hematology (ASH) Annual Meeting

On November 5, 2015 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported that three abstracts on the Company’s lead product candidate, BPX-501, an adjunct T cell therapy for allogeneic hematopoietic stem cell transplantation, were accepted for poster presentation at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Bellicum Pharmaceuticals, NOV 5, 2015, View Source;p=RssLanding&cat=news&id=2107373 [SID:1234507980]). The Company will also highlight data in an oral presentation from a preclinical study with its BPX-401 controllable CAR-T (CIDeCAR) product candidate for the treatment of B cell malignancies. The meeting will be held in Orlando, Florida December 5-8, 2015.

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Investor/Analyst Luncheon

Bellicum will also host an investor and analyst luncheon on Monday, December 7, 2015 from 12:15 – 1:15 p.m. EST at the Hyatt Regency Orlando. Management and select key opinion leaders, including lead Principal Investigator, Professor Francesco Locatelli, M.D., will review the BPX-501 Phase 1/2 clinical study data in pediatric patients with hematologic disorders. The luncheon will be webcast live and may be accessed from the News & Events section of the Bellicum website. An archived version of the webcast will be available for replay for at least two weeks following the event.

Full List of ASH (Free ASH Whitepaper) Presentations on Bellicum Programs

BPX-501:

Poster Presentation: "Clinical Outcome after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Results of a Phase I-II Trial"

Abstract Number: 1931
Session Name: 722. Clinical Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster I
Date: Saturday, December 5, 2015
Presentation Time: 5:30 PM – 7:30 PM EST

Poster Presentation: "Immune Reconstitution after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Phenotypic and Functional Results of a Phase I-II Trial"

Abstract Number: 3093
Session Name: 703. Adoptive Immunotherapy: Poster II
Date: Sunday, December 6, 2015
Presentation Time: 6:00 PM – 8:00 PM EST

Poster Presentation: "BPX-501 Cells (donor T cells transduced with iC9 suicide gene) Are Able to Clear Life-Threatening Viral Infections in Children with Primary Immune Deficiencies Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT)"

Abstract Number: 4299
Session Name: 703. Adoptive Immunotherapy: Poster III
Date: Monday, December 7, 2015
Presentation Time: 6:00 PM – 8:00 PM EST

Additional data on more BPX-501 patients to be presented at ASH (Free ASH Whitepaper).

BPX-401:

Oral Presentation: "Expression of MyD88/CD40 Drives In Vivo Activation and Proliferation of Chimeric Antigen Receptor-Modified T Cells That Can be Effectively Regulated By Inducible Caspase-9"

Abstract Number: 851
Session Name: 703. Adoptive Immunotherapy: Preclinical Studies
Date: Monday, December 7, 2015
Presentation Time: 5:30 PM EST

BPX-601:

Poster Presentation: "Inducible MyD88/CD40 Allows Rimiducid-Dependent Activation to Control Proliferation and Survival of Chimeric Antigen Receptor-Modified T Cells"

Abstract Number: 4295
Session Name: 703. Adoptive Immunotherapy: Poster III
Date: Monday, December 7, 2015
Presentation Time: 6:00 PM – 8:00 PM EST

Enhanced TCRs:

Poster Presentation: "Inducible MyD88/CD40 Enhances Proliferation and Survival of PRAME-Specific TCR-Engineered T Cells and Increases Anti-Tumor Effects in Myeloma"

Abstract Number: 1886
Session Name: 703. Adoptive Immunotherapy: Poster I
Date: Saturday, December 5, 2015
Presentation Time: 5:30 PM – 7:30 PM EST

Bellicum Program Licensed from Leiden University Medical Center:

Poster Presentation: "T Cell Receptor Gene Therapy Targeting the Intracellular Transcription Factor Bob1 for the Treatment of Multiple Myeloma and Other B Cell Malignancies"

Abstract Number: 3002
Session Name: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster II
Date: Sunday, December 6, 2015
Presentation Time: 6:00 PM – 8:00 PM EST

Stemline Therapeutics Announces Five Presentations at the American Society of Hematology (ASH) Annual Meeting; SL-401 Poster Will Include Clinical Data From Lead-In and Ongoing Expansion Stages of BPDCN Pivotal Trial

On November 5, 2015 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that it will present results from clinical and preclinical studies, including updated data from its ongoing SL-401 pivotal trial, at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held December 5-8, 2015 at the Orange County Convention Center in Orlando, FL (Press release, Stemline Therapeutics, NOV 5, 2015, View Source [SID:1234507996]).

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Five poster presentations, including one covering clinical data updates from the lead-in and ongoing expansion stages of the SL-401 pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), will be featured. Three preclinical presentations will highlight data around SL-401’s potential in additional indications as both a single agent as well as in combination. In addition, a poster demonstrating broad preclinical anti-cancer activity will be presented around SL-801, Stemline’s novel oral XPO1 inhibitor being advanced toward the clinic across multiple hematologic and solid tumor indications.

Ivan Bergstein, M.D., Stemline’s CEO, commented, "At the upcoming ASH (Free ASH Whitepaper) meeting, we plan to share updated clinical data from the SL-401 pivotal trial in BPDCN. Data from the abstract demonstrate that SL-401 offers a manageable safety profile over multiple cycles with high response rates. We have also gained important insights into the dosing and administration schedule which has enabled us to improve the safety profile of SL-401 in BPDCN." Dr. Bergstein continued, "We also plan to present additional data on BPDCN patients from both the lead-in as well as initial expansion stage of the ongoing pivotal trial, including patients enrolled since abstract submission." Dr. Bergstein concluded, "In addition, we believe that SL-401’s ability to induce responses quickly coupled with a manageable safety profile following multi-cycle administration may bode well not only for our single agent approach in relapsed/refractory BPDCN but also in future combination studies in larger indications, such as myeloma."

About SL-401 and SL-801

SL-401 is a targeted therapy directed to the interleukin-3 receptor (IL-3R) present on cancer stem cells (CSCs) and tumor bulk of BPDCN, AML, and other hematologic cancers. Stemline is evaluating SL-401 in multiple clinical programs, including an ongoing pivotal trial in BPDCN, as well as trials in additional hematological cancers. SL-801 is a novel oral, small molecule reversible XPO1 inhibitor that is expected to enter clinical development in early 2016 for both solid and hematologic cancers.

Five abstracts were accepted for the 2015 ASH (Free ASH Whitepaper) meeting, and the details on the presentations are listed below and available on the ASH (Free ASH Whitepaper) conference website:

Lead-in Stage Results of a Pivotal Trial of SL-401, an Interleukin-3 Receptor (IL-3R) Targeting Biologic, in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or Acute Myeloid Leukemia (AML)
Lead Author: Marina Konopleva, MD, PhD
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX

A Novel Agent SL-401 Triggers Anti-Myeloma Activity by Targeting Plasmacytoid Dendritic Cells: Implications for a Novel Immune-associated Mechanism
Lead Author: Arghya Ray, Ph.D.
The LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Department of Medical Oncology, Dana Farber Cancer Institute, Harvard Medical School, Boston, MA

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Patient-Derived Xenografts Are Faithful Genomic and Phenotypic Models of Primary Leukemia and Respond to the IL-3R targeting agent SL-401 In Vivo
Lead Author: Amanda Christie, B.A.
Department of Hematologic Oncology, Dana Farber Cancer Institute, Harvard Medical School, Boston, MA

CD123 immunostaining in systemic mastocytosis: differential expression in disease subgroups and potential prognostic value
Lead Author: Animesh Pardanani, MBBS, Ph.D.
Department of Hematology, Mayo Clinic College of Medicine, Rochester, MN

SL-801, a novel, reversible inhibitor of Exportin-1 (XPO1) / Chromosome Region Maintenance-1 (CRM1) with broad and potent anti-cancer activity
Lead Author: Janice Chen, Ph.D.
Stemline Therapeutics, Inc., New York, NY

10-Q – Quarterly report [Sections 13 or 15(d)]

(Filing, 10-Q, Celldex Therapeutics, NOV 5, 2015, View Source [SID:1234508041])

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MEI Pharma Highlights New Data from Phase II Clinical Studies of Pracinostat in Acute Myeloid Leukemia and Myelodysplastic Syndrome

On November 5, 2015 MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported the publication of new data from the Company’s Phase II clinical studies of the investigational drug Pracinostat in patients with previously untreated myelodysplastic syndrome (MDS) and elderly patients with acute myeloid leukemia (AML) (Press release, MEI Pharma, NOV 5, 2015, View Source [SID:1234508012]). Results from these studies were recently selected for oral presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando on December 7, 2015. The abstracts are now available on the ASH (Free ASH Whitepaper) website at www.hematology.org.

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(Logo – http: /photos.prnewswire.com/prnh/20140805/133834)

"The data contained within the abstracts released this morning point to certain trends worth highlighting," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "Since the unblinding of our randomized study in front-line MDS, we have learned that the combination of Pracinostat and azacitidine resulted in a high rate of discontinuations due to adverse events compared to azacitidine alone. These discontinuations occurred predominantly within the first two cycles of treatment and often before a response assessment could be performed, leading to a higher complete response (CR) rate overall with azacitidine alone. However, exploratory sensitivity analyses among patients who were able to tolerate treatment for at least four cycles (n=54) suggest that patients treated with Pracinostat plus azacitidine appear to derive benefit compared to azacitidine alone, with hazard ratios for progression progression-free survival (0.37), event-free survival (0.33) and overall survival (0.59) all favoring the Pracinostat plus azacitidine arm.

"The data from our open-label study in elderly patients with newly diagnosed AML," continued Dr. Gold, "demonstrate that many patients are achieving responses within the first two cycles and continue to improve with ongoing therapy, with fewer discontinuations due to adverse events than in our MDS study. Overall, 54% of patients (27 of 50) have achieved a clinical response with 42% (21 of 50) achieving a CR. The 60-day mortality rate in the study is 10% (5 of 50) and the one-year survival rate is estimated at 60%. All of these data points compare favorably to a recent study of azacitidine alone in this population1. Median overall survival, the most important measure in determining the development path forward for this combination, still has not been reached. We will continue to follow these patients and look forward to the presentation of updated overall survival data at ASH (Free ASH Whitepaper) next month."

The data contained within the abstracts listed below are as of the ASH (Free ASH Whitepaper) submission deadline on August 4, 2015. In accordance with ASH (Free ASH Whitepaper) policies, information that goes beyond that which is contained within these abstracts is embargoed until their presentation on December 7, 2015.

Title: Final Results from a Phase 2 Study of Pracinostat in Combination with Azacitidine in Elderly Patients with Acute Myeloid Leukemia (Abstract #453)
Session Name: 613. Acute Myeloid Leukemia: Clinical Studies: Advances in Therapy
Session Date: Monday, December 7, 2015
Session Time: 7:00 AM – 8:30 AM
Presentation Time: 7:30 AM

Title: A Randomized, Placebo-Controlled, Phase II Study of Pracinostat in Combination with Azacitidine in Patients with Previously Untreated Myelodysplastic Syndrome (Abstract #911)
Session Name: 637. Myelodysplastic Syndromes – Clinical Studies I
Session Date: Monday, December 7, 2015
Session Time: 6:15 PM – 7:45 PM
Presentation Time: 7:15 PM

About Pracinostat

Pracinostat is an orally available inhibitor of a group of enzymes called histone deacetylases, or HDACs. HDACs belong to a larger set of proteins collectively known as epigenetic regulators that can alter gene expression by chemically modifying DNA or its associated chromosomal proteins. Abnormal activity of these regulators is believed to play an important role in cancer and other diseases. Pracinostat has been tested in multiple Phase I and Phase II clinical studies in advanced hematologic diseases and solid tumor indications. The results of these studies suggest that Pracinostat has potential best-in-class pharmacokinetic properties when compared to other oral HDAC inhibitors, with side effects often associated with drugs of this class, including fatigue and myelofibrosis. Pracinostat has not been approved for commercial distribution in the U.S.