ASH Abstract: Imetelstat Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells

1654 Imetelstat, a Telomerase Inhibitor, Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells

Treatment of myelofibrosis (MF) patients with imetelstat (Imet), a telomerase inhibitor, has been reported to lead to clinical, morphologic and molecular remissions in a subset of patients (Tefferi A, et al. N Engl J Med. 2015; 373:908), suggesting that Imet has disease-modifying activity. The precise mechanism by which Imet induces such responses has however not been reported. In this study, we investigated the effects of Imet on MF hematopoietic stem/progenitor cells (HSC)/(HPC) to address this question.

Read more

Learn More about MF Clinical Trials

Impact BioMedicines Presents at ASH on Fedratinib

Impact Biomedicines Presents Analysis at the 2017 ASH Annual Meeting Suggesting that Fedratinib Did Not Increase Wernicke Encephalopathy Risk in Phase 2 and 3 Myelofibrosis Clinical Trials

SAN DIEGO–(BUSINESS WIRE)–Impact Biomedicines today presented a case review on fedratinib, a selective oral small molecule JAK2 kinase inhibitor that is being developed for the treatment of myelofibrosis (MF) and polycythemia vera (PV), in a poster session at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.

The poster titled “Case Series of Potential Wernicke Encephalopathy in Patients treated with Fedratinib,” demonstrated that patients treated with fedratinib in clinical trials did not experience a decrease in thiamine levels, and the prevalence of Wernicke Encephalopathy (WE) in the trials was less than originally perceived for patients with myeloproliferative neoplasms.

Read more

Learn more about clinical trials

Incyte Reports Four Year Phase 3 Data Analysis Shows Durability of Response of Jakafi (ruxolitinib) in Patients with PV

Incyte Corporation (NASDAQ: INCY) today announced new 208-week (4-year) follow-up data from the ongoing, global, multi-center, open-label Phase 3 RESPONSE study of Jakafi® (ruxolitinib) comparing the efficacy and safety of Jakafi with best available therapy (BAT) in patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU). The pre-planned data analysis showed a durable primary response to Jakafi in patients with PV who are resistant to or intolerant of HU and the overall safety profile for Jakafi remained consistent with previously reported 80-week RESPONSE data.1 The results were shared in an oral presentation today at the 59th American Society of Hematology (ASH) Annual Meeting 2017 in Atlanta, Georgia.

“With 30 months of additional follow-up, the four-year RESPONSE data analysis presented today at ASH further reinforces the potential of Jakafi as a long-term option for patients with PV,” said Peg Squier, M.D., Ph.D., Head of U.S. Medical Affairs at Incyte. “Given the few treatment options available to treat this chronic and progressive blood cancer, these long-term safety and efficacy data are meaningful to patients with uncontrolled PV.”

Read more

Go to Clinical Trials

Italfarmaco Announces Givinostat Clinical Trial Presentations at the 59th American Society of Hematology (ASH) Annual Meeting

MILAN–(BUSINESS WIRE)–Italfarmaco Group, a specialty pharmaceutical company, today announced both an oral and a poster presentation at the 59th American Society of Hematology (ASH) Meeting & Exposition held in Atlanta, Georgia from December 9 – 12, 2017. The presentations cover data from Italfarmaco’s Phase II development program for its proprietary compound Givinostat, in development to treat Polycythemia Vera, a rare blood disease with orphan drug designation. Italfarmaco will announce the results through a press release following the presentations.

Oral Presentation

Title: A Two-Part Study of Givinostat in Patients with Polycythemia Vera: The Maximum Tolerated Dose Selection and the Proof of Concept Final Results

Presenter: Prof. Alessandro Rambaldi

Session Name: 634. Myeloproliferative Syndromes: Clinical: Phase I/II Trials of Novel Agents in MPNs

Date & Time: Saturday, December 9, 2017: 4:00-4:15 PM EST

Location: Bldg C, Lvl 2, C208-C210 (Georgia World Congress Center)

Link to the ASH conference abstract: https://ash.confex.com/ash/2017/webprogram/Paper100916.html

Go To MPN Clinical Trials

ASH Presentation-Personalized Medicine in Blood Cancers

Foundation Medicine and Collaborators to Present New Data at the American Society of Hematology (ASH) Annual Meeting that Supports Use of FoundationOne®Heme to Advance Personalized Medicine in Blood Cancers

— New data demonstrate the value of comprehensive genomic profiling for informing clinical care and guiding use of targeted therapies, autologous stem cell transplantation and immunotherapy —

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Foundation Medicine, Inc. (NASDAQ:FMI) today announced that new data generated with FoundationOne®Heme, its comprehensive genomic profiling (CGP) assay for hematologic malignancies and sarcomas, will be presented at the American Society of Hematology (ASH) Annual Meeting. Data from a broad range of blood cancers, including acute myeloid leukemia (AML), myeloproliferative neoplasms (MPN), and non-Hodgkin lymphoma (NHL), including primary central nervous system lymphoma (PCL), demonstrate the value of integrating FoundationOneHeme into clinical care. The data presented is expected to demonstrate the potential for CGP to improve disease classification, to offer personalized prognostic information and to support therapeutic treatment decision making by informing treating physicians about the use of novel treatment options, including cancer immunotherapies.

Read more

Impact Biomedicines Closes First Tranche of Oberland Capital Financing Following FDA Meeting

Management team expands to accelerate fedratinib global manufacturing and business operations

SAN DIEGO — December 1, 2017— Impact Biomedicines (“Impact”) today announced that it has achieved the first milestone in the Company’s previously disclosed $90 million financing with Oberland Capital, triggering the closing on the first tranche of $20 million. Impact also announced the expansion of its management team to include Randy Adams as Senior Vice President of Commercial Operations and Jeff Barker as Senior Vice President of Global Technical Operations.

“The first tranche of this financing follows a positive meeting with the U.S. Food and Drug Administration (FDA) bringing this much needed potential treatment option for myelofibrosis closer to patients in need,” said John Hood, Ph.D., Chief Executive Officer of Impact Biomedicines. “With Oberland’s financial support, we have made some important investments to ensure that we are well-staffed and prepared for U.S. commercialization.”

Read more

ASH Abstract: MPNs and Depressive Symptoms, Diagnosis, and Associations

Patients with a myeloproliferative neoplasm (MPN) diagnosis experience a high systemic symptom burden and a spectrum of physical, financial, mental and emotional stressors. The impact of mood disturbances in MPN patient is not well understood. It was recently identified by our study team that symptoms of depression coexist with the prevalent and debilitating symptom of fatigue in patients with MPNs. The purpose of this study is to comprehensively describe the experience of depressive symptoms in MPN patients.

Methods: A 70-item internet based national survey regarding fatigue and mood symptoms was developed by MPN investigators and patient/advocates and refined by the Mayo Clinic Survey Research Center. The Patient Health Questionnaire-2 (PHQ) was completed by all respondents and utilized to assess symptoms of depression. Survey responses were compared between those who endorsed having current symptoms of depression (defined as a PHQ-2 score ≥ 3) and those without symptoms of depression (defined as a PHQ-2 score < 3).

Read more

Impact Biomedicines to Present Retrospective Data on Fedratinib at the 2017 ASH Annual Meeting

SAN DIEGO — November 27, 2017 — Impact Biomedicines today announced that retrospective data on fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV), will be presented in a poster at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.
The details of the presentation are as follows:
Session: 634 – Myeloproliferative Syndromes: Clinical: Poster III
Poster Title: Case Series of Potential Wernicke’s Encephalopathy in Patients treated with Fedratinib
Presenter: John Hood, Ph.D.
Date: Monday, December 11, 2017
Presentation time: 6:00-8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
About Impact Biomedicines
Impact Biomedicines (“Impact”) is pioneering the development of life changing treatments for patients with complex cancers. The Company’s pipeline is centered around fedratinib, a potent and highly selective oral small molecule, JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV).

ASH: 1663 Germline ERBB2 Variants Associate with MPNs

Background: The myeloproliferative neoplasms (MPN) and myelodysplastic syndrome (MDS) are usually sporadic diseases, however, familial cases are well-described. Approximately 10% of MPN cases display familial clustering, and there is a 5-7 fold increased risk of developing an MPN among first degree relatives of MPN patients. While familial MDS appears to be less common, identification of multiple predisposition genes has led to the incorporation of this entity into the most recent WHO classification of myeloid neoplasms. Identification of predisposing germline mutations in the myeloid malignancies has led to a better understanding of the pathophysiology of these diseases and improved clinical care. However, many familial MDS and MPN cases exist in which the inherited genetic lesion is unknown. Our recent investigation of a multigenerational family with inherited MDS/AML marked by erythroid hyperplasia identified a missense variant in ERBB3that co-segregated with the disease phenotype (1). In this study, we investigated a kindred with an autosomal dominant familial cancer syndrome characterized by both MPN and solid tumor malignancies.
Program: Oral and Poster Abstracts
Session: 635. Myeloproliferative Syndromes: Basic Science: Poster I
Saturday, December 9, 2017, 5:30 PM-7:30 PM
Bldg A, Lvl 1, Hall A2 (Georgia World Congress Center)

Evan M Braunstein, MD, PhD1, Donna Marie Williams, PhD1*, Igor Makhlin, MD1, Aparna Pallavajjala2*, Christopher D Gocke, MD2*, Nara Sobreira, MD3*, Kala Visvanathan, MD4*, Linda Resar, MD5, Jerry L. Spivak, MD1 and Alison R. Moliterno, MD1

1Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD
2Department of Pathology, Johns Hopkins School of Medicine, Baltimore, MD
3Department Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD
4Medical Oncology and Epidemiology, Johns Hopkins University, Baltimore, MD
5Division of Hematology, Department of Medicine, Johns Hopkins University SOM, Baltimore, MD

Read more