Reappraising the Approach to PV and ET

Description of recurrent genetic abnormalities in driver genes, a better appreciation of the key diagnostic role of bone marrow features, results of large epidemiologic studies, and landmark controlled clinical trials have resulted in a reappraisal of the approach to polycythemia vera and essential thrombocythemia, authors noted in a recent review.

Read more

The Effects of Myeloproliferative Neoplasm Symptoms on Quality of Life

A secondary analysis being presented at the 59th Annual Meeting of the American Society of Hematology in Atlanta, Georgia, found that all individual symptoms of myeloproliferative neoplasms (MPNs) correlate with quality of life (QoL).
“Patients with MPNs are faced with high disease-related symptom burden and QoL decrements,” wrote the authors.
Previous study results have shown that symptom burden measured from the MPN Symptom Assessment Form (MPN-SAF) Total Symptom Score has a strong correlation with QoL measured by the Global Health Status/QoL (GHS/QoL) scale of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30). However, according to the authors, an analysis of predictors of QoL in this population has not been performed.

Read more

Go to MPN Quality of Life Survey


PharmaEssentia Announces Favorable Two-year Results of Ropeginterferon Alfa-2b in PV at ASH

 At 24 months Ropeginterferon alfa-2b:

Demonstrated superiority over best available treatment
Achieved high rates and durable clinical and hematological response
Confirmed favorable safety and tolerability profile beyond 24 months
Further demonstrated disease modifying capability

PharmaEssentia is currently working with the U.S. FDA for submission of a biologics license application (BLA) for Ropeginterferon alfa-2b for Polycythemia Vera (PV) in the U.S.

AOP Orphan’s submission for marketing authorization of Ropeginterferon alfa-2b in the EU is currently under review by EMA

WALTHAM, Mass.Dec. 10, 2017 /PRNewswire/ — PharmaEssentia USA, a subsidiary of PharmaEssentia Corporation (Taipei Exchange: 6446), today announced the latest follow-up results of Ropeginterferon alfa-2b from the ongoing, long-term, follow-up trial CONTINUATION-PV (CONTI-PV) for patients with Polycythemia Vera presented during an oral presentation at ASH 2017. The CONTI-PV trial is being conducted by AOP Orphan Pharmaceuticals AG (AOP Orphan) in Europe.

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon. It is administered once every two weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide. PharmaEssentia discovered and manufactures Ropeginterferon alfa-2b and has exclusively licensed the rights for the novel molecule in the field of Myeloproliferative Neoplasms (MPNs) to AOP Orphan for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

Read more

View Current MPN Clinical Trials

The MPN Yoga Study: A Patient’s Story

MPN Patient Amy Wang Manning, Portland, Oregon

By Amy Wang Manning

As I typed one day at work this past spring, my left pinkie suddenly didn’t feel right. A moment later, my left thumb curled under; I couldn’t straighten it except by pulling it with my right hand. Then my entire left hand stiffened into a useless claw. And a tingling sensation was spreading rapidly up my left arm and to my shoulder. I told my boss I needed medical attention. Soon I was in a hospital emergency room, undergoing an MRI.
I didn’t have a stroke that March day. But several weeks later, a hematologist oncologist gave me a diagnosis I’d never heard of: essential thrombocytosis (ET), based on the clot I’d experienced, along with a platelet count of over 1 million and a positive test for the JAK2 mutation. He said I’d probably had ET for as long as 20 years. Suddenly, a lot of health symptoms and setbacks that I’d experienced over that time period made sense.
While I researched essential thrombocytosis online, I stumbled across a call for participants in a pilot study at Arizona State University. The researchers wanted to investigate whether patients with essential thrombocytosis and other myeloproliferative neoplasms could find some relief from symptoms such as pain, fatigue and insomnia by using a mobile app for guided meditation.
While I hadn’t experienced much pain, I’d been having bouts of fatigue and I’d struggled for years with insomnia, which seemed to be worsening. I had practiced yoga and found it helpful in managing my emotional, mental and physical health, so meditation seemed appealing. It certainly couldn’t hurt. And the eight-week study wasn’t asking much of participants: Fill out a few questionnaires, keep a daily sleep log, and wear a Fitbit to track my daily activity. I applied to the study and was accepted. The daily meditation sessions turned out to be very reasonable, just 10 minutes a day.
Now that the study’s finished, I feel calmer and more able to handle whatever comes my way with this disease. I continue to meditate with the app I tested. If nothing else, I am now better equipped to take a deep breath, let go of what I cannot control, and just focus on the moment – this moment, in which I am still here, living.

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:

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