PharmaEssentia Updates Progress of Ropeginterferon Alfa-2b Clinical Program

  • 36 month analysis of ropeginterferon alfa-2b (Ropeg) phase III clinical data in Polycythemia Vera (PV) demonstrates superiority across three key aspects of disease control, including complete hematological response (CHR), disease burden, and molecular response
  • A long-term development partner of Ropeg, AOP Orphan’s submission for marketing authorization of ropeginterferon alfa-2b in the EU is in the final stage of European Medicines Agency (EMA) regulatory review
  • PharmaEssentia is continuing to discuss with the US Food and Drug Administration (FDA) the best path forward to make ropeginterferon alfa-2b available to PV patients in the US
  • PharmaEssentia is planning to initiate a global clinical development program of ropeginterferon alfa-2b in Essential Thrombocythemia (ET)

Ropeginterferon alfa-2b maintains efficacy and safety profile in PV

Ropeginterferon alfa-2b is a novel, single isomer long-acting pegylated interferon in clinical development for treatment of patients with PV. At the 2018 American Society of Hematology (ASH) Annual Meeting, Prof. Heinz Gisslinger from the Medical University of Vienna, Austria presented the 36 month update of ropeginterferon alfa-2b in patients with PV (http://www.bloodjournal.org/content/132/Suppl_1/579).

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Go to MPN Clinical Trials

Making the Most of Social Media

Dr. Aaron Gerds and Dr. Ruben Mesa present on the use of social media at ASH 2018

After attending the ASH event in San Diego it was clear how the use of social media has influenced the medical community. It was even the subject of one session at ASH, chaired by MPN specialist, Dr. Aaron Gerds, Cleveland Clinic Taussig Cancer Institute. Both doctors and patients are struggling with the wise-use of social media. While Facebook appears to be the preferred source of information and support for MPN patients, Twitter is the primary tool for doctors and researchers to communicate on a broad scale. It creates a quick and easy way for doctors to reach out to peers and patients alike, and include links to valuable information that patients may not otherwise have access to without extensive google searchers. 

Dr. Gerds explained his reasoning for signing onto Twitter, ”as a specialist in MPNs I follow a group of hematologists, patients, and advocacy groups that tweet with the hashtag #MPNSM. The members of this community are all interested in new developments in our field, and we’re all there to start discussions about what these developments might mean for our patients and practices.” (read more about Dr. Gerds work on this subject) *

Regardless of your social media source, it is wise to proceed with caution. Not everything you read applies to your particular situation, diagnosis, symptoms or treatments. In response to feedback from concerned patients regarding the use of Facebook for medical information, MPN Advocacy and Education International talked with Dr. Naveen Pemmaraju, MD Anderson Cancer Center, who has researched the use of social media as a medical professional and offers his advice on how to interact responsibly with social media. 

Click here to view Dr. Pemmaraju’s Perspective on the use of Social Media

 

*Most items posted on Twitter include a hashtag (#), which is a way of sorting and filing information. #MPNSM, myeloproliferative neoplasm social media, is included in most significant MPN posts. Take a moment to sign up and search for #mpnsm to get an understanding of the conversations that go on under this

 

2018 ASH Papers & Abstracts

Click on the titles below to view the full summary of MPN-related abstracts from ASH

Researchers Identify Genetic Prognostic Predictors in MPNs

Survey of Integrative Medicine in MPNs (The SIMM Study-2)

Males with MPN Have Inferior Survival Compared to Females

MPNs in Patients below 25 Years Old at Diagnosis: A Retrospective International Cooperative Work

Disease Characteristics of Minority Patient Populations with

Polycythemia Vera: An Analysis from the Reveal Study

Pregnancy Outcomes in Patients with MPNs: A Systematic Review and Meta Analysis

 

Myelofibrosis:  Full listing of MF abstracts (scroll down to view all)

Essential Thrombocythemia: Full list of ET abstracts (scroll down to view all)

Polycythemia Vera:  Full list of PV abstracts (scroll down to view all)

Prevalence of Pulmonary Hypertension in Patients With MPN Lower Than Previously Reported

Pulmonary hypertension (PH) has been reported to be associated with myeloproliferative neoplasms (MPN) in 5% to 48% of MPN patients. Now, authors of the largest PH study in patients with Philadelphia chromosome-negative MPN have concluded that the prevalence of PH is lower than has been previously reported.

Pulmonary hypertension (PH) has been reported to be associated with myeloproliferative neoplasms (MPN) in 5% to 48% of MPN patients—a large variability likely related to the small cohorts of patients previously studied. Based on these earlier studies, both the European Society of Cardiology (ESC) and the European Respiratory Society recognized MPN as a specific cause of PH of unclear and/or multifactorial mechanisms. Now, authors of the largest PH study in patients with Philadelphia chromosome-negative MPN (Ph-MPN) have concluded that the prevalence of PH is lower (3.8%) than has been previously reported.

Several risk factors have been identified for developing PH among patients with Ph-MPN: obstruction of pulmonary vessels by circulating megakaryocytes, smooth muscle hyperplasia due to platelet-derived growth factor, and altered angiogenic status. There is an increased risk for thromboembolic events, and thus a high risk of pulmonary embolism leading to PH. Extramedullary hematopoiesis, a well-known complication of progressive massive fibrosis that involves the presence of circulating marrow progenitors in the lung parenchyma, may also cause PH.

The current prospective, observational cohort study, from March 2015 through June 2016, followed 158 patients with Ph-MPN (median age, 65 years; 50.6% were female; all were white; and the median duration of Ph-MPN at inclusion was 4 years). About half the subjects had polycythemia vera (PV), 34% had essential thrombocytosis (ET), 11% had primary myelofibrosis, 3% had post-ET myelofibrosis, and 2% had post-PV myelofibrosis. Transthoracic echocardiography (TTE) was performed on all 158 patients included in the study. When TTE results were abnormal, further investigations were performed according to current ESC guidelines. The primary study endpoint was the frequency of PH; the secondary endpoint was cause of PH. Once the etiology behind PH was established, no further tests were performed as part of the study.

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CTI BioPharma Provides Program Update on Pacritinib

CTI BioPharma Provides Program Update Following Regulatory Feedback on Pacritinib Development from the European Medicines Agency

– European regulatory opinion on marketing authorization application (MAA) for pacritinib now expected in the first quarter of 2019 –

SEATTLE, Nov. 26, 2018 /PRNewswire/ — CTI BioPharma Corp. (NASDAQ: CTIC) today announced that the Company has received a second round of questions related to the Day 180 List of Outstanding Issues, for the marketing authorization application (MAA) for pacritinib, from the European Medicines Agency (EMA). The Company plans to submit responses to the EMA, which will include data from the ongoing open label PAC203 trial, by the end of the year. In addition, the Company is preparing for an Oral Explanation meeting before the Committee for Medicinal Products for Human Use (CHMP). A decision by CHMP on the MAA is expected in the first quarter of 2019.

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Emerging Agents Propel Progress in MPN Paradigm

Onclive

Dr. Stephen Oh, MD, PhD

Ruxolitinib (Jakafi) has been an integral aspect of care for patients with myelofibrosis and polycythemia vera (PV), but updates in the development of more selective JAK inhibitors such as momelotinib, pacritinib, and fedratinib are showing encouraging progress in the treatment paradigm, explained Stephen Oh, MD, PhD.

“At this point, it’s impossible to say that momelotinib, pacritinib, or fedratinib is really a frontrunner. Rather, I’d say that they potentially offer slight distinctions between each other and ruxolitinib,” said Oh. “I could envision a scenario where if all of these agents were available, there could be specific niches for each of these particular drugs for specific types of patients.”

In 2011, the JAK1/2 inhibitor ruxolitinib (Jakafi) was approved by the FDA for patients with intermediate- and high-risk myelofibrosis. Three years later, it was approved for patients with PV who are intolerant or resistant to hydroxyurea. Although it shows significant symptom reduction in patients with PV, other agents need to be added to the armamentarium.

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Learn more about Ruxolitinib

 

Prevalence of Pulmonary Hypertension in Patients With MPN Lower Than Previously Reported

Pulmonary hypertension (PH) has been reported to be associated with myeloproliferative neoplasms (MPN) in 5% to 48% of MPN patients. Now, authors of the largest PH study in patients with Philadelphia chromosome-negative MPN have concluded that the prevalence of PH is lower than has been previously reported.

Pulmonary hypertension (PH) has been reported to be associated with myeloproliferative neoplasms (MPN) in 5% to 48% of MPN patients—a large variability likely related to the small cohorts of patients previously studied. Based on these earlier studies, both the European Society of Cardiology (ESC) and the European Respiratory Society recognized MPN as a specific cause of PH of unclear and/or multifactorial mechanisms. Now, authors of the largest PH study in patients with Philadelphia chromosome-negative MPN (Ph-MPN) have concluded that the prevalence of PH is lower (3.8%) than has been previously reported.

Several risk factors have been identified for developing PH among patients with Ph-MPN: obstruction of pulmonary vessels by circulating megakaryocytes, smooth muscle hyperplasia due to platelet-derived growth factor, and altered angiogenic status. There is an increased risk for thromboembolic events, and thus a high risk of pulmonary embolism leading to PH. Extramedullary hematopoiesis, a well-known complication of progressive massive fibrosis that involves the presence of circulating marrow progenitors in the lung parenchyma, may also cause PH.

Read more

 

Dose-Escalation Mitigates Risk of Grade 3/4 Adverse Events With Ruxolitinib for MF

Dose escalation may combat worsening anemia during early ruxolitinib therapy in patients with myelofibrosis, according to a recent study published in the Journal of Hematology and Oncology. Ruxolitinib improves splenomegaly and alleviates the symptoms of intermediate-2 or high-risk myelofibrosis. However, its use is associated with an increased risk of developing grade 3/4 anemia and/or thrombocytopenia, requiring additional dose reductions or transfusions.

The authors of the study aimed to preserve clinical benefit, but reduce hematologic risk early during treatment using dose escalation. The study was an open-label phase 2 study of 45 patients with myelofibrosis, 68.9% of whom had a Dynamic International Prognostic Scoring System score of 1 to 2, indicating intermediate disease risk. Patients received ruxolitinib 10 mg twice daily with increases in increments of 5 mg at 12 weeks and 18 weeks for a maximum dose of 20 mg. Symptom severity was assessed using the Myelofibrosis Symptom Assessment Form Total Symptom Score.

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Learn more about MF

 

Therapeutic Phlebotomy, Hydroxyurea Improve Overall Survival in Geriatric Polycythemia Vera

Treatments with therapeutic phlebotomy and hydroxyurea (HU) are associated with improved overall survival (OS) and decreased risk of thrombosis in older patients with polycythemia vera (PV), according to a recent retrospective cohort study published in Blood Advances.1

PV, a myeloproliferative neoplasm with a median age at diagnosis of 65 years, is associated with reduced OS and is characterized by an increased risk of thrombosis. Although current guidelines recommend therapeutic phlebotomy to maintain hematocrit below 45% for all patients with PV and additional cytoreductive therapy (such as HU or interferon, which can directly reduce counts of red cells, white cells, and platelets) for high-risk patients with PV,2 little has been known about the impact of these therapies in the real-world treatment setting. In addition, the study’s authors, led by Nikolai A. Podoltsev, MD, PhD, of Yale University, concluded that phlebotomy and HU were underused in their sample of older PV patients.

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