Recruiting: New Phase 2 Clinical Trial for MF Patients on Ruxolitinib

People with myelofibrosis who are currently receiving ruxolitinib therapy may be eligible for a new phase II clinical trial led by Dr. Ellen Ritchie.

Navitoclax is an investigational agent that inhibits a family of BCL proteins. These proteins block some of the enzymes that keep cancer cells from dying and by inhibiting these proteins, navitoclax may cause the cancer cells to die.  Ruxolitinib is approved by the FDA for the treatment of myelofibrosis. Ruxolitinib blocks a protein called Janus-associated kinases (JAK) which may help keep abnormal blood cells or cancer cells from growing.

The purpose of this study is to evaluate the addition of navitoclax to ruxolitinib in patients who have been receiving ruxolitinib alone. Ruxolitinib treatment alone has not been fully controlling disease which is evident by an enlarged spleen. Preclinical data suggest that the combination of navitoclax with ruxolitinib are synergistic and that navitoclax may help to overcome disease resistance to ruxolitinib.

The study will help to determine the effect of the combination of navitoclax plus ruxolitinib on your cancer. The study will evaluate how the efficacy and safety of the drug.

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A Veteran’s Story: The Frustrations of Filing a Claim with the VA

By Wayne E.

MPN Patient and Vietnam Veteran Wayne E.

I served in the USAF Security Service, 6924th Security Squadron, stationed in Da Nang, Vietnam for one year (1970-1971) and was exposed to the deadly Agent Orange/Dioxin. In 2007, after a simple pre-op blood test, I was diagnosed with essential thrombocythemia (ET). Upon further study I was told I had an incurable, but manageable, blood cancer, coupled with a gene mutation (JAK2). The word cancer scared me. I had never heard of ET and I was at a loss for what to do. I didn’t know where to go next. After much reading about these potentially deadly diseases, I found out I was one of many Vietnam Veterans who had an MPN.

In 2011, I filed my first claim with the VA. Until this filing, I was unable to get any substantial information from my primary care physician (PHP) or my hematologist/oncologist, as to what may have caused or contributed to my ET. They knew virtually nothing about Agent Orange. I contacted the National Institutes of Health, The Centers for Disease Control, and as many online medical sites as possible, all ending with a bigger question mark. Nothing could be explained to satisfy my inquiry.

It was by chance that I connected with a most remarkable group, MPN Advocacy and Education International. I could never thank them enough for the compassion and the understanding they extended to me.

After my initial rejection from the VA, I filed three more times and each time I was denied because MPNs are not on the “presumptive” list of Agent Orange-related illnesses. The same message I kept getting was I needed “clinical rationale” to support my claims. My doctors have not been able to provide me with this needed information. I don’t know what to do today. I understand there are many Vietnam vets that have won their appeals and now get benefits, but there are many others who were not approved and just gave up. I don’t plan to give up.

To my fellow Vietnam Veterans who may be dealing with one of these MPNs, don’t give up. If you have been denied, file an appeal. There is hope, comfort, and assistance available. With the help of MPN Advocacy and Education International.

 Learn more about filing a claim with the VA

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Imago BioSciences Receives FDA Approval of IND Application for the Treatment of Myeloid Malignancies

SAN CARLOS, Calif., Feb. 1, 2018 /PRNewswire/ — Imago BioSciences, a clinical-stage pharmaceutical company developing novel therapies for hematological and inflammatory diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND) application providing clearance to proceed with the clinical development of IMG-7289 in the U.S. The IND supports the company’s ongoing Phase 1/2 clinical trial of IMG-7289 for myelofibrosis (MF).

“There is a pressing need for novel approaches to the treatment of myeloproliferative disorders including myelofibrosis,” said Hugh Young Rienhoff, Jr. M.D., Imago’s Chief Executive Officer.  “We are pleased to have received FDA acceptance of our clinical trial protocol and look forward to the imminent expansion of this study into the United States.”

This Phase 1/2 open-label clinical trial is designed to assess the pharmacodynamics of IMG-7289, an oral inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1) in high-risk myelofibrosis patients aged 18 or older ( Identifier NCT03136185).  Assessments include measuring changes in spleen volume, patient reported total symptom scores, mutant allele burden, inflammatory cytokines and bone marrow fibrosis over the course of the treatment period.  The trial commenced in Australia in 2017 and will add multiple sites in the United States in 2018.

This is the second clinical trial of IMG-7289 sponsored by Imago BioSciences, Inc.  The first, evaluating IMG-7289 for the treatment of high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), was initiated in 2016 ( Identifier NCT02842827) and continues to enroll patients.

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Qiagen JAK2 Test Gets FDA Clearance for Additional Blood Cancer Types

NEW YORK (GenomeWeb) – Qiagen said after the close of the market on Tuesday that it received US Food and Drug Administration clearance for its Ipsogen JAK2 RGQ PCR Kit for additional use in the diagnosis of all myeloproliferative neoplasms (MPNs).

In March 2017 the FDA cleared the assay as a qualitative in vitro diagnostics test for the detection of the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood to aid in the diagnosis of the blood cancer polycythemia vera.

The new FDA clearance now covers two additional types of MPNs: essential thrombocythemia and primary myelofibrosis.

The assay runs on Qiagen’s Rotor-Gene Q MDx instrument, a component of the modular QiaSymphony family of laboratory automation products.

Qiagen said that it is the exclusive worldwide licensee of intellectual property covering the detection of the JAK2 V617F mutation for diagnostic purposes, and that it recently reached a settlement with an unspecified molecular diagnostics industry supplier in Europe, which agreed to stop selling its own JAK2 V617F test kit.

“We are eager to expand the use of our Ipsogen JAK2 assay, which is already available in Europe and other markets, for use in a wider range of patients in the US,” Thierry Bernard, senior vice president and head of Qiagen’s molecular diagnostics business area, said in a statement. The assay “makes it easier for hematologists and oncologists to follow recommended diagnostic testing algorithms and international guidelines for their patients suspected of having MPNs.”

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New Data Proves Role of Thrombosis in Patients with MPNs

New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).

MPNs include a variety of blood disorders, like myelofibrosis, polycythemia vera, and essential thrombocytosis, and patients with them have been reported to be at increased risk for thrombotic events. Pharmacologic treatment can stabilize the blood counts, but they generally provide only partial symptom improvement. Until recently, no population-based study has estimated the excess risk with matched control participants.

Malin Hultcrantz, M.D., PhD and colleagues, in an effort to address the gap in knowledge, evaluated data from 9,429 patients – 46% of whom were male – diagnosed with MPNs who reported to the Swedish Cancer Register between 1987 and 2009, and matched them to 35,820 control participants and compared their rates of arterial and venous thrombosis. Median age of study participants was 72 years, and a mong them, 3,001 had polycythemia vera, 3,462 had essential thrombocythemia, 1,488 had PMF, and 1,478 had MPN unclassifiable.

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Incyte and Syros to collaborate in cancer R&D

  • Gene expression-focused biotech Syros Pharmaceuticals, Inc. has snagged a collaboration deal with biopharma Incyte Corp. to develop potential therapeutics to treat rare bone marrow disorders known as myeloproliferative neoplasms (MPNs).
  • In return for $20 million upfront in cash and R&D funding, and $10 million in stock, Syros will use its platform technology to find targets in MPNs. Incyte gets options to exclusive rights for therapies modulating up to seven validated targets discovered under the collaboration.
  • If Incyte exercises these options, Syros could see up to $54 million in option fees, and up to $115 million for products against each of the targets, as well as low-single digit royalties.

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MPN Research Foundation Announces the Passing of its Chairman and Founder Robert Rosen

MPN Research Foundation announced today with great sadness the passing of its Chairman and Founder, Robert Rosen, age 74. Diagnosed with polycythemia vera in 1997, Rosen was shocked to discover that little research was being conducted on his condition and that there were no advocacy groups working to assist people with these rare blood cancers. Polycythemia vera (PV) is one of a cluster of blood cancers known as myeloproliferative neoplasms (MPNs), which includes PV, essential thrombocythemia (ET) and myelofibrosis (MF).

In 1999 Rosen, an established and highly successful Chicago businessman, formed the MPN Research Foundation to catalyze research and advance treatments for MPN patients. To date, Rosen’s MPN Research Foundation has awarded in excess of $12 million in MPN funding that has led to over 60 MPN-focused research projects, the advancement and development of new drug therapies, the publication of cutting edge research in numerous peer-reviewed scientific journals and the development of much needed patient advocacy initiatives, including effective outreach to the U.S. Food and Drug Administration, and highly beneficial education tools for the MPN community.

Andrew I Schafer, M.D., Director of the Richard T. Silver Center for Myeloproliferative Neoplasms (MPNs) at Weill Cornell Medical College, stated, “Because of Bob Rosen’s relentless tenacity, intense work ethic and limitless optimism, the MPNRF has been involved in every major scientific and medical advancement in the field of MPN research. Bob and the MPNRF have made immeasurable differences in the lives of people living with MPNs, the physicians who care for these patients, and the researchers who continue to search diligently for a cure.”

“There are no words to describe our sense of loss over Bob’s passing,” added Barbara Van Husen, MPNRF’s President and long-time friend of Rosen. “Our best and most significant tribute to Bob will be our continued efforts to accelerate and fund innovative research, create greater connectivity and resources for improving the lives of patients, and ultimately discover a cure for families living with an MPN. This was Bob’s mission and I’m confident that the MPNRF is in a strong position both professionally and financially to succeed with his goal.”

Bob’s daughter Molly Rosen Guy, also a director on the MPN Research Foundation board, said “Dad sought the only available curative treatment – a stem cell transplant – which is fraught with complications and often unsuccessful. His death is further evidence of the unmet need to find better ways to improve and extend the lives of patients with polycythemia vera and other blood cancers. Our work will continue.” A video produced by Molly about Bob’s diagnosis and her entry to the board can be seen here.

About the MPN Research Foundation
Founded by patients for patients, the MPN Research Foundation is a catalyst for research funding in pursuit of new treatments – and eventually a cure – for myeloproliferative neoplasms (MPNs). To date, the MPNRF has funded more than $12 million in MPN research.

Research funding is only part of what we do. The MPNRF is also dedicated to helping people living with MPNs change their prognosis by serving as a valuable source of education and resources in the MPN community.

Click here to make a donation in Bob Rosen’s name

Celgene to Acquire Impact Biomedicines, Adding Fedratinib to Its Pipeline of Novel Therapies for Hematologic Malignancies

  • Fedratinib is a highly selective JAK2 kinase inhibitor that is being evaluated for myelofibrosis and polycythemia vera
  • Fedratinib demonstrated clinical improvement in a phase III trial with treatment-naïve myelofibrosis patients and in a phase II trial with myelofibrosis patients resistant or intolerant to ruxolitinib
  • A New Drug Application (NDA) submission for fedratinib in myelofibrosis is planned for mid-2018

SUMMIT, N.J. & SAN DIEGO–(BUSINESS WIRE)–Celgene Corporation (NASDAQ:CELG) and Impact Biomedicines today announced the signing of a definitive agreement in which Celgene will acquire Impact Biomedicines, which is developing fedratinib for myelofibrosis and polycythemia vera. Under the terms of the agreement, Celgene will pay approximately $1.1 billion upfront and up to $1.25 billion in contingent payments based on regulatory approval milestones for myelofibrosis. Additional future payments for regulatory approvals in additional indications and sales-based milestones are also possible.

“Myelofibrosis is a disease with high unmet medical need as the number of patients who are ineligible for or become resistant to existing therapy continues to increase”

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Fedratinib, a highly selective JAK2 kinase inhibitor, was evaluated in 877 patients across 18 clinical trials. In a randomized, placebo-controlled, phase III pivotal trial (JAKARTA-1) for patients with treatment-naïve myelofibrosis, fedratinib demonstrated statistically significant improvements in the primary and secondary endpoints of splenic response and total symptom score, respectively. In an exploratory subgroup analysis, these improvements were observed regardless of a patient’s baseline platelet count.

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ASH: Stemline Presents on Recent Clinical Trial Results

Stemline Therapeutics Presents Detailed SL-401 Pivotal Data in BPDCN at ASH and Kicks Off its BPDCN Awareness Campaign; Updated Results From Ongoing Trials in Additional Malignancies Also Presented

NEW YORK, Dec. 13, 2017 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, presented detailed data from its SL-401 pivotal trial in BPDCN, as well as results from other ongoing trials in additional indications, at the 2017 American Society of Hematology (ASH) Annual Meeting and Exposition, held in Atlanta, GA. Presentations are available on the Stemline website,, under the Scientific Presentations tab…

SL-401: Phase 1/2 Trial in myeloproliferative neoplasms (MPN): chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF)

  • Key outcomes (ASH ’17 data)
    – SL-401 Phase 1/2 trial consists of a Stage 1 (lead-in, dose escalation) and Stage 2 (expansion); has enrolled 24 patients
    – No dose limiting toxicities (DLT) were identified and a maximum tolerated dose (MTD) was not reached. Most common TRAEs include hypoalbuminemia (33%), thrombocytopenia (33%), and fatigue (29%). Most common TRAEs (grade 3 or higher), include thrombocytopenia (24%) and anemia (19%)
    – Durable CR (14+ months) in CMML patient
    – 65% (11/17 evaluable) of CMML and MF patients had spleen reductions >25% (range 29% to 100%)
    – Durable SD in 4 patients (2 CMML, 2 MF) for 5+ to 8+ months. Three ongoing SD patients enrolled with baseline platelet counts <100,000, including 1 patient platelet count <50,000 are on treatment
  • Next Steps in MPN
    – Continue enrollment and patient follow-up
    – Favorable tolerability and preliminary signs of activity support both single agent and combination development strategies, including JAK-inhibitors and hypomethylating agents

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ASH: Sotatercept Boosts Hemoglobin in MPN-Associated Myelofibrosis

The investigational activin receptor IIA ligand trap sotatercept safely increases hemoglobin levels in patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis, both when used alone and in combination with ruxolitinib (Jakafi).

A phase II investigator-initiated trial of sotatercept in this population showed responses in 10 of 28 evaluable patients, said Prithviraj Bose, MD, of updated findings presented at the 2017 ASH Annual Meeting.

Anemia is present in about one-third of patients with myelofibrosis at diagnosis and eventually develops in all patients. Only 20% to 30% of patients respond to current therapy, such as danazol and erythroid-stimulating agents.

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