Incyte (INCY) Announces Positive Results on Jakafi for GVHD

Incyte Corp announced that the phase II trial, REACH1, evaluating lead drug Jakafi in combination with corticosteroids for the treatment of patients with steroid-refractory acute graft-versus-host disease (GVHD), met its primary endpoint.

The data from the study showed that Jakafi demonstrated an overall response rate (ORR) of 55% at day 28. Moreover, the number of patients achieving a response at any point of time, during the study, was 73%. Propelled by positive data from the REACH 1 study, Incyte now plans to file a Supplemental New Drug Application (sNDA) with the FDA for the label expansion of Jakafi, for the treatment of steroid-refractory acute GVHD, during the third quarter of 2018.

Incyte also plans to present full details from the study, at an upcoming scientific meeting.

We note that Jakafi, a first-in-class JAK1/JAK2 inhibitor, is already approved by the FDA for the treatment of people with polycythemia vera (PV), who have had an inadequate response to or are intolerant of hydroxyurea.

The drug is also indicated for the treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF and post–essential thrombocythemia MF. Incyte has a collaboration agreement with Novartis (NVSFree Report) for Jakafi. While Jakafi is marketed by Incyte in the United States, it is marketed by Novartis outside the country.

Jakafi’s performance has been impressive so far. In order to expand the patient population and increase the commercial potential of the drug, the company is working on expanding the drug’s label, further. In October 2017, the FDA approved a label update of the drug to include the addition of new patient-reported outcome (PRO) data from the COMFORT-I study as well as updating the warning related to progressive multifocal leukoencephalopathy.

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Small Number of Myelofibrosis Patients Develop Aggressive Lymphoma from JAK Inhibition

BY Brielle Urciuoli
PUBLISHED June 25, 2018

While JAK inhibitors have proven to be an effective treatment for patients with myelofibrosis, a type of myeloproliferative neoplasm (MPN), they may come with a severe downside – in particular, a 16-fold increase in the chance of developing a B-cell lymphoma, according to recent research published in the journal Blood.

“Many patients profit from this treatment and survive with good quality of life. Therefore, it is important to study potential long-term side effects of this therapy,” study author Ulrich Jager, M.D., head of the Division of Hematology and Haemostaseology at the Medical University of Vienna in Austria, said in an interview with CURE.

Researchers examined 626 patients with MPNs who were diagnosed and treated at the Medical University of Vienna between the years of 1997 and 2016. Of these patients, 69 with myelofibrosis were treated with a JAK1/2 inhibitor for lymphoma development, which included Jakafi (ruxolitinib), gandotinib, fedratinib or momelotinib.

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From Cleveland to Pittsburgh: A Busy Travel Schedule Worth Every Minute


Top left, Dr. James Rossetti, Pittsburgh Support Group Coordinator, Jean Diesch, with Ann Brazeau, Dr. Salman Fazal with Dr. John Mascarenhas and Dr. Raajit Rampal.

On June 7, MPN Advocacy & Education International held its first MPN Patient/Caregiver program in Pittsburgh. Support Group Coordinator, Jean Diesch, invited us many times and we were finally able to make it happen. Two local hematologists, Drs. Fazal and Rossetti joined Drs. Rampal and Mascarenhas for this program.

Dr. Rampal, Memorial Sloan Kettering, presented on new therapies in MPNs, The good news is the development of new JAK inhibitor therapies on the horizon for MPN patients. Click here to view new clinical trials.

Dr. John Mascarenhas, Mount Sinai, discussed new polycythemia vera therapies. Comparing the goals of PV patients to feel better, and the goal of investigators to take what they are doing in the lab and bring it to the patient. Dr. Mascarenhas believes clinical trial participation is the pathway to a cure. This resulted in an excellent discussion with the patients on what they can do to help. Dr. Mascarenhas urged patients to consider donating to a tissue bank. Mount Sinai has a donation program in place, click here to learn more.

Dr. James Rossetti, UPMC Hillman Cancer Center, gave an overview of bone marrow biopsies and aspirations, including how they are done, what is extracted and why, and what they can learn. Many patients have multiple biopsies over the course of their disease.  Biopsies are helpful in creating a baseline for assessing the progression of the disease. (There is a new method being researched to produce non-invasive biopsies, click here to learn more.)

Dr. Salman Fazal, Temple University School of Medicine, reviewed the findings from the MPN Landmark Study. This study highlighted the differences in treatment goals and perception of symptom burden between patients and the hematologists/oncologists who treat MPN patients. While patients listed slowing the progression of the disease as their top priority, physicians listed the reduction in symptom burden, thrombotic events, as their primary goal. For many physicians, this offered unique insights into a patient’s mindset. To learn more about the outcomes of this study click here.

MPN Field ‘Hitting Its Stride,’ But More Work Is Needed

New biomarkers are being investigated in myeloproliferative neoplasms (MPNs), pointing toward a future of advances in the field, according to David S. Snyder, M.D., associate chair and professor of the Department of Hematology and Hematopoietic Cell Transplantation at the City of Hope.

“The field of MPNs is hitting its stride with new molecular markers that are being defined,” Snyder said in an interview with OncLive, a sister publication of CURE.

Currently understood driver mutations include JAK2, CALR and NPL, but researchers are now looking at secondary mutations and their significance toward a patient’s prognosis. Snyder said that these findings will lead to targeted therapies, as they did for Jakafi (ruxolitinib), a JAK2 inhibitor that was the only FDA-approved drug that was developed for myelofibrosis.

Jakafi may also work for patients with a CALR mutation as a different mechanism, according to Snyder. He explained that there are two types of CALR mutations: type 1, which is a deletion in the protein; and type 2, which is an insertion into the protein. The CALR mutations ultimately feed through the JAK2-STAT pathway, similar to the JAK2 pathway.

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Effect of MPN Diagnosis on Work, Employment Status

Many patients experience a negative impact on work productivity and employment status after a diagnosis of myeloproliferative neoplasm (MPN), according to a study published in BMC Cancer.

The clinical adverse effects of MPNs (eg, polycythemia vera [PV], essential thrombocytopenia [ET], myelofibrosis [MF]) are well known; risks for cardiovascular and thrombotic events and various other symptoms are increased. The impact of illness on the workplace and careers, however, have yet to be explored.

For this study, researchers sent the cross-sectional online survey “Living with MPNs” to patients with a myeloproliferative neoplasm. The survey consisted of approximately 100 questions related to MPN, focusing on factors such as diagnosis, symptoms, and changes in employment, work productivity, and daily activities. The Work Productivity and Activity Impairment Specific Health Problem questionnaire (WPAI-SHP) was used to evaluate the effects of MPN on work productivity and activity for currently employed respondents, and the MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) was used to measure symptom burden.

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PV Treatment Landscape and QOL Challenges


Dr. Jeanne Palmer, MD

While there is not a large number of treatments for patients with polycythemia vera (PV), there are currently regimens in the first- and second-line setting that work well for this population, says Jeanne M. Palmer, MD.

For example, long-term follow-up data from the phase III RESPONSE study showed that patients who responded to the JAK2 inhibitor ruxolitinib (Jakafi) as a second-line treatment maintained those responses for up to 4 years. At 208 weeks, 41 patients (37%) originally on the ruxolitinib arm remained on treatment compared with no patients on the control arm. Additionally, 70% of patients who had clinicohematologic (CLHM) response maintained that status. There were no new safety signals compared with the previous follow-up at 80 weeks.

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View Dr. Palmer’s Presentation @ San Antonio MPN Patient Program

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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View MF Clinical Trials



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

 Learn more about Veterans and MPNs

Vanderbilt University Hosts MPN Advocacy & Education International

From Left, Dr. Palmer, Ken Rosen, Drs. Stein, Savona & Gerds and Ann Brazeau

“It is always a great honor to spend time with patients. Thank you for taking the mantle MPN Advocacy & Education International.” ~Dr. Michael Savona, MD

On Friday, April 27th, MPN Advocacy & Education International presented their first educational program in Nashville, Tennessee. Dr. Michael Savona, Associate Professor of Medicine and Director of Hematology Research at the Vanderbilt University Medical Center, invited us to Vanderbilt to engage MPN patients from several cities and states in that region of the country. MPN specialists, including Dr. Savona, gave valuable updates and spent quality time with patients and caregivers answering questions. Dr. Gerds, Assistant Professor in Medicine (Hematology and Oncology) at the Cleveland Clinic Taussig Cancer Institute, shared in detail why patients may not be feeling so well and offered advice on ways to ease some of their symptoms. Dr. Brady Stein, Assistant Professor, Northwestern Feinberg School of Medicine, presented excellent findings on gender differences. Dr. Savona offered an overview of MPNs including symptom management, treatments options, and clinical trials.

Additionally, Dr. Jennifer Powers, Senior Manager of Oncology Disease at Walgreens, imparted great direction and advice from her perspective as a patient and a pharmacist. Dr. Powers has firsthand knowledge of the challenges MPN patients endure accessing and paying for treatments. Ken Rosen, a family therapist and MF patient, added the key ingredient missing in so much of the information given to patients-ways to quiet ones brain and develop peaceful coping skills. His patient story revealed a forty year commitment to meditation and a vegetarian diet that he believes diminishes his constitutional symptoms. Thank you to all the attendees who made the effort to be at this event and to the presenters for taking the time to participate and talk with the patients.

“I want to thank you for putting together such a wonderful program.  I came away with not only important information, but a sense of support from everyone there. It was a great experience!” ~Patient Attendee

NOTE: An MPN Patient Support Group is forming in Nashville that will include those in surrounding cities and states. For more information please contact Michele Riley,

A special thank you to our generous sponsors for making these programs possible:




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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