Incyte Announces Pivotal Clinical Trial of Ruxolitinib for Treatment of ET

WILMINGTON, Del.–(BUSINESS WIRE)–Nov. 15, 2017– Incyte Corporation (Nasdaq:INCY) today announced that the first patient has been treated in the RESET pivotal trial evaluating ruxolitinib (Jakafi®) compared to anagrelide for the treatment of patients with essential thrombocythemia (ET) who are resistant to or intolerant of hydroxyurea (HU).

“We are pleased to treat the first patient in our pivotal trial evaluating ruxolitinib as a treatment for ET, a rare blood cancer that can lead to life-threatening complications,” said Steven Stein, M.D., Chief Medical Officer, Incyte. “We look forward to building on the clinical evidence for ruxolitinib and to advancing this trial to help address the needs of higher-risk patients with ET, who are resistant to or intolerant of HU and currently have limited treatment options.”

ET is a rare, chronic blood cancer, part of a group of related blood cancers known as myeloproliferative neoplasms (MPNs), characterized by increased platelet production, a white cell count above the normal range, persistently elevated platelet counts with normal red blood cell mass and the absence of prominent bone marrow fibrosis.1 An increased platelet count can increase the risk of thrombosis. Thrombosis can, in turn, lead to serious health problems including heart attack or stroke. Vascular complications and transformation to myelofibrosis (MF) or acute myeloid leukemia (AML) are the major causes of increased morbidity and mortality in patients with ET.2,3

About the RESET Study

The randomized, double-blind, double-dummy pivotal study (NCT03123588) is evaluating the safety and efficacy of ruxolitinib versus anagrelide as a treatment of patients with ET. The study is expected to enroll approximately 120 patients, 18 years or older, diagnosed with ET who are resistant to or intolerant of HU, with a screening platelet count of >650 × 109/L and white blood cell (WBC) count of >11.0 × 109/L.

The primary endpoint of this study is the proportion of patients who achieve platelet and WBC control over 1 year of follow-up. Key secondary endpoints include safety and tolerability and the proportion of patients who achieve complete remission (CR) or partial remission (PR). For more information about the study, please visit

About Jakafi® (ruxolitinib)

Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration, as Jakafi® (ruxolitinib), for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.

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Effective treatment of low-dose decitabine in MDS/MPNs

Objective: Primary myelofibrosis (PMF) is one of the Philadelphia negative myeloproliferative neoplasms (MPN). The main clinical features are obvious physical symptoms and symptomatic splenomegaly. It may be converse to leukemia and has a shortened life expectancy. Nowadays, the therapy for PMF is aimed at maintaining comfort and there is no curative treatment. PMF with myelodysplastic syndrome (MDS), called MDS/MPN-u, is rare and the treatment is complex. In this study, we want to discuss an effective treatment for MDS/MPN via a case report and literature review.
Materials and methods: A female patient was diagnosed with MDS/MPN through bone marrow cytology, immunology, cell genetics, molecular biology, and pathology. She received thalidomide and prednisone as initial treatment. Ten months later, the first-line therapy had failed, she presented with clinically relevant pancytopenia and increased blasts in bone marrow. Because decitabine is one of the first-line treatments for MDS and the patient was frail, she received low-dose decitabine as second-line therapy. Decitabine was administered at 15 mg/m2 once a week for 3 weeks, in a 4 week cycle. If there was improvement the treatment interval was prolonged.
Result: After one cycle, the blasts in bone marrow were decreased to 0.5%. After four cycles, she felt comfortable and hematological improvement was achieved. The treatment interval was prolonged. After eight cycles, the spleen reduced to 2 cm under the rib, and she achieved complete hematological remission. After ten cycles, the mutation of JAK2/V617F expression was decreased from 60.63% to 0.01%. During the therapy, the patient presented with grade III–IV hematological toxicity after the first two cycles, but there were no side effects after subsequent cycles.
Conclusion: Our research showed that low-dose decitabine may be an effective treatment for MDS/MPN, especially in improving physical symptoms and achieving hematological remission. Besides, it may be possible to reverse positive JAK2 mutation.

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Ruxolitinib vs Allogeneic SCT for Patients With MF According to Donor Availability


The present study will be a multicenter, prospective phase II-study comparing efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.


This study is a multicenter, prospective phase II-study compares efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.

In this study will further assess and compare the safety and efficacy of study treatments/ induction therapy in both study arms on spleen reduction, improvement of constitutional symptoms, QOL, toxicity, fibrosis regression, development of GvHD as well as chimerism, engraftment, relapse incidence, disease related mortality, outcome and overall survival.

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New MPN Progression Marker Project

The Progression Marker Project: A Rigorous Effort to Identify the most Relevant and Influential Markers for Therapeutic Intervention and Challenge the Natural History of Myeloproliferative Neoplasms

We Need Your Help

Only a very few genetic mutations have been linked to MPNs and despite the one approved therapy that targets the JAK2 mutation, this targeted therapy does not lead to a cure nor does it help every MPN patient.

The MPNs represent a group of diseases in the blood cancer space that affect normal blood cell production in the bone marrow. The bone marrow causes an overproduction of one or more blood cell types (red cells, white cells or platelets). MPN physicians are struggling to understand which mutations and / or biologic markers are the primary risk factors for disease progression and poor patient outcomes. Since risk-adapted therapy for some cancers is becoming standard of care, there is an urgent need for a precise risk stratification model/understanding to assist in therapeutic decision-making and research. While progress has been made, there remains a significant opportunity to improve patient stratification to optimize treatment and to develop new therapies for high-risk patients.

Results from this highly collaborative research effort, with the best and brightest in the MPN research space and industry partners, are expected to significantly advance our understanding of risk factors and molecular changes that lead to disease progression; shift the paradigm of treatment potentially delaying progression, and identify new drug targets for therapeutic intervention.

Why now?

“We need rigorous data to move physicians away from “watch and wait” to treatment strategies based on an understanding “why, how and when” people with MPNs should be treated. The Progression Marker Project is designed to provide us with this essential yet missing data.”
– Srdan Verstovsek, MD Anderson, Principal Investigator for the Progression Marker Project

Project Goals

  • Identify within the MPNs e.g., ET, PV and MF, the most relevant markers of disease progression and establish the most appropriate time to effectively intervene therapeutically
  • Identify which patients are at greatest risk to progress to MF
  • Provide patients with treatment strategies to prevent conversion to Myelofibrosis
  • Identify biomarkers and molecular changes that most commonly occur in this heterogenous population to identify novel or relevant therapeutic targets

The Strategy:

  • Convene a panel of thought leaders to establish the protocol and mechanisms for building the database and biobank required to meet the Project goals
  • Partner with industry, motivated patients and MPN researchers to provide the financial and professional resources to support and execute the Project
  • Enroll 500 PV and ET patients for a 5-year study
  • Share data with MPN researchers and industry partners in an open-source competitive challenge. This truly represents a chance not only to integrate available data and analytical approaches to tackle this important problem, but also provides the ability to benchmark potential methods to identify those with the greatest potential to yield patient care benefits in the future

Why Help?

The Progression Marker Project is expected to produce actionable results within 5 years by building a network of researchers sharing data and creating solutions to treating patients at risk for progression of their myeloproliferative neoplasms.

This project will lead to new treatments, new combinations of treatments and, new insights regarding the most appropriate time to treat patients.

The cost of the Project is estimated at $5.5 million and the impact to patients’ lives is estimated to be priceless.

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Blog: Living in Paradox

November: You Are Not Alone

By Marina Sampanes Peed

When people with a MPN gather, it is easy to forget that these blood cancers are rare. MPN advocates from two dozen countries describe patient experiences that are eerily similar:

  • Correct diagnosis follows one or more years of signs or symptoms.
  • Clinician who made diagnosis did not provide current information about the disease nor treatment options. (Some discount while others magnify the risks.)
  • Delayed in receiving appropriate treatment.
  • Many “Quality of Life” symptoms are not recognized and remain untreated, especially in women.
  • Access to and participation in clinical trials is difficult.
  • Support from the MPN patient community is highly valued.

Most hematologists rarely, if ever, encounter a patient with a MPN. Because the diseases manifest differently, experience with a few patients is not sufficient to develop expertise. If your local physician is not proactive, you may insist he/she confer with a MPN specialist regarding your care. The experts regularly consult with general practitioners and hematologists regarding management of their MPN patients.

If your local physician is not proactive, you may insist he/she confer with a MPN specialist regarding your care. The experts regularly consult with general practitioners and hematologists regarding management of their MPN patients.

The patient experience was a recurring theme in Frankfurt am Main, Germany October 27-29, 2017 when 43 patient advocates from 24 countries gathered with several clinicians at the 2nd MPN Horizons International Conference for MPN Advocates. More reports coming soon.

Here are just a few headlines:

“These diseases will eventually be called by their mutations rather than Essential Thrombocythemia, Polycythemia Vera, and Myelofibrosis.”

~ Martin H. Ellis, MD, Meir Medical Center and Tel Aviv University, Chairman of Israel MPN Working Group

“Patient Reported Outcomes are, in fact, doctor reported outcomes; not actually from the patient. There is work to be done.”

~ Sarunas Narbutus, European Patients Academy on Therapeutic Innovation (EuPATI)

“The aging of hematopoietic stem cells may play a role in the mutations, and the health of the niche where the stem cells reside is also important… There appears to be hierarchy among mutations and competition among them.”

~ Saghi Ghaffari, MD, PhD, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai

“When someone receives a MPN diagnosis, their cognitive map lights up based on beliefs, knowledge, expectations, psycho-emotional reactions, and personality traits.”

~ Ketti Mazzocco, PhD University of Milan, Department of Oncology and Hemato-oncology; European Institute of Oncology



Atlanta MPN Program: Interesting Speakers Share Valuable Information

Last week we hosted another MPN education program in Atlanta.  Being in Atlanta was a bit nostalgic and heartwarming.  Bonnie Evans, longtime partner and caregiver to Joe (now deceased), spoke about why she became involved on a grander scale in the MPN community.  The Atlanta support group, founded by Bonnie, is still going strong.  Marina Peed shared her experiences and journey as a patient and transplant survivor. Her unique challenges and ultimate cure led her to a new role as a strong advocate for MPN patients. Dr. Jennifer Powers, a pharmacologist and MPN patient, imparted valuable information for those struggling with insurers and outlined the benefits of specialty pharmacies. Maria Hanik gave an informative overview of the BMT program at Northside Hospital Cancer Institute.

Our clinicians and researchers, Drs. Rampal, Michaelis and Winton shared updates on current clinical trials, what MPNs look like and how the body reacts to polycythemia vera, essential thrombocythemia and myelofibrosis. The feedback we hear from patients after each educational program fuels our commitment to continue providing the best possible events and resources available.


Co-Pay & Insurance Assistance Resources


For Jakafi

Patient Access Network: Phone: 1-866-316-7263

PAN Foundation has co-pay assistance program for Myeloproliferative Neoplasms and many other diseases.

Good Days from CDF (formerly known as Chronic Disease Fund, Inc):     Phone:  1-972-608-7141

Good Days is a co-pay assistance organization and Myeloproliferative Neoplasms are covered.

HealthWell Foundation:

Phone:  1-800-675-8416

NeedyMeds: Phone: 1-800-503-6897

Partnership for Prescription Assistance:

Phone:  1-888-477-2669

Patient Advocate Foundation Co-Pay Relief Program:

Phone:  1-866-512-3861

CancerCare Co-Payment Assistance Foundation:

Phone:  1-866-552-6729

Rx Outreach:   Phone:  1-800-769-3880





Behind the Counter: Tips from Your Specialty Pharmacist

By Marina Sampanes Peed

Traditionally, cancer treatments were delivered by infusion in clinics and hospitals. Today, many are patient-administered with 30% expected to become available in pill/capsule form in the near future. MPN patients routinely self-administer Hydroxyurea, Pegylated Interferon-a, or Ruxolitinib.

At our Atlanta MPN Patient Education program, Jennifer Powers, PharmD, of Walgreens Specialty Pharmacy explained what these changes mean for MPN patients today. Cancer drugs are classified as “specialty medications” and their coverage varies by prescription drug plan. They are typically not available through retail pharmacies. And the cost to patients varied widely.

Check Your Drug Plan

Above, Dr. Jennifer Powers, PharmD, presents at the Atlanta MPN Patient/Caregiver Program

Dr. Powers urges everyone to look at the new prescription drug plans before Open Enrollment season each year. Make sure the medications you expect to require are covered in your plan. Both public (Medicare/Medicaid) and private, commercial drug plans are subject to changes.  The name of the plan and monthly premium may look the same, but the drugs covered, dollar amount covered, and co-pays may change. Their pharmacy contracts may also change. Each plan has tiers of medications, with different levels of coverage. The tier level of a drug varies by pharmacy, as do the pricing and financial requirements. Some require multiple prior authorizations, continued follow-up, and appeals for off-label use.

More Hoops for Patients

Before you can receive the specialty medication prescribed by your doctor, the Prescription Benefits Manager (PBM) that administers the plan must issue a Prior Authorization. It is quite common for them to reject the initial treatment request, so an appeal must be filed. If the drug is not on their “formulary” (list of drugs covered), it often takes more than one appeal before treatment is approved. Once approved, the medication is ordered and shipped to the specialty pharmacy, then delivered to the patient.

Often refills must also go through the review, denial, and appeal process. With new treatments coming to market and “off-label” use of medications for other illnesses, the process adds to patient fatigue and stress.

Your pharmacist will work with your doctor and assist you through this process.

Financial Toxicity is a Known Side Effect

Financial toxicity comes from the direct costs associated with treatment. There is little room in most personal budgets to absorb ongoing, expensive treatment. Out-of-pocket medical costs (deductible, co-pay, co-insurance) as a percentage of income for managing chronic illness can lead to credit card debt, medical debt, trouble paying for other necessities (housing, food).

The expense of treatment is shown to impact several health related outcomes: treatment compliance; quality of life; and response to treatment.

Some Financial Assistance is Available

There are several Prescription Assistance Programs (PAP) available to patients, each with its own requirements and limitations. The main eligibility criteria include: diagnosis; funding availability; existing insurance coverage; patient’s finances; and FDA approval or off-label use.

Dr. Powers encourages patients to appeal if their application is denied. Some are determined by the prior year’s tax return information; if the current financial situation is worse, explain it in the appeal.   If you exhaust a grant, apply again.

(See the list of resources at the end of this article.)

Importance of Medication Adherence

While it may seem obvious, taking medicine as prescribed is important to manage disease. Dr. Powers highlighted four common challenges for patients:

  1. Affordability: some patients “stretch” the dosing by taking a lower dose than prescribed or skipping a dose regularly because of cost. Some patients buy pills as they have cash available.
  2. Unpleasant side effects: some patients stop taking a medication because of initial side effects or ongoing side effects that affect their quality of life.
  3. Incorporate with Lifestyle: it is important to find the right medication schedule for you. Some are best taken at night so side effects are less noticeable; some may be easier to manage when taken a certain day of the week.
  4. Access to Information: simply knowing what a drug is doing for you, what can interfere with its effectiveness, or how to alleviate side effects can help a patient comply with the treatment plan. Learning about financial and social resources can also help.

Travel Planning Tips

When planning to travel, don’t wait until departure day to think about your prescription medications.

  • Ask your pharmacist about “vacation overrides” to be sure you have enough medicine while away from home.
  • Check if you need a new prescription or a refill.
  • If you need medication sent to a temporary location, make arrangements before you leave home.
  • Check if you need a doctor’s note regarding your diagnosis and treatment to take with you.
  • Get additional packaging, supplies, and ancillary medications.
  • Remember a cooler and ice packs if needed.
  • Check TSA / airline rules about carry-on injectable medications.

Co-Pay & Insurance Assistance Resources

For Jakafi

Patient Access Network: Phone: 1-866-316-7263

PAN Foundation has co-pay assistance program for Myeloproliferative Neoplasms and many other diseases.

Good Days from CDF (formerly known as Chronic Disease Fund, Inc):     Phone:  1-972-608-7141

Good Days is a co-pay assistance organization and Myeloproliferative Neoplasms are covered.

HealthWell Foundation:

Phone:  1-800-675-841

NeedyMeds: Phone: 1-800-503-6897

Partnership for Prescription Assistance:

Phone:  1-888-477-266

Patient Advocate Foundation Co-Pay Relief Program:

Phone:  1-866-512-3861

CancerCare Co-Payment Assistance Foundation:

Phone:  1-866-552-6729

Rx Outreach:   Phone:  1-800-769-3880




Cancer drug fedratinib attracts $90 million for Impact Biomedicines

Less than two weeks after raising $22.5 million, San Diego cancer drug developer Impact Biomedicines has closed a deal for up to $90 million more.

The investment from Oberland Capital will support the launch of fedratinib, a blood cancer medication Impact Biomedicines was formed to bring to market. An application to sell the drug in the United States is being prepared for submission.

Impact is readying manufacturing and logistics so the drug can be available immediately after approval. With strong evidence the drug works against the bone marrow cancers polycythemia vera and myleofibrosis, approval is likely, said John Hood, the company’s CEO.

Drug information will be posted at

Oberland Capital will make two $20 million payments, contingent on Impact meeting milestones. In exchange, Oberland will get royalties on fedratinib sales. If fedratinib is approved, Oberland will lend up to $50 million. The deal was announced Oct. 26.

Fedratinib was first developed by San Diego-based TargeGen, which French drugmaker Sanofi purchased in 2010 for up to $635 million on the drug’s promise.

But in 2013, just as clinical trials appeared to be successfully concluding, a few patients developed a neurological disorder. The Food and Drug Administration put a hold on development, and Sanofi immediately dropped the drug.

Hood and Dr. Catriona Jamieson, a UC San Diego oncologist/researcher, have looked ever since for a way to bring back fedratinib. Efforts began almost immediately, but negotiating rights to the drug from Sanofi and getting the needed funding took time.

Jamieson said fedratinib produced durable remissions in some patients that no other drug could replicate. A number relapsed and died after the drug was no longer available, said Jamieson, the company’s interim chief medical officer.

Having been presented evidence the disorder wasn’t caused by the drug, the FDA has released its hold, and can now consider an application to approve it.

The second round of financing was prompted in part by recent results of a fedratinib trial. Published in The Lancet Haematology, the study found that the drug significantly benefited patients with ruxolitinib-resistant or intolerant myelofibrosis. Sold under the name Jakafi, ruxolitinib is the only US-approved drug for myelofibrosis.

The financing was structured to avoid diluting Impact’s equity, Hood said. The company did grant Sanofi an equity stake of about 10 percent to secure worldwide rights to fedratinib, he said.

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New Patient Guidelines From the NCCN Offer Much-Needed Clarity Around a Group of Rare Blood Cancers

New NCCN Guidelines for Patients® cover the basics and beyond for patients and caregivers coping with myeloproliferative neoplasms.

Newswise — [FORT WASHINGTON, PA  — October 19, 2017] – Patients with blood cancers known as myeloproliferative neoplasms (MPN) have a new resource to help guide them through diagnosis and treatment, in the form of the National Comprehensive Cancer Network® (NCCN®)’s latest addition to the NCCN Guidelines for Patients®. This NCCN Guideline for Patients focuses on the three most-prevalent types of MPN: polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), which affect approximately 148,000, 134,000 and 13,000 patients in the United States, respectively. [1] Funding for these patient guidelines was provided through the NCCN Foundation® and the MPN Research Foundation.

“As a physician, I find it makes a difference when patients and caregivers have access to the information they need when making treatment decisions, to complement what they’re hearing from me,” explained Brady L. Stein, MD, MHS, Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Dr. Stein is a member of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Panel for MPN. “Sitting in the hematologists’ office can be an overwhelming experience. These patient guidelines provide the most comprehensive at-home resource available for people with these rare diseases. They cover everything from basic explanations to complicated decision-making around diagnostic confirmation, supportive care techniques, treatment sequencing, adverse effects, and more.”

According to Dr. Stein, it’s not uncommon for patients diagnosed with MPN not to understand at first that their condition technically represents a chronic form of blood cancer, or that it can progress. In fact, most patients and caregivers have never even heard of MPN prior to diagnosis.

“When I was first diagnosed with ET, I actually left the office feeling relieved,” said Christy, a patient living with MPN. “It wasn’t until my pharmacist explained I had a prescription for a chemotherapy drug, that I really had any idea that I had cancer. I just assumed I’d only be taking a blood thinner. I’m grateful that these patient guidelines are helping me to understand what’s going on in my body, and why this chemotherapy is the right treatment path for me.”

“Having this free information available online and on their smartphones is particularly important for patients who can’t just reach out to a friend or relative who’s been through the same experience,” said Robert W. Carlson, MD, NCCN Chief Executive Officer. “The goal is not just to make them feel more informed, but also less isolated.”

NCCN Guidelines for Patients and NCCN Quick Guide™ sheets—one-page summaries of key points in the patient guidelines—are written in plain language and include patient-friendly tools, such as suggested questions for doctors, a glossary of terms, and medical illustrations of anatomy, tests, and treatment. They are based on the same clinical practice guidelines used by health care professionals around the world to determine the best way to treat a person with cancer. Each resource features unbiased expert guidance from the nation’s leading cancer centers designed to help people living with cancer understand and discuss their treatment options with their providers.

The NCCN Guidelines for Patients and NCCN Quick Guide™ sheet for MPN are available to read and download for free online at and via the NCCN Patient Guides for Cancer mobile app. Printed editions can also be ordered from for a small fee.

NCCN currently offers NCCN Guidelines for Patients for the following: Brain, Breast, Colon, Esophageal, Kidney, Non-Small Cell Lung*, Ovarian, Pancreatic, Prostate, Rectal, Stomach and Thyroid Cancers; Acute Lymphoblastic Leukemia; Adolescents and Young Adults with Cancer; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia*; Distress/Supportive Care; Hodgkin Lymphoma; Lung Cancer Screening; Malignant Pleural Mesothelioma; Melanoma*; Multiple Myeloma*; Myelodysplastic Syndromes*; Myeloproliferative Neoplasms; Nausea and Vomiting/Supportive Care; Non-Hodgkin’s Lymphomas; Soft Tissue Sarcoma; and Waldenström’s Macroglobulinemia. * Indicates NCCN Guidelines with new updates coming soon.

NCCN Guidelines for Patients and NCCN Quick Guide™ sheets DO NOT replace the expertise and clinical judgment of the clinician.


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