CTI BioPharma Announces First Patient Enrolled in Phase 2 Trial of Pacritinib in Patients with Myelofibrosis who have Thrombocytopenia and who have been Previously Treated with Ruxolitinib

SEATTLE, Aug. 1, 2017 /PRNewswire/ — CTI BioPharma Corp. (NASDAQ and MTA: CTIC) today announced that the first patient has been enrolled in PAC203, a Phase 2 clinical trial of pacritinib in patients with primary myelofibrosis who have failed prior ruxolitinib therapy. PAC203 is designed to evaluate the dose response relationship for safety and efficacy (spleen volume reduction at 12 and 24 weeks) of three dose regimens: 100 mg once-daily, 100 mg twice-daily (BID) and 200 mg BID. The 200 mg BID dose regimen was used in the Phase 3 PERSIST-2 trial of pacritinib in patients with myelofibrosis. The trial is expected to enroll up to approximately 105 patients.

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Everyone’s MPN Is Different: What You Should Know About Genetic Mutations

Should you get a genetic test? Myeloproliferative neoplasm (MPN) experts, Dr. Alison Moliterno and Dr. Stephen Oh, help patients understand the driver mutations associated with MPNs such as JAK2V617F, calreticulin (CALR), MPL and others. The experts explain why you should know your genetic mutations and how this knowledge can impact your care and treatment. The panel also discusses if MPNs are hereditary and what causes mutations. You’ll also hear from Marsha, a patient advocate, as she shares her own story.

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Registration-Women & MPN

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3rd Annual Women & MPN Patient Education Program

Thank you for registering for the Women & MPN Patient/Caregiver Education Program on September 29, 2017 in Los Angeles, CA.  We look forward to seeing you.  To complete the registration you can use the link below to pay the registration fee.  Follow the brief instructions to link to the PayPal account.

NOTE: You do not need a PayPal account to make this payment, you can select the “Pay with Debit or Credit Card” option.

 If you are registering for you and a guest or 2 guests please use “Select Registration Option” from the drop down menu provided on the registration page


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Agent Orange’s Possible Link to Rare Cancer Type Sparks Advocacy Efforts

Exposure to Agent Orange — a toxic chemical combination used for deforestation during the Vietnam War — may be the cause of myeloproliferative neoplasms (MPNs) for hundreds of war veterans, according to MPN Advocacy and Education International.

“There was evidence very early that its use to exfoliate the jungle in Vietnam and other parts of the territory was having a grave impact on the health and safety of those exposed, including civilians,” Ann Brazeau, CEO of MPN Advocacy Network and Education International said in an interview with CURE.

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A Veteran’s Story Told By His Wife

Bill C. Veteran and MPN Patient

In January 2017, my husband, Bill, was enjoying his consulting business and writing a book that has been in his mind for years, when an annual physical changed everything. Some lab work was “off” according to the Veterans Administration (VA) physician so Bill sent the lab results to our personal physician, who is also our best friend. Within ten minutes after receiving it, our friend asked Bill to come to his clinic immediately.  Further blood tests yielded an initial diagnosis of Chronic Myeloid Leukemia (CML).

While we were reeling from that shock and trying to ground ourselves, our physician friend sent us to a local hematologist for additional work-ups and treatment.  Following a bone marrow test, we were further shocked to find out that the CML we had become somewhat resolved with was indeed Myelofibrosis (MF).  We sat in a dumb stupor trying to figure out what that was, how serious it was, where it came from, what we could do about it, etc.  We were encouraged to start on the only medication for Myelofibrosis, Jakafi, and were told the only drawback to the drug was its cost–$10,000/month!  We immediately started working with the VA for them to supply the medication.  After numerous telephone calls and in-person visits with both our hematologist and the VA (in a city 40 miles from our home), we secured VA support for Bill’s Jakafi.  It now routinely comes to our home in an innocuous package.  The initial symptoms Bill was experiencing responded to the Jakafi but so did the platelets and hemoglobin which are continuing to drop so we played with the dosage to, hopefully, continue to drive the white blood count down while keeping the platelet count and hemoglobin up closer to where they should be.  The drop in Jakafi was too drastic and symptoms immediately returned so our local hematologist moved Bill back to the original dosage. Symptoms once again are gone and platelets somewhat controlled but still very low. Hemoglobin is recovering which is good.

While all of this was going on, we decided, with the support of our local hematologist (who is wonderful realizing this is all about Bill and not about the hematologist’s ego), to go to Rochester, MN to Mayo Clinic to see one of the leading researchers in Myelofibrosis, Dr. Tefferi.  While there was no proactive guidance offered from this visit, we did learn that we should only approach researchers whose field of study is a fit between their interests and Bill’s current health status.  To that end, we went to Northwestern University in Chicago to see Dr. Brady Stein, another renowned Myelofibrosis researcher. He listened and answered all of our questions while assessing Bill’s fitness for ongoing clinical trials. His ultimate recommendation was for us to consider a transplant—again, another shock as we had hoped that we would have a variety of alternative treatments  Dr. Stein is concerned that Bill’s Next Gen Sequence report that showed other mutations limit the time he will have before he converts to Acute Myeloid Leukemia (AML). Since Bill is in such good health right now (ironically), Dr. Stein found him to be amongst 10% of people with MF that even qualify for a transplant consultation and while a transplant is a “rough ride” encouraged us to explore it.

To that end, we have met with Dr. Tom Chauncey who is the Program Director at the VA in Puget Sound-who along with the University of Washington at Seattle are the number one transplant center (particularly for people with MF) in the country. Dr. Chauncey was very generous with his time and counsel and offered to work with us as we continue to explore transplant options. With Dr. Stein’s support, we also will meet with the Director of the Northwestern University Transplant Program, Dr. Mehta.

Simultaneously, we filed a VA benefits claim related to Myelofibrosis, believing Bill’s exposure to Agent Orange most likely caused this illness, but we were denied.  Bill is a Vietnam Veteran having served in Quang Tri—I Corp from November, 1968 – November, 1969 and was exposed to Agent Orange/Dioxin.  While compiling our appeal information, we found numerous Citations where the VA had granted benefits for veterans (on appeal) who have been diagnosed with Myelofibrosis due to exposure to Agent Orange, so will be using that information to move our claim forward.  During the exploration for more information, we also discovered that the VA is finalizing a rule to add to the benefits structure Stem Cell Transplant coverage as well as treatment protocol to include myelosuppressive therapies of which Jakafi is one. This proposed change is set to take effect in FY18 (which begins as soon as October 1, 2017) which is exciting for veterans waiting for coverage of Myelofibrosis because, at least, some of the symptoms and associated therapies will be addressed.

MPN Advocacy & Education International continues to advocate for essential thrombocythemia, myelofibrosis and polycythemia vera to be included in the VA’s ‘presumptive’ list of illnesses related to Agent Orange exposure. Please click here if you are in the process of filing a claim or appealing a claim for more details.

Like many of you, we are sure, our world right now is exploring drugs in Phase II or III of clinical trials that are successful in producing remission in MF as well as other drug trials/existing drugs that will hold down the “blasts” that would otherwise convert Bill to AML. We are also exploring transplant centers, protocol, outcomes, experiences, etc. to get a better sense of whether that is something we even want to consider.

In the midst of all of this, we continue to work hard to enjoy our lives. Having a daughter with Down Syndrome who is now 43 years old taught us that nothing is ever guaranteed and that we would have to fight for anything and everything we wanted.  We “cut our teeth” on the fights for our daughter, Mindie, against insurmountable odds and won.  Now we are using those skills on Bill’s behalf.  While sad and scared, we remain determined that there are many, many opportunities for Bill to remain as healthy as he is today and live a long and enjoyable life beyond the current prognosis.   We know there is a lot to learn from all of you “in our same boat” and look forward to sharing stories and guidance between all of us.  In the interim, all of you touched by an MPN are in our thoughts and prayers.  Together we can change the face of these diseases!

 

 

MPNs: Translating New Discoveries Into Better Outcomes, Better Quality of Life

Despite the identification of JAK mutations and the development of targeted inhibitors, there remain significant unmet needs for patients with myeloproliferative neoplasms. Identification of the myeloproliferative neoplasm populations not currently benefiting from JAK inhibitor therapy highlights the therapeutic deficits still present in this heterogeneous stem cell malignancy. While JAK inhibition has provided significant benefits for patients with intermediate-2 or high-risk myelofibrosis and in patients with polycythemia vera in the second-line setting, JAK inhibitor monotherapy is not approved and not appropriate for all patients with myeloproliferative neoplasms. Continued investigation into additional JAK inhibitors, combination therapy, and novel pathway therapeutics remains key to improving outcomes for all patients with myeloproliferative neoplasms. While therapeutic advances in the JAK inhibitor arena or involving alternative pathways are crucial to improving outcomes in myeloproliferative neoplasms, it is also important to reconsider the role of constitutional symptoms in affected patients as an indication for treatment with agents, such as JAK inhibitors, that can mitigate these debilitating symptoms. In this review, we demonstrate the evolving landscape of clinical investigations that address the important therapeutic needs of patients with myeloproliferative neoplasms.

Introduction

Myeloproliferative neoplasms are a group of myeloid malignancies caused by a hematopoietic stem cell clonal proliferation, the main result of which is primarily either erythrocytosis in polycythemia vera, thrombocytosis in essential thrombocythemia, or progressive cytopenias and splenomegaly in primary myelofibrosis.[1] This group of neoplasms is characterized by a lack of the BCR-ABL fusion protein[2] that is associated with chronic myelogenous leukemia; instead, they are identified with one of three mutually exclusive mutations: JAK2 (Figure),[3] CALR,[4] or MPL.[5] The identification of these mutations has led, through the subsequent development of JAK inhibitors, to changes in diagnostic paradigms,[6,7] prognostication,[8,9] and therapeutic interventions.

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Participants Needed for Patient-Centered MF Study

Dear MPN Community:

Re: Additional MF Focused QoL study focused around Stem Cell Transplant

We would like to invite you to participate in the steering committee for a new study in which we seek funding from the federal Patient-Centered Outcomes Research Institute (PCORI).  This effort is separate (i.e. different PCORI funding mechanism and different trial), but complementary to the efforts of Ruben Mesa and his team focus focusing on alleviating MPN Fatigue in a randomized clinical trial.   This parallel effort includes representatives from the National Marrow Donor Program, and the Center for International Blood and Marrow transplant registry, and MPN investigators focused on stem cell transplant.

Our goal in this study is to comprehensively understand the quality of life in patients who have advanced myelofibrosis to help inform patients and caregivers regarding different therapeutic options they have.  As many patients consider different therapeutic options for advanced myelofibrosis, including clinical trials, standard supportive care, or allogeneic stem cell transplant, there is very little to guide them about the relative impact these options will have on their quality of life.  Although, as providers, we can give estimates of survival, we cannot advise how that survival will look.  In a disease such as myelofibrosis, where symptoms play a major role in the disease, what the patient experiences if often unknown.

We would like to understand this experience.  We plan on doing this through several efforts.  First, to guide our approach, we would like to survey patients regarding their feelings on transplant (link below).  This will explore what factors are important to them when considering a transplant, and why they have or have not considered this therapeutic option.  Using this information, we then will design a clinical trial that will evaluate the trajectory of symptoms and quality of life in patients with myelofibrosis.  This will help identify which patients are likely to benefit from transplant, and who may be likely to benefit from standard supportive care or clinical trials.  We may also identify different areas of concern that could be addressed by interventions or policy change.

The input from the MPN community at large will be critical in designing a subsequent trial to obtain the most relevant information to help patients.  We look forward to your insight and suggestions as they will be of tremendous help in designing the most informative trial we can.

Thank you in advance for your time and consideration.

Here is the link to the live survey: https://redcap2.mayo.edu/redcap/surveys/?s=mjAdrIxJtX

Sincerely,

Jeanne Palmer, MD

Director of MPN Transplant – Mayo Clinic in Arizona

Palmer.jeanne@mayo.edu

 

Ruben A. Mesa, MD Director, UT Health San Antonio Cancer Center

mesar@uthscsa.edu

Novartis Survey Reveals Burden of MPNs on Patients’ Daily Activity and Ability to Work, Calling for More Proactive Symptom Assessment

  • Findings show more than a quarter of myeloproliferative neoplasm (MPN) patients are managed with watchful waiting at diagnosis, despite one in five (22%) reporting moderate to high symptom burden[1]
  • Survey highlights potential disconnect between perceived symptom severity among UK patients and clinicians[2]
  • Data presented at 22nd Congress of the European Hematology Association (EHA) in Madrid, Spain

Novartis today announced new data from the MPN Landmark survey, with clinicians reporting more than a quarter of their MPN patients are managed with watchful waiting at diagnosis[1]. Although most patients in the survey reported low symptom burden, more than one in five (22%) reported moderate to high burden, highlighting the need for proactive and standardised symptom assessment at diagnosis and during the course of treatment[1].

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AOP Orphan and PharmaEssentia announce latest clinical results for Ropeginterferon alfa-2b in PV from 3 abstracts presented at EHA

AOP Orphan and PharmaEssentia announce latest clinical results for Ropeginterferon alfa-2b in Polycythemia Vera from 3 abstracts presented at the 22nd Congress of the European Hematology Association (EHA) in Madrid

– Long-term maintenance treatment of Polycythemia Vera (PV) patients with Ropeginterferon alfa-2b administered every 4 weeks is feasible, efficacious and well tolerated

– Home self-administration of individual doses with a pen is well accepted and expected to support adherence in the long-term treatment of PV-patients

– Disease modifying capability of Ropeginterferon alfa-2b and delay of disease progression in PV-patients is supported by latest clinical and molecular data

– Ropeginterferon alfa-2b is currently under EMA review for marketing authorization in the EU by AOP Orphan, PharmaEssentia intends to seek FDA approval for Ropeginterferon alfa-2b in the U.S.

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Global ET API Manufacturers, Marketed and Phase III Drugs Landscape, 2017-2018, Market Research Report

The Report, “Essential Thrombocythemia-Global API Manufacturers, Marketed and Phase III Drugs Landscape, 2016”, provides comprehensive insights about the marketed drugs, drug sales, Phase III pipeline drugs and their API Manufacturersacross the globe.

A key objective of the Essential Thrombocythemia Report is to understand the market and pipeline status of the drugs around the Essential Thrombocythemia to explore the generic development opportunities, licensing opportunities and to gain competitive advantage on designing pipeline startegies. The Report provides the historical and forecasted sales of the drugs till 2018.

The Report gives insights into patents providing the patent protection data and marketing exclusivity of all the drugs across the Essential Thrombocythemia.

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