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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Samus Therapeutics Announces Granted Orphan Drug Designation in Phase 1b Study in MF

BOSTON, June 13, 2018 /PRNewswire/ — Samus Therapeutics, Inc. (“Samus” or the “Company”), a privately held Boston-based biopharmaceutical company developing novel therapeutics and biomarkers targeting the epichaperome, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PU-H71 for the treatment of myelofibrosis. The Company also announced today that it has dosed the first patient in a Phase 1b dose-escalation study of PU-H71 in patients with myelofibrosis.

PU-H71, along with PU-AD, are novel therapeutics that, with high specificity, target the epichaperome, a foundational protein complex present in multiple disease states, including cancer and neurological disorders. Under conditions of abnormal stress, cells become biochemically ‘rewired’ to form a network of high-molecular-weight complexes known as epichaperomes. In cancer, these can function as a network to enhance cellular survival, irrespective of tissue of origin or genetic background. PU-H71 specifically targets the epichaperome, present in over half of all cancers tested by Samus and the Chiosis group at Memorial Sloan Kettering Cancer Center, to precisely interfere with the epichaperome function in diseased cells and to not affect normal cells. (Nature Reviews Cancer, 2018, doi:10.1038/s41568-018-0020-9; Nature, 538, 397-401, 2016).

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Exploring MPNs with Ruben Mesa, MD: What’s on the Horizon?

Ruben Mesa, MD, FACP

At the American Society of Clinical Oncology (ASCO) 2018 annual meeting in Chicago, Rare Disease Report® sat down with Ruben Mesa, MD, director at Mays Cancer Center at UT Health San Antonio and MD Anderson Cancer Center, to discuss myeloproliferative neoplasms and other rare cancers.

Rare Disease Report® (RDR®): As an expert in myeloproliferative neoplasms (MPNs), can you speak to your experience with MPNs and what your approach has been with these rare diseases?

Mesa: The matter of myeloproliferative neoplasms, as I tell my patients, [is they] are not rare or common cancers. What I share with folks in the era of precision medicine and precision oncology [is that] we find, to some degree, all patients have a rare disease. [That’s] because however their disease affects them, whether it’s something that you don’t hear much about, like myelofibrosis or polycythemia or even if it’s something [common] like breast cancer, there’s probably a thousand different subtypes, each one of which is kind of rare in and of itself.

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A Mother’s Story: Coping with a Sick Child

Young MPN patient “Jedi” with his companion Chewy

In the opening credits of the television show “The Fresh Prince of Bel Air” Will Smith sings, “This is the story/all about how/my life was turned/upside down.” This article is the story all about how OUR lives were turned upside down when our son, who we affectionately refer to as a “Jedi” because of his special blood, was diagnosed with a Myeloproliferative Neoplasm (MPN).

Our story is probably different from other adults/children with an MPN because Jedi wasn’t extremely sick before we discovered he had an MPN. For about two years, Jedi had experienced a variety of unexplained health issues — random fevers, flu, an estimated seven times over the preceding twelve months, and extreme pain in his legs. At the time, I attributed these conditions to allergies, or growing pains, things that boys normally experience at that age. This soon changed, however, when I took him in for his annual physical exam. The doctor suggested a blood test for Jedi after hearing about his recent health issues. I am not one who immediately agrees to testing, but I agreed when the doctor said to me, “if it was my son, I would do the test.”  A week later, the doctor called and told me the blood test was contaminated and to immediately re-test Jedi, which we did. Two weeks later, I received a phone call from him, who told me he believed Jedi had Essential Thrombocythemia (ET), a condition I had never heard used before. His doctor then recommended we see a specialist who specialized in ET. It can be hard to diagnose a child with a MPN, because it is so rare. However, the doctor was a General Practitioner and had seen it in other adults. Thus, Jedi didn’t get extremely sick before diagnosis as so many of the children do.

The following Monday, I called the recommended specialist. The scheduler answered the phone by stating the name of the organization, which was “something something oncology.” Her words, more specifically one word – oncology – startled me. I held it together long enough to explain who I was and to ask for an appointment. Once the call was completed, I hung up the phone, closed my door and cried. Oncology? Why was an oncologist being recommended to see my beautiful eight-year old?

The results of Jedi’s first bone marrow biopsy revealed he had the JAK2 gene mutation. Jedi asked what a gene mutation was. I frantically tried to explain a gene mutation to my child. My first instinct was to tell him he was a mutant. I knew his next question was going to be, “What is my superpower?” I didn’t have an answer for that question, but realized he is like a Jedi, who has midi-cholorians, or special blood. That is what I explained to him.

Pediatric MPN Specialist Dr. Nicole Kucin, MD, MS, New York Presbyterian Hospital/Weill Cornell Medicine. 

After initial difficulty finding a specialist who understood MPNs, we now have a talented team of specialists. He sees a local doctor every month. He also sees Dr. Nicole Kucine, MD, MS, an MPN specialist with Weill Cornell in New York City, click here to learn more. Dr. Kucine is performing a study on children with MPNs through the National Institutes of Health (NIH). If you have a child who has an MPN, I highly recommend contacting her. Last, Dr. Srdan Verstovsek (aka “Dr. V”) who is affiliated with MD Anderson in Houston, Texas, is part of the team. He is an Adult MPN specialist but performs a lot of work related to the JAK2 mutation. He has agreed to consult with Jedi’s Pediatric Hematologist, Dr. Michael Rytting, who is also at MD Anderson. As a result of Dr. V and Dr. Rytting’s recommendation, we have changed Jedi’s treatment plan from Hydroxyurea (HU) to Interferon.

In determining the appropriate treatment plan, some questions we asked were:

What are our options for treatments? What is the difference between each treatment? Are there timeframe limitations for each treatment? (The effectiveness of one of the drugs used to treat ET is limited to 5 years. That was information I did not know but extremely important to know given Jedi’s age – now nine years old!)

Are there any other patients using this same treatment? Have they experienced any side effects not listed on the medicine? Is the basis for our understanding of how this treatment affects the patient based on a different disease? For example, HU is commonly prescribed to patients with sickle cell anemia. Some doctors’ understanding of how HU impacts a patient is based on the their patients who have sickle cell anemia, which is a completely unrelated disease.

Additional questions to consider:

Is there a way to mitigate side effects?

Can we start with a lower dose and see if it works?

How long will it take for the medicine to start working?

What are the risks of not taking any medicine? What are the risks of taking this medicine?

Is there any research being done on these treatments?

In finding a local doctor, some questions we asked are:

Are you willing to work with other specialists in this field of medicine?

How do you propose to communicate with them?

Are you willing to follow the specialist’s instruction when treating my child?

Are you willing to consider diet as part of the treatment?

There a few ways to connect with other with MPN patients. I have gotten great information from Facebook support groups. Attending MPN conferences is another way to become informed and connected. We attended an MPN conference in February. Listening first-hand to specialists providing updates in the field and answering questions was like drinking from a firehose. Thankfully, MPN Advocacy & Education International posted the videos on the website, which allowed me the opportunity to repeatedly watch them to fully absorb the information the specialists provided, view conference videos. Being able to converse with the attendees at the conference was also extremely helpful. They shared their first-hand experiences and provided insight into what my child is going through. It is more difficult for a child to describe how he or she feels because what he or she experiences on an everyday basis is their “normal.” By sharing their experiences with me, the attendees were able to help me find the words to help my child describe how HE is feeling.

Part of this disease is a feeling of loneliness – for Jedi, Jedi’s brother (our other son), and my husband and me. Unlike more common disorders, finding and becoming part of a support group can be difficult for those with an MPN—especially since it is so rare in a child. That is why it is important for us to participate in conferences whenever possible. This Fall MPN Advocacy & Education Int’l is hosting a conference especially for children and young adults with MPNs. This is a fantastic opportunity for both parents and children to meet and get to know one another. We plan to attend this conference. Learn more about the Pediatric MPN event.

Finally, a plea to adults with an MPN. Please consider using the resources the MPN groups has provided, such as the tool that tracks symptoms. I know it can be concerning to share that information with a third party. (Believe me, I am wary of doing that myself.) But, any information YOU provide will help those that come behind you. Working together, we can collectively help each other and future generations better understand how to combat and defeat these diseases.



FDA Approve Investigational New Drug Application for CK0801 for Bone Marrow Failure Syndromes

HOUSTON, June 10, 2018 /PRNewswire/ — Cellenkos™, Inc., a clinical-stage biotechnology company, today announced that the United States Food and Drug Administration has cleared the Investigational New Drug (IND) application which allows for Cellenkos™ to proceed with a phase I clinical trial of CK0801, 3rd party cord blood derived regulatory T cells, in patients with bone marrow failure syndrome including aplastic anemia, myelodysplastic syndrome and myelofibrosis.  Cellenkos™ submitted its IND application on May 11, 2018.  The Phase I clinical trial of CK0801 will commence in the third quarter of 2018 at The University of Texas MD Anderson Cancer Center, Houston, Texas.

The clearance of this IND is a significant milestone for Cellenkos™ and for its’ platform technology, CK0801, as part of the overall clinical development plan of demonstrating adoptive cellular therapy with CK0801 to treat autoimmune diseases and inflammatory disorders”, said Founder and Chief Medical Officer, Simrit Parmar, MD.  “We are excited to launch the development of CK0801 as a novel cellular therapy approach to treat bone marrow dysfunction arising due to irregularities in the patient’s immune system.  CK0801 works to replace and replenish the defective regulatory T cells and blocks the uncontrolled inflammatory signal generated by the cytotoxic T cells against patients’ own tissues. Such an approach may lead to calming of the bone marrow inflammatory microenvironment as a result of the infused CK0801 which may then lead to the reversal of the hematopoietic arrest that will potentially translate into clinical improvement.”

Tara Sadeghi, Senior Clinical Director, added, “The US FDA’s clearance of this IND validates our commitment towards creating an ‘off-the-shelf’ cord blood derived regulatory T cell therapeutic platform for treating autoimmune diseases.  We are confident and eager that this Phase I study will confirm the safety of CK0801.  We remain grateful to all our colleagues and partners in academia, whose constant support and guidance allows us to take this step forward.”

About Bone Marrow Failure Syndromes
Bone marrow failure syndrome refers to the decreased production of one or more major hematopoietic lineages (white cells, red cells and platelets), which leads to diminished or absent hematopoietic precursors in the bone marrow.  Bone marrow failure remains an incurable disease except for those patients who are eligible to receive an allogeneic stem cell transplantation.  For a vast majority of these conditions, the role of immune dysfunction and increased inflammation in the bone marrow is increasingly recognized as important in both the inception as well as maintenance of the defective production of blood cells.

About CK0801
CK0801, is a first-in-class, cord blood-derived T-regulatory cellular product, developed using Cellenkos‘s proprietary manufacturing platform, that overcomes immune dysfunction by inhibiting key regulators of inflammation.  CK0801 contains healthy and robust regulatory T cells derived from donor cord blood units and adoptive therapy with CK0801 has the potential to replenish the immune system and impact patient care by providing novel, non-pharmacologic options.  CK0801 will be manufactured at the Cellenkos FDA registered manufacturing facility located in Houston, Texas.

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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.

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


MPN Advocacy and Education International hosted its 3rd MPN Patient/Caregiver program at the Cleveland Clinic on May 31. Dr. Aaron Gerds presented information on two MPN topics, Symptom Burden and MPNs 101.

Dr. Gerds helped explain the biology of MPNs and likened the JAK2 mutation to a broken thermostat. In this case, constantly triggering the body to turn up the heat (see slide). His discussion on symptom burden provided a keen insight into why patients endure extreme fatigue, itching and other constitutional issues. Dr. Gerds recommended reviewing the yoga study. The majority of patients who participated found some level of relief and improvements in their quality of life. (read more about the yoga study)


Dr. Betty Hamilton, Cleveland Clinic, presented on bone marrow transplants for MPN patients and how to determine the optimal time to proceed. Are the current treatments reducing the symptom burden? Is the patient healthy enough to recover? It is a matter of finding that point at which it will maximize survival and improve the patient’s quality of life. It isn’t age as much as the overall fitness of the patient. One of the most common issues for patient’s recovering from transplant is graft vs host disease, which affects more than half of transplant patients.

Dr. Kristen Pettit, University of Michigan, gave an overview of clinical trials and the benefits of participation. Patients who participate receive quality care from MPN experts.  The protocols in place are designed to ensure the utmost safety of the patient. Participating in a clinical trial is a big commitment. Patients should be aware of all the necessary requirement.

Click here for a list of questions to consider before participating in a trial

Click here for a list of current mpn clinical trials


Dr. Naveen Pemmaraju, MD Anderson, discussed what is on the horizon for MPN patients. He encouraged physicians to pay attention to what patient’s are dealing with beyond the visible symptoms. “No one understands the war going on inside your body.” MD Anderson is currently working on several clinical trials for MF patients:

Evaluation of Ruxolitinib in Combination With PU-H71 for Treatment of Myelofibrosis.

Testing of SL-401 in Advanced, High Risk MPN Patients


From left, Dr. Aaron Gerds, Dr. Naveen Pemmaraju, Dr. Kristen Pettit and Dr. Betty Hamilton

Structure of protein pair provides blueprint for future drugs

Walter and Eliza Hall Institute researchers have visualised for the first time how the protein SOCS1 ‘switches off’ cell signalling to dampen immune responses and block cancer growth.

The atomic-level structure of SOCS1 binding to its partner protein JAK could guide the development of drugs that alter disease-causing cell signalling pathways, and may have applications for treating some blood cancers, including leukaemias.

The research, led by Dr Nick Liau, Dr Nadia Kershaw, Associate Professor Jeff Babon and Professor Nick Nicola, was published in the journal Nature Communications.


– The SOCS1 protein binds to JAK proteins to ‘switch off’ cell signalling, which dampens processes including immune responses and cancer growth.

– Our researchers have used structural biology to visualise how SOCS1 binds to JAK proteins in never-before seen detail.

– The detailed structure may guide the development of new drugs that modify JAK activity, amplifying or dampening cell responses, with potential applications in cancer therapies.

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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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