Startup Impact Biomedicines Raises $22M to Bring Fedratinib to MF Patients

SAN DIEGO–(Business Wire)–Today, Impact Biomedicines (“Impact”) announced its launch to pioneer the development of life-changing treatments for patients with myeloproliferative neoplasms and other cancers. The Company’s pipeline is centered around fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV). In conjunction with this launch, Impact is pleased to share that the U.S. Food and Drug Administration (FDA) has removed the clinical hold placed on fedratinib and that the company has received $22.5 million from Medicxi through a Series A financing.

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3rd Annual Women & MPN Conference Videos

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We are pleased to have completed the 3rd annual Women & MPN conference on Friday, September 29th, in Los Angeles.  We had wonderful speakers and a lively panel discussion on issues impacting women and all those living with MPNs. One recurring theme was revealed by the newly diagnosed attendees; their physicians didn’t seem to know a lot about their ET, PV or MF and gave little support or direction to resources. MPN Advocacy & Education International is committed to a more vigorous outreach program to physicians and will continue to provide direction, education and resources to all patients and caregivers lacking the support they need.  Our special guest, Emmy-award winning actress, Finola Hughes, will be a great spokesperson for the MPN community because of her ability to reach much greater numbers through the media.


 

Panel Discussion


My Child is Being Evaluated for an MPN

How to Approach the Conversation with Your Child’s Hematologist

Dr. Nicole Kucin, MD, MS Assistant Professor of Pediatrics, Pediatric Hematology/Oncology, New York Presbyterian Hospital/Weill Cornell Medicine.

By Nicole Kucine, MD, MS
It can be difficult to know what to expect when your child is being evaluated for an MPN. There is limited guidance on the internet, which can make specialist visits overwhelming to families. As parents, you can expect that you and your child will be asked many questions about symptoms he or she might be having, including headache, abdominal symptoms, rashes, and itching. Your child will undergo a number of blood tests, including some genetic tests, as well as a bone marrow examination. Children who are having symptoms specific to a certain body area may need radiologic tests (such as an ultrasound or MRI.) You should feel free to ask anything you want, and make sure if you are searching the internet you look at reliable sources (such as the MPN Research Foundation, MPN Advocacy Education International, or cancer.gov.) Some of the questions I am often asked include the following:

Does my child have cancer?
This is a tricky question, and others may disagree, but I do not think of children with MPN as having cancer. In adults, the World Health Organization criteria considers MPN to be chronic forms of leukemia. We are still evaluating MPN in children, and at this point it is not clear that they are all the same disorders in kids as they are in adults. I view the classical MPN in children as chronic bone marrow disorders, and while they have the potential to transform to acute leukemia, this is not something that has been reported in the literature. By envisioning these as chronic illnesses, I think it helps to set the expectations for long-term follow-up and aiming for keeping day to day activities as normal for your child as possible.

Does my child need the bone marrow evaluation?
The answer to this is definitely yes. The bone marrow exam is an extremely important part of the diagnostic process. It can provide a lot of information about what is going on with your child’s blood cells at the source. Things like storage iron, fibrosis, and the appearance of the precursor blood cells are studied. The procedure itself is performed with anesthesia to make sure your child is asleep during the bone marrow test and doesn’t remember it. The pain following a bone marrow test is generally very mild, and children may not require any pain medicine or might require a dose of Tylenol.

What type of MPN does my child have?
The diagnostic criteria for the various types of MPN are based on years of study and data on adult patients. They include features like appearance of bone marrow cells, genetic findings, and lab criteria. Making the appropriate classification for adult patients is important for discussions of prognosis and treatment. We do not yet know if we can directly apply these criteria to children, and knowing the exact type of MPN each child has may not be possible. While it is important to gather all of this information in children, it may not be as important to specifically name the exact type of MPN, and “MPN, unclassifiable” is an appropriate diagnosis for a number of children. The decisions about how to counsel families, what treatments may be recommended, and what follow-up is needed, will be made based on a variety of findings.

Does my child require treatment for his or her MPN?
The answer to this question varies, as I do not believe treatment is required for all children with MPN. I usually determine the need for treatment based on an individual child’s symptoms and lab findings. I generally do not recommend treatment for children who are asymptomatic and have reassuring labs. Children with mild symptoms in the setting of a high platelet count can often benefit from low-dose aspirin, as long as they are not showing evidence of bleeding or acquired vonWillebrand disease. Children with high red blood cell counts can also benefit from low-dose aspirin or phlebotomy. When a child has a severe clinical event such as a blood clot, or does not have improvement of symptoms with initial therapy, then cytoreductive therapy is appropriate. Which medication is used should be decided based on a conversation with the family and the treating doctor. I have been asked about what is my “cutoff” for high platelet or red cell counts for treatment, and there isn’t a standard cutoff. For example, I don’t think an asymptomatic child with a platelet count of 1.3 million necessitates treatment if they are feeling well and otherwise healthy. If you asked 10 different hematologists, you would probably get 10 different opinions on when to treat, so I think it’s important to have a conversation with your hematologist about the risks and benefits of different treatments.

While it can be frustrating to be facing a rare disease, there is ongoing research to help us better understand these conditions in children and adolescents. Keeping an open mind and making sure you have good communication with your child’s hematologist is the most important thing you can do.

Contemporary Use of Interferon Therapy in the Myeloproliferative Neoplasms

The purpose of this article is to review the current evidence behind interferon therapy in patients with myeloproliferative neoplasms.

Recent Findings

Preliminary analysis suggests that interferon may be non-inferior to hydroxyurea in patients with polycythemia vera and essential thrombocytosis. Responses have been observed regardless of JAK2 mutational status, but the presence of non-JAK2 somatic mutations may negatively influence response rates.

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AMD in Patients With Chronic MPNs

Question  Do patients with chronic myeloproliferative neoplasms have an increased risk of age-related macular degeneration compared with the general population?

Findings  In a large Danish registry-based nationwide cohort study including 7958 patients with myeloproliferative neoplasms and 77 445 age- and sex-matched controls, the risk of age-related macular degeneration was increased for patients with myeloproliferative neoplasms, after adjustment for smoking and risk time.

Meaning  These data suggest that patients with myeloproliferative neoplasms have a higher risk of age-related macular degeneration than the general population, supporting the possibility that systemic alterations may be involved in the pathogenesis of age-related macular degeneration.

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Study Administers IMG-7289 to First Patients with MF

The first patients have been dosed in a Phase 1/2 clinical trial of IMG-7289 for the treatment of myelofibrosis (MF), according to Imago BioSciences.

The clinical-stage pharmaceutical company is developing novel therapies for hematological and inflammatory diseases. Myelofibrosis is characterized by the production of inflammatory cytokines, and IMG-7289 is an investigational lysine-specific demethylase 1 (LSD1) inhibitor intended to suppress that production.

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MPN Research Foundation Launches myMPN During Blood Cancer Awareness Month

Individuals living with Essential Thrombocythemia, Polycythemia Vera and Myelofibrosis are now invited to participate in the first ever patient advocacy driven registry for people with an MPN (myeloproliferative neoplasm). This new initiative is called myMPN and was launched in September at the beginning of Blood Cancer Awareness Month. Information about this project and the link to enroll can be found at http://www.mpnresearchfoundation.org/myMPN.

Through myMPN the MPN Research Foundation provides patients with an opportunity to share information about their experience with MPN with researchers. The “How do you feel today” survey records symptom data in real time. There is a “Health Event” survey for people who have had a change in diagnosis, medication change, hospitalization or other medical event they’d like to include in myMPN. Participants will be able to see how they compare to others in myMPN. Almost 100 people are already participating on a regular basis.

Dr. Ruben Mesa is the chair of myMPN’s steering committee which also includes representatives from Mayo Clinic, Guys and St. Thomas’ Hospital in London, Mt. Sinai School of Medicine, Oregon Health Sciences University, MD Anderson and Johns Hopkins University. The Steering Committee provided feedback and oversight for the protocol and surveys that are included in myMPN and will help set data access guidelines and lead the evolution of the surveys over time.

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September Blog: Answers to Your Intimacy Questions

Dr. Laura Michaelis, MD

How do I know if I’m having ET symptoms or menopause symptoms, i.e. night sweats, foggy brain etc.?

There is no one answer for this – and many of the symptoms of essential thrombocythemia may be confused with “normal” symptoms of menopause. However, there are some tell-tale signs that ET is the root cause. For example, night sweats are different than hot flashes. Night sweats typically occur while sleeping and characteristically cause soaking sweats – like you have to change your pajamas or the bedding. Night sweats are not subtle. In contrast, most women describe hot flashes as a flush that comes on at any time of the day (or night) and while uncomfortable, doesn’t typically cause profuse sweating. If you are having migraine headaches, visual auras, painful rash of the extremities — called “erythromelalgia,” these are more common with ET and warrant a discussion with your physician.

Sex is difficult since my diagnosis. My husband is cautious and doesn’t want to impose himself on me, and I cannot get my head into it. I’m consumed with thoughts of the disease and possible progression.  Can you offer an insights?

Many individuals, when faced with a life changing diagnosis, find that anxiety and depression about the diagnosis can affect the normal activities of life, whether or not it’s sexual activities or libido, work, sleep or other relationships. It is very, very common for people to experience grief reactions or event depression with diagnosis. Depression and anxiety are very treatable conditions.

In your situation, I would strongly recommend seeking out a psychotherapist or psychiatrist and discussing that your worries about your disease are impeding your normal function. You might also have your husband join you at some of these sessions. Finally, don’t forget to mention this to your hematologist as well, who might be able to give some advice with regard to a medical approach. The most stressful things in life: death, divorce, illness often cause humans to experience grief, which can also take the form of anxiety or depression. There is excellent treatment for this in the form of therapy and medication. Please talk to a professional.

I know intimacy is many things unrelated to sex but sometimes the fatigue overtakes me and I simply don’t care if I ever have sex. I also fall asleep when we try to be close.

Fatigue is a very common symptom of the MPNs, including the “lower-risk” MPNs including polycythemia vera and essential thrombocythemia. This can impede normal sexual activity and libido. One option is to try creative, non-medical, interventions. Think about what times of the day you are at your best. Perhaps timing your intimate encounters to be at a time of day when you feel more energized? Experiment with alterations in your diet – are there some foods that make you sleepy? If so, you can avoid those when you want to remain energized. Certainly, avoiding alcohol might help to prevent fatigue on “date nights.” A lack of libido can also be a sign of depression and certain

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