Update on Impact BioMedicine’s Fedratinib

ASH Highlights

Many patients wondered what happened to Fedratinib, the drug Sanofi had in trials several years ago.  Amid some controversy, the drug was shelved, although many patients were experience positive responses. Fedratinib is back. It is a selective JAK2 Inhibitor, whereas existing treatments target both JAK1 & 2. Spleen reduction will likely continue to be a primary endpoint. For those patients resistant to current treatments, Fedratinib could be a potential option in the near future.

In the JAKARTA phase 3 trial, fedratinib had a ≥35% reduction in spleen volume at week 24 in 46.9% of myelofibrosis patients and a ≥50% reduction in total symptom score in 37.1% of patients. The only grade >2 TEAE seen notably more often in 400mg fedratinib vs. placebo by cycle 6 placebo cross-over was anemia (common to all Jak2 inhibitors). We know there is a significant unmet need in the MF patient population and we’re working diligently to get fedratinib to market to address this unmet need.  In JAKARTA 2 fedratinib had a ≥35% reduction in spleen volume at week 24 in 55.4% of myelofibrosis patients that were previously intolerant or resistant to ruxolitinib treatment.

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ASH Highlights- Foggy Brain: Side Effect or Vitamin Deficiency?

Marina Sampanes Peed

Many of us in the MPN world have experienced:

  • read a page and can’t recall what you just read
  • forget events, tasks, or activities that you used to remember
  • difficulty in thinking clearly

We jokingly call it “chemo brain” and chalk it up to various medications. But what if it’s caused by something else?

According to Catriona Jamieson, MD, PhD, University of California San Diego Cancer Center, it is well-documented that deficiency of thiamine pyrophosphate deficiency (vitamin B-1) causes what we refer to as “chemo brain” or “foggy brain.” Extreme cases of thiamine deficiency can cause Wernicke’s encephalopathy (biochemical lesions on the brain), that affect vision, confusion, and memory.

Causes of thiamine (Vitamin B-1) deficiency

The body doesn’t produce essential vitamins; they must be ingested through eating healthy foods to maintain normal health.

Cancer cells metabolize faster than normal cells and they draw upon the body’s nutrient resources including glucose and micronutrients such as vitamins niacin, folic acid, pantothenic acid, pyridoxine, biotin riboflavin, and thiamine (Vitamin B1).

It’s quite common for patients to forget their nutritional needs when appetite is suppressed due to splenomegaly or certain medications.

Just one of these factors is enough to cause foggy thinking. When experiencing disease progression, the cancer cells are dominating the use critical nutrients.

Weigh the Risks

An association between cancer and low thiamine levels is demonstrated in several reports. The use of supplemental vitamins to modulate cancer rates has been promoted and discounted for years.

Some argue thiamine supplements may contribute to tumor cell survival, proliferation, and chemotherapy resistance.   Other studies suggest that very high dose thiamine produces growth inhibition of malignant cells.

What’s the answer?

Like many other elements, there is no single one size fits all answer. Talk with your hematologist about possible thiamine deficiency. If it’s low, proper dosage of Vitamin B-1 might clear the fog. Vitamins are medicine for your body, so It is important to make any changes with the knowledge and guidance of your physician.For more information check out these articles:

The Role of Thiamine in Cancer: Possible Genetic and Cellular Signaling Mechanisms

Linking Vitamin B1 with Cancer Cell Metabolism


MPN Expert Ruben Mesa on the Anemia-Based Data at ASH

At the 59th American Society of Hematology (ASH) Meeting and Exposition, Rare Disease Report caught up with Ruben Mesa, M.D., Director of the University of Texas Health Cancer Center.

In this video, he discusses the common problem of anemia that can come with myeloproliferative neoplasm (MPN)-associated myelofibrosis, how some of the medications administered can induce it, and the data pertaining to it that was presented at ASH.

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Ruxolitinib Beneficial in Hydroxyurea Resistant/Intolerant Polycythemia Vera

Data presented at the 2017 American Society of Hematology Annual Meeting (ASH 2017) are confirming previous studies suggesting that patients with polycythemia vera (PV) who fail hydroxyurea and are treated with ruxolitinib may have better hematocrit control. Researchers reported that patients treated with ruxolitinib appear to experience benefits in terms of hematocrit control, hematologic remission, and reduction in spleen size.

The RESPONSE study demonstrated that this potent JAK1/2 inhibitor results in superior response rates compared with best available therapy in controlling hematocrit and improving splenomegaly and symptoms in patients with PV whose disease was inadequately controlled with hydroxyurea. Following initial trials, an expanded-access phase 3b study was conducted looking at ruxolitinib in patients who were hydroxyurea resistant/intolerant. The study included patients who had no other treatment options available and were not eligible for any ongoing clinical trial.

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Treatment Decision Support Tool Analysis Suggests Many Clinicians Suboptimally Manage Patients With PV and MF

Many clinicians suboptimally manage patients with polycythemia vera (PV) and myelofibrosis (MF), according to an analysis of data captured from an online tool developed by Clinical Care Options (CCO) in collaboration with a panel of PV and MF experts. The analysis was presented by Ryan Topping, PhD, at the 59th ASH Annual Meeting & Exposition in Atlanta, Georgia.

Treatment for PV and MF continues to be refined, resulting in educational gaps for many healthcare providers (HCPs) who manage these conditions. In collaboration with a panel of PV and MF experts, CCO developed an online treatment decision support tool to assist HCPs in making optimal management choices for patients with PV and MF. HCPs were asked to enter patient case details into the decision support tool and were surveyed on their intended treatment for the case. They were then provided with management choices for that case from the expert panel.

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Questions to ask your Hematologist

ASH Abstract: Effective Treatment of Low-Doses Decitabine in MPNs with JAK2/V617F

Primary myeofibrosis (PMF) is one of the Ph negative myeloproliferative neoplasms (MPN). The mainly clinical features are obviously physical symptoms and symptomatic splenomegaly. It may be converse to leukemia and has shortened life expectancy. Nowadays, the therapy of PMF is aimed at maintaining comfort and there was no effective treatments. PMF complicated with myelodysplastic syndrome (MDS), which is named as MDS/MPN-u, is a rare case, and the treatments are confused. In this study, we want to discuss an effective treatment in MDS/MPN via a case therapy and literature review.

A 55-year-old woman presented with fatigue and chest distress for one month was admitted in our hospital. Physical examinations showed anemic appearance and splenomegaly which was four fingers under lib. A routine blood count test showed pancytopenia. A bone marrow examination showed dysplasia and fibrous tissue proliferation. The JAK2/V617F mutation was positive and the expression was 60.63%. The chromosome karyotype showed 47, XX, t (1; 20) (p11.2; q11.2), +9,-13, +21. She was diagnosed as PMF complicated with MDS (MDS/MPN) according to WHO 2016 version of hematologic neoplasms classification. She received thalidomide 100mg daily therapy combined with prednisone.

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Reappraising the Approach to PV and ET

Description of recurrent genetic abnormalities in driver genes, a better appreciation of the key diagnostic role of bone marrow features, results of large epidemiologic studies, and landmark controlled clinical trials have resulted in a reappraisal of the approach to polycythemia vera and essential thrombocythemia, authors noted in a recent review.

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The Effects of Myeloproliferative Neoplasm Symptoms on Quality of Life

A secondary analysis being presented at the 59th Annual Meeting of the American Society of Hematology in Atlanta, Georgia, found that all individual symptoms of myeloproliferative neoplasms (MPNs) correlate with quality of life (QoL).
“Patients with MPNs are faced with high disease-related symptom burden and QoL decrements,” wrote the authors.
Previous study results have shown that symptom burden measured from the MPN Symptom Assessment Form (MPN-SAF) Total Symptom Score has a strong correlation with QoL measured by the Global Health Status/QoL (GHS/QoL) scale of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30). However, according to the authors, an analysis of predictors of QoL in this population has not been performed.

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Go to MPN Quality of Life Survey


PharmaEssentia Announces Favorable Two-year Results of Ropeginterferon Alfa-2b in PV at ASH

 At 24 months Ropeginterferon alfa-2b:

Demonstrated superiority over best available treatment
Achieved high rates and durable clinical and hematological response
Confirmed favorable safety and tolerability profile beyond 24 months
Further demonstrated disease modifying capability

PharmaEssentia is currently working with the U.S. FDA for submission of a biologics license application (BLA) for Ropeginterferon alfa-2b for Polycythemia Vera (PV) in the U.S.

AOP Orphan’s submission for marketing authorization of Ropeginterferon alfa-2b in the EU is currently under review by EMA

WALTHAM, Mass.Dec. 10, 2017 /PRNewswire/ — PharmaEssentia USA, a subsidiary of PharmaEssentia Corporation (Taipei Exchange: 6446), today announced the latest follow-up results of Ropeginterferon alfa-2b from the ongoing, long-term, follow-up trial CONTINUATION-PV (CONTI-PV) for patients with Polycythemia Vera presented during an oral presentation at ASH 2017. The CONTI-PV trial is being conducted by AOP Orphan Pharmaceuticals AG (AOP Orphan) in Europe.

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon. It is administered once every two weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide. PharmaEssentia discovered and manufactures Ropeginterferon alfa-2b and has exclusively licensed the rights for the novel molecule in the field of Myeloproliferative Neoplasms (MPNs) to AOP Orphan for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

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