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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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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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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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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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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The MPN Yoga Study: A Patient’s Story

MPN Patient Amy Wang Manning, Portland, Oregon

Amy Wang Manning.

As I typed one day at work this past spring, my left pinkie suddenly didn’t feel right. A moment later, my left thumb curled under; I couldn’t straighten it except by pulling it with my right hand. Then my entire left hand stiffened into a useless claw. And a tingling sensation was spreading rapidly up my left arm and to my shoulder. I told my boss I needed medical attention. Soon I was in a hospital emergency room, undergoing an MRI.
I didn’t have a stroke that March day. But several weeks later, a hematologist oncologist gave me a diagnosis I’d never heard of: essential thrombocytosis (ET), based on the clot I’d experienced, along with a platelet count of over 1 million and a positive test for the JAK2 mutation. He said I’d probably had ET for as long as 20 years. Suddenly, a lot of health symptoms and setbacks that I’d experienced over that time period made sense.
While I researched essential thrombocytosis online, I stumbled across a call for participants in a pilot study at Arizona State University. The researchers wanted to investigate whether patients with essential thrombocytosis and other myeloproliferative neoplasms could find some relief from symptoms such as pain, fatigue and insomnia by using a mobile app for guided meditation.
While I hadn’t experienced much pain, I’d been having bouts of fatigue and I’d struggled for years with insomnia, which seemed to be worsening. I had practiced yoga and found it helpful in managing my emotional, mental and physical health, so meditation seemed appealing. It certainly couldn’t hurt. And the eight-week study wasn’t asking much of participants: Fill out a few questionnaires, keep a daily sleep log, and wear a Fitbit to track my daily activity. I applied to the study and was accepted. The daily meditation sessions turned out to be very reasonable, just 10 minutes a day.
Now that the study’s finished, I feel calmer and more able to handle whatever comes my way with this disease. I continue to meditate with the app I tested. If nothing else, I am now better equipped to take a deep breath, let go of what I cannot control, and just focus on the moment – this moment, in which I am still here, living.

ASH Abstract: Imetelstat Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells

1654 Imetelstat, a Telomerase Inhibitor, Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells

Treatment of myelofibrosis (MF) patients with imetelstat (Imet), a telomerase inhibitor, has been reported to lead to clinical, morphologic and molecular remissions in a subset of patients (Tefferi A, et al. N Engl J Med. 2015; 373:908), suggesting that Imet has disease-modifying activity. The precise mechanism by which Imet induces such responses has however not been reported. In this study, we investigated the effects of Imet on MF hematopoietic stem/progenitor cells (HSC)/(HPC) to address this question.

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Impact BioMedicines Presents at ASH on Fedratinib

Impact Biomedicines Presents Analysis at the 2017 ASH Annual Meeting Suggesting that Fedratinib Did Not Increase Wernicke Encephalopathy Risk in Phase 2 and 3 Myelofibrosis Clinical Trials

SAN DIEGO–(BUSINESS WIRE)–Impact Biomedicines today presented a case review on fedratinib, a selective oral small molecule JAK2 kinase inhibitor that is being developed for the treatment of myelofibrosis (MF) and polycythemia vera (PV), in a poster session at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.

The poster titled “Case Series of Potential Wernicke Encephalopathy in Patients treated with Fedratinib,” demonstrated that patients treated with fedratinib in clinical trials did not experience a decrease in thiamine levels, and the prevalence of Wernicke Encephalopathy (WE) in the trials was less than originally perceived for patients with myeloproliferative neoplasms.

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Incyte Reports Four Year Phase 3 Data Analysis Shows Durability of Response of Jakafi (ruxolitinib) in Patients with PV

Incyte Corporation (NASDAQ: INCY) today announced new 208-week (4-year) follow-up data from the ongoing, global, multi-center, open-label Phase 3 RESPONSE study of Jakafi® (ruxolitinib) comparing the efficacy and safety of Jakafi with best available therapy (BAT) in patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU). The pre-planned data analysis showed a durable primary response to Jakafi in patients with PV who are resistant to or intolerant of HU and the overall safety profile for Jakafi remained consistent with previously reported 80-week RESPONSE data.1 The results were shared in an oral presentation today at the 59th American Society of Hematology (ASH) Annual Meeting 2017 in Atlanta, Georgia.

“With 30 months of additional follow-up, the four-year RESPONSE data analysis presented today at ASH further reinforces the potential of Jakafi as a long-term option for patients with PV,” said Peg Squier, M.D., Ph.D., Head of U.S. Medical Affairs at Incyte. “Given the few treatment options available to treat this chronic and progressive blood cancer, these long-term safety and efficacy data are meaningful to patients with uncontrolled PV.”

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Secondary Myelofibrosis Recruiting Phase 1 Trials for DB00493 (Cefotaxime)

This pilot clinical trial studies the side effects of combination chemotherapy, total body irradiation, and donor blood stem cell transplant in treating patients with secondary myelofibrosis. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving combination chemotherapy and total body irradiation before a donor blood stem cell transplant helps to stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets.

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