Pacritinib Can Fill Unmet Need in Myelofibrosis

The investigational agent pacritinib (CTI BioPharma) holds promise as a treatment option for patients with myelofibrosis and baseline thrombocytopenia, and previous concerns that led to a hold on clinical trials have now been dispelled.

In a pivotal trial, PERSIST-2, pacritinib was significantly more effective than the best available therapy, including ruxolitinib (Jakafi, Incyte), in reducing splenomegaly and trended toward a reduction in total symptom score in patients with myelofibrosis and thrombocytopenia.

The study was published online March 8 in JAMA Oncology. Ruxolitinib, which was approved in 2011 and the first drug ever approved for myelofibrosis, is not safe for patients with low platelet counts.

“The label on ruxolitinib states it is for patients with platelet counts above 50,000, so it isn’t a viable option for those with thrombocytopenia,” said lead author, John Mascarenhas, MD, associate professor of medicine, hematology and medical oncology, at the Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai in New York.

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View Dr. Mascarenhas’ presentation at MPN Advocacy’s San Antonio Patient Program

Recruiting: New Phase 2 Clinical Trial for MF Patients on Ruxolitinib

People with myelofibrosis who are currently receiving ruxolitinib therapy may be eligible for a new phase II clinical trial led by Dr. Ellen Ritchie.

Navitoclax is an investigational agent that inhibits a family of BCL proteins. These proteins block some of the enzymes that keep cancer cells from dying and by inhibiting these proteins, navitoclax may cause the cancer cells to die.  Ruxolitinib is approved by the FDA for the treatment of myelofibrosis. Ruxolitinib blocks a protein called Janus-associated kinases (JAK) which may help keep abnormal blood cells or cancer cells from growing.

The purpose of this study is to evaluate the addition of navitoclax to ruxolitinib in patients who have been receiving ruxolitinib alone. Ruxolitinib treatment alone has not been fully controlling disease which is evident by an enlarged spleen. Preclinical data suggest that the combination of navitoclax with ruxolitinib are synergistic and that navitoclax may help to overcome disease resistance to ruxolitinib.

The study will help to determine the effect of the combination of navitoclax plus ruxolitinib on your cancer. The study will evaluate how the efficacy and safety of the drug.

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Imago BioSciences Receives FDA Approval of IND Application for the Treatment of Myeloid Malignancies

SAN CARLOS, Calif., Feb. 1, 2018 /PRNewswire/ — Imago BioSciences, a clinical-stage pharmaceutical company developing novel therapies for hematological and inflammatory diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND) application providing clearance to proceed with the clinical development of IMG-7289 in the U.S. The IND supports the company’s ongoing Phase 1/2 clinical trial of IMG-7289 for myelofibrosis (MF).

“There is a pressing need for novel approaches to the treatment of myeloproliferative disorders including myelofibrosis,” said Hugh Young Rienhoff, Jr. M.D., Imago’s Chief Executive Officer.  “We are pleased to have received FDA acceptance of our clinical trial protocol and look forward to the imminent expansion of this study into the United States.”

This Phase 1/2 open-label clinical trial is designed to assess the pharmacodynamics of IMG-7289, an oral inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1) in high-risk myelofibrosis patients aged 18 or older ( Identifier NCT03136185).  Assessments include measuring changes in spleen volume, patient reported total symptom scores, mutant allele burden, inflammatory cytokines and bone marrow fibrosis over the course of the treatment period.  The trial commenced in Australia in 2017 and will add multiple sites in the United States in 2018.

This is the second clinical trial of IMG-7289 sponsored by Imago BioSciences, Inc.  The first, evaluating IMG-7289 for the treatment of high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), was initiated in 2016 ( Identifier NCT02842827) and continues to enroll patients.

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New Data Proves Role of Thrombosis in Patients with MPNs

New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).

MPNs include a variety of blood disorders, like myelofibrosis, polycythemia vera, and essential thrombocytosis, and patients with them have been reported to be at increased risk for thrombotic events. Pharmacologic treatment can stabilize the blood counts, but they generally provide only partial symptom improvement. Until recently, no population-based study has estimated the excess risk with matched control participants.

Malin Hultcrantz, M.D., PhD and colleagues, in an effort to address the gap in knowledge, evaluated data from 9,429 patients – 46% of whom were male – diagnosed with MPNs who reported to the Swedish Cancer Register between 1987 and 2009, and matched them to 35,820 control participants and compared their rates of arterial and venous thrombosis. Median age of study participants was 72 years, and a mong them, 3,001 had polycythemia vera, 3,462 had essential thrombocythemia, 1,488 had PMF, and 1,478 had MPN unclassifiable.

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ASH: Stemline Presents on Recent Clinical Trial Results

Stemline Therapeutics Presents Detailed SL-401 Pivotal Data in BPDCN at ASH and Kicks Off its BPDCN Awareness Campaign; Updated Results From Ongoing Trials in Additional Malignancies Also Presented

NEW YORK, Dec. 13, 2017 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, presented detailed data from its SL-401 pivotal trial in BPDCN, as well as results from other ongoing trials in additional indications, at the 2017 American Society of Hematology (ASH) Annual Meeting and Exposition, held in Atlanta, GA. Presentations are available on the Stemline website,, under the Scientific Presentations tab…

SL-401: Phase 1/2 Trial in myeloproliferative neoplasms (MPN): chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF)

  • Key outcomes (ASH ’17 data)
    – SL-401 Phase 1/2 trial consists of a Stage 1 (lead-in, dose escalation) and Stage 2 (expansion); has enrolled 24 patients
    – No dose limiting toxicities (DLT) were identified and a maximum tolerated dose (MTD) was not reached. Most common TRAEs include hypoalbuminemia (33%), thrombocytopenia (33%), and fatigue (29%). Most common TRAEs (grade 3 or higher), include thrombocytopenia (24%) and anemia (19%)
    – Durable CR (14+ months) in CMML patient
    – 65% (11/17 evaluable) of CMML and MF patients had spleen reductions >25% (range 29% to 100%)
    – Durable SD in 4 patients (2 CMML, 2 MF) for 5+ to 8+ months. Three ongoing SD patients enrolled with baseline platelet counts <100,000, including 1 patient platelet count <50,000 are on treatment
  • Next Steps in MPN
    – Continue enrollment and patient follow-up
    – Favorable tolerability and preliminary signs of activity support both single agent and combination development strategies, including JAK-inhibitors and hypomethylating agents

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ASH: Sotatercept Boosts Hemoglobin in MPN-Associated Myelofibrosis

The investigational activin receptor IIA ligand trap sotatercept safely increases hemoglobin levels in patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis, both when used alone and in combination with ruxolitinib (Jakafi).

A phase II investigator-initiated trial of sotatercept in this population showed responses in 10 of 28 evaluable patients, said Prithviraj Bose, MD, of updated findings presented at the 2017 ASH Annual Meeting.

Anemia is present in about one-third of patients with myelofibrosis at diagnosis and eventually develops in all patients. Only 20% to 30% of patients respond to current therapy, such as danazol and erythroid-stimulating agents.

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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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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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Impact Biomedicines Closes First Tranche of Oberland Capital Financing Following FDA Meeting

Management team expands to accelerate fedratinib global manufacturing and business operations

SAN DIEGO — December 1, 2017— Impact Biomedicines (“Impact”) today announced that it has achieved the first milestone in the Company’s previously disclosed $90 million financing with Oberland Capital, triggering the closing on the first tranche of $20 million. Impact also announced the expansion of its management team to include Randy Adams as Senior Vice President of Commercial Operations and Jeff Barker as Senior Vice President of Global Technical Operations.

“The first tranche of this financing follows a positive meeting with the U.S. Food and Drug Administration (FDA) bringing this much needed potential treatment option for myelofibrosis closer to patients in need,” said John Hood, Ph.D., Chief Executive Officer of Impact Biomedicines. “With Oberland’s financial support, we have made some important investments to ensure that we are well-staffed and prepared for U.S. commercialization.”

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