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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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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Incyte and Syros to collaborate in cancer R&D

  • Gene expression-focused biotech Syros Pharmaceuticals, Inc. has snagged a collaboration deal with biopharma Incyte Corp. to develop potential therapeutics to treat rare bone marrow disorders known as myeloproliferative neoplasms (MPNs).
  • In return for $20 million upfront in cash and R&D funding, and $10 million in stock, Syros will use its platform technology to find targets in MPNs. Incyte gets options to exclusive rights for therapies modulating up to seven validated targets discovered under the collaboration.
  • If Incyte exercises these options, Syros could see up to $54 million in option fees, and up to $115 million for products against each of the targets, as well as low-single digit royalties.

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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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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

By 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.