Promedior Presents Positive Phase 2 Data for PRM-151 in Myelofibrosis at ASH Annual Meeting
— Company Initiates Second Stage of Phase 2 clinical study of the novel investigational anti-fibrotic immunomodulator, PRM-151, in patients with intermediate and high risk myelofibrosis —
Lexington, Mass., December 10, 2015 — Promedior, Inc.,today announced positive data from Stage 1 of an adaptive two-stage Phase 2 trial of PRM-151, a novel investigational anti-fibrotic immunomodulator, in patients with primary myelofibrosis (PMF), post-polycythemia vera MF (post-PV MF), or post-essential thrombocythemia MF (post-ET MF). PRM-151 was shown to be well-tolerated for at least 72 weeks, and some patients remaining on treatment for 72 weeks have had reductions in bone marrow fibrosis and/or improvements in anemia, thrombocytopenia, with reductions in transfusions, symptoms and spleen size. These study results were presented in an oral presentation by principal investigator Srdan Verstovsek, MD, PhD, at the ASH 2015 Annual Meeting on December 5, 2015.
Promedior also today announced the initiation of Stage 2 of the Company’s adaptive two-stage Phase 2 trial of PRM-151 in patients with PMF and post-ET/PV MF. This second stage of the Company’s randomized, double-blind, dose-ranging, two-stage Phase 2 study is designed to determine the efficacy and safety of three different doses of PRM-151 in approximately 84 patients with PMF and post-ET/PV MF. Promedior is initiating this second stage of this Phase 2 study based on data from the first stage of this Phase 2 study of PRM-151 in patients with MF, including the data presented at ASH 2015.
“I am very happy to see the continued benefits in patients with myelofibrosis remaining on PRM-151, including reductions in spleen size with longer treatment,” said Srdan Verstovsek, MD, PhD, Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and Principal Investigator for this Phase 2 trial. “Many of these patients had failed multiple other therapies, and PRM-151 has given them new hope for the future.”
PRM-151 is designed to target the underlying fibrotic pathology of myelofibrosis. In addition to the data presented at ASH 2015, clinical data from the first stage of this Phase 2 study of PRM-151 in patients with MF have also been presented at the American Society of Hematology (ASH) 2014 Annual Meeting, the 20th Congress of the European Hematology Association (EHA), and the American Society for Clinical Oncology (ASCO) 2014 Annual Meeting.
Phase 2 Study Design
The ongoing Phase 2 trial is a multi-center, two stage, adaptive design study to determine the efficacy and safety of PRM-151 as a single agent or added to a stable dose of ruxolitinib in patients with primary myelofibrosis (PMF), post-polycythemia vera MF (post-PV MF), or post-essential thrombocythemia MF (post-ET MF). Twenty-seven patients were enrolled in Stage 1 of the study. In this Stage 2 of the Phase 2 clinical study approximately 84 additional patients with intermediate-1, intermediate-2, or high risk MF who meet study eligibility requirements will be enrolled and randomized to treatment with single agent PRM-151 at doses of 0.3, 3, or 10 mg/kg IV. Each patient in this second stage of the Phase 2 study will participate in the study for approximately 44 weeks, and it is estimated that the study will be completed 2017. Efficacy will be assessed by evaluation of WHO bone marrow fibrosis grade, changes in hemoglobin, platelets, peripheral blood blasts, disease related symptoms, and spleen size, and safety will be evaluated from reported adverse events, scheduled physical examinations, vital signs, and clinical laboratory test results.
This second stage of the Phase 2 clinical study will be open at centers in the US, Canada, UK, Italy, Netherlands, France, and Germany, and participating Stage 1 investigators in the PRM-151 Phase 2 study include Srdan Verstovsek, MD, PhD (University of Texas MD Anderson Cancer Center, Principal Investigator for this Phase 2 trial), Jason Gotlib, MD (Stanford University), Ruben Mesa, MD (Mayo Clinic, Scottsdale), Vikas Gupta, MD (Princess Margaret Cancer Centre), John Mascarenhas, MD (Icahn School of Medicine at Mt. Sinai Hospital), Ronald Hoffman, MD (Icahn School of Medicine at Mt. Sinai Hospital), Ellen Ritchie, MD (Weill Cornell Medical College of Cornell University), Richard Silver, MD (Weill Cornell Medical College of Cornell University), and Lynda Foltz, MD (University of British Columbia). For additional details about this clinical trial, please visit www.clinicaltrials.gov.
Myelofibrosis (MF), a type of myeloproliferative neoplasm, is a serious, life-limiting cancer that is characterized by fibrosis of the bone marrow. Replacement of the bone marrow by scar tissue prevents the normal production of blood cells, leading to anemia, fatigue, and increased risk of bleeding and infection. Data show that bone marrow fibrosis grade is correlated with anemia, thrombocytopenia, peripheral blasts and shortened survival1,2.
Myelofibrosis affects approximately 18,000 people per year in the U.S., with a median age of 61-663. The only potentially curative treatment is allogeneic bone marrow transplant, which results in reversal of fibrosis and normalization of blood counts, but is a realistic option for only a small number of patients. Other currently available therapies have minimal, if any, impact on the underlying fibrosis, and often result in worsening in hemoglobin and platelets, important blood parameters which are directly linked to morbidity and mortality and remain the major unmet need in patients with myelofibrosis.
PRM-151, Promedior’s lead product candidate, is a recombinant form of an endogenous human protein, Pentraxin-2 (PTX-2), that is designed to be specifically active at the site of tissue damage. PRM-151 is an investigational agonist that acts as a monocyte/macrophage differentiation factor which is being investigated for prevention and potentially reversal of fibrosis. PRM-151 has shown anti-fibrotic activity in multiple preclinical models of fibrotic disease, including pulmonary fibrosis, acute and chronic nephropathy, liver fibrosis, and age-related macular degeneration.
PRM-151, an investigational compound, has Orphan Designation in the US and EU for treatment of IPF and myelofibrosis and Fast Track in the US for treatment of myelofibrosis.
Promedior is a clinical-stage immunotherapy company pioneering the development of targeted therapeutics to treat diseases involving fibrosis. Fibrosis occurs when healthy tissue is replaced with excessive scar tissue, compromising function and ultimately leading to organ failure. Fibrosis is a common feature of several rare diseases as well as more prevalent illnesses such as age related macular degeneration, diabetic nephropathy, nonalcoholic steatohepatitis (NASH), and several types of solid tumors.
Promedior has advanced its lead program (PRM-151) into clinical trials focused on two orphan fibrotic diseases, myelofibrosis and idiopathic pulmonary fibrosis. Promedior owns world-wide rights to PRM-151 and has a significant intellectual property estate.
In August, 2015, Bristol-Myers Squibb Company and Promedior, Inc. announced an agreement that grants Bristol-Myers Squibb an exclusive right to acquire Promedior and gain worldwide rights to PRM-151. Under the terms of the agreement, Bristol-Myers Squibb will make payments aggregating up to $1.25 billion that includes an upfront cash payment of $150 million as consideration for both the right to acquire Promedior and as payment for services in support of the MF and IPF Phase 2 clinical trials.
For additional information about Promedior, please visit www.promedior.com.
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1 Thiele, J., Kvasnicka, H.M., Ann Hematol 85: 226-232; 2006.
2 Vener, C., et al., Blood 111(4): 1862-5; 2008.
3 Mehta, J., Wang, H., Iqbal, S. U., Mesa, R., “Epidemiology of myeloproliferative neoplasms in the United States”, Leukemia & Lymphoma, Early Online: 1-6, 2013.
Note: Ruxolitinib is available under the trade names JAKAFI® and JAKAVI®, which are the registered trademarks of Incyte and Novartis, respectively.