|[May 30, 2014]
Promedior to Present Positive Preliminary Phase 2 Data for PRM-151 in Myelofibrosis at ASCO Annual Meeting
LEXINGTON, Mass. --(Business Wire)--
Inc., today announced positive preliminary data from its Phase 2 trial
of PRM-151, an anti-fibrotic immunotherapy, in patients with
myelofibrosis which demonstrated biologic activity with improvements
across clinically relevant measures, including bone marrow fibrosis,
hemoglobin, platelets, spleen, and symptoms. Clinical data showed
improvements in four independent treatment groups of myelofibrosis
patients who received PRM-151 weekly or monthly, either as a single
agent or in patients with no further improvements on a stable dose of
ruxolitinib1. Importantly, PRM-151 demonstrated safety and
tolerability both alone and in combination with ruxolitinib, with no
evidence of the myelosuppression commonly observed with other
treatments. Study results will be presented at the American Society for
Clinical Oncology (ASCO) 2014 Annual Meeting on June 2, 2014, at
1:15-5:00 pm CT, in S Hall A2.
"These early clinical data with PRM-151 in patients with myelofibrosis
are encouraging, and demonstrate the potential of the compound's novel
mechanism of action," 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. "The improvements seen in patients who have either
progressed on JAK inhibitors or were deriving no further benefit from
ruxolitinib speaks to the need for new therapies that target fundamental
mechanisms of the disease."
"These findings are very promising and we are particularly excited to
see improvements in bone marrow pathology in myelofibrosis patients
receiving PRM-151. We believe PRM-151's ability to precisely target the
fundamental fibrotic pathology validates its broad potential to treat
and reverse fibrosis in a wide range of fibrotic diseases," said Suzanne
L. Bruhn, PhD, President and Chief Executive Officer of Promedior. "We
will continue to work to move as quickly as possible to bring PRM-151
forward as a potential new treatment option for patients with fibrotic
diseases which have few, if any, treatment options today."
The preliminary Phase 2 data for PRM-151 were presented for 27 patients
with myelofibrosis, 18 of whom completed 24 weeks of therapy. PRM-151
demonstrated a 50% reduction in symptoms according to the MPN-SAF2
Total Symptom Score in 7 patients, 5 of which have persisted for =12
weeks and are therefore confirmed IWG-MRT3 Clinical
Improvement symptom responses; 5 reductions in bone marrow fibrosis by
=1 grade, with 2 of 3 patients confirmed 12 weeks later and 2 patients
pending confirmatory biopsy; a =20% reduction in spleen volume reduction
in 5 patients with one 50% reduction lasting 8 weeks; and improvements
in hemoglobin and platelets. Each treatment group demonstrated
improvements that met the pre-specified efficacy criteria for further
exploration of PRM-151 in the second stage of this adaptive Phase 2
trial. Fifteen out of 18 patients who have completed the 24 week study
are continuing treatment in a study extension.
In this study, PRM-151 was safe and well tolerated on weekly and monthly
dosing schedules, both alone and in combination with ruxolitinib, with
no evidence of myelosuppression. Most adverse events observed in the
study were Grade 1 or 2 and considered unrelated to PRM-151. Overall,
there were 14 severe adverse events (SAEs) in 5 study patients,
including 3 deaths, 2 from pneumonia and 1 from progressive multi-organ
failure. Other SAEs were abdominal pain, bone marrow biopsy site
hematoma, sialadenitis, gastroenteritis and respiratory syncytial virus.
The Company expects to report the complete first stage results of this
ongoing Phase 2 study by the end of 2014.
This 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). 27 patients were
enrolled in the first stage of the study; up to 80 additional patients
will be enrolled in the second stage.
Participating 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 (News - Alert), 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. Production of blood cells
shifts to the spleen and liver (extramedullary hematopoiesis), which
become enlarged, causing severe discomfort, inability to eat, and
weakness. Symptomatic myelofibrosis affects approximately 18,000 people
per year in the US, with a median age of 61-66.4 The only
potentially curative treatment is allogeneic bone marrow transplant,
which results in reversal of fibrosis and all symptoms, but is a
realistic option for only a small number of patients. Other currently
available therapies address the symptoms, but have minimal if any impact
on the underlying fibrosis.
PRM-151, Promedior's lead product candidate, is a recombinant form of an
endogenous human protein, Pentraxin-2 (PTX-2), that is specifically
active at the site of tissue damage. PRM-151 is an agonist that acts as
a monocyte/macrophage differentiation factor to prevent and potentially
reverse fibrosis. PRM-151 has shown broad anti-fibrotic activity in
multiple preclinical models of fibrotic disease, including pulmonary
fibrosis, acute and chronic nephropathy, liver fibrosis, and age-related
Phase 1a and 1b clinical studies in healthy subjects and IPF patients
have demonstrated that PRM-151 was well tolerated. Additionally, a Phase
1b study in patients with IPF showed encouraging
results in exploratory efficacy endpoints, which were presented in
an oral session at the 2013 Annual Meeting of the American Thoracic
Society5. Recent clinical data in myelofibrosis demonstrated
the potential of this immuno-oncology approach in fibrotic cancers.
is a clinical stage biotechnology company pioneering the development of
targeted therapeutics to treat diseases involving fibrosis. Fibrosis is
a harmful process that occurs in many diseases, when normal healthy
tissue is replaced with excessive scar tissue, compromising function and
ultimately leading to organ failure. Promedior's proprietary platform is
based upon Pentraxin-2, an endogenous human protein that is specifically
active at the site of tissue damage and, with an anti-fibrotic
immunotherapy approach, works to prevent and reverse fibrosis.
Promedior has successfully advanced its lead therapeutic candidate in
human clinical trials, and is initially focused on rare fibrotic
diseases, including myelofibrosis and idiopathic pulmonary fibrosis
(IPF). Promedior is backed by leading global healthcare venture
investors, has a significant intellectual property estate relating to
the discoveries and applications of Pentraxin-2 therapeutics and is led
by an experienced management team. For additional information about
Promedior, please visit www.promedior.com.
1. Ruxolitinib is available under the trade names JAKAFI® and JAKAVI®,
which are the registered trademarks of Incyte and Novartis, respectively.
2. Emanuel, R.M. et al., "Myeloproliferative Neoplasm (MPN) Symptom
Assessment Form Total Symptom Score: Prospective International
Assessment of an Abbreviated Symptom Burden Scoring System Among
Patients with MPNs," J Clin Oncol 30:4098-4103; 2012.
3. Tefferi, A., et al., "Revised Response Criteria for Myelofibrosis:
International Working Group- Myeloproliferative Neoplasms Research and
Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report,"
Blood 122(8): 1395-8; 2013.
4. Mehta, J., Wang, H., Iqbal, S. U., Mesa, R., "Epidemiology of
myeloproliferative neoplasms in the United States", Leukemia & Lymphoma,
Early Online: 1-6, 2013.
5. Van Den Blink, B. et al., "A Phase I Study Of PRM-151 In Patients
With Idiopathic Pulmonary Fibrosis", American Thoracic Society 2013
Annual Meeting, May 2013. Read More: http://www.atsjournals.org/doi/abs/10.1164/ajrccm-conference.2013.187.1_MeetingAbstracts.A5707
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