LONDON, June 18, 2019 /PRNewswire/ --
Arix Bioscience plc (LSE: ARIX)
("Arix"), a global venture capital company focused on investing and
building breakthrough biotech companies, notes that its portfolio
company Imara, Inc. ("Imara") has presented positive initial data
from its ongoing Phase 2a clinical trial evaluating the safety and
efficacy of IMR-687, its lead programme for the treatment of Sickle
Cell Disease, at the 24th Congress of the European Hematology
Association (EHA) in Amsterdam.
The announcement can be accessed on Imara's website
http://imaratx.com/who-we-are/news-press-releases/ and the full
text of the announcement from Imara is contained below.
About Arix Bioscience plc
Arix Bioscience plc is a global venture capital company focused
on investing in and building breakthrough biotech companies around
cutting edge advances in life sciences.
We collaborate with exceptional entrepreneurs and provide
capital, expertise and global networks to help accelerate their
ideas into important new treatments for patients. As a listed
company, we are able to bring this exciting growth phase of our
industry to a broader range of investors.
Arix Bioscience plc is listed on the Main Market of the London
Stock Exchange. For further information, please visit
http://www.arixbioscience.com
Imara Presented Positive,
Interim Phase 2a Data on IMR-687 for the Treatment of Sickle Cell
Disease at the 24th Congress of the
European Hematology Association
Company encouraged by
interim 13-week data in trial designed to assess
the safety and tolerability of IMR-687 and explore the potential
efficacy of IMR-687 on clinical outcome measures relevant to sickle
cell disease
CAMBRIDGE,
Massachusetts,
June 18, 2019 - Imara Inc., a
clinical-stage biopharmaceutical company developing novel therapies
for people living with sickle cell disease and other serious,
inherited blood disorders, today announced promising interim,
13-week Phase 2a data on IMR-687 that were presented at the
24th Congress of the European Hematology Association
(EHA) in Amsterdam. IMR-687 is an
investigational, orally administered, highly potent and selective
phosphodiesterase 9 (PDE9) inhibitor currently being evaluated as a
potential disease-modifying therapeutic for sickle cell disease
(SCD). Data presented at EHA demonstrated that treatment with
IMR-687 in adult patients was generally well tolerated. The data
also support the dual mechanism of action of IMR-687, with activity
seen across both red and white blood cell biomarkers.
"These initial Phase 2a data demonstrate the potential of
IMR-687 to significantly impact key biomarkers associated with the
pathology of this serious disease," said Biree Andemariam, M.D., Associate Professor at
UConn School of Medicine and Director of the New England Sickle
Cell Institute at UConn Health, and lead investigator for the
trial. "In the laboratory we saw that IMR-687 inhibition of PDE9
increases intracellular cGMP levels, increases fetal hemoglobin
expression, reduces sickling and hemolysis of red blood cells, and
does not induce neutropenia. The interim Phase 2a data reflect
trends that could be indicative of meaningful clinical translation
of these important measures in SCD."
The Phase 2a clinical trial is designed to evaluate the safety,
tolerability, pharmacokinetics and pharmacodynamics of escalating
doses of IMR-687 administered once daily for 16 to 24 weeks in two
groups of patients with SCD: those not on hydroxyurea (HU)
(Population A) and those receiving a stable dose of HU according to
the current standard of care (Population B). Blinded white cell
markers in Population A (n=19), along with red cell markers and
quality of life measures (ASCQ-Me®) (n=13) were analyzed. The
blinded interim analysis was conducted on the monotherapy IMR-687
(Population A) subgroup. At 5 weeks, in the 100 mg dose group of
Population A, there was a trend to reduced soluble P selectin
(sPsel), soluble vascular cell adhesion molecule-1 (sVCAM) and
myeloperoxidase (MPO) compared to placebo. At 13 weeks, in the 100
mg dose group of Population A, there was an increase (110%) in the
percent of F-cells, which are red blood cells that contain fetal
hemoglobin (HbF) which often precede rises in total HbF. A
corresponding decrease in absolute reticulocyte count and the
percentage of reticulocytes, with a trend towards improved pain as
measured by ASCQ-Me®, was also observed at 13 weeks in Population
A.
Blinded safety data for 27 patients in the Phase 2a clinical
trial demonstrated that treatment with IMR-687 was generally well
tolerated, with no clinically significant changes in white blood
cell counts and no evidence of neutropenia. There were no
treatment-related serious adverse events.
"We are encouraged by this interim Phase 2a analysis that
reinforces our belief in the potential of IMR-687 as a single oral,
once-a-day therapeutic," said Rahul D.
Ballal, Chief Executive Officer of Imara. "IMR-687
uniquely targets both red cell and white cell aspects of the
disease, and we are working to expeditiously advance this novel
therapy through clinical development, with a goal of delivering it
to patients with SCD who are in need of innovative treatment
options."
Imara expects to announce additional interim Phase 2a data in
the second half of 2019. Imara anticipates that these data will
include safety and potential acitivity in additional patients,
including patients in Population A that received higher doses
and/or longer treatment duration with IMR-687 as well as the first
data on patients in Population B.
IMR-687 Phase 2a Clinical Trial Design
IMR-SCD-102 is a Phase 2a, randomized, double-blind,
placebo-controlled study of IMR-687. It is designed to evaluate the
safety, tolerability, pharmacokinetics and pharmacodynamics of
escalating doses of IMR-687 administered once daily for 16 to 24
weeks in two groups of patients with SCD: those not on hydroxyurea
(HU) (Population A) and those receiving a stable dose of HU
according to the current standard of care (Population B).
Participants in Population A receive either IMR-687 or placebo for
a total of 24 weeks. On Day 1, subjects are randomized in a 1:1:1
ratio to receive oral IMR-687 50 mg, IMR-687 100 mg or placebo
daily for the first 12 weeks. Each participant's dose is doubled at
week 13 following the review of clinical safety data. Participants
in Population B are randomized in a 2:1 ration and receive either a
50 mg dose of IMR-687 or placebo, with dose escalation to 100 mg
(or placebo) after four weeks and review of the clinical safety
data. The Adult Sickle Cell Quality of Life Measurement Information
System (ASCQ-Me®), is also administered at baseline, 13 weeks and
at the end of the study.
About IMR-687
IMR-687 has been designed to address the underlying pathology of
sickle cell disease. An orally administered, highly potent and
selective phosphodiesterase 9 (PDE9) inhibitor, IMR-687 has the
potential to be a disease-modifying therapeutic for sickle cell
disease as well as other hemoglobinopathies. Pre-clinical data
demonstrate IMR-687 reduces both the sickling of red blood cells
and blood vessel occlusion that cause debilitating pain, organ
damage, and early mortality in affected patients. In a Phase 1
clinical trial in healthy volunteers, IMR-687 was demonstrated to
be well-tolerated. IMR-687 is currently in a Phase 2a clinical
trial in patients with sickle cell disease. IMR-687 has been
granted U.S. Orphan Drug Designation, U.S. Rare Pediatric
Designation and Fast Track Designation by the Food and Drug
Administration (FDA).
About Sickle Cell Disease
Sickle cell disease is a rare, genetically inherited condition
that alters hemoglobin, the protein in red blood cells that
transports oxygen throughout the body. The altered hemoglobin
distorts red blood cells into a sickle, or crescent, shape. Painful
episodes can occur when sickled red blood cells, which are stiff
and inflexible, get stuck in small blood vessels. These episodes
deprive tissues and organs of oxygen-rich blood and can lead to
vaso-occlusive crisis (VOC), acute chest syndrome (ACS), and
permanent damage to organs including the liver, spleen, kidney and
brain.
Sickle cell disease represents a critical unmet medical need
globally, as a rare disease in many parts of the world including in
the United States and as an
endemic condition in certain African countries.
About Imara
Imara Inc. is committed to transforming the lives of people with
sickle cell disease and other serious hemoglobinopathies, including
beta thalassemia, by developing oral small molecule therapeutics
that are designed to be easy to administer and use across the
world. Imara is currently advancing IMR-687, a highly selective,
potent small molecule inhibitor of PDE9 with a dual mechanism of
action targeting both red and white blood cells, in an ongoing
Phase 2a clinical trial in patients with sickle cell disease. In
March 2019, Imara closed a
$63M Series B with leading life
science investors New Enterprise Associates, OrbiMed Advisors, Arix
Bioscience plc, RA Capital, Rock Springs Capital, Pfizer Venture
Investments, Lundbeckfonden Ventures, Bay City Capital and
Alexandria Venture Investments. For more information, please
visit http://www.imaratx.com .