Imara Reports Favorable Preclinical and Phase 1 Data on IMR-687 in Sickle Cell Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Imara Inc. today announced it will report additional preclinical and
Phase 1 clinical data from the company’s lead compound, IMR-687, a
once-daily, oral therapy designed to address the underlying pathology of
sickle cell disease (SCD). In preclinical studies, IMR-687’s mechanism
of action was shown to positively impact both red and white blood cell
pathologies associated with the disease. The company has previously
reported the ability of IMR-687 to induce fetal hemoglobin (HbF) in
murine and human cells at ASH in 2017. Importantly, the data from a
recent Phase 1 clinical trial in healthy volunteers demonstrated IMR-687
to be safe and well-tolerated at the target dose that exceeded the
effective doses in cell and animal models of SCD, thereby clearing a
path to a Phase 2 study in adult patients with SCD. The new data will be
presented at the 6^th Annual Sickle Cell Therapeutics
Conference in New York.

“Data from our Phase 1 clinical trial demonstrate we are able to safely
dose subjects at levels shown to be therapeutically meaningful in
preclinical models,” said James McArthur, Ph.D., Founder, President, and
Chief Executive Officer of Imara. “We believe IMR-687 could represent an
important new treatment option for patients in need. Based on the
favorable data to date, we plan to initiate a Phase 2 clinical trial in
adult patients by the end of this year.”

In SCD, both red and white blood cells are affected, causing significant
pain and severe symptoms with potentially life-threatening consequences.
In a preclinical mouse model, IMR-687 reduced red blood cell sickling
and white blood cell adhesion associated with the disease. IMR-687 also
demonstrated a reduction in blood vessel occlusion, a hallmark of the
disease, which causes debilitating pain, organ damage, and early
mortality in patients with SCD. Additionally, preclinical studies showed
IMR-687 increased fetal hemoglobin (HbF); increases in HbF have been
shown to improve symptoms in SCD patients.

Existing treatment options for SCD are limited and there remains a
significant unmet need for new treatment options. The majority of
patients are treated with pain medications, fluids, and blood
transfusions, and experience frequent hospitalizations as a result. The
only approved treatments for SCD are hydroxyurea, approved more than a
decade ago, and Endari, an oral amino acid L-glutamine powder. Bone
marrow or stem cell transplants may be an option for younger patients
with severe SCD. However, these treatment options either come with
severe and potentially life-threatening side effects or have limited
efficacy.

About IMR-687

IMR-687 was specifically designed to address the underlying pathology of
sickle cell disease. An orally-administered, highly potent and selective
phosphodiesterase 9 (PDE9) inhibitor, IMR-687 is a potentially
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. A Phase 1 clinical trial in healthy volunteers showed IMR-687
to be safe and well-tolerated.

IMR-687 has been granted both U.S. Orphan Drug Designation and U.S. Rare
Pediatric 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.

About Imara

Imara
Inc., a Cydan
Development company, is dedicated to developing novel therapeutics
for patients with sickle cell disease. Imara is developing IMR-687, a
highly selective, potent small molecule inhibitor of PDE9, to treat
patients with sickle cell disease. The company was launched following an
18-month diligence and de-risking scientific collaboration between
orphan drug accelerator Cydan Development and H. Lundbeck A/S with $31M
Series A funding from life science investors NEA, Pfizer Venture
Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria
Venture Investments.

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