FDA Grants Fast Track Status to Imara’s IMR-687 Candidate Therapy for Sickle Cell Disease

IMR-687, an investigational therapy for sickle cell disease, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). It’s currently being evaluated in a Phase 2a trial in the U.S. and U.K.

The Fast Track process is meant to get new therapeutics that treat serious conditions and fill an unmet medical need to patients earlier. It facilitates the development and expedites the review and approval of new therapies.

“We believe that IMR-687 has the potential to transform outcomes for people living with sickle cell disease. It has been designed as an oral, once-daily therapy with a dual mechanism of action to address both red blood cell and white blood cell pathologies associated with the disease,” Rahul D. Ballal, CEO of Imara, which is developing IMR-687, said in a press release.

“The Fast Track designation granted by the FDA for IMR-687 is designed to allow us to advance this novel, investigative therapy more rapidly through increased opportunities for dialogue and collaboration with the Agency.”

IMR-687 is a potent and selective inhibitor of the enzyme phosphodiesterase 9 in red blood cells. According to Imara, it has the potential to be a disease-modifying therapy for sickle cell disease (SCD), and other hemoglobinopathies.

Earlier preclinical studies in cell and animal models showed that IMR-687 increases fetal hemoglobin, which prevents the assembly of sickled hemoglobin in red blood cells. As a result, it reduced red cell sickling and blood vessel occlusion — a cause of debilitating pain, organ damage, and early mortality in patients with the disease.

In a first-in-human Phase 1 clinical trial (NCT02998450) conducted in healthy volunteers, IMR-687 proved to be well-tolerated.

Imara is currently recruiting participants for a Phase 2 trial (NCT03401112) at several trial centers in the U.S. and the U.K., with a target enrollment of 50 adult patients with sickle cell disease.

The trial’s primary goal is to assess the safety and tolerability of IMR-687 administrated orally once daily, as well as its distribution and effects inside the body, a collection of parameters known as pharmacokinetics and pharmacodynamics, compared with a placebo control group. For more information, visit the clinical trial webpage here.

IMR-687 was granted Orphan Drug status and Rare Pediatric Disease Designation by the FDA in 2017.