In the European Union, this designation is given to investigative therapies for rare diseases — those that affect fewer than five in 10,000 people — to support treatment development. Treatments receiving the orphan drug designation must have sufficient clinical and non-clinical data backing up their ability to lead to clinically meaningful improvements.
The designation also comes with certain benefits, including clinical protocol assistance, access to a centralized marketing authorization procedure valid in all countries of the European Union, reduced regulatory fees, as well as 10 years of market exclusivity, if approved.
“We are pleased to have received Orphan Drug designation from the European Commission, reinforcing the unmet need for more effective treatment options for patients with sickle cell disease,” Rahul Ballal, PhD, president and CEO of Imara, said in a press release.
IMR-687 is a selective, oral inhibitor of phosphodiesterase 9 (PDE9), an enzyme found in red blood cells that normally destroys a signaling molecule called cyclic guanosine monophosphate (cGMP). In people with SCD and beta-thalassemia, another genetic blood disorder, the levels of cGMP are usually low.
By blocking PDE9 and increasing the levels of cGMP, IMR-687 is thought to reactivate the production of fetal hemoglobin — the protein that carries oxygen in newborns and is more effective in doing so than hemoglobin later in life.
Ultimately, by increasing the levels of fetal hemoglobin in red blood cells, IMR-687 is expected to ease the symptoms of SCD and beta-thalassemia.
Imara recently launched a Phase 2b trial called Ardent (NCT04474314) to assess the safety and efficacy of IMR-687 in adults (up to age 65) with SCD. The trial, which is still recruiting participants at two sites in the U.S., aims to enroll 99 patients.
During the trial, patients will be randomly assigned to receive one of two doses of IMR-687, or a placebo, once-a-day, for one year. Those who complete the study will also have the option to enroll in an extension study in which all will be given the therapy.
With the first participant in Ardent already dosed, Imara’s next priority is to perform a preliminary analysis of the study when 33 patients complete 24 weeks (six months) of treatment.
In addition to Ardent, a Phase 2a trial (NCT03401112) and its long-term extension study (NCT04053803) are currently assessing the safety, tolerability, and clinical efficacy of IMR-687 in adults with SCD.
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