REMEDY PHARMACEUTICALS is focused on therapies for acute CNS conditions. Remedy’s lead drug is CIRARA®, designed to reduce the formation of life-threatening CNS-related edema, lower mortality, improve patient lives and lessen healthcare costs.
CIRARA is a high affinity inhibitor of Sur1-Trpm4 channels, which are upregulated following ischemia and trauma. Opening of these channels can lead to edema and its downstream consequences. CIRARA is suitable for intravenous delivery at the bedside or even in an ambulance.
Large infarctions in the middle cerebral artery territory, known as Large Hemispheric Infarctions (LHI), frequently develop space-occupying edema, leading to midline shift, raised intracranial pressure, and herniation. This results in death or permanent disability. Edema is the primary cause of death in patients with LHI. Mortality ranges from 40-80%.
In a randomized, double-blinded Phase 2 study of LHI patients, edema was cut in half, 90-day mortality in the CIRARA group was more than halved as compared to the placebo-treated group – and this persisted out to one-year follow up.
At 90 days, CIRARA patients had an overall 29% improvement in 0-4 mRS as compared to placebo patients.
In 2017, Remedy sold the CIRARA program to Biogen (a multinational biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide) for $120 million upfront, plus potential milestone and royalty payments that, in total, could be several hundred million dollars or more.
Biogen began enrolling patients into a large, 680-patient phase 3 study of subjects with large hemispheric infarctions in 2018. To quote Biogen, “BIIB093 (CIRARA) has the potential to be the first major innovation in stroke in over 20 years.”
In 2019 Biogen started a second trial of CIRARA, a 160-patient phase 2 study in brain contusion. As Biogen has noted, “We believe that the shared pathophysiologic features of LHI and brain contusion exemplify the interconnectivity of neuroscience that we are leveraging as we continue to increase the depth and breadth of our pipeline.”
The REMEDY/CIRARA story is a perfect example of the potential of repurposing a drug.
CIRARA (glyburide) is an old diabetes drug that had been around for decades. We repurposed it, added IP (reformulated it into an IV, added a special dosing regimen), added several orphan indications, conducted a Phase 2 study and then packaged it up as a Phase 3-ready asset and sold the program for a substantial upfront payment, plus retained significant upside in the future through potential milestone payments and royalties.
By repurposing drugs, we can help reduce the time it takes to bring treatments to those most in need.
At age 32, Jaclyn had a large hemispheric infarction (148 cc lesion). Her LHI occurred the night before her oldest of three children was to start school for the first time.
Doctors warned her family to prepare for the worst. Transferred to a larger trauma hospital Jaclyn’s learned about a phase 2 trial using CIRARA in LHI patients. It was a double-blinded study. There was no way of knowing if she’d get drug or placebo. Her parents made the decision to enroll her anyway. Fortunately Jaclyn received CIRARA. She made a complete recovery.
Jaclyn’s story reminds us of the important mission we are on to help people afflicted with CNS-related edema to not only survive, but live life to the fullest.