Reviva Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to RP5063 as a potential treatment of pulmonary arterial hypertension (PAH). The product is already in clinical testing to treat psychosis.
“We are pleased to receive Orphan Drug Designation for RP5063, which emphasizes the significant need for new medications for patients suffering from PAH,” Laxminarayan Bhat, PhD, founder, president and chief executive officer of Reviva, said in a press release.
Based on preclinical and current clinical data, the company thinks RP5063 could be a first-in-class treatment for PAH, as well as for associated psychiatric symptoms like depression.
“Having already completed clinical studies phase 1, and phase 2 in patients with schizophrenia and schizoaffective disorders, we look forward to rapidly advancing this drug candidate into a phase 2 study in PAH patients. Many PAH patients also suffer from comorbid depression and psychosis which current medications do not address,” said Marc Cantillon, MD, the company’s chief medical officer.
In the Phase 2 trial (REFRESH; NCT01490086) in patients with schizophrenia and schizoaffective disorders, RP5063 showed consistent effectiveness with remission in acute schizophrenia, as well as promising results for comorbid negative, cognition, depression and mood disorders. Furthermore, RP5063 demonstrated a good safety and tolerability profile, with no reports of weight gain, or metabolic, cardiac or movement side effects.
RP5063, a new chemical entity, works through multimodal modulation of dopamine and serotonin receptors. Serotonin plays a key role in how the human brain, lungs and heart work, and serotonin signaling is thought to be involved in PAH development.
In preclinical studies in animal models of PAH, RP5063 has been shown to lower mean pulmonary arterial pressure, to decrease respiratory resistance, bring blood oxygen levels back to normal, and significantly reduce cytokine levels. These studies also showed that RP5063 reduced pulmonary arterial vessel wall thickness and muscular tissue.
“Based on the mechanism of action, demonstrated preclinical efficacy, and convenient delivery options for enhanced compliance, we believe that RP5063 could become a ‘first in class’ therapy for PAH,” Cantillon said.