Phase 1 Trial of R-107 for PAH Approved in Australia

According to a recent press release, the Australian Human Research Ethics Committee (HREC) has granted approval to Claritas Pharmaceuticals’ submission for R-107. Now, the company is free to move forward with a Phase 1 clinical trial of their nitric oxide-releasing compound, which comes in a liquid formula and treats pulmonary arterial hypertension (PAH). The first step is enrollment, which they began immediately.

The goal of this trial and the overall development of R-107 is to create an easier and more accessible way to administer nitric oxide, as the only option currently available is inhalation therapy. This formulation is expensive and needs to be administered by a trained respiratory therapist. If R-107 becomes available, PAH patients will be able to take their medication as a liquid delivered through a variety of options (e.g., capsule, injection, by suppository, nasal spray, by nebulizer, ointment).

Now, the phase 1 trial can move forward. It will be a single-center (conducted at Scientia Clinical Research), double-blind, and single-ascending dose (SAD) escalation study with the intention of evaluating R-107’s pharmacokinetics, tolerability, and safety when administered via intramuscular injection. 40 subjects are planned to enroll across 5 cohorts. If all goes well, R-107 will move forward with phase 2a trial.

About Pulmonary Arterial Hypertension (PAH)

PAH is a progressive form of high blood pressure that is characterized by the thickening of the pulmonary arteries. These arteries become blocked, which forces the heart to work harder to push blood through, which then leaves less oxygen-rich blood for the rest of the body. Additionally, the heart gets weaker as it has to work harder and harder to do its job. These issues lead to symptoms such as chest pain, fatigue, fainting, dizziness, shortness of breath, and swelling in the legs and ankles.

A mutated BMPR2 gene can cause this condition, although the use of street drugs or other diseases can also result in PAH. If the cause is genetic, the mutation is passed down in an autosomal dominant pattern. In terms of treatment, there is no cure; it focuses on slowing progression and addressing symptoms. Options include vasodilators, guanylate cyclase stimulators, sildenafil, endothelin receptor agonists, tadalafil, warfarin, high-dose calcium channel blockers, diuretics, oxygen therapy, digoxin, atrial septostomy, and a lung or heart transplant.

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