According to a recent press release from Respira Therapeutics, their investigational treatment for pulmonary arterial hypertension (PAH), RT234, has just been administered to the first patient in a phase 2b trial. RT234 comes in a powder formula and is administered through the use of an inhaler, making it a convenient and easy-to-use option for patients. Additionally, it is intended to alleviate classic PAH symptoms like shortness of breath, which allows patients to be more active and enjoy their lives with more freedom.
About PAH
First up, let’s understand what pulmonary arterial hypertension is. 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.
About the Trial
Titled VIPAH-PRN, this multi-center, dose-escalation, phase 2b trial was created to investigate RT234’s efficacy and safety. Specifically, it will evaluate the improvement of episodic symptoms alongside exercise capacity in PAH patients whose symptoms fall into New York Heart Association (NYHA) Functional Classes II and III. There will be two consecutive cohorts, both of which will utilize single dosing via an Axial Oscillating Sphere dry powder inhaler.
It has now officially begun, as the first participant has been dosed in the U.S. This marks an important step in drug development, and it also brings PAH patients one step closer to a new treatment option. It also addresses episodic symptoms, effects that many patients experience despite maintenance therapy. If clinical development is successful and RT234 becomes available to patients, it could allow them to be more active, which then goes on to improve mental and physical health.
