New Updates are Available on a Phase 2 Trial Evaluating CS1 for PAH

When it comes to finding novel ways to treat or cure cardiovascular diseases, Cereno Scientific is committed to making change. The clinical-stage biotechnology company works in the realm of both common and rare cardiovascular diseases. In a mid-May 2023 news release, the company shared updates from its clinical program for CS1, a new advanced reformulation of valproic acid. CS1 is being developed for pulmonary arterial hypertension (PAH) and is currently being evaluated in a Phase 2 clinical trial.

Cereno Scientific explains that CS1:

acts as an epigenetic modulator with anti-thrombotic, anti-inflammatory, anti-fibrotic, and pressure-relieving properties.

It has shown promise in preclinical and Phase 1 studies, as well as a good safety and tolerability profile. In the Phase 2 study, 16 patients have enrolled, though the company hopes to enroll up to 30. Only two have completed the study so far, but the initial data shows promise. The remainder of participants have either already had the CardioMEMS HF System implanted for monitoring and/or have began CS1 treatment. Within the trial, researchers hope to determine the ideal dose. Top-line data is expected by the end of this year.

About Pulmonary Arterial Hypertension (PAH)

The BMPR2 gene is believed to play a role in controlling cell growth, division, and apoptosis. But when this gene becomes mutated, health issues like pulmonary arterial hypertension can occur. PAH is a chronic and progressive form of high blood pressure in the lungs. Over time, it causes pulmonary arteries to thicken and harden, making it more difficult to circulate oxygenated blood. The heart has to pump harder to receive oxygen, which means that oxygen isn’t making it to the rest of the body. In addition to damaging the pulmonary arteries, PAH also causes the heart muscle to become weaker and fatigued. An estimated 500-1,000 new PAH cases are diagnosed nationwide each year and it is significantly more common in women between 30-60 years old. Although BMPR2 is a common cause of PAH, this condition may also be caused by recreational drug use, HIV, congenital heart disease or congestive heart failure, lung and liver diseases, and sickle cell disease. Symptoms can include:

  • Dizziness and/or fainting
  • An enlarged heart and liver
  • Swelling of the lower extremities
  • Chest pain
  • Fatigue
  • A cough that may produce blood
  • Hoarseness
  • Cyanosis (bluish tinge to the lips and skin)
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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