CS1 expanded access program for PAH now covers 10 patients
Cereno says more people may be enrolled in coming months
Cereno Scientific said it has enrolled nine additional people with pulmonary arterial hypertension (PAH) in its expanded access program (EAP) for investigational therapy CS1, bringing the total number of patients in the program to 10.
The patients will continue receiving CS1 after completing a Phase 2a clinical trial. The company said it may add more patients to the EAP, approved by the U.S. Food and Drug Administration (FDA) in 2024, in the coming months.
The additional long-term safety and efficacy data generated by the program will support the therapy’s documentation, help with required regulatory interactions, and inform future clinical trial planning.
“We are very pleased to see the high interest from patients and physicians to continue with the treatment of CS1 after the completed Phase IIa study,” Sten R. Sörensen, Cereno’s CEO, said in a company press release. “From a drug development perspective, it is very valuable for us to be able to collect long-term clinical data on CS1 as we move forward with our plans for the next clinical program.”
PAH is marked by pulmonary vascular remodeling, in which the cells that line the walls of the lung’s blood vessels grow uncontrollably. This narrows the arteries, restricts blood flow, and increases blood pressure, leading to symptoms including shortness of breath, chest pain, dizziness, and fainting.
Extending survival
CS1 is a reformulation of valproic acid, an anti-seizure medication reported to have pressure-relieving, anti-scarring, and anti-inflammatory properties. Cereno expects the therapy will reverse the pulmonary vascular remodeling that drives PAH and extend survival for patients.
The Phase 2a CS1-003 trial (NCT05224531) evaluated the safety and preliminary efficacy of CS1 on top of standard of care in 25 adults with PAH. Participants received one of three daily oral doses (480 mg, 960 mg, or 1,920 mg) for 12 weeks.
Top-line data showed that the therapy was safe and well tolerated and stabilized or improved REVEAL risk scores, a validated measure of several clinical factors that estimate mortality risk in PAH. Implanted CardioMEMS devices, which allow daily, noninvasive monitoring of arterial blood pressure, showed that 67% of patients sustained a reduction in pulmonary artery pressure.
If study physicians deem them suitable, patients who complete the Phase 2a trial can continue to receive CS1 in the EAP.
For certain patients in the EAP, the long-term impact of CS1 on the pulmonary arteries will be visualized using a noninvasive imaging technology called Functional Respiratory Imaging (FRI) made by Fluidda.
Cereno recently secured at least 250 million Swedish crowns (nearly $22.5 million) in loan financing to advance the EAP and receive FDA approval to continue testing CS1 in a Phase 2b or pivotal Phase 3 trial. Together, these would help support the therapy’s regulatory approval in PAH.
The loans will also help Cereno develop CS014, a medicine with a similar mechanism of action for the treatment of idiopathic pulmonary fibrosis, a condition characterized by the buildup of scar tissue in the lungs.
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