Cereno awarded compassionate use of CS1 for PAH
Ongoing Phase 2 trial is running at 10 clinics in U.S., recruiting in Sweden
The U.S. Food and Drug Administration (FDA) has approved Cereno Scientific’s investigational drug CS1 in an extension of the ongoing Phase 2 clinical trial testing it in people with pulmonary arterial hypertension (PAH). This expanded access is sometimes called compassionate use.
The Phase 2 trial (NCT05224531) is assessing the safety, tolerability, pharmacological properties, and preliminary efficacy of three doses of CS1. It’s expected to enroll 30 PAH patients, with topline data due around mid-year.
“I am very pleased that we now have received FDA’s approval for the Extended Access Program (EAP) to CS1 and delighted for the patients who will now be able to continue receiving CS1 as drug therapy providing potential clinical benefits,” Sten R. Sörensen, Cereno’s CEO, said in a company press release.
CS1 is a reformulation of valproic acid, an anti-seizure medication. Its therapeutic benefits for PAH are thought to be related to CS1’s pressure-reducing and anti-inflammatory, anti-scarring and anti-blood clotting properties. It works as an epigenetic modulator, meaning it makes changes to genes, but not to the DNA sequence itself, to block the enzyme histone deacetylase.
The ongoing Phase 2 trial is running at 10 specialist clinics in the U.S. and recruiting at a site in Sweden. The trial is using Abbott‘s CardioMEMS HF System, a wireless, implantable monitor to continuously measure pulmonary arterial pressure, or the blood pressure in the arteries of the lungs.
Effects of CS1 on PAH
The company has reported positive efficacy data. In the first patient who completed the study, 12 weeks (or about three months) of treatment decreased pulmonary hypertension by 30% and increased cardiac output, the amount of blood pumped by the heart in one minute, by 20%.
Moreover, the patient’s physical function improved to next to normal. The quality of CardioMEMS-collected data was satisfactory, with timely data transfers from the patient’s home to the clinic.
CS1 also led to a clinically meaningful reduction of pulmonary pressure in several other patients, Cereno said.
The EAP offers participants who’ve completed the Phase 2 study the option to continue on CS1. This enables the company to collect long-term safety and efficacy results in patients.
Having more data might enable Cereno to reach certain drug development milestones and obtain regulatory clearance for a Phase 2b/3 trial to support FDA approval.
“I’m excited to be able to extend the use of CS1 to patients with this deadly disease who are in critical need of additional high-quality supportive therapy” said Raymond Benza, MD, chief of PH for the Mount Sinai Health System and principal investigator of the trial. Benza is also a member of Cereno’s clinical steering committee and chairman of its scientific advisory board.
Sörensen said Cereno has secured a long-term supply of CS1 by entering a scale-up manufacturing agreement with CordenPharma that will make sure patients in the EAP have access to the therapy and its available for the next clinical study.