Cereno secures loan financing to continue developing CS1 for PAH
Funds to help next-phase testing of CS1, advance expanded access program
Cereno Scientific has secured loan financing to continue developing CS1, its lead therapeutic candidate for pulmonary arterial hypertension (PAH).
The funds, totaling at least 250 million Swedish crowns (nearly $23 million), will help the company reach its milestones into 2026. This includes the U.S. Food and Drug Administration (FDA) approving a next-phase clinical trial to continue testing CS1 in PAH and advancing its expanded access program for compassionate use, which was given the go-ahead by U.S. regulators this year.
“We are happy to announce this new financing, which secures our pipeline’s advancement into 2026,” Sten R. Sörensen, CEO of Cereno, said in a company press release. “With this financial runway, we are well-positioned to reach critical development milestones, including regulatory approval for the next trial of CS1 in PAH.”
In PAH, the pulmonary arteries that carry blood from the heart to the lungs become narrow, restricting blood flow and increasing blood pressure. This makes the heart work harder to pump blood through the body, which can lead to heart failure. PAH symptoms include shortness of breath, weakness, fatigue, chest pain, dizziness, and fainting.
CS1 is reformulation of anti-seizure medication valproic acid
Cereno expects CS1 — a reformulation of the anti-seizure medication valproic acid, which is reported to have pressure-relieving and anti-inflammatory properties — to reverse arterial narrowing and ease symptoms.
The financial agreement comes on the heels of top-line data from the Phase 2a CS1-003 trial (NCT05224531), which was designed to evaluate the safety and preliminary efficacy of CS1 in addition to standard of care in 25 adults with PAH. Participants were randomly assigned to receive one of three daily oral doses (480 mg, 960 mg, or 1,920 mg) for 12 weeks, or about three months.
Data showed the therapy was safe, well tolerated, and improved REVEAL risk scores, which combines several clinical assessments to estimate the risk of death in PAH. Moreover, two-thirds of patients (67%) had a sustained reduction in pulmonary artery blood pressure, as indicated by implanted CardioMEMS devices, which remotely monitor changes in pulmonary artery pressure, an early indicator of the onset of worsening heart failure.
Eligible patients who completed the Phase 2a trial may continue to receive CS1 as part of the expanded access program, which will collect long-term data on safety and efficacy.
Part of the new cash loan is conditional upon Cereno receiving FDA approval to continue testing CS1 in a Phase 2b or a pivotal Phase 3 clinical trial, which would help support the therapy’s regulatory approval in PAH.
CS1 has been granted orphan medicinal product designation by the European Commission and orphan drug status by the FDA for PAH. These designations support the development of therapies for rare diseases and come with certain incentives.
The loans will also support the development of Cereno’s CS014, a medicine with a similar mechanism of action for people with idiopathic pulmonary fibrosis, a condition marked by the buildup of scar tissue in the lungs. Recently, CS014 was found to reverse disease-causing alterations in the structure and arrangement of pulmonary arteries in a preclinical model of PAH.
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