TUE, SEP 29, 2020 17:30 CET
Cereno Scientific today announced that the company will advance the development of drug candidate CS1 as an epigenetic modulator of rare diseases with orphan drug potential. The initial focus will be on pulmonary arterial hypertension (PAH), a form of high blood pressure in the lungs. The company’s revised clinical development strategy for CS1 was formed together with top commercial, scientific and regulatory advisors in Europe and the US with insights from the American authority, US FDA. A Phase II study program is planned to be started during first half of 2021.
The clinical development program for the drug candidate CS1 in PAH is anchored in the Orphan Drug Designation (ODD) that was granted by the American regulatory agency US FDA in March 2020. The ODD status is a validation of the major unmet clinical needs within the treatment of PAH and that CS1 has fulfilled the criteria of showing a potential to provide significant benefit to these patients. Several incentives are associated with ODDs to facilitate the drug development for rare diseases, such as seven years of market exclusivity in the US if the drug is approved, FDA assistance in clinical trial design, and tax credits for qualified clinical trial costs. The Phase IIa study with CS1 in PAH is planned to be started during first half of 2021 and conducted in the US.
“We strongly believe that the newly revised strategy for CS1 brings a strengthened commercial potential and well-defined business case toward potential investors as well as partners,” said Sten R. Sörensen, CEO at Cereno Scientific. “We are confident in the expert-led comprehensive assessment complemented by the insights from the US FDA around the ODD request interactions, that has brought us here and excited for this next stage in the development of CS1. The rare disease space opens up for several new possibilities for us and we hope to ultimately be able to help PAH patients with a new disease-modifying treatment option.”
PAH is a rare progressive vascular disease leading to a poor quality of life for those affected. The disease is characterized by elevated pulmonary vascular resistance leading to high pulmonary arterial pressure, local arterial thrombosis, right heart failure and, ultimately, death. Despite recent treatment advances, existing treatments only improve function and a moderate delay of progression.
Preparations for the Phase IIa study in PAH with CS1 is underway with the aim to apply for regulatory permission to start the study at the end of this year. Cereno’s development program for CS1 in VTE/SPAF will be deferred to follow after the Phase II study program in PAH. The preclinical HDACi development program is not affected and proceeds according to plan.
For further information, please contact:
Daniel Brodén, CFO
Tel: +46 768 66 77 87
This information is information that Cereno Scientific AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 17:30 CET on September 29, 2020.
About Pulmonary Arterial Hypertension (PAH)
PAH is a specific form of pulmonary hypertension, a fatal disease with poor quality of life. The increase in pulmonary pressure is caused by thickening of the walls of the pulmonary arteries (blood vessels leading from the right side of the heart to the lungs) that become thick and stiff, narrowing the space for the blood to flow causing increased blood pressure in the lung, at later stages worsened by locally formed thrombosis. PAH is a serious, progressive disease with a variety of aetiologias that ultimately leads to right heart failure and poor lung function. The disease has major impact on the individuals functioning, as well as their physical, psychological and social well-being. There is currently no cure for PAH with the exception of lung transplantation, which at that point patients are often in a too precarious state. Today’s medication only improves function and a moderate delay of disease progression, causing the large unmet need for disease-modifying therapy.
About Cereno Scientific AB
Cereno Scientific is a leading clinical stage biotech company within cardiovascular epigenetic modulation. The lead drug candidate, CS1, is a Phase II candidate in development for the treatment of the rare disease pulmonary arterial hypertension (PAH) and thrombotic indications. CS1 is an HDAC (Histone DeACetylase) inhibitor that acts as an epigenetic modulator with anti-thrombotic, anti-inflammatory, anti-fibrotic and pressure-relieving properties, all relevant for PAH. A clinical phase II study program for CS1 in PAH is planned to start during first half of 2021 under its US FDA granted orphan drug designation (ODD) status. In addition, Cereno has a preclinical HDAC inhibitor development program targeted at treating cardiovascular diseases. The company is headquartered in AstraZeneca’s BioVenture Hub, Sweden, and has an office in Kendall Square in Boston, Massachusetts, US. Cereno is listed on the Swedish Spotlight Stock Market (CRNO B). More information on www.cerenoscientific.com.