Cereno Scientific is a clinical-stage biotechnology company developing treatments for cardiovascular disease. The ultimate goal is to improve the quality of life and prolong the lives of people suffering from rare and common cardiovascular diseases, and the work done so far suggests that goal is within reach.
CS1 – harnessing the potential of epigenetic modulation in PAH
The company uses new methods for its drug development, with a focus on offering disease-modifying treatments. The lead project, CS1, is an innovative reformulation of the well-established antiepileptic drug valproic acid and acts as a histone deacetylase inhibitor (HDACi) – an epigenetic modulator – with the potential to restructure blood vessels (reverse remodeling), improve fibrosis, inflammation and thrombosis, and ultimately lower pulmonary artery pressure. These properties set CS1 apart from conventional PAH treatments, which do not address the root cause of the disease.
Read more about how Cereno is driving a paradigm shift in this field thanks to this new therapeutic approach here.
Due to CS1's potential to affect multiple pathogenic mechanisms in PAH, Cereno has chosen to focus on this disease. PAH is a rare and devastating cardiovascular disease that causes an abnormal increase in blood pressure in the arteries of the lungs, which is mainly due to changes in the blood vessels that transport oxygen-poor blood from the heart to the lungs. This means that the right side of the heart has to work extra hard, which eventually leads to heart failure and death within just a few years. CS1 has the potential to be an effective, safe and disease-modifying drug, and the aim of developing CS1 is to offer improved quality of life and prolonged life for patients with PAH.
Orphan drug status for CS1
Given that PAH is so rare, combined with the significant medical need, the disease qualifies as an orphan indication from a regulatory perspective. Cereno was granted orphan drug designation (ODD) for CS1 by FDA years 2020, which gave the company several regulatory advantages that could lead to a faster and easier path to market approval in the US.
Now Cereno can enjoy similar benefits in the 27 EU countries, as CS1 was recently granted orphan drug designation (OMPD) by European Commission following a recommendation from European Medicines Agency (EMA). The main benefit of this is a ten-year market exclusivity after approval, similar to ODD, which offers seven years of exclusivity in the US. This regulatory milestone and subsequent market exclusivity complements the patent protection for CS1 in the EU and strengthens the business case for CS1 in Europe.
Phase II study with CS1
The news comes as Cereno continues its evaluation of the safety, tolerability, pharmacokinetics and exploratory efficacy of CS1 in a Phase II study in PAH. The study will also provide insights for the planning of future studies of CS1. Patient recruitment for the Phase II study was completed in July, based on a recommendation from the clinical steering committee, and topline data are expected in the third quarter.
On January 30, 2024, CS1 was approved by FDA for Expanded Access, or “Compassionate Use,” which allows patients who have completed the study the opportunity to continue taking the drug if it is deemed beneficial by the study investigators and patients. The first patient was recently dosed in the Expanded Access Program (EAP), and Cereno looks forward to more patients joining to benefit from continued CS1 treatment.
Read more about how EAP strengthens the case for CS1 here, and don't miss this BioStock interview with Cereno's CEO where he talks in depth about the benefits of EAP.
Chat with the CEO
BioStock contacted Cereno's CEO Sten R. Sörensen to hear more about the benefits of OMPD and how this status will affect CS1's continued development, as well as the commercial opportunities this opens up for the company.
Sten, how does orphan drug status (OMPD) benefit the development process for CS1?
– The OMPD offers Cereno significant benefits, including ten years of market exclusivity for CS1 after approval, as well as various benefits during the development process, such as fee reductions for marketing approval applications, scientific advice and protocol assistance, accelerated evaluation and assistance with regulatory procedures. These orphan drug incentives will collectively facilitate our efforts to provide CS1 to patients with PAH in all 27 EU countries in a timely manner.
How does this complement the orphan drug (ODD) status already granted by the FDA?
– The EU and the US are both large pharmaceutical markets, both in terms of patient numbers and total market value. We are pleased that CS1 now has enhanced protection, through market exclusivity upon approval, through both ODD and OMPD in the US and Europe. The market exclusivity complements our patents in these markets.
Regarding the market potential in PAH, what commercial opportunities does this latest regulatory milestone open up for Cereno?
– Together, ODD and OMPD provide a coordinated framework of incentives and regulatory support, which, combined with the strong and growing patent protection for CS1 in these markets, lays a solid foundation for market introduction once CS1 has received market approval. OMPD also ensures access to a centralized approval, with one application for all EU countries, providing a simpler path to approval and the opportunity for earlier return on investment for the company and our shareholders.
You recently announced that the first patient was dosed in the Expanded Access program, what does this mean?
– The EAP provides value both to patients suffering from PAH and to Cereno. Under the EAP, patients who have completed the Phase II study, after being deemed suitable and benefiting from CS1 treatment by the study investigators, are given the opportunity to continue CS1 treatment for PAH when no comparable or satisfactory alternative treatment options are available. The EAP also allows Cereno, under a formal FDA-approved protocol, to collect safety and efficacy data from long-term exposure to CS1 in patients with PAH, which can be used in regulatory discussions and the planning of a Phase IIb/III pivotal study.
What are the next steps in the Phase II study of CS1 in PAH that we can look forward to?
– Our next major milestone is to release topline results from the Phase II study. Based on a recommendation from the Study Clinical Steering Committee, we decided to end patient recruitment for the CS1-003 study by July 1. The Clinical Steering Committee concluded that there is sufficient data to evaluate the next steps of development. Topline results will be shared in the third quarter.