The European Commission has granted Orphan Medicinal Product Designation (OMPD) to the company’s lead drug candidate CS1 in the rare disease Pulmonary Arterial Hypertension (PAH).

“This designation offers significant advantages, including a 10-year market exclusivity period post-authorization and various benefits during the development process, facilitating our efforts to bring CS1 to patients with PAH in Europe. The EU OMPD complements the Orphan Drug Designation (ODD) granted by the US FDA in 2020, enhancing the protection and value of CS1 in these major markets. These market exclusivities, alongside our robust patent portfolio, are integral to our commercial strategy for CS1,” says Sten R. Sörensen, CEO, Cereno Scientific.

CS1

Drug candidate CS1 is an HDAC inhibitor that works through epigenetic modulation, being developed as a treatment for the rare disease PAH. CS1 has the potential to be an effective, safe and disease-modifying drug. CS1’s unique efficacy profile fits well with the pathogenetic mechanisms of PAH and is believed to be able to address today’s major unmet need for better treatment alternatives. The aim of CS1’s development is to offer improved quality of life and prolonged life for patients with PAH.

Cereno Scientific has over the last year reported encouraging findings from the ongoing Phase II study suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH.

Since January 30th, 2024, CS1 is approved by the FDA for Expanded Access, as an extension of the ongoing Phase II trial CS1-003 evaluating CS1 in PAH. The EAP gives patients that have completed the Phase II trial the opportunity to, after being judged suitable and to benefit from CS1 treatment by investigators, continue CS1 treatment of PAH when no comparable or satisfactory alternative therapy options are available. The EAP allows Cereno to, under a formal FDA-approved protocol, collect safety and efficacy data from long-term exposure to CS1 in patients with PAH. As such, this initiative not only supports the treatment of PAH patients but also enables Cereno to gather additional CS1 usage documentation for regulatory discussions and Phase IIb/III pivotal study design planning. On August 30, 2024, the Company announced that the first patient had been dosed under the EAP.

Patient recruitment to the Phase II trial CS1-003 was closed on July 1, 2024, based on a recommendation by the Study Clinical Steering Committee, which concluded that there is sufficient data for evaluating the next steps in development. Topline results will be shared in Q3, 2024.

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