Cereno Scientific is granted Orphan Medicinal Product Designation in the EU for CS1 in rare disease Pulmonary Arterial Hypertension

Cereno Scientific (Nasdaq First North: CRNO B), a pioneering biotech developing innovative treatments for rare and common cardiovascular disease, today announced that 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).

“I am very pleased to report that CS1 has been granted Orphan Medicinal Product Designation (OMPD) in the EU, covering all 27 EU countries. 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”, said Sten R. Sörensen, CEO, Cereno Scientific. 

About OMPD in the EU

The European Commission is responsible for granting applications, reviewed by the European Medicines Agency (EMA) for OMPD in the EU. To qualify for orphan designation, a drug must meet a number of criteria:

• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
• no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Companies that obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan drugs, and market exclusivity once the drug is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required.

About 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.

For further information, please contact:

Henrik Westdahl, Director IR & Communications

Email: henrik.westdahl@cerenoscientific.com  

Phone: +46 70-817 59 96

Sten R. Sörensen, CEO

Email: sten.sorensen@cerenoscientific.com

Phone: +46 73-374 03 74

About Cereno Scientific AB

Cereno Scientific develops innovative treatments for rare and common cardiovascular disease. The lead drug candidate, CS1, is an HDAC (histone deacetylase) inhibitor that acts as an epigenetic modulator with pressure-reducing, reverse-remodeling, anti-inflammatory, anti-fibrotic and anti-thrombotic properties. A Phase II trial is ongoing (patient recruitment closed on July 1st, 2024) to evaluate CS1’s safety, tolerability, and exploratory efficacy in patients with the rare disease pulmonary arterial hypertension (PAH). The study will also provide insights for planning the subsequent trial of CS1 in PAH. A collaboration agreement with global healthcare company Abbott allows Cereno to use their cutting-edge technology CardioMEMS HF System in the trial. Two initiatives performed during the Phase II trial have shown positive findings suggesting the potential clinical benefit of CS1 in PAH patients. These initial findings are, however, not a guarantee of the final trial results that are expected in Q3 2024. Since January 2024, we are delighted that the FDA´s Expanded Access Program will enable patients with PAH, a serious life-threatening disease condition, to gain access to CS1 where no comparable alternative therapy options are available. Cereno’s pipeline comprises two additional programs in development through research collaborations with the University of Michigan. Investigational drug CS014 is an HDAC inhibitor in Phase I development as a treatment for arterial and venous thrombosis prevention. The innovative drug candidate represents a groundbreaking approach to antithrombotic treatment. CS014 is a new chemical entity with a multi-fold mechanism of action as an epigenetic modulator – regulating platelet activity, fibrinolysis, and clot stability for the prevention of thrombosis without an increased risk of bleeding as documented in preclinical trials. On 28th of June, 2024, Cereno initiated a first-in-human Phase I trial of CS014. Preclinical candidate CS585 is an oral, highly potent and selective prostacyclin (IP) receptor agonist that has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular disease. While CS585 has not yet been assigned a specific indication for clinical development, preclinical data indicates that it could potentially be used in indications like Thrombosis prevention without increased risk of bleeding and Pulmonary Hypertension. CS585 was in-licensed from the University of Michigan in 2023. The Company is headquartered in GoCo Health Innovation City, in Gothenburg, Sweden, and has a US subsidiary; Cereno Scientific Inc. Based in Kendall Square, Boston, Massachusetts, US. Cereno is listed on the Nasdaq First North (CRNO B). The Certified Advisor is Carnegie Investment Bank AB, CA@carnegie.se. More information is on http://www.cerenoscientific.com