First patient dosed in Cereno Scientific’s Expanded Access Program with 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 first patient has been dosed in the Expanded Access Program (EAP) with CS1 in the rare disease Pulmonary Arterial Hypertension (PAH).
“We are delighted to announce that the first patient in the EAP has been dosed. For the first time a patient, who previously completed the Phase II trial in PAH, has been provided Expanded Access to CS1. This means that the patient will receive CS1 under a prolonged protocol, a decision based on perceived benefit from CS1 treatment judged by the patient and treating physician. We are very happy to be able to provide this extension of CS1 treatment to these patients in need of better therapy. The EAP will also provide for extensive data capture on long-term usage of CS1 in patients with PAH, which will increase our knowledge and our ability to have constructive discussions with regulatory authorities on our next phase of development, targeting pivotal trial”, said Sten R. Sörensen, CEO, Cereno Scientific.
“This is an important milestone for the EAP and we look forward to seeing more patients enrolled in this program, to benefit from continued CS1 treatment, as we simultaneously gather additional data on the long-term use of CS1”, said Dr. Rahul Agrawal, CMO and Head of R&D, Cereno Scientific.
Since January 30 2024, the EAP is approved as an extension of the Phase II trial evaluating CS1 in PAH, under a formal FDA protocol titled “Expanded Access, Open-Label, Safety Extension Study for Patients that Have Completed Parent Study CS1-003 and Who Are Judged by the Investigator to Benefit from Continued CS1 Treatment“. 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. 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.
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 Phase II trial suggesting a potential positive effect of drug candidate CS1 in patients with the severe rare disease PAH.
Patient recruitment to the Phase II trial CS1-003 was closed on July 1st, 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 efficacy in patients with the rare disease pulmonary arterial hypertension (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 Pulmonary Hypertension and thrombosis prevention without increased risk of bleeding. 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 www.cerenoscientific.com.