Cereno Scientific expands patent protection for PAH drug candidate CS1:s second and third patent family

Cereno Scientific (Nasdaq First North: CRNO B), a pioneering biotech developing innovative treatments for rare and common cardiovascular disease, today announced two new granted patents; a patent in CS1:s second patent family has been issued in New Zealand and a second patent in CS1:s third patent family has been issued in the US.

“I am delighted to announce two new patents for our leading drug candidate, CS1, in Phase II development for the rare disease Pulmonary Arterial Hypertension (PAH). Our continuous work in securing a strong IPR portfolio has resulted in yet another expansion of CS1’s patent protection, an important building block of CS1’s commercial strategy,” said Sten R. Sörensen, CEO, Cereno Scientific.

The newly granted patent in New Zealand for Cereno’s second patent family is titled “Valproic acid for the treatment or prevention of pathological conditions associated with excess fibrin deposition and/or thrombus formation” and has been issued as patent number 730307.

The newly granted patent in the US is the second granted patent for the third patent family, expanding the previous patent protection for drug candidate CS1. The patent is titled “Delayed Release Pharmaceutical Formulations Comprising Valproic Acid, and Uses Thereof” and has been given patent number 12,023,311 by the USPTO.

About CS1

The 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 with CS1 for patients with PAH.

CS1 is being evaluated in a Phase II trial for which recruitment was closed July 1 and topline results will be shared in Q3. 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.

Remarkable Patient Case with CS1 reported June 2023

A case study performed on the first patient having completed the trial at one of the study centers showed remarkable efficacy data. After 12 weeks of treatment with CS1, the patient showed a 30% reduction in pulmonary arterial pressure and a 20% increase in cardiac output. The patient’s overall functional status was changed from NYHA/WHO functional class II to I at the end of the treatment period, meaning that the patient had next to normal functional physical capacity with CS1 added to stable conventional therapy. In addition to the data related to the effects of CS1 in the PAH patient, the case study indicated that using the CardioMEMS permits safe daily remote monitoring of pulmonary arterial (PA) pressure over time in patients with PAH, permitting assessment of medication effectiveness on an individual patient level. 

 

Data Quality Control Review reported October 13th, 2023

Cereno reported in October 2023 that a Data Quality Control Review (DQCR), of data obtained by the CardioMEMS HF System from the first 16 patients, was concluded with positive findings. The data quality of the CardioMEMS measurements was found satisfactory with adherence to study protocol and with timely data transfers from the patient's home to the clinic. Efficacy findings showed a clinically meaningful reduction of pulmonary pressure in several patients, of a similar or greater magnitude as in the Patient Case.

EAP for CS1 Approved Jan 30th, 2024

Since January 30th, 2024, CS1 is approved by the FDA for Expanded Access, an extension of the ongoing Phase II trial evaluating CS1 in PAH. The Expanded Access Program (EAP) will provide Cereno with the opportunity to, under a formal FDA-approved protocol, collect safety and efficacy data from long-term exposure to CS1 in patients with PAH. 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 trial design planning. Currently site-specific contracts and IRB approvals are being progressed.

The final results of the trial may differ from the above findings, and they should not in any way be seen as a guarantee regarding the outcome and conclusions of the upcoming final Phase II trial results.

 

For further information, please contact:

Henrik Westdahl, Director IR & Communications

Email: [email protected]

Phone: +46 70-817 59 96

 

Sten R. Sörensen, CEO

Email: [email protected]

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 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 ongoing 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, [email protected]. More information is on www.cerenoscientific.com.