Cereno Scientific (Nasdaq First North: CRNO B), an innovative biotech pioneering treatments to enhance and extend life for people with rare cardiovascular and pulmonary diseases, today announced that a new patent has been granted in the US for drug candidate CS1's second patent family. Two patent applications have, additionally, been filed based on the encouraging efficacy signals observed in the recently completed Phase IIa trial of CS1 in the rare disease pulmonary arterial hypertension (PAH). These patent applications combined with the existing patent portfolio has the potential to extend the market exclusivity for CS1 in PAH to 2045.
"Securing a patent not only ensures that our innovative treatments are protected, but it strengthens our commercial position related to market exclusivity. We have already received orphan drug designation (ODD) in the US and Europe granting market exclusivity for 7 and 10 years, respectively. With these new patents, if granted, there is a possibility of further extending the market exclusivity for CS1 by about 7 years," said Sten R. Sörensen, CEO of Cereno Scientific. "A strong patent portfolio is an important aspect of continuing to build upon an attractive commercial positioning for CS1 and for Cereno Scientific."
The granted patent in the US is the third approved patent for the second patent family, which broadens the protection already obtained in this key market for drug candidate CS1. The patent is titled "Valproic acid for the treatment or prevention of pathological conditions associated with excess fibrin deposition and/or thrombus formation" and has been given patent number 12,245,999 by the US Patent Office (USPTO).
The two newly filed patent applications relate to the recently completed Phase IIa trial in PAH. The trial showed efficacy signals suggesting reverse vascular remodeling effects of CS1. This was accompanied by signals suggesting improved right ventricular function, functional class (NYHA) and quality of life (QoL) as well as improved REVEAL 2.0 risk score. These parameters indicate better patient outcomes, improvement and/or stabilization of disease progression, as well as improving patient function and prognosis. The trial also showed that CS1 has a good safety profile and is well-tolerated. An ongoing Extended Access Program (EAP) allowing patients to continue CS1 treatment will provide further insight into the long-term disease-modification effects of CS1. A sub-study of the EAP utilizes the non-invasive imaging technology Functional Respiratory Imaging (FRI) developed by Fluidda to visualize how long-term treatment of CS1 on top of standard therapy may impact changes in pulmonary arteries. Cereno Scientific intends to continue to explore the effects of CS1 on reverse vascular remodeling in further clinical development. A larger placebo-controlled Phase IIb trial is being planned to confirm and expand upon these effects.
For further information, please contact:
Tove Bergenholt, Head of IR & Communications
Email: tove.bergenholt@cerenoscientific.com
Phone: +46 73- 236 62 46
Sten R. Sörensen, CEO
Email: sten.sorensen@cerenoscientific.com
Phone: +46 73-374 03 74
About CS1
Drug candidate CS1 is a Class I HDAC inhibitor (HDACi) that works through epigenetic modulation, being developed as a safe, effective and disease-modifying treatment for the rare disease pulmonary arterial hypertension (PAH). CS1 targets the root mechanism of the disease, aiming to reverse the pathological vascular remodeling of the small lung arteries.
In a Phase IIa study, CS1 successfully met the primary endpoint of safety and tolerability. CS1 further showed compelling positive impact on exploratory parameters of clinical relevance over the 12-week treatment period. The Phase IIa data, together with preclinical data from the HDACi program supports reverse remodeling in small lung arteries. This provides a basis of assumption that CS1 may act with disease-modifying capacity in PAH. In preclinical cardiovascular disease models, HDAC-inhibitors have also shown anti-fibrotic, anti-inflammatory, pulmonary pressure-reducing, and anti-thrombotic effects. CS1's unique efficacy profile aligns well with the underlying mechanisms of disease that drives the progression of PAH, positioning it to address the critical unmet need for more effective treatment options. The goal of CS1's development is to enhance and extend life for patients with PAH. CS1 is part of Cereno's HDACi portfolio, untapping the potential of epigenetic modulation in rare cardiovascular and pulmonary diseases. CS1 has been granted orphan drug status in both the US and EU.
CS1 has been approved by the FDA for an Expanded Access Program (EAP) as an extension of the Phase IIa trial in PAH, which enables Cereno to obtain valuable long-term safety and efficacy data of CS1 in PAH patients. Ten patients are currently enrolled in the program, which allows patients who have completed the Phase IIa trial to continue CS1 treatment if deemed suitable by physicians in the study. Through a collaboration with innovative medical company Fluidda, their non-invasive imaging technology called Functional Respiratory Imaging (FRI) will be used on certain patients enrolled in the EAP to visualize how long-term use of CS1 influences changes in pulmonary arteries. The data obtained by the EAP supports required regulatory interactions and planning future Phase IIb or pivotal Phase III trials.
About Cereno Scientific AB
Cereno Scientific is pioneering treatments to enhance and extend life. Our innovative pipeline offers disease-modifying drug candidates to empower people suffering from rare cardiovascular and pulmonary diseases to live life to the full.
Lead candidate CS1 is an HDACi that works through epigenetic modulation, being developed as a safe, effective and disease modifying treatment for rare disease Pulmonary Arterial Hypertension (PAH). A Phase IIa trial evaluating CS1's safety, tolerability, and exploratory efficacy in patients with PAH demonstrated that CS1 was safe, well-tolerated and showed a positive impact on exploratory clinical efficacy parameters. An Expanded Access Program enables patients that have completed the Phase IIa trial to gain access to CS1. HDACi CS014, in Phase I development, is a new chemical entity with disease-modifying potential. CS014 employs a multi-modal mechanism of action as an epigenetic modulator, targeting key unmet needs in patients with rare disease Idiopathic Pulmonary Fibrosis (IPF). Cereno Scientific is also pursuing a preclinical program with CS585, an oral, highly potent and selective prostacyclin (IP) receptor agonist that has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular diseases. 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.
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 Scientific is listed on the Nasdaq First North (CRNO B). The Certified Adviser is Carnegie Investment Bank AB, certifiedadviser@carnegie.se. More information can be found on www.cerenoscientific.com.
