Cereno Scientific signs agreement with Fluidda, to evaluate the impact of its HDAC inhibitor CS1 on reverse remodeling of pulmonary vessels in patients with PAH
Cereno Scientific (Nasdaq First North: CRNO B), a pioneering biotech developing innovative treatments for rare and common cardiovascular disease, today announced that the Company has signed an agreement with medical technology company Fluidda on Respiratory Imaging solutions, with the aim to visualize signs of reverse remodeling of lead drug candidate CS1 in Pulmonary Arterial Hypertension (PAH) in a clinical setting.
“We have a vision to develop new therapies which address the root cause of cardiovascular disease as we believe this will provide high value to patients with respect to improvement of quality of life and survival. On the heels of our increasing knowledge of impact of reverse remodeling capacity of HDACi from our preclinical program CS014, together with our top line results from our Phase IIa study with our lead HDACi program CS1, I am excited to announce our partnership with Fluidda. This collaboration will allow Cereno to use Fluidda’s cutting-edge technology to visualize CS1’s ability for long-term reverse remodeling in PAH patients,” said Sten R. Sörensen, CEO, Cereno Scientific
“We are excited to collaborate with Cereno Scientific in tackling the significant challenges in the pulmonary hypertension field. Pulmonary hypertension remains a complex condition where comprehensive understanding of vascular disease including remodeling is critical for effective treatment. Fluidda's functional respiratory imaging (FRI) technology offers unique insights into the structural and functional changes in the lungs and blood vessels, allowing us to assess the impact of therapies more precisely. By partnering with Cereno, we aim to accelerate advancements in detecting and understanding these changes, ultimately improving patient outcomes,” said Jan De Backer, CEO Fluidda
CS1 is an HDAC inhibitor that works through epigenetic modulation, being developed as a safe, effective and disease modifying treatment for PAH. CS1 targets the root cause of the disease, aiming to reverse the pathological vascular remodeling of the small lung arteries, which is a hallmark feature of PAH.
On the 27th of September 2024 Cereno Scientific presented topline results from the Phase IIa trial evaluating the safety, tolerability and exploratory efficacy of CS1 in patients with PAH. CS1 was demonstrated to be safe, well-tolerated and showed a positive impact on exploratory clinical efficacy parameters including improvement of REVEAL risk score, functional class and mean Pulmonary Arterial Pressure (mPAP). CS1 Phase IIa study data, together with preclinical information, is consistent with reversing pathological remodeling.
Preclinical data
Previously published preclinical data has shown the ability of CS1s active substance VPA to both prevent and reverse remodeling in animal models of PAH.
On September 25, 2024, Cereno reported preclinical data out of the HDACi program. The study demonstrated that novel HDACi CS014 induced a robust, dose dependent reversal of the pulmonary vascular remodeling in an established preclinical PAH model. This included statistically significant reductions in small artery vessel occlusion, plexiform lesions and small vessel related fibrosis.
Cereno and Fluidda collaboration
To deepen the exploration of CS1’s impact on pathological vascular remodeling of small pulmonary arteries, Cereno is actively engaging with a leading PAH specialist to launch an investigator-initiated trial (IIT). This trial aims to harness Fluidda’s innovative, non-invasive imaging technology to visualize how long-term use of CS1 influences structural changes in pulmonary arteries. The trial seeks to provide valuable insights into CS1's potential to transform PAH treatment. Some patients enrolled in the Expanded Access Program are planned to be included in this IIT.
“This work will give us a deeper understanding of our lead candidate CS1 and further support us in optimizing the road to approval of CS1, and subsequently into the clinic, for the benefit of patients suffering from PAH”, said Dr. Rahul Agrawal, CMO and Head of R&D, Cereno Scientific.
CT-based Functional Respiratory Imaging (FRI) has been developed by Fluidda and enables the visualization and quantification of regional lung structures. FRI is not only able to assess the effect of respiratory therapies, it is also used to define respiratory disease stage, assess disease progression, and can act as a companion diagnostic in surgical procedures. Fluidda FRI technology can quantify regional structures of the lungs in great detail, making it a valuable tool in Cereno Scientific’s further development of CS1 in PAH. This allows the Company to study CS1’s effect and disease-modifying capacity through reverse remodeling in PAH patients.
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 holds the potential to be an effective, safe and disease-modifying drug, targeting the root cause of the disease by reverse remodeling.
In preclinical cardiovascular disease models, HDAC inhibitors have shown disease-modifying potential through reverse pathological remodeling, as well as anti-fibrotic, anti-inflammatory, pulmonary pressure-reducing, and anti-thrombotic effects. CS1’s unique efficacy profile aligns well with the underlying mechanisms of PAH, positioning it to address the critical unmet need for more effective treatment options. The goal of CS1's development is to improve quality of life and extend survival for patients with PAH.
CS1 is part of Cereno’s HDACi portfolio, untapping the potential of epigenetic modulation in CVD. CS1 has been granted orphan drug status in both the US and EU.
Since January 30, 2024, CS1 is approved by the FDA for Expanded Access, as an extension of the Phase IIa trial CS1-003 with CS1 in PAH. The EAP gives patients that have completed the Phase IIa 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.
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 IIa trial evaluating CS1’s safety, tolerability, and exploratory efficacy in patients with the rare disease pulmonary arterial hypertension (PAH) demonstrated that CS1 was safe, well-tolerated and showed a positive impact on exploratory clinical efficacy parameters. CS1 study data, together with preclinical information, is consistent with reversing pathological remodeling. A collaboration agreement with global healthcare company Abbott allowed Cereno to use their cutting-edge technology CardioMEMS HF System in the trial. 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 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. The drug candidate has also demonstrated a favorable profile in preclinical models of other cardiovascular diseases, such as PAH, with reverse remodeling of pulmonary arterial vessels and effects on vascular fibrosis. 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 Adviser is Carnegie Investment Bank AB, certifiedadviser@carnegie.se. More information is on www.cerenoscientific.com.
About Fluidda
Fluidda is the world leader in the field of Functional Respiratory Imaging. This technique combines HRCT scans and Computational Fluid Dynamics technology, which offers vast improvements by making clinical trials shorter, faster and thus, more cost effective.
FRI also helps patients and healthcare providers in offering a unique entry point in personalized medicine, by optimizing diagnosis, monitoring disease progression and the effects of therapy. Fluidda is both a CRO services company and a partner of healthcare professionals by offering worldwide evidence for better respiratory treatment. More information on www.fluidda.com.