Cereno Scientific pivots to rare disease Idiopathic Pulmonary Fibrosis (IPF) as the initial target indication for novel HDAC inhibitor CS014 – strengthens Company focus on rare diseases
Cereno Scientific (Nasdaq First North: CRNO B), a pioneering biotech developing innovative treatments for diseases with high unmet medical needs, today announced that the Company has selected rare disease Idiopathic Pulmonary Fibrosis (IPF) as the initial target indication for novel HDACi CS014. This decision follows recent preclinical results showing significant reductions in pulmonary artery occlusion and fibrosis. With this move, Cereno strengthens its focus on rare diseases, reinforcing its commitment to addressing the significant needs of patients affected by these conditions.
“At Cereno, our mission is to enhance and extend the lives of patients with diseases that have high unmet needs. IPF, a rare disease, is in dire need of new disease-modifying treatments - a need we believe CS014 can help address. Expanding our indication portfolio into additional rare diseases is a natural progression for us, building on the very promising foundation we've established with our pioneering effort with our lead program CS1 in Pulmonary Arterial Hypertension (PAH). We will leverage our expertise in orphan drug development to deliver meaningful benefits for both patients and our shareholders as we advance in this space”, said Sten R. Sörensen, CEO, Cereno Scientific.
IPF initial target indication for CS014
Idiopathic Pulmonary Fibrosis (IPF) is a rare, progressive, and fatal disease characterized by irreversible scarring (fibrosis) of the lungs. Patients experience significant symptoms, including a persistent dry cough, fatigue, and exertional dyspnea. Over time, this fibrosis leads to a gradual loss of lung function, ultimately resulting in respiratory failure. Mortality rates in IPF are comparable to those of severe cancers. There is currently no cure for IPF, and current treatments have severe tolerability issues. There is an outspoken need for new therapies that can halt and reverse disease progression, extend lifespan and improve quality of life of patients suffering from this disease.
Cereno’s HDAC inhibitor CS014 is a new chemical entity with a multi-modal mechanism of action as an epigenetic modulator. The scientific rationale for evaluating CS014 for the treatment of IPF is supported by preclinical studies, which have demonstrated that HDAC inhibitors can effectively reverse fibrosis in models of IPF. The studies also show that HDAC inhibitors prevent the pathological remodeling of pulmonary vessels that ultimately leads to pulmonary hypertension in many IPF patients. The rationale is further strengthened by our recently published preclinical data in an established model of PAH showing that CS014 has an effect on reversal of fibrosis and a dose-dependent beneficial effect on pathological vascular remodeling. Together, these findings indicate that CS014 has the potential to address the underlying pathophysiology behind the development of IPF.
Patients with IPF have an increased risk of developing venous thromboembolism. CS014 regulates platelet activity, local fibrinolysis, and clot stability for the prevention of thrombosis without increasing the risk of bleeding, as demonstrated in preclinical studies. This adds to the favorable profile of CS014 in relation to the unmet needs of IPF patients.
“IPF is characterized by an imbalance in HDAC activities, and HDAC inhibitors have been documented to alleviate the fibrosis in preclinical models of IPF. This, together with our recently published preclinical data showing that CS014 effectively reduces fibrosis in small pulmonary arteries gives us reason to believe that CS014 may be able to offer a disease modifying approach in IPF. Additionally, the anti-thrombotic effect of CS014 could be beneficial since IPF is associated with an increased risk of venous thromboembolism,” said Dr. Björn Dahlöf, CSO, Cereno Scientific.
“Driven by the ambition to enhance patient outcomes, we have identified IPF as a condition that aligns with the strengths of our novel HDAC inhibitor, CS014. Our goal is to provide a disease-modifying treatment for patients suffering from this rare and progressive disease, which currently has no cure,” said Dr. Rahul Agrawal, CMO and Head of R&D, Cereno Scientific
Cereno Scientific eyes pipeline focus on rare diseases
As a result of a chosen initial target indication for CS014, Cereno now advances a pipeline of three innovative drug candidates, two of which are being developed as disease-modifying treatments for rare diseases where high unmet needs persist.
Lead 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. Positive topline results from a Phase IIa trial were reported on September 27, 2024.
CS014 is a novel HDAC inhibitor with epigenetic effects, being developed as a disease-modifying therapy for rare disease IPF. A first-in-human Phase I trial was initiated in Q2 2024.
CS585, a novel IP receptor agonist, 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 for thrombosis prevention without increased risk of bleeding. A target indication for CS585 is currently being evaluated; rare diseases with high unmet medical needs are being considered.
Why increased focus on rare diseases
The key strategic rationale for Cereno’s increasing focus on rare diseases is based on the fact that there are significant unmet medical needs in rare and often fatal diseases. Cereno has the potential to deliver high treatment value to patients leveraging our pioneering portfolio and disease-modifying approach to address the root cause of such diseases. The strategic focus on rare diseases provides an attractive business model for biotech companies due to relatively shorter development timelines and less capital needed to reach market authorization and attractive incentives offered to companies developing orphan drugs. These include the possibility to obtain market exclusivity for 7 and 10 years in US and EU, respectively, through orphan drug status.
Cereno’s move to increase its focus on rare diseases follows on recent significant developments for the Company’s HDACi portfolio. In June this year a Phase I trial with CS014 was initiated and positive topline results, from the Phase IIa trial in rare disease PAH with lead candidate drug CS1, were reported on Sep 27. The Company now stands ready to execute on the new strategic direction for novel HDACi CS014 with focus on rare disease IPF as target indication and a completion of ongoing Phase I program by summer 2025. Further to advance the lead HDACi program CS1, by completing the analysis of the Phase IIa trial in rare disease PAH and subsequently initiate discussions with regulatory bodies aiming to pursue a pivotal trial in PAH.
About CS014
HDAC inhibitor CS014 is a new chemical entity with a multi-modal mechanism of action as an epigenetic modulator, under development for Idiopathic Pulmonary Fibrosis (IPF).
Preclinical studies of HDAC inhibitors show that these drugs can reverse fibrosis in models of IPF. Studies also show that these drugs prevent the pathological remodeling of pulmonary vessels that ultimately leads to pulmonary hypertension in many IPF patients. Preclinical studies of CS014 have demonstrated an effect on reversal of fibrosis and a dose-dependent beneficial effect on pathological vascular remodeling in an established model of PAH. Together, these findings indicate that CS014 has the potential to address the underlying pathophysiology behind the development of IPF.
CS014 has demonstrated, in preclinical studies, the ability to regulate platelet activity, local fibrinolysis, and clot stability, helping to prevent thrombosis without increasing the risk of bleeding. This supports CS014's potential to address key unmet needs in IPF patients.
Cereno’s HDAC inhibitor portfolio, capitalizing on the principle of epigenetic modulation, comprises Cereno’s lead drug candidate CS1 and the investigational drug candidate CS014.
A first-in-man, Phase I trial of CS014 was initiated in June 2024.
This information is information that Cereno Scientific AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 08:00 (CET) on October 17, 2024.
For further information, please contact:
Henrik Westdahl, Director IR & Communications
Email: mailto: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 diseases with high unmet medical needs. Lead drug candidate, CS1, is an HDAC inhibitor that acts as an epigenetic modulator, with pressure-reducing, reverse-remodeling, anti-fibrotic, anti-inflammatory and anti-thrombotic properties. CS1 is being developed as a disease-modifying treatment for the serious and life-threatening 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. 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. An Expanded Access Program enables patients that have completed the Phase IIa trial to gain access to CS1 when no comparable alternative therapy options are available. CS1 holds orphan drug designations in both the US and EU. HDAC inhibitor CS014, in Phase I development, is a new chemical entity being developed as a disease-modifying treatment for rare disease Idiopathic Pulmonary Fibrosis (IPF). Preclinical studies of HDAC inhibitors show that these drugs can reverse fibrosis in models of IPF. Studies also show that these drugs prevent the pathological remodeling of pulmonary vessels that ultimately leads to pulmonary hypertension in many IPF patients. Preclinical studies of CS014 have demonstrated an effect on reversal of fibrosis and a dose-dependent beneficial effect on pathological vascular remodeling in an established model of PAH. Together, these findings indicate that CS014 has the potential to address the underlying pathophysiology behind the development of IPF. CS014 has demonstrated, in preclinical studies, the ability to regulate platelet activity, local fibrinolysis, and clot stability, helping to prevent thrombosis without increasing the risk of bleeding. This supports CS014's potential to address key unmet needs in IPF patients. 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. A target indication for CS585 is currently being evaluated; rare diseases with high unmet medical needs are being considered. CS014 and CS585 are developed through research collaborations with the University of Michigan. 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 Scientific 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.