Impactful third quarter for Cereno
Swedish biotech Cereno Scientific has delivered a transformative Q3 2024, highlighted by positive phase IIa results for its lead candidate, CS1. Backed by a series of significant milestones, a strengthened focus on rare diseases and a recent SEK 250 million capital injection, Cereno is decisively advancing towards key milestones in 2025. At the same time, the company is progressing business development activities, actively evaluating potential partners for its pipeline of development.
Cereno Scientific is a clinical-stage biotech company developing treatments for rare diseases. Its portfolio includes three innovative drug candidates — CS1, CS014, and CS585 — currently undergoing evaluation in separate, parallel development programs.
A pivotal event in Cereno’s Q3 2024 was the announcement of topline results from the phase IIa trial of CS1, Cereno’s lead candidate being developed as a disease-modifying treatment for the rare disease pulmonary arterial hypertension (PAH). CS1, a histone deacetylase (HDAC) inhibitor acting through epigenetic modulation, was demonstrated to be safe and well tolerated, with no serious adverse events related to the candidate.
Compelling positive impact on exploratory clinical efficacy parameters
Key exploratory efficacy results were equally compelling. Patients treated with CS1, which was administered on top of standard of care medications, showed improvements in clinically relevant parameters such as the REVEAL Risk Score and NYHA Functional Class, two significant benchmarks for PAH management. Sustained reductions in mean pulmonary artery pressures were also observed, all positive indicators of CS1’s potential impact on improvement of physical capacity and other relevant parameters for patients.
Cereno’s in-depth analysis highlighted a subgroup of “remarkable responders”, with 25 percent of patients achieving large reductions in pulmonary vascular resistance (PVR) alongside increased right ventricular stroke volume — findings consistent with CS1’s proposed mechanism of reversing pathological vascular remodeling. This aligns with Cereno’s aim of developing a disease-modifying treatment that targets the root mechanism of PAH. Read more here.
During this quarter, the company also reported progress in the Expanded Access Program for CS1 in PAH, with the first patient being dosed with CS1. Insights from this study will guide Cereno in discussions with regulatory bodies and provide critical documentation for future trials.
Cereno partners with Fluidda
In September, Cereno partnered with functional respiratory imaging company Fluidda to enhance its understanding of CS1’s impact on structural vascular changes. The collaboration will incorporate Fluidda’s imaging technology to visualise CS1’s impact on long-term reverse remodeling and includes plans for an investigator-initiated trial, including patients in the Expanded Access Program for CS1.
Moving toward the next stage of CS1 development, the company is preparing for a phase IIb or pivotal phase IIb/III study, which Cereno aim to initiate in 2026.
CS1 obtains 10 years EU market exclusivity for PAH
In addition to the clinical progress in Q3, CS1 achieved Orphan Medicinal Product Designation (OMPD) for the indication PAH from the European Commission. This regulatory milestone provides 10 years of market exclusivity in the EU post-authorisation, complementing the 7-year exclusivity granted under Orphan Drug Designation in the US. Together with patent protections, these measures strengthen CS1’s commercial outlook and solidify its business case across global markets.
IPF indication announced for CS014
Regarding CS014, Cereno announced that Idiopathic Pulmonary Fibrosis (IPF) will be the initial target indication for the second HDAC inhibitor. IPF patients currently have no good treatment alternatives, and preclinical data on CS014 have shown that the candidate may have the potential to address the underlying pathophysiology of IPF and target unmet clinical needs. The drug candidate recently entered its phase I program’s multiple ascending dose stage. Results are expected in mid-2025, and the goal is to enter phase II in 2026.
Promising results with CS585
For prostacyclin agonist CS585, results were presented at the European Society of Cardiology in September and the American Heart Association Scientific Sessions in November, adding to the growing preclinical data package. The preclinical documentation indicates that platelet activation and clot formation is inhibited up to 24 hours after administering CS585.
Strengthened financial position
Post-Q3, Cereno announced a financing agreement securing at least 250 MSEK, extending its cash runway into 2026. The funding, sourced from Fenja Capital II A/S and US-based Arena Investors includes a combination of cash loans and convertible loans. This capital injection positions Cereno with a financial runway to be able to achieve key milestones for all three candidates in its pipeline: advancing CS1 through regulatory FDA approval for a Phase IIb trial or a pivotal Phase IIb/III trial in PAH, completing Phase I trial of CS014, progressing the Expanded Access Program (EAP) investigating long-term clinical use of CS1 in PAH, receiving regulatory approval for a CS014 Phase II trial in IPF as well as continuing preclinical development with CS585.
The financing also bolsters business development efforts, particularly discussions with potential partners and professional investors, to further support progress across its programs.
– The financing agreement provides a good foundation to reach key milestones and increases our ability to explore optimal strategic financial and pharma partnerships, Cereno CEO Sten R. Sörensen says.
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