What CS014 is and why it matters
CS014 is Cereno Scientific’s second clinical-stage program, developed as an oral, once-daily HDAC (histone deacetylase) inhibitor for pulmonary hypertension associated with interstitial lung disease (PH-ILD) — a serious condition with limited treatment options and poor prognosis.
PH-ILD arises when pulmonary hypertension develops as a complication of interstitial lung disease, combining fibrotic lung pathology with pulmonary vascular disease. This dual disease burden leads to reduced exercise capacity, accelerated progression, and increased mortality.
CS014 is designed to target core biological processes involved in both fibrosis and pulmonary vascular remodeling, with the ambition of delivering disease-modifying potential together with favorable safety and tolerability suitable for long-term use.
Designed for chronic treatment in complex cardiopulmonary disease
Patients with PH-ILD often live with chronic, progressive disease requiring long-term treatment and supportive care. CS014 is being developed with this reality in mind.
Key principles include:
- Once-daily oral administration, supporting ease of use and adherence
- A pharmacological profile intended for long-term treatment
- A focus on safety and tolerability, which is particularly important in fragile patient populations
These attributes are critical in a condition where treatment burden and tolerability can significantly affect quality of life.
Scientific rationale – addressing core cardiopulmonary biology
CS014 is being developed as a next-generation HDAC inhibitor with epigenetic modulation properties. CS014 builds on Cereno’s scientific platform in epigenetic modulation through histone deacetylase (HDAC) inhibition, while representing a new chemical entity with a multimodal profile. This multimodal approach aims to influence key processes implicated in both fibrotic lung disease and pulmonary vascular disease.
Supported by preclinical evidence, CS014 has shown activity against:
- Fibrosis (abnormal scarring of lung tissue)
- Vascular remodelling (changes in small vessels contributing to increased pulmonary pressure)
- Thrombosis and inflammation, which further drive disease progression
Rather than acting on a single downstream pathway, this broad mechanism may be particularly relevant in PH-ILD, where multiple pathological processes converge.
For a deeper scientific explanation, see
Formulation and treatment concept
CS014 is formulated for oral, once-daily administration, which supports:
- Convenience and adherence for patients living with progressive cardiopulmonary disease
- A profile suited to long-term treatment
- Integration into existing clinical practice, where oral add-on therapies are more feasible than frequent or invasive administration
This aligns with Cereno’s broader philosophy of developing therapies with favorable safety and tolerability, tailored for long term use.
Clinical evidence
Completed Phase I trial
CS014 has successfully completed a first-in-human Phase I study in healthy volunteers, which evaluated safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD).
Key outcomes included:
- Favorable safety and tolerability, with no unexpected safety signals
- Pharmacokinetic profiles supportive of once-daily oral dosing
- Exposure levels aligned with preclinical models suggesting potential biological activity
These results provide a solid clinical foundation for further development in PH-ILD.
Next step
Phase IIb during 2027 – planned in PH-ILD
Cereno Scientific is preparing for the start of a Phase IIb trial with CS014 in patients with PH-ILD in the first quarter of 2027.
A Phase I study is currently ongoing, designed as a regulatory bridging study (so-called PK bridging), which is expected to replace additional safety studies and a Phase IIa study, while enabling a more efficient development pathway toward potential market approval.
Results are expected in mid-2026 and will form part of the basis for the planned Phase IIb study.
PH-ILD at a glance
Pulmonary Hypertension associated with Interstitial Lung Disease
PH-ILD is a condition in which pulmonary hypertension develops in the setting of interstitial lung disease, combining lung fibrosis with increased pressure in the pulmonary circulation.
The coexistence of these disease processes leads to more severe symptoms, faster progression, and worse outcomes compared with interstitial lung disease alone.
Why CS014 stands out
CS014 brings together several important attributes:
- Targets key disease mechanisms involved in PH-ILD
- A next-generation HDAC inhibitor with multimodal properties
- Proprietary new chemical entity
- Once-daily oral dosing designed for long-term treatment
- Favorable safety and tolerability demonstrated in Phase I
- Positioned for development in a disease area with significant unmet medical need
Together, these elements position CS014 as a differentiated investigational therapy with the potential to address a significant unmet need in rare cardiopulmonary disease.
Other candidates
A HDACi
Drug candidate CS1
A HDACi, proprietary reformulation of VPA, being developed as a well-tolerated oral therapy with favorable safety profile and disease-modifying effects for the rare disease pulmonary arterial hypertension (PAH). A Phase IIa trial has successfully been completed and preparations for a Phase IIb is ongoing.
Learn more
Preclinical asset
Drug candidate CS585
A novel, selective and potent IP receptor agonist has demonstrated the potential to significantly improve disease mechanisms relevant to cardiovascular diseases. A research collaboration with the University of Michigan is ongoing with the aim of transitioning to Phase I.
Learn more