The broadened focus is intended to support a more clinically relevant Phase II program, strengthen the development potential of CS014, and address a patient population with very high unmet medical need, the company states.

CS014 has been developed with idiopathic pulmonary fibrosis (IPF) as the intended initial indication. Interstitial lung diseases (ILDs) comprise a group of fibrotic lung disorders, of which IPF is the most common and a key disease where CS014 remains highly relevant. A substantial proportion of patients with ILD, including many with IPF, go on to develop pulmonary hypertension, a complication associated with significantly worse prognosis. Broadening the development focus to PH-ILD reflects these disease characteristics and allows CS014 to be evaluated in patients where both fibrotic lung disease and pulmonary vascular pathology play a central role.

“Broadening the development focus for CS014 to PH-ILD is a natural and scientifically driven evolution,” says Sten R. Sörensen, CEO of Cereno Scientific. “It enables us to target a severe condition with very limited treatment options and is expected to strengthen the clinical and strategic positioning of CS014 without changing its underlying scientific rationale or long-term development focus. The underlying rationale for this strategic focus is that we believe it will enable us to get CS014 faster to market at lower cost and with a higher probability of success.”

The rare disease PH-ILD is a severe and life-limiting condition associated with markedly worse outcomes compared with fibrotic lung disease alone, including IPF without pulmonary hypertension. ILD patients who develop pulmonary hypertension experience reduced exercise capacity, faster disease progression and high mortality, while treatment options remain very limited and largely focused on symptom management rather than disease modification.

“CS014 targets key pathophysiological processes that are shared across fibrotic lung disease and pulmonary vascular disease, including vascular remodeling, fibrosis, thrombosis and inflammation,” said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific. “By broadening the development focus to PH-ILD, we can design a Phase II study that better reflects real-world disease biology and for patients with worse prognosis.”

CS014 is a novel, orally administered HDAC inhibitor with potential to address underlying disease mechanisms in severe cardiopulmonary diseases. With a completed Phase I study and a broadened development focus on PH-ILD, Cereno Scientific is advancing preparations for a Phase II study planned to be initiated in Q1 2027.