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, it states.

“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,” says Sten R. Sörensen, CEO, Cereno Scientific.

Increased focus on rare diseases

As a result of a chosen initial target indication for CS014, Cereno now advances a pipeline of three drug candidates, two of which are being developed as disease-modifying treatments for rare diseases where high unmet needs persist.

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, it states. “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,” the company states.

Cereno’s move to increase its focus on rare diseases also 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.