SeaBeLife receives Orphan Drug Designation (ODD) from EMA for treatment of acute liver failure

Roscoff, France, September 3, 2024 – SeaBeLife, a biotechnology company developing drug candidates intended to block cellular necrosis, today announces the granting of an Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for its drug candidate SBL01 in the treatment of acute liver failure. 

Acute liver failure (ALF) is a life-threatening disease which occurs when the liver suddenly loses its ability to function in a person without a pre-existing liver disorder, resulting in encephalopathy (brain dysfunction) and coagulopathy (bleeding disorder). ALF often affects young people and carries a high morbidity and mortality rate (85% when transplantation is not feasible). 

In the EU, the population of patients with ALF is estimated to be between 1.3 and 3.1 in 
10,000^[1]. This meets the EMA’s orphan designation threshold, which is targeted at conditions that affect no more than 5 in 10,000 people. With this ODD, SeaBeLife will be able to benefit from regulatory and financial incentives, including protocol assistance, reduced fees and market exclusivity for ten years upon approval.

According to the EMA’s committee for orphan medicinal products: “[SeaBeLife] has provided non-clinical data in a model of acute liver failure showing reduced hepatotoxicity and improved survival when SBL01 was used in combination with the currently authorized product in the applied for condition, as compared to the authorized product alone. The committee considered that this constitutes a clinically relevant advantage.”

“We are thrilled to receive the orphan drug designation from European authorities. This is an important recognition of our strong preclinical research dataset. It reinforces our determination to push ahead with our approach in treating acute liver diseases and serious ophthalmologic disorders,” said Morgane Rousselot, CEO and co-founder of SeaBeLife. “This achievement comes at a time when we are actively preparing a Series A fundraising with venture capital funds, institutional funds and family offices.”

SBL01 is a first-in-class small molecule ‘dual inhibitor of both necroptosis and ferroptosis’. Regulated cell death, such as necroptosis and ferroptosis, are key processes in acute liver failure where cells degenerate abruptly. The company’s molecule is capable of specifically inhibiting the induction cascade of these two cell death mechanisms and thereby enabling cellular protection and restoration of liver function.

About regulated cell death and SeaBeLife’s technology 

When a cell dies, there are several modes in which it can do so. In the case of certain pathologies, a phenomenon called necroptosis occurs, which is a form of regulated necrosis. Unfortunately, necroptosis results in inflammation, which damages surrounding tissue. SeaBeLife’s molecules have a unique property that make them particularly effective in fighting necroptosis; they also block another specific mode of regulated cell death, ferroptosis, making them double action inhibitors. 

In recent scientific literature, researchers have demonstrated that this dual action is essential to the inhibition of regulated necrosis in treating certain complex pathologies. The synergy between necroptosis and ferroptosis triggers cell death and rapid organ degeneration.

SeaBeLife’s approach – based on dual action molecules that simultaneously target two different pathological cell death pathways – is unique in tackling the complex crosstalk between them, unlike other treatments that tend to target a single pathway. As a result, SeaBeLife’s treatment options are more likely to prove effective and reduce the chance of resistance in patients.

^[1] The incidence data is derived from a large epidemiological study, run over a one-year period in a population of 124,511 people (Mei-Sheng Duh et al, 1999).