Enrollment of patients in the first clinical trial at the Saint Louis hospital in Paris, France, was initiated December 7th.
The targeted treatment, SXR1096, has the potential of becoming the first available treatment of Netherton syndrome and meet a great patient need, the company states. Sixera has diligently been developing a new series of specific inhibitors of the proteases (Kallikreins 5, 7 and 14) shown to be overactive in the skin of Netherton patients. The main drug candidate, SXR1096, has been formulated as a topical skin cream and will now be tested in 20 patients with Netherton syndrome at a few European specialist clinics. The phase I/II trial will verify the safety of the drug and test its effect as compared to placebo.
“We have reached an important milestone in our development of a targeted treatment for Netherton syndrome, a first of its kind. This is thanks to the tremendous joint efforts of our team in collaboration with a network of investigators, external consultants, contract research organisations – as well as patients and patient organisations,” says Maarten de Château, CEO, Sixera Pharma.
Sixera has in animal models of Netherton syndrome and in human skin from patients, ex vivo seen very promising results with its kallikrein inhibitors. Netherton syndrome is a genetic disease and patients are diagnosed based on absent or low expression of a protease inhibitor (LEKTI) in the outermost layer of the epidermis. The SXR1096 compound has been shown to be able to replace the function of the missing protease inhibitor.
As Netherton syndrome is a very rare disease, Sixera has applied for and been granted Orphan drug designation (ODD) both by the EMA and the FDA for the use of SXR1096 in Netherton syndrome. ODD renders the company market exclusivity upon approval and can also facilitate the interactions with regulatory agencies.
The title of the study is: “A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled, first-in-human (FIH) Proof of Concept (PoC) study to evaluate the safety and efficacy of topically applied SXR1096 cream in patients with Netherton syndrome (NS).”
Photo of Maarten de Château: Sixera Pharma