07 Dec Sixera initiates its first clinical trial on SXR1096 in Netherton Syndrome
with the potential to be the first available treatment of the rare and serious skin disease Netherton syndrome
Stockholm, December 7, 2021. The Swedish biotech company, Sixera Pharma (”Sixera”) has reached an important milestone today, as enrollment of patients in the first clinical trial at the Saint Louis hospital in Paris, France, was initiated. The targeted treatment, SXR1096, has the potential of becoming the first available treatment of Netherton syndrome and meet a great patient need.
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.
“I am thrilled to be able to offer this new treatment opportunity to our patients with Netherton syndrome. Several studies, also from my own lab, have shown that KLK 5, 7 and 14 are important players in the pathogenesis of Netherton syndrome”, said Alain Hovnanian, Principal investigator for the trial, professor, an expert on the disease and researcher at INSERM and Saint Louis Hospital, Paris.
“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”, said 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.
“To see that many years of work in the lab can eventually lead to a clinical application for patients who have a great unmet medical need, is a great success for all of us”, said Torbjörn Egelrud, professor emeritus in Dermatology at the University of Umeå, Sweden and co-founder of Sixera, who has been studying the role of different proteases in the skin since the 80’s.
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)”
For more information, please contact:
Maarten de Chateau, CEO Sixera Pharma, firstname.lastname@example.org
About Netherton syndrome:
Netherton syndrome is an inherited, genetic disease based on mutations in a gene coding for the protease inhibitor called LEKTI, that has a role in regulating the activity of proteases in the outermost layer of the skin, determining the pace at which skin is shedded. Individuals with this disease can have a red and scaling skin, that is dysfunctional – leading to loss of fluid over the skin as well as increased risk of developing allergies and serious infections. Itching is a prominent symptom. The hair is brittle, breaking off easily. Symptoms can vary between individuals and over time. The disease is lifelong. Treatment with SXR1096 is expected to restore the damaged skin barrier.
Currently, there is no targeted treatment for Netherton syndrome. Treatment is focused on alleviating the symptoms and preventing allergies and infections. Treatment with skin creams at least twice a day is a must for most patients.