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- W3036557626 abstract "Netherton Syndrome (NS) is a potentially life-threatening autosomal recessive disorder involving skin barrier breakdown, inflammation, and allergy. NS affects individuals from birth through adulthood, but standard interventions provide limited benefit and there are no FDA- or EMA-approved therapeutics. We summarize the Kallikrein Inhibitor in Netherton Syndrome (KINS) study, the first Phase 2/3, multicenter, randomized, double-blind, vehicle-controlled trial to evaluate the safety, efficacy and tolerability of a novel topical therapeutic (LM-030) in NS patients. NS is caused by loss of function mutations in the SPINK5 gene which encodes the serine protease inhibitor LEKTI. Hence, the epidermal barrier is broken down due to excessive activation of skin proteases, particularly kallikrein-related peptidase 5 and 7 (KLKs 5&7) and elastase 2 (ELA2). LM-030 (previously BPR277) is a low MW cyclic peptide with selective potent inhibition of KLK7 and moderate inhibition of ELA2, and favorable skin penetration with limited systemic exposure. As listed on the EU clinical trials site, a Phase 1/2 trial in 79 subjects was completed with daily topical application of ointment (predominantly 1%) onto defined target areas in healthy volunteers for 2 weeks or atopic dermatitis and NS patients for 4 weeks. The treatment was generally safe and well-tolerated. 7 of 15 NS patients exhibited a ‘treatment effect’ defined as a 2-point improvement from baseline in the Total Lesional Sign Score for Netherton Syndrome (TLSS-NS) vs 1 of 15 with vehicle control. Inhibitor concentrations in skin exceeded plasma levels by 1000-fold. These data support further evaluation of LM-030 in NS. The KINS study, targeted for initiation in the US and EU in mid-2020, will enroll over 100 patients. Additional design details will be presented in the presentation." @default.
- W3036557626 created "2020-06-25" @default.
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- W3036557626 date "2020-07-01" @default.
- W3036557626 modified "2023-10-18" @default.
- W3036557626 title "579 Rationale and design for the Kallikrein Inhibitor in Netherton Syndrome (KINS) pivotal clinical trial" @default.
- W3036557626 doi "https://doi.org/10.1016/j.jid.2020.03.589" @default.
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