- Active MC2-03 eye drop treatment resulted in a strong clinical improvement in severe dry eye patients over control arms both on CFS response (p=0.0090) and mean change from Baseline to Month 6 (p=0.0496)
- A comprehensive biomarker study confirms anti-inflammatory efficacy including a reduction of HLA-DR positive cells from Baseline to Month 6 (p=0.028)
- Data demonstrate favorable safety and tolerability profile, including low incidence of eye irritation
- The data will be discussed with regulatory authorities
Copenhagen, October 23rd, 2018 – MC2 Therapeutics A/S, a clinical-stage dermatology and eye care company, today announced topline results from the MC2-03-C1 Northern Lights Phase 2 trial evaluating the safety and efficacy of MC2-03 (PADciclo™) in moderate-to-severe dry eye (DED) patients. MC2-03 is an eye drop containing ciclosporin (CsA) formulated using MC2 Therapeutics’ proprietary PAD™ Technology to enable improved penetration into ocular tissues without the use of excipients that can cause ocular safety concerns. Topline results will be presented at the Ophthalmology Innovation Summit (OIS) in Chicago on October 25th, 2018.
In this randomized, double-masked, parallel arms, controlled six-month trial enrolling 263 adult patients, two active treatment arms (MC2-03 0.06% CsA and MC2-03 0.03% CsA) were compared to two control arms (MC2-03 vehicle and lubricant) as an add-on therapy to a standard-of-care lubricant. The trial was powered to evaluate safety and efficacy of the individual treatment arms, and to potentially show significant differences between the combined MC2-03 active arms versus the combined control arms.
The primary efficacy variable was the proportion of patients achieving 2-grade improvement in corneal fluorescein staining (CFS) on the modified Oxford scale at Month 6 (defined as CFS responders), which is recognized by regulatory authorities and experts as being clinically and medically relevant. The active arms MC2-03 0.06% CsA (n=68) and MC2-03 0.03% CsA (n=66) showed a similar and consistent trend for a higher proportion of CFS responders than MC2-03 vehicle (n=58) and lubricant (n=63) (full analysis set, n=255). A significant difference between MC2-03 0.06% CsA versus lubricant was observed when comparing patients with CFS response in both eyes (43.1% vs 22.8%, p=0.0289), which was confirmed when comparing the combined MC2-03 active arms to the combined control arms (39.5% vs 26.1%, p=0.0358).
Among the severe population (a total of 108 patients), 58 patients were randomized to the combined MC2-03 active arms. This group of patients with severe keratitis appeared to benefit more from active treatment as significantly more patients treated with active MC2-03 were CFS responders compared to the combined control arm (58.0% vs 31.3%, p=0.0090). This finding was confirmed in severe patients having CFS response in both eyes (42.0% vs 18.8%, p=0.0161), and further supported by the secondary endpoint variable Change in CFS from Baseline to Month 6 in the worse eye (p=0.0496).
The clinically and medically relevant improvement of corneal staining shown with active MC2-03 was substantiated by a comprehensive analysis of inflammatory biomarkers by impression cytology, including a significant difference observed in favor of MC2-03 0.06% CsA over MC2-03 vehicle in the reduction of HLA-DR positive cells from Baseline to Month 6 (p=0.028). HLA-DR is one of the best validated biomarkers of inflammation on the ocular surface and shown to be correlated to DED progression.
An attractive safety profile was confirmed across all arms with no serious adverse events reported to be related to the study medication (safety population, n=263). The most common adverse events related to the active arms MC2-03 0.06% CsA and MC2-03 0.03% CsA were ocular with few observed events of eye pain and eye irritation as the most common, confirming favorable tolerability of MC2-03 in this moderate-to-severe patient group.
“The results of the MC2-03-C1 Northern Lights trial are positive and extremely promising, especially in the most severe population where results go beyond our initial expectations” stated Jesper J. Lange, President of MC2 Therapeutics and he added: “In addition to our recently announced positive US Phase 3 results on MC2-01 PAD™ Cream for topical treatment of psoriasis these results underpin the ability of PAD™ Technology to redefine topicals”.
Professor, Consultant, MD DMSc Steffen Heegaard, University Hospital Rigshospitalet, the coordinating principal investigator in the trial commented: “There is still a need for new modalities in the treatment of severe dry eye disease as patients remain insufficiently managed despite an approved therapy. Importantly, MC2-03 was well-tolerated in the trial and has shown the potential to provide robust and clinically meaningful treatment for this underserved patient group.”
As soon as the full assessment of the data is completed, MC2 Therapeutics will discuss safety and efficacy data with regulatory agencies.
About MC2 Therapeutics A/S
MC2 Therapeutics A/S is a privately held clinical-stage dermatology and eye care company. Using its proprietary PAD™ Technology – a new class of vehicle – MC2 Therapeutics is developing a pipeline of new highly efficacious topical therapies designed for unique patient experiences.