MC2 Therapeutics announces initiation of a Phase 2 clinical trial in CKD-aP

• Trial based on the recent breakthrough discovery of a novel treatment paradigm for urea associated skin diseases
• First patients dosed in Europe in Phase 2 trial assessing the safety and efficacy of MC2-25 Cream in Chronic Kidney Disease-associated Pruritus (CKD-aP) stages 3-5 patients
• MC2-25 Cream has the potential to become the first approved treatment for ~30 million people in US and EU with chronic kidney disease associated pruritus stages 3 and 4
• Topline results are expected in Q1 2024

COPENHAGEN, Denmark, Sept. 14, 2022 /PRNewswire/ -- MC2 Therapeutics A/S, a commercial stage pharmaceutical company, engaged in research in skin biology and development of novel treatment paradigms for autoimmune and chronic inflammatory skin conditions, announces that the first patients have been dosed in its European Phase 2 trial, MC2-25-C1, evaluating the safety and efficacy of MC2-25 Cream compared to MC2-25 vehicle in treating moderate to severe CKD-aP in patients with CKD stages 3-5.

MC2-25 Cream is an innovative new drug candidate, addressing MC2's new biological treatment paradigm for treatment of urea associated skin diseases. MC2-25 Cream inhibits carbamylation in skin, and thereby potentially can provide relief of the severe skin dryness and debilitating itch, which CKD patients suffer from.

"The initiation of the Phase 2 trial with MC2-25 Cream in Europe marks the first step in our journey to leverage our scientific breakthrough discovery of how to treat urea associated skin diseases, for which there are currently limited or no approved therapies," says Jesper J. Lange, President and CEO of MC2 Therapeutics and added: "As part of our commitment to developing this novel treatment paradigm, we also plan to engage with regulatory authorities in the US in Q4 2022 as part of our global CKD-aP program."

"CKD-aP severely impacts the quality of life of CKD patients stages 3-5 with very few treatment options. There are no approved treatments for stages 3-4 patients and no therapies known to treat the damage in the skin caused by the disease. Based on the novel treatment paradigm targeting carbamylation in skin, MC2-25 Cream has the potential to become the first therapy addressing the origin of CKD-aP for millions of CKD patients," says Kieran McCafferty, MD, at Barts Health NHS Trust and Senior Lecturer at Queen Mary University London and principal investigator of the MC2-25-C1 trial and continues: "The trial has been endorsed by Kidney Research UK and is a highly appreciated opportunity for our CKD-aP patients to contribute to the clinical development of this exciting drug candidate."

About MC2-25 Cream

The active component of MC2-25 Cream is a di-peptide, which is formulated in MC2 Therapeutics' proprietary formulation and drug delivery system – PAD Technology™. MC2-25 Cream is an effective isocyanate scavenger showing >90 % inhibition of protein carbamylation and counteracting the morphological skin changes induced by carbamylation as demonstrated in an in vitro reconstructed human uremic skin model.

Isocyanate is a very reactive molecule known both to be neurotoxic and to readily react with proteins, peptides and amino acids present in the body and skin. In CKD patients carbamylation of amino acids and proteins is highly upregulated and believed to be a key contributor to the root cause of CKD-aP.

For more information about MC2 Therapeutics' breakthrough discovery of a novel biological treatment paradigm for urea associated skin diseases, please see Press Release of September 1^st, 2022.

About the MC2-25 Phase 2 Trial

The trial is a Phase 2, randomized, multicenter, double-blind, parallel-group trial in subjects with CKD-aP stages 3-5. The trial is expected to enroll approximately 108 patients in about 23 centers in Europe. Subjects will apply trial medication topically twice daily for 12 weeks.

The primary objective is to compare therapeutic efficacy of MC2-25 Cream versus vehicle cream, as well as to characterize the safety profile of MC2-25 Cream in subjects with CKD-aP. The primary efficacy endpoint is the mean change in weekly mean Worst Itch Numeric Rating Score (WI-NRS) from baseline to week 12. Secondary endpoints will be assessed, including treatment success according to responder endpoints, as well as various other patient and physician reported outcomes.

Topline results are expected in Q1 2024.

About CKD-aP

CKD-aP effects more than 35 million people in the US and EU, of which the vast majority (~30 million) are non-dialysis patients (stages 3 and 4) who currently have no approved treatment options. People suffering from CKD have severe dry skin and evidence of damaged nerve fibers in the skin. CKD-aP is a severe condition, which leads to an overall increased mortality risk, as well as higher frequency of depression and reduced quality of life.

About MC2 Therapeutics A/S

MC2 Therapeutics A/S is a privately held commercial stage pharmaceutical company committed to research of skin biology and development of novel treatment paradigms for people with autoimmune and chronic inflammatory skin conditions.

Its innovative approach to address complex challenges more effectively is anchored in deep understanding of the skin biology combined with learnings from the pathophysiology across disease segments. Fueled by an entrepreneurial mindset and creativity, MC2 Therapeutics aims to set new standards in treatment satisfaction for people with skin conditions.

For additional information on MC2 Therapeutics Group, please visit www.mc2therapeutics.com

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SOURCE MC2 Therapeutics