MC2 Therapeutics Announces Completion of Enrollment in MC2-25 Ph2 Chronic Kidney Disease-associated Pruritus Trial

MC2 Therapeutics Announces Completion of Enrollment in MC2-25 Ph2 Chronic Kidney Disease-associated Pruritus Trial

• MC2-25 CKD is a first-in-class drug candidate and a potential breakthrough in the understanding and treatment of urea associated skin diseases that has puzzled scientists for decades
• MC2-25 CKD uses a di-peptide as an iso-cyanate scavenger to inhibit carbamylation of amino acids and proteins in the skin
• MC2-25 CKD has the potential to become the world’s first approved treatment for pre-dialysis Chronic Kidney Disease-associated Pruritus (CKD-aP), a debilitating condition thought to affect a significant proportion of the ~800 million people globally who suffer from Chronic Kidney Disease (CKD)
• 111 patients enrolled in the multi-center trial across Europe with topline results expected in Q2 2024

Copenhagen, December 7^th, 2023 – MC2 Therapeutics, a commercial stage biotech company focused on developing novel treatment paradigms within immune-mediated and inflammatory conditions, today announced it has completed enrollment in its European Phase 2 trial assessing the safety and efficacy of MC2-25 CKD, an innovative new drug candidate for the treatment of urea associated skin diseases, incl. CKD-aP Stage 5 (dialysis) and stages 3 and 4 (pre-dialysis), the latter segment having no approved treatment options.

MC2-25 CKD presents a potential breakthrough in the understanding and treatment of urea associated skin diseases that has puzzled scientists for decades. It is a totally novel treatment paradigm based on a di-peptide selected to inhibit carbamylation of amino acids and proteins in skin, which is believed to be the causal factor in CKD-aP. MC2-25 CKD has an attractive treatment profile based on convenience and tolerability and has the potential to provide relief from both the severe skin dryness and the debilitating itch associated with CKD-aP, which negatively impacts the quality of life of a significant proportion of the ~800 million people globally who suffer from CKD stages 3-5.

The Phase 2 clinical trial is being conducted at multiple centers across Europe and is evaluating the safety and efficacy of MC2-25 CKD in patients with CKD-aP stages 3-5. 111 patients have been enrolled in the study and randomized in a double-blinded, placebo-controlled parallel-group trial to 12 weeks topical treatment with MC2-25 CKD or placebo.

Top line results from the study are expected in Q2 2024. MC2-25 is also being evaluated in a Phase 2a PoC trial in Vulvar Lichen Sclerosis.

Jesper L. Lange, CEO of MC2 Therapeutics said: “There is increasing evidence that carbamylation plays a central role in various urea associated skin diseases. We are pioneering new ground in this important field and strongly believe that MC2-25 CKD has the potential to finally bring meaningful change to the millions of CKD patients who suffer from debilitating itch and dryness of their skin. We look forward to the data next year and to completing our discussions with regulatory authorities to fast track this treatment to patients globally.”

Kieran McCafferty, MD, at Barts Health NHS Trust and Senior Lecturer at Queen Mary University London and principal investigator of the trial, added: “CKD-aP is a frequent, and highly debilitating symptom of many patients with advanced CKD, a progressive condition which affects around 10% of the general population. This new understanding of the role played by carbamylation in CKD-aP is very exciting and offers hope to millions of patients.”

About MC2-25 CKD
The active component of MC2-25 CKD is a di-peptide, which is formulated in cream based on MC2 Therapeutics’ proprietary formulation and drug delivery system: PAD Technology™. It is an effective isocyanate scavenger showing >90 % inhibition of protein carbamylation and counteracts the morphological skin changes induced by carbamylation as demonstrated in an in vitro reconstructed human uremic skin model.

Isocyanate is in equilibrium with urea and it 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.

About the MC2-25 CKD-aP Ph2 Trial
The trial is a Phase 2, randomized, multicenter, double-blinded, parallel-group trial in subjects with CKD-aP stages 3-5. The trial has enrolled 111 patients in multiple centers in Europe. Patients will apply the trial medication topically twice daily for 12 weeks.

The primary objective is to compare therapeutic efficacy of MC2-25 CKD compared to MC2-25 CKD vehicle and characterize the safety profile of MC2-25 CKD in subjects with CKD-aP stages 3-5. 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, incl. treatment success according to responder endpoints, as well as various other patient and physician reported outcomes. Topline results are expected in Q2 2024.

About CKD-aP
CKD-aP affects a significant portion of the ~800 million people globally suffering from CKD. The vast majority of patients (90%) are pre-dialysis (stages 3 and 4) who currently have no approved treatment options. People suffering from CKD-aP have severe dry skin and evidence of damaged nerve fibers in the skin. CKD-aP is a debilitating condition, which negatively impacts quality of life leading to higher rates of depression and an increased mortality risk.

About MC2 Therapeutics
MC2 Therapeutics is a commercial stage biotech company focused on developing novel treatment paradigms within immune-mediated and inflammatory diseases.

Its pioneering approach in immunology is anchored in a deep understanding of skin biology, clinical expertise and cross-silo thinking. Fuelled by an entrepreneurial mindset and agility, MC2 Therapeutics aims to set new standards in treatment satisfaction for people with immune-mediated and inflammatory conditions.

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

Contacts:
Lonni Goltermann, +45 2018 1111, investor@mc2therapeutics.com

Media: ICR Consilium
Amber Fennell, +44 20 3709 5700, MC2@consilium-comms.com

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