MC2 Therapeutics Announces First Patients Enrolled in Phase 2 Proof of Concept Clinical Trial in Vulvar Lichen Sclerosus

MC2 Therapeutics Announces First Patients Enrolled in Phase 2 Proof of Concept Clinical Trial in Vulvar Lichen Sclerosus

• MC2-25 VLS is a first-in-class drug candidate for urea associated skin diseases using a di-peptide as an iso-cyanate scavenger to inhibit carbamylation of amino acids and proteins in the skin
• MC2-25 VLS has the potential to become the first approved treatment for Vulvar Lichen Sclerosus (VLS), a debilitating chronic inflammatory disorder thought to affect millions of women globally
• Phase 2 proof of concept trial for MC2-25 VLS for VLS will recruit approx. 40 patients across multiple centres with topline results expected in H2 2024

Copenhagen, November 23, 2023 – MC2 Therapeutics, a commercial stage biotech company focused on developing novel treatment paradigms within immune-mediated and inflammatory conditions, today announced that the first patients have been enrolled in a Phase 2 PoC trial evaluating the safety and efficacy of MC2-25 VLS, an innovative new drug candidate for the treatment of urea associated skin diseases, incl. Vulvar Lichen Sclerosus (VLS) where there are no approved treatment options.

MC2-25 VLS is a novel treatment paradigm based on a di-peptide that inhibits carbamylation of amino acids and proteins in skin. MC2 Therapeutics believes that carbamylation of amino acids and proteins in the vulvar region by iso-cyanate is the root cause of VLS. MC2-25 VLS has the potential to provide relief from the severe itch, erosions, fissures and pain associated with VLS. This debilitating inflammatory condition is thought to affect millions of patients globally and studies have shown that ~3% of women with VLS eventually develop vulvar squamous cell carcinoma.

The Phase 2 clinical trial is being conducted at multiple centres across Denmark and will evaluate the safety and efficacy of MC2-25 VLS in patients with VLS. 40 patients will be recruited for the study and randomized 1:1 in a double-blinded, placebo-controlled parallel-group trial to 12 weeks topical treatment with MC2-25 VLS or placebo. The study is a Phase 2a PoC study. The data generated from this double-blinded, placebo-controlled PoC trial will inform the trial design for a larger Phase 2b trial.

“MC2-25 VLS has been developed based on our novel treatment paradigm targeting carbamylation in the skin and the active di-peptide component is formulated using our proprietary PAD Technology™. This delivers a highly attractive treatment profile based on ease of use and tolerability in sensitive, fissured skin,” said Professor Lars Iversen, CMO of MC2 Therapeutics. “As pioneers in uremic diseases, this trial represents a major step forward for MC2 in developing a safe and effective treatment for VLS that severely affects the quality of life of millions of women globally.”

Jesper J. Lange, CEO of MC2 Therapeutics said: “Female health remains a hugely underserved area with a distinct lack of approved drugs for female-specific conditions. We are proud to be pioneering MC2-25 VLS for the treatment of VLS as well as for the treatment of Chronic Kidney Disease associated Pruritus in an ongoing Phase 2 trial. In both cases we have the potential to radically change the treatment paradigm for these major indications with huge patient populations, further demonstrating our commitment to changing the immunology and inflammation landscape.”

About MC2-25 VLS
The active component of the new drug candidate MC2-25 VLS is a di-peptide, which is formulated in cream based on MC2 Therapeutics’ proprietary formulation and drug delivery system – PAD Technology™. It is highly tolerable in sensitive fissured skin and convenient to use. MC2-25 VLS 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 VLS patients, the vulvar is exposed to iso-cyanate via urine as urea is in equilibrium with iso-cyanate. MC2 Therapeutics believes that carbamylation of amino acids and proteins in the vulvar region by iso-cyanate is the root cause of Vulvar Lichen Sclerosus.

About the MC2-25 VLS Phase 2 Trial
The trial is a Phase 2, randomized, multi-centre, double-blinded, placebo-controlled parallel-group trial in subjects with VLS. The trial is expected to enrol 40 patients in multiple centres in Denmark. Patients are randomized 1:1 to either active treatment with MC2-25 VLS or placebo. Subjects will apply trial medication topically for 12 weeks.

The primary objective is to explore the efficacy of MC2-25 VLS compared to MC2-25 VLS vehicle in VLS. Secondary objectives are to explore the safety of MC2-25 VLS compared to MC2-25 VLS vehicle in VLS and to explore the burden of VLS on women’s lives. The primary endpoint is mean change in weekly mean Worst Itch Numeric Rating Score (WI-NRS) recorded in the patient’s diary from Baseline to Week 12 for MC2-25 VLS compared to MC2-25 VLS vehicle. Also, secondary endpoints will be assessed, incl. safety and other patient and physician reported outcomes. Topline results are expected in H2 2024.

About Vulvar Lichen Sclerosus
Vulvar Lichen Sclerosus is a common, chronic, inflammatory, cutaneous disorder that causes severe and distressing itch but also erosions, fissures and pain. It can lead to scarring, impaired sexual function, and malignancy. The true prevalence of VLS is unknown, and probably underestimated as VLS is thought to be underdiagnosed and undertreated (REF: Melnick et al. Int. J Womens Dermatol 2020;6:260-262).

While Lichen Sclerosus can affect any area of the body of both males and females, it has a predilection for female anogenital epithelium.

The typical VLS patient is a woman around 50 years of age with external genital itch, but it can affect at any age, incl. childhood. Treatment of VLS aims at controlling the symptoms, stopping further scarring and distortion, reducing the risk of cancer and improving the patient’s quality of life (REF: Perez-Lopez FR et al. Climacteric 2017;20:339-347). However, currently there is no approved treatment for VLS in Europe or the US and there is therefore a high unmet medical need for this debilitating disease.

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

Its innovative approach in immunology is anchored in a deep understanding of skin biology, clinical expertise and cross-silo thinking. Fuelled by an entrepreneurial mindset and creativity, 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