MC2 Therapeutics Gets Positive FDA Pre-IND Meeting Feedback, Updates Indication Expansion Strategy

MC2 Therapeutics, a biotech company specializing in novel treatments for immunology and inflammatory diseases, announced it has received positive feedback from a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA) regarding its drug candidate, MC2-32. This first-in-class, oral HSP90 inhibitor is being developed for the treatment of Hidradenitis Suppurativa (HS), a severe and chronic skin condition.

MC2-32 has demonstrated a wide range of HSP90 inhibition effects without the typical side effects associated with the drug class. The drug's specific tissue distribution supports a favorable clinical response and tolerability, as evidenced by a Phase 2a trial published in JAMA Dermatology in December 2023. Based on the FDA's initial feedback and ongoing pre-clinical studies, MC2 Therapeutics plans to file an IND for MC2-32 in HS by mid-2025.

The unique mechanism of MC2-32 makes it a promising candidate for treating various neutrophilic dermatoses and other immunology and inflammation (I&I) diseases. MC2 Therapeutics intends to explore MC2-32 in a Phase 2 trial for Pyoderma Gangrenosum (PG), a rare skin condition characterized by painful ulcers with significant unmet patient needs. Over 50,000 people in the US and Europe are affected by PG, presenting a substantial commercial opportunity.

Moreover, the potential of MC2-32 will be investigated for other diseases affecting the skin and other organs. Jesper J. Lange, CEO of MC2 Therapeutics, highlighted the significance of the FDA's feedback, marking a crucial milestone in the drug's clinical development. He emphasized the company's commitment to advancing the Phase 2b trial for HS as a step towards realizing MC2-32's full potential across multiple I&I conditions where current treatment options are limited.

Prof. Lars Iversen, CMO of MC2 Therapeutics, pointed out that MC2-32's novel mode of action, targeting multiple pro-inflammatory pathways and specific tissue properties, underscores its potential to address a broader spectrum of neutrophilic dermatoses and other I&I diseases beyond the skin. He expressed excitement about exploring this potential in PG, where no approved treatments currently exist.

In addition to MC2-32, MC2 Therapeutics is advancing the development of its iso-cyanate scavenger, MC2-25, for treating Vulvar Lichen Sclerosus (VLS). The company's Phase 2a proof-of-concept trial for this indication is on track, with top-line results expected in the fourth quarter.

MC2 Therapeutics' innovative approach to immunology is rooted in a deep understanding of skin biology, clinical expertise, and cross-disciplinary thinking. The company's pipeline includes two first-in-class drug candidates in Phase 2 clinical development, each with novel mechanisms of action and significant potential across multiple indications.

MC2-32, previously known as RGRN-305, is a new chemical entity and Heat Shock Protein 90 (HSP90) inhibitor. It modulates multiple inflammatory pathways relevant to HS and features specific tissue targeting, resulting in a favorable safety profile. MC2-32 has shown success in a Phase 2a double-blinded, placebo-controlled, proof-of-concept trial for HS and a small clinical trial for Plaque Psoriasis.

HSP90, a group of chaperone molecules involved in cellular processes, has demonstrated strong anti-inflammatory properties through its inhibition. MC2-32 selectively inhibits HSP90 isoforms HSP90α and HSP90β, affecting several pro-inflammatory pathways linked to HS's pathogenesis.

In September 2023, MC2 Therapeutics acquired global rights, excluding the greater China region, to exclusively license MC2-32 for all human indications from Regranion. HS is a debilitating, recurrent inflammatory skin disorder with significant unmet medical needs, often affecting areas such as the armpits, groin, and genitals, causing painful nodules, abscesses, and scarring.

PG, part of the neutrophilic dermatoses group, presents as painful ulcers that can vary in number and size, often merging into larger lesions. The classic PG presentation includes deep ulcers with purulent bases and irregular borders, expanding centrifugally.