CUDC-305

MC2 Therapeutics Receives Positive Feedback from FDA pre-IND Meeting and Updates on Indication Expansion Strategy On track to file IND in mid-2025 for a Phase 2b clinical trial in Hidradenitis Suppurativa (“HS”) with MC2-32, a first-in-class, oral drug candidate targeting pathways involved in numerous Immunology and Inflammation indications (“I&I") Comprehensive pre-clinical studies show that MC2-32 is highly relevant for neutrophilic dermatoses as well as other I&I diseases outside of the skin (I&I pipeline in a product) MC2 Therapeutics expects to start a Phase 2 trial in rare disease Pyoderma Gangrenosum in 2025 May 13th, 2024 - MC2 Therapeutics (“the Company”), a commercial stage biotech company focused on developing novel treatment paradigms for immunology and inflammatory diseases, today confirms it has received positive feedback from a pre-Investigational New Drug ("IND”) meeting with the US Food and Drug Administration (“FDA”) with respect to its first-in-class, oral drug candidate MC2-32 for the treatment of Hidradenitis Suppurativa (“HS”). MC2-32 is a first-in-class, oral HSP90 inhibitor with a unique and highly specific pharmacological profile that elicits a full spectrum effect of HSP90 inhibition, without the class side effects. Alongside its pro-inflammatory action, MC2-32 has a specific targeted tissue distribution, supporting good clinical response profile and tolerability, as demonstrated in a Phase 2a trial published in JAMA Dermatology in December 2023. Based on initial positive feedback from the FDA and subject to the completion of already initiated pre-clinical studies, MC2 Therapeutics expects to file an IND for MC2-32 in HS in mid-2025. MC2-32’s unique mode of action positions it well to address a multitude of other neutrophilic dermatoses as well as other I&I diseases. Consequently, MC2 Therapeutics is planning to explore MC2-32 in a Phase 2 trial in Pyoderma Gangrenosum (“PG”), a rare skin condition causing painful ulcers, for which there is a significant unmet patient need and no approved treatments. It is estimated that >50,000 people suffer from PG in the US and Europe alone, representing a very large commercial opportunity. The potential of MC2-32 will also be investigated in additional diseases in the skin and other organs. Jesper J. Lange, CEO of MC2 Therapeutics, commented: “Encouraging feedback from the FDA marks an important milestone in the continued clinical development progress of our oral drug candidate MC2-32 and is a testament to the high quality of work by our team. Proceeding to the initiation of our Phase 2b trial for HS will be the first step to exploit the full potential of MC2-32 in several I&I indications where patients are currently left with no or limited treatment options. We look forward to working closely with the FDA, our investigators and stakeholders to advance the next phase of development.” Prof. Lars Iversen, CMO of MC2 Therapeutics, commented: “MC2-32’s novel mode of action, targeting multiple pro-inflammatory pathways and with specific tissue targeting properties, highlights the potential to address a broader range of neutrophilic dermatoses as well as other I&I diseases beyond the skin. We are excited to explore this potential in PG, where no approved treatments exist, as well as other I&I diseases.” MC2 Therapeutics also continues to progress the development of its MC2-25 iso-cyanate scavenger for the treatment of Vulvar Lichen Sclerosus (“VLS”). The Company’s Phase 2a proof of concept trial for this indication remains on track, with topline results expected in Q4. MC2 Therapeutics is a commercial stage biotech company focused on developing novel treatment paradigms for neutrophilic diseases. Its pioneering approach in immunology is anchored in a deep understanding of skin biology, clinical expertise and cross-silo thinking. Its pipeline includes two first-in-class drug candidates both in Phase 2 clinical development, with novel modes of action and blockbuster potential in multiple indications (“I&I pipeline in a product”): MC2-32: an oral HSP90 inhibitor with unique tissue specific targeting and a new MOA that modulates multiple pro-inflammatory pathways and relevant immune responses. MC2-25: an iso-cyanate scavenger addressing carbamylation of proteins and amino acids in the skin by iso-cyanate, a dissociation product of urea. MC2-32 (previously RGRN-305) is a new chemical entity small molecule, Heat Shock Protein 90 (HSP90) inhibitor, with a proven capability to clinically attenuate inflammation through a novel mode of action. MC2-32 modulates multiple inflammatory pathways relevant for HS, while the exceptional pharmacological properties of MC2-32 (specific tissue targeting) provide a remarkably favorable safety profile. MC2-32 has been tested successfully in a Phase 2a2a double-blinded, placebo-controlled, proof-of-concept trial in Hidradenitis Suppurativa and a small clinical trial in Plaque Psoriasis. HSP90 is a group of chaperone molecules involved in several cellular processes, including cellular signaling and recent evidence has demonstrated that HSP90 inhibition has strong anti-inflammatory properties. MC2-32 selectively inhibits the two HSP90 isoforms, HSP90α and HSP90β and causes inhibition of several pro-inflammatory pathways involved in the pathogenesis of Hidradenitis Suppurativa. MC2 Therapeutics acquired global (ex-greater China region) option rights to exclusively license MC2-32 (formerly RGRN-305) for all human indications from Regranion in September 2023. HS is an immune-mediated debilitating, life long, recurring, inflammatory skin disorder with a high, unmet medical need. It is characterized by chronic, painful nodules, abscesses, and suppurating sinus tracts that in the most severe form cause significant scarring. Commonly affected body locations include the armpits, below the breasts, the groin, the genitals, perineal and gluteal regions. The pathogenesis of HS is complex and implicates activation of cells of both the innate and adaptive immune systems and involves several proinflammatory pathways. Broad immune modulation can therefore be a preferred strategy. PG is one of a group of autoinflammatory disorders known as neutrophilic dermatoses. It presents as a rapidly enlarging, recurrent and very painful ulcer. The number of ulcers can vary from one to over a dozen and sometimes they coalesce. The classic morphologic clinical presentation of PG in its fully developed form is a deep ulcer with a purulent base and irregular, undermined blue/purple borders which extends centrifugally. The content above comes from the network. if any infringement, please contact us to modify.