Kamari Pharma, a clinical-stage biotechnology company based in Ness Ziona, Israel, announced significant findings from two presentations on their novel
TRPV3 inhibitors,
KM-001 (topical) and
KM-023 (oral), at the 81st Annual Meeting of the Society for Investigative Dermatology (SID) held from May 15-18, 2024, in Dallas, Texas.
In the first presentation, titled "KM-001, a novel TRPV3 inhibitor, demonstrates safety and preliminary efficacy in Phase 1b clinical study for the treatment of
palmoplantar keratoderma," Professor Edel O’Toole of Queen Mary University of London led the discussion. This study evaluated the safety and efficacy of KM-001, a topical cream applied twice daily for twelve weeks. The study involved 15 patients, with eight participants from the UK and seven from Israel, suffering from either
pachyonychia congenita (PC) or
punctate palmoplantar keratoderma type 1 (PPPK1).
The trial demonstrated that KM-001 is safe, as no severe adverse events or systemic side effects were reported. Mild treatment-related adverse events did occur but did not necessitate discontinuation of the treatment. Efficacy results were promising, with an overall responder rate of 87%. Specifically, 88% of UK participants and 86% of Israeli participants showed improvement in at least one of six disease parameters. Additionally, nearly half of the patients showed improvements in at least two parameters, and a significant reduction in
pain was observed in 60% of PC patients, although PPPK1 patients typically do not report pain as a symptom.
The second presentation, "Novel TRPV3 inhibitors developed for the treatment of palmoplantar keratodermas, demonstrate safety and efficacy in preclinical models," was presented by Kamari's Chief Scientific Officer, Dr. Liora Braiman-Wiksman. This presentation covered preclinical results for both KM-001 and KM-023. The inhibitors were designed to address skin damage at both molecular and local levels by regulating calcium influx into the cells.
In 3D skin models of PC and PPPK1, KM-001 and KM-023 were effective in normalizing proliferation and differentiation markers and in enhancing epidermal barrier formation. In in vivo studies using DS-Nh mice with a gain-of-function TRPV3 mutation, KM-001 showed a remarkable improvement in skin histology and reduced scratching bouts significantly. Specifically, KM-001 showed a 70% improvement in skin histology compared to 30% in the vehicle group and an 80% reduction in scratching. KM-023 also demonstrated a significant reduction in keratoderma severity and scratching.
Based on these promising results, Kamari Pharma plans to move forward with Phase 2 development of KM-001 for treating palmoplantar keratoderma. Additionally, they intend to advance KM-023 into first-in-human studies for the oral treatment of
Olmstead syndrome, severe keratoderma, and
Ichthyosis.
Kamari Pharma is committed to developing innovative treatments for
rare and severe genetic skin diseases. Their lead molecules, KM-001 and KM-023, stand out due to their high specificity and selectivity as TRPV3 inhibitors. The management team at Kamari comprises industry leaders with extensive experience in drug discovery, dermatological pharmaceutical development, and rare disease drug development.
How to obtain the latest research advancements in the field of biopharmaceuticals?
In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!
