Pipeline

Expanding Across Rare Skin Indications

Focused Science. Broad Patient Impact. Global Outreach.

Discovery

Preclinical

Phase Ia

Phase Ib

in patients

Phase 2

Phase 3

Marketing approval

Olmsted Syndrome

Olmsted Syndrome is an ultra-rare genetic skin disease marked by severe thickening of the palms and soles, painful lesions, and inflammation causing sever mobility challenges, starting in early childhood. OS is caused by gain-of-function mutations in the TRPV3 ion channel.

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Olmsted Syndrome (TRPV3-pEDD)
‍Lanavatrep (oral KM-023)

Pachyonychia Congenita

 Pachyonychia Congenita is a rare inherited skin disorder characterized by painful plantar calluses and characteristic nail changes that can limit walking and daily activities. PC is caused by mutations in keratin genes, and over expression of TRPV3 resulting in increased TRPV3 signaling.

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Pachyonychia Congenita (pEDD-PC)
‍Lanavatrep (oral KM-023)

Ichthyosis

 Ichthyosis is a group of rare, severe inherited skin disorders characterized by dry, thickened, scaly skin caused by an impaired skin barrier. It is driven by mutations in genes involved in barrier formation and lipid processing, and can range from mild scaling to thick, rigid plates covering the entire body with widespread cracking affecting the face (inverted eyelids/lips) and restricting limb movement.

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Ichthyosis
‍Lanavatrep (oral)

Keratoderma

 Keratoderma (most commonly Palmoplantar Keratoderma, PPK) is a group of genetic skin diseases characterized by abnormal thickening of the skin on the palms and soles. It is caused by mutations in genes involved in keratin structure and epidermal differentiation, leading to painful fissures, inflammation, and impaired mobility that can significantly affect daily function.

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Keratoderma (pEDD)
‍KM-001 (topical)

pEDD = palmoplantar Epidermal Differentiation Disorder

Lanavatrep (Oral KM-023)

Safe and well-tolerated in a phase Ia clinical study

Phase Ia

No clinically meaningful safety findings.

No treatment-related adverse events in the Single Ascending Dose (SAD) part.

Only 2 treatment-related adverse events in the MAD cohorts which were infrequent, mild, and limited to isolated cases

No dose interruptions or discontinuations  due to adverse events.

Identifier: NCT07090889

Lanavatrep (Oral)

Enables systemic treatment of TRPV3-driven skin disease

Phase Ia

Lanavatrep reaches the skin within just four hours, achieving rapid target engagement.
Sustained exposure over 24 hours in healthy volunteers supports the potential for durable efficacy.

Identifier: NCT07090889