About AVX001

AVX001 is our drug candidate for the treatment of skin cancer, specifically actinic keratosis and basal cell carcinoma. The plan is to conduct a phase 2b and a phase 2a study in close collaboration with a partner.

AVX001 is a small molecule and selective inhibitor of an enzyme called cPLA2α that is playing a fundamental role in developing cancer and inflammatory diseases, such as actinic keratosis and basal cell carcinoma (BCC). AVX001 has been found, in clinical trials, to be safe and well-tolerated. It has shown to be ten times more potent in treating basal cell carcinoma than one of the most well-known products on the market, vismodegib (which is the active ingredient in the approved product for BCC, Erivedge). AVX001 is delivered into the skin in a cosmetically attractive gel.

  • Attacks the cancer on both immune cells and rapidly dividing cells
  • Shows clear effect, tested in four clinical studies, after only 4 weeks

The market

Actinic keratosis (AK) and basal cell carcinoma (BCC) are very common diseases, both are skin diseases that result from damage caused by sun rays. Actinic keratosis is by far the most common skin cancer condition, affecting up to 60 million people in the United States alone. Basal cell carcinoma is the most common type of skin cancer, with an estimated 4 million new cases each year in the United States alone.. There are few drug therapies available, and most are associated with moderate to severe side effects. Hence, we see a market opportunity for a safe and efficacious treatment with our clinically validated drug candidate AVX001.

The global market for basal cell carcinoma is estimated at

6.7 billion USD

Milestones

2024

Co-development/out-licensing agreements with partner.

2024

Additional skin tolerability studies to extend the treatment period, produce study medication and prepare the applications for clinical trials.

2025

First patient in the phase 2b study in AK and in 2a study in BCC clinical trials.

2026

Key results from the clinical trials.

Research

AVX001 is a highly potent and selective small molecule that blocks the activity of the key enzyme cPLA2α which is a key biomarker for poor prognosis in cancer. When blocking the cPLA2α enzyme activity with AVX001 the levels of arachidonic acid are reduced following regress of the tumor growth. Several key hallmarks of cancer development are affected, including programmed cell death, anti-tumor inflammation, inhibition of angiogenesis, and anti-tumor proliferation.

High cPLA2α levels correlate with metastasis and hence poor prognosis of several cancers. Even poor treatment outcomes of existing treatments have been shown to be correlated with high cPLA2α levels, and inhibiting cPLA2α in combination with existing standard of care treatments, for example radiation, increases the treatment response in preclinical models.

• Clinically validated drug candidate
• Well characterized mode-of-action
• Strong evidence for treating inflammatory driven cancers
• Skin friendly with excellent safety profile and limited skin reaction
• Cosmetically attractive gel formulation with high delivery of the drug candidate into the skin

Promising results in completed clinical trials

Clinical safety and efficacy studies of AVX001 have been completed in four phase 1 or 2 clinical studies. Two studies with a topical ointment formulation for the treatment of mild-to-moderate psoriasis, and two in a gel formulation for the treatment of atopic dermatitis and AK. In the latter, we conducted the Copenhagen Actinic Keratosis Study (COAKS), a 12-week single-center, randomized, vehicle-controlled, double-blind, hybrid clinical trial in adults with multiple AK lesions Olsen grade 1 or 2. In this study the AVX001 topical gel-formulation was found to be safe and tolerable, and effectiveness was observed for the highest dose. From the three other studies AVX001 was also found to be safe and effectiveness was shown in treating psoriasis patients.

AVX001 3% reduced the number of AK lesions

AVX001 showed overall effectiveness

AVX001 acts more effectively in more severe AK-grade-2

BCC cell proliferation is highly dependent on the so-called Hedgehog signaling pathway, and the marketed drug vismodegib has been developed to block this pathway. AVX001 has been investigated in human BCC cell line model. The experiments demonstrated a potent dose dependent effect, being ten times more potent as compared to vismodegib. These data form a strong rationale for the planned clinical study in superficial basal cell carcinoma.

Plan

The plan is to identify one or more licensing/development partners to continue the development of AVX001 and conduct a phase 2 study.

Bakgrund

AXV001 was developed by prof. Berit Johansen at NTNU in Trondheim, Norway and Professor Leif Skattebøl at Univerity of Oslo, Norway. The groundbreaking research of the key enzyme cPLA2α and its central role in inflammation and cancer has been performed in close collaboration with prof. Edward Dennis at University of California, San Diego and Professor Joseph Bonventre at Harvard Medical School, Boston.

The research of cPLA2α and the role of AVX001 in AK and BCC has been investigated by Professor Berit Johansen, NTNU and Professor Merete Hædersdal at the Danish Research Center for Skin Cancer, University of Copenhagen.