Dosing initiation in AGLE-102 topical treatment trial for DEB lesions

The first patient was dosed in a clinical trial evaluating Aegle Therapeutics' AGLE-102, an investigational topical treatment for dystrophic epidermolysis bullosa (DEB).

The Phase 1/2a study (NCT04173650) will evaluate the safety and efficacy of therapy applied topically to skin lesions compared to placebo in up to 10 people with DEB aged 6 years and older.

“We are very pleased to announce the initiation of this Phase 1/2a clinical trial for DEB,” said Shelley Hartman, CEO of Aegle, in a company press release. “Aegle is committed to developing new therapies to address the continuing unmet medical needs of this patient population.”

In DEB, mutations in the : COL7A1 Gene results in faulty or missing collagen type 7 (COL7), a protein that connects and anchors different layers of skin. Without COL7, the skin layers can separate, leading to blistering and scarring as the blisters heal.

AGLE-102 consists of tiny bags of cellular components called extracellular vesicles (EVs) that contain proteins, nucleic acids (including DNA and RNA) and other molecules derived from mesenchymal stem cells (MSCs), a type of stem cell found in the bone marrow. Biomolecules in EVs have been shown to have anti-inflammatory, immune-modulating and tissue-regenerating properties.

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Treatment mimics the body’s healing mechanism

To maintain their quality, EVs are collected from donor MSCs and isolated using a technology developed by Aegle.

MSCs can transform into other cell types or regulate the activity of neighboring cells, including increasing the production of COL7. In fact, preclinical research has shown the potential of MSC-derived EVs to stimulate COL7 production in cells. AGLE-102 has been shown to be effective in healing burns, according to the company.

“AGLE-102 is designed to mimic the body’s healing mechanism by delivering proteins, including collagen 7, and other key biomolecules such as nucleic acids that reduce inflammation, modulate the immune system and stimulate diseased cells to produce their own collagen 7,” said Evangelos Badiavas, MD, PhD, chief scientific officer at Aegle.

The EV-based treatment received rare pediatric disease designation and fast-track designation from the U.S. Food and Drug Administration. Such designations help speed the development of investigational drugs for serious or life-threatening childhood diseases that affect fewer than 200,000 people in the United States.

Aegle previously raised $4 million in clinical trial funding to test AGLE-102 and an additional $6.5 million in Series A funding to accelerate its clinical pipeline.

“AGLE-102 offers a unique, multi-faceted approach to treating DEB,” said Badiavas.